[Senate Hearing 107-127]
[From the U.S. Government Publishing Office]



                                                        S. Hrg. 107-127
 
                           STEM CELL RESEARCH
=======================================================================


                                HEARING

                                 OF THE

                    COMMITTEE ON HEALTH, EDUCATION,
                          LABOR, AND PENSIONS
                          UNITED STATES SENATE

                      ONE HUNDRED SEVENTH CONGRESS

                             FIRST SESSION

                                   ON


EXAMINING THE SCIENTIFIC AND ETHICAL IMPLICATIONS OF STEM CELL RESEARCH 
               AND ITS POTENTIAL TO IMPROVE HUMAN HEALTH

                               __________

                           SEPTEMBER 5, 2001
                               __________

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                                Pensions












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          COMMITTEE ON HEALTH, EDUCATION, LABOR, AND PENSIONS

               EDWARD M. KENNEDY, Massachusetts, Chairman

CHRISTOPHER J. DODD, Connecticut     JUDD GREGG, New Hampshire
TOM HARKIN, Iowa                     BILL FRIST, Tennessee
BARBARA A. MIKULSKI, Maryland        MICHAEL B. ENZI, Wyoming
JAMES M. JEFFORDS (I), Vermont       TIM HUTCHINSON, Arkansas
JEFF BINGAMAN, New Mexico            JOHN W. WARNER, Virginia
PAUL D. WELLSTONE, Minnesota         CHRISTOPHER S. BOND, Missouri
PATTY MURRAY, Washington             PAT ROBERTS, Kansas
JACK REED, Rhode Island              SUSAN M. COLLINS, Maine
JOHN EDWARDS, North Carolina         JEFF SESSIONS, Alabama
HILLARY RODHAM CLINTON, New York     MIKE DeWINE, Ohio

           J. Michael Myers, Staff Director and Chief Counsel

             Townsend Lange McNitt, Minority Staff Director

                                  (ii)

  






















                            C O N T E N T S

                               __________

                               STATEMENTS

                      Wednesday, September 5, 2001

                                                                   Page
Specter, Hon. Arlen, a U.S. Senator from Pennsylvania and Hon. 
  James Langevin, a Representative in Congress from Rhode Island.    11
Thompson, Hon. Tommy G., Secretary, U.S. Department of Health and 
  Human Services, Washington, DC.................................    15
    Prepared statement...........................................    21
    Attachment...................................................    23
Melton, Douglas, Thomas Dudley Cabot Professor in the Natural 
  Sciences, and Chair, Department of Molecular and Cellular 
  Biology, Harvard University; Karen Hersey, Senior Counsel for 
  Intellectual Property, Massachusetts Institute of Technology; 
  James F. Childress, Edwin B. Kyle Professor of Religious 
  Ethics, University of Virginia; Father Kevin FitzGerald, Doctor 
  Lauler Professor for Catholic Health Care Ethics and Associate 
  Professor, Department of Oncology, Georgetown University; and 
  John P. Chute, M.D., Head, Hematopoietic Stem Cell Studies 
  Section, NIDDK, Navy Transplantation and Autoimmunity Branch, 
  Naval Medical Research Center..................................    59

                          ADDITIONAL MATERIAL

Articles, publications, letters, etc.:
    Alpha-1 Foundation...........................................    88
    Don C. Reed..................................................    89
    Christopher C. Straub........................................    90
    Culture of Life Foundation...................................    92

                                 (iii)

  

























                           STEM CELL RESEARCH

                              ----------                              


                      WEDNESDAY, SEPTEMBER 5, 2001

                                       U.S. Senate,
       Committee on Health, Education, Labor, and Pensions,
                                                    Washington, DC.
    The committee met, pursuant to notice, at 9:40 a.m., in 
room SD-106, Dirksen Senate Office Building, Senator Kennedy 
(chairman of the committee) presiding.
    Present: Senators Kennedy, Dodd, Harkin, Mikulski, 
Jeffords, Wellstone, Murray, Reed, Edwards, Clinton, Gregg, 
Frist, Hutchinson, Warner, and Collins.

                  Opening Statement of Senator Kennedy

    The Chairman. Good morning. I would ask if our guests would 
kindly be seated so that we can move along with the hearing. We 
have an enormously important hearing today and some really 
extraordinary witnesses, and we are here to listen and to 
learn, and we are grateful to all those who have come to share 
their experience and their knowledge with us in this hearing 
this morning.
    It is a privilege to convene this hearing on the important 
issue of stem cell research. One of our greatest fears as human 
beings is that 1 day, we will learn that we or a loved one have 
cancer, Alzheimer's, diabetes, Parkinson's, heart disease, or 
any number of dread and deadly illnesses. But every day, 
thousands of Americans are stunned by such bad news. The phone 
rings, the doctor is on the line, and lives are changed forever 
by the awful news.
    Stem cell research holds the greatest promise of hope for 
millions of Americans who face these diseases. Research on 
these tiny cells may mean that the next time a doctor gives the 
bad news of a horrible disease, the doctor can also say that 
these diseases are now curable.
    So there is probably no more important work before this 
Congress than to support stem cell research, to provide life 
and hope to millions of Americans who would otherwise face 
lives of struggle, disability, and even death.
    President Bush has opened the door to Government funding 
for this important area of health research, and we look forward 
to hearing from Secretary Thompson about his plans and those of 
the administration in moving forward on stem cell research.
    The question before the Congress is whether the door is 
open wide enough; whether the stem cell lines identified by the 
administration are adequate and available for the research that 
is needed now to save lives.
    Today our committee will hear from an outstanding group of 
leaders in national policy, in science and ethics, and in the 
law. Their testimony will be a useful guide to the committee 
and to Congress as we consider action in this important area.
    We must make certain that stem cell research can fulfill 
its vast potential to improve the health and relieve the 
suffering of millions of Americans. President Bush has 
recognized the great value of this research with his recent 
decision allowing Federal funds for the work. But many in the 
scientific community are concerned that the President's 
decision establishes restrictive conditions on this critical 
research and will delay development of cures for dread diseases 
for many years, at the cost of countless lives and immeasurable 
suffering.
    Failure to seize this unprecedented medical opportunity 
would be a tragic betrayal of the hopes and dreams of millions 
of patients who expect us to do all we can to develop these new 
cures.
    The President has said that his policy, which limits 
federally-funded research to cell lines in existence before 
August 9, will make more than 60 cell lines available to 
researchers and that these cells will be adequate to conduct 
all needed research. But this conclusion is hardly clear.
    Scientists question whether many of these stem cell lines 
will actually be usable and available. The President's 
limitation gives monopoly power to only 10 organizations that 
will now control the supply of stem cells. Most of the existing 
stem cell lines would not meet Federal guidelines for safety if 
they were to be used in actual clinical work with human 
patients. These lines may deteriorate and become unusable in 
just a few years.
    New and more effective techniques for deriving stem cells 
may be developed but could not be used in federally-funded 
research under the President's guidelines.
    The questions about the President's policy are serious 
questions, and they deserve serious answers because the lives 
and health of millions of patients and their families are at 
stake. It would be unacceptable to offer these patients and 
families the promise of effective stem cell research but deny 
them the reality of it.
    Our committee today begins its oversight of stem cell 
research as it evaluates the need for legislation to assure 
vigorous, ethical stem cell research. I hope that our hearing 
will contribute to the understanding of these important 
scientific and ethical issues, and I look forward to the 
testimony of our witnesses and the comments of our colleagues.
    Senator Gregg?
    [The prepared statement of Senator Kennedy follows:]

                 Prepared Statement of Senator Kennedy

    It's a privilege to convene this hearing on the important 
issue of stem cell research. Today, our committee will hear 
from an outstanding group of leaders in national policy, in 
science, ethics and the law. Their testimony will be a useful 
guide to the committee and to Congress as we consider action in 
this important area.
    In recent years, remarkable progress has taken place in 
medical science. Advances in basic understanding of biology and 
the application of that knowledge to clinical practice have 
saved the lives and preserved the health of countless 
Americans. Many conditions that once caused disability or death 
can now be treated and even cured. Yet despite this continuing 
progress, the lives of far too many Americans are darkened by 
illness.
    Parkinson's Disease robs thousands of senior citizens of 
the power to control their own movements. Alzheimer's Disease 
impairs the mental abilities of far too many older Americans. 
Diabetes condemns children to a lifetime of insulin injections 
and the ever-present risk of coma or death. Spinal cord 
injuries confine thousands of our fellow citizens to 
wheelchairs. Cancer and heart disease exact an extraordinary 
toll of premature death on millions of Americans and their 
loved ones.
    But for each of these disorders--and for many other serious 
illnesses--there is the promise of help and hope from a 
revolutionary new medical development. Stem cells may 1 day 
liberate children with diabetes from their dependence on 
insulin injections--restore the damaged brains of patients with 
Parkinson's Disease or Alzheimer's Disease, allow the paralyzed 
to leave their wheelchairs, regenerate hearts damaged by heart 
attack or coronary artery disease, and provide new cancer 
treatments.
    This week, we have seen yet another example of the 
remarkable power of stem cells. Scientists at the University of 
Wisconsin announced that they have been able to turn stem cells 
into blood cells.
    We must make certain that stem cell research can fulfill 
its vast potential to improve the health and relieve the 
suffering of millions of Americans.
    President Bush has recognized the great value of this 
research with his recent decision allowing Federal funding for 
this important work. But many in the scientific community are 
concerned that the President's decision establishes restrictive 
conditions on this critical research and will delay development 
of cures for dread diseases for many years--at the cost of 
countless lives and immeasurable suffering. Failing to seize 
this unprecedented medical opportunity would be a tragic 
betrayal of the hopes and dreams of the millions of patients 
who expect us to do all we can to develop these new cures.
    The President has said that his policy, which limits 
federally funded research to cell lines in existence before 
August 9th, will make more than 60 cell lines available to 
researchers and that these cells will be adequate to conduct 
all needed research. But this conclusion is hardly clear.
     Far fewer than 60 stem cell lines may actually be 
available. The National Institutes of Health combed the globe 
to find stem cell lines in far-flung laboratories--but it now 
appears that many of these cell lines are untested, unproven 
and may have uncertain medical value. Even many of the 
researchers who developed these lines have said that many of 
them have not been proven to actually be stem cell lines.
     Of those that are truly stem cell lines, virtually 
none have undergone the rigorous safety testing needed to 
assure that they will be safe to transplant into patients. Most 
of the stem cell lines appear to have been mixed with cells 
from laboratory mice. Yet, just 2 weeks ago, the Department of 
HHS issued strict new safety guidelines for clinical use of 
human cells that had been mixed with animal cells. There is 
little evidence that existing cell stocks would meet these 
stringent criteria.
     Even cell lines that are safe and usable may not 
be available, because patent claims and legal restrictions may 
impede access to them. I know that Secretary Thompson has been 
working to resolve these difficulties, but many legal experts 
are skeptical that researchers will have sufficient access to 
viable stem cell lines.
     Researchers are also concerned that stem cell 
lines usable today may deteriorate and become unusable for 
research in a year or two. Under the President's policy, that 
could mean no stem cell lines at all would be available in the 
future for federally funded research.
    Stem cell research is in its earliest stages. We cannot 
know what remarkable breakthroughs will be made in the years to 
come. In particular, new ways of establishing cell lines may be 
developed that can unlock the medical potential of these cells 
far more effectively. Yet under the President's plan, NIH-
funded doctors would have no access to cells established with 
breakthrough techniques discovered after August 9th. Who knows 
what price we may pay by freezing medical progress as of this 
particular date? Imagine if we had imposed a similar 
restriction on the use of fetal tissue in 1954--a year before 
Jonas Salk announced that he had used fetal tissue in 
developing the polio vaccine that saved countless lives.
    These are serious questions, and they deserve serious 
answers, because the life and health of millions of patients 
and their families are at stake. It would be unacceptable to 
offer these patients and families the promise of effective stem 
cell research, but deny them the reality of it.
    Our committee today begins its oversight of stem cell 
research as it evaluates the need for legislation to assure 
vigorous, ethical stem cell research. I hope our hearing will 
contribute to the understanding of these important scientific 
and ethical issues, and I look forward to the testimony of our 
witnesses and the comments of our colleagues.

                   Opening Statement of Senator Gregg

    Senator Gregg. Thank you, Senator Kennedy. I thank you for 
holding this important hearing, which goes to a subject which 
has captured the Nation's attention because the potential for 
it is so immense. The potential is to address diseases which 
have plagued many citizens of our Nation and the world for 
years, for generations, and which now have a potential source 
of relief.
    Clearly, stem cell research creates the opportunity to 
provide treatments for thousands of Americans suffering from a 
variety of diseases including cancer, diabetes, Alzheimer's, 
Parkinson's, leukemia, spinal cord injuries, and various other 
areas of care.
    The therapeutic advances that have already been made by 
nearly 50 years of research on adult stem cells is very 
encouraging and deserves our continued and enthusiastic 
support, also. These therapies, unlike the unproven and 
untested potential application of embryonic stem cell 
therapies, which are at a minimum 5 and potentially 10 years 
away from clinical application, are actually being used today 
and are being used very successfully, and we should not forget 
that.
    We will hear today from Dr. Chute, who will talk about some 
of the applications of adult stem cell research and the 
therapies which have derived from adult stem cells. So as we 
embark on the road of addressing the issue of embryonic stem 
cell use, we certainly should not ignore the use of adult stem 
cells and should make that a priority of the focus of this 
committee also.
    Clearly, embryonic stem cells and the policies which the 
President have outlined, however, have become the issue which 
we are addressing as the priority in this hearing. I certainly 
congratulate the President for bringing forward this issue in 
the manner in which he has. He has presented a thoughtful and I 
think comprehensive approach to how we should proceed.
    The questions before this committee which we are to address 
are: 1) how is the President's policy going to work, and how is 
it going to be applied; and 2) we need to examine how we as a 
Federal Government should support the further research in the 
area of stem cells.
    Senator Kennedy has noted that the President has identified 
64 lines of potential stem cells which have been derived from 
embryonic cells. The issue, of course, is whether or not that 
is an adequate number and whether that number correctly 
addresses the issues which are involved, the questions of 
principles of life.
    This is not an easy issue for us to address as a political 
body, but it is clearly one which we must address. I believe 
the President has made a comprehensive and aggressive attempt 
to try to bring this issue forward in a way which will allow 
science to pursue the opportunities that are there. It is my 
belief that we should not act precipitously to expand or to set 
out on another course, but rather, see and determine what the 
effects of the President's initial proposal are. We do not yet 
really know whether the 64 lines which have been identified are 
going to be adequate and whether the science which will be 
developed from those lines will effectively address the needs 
of people who are suffering from diseases which may be relieved 
as a result of the use of those stem cells. We do not even know 
whether those stem cells are going to be available for 
application to humans because of the manner in which they have 
been derived and been maintained.
    These substantive questions have to be addressed as a 
threshold issue, I believe, before we start second-guessing the 
issue of whether the 64 lines is the appropriate number.
    So there is a lot for us to talk about, a lot for us to 
look at. I believe this hearing is really an entry-level 
discussion of the topic, but hopefully, the results of the 
hearing will be that we will set ourselves on a path of 
reaching conclusions which will move forward the great 
opportunities that lie with this new area of stem cell therapy.
    Thank you, Mr. Chairman.
    The Chairman. Thank you very much.
    Senator Harkin, we welcome your comments.

                  Opening Statement of Senator Harkin

    Senator Harkin. Thank you, Mr. Chairman, and I thank 
Senator Dodd for yielding his time.
    I will not take long, and I will ask that my statement be 
made a part of the record as well.
    The Chairman. It will be made apart of the record. We know 
that you and Senator Specter have conducted extensive hearings 
in your committee, so we are looking forward to your comments 
and to working with you.
    Senator Harkin. Thank you, Mr. Chairman.
    I applaud you for having this hearing; it is very timely. 
It is something that, as both you and Senator Gregg have said, 
has captured the public's attention and imagination all over 
this country.
    I think we have to pay very strong attention to whether we 
are opening the door, as you said, far enough to really be able 
to move aggressively in this research area. Certainly we are 
all concerned about the ethical implications of this new 
science, but we have wrestled with this over the last few 
years. We had an Ethics Committee at the NIH that came up with 
I thought very strong ethical guidelines that are now in 
existence at this point in time, but if there are others who 
have other views on this, we certainly welcome those also.
    I have become more and more convinced that we have to move 
forward on this basic research. Every week, new findings come 
out. Just yesterday, we saw the potential of using stem cells 
to make blood cells at the University of Wisconsin, and the 
potential that that alone holds for our country.
    So I think the concern that we have is whether or not, 
within the strictures of ethical guidelines, we can conduct 
good, sound science and use the massive resources of what my 
colleague and friend, Senator Specter, has often called the 
crown jewel of our Federal Government, that is, the NIH; can we 
use the power and the structure of the NIH and all the money we 
provide to the NIH to involve researchers all over this country 
to move this science forward as rapidly as possible, as I said 
again within ethical guidelines.
    That is really what I think we are about, and those are the 
questions that we have to answer. I think the essential 
question is whether there are 64 cell lines, whether they are 
viable, and whether they can actually lead to therapies in the 
future. I think these are the essential questions that we have 
to wrestle with.
    I thank you, Mr. Chairman, for holding this hearing, and I 
thank Senator Dodd for yielding me the time.
    [The prepared statement of Senator Harkin follows:]

                  Prepared Statement of Senator Harkin

    Thank you, Mr. Chairman. As you know, I've spent a great 
deal of time over the past two-and-a-half years thinking about 
stem cells. On the Labor, HHS, and Education Appropriations 
Subcommittee, Senator Specter and I have held nine hearings on 
this matter since December 1998, and we'll hold two more later 
this month.
    We've heard from scientists like Dr. James Thomson, the 
first researcher to isolate stem cells from early human 
embryos. We've heard from ethicists and religious leaders. 
We've heard from several of our Senate colleagues, who have 
offered their thoughtful perspectives on both sides of this 
issue. And we've heard from victims of the many devastating 
diseases that could be cured as a result of stem cell 
research--diseases like Parkinson's, diabetes, ALS, and 
Alzheimer's.
    After each one of those hearings, I've become more and more 
convinced that we need to move forward on this research. Every 
week, it seems, brings new findings about the potential of stem 
cells--just yesterday, in fact, scientists at the University of 
Wisconsin reported that they have figured out how to coax these 
cells into making blood.
    We need to power that research with Federal dollars, and we 
need to do it quickly, ethically, and without unnecessary red 
tape. That's why I welcomed President Bush's announcement last 
month in support of Federal funding for research on existing 
stem cell lines as a positive first step.
    Since then, scientists have raised a number of concerns 
about the President's plan. They question how many of the 64 
stem cell lines identified by the NIH will actually prove 
useful for research. And there's a possibility that all of 
these lines have been mixed with mouse cells--a situation that 
could make it dangerous to test them on humans.
    I share these concerns, but that's why I'm here today, and 
that's why my Appropriations Subcommittee will hold two more 
hearings later this month--to get some answers.
    Mr. Chairman, you've assembled an excellent panel of 
witnesses for us this morning, and I look forward to the 
testimony.
    The Chairman. Thank you very much, Senator Harkin.
    Senator Frist?

                   Opening Statement of Senator Frist

    Senator Frist. Thank you, Mr. Chairman and Senator Gregg, 
thank you for jumping right in with this hearing so that we can 
more closely and more clearly examine the important issues of 
embryonic stem cell research.
    On July 18, a little over a month ago, I announced my 
strong support for Federal funding of both embryonic and adult 
stem cell research. And, as we all know and have heard again 
and again through the press and through the various hearings 
that have been held, embryonic stem cell research holds great 
potential for advancing treatments for a broad range of 
diseases, illnesses, injuries, and conditions.
    Yet I think we need to be very, very careful at this 
juncture, early in our discussions--and yes indeed, it is early 
in the evolution of this relatively new science--not to 
oversell the promise of this research to the American people. 
We must recognize that the field of embryonic stem cell 
research is young, it is early, it is pioneering; it is not yet 
tested. The benefits of this research, although we all attach 
huge hope to this particular field, have not yet been realized, 
and they are just possibilities.
    I think we also need to recognize that there are millions 
of Americans, including myself, who hold very deeply-felt moral 
and religious concerns about research using stem cells derived 
from embryos.
    The whole topic is important for us to address in a 
straightforward way. It is important because we as a Government 
have not yet come to grips with what is the appropriate ethical 
and moral construct, how much oversight, what should the 
guidelines be, as we enter into a field, maybe for the first 
time--the one exception might be genetics--but for the first 
time enter into a field that will so profoundly affect the 
course of human life and disease by manipulation by human 
beings and altering those basic, fundamental building blocks of 
life, what makes life, what is living, and indeed what makes us 
human. We just have not had to address that in the past in much 
of science or medical science. This is the first time.
    We have the possibility of producing powerful treatments--
we talk about cures and treatments--as we address this in a 
stepwise fashion. However, there is also the possibility of 
unintended outcomes, of outcomes that are unanticipated--and 
yes, you could even throw harm or potential harm into those 
unintended outcomes.
    We are just beginning to understand the capacities of this 
science, the potential for this science, and therefore it is 
critical that we as public servants respond in a way that 
treats this pioneering research with awe and moral respect and 
great care.
    The one thing we have realized over the last several weeks 
is how little we know about the State of the art today. 
Although people can be critical of the way public policy is 
being developed, we know a lot more today than we did 4 weeks 
ago and than we did 8 weeks ago. The press has participated 
through the hearings that have been held previously and through 
this hearing today.
    All of this has made clear to me the lack of knowledge that 
we have as we address this issue of stem cells. Therefore, I 
think that, for the welfare of mankind, there is a moral 
imperative that we proceed with embryonic stem cell research 
but that we do so with caution and that we do so with 
restraint, remembering that it is untested and untried.
    I have argued in the past that we need to proceed within a 
fully transparent, carefully regulated framework that respects 
the potential of this science but, at the same time, respects 
the moral significance of the human embryo. Earlier this year, 
I set out 10 principles which I felt establish this larger 
ethical framework of oversight and regulation.
    I am very excited about the hearing today, the hearings 
that are planned among the various committees of the U.S. 
Senate and the United States Congress as we address these 
issues. I am 100 percent in support of what the President has 
put forward. For the first time--for the first time under 
President Bush's carefully balanced policy, NIH and NIH-funded 
scientists will be able to access embryonic stem cell lines 
that do hold the potential that we all understand is there.
    These issues are difficult because they involve life, human 
life, the intersection of science and religion that we simply 
have not had to address to this degree in the past. I believe 
the President of the United States has put forward a balanced 
approach that will allow stem cell research to begin 
immediately, right now, quickly, but to do so in a very careful 
way.
    We will also talk a little bit in the hearing about the 
stem cell registry, which I am delighted that the President has 
put forward, as well as the Bioethics Advisory Commission, 
which I think is critical as we go forward.
    Mr. Chairman and Senator Gregg, thank you for calling this 
hearing as we address these important issues.
    The Chairman. Thank you very much.
    If other members wish to include a statement in the record, 
they will be so included.
    [The prepared statement of Senator Jeffords follows:]

                 Prepared Statement of Senator Jeffords

    Mr. Chairman, thank you for holding this hearing on an 
extremely significant issue that will have a profound impact on 
all of us. I want to especially applaud the effort of our 
colleagues, Senator Specter and Senator Harkin for their 
leadership on this issue. The hearings they have held in the 
Appropriations Committee have been instrumental in focusing 
attention on the promise and hope of stem cell research.
    I also want to thank all of our witnesses and especially 
Secretary Thompson who, based on his experience as Governor in 
Wisconsin, has been able to provide so much guidance to the 
Administration.
    President Bush's proposal focuses on allowing Federal 
funding for research on a small subset of the stem cells 
derived from very early stage embryos. Although it is a good 
first step, we need to explore other options. We need to make 
sure that his proposal is strong enough to propel the research 
and not impede it.
    It is crucial that we allow our scientists to move forward 
with stem cell research because it holds the promise of 
providing answers for a host of diseases from Alzheimer's to 
heart disease.
    Because the possibilities to eradicate so many diseases and 
disabilities are endless, the promise has raised hope for so 
many Americans.
    Christopher Reeves, has encouraged all of us with his grit 
and determination to overcome the disability he suffers because 
of neurological damage. He knows all too well what stem cell 
research can do for him and thousands of others living with 
disabilities.
    Another brave American, with whom we are all familiar, is 
Michael J. Fox. By publicly facing his Parkinson's disease and 
becoming a spokesperson for stem cell research, he too has 
pointed to the profound promise of this research. These men and 
many other Americans have testified in front of Senator Specter 
and Harkin and in numerous other venues about the potential of 
stem cell research.
    Science has brought us to a fork in the road and I have 
confidence that we will take the right fork. We can approach 
this road morally and ethically and still continue to walk down 
a path that will save lives and minimize suffering.
    I want to thank our other witnesses as well. These are 
profound issues that require the best advice available so I'm 
looking forward to their statements. Thank you again for 
holding this hearing Mr. Chairman.
    The Chairman. We will hear now from two Members of 
Congress, first from Senator Specter, who with Senator Harkin 
has been conducting very extensive hearings in the 
Appropriations Committee on the NIH and has taken a very 
serious interest in this issue. We look forward to hearing from 
him, and after that, a good friend from my neighboring State of 
Rhode Island, Congressman Langevin, someone who has thought 
about this over a very considerable period of time and is one 
of the new very bright lights in the Congress of the United 
States who can help us all understand this issue better as 
well. We look forward to hearing from him after Senator 
Specter.
    Before we begin, I have a statement from Senator Collins.

