[Senate Hearing 107-499]
[From the U.S. Government Publishing Office]



                                                        S. Hrg. 107-499
 
                            STEM CELLS, 2001
=======================================================================

                                HEARINGS

                                before a

                          SUBCOMMITTEE OF THE

            COMMITTEE ON APPROPRIATIONS UNITED STATES SENATE

                      ONE HUNDRED SEVENTH CONGRESS

                             FIRST SESSION

                               __________

                            SPECIAL HEARINGS

                     JULY 18, 2001--WASHINGTON, DC
                     AUGUST 1, 2001--WASHINGTON, DC
                    OCTOBER 31, 2001--WASHINGTON, DC

                               __________

         Printed for the use of the Committee on Appropriations


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                      COMMITTEE ON APPROPRIATIONS

                ROBERT C. BYRD, West Virginia, Chairman
DANIEL K. INOUYE, Hawaii             TED STEVENS, Alaska
ERNEST F. HOLLINGS, South Carolina   THAD COCHRAN, Mississippi
PATRICK J. LEAHY, Vermont            ARLEN SPECTER, Pennsylvania
TOM HARKIN, Iowa                     PETE V. DOMENICI, New Mexico
BARBARA A. MIKULSKI, Maryland        CHRISTOPHER S. BOND, Missouri
HARRY REID, Nevada                   MITCH McCONNELL, Kentucky
HERB KOHL, Wisconsin                 CONRAD BURNS, Montana
PATTY MURRAY, Washington             RICHARD C. SHELBY, Alabama
BYRON L. DORGAN, North Dakota        JUDD GREGG, New Hampshire
DIANNE FEINSTEIN, California         ROBERT F. BENNETT, Utah
RICHARD J. DURBIN, Illinois          BEN NIGHTHORSE CAMPBELL, Colorado
TIM JOHNSON, South Dakota            LARRY CRAIG, Idaho
MARY L. LANDRIEU, Louisiana          KAY BAILEY HUTCHISON, Texas
JACK REED, Rhode Island              MIKE DeWINE, Ohio
                  Terrence E. Sauvain, Staff Director
                 Charles Kieffer, Deputy Staff Director
               Steven J. Cortese, Minority Staff Director
            Lisa Sutherland, Deputy Minority Staff Director
                                 ------                                

 Subcommittee on Departments of Labor, Health and Human Services, and 
                    Education, and Related Agencies

                       TOM HARKIN, Iowa, Chairman
ERNEST F. HOLLINGS, South Carolina   ARLEN SPECTER, Pennsylvania
DANIEL K. INOUYE, Hawaii             THAD COCHRAN, Mississippi
HARRY REID, Nevada                   JUDD GREGG, New Hampshire
HERB KOHL, Wisconsin                 LARRY CRAIG, Idaho
PATTY MURRAY, Washington             KAY BAILEY HUTCHISON, Texas
MARY L. LANDRIEU, Louisiana          TED STEVENS, Alaska
ROBERT C. BYRD, West Virginia        MIKE DeWINE, Ohio
                           Professional Staff
                              Ellen Murray
                              Jim Sourwine
                              Mark Laisch
                            Adrienne Hallett
                              Erik Fatemi
                               Adam Gluck
                       Bettilou Taylor (Minority)
                        Mary Dietrich (Minority)
                    Sudip Shrikant Parikh (Minority)

                         Administrative Support
                             Carole Geagley
                        Emma Ashburn (Minority)







                            C O N T E N T S

                              ----------                              

                         Tuesday, July 18, 2001

                                                                   Page

Opening statement of Senator Tom Harkin..........................     1
    Prepared statement...........................................     3
Opening statement of Senator Arlen Specter.......................     4
Letter from Senators Tom Harkin and Arlen Specter................     7
Letters from Senator Arlen Specter...............................     7
Letter from Tommy G. Thompson....................................     8
Opening statement of Senator Patty Murray........................     9
Statement of Hon. Orrin G. Hatch, U.S. Senator from Utah.........    11
    Prepared statement...........................................    13
Statement of Hon. Bill Frist, U.S. Senator from Tennessee........    15
Statement of Hon. Sam Brownback, U.S. Senator from Kansas........    18
    Letter from President George W. Bush.........................    19
    Letter from Christopher Currie...............................    21
    Letter from Julie Durler.....................................    22
Statement of Hon. Gordon Smith, U.S. Senator from Oregon.........    23
Opening statement of Senator Thad Cochran........................    28
Opening statement of Senator Kay Bailey Hutchison................    33
Statement of Lana Skirboll, Ph.D., Director, Office of Science 
  Policy, National Institutes of Health, Department of Health and 
  Human Services.................................................    34
Statement of Richard M. Doerflinger, associate director for 
  policy development, secretariat for pro-life activities, United 
  States Conference of Catholic Bishops..........................    36
    Prepared statement...........................................    38
Statement of Anton-Lewis Usala, M.D., founder, chairman, and 
  chief science officer, Encelle, Inc............................    44
    Prepared statement...........................................    46
Statement of William E. Gibbons, M.D., chairman, Jones Institute 
  for Reproductive Medicine......................................    48
    Prepared statement...........................................    49
Statement of Susan Lanzendorf, Ph.D., Eastern Virginia Medical 
  School.........................................................    50
    Prepared statement...........................................    51
Statement of Diane Krause, M.D., Ph.D., associate professor, Yale 
  Univer- 
  sity...........................................................    52
    Prepared statement...........................................    53
Statement of Mary J.C. Hendrix, Ph.D., professor and head, 
  Department of Anatomy and Cell Biology, University of Iowa 
  College of Medicine............................................    54
    Prepared statement...........................................    55
Statement of Michael D. West, Ph.D., president and chef executive 
  officer, Advanced Cell Technology..............................    58
    Prepared statement...........................................    59
Prepared statement of William L. Pierce, Ph.D., senior fellow, 
  Discovery Institute for Public Policy..........................    71
Prepared Statement of Senator Herb Kohl..........................    76

                       Wednesday, August 1, 2001

Opening statement of Senator Arlen Specter.......................    77
Statement of Maria Friere, Ph.D., Director, Office of Technology 
  Transfer, National Institutes of Health, Department of Health 
  and Human Services.............................................    78
    Prepared statement...........................................    79
Statement of Carl Gulbrandsen, Ph.D., managing director, 
  Wisconsin Alumni Research Foundation, president, WiCell 
  Research Institute, Inc........................................    83
    Prepared statement...........................................    84
Statement of Nigel M. de S. Cameron, Ph.D., executive chair, the 
  Centre for Bioethics and Public Policy, London, England........    94
    Prepared statement...........................................    96
Statement of Arthur L. Caplan, Ph.D., director, center for 
  bioethics, University of Pennsylvania..........................    97
Statement of Glenn McGee, Ph.D., assistant professor of 
  bioethics, philosophy and history and sociology of science, 
  University of Pennsylvania.....................................    99
Joint prepared statement of Arthur L. Caplan and Glenn McGee.....   102
Statement of Michael West, Ph.D., president and CEO, Advanced 
  Cell Technology................................................   104
    Prepared statement...........................................   107

                      Wednesday, October 31, 2001

Opening statement of Senator Arlen Specter.......................   123
Statement of Dr. Wendy Baldwin, Deputy Director for Extramural 
  Research, National Institutes of Health, Department of Health 
  and Human Services.............................................   124
    Prepared statement...........................................   126
Statement of Bert Vogelstein, M.D., professor of oncology and 
  pathology, John Hopkins Oncology Center; chairman, National 
  Research Council Institute of Medicine Committee on the 
  Biological and Medical Applications of Stem Cell Research......   129
    Prepared statement...........................................   130
Statement of Martin F. Pera, Ph.D., Monash University, Australia.   137
    Response to queries from Senators Specter and Harkin.........   140
Statement of Joseph Itskovitz, Ph.D., director, Rambam Medical 
  Center; professor, Technion University, Haifa, Israel..........   142
    Prepared statement...........................................   143
Statement of James Thomson, Ph.D., chief scientific officer, 
  WiCell Research Institute, Inc.................................   146
    Prepared statement...........................................   148
Statement of Carl Gulbrandsen, Ph.D., J.D., managing director, 
  Wisconsin Alumni Research Foundation, president, WiCell 
  Research Institute, Inc........................................   149
    Prepared statement...........................................   152








                               STEM CELLS

                              ----------                              


                         TUESDAY, JULY 18, 2001

                           U.S. Senate,    
    Subcommittee on Labor, Health and Human
     Services, and Education, and Related Agencies,
                               Committee on Appropriations,
                                                    Washington, DC.
    The subcommittee met at 9:41 a.m., in room SH-216, Hart 
Senate Office Building, Hon. Tom Harkin (chairman) presiding.
    Present: Senators Harkin, Murray, Landrieu, Specter, 
Cochran, Hutchison, and DeWine.

                OPENING STATEMENT OF SENATOR TOM HARKIN

    Senator Harkin. The Appropriations Subcommittee on Labor, 
Health and Human Services, and Education will come to order. 
This hearing of the Appropriations subcommittee will now 
proceed.
    In November 1998 the country learned that Dr. James Thomson 
and John Gearhart had isolated pluripotent stem cells from 
early human embryos and grown them in a culture. It marked a 
significant achievement in science and a new hope for a cure to 
many of the most cruel and debilitating diseases. One month 
later this subcommittee promptly held its first hearing on stem 
cells research and learned of the enormous potential this 
research had for the treatment of diseases like Parkinson's and 
Alzheimer's, ALS, Lou Gehrig's Disease, heart disease, and 
other diseases.
    Since then this committee has held six more hearings on 
this important subject, chaired by my good friend and then 
chairman Senator Arlen Specter. Today it is my turn, but the 
purpose will be the same. Senator Specter and I have been 
partners for a long time in our efforts to increase funding for 
medical research and in particular for stem cell research in 
particular.
    I have co-sponsored with Senator Specter a bill that would 
allow federally funded scientists to derive human stem cells 
from embryos under four conditions: first, the embryos must be 
obtained from an in vitro fertilization clinic; second, the 
donors must have provided informed consent; third, the embryo 
must no longer be needed for infertility treatments; and 
fourth, there can be no payment to the donors.
    The American Society of Cell Biology has estimated that 
about 100,000 human embryos are currently frozen in IVF clinics 
in excess of their clinical need.
    Let me be clear about why we are here and why we have 
introduced our bill and why we fought so hard to make sure that 
the Federal Government supports this research. We introduced 
this legislation because we want to save lives and we want to 
find cures for some of the most debilitating diseases that 
affect mankind. We have seen the human faces of these diseases. 
We have been moved by the testimony of doctors and patients, 
family members and advocates that have been touched by juvenile 
diabetes, Parkinson's, ALS, Alzheimer's. I particularly 
remember the poignant testimony of Mr. John Wagenaar from 
George, Iowa, who is suffering from Alzheimer's.
    That is really why we are here. This is not an abstract 
issue. It is about saving the lives of millions of human 
beings. I believe it is imperative that the Federal Government 
support this research. The government has an important role to 
play in basic science and basic science will always be 
underfunded by the private sector because this type of research 
does not immediately get products onto the market. There is no 
immediate profit, but there are tremendous long-term benefits. 
That is why the Federal Government has been involved in 
supporting basic research.
    Equally important are the strict ethical guidelines that 
will come with Federal funding. It is important to note that 
stem cell research in the private sector is not subject to 
Federal monitoring and these guidelines.
    This morning Dr. Lana Skirboll with the National Institutes 
of Health will release the NIH report which reviews the current 
state of the science of stem cell research. We received a copy 
of that report yesterday and it is clear when you read the 
report that stem cell research holds promise in the treatment 
of diseases.
    Some say that stem cell research is fine as long as you use 
just adult cells. I disagree. The NIH's report is clear on this 
important point. Embryonic and adult stem cells are different 
and both present immense research opportunities for potential 
therapies. I think it would be irresponsible to wait for years 
to determine the potential of adult stem cells before studying 
the benefits of embryonic stem cells.
    There are still a lot of unanswered questions. For example, 
are there enough existing stem cells to do the research that 
needs to be done, and what are the differences between adult 
and embryonic stem cells? At this hearing we will try to get 
some of the answers to those questions.
    We have a distinguished panel of experts before us today. I 
especially want to thank Dr. Mary Hendrix of the University of 
Iowa. Dr. Hendrix has been invaluable to my staff and me as she 
has patiently answered our questions about the science of stem 
cells.
    At many points in our history religion and science have 
intersected, and at every point we have paused to measure our 
morality and the ancient lessons of religion against our 
science and the new frontiers we explore, as well we should do 
that. Science must be infused with morality and humanity. When 
it is not, it can be more about amusing ourselves with our own 
ingenuity than pursuing real scientific breakthroughs that 
improve our lives.

                           prepared statement

    In the case of stem cell research, I strongly believe that 
we have measured the question carefully and that it is time to 
move forward. Where there cannot be new life, there can be new 
hope, new hope for thousands of Americans suffering from 
horrible and debilitating disease that withers the mind and the 
body and robs us of our loved ones. In the case of stem cell 
research, we can be true and loyal to our loved ones and true 
and loyal to our values. In fact, it would be an affront to our 
values if we did not proceed with caution and under ethical 
guidelines and investigate how stem cell research can better 
our lives and the lives of all people.
    [The statement follows:]

                Prepared Statement of Senator Tom Harkin

    Good Morning. This hearing of the Appropriations Subcommittee on 
Labor, Health and Human Services, and Education will now proceed.
    In November, 1998, the country learned that Drs. James Thomson and 
John Gearhart had isolated pluripotent stem cells from early human 
embryos and grown them in culture. It marked a significant achievement 
in science and a new hope for a cure to many of the most cruel, and 
debilitating diseases.
    A month later, this Subcommittee promptly held its first hearing on 
stem cell research--and learned of the enormous potential this research 
had for the treatment of Parkinson's, Alzheimer's, ALS, heart disease, 
and other diseases.
    Since then, this Subcommittee has held six more hearings on this 
important subject chaired by my good friend from Pennsylvania, Senator 
Specter. Today it's my turn--but the purpose will be the same. Senator 
Specter and I have been partners for a long time in our efforts to 
increase funding for medical research and for stem cell research, in 
particular.
    I have co-sponsored with Senator Specter a bill that would allow 
federally-funded scientists to derive human stem cells from embryos 
under three conditions: the embryos must be obtained from an IVF 
clinic; the donor must have provided informed consent; and the embryo 
must no longer be needed for infertility treatments. The American 
Society of Cell Biology has estimated that 100,000 human embryos are 
currently frozen in IVF clinics, in excess of their clinical need.
    Let me be clear about why we are here, why we have introduced our 
bill, and why we have fought so hard to make sure that the Federal 
government supports this research.
    We introduced this legislation because we want to save lives and to 
find cures for some of the most debilitating diseases that affect 
mankind. We have seen the faces of human face of these diseases. We 
have been moved by the testimony of doctors, patients, family members 
and advocates that have been touched by Juvenile Diabetes, Parkinson's, 
ALS and Alzheimer's. I particularly remember the poignant testimony of 
Mr. John Wagenaar, from George, Iowa who is suffering form Alzheimer's. 
That is why we are here. This is not an abstract issue. It is about 
saving the lives of millions of Americans.
    It is imperative that the Federal government support this research. 
The government has an important role to play in supporting basic 
science. Basic science will always be underfunded by the private sector 
because this type of research does not immediately get products onto 
the market. There is no immediate profit--but there are tremendous 
longterm benefits.
    Equally important are the strict, ethical guidelines that will come 
with Federal funding. It is important to note that stem cell research 
in the private sector is not subject to Federal monitoring.
    This morning, Dr. Lana Skirboll, with the National Institutes of 
Health, will release the NIH report which reviews the current state of 
the science of stem cell research. I received a copy of that report 
yesterday. It's clear, when you read the report that stem cell research 
holds promise in the treatment of disease.
    Some say stem cell research is fine, as long as you just use adult 
cells. I disagree, and the NIH report is clear on this important point: 
embryonic and adult stem cells are different and both present immense 
research opportunities for potential therapies. It would be 
irresponsible to wait for years to determine the potential of adult 
stem cells before studying the benefits of embryonic stem cells.
    There are still a lot of unanswered questions. For example, are 
there enough existing stem cell lines to do the research that needs to 
be done and what are the differences between adult and embryonic stems 
cells. At this hearing we will try to get some of the answers to those 
questions. We have a distinguished panel of experts before us today; I 
want to especially welcome Dr. Mary Hendrix, of the University of Iowa. 
Dr. Hendrix has been invaluable to my staff and me as she has patiently 
answered our questions about the science of stem cells.
    At many points in our history, religion and science have 
intersected. And at every point, we have paused to measure our morality 
and the ancient lessons of religion against our science and the new 
frontiers we explore. As well we should.
    Science must be infused with our morality and humanity. When it is 
not, it can be more about amusing ourselves with our own ingenuity than 
pursuing real scientific breakthroughs that improve our lives.
    In the case of stem cell research, I strongly believe that we have 
measured the question carefully, and that it is time to move forward. 
Where there cannot be new life, there can be new hope--new hope for the 
thousands of Americans suffering from horrible and debilitating disease 
that withers the mind and body and robs us of our loved ones. In the 
case of stem cell research, we can be true to our loved ones and true 
to our values. In fact, it would an affront to our values if we did not 
proceed, with caution, and investigate how stem cell research can 
better our lives and the lives of all Americans.
    I look forward to hearing from a number of Senate colleagues on 
this important issue. I want to welcome Senator Hatch, Senator Frist, 
Senator Smith and Senator Brownback who are taking time out of their 
busy schedules to testify before us this morning. But before we turn to 
their testimony, I yield to my friend and colleague, Senator Specter, 
for his opening remarks.

    Senator Harkin. I look forward to hearing from a number of 
our Senate colleagues who are here today on this important 
issue. I want to welcome Senator Hatch, Senator Frist, Senator 
Smith, Senator Brownback, who are taking time out of their busy 
schedules to testify before us this morning.
    Before I turn to them, I would yield to my friend and one 
of our great leaders on basic scientific research, Senator 
Specter.

               OPENING STATEMENT OF SENATOR ARLEN SPECTER

    Senator Specter. Thank you very much, Mr. Chairman. I thank 
you for your leadership and your work on this very important 
subject, as well as your work on increasing NIH funding. As you 
have outlined, when the stem cell issue became public in 
November 1998 this subcommittee immediately started a series of 
hearings. This is our eighth hearing, which is a very 
substantial number, and as these hearings have progressed and 
as the public has become better acquainted with the potential 
for stem cells for curing Parkinson's, delaying Alzheimer's, 
spinal cord injury, important on cancer, on heart ailments, and 
virtually all of the other maladies confronting the human race, 
there has been a groundswell of support for stem cell research. 
I think it is now an avalanche.
    I have talked to many, many of our colleagues in the 
Senate, and I had said last week that I thought there were 70 
votes in favor of stem cell research and now I believe it is in 
excess of 75. I thank our colleagues for coming here today. 
Senator Hatch, he is a very strong pro-life Senator who has 
gone into the lion's den in taking a stand in favor of stem 
cell research, and his testimony, which we will hear, is very, 
very important.
    Senator Frist--and not all of us agree on all aspects of 
this matter--is our doctor in residence. Senator Brownback has 
been candid in his opposition and we have debated this subject 
and doubtless will continue to do so.
    Senator Gordon Smith I think capsulated the matter. I quote 
him frequently. It may not be a good idea to quote him in his 
presence. He will speak for himself. But when he made the 
distinction between an embryo in the womb of a woman, where 
life eventuates, contrasted with an embryo in a laboratory 
dish, it is hard to top that kind of a presentation or 
rationale for no funding stem cell research.
    There is one matter that I feel constrained to comment 
upon, and that is the difficulty of this subcommittee in 
getting the unvarnished facts from the Department of Health and 
Human Services. In advance of our appropriation hearing with 
the National Institutes of Health, Senator Harkin and I wrote 
to the NIH Institute directors asking for their evaluation of 
stem cell research. There is no better time to get information 
than when a Federal agency is applying for an appropriation. 
That is a superb time to get information.
    But I am very distressed to have to report--and I have 
taken this up personally with the Secretary of Health and Human 
Services--that of the 15 letters which were submitted there 
were 21 deletions in 10 of the letters. I am going to put all 
of the deletions into the record because they are too long to 
go into at this time. But a couple are illustrative.
    [The information follows:]

    A total of 21 deletions were made in 10 of the 15 letters submitted 
to the Subcommittee by the Department. The deletions relate mainly to 
four issues:
  --How a ban on Federal funding for stem cell research would affect 
        current and future research.
  --The advantages of embryonic stem cells over adult stem cells, and 
        the need to compare the two.
  --Discussion of embryonic stem cell research in the private sector.
  --Concluding statements regarding the potential of embryonic stem 
        cells.
    A summary of the deletions are as follows:
How would a ban on Federal funding for stem cell research affect 
        current and future research?
    ``While many questions remain unanswered, the opportunities that 
would be lost, if there were a lack of NIH support, would be greater 
and devastating to this realm of research. This research offers 
tremendous opportunity to restore lost sensory function, including the 
regeneration of lost hair cells, as well as develop future therapeutic 
strategies important to every known human disease, including severe 
neurological disorders such as Alzheimer's and Parkinson's disease. It 
would be unfortunate if the ban on NIH support for human stem cell 
research results in a missed opportunity to restore hope and quality of 
life to affected individuals.''----James Battey, Jr., M.D., Ph.D., 
National Institute on Deafness and Other Communication Diseases
    ``A ban on funding for stem cell research would affect the National 
Institute of Dental and Craniofacial Research's ability to progress 
toward developing innovative solutions to complex conditions and 
diseases. Although research with adult stem cells has contributed to 
significant research advances, it has yet to be established that adult 
stem cells are as versatile as those derived from embryonic tissue. 
Thus a ban on embryonic stem cell research will limit our ability to 
understand the full potential of this therapeutic modality to treat the 
many complex conditions and diseases of interest to NIDCR.''----
Lawrence Tabak, D.D.S., Ph.D., National Institute of Dental and 
Craniofacial Research
    ``The ban on Federal Stem cell research would likely limit the use 
of this important research tool to non-federally funded projects, 
including those within the private sector and other countries.''----
Jack McLaughlin, Ph.D., National Eye Institute
    ``A Federal ban on human stem cell research is likely to hurt 
current and future research in at least two ways. First, we believe 
that a ban on this research would produce a chilling effect that would 
result in a decreased number of research grant applications on 
embryonic stem cells of animals, such as mice, in which most of the 
embryonic stem cell work has been done. . . . Although, it is difficult 
to prove cause and effect, the ban on human embryo research is likely 
to be the reason that we currently receive a very small number of 
applications per year on embryo research in mice and other animals. . . 
. Second, a Federal ban on human embryonic stem cell research would 
mean that research on this topic in the United States will be conducted 
in private laboratories without scientific and ethical oversight. . . . 
It also would be subject to public monitoring for compliance with 
ethical guidelines. In essence, Federal funding of this important 
research would open it to public scrutiny.''----Duane Alexander, M.D., 
National Institute of Child Health & Human Development
The advantages of embryonic stem cells over adult stem cells, and the 
        need to be able to compare the two.
    ``It is known that embryonic stem cells have vast potential to 
develop into tissues of any type, while only a small number of studies 
have indicated a possible limited potential for adult stem cells.'' . . 
. ``It is not known how long adult stem cells will survive and 
function, while embryonic stem cells do not have this limitation.'' . . 
. ``The use of adult stem cells for cell therapy at this time, without 
knowing how much better embryonic stem cells would perform, is 
considered by many researchers to be premature.'' . . . ``If embryonic 
stem cells are not used, optimal therapies may not be developed.''----
Allen Spiegel, M.D., National Institute of Diabetes and Digestive and 
Kidney Diseases
    ``Not knowing whether adult or embryonic stem cells will ultimately 
prove to be of value makes it important that research proceed using 
both types of stem cells.''----Claude Lenfant, M.D., National Heart, 
Lung, and Blood Institute
    ``In cancer patients, normal tissues can be damaged by both the 
disease and by the treatment (surgery, radiation, and chemotherapy). We 
already know that embryonic stem cells have all the characteristics 
that would be needed for regenerating healthy tissues but there is no 
way to establish the relative potential of embryonic stem cells to 
adult stem cells unless we can compare the two.''----Richard Klausner, 
M.D., National Cancer Institute
Discussion of embryonic stem cell research in the private sector.
    ``Researchers funded by the private sector have also reported 
progress with respect to the application of stem cell research to liver 
disease. It has been reported that scientists at Geron in California 
have coaxed their embryonic stem cell lines to produce `liver-like' 
cell. . . . It may well be that there are other such studies supported 
by private industry, which are not being reported in the published 
literature and of which we are therefore unaware.''----Allen Spiegel, 
M.D., National Institute of Diabetes and Digestive and Kidney Diseases
Concluding statements regarding the potential of embryonic stem cells.
    ``I share with my colleagues in the other Institutes enthusiasm for 
the great promise that stem cells hold for the treatment of disease, 
and NIGMS intends to continue its support for research into the 
fundamental genetic and cellular mechanisms that underlie this 
promise.''----Marvin Cassman, Ph.D., National Institute of General 
Medical Sciences
    ``Continuous stem cell research is critical to cancer research, 
based on the knowledge that cancer cells often have certain stem cell 
properties, in particular their arrest in development and the ability 
to renew themselves.''----Richard Klausner, M.D., National Cancer 
Institute
    ``I would like to conclude by stating that embryonic stem cells are 
truly remarkable cells. We are on our way to understanding how they 
form and how they can be used for the treatment of human diseases and 
disorders. The evidence we have at present indicates that they have 
enormous potential in this regard.''----Duane Alexander, M.D., National 
Institute of Child Health & Human Development
    ``Research on human stem cells offers the greatest potential in the 
eons of modern human history for mankind to experience a major 
breakthrough in the medical treatment or cure of a wide-range of 
devastating human diseases and disorders. . . A ban of Federal funding 
for stem cell research would deprive the American public of the 
benefits of 100 years of scientific research and 20 years of 
progressive stem cell research--the vast majority of which was paid for 
by Federal funds from the American public as an investment in health 
care research.''----Kenneth Olden, Ph.D., National Institute of 
Environmental Health Sciences
                                 ______
                                 

           Letter From Senators Tom Harkin and Arlen Specter

                                               U.S. Senate,
                                     Washington, DC, June 29, 2001.
Hon. Tommy Thompson,
Secretary, Department of Health and Human Services,
Washington, DC.
    Dear Mr. Secretary: We were surprised to read in the June 26, 2001, 
New York Times, an article written by Robert Pear, detailing the 
recently completed Stem Cell Research Study conducted by the National 
Institutes of Health.
    It is particularly troubling that several requests made by our 
respective staff to obtain a copy of the report, titled ``Stem Cells: 
Scientific Progress and Future Research Directions,'' were denied.
    Given our great interest in stem cell research, we request that the 
Department immediately provide us with a copy of the study for our 
review.
    Thank you for your attention to this matter.
            Sincerely,
                                                Tom Harkin,
                                                          Chairman.
                                             Arlen Specter,
                                         Ranking Republican Member.
                                 ______
                                 

                   Letter From Senator Arlen Specter

                                               U.S. Senate,
                                     Washington, DC, July 11, 2001.
Hon. Tommy Thompson,
Secretary, Department of Health and Human Services,
Washington, DC.
    Dear Secretary Thompson: I want you to know that I am very 
displeased with your decision not to turn over a copy of the report, 
``Stem Cells: Scientific Progress and Future Research Directions''.
    It is my view that our Subcommittee, or, at a minimum, the Chairman 
and Ranking Member, have an absolute right to that stem cell report.
    It is insufficient for me to be limited to having my staffer go to 
your office to read the report. I want to read it myself and I cannot 
reasonably come to your Department to read it.
    I also want you to know that I am displeased with the censoring by 
your Department of the responses by the NIH Institute Directors in 
answer to my May 4, 2001 letter posing specific questions regarding 
stem cell research. Those full letters should have been transmitted to 
the Subcommittee promptly and we should not have had the delays or the 
necessity to push you for these letters.
            Sincerely,
                                                     Arlen Specter.
                                 ______
                                 

                   Letter From Senator Arlen Specter

                                               U.S. Senate,
                                     Washington, DC, July 17, 2001.
Hon. Tommy Thompson,
Secretary, Department of Health and Human Services,
Washington, DC.
    Dear Secretary Thompson: At 9:45 a.m. this morning, I finally 
received a copy of the 202 page report entitled ``Stem Cells: 
Scientific Progress and Future Research Directions''.
    As you know, the Appropriations Subcommittee covering your 
Department has a stem cell hearing tomorrow morning at 9:30 a.m. I 
consider the response by you and your Department to my letter of July 
11, 2001 and the late availability of this report to be absolutely 
insulting.
    Since I am advised that you have declined the Subcommittee's 
request to testify tomorrow, I am writing to give you advance notice, 
in the event you wish to make some response, that I intend to comment 
on your Department's conduct and to put this letter and my letter of 
July 11, 2001 in the record.
            Sincerely,
                                                     Arlen Specter.
                                 ______
                                 

                     Letter From Tommy G. Thompson

                The Secretary of Health and Human Services,
                                     Washington, DC, July 17, 2001.
Hon. Arlen Specter,
U.S. Senate,
Washington, DC.
    Dear Senator Specter: I received your letter this afternoon and 
felt it was important to respond to you as quickly as possible. I take 
concerns about the responsiveness of the Department of Health and Human 
Services very seriously, which is why we are undertaking a variety of 
management reforms. I also can appreciate how passionately you feel 
about the issue of stem cell research. It is an important scientific, 
issue and the media frenzy surrounding it has only served to intensify 
an enormously emotional debate.
    However, I feel it is important to clarify some matters regarding 
the hearing being held by the Labor-HHS Appropriations subcommittee 
tomorrow, and the report prepared by NIH: ``Stem Cells: Scientific 
Progress and Future Research Directions.''
    When you originally approached us about both my participation in 
the hearing and obtaining copies of the report, we made what 
accommodations we could. As you may know, I have spent a several days 
each month working from the various operating divisions of this 
department. My intent is to learn as much as possible so that I can 
most effectively run this Department. Before receiving your invitation, 
I had committed to work from the Food and Drug Administration this 
week. My staff did work, however, to ensure you would have a 
representative of this Department to appear before the committee.
    The report that we sent to your committee this morning was 
developed at my request. I asked NIH to provide me with a review of the 
available science so that I am as well versed and understand this issue 
as well as possible. The undertaking was enormous. The scientists at 
NIH reviewed more than 1,200 documents and spoke to more than 50 
scientists.
    The report was not finalized until a few hours before I sent it to 
you. In fact, they worked throughout the weekend to endure it would be 
available to you and your committee before the hearing tomorrow. In 
addition, we allowed members of your staff to review a draft version of 
this report--before the scientists were ready to consider it final.
    As you know, I try to make myself available to personally address 
concerns about this Department. I sincerely hope that you and I can 
work together on this and other issues going forward.
            Sincerely,
                                                 Tommy G. Thompson.

    Senator Specter. Dr. Richard Klausner, the head of the 
National Cancer Institute: ``Continuous stem cell research is 
critical to cancer research.'' Not given to this subcommittee 
until we had to extract it like a bicuspid from the Department 
of Health and Human Services.
    Dr. James Batty [commented]. ``Opportunities would be lost 
if there were a lack of NIH support. It would be devastating in 
this realm of research.''
    Dr. Dwayne Alexander. ``A ban on this research would 
produce a chilling effect that would result in a decreased 
number of research grant applications.''
    Now, we may not agree with the administration, but Congress 
has a right to the facts and we ought not to be getting 
censored information.
    Then there is the issue of the report. I read about the 
report in the New York Times on June 27--I like to get my 
information from the Secretary, not the New York Times--and 
immediately wrote to the Secretary thereafter requesting the 
report. I could not get a copy of the report. Finally our staff 
had to go to the offices of HHS on the afternoon of July 3 and 
read it over there, no copies, no copies available to the 
subcommittee. Two of our staffers, Senator Harkin's and mine, 
had to share one report, after a long wait.
    So I wrote to the Secretary last week on July 11 and said, 
I would like a copy of the report, I would like to prepare for 
this hearing. Yesterday morning at 9:45 I got a copy of this 
202-page report. I am not too slow at reading, but that is just 
not right.
    Yesterday afternoon I got a copy--I wrote to the Secretary 
again and I am going to make copies for the record my letters 
of June 29, 2001, July 11, July 17, and Secretary Thompson's 
reply to me yesterday. But the Secretary does not deal with the 
censorship issue. He is going to have to deal with that yet. He 
said that we got a copy of the report just as soon as it was 
finalized.
    But I am told that, except for a few editorial changes and 
printing, this report was available last week and really the 
week before. I intend to get to the bottom of it. The Secretary 
and the administration do not have to agree with us, but they 
cannot keep the facts from us. I am not unmindful of the 
consideration that there may be some in the administration who 
are in disagreement with Secretary Thompson's personal views 
and that he may be under substantial pressure. But we have a 
constitutional government in America and we have status in the 
Congress to find out the facts and to make our judgments.
    I am hopeful we can work it out in a collegial way with the 
administration. But if we cannot, we are going to get to the 
bottom as to why we did not get this report in a timely way, 
why these facts were censored, and it is a matter for 
congressional consideration. The Congress still establishes 
public policy in the United States, subject to the President's 
concurrence or congressional override. We intend to pursue it.
    Thank you, Mr. Chairman.
    Senator Harkin. Thank you, Senator Specter.
    Senator Murray.

               OPENING STATEMENT OF SENATOR PATTY MURRAY

    Senator Murray. Thank you very much, Mr. Chairman, for 
holding this very important hearing. I think stem cell research 
offers a lot of hope for millions of patients and I really 
appreciate you and Senator Specter for your work to implement 
the NIH guidelines that support embryonic stem cell research, 
and I am proud to be a co-sponsor of the legislation that you 
have introduced to codify those guidelines.
    Mr. Chairman, delaying NIH support for stem cell research 
is going to delay hope for millions of patients struggling with 
diseases like MS, Parkinson's, diabetes, cancer, ALS, and 
spinal cord injuries. My father was diagnosed with multiple 
sclerosis when I was 15 years old. I lived with a family member 
with MS until he died a few years ago. I know the devastating 
impacts, the pain, the suffering, the agony, for the patient 
and the family, and the thought that there is going to be 
progress out there that could be stymied by action here in 
Washington, DC, to me is really frightening.
    I know how important this research is and how important it 
is that we can say to families in the future that they will not 
have to live with what my father lived with, to say to patients 
like my father that they would not have to live with this 
because we have research that can improve their lives, I think 
is extremely important.
    This is not about abortion. Stem cells are not a result of 
abortions. It is not about destroying life. It is about 
improving life and in many cases saving lives. Federal support 
of stem cell research does not legitimize abortion. It does not 
reduce the value of all human life. But it does give us a 
promising new avenue for research that I believe could save 
many lives.
    I do think we need to set some standards to guide this 
research and I believe the standards that have been developed 
by NIH are appropriate. I know that the former Administrator of 
NIH, Dr. Varmas, conducted an exhaustive process to develop the 
guidelines for support of stem cell research. Bioethical 
standards were high and every effort was made to separate the 
issue of abortion from the debate. NIH-supported research will 
ensure that these bioethical standards are followed.
    Mr. Chairman, without Federal guidelines the private sector 
will determine the direction of research and the ethical 
standards that are employed. I think this is an opportunity for 
us to help advance the science that millions of patients are 
waiting on. We should be taking a strong leadership role in 
this new exciting technology.
    My own State of Washington is where there are a lot of 
promising research institutes that are working on this: Fred 
Hutchinson Cancer Research, University of Washington. I have 
listened to the researchers, I have listened to the patients 
and families from across my State, and hundreds of them have 
contacted me to urge me to support embryonic stem cell 
research.
    So I want to thank all of the witnesses who have come here 
today to share their views and their concerns and to let you 
know that your testimony will help us implement sound research 
guidelines for NIH.
    So Mr. Chairman, again I thank you for your strong work on 
this, for your continued work on this, and I look forward to 
working with you.
    Senator Harkin. Thank you very much, Senator Murray.
    Senator DeWine.
    Senator DeWine. I have no comment, no opening statement.
    Senator Harkin. Thank you very much.
    Senator DeWine. I thank you for holding the hearing.
    Senator Harkin. Thank you, Senator DeWine.
    Now we turn to our distinguished group of Senators who are 
here today. We will go in order of seniority. Thus I would like 
to first recognize my good friend Senator Orrin Hatch of Utah, 
first elected to the Senator in 1976, ranking member on the 
Senate Judiciary Committee and member of the Finance, 
Intelligence, Indian Affairs, and Joint Economic Committees. 
Senator Hatch received his B.S. from Brigham Young University 
and his law degree from the University of Pittsburgh.
    Then I will go in order of Senator Hatch and then Senator 
Frist and Senator Brownback and Senator Smith. Senator Hatch, 
welcome to the committee. The floor is yours. Proceed as you so 
desire.
STATEMENT OF HON. ORRIN G. HATCH, U.S. SENATOR FROM 
            UTAH
    Senator Hatch. Well, thank you, Mr. Chairman. I thank you 
and Senator Specter for inviting me to testify today.
    As a long-time supporter of biomedical research, I applaud 
the bipartisan leadership this committee has demonstrated in 
working toward the goal of doubling the NIH research budget by 
2003. This investment in biomedical research is helping to 
usher in a new age of science in which the mysteries of human 
health and disease are unraveled.
    Today's hearing centers on a major opportunity presented to 
the biomedical research community, stem cell research. Nobel 
laureate and former NIH Director Harold Varmas has 
characterized the situation by saying that ``It is not 
unrealistic to say that stem cell research has the potential to 
revolutionize the practice of medicine.''
    I would like to take this opportunity to share with you how 
I came to my decision to support Federal funding for embryonic 
stem cell research. Over many months I devoted hours of study 
to this important issue, reflecting on my spiritual teachings, 
the law, the science, and the ethical issues presented by 
embryonic stem cell research. Let me be absolutely clear. I 
hold strong pro-life, pro-family values and strongly oppose 
abortion.
    I conclude that support of embryonic stem cell research is 
consistent with and advances pro-life and pro-family values.
    Let me emphasize four points for you this morning. First, I 
think that the support of this vital research is a pro-life, 
pro-family position. This research holds out promise for more 
than 100 million Americans suffering from a variety of 
diseases, including heart disease, multiple sclerosis, 
Parkinson's, Alzheimer's, ALS, cancer, and diabetes.
    Second, in the in vitro fertilization process it is 
inevitable that extra embryos are created, embryos that simply 
will not be implanted in a mother's womb. As these embryos sit 
frozen in a test tube outside the womb, under today's 
technology there is no chance for any of them to develop into a 
person. While I have no objection to considering ways to foster 
adoption of embryos, there are a host of issues associated with 
this which must be worked out.
    While these issues are being considered, the reality today 
is that each year thousands, and I am told the number may be 
tens of thousands, of embryos are routinely destroyed. Why 
should not these embryos slated for destruction be used for the 
good of mankind?
    Third, while I understand that many in the pro-life 
community will disagree with me, I believe that a human life, a 
human's life, begins in the womb, not in a petri dish or a 
refrigerator. It is inevitable that in the IVF process extra 
embryos are created that will simply not be implanted in a 
mother's womb. To me the morality of the situation dictates 
that these embryos, which are routinely discarded, be used to 
improve and extend and facilitate life. The tragedy would be in 
not using these embryos to save lives when the alternative is 
that they would be destroyed.
    Fourth, there is no guarantee that any stem cell research 
will reap the benefits we hope, but it is clear that embryonic 
stem cell research holds tremendous promise. Some hold out 
adult stem cell research as a good alternative. By all means we 
should continue adult stem cell research, but I do not believe 
it would be wise to cut off support for embryonic stem cell 
since many eminent scientists believe it is the more promising 
avenue of research.
    The committee will hear from scientific experts this 
morning. You will hear from NIH about the report, this very 
comprehensive report on stem cell research that the agency will 
formally issue today. While I am not a scientist, my 
preliminary reading of the report strongly suggests that 
embryonic stem cell research may have some substantial 
advantages over adult stem cells, at least at this stage of the 
research.
    Consider the following excerpts from the summary of the new 
NIH stem cell report: ``Stem cells in adult tissues do not 
appear to have the same capacity to differentiate as do 
embryonic stem cells or embryonic germ cells.'' Consider this 
next statement: ``Human embryonic stem cells can be generated 
in abundant quantities in the laboratory and can be grown, that 
is allowed to proliferate, in the undifferentiated or 
unspecialized state for many, many generations.''
    Then this last one: ``Researchers have had difficulty 
finding laboratory conditions under which some adult stem cells 
can proliferate without becoming specialized.'' Finally: 
``Current evidence indicates that the capability of adult stem 
cells to give rise to many different specialized cell types is 
more limited than that of embryonic stem cells.''
    However, it is also important to note what the NIH report 
does not say. It does not say that the promise of embryonic 
stem cell research obviates the need to pursue adult stem cell 
research. The report indicates that both embryonic and adult 
stem cell research holds great promise, and I believe that both 
avenues should be zealously pursued.
    In the end, it is my hope that we are able to conduct 
research that will improve and prolong human life. I truly 
believe that the cures for diseases like diabetes, Parkinson's, 
Alzheimer's, ALS, cancer, multiple sclerosis, heart disease, et 
cetera, can be found if we continue this promising research. 
That is why we must take advantage of all ethical and promising 
types of research.
    Before I close, I would like to comment on the work of the 
Jones Institute for Reproductive Medicine in Norfolk, Virginia, 
which is creating embryos in order to conduct stem cell 
research. I find the work of this clinic extremely troubling. 
To me this type of research is indicative of the problems we 
will continue to encounter if we do not allow Federal funding 
with strict research guidelines for embryonic stem cell 
research.
    As this case illustrates, without stringent NIH ethical 
requirements, we are opening the door to an array of different 
research standards, which I believe could create some very 
serious consequences.
    Mr. Chairman, today we stand on the threshold of a great 
opportunity. Embryonic stem cell research may be the single 
most important scientific discovery in all of our lifetimes. 
The most renowned scientists in the country have told us that 
this research holds forth the promise of treatments and perhaps 
cures for some of the most debilitating diseases affecting our 
entire Nation and the world.

                           prepared statement

    I think it would be a mistake to cut off Federal support 
for this research.
    I appreciate the opportunity to testify before your 
subcommittee and would be happy to answer any questions from 
members of the subcommittee, and I am sorry I went over just a 
little bit.
    [The statement follows:]

              Prepared Statement of Senator Orrin G. Hatch

    Mr. Chairman, thank you and Senator Specter for inviting me 
to testify today.
    As a long time supporter of biomedical research, I applaud 
the bipartisan leadership this Subcommittee has demonstrated in 
working toward the goal of doubling the NIH research budget by 
2003. This investment in biomedical research is helping to 
usher in a new age of science in which the mysteries of human 
health and disease are unraveled.
    Today's hearing centers on a major opportunity presented to 
the biomedical research community: stem cell research. As Nobel 
laureate and former NIH Director, Harold Varmus, has 
characterized the situation by saying that, it is not 
unrealistic to say that [stem cell research] has the potential 
to revolutionize the practice of medicine.
    I would like to take this opportunity to share with you how 
I came to my decision to support federal funding for embryonic 
stem cell research.
    Over many months, I devoted hours of study to this 
important issue, reflecting on my spiritual teachings, the law, 
the science, and the ethical issues presented by embryonic stem 
cell research.
    And let me be absolutely clear: I hold strong pro-life, 
pro-family values and strongly oppose abortion. I conclude that 
support of embryonic stem cell research is consistent with and 
advances pro-life and pro-family values.
    I would like to submit for the record copies of my letters 
to President Bush and Secretary Thompson in which I give my 
views on this issue.
    And I would also like to submit for the record a statement 
by former Secretary of Health and Human Services, Dr. Louis 
Sullivan in which he gives his support for embryonic stem cell 
research.
    Let me emphasize four points for you this morning.
    First, I think that support of this vital research is a 
pro-life, pro-family position. This research holds out promise 
for more than 100 million Americans suffering from a variety of 
diseases including heart disease, multiple sclerosis, 
Parkinson's, Alzheimer's, ALS, cancer, and diabetes.
    Second, in the in vitro fertilization process, it is 
inevitable that extra embryos are created, embryos that simply 
will not be implanted in a mother's womb. As these embryos sit 
frozen in a test tube, outside the womb, under today's 
technology, there is no chance for them to develop into a 
person.
    While I have no objection to considering ways to foster 
adoption of embryos, there are a host of issues associated with 
this which must be worked out. And while those issues are being 
considered, the reality today is that each year thousands and I 
am told the number may be tens of thousands--of embryos are 
routinely destroyed. Why shouldn't these embryos slated for 
destruction be used for the good of mankind?
    Third, while I understand that many in the pro-life 
community will disagree with me, I believe that a human's life 
begins in the womb, not in a petri dish or refrigerator.
    It is inevitable that in the IVF process, extra embryos are 
created that will simply not be implanted in a mother's womb. 
To me, the morality of the situation dictates that these 
embryos, which are routinely discarded, be used to improve and 
extend life. The tragedy would be in not using these embryos to 
save lives when the alternative is that they will be destroyed.
    Fourth, there is no guarantee that any stem cell research 
will reap the benefits we hope, but it is clear that embryonic 
stem cell research holds tremendous promise. Some hold out 
adult stem cell research as a good alternative. By all means, 
we should continue adult stem cell research. But, I do not 
believe it would be wise to cut off support for embryonic stem 
cell research, since many eminent scientists believe it is the 
more promising avenue of research.
    The Committee will hear from scientific experts this 
morning. You will hear from NIH about the report on stem cell 
research that the agency will formally issue today.
    While I am not a scientist, my preliminary reading of the 
report strongly suggests that embryonic stem cell research may 
have some substantial advantages over adult stem cells at least 
at this stage of the research.
    Consider the following excerpts from the summary of the new 
NIH Stem Cell Report:

    ``Stem cells in adult tissues do not appear to have the 
same capacity to differentiate as do embryonic stem cells or 
embryonic germ cells.''

           *       *       *       *       *       *       *

    ``Human embryonic stem cells can be generated in abundant 
quantities in the laboratory and can be grown (allowed to 
proliferate) in their undifferentiated (or unspecified state 
for many generations.''

           *       *       *       *       *       *       *

    ``. . . researchers have had difficulty finding laboratory 
conditions under which some adult stem cells can proliferate 
without becoming specialized.''

           *       *       *       *       *       *       *

    ``Current evidence indicates that the capability of adult 
stem cells to give rise to many different specialized cell 
types is more limited than that of embryonic stem cells.''

    However, it is also important to note what the NIH report 
does not say. It does not say that the promise of embryonic 
stem cell research obviates the need to pursue adult stem cell 
research. The report indicates that both embryonic and adult 
stem cell research hold great promise. I believe that both 
avenues should be zealously pursued.
    In the end, it is my hope that we are able to conduct 
research that will improve and prolong human life. I truly 
believe that cures for diseases like diabetes, Parkinson's, 
Alzheimer's, ALS, diabetes, multiple sclerosis, heart disease 
and cancer can be found if we continue with this research. 
That's why we must take advantage of all ethical and promising 
types of research.
    Before I close, I'd like to comment on the work of the 
Jones Institute for Reproductive Medicine in Norfolk, Virginia, 
which is creating embryos in order to conduct stem cell 
research. I find the work of the clinic extremely troubling.
    To me, this type of research is indicative of the problems 
we will continue to encounter if we don't allow federal funding 
with strict research guidelines for embryonic stem cell 
research. As this case illustrates, without stringent, NIH 
ethical requirements, we are opening the door to an array of 
different research standards, which I believe could create some 
serious consequences.
    Mr. Chairman, today we stand on the threshold of a great 
opportunity. Embryonic stem cell research may be the single, 
most important scientific discovery in our lifetimes. The most 
renowned scientists in the country have told us that this 
research holds forth the promise of treatments and perhaps 
cures for some of the most debilitating diseases affecting our 
nation, and the world. I think it would be a mistake to cut off 
federal support for this research.
    I appreciate the opportunity to testify before your 
Subcommittee and would be happy to answer any questions from 
members of the Subcommittee.

    Senator Harkin. Shut those lights off anyway. Do not pay 
any attention to those lights.
    Thank you very much, Senator Hatch. I do not know what your 
time frame is, but I would like to have all the Senators 
testify.
    Senator Hatch. I do need to go to Judiciary, but I will be 
glad to wait.
    Senator Harkin. I would appreciate that.
    Next we turn to Senator Bill Frist of Tennessee, elected to 
the Senate in 1994, who is the only physician serving in the 
U.S. Senate. He serves on Budget, Foreign Relations, and 
Health, Education, Labor, and Pensions Committees, received his 
undergraduate degree from Princeton University and his M.D. 
from Harvard Medical School.
    Senator Frist, please proceed as you so desire.
STATEMENT OF HON. BILL FRIST, U.S. SENATOR FROM 
            TENNESSEE
    Senator Frist. Thank you, Mr. Chairman. I want to thank 
both you and Senator Specter for the opportunity to share my 
views on what is a very challenging moral, ethical, and policy 
issue that is before us today and over the coming weeks.
    Mr. Chairman, it is easiest for me to make four points, but 
all four of these points stem from my personal experience of 
spending about 20 years in the field of training and practicing 
medicine, and before coming to the U.S. Senate the most 
significant aspect of my professional practice was the 
transplantation or transfer of living tissue from one 
individual to another on a daily basis, and on a nightly basis 
I dealt with issues surrounding life and death and health, 
obtaining informed consent in order to transfer tissues from an 
individual in many ways otherwise healthy to another, to the 
benefit of other people.
    Four points I would like to make:
    No. 1, I too am pro-life, oppose abortion, and in terms of 
my voting and policy record here in the U.S. Senate it is very 
consistent or 100 percent consistent with that position. I 
mention that philosophy because in every one of our cases we 
are going to come back and what we ultimately decide is going 
to be colored on our own spiritual beliefs, on our own moral 
beliefs, on the experience we have had. Mine happens to have 
been in the field of medicine and of science, where I spent 
these 20 years.
    Again just so people will understand, because it does color 
my views, there is no question about it, I believe that we have 
a normal progression of life that begins with fertilization, 
continues through the blastocyst phase, continues through the 
embryo phase, continues through the fetal stage, continues 
through what we know as birth to the child, to the adolescent 
and to the adult. It is a continuum to me.
    I do give moral significance to the embryo and I indeed 
give moral significance to the blastocyst, unlike some of the 
other comments that have been made earlier today.
    No. 2, I am a transplant surgeon. I have served on ethics 
committees of individual hospitals. I have served the United 
Network for Organ Sharing, which is the coordinating body, the 
registry for the transplantation of all human tissue or all 
human organs in the United States of America, a body that was 
set up by our U.S. Congress to coordinate, to ensure that there 
is full public accountability and transparency for the 
transplantation of tissues and organs.
    I have been author of scores of peer-reviewed papers in the 
medical literature on transplantation, the ethics surrounding 
transplantation as well. I, as an individual, wrestle with the 
decisions of life and death and health and healing. I have had 
the opportunity to routinely deal with transplanting tissue 
into six day old babies who would otherwise die without that 
transplant of a heart and transplanting people well into their 
fifties years of age.
    I have had the blessing to see the miracle, the miracle, 
that results from the transplantation of tissue, of taking a 
beating heart out of an individual who has healthy lungs and a 
healthy kidney and a healthy liver and taking that heart and 
placing it into another for the benefit of others.
    I have had the opportunity to see a very rigorous consent 
process which we developed in 1968 through the seventies, a 
rigorous consent process that is well established, which makes 
sure that we avoid the potential abuse that is inevitably 
associated with the use of a scarce tissue that literally gives 
life as well as hope to other individuals.
    Based on this experience, I am absolutely convinced, based 
on the knowledge and the experience, but also what we as policy 
makers that we can do, that we can address the use of living 
tissue, of living cells that otherwise would not be used, that 
otherwise would not be used--the words are tough, but 
discarded, disposed of. That particular subset of tissues I 
believe, with an appropriate ethical construct, we can use that 
tissue to the benefit of hundreds of others, thousands of 
others, maybe millions of others.
    It was not easy in transplantation. We addressed many of 
these issues in the 1960's and 1970's. We defined brain death 
in the year 1968. It was debated in Congress. It was debated in 
the scientific literature. That sort of public discourse we 
have to have today, I believe. That addressed issues of human 
beings at 15 years of age and 30 years of age and 50 years of 
age.
    But now we are much earlier in this life cycle, but we are 
addressing the same issues of informed consent, of using living 
tissue.
    The consent process itself is inadequate today. We 
absolutely must have a comprehensive consent process to avoid 
abuse, avoid the potential for commercialization, avoid the 
potential for incentives, of traveling down the so-called 
slippery slope.
    No. 3, is research. You know, we have to be very careful. 
Everybody will put this long list of diseases: Alzheimer's, 
Parkinson's, diabetes, diseases that we are all exposed to. We 
need to make it very clear to our colleagues and to people 
broadly, this is untried, untested research. Huge potential, 
huge potential, yes, but it is just that, it is potential. It 
is untried. It is untested. Yes, we need the research itself, 
but let us not put every hope for every disease to be cured by 
this one aspect of research. But huge potential.
    It has become to mean hope to most people and that is good, 
and I love it when people come up and say, that is the only 
hope that we have for the cure of my child. That is really not 
true. There are lots of other areas that we can explore as 
well. But it is important. It is evolving science. Whatever 
structure we set up, I believe we have to set it up in a way 
that will continue to address new ethical issues that are 
introduced.
    We have seen it in the newspapers over the last 2 weeks. We 
do not know what is being done today. We certainly do not know 
6 months from now, and whatever ethical construct we set up, we 
must have it ongoing and responsive as we go forward.
    There is the potential for abuse of this research. How far 
should we go? Scientists will say, let us just open it broadly. 
I again as a scientist want to say that we do have to recognize 
that this research can be abused. There is a lack of 
predictability. We must build in the safeguards.
    No. 4, and last point is Federal funding. I will say that 
we should fund adult stem cell research, we should increase 
adult stem cell research, but I also conclude that both 
embryonic stem cell research as well as the adult stem cell 
research should be Federally funded within a very carefully 
regulated, fully transparent framework that ensures the highest 
respect for the moral significance of the human embryo.
    I will just read very quickly ten components of a 
comprehensive framework that I think is very important. Again, 
this comprehensive framework in my mind is the only way that we 
should allow embryonic stem cell research to progress, because 
I believe, based on my experience with the transplantation of 
tissues, that only by having a comprehensive framework such as 
I outline will we be able to progress in a manner that is 
respectful of the moral significance of the human embryo and 
the potential of stem cell research to improve health.
    Those 10 components, and I will just read them:
    No. 1, I would recommend that we ban embryo creation for 
research. I believe that the creation of human embryos solely 
for research purposes should be strictly prohibited.
    No. 2, I would continue the funding ban on the derivation. 
I think we need to strengthen and codify the current ban on 
Federal funding for the derivation of embryonic stem cell.
    No. 3, I would ban human cloning. I would prohibit all 
human cloning to prevent the creation and exploitation of life 
for research purposes.
    No. 4, I would increase adult stem cell research funding.
    No. 5, I would provide funding for embryonic stem cell only 
from blastocysts that would otherwise be discarded. I would 
allow Federal funding for research using only those embryonic 
stem cells derived from blastocysts that are left over after in 
vitro fertilization and would otherwise be discarded.
    No. 6, require a rigorous informed consent process. I will 
not elaborate now on that, but I can tell you, based on the 
well-established consent process with transplantation, it is 
critical, it is critical that we address this in a thorough 
manner. It has not been addressed to date.
    No. 7, I would limit the number of stem cell lines. I would 
restrict the Federally funded research using embryonic stem 
cells derived from blastocysts to a limited number of cell 
lines.
    No. 8, I would establish a strong public research oversight 
system. This oversight mechanism is critical. We did it in 
transplantation in a very successful way. I believe we need a 
national research registry to ensure the transparent in-depth 
monitoring of Federally funded and Federally regulated stem 
cell research and to promote the ethical high quality research 
standards.
    No. 9, require ongoing independent scientific and ethical 
review. I do believe we need ongoing scientific review by the 
Institute of Medicine. I do recommend that we create an 
independent presidential advisory panel to monitor the evolving 
bioethical issues surrounding stem cell research and I believe 
that we should have annual reports to Congress.
    No. 10, I believe we need to strengthen and harmonize the 
fetal tissue research restrictions. Transplantation was a 
little bit later in life. We addressed that 20 to 30 years ago. 
Fetal tissue research we moved into about 6 or 7 years ago. Now 
we are moving earlier in that time line to embryonic stem 
cells, which I believe we should address in the manner that I 
have outlined. As we address the embryonic stem cells, which 
are the early precursor cells, I believe we need to go back and 
harmonize the process for fetal tissue research as well.
    Mr. Chairman, I thank you for the opportunity to outline 
what I view is an appropriate public policy response to one of 
the more challenging moral and ethical issues of our time.
    Senator Harkin. Senator Frist, thank you, as well as 
Senator Hatch, for two thoughtful statements and for your 
involvement in this issue.
    I now will turn to Senator Sam Brownback from Kansas, 
elected to the Senate in 1996. Senator Brownback serves on the 
Commerce, Science and Transportation Committee, as well as 
Judiciary and Foreign Relations. Law degree from the University 
of Kansas, B.A. from Kansas State University.
    Senator Brownback testified last year on this issue before 
this subcommittee. We welcome you back, Senator Brownback.
STATEMENT OF HON. SAM BROWNBACK, U.S. SENATOR FROM 
            KANSAS
    Senator Brownback. Thank you very much, Mr. Chairman, and 
it is a pleasure to be back. Thank you for inviting me to give 
another perspective on this particular issue.
    I want to start off with my thanks and appreciation to you 
and to Senator Specter for holding these hearings, for looking 
at this in depth, and also for your push for doubling the NIH 
funding, which I have strongly supported. I believe it is one 
of the great things that we have done during my tenure of being 
in Congress.
    We are on the edge of a frontier of great discovery, of 
great hope and opportunity for a number of people. That is one 
of the great things that we can do. Similar to the space 
program in the sixties that gave us vision, this one gives us 
great opportunity, and you have led that charge, and I 
appreciate your efforts.
    This is a fast-developing field that we are talking about 
now. The study that was just released, to which Senator Hatch 
pointed, I have not received an advance copy of that either to 
be able to look through. I have looked at the news report 
articles of it. I am hopeful of getting the information. They 
do point out in the report that they do not deal with the one 
issue that I raised to you last time that I testified in front 
of this committee, which I will raise again today.
    That is simply the issue that we all on this panel, I 
believe, all in this room, agree that this embryo is alive. The 
central question remains is it a life, or is it a mere piece of 
property to be disposed of as its master chooses? That is the 
central legal and ethical question that we have in front of us. 
It was there a year ago, it remains here today for us to 
examine: It is alive; is it a life?
    We really have to determine the answer to that question 
before we can proceed forward with any of the hope or the 
promise of what one might want to try to consider with this.
    The field is fast developing. Last week a group in Virginia 
announces that they were creating embryos for research 
purposes. They were creating the most hardy ones of the eggs 
and the sperm joined together to get the most robust stem cell 
line.
    I come from an agricultural background. Those are livestock 
terms, when you create a robust and hardy line, which is what 
they were talking about. I think that is a terrible thing that 
they are moving towards in the Virginia group.
    Last week we also had an announcement from Massachusetts 
that for a year now a group there has been working on human 
cloning. I want to talk about human cloning a little bit more 
in my discussion, because I think inevitably the direction that 
we are moving leads towards human cloning. I want to develop 
that point with you if I could in a moment.
    Second, we now have the other side of the face of this, and 
I would like to share, if I could, a face of the human embryo. 
We had testimony yesterday in the House side and a front page 
lead picture in the New York Times. There is a picture of two 
children adopted from an in vitro fertilization clinic and they 
are human and they are bouncing children today and they are 
beautiful children.
    Here is another one, if I could point to a picture of 
Hannah when she is conceived, when she is frozen, when she is 
adopted, implanted, the development takes place, and where she 
is in April at age 28 months. Hannah was frozen in an in vitro 
fertilization clinic and is another face I think that we should 
also be considering as we really discuss this issue. Clearly 
they are alive, are they a life.
    I applaud President Bush for his principled stand that he 
has taken to date on this issue. I think it is bold and quite 
principled in the stand for human life. I would like to submit 
to the record his letter that he sent to the Culture of Life 
Foundation May 18, 2001 of this year.
    Senator Harkin. Without objection, that will be included.
    [The information follows:]

                  Letter From President George W. Bush

                                           The White House,
                                      Washington, DC, May 18, 2001.
Mr. Robert A. Best,
President, Culture of Life Foundation, Inc.,
Washington, DC.
    Dear Mr. Best: Thank you for your letter about the important issue 
of stem cell research.
    I share your concern and believe that we can and must do more to 
find the causes and cures of diseases that affect the lives of too many 
Americans.
    That's why I have proposed to double funding for National 
Institutes of Health medical research on important diseases that affect 
so many American families, such as breast cancer. My proposal 
represents the largest funding increase in the Institutes' history. I 
also have called for an extension of the Research and Development tax 
credit to help encourage companies to continue research into life-
saving treatments.
    I oppose Federal funding for stem-cell research that involves 
destroying living human embryos. I support innovative medical research 
on life-threatening and debilitating diseases, including promising 
research on stem cells from adult tissue.
    We have the technology to find these cures, and I want to make sure 
that the resources are available, as well. Only through a greater 
understanding through research will we be able to find cures that will 
bring new hope and health to millions of Americans.
            Sincerely,
                                            George W. Bush,
                                                         President.