                 Prepared Statement of Senator Collins

    Mr. Chairman, thank you for calling this important hearing 
on the President's plan for providing federal funding for 
embryonic stem cell research. I am a strong supporter of stem 
cell research and want to commend President Bush and his 
Administration for not only allowing this potentially life-
saving research to move forward, but also for moving so quickly 
and aggressively to implement the plan.
    Stem cell research holds tremendous potential to treat and 
even cure a vast array of devastating diseases and conditions, 
ranging from Alzheimer's disease, to Parkinson's disease, to 
ALS, spinal cord injury and cancer. This research has 
tremendous promise for millions of American families, and I 
applaud the President for taking such a positive step forward.
    As the founder and co-Chair of the Senate Diabetes Caucus, 
I am particularly excited about the promise that stem cell 
research holds for a cure for juvenile diabetes, which has had 
such a devastating impact on millions of American children and 
their families. Early research has shown that stem cells have 
the potential to develop into insulin-producing cells to 
replace those that have been destroyed in people with Type 1 
diabetes. One of the major limitations to success in this 
research is the limited number of insulin-producing cells for 
transplantation--an obstacle which could be overcome through 
embryonic stem cell research.
    I do have some question, however, about the restrictions 
that the President has imposed in limiting this research to 
those stem cell lines that are currently in existence. As we 
will hear this morning, some researchers have expressed concern 
that there will not be enough stem cells available to support 
the hundreds and possibly thousands of research teams that 
stand ready to investigate them, which could potentially delay 
progress in bringing stem cell therapies into medical practice. 
Others have questioned whether the 64 stem cell lines that have 
been identified are all viable and of good quality and whether 
they would all be available to researchers.
    Mr. Chairman, stem cell research offers tremendous hope to 
those suffering or dying from devastating illnesses. This 
morning's hearing provides us with an excellent opportunity to 
examine these questions and issues further, and I look forward 
to the upcoming testimony.
    The Chairman. Senator Specter?

     STATEMENTS OF HON. ARLEN SPECTER, A U.S. SENATOR FROM 
   PENNSYLVANIA AND HON. JAMES LANGEVIN, A REPRESENTATIVE IN 
                   CONGRESS FROM RHODE ISLAND

    Senator Specter. Thank you very much, Mr. Chairman and 
members of this distinguished committee, for an opportunity to 
present some of the findings which the Appropriations 
Subcommittee on Labor, Health, Human Services, and Education 
has noted during the course of some nine hearings which began 2 
weeks after stem cells were first broached on the American 
scene in November of 1998.
    The President made a profound statement on August 9 and has 
made an important opening of the door; but there is a real 
question as to whether the door is open sufficiently, and there 
is a real question about the accuracy of the facts which were 
presented to the President by the Department of Health and 
Human Services.
    A key statement by the President related to 60 stem cell 
lines now expanded to 64, but in the intervening several weeks, 
it has become apparent that many of the lines cited are not 
really viable or robust or usable. For example, Gothenburg 
University in Sweden was reputed to have 19 lines, and they 
have at most 3. In India, the researchers were supposed to have 
7 lines; none is ready for research. The San Diego Consortium 
was reputed to have 9 lines; again, none is ripe for 
utilization.
    It is up to the congressional hearings to make a detailed 
examination as to the accuracy of the representations by HHS of 
robust, viable, and diverse lines.
    Then, there are the intricate questions of informed 
consent. And then, perhaps most fundamentally is the issue of 
therapy. It was not addressed in the President's statement, but 
it has come to light in the intervening weeks that all of the 
stem cell lines have had nutrients from mice and have had 
bovine serum. Under the FDA regulations, there cannot be a 
mixing of the species.
    Now, it is a complex matter, and there have been some 
exceptions, but I think it is going to be up to Congress in 
these hearings to make the determination as to what the facts 
are.
    I think it is very important to focus on the need for an 
independent review of all of these facts. The information given 
to the President has not been complete. HHS has insisted, for 
example, that there are 19 lines from Sweden when Dr. Lars 
Hamburger has said that he personally advised the Secretary to 
the contrary. So these are facts which we must determine for 
ourselves.
    I agree totally with what Senator Gregg has said, that we 
should not be precipitous. The issue as to the hope has been 
documented in a detailed manner by responses to letters which 
Senator Harkin and I sent to all the directors of the 25 
institutes of the National Institutes of Health, who wrote back 
on the enormous potential for stem cell research in so many 
lines. And I regret but think it is necessary to inform this 
committee that when those letters were transmitted to the 
subcommittee, we found that they had been censured, and that 
many of the very, very positive statements which had been made 
by the directors of the institutes, illustrative of which is 
Dr. James Bailey, omitted from the letters we got, that ``it 
would be unfortunate if the ban on the NIH support for human 
stem cell research results in a missed opportunity to restore 
hope and quality.'' Similar statements were made by Dr. 
Klausner as to cancer and many of the other institute 
directors.
    When the issue of research was omitted, that is something 
which I believe has to proceed apace at the present time. There 
is some conversation about let us do the basic research if the 
lines are sufficient. The hearings in our subcommittee 
suggested that you need 200 lines. And bear in mind that the 
Congress has been enormously generous with NIH. That 
appropriations process started again with our subcommittee, and 
so far, we have added more than $8 billion, and by the end of 
this year, on our anticipated doubling, we will have added $12 
billion to NIH funding. So there is ample funding to proceed.
    And let there be no mistake--we believe that there should 
be funding on adult stem cell lines, on cords, on placenta, on 
every line, so that science should have the full range of 
opportunity.
    Before the President's announcement, there was considerable 
sentiment in the Congress and a considerable head of steam that 
the existing prohibition on use of NIH funds on stem cells had 
to be changed. Some 64 Senators signed a variety of letters, 
saying they favored stem cell research, and more than a dozen 
others made commitments to stem cell research but did not want 
to put it in writing. It is up to the Congress to take a look 
at these hard facts, and I suggest with a sense of urgency.
    Karl Rove has been quoted as saying, ``The President 
equates the enormity, gravity, and magnitude of this decision 
to an issue of war and peace and whether to commit American 
troops.'' That is obviously a pretty strong statement.
    My own statement has been that I believe that this issue of 
stem cell research, with its potential to touch virtually every 
family in America, all of whom are afflicted with either 
Parkinson's or Alzheimer's or heart disease or cancer, that 
there is no more important issue facing Congress except the 
issue of weapons of mass destruction.
    I have a friend and constituent in Pittsburgh named Jim 
Kordi who suffers from Parkinson's. Whenever I see Jim Kordi, 
he carries an hourglass, to remind me that the sands of time 
are passing and that the days of his life are slipping away. 
That is a pretty emphatic message from the hourglass.
    So it seems to me that this is the kind of sense of urgency 
which ought to motivate this very distinguished committee and 
what we will be doing on two hearings later this month in our 
Appropriations subcommittee.
    Thank you, Mr. Chairman.
    The Chairman. Thank you very much, Senator Specter.
    Congressman Langevin?
    Mr. Langevin. Thank you, Mr. Chairman, members of the 
committee. I would like to thank you, Chairman Kennedy, and 
Senator Gregg, and of course, my senior Senator from Rhode 
Island, Senator Reed, and the entire HELP Committee for 
convening today's hearing on stem cell research.
    I am honored to be joining Senator Specter and Secretary 
Thompson, who will be testifying later, as well as several 
eminent cellular biologists, shedding light on the 
ramifications of President Bush's August 9 decision.
    Ladies and gentlemen, the issue that we face today is not 
whether to move forward with embryonic stem cell research but 
how. How do we ensure that all unnecessary barriers to the 
research and development of life-saving cures are removed? How 
do we establish parameters that provide ethical oversight of 
this most delicate issue? And how do we help as many people as 
possible as expediently as possible?
    Unfortunately, today these questions are being answered in 
the context of a policy that impedes the potential of this 
Nation's leading scientists.
    As many of you know, on November 7 last year, I became the 
first quadriplegic elected to the United States Congress. While 
my physical condition does not define me, it does affect me on 
a daily basis, providing me with a unique perspective on stem 
cell research.
    At the age of 16, I spent my summer vacation participating 
in the Warwick Police Cadet Explorer Program. I had dreamed of 
becoming a police officer or an FBI agent for most of my young 
life. But on August 22, 1980, my dream was shattered. I stood 
in a locker room with a fellow cadet, watching two members of 
the SWAT team examining a new weapon which they thought was 
unloaded. That weapon accidentally discharged, launching a 
bullet that ricocheted off a metal locker and entered my neck, 
severing my spinal cord and leaving me paralyzed for life--
perhaps until now.
    While embryonic stem cell research could give me the chance 
to walk again, please understand that I am here today not just 
for myself or others with spinal cord injuries but also to help 
alleviate the pain and suffering of millions of people whose 
lives could be saved, lengthened and dramatically improved by 
this research.
    Nearly half of all Americans could benefit from embryonic 
stem cell research, including the one million children with 
juvenile diabetes, the 8.2 million people with cancer, the 60 
million people who are struggling with heart disease, the 4 
million Alzheimer's sufferers, the 10 million people fighting 
osteoporosis, the 43 million arthritis suffers, the quarter of 
a million people with spinal cord industries, and the 30,000 
people suffering with Lou Gehrig's disease.
    Every family in America, ladies and gentlemen, has been 
touched by these diseases and conditions, and now we have the 
opportunity to offer them real hope.
    That is why I support using stem cells derived from excess 
frozen embryos that would otherwise be discarded, which would 
allow us to save, extend, and improve lives. Every year, 
hundreds of thousands of couples experience the joy of 
childbirth through in vitro fertilization, a process which 
unfortunately creates more embryos than can be used. To 
relegate these potentially life-saving cells to the trash heap 
after the arbitrary deadline of August 9 is simply wrong.
    While I applaud the door President Bush has opened with the 
new embryonic stem cell policy, I am frustrated with the 
discovery of just how little room it leaves for medical 
advancement. Despite NIH's recent disclosure of the 64 cell 
lines that existed before August 9, we are now learning that 
they are not all ``robust'' as once claimed, and some of these 
cells are still in development and cannot yet be classified as 
lines.
    Questions about the safety of using the cells in human 
trials are also surfacing because many researchers have mixed 
human cells lines with mouse cells, which poses the risk of 
infecting people with animal viruses.
    Finally, we must recognize that irrespective of the 
President's guidelines for the existing embryonic stem cell 
lines, the private sector in the United States as well as the 
public and private sectors abroad will continue to conduct 
research on stem cells that fall outside the parameters 
established by the Bush Administration.
    I would like to conclude with this. What will we do when an 
embryonic stem cell derived from the in vitro fertilization 
process after August 9 leads to a cure for heart disease, the 
number one cause of death in this country? Will we deny 60 
million Americans this life-saving cure? And worse, what if 
such a cure is found through the morally offensive procedure of 
creating embryos purely for harvesting stem cells?
    We must fund research on other cell lines besides the 64 
cell lines identified by NIH, and we must provide strong 
oversight of this research to ensure that it is conducted by 
ethical means that do not force us to wrestle with similar 
moral quandaries in the future.
    The administration's policy impedes unprecedented life-
saving research and raises critical ethical dilemmas that we 
must not ignore. Because embryonic stem cell research cannot 
deliver on its promise of therapeutic benefit for millions of 
people under this policy, I am forced and compelled to oppose 
it.
    I understand the struggle very well to balance a pro-life 
position with embryonic stem cell research. This is perhaps one 
of the most difficult decisions that I have ever had to make. 
Having come so close to losing my own life, I am reminded every 
day of how precious the gift of life truly is. That is why I am 
pro-life.
    However, nothing is more life-affirming than using what 
otherwise would be disposed of to save, extend, and improve 
countless lives. I urge my colleagues to open the door to 
research on all excess embryonic stem cells derived in the in 
vitro fertilization process and to do so with Government 
oversight that ensures ethical research procedures.
    Mr. Chairman, I believe that as a determined Nation, we 
have an obligation to get behind this research and to see it 
move forward and offer the hope of easing so much pain and 
suffering for so many million Americans.
    Thank you, Mr. Chairman.
    The Chairman. Thank you, Congressman, for a very moving 
presentation and statement, one that obviously includes a great 
deal of thought and examination on this issue from a personal 
perspective.
    And I thank Senator Specter, who has given important 
leadership on this issue.
    I think that unless there is a different opinion, I will 
excuse our two guests, thank them for their excellent 
statements, and ask that any questions to them be submitted in 
writing.
    Senator Reed. Mr. Chairman, can I simply say how proud we 
are in Rhode Island of our Congressman and how distinguished 
and effective he is with his principled discussion of these 
issues.
    The Chairman. You certainly may.
    Senator Reed. He was an extraordinary public leader in our 
State in terms of his management as the secretary of State 
before he came here. We are all very proud of Jim and thank him 
for his wonderful statement.
    Thank you, Mr. Chairman.
    The Chairman. Thank you.
    Thank you both very, very much.
    It is a pleasure to welcome Secretary Tommy Thompson to 
speak to our committee on stem cell research. His sense of 
timing is impeccable.
    The Secretary has a longstanding interest in this important 
issue since much of the groundbreaking research in this area 
was done at the University of Wisconsin. We look forward to 
hearing from him on how the Department of Health and Human 
Services plans to implement the President's proposal for 
funding stem cell research.
    I can say just personally that the Secretary spent a great 
deal of time thinking about this issue. I know that he has 
spoken with most of the members of our committee, and I know as 
well that he spent a good deal of time during the President's 
consideration attending many of the briefings and expressing 
his views on this matter.
    We know you have given a great deal of thought to this, Mr. 
Secretary, as you do to other issues. We welcome you to the 
committee and look forward to your testimony.

STATEMENT OF HON. TOMMY G. THOMPSON, SECRETARY, U.S. DEPARTMENT 
         OF HEALTH AND HUMAN SERVICES, WASHINGTON, DC.

    Secretary Thompson. Good morning, Mr. Chairman, Senator 
Kennedy, Senator Gregg, and all the other members.
    Let me start by thanking you for holding the hearing, and 
thank you all for being so interested in a subject that is 
very, very important to the American citizens and to the world. 
I certainly appreciate this opportunity to appear before this 
committee to talk about a subject that I am very personally 
deeply involved in and have been ever since Jamie Thomson 
discovered the embryonic stem cell procedure at the University 
of Madison 3 years ago, in 1998.
    I am accompanied by Lana Skirboll, director of the Office 
of Science Policy at the NIH; Mark Rohrbaugh, deputy director 
of the Office of Technology Transfer; Maria Ferrar, director of 
the Office of Technology Transfer--and this happens to be her 
last day as a Federal employee, and she is here to support this 
procedure; and also, Kathy Zoon, director of the Center for 
Biologics Evaluation and Research at the FDA.
    Let me begin by thanking all of you for your tremendous 
support of the Department of Health and Human Services and more 
specifically NIH. I want to thank you, Chairman Kennedy and 
Senator Gregg, for your support of research, all of you who 
have worked so hard to assist us in getting the dollars 
necessary to do the medical research at NIH, and for your 
support for the great potential of stem cell research, both 
adult and embryonic.
    I have submitted my written testimony for the record, Mr. 
Chairman, but there are some additional points that I would 
like take the opportunity to make this morning.
    All of us stand today at the precipice of a new era where 
science holds the promise of curing the most devastating 
diseases. And most important, President Bush is ushering 
America into this new era by opening the door to Federal 
funding of human embryonic stem cell research in an ethical and 
solemn manner.
    The President came to a thoughtful and deliberate decision 
that the administration will support policies that preserve and 
promote the sanctity of life while allowing important medical 
research to proceed.
    There is nothing easy about human embryonic stem cell 
research. It is a complex issue with great ethical and moral 
implications. It is an issue that speaks not only to our 
greatest hopes and passions as a society but also to who we are 
as a society. The moral considerations cannot and must not be 
lost in this debate.
    President Bush sent us on a wise and deliberate course with 
his decision to allow Federal funding of research on existing 
embryonic stem cell lines. His decision balanced our Nation's 
deepest respect for life with our highest hopes for alleviating 
human suffering.
    These existing stem cell lines no longer hold the potential 
for life, but they do hold the potential to save life. It is 
that potential to save life that we must tap and bring to 
fruition, Mr. Chairman and members.
    Our challenge now is to move beyond the halls of debate 
into the laboratories of science where we can do the basic 
research that will 1 day lead us to the therapies and the 
treatments for the most horrible maladies that plague humanity. 
This is an emotional debate. It is a debate that spawns a great 
deal of speculation and feeds many misunderstandings. My hope 
is that we can clear up the misunderstandings and recognize 
that the only way that we are going to resolve the speculation 
is to do the research and to find the answers.
    The first thing we must all understand is the underlying 
need to conduct the basic research into embryonic stem cells. I 
cannot stress this point enough. We need to create a 
fundamental base of knowledge about how these cells function 
and how they can be manipulated, as well as get the answers to 
many other scientific questions.
    Some people want to make the grand leap from the onset of 
federally-funded research to the cures for Parkinson's, 
Alzheimer's, and other diseases. If only it were that easy. It 
is easy to make such a leap in the emotion of this debate, but 
it is also inaccurate and unfair to do so.
    The cures for these diseases are not just around the 
corner--I wish they were. Before we can even get to the stage 
of credibly talking about therapies for diseases, we must 
complete the basic research. This will take years, possibly 3, 
5, and maybe even 8. No one knows for sure. And Senator Frist 
probably knows more than any of us about the time it took for 
transplants in heart surgery and how long it took to perfect.
    But we now have the ability to do the basic research with 
the stimulus of Federal dollars. The role of the Federal 
Government should be and will be to make sure that that basic 
research takes place. With the support of Congress, this is 
where our investments will be going. And as we are investing in 
the basic research of embryonic stem cells, we will continue to 
fund research into the other types of stem cells, including 
adult, cord blood, and placenta. There is great value in all 
the research, and we have so much yet to learn about how much 
each can contribute to treating these diseases.
    The private sector is going to continue to pursue stem cell 
research as well. And the logic of the American free enterprise 
system suggests that President Bush's decision is going to 
provide the incentive for the private sector to get more 
involved. Once the basic research is completed, the private 
sector will likely have great incentive to step in and 
transform the basic research into therapies for disease.
    It is in that context of basic research that we must then 
address the underlying question of whether we have enough 
embryonic stem cell lines to meet the eligibility criteria. We 
believe the answer is yes. Let me explain by first addressing 
how we arrived at this number and then why we believe the 
number is sufficient.
    So far, the National Institutes of Health has identified 64 
stem cell derivations that meet the President's eligibility 
criteria. The President never spoke about or drew any limits on 
these lines based on where they were in their development. 
Furthermore, we have consistently said that these lines are at 
various stages of development. I have spoken to that fact; the 
NIH has spoken to that fact, and the NIH white paper 
identifying these derivations makes that fact crystal clear.
    But unfortunately, and I believe unfairly, some are 
choosing to engage in word games or hear only parts of the 
story. What is most unfair is that some are trying to create 
conflict between myself, the NIH, and the Swedish scientists 
from the University of Gothenburg over the number of lines they 
have that qualify for Federal funding. They have 19 lines that 
qualify for Federal funding. That is what we said; that is what 
they have.
    Let me be perfectly clear that there is no misunderstanding 
and no conflict between us. We are on the same page. In fact, I 
talked to Dr. Hamburger yesterday at 5 o'clock in the 
afternoon.
    I would like to point out to the committee that this is the 
blastocyst, and this is at 5 to 8 days. The intercell mass 
takes 3 days, depending upon what procedure you use to take 
from the blastocyst to develop the cells. They are there in the 
proliferation stage in the petri dish. This is the 
characterization, and this is what you have to do to 
characterize if you are going to have a viable embryonic stem 
cell line.
    This is the cell line established. This period takes 6 to 8 
months from here, after it is removed, until it is perfected 
and before they start freezing the cells and are able to use 
them in the vials for further research.
    The scientists in Sweden have three cell lines at this 
stage that are completed. They have four lines in the 
characterization process right now, and they have 12 in the 
proliferation stage. That is 19. All 19 of those lines qualify 
under the President's remarks for funding. That means that they 
have been taken from the blastocyst and are able, before August 
9 at 9 p.m., to qualify for Federal funding. That is the 19, 
and all 64 meet those criteria. All 64 are at different stages 
of development.
    The Gothenburg scientists have 19 derivations that meet the 
eligibility criteria, and we have always acknowledged that most 
of these are in the earliest stages of development. We agree 
with their scientists that they only have three fully-developed 
lines, but they also agree with us that they have 19 in various 
stages that meet the eligibility criteria for funding. In fact, 
the scientists from Gothenburg had inquired if 50 other 
blastocysts that they own would qualify for Federal funding as 
well; they did not, because the embryo had not yet been 
destroyed.
    I spoke yesterday with Dr. Lars Hamburger to once again 
make sure we have the same understanding, and there is 
absolutely no disagreement or misunderstanding between us.
    Now let me explain why we believe that the stem cell 
derivations that we have identified are adequate and ample for 
basic research even though some are at various stages of 
development.
    First, we will not be taking applications for funding until 
after October 1. To go through the procedure at NIH will 
probably take 8 to 9 months to get the dollars out unless there 
is an amendment to an existing grant, which could be sooner. 
During that 8 to 9 months, a lot of those 12 lines from 
Gothenburg will have been fully characterized and fully 
developed, making those available but at different stages.
    I began by putting into perspective how much work can be 
done with the use of a small number of lines--and keep in mind 
that embryonic stem cells reproduce, and to the best of our 
knowledge right now, they do so endlessly.
    For the pasta two decades, there has been research done on 
embryonic stem cells from mice. Ninety percent--20 years of 
research on mice--90 percent of that research has taken place 
with just five mice lines.
    But a more impressive example is the work being done at the 
University of Wisconsin in Madison. UW scientist James Thomson 
was the first to isolate human embryonic stem cells and I 
believe probably has more experience and knowledge working with 
them than nay scientist in the world. He has done nearly all of 
his research using just two of his five stem cell lines.
    The Wisconsin Alumni Research Foundation which owns the 
five stem cell lines and licenses them through WiCell says it 
has enough to supply every researcher with a Federal grant. 
Those are not my words; that is WiCell, that is WARF, and that 
is what Jamie Thomson has indicated to them. So that is one 
owner with just five lines who indicates that they can feed all 
the scientists in the world at the present time wishing to 
engage in this research with Federal funds. That is a powerful 
statement, yet one that many are choosing to ignore.
    What cannot be ignored is the remarkable amount of work 
already being done on these few lines. As you know, the 
University of Wisconsin Medical School reported Monday that its 
researchers under the direction of Jamie Thomson have turned 
human embryonic stem cells into blood cells called 
hematopoietic blood cells. They did so using the WARF line. 
This breakthrough is a profound contribution to the research 
and understanding of stem cells.
    I asked Jamie last night ``How soon could you put that into 
therapy?'' He said, ``If everything broke, it would probably be 
4 years, more likely 5 years or 6 years.''
    The hematopoietic blood cells means that they have 
differentiated after they have been frozen the embryonic stem 
cells into a blood cell line, which makes it different then, 
after it is differentiated from am embryonic stem cell line. 
And even while a handful of lines can supply scores of 
researchers, the good news is that we have far more than just 
the five lines from the University of Wisconsin. We have 
identified dozens of already-developed lines--64 lines that 
meet the President's criteria for Federal funding--but dozens 
of lines that have already been fully characterized and are 
ready to be sent out to researchers.
    We have identified dozens of developed lines with the 
potential for many more to become fully developed and useful. 
As I indicated, it takes 6 to 8 months to fully perfect and 
develop an embryonic stem cell line from derivation. These 
lines come from five different countries--India, Sweden, the 
United States, Australia, and Singapore.
    Certainly we wish each of the derivations were fully 
developed; but we must appreciate and we must not underestimate 
the basic research value of those stem cells in developing 
stages. We can benefit from research into their development. So 
the 12 stem cell lines from Gothenburg University at this 
stage, at the proliferation stage or even at the 
characterization stage, can offer tremendous research on what 
stage they are in and how they may be further developed. So 
just because they have not been fully established as a cell 
line does not mean the research cannot go forward on different 
aspects of embryonic stem cells.
    Certainly we wish that each of the derivations were fully 
developed; but we must appreciate and we must not underestimate 
the basic research value of those stem cells in developing 
stages. But we could even benefit from research in their 
development.
    There are some who still wish and will argue that we do not 
have enough. Well, we disagree for the reasons outlined. The 
only way that you and I will be able to fully answer that 
question correctly is to do the research, get the scientists in 
and do the research. We need to move beyond the back-and-forth 
over the numbers and get to actual work and doing the basic 
research on the science.
    The President has singled out the embryonic stem cells that 
are most immediately available for research, and he has done so 
in an ethically sound manner. We must seize the moment, 
Senators, and take advantage of the opportunities for research 
that these cells present.
    Before I wrap up, let me just quickly touch on a few of the 
other hurdles we are clearing at NIH and the Department so that 
this research can go forward.
    First, the NIH is in the process of developing a stem cell 
registry and making it available so that scientists know 
exactly what lines are eligible and who and how they can 
approach those lines for access. We are working to make the 
registry available on NIH's website very soon, and I would be 
able to tell you today that we will have that website up 
sometime within the next 10 days to 2 weeks.
    Second, the FDA is making it clear that the use of mouse 
feeder lines or layers in the development of lines is not an 
insurmountable impediment to research including clinical 
trials.
    I have a letter here, Senator Kennedy, and one for you, 
Senator Gregg, that outlines for the committee FDA's policy on 
xenotransplantation. I would like to point out that the FDA has 
assured me that the issue is not unique to embryonic stem 
cells. In fact, at the present time, we have 13 INDs dealing 
with xenotransplantation--currently--not embryonic stem cells--
13 currently under development, new drugs using 
xenotransplantation.
    They have several investigations for new drugs beyond that 
for xenotransplantation, products currently in clinical trial. 
So scientists should not let this issue deter them from 
research.
    I am submitting this letter from the FDA for the record 
that outlines their stance on this issue.
    Third, we are aggressively tackling many of the proprietary 
issues regarding the stem cell lines and their availability. We 
are encouraged that the owners of the lines want to make them 
available for basic research and are working very closely with 
all of them.
    I would like to point out parenthetically that every one of 
the entities has been in to see NIH, and I have personally 
talked to them, and every one of them wants to cooperate and 
wants to contribute and wants this basic research to get 
started and continue.
    In fact, I am very pleased today, Senator Kennedy, to 
announce that we have negotiated as of yesterday afternoon a 
memorandum of understanding that includes an MTA, Material 
Transfer Agreement, that will accelerate research on stem cells 
within the scientific community. The National Institutes of 
Health and WARF, the WiCell Research Institute, signed that MOU 
last night which will allow for the research use of its five 
existing stem cell lines that meet the eligibility criteria. 
The NIH scientists will have these lines available to them; 
they will not be limited in the amount of research they do and 
will not be limited to the publication.
    This agreement allows scientists to access these cell lines 
for their own research, permits scientists to freely publish 
the results of that research, and allows the NIH to retain its 
ownership--its ownership--of any intellectual property that 
might arise from its research using those lines. Furthermore, 
the MOU provides for a simple letter of agreement to govern the 
transfer of cell lines with minimal administrative burden.
    This is a groundbreaking agreement that we are happy to be 
able to report to this committee this morning, that hopefully 
will serve as the model for making the other lines available. 
But it also gives an indication of how serious the owners of 
these lines are about making their products available for basic 
research. We will continue to work with all stem cell owners to 
address proprietary issues.
    Carl Gulbrandsen, of the Wisconsin Alumni Research 
Foundation is here today, and I would like to take this 
opportunity to publicly thank him, the folks at WiCell and the 
dedicated team from the NIH Office of Technology Transfer, 
headed up with Maria Ferrar, for the hard work they put into 
reaching this agreement so quickly.
    This agreement gives us even more momentum and incentive to 
get to work.
    In closing, thank you again for giving me the time and the 
opportunity to outline some important and fundamental issues 
regarding the President's decision to allow embryonic stem cell 
research to go forward. Yes, I am passionate about this; I am 
excited and enthusiastic, as you are, about the President's 
decision. We all should be. There is great potential for good 
from stem cell research. But there is also much work to be 
done.
    So let us come together and move forward. The only place 
where we are truly going to find the answers to all of our 
questions is in the laboratories of America and the world. 
President Bush has opened the laboratory door. Now let us get 
our best and our brightest scientists into the lab so that they 
can do the work.
    Thank you, Mr. Chairman.
    [The prepared statement and attachment of Secretary 
Thompson follow:]
                Prepared Statement of Secretary Thompson
    Mr. Chairman, Senator Gregg, and Members of the Committee, I am 
pleased to appear before you today to testify about the President's 
decision to permit Federal funding for research using human embryonic 
stem cell lines. I am accompanied by Lana Skirboll, Director of the 
Office of Science Policy at the NIH, Mark Rohrbaugh, Deputy Director of 
the Office of Technology Transfer at the NIH, and Kathy Zoon, Director 
of the Center for Biologics Evaluation and Research at the FDA.
    I am pleased to be here today because I believe that President Bush 
has made a wise decision, one that we can only guess at the import 
right now. This was not an easy decision, and as his words to the 
Nation made crystal clear, it was not a decision that was made lightly. 
President Bush is guided by a strong set of principles, and he, like 
all of us, strongly values human life. Importantly, no Federal funds 
will be used to support the destruction or creation of new embryos, and 
Federal funds will not be used to support research on stem cell lines 
that are derived from newly destroyed embryos. The principle that the 
Federal government should not encourage or sanction the destruction of 
embryos was a cornerstone of the President's decision.
    This President, by exhibiting such strong leadership, has helped to 
create a research environment that we all hope will lead to cures for 
such diseases as diabetes, Parkinson's disease, spinal cord injury, 
stroke, Lou Gehrig's disease, and Alzheimer's disease--just to name a 
few. President Bush has given hope to us all; the scientific community, 
those suffering from these devastating illnesses, and anyone who has 
ever seen a loved one suffer from a debilitating disease.
    Very early on in my tenure at the Department, I asked NIH to 
prepare a report on what was known about human embryonic stem cells: in 
short, to do a review of the science. What investigators found, and 
what the scientific community has been saying, is that today very 
little is known. The first human embryonic stem cell was isolated only 
a little over 3 years ago. Very few people in the world are working 
with this type of cell. Yet, in that short time, and combined with 
research that has been done on animal embryonic stem cells, we all have 
every right to feel hopeful about what scientists may be able to 
accomplish.
    Human embryonic stem cells are unique. They are capable of 
continuous self-renewal and of the ability to give rise to all cell 
types that comprise the human body. But at this point, we know almost 
nothing about their potential to treat disease. And we don't know how 
they compare to adult stem cells.
    On August 9, 2001, the President announced that Federal funds may 
be awarded for research involving the use of human embryonic stem cell 
lines that meet the following criteria:
     The derivation process was initiated prior to 9:00 p.m. 
EDT on August 9, 2001;
     The stem cells were derived from an embryo that was 
created for reproductive purposes and was no longer needed for such 
purposes; and
     An informed consent was obtained for the donation of the 
embryo, and that donation did not involve financial inducements.
    When NIH first presented its report on the State of the science to 
me in June, it had documented at least 30 lines. In follow-up 
discussions, it was clear that NIH had heard about the possibility of 
others, even though scientists had not yet published their findings 
regarding some of these other lines, and many were in the early stages 
of development. So I asked NIH to go back, talk to these scientists and 
companies, and find out everything it could about the stem cell lines 
that currently exist. What investigators at NIH found was that more 
stem cell lines existed, in various stages of development, than anybody 
had realized.
    Some were identified from publications and presentations at 
scientific meetings. Others were identified in the course of preparing 
the NIH report on stem cells through general discussions with the 
scientists who had worked in the field. In some cases, the 
organizations called NIH, and in others, information was provided to 
NIH during inquiries regarding compliance with the former NIH 
Guidelines.
    To date, investigators from ten laboratories in the United States, 
Australia, India, Israel, and Sweden have reported to the NIH that they 
have derived stem cells from 64 individual, genetically diverse early 
embryos. These human embryonic stem cell lines are currently viable, 
exhibit characteristic stem cell morphology, and have undergone at 
least several population doublings. The majority of these cell lines 
are reported to express all of the markers known to be associated with 
human embryonic stem cells.
    All 64 of these human embryonic stem cell lines, which are in 
various stages of development, meet the President's criteria and are 
therefore qualified for use in federally funded human embryonic stem 
cell research. The NIH has met or spoken extensively with each of the 
investigators responsible for the derivation of these cells. These 
scientists have expressed interest in working with the NIH and the 
research community to establish a research infrastructure that ensures 
the successful handling and use of these embryonic stem cells in the 
laboratory. How researchers will be able to use these stem cell lines, 
some of which are in the earliest stages of development, for laboratory 
research is a valid and important research question, and I encourage 
scientists to begin looking for an answer as soon as possible. 
Likewise, the question of how to determine the quality or usefulness of 
any cell line is an open question that also requires research, and we 
encourage investigators to apply for NIH grants to answer it.
    By allowing, for the first time, the use of Federal funds for 
research on human embryonic stem cells, the President has opened the 
door to a promising field of scientific opportunity for some of the 
best and the brightest investigators who conduct their work with the 
financial support of the Federal government.
    I am working with NIH to ensure that the scientific community will 
be able to use Federal funds to tap the extraordinary research 
potential of human embryonic stem cells. We are hoping to begin funding 
this research using a variety of mechanisms:. grants, contracts, 
cooperative agreements, and supplements to existing grants. Today, our 
goal is to ensure that cells are available and ready for distribution 
and to help and encourage researchers who are working with these human 
embryonic stem lines, which are in various stages of development, to 
conduct the additional research that will make these lines available 
for research use. There are challenges ahead.
    First, in order to facilitate the much-needed basic research using 
human embryonic stem cells, the NIH is creating a Human Embryonic Stem 
Cell Registry that will list the human embryonic stem cells that meet 
the eligibility criteria and provide basic information about the cells.
    And there is more to do. We need to encourage the further 
characterization of those lines that are in the earliest stages of 
development. We need to provide technical assistance and funds for the 
large-scale expansion of stem cell lines so that they are available to 
as many researchers as wish to use them. We need to minimize the 
administrative burden, with regard to requests for the distribution of 
cells, both for scientists who have derived these cells and researchers 
who wish to use them. And because these cells are challenging to work 
with, we need to determine in what manner we will provide training to 
researchers on how to maintain, grow and handle 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. 
Finally, and most importantly, NIH needs to do what it does best: fund 
the most promising ideas, gain new knowledge from research, and use 
that knowledge to improve human health.
    As with other types of research, recipients of NIH funding will be 
responsible for arranging access to particular cells that they 
determine are necessary for their research. The NIH is interested both 
in accessing cells for use in its intramural research program, as well 
as in facilitating access for the broader research community. The goal 
is to encourage the transfer of cells from qualified providers under 
acceptable conditions and with as minimal an administrative burden as 
possible.
    Recently, the NIH has met with investigators who have derived these 
cells to discuss these topics. At these meetings, the NIH has, on 
behalf of its intramural investigators, initiated negotiations with 
organizations that have derived human embryonic stem cells. Although 
the NIH does not have the authority to negotiate agreements on behalf 
of grantee institutions or third parties, it has been the NIH's 
experience in other cases that the agreement into which it enters may 
serve as a model for subsequent agreements negotiated by NIH-funded 
investigators, should their institutions choose to adopt it.
    Some scientists have asked about the effect that patents filed or 
issued over the past few years will have on human embryonic stem cell 
research. The issuance of patents on new discoveries need not adversely 
affect continuing research, provided that the patent owners devise a 
licensing and sharing strategy to allow basic research to proceed. 
Experience has shown that conditions imposed by patent owners can be 
crafted both to ensure research uses and to provide appropriate 
incentives for commercial development. Although the specific terms and 
conditions of availability must be determined between providers of the 
cells and the recipients, we are pleased by the willingness of the 
researchers who have derived cells to make them available for use by 
federally funded researchers.
    Although scientists will soon have the opportunity to explore the 
promise of human embryonic stem cells, I wish to make it very clear 
that this research must proceed responsibly and ethically. We have much 
to learn about these cells--much basic research that needs to be 
conducted. Clinical applications, which could possibly emerge only 
after considerable basic research, are years away. What is important 
now is that we begin the process of gaining a thorough and 
scientifically based understanding of the promise and potential of 
embryonic stem cell research.
    The NIH is now implementing the President's policy. It is our hope 
that federally funded investigators will take full advantage of this 
new opportunity to conduct research on existing human embryonic stem 
cells and explore the enormous promise of these unique cells, including 
their potential to produce breakthrough therapies and cures. At the 
same time, NIH will continue vigorously to support research on animal 
stem cells and human adult stem cells, including those found in 
umbilical cord blood, so that in the not too distant future we will be 
in position to understand the relative benefits and limitations of all 
types of stem cells. With the help of the scientific community, this 
research will mark the beginning of a new era in modem medicine.
    Mr. Chairman, it is time for federally funded scientists to begin 
the fundamental research that is needed to determine the true potential 
of stem cells. We will provide scientists with ample opportunities to 
fully investigate this potential. We urge the research community to 
begin these explorations with the profound hope that we stand at the 
threshold of a true breakthrough in our ability to treat disease and 
disability.
                               attachment
             Department of Health & Human Services,
                                       Rockville, MD 20857,
                                                 September 5, 2001.
Hon. Judd A. Gregg,
U.S. Senate,
Washington, DC. 20515-2101.

    Dear Senator Gregg: During the past few weeks, concerns have been 
raised about human embryonic pluripotent stem cells (HEPSC) and their 
eventual use in human clinical trials for a variety of therapies. The 
Food and Drug Administration would like to take this opportunity to 
clarify these issues. A similar letter is being sent to Senator Edward 
M. Kennedy.
    While many questions are being raised, the science has not advanced 
far enough to answer them yet. HEPSCs were first isolated in 1998, and 
the scientific research about them is in its infancy. The FDA stands 
ready to work with the scientific community as they near the stage of 
human clinical trials. We encourage investigators to work closely with 
us to ensure that testing occurs in the most expedient and safest 
manner.
    Most of the concerns raised to date are not unique to human 
embryonic stem cells. The use of irradiated mouse feeder layers in 
deriving HEPSC raises concerns that also occur for other 
xenotransplantation products. FDA regulations do not prohibit using 
mouse feeder layers to make HEPSC products for human clinical trials. 
Of course, appropriate testing and precautions are necessary. FDA has a 
number of active Investigations for New Drugs (INDs) for 
xenotransplantation products currently in clinical trials.
    Xenotransplantation products raise issues that several Public 
Health Service agencies address, including the Food and Drug 
Administration (FDA), Centers for Disease Control and Prevention (CDC), 
National Institutes of Health (NIH) and Health Resource Services 
Administration (HRSA). In order to provide guidance to maximize the 
safe conduct of xenotransplantation trials, these agencies together 
with staff from the Office of the Assistant Secretary for Planning and 
Evaluation (OASPE) developed the PHS Guideline on Infectious Disease 
Issues in Xenotransplantation (1/18/01). The PHS Guideline on 
Infectious Disease Issues in Xenotransplantation and several FDA 
Guidance Documents (see below) \1\ have defined xenotransplantation as 
``any procedure that involves the transplantation, implantation, or 
infusion into a human recipient of either (a) live cells, tissues, or 
organs from a nonhuman animal source, or (b) human body fluids, cells, 
tissues or organs that have had ex vivo contact with nonhuman animal 
cells, tissues or organs.''
---------------------------------------------------------------------------
    \1\ Guidance documents defining xenotransplantation:
     PHS Guideline on Infectious Disease Issues in 
Xenotransplantation, January 2001
     Draft Guidance for Industry: Source Animal, Product, 
Preclinical, and Clinical Issues Concerning Use of Xenotransplantation 
Products in Humans, February 2001
     Draft Guidance for Industry: Precautionary Measures to 
Reduce the Possible Risk of Transmission of Zoonoses by 
Xenotransplantation Product Recipients and Their Close Contacts, 
Through Whole Blood, Blood Components, Source Plasma, and Source 
Leukocytes, December 1999
     Guidance for Industry: Public Health Issues Posed by the 
Use of Nonhuman Primate Xenografts in Humans, April 6, 1999 (The actual 
wording of the definition of xenotransplantation in this document ``the 
use of live cells, tissues, or organs from a nonhuman animal source 
transplanted or implanted into a human, or used for ex vivo contact 
with human body fluids, cells, tissues or organs that are subsequently 
given to a human recipient.'')
---------------------------------------------------------------------------
    FDA regulates xenotransplantation products using the regulatory 
framework established for other biologics. Xenotransplantation products 
are, by their nature, also cellular therapies, and some are also gene 
therapies, and as biologics are subject to the provisions of the Food, 
Drug and Cosmetics Act and the Public Health Service Act and the 
appropriate regulations in 21 CFR. A number of clinical trials of FDA-
regulated xenotransplantation protocols, including several in which the 
contact with the animal cells is ex vivo, are currently enrolling 
patients, after adequately addressing FDA's concerns. Addressing FDA's 
concerns have included use of precautions such as: (1) testing the 
xenotransplantation product for infectious agents by appropriate 
methods including co-culture assays to detect potentially unknown 
viruses, (2) educating patients regarding the potential infectious 
disease risks and monitoring and testing them for xenogeneic infections 
following treatment, (3) educating xenotransplantation product 
recipients to not donate blood or tissues for use in humans, (4) 
educating patient contacts, and in some cases monitoring them, (5) 
maintaining appropriate records of treatment with xenotransplantation 
products, (6) maintaining archived biologic samples from the patient, 
animal source, and product in the event that a public health 
investigation is needed, and (7) maintaining animal sources of 
xenotransplantation products in a manner appropriate to reducing the 
infectious disease risks of the product when used in humans. This 
latter precaution might not be necessary if a long-term established, 
well-characterized cell line with adequate documentation is used in 
xenotransplantation.
    Thus, as intended and practiced, the FDA regulation of 
xenotransplantation products, while aimed first and foremost at 
safeguarding the public health, should not impose a substantial 
impediment to xenotransplantation product development, including HEPSC 
that are produced by culture in vitro with mouse cells. HEPSC, as with 
any other product, will be reviewed on a case-by-case basis to evaluate 
safety when an application for investigational use is submitted to FDA. 
In the meantime, sponsors planning the clinical use of HEPSC that have 
been developed by exposure to animal cells should visit the FDA 
Xenotransplantation Action Plan website () where links to all the published material and transcripts can 
be found. They are also encouraged to phone the agency (301 827-2000) 
with questions and concerns. Potential sponsors of any 
xenotransplantation products are often counseled directly by FDA 
personnel on how best to comply with FDA requirements on 
xenotransplantation.
            Sincerely yours,
                         Bernard A. Schwetz, D.V.M., Ph.D.,
                              Acting Principal Deputy Commissioner.
                                 ______
                                 