    Senator Brownback. Thank you.
    Mr. President, let me also say that I testify with a number 
of my colleagues today, and all of us share a deep and abiding 
desire to cure the diseases which plague humanity--as do the 
people on this committee. Can we? Are we on the brink of being 
able to solve such terrible diseases as Parkinson's, ALS, 
diabetes, juvenile diabetes, and cancer? I think we are, and I 
think there is a right route that we can go with this, and I 
think it is the adult stem cell route that does not have the 
ethical and moral questions that we have surrounding the 
embryonic, stem cells. Adult stem cell research is also showing 
a great deal of promise by the NIH reports and studies, and it, 
is also showing fewer of the problems that we are seeing with 
embryonic, stem cell research.
    Two weeks ago we had a report out in Science magazine 
saying the embryonic stem cells are not stable. I know my 
colleague Dr. Frist has pointed out that we should not hang all 
of our hopes on this research. It looks promising, but there 
are a lot of questions.
    We know from this report, the NIH report, the earlier 
studies, earlier reports released from it, that some of the 
embryonic stem cells are creating tumors. These are fast-
growing cells. In many respects they may be placing the new 
wine in old skins parable that we see in front of us. We are 
not certain, but we do see that in some of the early and 
preliminary results.
    Far from lagging behind the embryonic stem cell, the adult 
stem cell lines are already being used in human patients to 
assist in recoveries, from cancer and leukemia, restoring sight 
to the blind, curing severe combined immune deficiency, repair 
damaged bone and cartilage.
    On the contrary, recent animal trials using embryonic stem 
cells have shown a disturbing tendency for these cells to form, 
as I stated, uncontrollable tumors when transplanted. To my 
knowledge, and my contact with the scientific literature 
confirms this--I am not a scientist, but I do read--there is no 
embryonic stem cell work even close to treating humans today.
    I would also like to point out that many of the proponents 
of destructive embryo research are now advocating the so-called 
use of therapeutic cloning. I want to put this term on the 
table and I want to put this term into the debate because that 
is where this issue is headed. At a recent hearing that I held 
on the issue of human cloning, both the president of 
Biotechnology Industry Association and a representative of the 
American Society for Cell Biology emphasized their strong 
support for so-called therapeutic cloning as the ultimate 
source of embryonic stem cell that will not face rejection.
    Therapeutic cloning is where you take the young embryo, or 
in some cases you can take an egg, de-nuclei it, take DNA from 
my skin cell, yours, somebody here, insert it into the egg or 
the embryo, start it growing again for a period of several 
days, and then harvest--and I use that term again because we 
are talking almost in livestock terms here--harvest the stem 
cells that will genetically match the person for whom we seek 
to be able to fix, repair or replacement an organ.
    Both of these gentlemen testified that they are going to 
need and that they strongly support therapeutic cloning. This 
is the route they prefer to go to get stem cells that 
genetically match up.
    Dr. Rudolph Jaenisch testified that with therapeutic 
cloning no rejection will occur because these cells, which come 
from the cloned embryonic stem cell, are the same immunological 
makeup as the patient's cells. The testimony of both Dr. 
Jaenisch and Mr. Feldbaum recognizes that for the purposes of 
possible clinical applications, particularly to avoid possible 
tissue rejection, human cloning is the logical next step or so-
called therapeutic cloning. This means that live embryos 
created by researchers can be experimented on and then 
disemboweled at the leisure of the researchers for purported 
benefits of patients.
    This is truly a slippery slope. Cloning of humans should be 
and I believe it must be stopped. I say ``slippery slope'' 
because the current proposals seek to undo any principled 
limitation by rejecting true principle on this. The principle 
being denied in this case is the dignity of the young human, 
effectively making the human embryo equal to mere plant or 
animal life or property, or even livestock, to be disposed of 
according to human choice, governed by mere legal and pragmatic 
considerations.
    Is that really the direction we want to go? I want to cure 
these diseases, and I think we have an ethical route to go as 
well with the adult stem cells.
    How many of us were repulsed when we heard of cases in 
other countries, in China where people were executed and then 
their organs harvested? Now, you could say, well, they are 
being destroyed and somebody is going to be saved. Somebody is 
going to get a heart that needs a heart out of this. Yet do you 
not just repulse when you hear that? This is not the right way 
to go.
    The embryo is alive. Is it a life, or is it mere property? 
We really must address this question first and foremost.
    Finally, Mr. Chairman, I want to make the point of this. I 
have two letters from people who suffer from terrible diseases 
who do not want to see embryonic stem cell work move forward. I 
would ask for that submission into the record.
    [The information follows:]

                     Letter From Christopher Currie

                                                     July 17, 2001.
Hon. Sam Brownback,
U.S. Senate,
Washington, DC.
    Dear Senator Brownback: People who suffer from diseases shouldn't 
have to destroy their souls to save their bodies.
    Yet that's exactly what some medical-research advocates would have 
us do by persuading Congress to fund therapies that would involve 
killing living human embryos in order to treat victims of certain 
diseases.
    I am one of those disease victims. Diagnosed as a Type I diabetic 
at age 11, I've been insulin-dependent for the past 27 years. My 
treatment regimen consists of continuous insulin infusion through a 
wearable pump, at least four daily blood tests to measure plasma 
glucose levels, and careful control of diet, exercise and other daily 
activities.
    In addition to the threat of sudden death from insulin shock or 
ketoacidosis, I must contend with the advance of several complications 
of the disease that may lead to blindness, kidney failure, loss of 
limbs, chronic pain, stroke, heart disease, and, ultimately, death. 
Knowing that I can expect to lose a third or more of a normal lifespan, 
I worry about my wife and two young children, for whom I am the sole 
source of support.
    Stem-cell research may hold promise for diabetics like me who 
desperately hope for a cure. But treatments that depend on the 
destruction of human embryos will not help me or many thousands of 
patients like me. To do so would make us accomplices in the deliberate 
destruction of life for research purposes.
    It's easy enough for many to dismiss the claims upon our 
consciences from unseen and unheard human embryos, especially when 
they're focused on the conquest of a disease or perhaps the sufferings 
of a loved one. But when you enter into a relationship with that tiny 
human being, the relationship between donor and recipient, the moral 
weight of that little one is much harder to ignore.
    Would that person have had the chance to live, to love, to 
experience all the joys of life that I have, if she hadn't been killed 
to help me? These are the kinds of questions that torment the disease 
victim who contemplates a treatment purchased at the cost of another 
human life, and they are not so easily dismissed.
    In my view, Congress should spend our tax dollars on treatments 
that all taxpayers can support, and that all patients can accept 
without violating their consciences. Fortunately, there are 
alternatives, including research that involves stem cells taken from 
adult donors and umbilical cord blood.
    Congress need never sanction the destruction of some human lives in 
order to help others. Please honor your own consciences as you honor 
those of millions of others like me.
            Very truly yours,
                                                Christopher Currie.
                                 ______
                                 

                        Letter From Julie Durler

                                                     June 30, 2001.
Hon. Sam Brownback,
U.S. Senate,
Washington, DC.

RE: Funding for Diabetes Research

    Dear Senator Brownback: I was recently made aware of a hearing last 
week before the Senate Permanent Subcommittee on Investigations (Senate 
Government Affairs Committee) wherein actress Mary Tyler Moore and 
others requested increased funding for diabetes research and support 
for embryonic stem cell research which she called ``truly life 
affirming''.
    As an insulin-dependent diabetic myself for the past 16 years, I 
would like to see a cure for diabetes. I know that through research a 
cure can be found (or at the very least improvements in the treatment 
for diabetes). However, I do NOT support embryonic stem cell research 
to accomplish this goal. I believe it is wrong to use stem cells taken 
from aborted babies (or any embryo/fetus if it means the death of such 
child) for research. Stem cells taken from consenting adults for 
research is another matter, and one which I would support.
    What is your position on using embryonic stem cells for research 
for diabetes or other medical ailments? Will you support a ``truly'' 
life affirming stand and support increased funding for diabetes 
research ONLY if it DOES NOT provide for the use of embryonic stem 
cells in the research?
    Thank you for your time. I look forward to your response.
            Sincerely,
                                                   Julie M. Durler.

    Senator Brownback. I would point out as well that this is 
an issue of enormous magnitude. It should not be done in an 
appropriations bill. This should be done free-standing on the 
floor, with a lengthy debate. We are going into a new bold, 
some would refer to brave, new world. I think this is an area 
that we should have set on a free-standing bill with thorough 
discussion and debate, and not something that we would do as 
part of an appropriations process.
    If that were to occur, there would be a lot of vigorous 
debate on the floor about doing something of this magnitude and 
this nature in an appropriations bill.
    Thank you very much for your heart and for your patience.
    Senator Harkin. Thank you very much, Senator Brownback, 
again for a very thoughtful statement now and the one last year 
also.
    We now turn to Senator Gordon Smith, elected to the Senate 
in 1996, a member of the Energy and Natural Resources, Foreign 
Relations, Budget, Commerce, Science, and Transportation 
Committees. Senator Smith graduated from Brigham Young 
University, the same as Senator Hatch, and received his law 
degree from Southwestern University.
    Senator Smith, welcome. Please proceed.
STATEMENT OF HON. GORDON SMITH, U.S. SENATOR FROM 
            OREGON
    Senator Smith. Thank you, Mr. Chairman and colleagues of 
this committee.
    Each of us as U.S. Senators come to this place, this public 
place, as the sum of our beliefs, our experiences, and our 
values. None of us checked them at the door when we came here. 
I thought in the spirit of trying to be helpful to your 
deliberations I would share with you what I have experienced 
and what I believe.
    As a young boy I watched my Grandmother Udall die of 
Parkinson's disease. Growing up, I watched my cousin 
Congressman Morris Udall literally die in public of Parkinson's 
disease. Last April I buried my uncle Addison Udall of 
Parkinson's disease. Last weekend my brother-in-law Dan Daniels 
informed me that he now suffers from Parkinson's disease.
    In the experience of my life I have not been a stranger to 
hospitals and trying to provide care and comfort to those who 
suffer and seek to be well. So for me, this debate presents me 
with the ultimate question, one for which I believe I will be 
held accountable in this life and hereafter. That question is, 
when does life begin? Senator Brownback has stated it well.
    Some say it is at conception. Others say that it is at 
birth. For me in my quest to be responsible and to be as right 
as I know how to be, I turn to what I regard as sources of 
truth. I find this: ``And the lord God formed man of the dust 
of the ground and breathed into his nostrils the breath of 
life, and man became a living soul.'' This allegory of creation 
describes a two-step process to life, one of the flesh, the 
other of the spirit.
    Cells, stem cells, adult cells, are I believe the dust of 
the earth. They are essential to life, but standing alone will 
never constitute life. A stem cell in a petri dish or frozen in 
a refrigerator will never, even in 100 years, become more than 
stem cells. They lack the breath of life. As an ancient apostle 
once said: ``For the body without the spirit is dead.''
    I believe that life begins in the mother's womb, not in a 
scientist's laboratory. Indeed, scientists tell me that nearly 
one-half of fertilized eggs never attach to a mother's womb, 
but naturally sluff off. Surely life is not being taken here by 
God or by anyone else.
    For me, being pro-life means helping the living as well. So 
if I err at all on this issue, I choose to err on the side of 
hope, healing, and health. I believe the Federal Government 
should play a role in research to assure transparency, to 
assure morality, to assure humanity, and to provide the ethical 
limits and moral boundaries which are important to this issue. 
Those boundaries and limits must stop at a mother's womb, for 
again that is where life begins. The puritans called it a 
quickening, and the scriptures, I think speak to that effect as 
well.
    In conclusion, I say with deepest respect for those who 
have views different from my own, both theologically or 
scientifically, that I respect those views, but share with you 
my own views and my own experience in seeking cures for the 
most dreaded diseases on this planet. We are at a confluence 
between science and theology. I believe we must err on the 
broadest interpretations to do the greatest amount of good. It 
is in that spirit, Mr. Chairman, that I am here, and I thank 
you for this time.
    Senator Harkin. I am going to yield to Senator Specter for 
questions, but before I do I just want to say, I think in my 27 
years that I have been privileged to serve in both the House 
and the Senate, I do not believe I have ever heard testimony as 
powerful and as deeply thought out and as moving as what I have 
just heard from the four of you. Regardless of whether I may 
agree or disagree, I think you have touched me very deeply.
    I just want all who are in this room, I hope all who have 
heard your testimony and those who may be watching--I believe 
you really have raised the stature of the U.S. Senate. I think 
people who are listening and watching, I hope they have a 
renewed faith in and confidence that those of you and those of 
us who serve here do not take these issues lightly. These are 
things that we wrestle with on a daily basis, and we try to 
come up with what we hope are the best results and solutions 
that will enable us to adhere to our basic values and our 
religious beliefs and yet try to meet the needs of people who 
are suffering.
    So I just want you all to know that I really appreciate 
your testimonies and the thought and concern and the compassion 
which each of you have brought to this issue. You do us proud.
    With that, I will turn to Senator Specter.
    Senator Specter. Thank you, Mr. Chairman.
    Supplementing what Senator Harkin said, the U.S. Senate is 
frequently quoted as the world's greatest deliberative body. 
Occasionally people watch on C-SPAN2 have some reason to doubt 
that. But anybody who has heard this testimony I think would 
concur that the quality of deliberation is extraordinary in 
thoughtfulness.
    While Senator Brownback and I have some disagreements on 
this issue, I agree with him that this matter ought to be taken 
up with full debate. An appropriation bill may turn out to be 
the appropriate line because right now the prohibition comes 
from an appropriation bill out of this subcommittee from 1995. 
But on the substantive matters about having thorough 
consideration, I believe you are correct, Senator Brownback.
    I thank you, Senator Hatch, for your very, very thoughtful 
testimony, coming at the issue in so many directions with your 
experience and your stature in the Senate, and a strong pro-
life, pro-family advocate for so many years, having been 
elected here in 1976.
    Senator Gordon Smith, very profound as you articulate those 
views. Senator Frist, we are very appreciative for your 
appearance here today. You and I have discussed this matter on 
many, many, many, many occasions and I am glad to see your 
position coming forward today, and as you articulate 10 
standards, I agree with you about eight, but I want to discuss 
with you the two remaining.
    We will later here today testimony from Dr. Diane Krause, 
and let me lay out a couple of lines of concerns. Dr. Krause 
would take issue with the standards which you have articulated 
on number two and number seven, which are really overlapping: 
number two, the continue the funding ban on derivation, that is 
the Federal funding ban; and number seven, to limit the number 
of stem cell lines.
    Dr. Krause in her written testimony has raised this issue, 
that the proposals to limit funding research to only existing 
cell lines is far too limiting. Scientists need to compare 
multiple cell lines in order to better understand the common 
factors that give embryonic stem cells their incredible 
plasticity, as she put it.
    Dr. Lawrence Goldstein, professor of pharmacology at the 
Division of Cellular and Molecular Medicine at the University 
of California at San Diego, has raised a concern about the 
current number of stem cell lines being insufficient because 
you need more genetic variety. The current stem cell line 
established by NIH is only 30 and there are suggestions by 
other scientists in the field that you need a minimum of 200.
    Then let me put the second consideration before you all at 
the same time. When we talk about the NIH guidelines, they 
would not be met by the existing cell lines because embryos 
must have been frozen, there is not sufficient documentation of 
informed consent, which I think you are exactly right about, 
and the prohibition against creating for medical research 
purposes the cell lines is part of the NIH guideline. There 
again, I agree with you that if we do not legislate on this 
field in a comprehensive way we have the problems of cloning, 
which trouble everyone, and the problems of creating these 
embryos for medical research, which again are extraordinarily 
cloning.
    But in this area, Senator Frist, Dr. Frist, I think we join 
a critical issue which our colleagues will be looking at when 
we examine the scope of legislation on this important subject.
    Senator Frist. Thank you, Senator Specter. I do--let me 
comment on several of the points, and then I know that you have 
a lot of people to talk specifically about these.
    First of all, there are major differences with what I 
proposed and what you have in an underlying bill, and I think 
we pointed to that's where the differences are. I do not 
believe we should be funding derivation, which you mentioned. 
The consent process, the informed consent process, we 
absolutely must address, which I believe again it is something 
that we have not focused enough attention on.
    I believe we should ban human cloning, which is another of 
the issues which in your legislation you do not. And I do not 
believe that you ban the creation of embryos for research only. 
I really feel strongly on this publicly accountable oversight 
system.
    Those are sort of the big differences, I think. I summarize 
those right now so that the American people understand the 
differences. Cell lines, number one, it is worth talking to the 
scientists about that. What are cell lines? First of all, 
transplantation. When I transplant a human heart, I take it 
from one individual who otherwise has normal kidneys, lungs, 
blood is flowing, warm, the like, take that heart and put it 
into another person, and that helps that one individual.
    The really exciting thing about embryonic stem cells, it is 
not sort of a one for one. It is not like that you have to take 
a blastocyst, which is really 20 to 30 inner cells coupled with 
an outer supportive framework. You do not have to take one of 
those to do one--help one research project and then take 
another one to help another research project or another. The 
great power and potential--again, it is unpredictable--of these 
is the fact that these will perpetuate themselves forever and 
that once you take a cell line, that cell line you can grow and 
you can grow it in an identical fashion and share with 
researchers all over the world, as long as that informed 
consent. That is the real power.
    Therefore, how many cell lines do we have today? We can 
talk more about that with the scientists, but we maybe have 7, 
10, 12 cell lines today. That is a pretty good amount. Some 
will say you can limit it just to that. I would propose that we 
have a discussion with the scientists to say, how many do you 
really need. The point is you do not need thousands and 
therefore you do not need thousands of blastocysts to do this. 
It might be 20, it might be 30, it might be 100, or you 
mentioned 200 cell lines. What is a cell line? A cell line is 
in essence the genetic--or the makeup of a single blastocyst or 
embryo.
    I think that is an important concept because people have in 
their image that all these embryos are going to be created and 
they are living and there is going to be destruction of these 
embryos that is going to go on millions and millions and 
thousands of times. It is just important for people to 
understand you do not need unlimited cell lines. Exactly how 
many, I think it is worth talking to the scientists about.
    The informed consent process we mentioned. I will stop. I 
should probably stop there, but I think this larger moral, 
ethical construct which is inadequate in NIH guidelines today, 
using informed consent as one example, absolutely must be 
addressed. Therefore, I would urge also that we do not rush 
with a very narrow bill on an appropriations bill that does not 
take that into consideration.
    Senator Specter. Senator Frist, the question as to how many 
cell lines let us leave open. Let us get the scientific 
interpretation. The information I have heard is that we need a 
lot more than 7 to 12. Two hundered may be a line. Let us rely 
on the scientists on that.
    The one point where I do think we have a fundamental 
disagreement is on the Federal funding. The current status of 
the appropriations law is no Federal funds may be used on stem 
cell derivation from embryos, and that has been interpreted by 
an opinion from general counsel in HHS a couple of years ago to 
prohibit Federal funding to extract stem cells from embryos, 
but not to prohibit Federal funding to research on the stem 
cells once they have been extracted.
    Senator Frist. Yes, sir.
    Senator Specter. As I understand your testimony, you want 
to maintain the ban, which would limit Federal funding to 
extract stem cells from embryos.
    Senator Frist. That is correct.
    Senator Specter. We are prepared on the Specter-Harkin bill 
to yield to you on 8 of your 10 points, making it the Frist-
Specter-Harkin bill. But I think we are not prepared--I will 
let Senator Harkin speak for himself--on the issue of funding 
for extracting stem cells from embryos, because of our 
concerns.
    Everybody has applauded what we have done, the leadership 
from this subcommittee on increasing NIH funding from $12 
billion to more than $20 billion. If this year's idea goes 
through, we will have doubled it. But when you use the private 
sources, first of all, in a marketplace society they will do 
for profit. When NIH is doing the research on extracting stem 
cells from embryos, they are subject to the ethical 
considerations and also to the brilliance within the NIH family 
and the grants which they may allow.
    So, moving right to the core of the issue, how can you 
adequately deal with this vital, vital subject if you do not 
have these NIH funds, which we have increased so dramatically, 
available for the critical aspect of extracting stem cells from 
embryos?
    Senator Specter. Yes, sir. Very quickly, the overall 
ethical-moral construct, including the bioethics advisory 
committees, oversight, registry, would apply quite broadly. I 
think it is absolutely critical that you link the last question 
to this question. We do not need an unlimited number of stem 
cell lines. Therefore, for each experiment, for each 
investigation, you do not have to go out and take another 
blastocyst, take out those 20 or 30 cells, grow them, and start 
from scratch. That is why this is so different in terms of the 
power and the potential.
    You just said I am not sure whether we need 7 cell lines or 
25 cell lines or 50. You could also argue you should be doing 
it in animals instead of humans as you determine just that and 
make these comparisons, but that is a separate issue. But let 
us just say you need 100 cell lines. I think if you talk to 
enough scientists they will say, for right now, given where we 
are in terms of the evolution of science, the potential for 
abuse, the lack of an ethical construct in which to both debate 
and carry on public discourse, that you do not need 200 cell 
lines.
    But even if, let us say you needed 100 cell lines. The 
derivation of the cells, the taking of 20 cells out, would have 
to occur 20 times or 40 times or 60 times or 80 times. It is 
not an ongoing process because once those cells are taken out 
you can do thousands of experiments with thousands of 
investigators all over the world.
    That is why it is important. So the derivation to me, I 
think you are exactly right, we have heard of the great moral 
challenges we have today in terms of are you destroying. 
Senator Brownback, that is a human being to Senator Brownback. 
You are destroying that, and you are. It is going to be 
discarded otherwise, but in his mind.
    Therefore, to take Senator Brownback's taxpayer dollars or 
the dollars of his constituent to do something, even on a very 
limited basis, that is against his moral conscience, we are not 
ready to do as a society.
    Senator Specter. Well, Dr. Frist, I understand what you are 
saying and I am prepared to, as you seem to be, rely upon the 
scientists to tell us how many cell lines we need. But I am not 
prepared to leave it to the private sector to extract the stem 
cells from the embryos. I think that is where we have our 
disagreement.
    I think that with the NIH expertise and their ethical 
standards and the funding which the Congress has provided, all 
of those resources ought to be brought to bear on the removal 
of the stem cells from the embryos on this very important 
subject.
    I thank you very much, Dr. Frist. There are a lot of 
questions which could be asked of Senator Hatch and Senator 
Brownback and Senator Smith, but I will yield that chore to 
others.
    Senator Harkin. Thank you very much, Senator Specter.
    Senator Murray.
    Senator Murray. Thank you, Mr. Chairman. I do not have any 
questions at this time. I just want to thank all of our 
colleagues for obviously a very courageous and thoughtful 
discussion on a very difficult issue. I think you have helped 
us move the debate forward and I appreciate it.
    Senator Harkin. Senator DeWine.
    Senator DeWine. No questions, Mr. Chairman. Thank you very 
much.
    Senator Harkin. Senator Cochran.

               OPENING STATEMENT OF SENATOR THAD COCHRAN

    Senator Cochran. Mr. Chairman, I just want to thank the 
Senators who are here today. I think this is a defining moment 
for the Senate. We are blessed to have these four Senators 
available to us for counsel and advice and information. I am 
pleased we also have technical and scientific guidance from 
others in this process.
    It is truly a matter of life or death. This issue deserves 
our best efforts to learn the facts and act no them, to serve 
the public interest. This is not an issue where we try to be 
just politically correct. Our obligation is more than that. We 
just use our best judgment, enlightened by the best evidence.
    This hearing is a very important step in that process, and 
I appreciate the seriousness with which the committee 
leadership has undertaken to outline an agenda and select 
witnesses to come before us to see that we do the best we 
possibly can on this issue.
    Thank you.
    Senator Harkin. Thank you, Senator Cochran.
    I just would just follow up with what Senator Specter was 
saying. I am glad you clarified, Senator Frist, on limiting the 
cell lines. Now I have a better understanding of what you mean 
by that. But I still have a problem with number two, as Senator 
Specter does. While I agree we do not need an unlimited number 
of cell lines, I wonder about the logic that would allow our 
Federal researchers to work on stem cells, but not to derive 
them. It is sort of, you go ahead and do it, then you give them 
to me, but I do not want to have my hands in it. I do not 
understand that logic. I do not want to be flip about it, but 
it is like, do not tell me what bank you robbed, just give me 
the money.
    I do not want to be flip about it, but I do not understand 
the logic of saying you can work on the cell lines, but you 
cannot derive them.
    Senator Frist. Right. I think the question is very 
legitimate, and for me it is a matter of Federal funding. That 
is the issue that we are talking about. Right now, given where 
we are today in terms of the derivation, when there are a 
significant number of people--and I believe life does begin 
with fertilization. I give moral significance throughout, and 
that is based on the spiritual values and moral values and 
religious values and the medical view that I have.
    Therefore, I think, in terms of taking taxpayer dollars of 
probably 40 percent of the people that are listening to us 
right now and saying that we are going to use that very 
specifically, a part of the process which in truth is very 
small when you look at the research that is going to be done, 
very small--and that is very clear for us to understand. The 
derivation part of it, it does not take that many. We talk 
about maybe 10, 15, 20, maybe 200 cell lines. It is a very 
small part.
    So I think from a moral standpoint it is not right to take 
taxpayer dollars and use that when there are--and I do not know 
what the percentage is, but 40 percent of most people's 
constituents would say it is not the right thing to do, when it 
is not going to slow down the research. I do not believe it 
will slow down the research if you do not, if you do not pay 
for it.
    Senator Harkin. Does anybody have any other thoughts?
    Senator Hatch. Could I just add one thing?
    Senator Harkin. Yes, yes.
    Senator Hatch. It is my understanding that the in vitro 
fertilization process is basically completely private sector, 
although there has been some research done in the Federal 
Government. Maybe I am wrong on that, but I believe I am 
correct. If that is the case, that is where these cells are 
coming from.
    Now, look. I think Senator Frist was very cogent in his 
whole discussion of this. But what it comes down to probably is 
a political process problem, that should the Federal Government 
be paying to extract these cells from a blastocyst, which then 
of course is discarded, and which would have been discarded 
anyway if we had not taken the pluripotent cells from it.
    The House has, I think, has been unwilling to have Federal 
funding of any type of abortion approach, and to some in the 
House they feel this is a similar situation. So I think Senator 
Frist has really described the political situation quite well. 
But as far as stem cell lines, what I have been told is that 12 
to 20 would not be enough, but approximately around 100 would 
probably be sufficient. Senator Specter, you said 200 and I am 
sure there are some scientists who do say that. But the ones 
that I have chatted with have indicated about 100 stem cell 
lines would be adequate.
    But I think that basically, if the Federal Government is 
not doing the in vitro fertilization process itself, they are 
going to get these cells primarily from the private sector. But 
what Senator Frist is saying--and I do not mean to speak for 
him; it is just that I agree with him on all 10 of his points. 
What he is bringing out is that this work has to be done under 
the strictest guidelines and rules and regulations, and NIH has 
those, although they may be enhanced through these hearings and 
other considerations by Congress.
    If that is done, that will go a long way to alleviate some 
of the concerns of some people in our society. But I am 
concerned about the fact of getting the Federal Government into 
the whole process.
    Senator Harkin. Just on that one point, the bioethics 
committee that was set up that was headed by Dr. Shapiro of 
Princeton University--if you look at the list of all of the 
people that are on there, I mean, these are some of our best 
ethicists, philosophers, religious leaders in this country, and 
they came up with these guidelines. Maybe they can be enhanced. 
I do not know. But I thought they were pretty well thought out 
guidelines that NIH had come up with by this distinguished 
group of citizens.
    Senator Hatch. I have no problems with those. I have no 
problems with those guidelines.
    Senator Smith. Mr. Chairman, on your point----
    Senator Harkin. Yes, Senator.
    Senator Smith. I think you are exactly right, that if we 
are going to incentivize this, if we are going to pay for the 
product, but we do not want our hands in the process, we are 
doing the same thing with two steps that we are in one. I 
recognize my colleagues look at this somewhat differently, but 
it is a just give me the money, do not tell me where it came 
from, type of process. That is why I think you cannot go that 
route if you are deeming that this is an inappropriate thing to 
do in the first place. You cannot incentivize the product and 
then say, I do not want to look at the process.
    Senator Harkin. The other question I have is, again for any 
of you, Senator Frist, is if you say, well, we cannot have NIH 
fund the derivation, but we can do the research, if the 
derivation is done in the private sector, that does cost money. 
What incentive will the private sector have to derive the stem 
cells if all they are going to do is give them free to NIH? Or 
is NIH going to have to purchase these? Would that be 
acceptable?
    Senator Frist. Mr. Chairman, I think that would be 
acceptable, but I think the larger issue--in terms of purchase, 
I do not think--that sort of thing in terms of science is not 
uncommon. In transplantation, for example, which is the 
transfer of human tissue from a live entity to another live 
entity, but actually similar in many ways--again, you will see 
I call upon this, but about 30 years ago we had to decide, 
after 2,000, 3,000, or 4,000 years of thinking death is the 
heart and the lung when they stop, we had to redefine it as 
brain death with everything else normal.
    I think we are going through a little bit similar process 
as we work through the definitions. There are two parallels. 
One is that the tissue from transplantation before I take out 
the human heart of a body that is otherwise normal except there 
is one organ gone, but everything else is normal, before I take 
that heart out you go through this consent process, which is 
inadequate in the statute now. The National Bioethics Advisory 
Committee looked at it, so it is inadequate, so it does need to 
be strengthened.
    That body is going to be going into a grave or discarded or 
disposed of, but that is living tissue itself. A lot were 
thinking--or I think we should think of it in a parallel way, 
in that the limitation that I place is in vitro fertilization. 
If a woman gives 20 blastocysts, has 5 implanted, 4 or 5 
implanted, and there are 15 that she freezes, and that after an 
adequate consent process is set up she makes a decision whether 
to offer those, say 20 years later or 10 years later, for 
adoption--perpetual freezing, not many people are going to want 
to do that because they usually charge $1,000 or $500 or 
something a year to freeze them. A lot of people probably are 
not going to do that.
    Those embryos today are discarded. You turn the freezer off 
and that is it. I think we do--I think we should today, because 
there is so much uncertainty, limit the research to the use of 
those cells, similar to transplantation, that otherwise 
absolutely, with full public disclosure, full transparency, 
full oversight by the Federal Government, is going to be 
discarded and not used.
    Senator Harkin. You are not opposed to in vitro 
fertilization?
    Senator Frist. No, sir.
    Senator Harkin. Senator Smith?
    Senator Smith. No, sir.
    Senator Harkin. Senator Hatch?
    Senator Hatch. No, sir.
    Senator Harkin. Senator Brownback?
    Senator Brownback. No.
    Senator Harkin. No. I guess my question is that obviously 
in vitro fertilization is going on right now.
    Senator Frist. Yes, sir.
    Senator Harkin. So there will be, as you point out, left-
over embryos, in essence.
    Senator Frist. Thousands and thousands and thousands.
    Senator Harkin. So if you are just saying--and this is a 
question I do not know, I am going to ask the scientists who 
are coming up here later on, that I understand that somehow the 
ones that are frozen, that there is a problem in the derivation 
of the stem cells.
    Senator Frist. We do not know what the freezing process 
does long-term with those cells. We do not know--also, we 
implant those cells in women, or women have them implanted. But 
in most of the consent forms that are given to women, you do 
not know what the freezing process long-term will do, and 
therefore scientists will come forward and say just freezing 
may drop back some of the totipotential or pluripotential 
components of the cell.
    Senator Harkin. Thank you. You are way ahead of me on that.
    So if in vitro fertilization continues on and a woman and a 
man, the donor, if they have left over embryos at that point 
that are not frozen, could those be used to extract cell lines, 
again going through the ethical guidelines that are set up? We 
were focused so much just on the ones that are frozen. How 
about the ones that are all obviously being generated, I do not 
know how often, in an in vitro fertilization.
    Senator Frist. We are getting pretty technical there. In my 
own mind, IVF involves the creation of surplus--and these words 
are hard for people, discarding, disposing, and surplus. But 
that is the nature of in vitro fertilization today. You have 
to, and so you are going to have 10 or 15. People elect either 
to freeze them or to discard them immediately.
    So from my standpoint, from an ethical standpoint, they do 
not have to be frozen before you use them, as long as you have 
an appropriate consent. I think in transplantation at least, 
and I think it is important here, the consent process, you 
cannot have consent made under duress or when other decisions 
are being made. So I am not sure at the time you are deciding 
whether or not you are going to have children and when you are 
concerned about it and focused on it, that is the best time to 
be making this decision.
    I almost think it should be a two-step consent process. You 
should say, I am going to do IVF, I am going to freeze the 
cells, and at some later point in time go through a consent 
process of saying adoption, discarding, turning the freezer 
off.
    Senator Specter. A final comment, Mr. Chairman. I agree 
with Senator Brownback's reasoning, but come to a different 
conclusion, Senator Frist. When you say that you are prepared 
to have the Federal Government buy the extracted stem cells 
from the embryos, if the Federal Government is going to pay 
private concerns which have extracted embryos from stem cells 
and the taxpayers' money is being used, it is really not even 
indirect. It is very direct. Where is the money coming from?
    But I come to the different conclusion, that if you are 
prepared to do that, Senator Frist, it makes more sense for NIH 
to be involved in the extraction of the stem cells from the 
embryos. A big issue which has arisen here which this 
subcommittee has looked at otherwise is the issue of the 
patents. There are a lot of people out there who want to make a 
lot of money, and if they are extracting the stem cells from 
the embryos and they are going to sell them to the Federal 
Government, it is going to be a lot more costly.
    But if we are going to have the Federal Government pay the 
private concerns, it seems to me much wiser to have NIH do it 
directly.
    Thank you very much.
    Senator Frist. One more statement about transplants. On the 
transplant field, the Federal Government does not pay for all 
transplantation of tissue, nor did it in the research component 
of it. We do have an oversight mechanism where every heart that 
I transplant or any transplant surgeon transplants or lung or 
kidney goes through a registry, where there is appropriate 
ethical oversight.
    I think that that is a similar sort of approach. But I do 
not think we need to pay for everything. In terms of research--
I know you are going to keep arguing for it, I am going to keep 
arguing against it. But I do not think you need to pay for 
every aspect of research, just like the Federal Government does 
not pay for all of transplantation. Through Medicare we pay for 
transplantation and Medicaid, most places do. But there is a 
huge private sector out there today in terms of the transfer of 
tissue. So I do not think you have to pay for all of it.
    Senator Harkin. Well, again I thank you all very much. You 
have just been great and I want to thank you. I especially want 
to thank Senator Smith. I went back and read Genesis after you 
talked to me yesterday and you are right. It gave me a whole 
new way of looking at this. I had not thought about that 
before, and I would commend others to think about what you said 
today in your statement. I appreciate it very much.
    Senator Specter. Senator Harkin, Senator Hutchison has 
joined us.
    Senator Harkin. Did you want to ask questions? I was going 
to let the Senators leave.
    Senator Hutchison. I did want to ask a question.
    Senator Harkin. I would recognize Senator Hutchison.

           OPENING STATEMENT OF SENATOR KAY BAILEY HUTCHISON

    Senator Hutchison. Thank you, Senator Harkin, and I 
apologize for being late. I was on the floor.
    But I wanted to make a brief comment and then I did want to 
ask Senator Frist a question. One of the things that I have 
found through my personal experiences with a family member with 
cancer is that much of the research on cancer by private 
companies is done on diseases where there would be enough 
patients for the return to the private company that is making 
the investment, that many of the diseases that do not have a 
large patient number have been not focused on.
    That is where you think the NIH can make such a huge 
contribution, and our commitment in Congress to double the 
funding of NIH I think makes it important for us to look at all 
of the ways that we can assure that NIH will have the tools it 
needs to do the proper research. So I think that is why this is 
a very important issue.
    I wanted to ask one question of Senator Frist, because it 
is my understanding that the longer an embryo from in vitro 
fertilization is frozen the less likely it is to be as viable. 
Maybe there is a 5-year window or so. My question is, if it is 
not viable as a potential life, is it still viable to be a stem 
cell donor?
    Senator Frist. Thank you, Senator Hutchison. We talked a 
little bit about that, and the scientists can comment more on 
that later. The standard consent forms today, because the 
science is just evolving in terms of IVF--we know the great 
miracles that can take place because of it. We know that 
surplus embryos are created and the option is there to freeze 
these embryos in case the first therapeutic trial does not work 
and you can use them later.
    What we need to start thinking about socially as a society 
and as policymakers is what the ultimate destiny of those 
embryos are. We have inadequate oversight today and that is why 
I keep arguing, let us be very careful, let us make sure we 
look at the large ethical picture. We do not know what freezing 
actually does long-term and that is why in the consent form 
usually, the consent forms for an IVF, in vitro fertility 
clinic, will basically say something to that effect.
    Therefore, we do not really know because we have not done 
the research in terms of whether or not the long-term viability 
will in some way slow down what is called the pluripotential 
nature of these cells or their ability to copy themselves over 
time. We just do not know at this juncture.
    I should also say, we do not have to do all this research 
in human blastocyst cells. Before I figured out an operation, 
before I did my first heart-lung operations, I did not do it in 
human beings. I did heart-lung surgery in animal models. Let us 
just not forget, we are talking about research that is hugely 
promising, that every one of us have heard from constituents 
that the answer is going to be and the hope and the tremendous 
cure is going to be of these cells. That is overselling it 
today, maybe for every disease. It is just unrealistic.
    The hope is there, but it is unpredictable. It is like any 
new science, it is evolving, and therefore we have to be very 
careful. I would encourage us not to forget that there are 
other models besides human models to be doing this research.
    Senator Hutchison. Thank you, Mr. Chairman.
    Senator Harkin. Thank you very much, Senator.
    Again, I thank all of you for being here, for your time, 
and for your thoughtfulness and your suggestions to us on how 
we should proceed.
STATEMENT OF LANA SKIRBOLL, Ph.D., DIRECTOR, OFFICE OF 
            SCIENCE POLICY, NATIONAL INSTITUTES OF 
            HEALTH, DEPARTMENT OF HEALTH AND HUMAN 
            SERVICES
    Senator Harkin. Let me now call up Dr. Lana Skirboll, 
Associate Director for Science Policy and the Director of the 
Office of Science Policy in the NIH. Dr. Skirboll is 
responsible for the development of NIH's guidelines for 
research using human pluripotent stem cells and led the team 
that wrote the report that we have in front of us here today.
    Dr. Skirboll received her Ph.D. from Georgetown University 
Medical School and conducted her post-doctoral work at the Yale 
University School of Medicine. Dr. Skirboll, as you probably 
know, we are running way behind time and I am going to ask if 
you could hopefully summarize in 5 minutes, I would sure 
appreciate it. Then we will call on the next panel.
    Dr. Skirboll. Yes, I would be happy to do that. Good 
morning, Mr. Chairman, Senator Specter, and members of the 
subcommittee. I am Dr. Lana Skirboll, Director of Science 
Policy at the National Institutes of Health. Thank you for 
giving me the opportunity today to testify on our report 
entitled ``Stem Cell Scientific Progress and Future Research 
Directions.''
    The report is based on a review of more than 1,200 
scientific articles and interviews with over 50 leading private 
and public sector scientists conducting stem cell research. The 
report addresses both animal and human stem cell biology. It 
reviews research on adult stem cells as well as stem cells 
derived from embryos and fetal tissue.
    A stem cell, as you heard this morning, is unique. Unlike 
most cells, it has the capacity to both renew itself and to 
give rise to specialized cell types, like heart muscle and 
blood. Research on stem cells is not new. Scientists with the 
support of NIH have been studying stem cells for many years. In 
fact, animal research revealed more than two decades ago that 
there is a class of stem cell called the embryonic stem cell 
which has exceptional capabilities. It can make copies of 
itself indefinitely in culture and it can develop into almost 
all of the bodies many cell types.
    But until recently a human cell with these qualities has 
been elusive. In 1998, for the first time researchers were able 
to isolate this type of human stem cell from the very early 
human embryo.
    About the same time that human embryonic stem cells were 
first isolated, research was also revealing exciting 
information about previously unsuspected qualities of the adult 
stem cell. Adult stem cells are also unspecialized. They are 
present in many tissues and organs in the body, but they are 
rare in those tissues. The job of the adult stem cell is to 
develop into specialized cells of the tissue in which it 
resides, usually for the purpose of replacing damaged or lost 
cells.
    For example, the hematopoietic stem cell, which I think 
most of you have heard of, does the important work of ensuring 
a continual supply of new blood cells. Recently, scientists 
have found adult stem cells in more tissues than previously 
thought, like brain. They have also reported that adult stem 
cells from one tissue can develop into cell types that are 
characteristic of other tissue. This new characterization is 
called plasticity.
    Taken together, these new findings have generated much 
excitement. You have had many hearings about that, about the 
potential of both embryonic and adult stem cells in the 
development of cell-based therapies. But in order to develop 
such cell therapies, what do we really need to do? What do 
scientists need to accomplish?
    First, we need cultured cell lines that are pure, cell 
lines that in fact are well characterized and identical for 
safety reasons. We need cell lines that are diverse, that have 
the capacity to develop into as many kinds of cells that are 
possible to replace tissues that are destroyed or damaged from 
disease. And we need cells that proliferate, can make 
sufficient quantities in culture, so that many patients will 
have access to them.
    With these goals in mind, what does the report show about 
whether human adult or human embryonic stem cells are 
equivalent in their potential for generating replacement cells 
in tissues? First, with regard to purity, using embryonic stem 
cells researchers can generate pure cell lines in culture, 
while, with very few exceptions, adult stem cells in culture 
are a mixed cell population.
    Second, with regard to the ability to develop into many 
kinds of cells for treatment, embryonic stem cells in culture 
are pluripotent. Although certain kinds of adult stem cells 
have recently been shown to be plastic, no adult stem cell has 
been shown to be pluripotent in culture.
    Finally, what about that important ability of these cells 
to make many copies of themselves? Human embryonic stem cells 
have been shown to remain unspecialized and proliferate 
indefinitely in culture, potentially yielding quantities needed 
for transplantation, while the capacity of adult stem cells to 
proliferate in culture and remain unspecialized is limited.
    The report also addresses what we know and do not know, and 
there is much, about the ability of these cells to 
differentiate into specialized cells and ultimately to 
function. Three things we can say for sure: Both stem cell 
types may be useful in developing cell-based therapies; two, 
these kinds of stem cells are different; and three, right now 
there are more unanswered questions than there are answers.
    Scientists all agree that stem cell research holds enormous 
promise to lengthen and improve the quality of life for many 
patients suffering from perhaps a broad spectrum of diseases: 
spinal cord injuries, Parkinson's disease, heart disease, 
kidney disease, liver failure, multiple sclerosis, Alzheimer's 
disease, and diabetes, to name a few.
    In sum, because we do not know from which stem cell type 
the best therapies will come for these diseases, scientists 
believe the door should be left open to conduct research on 
both embryonic and adult stem cells.
    I would be pleased to answer any questions you may have. 
Thank you.
    [The information follows:]

    [Clerk's note.--The report ``Stem Cells: Scientific 
Progress and Future Research Directions'' can be found on line 
at http://www.nih.gov/news/stemcell/scireport.htm]

    Senator Harkin. Dr. Skirboll, thank you. In the interest of 
time, I think if I could ask you to stay at the table and let 
me bring up the next panel.
    Would you please put up the name plates here. Mr. Richard 
Doerflinger, associate director for policy development at the 
Secretariat for Pro-Life Activities, the National Catholic 
Conference--the National Conference of Catholic Bishops, I am 
sorry. Anton-Lewis Usala, M.D., founder, chairman, and chief 
scientific officer for Encelle, Incorporated; Diane Krause, 
M.D., Ph.D., associate professor of pathology and medicine, the 
Yale University School of Medicine; Mary Hendrix, Ph.D., 
professor and head of the department of anatomy and cell 
biology at University of Iowa; William Gibbons, M.D., chairman 
of the Jones Institute for Reproductive Medicine; Susan 
Lanzendorf, Ph.D., associate professor at the Jones Institute 
for Reproductive Medicine at the Eastern Virginia Medical 
School; Michael West, Ph.D., president and CEO of Advanced Cell 
Technology.
    Let me see where we are here. Where is Dr. West?
    He was here. He maybe just stepped out and he will be back.
    Let me start. Then I will start as I called them off. In 
fact, I may not go in the same order. I will just go from left 
to right. I will start with Mr. Doerflinger.
    Again, I would like to ask--I hate to do this because this 
is something I could spend all day on. But if you could limit 
it to 3 or 4 minutes, I would sure appreciate it. Just give us 
the basic impetus of what you want us to know, and then we will 
open it up for questions.
    Mr. Doerflinger.
STATEMENT OF RICHARD M. DOERFLINGER, ASSOCIATE DIRECTOR 
            FOR POLICY DEVELOPMENT, SECRETARIAT FOR 
            PRO-LIFE ACTIVITIES, UNITED STATES 
            CONFERENCE OF CATHOLIC BISHOPS
    Mr. Doerflinger. Thank you, Mr. Chairman.
    Our title, by the way, now is the United States Conference 
of Catholic Bishops. We changed the name the beginning of July.
    Senator Harkin. It is not ``National Conference''?
    Mr. Doerflinger. Right. We decided to recognize other 
nations as sovereign nations as well.
    In our view, forcing U.S. taxpayers to subsidize research 
the relies on deliberate destruction of human embryos for their 
stem cells is illegal, immoral, and unnecessary. Obviously, we 
agree with Senator Specter's comment that the current NIH 
guidelines, which have the NIH arrange for the destruction of 
embryos and then pretend to know not where--not to know where 
they came from, is hypocrisy and an evasion of the law, not a 
way of implementing the current appropriations rider, and 
therefore we certainly think that, out of respect for the law, 
the administration should find the NIH guidelines to be 
contrary to the statute, whatever else Congress may try to pass 
as legislation.
    We think the represent is immoral. It violates the central 
tenet of all civilized codes of human experimentation beginning 
with the Nuremberg Code, approving doing deadly harm to a 
member of the human species solely for benefit to others. Aside 
from the fuller respect for human life across the board that 
the Catholic Church and others have promoted, even President 
Clinton's National Bioethics Advisory Commission, which has 
been given high accolades in the panel already, even to the 
point of fabricating religious leaders on the commission, which 
there were none of, the National Bioethics Advisory Commission 
conceded that the early human embryo is a form of developing 
human life that deserves our respect.
    Where we disagree is whether the idea of scooping out the 
cells of a living embryo and throwing away the shell is a way 
of showing respect.
    Finally, this proposal is unnecessary because adult stem 
cells and other alternatives are already achieving some of the 
goals for which embryonic stem cells have been proposed and new 
clinical uses are constantly being discovered. In that light, I 
think the National Bioethics Advisory Commission made a very 
interesting statement, that because of the claims of human life 
and the respect it deserves, that using these embryos for 
research is completely unjustified if there are morally less 
problematic alternatives available.
    We know even from the NIH report, which was written by 
people with a particular ideological drive on this issue--it 
is, after all, the NIH that first recommended, its institutes 
unanimously recommended in 1994, a full range of Federal 
funding for destructive embryo research, including research 
that involves the special creation of embryos solely for 
research, which has been condemned by many others.
    Even the NIH has conceded that adult stem cells are 
extremely useful and it is now unpredictable which will be most 
useful for various purposes. If that is the case, in this 
period of uncertainty it would be morally irresponsible to 
cross this moral Rubicon and begin acting as though there is no 
moral difference. Obviously, if embryo research is the last 
resort, if even President Clinton's advisors said that other 
avenues need to be exhausted first, then pursuing both at the 
same time and forcing millions of morally opposed taxpayers to 
fund it would be to force all the taxpayers to act as though 
they agree the that embryo is absolutely nothing in terms of 
moral status.
    In fact, it would force us to pretend to agree with the 
chairman of the Juvenile Diabetes Foundation, who testified 
last year that the human embryo is more like a goldfish than a 
human being.
    A subtly different argument from the argument that the 
embryo is not a life has been offered by those who simply say 
these would be discarded anyway. The problem with that argument 
is that if it is an argument that is supposed to presuppose 
that other embryos really are human lives that deserve respect, 
it articulates a moral principle that is horrific for every 
patient subjected to human research.
    Currently we do not kill terminally ill patients for their 
organs, though they will die soon anyway, and Federal law 
prohibits federally funded researchers from doing any harm to 
an unborn child slated for abortion, though that child will 
soon be discarded anyway.
    In any case, the claim that the embryos to be destroyed 
under the NIH guidelines will necessarily be destroyed anyway 
is actually a canard. That is not in the guidelines. It covers 
embryos found to be in excess of clinical need and the option 
of destroying them for their stem cells clearly stated should 
be offered as an option along side all the other options, 
including the option of adopting from another couple or saving 
for one's own later use.
    Finally, I think that the argument that somehow by funding 
this we prevent more of it is a bizarre argument to me, because 
already the NIH guidelines in its limited proposal for this has 
quickly led to a slippery slope in which we are discussing 
legislation to fund not only the destruction of these embryos 
for their cells, but the use of specially created embryos for 
research as well, as in the Specter-Harkin bill.