    The Chairman. Thank you very much, Mr. Secretary, for your 
presentation and for the obvious thought and commitment that 
you have reflected in the presentation and your knowledge about 
this issue. That will be of great help to all of us and to the 
country in terms of developing policy.
    We will try to have 7-minute rounds; I think it will take 
at least that amount of time to ask a few questions.
    First--and I want go through very quickly--you talked in 
the presentation this morning about 64 derivations on the stem 
cells. The President talks about as a result of the private 
research, more than 60 genetically diverse stem cells already 
in existence. Maybe it is semantics--I do not think it is--but 
there was a difference in terms of those two concepts.
    Let me get to my first question. If the current stock of 
stem cells proves inadequate for effective research, or if 
there are no new breakthroughs in ways to extract stem cells, 
or if the existing cell colonies are unavailable due to patent 
restrictions, would you then consider revising the 
administration's policy to ensure that doctors will have access 
to the stem cells they need to develop the cures of the future?
    Secretary Thompson. Senator, we feel very strongly that the 
cells are available. We feel that we have already knocked down 
the proprietary concerns with the first MOU, and we think there 
are many available.
    There is also another procedure called ``subclonal'' that 
allows for the subcloning of these existing stem cell lines for 
further research being done. So we think it is adequate, and we 
do not believe that we would advise the President to change his 
policy.
    The Chairman. Well, as you know, there are many scientists 
who disagree with you about the adequacy and the availability 
of the current stem cell lines, including some of the 
scientists who developed the lines in the first place.
    As you pointed out, we only discovered human stem cells 
approximately 2\1/2\ to 3 years ago, and no one can know what 
new advances are just around the corner. We have seen repeated 
examples of the progress that scientists can make in a few 
short years--for example, it took 5 years after the start of 
the Human Genome Project just to determine the DNA sequence. 
Only 5 years after that, they sequenced the entire genetic 
code. Wouldn't it be a tragic mistake to freeze this field and 
its progress to August 9, when we do not know what improvements 
are going to come down the pike?
    Secretary Thompson. Senator, the only real answer to that 
is that we have to do the research; we have to do the basic 
research. There has not even been the comparative research done 
between embryonic, adult, placenta, and cord blood stem cell 
lines. This research has got to be done.
    I do not know if you or I or any scientist will be able to 
say at this point in time that the breakthroughs are going to 
be that quick or whether these stem cell lines are going to be 
enough. I think that they are. I really believe that. And I 
think there is adequacy to do it. But the only way that we can 
get these answers is to get our researchers and our scientists 
into the laboratory to start doing that basic research.
    The Chairman. I agree with you, and I think we all want 
them in the laboratory doing as well as they can, but as you 
well know, the stem cells receive their nutrients from mice 
tissue at the present time.
    Secretary Thompson. Mice layers; right.
    The Chairman. Yes. I will come back to this. There may be 
the development of a technology that could remove those once 
the determination is made about what those nutrients are. We 
would be denied under the August 9 restrictions, or the 
researchers would be, from taking advantage of breakthroughs in 
terms of new technologies in terms of the derivation of the 
stem cells, would we not?
    Secretary Thompson. Some scientists are working on that 
particular question as we speak, Senator.
    The Chairman. But they would be precluded from taking 
advantage of that new technology under the August 9 
restriction, would they not?
    Secretary Thompson. We do not believe so. We think that 
the----
    The Chairman. Since all the current cells now derive their 
nutrition from mice cells--all of them now----
    Secretary Thompson. That is correct.
    The Chairman [continuing]. And I will get to the question 
about that issue.
    Secretary Thompson. I am not sure----
    The Chairman. Just let me finish.
    Secretary Thompson. OK.
    The Chairman. There may very well be the ability to derive 
those products as stem cells without using the mice; under the 
August 9th restriction, that would not be possible as I 
understand it.
    Secretary Thompson. All I can say in answer to that is that 
one of the scientists did tell us that they think they have 
developed a system using pre-August 9 embryo derivations 
without mouse line. I am not at liberty and I do not know for 
sure. That was something that was said in passing in one of the 
discussions.
    The Chairman. Well, it was an example of what might happen.
    Now, here in this book are the CDC guidelines for 
transplanting animal tissues into human beings. ``All cells,'' 
it says in these guidelines--and this was published August 24--
``All cells that come in contact with human cells designed to 
be transplanted should come from animals that are from a colony 
that is continually monitored for infectious diseases or from a 
colony that was not allowed to interbreed with animals outside 
the colony or routinely tested for infectious diseases or 
quarantined for 3 weeks prior to procurement of the cells.''
    Now, virtually all of the stem cells approved under the 
President's plan were grown in the presence of mouse cells. 
these mouse cells were almost certainly not procured from 
animals treated consistent with the guidelines described above.
    Secretary Thompson. All I can tell you, Senator, is that 
these are questions that I raised with FDA, and they have 
indicated to me that they have many current clinical trials and 
INDs going on using xenotransplantation, and they have 
indicated to me--they are the experts--that they do not see 
this as really an impediment. They will be questioning it, they 
will be supervising it and doing the investigation, but as this 
letter points out that I am submitting for the record, Senator, 
it indicates that they do not believe this is a real 
impediment.
    The Chairman. This is again an area where research takes 
strong exception with the nature of the research that is being 
done.
    Let me come to another area, and this is the final area 
that I will have a chance to mention given the time. Under your 
policy, there are 10 organizations that are allowed to provide 
the stem cells to federally-funded researchers. People complain 
about OPEC being a monopoly, but even they have 11 members. I 
know that you are working hard to get adequate agreements, and 
I commend you for the agreements that you have announced here. 
You ought to be commented on that. The concern will be how many 
other agreements will be able to be signed, and that is 
basically what we want to do, because without signed agreements 
from the suppliers, it is hard for me to share the confidence 
that the problems will be satisfactorily resolved.
    You have 10 organizations; you have been able to sign up 
one or two--one--whatever it is. You have these organizations 
that basically have control on these items. How can you give us 
the assurance given that kind of restriction that there will be 
availability and accessibility for researchers to do the job 
that is out there to be done?
    Secretary Thompson. We have talked to all the entities, all 
10 of them, and all have indicated that they would like to see 
the basic research go on. All of them have cooperated. WiCell 
is the one that has the patent in the United States at the 
present time. They are the one that has signed the MOU plus the 
Material Transfer Agreement letter as of yesterday, and they 
have also indicated, Senator, that they have five cell lines 
available right now, and they have also indicated that there 
will be no limitation on the scientists who want to apply to 
use those lines and that those liens will then be able to be 
used in research, and it will not be limited to the 10 
entities. Any scientist--and they have already reached an 
agreement with the scientists at NIH to make them available for 
the NIH scientists as of today.
    The Chairman. Senator Gregg?
    Senator Gregg. Mr. Secretary, just as a threshold issue 
here, I want to clarify the situation of adult stem cell 
funding. Is it your intention and can you assure us that funds 
will not be diverted from adult stem cell funding research into 
fetal activity, fetal and embryo stem cell?
    Secretary Thompson. Absolutely not. That is the beauty of 
the President's policy, Senator Gregg, that the basic research 
on the comparison of adult stem cells, embryonic stem cells, 
placenta flat and cord blood has never been done. The best 
thing we can do is to do that basic research and get it started 
in the laboratories. And no, there will be no reduction on 
adult stem cells. We used $250 million last year on stem cell 
research, and I can assure you there is going to be more 
available for all forms of stem cells including and most 
important, adult stem cells and embryonic stem cells.
    Senator Gregg. I think that that is very important. We have 
doubled, as was mentioned, the funding for NIH as a result of 
the efforts of Senator Harkin, Senator Specter, and a lot of it 
was really started by Senator Mack when he was serving in the 
Senate. There is certainly a lot of money at NIH these days, 
and it seems to me that with this exciting new area of therapy, 
we should be able to fully fund especially the adult area which 
is already producing results. We actually have physical 
therapies that benefit from adult stem cells right now today.
    Secretary Thompson. We will not limit that at all. In fact, 
I talked with the NIH scientists as of last night and 
yesterday, and they have indicated they have enough money 
available for all good research projects. Also, because of this 
hearing and because of the President's decision and all of the 
publicity around embryonic stem cells, there is a tremendous 
amount of interest from scientists, researchers, and 
investigators around the country and the world who want to 
apply.
    Senator Gregg. Let me go through a couple of elementary 
questions, because I think there are some initial policy issues 
that we have to get clear before we go into the substance of 
some of the issues that Senator Kennedy was talking about.
    As I understand it, without Federal dollars, research on 
stem cells can proceed carte blanche; correct?
    Secretary Thompson. That is correct.
    Senator Gregg. So explain to us if you would why it is 
important to have Federal funds involved in this research.
    Secretary Thompson. Because the private dollars will want 
to go as quickly as possible to a therapy. It is more important 
to do the basic research at the beginning, the kind of research 
that Jamie Thomson and other scientists who are the forerunners 
in this research field are doing. That comparison that I talked 
about in response to a previous question that you asked, 
Senator, needs to be done, and that kind of basic research 
comparison adult, embryonic and all the other stem cell lines 
will not be done just by the private sector. The Federal 
dollars allow that. That is point number one.
    The second point is that Federal research dollars when they 
do this research encourages other scientists to get involved. 
And once the basic research is done, the privates come in and 
do more of the therapy research. So it is sort of a building 
block. That is why it is so important to get those Federal 
research dollars out quickly and into the laboratories as soon 
as possible.
    Senator Gregg. That is an important point to make.
    Second, what is the role of patents in this exercise? This 
company, Geron, which I am not familiar with, has the patents 
from the Wisconsin projects to the extent there is 
commercialization. What do you see as the role of patents in 
this exercise?
    Secretary Thompson. First of all, Geron does not have a 
patent, Senator Gregg. WiCell is a subsidiary----
    Senator Gregg. Or a license.
    Secretary Thompson. They have the license. Geron has the 
license of three of the five lines, and they have already 
reached an agreement with WiCell to make those lines available. 
That is part of the agreement, part of the MOU. Really, WARF 
and WiCell are directing this. They made the arrangements with 
NIH yesterday by signing the MOU and the Material Transfer 
Agreement letter to make these available to any scientist in 
the world who wants to apply through NIH.
    Senator Gregg. I am talking more philosophically. What is 
the role of patents in this?
    Secretary Thompson. Patents will come into play much more 
importantly when the therapies are developed and they go into 
commercial products; hopefully, as soon as possible.
    Senator Gregg. So in the basic research area, you do not 
see that as being an issue.
    Secretary Thompson. I do not see that as much of a 
restriction, no--based upon the agreement signed yesterday.
    Senator Gregg. Can you explain why you picked the date 
August 9 at 9 p.m.?
    Secretary Thompson. Because that is the time the President 
made his speech. And it was not my decision; it was the 
President's decision.
    Senator Gregg. And what is the implication of that date, in 
your opinion?
    Secretary Thompson. The implication is that there are 
embryonic stem cell lines available for basic research, there 
is money available for that basic research, and it allows us 
now to open the door to the laboratories, to get the money out, 
and to get the comparative and basic research done which is so 
important in this whole embryonic field of stem cells.
    Senator Gregg. I believe the key to that decision was that 
you believe the embryonic stem cell lines that are out there 
can be used in a manner which, because they replicate 
themselves limitlessly, I think was the term you used----
    Secretary Thompson. Endlessly.
    Senator Gregg [continuing]. Endlessly--that because of 
that, there is no need to go past that date in order to find at 
this time adequate stem cells for the research that is going 
on. Is that correct?
    Secretary Thompson. That is correct. They are pluripotent, 
which means they replicate, and as long as they do not 
differentiate, those cell lines are able to be used for basic 
research. And Jamie Thompson--we have five cell lines, and he 
is sort of the creator, the father, of the embryonic stem cell 
lines, and he personally uses about two of his stem cell lines 
for his research. As far as embryonic stem cell research, it 
has been done for 20 years on mice, and 90 percent of that 
research has been done on five cell lines.
    Senator Gregg. And it is also, as I understand, the 
position of FDA as presented to us in this letter--and I have 
not read it yet, but you are characterizing it--that the 
xenotransplantation issues are not a problem when you move from 
basic research to therapy, and they are not going to be a 
problem for the FDA as to----
    Secretary Thompson. FDA will certainly be looking at every 
facet of the procedures as we go along, but they also have 
currently 13 INDs going on with different aspects of 
xenotransplantation, and they have said that that should not 
act as the impediment or reluctance to proceed on the research. 
But they will continue to supervise, they will continue to make 
sure that it meets all of the safety rules and regulations that 
CDC and FDA have put out.
    Senator Gregg. Let me ask you another question which I 
think raises the issues that get to the issue of when--well, my 
time is up, so I am not going to get into that.
    The Chairman. Senator Dodd?
    Senator Dodd. Thank you very much, Mr. Chairman.
    Mr. Secretary, welcome.
    Secretary Thompson. Thank you, Senator Dodd.
    Senator Dodd. It is good to have you with us today, and I 
appreciate your deep interest in this and your obvious 
knowledge of it. That is obviously helpful, and I am pleased as 
well that you have some wonderful folks from NIH with you here.
    This is a tremendously important issue as you and others 
have pointed out. I have just a couple observations. I have 
asked unanimous consent that my opening statement be included 
in the record. Obviously, we are not going to resolve this 
issue specifically with a piece of legislation or even a speech 
on August 9. This is going to be an ongoing effort, and my hope 
would be that whatever policies are developed, and whatever 
finally comes out of this debate in the short term, that a 
decision will be made to revisit this issue periodically, 
almost sunset decisions, so that we will have the ability to 
come back and review decisions that have been made to determine 
whether in fact, since we are wandering or heading into 
uncharted areas, to put it mildly; I would hope that would be 
part of the consideration.
    It causes me to comment and ask you to further comment 
yourself on Senator Kennedy's questions regarding these mouse 
feeder cells--and I hear you, and I know you are confident and 
hopeful that the research will be there to be able to extract 
these mouse feeder cells so as not to contaminate these stem 
cells in any way that would jeopardize human life.
    Secretary Thompson. That is correct.
    Senator Dodd. But there is the possibility that that will 
not happen. Just as you hope that it will, we all understand 
that it may not work. So my question to you to conclude this 
line of questioning would be if in fact that is not the case, 
will the administration go back and revisit the August 9 
deadline to determine whether we can develop some new lines.
    Secretary Thompson. It is going to take such a long period 
of time to go from the laboratory to therapy to human clinical 
trials, Senator Dodd, that--everybody, including myself, has 
great anticipation and hope that we are going to be able to 
move directly to a cure for Parkinson's and so on and so forth, 
but it is going to take 5 to 8 years.
    Yesterday, the hematopoietic blood cells were developed, 
and it is probably going to take 5 years----
    Senator Dodd. I understand that.
    Secretary Thompson [continuing]. And during that period of 
time, I am sure that what I say today and the questions that 
you ask will be sort of arcane and will probably be ludicrous 
by the time we look at it 5 years from now, Senator.
    Senator Dodd. It is always dangerous when Congress--we all 
get worried about mad scientists--nothing is more frightening 
to me than Congress trying to be a scientist. So I appreciate 
what you are saying. The point I am trying to get at is sort of 
a mindset from the administration standpoint as to whether or 
not there is the flexibility to come back and revisit issues. I 
realize we cannot answer these questions, and it will take 
time, but I am trying to elicit from you as the spokesman for 
the administration your policy questions so that if in fact we 
are unable to extract the mouse feeder cells, thereby making 
these lines precarious at best, will the administration 
reconsider its August 9 deadline?
    That is really my question. I understand all the scientific 
issues that we cannot answer today.
    Secretary Thompson. The administration will not reconsider 
its deadline as far as the destruction of embryos.
    Senator Dodd. OK.
    Secretary Thompson. It will reconsider all the policies and 
all the procedures and research going into embryonic stem cells 
on an ongoing basis as we find new things----
    Senator Dodd. I understand.
    Secretary Thompson [continuing]. But the destruction of 
embryos for new embryos--no, it will not.
    Senator Dodd. OK. Let me if I can--I do not know if you 
heard Senator Specter or had a chance to read his statement, 
but he made some comments in there regarding the viability of 
the various lines that exist and raised questions about the 
Gothenburg University 19 lines, which you have addressed to 
some degree in your conversations apparently as late as 
yesterday with one of the scientists there. But I read a 
statement today that says that out of those 19--and I am 
quoting here from Hamburger, who I guess is the same fellow you 
talked to----
    Secretary Thompson. Dr. Hamburger, right.
    Senator Dodd [continuing]. He says--and I am quoting him--
``If we get three good lines out of them, we will be 
satisfied''--out of the 19.
    How many lines do we need in order to--what is the minimum 
viable lines at the end of this process that we would need in 
order for there to be a viable level of research to be 
conducted?
    Secretary Thompson. We believe that the five lines that 
currently exist will be able to get us a long way down the road 
doing the basic research, but there are many more. We feel 
right now that roughly, there are 25, 24 or 25, full cell lines 
established, and there are 64 in the various phases from the 
proliferation to the characterization to the cell line 
established, and out of those remaining ones, we think there 
will be a lot more that will be established that will be able 
to use the vials at the end where the cells are frozen and be 
able to send out to researchers around the world.
    Senator Dodd. In fact, you may want to--and I am just 
trying to understand what minimums are, and I agree with you, I 
am hopeful that is the case as well--but I want to get some 
sense of what is the bottom line that we are looking at here. 
If in fact we are only getting three, four, or five, would that 
be adequate, and your statement is that you believe it is. AT 
the end of all of this, at the end of all the 64 that are out 
there, the potential that are out there, and you go through the 
steps which you have very clearly outlined here, if at the end, 
in that last step, there are five viable lines, your answer to 
my question is that that is more than adequate?
    Secretary Thompson. We have 25, 24 or 25, adequate right 
now, so I am confident that that is enough, but I am also 
confident that there are going to be many more of the 64 that 
will be available.
    Senator Dodd. In the minute remaining, if I can--and again, 
for those of us up here who are trying to learn, this is pretty 
complicated stuff, and Bill Frist and others whom many of us 
listen to very carefully when they speak on these issues--and I 
have been fascinated from a personal standpoint with cord blood 
research and have been doing a lot of work in the last few 
days, and NIH has been very, very, helpful, and I have talked 
with them at length about the subject matter, and there is some 
great potential in cord blood research--maybe that is a subject 
for another discussion later on--but the position the 
administration has taken, that as of August 9, only those 
embryos that have already been moved in this process are the 
ones to be considered--I think all of us here would tell you 
that if there were any issue raised about whether somehow we 
were going to create a ``hatchery,'' producing embryos 
specifically for developing stem cells, I think we would 
vehemently oppose it, every one of us. I do not know of anyone 
who would take a different point of view. On the issue, 
however, of embryos being developed as a result of the 
wonderful advances in the science of in vitro fertilization, 
there are existing embryos out there, many of which will be 
destroyed. Does the administration take the position that we 
ought to pass legislation or that they are going to offer 
legislation that would ban the destruction of these embryos 
that are sitting there today that will be discarded?
    Secretary Thompson. I do not think the administration has 
taken a position on that, Senator Dodd, but I would like to 
point out that those embryos right now--in order to establish a 
good line, it takes a lot of scientific ability to do that.
    Senator Dodd. I understand.
    Secretary Thompson. And I have discussed that with Jamie 
Thomson, and he says a good line takes, with optimum 
conditions, 6 months, but it is usually closer to 8 months.
    So that right now, we have these 64 in different stages, 24 
that are almost fully developed. It would take another 8 months 
to be able under the best of circumstances to establish a 
brand, new line.
    All I am saying is that we should get the research dollars 
out there for these basic lines. And it is going to take us 
some time, because the procedure at NIH to put out grant 
dollars as they tell me will be 8 to 9 months from now. So 
during this 8 to 9 months, several of those remaining from 24 
to 64 are going to be fully developed, so there will be many 
more lines available for research and for development.
    Senator Dodd. I thank you, Mr. Secretary. My time is up, 
but just a brief, quick comment. My concern--and others will 
express their views--is with your answer to the question of 
whether you would revisit at all the August 9 deadline if in 
fact it turns out that we are not able to remove the mouse 
feeder cells. The rigidity of that concerns me, and also the 
decision--if we are going to draw ethical lines here, if we are 
going to discard cells, there will be no effort to bad the 
discarding of those unused embryos, then we are blurring the 
lines further, it seems to me, and creating some great 
potential conflict here. So again, I appreciate your 
involvement, your determination, and your passion about the 
issue and look forward to an ongoing dialogue.
    Secretary Thompson. Thank you, Senator Dodd.
    The Chairman. Senator Frist?
    Senator Frist. Thank you, Mr. Chairman.
    I briefly want to explore, since you have had so many 
conversations, the relationship between public and private 
funding. Right now, of the 64 cell lines, or the cell lines 
that are out there, all been developed with private funding, as 
of today--the research.
    Secretary Thompson. No, I cannot answer that in the 
affirmative, because some of the universities in Sweden and 
some of the lines in India and Australia I am sure could have 
had public funding.
    Senator Frist. But in the United States, we have not been 
funding----
    Secretary Thompson. In the United States, yes, that is 
correct.
    Senator Frist [continuing]. The creation of these cell 
lines.
    Secretary Thompson. I do not know if they have or they have 
not internationally, Senator Frist. I cannot answer that.
    Senator Frist. As we look ahead, we know that this science 
is moving fast.
    Secretary Thompson. Yes, it is.
    Senator Frist. It is hard even to project how long all 
these lines are going to be available, what the research is 
going to be like, what the breakthroughs are going to be, what 
the roadblocks are going to be. There is just no way to know, 
and we can debate it.
    We are going to hear the argument again and again that we 
ought to have sort of a freestanding, let us use all the cells, 
and we need unlimited cell lines.
    I think that what we have learned a lot recently is that 
you do not need unlimited cell lines, that there is a limited 
number, and now, unlike 4 weeks ago, we are saying is it 64 or 
20. At Senator Specter and Senator Harkin's hearing about a 
month and a half ago, we initiated a lot of this discussion 
about how many cell lines do you need. It is different than 
transplantation, where you take the heart out of one person, 
you transplant it into another, and it helps them. These cells 
are self-perpetuating. So we have come a long way in the 
debate.
    What is interesting as I listened broadly is that we have 
Federal funding, which is important; it legitimizes the field 
and gives you some control in terms of ethical oversight and 
prioritization. But at the same time, we have the private 
sector which, if this promise is real, is going to be heading 
off full-steam.
    Secretary Thompson. Absolutely.
    Senator Frist. And by having some public funding, in fact 
we are going to accelerate that because it legitimizes the 
field.
    What I want to make sure of, and what I have argued in the 
last several months, is that we have the ethical oversight. 
That will come through the hearing process here, but we need 
not just the ethical but the moral, ethical, and scientific 
oversight of what is going on broadly, because the drive for 
science is powerful. It is to create, it is to discover, and 
that is good. On the other hand, as I said earlier in my 
opening statement, the potential for unintended consequences is 
there. And we are dealing with the creation of life, the 
manipulation of the basic building blocks of life, and what I 
am concerned about--and we have not talked about, and we do not 
have time to go into it in much detail now--is that larger 
ethical oversight.
    The registry is a step in the right direction because that 
will give you at least a handle on the State of the art, 
private and public. The Bioethics Commission--at some point, I 
would like to hear more about that in terms of what the plans 
actually are in terms of oversight, what the focus will be, and 
the composition--you do not have to go into that right now, but 
again, I would think that they are going to look at the public 
sector as well as the private sector as we go forward.
    Secretary Thompson. Yes.
    Senator Frist. And third, I am interested in scientific 
oversight and how we as a Nation--should we legislate it; do 
you do it through HHS--provide the appropriate scientific 
oversight. Right now, we are arguing--is 64 too many, or where 
is it in terms of the 64; is it 19, is it 5, is it 4--it is 
good because we are asking the questions, but we need ongoing 
oversight so that 3 months from now, when we find out that 
there are 200 cell lines, or there are 5, appropriate decisions 
can be made, or appropriate input is received.
    And I guess what I would like to introduce as a question is 
what are we doing not just as far as Federal funding for the 60 
cell lines, but in terms of getting the larger ethical, moral, 
and scientific construct both to oversee--not regulate, but 
oversee--and monitor the issues of both public and private 
sectors?
    Secretary Thompson. That is a very good question, and let 
me respond this way, Senator. That is the reason why the 
President set up the Bioethics Committee, to look at those 
questions and to be able to make the recommendations to the 
administration on those particular questions that you have 
raised.
    In regard to the Department of Health and Human Services 
and NIH, our responsibility is to implement the policy. We are 
doing several things to implement that policy. We are setting 
up the registry. We are talking to the scientists about whether 
or not we should have a repository of these cells at NIH. Some 
scientists would like that, other scientists are fearful of 
that. We have not been able to do that.
    The third thing is to negotiate MOUs and Material Transfer 
Agreements, which we have already started.
    So we are already a long way down the road in dealing with 
this implementation question. The ethical questions certainly 
will be considered as we go along, through the Department of 
Health and Human Services, but the overall review and direction 
and new policies on these will come out of this new commission 
set up by the President, headed by Dr. Kass.
    Senator Frist. Thank you.
    Thank you, Mr. Chairman.
    The Chairman. Senator Harkin?
    Senator Harkin. Thank you, Mr. Chairman.
    Again, Secretary Thompson, I appreciated the conversation 
we had on the night the President made his decision. I have 
given my support to the President's decision. I think that he 
has moved us ahead in this area. Not being a scientist but 
having been involved with NIH for many, many years, I have been 
watching very carefully the development of this issue over the 
last couple of weeks.
    There is a lot out there that we do not know and do not 
understand; a lot of controversy about these cell lines and how 
many Sweden has or India has. We do not really have all the 
answers yet, but I think it is incumbent on us to continue to 
delve into this to see if we can shed more light on it, to 
expand our knowledge of exactly what we are doing and what we 
are talking about.
    Again, I am not saying that I do not doubt for a minute 
that the President did not give a lot of thought and a lot of 
time and consideration to this; it is obvious that he did. But 
a lot of us have been giving thought and time and consideration 
to this for years, and we still do not have all the answers.
    So I am not going to take the position that what the 
President said on August 9 is the final end-all and be-all of 
everything, because even if he did give some consideration and 
time and thought to it, he could not have given--I mean, 
because of new things that are opening up and new things that 
are happening, we have to be able to adapt to this new science.
    So my position is going to be one of basically just 
continuing to ask questions to try to find out whether or not 
under the ethical guidelines that we have established under the 
Bioethics Commission--by the way, most of which the President 
followed; the only difference was the timing, the 9 p.m. on 
August 9--but the other guidelines are all the same--informed 
consent, no monetary consideration, and only using the embryos 
for----
    Secretary Thompson. The excess.
    Senator Harkin [continuing]. And not using any therapeutic 
type of embryo development. So, basically, it is all the same 
except for that time line; it is basically the same. And we may 
want to think about that time line, too, at some other point.
    But I guess my concern is along the lines of what you said 
about the university in Sweden--and I have talked extensively 
with my colleague Senator Specter on this. Yes, there are three 
developed lines. I did understand that before I came to the 
hearing today. But the real open question is whether or not 
early on in the process, those cells that were extracted will 
lead to viable cell lines, and we do not know that yet.
    Secretary Thompson. We do not know that.
    Senator Harkin. We just do not know that. I am glad to hear 
you--you agree with that, that we do not know that yet?
    Secretary Thompson. We do not know that for sure.
    Senator Harkin. We can hope they do, obviously, but if they 
do not, and if we find out that in other cases in India and 
some of these other places, that the lines are not fully 
developed, then we might wind up with something quite less than 
60 or 64; we might wind up with something quite a bit less than 
that. Is that correct?
    Secretary Thompson. That is possible, Senator Harkin, but 
at the same--if I could answer in a little bit more detail, 
there are dozens already in existence, and the time line--it 
takes 6 to 8 months to develop a good cell line. There are four 
in the characterization stage from Sweden and 12 over here that 
have just been derived. The four look very promising, and the 
12--there are certainly some, I am sure, that will fail and 
some that will succeed. I cannot give you that number. But 
there are dozens that are available right now.
    Even at this stage, there is some great research that can 
be done on those 12, and those are questions for the scientists 
to do, and we have not seen that kind of research done yet.
    The President has allowed for the Federal dollars now to 
flow toward research in all of these areas, and hopefully soon 
to get to some therapies.
    Senator Harkin. And that is the second thing--it has to do 
with the therapies--and we will be delving into this more in 
our committee hearings in the future also. But you responded to 
I think it was either Senator Kennedy or Senator Gregg when 
they asked about the fact that the nutrient supply used for the 
development of these cells was evidently mouse cells. I did not 
know that before, but I guess this is true in almost--in every 
case.
    The Chairman. All.
    Senator Harkin. All of them.
    Secretary Thompson. Yes.
    Senator Harkin. So there is a contamination problem that I 
was not aware of before. I fully admit that I was not aware of 
this contamination problem.
    And if we are looking at just what happened yesterday at 
the University of Wisconsin and the fact that they are able to 
at least initially take some of these embryonic stem cells and 
move them into blood lines, that is pretty astounding.
    Secretary Thompson. It is exciting.
    Senator Harkin. That is astounding. In this short a period 
of time, we have done this. Mind you, they only derived these 
cells 3 years ago.
    Secretary Thompson. Three years ago.
    Senator Harkin. And now we are making red and white blood 
platelets out of it.
    Secretary Thompson. Platelets, right.
    Senator Harkin. That is pretty amazing.
    But if there is a contamination problem, we could go down 
this line for, as you say, the next 5 years or so, but be 
working with things that, while they might prove out to be 
beneficial therapeutically, would not be able to be used 
because prior on, they were grown with a nutrient out of mouse 
cells.
    It would seem, then, that we would have to go back and 
almost start all over again. Why waste all that time? My big 
concern is that I did not know and was not aware that all of 
these cell lines were grown in mouse cell nutrients as a basis, 
and that disturbs me. We need to ask the scientists more about 
what that means in terms of the therapies and how soon we can 
derive those therapies if in fact they are contaminated.
    Secretary Thompson. Can I just answer in two ways? First, 
when we started on the 64 lines, nobody knew--you did not know, 
I did not know, nobody knew--how many lines were available. I 
asked NIH to make a complete inventory of the lines--not at 
what place they were but whether or not the embryos had been 
derived as of August 9.
    NIH did it once; they did it the second time after the 
President's speech; and the third time is coming in and talking 
to them. We found that there are 64, but they are in different 
stages; but there are dozens that have already been through the 
cell line established. That is number one.
    At the bottom of the petri dish is where the mouse layers 
are placed. And it is sort of a traumatic experience for these 
cells as I understand it when they are taken away from the 
blastocyst and removed and put into the culture; they have to 
have something in order to grow and be able to replicate. That 
is probably the most difficult portion, and according to Dr. 
Thomson, that is where a lot of scientists failed. They need to 
be able to do that.
    Then, FDA has established policies, and they have looked at 
this procedure. Kathy Zoon is here. She is in charge of this, 
and she has indicated that right now, we have 13 INDs, 
Initiatives for New Drugs, that have already been started using 
xenotransplantation. And she does not believe that this alone 
is going to prevent moving from here to therapy, and neither 
does Dr. Jamie Thomson.
    Senator Harkin. I appreciate that, but we need to delve 
into this further.
    Secretary Thompson. Yes. And the blood cells, the 
hematopoietic blood cells--I talked to Dr. Thomson yesterday, 
and he said that it is going to take probably 3, 5, 6, 7 years.
    Senator Harkin. But those are still contaminated lines.
    Secretary Thompson. I do not classify them as contaminated.
    Senator Harkin. Well----
    Secretary Thompson. They have the cell----
    Senator Harkin. There is a cloud over them. There is a very 
dark cloud there.
    Secretary Thompson. I do not want to argue with you because 
I am not a scientist, and I cannot know----
    Senator Harkin. Well, I am not, either, I am not either, 
but----
    Secretary Thompson [continuing]. But I do not think they 
are contaminated, because I think they are going to be very 
useful and usable. I cannot say for sure. All I can tell you is 
what I talk to people and they tell me, Senator Harkin.
    Senator Harkin. We have got to follow up with the 
scientists on this. I do not know.
    Secretary Thompson. Yes. The scientists have got to tell us 
that. That is why it is so important to get the scientists in 
the laboratory to start answering these questions, Senator.
    The Chairman. As the Senator is winding up, as I understand 
it, mad cow resulted from a completely unanticipated transfer 
of infectious agent from beef to humans. No one anticipated 
that, no one knew about it. That is why these guidelines that 
you have issued in the CDC are so important, to make sure that 
we do not have unanticipated transfer and why those guidelines 
are so important. These stem cells that you have outlined here 
and the President, I do not believe meet those kinds of 
requirements, and looking through the letter that you sent 
here, I do not think that should give us much satisfaction. But 
we will have another chance to come back to it.
    Secretary Thompson. Senator, could I respond?
    The Chairman. Yes, please.
    Secretary Thompson. The truth of the matter is that the 
embryonic stem cells probably would not survive--would probably 
not survive--the removal from the blastocyst into the culture 
dish without the mouse layers that the cells rest upon and get 
the nutrients from. In order for us to do this research, we 
have to have this, and it is going to take 3 to 5 years to find 
out all of these answers.
    The truth of the matter is we have got to get this basic 
research done, because the questions you ask are good ones, 
Senator Kennedy, and I cannot answer them, but I do know that 
the embryonic stem cells would not grow and replicate without 
the mouse layers, because they provide the security and the 
proteins.
    The Chairman. But Mr. Secretary, they may very well have to 
use the mouse cells, but not these mouse cells--mouse cells 
that meet the kinds of requirements that CDC has outlined that 
might take 6 or 8 months. But the ones that are now being 
identified in these 64 lines do not meet the requirements of 
the protections that have been announced by your own agency and 
CDC; they just do not do that. We will spend more time on this, 
but the August 9 date arbitrarily says that we are only going 
to be able to use those that are already out there when, as you 
have said here, we could develop these in between 6 to 8 
months, and we could do it with mice that meet the requirements 
of the CDC. But we are not going to be able to do that because 
we have the August 9 deadline, nor will we be able to even 
consider research that might be able to detect the kinds of 
nutrients that are so important in the mouse cells--that is 
what it is; it is these nutrients--and many of the researchers 
that I have talked to say we will be able to find out what 
those nutrients are, and we might not even need those mouse 
cells down the road, but we are going to be prohibited from 
using those cells in the future because of the August 9 
deadline as well. These are some of the concerns.
    Secretary Thompson. But Senator, the basic research can be 
done with the Federal dollars. On the private research dollars, 
there is no prohibition or inhibition whatsoever on private 
research coming in and using a new procedure and getting it 
ready for therapy, getting it ready for the development of a 
pill or a device.
    The Chairman. Senator Warner?
    Senator Warner. Thank you, Mr. Chairman. I wish to commend 
you and our distinguished ranking member for initiating this 
hearing.
    Those of us who have been privileged to attend this hearing 
I think will remember it in years to come, because this is an 
historic threshold for the Congress. This is the first full 
committee hearing on a subject that will place challenges to 
our Republic unlike any I have seen, literally, in the 23 years 
I have been privileged to serve in the Senate.
    We are fortunate to have you at the helm at this particular 
time. You bring to your position vast experience in diverse 
subjects, and most importantly as Governor, caring for people. 
That is what the bottom line is in this.
    And I commend the President, even though I and others 
stepped out somewhat ahead of the President in a letter--which 
I would ask unanimous consent be included as part of the record 
today----
    The Chairman. It will be so included.
    Senator Warner [continuing]. Where 11 Republican Senators 
took a stance on this early on.
    [Letter follows:]
                                       U.S. Senate,
                                      Washington, DC 20510.
The President,
The White House,
Washington, DC 20510.