                           prepared statement

    Obviously, once you fund a little of this you get a lot 
more of it. The only thing we can be sure of if we fund it is 
that there is going to be a lot of destruction going on to 
benefits that are at best speculative and we think can be 
better met by other research that destroys no one.
    We think that the President and the Congress should unite 
to support promising medical research that everybody can live 
with.
    Thank you.
    [The statement follows:]
              Prepared Statement of Richard M. Doerflinger
    I am Richard M. Doerflinger, Associate Director for Policy 
Development at the Secretariat for Pro-Life Activities, United States 
Conference of Catholic Bishops. I am grateful for this opportunity to 
present the Catholic bishops' grave concerns on this critically 
important issue.
    In our view, forcing U.S. taxpayers to subsidize research that 
relies on deliberate destruction of human embryos for their stem cells 
is illegal, immoral and unnecessary.
    It is illegal because it violates an appropriations rider (the 
Dickey amendment) passed every year since 1995 by Congress. That 
provision forbids funding ``research in which'' human embryos (whether 
initially created for research purposes or not) are harmed or destroyed 
outside the womb.\1\ National Institutes of Health guidelines approved 
by the Clinton Administration nonetheless give researchers detailed 
instructions on how to obtain human embryos for destructive cell 
harvesting, if they wish to qualify for federal grants in ``human 
pluripotent stem cell research.'' \2\ Clearly, obtaining and destroying 
embryos is an integral part of this project, even if the specific act 
of destroying embryos does not directly receive federal funds. By 
implementing these guidelines, the federal government would encourage 
researchers to conduct destructive embryo experiments that are 
punishable as felonies in some states.\3\
---------------------------------------------------------------------------
    \1\ Section 510 of the Labor/HHS appropriations bill for fiscal 
year 2001, H.R. 5656 (enacted through Section 1(a)(1) of H.R. 4577, the 
fiscal year 2001 Consolidated Appropriations Act, Public Law 106-554).
    \2\ National Institutes of Health Guidelines for Research Using 
Human Pluripotent Stem Cells, 65 Fed. Reg. 51976-81 (August 25, 2000).
    \3\ See Fact Sheet, ``The NIH Proposal for Stem Cell Research Is a 
Crime,'' www.usccb.org/prolife/issues/bioethic/states701.htm.
---------------------------------------------------------------------------
    This proposal is immoral because it violates a central tenet of all 
civilized codes on human experimentation beginning with the Nuremberg 
Code: It approves doing deadly harm to a member of the human species 
solely for the sake of potential benefit to others. The embryos to be 
destroyed by researchers in this campaign are at the same stage of 
development as embryos in the womb who have been protected as human 
subjects in federally funded research since 1975.\4\ President 
Clinton's National Bioethics Advisory Commission (NBAC) and its 1994 
predecessor, the NIH Human Embryo Research Panel, conceded that the 
early human embryo is a form of developing human life that deserves our 
respect.\5\ Treating human life as mere research material is no way to 
show respect.
---------------------------------------------------------------------------
    \4\ Federal regulations on Protection of Human Subjects include 
protections for the human fetus, ``from the time of implantation.'' 45 
CFR Sec. 46.203 (c). Implantation generally begins about six days after 
fertilization, at the blastocyst stage of human development.
    \5\ ``We believe that most Americans agree that human embryos 
should be respected as a form of human life . . .'' National Bioethics 
Advisory Commission (NBAC), Ethical Issues in Human Stem Cell Research 
(September 1999) at 2.
---------------------------------------------------------------------------
    Finally, this proposal is unnecessary because adult stem cells and 
other alternatives are already achieving some of the goals for which 
embryonic stem cells have been proposed, and new clinical uses are 
constantly being discovered.\6\
---------------------------------------------------------------------------
    \6\ See Fact Sheet, ``Current Clinical Use of Adult Stem Cells to 
Help Human Patients,'' www.usccb.org/prolife/issues/bioethic/
adult701.htm.
---------------------------------------------------------------------------
    In our view, human life deserves full respect and protection at 
every stage and in every condition. The intrinsic wrong of destroying 
innocent human life cannot be ``outweighed'' by any material 
advantage--in other words, the end does not justify an immoral means. 
Acceptance of a purely utilitarian argument for mistreating human life 
would endanger anyone and everyone who may be very young, very old, 
very disabled, or otherwise very marginalized in our society. However, 
even the Clinton Administration's bioethics advisors, who denied human 
embryos the moral status of ``person,'' concluded that they could only 
be destroyed for research as a last resort, if no alternative course 
existed.\7\
---------------------------------------------------------------------------
    \7\ ``In our judgment, the derivation of stem cells from embryos 
remaining following infertility treatments is justifiable only if no 
less morally problematic alternatives are available for advancing the 
research.'' NBAC, note 5 supra at 53.
---------------------------------------------------------------------------
    It cannot be denied that these alternatives are available. To be 
sure, further study will be needed to determine their full potential. 
But to fund destructive embryo research now, alongside these morally 
acceptable alternatives, would be to deny any moral status at all to 
human embryonic life. For that is what we would do if there were no 
moral issue at stake. Funding embryonic stem cell research here and now 
will force all taxpayers to act as though they agree with the 
international chairman of the Juvenile Diabetes Foundation that human 
embryos have no more value or dignity than a goldfish.\8\
---------------------------------------------------------------------------
    \8\ ``The embryos that are being discussed, according to science, 
bear as much resemblance to a human being as a goldfish.'' Mary Tyler 
Moore, Testimony on behalf of the Juvenile Diabetes Foundation before 
the Senate Appropriations Subcommittee on Labor, Health and Human 
Services and Education, September 14, 2000.
---------------------------------------------------------------------------
    This view of the human embryo as a goldfish has apparently garnered 
support from some members of Congress who have generally opposed 
abortion. Their claim is that human life does not begin until placed in 
a mother's womb. Biologically, however, this is an absurd claim. An 
embryo's development is directed completely from within--the womb 
simply provides a nurturing environment. Scientists tell us it would be 
technically possible to nurture a human embryo in a man's body by 
abdominal pregnancy, or in a mammal of another species, or even 
(someday) in an artificial womb.\9\ Upon being born could such a person 
morally be killed for his or her stem cells, because he or she never 
lived inside a woman's womb?
---------------------------------------------------------------------------
    \9\ Testimony of Lee M. Silver, Ph.D., before the House Government 
Operations Subcommittee on Human Resources and Intergovernmental 
Relations, July 14, 1988; R. Rowland, Living Laboratories: Women and 
Reproductive Technologies (Indiana University Press 1992) at 288-9.
---------------------------------------------------------------------------
    A subtly different argument has also emerged to try to justify 
using embryos from fertility clinics for destructive experiments. While 
human embryos ordinarily deserve respect, goes this argument, these 
particular embryos do not, because they ``would be discarded anyway'' 
by their parents. But this is, to say the least, fallacious reasoning. 
If parents were neglecting or abusing their child at a later stage, 
this would provide no justification whatever for the government to move 
in and help destroy the child for research material. We do not kill 
terminally ill patients for their organs, although they will die soon 
anyway, or even harvest vital organs from death row prisoners, although 
they will be put to death soon anyway. Federal law prohibits federally 
funded researchers from doing any harm to an unborn child slated for 
abortion, though that child will soon be discarded anyway (see 42 USC 
Sec. 289g). If people's value depends entirely on the extent to which 
other people ``want'' them, they have no inherent value at all. So on 
reflection, this argument ultimately reduces to the argument of 
``embryo as goldfish.''
    The argument also rests on a false premise. The embryos slated for 
destructive research under the NIH guidelines are those deemed to be 
``in excess of clinical need'' by fertility clinics. This simply means 
that they are not needed or wanted by their parents for reproduction at 
present. Parents in this situation are routinely offered several 
options, including: saving the embryos for possible later use (by far 
the most frequently chosen), discarding them, or donating them to 
another couple so they can have a child. The NIH guidelines require 
that these parents be asked to consider donating their embryos for 
destructive cell harvesting at the same time that they are offered 
these other options.\10\ Some couples who would otherwise have allowed 
their embryonic children to live--in their own family or another--will 
instead have them killed for government research. That is why the 
adoptive couples of some of these former ``frozen embryos'' have filed 
suit against the guidelines.\11\
---------------------------------------------------------------------------
    \10\ Parents must be asked about having their embryos destroyed for 
federally funded stem cell research ``only at the time of deciding the 
disposition if embryos in excess of the clinical need.'' National 
Institutes of Health, note 2 supra at 51980 (emphasis added). 
Proponents seem to assume that the option of destructive research is to 
be offered after parents have decided to have the embryos discarded. 
Read strictly, the guidelines actually forbid clinics to do this.
    \11\ Nightlight Christian Adoptions v. Thompson (D.D.C. filed March 
8, 2001).
---------------------------------------------------------------------------
    We have presented our position on this issue at length in other 
testimony.\12\ In the remainder of this testimony we would like to 
comment on recent developments, including new evidence that proponents 
of destructive embryo research have misrepresented or distorted the 
facts to serve their political goal.
---------------------------------------------------------------------------
    \12\ For past testimony, including our public comments on the NIH 
guidelines and our testimony before this subcommittee in December 1998 
and January 1999, see: www.usccb.org/prolife/issues/bioethic/
biotest.htm.
---------------------------------------------------------------------------
    new developments in alternatives to embryonic stem cell research
    Since we testified before this subcommittee in 1999, startling 
advances have been made in adult stem cell research and other non-
embryonic avenues for repairing or replacing damaged organs and 
tissues. The field of ``tissue engineering'' using adult cells has 
exploded as researchers move toward rebuilding ears, tracheas, and even 
hearts.\13\ Adult stem cells have successfully treated hundreds of 
thousands of patients with cancer and leukemia; they have repaired 
damaged corneas, restoring sight to people who were legally blind; they 
have healed broken bones and torn cartilage in clinical trials; they 
are being used to help regenerate heart tissue damaged by a cardiac 
arrest.\14\ Adult bone marrow stem cells were responsible for the first 
completely successful trial of human gene therapy, helping children 
with severe combined immunodeficiency disease to recover an immune 
system and safely leave their sterile environment for the first 
time.\15\ Adult cells from a young paraplegic woman's own immune 
system, injected into the site of her spinal cord injury, have 
apparently cured her incontinence and enabled her to move her toes and 
legs for the first time--``generating hope for those with spinal-cord 
injuries around the world,'' as one news report observes.\16\
---------------------------------------------------------------------------
    \13\ J. D'Agnese, ``Brothers with heart,'' Discover, July 2001 at 
36-43, 102.
    \14\ For documentation see www.stemcellresearch.org, the Web site 
of Do No Harm: The Coalition for Americans for Research Ethics, 
especially ``Current Clinical Applications of Adult Stem Cells'' 
(www.stemcellresearch.org/currentaps.pdf) and ``Letter to Ruth 
Kirschstein, Ph.D., Acting Director of the National Institutes of 
Health'' (www.stemcellresearch.org/kirschstein.pdf).
    \15\  M. Cavazzana-Calvo et al., ``Gene Therapy of Human Severe 
Combined Immunodeficiency (SCID)-X1 Disease,'' 288 Science 669-72 (28 
April 2000).
    \16\ K. Foss, ``Paraplegic regains movement after cell procedure,'' 
The Globe and Mail (Toronto), June 15, 2001 at A1.
---------------------------------------------------------------------------
    Finally, adult pancreatic islet cells from cadavers have been used 
to reverse juvenile diabetes in fifteen patients, and further human 
trials are being planned at several centers in the United States. At 
the annual meeting of the American Diabetes Association on June 24, 
researchers announced that all patients benefited from the transplants, 
and nine have remained ``insulin free'' for a median period of eight 
months--with some patients requiring no injections for up to two 
years.\17\
---------------------------------------------------------------------------
    \17\ E. Ryan et al., ``Glycemic Outcome Post Islet 
Transplantation,'' Abstract #33-LB, Annual Meeting of the American 
Diabetes Association, June 24, 2001. See: http://38.204.37.95/am01/
AnnualMeeting/Abstracts/NumberResults.asp?idAbs=33-LB.
---------------------------------------------------------------------------
    Hailed by experts as a ``remarkable advance,'' this breakthrough 
has also received enthusiastic attention from Lee Ducat, founder of the 
Juvenile Diabetes Foundation (JDF). ``There's still a lot to be 
learned, but this is the most optimistic I've been in 30 years,'' she 
says. ``To take patients who are terribly ill and going in and out of 
shock and give them a normal life . . . this is an unbelievable result. 
They say they never knew what feeling normal is all about.'' \18\
---------------------------------------------------------------------------
    \18\ M. McCullough, ``Islet transplants offer hope that diabetes 
can be cured,'' Philadelphia Inquirer, June 22, 2001 at A1.
---------------------------------------------------------------------------
    Yet this good news has gone largely unnoticed by the current 
leadership of the JDF. Instead the organization is focused on diverting 
funds toward a misleading ad campaign to persuade Americans to support 
killing human embryos for their stem cells.
    Neglect--even misstatement--of recent scientific data was also 
evident in last year's testimony before this subcommittee by the 
Christopher Reeve Paralysis Foundation. Mr. Reeve, on behalf of the 
Foundation, testified that adult stem cells are no substitute for 
embryonic cells because they cannot be ``pluripotent'' but are confined 
to a narrow range of specialization. Yet a few weeks after that 
hearing, researchers funded by the NIH and the Christopher Reeve 
Paralysis Foundation published a study indicating that adult bone 
marrow stem cells ``may constitute an abundant and accessible cellular 
reservoir for the treatment of a variety of neurologic diseases.'' The 
first sentence of the published study states: ``Pluripotent stem cells 
have been detected in multiple tissues in the adult, participating in 
normal replacement and repair, while undergoing self-renewal.'' \19\ 
The authors cite eleven other studies in support of this observation. 
Their article, prepared under the aegis of Mr. Reeve's foundation, was 
received for publication in March 2000, before Mr. Reeve testified in 
April that adult stem cells cannot be pluripotent.
---------------------------------------------------------------------------
    \19\ D. Woodbury et al., ``Adult Rat and Human Bone Marrow Stromal 
Cells Differentiate Into Neurons,'' 61 J. of Neuroscience Research 364-
70 (2000) at 364 (emphasis added).
---------------------------------------------------------------------------
    An author of that study, Dr. Darwin Prockop, told this subcommittee 
last year that the implications of his work should not be overstated 
and that he himself supports funding both embryonic and adult stem cell 
research. However, medical and patient groups have now tilted the 
pendulum so far toward outright denial of the facts about the promise 
of adult stem cell research that Dr. Prockop recently felt obliged to 
correct the record. Responding to an article that questioned the 
benefits of adult stem cells, he notes:

    ``More than 20 years ago, Friedenstein and then others grew adult 
stem cells from bone marrow called mesenchymal stem cells or marrrow 
stronal cells (MSCs). MSCs differentiate into bone, cartilage, fat, 
muscle, and early progenitors of neural cells. Human MSCs can be 
expanded up to a billionfold in culture in about 8 weeks. Preliminary 
but promising results have appeared in the use of MSCs in animal models 
for parkinsonism, spinal cord defects, bone diseases, and heart 
defects. Also, several clinical trials are in progress. In addition, 
there are promising results with other adult stem cells that perhaps we 
may yet learn how to grow effectively.'' \20\
---------------------------------------------------------------------------
    \20\ D. Prockop, ``Stem Cell Research Has Only Just Begun'' 
(Letter), 293 Science 211-2 (13 July 2001) (citations omitted).

    Perhaps the most troubling and unwarranted fixation on embryonic 
stem cells to the exclusion of all other approaches has been exhibited 
by the Parkinson's Action Network (PAN). This group has declared that 
it actively opposes a new bill introduced by Congressman Chris Smith, 
which would authorize $30 million a year in new funding for stem cell 
research and establish a national stem cell bank for research and 
possible treatments (Responsible Stem Cell Research Act of 2001, H.R. 
2096). While this bill places no restrictions on embryonic stem cell 
research--indeed, does not mention such research one way or the other--
PAN believes that this much-needed additional funding for promising 
medical research must be rejected because it does not include stem 
cells obtained by destroying embryos. By this logic PAN would have to 
oppose all current NIH funding for Parkinson's research, which has 
never included funding for embryonic stem cell research.
          new disappointments in embryonic stem cell research
    In the past two years, initial enthusiasm over embryonic stem cells 
has been dampened in the scientific community by some sober 
realizations, even as patient groups organize public campaigns based on 
earlier assumptions.
    First, these cells are not as easy to maintain in the laboratory as 
once thought. Researchers call them ``tricky'' and ``more tedious to 
grow'' than their mouse counterparts, as well as ``really difficult'' 
to direct toward more specialized cells.\21\ The dream of ``immortal'' 
cell lines that will easily provide unlimited supplies of any kind of 
tissue remains a dream.
---------------------------------------------------------------------------
    \21\ G. Vogel, ``Stem Cells: New Excitement, Persistent 
Questions,'' 290 Science 1672-4 (1 December 2000) at 1674.
---------------------------------------------------------------------------
    Second, a new study of problems in cloning suggests that embryonic 
stem cells are ``surprisingly genetically unstable'' in mice and 
perhaps in humans as well. This ``may complicate efforts to turn the 
cells into cures,'' and interfere with efforts to produce all-purpose 
cell lines that could reliably become tissue of any desired type. ``You 
may have to establish hundreds of lines to get the few you'd want to 
have,'' Dr. John Gearhart of Johns Hopkins University now says. 
Establishing hundreds of these cell lines could require destroying many 
thousands of human embryos, and replenishing them with thousands more 
when the original cell lines become too unstable for further use. 
Perhaps most troubling is the news that these researchers deleted from 
their final paper a reference to this problem, believing that any 
public acknowledgment of such setbacks has become too ``politically 
sensitive.'' \22\ We can only wonder how much of this kind of 
information is being withheld without detection. We have reached a 
stage in this discussion where, on the side supporting destructive 
embryo research, science is becoming subservient to politics.
---------------------------------------------------------------------------
    \22\ R. Weiss, ``Clone Study Casts Doubt on Stem Cells,'' The 
Washington Post, July 6, 2001, A1 and A9.
---------------------------------------------------------------------------
    Third, the chief advantage universally cited for embryonic stem 
cells--their ability to grow and differentiate into all the more than 
200 kinds of cells and tissues in the human body--is proving to be a 
major disadvantage for transplantation into living bodies. For it is 
very difficult to make these cells stop turning into all kinds of cells 
and tissues. In recent studies, embryonic stem cells (or partially 
differentiated cells arising from them) ``stayed in a disorganized 
cluster, and brain cells near them began to die.'' \23\ Says 
bioethicist Glenn McGee, who supports of embryonic stem cell research:
---------------------------------------------------------------------------
    \23\ G. Vogel, note 21 supra at 1674.

    ``The emerging truth in the lab is that pluripotent stem cells are 
hard to rein in. The potential that they would explode into a cancerous 
mass after a stem cell transplant might turn out to be the Pandora's 
box of stem cell research.'' \24\
---------------------------------------------------------------------------
    \24\ E. Jonietz, ``Innovation: Sourcing Stem Cells,'' Technology 
Review, January/February 2001, http://209.58.177.220/articles/jan01/
innovation__jonietz__printable.html.

    By contrast, though non-embryonic stem cells seem harder to direct 
to form tissues of different categories, they seem much more docile to 
their environment. Upon reaching a particular kind of tissue, they 
receive signals as to the kind of tissue needed and produce only that 
tissue. They may be ``easier to manage,'' and therefore far safer for 
clinical use in humans, than embryonic cells.\25\ After all, adult stem 
cells are already found throughout the human body, already provide a 
built-in repair kit for repairing and regenerating human tissue, and 
have already safely treated hundreds of thousands of patients. 
Understanding and stimulating this natural ability may be a far more 
promising avenue than efforts to harness and control cells that simply 
do not belong in an adult body in the first place--cells with a 
tendency to form tumors, in an apparent effort to turn back into a 
complete embryo.
---------------------------------------------------------------------------
    \25\ G. Vogel, ``Can Old Cells Learn New Tricks?'', 287 Science 
1418-9 (February 25, 2000) at 1419; L. Johannes, ``Adult Stem Cells 
Have Advantage Battling Disease,'' Wall Street Journal, April 13, 1999 
at B1.
---------------------------------------------------------------------------
    The kind of exaggerated claims now made for embryonic stem cells 
have been seen in this Congress before. A decade ago it was fetal 
tissue from abortions that was hailed as the magic bullet that might 
cure diabetes, Parkinson's disease and many other conditions in a few 
years if only federal funds were provided. By the time such funds were 
approved in 1993, however, it was already becoming clear that fetal 
tissue from abortions would be largely useless in treating diabetes. 
Millions of taxpayers' dollars were diverted toward fetal tissue 
transplant trials for Parkinson's disease--and the final results were 
not only disappointing but ``devastating,'' according to the New York 
Times. The implants ``failed to show an overall benefit,'' and in 15 
percent of the patients actually produced ``nightmarish'' symptoms as 
the immature cells produced dopamine in uncontrollable amounts.\26\ The 
chief result of the campaign for fetal tissue research by some 
Parkinson's disease groups is that a significant number of Parkinson's 
patients may now be incurably worse off than before.
---------------------------------------------------------------------------
    \26\ G. Kolata, ``Parkinson's Research Is Set Back By Failure of 
Fetal Cell Implants,'' The New York Times, March 8, 2001 at A1, A12.
---------------------------------------------------------------------------
    Will embryonic stem cells prove to be equally disappointing or even 
disastrous? No one knows. However, a tragic occurrence following one 
particular fetal tissue transplant for Parkinson's disease should give 
us pause. Some of the tissue placed in this man's brain may have been 
from an earlier gestational age than is customary in American clinical 
trials--that is, it may have been more embryonic than fetal in nature. 
Within two years after the transplant this man died mysteriously--and 
an autopsy revealed that masses of ``nonneural tissue'' such as skin 
and hair had filled the ventricles of his brain and cut off his 
breathing. Researchers theorized that this tissue may have remained 
``pluripotent'' and differentiated uncontrollably to cause the 
patient's death.\27\
---------------------------------------------------------------------------
    \27\ R. Folkerth and R. Durso, ``Survival and proliferation of 
nonneural tissues, with obstruction of cerebral ventricles, in a 
parkinsonian patient treated with fetal allografts,'' 46 Neurology 
1219-25 (May 1996).
---------------------------------------------------------------------------
    At the very least, past experience argues in favor of greater 
humility than some researchers and organizations are now showing in 
their campaign for destructive embryo research. To quote two 
bioethicists who do not oppose such research on moral grounds, ``much 
of the hype that surrounded the debate about the clinical value of 
fetal tissue implants was exactly that--hype. This ought to be kept in 
mind by those now engaged in the debate over stem cell research.''\28\
---------------------------------------------------------------------------
    \28\ A. Caplan and G. McGee, ``Fetal Cell Implants: What We 
Learned,'' Hastings Center Report, May-June 2001 at 6.
---------------------------------------------------------------------------
                      the slippery slope in action
    Finally, recent developments highlight a point made by opponents of 
embryonic stem cell research for years: Once our consciences are numbed 
to the moral wrong of using so-called ``spare'' human embryos for 
research, our society will move on to even more egregious abuses. The 
Jones Institute for Reproductive Medicine in Virginia has announced 
that it is using donated eggs and sperm to create human embryos solely 
to destroy them for stem cell research.\29\ Moreover, Advanced Cell 
Technology (ACT) in Massachusetts has announced it is trying to make 
human embryos by somatic cell nuclear transfer (cloning) for the same 
purpose.\30\
---------------------------------------------------------------------------
    \29\ S. Stolberg, ``Scientists Create Scores of Embryos to Harvest 
Cells,'' The New York Times, July 11, 2001 at A1.
    \30\ A. Regalado, ``Experiments in Controversy,'' Wall Street 
Journal, July 13, 2001 at B1.
---------------------------------------------------------------------------
    In the past, this further step--that of creating life in the 
laboratory for the sole purpose of destroying it--was supported by the 
NIH, but widely condemned even by abortion supporters in Congress and 
editorial boards across the country. President Clinton refused funding 
for this approach, and the Washington Post editorialized:

    ``The creation of human embryos specifically for research that will 
destroy them is unconscionable . . . [I]t is not necessary to be 
against abortion rights, or to believe human life literally begins at 
conception, to be deeply alarmed by the notion of scientists' purposely 
causing conceptions in a context entirely divorced from even the 
potential of reproduction.'' \31\
---------------------------------------------------------------------------
    \31\ Editorial, ``Embryos: Drawing the Line,'' The Washington Post, 
October 2, 1994 at C6.

    Despite this strong consensus against creating embryos to destroy 
them, those actually involved in embryo research no longer see any 
serious ethical problem in it. Now the American Society for 
Reproductive Medicine (ASRM), which published the Jones study in its 
journal, says the study is ``not inappropriate'' and is in accord with 
ASRM's ethical guidelines. Some even argue that such research is 
morally superior to the use of ``spare'' embryos, because the egg and 
sperm donors understand from the beginning what the embryos will be 
used for.
    Similarly, when ACT testified before this subcommittee in December 
1998, it was virtually alone in insisting that success in embryonic 
stem cell research would require moving on to human cloning to make 
genetically matched tissues for each patient. However, the nation's 
leading for-profit group promoting embryonic stem cell research, the 
Geron Corporation, soon acquired the Roslin Institute in Scotland to 
combine its own expertise in embryonic stem cell research with Roslin's 
expertise in cloning.\32\ The president of Geron recently testified to 
a House subcommittee that allowing the special creation of human 
embryos by cloning will be ``essential'' to the future of embryonic 
stem cell research.\33\
---------------------------------------------------------------------------
    \32\ L. Krieger, ``Clone Coup: Geron Buys `Dolly' Biotech Pioneer 
for Technology That May Have Worldwide Medical Use,'' San Jose Mercury 
News, May 5, 1999 at 1C.
    \33\ ``Somatic cell nuclear transfer research is essential if we 
are to achieve our goals in regenerative medicine.'' Testimony of 
Thomas Okarma before the House Energy and Commerce Subcommittee on 
Health, June 20, 2001. During the question session at this hearing, Dr. 
Okarma made it clear that he was speaking of the use of this technology 
to create genetically tailored human embryos for research.
---------------------------------------------------------------------------
    These groups have engaged in embryo research long enough to deaden 
all sensitivity to the fact that they are dealing with human life. If 
the federal government funds even a limited amount of research that 
relies on destroying human embryos, this deadening of consciences will 
occur on a wider scale and with government approval.
    The Coalition for the Advancement of Medical Research, which favors 
federal funding of embryonic stem cell research, has argued that these 
developments actually show that the Bush Administration should proceed 
with the funding. To stop such abuses, goes the argument, the federal 
government must fund embryo research so it will have the authority to 
set limits.\34\
---------------------------------------------------------------------------
    \34\ Press Release, ``Development of Stem Cells from Fertilized 
Eggs Created for Research Demonstrates Need for Oversight,'' Coalition 
for the Advancement of Medical Research, www.stemcellfunding.org/
fastaction/news.asp?id=52.
---------------------------------------------------------------------------
    But the first groups to make this claim were groups that favor 
destructive embryo research, including groups closely associated with 
the Jones Institute's abuses. ASRM, which has given the ethical ``green 
light'' to the Jones study and published the results in its own 
journal, is an active member of the Coalition for the Advancement of 
Medical Research. So we are being told how to prevent special creation 
of embryos by the leading groups that favor and even perform it!
    The argument that one must fund this research to regulate it is 
also absurd on its merits. The Jones study was done entirely with 
private funds, because for five years Congress has clearly prohibited 
funding of all destructive embryo research. If the federal government 
begins to fund some destructive research, it will be able to set 
standards for the research it chooses to fund, but the privately funded 
Jones study will remain untouched. In fact, such a policy change will 
signal that the government is moving in the Jones Institute's direction 
on this issue. It will soon become apparent that the government must 
fund research involving special creation of embryos for research--that 
is, must fund the very abuse it claims to oppose--in order to set 
standards for such research. Even then, those choosing not to obey such 
standards will simply conduct that part of their research with private 
funds--and encourage the federal government to catch up with their 
advanced thinking, as it already will have done on the subject of 
destroying ``spare'' embryos. Indeed, supporters of embryo research in 
Congress have already introduced legislation that could fund research 
using specially created ``research embryos,'' to take this next step 
(Stem Cell Research Act of 2001, S. 723).
    We know that destructive embryo research can be regulated or even 
prohibited without funding it. As noted earlier, nine states now ban 
all such research, whether publicly or privately funded.\35\ The state 
of Virginia itself has banned the use of cloning to make human embryos 
for research, and is considering a response to the Jones Institute's 
project for making research embryos by in vitro fertilization.\36\ And 
the Food and Drug Administration, without funding any part of in vitro 
fertilization, recently wrote to in vitro fertilization clinics engaged 
in new reproductive techniques to remind them that such technologies, 
albeit privately funded, are subject to federal regulation.\37\
---------------------------------------------------------------------------
    \35\ See note 3 supra.
    \36\ Code of Virginia, Sec. 32.1-162.21 and Sec. 32.1-162.22 
(Enacted 2001); C. Timberg, ``Va. to Examine Embryo Research,'' 
Washington Post, July 14, 2001 at B1.
    \37\ R. Weiss, ``FDA to Regulate Certain Fertilization 
Procedures,'' Washington Post, July 11, 2001 at A2.
---------------------------------------------------------------------------
                               conclusion
    Like the argument that human embryos are not members of the human 
race, arguments that destroying them is necessary for medical progress 
or that funding such destruction is needed to prevent broader abuse 
cannot be sustained. With these arguments out of the way we can return 
to the real issue at stake: Should the federal government subsidize--
and force millions of morally opposed taxpayers to subsidize--research 
that requires the destruction of innocent human life? We hope that the 
President and Congress will answer that question in the negative, and 
will unite instead to support promising medical research that everybody 
can live with.

    Senator Harkin. Thank you, Mr. Doerflinger.
    Dr. Usala.
STATEMENT OF ANTON-LEWIS USALA, M.D., FOUNDER, 
            CHAIRMAN, AND CHIEF SCIENCE OFFICER, 
            ENCELLE, INC.
    Dr. Usala. Thank you, Senators.
    For the record, I would like to state that I resigned as 
chairman of my company in December and that the views I am 
about to express are my own as a medical scientist, not those 
of my company.
    I appear before the subcommittee to discuss embryonic stem 
cell research and alternative technologies that are currently 
under development in the company I founded. Our focus was to 
develop a method to regenerate and replace damaged patient 
tissues as a treatment for various human diseases.
    I developed a proprietary scaffolding based on the 
structure of embryonic tissue scaffolding, to which the adult 
cells can attach. This proprietary injectable scaffolding has 
developed apparent--that my company has developed, apparently 
enables regeneration of a specific kind of tissue called 
mesenchymal tissue. One of three embryonic germ layers, 
mesenchymal tissue gives rise to connective tissue, blood 
vessels, bone, cartilage, and parts of skin.
    In pre-clinical animal studies, we have demonstrated 
regeneration of blood vessels and skin in animals suffering 
from chronic diabetic and surgical extremity lesions. We are 
currently nearing completion of our FDA-approved first human 
clinical trial with what I believe are exciting results. We 
have not yet submitted the final report to the Food and Drug 
Administration and the data has not yet been peer reviewed and 
I would like to make that point for the record.
    However, photos of chronic diabetic foot ulcer wounds in 
some of the patients treated with a single injection of our 
scaffolding appear to show rapid, well vascularized tissue 
regeneration and closure of these ulcers within 2 to 8 weeks. 
These ulcers were refractory to multiple forms of therapy and 
were unhealed from between 2 and 12 years prior to our 
treatment.
    Several years ago, children with diabetes came to the House 
and Senate to request lifting the ban on fetal tissue research. 
At the time, many at the NIH believed that the less 
differentiated fetal pancreatic cells would be a better source 
for human transplantation as they should be less immunogenic. 
This too was hailed as a medical miracle.
    Subsequent studies, both in the United States and Europe, 
demonstrated this not to be the case. Successful 
transplantation studies in Canada using adult cadaveric 
pancreas cells have removed fetal tissue sources from the 
limelight.
    Having diabetes 42 of my 43 years, I can bear witness to 
the fallacy of consensus medical thinking. As a child, I 
remember my physician telling my mother that multiple NIH-
funded studies showed that blood sugar control did not make any 
difference into whether or not I would develop vascular 
complications. Indeed, when I was in medical school early in 
the eighties this same NIH dogma was being taught.
    At 10 years of age, I reasoned that nature kept blood 
sugars within a normal range for a reason and I surreptitiously 
injected myself with fast-acting insulin at meals to prevent my 
blood sugar from rising. People and physicians who thought like 
I did were labeled as extremists. It was not until the early 
1990's that reasonably well designed studies demonstrated that 
in fact blood sugar control is the single largest determinant 
as to whether a child will develop complications such as kidney 
failure and blindness. However, hundreds of thousands of 
children developed renal failure and died between 1960 and 1990 
because of this medical consensus mistake.
    As I testified last September before this committee, the 
mass of cells that begins the process of specific 
differentiation occurs very shortly after conception. The 
promotion of a specific and integrated genome pathway results 
in the beginning of that particular species of animal. The 
embryos that are fertilized in vitro differentiate and 
integrate their cellular signals in a specific way that are 
human. When they acquire rational thought and feeling is as yet 
debatable. When they are defined as human is not.
    All societies are ruled by laws, even unjust societies. The 
difference between a just and an unjust society is the set of 
precedents the society chooses to use in establishing its laws. 
In my view the United States is a uniquely just society, being 
the first government in the history of humankind where the 
rights of the individual supersede the perceived right of the 
State. This is the foundation that was established by the first 
10 amendments to our Constitution.
    Should human embryonic stem cell research be funded, it 
will be the first time in U.S. history that the Federal 
Government has determined the best use for a human being. This 
would be a cataclysmic paradigm shift. The perceived right of 
the state will have superseded the right of the individual. 
Even during the horrific times of slavery, the Federal 
Government did not fund programs using human beings for state 
purposes, although clearly individuals did.
    Enthusiastic medical consensus on a promising idea does not 
make any potential therapy a medical breakthrough, and a 
congressional subcommittee is probably not the ideal forum for 
discussing the scientific validity of one form of research 
compared to another. Existing regulatory bodies such as 
internal review boards and the Food and Drug Administration are 
better equipped to assess the validity and safety of such data.
    However, I am grateful that this subcommittee has taken the 
time and effort to hear the scientific as well as the societal 
case regarding human embryonic research. It is one of only a 
few appropriate forums to link all of these arguments together 
to best serve the national interest on all levels.

                           prepared statement

    In conclusion, the suffering caused by as yet incurable 
diseases can more quickly and with more certainty be alleviated 
by the multiple efforts that do not require a paradigm shift in 
our national character. Such a paradigm shift would necessarily 
result from Federal funding of human embryonic stem cell 
research.
    Thank you.
    [The statement follows:]
                Prepared Statement of Anton-Lewis Usala
    Senators, I appear before this subcommittee to discuss embryonic 
stem cell research, and alternative technologies that are currently 
under development in the company I founded. Our focus was to develop a 
method to regenerate and replace damaged patient tissues as a treatment 
for various human diseases. I developed a proprietary scaffolding, 
based on the structure of embryonic tissue scaffolding, to which adult 
cells can attach. The hypothesis that we first tested in multiple 
tissue culture, animal, and toxicology studies, was that upon binding 
to this embryonic-like structure, patient cells would be signaled to 
promote areas of the genome that are normally only promoted during 
embryogenesis and early fetogenesis. During this time, cells that are 
not committed to becoming a specific kind of cell, explosively 
differentiate into specialized tissues. As they do, they make 
connections and are modulated to integrate with all surrounding cells 
in a manner that is species specific.
    The proprietary scaffolding that my company has developed 
apparently enables regeneration of a specific kind of tissue called 
mesenchymal tissue. One of the three embryonic germ layers, mesenchymal 
tissue gives rise to connective tissue, blood vessels, bone, cartilage 
and parts of skin. In pre-clinical animal studies, we have demonstrated 
regeneration of blood vessels and skin in animals suffering from 
chronic diabetic and surgical extremity lesions.
    Years are required for an idea to be tested first in the petri 
dish, then in controlled animal efficacy studies, then in safety 
studies. Years are required to build the infrastructure to safely and 
reproducibly build a product for human clinical trials. Immense amounts 
of time and money are required to efficiently put the pieces into place 
that will give the Food and Drug Administration confidence that the 
risks for patients in experimental clinical trials are worth the 
potential benefit.
    My company is currently nearing completion of its first human 
clinical trial, with what I believe are exciting results. We have not 
yet submitted the final report to the Food and Drug Administration, and 
the data have NOT yet been peer reviewed. However, photos of chronic 
diabetic foot ulcer wounds in some of the patients treated with a 
single injection of our scaffolding appear to show rapid, well 
vascularized tissue regeneration, and closure of the ulcers within 2 to 
8 weeks. These ulcers were refractory to multiple forms of therapy and 
were unhealed for 2 to 12 years prior to our therapy.
    Yesterday, on the Today show, a segment was aired showing a mother 
injecting insulin into her four year old diabetic child. The mother 
stated she was extremely supportive of embryonic stem cell research if 
it could cure her child. It is not honest to bring before this 
Committee people such as myself, who have chronic illnesses for which 
there is no cure, as a valid argument for funding human embryonic stem 
cell research. There are many alternative paths; and if there is a 
legal or ethical reason not to conduct this research, public resources 
can be all the more effectively focused on those alternative paths.
    There are private companies in human clinical trials using porcine 
derived neurons as a treatment for Parkinson's disease. The first 
project my company embarked upon also utilized porcine tissue sources 
as a treatment for diabetes. We showed remarkable pre-clinical success 
in transplanting porcine tissue into diabetic dogs, without 
immunosuppressive therapy, and maintaining the tissue without 
rejection. Others in Europe and New Zealand have conducted human 
clinical trials utilizing microencapsulated porcine tissue. Pre-
clinical animal studies utilizing adult human stem cells have to date 
demonstrated at least as much efficacy as human embryonic stem cells, 
and the advantages of using embryonic stem cells is only theoretical at 
this point in time.
    There is little data to support, or infer, that embryonic human 
stem cells have any advantages over adult human stem cells in medical 
research. As a scientist in the field of human tissue regeneration, it 
is clear to me that integrating functional new tissue, not simply 
healthy tissue, into a diseased area requires integration of hundreds 
if not thousands of signals. Several years ago, children and patients 
with diabetes came to the House and Senate to request lifting the ban 
on fetal tissue research. At the time, many at the NIH believed the 
less differentiated fetal pancreatic tissues would be a better source 
for human transplantation as they should be less immunogenic. This, 
too, was hailed as a medical miracle. Subsequent studies, both in the 
United States, and in Europe, demonstrated this not to be the case, and 
the successful transplantation studies in Canada, using adult cadaveric 
pancreatic cells, have removed fetal tissue sources from the limelight.
    Having diabetes 42 of my 43 years, I can bear witness to the 
fallacy of consensus medical thinking. As a child, I remember my 
physician telling my mother that multiple NIH funded studies showed 
blood sugar control did not make any difference in whether or not I 
would develop vascular complications. Indeed, when I was in medical 
school in the early eighties, this same dogma was being taught.
    At ten years of age, I reasoned that nature kept blood sugars 
within a normal range, and I surreptitiously injected myself with fast 
acting insulin at meals to prevent my blood sugar from rising (as 
occurs in patients without diabetes). People who thought like I did 
were labeled as extremists. It was not until the early 1990's that 
multiple reasonably well designed studies demonstrated that in fact, 
blood sugar control is the single largest determinant as to whether a 
child will develop complications such as kidney failure and blindness. 
However, hundreds of thousands of children developed renal failure 
between 1960 and 1990 because of this medical consensus mistake.
    We all agree that the diseases for which cures are sought through 
embryonic or adult stem research are responsible for a great deal of 
human suffering, as well as economic cost to the nation. According to 
the National Bioethics Advisory Commission, most of us also agree that 
human embryos are deserving of respect. It is for that last reason that 
these hearings are being held.
    As I testified last September before this subcommittee, the mass of 
cells that begins the process of specific differentiation occurs very 
shortly after conception. The promotion of a specific and integrated 
genome pathway results in the beginning of that particular species of 
animal. The embryos that are fertilized in vitro differentiate and 
integrate their cellular signals in a specific way that are human. When 
they acquire rational thought or feeling is as yet debatable; when they 
are defined as human is not.
    The real question before Congress is whether or not this research 
should be funded by the federal government. Whether it is 
scientifically valid is not an issue that Congress can resolve--for if 
there is no legal or ethical issue, it should be considered by the 
relevant regulatory bodies as any other approach is considered. 
However, the legal and ethical issues are paramount. As Dr. Frank 
Young, a former FDA Commissioner and physician scientist says:

    ``I believe that the only defensible position is that life begins 
at conception whether in the petri dish or the uterus. To destroy 
embryos for only potential benefits that are promised to suffering 
people before the work is done in animals is misleading, inappropriate 
and in my opinion utilitarian ethics. We do pre-clinical tests of 
medicines in animals rather than rush into humans to avoid mistakes as 
much as possible. Why rush into experiments or trials with ES before 
completing animal studies and exploring adult stem cells?''

    As I said before this subcommittee in September, all societies are 
ruled by law, even unjust societies. The difference between a just and 
an unjust society is the set of precedents the society chooses to use 
in establishing its law. In my view, the United States is a uniquely 
just society, being the first government in the history of humankind 
where the rights of the individual supersede the perceived right of the 
State. This is the foundation that was established by the first ten 
amendments to our Constitution. Should human embryonic stem cell 
research be funded, it will be the first time in U.S. history the 
Federal government has determined the best ``use'' for a human being. 
This would be a cataclysmic paradigm shift. The perceived right of the 
State will have superseded the right of the individual. Even during the 
horrific times of slavery, the Federal Government did not fund programs 
using human beings for State purposes (although clearly individuals 
did).
    Enthusiastic medical consensus on a promising idea does not make 
any potential therapy a medical breakthrough, and a congressional 
subcommittee is probably not the ideal forum for discussing the 
scientific validity of one form of research compared to another. 
Existing regulatory bodies, such as Internal Review Boards and the Food 
and Drug Administration, are better equipped to assess the validity and 
safety of such research data. However, I am grateful that this 
subcommittee has taken such time and effort to hear both the 
scientific, as well as the societal, case regarding human embryonic 
research. It is one of only a few appropriate forums to link all of 
these arguments together to best serve the national interest on all 
levels.
    In my view, the suffering caused by as yet incurable diseases, can 
more quickly and with more certainty, be alleviated by the multiple 
efforts that do not require a paradigm shift in our national character. 
Such a paradigm shift would necessarily result from Federal funding of 
human embryonic stem cell research.

    Senator Harkin. Dr. Usala, thank you very much.
    Now we will turn to Dr. Gibbons. Dr. Gibbons.
STATEMENT OF WILLIAM E. GIBBONS, M.D., CHAIRMAN, JONES 
            INSTITUTE FOR REPRODUCTIVE MEDICINE
    Dr. Gibbons. Thank you. My name is William Gibbons. I am 
chairman of the Department of Obstetrics and Gynecology at 
Eastern Virginia Medical School, within which resides the Jones 
Institute of Reproductive Medicine. I am here today with Dr. 
Susan Lanzendorf, the lead scientist on the recently published 
work concerning the experience of the Jones Institute with 
human stem cells. Dr. Lanzendorf and I would like to thank the 
committee for the opportunity to participate in the dialogue 
concerning the use of human stem cells.
    I am a reproductive endocrinologist. Dr. Lanzendorf is a 
Ph.D. reproductive scientist. We are not here today to indicate 
that we have all of the answers regarding this very complex 
scientifically and ethically challenging process. The Jones 
Institute has initiated some studies involving human embryonic 
stem cells, research which we are hopeful may lead to the 
realization that the potentially staggering benefits of stem 
cell therapy to many patient conditions.
    This project was initiated after multiple levels of input 
from bioethicists, clerics, lay leaders, and legal scholars. It 
was debated at all levels of our institution, including the 
multidisciplinary approval of the institutional review board at 
the Eastern Virginia Medical School.
    Was there unanimity? No. Was there consensus? Yes.
    In addition, we have followed the guidelines of committees 
which have included the late 1970's advisory panel to the 
Department of Health and Human Services, the ethics panel of 
the American Society of Reproductive Medicine, and the 1994 
Special Panel to the National Institutes of Health.
    The path chosen by the Jones Institute was done for its 
clarity of informed consent, psychological assessment of 
participants, and to optimize the medical success by focusing 
on a volunteer population that was not infertile and was 
younger than our infertile population, as oocyte and egg 
quality is dependent upon a woman's age.

                           prepared statement

    To see the clinical benefits of this process, it is going 
to require tremendous resources, perhaps best coming from both 
the public and the private sector. Before Dr. Lansdorf speaks 
to her results, I would ask this powerful forum to allow the 
continuation of this controversial, but critically important, 
research.
    Thank you. Dr. Lanzendorf.
    [The statement follows:]
                Prepared Statement of William E. Gibbons
    My name is William E. Gibbons, Chairman of the Department of 
Obstetrics and Gynecology at the Eastern Virginia Medical School, 
within which resides the Jones Institute for Reproductive Medicine. I 
am here today with Dr. Susan Lanzendorf, the lead scientist on the 
recently published work concerning the experience of the Jones 
Institute with stem cells. Dr. Lanzendorf and I would like to thank the 
committee for the opportunity to participate in the dialogue concerning 
the use of human stem cells.
    I am a reproductive endocrinologist and Dr. Lanzendorf is a Ph.D.-
reproductive scientist. We are not here today stating that we have all 
the answers to this complex scientific and ethically challenging 
process. The Jones Institute has initiated some studies involving human 
embryonic stem cell research which we are hopeful may lead to the 
realization of the potentially-staggering benefits that stem cell 
therapy may hold for many patient conditions, including Alzheimer's 
disease, diabetes and others. This project was initiated after multiple 
levels of input from bioethicists, clerics, lay leaders and legal 
scholars. It was debated at all levels of the institution including the 
multidisciplinary approval of the Institutional Review Board at Eastern 
Virginia Medical School. Was there unanimity: No. Was there consensus: 
Yes. In addition, we followed the guidelines of committees including; 
the late 70's advisory panel to the Department of Health and Human 
Services, the Ethics Panel of the American Society of Reproductive 
Medicine, the 1994 special panel of the NIH, and others.
    The Jones Institute chose this path for its clarity of informed 
consent, psychological assessment of participants, and to optimize the 
medical success by focusing on a volunteer population that was not 
infertile and that were younger than our infertility population, as 
oocyte or egg quality is a function of a woman's age.
    To see the clinical benefits from this process it is going to 
require tremendous resources, perhaps best coming from both the public 
and private sectors. Before Dr. Lanzendorf speaks to her results I 
would ask this powerful forum to allow the continuation of this 
controversial, but critically important research.

    Senator Harkin. Thank you, Dr. Gibbons.
    Dr. Lanzendorf.
STATEMENT OF SUSAN LANZENDORF, Ph.D., EASTERN VIRGINIA 
            MEDICAL SCHOOL
    Dr. Lanzendorf. Thank you for this opportunity to discuss 
our work with human embryonic stem cells. As Dr. Gibbons has 
explained, I am a reproductive scientist and my main area of 
interest is fertilization and the study of early stages of 
embryo development.
    In the mid-1990's it became clear that science was moving 
forward in the potential uses of embryonic stem cells. 
Embryonic stem cells were being produced in many different 
animal species and it was becoming clear that they may one day 
provide a means for curing numerous diseases. My colleagues and 
I joined many scientists around the world who began evaluating 
ways to study human stem cells.
    Some scientists directed their efforts toward harvesting 
embryonic germ cells from aborted fetuses, evaluating them for 
their ability to develop into different cell types. Other 
investigators chose to initiate embryonic stem cell lines from 
human embryos obtained following infertility treatment.
    In 1994 we also began evaluating methods for initiating 
human embryonic stem cell lines. We also looked at embryos 
donated by infertile couples as a source of study material. 
However, we found that few such embryos existed at our center. 
While it is true that many couples no longer wishing to store 
their embryos do request that they be destroyed, it is usually 
their wish that the embryos be discarded without further 
evaluation. Therefore, these embryos would not be available for 
embryonic stem cell research.
    Another option that we evaluated was the use of donated 
gametes for the production of embryos, from which we could 
harvest the stem cells. The evaluation of this possible source 
was not entered into lightly and the debates and discussions 
that followed happened over a number of years. In fact, it was 
about 3 years from the time we began the discussion to the 
performance of the first egg retrieval.
    The study published in Fertility-Sterility was solely 
investigational. It was our goal to evaluate the laboratory 
conditions required to produce and maintain human embryonic 
stem cell lines. The fact that the embryos used in these early 
studies were obtained from healthy, fertile volunteers most 
certainly aided us in our studies. These findings have also 
allowed us to modify our techniques and obtain institutional 
approval for further studies to investigate how human embryonic 
stem cell may one day cure diseases.
    The use of donated gametes to produce embryonic stem cells 
was the path we chose to begin our investigation. I personally 
feel that this method is ethical in that the donors of the 
gametes specifically consent to what they are being used for 
and that the embryos that are produced were never done so for 
the purpose of creating an individual. However, I also 
understand that there are other avenues available for the 
production of human stem cells.

                           prepared statement

    Over the past week I have been amazed at the number of 
individuals struggling with diabetes, spinal cord injury, and 
other conditions who have contacted by colleagues and me 
offering to donate their gametes and embryos to further this 
research. Regardless of how it progresses, we urgently request 
that resources be made available to continue the research and 
endeavors of the scientist community in the area of human 
embryonic stem cells.
    [The statement follows:]
                 Prepared Statement of Susan Lanzendorf
    Thank you for this opportunity to discuss our work with human 
embryonic stem cells.
    As Dr. Gibbons has explained, I am a reproductive scientist and my 
main area of interest is fertilization and the study of the early 
stages of embryo development. In the mid-1990's, it became clear that 
science was moving forward in the potential uses of embryonic stem 
cells. Embryonic stem cells were being produced in many different 
animal species and it was becoming clear that they may one day provide 
a means for curing numerous diseases.
    My colleagues and I joined many scientists around the world who 
began evaluating ways to study human stem cells. Some scientists 
directed their efforts towards harvesting embryonic germ cells from 
aborted fetuses, evaluating them for their ability to develop into 
different types of cells. Other investigators chose to initiate 
embryonic stem cell lines from human embryos obtained following 
infertility treatment.
    In 1994, we also began evaluating methods for initiating human 
embryonic stem cell lines. We also looked at embryos donated by 
infertile couples as a source of study material. However, we found that 
few such embryos existed at our center. While it is true that many 
couples no longer wishing to store their embryos do request that they 
be destroyed, it is usually their wish that the embryos be discarded 
without any further evaluation. Therefore, these embryos would not be 
available for embryonic stem cell research.
    Another option that we evaluated was the use of donated gametes for 
the production of embryos from which we could harvest the stem cells. 
The evaluation of this possible source was not entered into lightly and 
the debates and discussions that followed happened over a number of 
years. In fact, it was about 3 years from the time we began the 
discussion to the performance of the first egg retrieval.
    The study published in Fertility and Sterility was solely 
investigational. It was our goal to evaluate the laboratory conditions 
required to produce and maintain human embryonic stem cell lines. The 
fact that the embryos used in these early studies were obtained from 
healthy, fertile volunteers most certainly aided us in our studies. 
These findings have also allowed us to modify our techniques and obtain 
institutional approval for further studies to investigate how human 
embryonic stem cells may one day cure diseases.
    The use of donated gametes to produce embryonic stem cells was the 
path we chose to begin our investigations. I personally feel that this 
method is ethical in that the donors of the gametes specifically 
consent to what they are being used for and that the embryos that are 
produced were never done so for the purpose of creating an individual. 
However, I also understand that there are other avenues available for 
the production of human stem cells. Over the past week, I have been 
amazed at the number of individuals struggling with diabetes, spinal 
cord injury and other conditions who have contacted my colleagues and 
me offering to donate their gametes and embryos to further this 
research. Regardless of how it progresses, we urgently request that 
resources be made available to continue the research endeavors of the 
scientific community in the area of human embryonic stem cells.

    Senator Harkin. Thank you very much, Dr. Lanzendorf.
    Now Dr. Krause.
STATEMENT OF DIANE KRAUSE, M.D., Ph.D., ASSOCIATE 
            PROFESSOR, YALE UNIVERSITY
    Dr. Krause. Chairman Harkin, ranking member Specter, and 
members of the Senate Appropriations Committee: I am Dr. Diane 
Krause. I am an Associate Professor at Yale University School 
of Medicine, where I work on adult-derived stem cells. My 
objective here today is to describe why Federal funds need to 
be applied to embryonic stem cell research. I am speaking not 
only for myself, but also for other members of the scientific 
and medical community, and specifically on behalf of the 
American Society of Hematology, which has over 10,000 members 
united by their commitment to understanding and curing blood 
disorders.
    Recently, my colleagues and I published data providing the 
strongest evidence yet that adult stem cells may be used to 
repair multiple organs. We showed that in mice adult-derived 
bone marrow cells make not only blood, as was expected, but 
also produce mature cells of the liver, the lung, the 
intestine, and the skin.
    While I am very excited about this research, it is 
important that the subcommittee understand that adult stem cell 
research is not a substitute for embryonic stem cell research. 
I am deeply concerned that people seeking to end Federal 
funding for human embryonic stem cell research are using my 
data as justification for discontinuing this Federal funding. 
This interpretation is not only stunningly premature, but 
potentially undermines the development of adult stem cell 
therapeutic options. In fact, the progress made in studying 
adult human stem cells relies on what has been learned from 
embryonic stem cell studies.
    I would like to discuss the importance of embryonic stem 
cell research and the need for the administration to allow 
Federally funded embryonic stem cell studies to proceed. It is 
my testimony that these two areas of research together will 
lead to effective and safe treatments for life-threatening 
diseases.
    There are three main reasons why embryonic stem cells need 
to be funded.
    First, embryonic stem cells are unique in that they can be 
grown in vitro, outside the body, and maintain the capacity to 
become any type of cell in the body. In contrast, adult stem 
cells have not yet been grown in vitro and it is not yet known 
whether adult stem cells have the same ability as embryonic 
stem cells do to become all cell types.
    Second, in order for scientific discovery to continue 
rapidly in this field, both adult and embryonic stem cells will 
need to be studied and compared. At present, we can obtain far 
more information from embryonic stem cells, which are the 
experts in versatility, than from rare adult-derived cells.
    Third, no one can predict which line of investigation will 
lead to development of effective and safe treatments for human 
diseases. In fact, it is likely that critical therapies for 
different diseases will derive from different research avenues. 
To eliminate one avenue simply because the other has begun to 
show promise is to speculate on the basis of too little data on 
matters which may be life-threatening or life-preserving for 
large numbers of people.
    I urge you to take into consideration the long-term 
objectives of embryonic stem cell research, to cure and 
eliminate life-threatening diseases. Any possible compromise, 
such as that attributed to the administration, that allows 
Federally funded research using only existing cell lines, is 
far too limiting for multiple reasons. Perhaps the most 
important reason is that scientists need to compare multiple 
cell lines in order to better understand the common factors 
that give embryonic stem cells their incredible versatility.

                           prepared statement

    In closing, embryonic stem cell research should receive 
Federal funding because work on these cells is invaluable and 
work on adult-derived stem cells is just beginning. To close 
off one avenue because of premature assumptions about the other 
is to play odds with people's lives.
    Thank you very much for providing me this opportunity to 
speak with you.
    [The statement follows:]
                 Prepared Statement of Dr. Diane Krause
    Chairman Harkin, Ranking Member Specter, and members of the Senate 
Appropriations Subcommittee: I am Dr. Diane Krause and I am an 
associate professor at Yale University School of Medicine where I work 
on adult stem cells. My objective here today is to describe why Federal 
funds need to be applied to embryonic stem cell research. I am speaking 
not only for myself, but also for other members of the scientific and 
medical community and specifically on behalf of the American Society of 
Hematology which has over 10,000 members united by their commitment to 
understanding and curing blood disorders.
    My colleagues and I recently published data providing the strongest 
evidence so far that adult stem cells could be used to repair multiple 
organs. We showed that, in mice, adult-derived bone marrow cells make 
not only blood, as expected, but also produce mature cells of the 
liver, the lung, the intestine and the skin.
    While I am very excited about this research, it is important that 
the Subcommittee understand that adult stem cell research is not a 
substitute for embryonic stem cell research. I am deeply concerned that 
people seeking to end federal funding for human embryonic stem cell 
research have inappropriately used my data as justification for 
discontinuing federal funding for embryonic stem cell research. This 
interpretation is not only stunningly premature, but potentially 
undermines the development of adult stem cell therapeutic options.
    In fact, the progress made in studying adult stem cells relies on 
what has been learned from embryonic stem cell studies. Today I would 
like to discuss the importance of embryonic stem cell research and the 
need for the Administration to allow federally funded embryonic stem 
cell studies to proceed. It is my testimony that these two areas of 
research together will lead to effective and safe treatments for life-
threatening diseases.
    We all agree that it would be optimal if we could bypass this 
controversial issue of using embryonic stem cells by using adult 
derived stem cells exclusively. However, we also agree that it is 
important to develop treatments and cures for human diseases and 
progress toward this goal will be slowed considerably if embryonic stem 
cell work is inhibited!
    There are three main reasons why embryonic stem cell studies need 
to be funded.
    First, embryonic stem cells are unique in that they can be grown in 
vitro (outside of the body), and maintain the capacity to become any 
type of cell in the body. In contrast, adult stem cells have not yet 
been grown in vitro, and it is not yet known whether adult stem cells 
have the same ability as embryonic cells to become all cell types.
    Second, in order for scientific discovery to continue rapidly in 
this field, both adult and embryonic stem cells will need to be studied 
and compared. At present, we can obtain far more information from 
embryonic stem cells, which are the ``experts'' in plasticity, than 
from rare adult derived cells.
    Third, no one can predict which lines of investigation will lead to 
development of effective and safe treatments for human disease. In 
fact, it is likely that critical therapies for multiple different human 
diseases will derive from different research avenues. To eliminate one 
avenue simply because the other has begun to show promise is to 
speculate on the basis of too little data on matters which may well be 
life threatening or life preserving for large numbers of people
    I urge you to take into consideration the long-term objectives of 
embryonic stem cell research--to cure and eliminate life-threatening 
disease. Any possible compromise, such as that attributed to the 
Administration that allows federally funded research using only 
existing cell lines is far too limiting. Perhaps the most important 
reason is that scientists need to compare multiple cell lines in order 
to better understand the common factors that give embryonic stem cells 
their incredible plasticity.
    In closing, embryonic stem cell research should receive federal 
funding because work on these cells is invaluable and work on adult 
derived stem cells is just beginning. To close off one avenue because 
of premature assumptions about the other is to play the odds with 
people's lives.
    Thank you very much for providing me this opportunity to address 
you on this critical issue.

    Senator Harkin. Thank you very much, Dr. Krause.
    Now we go to Dr. Hendrix.
STATEMENT OF MARY J.C. HENDRIX, Ph.D., PROFESSOR AND 
            HEAD, DEPARTMENT OF ANATOMY AND CELL 
            BIOLOGY, UNIVERSITY OF IOWA COLLEGE OF 
            MEDICINE
    Ms. Hendrix. Thank you. Chairman Harkin, Senator Specter, 
and distinguished members of the subcommittee: I am Dr. Mary 
Hendrix and I am honored to be here today to present a 
scientific perspective on embryonic stem cell research. As a 
scientist conducting cancer research and as the immediate past 
president of the Federation of American Societies for 
Experimental Biology, or FASEB, I speak to you today on behalf 
of FASEB's 21 member societies and more than 60,000 member 
scientists.
    First I would like to briefly summarize the current state 
of human embryonic stem cell research in the United States. The 
human embryonic stem cells that we are talking about are 
isolated from a very early embryo called the blastocyst, which 
is so small that it can fit on the tip of a sewing needle. The 
inner cell mass from which the stem cells are derived is 
composed of 30 to 34 undifferentiated cells. These embryonic 
stem cells of the inner cell mass cannot form a human being, 
not even when implanted into a woman's womb.
    This scientist observation has sparked an emotional and 
profound debate about whether Federal funds should be used to 
support this new and promising area of research, and right now 
our leading medical institutions are prevented from conducting 
stem cell research, severely limiting the flow of information. 
There is great promise, as we have heard, in human embryonic 
stem cell research.
    First, human embryonic stem cells are self-renewing. So 
far, the cell lines derived appear to have virtually unlimited 
replication capacity.
    Second, human embryonic stem cells are pluripotent, that is 
they can differentiate into many diverse cell types that 
comprise the human body. Understanding how these particular 
cells develop will allow us to direct their differentiation 
into specific cell types and tissues. We now know that the 
development is governed by the intricately choreographed 
interactions of hundreds of genes, and with this research we 
hope to realize the true potential of the fully sequenced 
genome.
    Stem cells may also provide cures and therapies for many of 
the diseases that plague humanity. Let me just mention a few. 
Patients with Alzheimer's and Parkinson's disease might be 
treated by replacing dead cells with neurons grown from 
embryonic stem cells. The most hopeful near-term example of the 
promise of stem cell research is in type 1 or juvenile 
diabetes, where new pancreatic islet cells, cells that produce 
the insulin in the body, can be transplanted into diabetes 
patients.
    In my own field of cancer biology research, stem cells hold 
great promise. Stem cells can renew themselves indefinitely and 
may shed new light on the uncontrolled growth of cancer cells. 
In the realm of gene therapy for cancer, if embryonic stem cell 
research is allowed to proceed it might allow us to engineer 
cells and tissues that are resistant to the most effective, but 
also the most toxic, cancer therapies. That would allow normal 
tissues to be protected while cancer cells are selectively 
destroyed.
    Existing embryonic stem cell lines are a valuable resource, 
but they are insufficient to explore adequately the vast 
therapeutic potential of stem cell research. At this time we do 
not know precisely how many human embryonic stem cell lines are 
available for study and we do not know if these existing cell 
lines are genetically diverse enough. The scientific community, 
like the public, is divided on the issue of how many embryonic 
stem cell lines will be needed to address current and future 
scientific questions.
    Whatever the needs may be, the NIH can provide the 
oversight necessary to regulate the ethical considerations 
involved in all aspects of this research.
    I would like to conclude by saying, we at FASEB strongly 
support allowing human embryonic stem cell research to go 
forward under the pending NIH guidelines and, second, based on 
our current knowledge, while adult stem cell research is highly 
promising and should be pursued, embryonic stem cells have 
greater potential.

                           prepared statement

    We are grateful to you, Mr. Chairman, and to your colleague 
Senator Specter for championing the opportunities to conduct 
human embryonic stem cell research. We hope it can be done in 
an open manner so that the public can see exactly what is going 
on.
    Thank you.
    [The statement follows:]

                Prepared Statement of Mary J.C. Hendrix

    Chairman Harkin, Senator Specter, and distinguished Members of the 
Subcommittee, I am Mary Hendrix, and I am honored to be here today to 
present a scientific perspective on embryonic stem cell research. As a 
scientist conducting cancer research, and as the immediate past 
president of the Federation of American Societies for Experimental 
Biology or FASEB, I speak to you today on behalf of FASEB's 21 member 
societies and their more than 60,000 scientist-members.
                     the current state of research
    First, I would like to summarize the current state of human 
embryonic stem cell research in the United States.
    In 1998, a milestone in biomedical research was achieved. We 
learned that cells isolated from the inner cell mass of a very early 
human embryo (4-5 days after fertilization), and grown in culture under 
special conditions, could develop into the many different cell types of 
the human body. This very early embryo (called the blastocyst) is so 
small that it can fit on the tip of a sewing needle. The inner cell 
mass within it, and from which stem cells are derived, is comprised of 
30-34 undifferentiated cells. These embryonic stem cells of the inner 
cell mass cannot form a human being, not even when implanted into a 
woman's womb.
    This scientific observation has sparked an emotional and profound 
debate about whether federal funds should be used to support this new 
and promising area of biomedical investigation. Right now, our leading 
medical institutions are prevented from conducting stem cell research, 
severely limiting the flow of information.
           the promise of human embryonic stem cell research
    There is great promise in human embryonic stem cell research, 
because we might learn how to grow specialized cells for therapeutic 
purposes. This is possible because of two unique attributes of these 
cells.
    First, human embryonic stem cells are self-renewing. So far, the 
cell lines derived from the pioneering work of Dr. James Thomson of the 
University of Wisconsin have undergone more than 300 population 
doublings and appear to have virtually unlimited replication capacity 
based on the expression of certain cellular and genetic markers.
    Second, human embryonic stem cells are pluripotent, that is, they 
can differentiate into many of the diverse cell types that comprise the 
human body. This capacity for replication, coupled with the property of 
pluripotency provides researchers an extraordinary opportunity. 
Understanding how these particular cells develop may allow us to learn 
how to direct their differentiation into specific cell types or 
tissues.
    Embryonic stem cells might also provide part of the answer to the 
fundamental mystery of human biology: how does an early blastocyst 
develop into the multitude of cells that become the tissues, organs and 
limbs of an adult? We know now that this development is governed by the 
intricately choreographed interactions of dozens, even hundreds of 
genes. Stem cell research is allowing scientists to understand how 
genes interact during human development. With this research, we can 
realize the true potential of the fully sequenced human genome.
    In addition to revealing the secrets of human development, stem 
cells may provide cures and therapies for many of the diseases that 
plague humanity. Let me mention just a few here.
             neurodegenerative diseases and neuronal injury
    Among the most heart-wrenching human afflictions are diseases in 
which cells of the brain and nervous system degenerate and die, for 
example, in patients suffering from Alzheimer's and Parkinson's 
diseases. In the case of Alzheimer's disease, dead and dysfunctional 
cells in the hippocampal and cortical regions of the brain might 
actually be replaced by transplanted, specialized neurons developed 
from embryonic stem cells. Similarly, Parkinson's disease, which is 
characterized by the death of dopamine-transmitting neurons, might be 
treated by replacing dead cells with neurons grown from embryonic stem 
cells.
    Recent research performed in rodents suggests that pluripotent stem 
cells may help repair the damaged nervous system. In this very exciting 
research, paralyzed rodents with spinal cord injuries have regained 
some degree of mobility following transplantation of oligodendrocytes 
derived from mouse embryonic stem cells.
                  transplantation and type i diabetes
    The most hopeful near-term example of the promise of stem cell 
research is in type-I or juvenile diabetes. Using embryonic stem cells 
and our understanding of how they differentiate, we may be able to give 
patients new pancreatic islet cells, the cells that produce insulin in 
the body, and thereby provide a cure for juvenile diabetes.
                                 cancer
    In my own field of cancer biology, stem cell research holds great 
promise. That special intrinsic property of stem cells, their ability 
to renew themselves indefinitely, may shed light on the similar 
uncontrolled growth of cancer cells. By understanding how embryonic 
stem cells are able to replicate themselves, we might be able to 
understand the cellular mechanisms by which tumor cells become immortal 
and grow out of control until they kill the patient.
    In the realm of gene therapy for cancer, if embryonic stem cell 
research is allowed to proceed, it might be possible to ``engineer'' 
cells and tissues that are resistant to the most effective, but also 
most toxic, cancer therapies, so that normal tissues would be protected 
while cancer cells are selectively destroyed. By the same principle, it 
might also become possible to design cells that generate antibodies 
against cancer cells, effectively programming the patient's immune 
system to attack deadly tumor cells.
            adult stem cells hold promise but have drawbacks
    Let me conclude my discussion on the promise of embryonic stem cell 
research by addressing adult stem cells. I want to emphasize that 
embryonic and adult stem cell research are both extremely promising and 
should be federally supported. In fact, they are complementary to each 
other but may not be interchangeable. While adult stem cells may indeed 
hold great potential, our current understanding is that they have 
several major drawbacks.
    First, it is difficult to identify and isolate adult pluripotent 
stem cells. Second, adult stem cells appear to be much more restricted 
in their ability to differentiate into different cell types in the 
body, and it remains to be proven whether adult stem cells can truly 
give rise to all cell types in the body. Finally, the ability of adult 
stem cells to replicate is not as robust as embryonic stem cells.
    Mr. Chairman and Members of the Subcommittee, while it is possible 
that pluripotent adult stem cells may exist and that additional 
research might reveal their sources in the body, we have pluripotent 
embryonic stem cells now. The potential of adult stem cells remains 
only a hope, and that's why federally-funded embryonic stem cell 
research, which is far more likely to lead to new knowledge and 
therapies quickly, must be allowed to proceed.
   the nih guidelines for research using human pluripotent stem cells
    Mr. Chairman, having concluded my overview of the current state of 
human embryonic stem cell research, I would like to turn to the current 
NIH Guidelines for Research Using Human Pluripotent Stem Cells. Let me 
state from the outset that FASEB strongly endorses these NIH 
Guidelines. These draft guidelines were presented to the public and the 
scientific community for formal review in 1999, and the revised 
guidelines issued by NIH last year reflect the advice and ethical 
judgements of scientists and other concerned citizens.
    Under the NIH Guidelines, research using pluripotent embryonic stem 
cells is eligible for Federal funding. This research can only be 
supported if the embryonic stem cells are derived without Federal funds 
and are certified to have been derived from embryos in excess of 
clinical need for in vitro fertilization procedures. Research on 
embryonic stem cells derived from fetal tissue may also be federally 
supported under the NIH Guidelines.
    Please allow me to make an important point about the virtue of the 
NIH Guidelines: Federal funding means medical progress under Federal 
oversight. Scientists working under the NIH Guidelines and with Federal 
oversight will be allowed to conduct the research and provide the cures 
and therapies for which we all hope.
               existing stem cell lines are insufficient
    Existing embryonic stem cell lines are a valuable resource, but 
they are insufficient to explore adequately the vast therapeutic 
potential of stem cell research. At this time, there are approximately 
30 human embryo-derived cell lines available for study. We do not know 
if these existing cell lines are genetically diverse enough.
    The scientific community, like the public, is divided on the issue 
of how many embryonic stem cell lines will be needed to address current 
and future scientific questions. Whatever the needs may be, the NIH can 
provide the oversight necessary to regulate the ethical considerations 
involved in all aspects of this research.
                               conclusion
    We are grateful to you Mr. Chairman, and your colleague Senator 
Specter, for championing opportunities for researchers to conduct human 
embryonic stem cell research in an open manner--with government support 
and oversight.
    The public has every right to know exactly what type of human 
embryonic stem cell research is being performed in our country. For 
that to happen, the government must provide funding and the appropriate 
oversight for these new research opportunities. In the absence of 
Federal support and oversight, this exciting line of research will 
occur only behind closed doors. To preclude such an approach is to 
delay the prospect of life enhancing biomedical breakthroughs.

    Senator Harkin. Dr. Hendrix, thank you very much.
    Now we turn to Dr. Michael West, president and CEO of 
Advanced Cell Technology. Dr. West.
STATEMENT OF MICHAEL D. WEST, Ph.D., PRESIDENT AND CHEF 
            EXECUTIVE OFFICER, ADVANCED CELL TECHNOLOGY
    Dr. West. Thank you, Mr. Chairman and members of the 
committee. My name is Michael West. I am the president and CEO 
of Advanced Cell Technology, a biotechnology company in 
Massachusetts. Prior to Advanced Cell, I was the founder of 
Geron Corporation in Menlo Park, California, and beginning in 
1995 organized an effort in collaboration with Jamie Thompson 
and John Gearhart to isolate the human embryonic stem cell and 
human embryonic germ cell.
    It may be of surprise and interest to know that I hold a 
pro-life position and, consistent with that, believed at that 
time and still do that the effort to isolate embryonic stem 
cells and to use them in medicine is moral and is consistent 
with the pro-life position. I would like to explain why and to 
clarify some misinformation. I feel that misinformation is our 
enemy more than anything else.
    To begin with, what are we so excited about with these 
embryonic stem cells? It has been mentioned frequently and in 
the report submitted by the NIH that these cells stand at the 
base of the tree of life, of cellular life, in that they can 
branch out, forming any cell and tissue in the body. So cells 
that at least to this date have never been made from an adult 
stem cell, like a beta cell for diabetes and so on, or dopamine 
for Parkinson's, can be obtained from these cells.
    Not only can any cell type be made from these cells, but 
even complex tissues, which is quite amazing in the history of 
science and medicine. These cells will self-aggregate, for 
instance, into intestine. They could be used to repair damaged 
intestine in colon cancer, as an example.
    Beginning in 1995, it became clear that we had a major 
unsolved problem, however. The problem is these are all cells 
that are not us. Our bodies would reject them as foreign. With 
the cloning of Dolly in 1997, it became clear that mankind in 
this decade had been given two enormous possibilities: one, to 
make any cell type for a person who is sick; second, the door 
is now open to find a way, an ethical and a carefully designed 
way, to give back a patient their own histocompatible, meaning 
their own cells that can be accepted by the body itself.
    This is an enormous opportunity and gift to mankind. The 
question is how do we take this gift and translate it into 
something that we all can agree on is a good and ethical use of 
technology? An important part of this debate--and we have heard 
it several times today--is what is this thing called the 
blastocyst embryo?
    I have a diagram here on the chart. An important part of 
this debate, first, is there is a constant misuse of 
information about what is a life or human life. Human cells are 
alive, we know this. We evolved from single cell organisms. 
These are living entities. So to say that there is human life 
in a sperm cell or an egg cell is correct. You could indeed 
adopt a sperm or an egg cell and bring in to show in testimony 
that we have created a human being from these cells.
    But we agree a sperm or an egg cell is not a human life. 
With the development of a multi-cellular animal, which is a 
human being, from the fertilized egg, we have this first 
aggregation of cells called the blastocyst. These cells on the 
outside will become the placenta. The cells on the inside are 
the subject of debate. If put in culture, they are simply an 
embryonic stem cell. An important point: these cells--and this 
is I think a central issue for this committee and all of us to 
grapple with. These cells have not committed to becoming any 
cell of the body or indeed somatic cells at all. They can still 
become a sperm or an egg cell.
    Second and very importantly, I think it goes to the heart 
of this issue: This blastocyst embryo has not individualized. 
It can become identical twins. If the intercell mass separates 
into two separate balls of cells, you will have identical twins 
that have, what is said, share the corionic sac, but have 
separate amniotic sacs. If two primitive streaks form no the 
intercell mass, you will have identical twins that share the 
same amniotic sac.
    These are lessons from nature that teach us that not only 
are these cells not body cells of any kind, they have not even 
become an individual, and to ascribe to unindividualized cells 
the status of a person is a logical inconsistency.
    We have heard references to the Bible and tradition is to 
help guide us moving forward into the future. There are two 
verses I would like to mention. One is in the book of 
Corinthians the Apostle Paul says: ``When I was a child, I 
spake as a child, I understood as a child, I thought as a 
child. But when I became a man, I put away childish things.''
    I think it is important for us as the leading country in 
technology to have a mature, reasoned, and I think 
dispassionate debate of these issues. We have been given two 
talents of gold, to recall another biblical parable, two gifts 
to mankind which are of great merit: the cell that can form any 
cell tissue in the body and I think this rather remarkable and 
unexpected gift of being able to return a cell back in time, in 
the tiny time machine of nuclear transfer, to make these cells 
identical to a patient.

                           prepared statement

    I would encourage the United States, as we have in the past 
in our exploration of space, for instance, to shrug off 
accusations that we are building a modern tower of Babel in 
reaching for the heavens and to touch the moon. We shrug off 
those fears and superstitions, and with good intentions and 
proper moral and ethical debate we bravely move into the future 
to try to find a way to alleviate human suffering.
    Thank you.
    [The statement follows:]

                 Prepared Statement of Michael D. West

    Mr. Chairman and members of the Subcommittee on Labor, Health and 
Human Services, Education, and Related Agencies, my name is Michael D. 
West and I am the President and Chief Executive Officer of Advanced 
Cell Technology a biotechnology company based in Worcester, 
Massachusetts. A copy of my curriculum vitae is presented in Appendix 
A.
                              introduction
    I am pleased to testify today in regard to the new opportunities 
and challenges associated with human embryonic stem (ES) cell and 
nuclear transfer (NT) technologies.
    I will begin by describing the bright promise of these twin and 
interrelated technologies and then attempt to correct some 
misunderstandings relating to their application in medicine.
    It may be useful to point out that I think of myself as pro-life in 
that I have an enormous respect for the value of the individual human 
life. Indeed, in my years following college I protested abortion 
clinics. My goal was not to say to women that they did not have the 
right to choose. My intent was simply to urge them to reconsider the 
destruction of a developing human being. Despite my strong convictions 
about the value of the individual human life, in 1995 I organized the 
collaboration between Geron Corporation and the laboratories of Dr. 
James Thomson and John Gearhart to isolate human embryonic stem cells 
and germ cells from living human embryos and fetuses. My reasons were 
simple. These technologies are entirely designed to alleviate human 
suffering and to save human life. They are, in fact, pro-life. The 
opponents that argue they destroy human lives are simply and tragically 
mistaken. Let me explain why this is the case.
Human ES cells
    We are composed of trillions of individual living cells, glued 
together like the bricks of a building to construct the organs and 
tissues of our body. The cells in our bodies are called ``somatic 
cells'' to distinguish them from the ``germ line'' or reproductive 
cells that connect the generations. We now know that life evolved from 
such single-celled organisms that dominated all life some one billion 
years ago.


Figure 1. The Distinction Between the Life of Cells and the Life of and 
 Individual Human Being in the Human Life Cycle. Biological, that is, 
    ``cellular life'' began with the origins of life on earth. The 
  beginning of the life of an individual human being is linked to the 
appearance of somatic cells, that is, cells committed to form the human 
                                 body.

    Therefore, in answer to the question of when life begins, we must 
make a critical distinction. Biological life, that is to say, 
``cellular life'' has no recent beginnings. Our cells are, in fact, the 
descendents of cells that trace their beginnings to the origin of life 
on earth. When we speak of an individual human life, we are speaking of 
the communal life of a multicellular organism springing from the 
reproductive lineage of cells. The individual human life is composed of 
cells committed to somatic cell lineages. All somatic cells are all 
related in that they originate from an original cell formed from the 
union of a sperm and egg cell.
    The fertilization of the egg cell by a sperm leads to a single cell 
called the ``zygote''. From this first cell, multiple rounds of cell 
division over the first week result in a microscopic ball of cells with 
very unusual properties. This early embryo called the ``preimplantation 
embryo'' has not implanted in the uterus to formally begin a pregnancy.
    It is estimated that approximately 40 percent of preimplantation 
embryos formed following normal human sexual reproduction fail to 
attach to the uterus and are simply destroyed as a result.


 Figure 2. The Blastocyst Stage of the Preimplantation Embryo. At the 
  blastocyst stage of the preimplantation embryo, the external cells 
 called the ``trophectoderm'' are destined to attach to the uterus and 
 form the placenta. The remaining cells, the Inner Cell Mass (ICM) are 
completely undifferentiated and have not committed to any somatic cell 
                                lineage.

    From the above it should be clear that at the blastocyst stage of 
the preimplantation embryo, no body cells of any type have formed, or 
even more significantly, there is strong evidence that not even the 
earliest of events in the chain of events in somatic differentiation 
have initiated. A simple way of proving this is by observing subsequent 
events.
    Should the embryo implant in the uterus, the embryo, at 
approximately 14 days post fertilization will form what is called the 
primitive streak, the first definition that these ``seed'' cells will 
form an individual human being as opposed to the forming of two 
primitive streaks leading to identical twins. Rarely two primitive 
streaks form that are not completely separated leading to conjoined or 
Siamese twins. In addition, rarely, two separately fertilized egg cells 
fuse together to form a single embryo with two different cell types. 
This natural event leads to a tetragammetic chimera, that is a single 
human individual with some of the cells in their body being male from 
the original male embryo, and some cells being female from the original 
female embryo. These and other simple lessons in embryology teach us 
that despite the dogmatic assertions of some theologians, the evidence 
is decisive in support of the position that an individual human life, 
as opposed to merely cellular life, begins with the primitive streak, 
(i.e. after 14 days of development). Otherwise we are left with the 
logical absurdity of ascribing to the blastocyst personhood when we 
know, scientifically speaking, that no individual exists (i.e. the 
blastocyst may still form identical twins).


  Figure 3. The lack of Individuation of The Blastocyst Stage Embryo. 
Lessons from nature indicate that the blastocyst-stage preimplantation 
 embryo has not individualized. On the left fraternal or nonidentical 
  twins form from independently-fertilized egg cells. Identical twins 
 form from a single ICM breaking into two ICMS (center diagram) or by 
       two primitive streaks forming on one ICM (right diagram).

    Human ES cells are nothing other than ICM cells grown in the 
laboratory dish. Human ES cell technologies may greatly improve the 
availability of diverse cell types. Human ES cells are unique in that 
they stand near the base of the developmental tree. These cells are 
frequently designated ``totipotent'' stem cells, meaning that they are 
potentially capable of forming any cell or tissue type needed in 
medicine. These differ from adult stem cells that are ``pluripotent'' 
that is, capable of forming several, but only a limited number, of cell 
types. An example of pluripotent adult stem cells are the bone marrow 
stem cells now widely used in the treatment of cancer and other life-
threatening diseases.


  Figure 4. Human Embryonic Stem (ES) Cells are Inner Cell Mass (ICM) 
   Cells Cultured in the Laboratory Dish. Human ES cells were first 
   cultured for long periods of time in the laboratory by Dr. James 
           Thomson of the University of Wisconsin at Madison.

    Some have voiced objection to the use of human ES cells in medicine 
owing to the source of the cells. Whereas the use of these new 
technologies has already been carefully debated and approved in the 
United Kingdom, the United States lags disgracefully behind. I would 
like to think it is our goodness and our kindness that generates our 
uncertainty over these new technologies. Indeed, early in my life I 
might have argued that since we don't know when a human life begins, it 
is best not to tamper with the early embryo. But, with time I learned 
the facts of human embryology and cell biology. As the Apostle Paul 
said: ``When I was a child, I spake as a child, I understood as a 
child, I though as a child: but when I became a man, I put away 
childish things.'' (I Cor 13:11) In the same way it is absolutely a 
matter of life and death that policy makers in the United States 
carefully study the facts of human embryology and stem cells. A child's 
understanding of human reproduction could lead to disasterous 
consequences.
    With appropriate funding of research, we may soon learn to direct 
these cells to become vehicles of lifesaving potential. We may, for 
instance, become able to produce neurons for the treatment of 
Parkinson's disease and spinal cord injury, heart muscle cells for 
heart failure, cartilage for arthritis and many others as well. This 
research has great potential to help solve the first problem of tissue 
availability, but the technologies to direct these cells to become 
various cell types in adequate quantities remains to be elucidated. 
Because literally hundreds of cell types are needed, thousands of 
academic research projects need to be funded, far exceeding the 
resources of the biotechnology industry.


 Figure 5. Using Embryonic Stem (ES) cells in human therapy. Human ES 
cells are immortal while cultured in the undifferentiated state and can 
    theoretically lead to any cell or tissue type in the human body.

    As promising as ES cell technology may be, it does not solve the 
second problem of histocompatibility. Human ES cells obtained from 
embryos derived during in vitro fertilization procedures, or from fetal 
sources, are essentially cells from another individual (allogeneic). 
Several approaches can be envisioned to solve the problem of 
histocompatibility. One approach would be to make vast numbers of human 
ES cell lines that could be stored in a frozen state. This ``library'' 
of cells would then offer varied surface antigens, such that the 
patient's physician could search through the library for cells that are 
as close as possible to the patient. But these would likely still 
require simultaneous immunosuppression that is not always effective. In 
addition, immunospuppresive therapy carries with it increased cost, and 
the risk of complications including malignancy and even death.
    Another theoretical solution would be to genetically modify the 
cultured ES cells to make them ``universal donor'' cells. That is, the 
cells would have genes added or genes removed that would ``mask'' the 
foreign nature of the cells, allowing the patient's immune system to 
see the cells as self. While such technologies may be developed in the 
future, it is also possible that these technologies may carry with them 
unacceptably high risks of rejection or other complications that would 
limit their practical utility in clinical practice.
    Given the seriousness of the current shortage of transplantable 
cells and tissues, the FDA has demonstrated a willingness to consider a 
broad array of options including the sourcing of cells and indeed whole 
organs from animals (xenografts) although these sources also pose 
unique problems of histocompatibility. These animal cells do have the 
advantage that they have the potential to be genetically engineered to 
approach the status of ``universal donor'' cells, through genetic 
engineering. However as described above, no simple procedure to confer 
such universal donor status is known. Most such procedures are still 
experimental and would likely continue to require the use of drugs to 
hold off rejection, drugs that add to health care costs, and carry the 
risk of life-threatening complications.
                          therapeutic cloning
    A promising solution to this remaining problem of 
histocompatibility would be to create human ES cells genetically 
identical to the patient. While no ES cells are known to exist in a 
developed human being and are therefore not available for treatment, 
such cells could possibly be obtained through the procedure of somatic 
cell nuclear transfer (NT), otherwise known as cloning technology. In 
this procedure, body cells from a patient would be fused with an egg 
cell that has had its nucleus (including the nuclear DNA) removed. This 
would theoretically allow the production of a blastocyst-staged embryo 
genetically identical to the patient that could, in turn, lead to the 
production of ES cells identical to the patient. In addition, published 
data suggests that the procedure of NT can ``rejuvenate'' an aged cell 
restoring the proliferative capacity inherent in cells at the beginning 
of life. This could lead to cellular therapies with an unprecedented 
opportunity to improve the quality of life for an aging population.
    The use of somatic cell nuclear transfer for the purposes of 
dedifferentiating a patient's cells for purposes of obtaining 
undifferentiated stem cells has been designated ``Therapeutic Cloning'' 
or alternatively, ``Cell Replacement by Nuclear Transfer''. This 
terminology is used to differentiate this clinical indication from the 
use of NT for the cloning of a child which in turn is designated 
``Reproductive Cloning''. In the United Kingdom, the use of NT for 
therapeutic cloning has been carefully studied by their Embryology 
Authority and formally approved by the Parliament.
                         ethical considerations
    Ethical debates often center over two separate lines of reasoning. 
Deontological debates are, by nature, focused on our duty to God or our 
fellow human being. Teleological arguments focus on the question of 
whether the ends justify the means. Most scholars agree that human ES 
cell technology and therapeutic cloning offer great pragmatic merit, 
that is, the teleological arguments in favor of ES and NT technologies 
are quite strong. The lack of agreement, instead, centers on the 
deontological arguments relating to the rights of the blastocyst embryo 
and our duty to protect the individual human life.
    I would argue that the lack of consensus is driven by a lack of 
widespread knowledge of the facts regarding the origins of human life 
on a cellular level and human life on a somatic and individual level. 
So the question of when does life begin, is better phrased ``when does 
an individual human life begin.'' Some dogmatic individuals claim with 
the same certainty the Church opposed Galileo's claim that the earth is 
not the center of the universe, that an individual human life begins 
with the fertilization of the egg cell by the sperm cell. Like previous 
vacuous pronouncements, this is simply not based in fact or, for that 
matter, without basis in religious tradition.
    All strategies to source human cells for the purposes of 
transplantation have their own unique ethical problems. Because 
developing embryonic and fetal cells and tissues are ``young'' and are 
still in the process of forming mature tissues, there has been 
considerable interest in obtaining these tissues for use in human 
medicine. However, the use of aborted embryo or fetal tissue raises 
numerous issues ranging from concerns over increasing the frequency of 
elected abortion to simple issues of maintaining quality controls 
standards in this hypothetical industry. Similarly, obtaining cells and 
tissues from living donors or cadavers is also not without ethical 
issues. For instance, is it morally acceptable to keep ``deceased'' 
individuals on life support for long periods of time in order to 
harvest organs as they are needed?
    The implementation of ES-based technologies could address some of 
the ethical problems described above. First, it is important to note 
that the production of large numbers of human ES cells would not in 
itself cause these same concerns in accessing human embryonic or fetal 
tissue, since the resulting cells have the potential to be grown for 
very long periods of time. Using only a limited number of human embryos 
not used during in vitro fertilization procedures, could supply many 
millions of patients if the problem of histocompatibility could be 
resolved. Second, in the case of NT procedures, the patient may be at 
lower risk of complications in transplant rejection. Third, the only 
human cells used would be from the patient. Theoretically, the need to 
access tissue from other human beings could be reduced.
    Having a knowledge of a means to dramatically improve the delivery 
of health care places a heavy burden on the shoulders of those who 
would actively impede ES and NT technology. The emphasis on the moral 
error of sin by omission is widely reflected in Western tradition 
traceable to Biblical tradition. In Matthew chapter 25 we are told of 
the parable of the master who leaves talents of gold with his servants. 
On servant, for fear of making a mistake with what was given him, 
buries the talent in the ground. This servant, labeled ``wicked and 
slothful'' in the Bible, reminds us, that simple inaction, when we have 
been given a valuable asset, is not just a lack of doing good, but is 
in reality evil. There are times that it is not better to be safe than 
sorry.
    Historically, the United States has a proud history of leading the 
free world in the bold exploration of new technologies. We did not 
hesitate to apply our best minds in an effort to allow a man to touch 
the moon. We were not paralyzed by the fear that like the tower of 
Babel, we were reaching for the heavens. But a far greater challenge 
stands before us. We have been given two talents of gold. The first, 
the human embryonic stem cell, the second, nuclear transfer technology. 
Shall we, like the good steward, take these gifts to mankind and 
courageously use them to the best of our abilities to alleviate the 
suffering of our fellow human being, our will we fail most miserably 
and bury these gifts in the earth? History and many thousands of 
suffering fellow human beings will stand in judgment of our response to 
this great challenge. I urge you to stand courageously in favor of 
existing human life. The alternative is to inherit the wind.

    Senator Harkin. Dr. West, thank you very much.
    Again, thank you all very much for being here today and for 
waiting so patiently. I want to say that all of your written 
statements will be made part of the record in their entirety.
    I just have a couple of questions. Dr. Skirboll, again I 
want to follow up on this question to make sure I understand it 
completely. Does it make any sense to allow Federally funded 
researchers to work with embryonic stem cells already derived, 
but not to allow those researchers to derive the stem cells in 
their own labs?
    Dr. Skirboll. I think if I understand your question, 
Senator, you are asking about Federal funding of derivation 
with that?
    Senator Harkin. Yes.
    Dr. Skirboll. I think there are several things that need to 
be considered with regard to Federal funding for derivation, 
questions that have actually not been thoroughly explored. 
There may well be intellectual property issues with regard to 
patenting and the patenting of the technology that may or may 
not allow for Federal funds to go toward derivation. I am not 
an expert in that arena. I think I suggest that we would need 
to look at that more carefully.
    Senator Harkin. I will ponder that.
    Dr. Hendrix, as the head of FASEB and as a distinguished 
researcher yourself, you know the science. As a federally 
funded researcher, if you decided to do research today using 
embryonic stem cells, but you were limited to using those lines 
now in existence, what problems would that present for you? Are 
there sufficient existing stem cell lines that they offer 
sufficient diversity? Are there patent issues?
    We heard the discussion, you heard the discussion this 
morning, is it 30, is it 50, is it 100, is it 200. Do we have 
any idea at all how many lines that we think might be 
sufficient?
    Ms. Hendrix. Mr. Chairman, in the absence of formal 
oversight, we have no idea right now precisely how many cell 
lines are available for study. My concerns as a scientist--if I 
did not have the opportunity to understand precisely how these 
cell lines were derived, if I did not have the opportunity to 
derive them in my own lab, or if I did not know precisely the 
source of these precious materials, I would have a problem 
conducting an experiment and also interpreting the results of 
an experiment.
    So to specifically answer your question, we do not know how 
many lines are available right now. We have heard there may be 
up to 30 lines available. We have heard from scientists such as 
Dr. Gearhart from Hopkins. He feels that we could use at least 
100 cell lines and that would give us the genetic diversity 
that we would need to look at many different aspects from these 
cells.
    But at this point we really do not know, and that is the 
honest answer. We do not know exactly how many lines are 
available for study and how many we will need to answer our 
current questions and our future questions.
    Senator Harkin. Would there ever be a point in time where 
we might know that? I mean, both Dr. Frist and others said 
earlier today that you do not need thousands and thousands and 
thousands of lines. But is it tens and tens or hundreds and 
hundreds, or what? I mean, how do we finally figure this out?
    Ms. Hendrix. If we were to consult a panel of experts in 
this area right now, the number that people may settle on may 
be a number such as about 100. However, until we actually are 
involved in the science of looking and examining those cell 
lines, we will not know if they are sufficient for all of our 
activities. One possibility where a template currently exists 
is that when scientists are funded by the Federal Government to 
develop research tools such as antibodies or animal models or 
discover new genes, they are obliged because of Federal funding 
to share those resources in publications with all other 
researchers.
    Something we might consider and the panel might consider is 
if this research could be Federally funded, that researchers 
would be allowed to derive lines, and they must share them with 
others. This template already exists for other research tools.
    Senator Harkin. My time has run out. In my second round I 
just want to ask Dr. West a question about therapeutic claims, 
but I will get back to that.
    Senator Specter.
    Senator Specter. Dr. Skirboll, thank you for your work in 
this area. I note in the comment in the report that diseases 
that might be treated by transplanting human cells derived from 
stem cells include Parkinson's disease, diabetes, traumatic 
spinal cord, Duchenne's, muscular dystrophy, heart failure, and 
osteogenesis imperfecta. However, treatment for many of these 
diseases require that human cells be directed to differentiate 
into specific cell types prior to transplantation. The research 
is occurring in several laboratories, but is limited because so 
few laboratories have access to human stem cells.
    That is a pretty flat statement that we need more stem 
cells to be extracted from embryos, right?
    Dr. Skirboll. I think what we were referring to there, 
Senator, was we need more research on stem cell lines to show 
that we can differentiate them into those many cell types that 
would affect those various diseases.
    Senator Specter. Are there adequate stem cells available at 
the present time for NIH to do research without having 
derivation paid for by the Federal Government? Let us come 
right down to the nitty-gritty. I am disappointed that your 
report did not deal with that. That is the crucial issue. The 
last administration said you could pay for them once extracted, 
and legislation is pending to have Federal moneys pay for 
extracting them.
    How about it? Is it not a fact, a good hard cold fact, that 
you need to have Federal funding to get adequate number of stem 
cells from the embryos if this research is to continue?
    Dr. Skirboll. Well, I think it is a fact that we need, as 
Dr. Hendrix just testified, I think it is a fact that we need 
more than the existing cell lines to do all the research that 
is pending. A lot of research could be conducted with the cell 
lines that exist. Let me be clear. But more cell lines by 
genetic diversity. They took the issues in cell lines, we do 
know whether cell lines are different in their ability to 
proliferate, in their ability to differentiate, all of the 
things that matter when we get to treatment.
    Senator Specter. Well, how will there be an adequate number 
of stem cells if we do not get that massive Federal budget that 
this subcommittee has taken the lead on? You only had $12 
billion a couple years ago. Now you have got more than $20 
million.
    Dr. Skirboll. Yes, sir.
    Senator Specter. We did not take the lead in providing that 
research funding for you not to do research, not to be 
scientists. Is there any conceivable excuse for NIH not to 
actively seek to get stem cells and have the Federal funding 
available to extract them from embryos?
    Dr. Skirboll. I think in the order of science, what we need 
access to is the stem cells to do the research. I agree with 
you there, Senator. I do not think NIH at this point has a 
position as to whether Federal funding for the derivation of 
the stem cells in order to get those cells into the 
laboratories of Federal investigators is necessary.
    As I stated to Senator Harkin, I think there are some 
intellectual property and patenting issues that may or may not 
affect the ability of Federal funds to go toward that.
    Senator Specter. We will take care of that. That is a red 
herring. We are not going to have any problem with the 
intellectual property rights. If necessary, we will legislate 
on that subject if they are out there profiteering.
    But the concern I have is that your report is a quasi-
political document when it does not tackle the question of 
Federal funding to extract stem cells from embryos. But I am 
not asking you for a political position. I am asking you a 
scientific question. You are the leader of this report.
    Dr. Skirboll. Yes.
    Senator Specter. It is pretty apparent on the face of it 
that there are not enough stem cells to conduct the research 
necessary, and that we have given you all that funding. Maybe 
we ought to take the funding back if you are not going to use 
it for important scientific purposes.
    Dr. Hendrix is frowning. But we did not give you that 
funding not to use it for important scientific purposes.
    Dr. Skirboll. I see your point, Mr. Specter.
    Senator Specter. Okay. Let me go to a harder point. When 
was this report substantially completed, Dr. Skirboll?
    Dr. Skirboll. We delivered a draft of the report to the 
Secretary of Health and Human Services on the 19 of June, 
slightly less than a month ago.
    Senator Specter. How many changes were made, except for 
editorial comments and punctuation and a little polishing, 
after June 19 when you submitted the report?
    Dr. Skirboll. Well, the report was submitted to the 
Secretary on the 19 of June. It was my understanding it was 
being reviewed in the Office of the Secretary. Only last week 
did I have the opportunity--and I think the Secretary has many 
obligations, many interests--to brief the Secretary on that 
report. Shortly after that briefing----
    Senator Specter. Last week? You submitted it on June 19 and 
you had access to the Secretary during the week of July 9?
    Dr. Skirboll. 3 weeks, 3\1/2\ weeks after the report was 
submitted--you yourself agree it is a long report to review--I 
had an opportunity to brief the Secretary. After that briefing 
and, frankly, because of this hearing, we moved expeditiously, 
my office moved expeditiously, to move that report from its 
draft form, which looked significantly different than this, to 
the final report which is published and handed to you last 
night.
    Senator Specter. Well, was it substantially completed in 
draft form?
    Dr. Skirboll. It was substantially completed in draft form, 
yes, sir.
    Senator Specter. Well, we would like to have a copy of that 
draft report to compare it to what was finally completed. The 
Secretary wrote to me yesterday after I told him that I was 
displeased with his censorship and displeased with his not 
responding to the letter which Senator Harkin and I sent him on 
June 29 and then our efforts to look at the report, where two 
staffers had to look at one report over in your offices. Then I 
wrote him on July 17, and I did not get a copy until 9:45 
yesterday, hardly in a position to be adequately prepared for 
this hearing.
    But we are requesting of you a copy of that draft report, 
because the Secretary has represented to me that it was not 
completed until--let me not have any chance of misquoting. His 
letter to me late yesterday: ``The report was not finalized 
until a few hours before I sent it to you.'' So we want to get 
to the bottom of that, to see what ``finalize'' means. You have 
said it was substantially complete, because we are not going to 
wait forever.
    I know the Secretary is a busy man, and you give it to him 
on June 19 and he cannot take it up until the week of July 9. 
But some of us on this subcommittee have some other 
responsibilities as well. But as I say, we will get to the 
bottom of it.
    But as to your work, I thank you.
    Dr. Skirboll. Thank you, Senator.
    Senator Harkin. Senator Hutchison.
    Senator Hutchison. Thank you, Mr. Chairman.
    I want to continue one of the earlier points that was made 
by Senator Specter, and that is the availability of adult stem 
cells. I am struck, just because of my personal knowledge, that 
we would never be in a position to have enough stem cells 
donated by adults because of the pain involved in extracting 
stem cells.
    It seems to me that when you talk about adult stem cells 
being adequate, which is the argument being made by some, is 
not the availability always going to be in question because of 
the pain involved in extracting stem cells for someone to 
donate just to an unknown? Whereas donating blood is very 
common, very easy, painless, stem cell donations are not so, 
and the number of people willing to donate to a bank are very 
small.
    So you have a situation where a patient does not have an 
appropriate family donor, which is certainly very common. Is 
there a question in your mind about continued availability of 
adult cells in numbers that would be adequate? I would open it 
to anyone who would like to answer. Yes, I would like Dr. 
Doerflinger and then if there is another answer.
    Mr. Doerflinger. Thank you, Senator. Well, Senator, the 
current bone marrow transplantation using stem cells can be an 
arduous process. It involves taking bone marrow from ten 
different places on a person's body and that is a painful 
process. One of the recent advances in the NIH's own stem cell 
biology laboratory is a tremendous advance in culturing very 
small quantities of these bone marrow stem cells to the point 
where they can grow them an order of magnitude, ten times, 
maybe more. They are doing that in primates now and moving 
toward a clinical trial.
    So that a bone marrow transplant, instead of having to use 
almost a liter of material, you only need one injection, one 
site, and a very small vial of material.
    Another particularly promising source of stem cells that 
early indications are may be more versatile than adult cells 
are stem cells from umbilical cord blood and from placentas. 
Four million of each of those is thrown away now without being 
used after live births. There is no moral problem with them and 
they provide a very abundant supply.
    I think one of the cutting edges now of research, though, 
is really going to be stimulating the stem cells that are 
resident in our own bodies to do their job better and to home 
in on sites of damage, in which case you would not need stem 
cell banks at all.
    Senator Hutchison. I would like to ask if there is another 
view, because I would like, in addition to knowing the answer 
to my original question--our Texas legislature has just set up 
a cord blood bank for the State that would do exactly as Dr. 
Doerflinger mentioned. It would be a bank from which anyone 
could come that did not have a proper donor.
    But my question is this: Is that as effective as the stem 
cells from embryos about which we are speaking today? Dr. 
Krause?
    Dr. Krause. I think that the question and the first answer 
both signify a lot of misrepresentation and misunderstanding 
that is going on in the field. ``Stem cell'' is not a 
definition of anything. There needs to be a word in front of 
that what that stem cell is capable of doing. An embryonic stem 
cell is capable of forming any cell in the body. A bone marrow-
derived stem cell until recently we thought only made blood. It 
might have additional possibilities. Cord blood stem cells are 
similar to those from the bone marrow in that we know that they 
can produce blood. We have no idea whether cord blood stem 
cells have the versatility of the bone marrow-derived cells 
that I showed were present or of those that are embryonic stem 
cells. We do not have any idea.
    Cord blood is cord blood. Liver stem cells make liver. Lung 
stem cells make lung. Bone marrow stem cells make blood. The 
versatility that an embryonic stem cell has is unique. However, 
there are recent inklings of data, like that from my 
laboratory, that there might be rare adult-derived cells that 
have some degree of versatility.
    It is not painful to obtain these cells, but we simply do 
not know how to obtain these cells. We have never grown them in 
culture. We do not know how to expand them. Everything is 
unknown. We need embryonic stem cell research because these are 
the experts at versatility to show us what to do with adult-
derived cells. But we are mixing apples and oranges in here to 
say cord blood, bone marrow, and embryo stem cells are all stem 
cells.
    Senator Hutchison. Well, if I could just finish this point, 
do you think there is a lack of availability of adult stem 
cells because of the pain involved at this point in donating? 
Second, is there a possibility that the cord blood could be as 
effective as the embryonic stem cells?
    Dr. Krause. To answer your first question, in terms of 
painful to donate, I think you might be thinking about donating 
your eggs, the gametes, because we do not know where the 
versatile, multipotent adult-derived stem cells live. So we 
cannot say that it is painful to get them. Perhaps they are 
available in the bone marrow, perhaps they are available in 
fat. We just do not know.
    Senator Hutchison. Collecting them from the bone marrow is 
painful.
    Dr. Krause. But we do not know where they are.
    Senator Hutchison. It is very hard to get people to do that 
for an unknown donee, is what I am saying.
    Dr. Skirboll. I think it is important to keep in mind that 
one of the major barriers with adult stem cells is getting them 
to proliferate in vitro so that you can get them to, even if 
they can differentiate into other cell types that might be used 
in therapy, you still have to get enough cells to be used in 
treatment.
    So your point being taken about how many and how many adult 
stem cells you might need in order to get the treatment, 
embryonic stem cells, as I stated earlier, replicate 
indefinitely in culture. The other important thing about stem 
cells taken from bone marrow is that they are not pure, they 
are a population. We do not know--we have not isolated a single 
adult stem cell.
    We get back to this issue of using a stem cell for 
treatment in the heart. You certainly would not want a mixed 
population to be injected into your heart. I do not think the 
FDA would allow that, either. So this issue of purity and 
proliferation are extremely important. We are not there with 
adult stem cells.
    Dr. Krause. So to address your two questions, no, I do not 
think that pain is one of the limitations to obtaining adult-
derived stem cells. It is more that we do not know where they 
are, how do you get them and how to grow them.
    The second thing, in response to your cord blood question, 
is cord blood may have potential to do things other than making 
blood, but we have no idea.
    Senator Hutchison. But you think we should pursue it?
    Dr. Krause. Absolutely.
    Senator Hutchison. In tandem with the other possibilities.
    Thank you, Mr. Chairman.
    Senator Harkin. Thank you, Senator Hutchison.
    I said, Dr. West, I wanted to ask----
    Senator Hutchison. Mr. Chairman, excuse me. I am sorry. 
Could I submit two pieces for the record from Dr. William 
Pierce who is making statements on the adoption of the 
embryonic stem cells? He is the former President of the 
National Council for Adoption, and I would like to submit those 
for the record.
    Senator Harkin. Sure. Without objection, that will be done.
    [The information follows:]

    Prepared Statement of William L. Pierce, Ph.D., Senior Fellow, 
               Discovery Institute for Public Policy \1\

    Because of more than 30 years' experience in the child welfare 
field, 20 of which were as President of the National Council For 
Adoption, perhaps some comments on the current controversy over Federal 
funding for embryonic stem cell research and the recommendation that 
``embryo adoption'' be widely from my vantage point will be helpful.
---------------------------------------------------------------------------
    \1\ This statement does not necessarily reflect the views of 
Discovery Institute. For more information, contact Discovery 
Institute's Washington, D.C., Office at 202-299-0055.
---------------------------------------------------------------------------
    The debate over stem cell research is raging and one of the reasons 
is that in the pluralistic society of the United States there are 
profound theological and religious differences on issues directly 
related to the debate.
    The most important difference of opinion, that surprises many, is 
between those who are identified as ``prolife''--such as Sen. Orrin 
Hatch (R-UT) and Rep. Chris Smith (R-NJ). The difference of opinion 
does not surprise people like Rep. Henry A. Waxman (D-CA), the Ranking 
Member of the House Committee on Government Reform. Rep. Waxman made 
the point clearly at a hearing July 17 on stem cell research before his 
Committee's Subcommittee on Criminal Justice, Drug Policy and Human 
Resources, Chaired by Rep. Mark Souder (R-OH).
    Rep. Waxman said: ``In closing, I want to acknowledge that some 
people do differ in this area. Some people believe that a fertilized 
egg (whether it is inside a womb or inside a test tube) is the same as 
a human being. They also oppose IVF [in vitro fertilization] as it is 
generally practiced, as well as some or all methods of family planning. 
I do not question their sincerity. But I sincerely do not agree.''
    Neither does Sen. Hatch, who said in his testimony July 17 that ``. 
. . I believe that human life begins in the womb, not a petri dish or 
refrigerator.'' Sen. Hatch's views reflect the beliefs of his church, 
the Church of Jesus Christ of Latter-day Saints, also known as Mormons. 
Sen. Hatch's Church does not ``strongly discourage'' IVF as, for 
instance, it does so-called ``surrogate motherhood.'' Mormons leave the 
matter of IVF to the judgment of the husband and wife. Because Mormons 
do strongly discourage surrogate motherhood, it was no surprise when 
Sen. Hatch's statement mentioned that ``. . . the embryo adoption issue 
could raise a whole host of new legal issues. There are also religious 
issues--for example, there are people, some in my own faith, who 
seriously question the notion of surrogate motherhood.''
    Rep. Chris Smith, by contrast, testified that ``These littlest of 
human beings aren't potential life--but life with vast potential.'' 
Rep. Smith's views reflect the beliefs of his church, the Roman 
Catholic Church. The Roman Catholic Church strongly condemns IVF, based 
on the view that human life begins at conception, not implantation.\2\ 
Nor are Roman Catholics alone in this belief. At the press conference 
held by the Family Research Council on July 16, the list of speakers 
was headlined ``LIVING PROOF: HOW CHILDREN'S DRAMATIC STORIES SHOW LIFE 
BEGINS AT FERTILIZATION.'' The remarks of Ken Connor, President of the 
Family Research Council, reflect his religious beliefs. Connor used the 
words ``embryonic human beings'' in his statement and asked the 
question, ``Are the risks to tiny human beings created by man-made 
conception and frozen storage acceptable?''
---------------------------------------------------------------------------
    \2\ Disclosure: the author is an observant, practicing Roman 
Catholic.
---------------------------------------------------------------------------
    As Rep. Waxman pointed out, these differences of belief are at the 
heart of the debate over not just embryonic stem cell research but of 
IVF itself and the ``excess'' human embryos that are routinely produced 
under current IVF protocols. No amount of Congressional testimony or 
research by the National Institutes of Health will settle questions 
that are theological or religious.
    Since science cannot settle for all Americans what is a question of 
religious beliefs, it is clear why one group of legislators and a 
corresponding group of witnesses strongly object to the use of their 
tax dollars to fund experiments on ``tiny human beings,'' in the words 
of the Family Research Council. And it is equally clear why those who 
do not share those religious beliefs are pressing forward with the case 
to spend Federal funds on this research.
    Given the profound differences of opinion about the religious 
content of the controversy, the debate can still take place around 
other issues. At the July 17 hearing, there were strongly contrasting 
viewpoints from the scientists present not only on IVF but on whether 
research needs to go forward at this time on embryonic stem cells and 
whether the U.S. government should be involved in such research.
    The questions on IVF include matters such as the creation of 
``excess'' human embryos and the issue of the length of time these 
embryos can be stored. Testimony to the effect that non-human embryos 
have been frozen for 25 years suggests that the question is not so much 
a matter of ``excess'' but rather of the costs of storage. Perhaps a 
temporary solution to part of the current problem would be to find ways 
to relieve people of the burden of paying seemingly high storage fees, 
so that there is no financial pressure to thaw the embryos. Let's put a 
hold on these frozen embryos since no further apparent harm would be 
done to them in the process.
    IVF techniques themselves could be changed to provide that 
reproductive medical experts no longer extract more ova than can be 
safely fertilized and implanted, without freezing. Let's urge those 
contemplating IVF to take a different approach, one that may impact 
consenting adults rather than embryos who cannot give consent.
    There is no question but that research on embryonic stem cells is 
going to go forward, if not in the United States, then in the United 
Kingdom and other countries. This fact should not stampede U.S. 
policymakers. It is not unique for one country to engage in actions 
that most other countries question, or condemn. One could easily 
recount examples not just from medicine, and not just from the Nazi 
era, but from other arenas of human activity. For instance, despite the 
fact that the United States outlawed human slavery more than 100 years 
ago, as did most nations, we are today confronted with slavery in at 
least one African country, Sudan. The point is: just because some other 
country main gain some purported advantage by engaging in questionable 
activities, that is no reason why U.S. policy should react in blind 
competitive activity. This is a reverse ``brain drain'' that cannot be 
stopped, given the freedom to travel and the nature of free enterprise. 
In the view of some of us, this is no loss because the researchers at 
least will not be conducting questionable experiments in U.S. 
laboratories.
    Such research may even continue in the United States, under private 
funding. Much was made of this issue in the July 17 hearing, as if such 
research is inherently suspect. No such outrage was expressed and no 
major negative results flowed from the competition between Federal 
researchers and privately-funded researchers to decode the human 
genome. This is not to say that ethically illicit research should go 
forward without the strongest appropriate objections being raised, 
whether funded by tax dollars or private sources, but rather to say 
that some of the objections raised appear disingenuous.
    At the July 17 hearing, an alternative for frozen human embryos was 
discussed at length: so-called ``embryo adoption.'' Although there 
seems to be no reason not to move forward with embryo adoption at this 
time, especially given the alternatives, there are many unanswered 
questions which need to be explored. Some of those questions were 
included in a Statement for the Record provided to Chairman Souder's 
staff in advance of the July 17 hearing. Copies of the statement are 
available by email by contacting [email protected].

    Senator Harkin. Dr. West, what you are trying to do--clear 
this up for me. What you are trying to do is to say that, while 
there may be some rejection possibilities of cell lines that 
might be developed for a certain illness, diabetes or whatever, 
that what you are trying to do is to say that by taking the 
person's own cells and--I am searching for that word because I 
am not a scientist--and doing something, that way you match 
them up perfectly.
    Would you tell me again how you differ from what we are 
talking about in terms of embryonic stem cell research.
    Dr. West. Well, there was this discussion that has been 
ongoing about do we have sufficient numbers currently of human 
ESL lines to allow research to go forward. Well, one would be a 
sufficient number to allow some research to move forward. More 
lines would be useful to allow more genetic background and 
diversity. So if there are differences in the genes of all of 
us, we are drawing different genotypes, different kinds of 
genetic backgrounds. That would be of some marginal benefit.
    I think what may be confusing is some people when they are 
talking about large, very large numbers of these lines, they 
are thinking about we might want to bank away maybe 10,000 ESL 
lines to have sufficient genetic diversity so that we might be 
able then, for a patient that is in heart failure, to take cell 
line number 10,001 that most closely matches their own genetic 
background, so that with mild immunosuppression or less severe 
drug therapies they may be able to long-term accept those 
cells. So the idea of making large banks of these cells may be 
driving some of these estimates upwards.
    What I was pointing out is that we really need to grapple 
with this opportunity that has been given to mankind by nuclear 
transfer. It teaches us that, as has been talked about with 
adult stem cells, all cells in the body, given the right 
environment, are completely plastic. We know an egg cell can 
return a cell back to an embryonic state. So you could 
conceivably see a day when we will be able to offer people who 
is the dream of transplantation medicine, to offer a patient of 
any type cells of any kind that are genetically matched to that 
patient so they do not have the risk of long-term 
immunosuppressive therapy, which causes cancer and other 
complications.
    Senator Harkin. So what you do is you just take the cell 
from, say, some cells from my body or cell from my body and you 
would have a somatic cell transfer into the egg, is that right? 
Is that what you do?
    Dr. West. There are a whole host of opportunities here. The 
simple example which I think is easy to point to is that in 
animal cloning we simply take a skin cell and some other cell, 
put it in an egg cell whose DNA has been removed, and we can 
create a whole genetically identical copy of that animal. What 
that teaches you is a simple cell biology lesson. You have 
taken a skin cell back in time, back to this embryonic state, 
from which you could then also make the stem cells.
    Would it have to be done that way? Perhaps. There are other 
opportunities which we could explore at a future point. Nuclear 
transfer points the way to a relatively straightforward path of 
solving transplantation histocompatibility. There may be other 
ways around that.
    Senator Harkin. Senator DeWine, do you have any questions?
    Senator DeWine. Mr. Chairman, thank you very much. I 
apologize, I had another meeting.
    I wonder if I could ask the panel if you could give us some 
idea of how much private funding is currently going into human 
embryonic research. Anybody have any ideas? The question may 
have been asked and I apologize if it was.
    Dr. West. For the private sector, I could speak to some 
extent on that.
    Senator DeWine. Could you maybe kind of break it out into 
how it is being used?
    Dr. West. When you said embryonic research, did you mean 
embryonic stem cell research or embryonic research in general?
    Senator DeWine. I would like both if you could.
    Dr. West. Embryonic research in general, of course, 
emcompasses the whole field of in vitro fertilization.
    Senator DeWine. Sure.
    Dr. West. But on the stem cell front, it is extremely 
small. It is nearly microscopic. There are two companies in the 
United States focused on this, whose total expenditures are in 
the range of probably $10 to $15 million a year in this 
particular area--no, probably more than that.
    Senator DeWine. $10 to $15 million apiece?
    Dr. West. In a year, total.
    Senator DeWine. $10 to $15 million a year.
    Dr. West. In the United States in the private sector. The 
problem of course with that is that it sounds like a 
significant amount of money, but we are looking at a project to 
make hundreds of cell types in the human body. The budget there 
is in the billions of dollars, to adequately address the 
problem.
    Senator DeWine. Anyone else have another comment on that?
    Mr. Doerflinger. I do know, Senator, that one wealthy donor 
recently left a legacy of $58.5 million to one institution, 
Johns Hopkins University, solely to do stem cell research. I 
think they are going to do a great deal of the embryonic 
variety. So there is certainly more than $10 or $15 million out 
there. This was just one institution in the private sector.
    I dare say that if the patient groups and organisms that 
are spending their money on ad campaigns to force all the rest 
of us to pay for this would spend the money instead on the 
research, there would be a lot more available.
    Senator DeWine. I would assume that with the publicity, the 
current public focus on this, that the amount as well as the 
obvious potential for drug companies and other companies' 
research folks to eventually make money, I would assume that 
the amount of money spent on this is going to continue to grow 
very significantly. Does anyone disagree with that?
    Mr. Doerflinger. I think, Senator, that if you are to 
depend--it is going to depend on what research shows. There 
have been some very recent indications that there are more 
problems with embryonic stem cells than people thought, in 
terms of genetic instability and tendency to form tremors, a 
pretty complete failure for the cells to work in diabetes mice. 
They only produced 2 percent of the insulin needed.
    So if those things do not pan out, private investors, who 
are sensible about the use of their money, may pull out of the 
field.
    Senator DeWine. But conversely--we are sitting here. We do 
not know. You are the experts, I am not. But obviously, if the 
opposite is true and it does begin to pan out, we assume the 
money will follow; will it not, at least to some extent it will 
follow.
    Dr. Usala. Senator, my company is a venture company-funded. 
Our evaluation is $20 million at this point. Right now there is 
not a lot of interest in private investment in stem cells. The 
research is far too early to suggest to those that invest in 
this kind of thing that it would make a medical product. To 
make a medical product takes years of basically research, which 
I think all the scientists here are very enthused to get into, 
but on top of that you have to go through the pre-clinical 
testing, the toxicology, everything else.
    Anywhere along the way, there can be a problem that would 
prevent an exciting idea to becoming a medical therapy. So at 
this point I would say there is very little interest in the 
biotech venture community for investing in something like this.
    Senator DeWine. Anybody else?
    Dr. Skirboll. I think it is important to not put the sole 
emphasis here on issues of private funding versus Federal 
funding.
    Senator DeWine. But I am not. I am just, asking just asking 
a question that I did not know the answer to, just a question.
    Anybody else? Yes?
    Dr. Usala. I would say there is far more interest in 
private financing in other forms of tissue regeneration, like 
my company is undergoing, just because those are much closer, a 
lot, to clinical trials. So the prospect for therapies that do 
not have to go through this very long and risky from a 
financial backing point of view, the venture community is much 
more likely to supplement.
    Senator DeWine. Thank you very much.
    Mr. Chairman, thank you very much.
    Senator Harkin. Thank you, Senator DeWine.
    Again, I thank you all very, very much for your efforts on 
this subject, the time and expertise you have given to it. 
Thank you for being so patient this morning. Thank you for all 
your wonderful testimony.

                     ADDITIONAL SUBMITTED STATEMENT

    The subcommittee has received a statement from Senator Kohl 
which will be placed in the record at this point.
    [The statement follows:]

                Prepared Statement of Senator Herb Kohl

    Thank you, Mr. Chairman. I commend you and Senator Specter for 
holding this hearing today, and I want to thank both of you for your 
tireless efforts during the past few years to educate the Senate and 
the public about stem cell research.
    I realize that people on both sides of this debate have strongly 
held beliefs about whether or not embryonic stem cell research should 
go forward. This issue raises ethical and moral questions that cannot 
be taken lightly. During the past several years, I have listened 
carefully to both sides, and I have come to the conclusion that it is 
time for stem cell research to go forward, and it is time for the 
Federal government to play an active role in the conduct and oversight 
of that research.
    Every day, my constituents from Wisconsin write, call, and visit my 
office and describe the terrible and debilitating and life threatening 
diseases they endure. These are families who are suffering from the 
ravages of Alzheimer's Disease, cancers, diabetes, Parkinson's Disease, 
spinal cord injuries and countless others.
    These families rightly demand more Federal help and funding for 
biomedical research. They need cures now, and they want their 
government to pursue every ethical avenue to help them. It would be 
shameful to let these families continue to suffer, while we know full 
well that there is promising new research that could improve or save 
their lives.
    Every year, Congress works toward its goal of doubling funding for 
NIH. This Subcommittee works hard to find the money to do it and I 
support this goal. But if we are serious about finding cures and 
helping our families, then how can we ignore one of the most promising 
avenues of research? How do we explain that to our families?
    Of course, as this research moves ahead, I agree we must also be 
sure that it's conducted appropriately. Currently, stem cell research 
is conducted by privately-funded researchers and there is practically 
no oversight. In recent weeks, we have seen news accounts that raise 
serious ethical and moral questions about the way some stem cell 
research is being conducted. Federal funding and oversight would shed 
light on these practices and provide a framework to make sure stem cell 
research is done in a way that the American public can support.
    I am proud that the early breakthroughs in stem cell research 
happened in my home State at the University of Wisconsin in Madison. It 
is clear to me that we must now go forward nationally, and we must do 
so with the active oversight of the Federal government to make sure 
that stem cell research is conducted in a moral and ethical manner.
    I hope that this hearing will shed additional light on the pressing 
need for stem cell research. And I hope that President Bush comes to 
the same conclusion I did: that helping families with tragic diseases 
is the ethical and moral choice here.

                          SUBCOMMITTEE RECESS

    Senator Harkin. Thank you all very much for being here, 
that concludes our hearing.
    [Whereupon, at 12:18 p.m., Tuesday, July 18, the 
subcommittee was recessed, to reconvene subject to the call of 
the Chair.]









                               STEM CELLS

                              ----------                              


                       WEDNESDAY, AUGUST 1, 2001

                           U.S. Senate,    
    Subcommittee on Labor, Health and Human
     Services, and Education, and Related Agencies,
                               Committee on Appropriations,
                                                    Washington, DC.
    The subcommittee met at 9:30 a.m., in room SD-192, Dirksen 
Senate Office Building, Hon. Arlen Specter presiding.
    Present: Senator Specter.