    Dear Mr. President: We strongly urge you to continue the last 
Administration's policy of using Federal funds for research on human 
stem cells after these cells have been derived from embryos. In 
addition, we strongly urge you to support legislation which would 
remove the existing ban on the use of Federal funds to derive stem 
cells from embryos.
    On the issue of stem cell research, we think our colleague, Senator 
Gordon Smith, went to the heart of the matter when he pointed out the 
difference between an embryo in a petri dish, which would not produce 
human life, as opposed to an embryo in the womb of a woman? where 
further development would produce life.
    The essential consideration is that there are many excess embryos 
created for the purpose of vitro fertilization.The only issue is 
whether these embryos will be discarded or used for stem cell research 
to save lives. Stem cell research has demonstrated a remarkable 
capacity of these cells to transform into any type of cell in the human 
body. Stem cells could be transplanted to any part of the body to 
replace tissue that has been damaged by disease, injury or aging. If 
scientists are correct, stem cells could be used to treat and cure a 
multitude of maladies such as Parkinson's, Alzheimer's, diabetes, ALS, 
heart disease, spinal-cord injury, all types of cancers, burns, stroke, 
macular degeneration, multiple sclerosis, muscular dystrophy, 
autoimmune diseases, hepatitis and arthritis.
    Current law prohibits Federal funding to create human embryos for 
research purposed through cloning, or through any other means. We do 
not object to these important prohibitions. However, creating embryos 
for research purposes is entirely different from using spare embryos 
left-over from infertility treatments. These spare embryos are now 
destined to be thrown away. Rather than discarding them, we support 
using these embryos in medical research to treat and cure disease.
            Sincerely,
                                             Arlen Specter.
                                            Strom Thurmond.
                                               John Chafee.
                                             Olympia Snowe.
                                   Ben Nighthorse Campbell.
                                              Gordon Smith.
                                             Susan Collins.
                                               Ted Stevens.
                                      Kay Bailey Hutchison.
                                                Orin Hatch.
                                                Dick Lugar.
                                               John McCain.
                                               John Warner.
                                 ______
                                 

    Senator Warner. So I look upon this as, not unlike our 
great pastime, a football field--at one goalpost, those 
standards of ethics, morality, and religion which have made 
this Nation the strong Nation and the envy of the world that we 
are today--and we have got to protect those in the future. At 
the other end of the field is the goalpost of alleviating human 
suffering and the need for this Nation to move ahead on the 
forefront of science.
    My particular area in this Senate is national defense. Our 
country is a superpower, not because we desire to be, but 
because the rest of the world has ceded to us that 
responsibility. We are working tonight on a military budget for 
the United States which is greater than cumulatively adding up 
all the other budgets of the world. It is astonishing. But we 
have taken that responsibility on.
    The question before this President and successive 
Presidents, the question before this Congress and successive 
Congress is are we going to be a superpower in this exciting 
science, or are we going to put in a lot of arbitrary 
standards? If that is the case, what will happen is two things. 
If our Federal system--the President setting down the 
leadership, the Congress enacting the laws to support our 
President, and the courts trying to fairly arbitrate the 
differences of views--all three of our entities of Government 
will work together on this--but if we fail to convey to the 
American people and the rest of the world that we are fair and 
have objectivity and a realistic approach to this, the Federal 
Government will be left in a cloud of dust as the private 
sector marches off on this. We will also no longer be a nation 
of importers of brain power and scientists; our own scientists 
I am fearful will go abroad to where they can work on this. 
Then, we will lose as a nation that degree of oversight and 
control over our standards of ethics and morality if they all 
depart. Then, that science will come back into the United 
States, either legally or illegally, because you are just not 
going to stop the advancement. So this is a whale of a 
challenge.
    Do you basically agree with that sort of philosophical 
approach that I have, and if so, what can we do to keep the 
private sector in a fair set of rules to do those things that 
perhaps our Federal Government will not do for the moment and 
to keep our scientists from leaving this country and going 
abroad?
    Secretary Thompson. I think your comments were very 
thoughtful and very passionate, and I could not disagree with 
anything you said, Senator, and applaud you for saying it.
    I would like to only clarify that prior to August 9, there 
were no Federal research dollars available. As of the 
President's speech there is money available. We made an 
inventory, and we have found that 64 embryonic stem cell lines 
exist that meet the President's criteria--that means for 
Federal funds.
    They are in different degrees of development. Some are in 
the proliferation stage, some are in the characterization, some 
are in the cell line established. But they are all available 
for research.
    The mouse research on embryonic stem cells has been going 
on for 20 years, and 90 percent of that work has been done on 
five lines. Dr. Jamie Thomson, who discovered the embryonic 
stem cells, has five lines, but he uses for his research 
purposes two lines, and he has lines available.
    And WiCell, who has the patent, has indicated they have 
enough cell lines available for any researcher in America who 
wants to do the research.
    I think it is a tremendous opportunity. This whole area of 
embryonic stem cells opens the door. Everybody who has some 
sort of malady or has a loved one or a dear friend who is 
suffering from dementia or cancer or so on is looking for this 
as the panacea. We have to be careful not to indicate that a 
cure is right around the corner. It is going to take several 
years. We need now to get the Federal research dollars and the 
basic research done, Senator, so that private dollars can come 
in and develop the therapies once they are developed.
    I believe that there is more interest because of this 
hearing, more interest because of the President's speech, more 
interest because of all the discussions around the country 
about embryonic stem cells, and that people know more about 
human biology and physiology than they ever have before, which 
is in and of itself a positive. And I think that scientists now 
calling NIH are excited about the potential.
    I cannot answer all the questions, and none of the 
individuals up there can at this point in time. The important 
thing is to get the research started and make the comparisons 
so we know some of the answers, so we can come back here 2 
years or 3 years from now and talk about this issue.
    Senator Warner. You are the President's principal advisor, 
and as I said, we are fortunate to have you. And I commend the 
President even though I personally feel somewhat that we should 
move a step further.
    But the point is let us take one adjunct question, and that 
is human cloning. I am totally opposed. The President is 
totally opposed.
    Secretary Thompson. So is the President, and so am I.
    Senator Warner. Should we effect here in the Congress rigid 
standards of law and indeed attach criminal penalties to that 
one area?
    Secretary Thompson. I think we should prohibit human 
cloning, Senator.
    Senator Warner. And indeed go so far as to attach criminal 
penalties?
    Secretary Thompson. That is a decision for the Senators.
    Senator Warner. I thank the chair. I thank the witness.
    The Chairman. Senator Mikulski?
    Senator Mikulski. Thank you very much, Mr. Chairman.
    Mr. Chairman, I would like to ask unanimous consent that my 
opening statement be included in the record.
    The Chairman. It will be so included.
    [The prepared statement of Senator Mikulski follows:]

                 Prepared Statement of Senator Mikulski

    Thank you, Mr. Chairman, for holding this important hearing 
on stem cell research today. I am happy to have the opportunity 
to hear from Senator Specter, Congressman Langevin, Secretary 
Thompson, and from experts on this issue. The President had a 
difficult decision before him, and I appreciate the 
deliberative, responsible way he has approached it. I know that 
stem cell research is an extraordinarily complex scientific and 
ethical issue, and I am pleased that the President took such 
great care in making his decision.
    One of the reasons I became a U.S. Senator was to save 
lives. Scientists tell us that stem cells may help lead to 
cures for diseases ranging from Alzheimer's to diabetes. Stem 
cell research could save lives. We owe it to every American 
suffering from these diseases to explore the scientific 
possibilities in front of us.
    First, I share the President's opposition to human cloning. 
I also agree that we should use our resources to investigate 
the research possibilities of stem cells derived from adults 
and from umbilical stem cells.
    Yet I disagree with the President's decision to allow 
Federal funding for embryonic stem cell research only in the 
most narrow way. I'm concerned that he has created an enormous 
loophole for researchers in private, profit making firms. 
Embryonic stem cell research will go forward with or without 
Federal funds. The President's decision leaves the majority of 
embryonic stem cell research to private firms in an unregulated 
atmosphere. My position is that there should be Federal support 
for embryonic stem cell research. With Federal funds comes 
Federal scrutiny, regulation, and transparency.
    Stem cell research offers us a cornucopia of opportunity 
that may lead to treatments or even cures for diseases like 
Alzheimer's, diabetes, Parkinson's and more. Public funding--
and the public oversight that goes with it--ensures that this 
research is conducted within a rigorous ethical framework. We 
can have both sound science and sound ethics.
    Again, I thank the Chairman for calling this hearing on 
stem cell research. I look forward to working with my 
colleagues and the Administration on this important issue, and 
I look forward to hearing from our witnesses. Thank you.
    Senator Mikulski. Mr. Secretary, I just want to really 
welcome you today and thank you, in your 6 months in office, 
for all the courtesy and collegiality and how responsive you 
have been to many issues that I have raised with you. I want to 
thank you for that.
    I also want to thank you for inviting the women in the 
Senate to meet with you to talk about a women's health agenda.
    I must say that I am impressed with the mastery that you 
have of the technical issues here. For a while there, I thought 
you were defending a Ph.D. in biology.
    Secretary Thompson. I think I could almost write one right 
now, Senator.
    Senator Mikulski. Yes, really. You can see that you have 
really put a lot of work into this personally and are very 
hands-on about it.
    Let me go right to my question, which follows along the 
line of Dr. Frist. First, in terms of President Bush's 
position, we thank him for his deliberativeness. I too oppose 
human cloning, support the adult umbilical stem cells, and am 
glad that he took the next step in embryonic.
    Let me tell you what my concern is. My concern is that the 
Bush framework creates a loophole, and the loophole is that 
embryonic research from discarded embryos will go on anyway, 
but that it will go on within the private sector environment, 
unscrutinized and unregulated, therefore lacking any type of 
transparency.
    The fears that I have about that are twofold--number one, 
that big breakthroughs could come from those who hold the reins 
of profitmaking rather than being in a large public domain and 
therefore could not only profit but even profiteer by it.
    No. 2, in an unregulated and nontransparent atmosphere, I 
am also worried about some of those grim things that President 
Bush is worried about. The biological revolution is stunning in 
terms of its opportunities, but it is also stunning in the way 
it can present to us a ghoulish, Huxley-like environment.
    I wonder what you think about this loophole and the 
concerns that I have about this, and where do you see us 
heading in that, really, so much of the research could go on in 
this unregulated, untransparent, very profitmaking environment?
    Secretary Thompson. Senator Mikulski, first off, thank you 
for your kind words. I appreciate it very much.
    The President set up this Bioethics Commission headed by 
Dr. Kass to do just exactly what you are talking about--to 
develop the theories, encourage the adoption of laws, to make 
sure that the profiteering and other problems that you have 
indicated will not exist. Saying all that, before we get there, 
I do not believe that the privates are going to do the basic 
research; they never really have. The basic research is not 
what it is going to get them the money. The basic research is 
what NIH does; it is what Johns Hopkins does, it is what 
University of Massachusetts and University of Wisconsin and all 
the others do. They are the ones who are going to have to do 
the basic research. Once that basic research is developed, I 
believe that then, the private dollars are going to come in and 
try to take that basic research--but we will have intellectual 
property rights to that. Geron, WARF, NIH--the agreement we 
signed yesterday, the MOU that we signed with WARF, allows us 
to maintain our intellectual property rights on our discoveries 
at NIH, which is exciting, and the publications that are going 
to be going on in this area.
    It is so important for us to get this research done. I 
cannot scientifically tell you that there will not be 
profiteering or a problem existing as you point out. I cannot 
say unequivocally that that is impossible. I can tell you that 
we are trying to contain any of those problems, and we are sure 
that they are in the future and not right now. What we need to 
do is get our best scientists to get the Federal dollars to do 
that basic research that is just crying for discovery, I 
believe.
    Senator Mikulski. Thank you very much, Mr. Secretary. As I 
understand it, the thrust of your presentation is that 
essentially, this is a work in progress, the stem cell 
research, both from an administrative perspective as well as 
setting our bioethical standards. We have a benchmark. 
Actually, we have broad agreement on a certain bioethical 
framework. Much is made of the prickly distinctions, but by and 
large, I think we are all agreed that we do not want grim and 
ghoulish things to go on.
    But as I understand it, while NIH begins its role, this 
Bioetehics Commission will also commence its role and be 
looking at the issues of the greater in-depth of a bioethical 
framework as well as evaluating these breakthroughs. Am I 
correct in that?
    Secretary Thompson. You are absolutely correct.
    Senator Mikulski. And in other words, we are going to be 
continually reviewing it.
    Secretary Thompson. Plus you cannot forget that Congress is 
also very much involved in holding hearings on this subjects as 
well, and making laws.
    Senator Mikulski. But my point is that Congress will 
continue to hold hearings based on now what NIH is doing and 
also the work of this commission.
    Secretary Thompson. Right.
    Senator Mikulski. But essentially, this is the framework 
for embarking on this biological revolution.
    Secretary Thompson. That is correct.
    Senator Mikulski. Now a technical question--and I think 
that is good; I think we are going to have to hold periodic 
hearings on this--these stem lines that we have talked about, 
do they have a shelf life, and if they do, this could present 
great dilemmas for the Bush position.
    I take the position of being allowed to do research on 
discarded embryos where there has been informed consent to do 
so and oppose creating embryos for therapeutic reasons. But 
what do we know about the shelf life, or should we save it for 
the ``bio gurus'' here?
    Secretary Thompson. We know that for 3 years, the first 
embryonic stem cell line is still replicating. Dr. Thomson says 
that he believes they can continue to replicate indefinitely, 
and other scientists have said the same thing. I am not a 
scientist, and I have to take what they tell me as the gospel--
but I believe that they will.
    Now, can I say conclusively that there is no shelf life? I 
cannot say that. I can tell you that they are usable as long as 
they are not differentiated. When the cell starts 
differentiating into a particular part of the body, then, like 
the hematopoietic blood cells--what has taken place at the end, 
after the embryonic stem cell line has been frozen, they have 
taken the cells out, and they have then differentiated those 
cell lines into blood cells, which is really exciting, creating 
blood, white corpuscles, red, and platelets. That to me is 
truly exciting, and it is a tremendous breakthrough, but 
whether or not it is going to be able to be used in therapy 
within 3 years, 5 years or 6 years, I do not think anybody can 
say at this point in time, Senator Mikulski.
    Senator Mikulski. Yes. There are scientists, many of whom 
are involved in cellular research, at Hopkins who apprise me 
that they do believe that these cells do have a shelf life. But 
I think that you are saying that the science is so new, we are 
going to have to see this, and should it have a shelf life, 
confront those issues, because both the Commission and NIH will 
have had a chance to do their work.
    By the way, how are we doing on getting new heads of NIH 
and FDA? You have great acting directors.
    Secretary Thompson. I have made my views known to Senator 
Kennedy and to the White House, and I will hopefully hear good 
news from both of those people.
    The Chairman. I hope to have good news, too.
    Senator Mikulski. Well, like E.F. Hutton, I cannot wait 
until I hear the news since both of these flagship agencies are 
located in my State.
    Secretary Thompson. I know. I am as anxious as you are, 
Senator Mikulski.
    Senator Mikulski. Thank you, and let me know if I can help 
move the process along--again.
    Secretary Thompson. I appreciate that.
    Senator Mikulski. Ted, can we talk later?
    The Chairman. We will talk. [Laughter.]
    Senator Murray?
    Senator Murray. Thank you very much, Mr. Chairman, and 
thank you for having this hearing.
    Clearly, this is a very complex issue, and I appreciate all 
the knowledge that you have developed on this and the 
considerable amount of effort you have taken on a very 
difficult subject. Certainly the public is very, very 
interested in what we are doing and how we are moving forward 
from many different perspectives--obviously, from moral and 
religious, but also from a perspective that they do not want us 
limiting what we can do in the future in terms of life-saving 
processes that can be developed from what we set out here 
today.
    I share the concern of Senator Mikulski in terms of private 
research. You have said very clearly that the administration 
has said that there will be no further destruction of embryos 
after the August 9 deadline, and I am worried about what kind 
of impact this will have on our research. I understand the 
commission that you have set out and that you see the Federal 
Government and NIH doing sort of basic research and then 
allowing private interests to move further. But you yourself 
know that private industry has a different motive than the 
Federal Government, which is why we have NIH and why we do 
research and why we put money into it. We can do the basic 
research with the best of intentions that the development will 
occur for diseases like multiple sclerosis or Parkinson's or 
other diseases, but private companies may well see that 
marketing and profit comes from male baldness and may set aside 
what we see as a public interest.
    I am very concerned that a commission set up to look at 
bioethical standards is not going to--they cannot determine 
what profit means, but that is what private industry will do.
    How do you reconcile this August 9 deadline with what 
private interests are going to want to do once we have done the 
basic research?
    Secretary Thompson. I clearly can reconcile it because the 
private sector would not do the basic research that I think is 
so important. I do not think the private sector would spend its 
time or money making the comparison of embryonic stem cells 
versus adult stem cells versus placenta versus cord blood. I 
think that that kind of research needs to be done for you and 
for me to make the proper decisions, and I do not think the 
private sector will do that.
    That is why, prior to August 9, the Federal research was 
not being done. The President----
    Senator Murray. But you are limiting us to that basic 
research rather to what we can develop maybe in the future, 2, 
5, 10 years from now, on specific diseases.
    Secretary Thompson. No, I am not, Senator Murray, because 
the MOU that we signed yesterday with WiCell allows us to have 
the five cell lines available right now for research and that 
the scientists at NIH will be able to keep their intellectual 
property rights and will be able to publish on what they 
discover. That may be a therapy on baldness, as you have 
indicated--I do not think that that is what we are that 
concerned with right now; we are more concerned with----
    Senator Murray. But it may be the best marketing and the 
best profit for somebody.
    Secretary Thompson. But that is for the private sector.
    Senator Murray. Right, but we have to protect the public 
interest, and my concern is that having an August 9 deadline 
set, with no intention of ever going back and looking at it 
again, may very well preclude some of the important research we 
talk about at every hearing we have on this, that we tell our 
constituents that it may be available in the future for 
diseases that touch many, many families, that it will never be 
developed because we had a hands-off approach once the basic 
research is completed.
    Secretary Thompson. I cannot answer that question except to 
say that we think there is ample supply of embryonic stem cell 
lines to do the basic research for the areas that you are 
concerned about for your citizens and that I am concerned about 
for all Americans and that the President is concerned with. He 
has just drawn a moral line, an ethical line, saying that there 
will be no further destruction of embryos that will get Federal 
funding of dollars. And that is probably something that you and 
I would never agree on.
    Senator Murray. And I guess I am just stating a concern 
here.
    Well, how do you see this affecting university research, 
which is often a collaboration of both private and Federal 
dollars?
    Secretary Thompson. I think that that is going to have to 
be worked on, but I am confident that----
    Senator Murray. How will it be worked on? Do we have a 
process in place to look at that?
    Secretary Thompson. We do not as of yet, but we are working 
on it, Senator Murray. As you know, 75 to 80 percent of NIH 
dollars go back to universities for research dollars.
    Senator Murray. Right, and it is often used in 
collaboration with private money. If private money uses stem 
cell lines that were developed after August 9, will we be 
precluded from doing collaborative research at universities on 
that?
    Secretary Thompson. That question has not been raised, 
Senator, and a decision has not been made.
    Senator Murray. I think it is an important one.
    Secretary Thompson. I cannot answer that.
    Senator Murray. Thank you, Mr. Secretary.
    Thank you, Mr. Chairman.
    The Chairman. Thank you very much, Senator Murray.
    Senator Clinton?
    Senator Clinton. Thank you, Mr. Chairman, and thank you, 
Mr. Secretary.
    I think, as probably all of us feel after these couple of 
hours together, there are a lot of unanswered questions which 
we will look forward to struggling through with you and those 
with whom you consult because clearly, as Senator Warner said, 
this is an issue that has such great importance and resonance 
not only throughout our country but throughout the world.
    I am still at the level of acquiring information and trying 
to get it straight, because there are just a lot of 
contradictory attitudes that are being expressed about what we 
have and what we do not have. I think it is important that we 
have a factual base.
    You ere not here, Mr. Secretary, for Senator Specter's 
testimony, but I am sure that your staff will fill you in and 
give you a copy of it. He certainly takes the position that is 
I think somewhat different from the one that you have expressed 
about what the viability and accessibility of the stem cell 
lines are.
    We need to come to some basic agreement about what the 
evidence shows, and I hope that you will work with us on that, 
because certainly, Senator Specter and Senator Harkin have held 
a number of hearings on this issue going back now nearly 2 
years, and Senator Specter has a different basis of information 
available to him than what you have presented. So I hope that 
we can work through that so we all know what we are talking 
about.
    Second, the serious question that has been raised about the 
potential impact, if not contamination, from the mouse cells--
and also, I believe blood serum from cattle is also used to 
nourish these cells in some of the settings--is one that we all 
have grave issues about. We are concerned that our Government's 
own standards about how to treat the introduction of animal 
elements into human material have not necessarily been followed 
in the various locations where we have identified these stem 
cells. So that clearly, we have a lot more work to do on that, 
because as you point out, the research is going to take a 
number of years, and I think many of us would be very 
disappointed if, at the end of all those years, the work could 
not be useful to the alleviation of human suffering because of 
the contamination that affected these cells.
    One other question that I need some guidance about is in 
the President's speech, I believe he said he supported the 
House bill. As I understand the House bill, it not only bans 
reproductive and therapeutic cloning in the United States and 
certainly any Federal involvement in such efforts, but it goes 
a step further and says that if, in England or Germany or 
Japan, any therapy or treatment is created from therapeutic 
cloning, that treatment could not be imported into the United 
States.
    Am I understanding that correctly in both the House bill 
and the President's position?
    Secretary Thompson. Senator Clinton, I have not done any 
research on that subject, I am sorry to say, so I do not 
particular want to answer that question at this point in time. 
I would be more than happy to do the research and call you or 
write you with an answer on that.
    But I would like to hopefully make a correction, and not to 
be confrontational. On the contamination, I do not believe that 
there is contamination. Just because these cells rest on a 
mouse layer of cells does not mean that they are contaminated.
    I have talked to FDA, I have talked to NIH, and it is such 
a traumatic experience for these cells to be taken out of the 
embryo that they have to have this cushion----
    Senator Clinton. Mr. Secretary, I understand that, but the 
point that was made this morning--and I am just trying to get a 
base of information so that I can make the best possible 
decisions--but as I understood the chairman's point this 
morning, our Government has issued guidelines about how mouse 
nutrients are to be injected into any kind of human material. 
So that clearly, we know what happens--the xenotransplantation 
efforts that you referred to are ongoing. I read the letter 
that was distributed. Frankly, it still is a little concerning 
to me, because the long list of steps to be taken to guard 
against any kind of untoward consequences of 
xenotransplantation is a little bit daunting to me. But putting 
xenotransplantation to one side, as I understand what the 
chairman said, our Government has issued very strict guidelines 
about the introduction of mouse nutrients into the human 
material, and we have no guarantee that these stem cells in 
India or in Sweden or anywhere else have followed the American 
Government's guidelines, do we?
    Secretary Thompson. We do not.
    Senator Clinton. We do not. So I do not think any of us 
wants to be confrontational. We are trying to understand what 
it is we are doing.
    Secretary Thompson. I just----
    Senator Clinton. So if our Government has rules about how 
to avoid contamination that they have issued to researchers 
about how to use mouse nutrients, I think it is a fair 
conclusion to draw that in the absence of following those 
rules, some might conclude that contamination has or could 
occur. That is my only point.
    So my only point is that as we go forward to try to 
understand what it is we are doing here, we need to be open. We 
cannot close our minds. This is not an inquisition. This is not 
trying to determine who is right and who is wrong. We are 
trying to figure out what we are doing. And there have been 
many questions raised since the President's speech that deserve 
an answer. The scientific community deserves an answer, the 
Congress deserves an answer, and certainly people who are out 
there wondering about this deserve an answer.
    So on those several points, I would appreciate additional 
information.
    Also, as we look at this, the question that was raised by 
Senator Mikulski, which I think is a very important one, is 
that we have chosen to adopt a different approach than Great 
Britain, for example. As I understand it, they have an 
Embryonic Research Advisory Board that attempts to govern both 
public and private sector investments in this research. We have 
adopted a different approach. We have adopted these 
restrictions on Federal funding and very few on private 
funding. And I take seriously your point and happen to agree 
with it that the basic research is likely to be done with the 
public sector dollars. That, though, raises some of the 
concerns that scientists have suggested to me, that is, that it 
is very difficult to do the kind of basic research without 
those public dollars. That really is the core of whatever 
therapeutic use can come from this research.
    So that we are concerned, I think, about the potential 
shelf life of these stem cell lines, the specialization that 
can occur, rendering them useless for the research, the 
numerous references to the mouse lines that have been 
developed--we do not know how many mouse lines it took to get 
those that are now replicable and usable. We are really out 
there in the dark, trying to figure it out. If we are going to 
make an analogy to the mouse lines, we ought to have as much 
information as we can to know if it was 1,000 mouse lines that 
eventually produced five viable mouse lines, or was it 64 mouse 
lines that produced five viable mouse lines. I do not have any 
idea.
    So I think that every one of us is grateful for the hard 
work that you and the NIH and the professional staff have done, 
and we are grateful that the President had a deliberative 
process that led at least to the door being opened. But as we 
learn more about these 64 stem lines, as we try to figure out 
how to reconcile Senator Specter's very strong statement--it 
was a passionate statement this morning about what he believes 
to be the facts about these stem lines--with your very 
optimistic statement about the stem lines and the fact that if 
we go with the optimistic view, which is that this is all we 
need, and this is all we are going to get, how many years do we 
lose, what do we give up--the questions that Senator Warner 
raised.
    So I think, Secretary Thompson, that many of us are still 
in the asking questions phase, and if I may, just one final 
question. You have said several times, and you said in response 
to Senator Murray that you envisioned private researchers 
taking up where the publicly-funded basic research leaves off. 
What thought has been given to how these private researchers 
will be able to overcome the proprietary rights issues as they 
engage in commercial development? With the kind of memorandum 
of understanding that you have reached between the Government 
and the research institutions--how will the private researchers 
be able to do that?
    Secretary Thompson. Senator, first off, I did not mean to 
imply anything. I just did not want to leave this hearing 
saying that all of these lines are contaminated, and I do not 
think they are.
    Second, you raised a lot of points. I do not know the 
answers. I can tell you that I have been involved in this thing 
since a month after Jamie Thomson discovered--first as a 
Governor; I have been an advocate and a passionate believer 
that this shows great promise, and I still believe that.
    We have to do the research, and we have to do the basic 
research as you have indicated, to find out how can these cells 
be used. Adult stem cells may be more usable, more placid, than 
embryonic stem cells. That comparison has not been made; that 
has to be done.
    In regard to the patent rights, I have talked to the people 
at NIH, and they tell me that this is no different, that the 
MOU and the statement of material transfer allow for this 
research to continue. The intellectual rights, the research 
writing of the scientists, will be able to continue and will be 
saved for NIH; but once they develop a product, like they do 
any other product, that does not use embryonic stem cell lines, 
the private sector and the patent laws and the commercial laws 
of America take over, and they will have to negotiate with 
WiCell or get a license to use that. But that is no different 
than any other product that has been developed by NIH or by the 
private sector when there is an already-existing patent out 
there.
    Senator Clinton. Thank you.
    Thank you, Mr. Chairman.
    The Chairman. Thank you very much.
    Senator Edwards?
    Senator Edwards. Good morning, Mr. Secretary.
    Secretary Thompson. Good morning, Senator Edwards. How are 
you?
    Senator Edwards. I am fine. We appreciate your passion 
about this issue and the work you have done on it in the past. 
There were a number of us who had concerns when the President 
made his speech about the rigid guidelines that he established, 
understanding that there are serious scientific and ethical 
questions that have to be resolved, and how those guidelines 
would be applied in the context of a very new science. I think 
all of us recognize that this is a very new and rapidly 
developing science.
    Secretary Thompson. And very exciting.
    Senator Edwards. And very exciting, potentially very 
exciting.
    I think our concern is that we want to make sure that that 
potential in fact is realized. At the time the President spoke 
and shortly thereafter when you talked about the subject, you 
talked about 60 some-odd stem cell lines being available and 
the nature of the research that you thought could be done based 
on those stem cell lines. Let me ask you first are you aware--
have you actually been on site where these stem cell lines are 
located--and we have a list of where they are across the 
country and across the world. Have you actually been on site 
and examined the stem cell lines?
    Secretary Thompson. No, I have not. I have been at the 
University of Wisconsin lab many times, but I have not been 
anyplace else, if that is your question.
    Senator Edwards. The American Society for Cell Biology has 
established four criteria that they think should apply in this 
context in order to satisfy the research that needs to be done. 
The first is that the cell line be available to publicly-funded 
scientists both at the NIH and at universities around the 
country.
    The second is that the owners of the stem cell lines not 
impose restrictions that would limit that research.
    Third is that they have growth and handling characteristics 
that are compatible with quality research.
    And fourth is that they retain the capacity to generate 
every adult cell type.
    They are pretty simple, actually. One is to make sure it is 
available to the scientists who need to do the research; 
second, that there are not legal and other restrictions that 
would limit the ability to do the research; third, that the 
stem cell lines themselves are usable for the research that 
needs to be done; and fourth, that they have the capacity to 
generate the adult cell types that need to be generated.
    Do you know as you sit here today how many of the stem cell 
lines that meet your test also meet those four simple tests?
    Secretary Thompson. I know that 64 meet the President's 
guidelines in regard to Federal funding. There are 64 that we 
have inquired about and re-inquired about and have met with 
personally.
    Senator Edwards. Can I interrupt you--do you think that 
those are reasonable tests, the tests that they lay out, those 
four simple tests?
    Secretary Thompson. I think that those four are adequate, 
and I think that they will support----
    Senator Edwards. You think they are reasonable.
    Secretary Thompson. They are reasonable.
    Senator Edwards. OK. Can you tell me as you sit here 
today--and you and the President have already established the 
limitations--can you tell me as you sit here today how many 
stem cell lines meet those four tests?
    Secretary Thompson. Dozens.
    Senator Edwards. What does that mean, ``dozens''--25, 30? 
How many?
    Secretary Thompson. I would say--dozens--I would say 20 to 
25 at this point in time. But--you were not here--there are 
three stages that these embryonic stem cell lines go through. 
The first one is the proliferation, and there are several of 
those 64 lines that are in the proliferation line; there are 
several in the characterization line, which is over here; and 
several are in the ``cell line established.'' It takes 6 to 8 
months to develop a good embryonic stem cell line, and there 
are some of these that have not met the full cell line 
establishment criteria yet--they will be by the time they apply 
for the money, and the money goes out at NIH. That is going to 
take somewhere between 8 or 9 months to get the money out.
    To give you an example, in Sweden, Gothenburg University 
has 19 lines, Senator Edwards. Three of them are in the ``cell 
line established,'' which means they are ready right now for 
research and that further research could be done. Four of them 
are in characterization, and 12 are in proliferation.
    Over the course of the next 6 to 8 months, several will 
move from proliferation to characterization to cell line 
established, and that is during the process that they will be 
applying for Federal research dollars.
    To tell you exactly today how many, I cannot tell you. If I 
were pressed, I would say there would be 24 to 25 that would 
meet the ``cell line established'' criteria, Senator Edwards.
    Senator Edwards. That leads me to my next question. When I 
originally asked you, you said ``dozens,'' and then, when I 
asked you a follow-up question about it, you said ``24 or 25,'' 
something in the----
    Secretary Thompson. That is what----
    Senator Edwards. How do you know that those 24 or 25 meet 
this reasonable, what you described as reasonable criteria that 
the cell biologists have established?
    Secretary Thompson. Basically, because the MOUs that we 
have discussed with the 10 entities that have them have 
indicated they want to share with the scientists; two, that 
they can be used; three, that they replicate or are 
pluripotent, which is the scientific word for that. And I do 
not know what the fourth one was, but as I understand it, they 
meet those four qualifications.
    Senator Edwards. Let me ask a follow-up question to that. 
How many genetically diverse stem cell lines, again as we sit 
here today--not the potential, but that we actually know 
about--have the ability to regenerate themselves indefinitely? 
How many do you know about today?
    Secretary Thompson. We think all 64.
    Senator Edwards. OK. So it is some of the other tests that 
you are not certain whether they are----
    Secretary Thompson. Yes. The other characterizations, the 
other things like the freeze-thaw testing, the cell marker 
assays, which are SSEA-3's and 4's and PTA-61's and 80's, and 
the chromosome analysis, and the testing for pluripotency--we 
are not sure about all of those things. But we do know that in 
order for this to be proliferating, they have already started 
proliferating right after they have been removed from the 
blastocyst and are in the cells in the culture dish. They are 
proliferating right then. So all 64 lines have proliferated; we 
do know that for a fact.
    Senator Edwards. But you, HHS, NIH, have not physically 
been to the sites to actually examine the cells yourselves?
    Secretary Thompson. No.
    Senator Edwards. Do you plan to do that?
    Secretary Thompson. I probably will not, but I know the 
scientists will be doing an exhaustive study, which they 
already are. We are in the process of setting up the registry. 
We have signed--you were not here--but we did sign an MOU and a 
Material Transfer Agreement yesterday with Wisconsin Alumni 
Research Foundation that allows for their five embryonic stem 
cell lines to be made available to the NIH scientists right 
now.
    Senator Edwards. Are those five lines available to the 
nonNIH scientists around the country, publicly-funded 
scientists?
    Secretary Thompson. Yes. Carl Gulbrandsen is here, and you 
can ask him that question, but he has indicated that, yes, any 
scientists who want those lines----
    Senator Edwards. And what do we know about the availability 
of all the other stem cell lines?
    Secretary Thompson. All we know--all I can tell you, 
Senator Edwards, is that the entities have been in to meet with 
Dr. Lana Skirboll and her staff, and I have personally met with 
them. I have not negotiated with them, but I have met with 
them, and they have all indicated to Dr. Lana Skirboll and to 
the staff at NIH that they want to make their cell lines 
available to scientists and for NIH to get the basic research 
done. They are excited.
    Senator Edwards. But at this point, who controls that 
decision? Do they control it?
    Secretary Thompson. The entities, the entities.
    Senator Edwards. We as a nation have left to them control 
over what kind of research can be done on these cell lines?
    Secretary Thompson. No, no. They own it; I mean, they have 
developed them, so they have control over them. And now what we 
are doing is----
    Senator Edwards. So we have created through this decision 
by you and the President a monopoly in those people who have 
existing cell lines. If I understand you correctly, what you 
are saying is that they now have control over what research, if 
any, can be done by--it is all well and good that they are 
well-intentioned about it--I appreciate that--but suppose they 
decide that no, in fact, they are not going to permit the 
research to take place, or they are only going to allow the NIH 
to do it, they are not going to allow the other scientists to 
do it?
    Secretary Thompson. That is not what I have said, Senator. 
What I have said is that they have all indicated that they are 
in agreement and want to cooperate not only with NIH but with 
other scientists to do the basic research on their stem cell 
lines.
    WARF has gone a step further. They have negotiated now with 
NIH an actual agreement which was signed yesterday with NIH to 
make their five cell lines that Jamie Thomson, the father of 
embryonic stem cells, has developed. He has licensed those to 
WARF; WARF in turn has licensed them or has given the 
permission to be used by NIH and other scientists through an 
MOU and a Material Transfer Agreement for any type of research 
that scientists want to do on those.
    The second thing that we are doing is developing a registry 
at NIH, making all of these 64 lines hopefully available for 
scientists around the world to be able to pick the line that 
they want to use for their research projects when they apply 
for Federal research dollars.
    Senator Edwards. The critical word there was ``hopefully,'' 
and I know my time is up, but my obvious concern is--and I 
think it is a critical issue in this ongoing process--that by 
establishing the criteria that the President established, he 
limited the number of lines that are even potentially available 
and left in the hands of the people who presently have the 
proprietary control of those lines the decisionmaking about 
whether they are going to be publicly available. We cannot 
force them to make them available; they have a legal right to 
do whatever the choose to do. And I appreciate that they are 
well-intentioned, but it certainly has not been an unusual 
thing in the past to have people act out of their own personal 
financial best interest, and it would not be a shocking thing 
to see that happen in this case.
    So I think the restrictions that have been imposed 
obviously create ongoing problems. I am hopeful with you that 
we will be able to get these folks to make these stem cell 
lines available, but I think it is a serious question as we sit 
here today.
    I thank you, Mr. Secretary, for your answers.
    Secretary Thompson. Thank you. All I can tell you in 
answer, Senator Edwards, is that we have five--the first one we 
have negotiated with, which is the one that has the patent in 
America, is the one we have reached an agreement with, and we 
feel very good about that, and we think the other ones will 
follow through.
    I cannot promise you today that we are definitely going to 
have them all signed up next week, but we are working on them, 
and we are working as fast as we possibly can to reach an 
agreement with them.
    Senator Edwards. Thank you, Mr. Secretary.
    Thank you, Mr. Chairman.
    The Chairman. Thank you.
    Mr. Secretary, I will just make a very brief comments, and 
if others wish to ask a final question, obviously, they are 
welcome to.
    No one could sit through the course of this hearing since 
9:30 and not know of your own very strong commitment in terms 
of research in this area and your belief that this offers some 
enormous opportunities and hope for families across this 
country in regard to these dreaded diseases that afflict every 
family in America--every family in America. That is very 
commendable. The President is fortunate to have the kind of 
advocate and advocacy that you have expressed here.
    There are those who view this situation somewhat 
differently, and we are here trying to listen as we have 
carefully to your presentation, made in a very convincing way. 
Will there be the availability of stem cells for the research 
and the opportunities in an area that was really unthought of, 
or maybe thought of but not in reality until 2\1/2\ or 3 years 
ago, and with the best of the research that is done at NIH. 
Will there be sufficient product out there to permit 
opportunities for breakthroughs?
    You make a very convincing and compelling case that there 
is, and I think there have been some legitimate issues and 
questions raised that cause us some concern. I think one of 
those issues that I raised very briefly, as others have, is the 
question of the conditions of safety and the issue of the use 
of nutrients from mice and the fact that in these various stem 
cell lines that you talk about here, you would be hardpressed 
to think that any of those followed the very strict guidelines 
that were issued on August 24th of this year on mice, from a 
colony that is continuously monitored for infectious disease. 
We know about mad cow disease; no one expected that that kind 
of infectious disease would be able to move from animals to 
human beings from a colony that was not allowed to interbreed 
with animals outside the colony and was routinely tested for 
infectious diseases and quarantined for 3 weeks prior to the 
procurement of cells.
    Now we are told that we have to stay with these cells; even 
if we are able to take mice and create new stem cell lines 
further after August 9 that would meet all the requirements 
that the administration and CDC have put out, you are saying, 
``No, no, you cannot do it. August 9th is it.'' And even if we 
are able to discover after 2\1/2\ years that we may be able to 
do it without nutrients from mice--maybe other kinds of 
nutrients would save us that kind of risk--we are told that we 
cannot do it because it is after August 9.
    We commend you for the arrangements that you have made in 
being able to get one of the ten suppliers to make a strong 
commitment to make available their stem cell lines, and we are 
impressed by your own work in the past and saying that you have 
been in touch with all the others, and you believe that they 
will make them available. I think most of us would say that no 
one underestimates your ability to talk to someone and get an 
understanding and form an impression about whether that is for 
real or not, and I have a lot of respect for that. I think many 
would say we ought to get it in writing, because what we have 
seen as I understand it from similar situation is that there 
has not been the kind of readily available access when similar 
situations have been out there in terms of research at the NIH.
    I think most of us have been around here long enough to see 
what OPEC was able to do to our economy and the impact that it 
has had on our society when they get together, 11 of them. And 
we are talking not about 11 countries here, we are talking 
about some countries, but basically 10 entities, and that 
raises concerns.
    So these are all important issues. We had comments about 
the patents, and you have commented about that, and I know you 
have worked on it. So these issues do raise some serious 
questions, but as far as I am concerned, I want to take what 
you have said here today and hear from our next panel their 
reactions, and then to work with you.
    I do find at the end of the day that an August 9 deadline, 
sort of a drop-dead date, and the unwillingness, in spite of 
what follows in terms of breakthroughs in the areas of 
research, would serve as a barrier to important progress in 
terms of cancer or Alzheimer's, very troublesome.
    But we have learned a good deal this morning, and I have 
been enormously impressed by your presentation. We will keep in 
close touch with you on this issue. We want no surprises--there 
could not be in any event. We want to work with the 
administration. We again thank the President for addressing it. 
It is just a desire to make sure that we are going to be able 
to get done what he has said he wanted done and what I think 
offers the greatest hope for the American people.
    Senator Dodd?
    Secretary Thompson. Can I just say thank you, Senator 
Kennedy, for your tremendous interest and passion on this 
subject. We may not totally agree on whether or not the August 
9 deadline is correct or not, but I think we both agree that we 
need to get the research done and the questions answered----
    The Chairman. Yes, yes.
    Secretary Thompson [continuing]. And I thank you for your 
passion and your advocacy on this issue, and I want to publicly 
State that I support that and want to work with you as much as 
I possible can to accomplish that.
    The Chairman. Thank you very much.
    Senator Dodd. Mr. Secretary, just to pick up on some of 
Senator Kennedy's comments--and Senator Clinton used the right 
word--this is not an ``inquisition'' at all. Obviously, there 
is deep interest in the subject matter not only here, obviously 
in the scientific community, and as you have heard, painfully, 
many cases from people at-large around the country who do not 
understand all of this. The news media may be in a sense hyping 
a bit what the potential is, but understandably, when you have 
a family member or a loved one who is suffering, and you hear 
about the potential to be able to deal with Alzheimer's or 
diabetes and other such illnesses, it is very, very exciting.
    I for one want to thank you. You are in the box here, and 
we are raising the questions to you, but I think you deserve a 
great deal of credit for bringing this as far along as you 
have. There are some of us here who would have liked to see the 
administration go a bit further with this, in my view--not that 
I wanted to see it all; I strenuously oppose the idea of 
somehow creating what someone once used the word 
``hatcheries''; I would vehemently oppose that--but I also 
understand that with the ability to provide people with 
families through in vitro fertilization, and that there are 
excess embryos here--and I raised with issue you earlier as to 
whether or not there is then going to be a position taken on 
banning the destruction of those excess embryos; and if there 
is, I suppose that would be consistent with the view that they 
cannot be used. If it is not, it raises the quandary--if you 
are not opposed to their destruction, how could you be opposed 
to using them, so that if in fact these other lines that 
hopefully are going to do exactly what you have described do 
not pan out, we have another source there to deal with.
    But I think you have done a tremendous job, and you are 
right--we now have a position taken by this administration to 
publicly finance research, having drawn lines--and I have 
difficulty with the arbitrariness of August 9 and 9 p.m.--but 
nonetheless, this is breakthrough, this is good, and we ought 
not allow this hearing to end on a note that somehow, while 
there are still some disagreements over whether or not after 
August 9, there are some additional embryos to be used--this is 
very positive.
    So I did not want the hearing to end without expressing my 
gratitude to you. I suspect you had an awful lot to do with 
this, the fact that there was that speech on August 9, and I 
suspect you had a lot to do with the fact that there was some 
breakthrough here, moving the line a bit further than I suspect 
some inside the White House wanted it to be moved. You do not 
have to answer that; I just have my own suspicions about it, 
and I am grateful to you, and a lot of other people are.
    And I know that you have a first-rate team. I cannot tell 
you how excited I am about the work that is being done in adult 
stem cell research. I have had some wonderful conversations 
with my friend from Tennessee who has been enlightening me on 
cord blood stem cells and what that can mean. And at NIH, I 
have had wonderful conversations with the acting director out 
there about how realistic cord blood stem cells might be. 
Nonetheless the work that is being done is very exciting.
    So I thank you for being here today and thank you for the 
work that you have done.
    Obviously, some of the questions that have been raised, 
Senator Clinton's questions and Senator Kennedy's, we need to 
follow up on pretty quickly, because if it turns out that you 
are wrong, and we cannot extract these things, we will have to 
revisit this issue. I know you do not want to admit that this 
morning, but the only thing you said that worried me is that we 
will never reconsider the August 9th date, and I am not going 
to ask you to repeat that because I like to believe you might 
want to rethink it. So if we have to come back to that, we will 
have to come back to it, and we will talk to you when that 
occurs.
    Thank you.
    Secretary Thompson. Senator Dodd, thank you, and I thank 
all the members of the committee.
    I will tell you this has been a great discussion. The 
beauty of it is that this holds so much promise for everybody 
out there who has a loved one suffering from breast cancer or 
dementia or anything else and is waiting for the possibility. 
We just cannot get the information out there that the cure is 
just around the corner; we have to get this basic research 
done.
    Senator Dodd. I agree.
    Secretary Thompson. The President has allowed that to 
continue. I am excited about it, and I know that you are, 
Senator Dodd, and I thank you very much for all of your 
comments and all of your questions today.
    The Chairman. Senator Frist?
    Senator Frist. Mr. Chairman, if I could have just 2 
minutes. I again want to thank Secretary Thompson and the 
President for a lot of thought, a lot of consideration, a lot 
of individual meetings--and this issue deserves it because it 
is, again, as we look at science one of the few issues that we 
have had to face, that humanity has had to face, that has the 
opportunity for both promise, altering the basic building 
blocks of life, but also that could have unintended 
consequences. I want to thank you for that.
    I do believe that we should expeditiously implement the 
policy put forth by the President, which is carefully crafted. 
As this moves forward, it will be important, again because 
science changes so rapidly, that we continuously reevaluate 
both the progress and the needs of this research.
    If there is one thing that has come out from your comments 
and the comments in the last several weeks, it is that this is 
an uncharted area of scientific inquiry. This is clear whether 
we are talking about the number of necessary cell lines or 
about the mouse cells and xenotransplantation issue, which we 
have addressed in one context of transplanting whole organs but 
not quite as much in cells. It demands an ongoing public 
discussion among the policymakers, like those here today, the 
scientific community, whom we will hear from shortly, 
ethicists, the religious leaders, and the American people. It 
is absolutely critical to have that discussion on an ongoing 
basis--not just today, but on an ongoing basis--as science does 
change and progress.
    We are going to have to wait several years before we know 
whether embryonic stem cell research is going to yield the 
promise that we all hope it will. In the meantime, I believe we 
should move forward expeditiously in implementing the 
President's policy and continue to examine the progress closely 
over the coming months and years.
    Secretary Thompson. Thank you.
    The Chairman. Thank you very much.
    Senator Clinton?
    Senator Clinton. Secretary Thompson, I want to thank you as 
well. I would not want you to leave thinking that because I and 
others have raised some very tough issues, we do not totally 
respect what you are doing and have done.
    I also want to thank our colleague, Senator Frist, because 
he also has had some things to say and do that have led us to 
this point, and we are very grateful for that.
    But we need this kind of open, honest dialogue. This is a 
decision which, because of our living in a global media age, we 
are bringing in millions of people to be part of. It is not 
going to be made by scientists in a closed lab or by Senators 
behind closed doors. This is a society decision that has to be 
addressed in that way, and I know, given your background and 
what you have done with this particular issue and in general, 
that you understand that and will be working to lead public 
opinion as well.
    Thank you very much.
    Secretary Thompson. Thank you.
    The Chairman. Thank you, Mr. Secretary.
    Secretary Thompson. Thank you.
    The Chairman. The hour is late, but we will try to get 
started with our panel, if we could. We will invite Dr. Douglas 
Melton, who is chairman of the Department of Molecular and 
Cellular Biology at Harvard University and a leading stem cell 
researcher and has made numerous important discoveries in 
diabetes research. Dr. Melton was honored for his outstanding 
accomplishments in medical research by being named to the 
National Academy of Sciences in 1995.
    Ms. Karen Hersey is senior counsel for intellectual 
property at the Massachusetts Institute of Technology. She can 
speak from extensive professional experience on the 
complications that can arise in seeking access to essential 
medical research materials.
    Dr. James Childress is the Edwin Kyle Professor of 
Religious Ethics at the University of Virginia. Dr. Childress 
served with distinction on the National Bioethics Advisory 
Commission whose report on stem cells is a thoughtful guide to 
the complex ethical issues raised by this research.
    It is a special pleasure to welcome Dr. Childress, who was 
formerly the Joseph P. Kennedy Senior Professor of Christian 
Ethics at Georgetown University and has been a great leader in 
all areas of bioethics and is someone whom I have respected 
over a long period of time.
    Dr. Kevin FitzGerald is the Doctor Lauler Professor for 
Catholic Health Care Ethics at Georgetown University and an 
associate professor of oncology. He has written extensively 
about the moral-ethical issues raised by new advances in 
medicine.
    And Dr. John Chute conducts research on adult stem cells at 
the Bethesda Naval Medical Center. I believe that research on 
adult stem cells should proceed in parallel with a vigorous 
research program on embryonic stem cells. We welcome his 
testimony on this important topic.
    It is a pleasure to have all of you, and we look forward to 
moving ahead.
    I will ask Dr. Melton if he would be good enough to start.