               OPENING STATEMENT OF SENATOR ARLEN SPECTER

    Senator Specter. Good morning, ladies and gentlemen. The 
hearing of the Appropriations Subcommittee on Labor, Health and 
Human Services, and Education, and Related Agencies will now 
proceed. Senator Harkin, who is chairman, is managing the 
agriculture bill which is on the floor of the Senate, at the 
moment, so I have just been informed that he will not be able 
to be present.
    We may have to abbreviate the hearing to some extent 
because of the agriculture debate. I filed an amendment 
yesterday on an issue regarding dairy compacts, and the floor 
action is always unpredictable when we schedule these hearings. 
As you might suspect, the Senate floor action takes precedence 
over hearings because it involves votes and the disposition of 
legislation. However, we will now proceed, and go as far as we 
can on the hearing.
    We are going to take up today the questions of patent and 
ethical issues relating to human embryonic stem cells. This is 
the ninth hearing which this subcommittee will have held. When 
the news about the embryonic stem cells burst upon the public 
scene in November of 1998, the subcommittee held a hearing very 
promptly thereafter, in early December, and we have had a whole 
series of hearings on this very, very important subject.
    In my judgment, there is no issue before the Congress more 
important than embryonic stem cells and the potential to cure 
or ameliorate diseases for millions of people. I believe that 
the tremendous funding which has been provided for the National 
Institutes of Health, where this subcommittee has taken the 
lead in adding $8.5 billion. We have in the budget an 
additional $3.4 billion more for fiscal year 2002, which will 
in effect double the NIH funding over a 5-year period or fiscal 
year 2003.
    The Department of Health and Human Services general counsel 
has ruled that while Federal funding may not be used to extract 
stem cells from embryos, Federal funding may be used to 
research on stem cells once extracted. Senator Harkin and I 
introduced legislation a long time ago which would free the 
prohibition against limiting Federal funding, and I think that 
is very, very important. There is an enormous momentum, in my 
opinion, in America today to proceed with stem cell research 
because of the phenomenal benefits which have been demonstrated 
on Parkinson's and spinal cord, and the potential on 
Alzheimer's and so many other ailments. The only thing I have 
not heard that stem cells can handle is the common cold, which 
is plaguing me at the moment, as you might have noticed.
STATEMENT OF MARIA FRIERE, Ph.D., DIRECTOR, OFFICE OF 
            TECHNOLOGY TRANSFER, NATIONAL INSTITUTES OF 
            HEALTH, DEPARTMENT OF HEALTH AND HUMAN 
            SERVICES
    Senator Specter. We will proceed now to call Dr. Maria 
Friere, and Dr. Carl Gulbrandsen. Would you step forward? The 
time allotted is 5 minutes, and we would ask you to remain 
within that time frame.
    Dr. Friere is the Director of the Office of Technology 
Transfer at the National Institutes of Health, oversees the 
patenting and licensing activities for NIH, and is responsible 
for the development and implementation of technology transfer 
policies and procedures for the Department of Health and Human 
Services. She has a Ph.D in biophysics from the University of 
Virginia. We thank you for joining us, and we look forward to 
your testimony.
    Dr. Friere. Good morning, Senator. I am here today to 
address how intellectual property considerations affect basic 
science and the future development----
    Senator Specter. Senator Thurmond says, will you pull the 
microphone a little closer?
    Dr. Friere. Are we better now?
    Senator Specter. Yes, you are.
    Dr. Friere. So I am here today to address how intellectual 
property considerations affect basic science and the future 
developments of products for public health. This morning, 
Senator, I will focus on three issues, how technology is 
transferred from the not-for-profit sector to the private 
sector, how this applies in the case of stem cell technology, 
and on the implications for basic research.
    Let me start briefly by discussing how laws enacted over 20 
years ago that encourage university and Government laboratories 
to commercialize their research. These are the Bayh-Dole act 
and the Stevenson-Wydler Innovation Act, including one of its 
amendments, the Federal Technology Transfer Act. In general, 
these laws allow Government laboratories and recipients of 
Government funding to take ownership to their inventions. They 
also impose certain obligations to promote utilization, 
encourage commercialization, and ensure public availability.
    As we will hear in a few minutes, the University of 
Wisconsin provides us with a good example of how the Bayh-Dole 
act is implemented. Early work by Dr. Thomson on nonhuman 
primates such as rhesus monkeys was federally funded. In 
accordance with the law, the invention on the primate stem 
cells was disclosed to the NIH. The university filed a patent 
application with the U.S. Patent & Trademark Office, and the 
technology was licensed to Geron.
    Because Federal funds were used, the Government retains a 
nonexclusive royalty-free license to the technology. This right 
is limited, however, to use by or on behalf of the Government. 
In contrast, when research is funded entirely by the private 
sector, as is the case with research on the human embryonic 
stem cells, the Government has no retained license, and it is 
strictly a private matter whether and under what terms new 
intellectual property is made available to others.
    Let me point out, however, that while some patents may have 
very broad claims, it is usually not the patent that raises 
concern for the biomedical research community, but rather, the 
way in which the patent holder chooses to exercise its rights.
    For example, the discovery may be a research material or a 
new procedure, primarily useful as a means to conduct further 
research. Such discoveries are known as research tools. These 
tools may be patentable, and they have economic value to the 
holder of the patent. In our view, however, the value to 
society is greater when such research tools are widely 
available to scientists. Therein lies the quandary, how best to 
achieve the balance between commercial interest and the public 
interest.
    So how do issues of intellectual property relate to the 
research in stem cells? Well, as you may know, there are 
important patents that have issued on stem cells. I will not go 
into the details of these patents, as in a few minutes we will 
have the opportunity to hear directly from the patent holder. 
In general, however, the ability of scientists to realize the 
potential of this technology will rest in part on how the 
owners of the patents choose to exercise their intellectual 
property rights.
    It is important to note that the NIH has limited authority 
over the patenting and licensing activities of our contractors 
and grantees, as mandated by the Bayh-Dole act. Therefore, with 
regard to the stem cell patents and patent applications, it 
would be most appropriate to address questions to the owners 
and licensees of this technology as to what terms and 
conditions will be required from those who desire to use these 
rights.
    In our view, the license or material transfer agreement can 
be crafted to ensure that both research continue and commercial 
uses are preserved. In fact, the NIH has urged, and will 
continue to urge patent owners and exclusive licensees to 
ensure continuing availability of the technology under terms 
that do not limit basic research or encumber future products.

                           PREPARED STATEMENT

    Mr. Chairman, I am grateful for the opportunity to discuss 
our views, and I would be pleased to answer any of your 
questions.
    [The statement follows:]
                   Prepared Statement of Maria Freire
    Mr. Chairman and members of the Subcommittee, I am Maria Freire, 
Director of the Office of Technology Transfer. I am pleased to appear 
before you today on behalf of the National Institutes of Health to 
address how intellectual property considerations affect basic science 
and the future development of products for public benefit.
    Given the complexity of these issues, it is important to understand 
how the transfer of federally funded technology from the not-for-profit 
sector to the for-profit sector is accomplished. To do so, I will 
briefly discuss the successful process established by Congress in the 
1980's that governs the commercialization of federally funded 
biomedical research.
the bayh-dole act, stevenson-wydler technology innovation act of 1980, 
 and amendments, including the federal technology transfer act of 1986 
                                 (ftta)
    Over twenty years ago, Congress enacted a series of laws that 
encourage government-owned and government-funded research laboratories 
to pursue the commercialization of the results of their research. These 
laws are the Bayh-Dole Act of 1980 and the Stevenson-Wydler Innovation 
Act of 1980, including one of its amendments, the Federal Technology 
Transfer Act of 1986 (FTTA). The Bayh-Dole Act addresses intellectual 
property rights in federally funded grants, contracts and cooperative 
agreements, while Stevenson-Wydler and the FTTA address intellectual 
property of government laboratories. The goal of these laws is to 
promote economic development, enhance U.S. competitiveness and benefit 
the public by encouraging the commercialization of technologies 
developed with federal funding. Generally, these laws allow government 
laboratories and the recipients of government funding to elect to 
retain title to their inventions. They also impose certain obligations: 
promoting utilization, encouraging commercialization and ensuring 
public availability of these technologies.
    I am pleased to say that the goals set by Congress under these laws 
have been achieved and that, in the biomedical arena, the impact of 
these statutes has been critical. In fact, many experts believe that 
the biotechnology industry was spawned from the close interaction 
between academia and industry. It is widely recognized that the Bayh-
Dole Act and the FTTA continue to contribute to the global leadership 
of the U.S. biomedical enterprise, and governments around the world are 
emulating these laws in the hopes of promoting economic development in 
their own nations.
    New products developed under this system benefit patients daily and 
provide hundreds of scientists with the tools required for further 
discovery in support of our public health mission. For example, 
inventions arising from the NIH intramural program alone have 
contributed to over 150 products on the market, including diagnostic 
kits, vaccines, therapeutic drugs and dozens of antibodies, cell lines 
and other research tools. Similarly, the transfer of technology arising 
from the NIH extramural program has contributed significantly to new 
products and fostered economic development.
    To accomplish the transfer of technology, universities have relied 
on authorities granted to them by the Bayh-Dole Act. The Act permits 
the grantee to retain title to intellectual property developed with 
federal funds and to license its rights to for-profit entities. It 
should be noted that, as provided by the Act, the Government does not 
have any direct control over patenting and licensing activities related 
to discoveries resulting from NIH funded research.
    Patents provide the right to exclude others from making, using, 
selling, offering for sale, or importing a new invention for the life 
of the patent. This is society's reward to the owner for teaching 
others how to make and use the invention claimed in the patent. In the 
biomedical field, patents are extremely valuable to companies, 
particularly small companies. They provide a means of securing 
investment income by establishing the company's preeminence in a 
particular area of technology. Parties interested in practicing an 
invention in which they have no ownership may obtain rights to the 
invention by entering into a licensing agreement with the patent owner. 
A license is a contract with binding commitments on each party, usually 
involving compensation. A license does not grant title to the 
invention. Licenses can be exclusive, when only one licensee is 
permitted to benefit from the use of the technology, or non-exclusive, 
when more than one licensee is permitted to benefit from such rights.
    As this Subcommittee well knows, new drugs and vaccines are costly 
to develop, and companies are unlikely to invest in further research 
and development without some promise of future product exclusivity. 
When Congress gave federal grantees the ability to patent and 
exclusively license government-funded inventions, the private sector 
turned its attention toward publicly supported research as a new source 
of potential products. The value to the public resides in the 
generation of new drugs, vaccines, and medical devices. These 
activities have also stimulated economic development and the creation 
of new jobs in the United States.
    Whenever federal funds are used to support a new discovery by 
contractors and grantees, the government has a non-exclusive, royalty-
free right to use the patented technology by or on behalf of the 
government. This would allow the government laboratories and 
contractors the right to use the patented technology for further 
research. In addition, in dealing with this invention the federal 
grantee or contractor must ensure that the goals of the Bayh-Dole Act--
utilization, commercialization, and public availability--are 
implemented.
    When research is funded entirely by the private sector, the 
government has no statutory license, and it is strictly a private 
matter whether, and under what terms, new intellectual property is made 
available to others for commercial or research purposes.
    Mr. Chairman, as we have discussed with this Subcommittee before, 
it is not merely the existence of a patent that raises concern for the 
biomedical research community. The concern mostly arises when the 
patent holder chooses to exercise its rights through licensing or other 
contractual agreements in a manner inconsistent with the advancement of 
basic research. For example, many new inventions are not final 
products. The discovery may be a research material or a new method or 
procedure, primarily useful as the means to conduct further research. 
Such discoveries are commonly known as research tools. There is little 
doubt that many research tools may be patentable and that they are of 
economic value to the holder of these rights. There is also little 
doubt that the value to society is greatest when such research tools 
are made widely available to scientists.
    For example, a license that provides complete exclusivity to a 
technology that is also a research tool may result in some product 
development in the short-term, but it will close off opportunities to 
advance science and develop other products in the long-term. The only 
way to maximize the benefit to the public is to ensure that both 
research use and the potential for commercial development are 
preserved.
    The professionals working in the specialized field of biomedical 
licensing strive to promote a balance between commercial interests and 
the public interest. In those instances where a research tool can also 
become a therapeutic product, licenses can be, and are, carefully 
crafted by scope, application and field to allow use by the research 
community without destroying a company's commercial incentive to 
develop the product.
    Careful licensing that preserves this balance, however, has not 
always been the case. The NIH has been concerned for some time about 
the potential adverse effects of restrictive licensing practices on 
access to research tools. In response to concerns from the scientific 
community, NIH has published guidelines on the sharing of biomedical 
research resources entitled ``Principles and Guidelines for Recipients 
of NIH Research Grants and Contracts.'' This document helps ensure open 
sharing of research tools to maintain the robust research enterprise in 
this country.
                           stem cell research
    How do issues regarding intellectual property considerations relate 
to research on stem cells? The issuance of patents by the U.S. Patent 
and Trademark Office may not necessarily have an adverse effect on 
continuing research and often promotes the development of new 
therapeutics, diagnostics and research tools, including cells. If 
patent owners devise a licensing and sharing strategy that will allow 
basic research to continue unencumbered while preserving appropriate 
commercial value, they will help ensure that such research tools are 
broadly available to the research community. The terms and conditions 
on the use and the transfer of material, such as these cells, would be 
set forth in an agreement commonly called a Material Transfer 
Agreement, or MTA.
    MTAs are vehicles used to transfer proprietary materials between 
and among the for-profit and not-for-profit sectors. Most MTAs are 
simple, 1- to 2-page agreements. MTAs can, however, impose obligations 
or restrictions that can stifle the broad dissemination of new 
discoveries, slow the technology transfer process and limit future 
avenues of research and product development. Examples of such 
obligations include so-called ``reach-through'' provisions that may: 
(1) give the provider of a material ownership of new inventions 
developed by the recipient; (2) require royalty payments by the 
recipient to the provider on inventions discovered by the recipient 
that are not covered by the provider's patent; or, (3) require the 
recipient to give the provider an option to exclusive rights to any new 
intellectual property arising from recipient's use of the material.
    NIH's experience has shown, however, that conditions imposed by 
patent owners--whether in a license or an MTA--can be crafted to ensure 
both research uses and commercial development. For example, the NIH 
strategy is to negotiate non-exclusive licenses for its intramural 
technologies whenever possible. This allows more than one company to 
develop products using a particular technology, products that may 
ultimately compete with each other in the marketplace. We recognize 
that companies need an exclusive market to offset the risk, time, and 
expense of developing biomedical diagnostic or therapeutic products. 
However, companies do not necessarily need to achieve that position 
solely by exclusively licensing a government technology used to develop 
the product. Instead, companies are frequently able to add their own 
proprietary technologies to the invention licensed from the government 
to ultimately achieve some level of uniqueness and exclusivity for the 
final product.
    If non-exclusive licensing does not provide enough incentive for 
the company to develop a product, and it often does not for a potential 
therapeutic application, NIH will award exclusivity for specific 
indications or fields of use, based on the license applicant's 
commercial development plans at the time of the application. NIH also 
provides for exclusive licensees to grant sublicenses to broaden the 
development possibilities when necessary for the public health. 
Finally, NIH insists on the continuing unencumbered availability of the 
licensed technology to the not-for-profit scientific community for 
further research.
    Experience over the last 20 years has shown that to maximize public 
health benefit, the balance between exclusivity and access must be 
carefully maintained, and research uses of new technologies must be 
preserved. These concepts form the basis for the licensing policies of 
the NIH, as well as for the proposed guidelines for our grantees 
mentioned above.
    Over the past few years, NIH has, however, faced situations in 
which the patent holder was willing to allow basic research to continue 
only under terms that were inconsistent with our research tools 
principles and guidelines. To resolve such a situation, NIH negotiated 
acceptable terms with the patent holder so that scientists could 
continue to use the tools for basic research while protecting the 
company's commercial rights. In fact, as part of these agreements, the 
terms and conditions were extended to NIH grantees, should they choose 
to take advantage of these more favorable terms.
    The experience since the inception of the guidelines has been that, 
while the process between not-for-profit entities has been streamlined, 
there is still work to be done when transfer of tools happens between 
the for-profit and not-for-profit sectors. We are hopeful that 
continued dialogue between these two constituencies will eventually 
result in an understanding that would permit the research enterprise to 
continue to flourish without undue impediments.
    Finally, it is important to remind the Subcommittee that the NIH 
has limited authority over the patenting and licensing activities of 
our contractors and grantees. Therefore, with regard to the stem cell 
patents and patent applications, it would be appropriate to address 
questions to the owners and licensees of this technology as to what 
conditions they may apply to those who desire to use the intellectual 
property.
                                summary
    Congress has enacted legislation for recipients of federal funding 
that encourages the utilization, commercialization and public 
availability of federally funded inventions. Grantees and contractors 
have exercised the broad discretion awarded them by the law and sought 
to achieve these goals through the patenting and licensing of new 
inventions that arise through the use of federal funds. The government 
has only limited authorities over these activities. If the research is 
entirely funded by the private sector, the government has no statutory 
license and is not involved in patenting or licensing decisions. 
Exclusive licensing, without regard to research uses, can impede rather 
than enhance utilization and public availability of certain types of 
inventions, such as research tools. Strategic licensing can alleviate 
potential problems. Indeed, many grantees provide for the continuing 
availability of exclusively licensed subject matter to researchers in 
order to ensure progress of biomedical research. The NIH has urged, and 
will continue to urge, patent owners and exclusive licensees to ensure 
continuing availability of a technology under terms that do not limit 
basic research or encumber future products. The key to the use of a 
technology is in the manner in which holders of existing patents 
exercise their rights through licensing and other contractual 
agreements.
    Mr. Chairman, thank you for the opportunity to provide this broad 
background on the effects of patents and licenses on the advancement of 
science and medicine.

    Senator Specter. Thank you very much, Dr. Friere. We will 
have some questions, but first we will proceed to Dr. 
Gulbrandsen, managing director of the Wisconsin Alumni Research 
Foundation and president of WiCell Research Institute, a 
nonprofit subsidiary.
    He received his bachelor's from St. Olaf, and J.D. from the 
University of Wisconsin, and Ph.D. in psychology from the 
University of Wisconsin Madison. Thank you very much for 
joining us, Dr. Gulbrandsen, we look forward to your testimony.
STATEMENT OF CARL GULBRANDSEN, Ph.D., MANAGING 
            DIRECTOR, WISCONSIN ALUMNI RESEARCH 
            FOUNDATION, PRESIDENT, WICELL RESEARCH 
            INSTITUTE, INC.
    Dr. Gulbrandsen. Thank you, Mr. Chairman.
    Mr. Chairman and members of the subcommittee, I am pleased 
to appear before you to discuss the role of the Wisconsin 
Alumni Research Foundation, or WARF, and the WiCell Research 
Institute in supporting the important research necessary to 
move the science of embryonic stem cells forward.
    I want to thank Chairman Harkin and Senator Specter for 
their vision and commitment to the ES cell research. Your work 
on this issue has given hope to millions of Americans who 
suffer from degenerative diseases and has also provided 
inspiration for researchers in the laboratory.
    WARF is an independent, nonprofit supporting organization 
for the University of Wisconsin Madison, and is the patent 
management organization also for the University of Wisconsin 
Madison. From its inception, WARF's mission has been to support 
scientific research at the university for the benefit of the 
university and the public.
    WARF carries out its mission by licensing university 
inventions and returning the proceeds to fund further research 
at the university, but equally important to the money returned 
to the university is the fact that throughout its existence the 
inventions licensed by WARF have functioned to improve the 
condition of mankind.
    WARF owns two issued patents covering primate embryonic 
stem cells, and has other applications pending. The first 
patent covers primate embryonic stem cells, including the human 
ES cell, and the method for deriving and maintaining those 
cells. The research underlying that patent includes research on 
nonhuman primates funded by NIH, and the patent carries the 
appropriate notice that the Federal Government has an interest 
in that patent. The second patent is specific to the human ES 
cell, and the research underlying that patent is entirely 
privately funded.
    WARF has never used its patents to block research, and does 
not intend to do so with embryonic stem cell patents. WARF has 
granted both research and commercial licenses under its 
patents, and will continue to do so under terms that are fair 
to all concerned, including the general public. Our goal is to 
facilitate research and development of stem cell technology, 
not impede it. In that regard, WARF supports legislation to 
permit derivation of new cell lines, and would welcome the 
opportunity to license centers for derivation of ES cells under 
WARF's patents.
    From the beginning, the distribution of the ES cells for 
research purposes was of critical importance to the university 
and WARF. In light of the complication of Federal law, the 
university asked WARF to establish a privately funded institute 
to support the distribution of and research on human ES cells.
    In response to the university's request, WARF formed WiCell 
Research Institute as a nonprofit subsidiary of WARF. Dr. James 
Thomson is the scientific director of WARF, and Dr. Nancy 
Black, who is here with me, is the manager of WiCell. Excuse 
me, I misspoke. Dr. James Thomson is the scientific director of 
WiCell.
    WiCell distributes human embryonic stem cells under a 
material transfer and license agreement. The fee charged to the 
researcher for the license is returned to the institute and 
helps defray but does not cover the cost associated with making 
embryonic stem cells widely available. The WiCell agreement 
provides a research license. Researchers are free to publish 
and patent their discoveries.
    If the researcher or her institution wishes to 
commercialize a discovery made under the agreement, an 
appropriate commercial license may be required. It may be that 
such commercial rights must be obtained from WARF, or from 
WARF's licensee, Geron. If such rights are not required, the 
researcher or her institution may commercialize the discovery 
as they choose.
    Geron is one of two commercial licensees under WARF's 
embryonic stem cell patents. Geron was instrumental in 
providing needed funding for the human embryonic stem cell 
research that led to the Thomson discovery. In return for that 
funding, Geron was granted certain exclusive and nonexclusive 
rights under the patents. Geron's exclusive rights are limited, 
are in the diagnostic and therapeutic fields, limited to six 
defined cell types.
    WARF's license agreement with Geron contains development 
requirements. The development requirements give Geron the right 
to terminate the license granted to a commercial partner for 
lack of active development. To maintain its rights, Geron must 
develop commercially viable products.
    It should be noted that Geron has no right to inventions 
made by researchers who receive ES cells from WiCell. Geron 
also has no right to distribute ES cells to third parties not 
associated with Geron. That right has been reserved for WiCell.
    WiCell currently has one other commercial licensee who has 
licensed the right to conduct internal research under the ES 
patents and use the human ES cells. That licensee, Bresagen, 
Limited, has recently announced the derivation of four 
additional cell lines under license from WiCell.

                           PREPARED STATEMENT

    WARF, WiCell, and the UW Madison believe in and are excited 
about the future of medicine utilizing the ES cell technology. 
Our goal is to see this technology widely disseminated and 
developed. We believe that our licensing practices reflect that 
goal. We hope that Federal funding will increase the number of 
researchers who work with ES cells, and that these researchers 
will bring the tomorrow of medicine closer today.
    Thank you.
    [The statement follows:]
                 Prepared Statement of Carl Gulbrandsen
                              introduction
    Mr. Chairman and members of the Subcommittee, I am pleased to 
appear before you to discuss the role of the Wisconsin Alumni Research 
Foundation (``WARF'') and the WiCell Research Institute (``WiCell'') in 
supporting the important research necessary to move the science of 
Embryonic Stem Cells (``ES Cells'') forward. Virtually every realm of 
medicine might be touched by this innovation. Because of this enormous 
promise, WARF and WiCell are committed to proceeding with this research 
and to providing scientists with ES Cells. Because of concerns over 
derivation and use of ES Cells, WARF and WiCell feel a special 
responsibility to conduct our mission ethically and legally. The 
absence of NIH funding and other federal restrictions are slowing the 
progress of research that could alleviate a great deal of human 
suffering.
    I want to thank Chairman Harkin and Senator Specter for their 
vision and commitment to human ES Cell research. Your work on this 
issue has given hope to the millions of Americans who suffer from 
Parkinson's, diabetes, spinal cord injuries and heart disease. Your 
tireless efforts also provide inspiration for researchers in the 
laboratory attempting to turn the potential of ES Cells into real 
treatments and cures for diseases that until now were thought to be 
untreatable. Dr. James A. Thomson from the University of Wisconsin-
Madison (the ``UW-Madison''), the first researcher to isolate human ES 
Cells, testified at your first hearing and detailed the method of 
deriving the ES Cells and the tremendous potential of these cells for 
studying human development and birth defects. It is an honor to speak 
to the Committee today about the efforts of WARF and WiCell to promote 
ES Cell research and to make ES Cells widely available to researchers 
across the United States and throughout the world.
                     background on warf and wicell
    The Wisconsin Alumni Research Foundation is an independent, non-
profit, supporting organization for the University of Wisconsin-Madison 
(UW-Madison). Recently, WARF celebrated its 75th anniversary. From its 
inception, WARF's mission has been to support scientific research at 
the UW-Madison for the benefit of the UW-Madison and the public. WARF 
carries out its mission by licensing university inventions and 
returning the proceeds to fund further research at UW-Madison. But 
equally important to the money returned to the UW-Madison is the fact 
that throughout its existence, the inventions licensed by WARF have 
improved the condition of humankind. WARF's licensed inventions have 
cured rickets, treated bone disease and psoriasis; Warfarin or coumadin 
was patented and licensed by WARF and is still the major therapeutic 
blood thinner for vascular disease and stroke prevention; the UW 
solution, another WARF invention, is widely used to preserve organs for 
transplant; and most recently human embryonic stem cells promises 
regenerative therapies for previously untreatable conditions.
    The UW-Madison is unique in its policies regarding faculty 
ownership of intellectual property. Researchers at UW-Madison are 
presumed to own the intellectual property rights to any invention 
discovered during the course of their employment, barring an obligation 
to assign the invention because of federal or industrial funding. If 
federal funding is used in the conception or reduction to practice of 
an invention, the researcher is required to disclose the invention to 
the UW-Madison and the provisions of the Bayh-Dole Act apply (35 U.S.C. 
200 et. seq.). In that case, WARF is the designated patent management 
organization for UW-Madison and receives assignment of any federally 
funded invention. It is important to note that federal funds were not 
involved in the work of Dr. Thomson when he made the critical ES Cell 
breakthrough in 1998. In fact, federal law prohibited NIH funding of 
research on embryos, even those in excess of clinical need after in 
vitro fertility treatment.
    Dr. Thomson, therefore, was not required to assign his invention to 
WARF. He could have patented the stem cell discovery in his own name 
and sold all the rights to a private company. It is a testament to Dr. 
Thomson that he voluntarily brought this important technology to us so 
that his discovery would be patented and licensed in a manner that 
would provide for wide distribution of the ES Cell and insure that the 
highest of ethical standards were followed.
    From the beginning, the distribution of the ES Cell for research 
purposes was of critical importance to UW-Madison, WARF, and WiCell. In 
light of the complications of federal law, the UW-Madison asked WARF to 
support a laboratory that was free of federal funds to support 
distribution and research on ES Cells. In response to the UW-Madison's 
request, WARF formed the WiCell Research Institute as a non-profit 
subsidiary in October of 1999 to advance ES Cell research. WiCell's 
mission is two fold: (1) to provide ES Cells for research purposes and 
(2) to engage in research in the human ES cells utilizing the expertise 
of the UW-Madison community. Dr. Thomson is the Scientific Director of 
WiCell and Dr. Nancy Block is the General Manager.
    To date, WARF has spent well over a $1 million to establish WiCell 
and for maintenance and distribution of the ES Cells. WiCell has 
already outgrown its physical facility and has plans for a five fold 
increase in lab space. This increase is necessary because of the 
tremendous interest by researchers at UW-Madison in working with ES 
Cells. WiCell exists because of the federal prohibition on embryo 
research. The federal prohibition has made it necessary to duplicate 
space, equipment, personnel and utilities to carry out ES Cell 
research. Without such a prohibition UW-Madison would perform WiCell's 
research functions.
    Recently, Dr. Roger Pederson, an early collaborator of Dr. Thomson, 
announced that he is leaving the University of California-San Francisco 
(``UCSF'') for Great Britain due to the difficulties associated with 
performing ES Cell research in the United States. This happened only 
after UCSF tried unsuccessfully to accommodate federal restrictions 
within its campus. Who knows how many other investigators may follow 
that lead or may be reluctant to pursue a career in ES Cell research.
    The Committee should know that the ES Cells that are currently 
available from WiCell do not meet the guidelines in the proposed NIH 
regulations. The present cell lines were derived from excess embryos 
that were created for reproductive purposes and not for purposes of 
research. They were, however, derived well before the guidelines were 
published. The current ES Cell lines do meet the spirit of the 
regulations but not the technical letter. WARF and WiCell are committed 
to deriving additional lines that will meet the NIH guidelines but we 
cannot promise a date by which new cell lines will be available.
                            patent position
    WARF has never used its patents to block research and does not 
intend to do so with its stem cell patents. WARF is the owner by 
assignment from the sole inventor, Dr. Thomson, of two issued patents 
in the area of ES Cells. The first, U.S. Patent No. 5,843,780, (the 
``'780 Patent'') issued December 1, 1998 with claims to primate ES 
Cells. The '780 Patent covers any primate ES cells (including humans as 
primates) which: (i) are stable and capable of proliferation in 
culture, (ii) maintain a normal karyotype containing all chromosomes 
characteristic of the primate species, (iii) maintain the potential to 
differentiate into all three embryonic tissue types (endoderm, 
mesoderm, and ectoderm tissues), and (iv) will not differentiate when 
cultured on a fibroblast feeder layer. This is characteristic of all 
normal ES Cells. In addition, the '780 Patent covers a method of 
isolating an ES Cell comprised of the current known method for 
culturing ES Cells.
    The second patent, U.S. Patent No. 6,200,806 (the ``'806'' Patent) 
issued March 13, 2001 and is a divisional of the '780 Patent 
(collectively the ``ES Cell Patents''). The '806 Patent covers any ES 
cells with the same characteristics of those claimed in the '780 Patent 
but it specifically claims human ES Cells. Also, the '806 Patent claims 
the method for culturing ES Cells claimed in the '708 Patent but it 
again specifically claims the method for culturing human ES Cells.
    It is anecdotally interesting to note that while WARF's ES Cell 
Patents currently cover the most basic characteristics and the current 
method of culturing ES Cells, a cursory search of patent databases 
reveals that there are 95 issued patents in the United States with 
``stem cells'' in their title and there are at least 638 U.S. patents 
that incorporate ``stem cells'' somewhere in their claims. In addition, 
there are hundreds of additional related issued patents covering cell 
culture, growth factors, extra cellular matrices and cell 
differentiation. This is no different than the patent landscape in any 
other biomedical area of research. There is patent protection in almost 
every facet of our lives, from the cereal consumed in the morning, to 
the alarm clock set before bed at night. Scientific innovation in the 
United States is advancing faster than it ever has. Patents facilitate 
and enable this advance.
    WARF does not believe the ES Cell Patents are a disincentive for 
researchers in any respect. WARF and WiCell are committed to making the 
ES Cells and licenses under the ES Cell Patents widely available to 
academic researchers and industry.
                dissemination of es cells to researchers
    WARF, through its subsidiary WiCell, is committed to making the ES 
Cells widely available to researchers, both academic and commercial, 
throughout the world. If federal funding is provided for ES Cell 
research, the process of distribution would continue at WiCell 
unchanged from present practice. WARF supports legislation to permit 
derivation of new cell lines and would welcome the opportunity to 
license centers for derivation under WARF's ES Cell Patents.
    WiCell provides ES Cells for research purposes under its standard 
Materials Transfer & License Agreement (``MTA''). The MTA contains 
research restrictions as provided in the NIH ES Cell guidelines 
published last August and from the University of Wisconsin-Madison 
Bioethics Committee. The MTA requires a research plan and yearly 
reporting to enable WiCell to monitor proper use of the ES Cells. 
Researchers entering into the MTA are provided frozen ES Cells in two 
vials sent along with protocols to assist the researcher in culturing 
the ES Cells.
    To date, WiCell has about 30 MTAs executed for the human ES Cells 
with academic and nonprofit research institutions. Currently, WiCell 
has approximately 100 additional MTAs in various stages of negotiation. 
WiCell anticipates the number of requests for human ES Cells will 
increase significantly when federally funded scientists are permitted 
to conduct research using the human ES Cells.
    The MTA for ES Cells allows researchers to do research and patent 
and publish any discovery made using the ES Cells. In order to 
accomplish the goal of making the ES Cells widely available at a low 
cost to researchers without prolonged negotiation, WiCell provides its 
standard MTA for research use only. Commercialization requires an 
appropriate commercial license. That license may be obtained from 
WiCell, WARF or, in areas for which Geron has rights, from Geron. The 
arrangement separating research and commercial licenses allows WiCell 
to grant research licenses at a price below WiCell's cost of producing 
ES Cells lines.
                  geron and other commercial licensees
    WARF has two license agreements with commercial entities. The 
primary license is to Geron Corporation. The license agreement grants 
exclusive rights to therapeutic and diagnostic products for six cell 
types. WARF's license agreement with Geron contains development 
requirements. The development requirements give WARF the right to 
terminate the license granted to a commercial partner for lack of 
active development. To maintain its rights, Geron must develop 
commercially viable products. These products are those that so many 
hope will increase the capacity to cure conditions that are presently 
incurable. WARF believes that the development requirements are 
significant in pushing this vital technology forward for the benefit of 
the public.
    There has been a suggestion and a concern that research conducted 
in an area that Geron has licensed exclusively only benefits Geron. 
WARF believes this suggestion is not valid but appreciates the concern. 
First, it should be emphasized that Geron has no rights to inventions 
made by researchers who receive ES Cells from WiCell. Second, as stated 
previously, such researchers or their institutions are free to patent 
inventions arising from research using the ES Cells. It may be that to 
commercialize an invention, a researcher will need to obtain rights 
from Geron. However, the converse is also true. If Geron wants to use 
an invention created through such research, Geron will have to license 
those rights from the researcher--presumably under reasonable 
commercial terms.
    WiCell currently has only one other commercial licensee who has 
licensed the right to conduct internal research under the ES Cell 
Patents and using the human ES Cells. This licensee, Bresagen, LTD., 
has recently announced the deriviation of four additional cell lines 
under license from WiCell.
    WiCell has and will continue to make research licenses on ES Cells 
available. Commercial licenses to make research, therapeutic and 
diagnostic products in any cell type other than Geron's six cell types 
are also available. If a company with a research license discovers a 
therapeutic or diagnostic product in the area of one of Geron's six 
cell types, the licensee may need a license from Geron to commercialize 
the invention. Geron has the right to sublicense in its areas of 
exclusivity and Geron has assured WARF and WiCell that it will do so on 
reasonable commercial terms.
    WARF, WiCell and the UW-Madison believe in and are excited about 
the future of medicine utilizing ES Cell technology. Our goal is to see 
this technology widely disseminated and developed. We believe that our 
licensing practices reflect this goal. We hope that federal funding 
will increase the number of researchers who work with ES Cells and that 
these researchers will bring the tomorrow of medicine closer to today.

    Senator Specter. Thank you, Dr. Gulbrandsen.
    Dr. Gulbrandsen, how many entities, if you know, own 
patents on stem cells?
    Dr. Gulbrandsen. We did a preliminary search just recently, 
and I can tell you that I think there are over 65 patents that 
have stem cells in the title. The number of entities I think 
that are doing work in the stem cell area are at least in the 
half-dozen to dozen range in the United States.
    Senator Specter. 65 patents and research entities in the 
half-dozen to dozen range, you say?
    Dr. Gulbrandsen. Yes.
    Senator Specter. Is there any limitation on what someone 
may acquire by way of patent when you have something so 
fundamental to medical research, and with such widespread 
application for humanity, to have it available as a restricted 
property right, an intellectual property right?
    Dr. Gulbrandsen. Well, I think the limitation under the 
patent law is that it be described in such a way that somebody 
can make and use it, and it has to be distinctly claimed.
    Senator Specter. Someone can make it, use it, and what?
    Dr. Gulbrandsen. It has to be described sufficiently in the 
application that somebody can duplicate that invention, that 
they can make and use it, and then the claims are really 
limited to what is described in the application.
    Senator Specter. So there is no limitation as to what kind 
of intellectual property right may be obtained through a 
patent, even if it has enormous implications for society, human 
welfare?
    Dr. Gulbrandsen. Well, under the patent law there have been 
limitations that have brought into play where the public 
interest is really in jeopardy.
    Senator Specter. What are those limitations as you--let me 
finish the question.
    Dr. Gulbrandsen. I am sorry.
    Senator Specter. As you articulate it, where the public 
interest is in question?
    Dr. Gulbrandsen. My recollection--and I teach patent law at 
the University of Wisconsin Madison. My recollection is----
    Senator Specter. You teach patent law?
    Dr. Gulbrandsen. Yes--is that there is one case. In the 
Milwaukee sewage case--and this has to do not with what the 
Federal Government, or what the patent office issues as a 
patent, but whether the courts will enforce a patent, and they 
of course do have the right to not enforce a patent if, in the 
interest of public health and safety, enforcing that patent 
would jeopardize those interests, so in the Milwaukee sewage 
case, enforcing the patent meant that sewage was going to be 
dumped into Lake Michigan, and in that case the court would not 
enforce the patent.
    Senator Specter. Do you know what court that was?
    Dr. Gulbrandsen. I think it was the Eastern District Court 
in Wisconsin.
    Senator Specter. Well, do you think that kind of a 
generalization, that patent rights would be limited where 
public interest is involved, would be applicable where you have 
such enormous potential on the stem cells for alleviating so 
many diseases?
    Dr. Gulbrandsen. I think--certainly I am biased, Senator. I 
think that in the stem cell area that the opportunity for 
bringing these products to market to benefit mankind is going 
to be enhanced with the fact that WARF has patented this and is 
working very hard to distribute these cells to others to do 
research and that you do have an entity that is committed to 
serving the public good holding these patents.
    Senator Specter. Well, intellectual property rights are 
very fundamental in our society, beyond any question. They are 
provided for in the Constitution. Where you have such an 
unusual situation as patents on stem cells--and the 
ramifications are very complicated. I do not know that they are 
yet fully understood as to where you are going, and I 
appreciate your declaration of bias. You represent a patent 
holder. You have a right to be biased.
    This subcommittee is concerned about how these techniques 
will be applied to cure diseases, and we would be looking for 
an exception that you state, where there is a public interest, 
and it may not be necessary, if there is licensing, and if 
there is an open attitude on the part of the patent holders to 
permit others to utilize them for the public interest, but if a 
tight hold is maintained for the profit motive, that is another 
matter.
    What is your view on the issue, Dr. Friere, about limiting 
patent rights where there is an overwhelming, supervening 
public interest?
    Dr. Friere. Well, Senator, that is of course a critical 
question and one that, as a public agency, we really take very 
seriously. We do sometimes, in fact, in the United States have 
patents issued that are very broad in scope, and have 
tremendous implications for these kinds of technologies.
    In my experience, we have been able to work through by 
material transfer agreements or licensing agreements the issues 
of intellectual property such that you are able to maintain the 
research balance and the commercial balance. It is my hope----
    Senator Specter. That has been your experience, but you 
have not had anything as dramatic as stem cells, have you?
    Dr. Friere. No, not in stem cells.
    Senator Specter. You did not look like you were old enough 
to have had experience with something as dramatic as stem 
cells.
    Dr. Friere. No. We have had some experience, for example, 
with in vivo/ex vivo gene therapy in which we were issued a 
very broad claim on gene therapy, and that technology was 
licensed very broadly, after--it was licensed exclusively by 
us, and then it was made available very broadly to companies, 
so the system does have checks and balances in place, but you 
are correct, I have not had to deal with stem cells.
    Senator Specter. Dr. Gulbrandsen, I have been advised by 
staff that any researcher who derives human embryonic stem 
cells in his lab would be infringing on WARF's patent. Is that 
true?
    Dr. Gulbrandsen. Yes.
    Senator Specter. I am further advised that WARF has not 
indicated that it will license the right to derive stem cells 
in other labs, is that true?
    Dr. Gulbrandsen. No. It has indicated in both what I have 
said here this morning and in our written statement that we 
would be happy to license people to derive stem cells.
    Senator Specter. Have you licensed people to do that?
    Dr. Gulbrandsen. Yes.
    Senator Specter. At what cost?
    Dr. Gulbrandsen. This was a commercial entity, and the cost 
is proprietary.
    Senator Specter. The cost is what?
    Dr. Gulbrandsen. The cost is a confidential matter between 
WARF and the commercial----
    Senator Specter. Is there just one license which has been 
made available?
    Dr. Gulbrandsen. At this date there are two commercial 
licenses.
    Senator Specter. Two commercial licenses where WARF has 
been paid for the licensing rights?
    Dr. Gulbrandsen. That is right.
    Senator Specter. To what extent has NIH research been 
involved, Dr. Friere, which could be linked to the benefits or 
to the patents which WARF has obtained?
    Dr. Friere. The original primate patent with the rhesus 
monkeys work by Dr. Thomson was federally funded, and we do 
have some rights to that original patent, as Dr. Gulbrandsen 
mentioned.
    Senator Specter. Has Dr. Thomson--specify the relationship, 
if any, between Dr. Thomson and WARF.
    Dr. Friere. Dr. Thomson is--well, I should actually have 
you answer the question, if he is one of your researchers.
    Dr. Gulbrandsen. Dr. Thomson is a faculty member at the 
University of Wisconsin Madison. He is a collaborator to 
WiCell, and he is the scientific director for WiCell.
    Senator Specter. What is the relationship specifically 
between WiCell and WARF, Dr. Gulbrandsen?
    Dr. Gulbrandsen. WiCell is a wholly owned subsidiary of 
WARF.
    Senator Specter. Well, would you elaborate, Dr. Friere, on 
what NIH has done with respect to funding Dr. Thomson which 
would give NIH some proprietary interest?
    Dr. Friere. The NIH funded the very early work that Dr. 
Thomson did in monkeys. This was the work that allowed him to 
first look at this technology and, in fact, the original patent 
of the derivation of stem cells and of the actual cells was 
based on that primate work.
    Senator Specter. Do you know how much funding there was 
from NIH?
    Dr. Friere. No, but I can easily find out.
    Senator Specter. Would you provide that to the 
subcommittee?
    Dr. Friere. Yes, I will be happy to do that.
    Senator Specter. And as you see it, does that give NIH an 
interest in what Dr. Thomson has developed?
    Dr. Friere. Yes, by law, when we fund research, the 
Government retains a royalty-free, nonexclusive license to 
practice the invention on behalf of the Government.
    Senator Specter. A royalty-free, nonexclusive license?
    Dr. Friere. Yes.
    Senator Specter. So you are saying that NIH would have a 
license to work on deriving embryonic stem cells?
    Dr. Friere. We would have a license under the patent, if 
this was something that was permitted to do, we would have a 
license for Government purposes.
    Senator Specter. You mean if Federal funding could legally 
be used to extract stem cells from embryos?
    Dr. Friere. Right, and that means that we could use that, 
but only for Government purposes, and Government purposes are 
defined for work, for example, within the NIH and NIH 
contractors. It does not extend to our grantees.
    Senator Specter. It does not extend to your grantees?
    Dr. Friere. No.
    Senator Specter. How do you distinguish between a 
contractor and a grantee?
    Dr. Friere. A contractor is an award made for a specific 
Government purpose. A grantee is initiated by the researcher, 
so it is really a different system.
    Senator Specter. Well, the contractor provision would give 
a fairly broad swath to what could be done with NIH funding.
    Dr. Friere. I would have to look exactly into what that 
would allow us to do, Senator.
    Senator Specter. Why the distinction between contractor and 
grantee?
    Dr. Friere. That has always been the way that Government 
rights have been interpreted, have been interpreted to limit 
the rights to the contractor.
    Senator Specter. Well, would you provide the subcommittee 
with the specifics on that?
    Dr. Friere. I will be happy to.
    Senator Specter. We may be involved in a very high finance 
issue here, where the semicolons turn out to be very, very 
important.
    Dr. Friere. I will be happy to do so.
    Senator Specter. Dr. Gulbrandsen, would you agree with what 
Dr. Friere said?
    Dr. Gulbrandsen. Yes, I would, and I would say again that 
WARF supports the establishment of centers for derivation of 
stem cells and is eager to license those centers.
    Senator Specter. And you are prepared to license other 
laboratories which seek to derive human embryonic stem cells?
    Dr. Gulbrandsen. Yes.
    Senator Specter. But at a price.
    Dr. Gulbrandsen. Well, at a price that is fair.
    Senator Specter. But confidential.
    Dr. Gulbrandsen. That is right.
    Senator, I might add that with respect to WARF's patents I 
think the evidence is that WARF has acted responsibly with 
respect to its patents, and we have also put a tremendous 
amount of effort in moving this whole program forward. The 
research that has been done at Wisconsin in the embryonic stem 
cell area is really quite remarkable, and the research done by 
Geron is also quite remarkable, and so you know, we are not 
sitting on these patents.
    We are using these patents, and we are licensing them, and 
I would like to emphasize that Geron does not have the 
exclusive rights to these patents, and we are going to be 
looking at licensing others.
    Senator Specter. You do not have exclusive rights to the 
patents. Who else has rights to the patents?
    Dr. Gulbrandsen. What I am saying is, Geron does not have 
total exclusivity with these patents. They have exclusive 
rights in certain defined areas, but there are many other areas 
that are available for licensing and WARF intends that this 
technology gets developed to benefit humankind.
    Senator Specter. Well, are you saying that there are others 
besides WARF which have patent rights in the field?
    Dr. Gulbrandsen. No, not in the human embryonic stem cell 
area.
    Senator Specter. So WARF has the only patents in the human 
embryonic stem cell area?
    Dr. Gulbrandsen. That cover the human embryonic stem cell 
that I am aware of. I assume that Johns-Hopkins has patents 
covering the human--what do they call it, the embryonic germ 
cells. I am assuming that Johns-Hopkins has filed patents on 
that.
    Senator Specter. Well, this subcommittee is going to be 
looking at the specifics, and Dr. Friere, I would like you to 
provide the materials which I have asked for, and any other 
materials which would bear upon potential NIH rights to have 
research done because of the contribution which you made to Dr. 
Thomson.
    [The information follows:]

    You asked for clarification of the distinction between a grantee 
and a contractor and their respective right to use patented technology 
under grants and contracts.
    Contracts are used when the principal purpose of the instrument is 
to acquire (by purchase, lease, or barter) property or services for the 
direct benefit of the Government. 31 U.S.C. 6303. Grants are used when 
the principal purpose of the relationship is to transfer a thing of 
value to the recipient to carry out a public purpose of support or 
stimulation instead of acquiring property or services for the direct 
benefit or use of the Government, and substantial involvement between 
the Government and the recipient in carrying out the activity is not 
contemplated. 31 U.S.C. 6304. In accordance with the Bayh Dole Act, 35 
U.S.C. 200-212, the Government has a nonexclusive, nontransferable, 
irrevocable, paid-up license to practice or have practiced for or on 
behalf of the United States any invention conceived or first actually 
reduced to practice in the performance of a grant or contract for the 
performance of experimental, developmental, or research work.
    The government use license obtained from recipients of Federal 
funds may be used by the government for its own purposes, which have 
been determined to extend to government contractors, but not to 
grantees. As noted, grants are an assistance mechanism, where the 
grantee receives financial support for a particular activity, rather 
than a mechanism for the government to accomplish its own ends by 
setting the scope and performance of the work to be performed. Chapter 
10 of Principles of Federal Appropriations Law, 2nd ed., U.S. General 
Accounting Office, provides additional guidance (p. 10-6): ``Grant 
needs, by definition, are not needs for goods or services required by 
the Federal government itself. The needs are those of a nonfederal 
entity, whether public or private, which the Congress has decided to 
assist as being in the public interest.'' We are aware of no Federal 
court decisions directly interpreting the scope of the government's 
license under Bayh-Dole.

    Senator Specter. Dr. Gulbrandsen, the subcommittee would 
appreciate it if you would identify to the extent you can other 
parties who have patents on intellectual property rights.
    Dr. Gulbrandsen. Certainly.
    Senator Specter. And give us supporting data, to the extent 
you can, as to the positions that you have taken which have 
made this information available to others, and your general 
practices which, as you articulated, you consider to be 
responsible. We would like to take a close look at it, because 
it may be that the Congress will need to legislate in this 
area. I do not know.
    [The information follows:]

    WARF has never used its patents to block research and does not 
intend to do so with its stem cell patents. WARF is the owner by 
assignment from the sole inventor, Dr. Thomson, of two issued patents 
in the area of ES Cells. The first, U.S. Patent No. 5,843,780, (the 
``780 Patent'') issued December 1, 1998 with claims to primate ES 
Cells. The '780 Patent covers any primate ES cells (including humans as 
primates) which: (i) are stable and capable of proliferation in 
culture, (ii) maintain a normal karyotype containing all chromosomes 
characteristic of the primate species, (iii) maintain the potential to 
differentiate into all three embryonic tissue types (endoderm, 
mesoderm, and ectoderm tissues), and (iv) will not differentiate when 
cultured on a fibroblast feeder layer. This is characteristic of all 
normal ES Cells. In addition, the '780 Patent covers a method of 
isolating an ES Cell comprised of the current known method for 
culturing ES Cells.
    The second patent, U.S. Patent No. 6,200,806 (the ``806'' Patent) 
issued March 13, 2001 and is a divisional of the '780 Patent 
(collectively the ``ES Cell Patents''). The '806 Patent covers any ES 
cells with the same characteristics of those claimed in the '780 Patent 
but it specifically claims human ES Cells. Also, the '806 Patent claims 
the method for culturing ES Cells claimed in the '708 Patent but it 
again specifically claims the method for culturing human ES Cells.
    It is anecdotally interesting to note that while WARF's ES Cell 
Patents currently cover the most basic characteristics and the current 
method of culturing ES Cells, a cursory search of patent databases 
reveals that there are 95 issued patents in the United States with 
``stem cells'' in their title and there are at least 638 U.S. patents 
that incorporate ``stem cells'' somewhere in their claims. In addition, 
there are hundreds of additional related issued patents covering cell 
culture, growth factors, extra cellular matrices and cell 
differentiation. This is no different than the patent landscape in any 
other biomedical area of research. There is patent protection in almost 
every facet of our lives, from the cereal consumed in the morning, to 
the alarm clock set before bed at night. Scientific innovation in the 
United States is advancing faster than it ever has. Patents facilitate 
and enable this advance.
    WARF does not believe the ES Cell Patents are a disincentive for 
researchers in any respect. WARF and WiCell are committed to making the 
ES Cells and licenses under the ES Cell Patents widely available to 
academic researchers and industry.

    Senator Specter. We are on very unusual ground here when we 
talk about stem cells, and I congratulate what has been done by 
your organizations, Dr. Gulbrandsen, and the University of 
Wisconsin. We think it has been tremendous, and you have 
advanced scientific causes tremendously, and I do not in any 
way question your responsible approach to what is going on.
    We would just like to get a handle on the details, because 
we had a hearing a couple of weeks ago. A representative of HHS 
made a representation that the patents would obstruct NIH 
research, and that is why we convened this hearing on that 
aspect so that we could get involved in the details.
    Dr. Gulbrandsen. Senator, I appreciate that. I do not 
believe the patents will obstruct either the research or the 
commercial development, and we are more than happy to work with 
your office as we are happy to work with NIH. We consider NIH a 
very good partner at the University of Wisconsin, and I think 
we all want the same goal. We want to see this technology used 
to benefit humankind.
    Senator Specter. That is the common object.
    Well, I am told Dr. Friere, that you are stepping down from 
your post at NIH to become CEO of a nonprofit company dedicated 
to finding a cure for tuberculosis. Why did you choose a 
nonprofit company when profits appear to be so plentiful?
    Dr. Friere. Senator, I have a not-for-profit streak in me.
    Senator Specter. Well, you fit in very well with Congress 
in that event.
    Dr. Friere. Thank you.
    Senator Specter. And they are lucky to have you, Dr. 
Gulbrandsen. You are both a Ph.D. and a J.D., you teach patent 
law and you make all the discoveries. That is a pretty good 
combination, pretty hard to beat. Nobody around here to my 
knowledge, not even Dr. Frist, can compare with that, but 
perhaps Senator Frist can.
    Thank you very much, and we will take about a 2-minute 
recess, and will then convene the second panel, if the 
witnesses will come forward.
    Dr. Gulbrandsen. Thank you.
    Senator Specter. Thank you.
    We will reconvene. Thank you very much for coming, 
gentlemen.
    Let us start with Dr. Nigel Cameron, who is executive chair 
of the Centre for Bioethics and Public Policy. Educated at 
Cambridge and Edinburgh, Dr. Cameron founded the International 
Journal of Ethics and Medicine, and has authored a series of 
books on bioethics. I might just point out that Dr. Cameron has 
a somewhat different perspective. We have made these hearings 
balanced so that all points of view would be expressed.
    Senator Sam Brownback has been a frequent witness here, as 
have others, and my views on stem cell research are fairly 
well-known, but the subcommittee wants to hear from all points 
of view, and have it as balanced as possible so that there can 
be as thorough a discussion for the public as possible.
    So we are glad you came, Dr. Cameron, and the floor is 
yours. There is a 5-minute time limit, let me repeat, and we 
would like to observe that. Dr. Cameron, proceed.
STATEMENT OF NIGEL M. DE S. CAMERON, Ph.D., EXECUTIVE 
            CHAIR, THE CENTRE FOR BIOETHICS AND PUBLIC 
            POLICY, LONDON, ENGLAND
    Dr. Cameron. Thank you very much, Senator. I do appreciate 
that welcome, and the invitation. Part of the problem we face 
in this discussion is to identify what the issue is in which we 
are engaged in debate, and it seems to me very plain that the 
issue is this, whether we should be using members of our own 
species, our own kind, homo sapiens sapiens, in whatever stage 
of biological existence, for a purpose that is other than the 
good of the individual concerned, whether we should sanction 
the use of ourselves as experimental subjects.
    Let me offer four brief observations on our dilemma. First, 
until recently, it was widely agreed that human embryos should 
never be manufactured in order to be destroyed through 
experiment, however worthy the experiment. This principle, for 
example, is enshrined in the one international bioethics 
treaty, the European Convention on Biomedicine and Human 
Rights, and was memorably captured some years ago in a ringing 
editorial in The Washington Post.
    The creation of human embryos specifically for research 
that will destroy them is unconscionable, yet the Jones 
Institute has now brazenly announced that they have done just 
that, and the debate seems to be moving on.
    The problem, of course, is the challenge of consistency. 
May a line be drawn that will permit experimentation on 
clinically spare embryos, a line that will stand in the face of 
mounting commercial and clinical opportunity? This is, of 
course, the compromise that has been floated in various 
quarters, most notably and most seriously by Senator Frist.
    The level of support for embryo cloning to order in the 
Greenwood-Deutsch bill and now the timely ocular proof of the 
Jones Institute suggests the final naivety of such well-
intentioned policy hopes, since in the minds of most of those 
who lead the call for spare embryo research, there is only a 
modest distinction between spare embryo research and the new 
Jones approach, a distinction which falls far short of the kind 
of language captured by the Post in its use of a term like, 
unconscionable.
    Second, I do not propose to get involved in the extensive 
debate about the relative merits of embryonic and adult stem 
cell work. Plainly, some and perhaps all of the good things 
prophesied to come from the one may come from the other. It is 
ironic, and to my mind to be regretted that this debate has 
sometimes seemed to be central to our discussion of the ethics 
of embryonic stem cell work, as if somehow showing that there 
was another way to get these benefits somehow decided the moral 
questions. Moral questions, it seems to me, need to be decided 
on their own merits.
    Third, I do believe that in this discussion we are in 
danger of losing sight of the middle ground in the assessment 
of the early embryo. That is to say, this is not a rerun of 
Roe. This is not essentially a debate about the implications of 
our stance on the abortion issue.
    It is by no means necessary to take the view, moreover, 
that the early embryo is a full human person, however we define 
that term, in order to be convinced that deleterious 
experimentation is improper, indeed, the deeply held, widely 
held until very recently, view that creating embryos for the 
purpose of destructive research indicates a strong, intuitive 
commitment of persons, many of whom are not traditionally pro-
life, that the early embryo deserves profound respect. We seem 
to be moving on also from that view.
    Fourth, let me share my dismay at the degree to which this 
debate has sometimes degenerated into an iteration and 
reiteration of the potential benefits of this kind of 
experimentation, as if those who oppose public funding for what 
they consider unethical research are either ignorant of or 
heedless toward disease and its sufferance, and it does seem to 
me that the use of celebrities as an alternative argument is an 
unfortunate attempt to short-circuit the moral assessment of 
means by the crass assertion of ends.

                           PREPARED STATEMENT

    At the heart of our conception of civilization lies the 
notion of restraint. There are things that we will not do, 
whatever benefit they may bring, things we shall never do, 
though the heavens fall. As we stand at the threshold of the 
biotech century, we could hardly confront a decision that is 
more onerous, since the promised benefits from this technology, 
as from many other potentially unethical technologies, may be 
great, if that is, of course, simply to focus the moral 
question. It has become alarmingly hard to insist that whatever 
the potential benefit of a particular course of action, the 
means must be patently ethical. Human dignity, Mr. Chairman, is 
finally indivisible.
    Thank you very much.
    [The statement follows:]

              Prepared Statement of Nigel M. de S. Cameron
    My name is Nigel Cameron. I have worked for 20 years in bioethics, 
founded the journal Ethics and Medicine in 1983, and am currently 
involved in bioethics projects in both Europe and the United States. It 
is a privilege to be invited to testify today.
    Two great questions confront the human race at the start of the 
biotech century. The second, presently only on the horizons of our 
thinking and yet of incalculable import, will focus our growing 
capacity to design, determine, transform ourselves and our nature; the 
incremental progression toward the so-called ``post-human'' future. The 
first question is the one that confronts us today: whether we should 
use members of our own kind, Homo sapiens sapiens, in whatever stage of 
biological existence, for a purpose that is other than the good of the 
individual concerned; whether we should sanction the use of ourselves, 
in however early a form, as experimental subjects whose final end is 
destruction.
    Let me offer four observations on our dilemma.
    First, it seemed until recently to be widely agreed that human 
embryos should never be manufactured simply in order to be destroyed 
through experiment, however worthy the experiment. This principle is, 
for example, enshrined in the European Convention on Biomedicine and 
Human Rights, the one international bioethics treaty; and was memorably 
captured some years ago in a Washington Post editorial in the ringing 
phrase: ``The creation of human embryos specifically for research that 
will destroy them is unconscionable.'' Yet the Jones' Institute has 
brazenly announced that they have done just that. And as Charles 
Krauthammer's recent pro-stem-cell research piece notes, the cloning 
debate has focused the same issue. The chorus of support for Greenwood-
Deutsch has been fed precisely by a scientific-industrial community 
eager to clone and destroy embryos for scientific-industrial purposes.
    The problem, of course, is one of drawing lines; the challenge of 
consistency. May a line truly be drawn that will permit experimentation 
on clinically ``spare'' embryos, a line that will stand forever and in 
the face, we may expect, of mounting commercial and clinical 
opportunity that argues for their creation to order? That is of course 
the compromise that has been floated in various quarters, most notably 
and seriously by Senator Frist. The level of support for embryo 
cloning-to-order in Greenwood-Deutsch, and now the timely ``ocular 
proof' of the Jones Institute, suggests the naivete of such policy 
hopes, since in the minds of most of those who lead the call for 
``spare'' embryo research only there is only a modest distinction 
between this politic option and the Jones way. It is a distinction that 
falls far, far short of what the Post designated ``unconscionable.'' It 
is not, as we might put it, that we believe that further dominoes will 
fall; they are falling all around us. For the logic of the experimental 
abuse of ``spare'' human embryos depends ultimately on so meager a 
valuation of the embryo itself that their creation-to-order is 
inevitable. If the embryo is at base object and not in any sense 
subject, what is to prevent it? It is reported that one celebrity 
recently announced here on the Hill and in defense of embryonic stem-
cell research that the embryo is of similar moral standing to a 
goldfish.
    Second, I do not propose to get drawn into the extensive debate 
surrounding the relative merits of embryonic and other, typically 
adult, stem-cells. Plainly, some and perhaps all of the good things 
that are prophesied to be the fruit of embryonic stem-cells may be 
attained using adult cells or other means. It is ironic, and to be 
regretted, that this debate has sometimes seemed to hinge on whether 
adult stem-cell work is likely to be as fruitful as the embryonic kind, 
as if the moral question, while of some weight, could be discounted by 
a certain evaluation of likely relative clinical outcomes. This is a 
profound moral debate about what we will and will not do to our own 
kind, for whatever alleged benefit.
    Third, I believe that we are losing sight of the middle ground. By 
that I mean that it is by no means necessary to take the view that the 
early embryo is a full human person in order to be convinced that 
deleterious experimentation is improper. There are many possible 
grounds for such a view--that we do not know if the embryo possesses 
full human dignity and should therefore be prudent; that the embryo 
possesses the potential to be a full human person and that such inbuilt 
potentiality entails profound respect, a view widely held and deeply 
threatened in this debate; or that membership in our species is enough 
to distinguish the human embryo from all other laboratory artifacts. 
Indeed, the widely held view that embryos should not be specially 
created for experimental purposes itself reveals a strong if undefined 
disposition to protect the embryo from abuse.
    Fourth, let me share my sense of dismay at the degree to which this 
debate has sometimes degenerated into an iteration and reiteration of 
the potential benefits of this kind of experimentation, as if those who 
oppose public funding for what they consider unethical research are 
either ignorant of or heedless toward disease and its sufferers. The 
celebrity argument is a sham, an attempt to short-circuit the moral 
assessment of means by the crass assertion of ends. It is an 
embarrassment to the cause of ethics in public policy.
    For the question we face is distinctly ethical in character. At the 
heart of our conception of civilization lies the principle of 
restraint: that there are things we shall not do, shall never do, even 
though they may bring us benefit; some things we shall never do, though 
the heavens fall.
    As we stand on the threshold of the biotech century, we could 
hardly confront a decision that is more onerous, since the promised 
benefits from this technology may be great. Yet that is of course 
simply to focus the moral question. If there are things that we should 
not do, it is easy for us to refuse to do them when they offer no 
benefit. When the benefit they offer is modest, the choice is still not 
hard. The challenge to morals and to public policy lies precisely here, 
where the benefits seem great. Yet it is here also that our intuitive 
respect for the early embryo requires us to pay a price. In a culture 
fixated with the satisfaction of its needs and the healing of its woes, 
it has become hard even to say that we shall never, for whatever 
benefit, experiment on our own kind? Shall we do evil, that good may 
come?