STATEMENTS OF DOUGLAS MELTON, THOMAS DUDLEY CABOT PROFESSOR IN 
 THE NATURAL SCIENCES, AND CHAIR, DEPARTMENT OF MOLECULAR AND 
  CELLULAR BIOLOGY, HARVARD UNIVERSITY; KAREN HERSEY, SENIOR 
 COUNSEL FOR INTELLECTUAL PROPERTY, MASSACHUSETTS INSTITUTE OF 
  TECHNOLOGY; JAMES F. CHILDRESS, EDWIN B. KYLE PROFESSOR OF 
    RELIGIOUS ETHICS, UNIVERSITY OF VIRGINIA; FATHER KEVIN 
 FITZGERALD, DOCTOR LAULER PROFESSOR FOR CATHOLIC HEALTH CARE 
    ETHICS AND ASSOCIATE PROFESSOR, DEPARTMENT OF ONCOLOGY, 
      GEORGETOWN UNIVERSITY; AND DR. JOHN P. CHUTE, HEAD, 
     HEMATOPOIETIC STEM CELL STUDIES SECTION, NIDDK, NAVY 
TRANSPLANTATION AND AUTOIMMUNITY BRANCH, NAVAL MEDICAL RESEARCH 
                             CENTER

    Mr. Melton. Good afternoon, Chairman Kennedy and Senator 
Frist. Thank you for inviting me to speak here today about 
human embryonic stem cells.
    In the last 3 years, the potential of these cells has been 
widely debated in the public, and rightly so. The subject 
forces us all to revisit the question of when life begins, and 
we have to scrutinize the crossroads between scientific 
inquiry, our efforts to improve the human condition, and our 
moral and ethical responsibilities to preserve human dignity.
    I am not here to testify on the moral, religious or 
political aspects of this research. I appear before you as a 
scientist and as the father of a young boy with Type 1 or 
juvenile diabetes. I will furthermore not speak to you about 
the human burden of a diabetes and will simply say that I work 
on human embryonic stem cells to try to treat or cure diabetes. 
My remarks today will therefore be confined to the scientific 
potential of these cells and the implementation of the 
President's plan about which we have already heard.
    In my written testimony which I would like submitted for 
the record, I summarize the properties of embryonic stem cells 
and put that research in a larger context of recent biological 
activities and studies at the NIH.
    Let me just say now that the ability of these cells to make 
any part of the body is what holds their promise for therapies. 
We have already heard about the numerous diseases that can be 
potentially treated--Parkinson's, Alzheimer's, osteoporosis, 
and the disease that has my full attention, juvenile diabetes. 
And I would simply like to say at this point that while adult 
stem cells have some similar properties, based on what we know 
today, adult stem cells do not have all the properties of 
embryonic stem cells.
    To give an example, there is no credible evidence for the 
isolation or growth of an adult pancreatic stem cell, and that 
alone justifies, in my view, the work on embryonic stem cells.
    I do not need to remind you all that the President made an 
important speech on August 9, and I would simply like to 
comment on that date as I feel it has important implications 
for the implementation of his plan.
    That date was not chosen for scientific reasons, and its 
arbitrary selection will unquestionably have an effect on the 
progress of research. For example, as we have already heard, it 
will not be possible for federally-funded researchers to 
explore new ways to derive human embryonic stem cells, stem 
cells that have a broader genetic diversity, or perhaps were 
grown in the absence of mouse tissue, for example, grown with 
human as opposed to mouse tissue.
    Nevertheless, as Secretary Thompson has rightly pointed 
out, the door has been opened, and some research can now be 
done. I would like to address two issues and make a proposal 
for the implementation of the President's plan.
    One of the things I feel the committee has struggled with 
is the lack of information about these 60-plus cell lines, and 
legitimate questions were asked about them. Let me simply say 
that scientists are by their nature inquisitive and skeptical, 
and we hold dear the practice of publishing results following 
an independent review by qualified experts.
    Moreover, by publishing results, scientists generally agree 
that the reagents reported therein, including cells, are to be 
made available and shared with the research community. In this 
way, the results can be independently verified, and new 
directions and discoveries can be explored.
    The problem with the present case is that only a handful of 
these 60-plus embryonic cell lines have been published, so it 
is not yet possible to give evaluation or comment on the 
quality of the lines. Nonetheless, legitimate questions can be 
asked about their growth, differentiation potential, age, and 
purity. These issues have already been raised this morning.
    What I can say is that decades of experience with mouse 
embryonic stem cells show that they lose their differentiation 
potential, become contaminated, accumulate mutations, and tend 
toward spontaneous differentiation or uncontrolled 
differentiation after a certain period. This is related to the 
question that Senator Mikulski raised about shelf life.
    Stated otherwise, there is incontrovertible evidence that 
old mouse embryonic stem cells do not have the same potential 
that young ones do. If I were to give an analogy with a human, 
it is true that these lines can grow forever and are in that 
sense immortal, but they lose their potential--a 150-year-old 
may still be alive but does not have the same potential as a 
20-year-old.
    I hasten to add that I am not criticizing the NIH nor the 
scientists who have reported the isolation of these 60 stem 
lines. Indeed, the scientists have not published their work, 
and they may well wish to further characterize the cells before 
doing so. It is therefore, in my opinion, too early to tell how 
many of the 60 lines are truly useful. Preliminary indications 
nevertheless suggest that the final number will be 
significantly less than 60. If the available lines have been 
extensively grown and have a high passage number, that will 
further reduce their value.
    Let me now turn to the question of availability. A separate 
issue concerns whether these lines will be made available, and 
we have already heard that the entities that have derived the 
lines have proprietary and commercial interests. Experience 
shows that the negotiation of transfer from those who own the 
reagent to federally-funded scientists can be slow, expensive, 
and sometimes accompanied by onerous restrictions. It is 
obvious that the legitimate interests of companies may not 
coincide with scientists' research plans and our Nation's 
public health policy.
    I was delighted to hear that Secretary Thompson has made 
progress with WARF in establishing one such relationship, but 
it is yet unclear whether he will be successful in doing so 
with the other entities.
    To get to my final point, I would like to make a suggestion 
which I have made before to Secretary Thompson and the NIH, 
which is a plan to move forward that I think will be most 
effective given the limitations presented to the scientific 
community.
    Specifically, I suggest that the NIH create a repository, 
not a registry, for the 60 embryonic cell lines. The NIH could 
collect the lines, determine their quality, and certify them 
for distribution to qualified researchers. Equally important, 
this plan would have the NIH negotiate favorable terms with all 
of the suppliers that could be set out in the Material Transfer 
Agreement.
    At the moment, it is very difficult for scientists to 
individually negotiate such arrangements, and the Federal 
Government and the NIH are in an immeasurably stronger position 
than are individual investigators to obtain the human embryonic 
stem cell lines from suppliers. In that way, as I have said, 
they could verify their quality and arrange for their 
distribution.
    I would like to know whether the NIH would be willing to 
consider doing that given that their resources far exceed those 
of individual investigators.
    In conclusion, Mr. Chairman, I think the President and 
Secretary Thompson have proposed a plan that will allow 
federally-supported researchers to begin to explore work on 
human embryonic stem cells and work toward a cure. It is an 
important step. If my remarks today seem cautious, the reason 
is the uncertainty I have about the quality, availability, and 
longevity of the cell lines. Assuming that some of the 60 lines 
are made available, federally-supported scientists can work to 
understand how these cells can be directed to differentiate. 
This will lead to new insights into human biology and disease.
    However, it seems to me perfectly clear that as these 
studies progress to the point where clinical applications can 
begin, I expect the plan will have to be revisited, principally 
because the viability or utility of the 60 cell lines will have 
been exhausted by that point.
    In closing, I thank you and the committee once again for 
the privilege of speaking to you about this important area of 
biology.
    The Chairman. Thank you very much.
    [The prepared statement of Mr. Melton follows:]
                  Prepared Statement of Douglas Melton
    Good afternoon Chairman Kennedy, Senator Gregg and other 
distinguished members of the committee. It is my pleasure to appear 
before you today to speak about human embryonic stem cells.
    Mr. Chairman, before I begin my remarks on stem cells, I want to 
take this opportunity to thank you and the other members of this 
subcommittee for your leading role in supporting the NIH. I thank you 
on behalf of the Nation's scientists who work to understand the basic 
principles of life and to cure human disease.
    In the last 3 years, and increasingly so in the past few months, 
the potential of human embryonic stem cells has been widely debated in 
the public and rightly so. This subject forces us all to revisit the 
question of when life begins. We have to scrutinize the crossroads 
between scientific inquiry, our efforts to improve the human condition, 
and our moral and ethical responsibilities to preserve human dignity. 
Not surprisingly, a subject that combines the science of life's 
beginnings with politics and religion has captured the Nation's 
attention. Indeed, this topic was recently the subject of President 
Bush's excellent speech on 9 August.
    I am not here to testify on the moral, religious or political 
aspects of human embryonic stem cell research. I appear before you as a 
scientist and as the father of a young boy with Type I or juvenile 
diabetes. I will furthermore not speak to you about the human burden of 
this disease, but simply say that I work on human embryonic stem cells 
with the aim of providing a cure for diabetes. My remarks today will be 
confined to the scientific potential of human embryonic stem cells and 
the implementation of the President's plan.
    While I'm certain the committee is well aware of the potential uses 
for human embryonic stem cells, allow me to briefly put this research 
in context. In the last century, biologists showed that genes are the 
units of development and heredity, discovered that genes are made of 
DNA, and recently completed sequencing the DNA that comprises the human 
genome, that is, sequencing the DNA of all human genes. This monumental 
achievement will stand as one of the most important scientific triumphs 
from the last century. Knowing the sequence of DNA allows scientists to 
uncover the basis for development, heredity and disease and challenges 
us to understand how the code of life is read or interpreted. At the 
same time, this enduring achievement should not cause us to forget that 
the unit of life is not DNA nor the gene, but rather the unit of life 
is the cell. Cells are alive and reproduce and among cells, stem cells 
are unique. Embryonic stem cells are special because they can reproduce 
to make more of themselves and they have the remarkable capacity to 
make any kind of cell in the body. One might think of them as the fire 
hydrant of all cells, having the capacity to renew or replenish lost 
cells and tissues. Understanding how these cells can duplicate 
themselves and how they specialize to make all types of cells will 
undoubtedly reveal important insights into human biology and disease.
    The ability of stem cells to specialize or differentiate into any 
kind of cell is what holds their enormous therapeutic promise. Many of 
the diseases that currently plague our society are diseases of cellular 
deficiency, diseases in which one particular cell type is missing or 
defective. These diseases include Parkinson's, Alzheimer's, 
osteoporosis, some cancers and the one that has my full attention, 
juvenile diabetes. It has been estimated that as many as one hundred 
million Americans are affected by these diseases. Stem cells have the 
potential to replace the missing or deficient cells and it follows that 
the Nation's scientists and those suffering from diseases are anxious 
to aggressively pursue this research. I should like to note that while 
embryonic stem cells have a much broader potential for growth and 
differentiation than do adult stem cells, research on both adult and 
embryonic stem cells is warranted; it's too early to know which type of 
stem cell will be most useful. Whereas the last century of biology can 
be said to have focused on the gene and the sequence of DNA, I believe 
this century will see biologists come to understand and harness the 
unit of life, the cell, specifically stem cells.
          president bush's plan for sixty embryonic cell lines
    President Bush has made clear his commitment to support research on 
human embryonic stem cells, highlighting the importance of this 
research. The President's plan provides the opportunity to advance 
embryonic stem cell research in the US, at least for a few years, and 
as such his plan marks an important commitment. The Honorable Tommy 
Thompson has worked diligently for this research and his continued 
leadership will be critical in moving forward with the President's 
plan.
    For this field the date of the President's speech, 9 August 2001, 
is important because only stem cell lines in existence at that time, 
estimated to be about sixty, are eligible for Federal support. This 
date was not chosen for scientific reasons and its arbitrary selection 
will have an effect on the progress of research. For example, it will 
not be possible for federally funded researchers to explore new ways to 
derive human embryonic stem cells nor work with cells that have been 
isolated without possible contamination from mouse or other supporting 
cells. Nevertheless, it is now possible for the Nation's researchers to 
initiate studies on how embryonic stem (ES) cells differentiate and we 
can begin to explore their therapeutic potential.
    Looking ahead to how the plan will work, I turn to two issues: the 
quality of the sixty cell lines and their access or availability.
             quality of the human embryonic stem cell lines
    Scientists are, by their nature, inquisitive and skeptical and we 
hold dear the practice of publishing results following an independent 
review by qualified experts. Moreover, by publishing results, 
scientists generally agree that the reagents reported, including cells, 
are available to be shared with the research community. In this way 
results can be independently verified and new directions and 
discoveries can be explored. In the present case, only a handful of the 
sixty+ embryonic cell lines have been published so it is not yet 
possible to evaluate or comment on the quality of cells. Nonetheless, 
legitimate scientific questions about the growth, differentiation 
potential, age, and purity of the lines must be considered. Decades of 
experience with mouse embryonic stem cells have shown that ES cells can 
lose their differentiation potential, become contaminated, accumulate 
mutations, and tend toward spontaneous or uncontrolled differentiation. 
The fact that mouse ES cells lose their full potential with increasing 
age or passage number is only one reason to believe that the sixty+ 
cell lines will not be sufficient for the years of research required to 
investigate therapies with these cells. Looking ahead to clinical 
applications, including transplantation and the problem of 
immunological rejection, there will certainly be a need for broader 
genetic diversity of cell lines. There may also be a need for cell 
lines that have been isolated without the use of mouse feeder layers.
    I hasten to add that I am not criticizing the NIH nor the 
scientists who have reported the isolation of the sixty human embryonic 
stem cell lines. Indeed, the scientists have not published their work 
and they may well wish to further characterize the cells before doing 
so. It is therefore too early to tell how many of the sixty+ lines are 
truly useful embryonic stem cell lines. Preliminary indications from 
press reports do suggest that the final number will be significantly 
less than sixty. If the available lines have been grown extensively and 
have a high passage number that will further reduce their value.
                              availability
    A separate issue concerns whether the cell lines will be made 
available to federally funded researchers in a timely manner and 
without restrictions on their use for research. It is noteworthy that 
most of the entities that have isolated the sixty+ human embryonic stem 
cell lines are companies with proprietary and commercial interests. In 
addition, there are relevant patents on some of the cells that may 
further restrict their distribution and use. Experience shows that the 
negotiation of transfer from those who own a reagent to federally 
funded scientists can be slow, expensive, and sometimes accompanied by 
onerous restrictions on use. It is obvious that the legitimate 
interests of companies may not coincide with scientist's research plans 
or our Nation's public health policy.
    I believe this problem of access is likely to be quite serious. The 
NIH plan to create a registry of cells will leave it to individual 
investigators to negotiate for transfer of the cells. This places a 
heavy burden on researchers and one can anticipate, at a minimum, 
significant delays. In some cases the terms of the transfer may be too 
restrictive to allow scientists access to the material. Finally, I note 
that some of the potential suppliers have already indicated that they 
lack the resources and incentive to prepare their cells for 
distribution.
            create a federal repository for human stem cells
    I would like to suggest a plan that addresses both of these issues. 
Specifically, I suggest that the NIH create a repository, not a 
registry, for the sixty+ embryonic cell lines. The NIH could collect 
the cell lines, determine their quality, and certify them for 
distribution to qualified researchers. Equally important, this plan 
would have the NIH negotiate favorable terms with the suppliers, set 
out in a Material Transfer Agreement, so that scientists could use the 
cells for research purposes. The Federal government and the NIH are in 
an immeasurably stronger position than are individual investigators to 
obtain the human embryonic stem cell lines from suppliers, verify their 
quality, and arrange for their distribution.
                                summary
    In conclusion, Mr. Chairman, I think that President Bush and 
Secretary Thompson have proposed a plan that will allow federally 
supported scientists to begin to explore the potential of human 
embryonic stem cells and work toward a cure for numerous diseases. This 
is a very important step forward. If my remarks today seem cautious, 
the reason is the uncertainty about the quality, availability and 
longevity of the cells. Assuming that some of the sixty cell lines are 
made available, federally supported scientists can work to understand 
how these cells can be directed to differentiate. As the studies 
progress to the point where clinical applications can begin, I expect 
the plan will have to be revisited because the viability or utility of 
the sixty+ cell lines will have been exhausted.
    In closing, I thank you and the Committee once again for the 
privilege of speaking to you about this important area of biology.