STATEMENT OF ARTHUR CAPLAN, Ph.D., DIRECTOR, CENTER FOR 
            BIOETHICS, UNIVERSITY OF PENNSYLVANIA
    Senator Specter. Thank you, Dr. Cameron. We turn next to 
Dr. Arthur Caplan, director of the Center for Bioethics, 
University of Pennsylvania, professor of molecular and cellular 
engineering, and professor of philosophy, B.A. from Brandeis, 
Ph.D. from Columbia. Thank you for joining us, Dr. Caplan, and 
the floor is yours.
    Dr. Caplan. Thank you, Senator. It is a pleasure to have 
this opportunity to testify to the committee and to a fellow 
Pennsylvanian.
    I think in some ways I, too, am going to follow Dr. 
Cameron's lead. I am not going to spend any time today in my 
brief remarks going over the benefits, be they from 
celebrities, or be they from scientists, or be they simply from 
patients who are suffering with disability or disease. I think 
we can concede that stem cell research is promising. I think 
there may be a long road to travel to deliver on that promise, 
but nonetheless, it seems to me the promise is there, and that 
can be conceded regardless of discussions about possible 
alternative strategies.
    What I would like to do is focus instead on a couple of 
ethical points about how I would frame this debate. I have been 
following it closely, in fact, since I first came before this 
committee about 9 months ago, I think, to talk about this 
issue, and like Dr. Cameron I, too, have been a little bit put 
off by some of the tone of the debate. I think people who are 
arguing for respect for human life are commanding a moral 
position that is deserving of careful listening. I appreciate 
the committee soliciting all opinion, but at the end of the 
day, I think the framework being articulated is not correct.
    It would be wrong for sure, morally, to say that we can 
benefit people who are in need, or future generations, by 
killing some people today. There is no doubt that a principle 
we should not break is that we should not murder to benefit. As 
the Senator knows, this has been a major issue in a related 
area that he and I have had a chance to talk about, organ 
donation. We know that we can benefit by making kidneys and 
livers and hearts available from deceased persons, but we also 
know that we must not hasten death, cause death, or in any way 
be involved with death, bringing it about, in order to do the 
benefit.
    On the other hand, people die from many tragic reasons, and 
we do try, then, to approach individuals and see whether they 
wish to make something good happen out of these tragic 
circumstances. There may be suicides, there may be murders, 
there may be child abuse. There are all kinds of conditions 
that sadly produce the availability of cadaver organs.
    I believe that in this debate there is a moral equation 
that does not hold, and that is that embryos are either 
persons, or to be treated as human beings from the moment of 
conception. I think factually this is not the case. I think 
that what we are talking about and what most Americans believe 
is that we have something that is a potential or possible 
person in the right circumstances. In a dish, in a freezer, 
that potentiality will go nowhere.
    If we look at the circumstance of embryos, and if we look 
at what our biologists are telling us about understanding the 
genetics involved in development, we know that many embryos are 
not ever going to become persons, no matter what we did to 
them. It is why infertility treatment is so difficult.
    The fact that embryos may have genetic errors, if you will, 
blueprint problems, and many do, and that increasingly, as 
women age, those are more manifest, making fertility impossible 
after one reaches their fifties, is an indication that not all 
embryos have potentiality, so one premise I would put before 
you and the subcommittee to ponder is that not all embryos do 
have the potential to become human beings. We know this.
    Second, when we store, freeze, and put them aside, it is 
often because in the opinion of physicians, these particular 
embryos are not likely to become persons, and the longer they 
are kept frozen and stored somewhat diminishes that potential 
even further.
    That means that what we are talking about to begin with is 
not, if we propose to destroy embryos, necessarily killing. We 
are talking instead about, if you will, the destruction of 
potential, possible persons. We are also talking, if you will, 
about the destruction of many things that have no possibility 
of becoming persons.
    If I am to make a trade-off, then, the other principle I 
have to follow is, do we make things with the intent of 
destroying them, or have we a situation where for good motives, 
people trying to have children, for good reasons, people 
wanting to have babies, these entities are created and exist, 
but are no longer wanted. As the Senator is well aware, there 
is something like 100,000 of these, at a minimum, around the 
United States. Their fate is never to become children.
    I see that as somewhat analogous to the situation with 
transplant. No one set out to make this situation occur. It is 
a byproduct of our ignorance and inability to successfully help 
people who want to have children. We overproduce embryos. They 
are left behind, and they are ultimately going to be destroyed.
    If we grant, then, that not all embryos have the potential 
to become persons, then not all embryos are persons, and that 
we have, if you will, an enormous number of embryos that never 
will become persons, and trying to make some good happen from 
the reality of the existence of those stored embryos, which 
abound, leads me to one last observation.
    We are talking here about research that will involve 
embryonic stem cells, but we are talking also about research 
that is relatively new, that relatively few people can do. I 
have to report to the Senator that in trying to figure out--and 
you will see this in my written testimony--what number of 
embryos are we talking about for the next few years to 
demonstrate the feasibility of this research, I would estimate, 
if 15 researchers worked with five embryo cell lines, we might 
be talking about something less than 200 embryos out of 100,000 
frozen that their fate is destruction, or permanent storage. It 
seems to me the moral equation comes out in favor of those who 
are real, here and now, with real needs and real disabilities 
and real problems. That promise should be delivered on.
    So I would argue, using those facts, and the moral 
principle that making something good happen out of the reality 
of something unfortunate exists, the surplus, absolute huge 
number of embryos that already exist, if we are in a situation, 
what we are talking about then is possibility of potentiality 
that will never be actualized, I think it is a trade that this 
committee should pursue aggressively in order to bring benefits 
to the American people.
    Senator Specter. Thank you very much, Dr. Caplan.
STATEMENT OF GLENN McGEE, Ph.D., ASSISTANT PROFESSOR OF 
            BIOETHICS, PHILOSOPHY AND HISTORY AND 
            SOCIOLOGY OF SCIENCE, UNIVERSITY OF 
            PENNSYLVANIA
    Senator Specter. We turn now to Dr. Glenn McGee, assistant 
director for education and assistant professor of bioethics, 
philosophy, history, and sociology at the University of 
Pennsylvania, editor-in-chief of the American Journal of 
Bioethics, B.A. from Baylor, and Ph.D. from Vanderbilt. Thank 
you for joining us, Dr. McGee, the floor is yours.
    Dr. McGee. Thank you very much for inviting me, Senator, 
and for the opportunity to testify before the subcommittee.
    As a scholar of bioethics, ethical issues and human cloning 
and stem cell research in particular, my role today----
    Senator Specter. Tell me for the record how your laptop is 
working here. It does not appear in the printed text. I think 
it would be of some interest if anybody ever reads this record.
    Dr. McGee. I am not sure I follow the question.
    Senator Specter. Your mechanism for presenting your 
presentation. What is it, and how are you using it?
    Dr. Caplan. He has got a battery-driven laptop.
    Senator Specter. Dr. Caplan has clarified it totally. You 
may proceed, doctor.
    Dr. Cameron. It is not potential, it is actual.
    Dr. McGee. That is right, and it has rights.
    My role today as--I would like to construe it as to briefly 
discuss the question of self-regulation in human embryonic stem 
cell research. Like my colleague, Dr. Caplan, I have authored a 
number of articles about stem cell research ethics in general, 
and participated in a number of ethical debates within the FDA 
Panel on Molecular and Genetic Devices, on which I serve, but 
from August of 1999, when I was invited to join the ethics 
advisory board of Advanced Cell Technology, one of the 
companies that works in the area that we are discussing today, 
until October of 2000, I was active in a relatively new form of 
discussion and debate about ethics, and that is the corporate 
ethics advisory approach.
    Although I am a proponent of stem cell research, and have 
argued in print that stem cell research, including research 
involving embryos, should be permitted and funded, in October I 
reluctantly resigned from my role at Advanced Cell Technology 
for two very specific reasons.
    First, I have concern over corporate decisions in stem cell 
research in particular not to share all of the information 
about ethically relevant activities in stem cell research with 
the ethics advisory board that is convened to help the company, 
or in due course to share the full amount of information 
available with the public so that public debate can be 
developed, and second, because I was concerned about what I 
viewed as the excessive pursuit of intellectual property in 
stem cell research.
    To be clear, I am not opposed in principle to the creation 
of embryos for research using nuclear transfer, or to research 
that uses stored or made embryos. I believe that at some point 
in the future there may be significant therapeutic benefits to 
be derived from what is being termed therapeutic cloning, and I 
worked with the British Government in 2000 on what became their 
policy of carefully monitored nuclear transfer research.
    But what I am concerned about today is the danger that 
industry self-regulation in the area of nuclear transfer may 
not be easily ameliorated by Federal funding. It seems to me 
extremely problematic that the early stem cell research 
currently underway has been driven in the main by small 
business, because at this early stage the only real resources 
that small stem cell companies can hope to build up are 
patents.
    If stem cell research is tied up at this stage by patents 
and licensing agreements, even if those patents are held by 
universities who trade them in fair and honest ways, the effect 
will be to hamper and slow research, but moreover, it will tax 
any Federal dollars for stem cell research in what I think you 
could argue is an unacceptable way.
    At this point, one could offer any number of percentages, 
but arguably as much as 30 to 50 percent of Federal funding for 
stem cell research might flow directly or indirectly to small 
stem cell companies through the fees that they are allowed to 
assess to any funded researcher.
    More importantly, I am concerned about the risks of 
allowing a small biotechnology company, whether inside or 
outside the university, to control and license stem cell 
technologies. This is aggravated, moreover, by the fact that 
the companies with the most valuable portfolio of stem cell 
patents are also companies that, it has recently been reported 
in the media, are tiny operations with little capital or 
strategic flexibility.
    Whether or not there is Federal funding of therapeutic 
cloning, it seems to me there must be much more oversight over 
nuclear transfer technologies and specifically over the control 
of these technologies by a few people in small business.
    Perhaps it is because small companies are so innovative and 
important in the overall scope of stem cell research that at 
least two stem cell companies created ethics advisory boards to 
help them deal with the kind of controversy that corporate-
driven basic research would necessarily create. I can only 
relay my own experience on such a board, which I think is in 
some ways instructive.
    In my time on the ethics advisory board of Advanced Cell 
Technology, I do not believe that the relatively new mechanism 
of corporate ethics advisory boards performs the role of self-
regulation effectively. I was concerned that ethics advisory 
boards, including the one on which I served, which many in 
industry have now pointed to as a way to promote ethical 
practice, will not be able to work in an objective way, or in 
the broader societal interest.
    My concern is not that ethicists are bought by companies, 
or about research conducted within companies to determine 
whether or not ethical problems exist. Indeed, both of these 
are to be, I think, considered separately. Instead, I am 
concerned that ethics boards and their chairs are being asked 
by these companies to play roles that are, in my view, not 
appropriate to the ideals of bioethics or, more importantly, of 
science in the public interest.
    Their roles have shifted, at least in my own experience, 
from the original conception of an ethics advisory board in its 
Government capacity, which is a research body convened to 
evaluate the activities of the organization and publish those 
to peers, into a very different role, that of lobbyists or 
public relations. In my view, industry self-regulation through 
boards thus serves neither the interests of the companies nor 
the interests of ethics.
    Stem cell corporate ethics advisory boards are attractive 
to ethicists precisely because, in the world of stem cells, you 
cannot study the research without understanding how it is 
conducted. In my view, this was an important reason to 
participate in this research and in this way.
    But whatever good intentions the company may have in terms 
of providing that kind of access, the usefulness of ethicists 
working in companies on ethics advisory boards is at least in 
part the fact that their papers and statements on behalf of the 
company have the effect of insulating the company against 
criticism from other ethicists, who will not be comfortable 
attacking their peers, whose work is coming out of such a 
vulnerable position.
    Briefly, a final point. I think that in order for the 
ethics advisory board as a mechanism for self-regulation to 
work, it would have to do several things, and to do them 
openly. First, to foment public discussion and debate, to hold 
open meetings that are well-publicized in advance, to name its 
members.
    Second, to advance discussion of corporate research in the 
bioethics community before companies begin their work, not 
afterwards.
    Third, to insist on full knowledge of corporate activities, 
including disclosure of any experiments that might receive 
ethics scrutiny. In my case, I found out about one very 
controversial experiment the day after it was released from 
embargo. The ethics advisory board had not been notified that 
cell technology was working on, for example, cloning a gauer.
    Fourth, to be open about the affiliations, 
responsibilities, and expertise, or partisanship of any members 
of the committee.
    Fifth, to be fully funded by the committee not just an 
unfunded declaration.

                           PREPARED STATEMENT

    And finally, to discuss only those issues about which it 
has written or debated, and to insist on rigorousness about 
that role.
    I think that ethics advisory boards are the best model 
industry has offered for self-regulation, and in my own 
experience, working in a number of different capacities, 
including my single experience on an ethics advisory board, I 
think we will find that this is insufficient.
    [The statement follows:]
      Joint Prepared Statement of Arthur L. Caplan and Glenn McGee
    We are very pleased to have the opportunity to address this 
subcommittee on the subject of stem cell research. One of us has had 
the privilege of speaking on this subject at an earlier hearing and 
since that time we have followed with keen interest the thoughtful 
efforts by this subcommittee to address the complex moral, regulatory 
and scientific issues associated with stem cell research.
    As some of the members of this subcommittee know, we have tried for 
many years to wrestle with the question of the morality of using cells 
from fetal and embryonic sources for medical research. In thinking 
through the ethics of such research activity it is our view that 
ethical principles and values, which have broad support in our society, 
ought to guide the initiation and direction of such research.
    In the case of embryonic stem cell research in particular, it is 
not morally acceptable to decide that embryonic stem cell research will 
be done and then set off in search of those experts, values and 
principles that will support such a policy. Unfortunately, that appears 
to be what has happened with respect to some of the ethical analyses 
that private companies who wish to engage in fetal or embryonic 
research. Moreover, while we are in no way opposed to companies seeking 
ethical advice and input from many sources we do not see these 
activities as in anyway a substitute for or an alternative to 
government interest and responsibility for stem cell research and 
research in related areas such as therapeutic cloning. In our view we 
should try and understand what the relevant facts are about embryonic 
stem cell research. Then an effort must be made to see whether there 
are ethical values and principles that command broad consensus relevant 
to this issue. Then, having ascertained the facts about the proposed 
research and the values that we as a society are trying to adhere to 
with respect to undertaking research or therapy involving stem cells or 
cloned embryos it should be possible to offer an evaluation about 
whether Federal support should be offered, whether it is ethical to 
undertake the research at all regardless of funding source, what limits 
should be placed on research if any and what role the government should 
take in providing oversight and accountability to these activities.
              the facts about embryonic stem cell research
    In asking whether it is ethical for the Federal government to 
support research on embryonic stem cells it is important to be clear 
about what is being undertaken. Research on embryonic stem cells 
requires the destruction of human embryos. There is no avoiding that 
fact. This single fact makes embryonic stem cell research ethically 
troubling. One does not have to be a member of the ``pro-life'' 
community to understand that destroying human cells that have the 
potential to become human beings represents an action that requires 
ethical justification.
    Why undertake research on embryonic stem cells? The answer is that 
these cells show great promise as a source of cell lines and tissues 
that could be used to repair or replace damaged cells, tissues and 
organs in fetuses, babies, children and adults who have a variety of 
ailments and diseases. Many of the ailments that plague humankind are 
caused by damage to our cells and tissues. Parkinsonism, juvenile 
diabetes, traumatic brain injuries, spinal cord injuries, Canavan's 
disease, sickle cell disease and cystic fibrosis are but a few such 
disorders. Since many human cells and tissues do not have the capacity 
to regrow or rejuvenate or can only do so at a very slow rate it would 
be a boon in the battle against disease and disability to have a source 
of cells that could be grown to be transplanted into damaged areas of 
the human body and once there perform the requisite functions necessary 
for human health.
    Some critics of embryonic stem cells research have acknowledged the 
potential benefit of conducting research which could provide a source 
of transplantable cells for all manner of diseases and disorders but 
they say there are alternatives to using embryonic stem cells for this 
purpose. Adult stem cells, such as those that are used by the body to 
replace damaged skin or muscle or to continuously rejuvenate our blood 
cell might be able to be transformed into cells lines that can make the 
kind of bounty of cell types that might be possible to obtain from 
embryonic stem cells.
    The fact is that no one can be sure what research on adult stem 
cells will produce in terms of being able to manufacture cells to 
repair cells in those with diseases because this research is very much 
in its infancy. It is absolutely true that embryonic stem cell research 
is also so new that it can only accurately be described as promising. 
But, there is no way that anyone can know today if research on adult 
stem cells will prove capable of making the kind of universal cell 
types that researchers hope to create from embryonic stem cells. Adult 
stem cell research is neither an alternative to nor a substitute for 
embryonic stem cell research. If the goals are to repair broken, 
damaged or dying cells in human beings than both lines of research must 
be pursued.
    One other fact becomes important in trying to formulate an ethical 
assessment of the morality of Federal support for embryonic stem cell 
research--embryonic stem cell research is at best a promise. Almost no 
sustained work has been done in this area. While there are private 
companies and clinics that are eager to pursue this avenue of work they 
have not published or made public any information that should lead 
anyone to think that therapies are in the offing. Nor has any of the 
work on embryonic stem cells done in the public sector using animal 
models done anything more than show potential for therapies. The facts 
are that relatively few scientists have done relatively little research 
using human embryonic stem cells.
    That said, it becomes very important to make clear the number of 
embryos that would be utilized should the Federal government decide to 
support embryonic stem cell research. In talking with scientists and 
veterinarians who know quite a bit about stem cell research it has 
become clear to me that there are no more than thirty researchers who 
might seek Federal support for research using human stem cells in the 
next few years. If one presumes that the Federal science agencies, 
mainly the NIH, were to give grants for fifty percent of the proposals 
received (a generous estimate) then there would probably be an average 
of 15 researchers conducting research with human embryonic stem cells a 
year for the next three years. If each one of these scientists were to 
use on average five human embryos for each study then the debate about 
Federal funding of human embryonic stem cell research is a debate about 
the utilization of less than one hundred human embryos a year for three 
years.
    There are some other facts that are equally relevant about human 
embryo stem cell research. There are four ways to get embryonic stem 
cells--from newly created human embryos made specifically for that 
purpose, from newly created human embryos made in order to help 
infertile couples have children, from frozen human embryos and from 
human embryos that have been created through the process of cloning 
following the steps used to create Dolly the cloned sheep. And each 
source is currently being utilized or could easily be utilized.
    At least one clinic, the Howard Jones Institute at Eastern Virginia 
Medical Center in Norfolk has reported that it has publicly stated that 
its scientists have made human embryos solely for the purposes of stem 
cell research. A few infertility programs that I have contacted have 
told me that they offer couples who are seeking infertility treatment 
the option of putting any embryos that will not be implanted for the 
purpose of reproduction the option of donating them for the purposes of 
research. At least one private company has stated that it has made 
human embryos by the process of cloning to use for procuring human 
embryonic stem cells. And a number of researchers have indicated an 
interest in utilizing human embryos that have been stored and remain 
unclaimed for many years as a source of stem cells.
    There is one fact that is not at all clear and that is what is 
created when human DNA from an adult cell is used to make a clone. 
While some believe that starting a clone is equivalent to making a 
human embryo, others do not. We have referred to this puzzle as the 
``what is in the dish dilemma''. Our view is that there is no certainty 
that a human clone at the embryo stage can turn into a viable human 
being. There is evidence from the animal world to suggest that the 
chances of being able to do that are poor. It is also the case that if 
the cloned human embryo is not put into a human uterus then it cannot 
become a human person.
    We are troubled by the idea of making therapeutic clones solely for 
research purposes since we are not really sure what it is that is being 
created. Without such certainty and in the absence of any agreement 
about what this new technology creates it is difficult to argue that it 
makes sense from the point of scientific research to ban this modality 
of acquiring stem cells for research or therapy. Before any legislation 
is enacted to prohibit, fund or encourage therapeutic cloning it would 
seem very prudent to have a committee or commission established to 
formulate an answer to the question of what moral standing should be 
accorded to human embryos made by nuclear transfer cloning techniques. 
While some may want to have a ban now on he grounds that the creation 
of any such clones is equivalent to the creation of human embryos more 
sustained deliberation may lead us to conclude that these ``starter'' 
clones lack the capacity to become human beings and as such may turn 
out to be a less ethically contentious source of stem cells for 
research.
                     ethical values and principles
    In 1996 Caplan wrote an article with George Annas and Sherman Elias 
in the New England Journal of Medicine in which we argued that research 
involving embryos was ethical if the embryos used for research were 
taken from those stored and frozen in the United States and other 
Nations. In taking this position we argued against the view advanced by 
various scholars and Federal panels that the source of human embryos 
did not matter. We believe this ethical position was correct then and 
remains correct today. The facts associated with human embryo stem cell 
research to demonstrate the feasibility of using stem cells for 
therapeutic purposes are consistent with the moral principles involved 
in restricting the source of such cells and tissues to frozen embryos 
left behind by the tens of thousands at infertility clinics in this 
country and other countries.
    Before arguing for this position, we want to say that Americans 
should also understand that those who believe that all research on the 
human embryos are deserving of nothing short of respect. It is a sad 
fact about the tone of our ethical debate on this issue and on all 
matters pertaining the to the handling of embryos and fetuses we all 
too often find ourselves engaged in interactions which insult, 
deprecate or dismiss out of hand the moral concerns of those with whom 
we disagree. We understand the moral position that holds that all human 
life is sacred and that it is wrong to sacrifice one human life to save 
another or help another. We believe this is not what is required with 
respect to human embryo stem cell research but the moral sentiment 
underlying the position is absolutely sincere and is deserving of both 
a full hearing and praise for the moral value it articulates.
    The values and principles that seem operative in thinking about 
what to do concerning the facts of stem cell research are that we 
should not kill in order to advance the welfare of those in need, we 
should not commercialize or commodify the process of procreation and 
human reproduction, we should accord all human cells and tissues 
respect by controlling how they are used and what can be done to them 
and with them and we must weigh seriously the health care needs of 
those who have diseases and disabilities carefully against the value of 
respect according to cells, embryos and the procreative process.
    We believe that in weighing the balance of values and principles 
that human embryos and cloned human embryos are at most potential or 
possible human beings. They are not human beings from the moment of 
conception. Nor are they in any sense persons from the moment of 
creation or conception. Many embryos cannot become persons due to 
errors in their biology. Most human clone embryos most certainly lack 
the capacity to become human beings. And without being put into a 
uterine environment no embryo, regardless of how it is created or where 
it is obtained, can have its potentiality actualized.
    We believe that in weighing the needs of children and adults who 
are paralyzed, demented or dying the needs of those are actual persons 
has greater moral standing than the potentiality inherent in embryos or 
clones. Thus, given the relatively small number of human embryos that 
would be required to establish the feasibility of human stem cell 
therapy, given the position that killing a potential or possible human 
being is not the same as murder, given the fact that medicine must, 
sadly, sometimes do harm in order to do good, given the potential for 
benefit in the form of assistance to real, living babies, children and 
adults we favor government sponsorship of a limited amount of human 
stem cell research. The best way for this work to proceed would seem to 
be to utilize embryos already in existence rather than to create news 
ones solely for the purposes of research. Without more consensus on the 
metaphysical status of cloned human embryos it makes better moral sense 
to proceed with research on embryos already in existence that are 
destined for destruction or never to be used for the purposes of 
procreation.

    Senator Specter. Thank you very much, Dr. McGee.
STATEMENT OF MICHAEL D. WEST, Ph.D., PRESIDENT AND CEO, 
            ADVANCED CELL TECHNOLOGY
    Senator Specter. We will turn now to Dr. Michael West, 
president and CEO of Advanced Cell Technology, a biotechnology 
company based in Worcester, Massachusetts, he has a master's 
degree from Andrews, Ph.D in cell biology from Baylor College 
of Medicine, and this is his third appearance before this 
subcommittee. Thank you very much for joining us, Dr. West.
    Dr. West. Thank you, Mr. Chairman, and members of the 
subcommittee. I have submitted my written testimony, so if I 
may, I will just make a few salient points.
    Senator Specter. Your written testimony, as well as all 
other written testimony, will be made a part of the record in 
full.
    Dr. West. Thank you. I would like to then summarize three 
major points, first just to briefly address I think maybe some 
useful points of science, and then secondly, on patent law, and 
then third on ethics, if I may.
    First, on the scientific front, I think it is useful to 
point out that mankind occasionally is given gifts, things that 
can greatly advance the human condition. I think we have been 
given two in just recent history. The first we have talked 
about at some length already this morning, the human embryonic 
stem cell.
    The opponents of the technology point out, or try to point 
out that we do not need this technology, these cells are not 
unique. There are other cells that do what these cells do. This 
is a misstatement of fact. The human embryonic stem cell is 
unique in at least two regards. One, it stands at the base of 
the developmental tree of cellular life. It can become any cell 
in the human body. As we say, it is totipotent. It has total 
power. This is unprecedented and a unique property for these 
cells.
    As an example, the cells can even assemble into a complex 
tissue, such as a complete intestine, with all the cells of an 
intestine. No other cell has ever come even close to this in 
the history of medicine.
    Besides the ability of these cells to become any cell in 
the human body, they have a second and equally important 
property. They have a property we call cellular immortality. 
This means that, unlike all the cells in the human body ever 
cultured to date, they can grow indefinitely. They stay young 
in a way that our reproductive cells stay young, making babies 
that are born young.
    Cells that result from these cells would be predicted to be 
young, having their full replicative life span ahead of them, 
so for an aging population, which is increasingly a focus of 
our national concern, these technologies, because this 
technology in particular, embryonic stem cells, because of all 
these unique properties, may be uniquely poised to address a 
very large health concern for the United States and the rest of 
the world.
    A second gift we have been given is this miracle we call 
cloning, or nuclear transfer. Nuclear transfer was a surprise 
to science. It tells us that a cell from our body, even an aged 
cell, can be taken back in time, in a little time machine--it 
is only big enough to hold a single cell, but it can take a 
cell back in time to an embryonic state, where that cell can 
then, as I just pointed out, become any cell in the body, but 
it also can be rejuvenated in the process, giving an aged 
patient back young blood cells, for instance, or young skin 
cells to help with geriatric skin ulcers.
    So in summary, on the science side we have been given a 
great opportunity. How do we best implement this for the 
benefit of humanity?
    On the legal front, opponents of this new technology point 
out that there should be no patents, or challenge the 
patentability of the technologies. It is important to point out 
that we have several basic assumptions I think we are operating 
from on the legal front, and forgive me for opining on law. I 
am not a lawyer, I am a scientist and a businessman, but in 
1980, the Supreme Court decided the Diamond v. Chakrabarty 
case, saying that life forms are patentable inasmuch as they 
involve human intervention.
    Maybe the most salient case goes back to a very early U.S. 
patent by Louis Pasteur. He patented in a very comparable way 
to the human embryonic stem cell patent a composition on living 
cells unaltered except for their isolation. He patented the 
isolated yeast cell, free of organic pathogens, useful in 
manufacture, one of the first U.S. patents issued in the field 
of biotechnology.
    Historically important portal technologies do not interfere 
with commercial development products that can help people who 
are sick. We can look, for instance, at recombinant DNA, which 
was managed by Stanford University. Although patented, it was 
freely available, and has generated now the majority of the 
current biotechnology industry, which has clearly benefitted 
mankind.
    Clearly an issue is availability, free availability for 
academic researchers in particular. In that regard, I would 
just like to point out to the committee, when I first 
approached WARF in 1995 about sponsoring research in this area, 
the first words out of WARF's mouth were exactly to that point, 
nonexclusivity, free access, to their credit.
    Senator Specter. Would you repeat that, Dr. West? What were 
the first words out of WARF's mouth?
    Dr. West. Out of WARF's mouth to me, as a corporate 
officer, when I approached them to sponsor this research, to 
obtain the human embryonic stem cells with private money, I 
requested an exclusive license, which is not unusual. Indeed, I 
would say the vast majority of such arrangements grant 
exclusivity.
    Senator Specter. You asked for an exclusive license, and 
what was the response?
    Dr. West. No--they refused, and what they stated was that 
they would be glad to talk about nonexclusive licenses, but 
they felt that as a matter of policy these cells should be 
licensed nonexclusively and be freely available, because of the 
potential breadth of the technology.
    In the interests of time here, I will just point out, on 
the ethics front, one quick point. Much of the debate about 
these technologies is about human life. It is critical to 
understand that I think the differences of opinion are about 
human cellular life. All cells in the human body are alive. 
Many of us do not want to agree with this, but we evolved from 
single-celled animals. These are living entities. When a 
scientist talks about life, or human life in a dish, he does 
not mean a human life. It is a fundamental distinction which 
goes to the heart of the debate.
    So in summary, I think even the critics of these new and 
emerging technologies will admit, teleologically, these 
technologies have the potential to save millions of human 
lives. I think this is an issue of grave concern, and should 
cause us to pause.

                           PREPARED STATEMENT

    Yesterday's vote by the House, with 2 hours of debate, I 
think was not a good indication of the relative graveness of 
this issue, and I would respectfully request that the Senate, 
the greatest deliberative body in the world, would take this 
issue with a great deal of caution and a careful deliberation. 
As a leader of world technology, I think it is the 
responsibility of the United States, it is our challenge, 
indeed, it is our duty to do so.
    Thank you.
    [The statement follows:]

                 Prepared Statement of Michael D. West

    Mr. Chairman and members of the Subcommittee, my name is Michael D. 
West and I am the President and Chief Executive Officer of Advanced 
Cell Technology, Inc., a biotechnology company based in Worcester, 
Massachusetts. A copy of my curriculum vitae is presented in Appendix 
A.
                              introduction
    I am pleased to testify today regarding human embryonic stem cell 
and nuclear transfer technology and their applications in medicine. I 
would like to first speak to the potential benefits of this emerging 
science, and then speak to some of the questions and concerns that have 
been voiced.
             the potential benefits of es and nt technology
    Human Embryonic Stem (ES) cells are unique in the history of 
medical research for at least two reasons. First, they alone are 
totipotent stem cells. By stem cells, we mean cells that can branch out 
like the stems of a tree, becoming other cell types. By ``totipotent'' 
we mean to say that they stand near the base or ``trunk'' of the 
developmental tree and so are capable of forming any cell or tissue 
type needed in medicine. In addition to forming any cell type, they are 
unique in their ability to self-assemble into complex multicellular 
tissues such as intestine, full thickness skin, kidney tissue, and so 
on. They differ in this respect from adult stem cells that are 
``pluripotent''--that is, capable of forming several, but only a 
limited number, of cell types. One can think of adult stem cells as 
limbs further out on the branches of a tree. While able to branch out 
in several different directions, only the trunk of the tree branches 
out into every leaf and limb. An example of adult pluripotent adult 
stem cells are the bone marrow stem cells now widely used in the 
treatment of cancer and other life-threatening diseases.
    The second distinguishing feature of ES cells is the ease with 
which they can be purposefully modified in a precise manner. This 
precise genetic modification is designated ``gene targeting''. The 
enhanced ability of ES cells to be modified with precision likely opens 
the door to many hundreds of clinical applications making human cells 
of any kind, genetically modified in any way to ``heal'' mutations in 
genes, something never before possible in medicine.
    These two unique characteristics of human ES cells open the door to 
manifold novel therapeutic strategies. It may not be an exaggeration to 
state that the combination of the ability to precisely genetically 
modify these cells by targeted modifications and the ability to make 
any cell type may have as profound an application in medicine as the 
ability to arrange electrical components has made in the electronics 
industry.
    To attempt to name every disease that potentially could be treated 
using this technology would require a larger report. Here are just a 
few examples. Neurons could be manufactured to treat degenerative 
diseases such as Parkinson's and spinal cord injury. Gene targeting to 
find and ``heal'' mutations could be used to manufacture neuronal stem 
cells for childhood retardation from diseases like Rett syndrome. Heart 
and skeletal muscle cells could be used for heart failure and age-
related skeletal muscle wasting, and targeted genetic modification 
could be useful in muscular dystrophy. Blood forming cells would be 
useful in bone marrow grafting after cancer treatments, and anemias. 
Precision genetic modification could lead to better therapies for 
inherited blood cell disorders such as sickle cell anemia and 
infectious diseases such as AIDS.
    I would argue that the debate over the number of human ES stem cell 
lines approved for Federal funding largely misses the point. Human ES 
cells obtained from IVF preimplantation embryos are not identical to 
the patient, that is they are ``allogeneic''. We should expect that 
such cells derived from the 20-60 approved lines would be rejected by 
the patient's immune system. The primary purpose in funding human ES 
cell research is not just the pure pursuit of human knowledge, but 
rather to accelerate the delivery of novel therapeutics to afflicted 
people. We must address from the beginning how we are going to make 
these cells useful in transplantation.
                the use of nuclear transfer in medicine
    The recent success in the cloning of animals from body cells 
demonstrates that the transfer of a body cell into the environment of 
an egg cell can ``reprogram'' it back to an embryonic developmental 
state. We have recently demonstrated that such technology actually 
rebuilds the replicative lifespan as well, suggesting that ``young'' 
cells can be derived from ``old'' cells. This is a profound development 
and perhaps the ideal solution for making real the longstanding dream 
of transplantation medicine; namely, to be able to offer any patient, 
even an aged patient, young healthy embryonic stem cells of from which 
any kind of cell could be make all of which would be their own cells, 
not expected to be rejected by their immune system.
    Nuclear transfer offers an important solution of the problem of 
tissue rejection. Every year many thousands of people die for the 
inability to liver, kidney, or other tissue with the right 
constellation of markers to allow it to accepted by the body as self. 
It is estimated that three thousand people a day die from degenerative 
disease potential addressed by therapeutic cloning. This new procedure 
would begin with the patient donating living cells to a physician, who 
would then reprogram them back to a totipotent state using the cloning 
procedure. This is called therapeutic cloning, to distinguish it from 
reproductive cloning which is designed to clone an entire human being. 
Therapeutic cloning does not involve the cloning of a human being, it 
involves the medical use of cloning to make living cells. The cells and 
tissues made from these cloned stem cells would be expected to be 
grafted stably for the life of the patient without immunosuppression.
                  responses to concerns and objections
    (1) The preimplantation embryo is a human life and to use 
therapeutic cloning is to ``clone and kill''.
    Answer. In the first few days following the fertilization of an egg 
cell by a sperm cell, there develops a microscopic ball of cells called 
a preimplatation embryo. This embryo is destined to die unless it 
implants in the uterus to form a pregnancy. Indeed, it is estimated 
that 50-80 percent of these preimplantation embryos naturally formed in 
a woman's body never implant and therefore die, naturally. Prior to day 
14, the preimplantation embryo has no body cells of any kind, and, in 
fact, has no cells even committed to somatic cell lineages. Indeed, the 
embryo has not individualized. Once this ball of cells attaches to a 
uterus, one or even two or more individuals can form from it. It is 
therefore proper to say that it is not yet an individual. At ACT, we 
neither allow cell development beyond day 14, nor do we implant the 
cells in a uterus.
    (2) Therapeutic cloning is merely theoretical; there is no reason 
to suggest it will work.
    Answer. There are published reports of success of therapeutic 
cloning research in at least two mammalian species; namely mice (1-2). 
While never performed in a human, the animal data suggests that 
therapeutic cloning has great promise. The National Academy of Sciences 
has formally recommended in a report titled ``Stem Cells and the Future 
of Regenerative Medicine'' as follows:

    ``Recommendation: In conjunction with research on stem cell biology 
and the development of potential stem cell therapies, research on 
approaches that prevent immune rejection of stem cells and stem cell-
derived tissues should be actively pursued. These scientific efforts 
include the use of a number of techniques to manipulate the genetic 
makeup of stem cells, including somatic cell nuclear transfer.''

    (3) Allowing therapeutic cloning would cause a ``slippery slope'' 
effect, whereby regulating human reproductive cloning would not be 
possible.
    Answer. In reality the procedures to clone a human being are well 
known in the scientific literature. The widespread use of therapeutic 
cloning would not significantly increase the likelihood of the success 
of an effort to clone a human being. In addition, laws can easily be 
written to allow one and prohibit the other as reproductive cloning 
requires the transfer of a cloned preimplantation embryo into a uterus.
    (4) Therapeutic Cloning will lead to ``embryo farms''.
    Answer. Therapeutic cloning guidelines could easily be constructed 
to limit development to less than 14 days as is the current practice 
with in vitro fertilization.
                                summary
    In conclusion, nuclear transfer and human embryonic stem cell 
technology offer novel pathways to develop lifesaving therapies that 
will impact the lives of millions suffering from such diseases as 
Parkinson's disease, diabetes, arthritis, heart disease, kidney 
failure, spinal cord injury, liver failure, skin burns, blood cell 
cancers, to name only a few. The gravity of this issue calls for a 
compassionate, reasoned, and dispassionate debate. History will judge 
us harshly if we as a society fail to recognize and deliberate 
carefully upon a medical technology that could so powerfully alleviate 
the suffering of our fellow human being.

    Senator Specter. Thank you, Dr. West.
    Dr. Cameron and Dr. Caplan have a joint issue on the core 
concern, with Dr. Cameron being opposed to research on stem 
cells, and extracting stem cells from embryos, and Dr. Caplan 
raising the counter factors, and by saying that many of these 
embryos cannot produce life, and they are going to be either 
destroyed or permanently frozen.
    Dr. Cameron, what is your response to that basic point, 
that for physiological reasons, many of these embryos cannot 
produce life, or will be destroyed? Why not use them to save 
lives?
    Dr. Cameron. Well, Senator, one or two reflections. One is, 
Dr. Caplan in fact kept using the term person. I may have used 
the term once, but I am making no case here that the early 
embryo is a person. A person is a legal concept. It is also a 
complex philosophical complex. My point is that the early human 
embryo is a member of the human species. Plainly, it is 
undifferentiated. I mean, plainly, it has yet to go through a 
whole series of transformations to become----
    Senator Specter. The early embryo is a member of the human 
species?
    Dr. Cameron. That does seem to me a matter which is common 
to our understanding of the biology here, and in that context, 
plainly, certain embryos will not flourish, either because they 
are deformed, or because they are prevented from being in the 
maternal in utero environment in which they would flourish.
    But mortality is a universal principle, and it is no 
comment on the dignity of any human individual that every human 
individual will die, and so it seems to me that for us to 
afford the earliest, youngest members of our species a dignity 
in which they are placed beyond the bounds of destructive 
experimentation is a fundamental component in our civilized 
vision of human dignity.
    Senator Specter. So you characterize the embryo, even if it 
has no potential to develop into a baby, a child, not in the 
womb of a woman, but as a member of the human species in that 
form, and therefore you think morally it is inappropriate to 
extract the stem cells. That is the essence of your concern.
    Dr. Cameron. It is the essence of my concern, and I make 
one further comment, Senator. We use the word potential very 
freely in this discussion, and it can mislead us, because there 
are several different senses of potentiality, and my 
understanding--I am not a biologist, but my understanding is 
that every advance we have in modern genetics has clarified our 
understanding that the potentiality is in-built. Given a lack 
of malformation, given an appropriate environment, the embryo 
will, indeed, go on to become someone who is palpably one of 
us.
    Senator Specter. But if you have malformation--you say, two 
factors, the absence of malformation and the environment, the 
womb of a woman, and if you have malformation, then what?
    Dr. Cameron. Well, if you have--I mean, as every case of 
disease and deformity, death will come sooner, but the 
continuity is stronger than every discontinuity.
    Senator Specter. So you think even with malformation you 
have a human specie which ought to run whatever course nature 
gives it.
    Dr. Cameron. Indeed, sir, and we have, of course, accepted 
principles for human experimentation and also for the use of 
human cadavers, involving consent issues and so on. It seems to 
me that the earliest members of our species need to fit within 
that framework of ethical understanding.
    Senator Specter. And in the absence of an environment, the 
womb of a woman, still you say, let nature take its course, or 
whatever happens to that form of human species?
    Dr. Cameron. I am not happy that we bring into being human 
embryos who do not have the prospect of a maternal environment, 
but given that we do bring into being for all sorts of good, 
and now bad, reasons, it seems to me they need to be treated 
with a dignity which relates to who they are in themselves, and 
not to the context in which we place them.
    Senator Specter. Dr. Caplan, I am sure you have some 
observations on our discussion with Dr. Cameron just now.
    Dr. Caplan. Well, I would not refer to frozen embryos as 
potential members of the youngest members of our species. I 
still believe that they are possible members, but in actuality, 
what has been stored because it did not look right, what has 
been kept for more than 4 or 5 years, which most of these 
100,000 embryos have now been, has no chance of having its 
potentiality actualized.
    Senator Specter. No chance of----
    Dr. Caplan. No one that I know of who operates an 
infertility clinic would say, if you want to have a baby, I am 
going to get a 5-year-old frozen embryo that probably was put 
aside because it did not look right, and make that the basis 
for my attempt to give you a child.
    Senator Specter. Well, all right, not a baby, but Dr. 
Cameron has a very--has a different definition of the human 
specie. How do you deal with that ethically?
    Dr. Caplan. I would distinguish between a blueprint and a 
house. I think that embryos carry instructions. They tell us 
things about how a house might be built. They tell us things 
about what sorts of ways to arrange the parts and the wood and 
the nails and the roof and so forth, but they are not houses.
    So from my point of view, when we are talking about 
potentiality, when we are talking about possibility, what 
embryos become are instruction manuals, and we can actualize 
them, we can put them in the environment in which the other 
requisite components will then be manipulated to make 
something, but basically what I would say is, it is more akin 
to a situation where you have possibility, as a blueprint 
inherently might, in the right circumstances, be used to turn 
into something like a house or a building.
    And then if you say, but your blueprint is damaged, and it 
is deteriorating because you have not used it, and you have 
kept it in a circumstance when it is going to lose some of its 
information, and when it was actually made it came from someone 
who unfortunately produced embryos that were somewhat 
deficient, I think the potentiality is wavering, so I do not 
accept the idea that those entities are members of the human 
species membership.
    Senator Specter. Dr. West, on therapeutic cloning, can the 
entity created be implanted in the womb of a woman and produce 
a baby?
    Dr. West. That is a very large question, and certainly, I 
think----
    Senator Specter. What kind of question?
    Dr. West. It is a very large question you have asked. I 
think if you asked it in more specific subsets, is it possible 
to clone a human, so is it possible to make a cloned 
preimplantation embryo and put it into the uterus of a woman 
and create a pregnancy, we think it is possible. It has never 
been done.
    Some species, despite repeated attempts, have not yet been 
cloned, and so we do not know how easy it would be to obtain a 
human pregnancy.
    Senator Specter. You say some species have not been cloned. 
We have had Dolly cloned, the sheep.
    Dr. West. Right, but I mean----
    Senator Specter. Anything else cloned?
    Dr. West. Well, it took years to clone a pig, despite 
repeated efforts, although that now has been cloned as well.
    Senator Specter. A pig?
    Dr. West. Pigs, yes.
    Senator Specter. P-i-g, pig?
    Dr. West. Pig, as in oink, oink, pig.
    Some species have been more difficult. It is purely 
speculation as to how easy it would be to take a pre-
implantation--cloned, as we say, reconstructed----
    Senator Specter. Run through the stages of therapeutic 
cloning which could lead to the cloning of a person.
    Dr. West. Right, so the most straightforward would be 
simply to recapitulate the work in the cloning of animals for 
therapeutic purposes. This is the context that is normally 
described, but then I will briefly mention the various shades 
of gray, and I think this is the part of the debate that needs 
to be carefully considered.
    The first would be, the lessons from cloning is, you take a 
skin cell, put it inside of an egg cell whose DNA has been 
removed. The egg cell, like the computer, initializing a 
computer disk says, you are not a skinless cell. You are back 
in an embryo, you are young again. You get a pre-implantation 
embryo, as is made in IVF, but in the case of cloning we call 
these reconstructed embryos, and placed back into a uterus, and 
the embryo is healthy. You would then get implantation, and 
then individuation in the beginning of a cloned animal, with 
kidneys, heart, liver.
    So what this teaches us is, there are a pathway to take a 
skin cell, take it back in time, and then make the specific 
cells and tissues. The way we would envision doing it using 
that model, is, we take a patient that has diabetes, take a 
skin cell from them, use a donated human or potentially 
nonhuman egg cell to reprogram the cell, making the pre-
implantation embryo, making embryonic stem cells from that 
embryo, and then making all these cells and tissues for the 
patient. That is model one.
    But then there are at least a half-dozen other 
possibilities, some of which would not create an entity that I 
think most rational people would call an embryo, as evidenced 
by the fact they could not create a pregnancy.
    Senator Specter. Dr. West, let me give you the staff 
articulation of your company's research. Women were paid for 
egg donations and signed inform consent forms stating that 
their specimens would be used for research purposes. Scientists 
remove the nucleus, the genetic material from the donated egg, 
and insert genetic material from the skin cell of the patient, 
thereby creating the first cell of an embryo that is 
genetically identical to the patient. That constitutes the 
therapeutic cloning. Researchers can then coax the embryo to 
divide to become the cells of a heart, liver, or any other 
organ, and could be transplanted into the patient without a 
concern for rejection.
    Now, it is noted this has not yet been performed 
successfully, but is that the way you would clone an identical 
person to the woman who has donated the egg?
    Dr. West. That is a way, a very simple-minded one. There is 
other variations on this theme. Let me point out one very 
briefly to exemplify the numbers of possibilities in front of 
us, which have not been publicly discussed.
    Another technology would be to do the reverse procedure, to 
take bits of an egg cell, so take an egg cell, which I think 
everyone agrees is not a human life, to take an egg cell, 
remove its DNA, and then take small packages of the cytoplasm, 
the substance of the egg cell, which is capable of 
reprogramming a cell, and put those packages of protein, 
cytoplasm, on top of the patient's skin cell to take them back 
to an embryonic state in a way that would not create an embryo.
    So this is just one example of a host of possibilities, and 
to say that we are going to in a blanket way, and I think an 
unprecedented way, ban a whole field of medical research is 
unwise.
    Senator Specter. Well, if you come back to the cloning of a 
person, is the way that I described from my staff's summary a 
way realistically--I know it has not been done, but the 
potential for cloning a person who would be identical to the 
woman who gave the egg?
    Dr. West. Identical to the donor of the somatic cell, which 
is I think what you meant to say. The egg, the person who 
donated the egg would not be the clone, necessarily. The egg is 
a means of reprogramming a body cell, like a skin cell, so it 
is the person----
    Senator Specter. You say cloning is not necessarily 
identical to the donor.
    Dr. West. The donor of the genetic information, which goes 
into the egg cell, would be the individual cloned.
    Senator Specter. Would it be identical?
    Dr. West. It would for all practical purposes be an 
identical twin. The purists point out that there are slight 
differences.
    Senator Specter. Do you think that there ought to be a 
statute prohibiting that?
    Dr. West. When this came up for a vote in the Senate in 
1998 I testified to a hearing in the House on this, and I 
stated publicly I thought there should be a law against the 
cloning of human beings. Like all areas of science and 
medicine, you start realizing the suffering of patients, and I 
have softened somewhat on that position, but I think most of us 
in the field believe that there is this mountain of opportunity 
in medicine to have this in any way interfered with by this 
phantom of human cloning----
    Senator Specter. Well, do you still believe there should be 
a prohibition against cloning a human being?
    Dr. West. I think I would lean in that direction, although 
even there I would argue we should be slow-going. Reproduction 
is an important part of human life, and there are couples that 
cannot reproduce any other way. I would support such a----
    Senator Specter. Dr. Cameron, I am pretty sure of your 
answer, but state it for the record.
    Dr. Cameron. I am pleased to state it is yes. It seems to 
me that cloning a human being is something which should never 
have been allowed to happen. Certainly, there is such a wide 
consensus that we do not want a human baby born, but back of 
that, of course, we now have the possibility of industrial 
production of human embryos for research using the cloning 
technology.
    And since my original homeland of Scotland, I come from a 
few miles away from where Dolly was cloned, I feel a sense of 
curious pride in that extraordinary event, and yet have deep, 
deep apprehension as I see the technology being applied to our 
own kind.
    Senator Specter. Dr. Caplan, cloning people, what is your 
view?
    Dr. Caplan. Well, I think we should absolutely prohibit 
human cloning simply on the grounds that the animal data shows 
us that we would be doing something very unsafe and very 
dangerous. The toll in terms of stillborns, deformed animals in 
the cloning process--and to answer your earlier question--of 
cows, pigs, bulls, mice, has been terrible, and it would 
basically amount to unethical human experimentation, simply on 
the grounds that you would risk killing somebody if you were 
able to do it.
    I might add one other point here. I am not sure human 
beings can be cloned. I know the media loves to speculate about 
that, and in many ways if you listen to popular discussion 
people believe it has already been done, or it will be done 
soon. There is every reason to think that some of the problems 
using bull DNA from body cells, and the handshake problems 
between egg and DNA may make it impossible to clone ourselves.
    So I would say ban it. I am not sure that it is possible, 
but the way I think it could be prohibited and controlled is to 
simply say, you may not put any cloned human embryo into a 
woman's uterus, or attempt to try and grow it for the purposes 
of reproduction.
    Senator Specter. It is 11:00. We are going to have to take 
a very short break, just about 2 or 3 minutes.
    Dr. McGee, what are your views on human cloning?
    Dr. McGee. Well, I find myself in agreement both with Dr. 
West and Dr. Caplan in that I think it is clearly a----
    Senator Specter. How can you be in agreement with both of 
them? Dr. West said he's leaning, Dr. Caplan's opposed. We are 
going to come back to Dr. West to see the degree of leaning.
    Dr. McGee. Let me think about that, but I think it is clear 
that the question as to whether or not there should be a 
prohibition on clinical reproductive cloning of a human, an in 
vitro fertilization clinic making a human clone is somewhat a 
detraction. I mean, there is no evidence that----
    Senator Specter. A detraction?
    Dr. McGee. A detraction.
    Senator Specter. A detraction.
    Dr. McGee. Yes. I think it is clear that it would be 
extraordinarily difficult to do it, and that the mounting 
debate on that question has really drawn attention away from 
stem cell research, which is the much, much more important and 
likely development in the short-term future.
    Moreover, I think as was discussed the first time this was 
brought up----
    Senator Specter. There is no doubt that the arguments for 
stem cell research, those who propose it are quickly in the 
sound bite dialogue of Sunday morning talk shows, has moved 
over to cloning. Then you move from therapeutic cloning to 
cloning people, and you start to get on very dangerous ground 
politically. It would be pretty hard to get stem cell research 
if people are equating it with cloning, but that is why it is 
important to have clarification. We may spend more time at this 
hearing than the House of Representatives did before having 
400-plus people vote on it. But go ahead, Dr. McGee. Where do 
you stand?
    Dr. McGee. I think I agree with you. I guess my position is 
that I believe it is not--I think it is not a bad idea to ban 
reproductive human cloning because of the obvious safety 
concerns and the continuing national debate about whether or 
not it is appropriate.
    Senator Specter. It is not a bad idea.
    Dr. McGee. I think, though, that because there is no 
imminent danger of a human clone being produced, media 
attention to the contrary, as Professor Caplan suggested, that 
it might be better to concentrate, as you have this morning, on 
how to describe what it is that is being done in stem cell 
research.
    I mean, this notion of a therapeutic clone is a misnomer, 
in my opinion, and what we are really talking about, as Dr. 
West pointed out, is what you call what you have made when you 
use nuclear transfer. Professor Caplan and I have called this 
from time to time the what's-in-the-dish problem. That is, if 
you, as Dr. West just suggested, organize the bulk of an egg 
around an adult cell, you will have produced what many 
embryologists would call an embryo. Some might not.
    The question is, who is to define that, and it is not a 
simple, scientific problem. It is clearly one that is very 
complicated, and I do not think Dr. West is right in saying 
that every rational person would agree that nuclear-transfer-
derived embryos are not embryos at all.
    Senator Specter. Well, you lost me on your last answer. Do 
you want to repeat it?
    Dr. McGee. I think the problem to be discussed about 
therapeutic cloning is the problem of how to responsibly 
respect the view of those----
    Senator Specter. Are you saying that therapeutic cloning 
cannot be differentiated on a bright line from human cloning?
    Dr. McGee. Yes. I think--well, I think there is one clear 
bright line, and it is the one Dr. Caplan identified. That is, 
something is or is not implanted in a uterus.
    Senator Specter. Well, what is your view on therapeutic 
cloning?
    Dr. McGee. I think it would be very difficult to define it, 
and the reason for that is because what is at stake in the 
debate about funding, as I understand it, funding stem cell 
research, is when you have an embryo, not when you have a 
clone, and so many of the technologies that are being developed 
in small companies right now, members of ethics advisory boards 
for those companies, for example, have been quoted to the 
effect that they are not making embryos, that they are really 
making, to quote one chair of an ethics advisory board, an 
activated egg, and I think it is very difficult--I think it is 
unfortunate that that is being captioned as a problem for 
industry.
    Senator Specter. Dr. Caplan, what is your view on 
therapeutic cloning?
    Dr. Caplan. I think we should absolutely prohibit any 
insertion of a clone into a human uterus, that any attempt to 
produce or derive cloned human cells for reproduction should be 
prohibited. I think we should not at this time prohibit 
therapeutic cloning for research purposes. I think that that 
avenue should be left open.
    Senator Specter. Define therapeutic cloning, what you think 
should be permitted.
    Dr. Caplan. I think we should at the present time allow 
scientists to transfer DNA from body cells into nucleated eggs 
of human beings, allow them to start to grow those entities to 
see what happens. I do not think we know very much about their 
potential. I do not think we know very much about what they can 
offer. I think that anyone who would put such a creation inside 
a human being, a woman, to try and turn that into a person, 
should be prohibited and restricted from doing so.
    And as my colleague Dr. McGee was saying, we know from 
Dolly that we can transfer DNA from your skin, from your lip, 
into an egg, a human egg from which the DNA has been taken out, 
and we can put that DNA in, but there are other ways to make 
DNA and egg material come together. That is what Dr. West was 
also starting to talk about. I would allow that research for 
the time being under very tight control, very tight regulation, 
to make sure that no one ever attempted to undertake human 
reproduction using any of these techniques.
    Senator Specter. They have just rung the bell for a vote, 
so I think it might be a good idea if we moved to the final 
subject on the agenda, and that is the issue which was joined 
by Dr. McGee and Dr. West on this business about the advisory 
board.
    Dr. West, what do you think about Dr. McGee's enumeration 
of standards for an ethics advisory board?
    Dr. West. Well, I appreciate Dr. McGee's concerns. What I 
attempted to do back in the mid-nineties, when I initiated this 
work in this area with Geron first, and then later now Advanced 
Cell, was put in place, ethics advisory boards in both cases, 
to advise us, where we were blindsided to provide support in 
charting the course through these uncharted waters, and we 
purposely--I have historically invited people, without knowing 
their perspectives, people who were opinionated, who had the 
power of the pen--I did not feel in the corporate concern we 
could give them veto power, because there are certain fiduciary 
duties corporations have, but we gave them the power of the 
pen.
    Senator Specter. Dr. West, let us move toward the core of 
the controversy that you had with Dr. McGee. I would like to do 
it in a more leisurely way, but once the votes start it is very 
hard to reconvene. Dr. McGee, what were your specific 
objections that led you, as I understand it, to resign from the 
ethics advisory board of Dr. West's company?
    Dr. McGee. Well, the straw that broke the camel's back for 
me was that I was involved in discussion, public discussion 
about the cloning of an endangered, rather distinct animal, the 
gauer, by Advanced Cell Technology. I did not know at the time 
I was discussing the question about this animal cloning project 
with a number of members both of my profession and of the media 
that Advanced Cell Technology had done the work. They kept it 
secret even from the ethics advisory board, despite the fact 
that this was very important, and very controversial work.
    Senator Specter. Dr. West, is it true that it was kept 
secret from the ethics advisory board?
    Dr. West. No. The chairman of our ethics advisory board, 
Ryan Green, did know. I apologize to Glenn. I simply felt that 
the cloning of an endangered animal was a humane and good use 
of technology, and it did not occur to me that that was a 
matter of urgent concern for our EAB, and so we did not discuss 
it with every member of our EAB, and that was wrong.
    Senator Specter. How many members of the ethics advisory 
board, Dr. McGee, are there?
    Dr. McGee. Well, as I noted in my comments, Senator, I 
think that is part of the question, because ethics advisory 
boards do not operate with any kind of disciplinary or 
professional or Federal or State standards. They operate in 
very different ways, and so the Advanced Cell Technology ethics 
advisory board's membership is kept secret, so I honestly do 
not know how many members there are, or their identities.
    Senator Specter. Dr. West, how many members?
    Dr. West. Nine, currently.
    Senator Specter. Dr. Caplan, I am advised that you had 
served on the Advanced Cell Technology scientific board, and 
what was your experience there?
    Dr. Caplan. Well, I served for a limited time. They did not 
choose to call upon me for any comment or advice, so I left. It 
was not out of any disagreement, disappointment. I just felt 
they were not using the board, so I decided that that was not a 
good use of my name or time.
    But I should add that I support the idea of ethics advisory 
boards. I think they are good when companies try to create 
them. I do not, however, personally believe that they are a 
substitute for Government oversight. They are commendable. It 
is great to get opinion. It is nice to have input. It is a nice 
thing, but I see them as no substitute for what this 
subcommittee is trying to accomplish.
    Senator Specter. The staff reports to me were that you 
resigned from the ACT scientific board because you were not 
consulted. You felt you were being used as a front man. Is that 
an accurate characterization?
    Dr. Caplan. There is a danger of having your name out there 
when you are not consulted, so I was worried about that.
    Senator Specter. Response, Dr. West.
    Dr. West. Only that we invited Dr. Caplan to be on our 
scientific advisory board, and he left before we had convened a 
scientific advisory board meeting.
    Senator Specter. But as to the experience you had with Dr. 
McGee, you say you should have consulted him. That was an error 
on the part of the company.
    Dr. West. Yes. We feel that cloning can be used to save 
endangered animals without bringing them into captivity, so you 
can literally take a cell from an animal in the wild and we 
thought this was a humane use of technology, and it frankly did 
not occur to me that it was a priority issue for our ethics 
advisory board, but they certainly should have been notified, 
and that was an oversight on our part.
    Senator Specter. Now, Dr. West, what is your view as to Dr. 
Caplan's contention that there ought to be governmental 
regulation?
    Dr. West. Well, in the corporate sector, of course, we 
always worry about such regulations, both because we like to go 
out and change the world and do not like to be slowed down, and 
of course secondly there is the issue of undue intervention in 
the business sector as a matter of principle.
    Given the importance of this area, however, we are far more 
interested in helping people who are sick and seeing this 
technology fully utilized, and I think there is a great 
eagerness, at least on our part, to find a pathway to benefit 
people who are sick.
    Senator Specter. So you think appropriate governmental 
regulation would be warranted?
    Dr. West. Absolutely.
    Dr. Cameron. Senator, could I add a comment here, please?
    Senator Specter. Sure.
    Dr. Cameron. It does seem to me that one matter common to a 
number of us here is the enormous importance of developing a 
regulated regime.
    The huge changes taking place in the last 10 years with the 
privatizing of major areas of bioscience out of the public 
domain, out of the major universities, into corporations, means 
that the use of ethics advisory boards as private, essentially 
public relations advisory opportunities for companies, perhaps 
well-intentioned, but that is the net effect of having these 
names on their letterhead, can be quite misleading, and 
certainly is no substitute for the development of a regulatory 
regime which will build public confidence in what the biotech 
industry is seeking to do.
    This seems to me to be crucially important. We have to have 
transparent ethical frameworks leading to regulation within 
which these activities are carried on.
    Senator Specter. The time has almost expired on the vote, 
so I am going to have to excuse myself, and there are some 
other issues that I want to take up, so we will stand in 
recess, and I will return as fast as I can, hopefully within 15 
minutes.
    We will resume.
    Dr. West, I have just been advised that you were explaining 
during the break about a patent which you had obtained.
    Dr. West. I am sorry, would you remind me? I am sorry, I do 
not remember, which patent?
    Senator Specter. It only happened 5 minutes ago.
    Dr. West. I know, I am sorry. Which patent?
    Senator Specter. Do you want the question to be more 
specific?
    Dr. West. A patent which we have obtained?
    Senator Specter. I will try to find out more of the 
details. I just got a thumbnail on it.
    Ms. Taylor. You were explaining to a reporter where you 
would take an egg, an unfertilized egg, crack open that egg----
    Dr. West. Oh, I am sorry, yes. You said patent you had 
obtained, and that confused me. We have a patent application 
pending on an alternative to the traditional way of thinking 
about the medical uses of cloning.
    The alternative is, you know, it is turning the arrow in 
reverse. When we talk about nuclear transfer, which is the 
scientific term used for cloning, we are referring, again, to 
recapitulate, an egg cell, remove its DNA, and then we do 
nuclear transfer. We take the genetic information from, 
typically, a body cell and put it into the egg cell, and that 
is why it is called nuclear transfer.
    What occurred to our scientists is, it may be possible to 
do the reverse, turning the arrow in the other direction and 
doing O plasmic transfer so--but this is still largely 
theoretical, and the reverse would be taking the proteins in 
the egg cell that do the work of reprogramming, packaging them 
up in like, let us say, water balloons, and dropping them on a 
patient's cell, so it is the reserve transfer, it is plasmic 
transfer of O plasm, being the material of the egg cell.
    That would lead to, we believe, undifferentiated cells in a 
flat layer on a dish, which although may be embryonic in the 
sense that they express the genes of the early embryo, would 
not have the architecture of an embryo, would not create a 
pregnancy if put into a uterus.
    Senator Specter. Let us come back to the issue as to 
governmental regulation. Dr. Caplan, what do you think a 
statute should provide by way of regulation?
    Dr. Caplan. Well, I think the key features to look for in 
terms of overall oversight of therapeutic cloning stem cell 
research are first, openness and transparency. I think that one 
of the problems we have is that much of the commercial work is 
done in private. It is up to the company to release it. We 
already encountered some issues about price and availability of 
license, the earlier comments.
    Senator Specter. What should be done in the regulation as 
to price or availability of patents?
    Dr. Caplan. I think basically the regulation should drive 
to maximize the availability of the good from these techniques 
by making sure that prices are reasonable and affordable and, 
most particularly----
    Senator Specter. Well, how can you do that?
    Dr. Caplan. Well, most particularly by no charge to persons 
who are doing research, pure research without intent to 
commercialize or manufacture.
    Senator Specter. Well, this, of course, is my field, not so 
much yours.
    Dr. Caplan. Yes.
    Senator Specter. I do not know that you can legislate to 
somebody who has a property right that there will be no charge.
    Dr. Caplan. Well, I think if the goal is simply to 
undertake work for scientific knowledge, we would want to make 
sure that without that commercial intent, then that information 
should be broadly available.
    I might add, Senator, you recall with the human genome the 
same issues were raised about patenting genes, making them 
available for research purposes, so I will defer to your vast 
expertise much more than mine about what can be done. What I 
would like to see happen is roadblocks to basic research taken 
down or minimized.
    The second thing I would like to see is some guarantee that 
we do not have any embryos at the present time manufactured. I 
think what the Jones Clinic did in making embryos for 
production is not necessary, is wrong. I would like to see 
regulatory oversight there about where material comes from.
    I would also like to see regulation, if you will, that 
makes sure that we do not have redundancy in research, so that 
if we are going to try and use these resources carefully and 
respectfully, we use them in the best hands, that we make sure 
that the peer review is absolutely solid on the science end, 
and that we are not allowing for unnecessary duplication, if 
you will. We want to try and keep what has to be done to a 
minimum.
    I think the last area of regulation that concerns me is 
that we take a long, hard look at present at what our patent 
policy should be in this area. We have got some claims made. 
Fair enough. There are other techniques emerging. Some of what 
Dr. West is talking about may move around certain patents that 
allow certain therapeutic cloning techniques to take place, but 
I would like to see the NIH role clearly established and 
articulated, so that a public interest is served in terms of 
not holding up the potential of stem cell research.
    Senator Specter. What would you like to see this 
legislation say as to the NIH role?
    Dr. Caplan. Hold any new patents, control them, basically 
make them available, that its funding is going to be contingent 
on availability.
    Senator Specter. Dr. Cameron, anything you would suggest on 
legislation?
    Dr. Cameron. Well, it seems to me my position would be that 
in this area we should seek to protect the early embryo from 
this sort of treatment.
    I must say, I was--perhaps I could ask Dr. Caplan to 
clarify. Was he actually saying that the early embryo was not a 
member of the human species? This seemed to be the entailment 
of his remarks earlier, which I thought was somewhat 
extraordinary.
    Dr. Caplan. Yes. I do not think they are persons, and I 
think membership in the human species gives them an equivalence 
to adults, to you and me, that I do not accept.
    Dr. Cameron. I would certainly take the view that we should 
protect the early embryo, but in the context, and part of the 
problem with our having discussions in this way now, is that we 
need also to be grappling with the much wider issues involved 
in biotechnology, and with the privatizing of the biosciences.
    I mean, there has been almost no discussion in this context 
of where interests lie, of the way in which, for as what ACT is 
saying, in speaking about helping patients, they are also, of 
course, speaking about helping their stockholders, and there 
are huge financial interests at stake in this industry which 
make it a very urgent matter for there to be a comprehensive 
regulatory regime which the industry has so far proved very 
effective at discouraging.
    Senator Specter. Dr. Cameron, thank you very much.
    Dr. Cameron. Thank you very much.
    Senator Specter. Yes. Dr. Gulbrandsen, do you agree with 
what Dr. Caplan said about free licensing for science? Why 
don't you pull your chair forward.
    Dr. Gulbrandsen. What WARF has tried to do, Senator, is 
make the embryonic stem cell as freely available for research 
purposes as we can. We thus far have distributed this stem cell 
to over 30 researchers in institutions around the world. We 
have ongoing negotiations with about 100 additional material 
transfer agreements that are close to finalizing, and those 
will be provided to researchers.
    Senator Specter. Are they provided to researchers without 
cost?
    Dr. Gulbrandsen. They are provided to researchers with a 
one-time-only fee of $5,000, to try and help cover expenses. It 
does not begin to cover our expenses. WiCell is truly a not-
for-profit organization. We are not making any money, and we 
have spent a lot of money on WiCell, and the purpose of that is 
to distribute this material.
    Senator Specter. How about WARF? Is WARF making money?
    Dr. Gulbrandsen. WARF has not made any money from WiCell or 
from the material transfer agreements that have been 
distributed. These have been made available below cost, and we 
will continue to do that.
    Senator Specter. Let me just cover one other subject. When 
Dr. West testified about what you had sought on a license from 
WARF, would you amplify what your answer was to that?
    Dr. West. Well, in 19--I believe it was 1995, I approached 
first the scientist in Wisconsin, Jamie Thomson, and then WARF, 
about the possibility of sponsoring research in an attempt to 
obtain the human embryonic stem cell based on Dr. Thomson's 
expertise in deriving these cells from nonhuman primates, and 
as is typical in biotech, in fact nearly universal, is 
universities and companies can come to an understanding that if 
the university--sorry, if the company puts forward, for the 
company what is a lot of money, maybe hundreds of thousand of 
dollars for a speculative project, that the company would be 
rewarded with an exclusive license for the life of the patent.
    Unlike all of my previous experiences, maybe 20 or 30 
licensing arrangements with universities, WARF from the very 
beginning refused, so having stayed 2 or 3 days in a hotel in 
Madison, I left at that time without an exclusive license.
    Senator Specter. Why was a nonexclusive license 
insufficient for you?
    Dr. West. Why was it insufficient?
    Senator Specter. Yes.
    Dr. West. Well, the concern is, of course, in biotech you 
need to be able--despite the cynicism about profit-making, you 
know, biotech is a very speculative business. Profits being 
maybe 10 years away, hundreds of millions of dollars in the red 
before you can make a profit. Investors want to know you have 
some insurance against unfair competition, so exclusivity is 
critical.
    Given the importance of the technology, I was willing to 
accept a nonexclusive arrangement. That has subsequently been 
amended.
    Senator Specter. You did get a nonexclusive arrangement.
    Dr. West. Originally. The agreement between the university 
and Geron was subsequently amended after I left Geron, and I 
could not speak to the current arrangement.
    Senator Specter. Dr. Gulbrandsen, what was WARF's thinking 
on the rejection of the request for an exclusive license?
    Dr. Gulbrandsen. Again,the concern was wide availability of 
this material for research purposes, and not being able to 
commit ourselves at this time that an exclusive license would 
be an appropriate way to move forward with this technology.
    Senator Specter. How did Geron fit into that, exactly?
    Dr. Gulbrandsen. I am not quite sure I understand what your 
question is. Geron provided the--when Dr. Thomson had succeeded 
with the rhesus monkey embryonic stem cells, Geron arrived and 
said, can you repeat this type of research with a human 
embryonic stem cell, and we were unable to use any Federal 
dollars for that, and so Geron provided critical funding at a 
critical time.
    Senator Specter. Anybody care to add anything?
    Well, thank you all very much. We are certainly on the 
frontier here, or maybe a little beyond.
    Dr. Gulbrandsen. Senator, I would like to add one other 
comment.
    Senator Specter. Sure.
    Dr. Gulbrandsen. And that is, I do not believe in any way 
that patents have impeded anything here. The greatest 
impediment in this research has been the lack of Federal 
funding, and if we had Federal funding, we would see this 
research advance much quicker than it is right now.
    Senator Specter. Well, there are many of us who are working 
on that very hard----
    Dr. Gulbrandsen. Thank you very much.
    Senator Specter (continuing). Trying to get that 
accomplished. We have added $8.5 billion to NIH, and we are 
going to come up to, I think, $12 billion in fiscal year 2002, 
and there is a lot of interest, and I think NIH has done 
spectacular work. There are fascinating horizons on stem cells, 
and I am aware, as Dr. Cameron has articulated, of ethical 
concerns, and I think it has been a very pointed discussion 
between Dr. Cameron, Dr. Caplan, Dr. McGee and Dr. West, and 
also between Dr. West and Dr. Gulbrandsen.
    It has been an interesting hearing, and I think a 
productive hearing, and I think we have exceeded the 2-hour 
debating time which the House undertook before voting on this 
measure.

                          SUBCOMMITTEE RECESS

    Thank you all very much for being here, that concludes our 
hearing.
    [Whereupon, at 11:53 a.m., Wednesday, August 1, the 
subcommittee was recessed, to reconvene subject to the call of 
the Chair.]


                               STEM CELLS

                              ----------                              


                      WEDNESDAY, OCTOBER 31, 2001

                           U.S. Senate,    
    Subcommittee on Labor, Health and Human
     Services, and Education, and Related Agencies,
                               Committee on Appropriations,
                                                    Washington, DC.
    The subcommittee met at 9 a.m., in room SD-124, Dirksen 
Senate Office Building, Hon. Arlen Specter presiding.
    Present: Senator Specter.

               OPENING STATEMENT OF SENATOR ARLEN SPECTER

    Senator Specter. Good morning, ladies and gentlemen. It is 
9 a.m., and we will proceed with this hearing of the 
Appropriations Subcommittee for Labor, Health, Human Services, 
and Education.
    This is the 11th hearing of the subcommittee on the issues 
relating to stem cells. Shortly after stem cells burst upon the 
scene in November of 1998, this subcommittee held a hearing to 
explore their potential in early December of 1998, and in the 
interim we have inquired into many of the facets of this 
remarkable procedure for combatting major diseases confronting 
medical science.
    There has been a considerable amount of controversy by some 
who claim that by extracting stem cells from embryos, that 
there is the destruction of lives. The fact of the matter is 
that there are many embryos created for in vitro fertilization, 
and only a few are used, and the rest are discarded. If there 
were any possibility that these embryos could produce lives, I 
certainly would be opposed to the destruction of any embryo 
that produced the stem cells, but that is not the fact.
    In the bill on Labor, Health, Human Services and Education, 
we have appropriated $1 million as a start for stem cell 
adoption. If there is any opportunity for these embryos to 
produce human life, and there are those who would be willing to 
adopt the embryos and take the next step forward, that is 
something which certainly ought to be encouraged. This 
subcommittee is very anxious to promote that.
    However it is calculated, there are many of these embryos 
which will end up being discarded and it is my view, and the 
view that has been expressed based on subcommittee's findings, 
that these embryos which are to be discarded ought to be used 
to save lives.
    The President made a decision that existing stem cell lines 
as of 9 p.m. on August 9 could be federally funded, and 
immediately thereafter the questions arose as to whether those 
stem cell lines were sufficient to carry on the requisite 
scientific research, and we are proceeding today to make 
further inquiry into that subject.
    The appropriations bill for Labor, Health, Human Services 
and Education is on the floor today. After this hearing was 
scheduled the majority leader scheduled the Labor-HHS bill for 
floor action, so we are going to have to proceed in a very 
strained time frame to conclude this hearing by 10:15, and so 
my opening statement will be similarly abbreviated.
STATEMENT OF DR. WENDY BALDWIN, DEPUTY DIRECTOR FOR 
            EXTRAMURAL RESEARCH, NATIONAL INSTITUTES OF 
            HEALTH, DEPARTMENT OF HEALTH AND HUMAN 
            SERVICES
    Senator Specter. At this time we are going to turn to Dr. 
Wendy Baldwin, the Deputy Director for Extramural Research at 
the National Institutes of Health, a graduate of Stetson 
University with her bachelor's, and a Ph.D from the University 
of Kentucky. Prior to obtaining her present position, Dr. 
Baldwin served as Deputy Director of the National Institute for 
Child Health and Human Development at NIH. We are going to ask 
all the witnesses to abide by the 5-minute limitation so that 
we can conclude the hearing, as I said, by 10:15.
    Welcome, Dr. Baldwin. We look forward to your testimony.
    Dr. Baldwin. Thank you, Senator Specter. I am pleased to 
appear before you today to inform you about steps the NIH is 
taking to implement the President's decision to commit Federal 
funding for research on human embryonic stem cells. As you 
know, on August 9, President Bush opened the door for Federal 
research in this very important, exciting, and promising area 
of research, and since that day, we have been laying the 
groundwork so that Federal funding for such research can begin.
    We have met with the investigators and the providers of 
stem cells, and we have been taking many different steps to get 
this process underway and to let the research community know 
how to interact with the NIH and how to avail themselves of 
this new opportunity in science.
    One of our first actions was to address the issue of the 
licensing agreement with the holder of the U.S. patent on 
embryonic stem cell technologies. On September 1, the Public 
Health Service signed a memo of understanding with WiCell 
Research Institute. This is a very important step, and while 
that agreement was for the PHS researchers, in fact it sets the 
stage for how researchers all around the world will be able to 
access this technology and be able to do the research.
    That MOU is available on our web site. This is not only a 
tremendous step forward for that particular set of cell lines, 
but we think it also sets the standard. We expect that is going 
to serve as a model agreement. That is very important step 
sence people have to be able to get access to these lines.
    Our next task was the establishment of a web-based human 
embryonic stem cell registry, which would list all of those 
lines that were eligible under the President's policy 
announcement. This registry has to be something that not only 
reflects that policy statement but is also useful to the 
investigator community. Right now, we are working on getting 
that tuned up, getting information available, making sure that 
it is presented in a way that the research community will know 
who to talk to and how to get access.
    Once that registry is in place, we are going to begin 
funding research in this area. While the beginning of this 
research activity had a very unusual beginning--with a 
presidential statement--it is my goal to mainstream our support 
for research on human embryonic stem cells as soon as possible.
    We have had a number of things to do. As we talked with the 
derivers, it was clear that some needed infrastructure support. 
The cell lines have to be functionally available, and there may 
be infrastructure needs.
    We have also heard about needs for technical assistance. 
Again, that will vary by different investigators, but it is 
something we must pay some attention to.
    We are looking also for ways to minimize administrative 
burden. Getting access to these lines is much like getting 
access to other research resources. We feel that there are 
steps we can take to minimize some of the administrative 
burden, if only to provide easy access to the guidance that 
people might need. If they need to import cell lines, they need 
to consider FDA, USDA, and CDC regulations. We think we can be 
helpful there to them. We are going to add information like 
that to our web site.
    We know that the cell lines can be challenging to work 
with. We can provide a variety of training opportunities, not 
just for the external community but also for our research 
administrators who will have new issues to deal with as grants 
seek funds to do research with these cell lines. So there is 
much to do. Over the last few months, this has moved the NIH 
from an area of policy development to implementation. That is 
really why I am here today, because it is in my office that we 
deal with the different hurdles we have to get over before we 
can actually make grant awards in this arena.
    We are going to work within the framework of the 
President's statement. But within that framework and with our 
registry posted, investigators will be able to use the full 
range of funding mechanisms. They will be able to apply all 
their knowledge and skills of dealing with research resources 
and dealing with application procedures and review and funding 
issues, so we are very pleased that this is going to give us an 
opportunity to really mainstream this work.

                           prepared statement

    We expect to make our first grant awards in 2002. 
Investigators who are already funded can use our options for 
administrative supplements or even rebudgeting to begin work 
quickly. We are eager to explore the enormous scientific 
opportunities here. There are many steps that have to be taken 
so that we can make this resource available in a way that is 
truly useful to the research community and provides enough 
guidance for them that they actually know what to do to 
interact with the NIH.
    So there is a great deal of work to do, and we are poised 
to do it, and I would be happy answer any questions that you 
have.
    [The statement follows:]
                Prepared Statement of Dr. Wendy Baldwin
    Mr. Chairman, Senator Specter, and Members of the Committee, I am 
pleased to appear before you today to testify about NIH's progress 
toward the implementation of the President's decision to permit federal 
funding for research using human embryonic stem cell lines.
    As you know, on August 9, President Bush opened the door for 
federal support of this exciting and promising area of research. Since 
that day, NIH has been laying the groundwork so that federally-funded 
research can begin. We have met with the investigators from all over 
the world who are responsible for development of the existing stem cell 
lines that are eligible for federal funding. They are cooperating with 
the NIH to create a framework for researchers to conduct much needed 
basic research on these cells.
    One of our first actions was to address the issue of licensing 
agreements with the holder of the U.S. patent of human embryonic stem 
cell technologies. On September 4, the Public Health Service (PHS) 
signed a Memorandum of Understanding (MOU) with the WiCell Research 
Institute of Madison, Wisconsin, (a subsidiary of WARF) for use by PHS 
researchers of WiCell's five existing human embryonic stem cell lines. 
The MOU permits PHS scientists, such as those working in the NIH 
intramural program, to publish freely the results of their research and 
permits the PHS to retain ownership to any new intellectual property 
that might arise from the conduct of such research. In addition, the 
MOU provides a ``Simple Letter of Agreement'' to govern the transfer of 
cell lines to individual laboratories with minimal administrative 
burden. Furthermore, WiCell has agreed to make stem cells available to 
PHS grantees under the same terms and conditions as those provided to 
PHS scientists. This agreement represents an important first step in 
allowing investigators to obtain the cells for their laboratory work 
under terms that are consistent with NIH's policy of shared research 
resources. We expect the agreement with WiCell will serve as a model 
for additional agreements with other sources of stem cell lines.
    Our next task was the establishment of a web-based Human Embryonic 
Stem Cell Registry to list all of the cells that meet the eligibility 
criteria. The registry will include information about all the cell 
lines that are currently available for research. In order to establish 
the on-line registry, we have been obtaining assurances that all of the 
President's criteria were met, and gathering necessary information 
about these sources so that researchers can contact them directly for 
information. Thus, investigators will be able to discuss obtaining 
cells for their research and those who apply for federal funding can 
refer to one of the eligible sources in their funding application. I am 
pleased to announce that this registry will soon be operational and 
will be available to the public through the Internet.
    When the registry is in place, NIH will begin funding this research 
using a variety of mechanisms: grants, contracts, cooperative 
agreements, and supplements to existing grants. There are many exciting 
avenues of research to be explored, and there certainly are many 
investigators prepared to apply for funding. In addition, we are 
talking with the different providers of stem cells to determine what 
might be needed to establish a research infrastructure that ensures the 
successful handling and use of these embryonic stem cells in the 
laboratory. We may need to provide technical assistance and funds for 
the expansion of stem cell lines so that they are available to as many 
researchers as wish to use them. We will try to minimize the 
administrative burden, in regard to requests for the distribution of 
cells, both for scientists who have derived these cells and researchers 
who wish to use them. For example, when researchers import biological 
products many of these derivers are outside the United States they must 
follow USDA, FDA and CDC regulations. We will be adding information to 
our web site to facilitate this process. Because these cells are 
challenging to work with, we need to determine in what manner we will 
provide training opportunities to researchers on technical approaches 
to grow and maintain these cells in their own labs. NIH may hold 
workshops and conferences to encourage broad scientific dialogue about 
research ideas, and the identification and resolution of technical 
problems inherent to any new arena of research. There is considerable 
work to do.
    Over the past six months the NIH effort regarding research on human 
embryonic stem cells has moved from policy development to program 
implementation. While this is an unusual initiative in that it is based 
on a Presidential announcement, now is the time to bring this research 
into the mainstream of NIH funding activities. Working within the 
framework of the President's statement, investigators will be able to 
use the full range of funding mechanisms and begin to apply their usual 
practices regarding the acquisition and use of a research resource, in 
this case the stem cells listed on the NIH Registry.
    Mr. Chairman, NIH is now poised to begin funding this research. We 
expect that NIH will make the first grant awards in 2002. In some 
cases, investigators who are already receiving funding for projects in 
research areas that are closely related to embryonic stem cell biology 
may seek supplemental funds or permission from NIH to utilize existing 
funds in these closely related areas. We are eager to explore the 
enormous scientific promise of these unique cells. There is much basic 
research to do, and it is time to move forward.
    I am happy to answer any questions you may have.

    Senator Specter. Thank you very much, Dr. Baldwin.
    The September 7 issue of the Wall Street Journal reported 
that the in vitro fertilization clinic from which Reliance Life 
Sciences obtained their embryos was, as the Journal put it, the 
most mysterious in Bombay. The article claims that the clinic 
opened at the earliest in March of this year, and at the latest 
in May, and the article goes on to say the clinic, quote, would 
have to work unusually fast to produce excess embryos that 
quickly in 6 months. The question is, how did they obtain spare 
embryos in 6 months, when you can not have spare embryos until 
the donors have at least one successful pregnancy, which takes 
9 months.
    Are you familiar with that article, Dr. Baldwin?
    Dr. Baldwin. Yes, sir.
    Senator Specter. How do you explain it?
    Dr. Baldwin. Well, we have interacted with every one of the 
providers, including Reliance. Reliance is a very large entity. 
It is not a quickly or recently formed entity. We have an 
assurance from them that they have, in fact, met the 
President's criteria.
    Senator Specter. You have an assurance from Reliance, but 
have you made an independent inquiry in light of the 
substantial questions raised in this article?
    Dr. Baldwin. We will be making a trip to India in the next 
month.
    Senator Specter. The answer is, you have not yet made an 
inquiry?
    Dr. Baldwin. We have made an inquiry. We have been 
satisfied by that inquiry. If you ask whether we have been on 
site----
    Senator Specter. What inquiry did you make?
    Dr. Baldwin. We have met with the Indian derivers through 
videoconferencing. We have discussed these issues with them, 
and have satisfactory responses from them.
    Senator Specter. What responses have you had on these 
issues?
    Dr. Baldwin. I would have to provide the detail of that for 
the record. That discussion was at the policy stage, before 
that activity moved into my office. I would prefer to provide 
you a more detailed answer to that.
    We did summarize after each of our meetings with the 
providers that every one of them had, in fact, satisfied the 
President's criteria. In fact, they were quite forthcoming 
about their procedures.
    Senator Specter. I have heard your generalizations, but we 
want the specifics. If you are unable to provide them at this 
time, then you submit them to the committee.
    Dr. Baldwin. I would be happy to submit them.
    Senator Specter. By the end of the week?
    Dr. Baldwin. By the end of the day.
    Senator Specter. By the end of the day?
    Dr. Baldwin. Absolutely.
    Senator Specter. A question has been raised about the 
Swedish stem cells which make up almost half of the stem cell 
derivations, according to information provided to the 
subcommittee. There are barriers from the Gotebourg, Sweden 
group. The availability of the number of stem cell lines for 
U.S. researchers is restricted by the donors, since the 
original donation was for a period of 6 months, and the donors 
have a right to reclaim the donated cells. Is that correct?
    Dr. Baldwin. Yes, that is correct. But in Gotebourg they 
are going back to reconsent the donors so that they could meet 
our eligibility criteria. They would be doing that under any 
circumstances, and it is obviously essential for the NIH 
purposes. We have met with them. We do not feel there is going 
to be any problem in that regard.
    Senator Specter. But they do not now have the donor's 
consent?
    Dr. Baldwin. Not for all of them. They had them at the time 
that they presented their material. The question in the consent 
form, where they said that they would only be used for 6 
months, was an issue. That is a problem for them, as well as 
for any uses that might be made of them at the NIH. So they 
have told us that they are, in fact, going back to each one to 
reconsent.
    Senator Specter. Is it true the donors have the right to 
reclaim the donated cells?
    Dr. Baldwin. I believe it is.
    Senator Specter. So these are unanswered questions as to 
whether those cell lines are really available.
    Dr. Baldwin. Yes. We know that three of them are available.
    Senator Specter. What's that?
    Dr. Baldwin. We know three of them are available now.
    Senator Specter. Has the period of time expired for the 
donors to reclaim them?
    Dr. Baldwin. That is a level of detail--again, I would like 
to provide that for the record.
    Senator Specter. Well, thank you very much, Dr. Baldwin. If 
you would stay with us, there may be some more questions which 
arise during the course of the hearing, when we hear from the 
other witnesses. If you could provide those materials to us by 
the end of the day, that would be very helpful.
    Dr. Baldwin. Thank you.
    Senator Specter. I would now like to hear from Dr. Bert 
Vogelstein, president of the Biological and Biomedical 
Associations of the Stem Research Committee, National Academy 
of Science.
    Dr. Vogelstein is professor of oncology at John Hopkins, 
where he holds a joint appointment in molecular biology and 
genetics, a bachelor's degree from the University of 
Pennsylvania--was that the College or the Wharton School?
    Dr. Vogelstein. College.
    Senator Specter. Good for you, and an M.D. from John 
Hopkins University School of Medicine. Do you recall your class 
slogan from Penn, Dr. Vogelstein?
    Dr. Vogelstein. No, I do not--sorry.
    Senator Specter. Thank you for joining us, Dr. Vogelstein. 
We look forward to your testimony.
STATEMENT OF BERT VOGELSTEIN, M.D., PROFESSOR OF 
            ONCOLOGY AND PATHOLOGY, JOHN HOPKINS 
            ONCOLOGY CENTER; CHAIRMAN, NATIONAL 
            RESEARCH COUNCIL INSTITUTE OF MEDICINE 
            COMMITTEE ON THE BIOLOGICAL AND MEDICAL 
            APPLICATIONS OF STEM CELL RESEARCH
    Dr. Vogelstein. Thank you. I am here today as the chairman 
of the National Research Council Institute of Medicine 
Committee on the Biological and Medical Applications of Stem 
Cell Research.
    We issued a report whose purpose was to look at how 
promising stem cell research really is, how far away we are 
from practical, therapeutic applications of this research, and 
what factors might either promote or hinder progress in the 
development of this research in the future.
    We have made several recommendations, but in the interest 
of time I will just briefly mention two of the most important 
of them. First, while much can be learned from existing 
embryonic stem cell lines if they are made widely available for 
research, several concerns about these lines strongly suggested 
that we will need to develop new stem cell lines in the future.
    Second, a substantial obstacle to the success of 
transplantation of these cells is the immune reaction of the 
patient's body to cells that it perceives as foreign. One of 
the most promising ways to overcome this obstacle is with a----
    Senator Specter. Would you repeat the obstacle? I did not 
understand it.
    Dr. Vogelstein. Yes. Most people are excited about this 
research because of their potential medical implications, and 
particularly for transplantation for diseases like Parkinson's 
disease, diabetes, et cetera. These cells will have come in 
general from another person, and they will in general be 
rejected by the host, the person they are transplanted into. 
That will become eventually one of the most difficult obstacles 
to actually using this research to help people.
    Now, how do we overcome that? There are several potential 
strategies, but perhaps the most promising of these is through 
the creation of stem cells using a technique known as somatic 
cell nuclear transfer. This technique is also called 
therapeutic cloning. It should not be confused with 
reproductive cloning. The purpose of somatic cell nuclear 
transfer, or SCMT, or therapeutic cloning, is to produce cells 
in culture, not to produce people.
    Senator Specter. It should not be confused with what kind 
of cloning?
    Dr. Vogelstein. Reproductive cloning. Reproductive cloning 
purpose is to produce people. Therapeutic cloning purpose is to 
produce cells in a tissue culture dish.
    Senator Specter. It is to produce cells?
    Dr. Vogelstein. Cells and tissues, heart cells, brain 
cells, pancreatic cells, and those cells then can be 
transplanted into people with a variety of diseases that would 
otherwise be lethal. Because these cells come from the person 
whom they are going to be transplanted into, they are 
genetically identical and should not be rejected. That is how 
they overcome the rejection problem.

                           prepared statement

    Thank you for the opportunity to testify. I would like my 
statement to be put into the record, together with our report 
from the National Research Council, and I would be happy to try 
to answer any questions you might have.
    [The statement follows:]

               Prepared Statement of Dr. Bert Vogelstein

    Good morning, Mr. Chairman, and members of the Committee. My name 
is Bert Vogelstein and I am a Professor of Oncology and Pathology at 
the John Hopkins Oncology Center and a Howard Hughes Medical Institute 
Investigator. I am here today as the chairman of a National Research 
Council and Institute of Medicine Committee on the Biological and 
Biomedical Applications of Stem Cell Research which yesterday released 
the report: ``Stem Cells and the Future of Regenerative Medicine.''
    Stem cell research gives hope to millions of Americans and people 
around the world who suffer from debilitating illnesses such as 
diabetes and Parkinson's disease-or who have suffered injuries to their 
spinal cords or other parts of the body-that new treatments and perhaps 
even a cure will someday make them well again.
    Given that promise, as well as the ethical controversies it 
generates, the National Research Council and Institute of Medicine 
decided it would be a good idea to form a committee to take a look at 
how promising stem cell research really is; how far away we are from 
practical, therapeutic applications; and what factors might either 
promote or hinder progress in the development of stem cell therapies.
    Our committee also took into consideration the fact that there are 
diverse views held in our society about the ethical controversies stem 
cell research raises. We included an expert in bioethics on our 
committee and invited philosophers, ethicists, religious leaders, and 
legal experts to a workshop we held on the scientific and ethical 
issues of stem cell research this past summer so we could listen to 
their views.
    It is important to note that none of the members of our committee 
are conducting stem cell research ourselves, and none have financial 
interests in stem cell research. This was to assure that none of us had 
a vested self-interest in the outcome of this report.
    Realizing the importance of stem cell research, The National 
Academies not only initiated but also funded most of this study itself, 
with additional support from the Ellison Foundation, to whom we are 
grateful.
    It should be pointed out that we recognize that the role of the 
National Academies is to advise public policy, not set it. The purpose 
of our report was to tell policymakers what we know about the potential 
of stem cell research based on the best available science, but it is up 
to the government, and really all of society, to consider our advice 
and make decisions.
    Let me give you a brief description of what stem cells are and what 
we know about their potential to be used as medical therapies, before I 
get to our findings and recommendations.
    Stem cells are unspecialized cells that can renew themselves 
indefinitely, and under the right conditions, become, or differentiate 
into, cell types with specialized functions. They can be found in an 
embryo in the very early stages of development, in some fetal tissue, 
and in some adult organs.
    Isolating adult stem cells is very difficult and there is only 
preliminary evidence that they can be turned into tissue characteristic 
of organs other than the ones from which they were taken. On the other 
hand, since 1998, we have been able to grow embryonic stem cells in the 
laboratory. In addition, embryonic stem cells are known to have the 
ability to differentiate into virtually all cell types. Researchers 
have had some success using transplanted embryonic stem cells from mice 
to restore some lost functions in diseased or injured animals. We found 
these studies to be promising, but not definitive, evidence that 
similar treatments can be successful in humans.
    Let me now briefly summarize the committee's main recommendations:
    First, although experiments in mice and other animals are 
necessary, they are not sufficient for realizing the full potential of 
stem cells to lead to tissue-replacement therapies for humans. Studies 
with human stem cells are absolutely necessary.
    Second, although stem cell research is on the cutting edge of 
science today, it is still in its infancy. Current scientific data 
indicate that there are important biological differences between adult 
and embryonic stem cells and among adult stem cells found in different 
types of tissue. The therapeutic implications of these biological 
differences are not yet clear. Adult stem cells from bone marrow have 
so far provided most of the examples of successful therapies for 
replacement of diseased or destroyed cells. However, their potential 
for fully differentiating into multiple tissue types is still poorly 
understood and remains to be clarified. In contrast, embryonic stem 
cells studied in animals clearly are capable of developing into 
multiple tissue types and are capable of long-term self-renewal in 
culture. Because the application of stem cell research to therapy for 
human disease will require much more knowledge about the biological 
properties of all types of stem cells, research on both embryonic and 
adult human stem cells should be pursued.
    Third, while much can be learned from existing embryonic stem cell 
lines if they are made widely available for research, concerns about 
the eventual accumulation of genetic mutations in these lines, and the 
fact that most have been cultured with animal cells and serum, means 
they need to be monitored very closely and that we will need to develop 
new stem cell lines in the future.
    Fourth, human stem cell research that is publicly funded and 
conducted under the highest standards of open scientific exchange, 
peer-review, and public oversight offers the most efficient and 
responsible means to achieve medical breakthroughs. Stem cell science 
is still in the early stages where much more basic research is needed. 
Although research by private, for-profit companies will eventually play 
a critical role in translating the fruits of basic research into actual 
medical therapies, it is likely to take years to yield commercial 
products. Without public funding for basic research to get us to that 
point, progress is likely to be hindered.
    Fifth, proposals for federal grants to work on human embryonic stem 
cells should be justified on the soundest scientific grounds and should 
be strictly scrutinized for compliance with existing and future 
federally-mandated ethical guidelines.
    Sixth, we recommend that to assist in providing scientific and 
ethical oversight, a national advisory body composed of leading 
scientists, ethicists, and other stakeholders be established at the 
National Institutes of Health. This group could evaluate the technical 
merit of research proposals, monitor potential risks to research 
subjects, and ensure compliance with the law and ethical standards.
    Our final recommendation is that in conjunction with research on 
stem cell biology and the development of stem cell therapies, research 
on the problem of transplant rejection should also be actively pursued. 
A substantial obstacle to the success of transplantation of any cells, 
including stem cells and their derivatives, is the immune reaction of a 
patient's body to cells that it perceives as foreign. Multiple 
approaches to reducing this problem should be explored, including ways 
to manipulate the genetic makeup of the stem cell tissue to make it 
less likely to provoke an immune reaction, and the creation of stem 
cells using a technique known as somatic cell nuclear transfer. This 
involves taking the DNA from a cell of a patient in need of a 
transplant, inserting it into an egg cell that has had its nucleus 
removed, and triggering cell division. The resulting stem cells and 
tissue that can be obtained from this procedure would be genetically 
identical to the patient's, and would in theory not be rejected by the 
patient's immune system when transplanted into him or her.
    This procedure should not be confused with reproductive cloning 
which utilizes a similar technique for the purpose of implanting an 
embryo and creating a child. The issue of reproductive cloning is being 
looked at by another National Academies committee, which will issue its 
findings soon.
    I would like to end by emphasizing again that stem cell research is 
in its infancy. Our committee is respectful of the wide array of 
social, political, legal, ethical, and economic issues that must be 
considered in policy-making in a democracy, and we have been impressed 
by the commitment of all parties in this debate to life and health, 
regardless of the different conclusions they draw. We hope our report, 
by clarifying what is known about stem cells and how best to realize 
their potential, will be a useful contribution to the discussion of 
this important issue.
    Thank you for this opportunity to testify. I would like my 
statement to be put into the record, and I will be happy to answer any 
questions the Committee might have.
                                 ______
                                 

           Stem Cells and the Future of Regenerative Medicine

                           executive summary
    Stem cell research offers unprecedented opportunities for 
developing new medical therapies for debilitating diseases and a new 
way to explore fundamental questions of biology. Stem cells are 
unspecialized cells that can self-renew indefinitely and also 
differentiate into more mature cells with specialized functions. 
Research on human embryonic stem cells, however, is controversial, 
given the diverse views held in our society about the moral and legal 
status of the early embryo. The controversy has encouraged provocative 
and conflicting claims both inside and outside the scientific community 
about the biology and biomedical potential of both adult and embryonic 
stem cells.
    The National Research Council and Institute of Medicine formed the 
Committee on the Biological and Biomedical Applications of Stem Cell 
Research to address the potential of stem cell research. The committee 
organized a workshop that was held on June 22, 2001. At the workshop, 
the committee heard from many leading scientists who are engaged in 
stem cell research and from philosophers, ethicists, and legal 
scholars. (Audio files of the speakers' presentations are available at 
the workshop web site, www.nationalacademies.org/stemcells.)
    The participants discussed the science of stem cells and a variety 
of ethical and other arguments relevant to public-policy as it applies 
to stem cells. The committee considered the information presented, 
explored the literature on its own, and contemplated the substance and 
importance of the preliminary data from recent stem cell experiments. 
The committee's deliberations on the issues led to the following 
conclusions and recommendations.
  --Experiments in mice and other animals are necessary, but not 
        sufficient, for realizing the potential of stem cells to 
        develop tissue-replacement therapies that will restore lost 
        function in damaged organs. Because of the substantial 
        biological differences between animal and human development and 
        between animal and human stem cells, studies with human stem 
        cells are essential to make progress in the development of 
        treatments for human disease, and this research should 
        continue.
  --There are important biological differences between adult and 
        embryonic stem cells and among adult stem cells found in 
        different types of tissue. The implications of these biological 
        differences for therapeutic uses are not yet clear, and 
        additional data are needed on all stem cell types. Adult stem 
        cells from bone marrow have so far provided most of the 
        examples of successful therapies for replacement of diseased or 
        destroyed cells. Despite the enthusiasm generated by recent 
        reports, the potential of adult stem cells to differentiate 
        fully into other cell types (such as brain, nerve, pancreas 
        cells) is still poorly understood and remains to be clarified. 
        In contrast, studies of human embryonic stem cells have shown 
        that they can develop into multiple tissue types and exhibit 
        long-term self-renewal in culture, features that have not yet 
        been demonstrated with many human adult stem cells. The 
        application of stem cell research to therapies for human 
        disease will require much more knowledge about the biological 
        properties of all types of stem cells. Although stem cell 
        research is on the cutting edge of biological science today, it 
        is still in its infancy. Studies of both embryonic and adult 
        human stem cells will be required to most efficiently advance 
        the scientific and therapeutic potential of regenerative 
        medicine. Moreover, research on embryonic stem cells will be 
        important to inform research on adult stem cells, and vice 
        versa. Research on both adult and embryonic human stem cells 
        should be pursued.
  --Over time, all cell lines in tissue culture change, typically 
        accumulating harmful genetic mutations. There is no reason to 
        expect stem cell lines to behave differently. In addition, most 
        existing stem cell lines have been cultured in the presence of 
        non-human cells or serum that could lead to potential human 
        health risks. Consequently, while there is much that can be 
        learned using existing stem cell lines if they are made widely 
        available for research, such concerns necessitate continued 
        monitoring of these cells as well as the development of new 
        stem cell lines in the future.
  --High quality, publicly funded research is the wellspring of medical 
        breakthroughs. Although private, for-profit research plays a 
        critical role of translating the fruits of basic research into 
        medical advances that are broadly available to the public, stem 
        cell research is far from the point of providing therapeutic 
        products. Without public funding of basic research on stem 
        cells, progress toward medical therapies is likely to be 
        hindered. In addition, public funding offers greater 
        opportunities for regulatory oversight and public scrutiny of 
        stem cell research. Stem cell research that is publicly funded 
        and conducted under established standards of open scientific 
        exchange, peer review, and public oversight offers the most 
        efficient and responsible means of fulfilling the promise of 
        stem cells to meet the need for regenerative medical therapies.
  --Conflicting ethical perspectives surround the use of embryonic stem 
        cells in medical research, particularly where the moral and 
        legal status of human embryos is concerned. The use of 
        embryonic stem cells is not the first biomedical research 
        activity to raise ethical and social issues among the public. 
        Restrictions and guidelines for the conduct of controversial 
        research have been developed to address such concerns in other 
        instances. For example, when recombinant-DNA techniques raised 
        questions and were subject to intense debate and public 
        scrutiny, a national advisory body, the Recombinant DNA 
        Advisory Committee, was established at the National Institutes 
        of Health (NIH) to ensure that the research met the highest 
        scientific and ethical standards. If the federal government 
        chooses to fund research on human embryonic stem cells, a 
        similar national advisory group composed of exceptional 
        researchers, ethicists, and other stakeholders should be 
        established at NIH to oversee it. Such a group should ensure 
        that proposals to work on human embryonic stem cells are 
        scientifically justified and should scrutinize such proposals 
        for compliance with federally mandated ethical guidelines.
  --Regenerative medicine is likely to involve the implantation of new 
        tissue in patients with damaged or diseased organs. A 
        substantial obstacle to the success of transplantation of any 
        cells, including stem cells and their derivatives, is the 
        immune-mediated rejection of foreign tissue by the recipient's 
        body. In current stem cell transplantation procedures with bone 
        marrow and blood, success can hinge on obtaining a close match 
        between donor and recipient tissues and on the use of 
        immunosuppressive drugs, which often have severe and life-
        threatening side effects. To ensure that stem cell-based 
        therapies can be broadly applicable for many conditions and 
        individuals, new means to overcome the problem of tissue 
        rejection must be found. Although ethically controversial, 
        somatic cell nuclear transfer, a technique that produces a 
        lineage of stem cells that are genetically identical to the 
        donor, promises such an advantage. Other options for this 
        purpose include genetic manipulation of the stem cells and the 
        development of a very large bank of embryonic stem cell lines. 
        In conjunction with research on stem cell biology and the 
        development of stem cell therapies, research on approaches that 
        prevent immune rejection of stem cells and stem cell-derived 
        tissues should be actively pursued.
    The committee is aware of and respectful of the wide array of 
social, political, legal, ethical, and economic issues that must be 
considered in policy-making in a democracy. And it is impressed by the 
commitment of all parties in this debate to life and health, regardless 
of the different conclusions they draw. The committee hopes that this 
report, by clarifying what is known about the scientific potential of 
stem cells and how that potential can best be realized, will be a 
useful contribution to the debate and to the enhancement of treatments 
for disabling human diseases and injuries. On August 9, 2001, when 
President Bush announced a new federal policy permitting limited use of 
human embryonic stem cells for research, this report was already in 
review. Because this report presents the committee's interpretation of 
the state of the science of stem cells independent of any specific 
policy, only minor modifications to refer to the new policy have been 
made in the report.
                            recommendations
    1. Studies with human stem cells are essential to make progress in 
the development of treatments for human disease, and this research 
should continue.
    2. Although stem cell research is on the cutting edge of biological 
science today, it is still in its infancy. Studies of both embryonic 
and adult human stem cells will be required to most efficiently advance 
the scientific and therapeutic potential of regenerative medicine. 
Research on both adult and embryonic human stem cells should be 
pursued.
    3. While there is much that can be learned using existing stem cell 
lines if they are made widely available for research, concerns about 
changing genetic and biological properties of these stem cell lines 
necessitate continued monitoring as well as the development of new stem 
cell lines in the future.
    4. Human stem cell research that is publicly funded and conducted 
under established standards of open scientific exchange, peer-review, 
and public oversight offers the most efficient and responsible means to 
fulfill the promise of stem cells to meet the need for regenerative 
medical therapies.
    5. If the federal government chooses to fund human stem cell 
research, proposals to work on human embryonic stem cells should be 
required to justify the decision on scientific grounds and should be 
strictly scrutinized for compliance with existing and future federally-
mandated ethical guidelines.
    6. A national advisory group composed of exceptional researchers, 
ethicists, and other stakeholders should be established at NIH to 
oversee research on human embryonic stem cells. The group should 
include leading experts in the most current scientific knowledge 
relevant to stem cell research who can evaluate the technical merit of 
any proposed research on human embryonic stem cells. Other roles for 
the group could include evaluation of potential risks to research 
subjects and ensuring compliance with all legal requirements and 
ethical standards.
    7. In conjunction with research on stem cell biology and the 
development of potential stem cell therapies, research on approaches 
that prevent immune rejection of stem cells and stem cell-derived 
tissues should be actively pursued. These scientific efforts include 
the use of a number of techniques to manipulate the genetic makeup of 
stem cells, including somatic cell nuclear transfer.

    [Clerk's note.--The report from the National Research 
Council: ``Stem Cell Research and the Future of Regenerative 
Medicine'' can be found on the web at http://www.nap.edu/books/
0309076307/
html/.]
    Senator Specter. Dr. Vogelstein, you start off saying that 
there is a need to develop new lines. Why?
    Dr. Vogelstein. There are several reasons for that. The 
currently existing lines, a relatively small number, are 
problematic because they accumulate mutations as they grow, and 
eventually they need to be used.
    Senator Specter. Accumulate mutations? What do you mean by 
that?
    Dr. Vogelstein. Genetic defects. Every time a cell divides 
in tissue culture, it accumulates mutations. These mutations in 
aggregate may cause certain problems, especially when it is 
realized that they may not be suitable for patients for another 
10 years or so.
    Senator Specter. May not be suitable for patients for 
another 10 years or so?
    Dr. Vogelstein. Yes.
    Senator Specter. Why is that?
    Dr. Vogelstein. This research is really in its infancy. By 
the time that enough research is done to allow transplantation 
with these cells, it is likely to be roughly 10 years.
    Now, one cannot estimate that exactly, but that is 
consistent with Secretary Thompson's----
    Senator Specter. So these mutations would be pronounced 
over a protracted period of time? They would not be usable?
    Dr. Vogelstein. That is one possibility, and it might be 
preferable to use fresh cells which have not accumulated as 
many mutations. That is one reason.
    A second reason is that all of the existing lines have been 
cocultured with animal cells, or animal products, which present 
certain potential hazards.
    There are other problems, too, but perhaps the main, or one 
of the most important points is that new technologies for 
generating these cells will undoubtedly continue to be 
developed over the coming years, and if one is forced to use 
only the stem cell lines that are now in existence, one 
precludes using new stem cell lines that may be better. As an 
example, it would be like forcing people to listen to vinyl 
records instead of using eight-tracks or CD's even when the 
technology becomes better, and when one realizes that this 
issue is much more important than sound quality, that may be 
quite limiting in the long term.
    Senator Specter. Any more reasons why we need to develop 
the new stem cell lines?
    Dr. Vogelstein. I think those are the main ones.
    Senator Specter. When you mentioned reason No. 2, that they 
are cocultured with animals, you are referring to the mouse 
feeder issue?
    Dr. Vogelstein. The mouse feeder cells as well as they have 
been cultured in serum generally derived from cows.
    Senator Specter. And does that preclude their use in 
therapy?
    Dr. Vogelstein. It does not necessarily preclude their use, 
but it certainly, at least theoretically, provides a hazard, a 
zoonotic hazard if there are any viruses or other pathogens 
that were in the mice or the cows that could have been 
transmitted to these cells, and it is a potential danger. It 
obviously would be preferable if they had never been cultured 
with any animal produces.
    Senator Specter. There is an amendment which is likely to 
be filed with our pending bill which I discussed yesterday with 
Senator Brownback. The thrust of it would be to ban therapeutic 
cloning, both public and private, and to impose a penalty of $1 
million and a prison term. What effect do you think such an 
amendment, if enacted into law, would have on stem cell 
research?
    Dr. Vogelstein. Well, as our report noted, one of the most 
important ways to actually be able to use stem cells for 
medicine may involve embryonic stem cells created from 
therapeutic cloning techniques, and if one were to preclude 
that, the implication could mean that these regenerative 
medicine applications will never occur, and that the people 
with a variety of terrible diseases will never be able to 
benefit from this research. That is a possibility.
    Senator Specter. Are you suggesting it might discourage 
such research?
    Dr. Vogelstein. I think that is an understatement.
    Senator Specter. Well, I asked you a question about what 
the effect would be, and did not get very much of a direct 
answer. I expected a fair amount of outrage. What happened to 
your Pern class? Which class was it, by the way?
    Dr. Vogelstein. 1966.
    Senator Specter. I was district attorney at that time, 
making the streets safe in West Philadelphia.
    Dr. Vogelstein. I remember that.
    Senator Specter. Which was no easy job.
    Dr. Vogelstein. It would completely preclude such research, 
and the implications might well be that it would completely 
preclude the use of this whole technology, embryonic stem 
cells, for real medical applications. There is a great danger 
in that policy.
    Senator Specter. Senator Brownback's amendment further bans 
any importation of any product derived from cloning. This 
undefined provision could keep Americans from benefiting from 
cures that may be developed from using therapeutic cloning 
outside of the United States. What impact would you see if it 
became law that you could not import any product derived from 
therapeutic cloning?
    Dr. Vogelstein. Again, it depends on what lines and 
reagents are developed outside the country, but since the 
current biomedical research is really an international effort, 
it is reasonably likely that the most useful lines and reagents 
will be developed outside of the United States and, if so, this 
policy would again inhibit or perhaps entirely preclude using 
the best technology to correct these diseases in people.
    Senator Specter. Dr. Vogelstein, elaborate on the kinds of 
ailments which could be affected by therapeutic cloning. You 
mentioned pancreatic cells. What else?
    Dr. Vogelstein. Pancreatic cells for diabetes.
    Senator Specter. Pancreatic cells for diabetes?
    Dr. Vogelstein. Yes. Nerve cells for Parkinson's disease.
    Senator Specter. Would you repeat that?
    Dr. Vogelstein. Nerve cells for Parkinson's disease and 
Alzheimer's disease. Nerve cells for spinal cord injuries. 
Blood vessel cells for certain types of heart diseases, as well 
as heart cells, and I think one of the important things to note 
is that most of the diseases that I just mentioned, as well as 
many others which are catalogued in our report, there is no 
cure, and the only real hope for these people, at least 
according to current medical opinion, is through regenerative 
medicine.
    Senator Specter. Through what?
    Dr. Vogelstein. Through regenerative medicine.
    Senator Specter. No cure for what?
    Dr. Vogelstein. For any of these diseases, Alzheimer's 
disease, Parkinson's disease, a variety of spinal cord 
injuries, certain types of diabetes, many others.
    Senator Specter. So the only hope is through regenerative--
--
    Dr. Vogelstein. The only hope on the horizon is through 
transplantation of these stem cells, and the other important 
point to mention, which is again in our report, the details, 
but these are not rare diseases. We are talking about diseases 
which affect millions of people in the United States alone.
    Senator Specter. You ticked them off. Diabetes, 
Parkinson's, Alzheimer's, spinal cord injuries, heart--are 
there others?
    Dr. Vogelstein. Yes. There is a whole list of them.
    Senator Specter. We found it on page 6. Thank you very 
much. I want to have this in hand for the debate which we are 
going to have a little later today.
    Dr. Vogelstein, what would be a more accurate and less 
inflammatory name than therapeutic cloning which would say 
about the same thing?
    Dr. Vogelstein. Well, in our report----
    Senator Specter. You should have consulted with your public 
relations people before you put cloning in the title.
    Dr. Vogelstein. In our report I actually refer to it as 
somatic cell nuclear transfer, or SCNT.
    Senator Specter. Repeat that again.
    Dr. Vogelstein. Somatic cell nuclear transfer. The acronym 
is SCNT, and we purposely used that term because of the great 
confusion between therapeutic and reproductive cloning, which 
is very unfortunate, because they are really quite different.
    Senator Specter. Well, Dr. Vogelstein, thank you very much 
for your testimony, and thank you for the work of the National 
Academy of Sciences. We appreciate it.
    If you would stay with us we may have some more questions 
as we hear from the other panelists.
    Dr. Vogelstein. Thank you very much.
    Senator Specter. We would now like to hear from panel 3, 
Dr. Marti Pera, Dr. Joseph Itskovitz, Dr. James Thomson, Dr. 
Carl Gulbrandsen. Dr. Pera is associate professor, senior 
research fellow at Monash University in Melbourne, Australia, 
and earned his master's from Oxford and his Ph.D. from George 
Washington University.
    Thank you for joining us, Dr. Pera. May I first inquire if 
you are here for some conference, or here for some other 
purpose?
    Dr. Pera. I am actually here on holiday, visiting my 
family.
    Senator Specter. That is the best of all reasons. We are 
glad to have you available to testify today. Please proceed.
STATEMENT OF MARTIN F. PERA, Ph.D., MONASH UNIVERSITY, 
            AUSTRALIA
    Dr. Pera. Thank you, Mr. Chairman, for the opportunity to 
come and speak to you today. In my testimony I will respond 
directly to queries raised by your committee on our stem cell 
research and related matters.
    The first question related to the current status of stem 
cell lines derived by our group in collaboration with Professor 
Bundus at the National University of Singapore. Our group has 
derived six independent stem cell lines. The lines were 
developed from embryos developed with informed consent by 
couples undergoing in vitro fertility treatment. They were 
surplus for clinical need. They had no financial inducement 
offered for their donation. All of these cell lines have been 
placed on the NIH registry of human embryonic stem cells.
    Of the six cell lines, four have been sufficiently 
characterized to validate their identification in light of our 
present understanding of the biology of primate ES cells. These 
criteria include the demonstration that the cells express 
molecules on the surface of a particular set of genes 
characteristic of pluripotent stem cells, that they maintain 
normal genetic makeup as evidenced by normal chromosome number 
and morphology, that they are able to differentiate into a wide 
range of cells in vitro when grafted into experimental animals, 
and that they retain these properties for at least 150 
population doublings, or generations, in vitro.
    The remaining two cell lines have been successfully 
cultivated for 50 population doublings, and have been 
successfully cryopreserved. We are now completing the 
characterization of these remaining cells lines, which should 
be finished in the first half of next year.
    The second question related to our definition of a 
successful derivation of an ES cell line. We consider that an 
attempt at derivation is successful in yielding a new line when 
the cell line has met the criteria I just outlined and has been 
successfully cryopreserved on at least three separate 
instances. Successful cryopreservation means the cell can be 
recovered in a viable form from frozen stock, and new cultures 
which also meet the criteria for ES cells may be initiated from 
the frozen stock.
    A third question related to the adequacy of presently 
available cell lines to support basic research on stem cells. 
While at present we cannot be certain that all of the cell 
lines currently available for federally funded research will 
meet all of the above criteria listed for validation as ES cell 
lines, with the exception of those described in the literature 
by Dr. James Thompson and ourselves. However, if we make a 
reasonable assumption that there are at least 20 cell lines 
available that will meet these criteria, that these cell lines 
will be widely and successfully distributed throughout the 
academic community, then such a panel of cell lines would 
probably be adequate to conduct much of the basic research into 
ES cell biology that is a prerequisite to any clinical 
application of these cells.
    Senator Specter. Probably adequate to conduct much of the 
basic research?
    Dr. Pera. Yes.
    Senator Specter. That leaves two questions in your answer, 
probably adequate, and much of the research. Do you think the 
scientific community ought to rely on that kind of a judgment, 
probably, for much, and not with certainty for all?
    Dr. Pera. In my opinion, the door should be left open to 
the derivation and the use of new cell lines for research.
    Senator Specter. Thank you.
    Dr. Pera. Regarding the use of existing cell lines in 
clinical therapies, it is possible that available cell lines 
will be useful in clinical therapies. However, to my knowledge 
the existing cell lines have been derived using mouse feeder 
cell support, and the Food and Drug Administration has 
indicated that any cell line produced for clinical use from 
such cells would be regarded for regulatory purposes as a 
xenotransplant.
    Now, such a classification of this cell as a xenotransplant 
would not prohibit its use, but would place incumbrances on its 
use, and if actual hazards such as the transmission of 
pathogens from the animal cells were, in fact, documented, then 
it is likely that the use of the cell lines would be curtailed, 
so in my view it is almost certain that additional cell lines 
will have to be derived for clinical therapy in the future 
because of possible hazards associated with coculture of the 
stem cells with animal cells, and because cell lines 
representative of a greater degree of genetic diversity may 
well be required to circumvent problems of tissue rejection.
    Senator Specter. When you say greater diversity, what do 
you have in mind there, Dr. Pera?
    Dr. Pera. Greater genetic diversity. We all carry a 
particular set of antigens that causes rejection of those cells 
in a noncompatible individual, and that is caused by the 
essential genetic diversity of people, and we may need more 
cell lines more broadly representative of a wide range of 
genetic background.
    Senator Specter. Are you thinking there about ethnic lines, 
about racial lines?
    Dr. Pera. I am talking about genetic diversity in general, 
but racially as well.
    Senator Specter. Well, what does genetic diversity mean 
beyond racial diversity, or ethnic diversity?
    Dr. Pera. Well, within any racial or ethnic group there is 
a very wide range of what we call tissue histocompatibility 
antigens that are encoded by our DNA, so that while some races 
have a narrow range of these, in others there are many 
thousands of combinations in any given ethnic group.
    Senator Specter. So that is even when you are dealing with 
no racial or ethnic diversity?
    Dr. Pera. That is correct, yes.
    Senator Specter. How many cell lines do you think would be 
required to solve this issue of genetic diversity?
    Dr. Pera. That is a very difficult question to answer.
    Senator Specter. That is why we have you here, Dr. Pera.
    Dr. Pera. Unfortunately, I am not a transplantation 
immunologist, but the answer to that question depends in part 
on, I think, the clinical problem. In some clinical instances 
you may be transplanting the cells into a site where 
immunorejection is not such a great problem. It depends in part 
on how visible the stem cell derived cells are to the immune 
system, and it depends in part on how sanguine you are about 
our future prospects to manipulate the immune system.
    Senator Specter. So with all those questions, would you say 
it is a fair conclusion that a great many lines may be 
necessary if scientists are to have a full range of opportunity 
for research?
    Dr. Pera. In our present understanding, I think that would 
be a fair answer, yes.
    The fifth question related to our plans to derive 
additional cell lines, and the answer is yes, we and our 
collaborators will certainly derive additional cell lines in 
the future to meet some of these clinical applications, as 
noted above.
    A further reason for deriving additional cell lines is that 
since our knowledge of embryonic stem cells is constantly 
expanding, and our current techniques for derivation and 
maintenance of these cell lines are almost certainly 
suboptimal, it may be that our present methodology is somehow 
selecting for cells which are defective in some way that has 
not yet become manifest. Thus, in the future, guided by a 
better understanding of stem cell biology, we might wish to 
derive new cell lines with improved properties.
    We were asked about obstacles to the distribution of cell 
lines, and to date the main obstacles we have faced in our 
efforts to distribute our cells have been limitations on our 
resources, which have really hampered our ability to produce 
cells for distribution and to train scientists in recipient 
laboratories.
    These obstacles are likely to become less significant in 
the coming years, since we have received support from our 
Victorian State government to set up a small laboratory for 
stem cell production and training. Intellectual property issues 
have delayed completion in some instances of materials transfer 
agreements for the cell lines, but we have been pretty flexible 
in the approach to this matter, and it has not proven yet to be 
a major impediment.
    The seventh question related to our interaction with the 
National Institutes of Health, and we did meet with officials 
from the NIH and Secretary Thompson in the summer of this year 
to discuss plans for listing of our cell lines on the registry.
    The final question relates to recent progress in embryonic 
stem cell research, and looking globally, I think that within 
the past year several laboratories have reported the derivation 
of a wide range of cell types from spontaneously differentiated 
cultures, clearly, nerve cells, cardiac muscle cells, insulin-
producing cells, and blood stem cells.
    In particular, I think that substantial progress has been 
made in obtaining neural precursors from cultures of embryonic 
stem cells. We have obtained highly enriched populations of 
nerve cell precursors, and we have been able to show that these 
can undergo normal differentiation and integration into the 
brain when grafted into the nervous system of experimental 
animals.
    More recently, we have identified a key regulatory molecule 
that is active in controlling the early phases of stem cell 
differentiation, and shown that modulation of the signaling 
pathway can direct differentiation of stem cells toward the 
nerve cell lineage, so that in summary, in upcoming months I 
think we can expect to see more work which will enhance our 
ability to control the growth and differentiation of embryonic 
stem cells, and I think that this work will lead to a much more 
efficient production of specific, specialized cells required in 
research and regenerative medicine.
    [The information follows:]
          Response to Queries from Senators Specter and Harkin
    Current status of cell lines derived by our group.--In 
collaboration with Professor Ariff Bongso at the National University of 
Singapore, our group has derived six independent human embryonic stem 
(ES) cell lines from human blastocysts. These lines were developed from 
embryos donated with informed consent by couples undergoing in vitro 
fertility treatment. The embryos were surplus to clinical requirements, 
and no financial inducement was offered in return for their donation. 
All of these cell lines have been placed on the National Institutes of 
Health Human Embryonic Stem Cell Registry. Of the six cell lines, four 
have been sufficiently characterised to validate their identification 
as human ES cell lines in light of our present understanding of the 
biology of primate ES cells. The criteria for validation include the 
demonstration that the cells express certain specific marker molecules 
on their surface and a particular set of genes that are characteristic 
of pluripotent cells, that they maintain a normal genetic makeup as 
evidenced by normal chromosome number and morphology, that they are 
able to differentiate into a wide range of body cells in vitro and when 
grafted into experimental animals, and that they retain these 
properties for at least 150 population doublings in vitro. The 
remaining two cell lines have been successfully cultivated for 50 
population doublings in vitro, and they have been successfully 
cryopreserved. Our laboratory is presently completing the 
characterisation of these remaining two cell lines, a task that should 
be finished in the first half of next year. There is at present no 
reason to believe these remaining two cell lines will not meet the 
criteria for validation listed above, but we must complete the 
necessary tests to ensure that they do.
    Definition of successful derivation of an ES cell line.--We 
consider that an attempt at derivation is successful in yielding a new 
ES cell line when the cell line has met the above criteria and has been 
successfully cryopreserved on at least three separate instances. 
Successful cryopreservation means that the cells can be recovered in a 
viable form from frozen stock and new cultures which meet the criteria 
for ES cells may be initiated from the frozen cells.
    Adequacy of presently available cell lines to support basic 
research on stem cells.--At present, we cannot be certain that all of 
the cell lines currently available for federally funded research will 
meet all the above listed criteria for validation as ES cell lines, 
with the exception of those described in the literature by Dr. James 
Thomson and ourselves. However, if we make a reasonable assumption that 
there are at least twenty cell lines available that will meet these 
criteria, and that these cell lines will be widely and successfully 
distributed throughout the academic community, then such a panel of 
cell lines will probably be adequate to conduct much of the basic 
research into ES cell biology that is a prerequisite to any clinical 
application of these cells.
    Use of existing cell lines in clinical therapies.--It is possible 
that currently available cell lines will be useful in clinical therapy. 
However, to my knowledge the existing cell lines have been derived 
using mouse feeder cell support, and the Food and Drug Administration 
has indicate that any cell produced for clinical use from such lines 
would be regarded for regulatory purposes as a xenotransplant. Such 
classification of a cell line as a xenotransplant would not prohibit 
its use, but would place encumbrances on such use, and if actual 
hazards such as transmission of pathogens from the animal cells were in 
fact documented, then it seems likely the use of the cell lines would 
be curtailed. It is certain that additional cell lines will have to be 
derived for clinical therapy in future, because of possible hazards 
associated with co-culture of the existing stem cells with animal 
cells, and because cell lines representative of a greater degree of 
genetic diversity may well be required to circumvent problems of tissue 
rejection.
    Derivation of additional cell lines.--We and our collaborators will 
probably derive additional cell lines in the future, mainly to meet the 
requirements of clinical applications as noted in 4 above. A further 
reason for deriving additional cell lines is that since our knowledge 
of ES cells is constantly expanding, and our current techniques for 
derivation and maintenance are almost certainly suboptimal, it may be 
that our present methodology somehow is selecting for cells which are 
defective in some way that has not yet become manifest. Thus in future, 
guided by a better understanding of stem cell biology, we might wish to 
derive new cell lines which will have improved properties. Our main 
priority at present however, is to carry out the basic research which 
is an essential prerequisite to clinical use of ES cells or their 
derivatives, and we are doing this with our existing cell lines.
    Obstacles to the distribution of cell lines.--To date, the main 
obstacles we have faced in our efforts to distribute our ES cells to 
workers wishing to conduct research in this area have been limitations 
on our resources, which have hampered our ability to produce cells for 
distribution and to train scientists in recipient laboratories. These 
obstacles will be less significant in the coming year, since we have 
received support from the Victorian state government to set up a small 
laboratory for stem cell production and training. Intellectual property 
issues have delayed completion of Materials Transfer Agreements for the 
cell lines in some cases, but we have been flexible in our approach to 
this matter and it has not proven to be a major impediment. ESI Pte., 
the company which controls intellectual property rights to our cell 
lines, continues to seek means to expedite distribution of these cells.
    Interaction with the National Institutes of Health.--We met with 
officials from the National Institutes of Health and Secretary Thompson 
in August of this year to discuss plans for listing of our cell lines 
on the registry.
    Recent progress in human ES cell research.--Within the past year, 
several laboratories have reported the derivation of a wide range of 
cell types from spontaneously differentiating cultures of human ES 
cells. These include nerve cells, cardiac muscle cells, insulin 
producing cells, and blood stem cells. In particular, substantial 
progress has been made in obtaining neural precursors from cultures of 
embryonic stem cells. We have obtained highly enriched populations of 
neural progenitors from human ES cell cultures, and have shown that 
these progenitors undergo normal differentiation and integration when 
grafted into the nervous system of experimental animals. More recently 
we have identified a key regulatory molecule active in the early phases 
of human ES cell differentiation, and we have shown that modulation of 
this signaling pathway can direct differentiation of ES cells towards 
the neuronal lineage. In upcoming months we can expect to see more work 
which will enhance our ability to control the growth and 
differentiation of ES cells; this work will lead to much more efficient 
production of specific specialised cells required in research and 
regenerative medicine.

    Senator Specter. Dr. Pera, are there any laws in Australia, 
to your knowledge, which preclude extracting stem cells from 
embryos?
    Dr. Pera. Yes, there are. It varies from State to State in 
Australia. In our own State of Victoria that sort of 
manipulation of an embryo is prohibited, and so we collaborated 
with groups from overseas to do that phase of the work.
    In other States there are no restrictions. We have recently 
had a parliamentary committee of inquiry report on stem cells 
and related matters, and their report was, in fact, very 
favorable. It recommended stem cell research and endorsed 
derivation of new cell lines, and the idea is that with that 
recommendation the Federal Government will get uniform 
legislation.
    Senator Specter. So you got the stem cell lines from 
overseas?
    Dr. Pera. That is right. They came from Singapore.
    Senator Specter. From Singapore?
    Dr. Pera. That is correct.
    Senator Specter. And do you receive any governmental 
funding?
    Dr. Pera. We are just now receiving governmental funding. 
We have a program grant.
    Senator Specter. From Australia?
    Dr. Pera. It is actually funded by the Juvenile Diabetes 
Foundation, but through our National Health and Medical 
Research Council.
    Senator Specter. Has NIH made our Federal funding available 
to you?
    Dr. Pera. It is certainly open to us to apply for those 
funds, and we intend to do so.
    Senator Specter. Well, thank you very much, Dr. Pera. We 
appreciate your being here.
    You have six lines, four characterized and two 
uncharacterized?
    Dr. Pera. That is correct.
    Senator Specter. And what is your expectation as to the 
utility of the two uncharacterized lines?
    Dr. Pera. From what we have been able to observe of them so 
far, they will be very similar in their properties to other 
cell lines, and we expect they will be validated as bona fide 
embryonic stem cell lines. We are doing that at the moment, and 
we hope to have the answer in the early part of next year.
    Senator Specter. Thank you. I will turn now to Dr. Joseph 
Itskovitz, professor at Technion University, Haifa, Israel, and 
director of the Rambam Medical Center. Thank you for joining 
us, Dr. Itskovitz. Let me inquire preliminarily what brings you 
to the United States, something beyond this hearing? Are you 
here in the United States solely for this hearing?
    Dr. Itskovitz. We are also meeting with NIH people.
    Senator Specter. Thank you for joining us. We look forward 
to your testimony. The floor is yours.
STATEMENT OF JOSEPH ITSKOVITZ, Ph.D., DIRECTOR, RAMBAM 
            MEDICAL CENTER; PROFESSOR, TECHNION 
            UNIVERSITY, HAIFA, ISRAEL
    Dr. Itskovitz. Thank you, Mr. Chairman. First of all, I am 
director for OB/GYN of the Rambam Medical Center.
    Mr. Chairman, Senator Specter, members of the committee, I 
am pleased to appear before you today to testify on human 
embryonic stem cell research. I represent a group of 20 
scientists and students currently involved in human embryonic 
stem cell research in Haifa for the last 3 years.
    In September 1997, we established collaboration with Dr. 
James Thomson, leading to the derivation to the first five 
human ES cell lines, H1, H7, H9, H13, and H14 in Madison, and 
also the clonal derivation from a single derivation of H9.1 and 
H9.2 in human ES cell lines.
    In July 2000, we derived three cell lines, I3, I4, I6, and 
then an additional line, J3 was established in January 2000 in 
Haifa from frozen cell embryos.
    The lines met President Bush's criteria. Three cell lines, 
I6, I3, and J3 are well-characterized. They show characteristic 
morphology of ES. They have already been continued in the 
culture for at least 10 months. They express markers shown to 
be associated with human ES, and maintain normal clarity. The 
cells form antibodies in suspension in deficient mice which 
show formation of tissues from all three germ layers.
    At least one single cell clone was derived from each of 
these cell lines. J3 is a unique cell line. It was derived from 
a culture in vitro for an additional week. There is a distinct 
morphology, and cells grow faster than other cell lines of the 
H or I series. It may represent a later stage of cell of the 
primitive ectoderm. The unique characteristics of specific 
pathways of differentiation is currently being examined.
    In addition, we continue the culture H cell lines that 
originated from Madison. Altogether, we have in our laboratory 
in Haifa nine parental lines and many clonal lines, cultures 
continuously from the period between 10 months and 2 years. In 
August 2001, two studies conducted in Haifa were reported. The 
first study demonstrated that each ES cells can differentiate 
into myocytes with structural and functional properties of 
cardiomyocytes. This was reported by Kehat in the second paper 
by Assady. She demonstrated insulin production by human ES 
cells, reported, published in Diabetes.
    These reports validate the H ES cell's model system as a 
potential basis for enrichment of human cardiomyocytes and 
islet cells, a possible future source for cell replacement 
therapy.
    More recently, we have directed differentiation of human ES 
cells to insulin-secreting structures similar to pancreatic 
islets by using a modified method of Ron McKay's protocol. This 
was done by Segev. Furthermore, Assady unpublished, she has 
generated pluripotent undifferentiated cell lines transgenic 
for a reporter driven by the insulin promoter, an approach that 
can be used to monitor better cell differentiation and to 
isolate a rich population of insulin-producing cells for 
transplantation therapy.
    In Israel, the Bioethics Advisory Committee of the Israel 
Academy of Sciences and Humanities has approved both the 
derivation of human ES cells and also research into therapeutic 
cloning. The Rambam Medical Center and the Technion School of 
Medicine view the embryonic stem cell project to be of major 
interest for basic and applied research.

                           prepared statement

    Prior to September 11, we started a first round of 
discussions with our colleagues at various institutes and 
centers at the NIH. We are warmly accepted and intend to 
continue our discussion in order to generate collaborative 
research programs with NIH scientists and to obtain support for 
our internal research activity and the necessary supporting 
infrastructure.
    [The statement follows:]
               Prepared Statement of Dr. Joseph Itskovitz
    Mr. Chairman, Senator Specter, and Members of the Committee, I am 
pleased to appear before you today to testify on human embryonic stem 
cell research. I represent a group of 20 scientists currently involved 
in human embryonic stem (hES) cell research in Haifa for the last 3 
years. In September 1997, we established a collaboration with Dr. James 
Thomson leading to the derivation of the first 5 hES cell lines in (H1, 
H7, H9, H13, H14) in Madison (Thomson et al., Science 282:1145, 1998) 
and also the clonal derivation from a single cell of H9.1 and H9.2 hES 
lines (Amit et al., Development 227:271, 2000).
    In July 2000, we derived 3 cell lines 13, 14, 16, and an additional 
line (J3) was established in January 2000 all in Haifa. The lines met 
President Bush's criteria. Three cell lines (I3, I6, and J3) are well 
characterized. They show characteristic hES morphology, have already 
been continuously cultured for at least 10 months, express markers 
known to be associated with hES and maintain normal karyotype. The 
cells form embryoid bodies in suspension and create teratomas in 
immune-deficient mice which show formation of tissues from all 3 germ 
layers. At least one single cell clone was derived from each of these 
cell lines. J3 is a unique cell line. It was derived from an embryo 
cultured in vitro for an additional week, has a distinct morphology, 
and the cells grow faster than the other hES cell lines of the H or I 
series. It may represent a later stage of cells of the primitive 
ectoderm. The unique characteristics for specific pathway of 
differentiation is currently being examined. In addition we continue to 
culture the H cell lines that originated from Madison. All together we 
have in our laboratory in Haifa 9 lines being cultured continuously 
from a period between 10 months and 2 years.
    In August 2001, two studies conducted in Haifa were reported. The 
first study, demonstrated that hES cells can differentiate into 
myocytes with structural and functional properties of cardiomyocytes 
(Kehat et al., J Clin Inv 108:407:2001). In the second paper, Assady et 
al., demonstrated insulin production by hES cells (Diabetes 50:1691, 
2001). These reports validate the hES cell model system as a potential 
basis for enrichment of human cardiomyocytes and islet cells, a 
possible future source for cell replacement therapy.
    More recently we have directed differentiation of hES cells to 
insulin-secreting structures similar to pancreatic islets by using a 
modified method of Ron McKay's protocol (Segev et al., unpublished). 
Furthermore, Assady et al., (unpublished) have generated pluripotent 
undifferentiated hES cell clones, transgenic for a reporter driven by 
the insulin promoter, an approach that can be used to monitor 
(-cell differentiation, and to isolate enriched populations of 
insulin-producing cells for transplantation therapy.
    In Israel, the Bioethics Advisory Committee of the Israel Academy 
of Sciences and Humanities has approved both the derivation of hES 
cells and also research into therapeutic cloning.
    Rambam Medical Center and the Technion's School of Medicine view 
the embryonic stem cell project to be of major interest for basic and 
applied research. Prior to September 11 we started the first round of 
discussions with our colleagues at various Institutes and Centers at 
the NIH. We are warmly accepted and intend to continue our discussions 
in order to generate collaborative research programs with NIH 
scientists and to obtain support for our internal research activity and 
the necessary supporting infrastructure.

    Senator Specter. Thank you for that very fine statement, 
doctor. Your timing was the closest I have seen. You finished 
your 5 minutes with 1 second left.
    In Israel, extraction of stem cells from embryos is lawful?
    Dr. Itskovitz. Yes.
    Senator Specter. Have you received any funding from the 
Government of Israel?
    Dr. Itskovitz. Not for funding, not for derivation of the 
embryos. This was done, I would say, until now the money for my 
departmental budget, but currently further money is available 
for research on the human ES stem cell research.
    Senator Specter. Are you discussing with NIH the 
possibility of funding from NIH?
    Dr. Itskovitz. Definitely.
    Senator Specter. Which diseases will be available for stem 
cell treatment first, Dr. Itskovitz? What diseases do you look 
forward to working on from the stem cell derivation?
    Dr. Itskovitz. As I mentioned earlier, there is some 
preliminary data which is relevant mainly to insulin-producing 
cells for diabetes, Type II diabetes. The derivation of cardiac 
cells for the repair of cardiac damage in heart patients, and 
we are also doing some work now currently on blood vessel 
differentiation, actually in differentiation to construct blood 
vessels.
    Senator Specter. Does your laboratory or any of your 
collaborators plan to work on therapeutic cloning?
    Dr. Itskovitz. Yes.
    Senator Specter. What do you think the impact would be if 
we passed a law in the United States prohibiting therapeutic 
cloning and provided a jail sentence and a big fine, and 
prohibited the importation of any materials resulting from 
therapeutic cloning?
    Dr. Itskovitz. It is, I believe, now it is hard to predict, 
but I think that this technology should be left open for 
research. It is really difficult to appreciate now how this 
will bring us to the clinic to alleviate or overcome all of the 
problems of rejections that was mentioned before we can have a 
lot of information generated from animal studies regarding the 
therapeutic cloning, but definitely I think that this should be 
left open for further discussion with the public.
    Senator Specter. Have you detected any differences between 
cell lines derived from frozen, as contrasted with fresh stem 
cell lines?
    Dr. Itskovitz. All of the cell lines were derived from 
frozen embryos.
    Senator Specter. Do you think that limiting the research to 
the stem cell lines in existence as of 9:00 p.m. on August 9 is 
sufficient for the needs of the scientific community?
    Dr. Itskovitz. Currently I believe it is sufficient even 
for the near future, because an enormous amount of data can be 
generated from the current available cell lines, even if there 
are only 30, not 60, but in addition I believe that they should 
also leave the door open to generate new cell lines in the 
future.
    I am not sure when and how soon, but the cell lines 
currently available will allow us to proceed very significantly 
with the basic research, and even with applied research, and 
maybe even bring us to the clinic, but definitely we should 
leave the door open in the future to generate a different type 
of cell line that I have already alluded to. One of them was 
established in my lab, and also in the case that we have cell 
lines that would not be compatible for therapeutic purposes 
after FDA reviewing and revision of the current cell lines that 
are available.
    Senator Specter. Well, are the existing lines all adequate 
for therapy, considering the contamination, so to speak, from 
mouse feeders and bovine, et cetera?
    Dr. Itskovitz. There are no indications of contaminations. 
This should be checked, but basically, principally speaking 
this does not preclude the use of the cells as a transplant in 
humans. This was, at least by this being announced by the FDA--
--
    Senator Specter. You say it does pose a problem?
    Dr. Itskovitz. Potentially it may cause a problem. If the 
FDA will not approve the current cell lines based on the 
definition that they are having for clinical purposes it is 
still enigmatic.
    Senator Specter. Do you think FDA should approve their use 
for therapy?
    Dr. Itskovitz. The current?
    Senator Specter. The stem cell lines that have been 
developed with mouse feeders or bovine.
    Dr. Itskovitz. If they are checked, and they would be 
disease-free, and other issues related to exposure would be 
settled, the FDA would be satisfied with it, it is okay.
    Senator Specter. But you think even beyond that there is a 
need to have the availability of new cell lines in the future?
    Dr. Itskovitz. In the future, yes.
    Senator Specter. Thank you very much, doctor.
    We turn now to Dr. James Thomson, scientific director of 
WiCell Research Institute, chief of pathology at the Wisconsin 
Regional Primate Research Center, assistant professor of 
anatomy, University of Wisconsin Medical School, B.S. from 
Illinois, Ph.D. and DVM from University of Pennsylvania.
STATEMENT OF JAMES THOMSON, Ph.D., CHIEF SCIENTIFIC 
            OFFICER, WiCELL RESEARCH INSTITUTE, INC.
    Dr. Thomson. Thank you, Mr. Chairman, for the opportunity 
to appear today to discuss human embryonic stem cell research. 
Today we are here because of your efforts, and we deeply 
appreciate your support.
    The main issue today is whether the existing human ES cell 
lines that were derived prior to President Bush's policy 
announcement of August 9, 2001 are sufficient for human ES cell 
research to fulfill its promise. I believe that much of the 
recent debate about the actual number of existing cell lines 
has been misdirected. If there are only a couple of dozen such 
cell lines that are widely available to American investigators, 
it is likely that much of the basic research that must be done 
in order to develop new therapies can be accomplished. These 
cells can be expanded without apparent limit. Therefore, a 
reasonably small number of cell lines can supply the research 
needs of a large number of investigators.
    The University of Wisconsin and the Wisconsin Alumni 
Research Foundation are both committed to seeing that the five 
human embryonic stem cell lines I have developed are shared 
widely with the research community, and we have already begun 
sharing these cell lines with investigators. I believe the 
existing human ES cell lines will support most of the basic 
research needs of U.S. investigators.
    However, the existing human ES cell lines will not fulfill 
their promise unless NIH begins aggressively to fund this area 
of research. As of today, the NIH human embryonic stem cell 
registry is not yet up and running, and this is necessary to 
initiate funding. We and others have already provided NIH with 
the required documentation for the existing cell lines, and 
researchers are anxious to start submitting grant proposals in 
this area. Anything that this committee can do to facilitate 
this process will make a big impact.
    There has recently been given a great deal of press 
attention given to the fact that human ES cell lines were 
derived in contact with cells from mice and with protein 
products from other species, including bovine serum. The 
intermingling of protein sources from multiple species raises 
legitimate safety concerns for future therapeutic products 
based on human ES cells because a possibility exists that 
pathogens could be transmitted between these species.
    At the time that I was deriving our five human ES cell 
lines, I was consciously deriving them for research purposes, 
not for therapeutic purposes. I believe that future derivations 
can be done in a more controlled manner that would satisfy the 
FDA's safety concerns for therapeutic products.
    Today, however, we do not yet know how to derive and grow 
human ES cells in the complete absence of these foreign cells 
or protein products.
    Senator Specter. Let me interrupt you at that point, Dr. 
Thomson, to pinpoint the question as to whether the existing 
lines with a mouse feeder and the bovine aspects are adequate 
for therapy.
    Dr. Thomson. I think that is where the big difference is. I 
think for basic research the existing cell lines are probably 
adequate. For therapy, they are probably not. The existing cell 
lines may well be approved by FDA. There is historical 
precedence for them approving human health cell lines,not ES 
cell lines, with the same kinds of problems, but if I was a 
patient and I had the choice between cell lines derived on use 
already done and outside cell lines under more controlled 
conditions----
    Senator Specter. And if you were a patient and told the 
only ones available were the ones----
    Dr. Thomson. If they are the only ones available, sure, but 
I would like to have that choice. It would be useful to derive 
more in the future.
    Senator Specter. Because the risks involved with the mouse 
feeders and the bovine----
    Dr. Thomson. That is right. You can test them extensively 
for everything that is known, and if you miss something because 
it is unknown, it is still a potential problem, and FDA deals 
with those risks all the time, but it clearly would be 
preferable to derive them in complete absence of those 
problems.
    Senator Specter. Thank you.
    Dr. Thomson. However, today we do not yet know how to 
derive stem cell lines that lack those problems. There is an 
active area of research in several lines, and I would guess 
over the next several years we will be able to derive new cell 
lines that do not have those problems. Because clinical trials 
based on human ES cells may be several years away, the several 
concerns about existing cell lines will not have any direct 
impact on initial basic research.
    The President's decision does not affect the private 
sector's ability to derive new human ES cell lines, and I am 
confident that as improved human culture conditions are 
established the private sector will derive additional cell 
lines. However, I believe that the major innovative 
breakthroughs in stem cell research will continue to occur in 
academic research centers. New therapies are usually introduced 
into these clinics by major academic medical centers, but the 
ability of these medical centers to use new human ES cell lines 
derived by the private sector could be severely limited by the 
President's decision.
    Academic medical centers receive Federal support, and the 
overhead from such Federal support could interfere with a 
physician's ability to use new human ES cell lines in clinical 
trials even if those clinical trials are privately funded. This 
is a serious concern that must be addressed if the President's 
policy remains in place once clinical trials are initiated.
    Senator Specter. Would you repeat your last statement 
please, Dr. Thomson?
    Dr. Thomson. Academic centers receive NIH support for a 
variety of purposes. They get overhead for that that supports 
everything from the lights to the heating system. The 
President's decision is interpreted very rigorously, that 
overhead intermixing with new cell lines could be a very big 
problem. It has been a very big problem up to now. We have had 
to separate our labs into separate places so they do not have 
the overhead problems, but you cannot separate the clinical 
trials from that overhead problem. It would be very difficult 
to do.
    So as new cell lines are derived----
    Senator Specter. You are saying the President's policy does 
not take into account overhead?
    Dr. Thomson. If new cell lines that are derived that are 
clinically more useful, even if they are privately funded in 
academic medical centers, it would be difficult for people to 
use them if the current policy continues.
    Senator Specter. Why is that?
    Dr. Thomson. Because NIH money is intermingled into 
overhead, and that would be a potential support for clinical 
trials in an indirect way, so that is a problem that has to be 
addressed if this policy stays.
    Finally, I believe the study of cells, both adult and 
embryonic, will revolutionize human medicine, but that this 
revolution will occur rapidly only if the proper infrastructure 
is provided to nurture it. The debate about whether adult or 
embryonic stem cells is better is a political debate not shared 
by mainstream stem cell biologists. For example, the knowledge 
gained from the study of embryonic stem cells will almost 
certainly advance the clinical utility of stem cells.

                           prepared statement

    The development of clinical applications of stem cells 
requires novel collaborative interactions between scientists 
and physicians with diverse backgrounds. Multiple national stem 
cell research centers that support both embryonic and adult 
stem cells would significantly accelerate the field by 
providing a collaborative environment for the development of 
stem-cell-based therapies.
    I look forward to your questions. Thank you.
    [The statement follows:]
                Prepared Statement of Dr. James Thomson
    I want to thank both Senators Specter and Harkin for the 
opportunity to appear here today to address issues concerning human 
embryonic (ES) stem cell research. Additionally, I would not only like 
to thank these Senators, but Secretary Thompson as well, for their 
continuing support of human embryonic stem cell research. Today we are 
here because of your diligent efforts. I deeply appreciate your 
support.
    The main issue to address today is whether the human ES cell lines 
that were derived prior to President Bush's policy announcement of 
August 9, 2001, are sufficient for human ES cell research to fulfill 
it's promise. I believe that much of the recent debate about the actual 
number of existing cell lines has been misdirected. If there are only a 
couple dozen cell lines that are widely available to American 
investigators, it is likely that much of the basic research that must 
be done in order to develop new therapies can be accomplished. These 
cells can be expanded without apparent limit. Therefore, a reasonably 
small number of cell lines can supply the research needs of a large 
number of investigators. The University of Wisconsin and the Wisconsin 
Alumni Research Foundation are both committed to seeing that the five 
human embryonic stem cell lines that I have derived are shared widely 
with the research community, and we have already begun sharing them 
with other investigators. I believe the existing human ES cell lines 
will support most of the basic research needs of U.S. investigators.
    However, the existing human ES cell lines will not fulfill their 
promise unless NIH begins to aggressively fund this area of research. 
As of Monday, October 29, 2001, the NIH Human Embryonic Stem Cell 
registry, necessary to initiate funding, had not yet been posted. We, 
and others, have already provided NIH with the required documentation 
for existing cell lines, and researchers across the country are anxious 
to start submitting grant proposals in this area. Anything that this 
committee can do to facilitate that process would make a big impact.
    There has recently been a great deal of press attention given to 
the fact that existing human embryonic stem cell lines were derived in 
contact with cells from mice and with protein products from other 
species, including bovine serum. The intermingling of protein sources 
from multiple species raises legitimate safety concerns for future 
therapeutic products based on human ES cells, because the possibility 
exists that pathogens could be transmitted between species. At the time 
that I was deriving our five human ES cell lines, I was consciously 
deriving them for research purposes, not for therapeutic purposes. I 
believe that future derivations can be done in a more controlled manner 
that would satisfy the FDA's safety concerns for therapeutic products. 
Today, however, we do not yet know how to derive and grow human ES 
cells in the complete absence of these foreign cells or protein 
products, and this is an area of active research in several labs. Thus, 
today we are not yet able to derive new human ES cell lines that lack 
these potential problems.
    Because clinical trials based on human ES cells may be several 
years away, these safety concerns about existing cell lines will not 
have any direct impact on initial basic research. The President's 
decision does not affect the private sector's ability to derive new 
human ES cell lines, and I am confident that as improved culture 
conditions are established, the private sector will derive additional 
cell lines. However, I believe that the major innovative breakthroughs 
in stem cell research will continue to occur in academic research 
centers. New therapies are usually introduced into the clinics by major 
academic medical centers, but the ability of these medical centers to 
use new human ES cell lines derived by the private sector in clinical 
trials could be limited by the President's decision. Academic medical 
centers receive federal support and the over-head from such federal 
support could interfere with a physician's ability to use new human ES 
cell lines in clinical trials, even if those clinical trials are 
privately funded. This is a serious concern that must be addressed if 
the President's policy remains in place once clinical trials are 
initiated.
    Finally, I believe that the study of stem cells, both adult and 
embryonic, will revolutionize human medicine, but that this revolution 
will only occur rapidly if the proper infrastructure is provided to 
nurture it. The debate about whether adult or embryonic stem cells are 
``better'' is a political debate not shared by mainstream stem cell 
biologists. For example, the knowledge gained from the study of 
embryonic stem cells will almost certainly advance the clinical utility 
of adult stem cells. The development of clinical applications of stem 
cells will required novel collaborative interactions between scientists 
and physicians with diverse backgrounds. Multiple National Stem Cell 
Research Centers that support both embryonic and adult stem cell 
research would significantly accelerate the field by providing a 
collaborative environment for the development of stem cell-based 
therapies.

    Senator Specter. Thank you, Dr. Thomson.
    We now turn to Dr. Carl Gulbrandsen, managing director of 
the Wisconsin Alumni Research Foundation, and president of 
WiCell Research Institute. He received his bachelor's degree 
from St. Olaf, Ph.D. from the University of Wisconsin, and J.D. 
from the University of Wisconsin Law School.
    Thank you for joining us, Dr. Gulbrandsen. The floor is 
yours.
STATEMENT OF CARL E. GULBRANDSEN, Ph.D., J.D., MANAGING 
            DIRECTOR, WISCONSIN ALUMNI RESEARCH 
            FOUNDATION, PRESIDENT, WiCELL RESEARCH 
            INSTITUTE, INC.
    Dr. Gulbrandsen. Thank you, Mr. Chairman. Mr. Chairman, 
members of the subcommittee, I am pleased to appear before you 
again to discuss the role of the Wisconsin Alumni Research 
Foundation, and its not-for-profit subsidiary, WiCell Research 
Institute, supporting the important research necessary to move 
the science of embryonic stem cells forward. I would like to 
once again personally thank Chairman Harkin and Senator Specter 
for their continued commitment to human embryonic stem cell 
research.
    This testimony was prepared for the hearing originally 
scheduled for September 12, 2001. I would like to take a moment 
to offer our sympathies to the families of the victims at the 
World Trade Center, the Pentagon, and passengers of the flight 
that crashed in Pennsylvania. Those who have been exposed to 
anthrax and have died from anthrax infection also should have 
our deepest sympathies.
    We at WiCell and the University of Wisconsin Madison 
involved in stem cell research are attempting to continue to 
move the program forward. While we are aware of the new 
landscape, we trust that the NIH and researchers all over the 
country will continue to pursue the promise of stem cell 
research.
    Since August 1, 2001, when I was last before this body, a 
significant barrier to forward movement of the ES cell research 
has been removed. In that regard, Secretary Tommy Thompson 
deserves special thanks. His counsel to the President was 
clearly instrumental in guiding the President to decide on 
August 9 that embryonic stem cell research should receive 
Federal funding. After the August 9 presidential decision and 
before September 11 it was evident that the leadership of Tommy 
Thompson had made funding of embryonic stem cell research a 
high priority at NIH.
    WARF and WiCell responded to that by negotiating and 
executing a memorandum of understanding with the United States 
Public Health Service that many commentators hailed as ground-
breaking. I am here today to explain that agreement to you.
    The agreement with the Public Health Service does two 
important things. First, under the agreement WiCell agrees to 
provide its human embryonic stem cells, which I will refer to 
as Wisconsin materials, to federally funded researchers at low 
cost and with few restrictions.
    Second, it provides at no cost an automatic limited 
noncommercial license under WARF's embryonic stem cell patents 
to Public Health Service researchers using Wisconsin materials 
and to third parties who provide human embryonic stem cells to 
researchers under similar terms as the agreement we have with 
PHS as well as to those researchers receiving the third party 
materials.
    Let me explain the agreement in more detail. The purpose of 
this agreement is to make the Wisconsin materials available to 
researchers at PHS and to federally funded researchers at 
universities and other research institutions as easily as 
possible. It is significant to note that the agreement with the 
Public Health Service does not require what is commonly 
referred to as reach-through rights. WARF and WiCell have a 
mission to serve the public good. In the interest of that 
mission we have provided research access to our patent rights 
and to the actual human embryonic stem cells cultured by Dr. 
Thomson.
    These cells are the gold standard. They meet the four 
criteria outlined by the American Society for Cell Biology. It 
should also be noted that these cells were derived using only 
private funds and are the private property of WARF. 
Nevertheless, WARF and WiCell are making these cells available 
at low cost with few restrictions to assist scientists in 
moving the research forward as quickly as possible. Scientists 
receiving Wisconsin materials under the agreement we have with 
PHS are free to publish and patent without consent whatever 
they discover using Wisconsin materials. The recipient 
institution will own such patents, and no commercial rights 
under those patents are owed back to WiCell.
    What restrictions are in the agreement with PHS? The 
bioethical restrictions are those imposed by the University of 
Wisconsin Bioethics Committee and representations to donors of 
embryos used to derive the Wisconsin materials that some 
experiments would not be done with donated embryos. Under these 
restrictions, the researcher receiving Wisconsin material 
agrees not to mix the materials with an intact embryo and place 
the material in a uterus, or attempt to make an embryo with the 
material.
    The agreement with the Public Health Service does not 
permit diagnostic or therapeutic use of the Wisconsin 
materials. There are two principal reasons for this 
restriction, safety and liability. The Wisconsin materials are 
research materials. While we hope that research using the 
Wisconsin materials will ultimately lead to life-saving 
therapies, at the present time there is no certainty that the 
Wisconsin materials themselves are suitable for diagnostic or 
therapeutic use. They were not prepared under the conditions 
that the FDA ordinarily requires for commercial diagnostic or 
therapeutic products. It would be irresponsible for WiCell to 
allow such use with its materials at this time.
    In fact, virtually all research material transfer 
agreements being used in the United States today have similar 
restrictions. In view of that, a failure by WiCell to require 
such a restriction would expose it to an unreasonably high risk 
of liability should an accident occur.
    The Wisconsin materials may not be used under the agreement 
that we have with the PHS in a research program where a non-
Federal research sponsor requires a grant back of commercial 
rights. Under such circumstances, the researcher or the sponsor 
will need a commercial license from WiCell. If such 
circumstances exist, under the agreement that we have with PHS 
WiCell has agreed to provide such a license under terms no less 
favorable than other similar commercial licenses to the extent 
that such rights are available.
    The Wisconsin materials may not be transferred to a third 
party without WiCell's written consent. However, WiCell has 
agreed under the agreement with the PHS to make materials 
available to such third parties under the simple letter 
agreement for teaching or noncommercial research purposes. This 
prohibition against transfer is in large part due to WiCell's 
obligations to assure that the Wisconsin materials are not used 
in violation of the bioethical restrictions.
    Beyond this, the researcher agrees to follow all applicable 
statutes, regulations, and guidelines related to handling use 
and disposal of the materials. The agreement does require an 
annual certification by the Public Health Service and the 
researcher that they are in compliance with those restrictions.
    WARF, WiCell, and the University of Wisconsin Madison 
believe in and are excited about the future of medicine 
utilizing embryonic stem cell technology. By allowing Federal 
funding for this research, the Government has taken an 
important first step. The next step is to provide sufficient 
funds to make a difference.
    According to the testimony at the Kennedy hearing in early 
September, approximately $250 million of NIH funding is 
currently spent on adult stem cell research. I would hope that 
at least that amount and preferably more would be devoted to 
embryonic stem cell technology.

                           prepared statement

    Our goal is to see this technology widely disseminated and 
developed, and we believe that our licensing practices and the 
recently signed agreement with PHS reflect that goal. We know 
that Federal funding will increase the number of researchers 
who work on embryonic stem cells, and that these resources will 
bring the tomorrow of medicine closer to today.
    Thank you.
    [The statement follows:]
             Prepared Statement of Dr. Carl E. Gulbrandsen
    Thank you, Mr. Chairman. Mr. Chairman and members of the 
subcommittee, I'm pleased to appear again before you to discuss the 
role of the Wisconsin Alumni Research Foundation and its not-for-profit 
subsidiary, WiCell Research Institute, in supporting the important 
research necessary to move the science of embryonic stem cells forward. 
I would like to once again personally thank Chairman Harkin and Senator 
Spector for their continued commitment to Human Embryonic Stem Cell 
(ES) research.
    This testimony was prepared for the hearing originally scheduled 
for September 12, 2001. I would like to take a moment to offer our 
sympathies to the families of the victims, at the World Trade Center, 
the Pentagon, and the passengers on the flight that crashed in 
Pennsylvania. Those who have been exposed to anthrax and have died from 
the anthrax infection also have our deepest sympathies. We at WiCell 
and the University of Wisconsin-Madison that are involved in stem cell 
research are attempting to continue to move the program forward. While 
we are aware of the new landscape we trust that the NIH and researchers 
all over the country will continue to pursue the promise of stem cell 
research.
    Since August 1, 2001, when I was last before this body, a 
significant barrier to forward movement of ES cell research has been 
removed. In that regard, Secretary Tommy Thompson deserves special 
thanks. His counsel to the President was clearly instrumental in 
guiding the President to decide on August 9, that embryonic stem cell 
research should receive federal funding. After the August 9th 
Presidential decision, and before the September 11 attack, it was 
evident that the leadership of Tommy Thompson had made funding of 
embryonic stem cell research a high priority at NIH.
    WARF and WiCell responded to that priority by negotiating and 
executing a Memorandum of Understanding (MOU) with the U.S. Public 
Health Service (hereafter referred to as ``MOU'') that many 
commentators hailed as groundbreaking. I am here today to explain that 
agreement to you.
    The MOU does two important things: first, under the agreement 
WiCell agrees to provide its human embryonic stem cells (``Wisconsin 
Materials'') to federally funded researchers at low cost and with few 
restrictions; second, it provides at no cost, an automatic, limited, 
non-commercial license under WARF's embryonic stem cell patents to 
Public Health Service (PHS) researchers using Wisconsin Materials and 
to third parties who provide human embryonic stem cells to researchers 
under similar terms as the MOU as well as to those researchers 
receiving those third party cells.
    Let me explain the agreement in more detail. The purpose of this 
agreement is to make the Wisconsin Materials available to researchers 
at the PHS and to federally funded researchers at universities and 
other research institutions as easily as possible. It is significant to 
note that the MOU does not require what are commonly referred to as 
reach-through rights. WARF and WiCell have a mission to serve the 
public good. In the interest of that public good we have provided 
research access to our patent rights and the actual Human Embryonic 
Stem Cells created by Dr. Thomson. These cells are the gold standard. 
They meet the four criteria outlined by the American Society for Cell 
Biology. It should also be noted that these cells were derived using 
only private funds and are the private property of WARF. Nonetheless, 
WiCell is making these cells available at low cost with few 
restrictions to assist scientists in moving the research forward as 
quickly as possible. Scientists receiving Wisconsin Materials under the 
MOU are free to publish and patent, without consent, whatever they 
discover using Wisconsin Materials. The recipient institution will own 
such patents and no commercial rights under those patents will be owed 
to WiCell.
    What restrictions are in the MOU? The bioethical restrictions are 
those imposed by the University of Wisconsin Bioethics Committee and 
representations to donors of embryos used to derive the Wisconsin 
Materials that some experiments would not be done with their donated 
embryos. Under these restrictions, the researcher receiving Wisconsin 
Materials agrees not to mix the Material with an intact embryo, implant 
the Material in a uterus or attempt to make an embryo with the 
material.
    The MOU does not permit diagnostic or therapeutic use of the 
Wisconsin Materials. There are two principal reasons for this 
restriction--safety and liability. The Wisconsin Materials are research 
materials. While we hope that research using the Wisconsin Materials 
will ultimately lead to life saving therapies, at the present time 
there is no certainty the Wisconsin Materials themselves are suitable 
for diagnostic or therapeutic use. They were not prepared under the 
conditions that the FDA ordinarily requires for commercial, diagnostic 
or therapeutic products. It would be irresponsible for WiCell to allow 
such use with its materials at this time. In fact, virtually all 
research material transfer agreements being used in the United States 
today have similar restrictions. In view of that, a failure by WiCell 
to require such a restriction would expose it to an unreasonably high 
risk of liability should an accident occur using Wisconsin Materials.
    The Wisconsin Materials may not be used under the MOU in a research 
program where a non-federal research sponsor requires a grant back of 
commercial rights. Under such circumstances, the researcher or sponsor 
will need a commercial license from WiCell. If such circumstance 
exists, under the MOU, WiCell has agreed to provide such a license 
under terms not less favorable than other similar commercial licenses 
to the extent such rights are available.
    The Wisconsin Materials may not be transferred to a third party 
without WiCell's written consent; however, WiCell has agreed under the 
MOU, to make materials available to such third party under the MOU and 
Simple Letter Agreement for teaching or non-commercial research 
purposes. This prohibition against transfer is in large part due to 
WiCell's obligations to assure that the Wisconsin Materials are not 
used in violation of the bioethical restrictions.
    Beyond this, the researcher agrees to follow all applicable 
statutes, regulations and guidelines relating to handling, use and 
disposal of such materials. The MOU does require an annual 
certification by PHS and the researcher that they are in compliance 
with these restrictions.
    WARF, WiCell, and the University of Wisconsin-Madison believe in 
and are excited about the future of medicine utilizing ES cell 
technology. By allowing federal funding for this research the 
government has taken an important first step. The next step is to 
provide sufficient funds to make a difference. According to testimony 
at the Kennedy Hearing in early September, approximately $250 million 
of NIH funding is currently spent on adult stem cell research. I would 
hope that at least that amount and preferably more would be devoted to 
ES cell technology.
    Our goal is to see this technology widely disseminated and 
developed. We believe that our licensing practices and recently signed 
MOU reflect that goal. We know that federal funding will increase the 
number of researchers who work with ES cells and that these researchers 
will bring the tomorrow of medicine closer to today.

    Senator Specter. Thank you very much, Dr. Gulbrandsen.
    Are you saying, in effect, that your companies have made 
available your patents in an unlimited way for basic research?
    Dr. Gulbrandsen. That is right.
    Senator Specter. But not for the application to therapy?
    Dr. Gulbrandsen. For commercial purposes that need to use 
our patents they will need to have commercial license from us.
    Senator Specter. They will have to have a commercial 
license from your company, but I hear the reasons you have 
given that you are concerned about civil liability.
    Dr. Gulbrandsen. I understand. Yes.
    Senator Specter. Any other reason?
    Dr. Gulbrandsen. No. The restriction in the memorandum of 
agreement to use of these materials for diagnostic or 
therapeutic purposes is principally a liability issue.
    Senator Specter. Well, it seems to me that if you make 
available patents and somebody else undertakes activity which 
results in damage, it would not be your responsibility, but I 
am not going to second-guess your lawyers. We really have not 
come to the application for therapy at this point. Would your 
company be willing to consider at some future time licensing 
for therapy as the matter progresses and therapy becomes more a 
current issue?
    Dr. Gulbrandsen. Absolutely, and the difference here is not 
just a patent license, it is actually providing the materials. 
If we were just licensing the patent, the liability issue is 
much less, but in this case we are providing materials.
    Senator Specter. You are providing materials? What do you 
mean?
    Dr. Gulbrandsen. The embryonic stem cells themselves, and 
so under our strict liability laws in this country those 
materials are dangerous. We would be liable.
    Senator Specter. Well, maybe so, and maybe not, but are you 
saying in effect that if the liability issue could be resolved 
so that there would not be liability for your companies, that 
you would be willing to issue licenses for therapy as well?
    Dr. Gulbrandsen. Absolutely.
    Senator Specter. What is the current status of WiCell's 
negotiations with the Geron Corporation regarding exclusive 
commercial licensing of the several additional cell types?
    Dr. Gulbrandsen. We are presently in litigation with Geron. 
The issue of their option rights to add additional cell lines 
is one of the issues in the lawsuit.
    Senator Specter. What is the essential dispute there, the 
essence of the dispute?
    Dr. Gulbrandsen. The essence of the dispute is that under 
the license agreement Geron had a license agreement that 
extended at the end of July, that expired at the end of July, 
to add additional types of cells to their exclusive fields of 
use and diagnostics and therapeutics. We unsuccessfully 
negotiated with them. The option in our mind expired. They are 
disputing that, and so we turned to the court to settle the 
issue.
    Senator Specter. Where is that case pending?
    Dr. Gulbrandsen. That is in the Western District of 
Wisconsin.
    Senator Specter. How many memoranda of understanding have 
been negotiated with academic research centers?
    Dr. Gulbrandsen. Since the signing of the memorandum of 
understanding with the Public Health Service we have sent out 
44 memoranda, or I guess 46 memoranda to other institutions.
    Senator Specter. How about with private companies?
    Dr. Gulbrandsen. Private companies, we are presently 
discussing with three of the cell type owners a license.
    Senator Specter. What compensation do you get from that, 
any?
    Dr. Gulbrandsen. From which?
    Senator Specter. From the memoranda, from letting others 
have your patents.
    Dr. Gulbrandsen. From academic researchers that are 
receiving Federal dollars the fee is a one-time up-front $5,000 
fee to transmit the stem cells to them.
    Senator Specter. You must have very considerable legal fees 
in connection with all of these memoranda of understanding, do 
you not?
    Dr. Gulbrandsen. This is a not-for-profit, truly a not-for-
profit. We are losing money on this. Yes, the fees are 
extensive. I did not mean to be flippant. We have invested an 
enormous amount of money in human embryonic stem cell research.
    Senator Specter. Dr. Thomson, let me come back to you for a 
question. We are expecting another amendment on the Labor-HHS 
bill which would make unlawful any attempt to combine a human 
gamete with an animal gamete, or to combine human genetic 
material with the egg of an animal. This sort of hybrid raises 
a lot of questions, and has an alarming sound. Are there any 
scientifically valuable experiments that would be affected by 
this kind of a ban?
    Dr. Thomson. Yes. There is a fertility assay that uses 
human sperm, and it is an assay to detect fertility of the 
sperm. That is an existing assay that has been used for years 
now. There is probably other assays like that, that would be 
affected by that wording.
    Senator Specter. Are you saying that that kind of a 
prohibition would be a significant limitation on scientific 
research?
    Dr. Thomson. I think to the people conducting those 
experiments, yes.
    Senator Specter. Can you elaborate your reasons so that I 
could repeat them on the floor of the Senate?
    Dr. Thomson. Sir, there is an existing assay----
    Senator Specter. If I find them persuasive, that is.
    I would like to understand what you are saying.
    Dr. Thomson. There is an existing assay to test the 
fertility of human sperm, and it is based on whether it will 
actually complete some of the process of fertilization with a 
hamster, and that product does not divide, it does not turn 
into an embryo, but what you just said would ban that, and it 
is an assay simply to look at fertility. I do not know how 
widely it is used. So that is one assay that has already been 
done in labs, and that would be banned by that wording.
    The thing that is probably intended to ban is taking a 
somatic nucleus and transferring it to a rabbit oocyte----
    Senator Specter. Start again and explain it once more.
    Dr. Thomson. Which one, the fertility assay?
    Senator Specter. What the impact would be on the 
prohibition which I just read to you.
    Dr. Thomson. There is already an existing assay which is 
used in clinics.
    Senator Specter. An existing----
    Dr. Thomson. An existing assay based on the combination of 
human sperm and hamster oocytes, a hamster egg, and it is 
simply to see if that sperm has the ability to fertilize 
something. Now, the product of that cannot divide.
    Senator Specter. The human sperm and the egg of an animal?
    Dr. Thomson. That is right.
    Senator Specter. And the purpose of that is to test the 
potency of the human sperm?
    Dr. Thomson. Right, and since I am not in that field I do 
not know how widely it is used, but it is an existing assay.
    Senator Specter. It is not used to create a new entity?
    Dr. Thomson. No. The combination apparently does not 
divide, and does not turn into an embryo, but it is enough to 
tell whether the sperm can penetrate.
    Senator Specter. That kind of a combination would not be 
successful in creating another entity?
    Dr. Thomson. Not a hybrid, no, but by the wording you said, 
that would be banned. More generally there is an interest in 
some groups to do nuclear transfer from human somatic cells to 
animal oocytes to do essentially therapeutic cloning, which was 
described before, to make ES cell-like lines from that product.
    Senator Specter. Would you repeat that, please?
    Dr. Thomson. There is an interest in using human oocytes, 
taking a nucleus from a patient, putting it into that animal 
oocyte, and letting the product grow to an appropriate stage 
and make an embryonic stem cell line. That embryonic stem cell 
line, if you can do that, and nobody has shown you can do that 
yet, would be matched to that patient, and you would get around 
the rejection problem.
    There are several groups that believe it might be possible. 
There is no convincing evidence that scientifically that works 
yet, but that ban would prohibit a potentially promising area 
of research.
    Senator Specter. Dr. Thomson, your five stem cells lines 
are among the oldest. Have they shown any signs of aging so as 
to be less useful?
    Dr. Thomson. No, but we probably have not tested them in 
the detail that is useful. Dr. Vogelstein mentioned that as you 
culture things, mutations occur. Most of those mutations would 
be likely very subtle things. They would be very difficult to 
detect, but they may have an impact on clinical applications, 
and so the level of detail that we have studied the cells would 
not allow us to see those changes yet. We have not seen any 
changes.
    Senator Specter. Dr. Baldwin, would you mind stepping 
forward again, please?
    Dr. Baldwin, Dr. Thomson raises an issue about the NIH 
registry, which is not up and running yet, and said if the 
subcommittee could do something about that, it would be 
enormously helpful. Is that correct, Dr. Thompson?
    Dr. Thomson. Correct.
    Senator Specter. Why isn't the NIH registry up and running?
    Dr. Baldwin. We have been spending a lot of time working on 
the structure of the registry, ensuring that the information 
that is in the registry is accurate and useful, and dealing 
with other policy issues. We had other guidelines in place. We 
have to rescind those guidelines. We have been trying to get 
all of those pieces in place. We are very close. I think what 
you have already heard today is how much work has gone on since 
August 9.
    Some of this activity we thought was moving along very 
briskly at the beginning of September. Our agenda collided with 
the world's agenda, and I cannot tell you anything other than 
we are just delayed. We are a little behind where I thought we 
would be. I think we are very close, and people are working 
very hard in putting all of the different pieces in place.
    Senator Specter. Are you saying the other responsibility of 
HHS has impeded getting the national registry up and running?
    Dr. Baldwin. Everything at the NIH has been affected by 
changes in security efforts on bioterrorism, people being drawn 
off to work on other issues that we did not anticipate, none of 
us anticipated.
    Senator Specter. When do you expect the national registry 
to be up and running?
    Dr. Baldwin. I think it will be up fairly shortly. We have 
solved all of the procedural and programmatic issues.
    Senator Specter. Within a week?
    Dr. Baldwin. I think that is a reasonable expectation.
    Senator Specter. What else would you like to see done, Dr. 
Thomson?
    Dr. Thomson. I think that is a big one. There is a lot of 
investigators that want to apply for funding, and they have to 
wait for that.
    Senator Specter. That will satisfy you today?
    Dr. Thomson. I am not easily satisfied.
    Senator Specter. Dr. Baldwin, what about the issue that Dr. 
Thomson raises about the overhead issue that--well, you 
understand it without my reformulating it. Just respond to the 
question which you understand.
    Dr. Baldwin. I think the question of how indirect costs----
    Senator Specter. Just a minute. Dr. Thomson, will you state 
the issue again so we have it clearly identified on the record?
    Dr. Thomson. The issue is that when an institution receives 
NIH funding it gets overhead to pay for buildings and 
infrastructure, and if you have a project that the President 
says you cannot use Federal funding for this, the cell lines 
would be something you could not use that money for, there is 
no way to separate the lighting and the heating from indirectly 
supporting those cell lines.
    Senator Specter. What is the answer to that, Dr. Baldwin?
    Dr. Baldwin. That is current policy, and we know that some 
entities have set up separate facilities, or spun off separate 
entities so they can do work that does not have any commingling 
with Federal funds.
    Senator Specter. Is that not really impractical?
    Dr. Thomson. When it gets to clinical trials it is very 
impractical. It is impossible.
    Senator Specter. It seems to me it is, Dr. Baldwin. If you 
have an institution, to require that it be totally separate, a 
new building, different people, different arrangements 
totally----
    Dr. Baldwin. Certainly, at the clinical application stage 
that would be a big challenge. I think what we have heard this 
morning is there is a great deal of basic research that needs 
to be done on the cells lines that will be available through 
the registry. We have really talked today, I think if you 
sketched out all the research recommendations, about a decade's 
worth of research. As you have heard from me, my interest right 
now is to get the registry up so we can begin to fund research 
grants doing that basic research, primarily.
    Senator Specter. Dr. Thomson, how soon do you think this 
will be a practical problem?
    Dr. Thomson. Well, I think within the next 3 years.
    Senator Specter. The next 2 years?
    Dr. Thomson. The next 2 to 3 years, and the reason is, 
investigators all over the world are improving the culture 
conditions of these cells, and there have been improvements 
since I originally derived them, and they continue to improve. 
I would guess over the next 2 to 3 years we would have 
extremely well-defined conditions that do not have all these 
problems with protein sources.
    The other thing that happens over the next 2 to 3 years 
there will be specific kinds of cells that will have already 
been derived in therapeutically useful amounts, and that does 
not mean they can go to clinics right away, but it means that 
people have to initiate the dialogue with FDA to get approval 
for their cell lines, and within 3 years it will be very useful 
to have new cell lines that do not have the current problems.
    Senator Specter. Well, Dr. Baldwin, you are in luck. You do 
not have to produce an answer by the end of today, like those 
other questions, or in a week, like the registry. You have a 
period of time, but I would like you to take it back to NIH and 
to HHS and address the issue. We have to find a way of solving 
that without requiring the recipients to have totally separate 
facilities.
    Well, I think this session has been very helpful. This is 
our eleventh hearing on the subject. It may be that if we have 
a sufficient number of hearings, that we will not have to ask 
you to repeat your answers several times, we will be able to 
start to understand them the first time around, but this is a 
matter of great complexity, and it is a matter of great, great 
importance.
    We are preoccupied, beyond any question, with the terrorism 
threats and the anthrax, and we had to postpone the hearing 
before from September 12, obviously, but we wanted to move 
ahead today. Although this kind of a hearing is important, as 
important as it is, it is not going to get a whole lot of 
public attention because of the bioterrorism problems, but we 
are dealing with a great many lives that can be saved here, and 
I think it is important to get that registry up and running and 
to move ahead.
    This subcommittee intends to pursue this matter with real 
diligence, and we will have the issue on the floor today and 
the information which you have given here is going to be 
helpful as we face up to these amendments.
    I will have another private conversation with you, Dr. 
Thomson, on this issue, if you would stand by. That would 
conclude our hearing. Thank you.

                         CONCLUSION OF HEARINGS

    Thank you all very much for being here, that concludes our 
hearings.
    [Whereupon, at 10:30 a.m., Wednesday, October 31, the 
hearings were concluded and the subcommittee was recessed, to 
reconvene subject to the call of the Chair.]

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