    The Chairman. Ms. Hersey?
    Ms. Hersey. Good afternoon, Mr. Chairman and distinguished 
members of the committee.
    My name is Karen Hersey. I am senior counsel for 
intellectual property at the Massachusetts Institute of 
Technology. It is my understanding that I have been invited 
here today for the purpose of providing you with an 
introduction and an overview of the subject of Material 
Transfer Agreements, known as MTAs, about which you have 
already heard this morning, and the role that they 
traditionally play in the exchange of materials for scientific 
research.
    As we already know from our colleagues at the University of 
Wisconsin, there is every expectation in the academic community 
that access to stem cells for research will involve the 
execution of a Material Transfer Agreement between the stem 
cell provider and the organization requesting them.
    MIT does not have a medical school, and in terms of the 
numbers of materials that we request access to, it is small 
compared with those institutions that have larger faculties, 
larger student bodies, and medical schools. However, MIT's 
faculty, students, and staff are engaged in a substantial 
volume of research in biology, biotechnology, bioengineering, 
and so forth, where biological materials are used every day to 
investigate and teach areas of advanced science.
    Like every research unit, whether academic or industrial, 
MIT investigators depend upon the availability of and the 
access to new materials developed by others to move their 
research forward. The materials are often proprietary to their 
owners and not commercially available, or they may be 
commercially available at a very steep price that the 
university generally does not have to pay only if it accepts 
materials under restrictive conditions. And in some cases, the 
materials will be made available to the academic and industrial 
communities under a common set of terms that we in academic 
find ourselves trying to work with in a contractual framework 
that is not suited to our environment.
    Twenty years ago when I first joined MIT as a licensing 
attorney, the term ``Material Transfer Agreement'' was 
virtually unknown, or at least unknown to those of us in the 
administration. We can reconstruct from the lament we often 
hear recalling the ``good old days'' that materials were 
transferred from scientist to scientist and from organization 
to organization through a phone call or a verbal handshake. 
From time to time, one-page documents or releases arrived, 
generally containing warnings that the materials might be 
toxic, that they were not to be used for human subject 
research, and requiring the receiving party to release the 
sending party from all responsibility for use of the materials.
    These one-page notifications were almost always signed by 
the investigator receiving the materials. A check of our 
database shows that MIT institutionally signed a total of eight 
MTAs in 1989.
    That was yesterday. Today the story is far different. My 
office at MIT has overall responsibility for incoming material 
transfer activity at MIT. We have one attorney and one 
paralegal negotiating more than 75 Material Transfer Agreements 
a year. We have a database where all MTAs, their status as 
active or inactive, the lab where the materials are placed, and 
the responsible investigator are logged, and where the 
restrictions on use of the materials are also logged. There are 
over 600 entries covering materials in active use, and we are a 
relatively small university. The larger State universities and 
medical schools are looking at exponential increases in these 
numbers.
    So what has happened to change the simple handshake between 
scientists into a fullblown negotiation between organizations, 
often taking months? No doubt there will be any number of 
theories out there that might be supported, but as I consider 
events over the last decade and a half, it leads me to 
attribute the change to three factors.
    The first is the explosion of the biotechnology industry 
and the recognition by both industry and the academic community 
during the 1980's and 1990's that certain combinations of the 
materials, certain methods of producing them or methods of 
using them, could in fact be patented.
    From then on, the materials, especially those that were 
unique, took on an added value, and the transfer of them under 
terms that would protect intellectual property rights of the 
owner became singularly important.
    The second was the companion recognition in the late 1980's 
that the sharing of materials might just translate into an 
advantageous business opportunity for the owner. If materials 
could be cast among academic scientists, industry bench 
scientists, and Government researchers, might there not be 
interesting knowhow, improvements, and new discovers that could 
be reeled back in if the materials owner implanted hooks into 
its agreements?
    Third, it is clear from the negotiations we conduct now 
that fear also played a role in the demise of the traditional 
transfer by handshake. That is, fear of deep-pockets liability 
if materials were misused and also fear that a potential 
business opportunity for the materials owner might be thrown 
away if materials were sent out with no hooks applied.
    That background should provide a clearer understanding of 
why, in today's typical Material Transfer Agreement received by 
an academic institution, it is now common that some combination 
of limitations, restrictions, give-backs or reach-throughs will 
be found. Before the materials can be introduced into 
scientific research programs, the terms under which the owner 
is willing to make them available will need to be reconciled 
with the proposed scientific research program, the sources of 
funding for it, and the institutional policies having to do 
with freedom to publish, the importance of sharing research 
findings with colleagues, and technology transfer.
    If I may, I would like to just take a very short time to 
take you through some of the very common but problematic terms 
that we are apt to see in the agreements that must be signed 
before the transfer of materials can be accomplished.
    I would like to say that except as between nonprofits, it 
is totally unusual for us to see a one-page Material Transfer 
Agreement unless the type size is minute. Most often these 
agreements will run well beyond the two-page quick transaction 
limit. They will commonly define or identify the materials they 
cover and routinely expand that definition of materials to 
subsume any progeny that might be split off or replicated. 
While that is clearly understandable, unfortunately, all too 
often the definition of ``the materials'' is expanded by the 
owner to encompass ``modifications and derivatives'' of them. 
This is where the problems for the receiving scientists are 
likely to begin, for if the owner owns the materials under this 
expanded definition, the owner also ends up with ownership of 
the modifications and the derivatives made by the receiving 
scientist. The ownership problem is exacerbated if the 
materials provider also wants ownership rights to all 
improvements, inventions or discoveries developed as a result 
of using the materials.
    If there is not an ownership issue, there is most likely a 
licensing issue. Materials owners may require an exclusive 
license, royalty-free, to ``all inventions and research 
results'' made through use of the materials; or they may 
require open-ended options or first refusal rights to license 
inventions relating to use of the materials.
    The Chairman. We can give you another minute or two to wrap 
up.
    Ms. Hersey. Thank you, Senator.
    At a minimum, if the materials provider is a commercial 
company, a university may expect to grant that company a 
royalty-free, nonexclusive license.
    I would just like to end by giving you a couple of 
observations that might help as you look at or contemplate the 
kinds of agreements that may come in with the materials that 
are going to be used by NIH research.
    We do have at MIT a slightly more difficult time with 
material transfer agreements coming in from foreign 
organizations. There is a problem with control over use of the 
materials, intellectual property rights, and in fact, some of 
the materials are not owned by the foreign institutions at all 
but by the faculty, the scientists who develop them.
    Finally, how do material transfer agreements really affect 
the scientific work of a university? They tend to be 
comprehensive legal agreements presenting unique and complex 
issues for a university. That means they are time-consuming to 
negotiate, as you have already heard, and hold up research 
efforts. They often contain ownership, licensing and reach-
through terms and conditions that are inconsistent with Federal 
requirements attached to federally-funded research. They may 
prohibit or restrict publication of research in a way that is 
unacceptable for an academic institution. They may discourage 
innovation because in fact the materials provider will control 
commercialization rights.
    Thank you very much.
    The Chairman. Thank you very much, Ms. Hersey.
    [The prepared statement of Ms. Hersey follows:]
                   Prepared Statement of Karen Hersey
    Mr. Chairman and distinguished members of the committee. My name is 
Karen Hersey. I am Senior Counsel for Intellectual Property at the 
Massachusetts Institute of Technology. I am pleased to have the 
opportunity today to provide you with an introduction to the subject of 
material transfer agreements and the role they traditionally play in 
the exchange of materials for scientific research. As we already know 
from our colleagues at the University of Wisconsin, there is every 
expectation in the academic community that access to stem cells for 
research will involve the execution of a material transfer agreement 
between the stem cell provider and the organization requesting them.
    MIT is a small institution compared with most other U.S. research 
universities and we do not have a medical school. However, MIT's 
faculty, students and staff are engaged in a substantial volume of 
research in biology, biotechnology, bioengineering, biochemistry and 
related disciplines where biological materials, including cell lines, 
plasmids, vectors, sequences, monoclonal antibodies and others are used 
every day to investigate and teach areas of advanced science. Like 
every research unit, whether academic or industrial, MIT investigators 
often depend upon the availability of, and access to, new materials 
developed by others to move their research forward. The materials are 
often proprietary to their owners and not commercially available; or 
they may be commercially available at a steep price that the university 
generally does not have to pay if it accepts the materials under 
restrictive conditions; and, in some cases, the materials will made 
available to the academic and industrial communities under a common set 
of terms and we, in academia, find ourselves trying to work within a 
contractual framework not suited to the academic environment.
                               background
    Twenty years ago, when I first joined MIT as a licensing attorney, 
the term ``material transfer agreement'' was virtually unknown, at 
least unknown to the administration. We can reconstruct, from the 
lament we often hear recalling the ``good old days'' that materials 
were transferred from scientist to scientist and from organization to 
organization through a phone call and a verbal handshake. From time to 
time, one page documents or releases arrived that generally contained 
warnings that the materials might be toxic, were not to be used on 
human subjects, and required the receiving party to release the 
materials owner of all responsibility for any use of the materials. 
These one-page notifications were almost always signed by the 
investigator receiving the materials. A check of our database shows 
that N41T institutionally signed a total of 8 MTAs in 1989.
    That was yesterday. Today, the story is far different. My office at 
MIT has overall responsibility for the in-coming material transfer 
activity for MIT. We have one attorney and one paralegal negotiating 
more than 75 material transfer agreements each year. We have a database 
where all MTAs, their status as active or inactive, the lab where the 
materials are placed, and the responsible investigator are logged. 
There are over 600 entries covering materials in active use--and we are 
a very small university. The larger State universities and medical 
schools are looking at exponential increases in these numbers.
    What has happened to change a simple handshake between scientists 
into a full-blown negotiation between organizations, often taking 
months? No doubt there will be any number of theories that might be 
supported, but as I consider events over the last decade and half, my 
experience leads me to attribute the change to three main factors.
    The first is the explosion of the biotechnology industry and the 
recognition by both industry and the academic community during the 
1980s and early 1990s that certain combinations of these materials, 
certain methods of producing them or methods of using them, in fact, 
could be patented. From then on, the materials, especially those that 
were unique, took on an added value and the transfer of them under 
terms that would protect intellectual property rights of the owner 
became singularly important. The second was the companion recognition 
by the late 1980s that the sharing of materials might just translate 
into an advantageous business opportunity. If materials could be cast 
among academic scientists, industry bench scientists, and government 
researchers might there not be interesting know how, improvements, and 
new discoveries that could be reeled back in if the materials owner 
implanted hooks into its agreements. Third, it is clear from the 
negotiations we conduct now that fear also played a role in the demise 
of the traditional ``transfer by handshake''. That is, fear of ``deep 
pockets'' liability if materials were misused and also fear that a 
potential business opportunity for the materials owner might be thrown 
away if materials were sent out with no hooks applied.
    This background should provide a clearer understanding of why, in 
today's typical material transfer agreement received by an academic 
institution, it is now common that some combination of limitations, 
restrictions, give-backs or reach-throughs will be found. Before the 
materials can be introduced into scientific research programs, the 
terms under which the owner is willing to make them available will need 
to be reconciled with the proposed scientific research program, the 
source of funding for it, and institutional policies having to do with 
freedom to publish, the importance of sharing of research findings with 
colleagues, and technology transfer.
                        common mta terms, today
    First, let me say that, except as between nonprofits as I will 
explain later, it is very unusual for us to see a one-page material 
transfer agreement today, unless the type size is minute. Most often 
these agreements run well beyond the two-page quick transaction limit. 
They will commonly define or identify the materials they cover, and 
routinely expand the definition of the materials to subsume any 
``progeny'' that might be replicated. Unfortunately, all too often the 
definition of what are ``the materials'' is expanded by the owner to 
encompass ``modifications and derivatives'' of them. This is where the 
problems for the receiving scientist are likely to begin; for, if the 
owner owns the materials, the owner also ends up with ownership of the 
modifications and derivatives made by the receiving scientist. The 
ownership problem is exacerbated if the materials provider also wants 
ownership rights to all improvements, inventions or discoveries 
developed as a result of using the materials.
    If there is not an ownership issue, there most likely is a 
licensing issue. Materials owners may require an exclusive license, 
royalty-free, to ``all inventions and research results'' made through 
use of the materials. Or they may require open-ended options or first 
refusal rights to license inventions relating to use of the materials. 
At a minimum, if the materials provider is a commercial company, a 
university may expect to grant the company a royalty-free, nonexclusive 
license to inventions made as a result of using the materials. As a 
result, the promise of an MTA-encumbered invention as a viable business 
opportunity for the inventor and his or her institution is 
significantly diminished.
    Where ownership and licensing issues can be successfully maneuvered 
around, other limitations, restrictions or encumbrances often result in 
additional obstacles. For instance, the materials provider may require 
the researcher to hold all results learned from using the materials in 
confidence, may require a right of review and approval for any 
publication dealing with the materials, may require a right of approval 
over any transfer to a third party of the materials, their progeny, 
derivatives, improvements and so forth, may tag on ``reach-through'' 
rights to second and third generation uses of the materials or 
derivatives of them in terms of royalty-sharing or first commercial 
rights to exploit. It should not be a surprise that the list of 
encumbrances generally grows with the perceived value of the materials 
as a business opportunity.
                          general observations
    I will finish up this short discussion of the role material 
transfer agreements play in current-day research efforts with a very 
few general comments.
     The transfer of materials from university to university or 
nonprofit to nonprofit is most often accomplished under a one-page 
document called a Uniform Biological Material Transfer Agreement, or 
UBMTA. This is a simplified method of transfer agreed upon among the 
U.S. research universities during the early 1990s with the help of NIH. 
The providing university does not attach strings to the use of the 
materials, but, in some cases does have an interest in where they may 
be transferred by the receiving university. An attempt was made at the 
same time to convince industry to join in a simplified transfer 
mechanism, but it was not successful.
     At MIT we have a slightly more difficult time with 
material transfer agreements from foreign organizations or companies. 
They seem to be concerned with maintaining control of their properties 
either by claiming ownership of derivatives and modifications or by 
prohibiting the receiving scientist from obtaining any independent 
intellectual property rights through uses of the materials. And, 
significantly, we find restrictions placed on uses, ownership and 
further transfer because foreign universities or institutes often do 
not own materials, or do not have the right to control the distribution 
of them. Unlike the situation with U.S. universities, in Europe and 
elsewhere materials are often the property of the scientist or 
professor who developed them.
     Where materials are patented there is an extra layer of 
complications that arises. The university community will very often be 
successful in getting a noncommercial research right or license to use 
a patent covering the materials, but this is as far as it goes. Any 
future commercial use requires a license from the patent holder or no 
license rights at all may be available, hence any commercial use that 
would infringe the patent is cut off all together.
     Finally, how do the material transfer agreements really 
effect the scientific work of a university? They tend to be 
comprehensive legal agreements often presenting unique and complex 
issues for a university. That means they are time consuming to 
negotiate and hold up research efforts. They often contain ownership, 
licensing or reach-through terms and conditions that are inconsistent 
with Federal requirements attached to federally-funded research. They 
may prohibit or restrict publication of research in a way that is 
unacceptable for an academic institution. They may discourage 
innovation because the control of the research output rests with the 
materials provider or, at a minimum, establishes a preference for the 
provider to exploit new discoveries made using the materials. Last, and 
a subject we've not considered at all today, they may contain 
indemnification obligations that amount to strict liability and are 
either not within the statutory authority of a State institution to 
accept or present an unacceptable risk to the endowment of a private 
institution.
    This concludes my brief remarks on a complex subject. I am most 
appreciative of the Committee's indulgence and will be pleased to 
answer any questions you may have.

    The Chairman. Dr. Childress?
    Mr. Childress. Good afternoon, Mr. Chairman and members of 
the committee.
    I am James Childress. I teach religious ethics and 
biomedical ethics at the University of Virginia and also serve 
on the National Bioethics Advisory Commission.
    I have been asked to present my own views on the ethical 
issues in human stem cell research. In doing so, I will 
sometimes draw on NBAC's report on this topic along with its 
recommendations, which I as a commissioner helped prepare and 
also endorsed.
    I will briefly summarize several points from my longer 
written testimony and would respectfully request that that 
testimony be entered into the record.
    The Chairman. All of the testimony will be included.
    Mr. Childress. I thank you very much.
    I appreciate the thought and consideration that went into 
President Bush's policy, but other more flexible policies are 
also ethically acceptable and even preferable.
    President Bush's policy suggests that it is ethically 
acceptable to use Federal funds for research on stem cell lines 
that were derived prior to his announcement on August 9 if the 
derivation also met certain ethical requirements--specifically, 
that donors or embryos that were created solely for 
reproductive purposes must have given informed consent without 
any financial inducements.
    If this policy is ethically acceptable and satisfies basic 
ethical standards--and I believe it does--it should also be 
ethically acceptable to do the same thing prospectively--that 
is, to provide Federal funds for research on stem cell lines 
derived in the future, after August 9 as well as before, within 
the same ethical guidelines. The prospective policy would offer 
greater and needed flexibility, especially in view of the 
scientific uncertainty about the value of the approved cell 
lines. And it would be ethically preferable because it would 
increase the possibilities for important research without 
violating ethical standards.
    This prospective policy can be undertaken without 
sanctioning or encouraging further destruction of human 
embryos. Those were legitimate, major concerns in President 
Bush's statement. We can establish effective ethical safeguards 
to ensure that a couple's voluntary decision to destroy their 
embryos is voluntary and informed, or that their decision to 
donate them for research is voluntary, informed, and 
uncompensated. The research, then, would only determine how the 
destruction occurs, not whether it will occur.
    In making these points, I want to stress that no consensus 
exists among religious and secular moral traditions in our 
society about the moral status of the unimplanted human embryo. 
Public policy in our pluralistic society has to respect diverse 
fundamental beliefs, and yet it must not be held hostage to any 
single view of embryonic life.
    Whichever policies are finally adopted to enable stem cell 
research to go forward within ethical limits, we will need a 
very strong public body to review protocols for deriving stem 
cells from embryos and to monitor these research. Perhaps the 
Council on Bioethics which President Bush has announced can 
fulfill these functions. If not, some other public body will be 
needed. For example, the UK has established a statute, the 
Human Fertilization and Embryology Authority, which would use 
all embryo research as well as licensing of reproductive 
technology and fertility clinics. It reviews all embryo 
research in public and private arenas. Congress might consider 
that model for our society as well.
    In a recent editorial in Science, ethicist LeRoy Walters 
stressed that ``Government's and their advisors should be 
humble and flexible, but also decisive and courageous.'' We 
must carefully scrutinize claims of scientific promise, but we 
must not unduly constrain research that may help alleviate 
human suffering and reduce the number of premature deaths.
    Indeed, we have a collective moral duty to try to alleviate 
human suffering and reduce premature deaths just as we have a 
collective moral duty to respect important ethical limits in 
dealing with developing human life.
    We must also provide clear and stringent ethical safeguards 
in stem cell research, along with strong review and oversight. 
And I think in the final analysis, we must avoid unduly rigid 
rules that appear to be arbitrary and inconsistent.
    Thank you very much for your attention. I will be glad to 
answer any questions.
    The Chairman. Thank you, Dr. Childress.
    [The prepared statement of Mr. Childress follows:]
                Prepared Statement of James F. Childress
    Good morning, Mr. Chairman and members of the committee. I am James 
Childress. I teach in the Department of Religious Studies, the Medical 
School, and the Institute for Practical Ethics at the University of 
Virginia. I am also a member of the National Bioethics Advisory 
Commission (NBAC). I have been asked to present my own views on the 
ethical issues in the debate about public policies toward human stem 
cell research, especially embryonic stem cell research. Even though I 
am not testifying on NBAC's behalf, I will sometimes draw on NBAC's 
report and recommendations, which as a commissioner I helped to prepare 
and also endorsed. \1\
---------------------------------------------------------------------------
    \1\ See National Bioethics Advisory Commission, Ethical Issues in 
Human Stem Cell Research, Vol. 1: Report and Recommendations 
(Rockville, MD, September 1999). In November 1999, I presented NBAC's 
views at the hearing on ``Embryonic Stem Cell Research'' before the 
Subcommittee on Labor, Health and Education of the Senate 
Appropriations Committee. I request that the Executive Summary of 
NBAC's report be entered into the public record of today's hearing.
---------------------------------------------------------------------------
                     an ethically acceptable policy
    I very much appreciate the thought and consideration that went into 
President Bush's announced policy on the use of Federal funds in human 
embryonic stem cell research, but I would argue that other, more 
flexible policies are also ethically acceptable, and even preferable. 
Consider three possible options:
    (1) Provide Federal funds for research on cell lines derived (using 
non-Federal funds) from embryos prior to August 9, 2001 within certain 
ethical guidelines (President Bush's announced policy).
    Provide Federal funds for research on cell lines derived (using 
non-Federal funds) from embryos, earlier or in the future, within 
certain ethical guidelines (NIH's earlier proposed policy).
    Provide Federal funds for both the derivation of, and research on, 
cell lines derived from embryos within certain ethical guidelines 
(NBAC's recommendation).
    President Bush's announced policy (#1) suggests that it is 
ethically acceptable to use Federal funds for research on stem cell 
lines that were derived, using non-Federal funds, prior to his 
announcement on August 9, if the derivation also met certain ethical 
requirements, including the informed consent of donors of embryos 
created solely for reproductive purposes and the absence of financial 
inducements. \2\ If policy #1 is ethically acceptable--as I believe it 
is--then it should also be ethically acceptable to do the same thing 
prospectively (policy option #2). That is, it should be ethically 
acceptable to provide Federal funds for research on stem cell lines 
derived in the future, after August 9 as well as before, with non-
Federal funds and within the same ethical guidelines. This prospective 
policy would offer greater--and needed--flexibility for the short-term 
and long-term future. And it would be ethically preferable because it 
would increase the possibilities for important research, without 
violating relevant ethical standards. \3\
---------------------------------------------------------------------------
    \2\ See President George W. Bush, ``Stem Cell Science and the 
Preservation of Life,'' New York Times August 12, 2001.
    \3\ This is similar to the policy NIH initially proposed in 
December 1999 and further refined in 2000.
---------------------------------------------------------------------------
    President Bush's statement noted that the first policy (#1), which 
includes about sixty stem cell lines (about which there is some 
scientific uncertainty and controversy), \4\ ``allows us to explore the 
promise and potential of stem cell research without crossing a 
fundamental moral line by providing taxpayer funding that would 
sanction or encourage further destruction of human embryos that have at 
least the potential for life.'' However, I believe that ethically we 
can provide Federal tax funds for research on stem cells derived after 
as well as before August 9, using non-Federal funds, and that this can 
be accomplished without sanctioning or encouraging further destruction 
of human embryos. To do so, we must establish effective ethical 
safeguards. Those safeguards should ensure to the greatest extent 
possible the couple's voluntary and informed decision to destroy their 
embryos--rather than use them or donate them to another couple--and 
their voluntary and informed decision to donate them for research. The 
research would then determine how the destruction occurs, not whether 
it will occur; as matters stand in most jurisdictions, couples may 
determine how to dispose of their embryos.
---------------------------------------------------------------------------
    \4\ See ``President Bush's Stem Cell Policy,'' A Statement of the 
American Association for the Advancement of Science, August 17, 2001.
---------------------------------------------------------------------------
    It is possible to go further than either of these first two 
policies and recommend, as NBAC did, a third option--the provision of 
Federal funds for both the derivation of stem cells from embryos and 
research on those cell lines, again in accord with ethical 
requirements. One argument for this option is that a strict separation 
between derivation and use would adversely affect the development of 
scientific knowledge. For instance, the methods for deriving embryonic 
stem cells may affect their properties, and scientists may increase 
their understanding of the nature of such cells in the process of 
deriving them.
    In short, I see no ethical reason for limiting Federal funding to 
research with cell lines derived by some arbitrary date, as long as 
future derivation, with non-Federal funds (option #2) or Federal funds 
(option #3), also respects the same moral limits and we establish 
effective ethical safeguards. Indeed, our collective moral duty to 
alleviate human suffering and reduce the number of premature deaths 
provides a strong ethical reason to support this research, within moral 
limits.
                         respect for the embryo
    There is widespread agreement, as NBAC observed, that ``human 
embryos deserve respect as a form of human life,'' but, at the same 
time, sharp disagreements exist ``regarding both what form such respect 
should take and what level of protection is required at different 
stages of embryonic development.'' \5\ At the very least this 
``respect'' implies
---------------------------------------------------------------------------
    \5\ See NBAC, Ethical Issues in Human Stem Cell Research.
---------------------------------------------------------------------------
     that early embryos should not be used unless they are 
necessary for research,
     that embryos remaining after in vitro fertilization (IVF), 
as well as cadaveric fetal tissue, should not be bought or sold, and
     that alternative sources of stem cells should 
simultaneously be explored.
    Indeed, given the promise of this research, and the uncertainty 
about which stem cells might be adequate and which might be superior 
for various purposes, research on stem cells derived from different 
sources should be eligible for Federal funding. The goal of realizing 
the therapeutic promise of stem cell research is ethically significant. 
It is also ethically important to treat the different sources of stem 
cells with appropriate respect.
    One interpretation of appropriate respect for early embryos would 
rule out their deliberate creation in order to use them in research. I 
supported NBAC's recommendation that, at this time, Federal agencies 
should not fund research involving the derivation or use of embryonic 
stem cells from embryos made solely for research purposes, whether they 
were made by IVF or by somatic cell nuclear transfer into oocytes. 
However, in this area, it is ethically dangerous to say ``never.'' For 
now, it appears to be possible to develop enough cell lines without 
creating more embryos, and there appears to be no need for nuclear 
transfer unless and until therapy is possible, at which time matched 
tissue may be needed. As the science develops, it may be necessary to 
revisit the question about so-called ``therapeutic cloning.'' \6\
---------------------------------------------------------------------------
    \6\ NBAC's report on human stem cell research anticipated that 
privately-funded research would use deliberately created embryos, 
whether created through IVF or cloning, and that careful monitoring of 
this private sector research would enable the Federal government to 
determine when, if ever, the time has come to fund the creation of 
embryos for research.
---------------------------------------------------------------------------
              diversity of religious and secular positions
    Views about appropriate respect for the embryo hinge on convictions 
about the embryo's moral status. As a scholar in religious ethics, I 
have been fascinated by the diverse religious views on human embryonic 
stem cell research, both across traditions and within traditions. On 
May 7, 1999 NBAC convened a meeting at Georgetown University to hear 
presentations on religious perspectives relating to human stem cell 
research. Altogether eleven scholars in Roman Catholic, Jewish, Eastern 
Orthodox, Islamic, and Protestant traditions presented formal testimony 
that day, and two others made statements in the public comment period. 
Their statements, as well as later statements of other traditions 
(e.g., the Mormon tradition), reveal significantly different 
perspectives on the ethical acceptability of research on unimplanted 
human embryos. Even when opposed to abortion, different religious 
positions may reach divergent moral conclusions about human embryonic 
stem cell research. Their different conclusions follow, in part, from 
different premises about the moral status of the early embryo existing 
outside a woman's womb.
    No consensus exists among religious traditions or secular moral 
traditions--about the moral status of the extracorporeal embryo. This 
diversity sets the context for an ethical assessment of public policy 
toward human embryonic stem cell research. An ethical public policy in 
our pluralistic society has to respect diverse fundamental beliefs. And 
yet it must not be held hostage to any single view of embryonic life.
           stem cell lines derived from aborted fetal tissue
    Another possible source of stem cells--human embryonic germ cells 
from aborted fetuses--has received scant attention recently. \7\ 
However, precedent exists in U.S. policies for providing Federal funds 
to support research on cell lines derived from aborted fetuses. This 
precedent appears in the framework developed for the use of cadaveric 
fetal tissue in transplantation research. \8\ This framework seeks to 
separate as much as possible a pregnant woman's decision to abort from 
her decision to donate fetal tissue for research. The rationale for 
this separation is to avoid any possibility, however slight, that the 
opportunity to donate fetal tissue in federally funded research could 
provide an additional motivation for a woman to have an abortion.
---------------------------------------------------------------------------
    \7\ See Alta Charo, ``Bush's Stem Cell Decision May Have Unexpected 
and Unintended--Consequences,'' The Chronicle of Higher Education, 
Tuesday, August 14, 2001.
    \8\ For the debate about human fetal tissue transplantation 
research, see the discussion in James F. Childress, Practical Reasoning 
in Bioethics (Bloomington, IN: Indiana University Press, 1997).
---------------------------------------------------------------------------
    Several ``ethical safeguards'' were erected in order to prevent the 
use of fetal tissue in federally funded transplantation research from 
encouraging abortions. For example, these safeguards separate the 
consent process for abortion from the consent process for the donation 
of fetal tissue for research, and prohibit the donor of fetal tissue 
from designating the recipient of the transplant. These ethical 
guidelines, which appear to have been effective in human fetal tissue 
transplantation research, should now be extended to stem cell research 
as well, as NBAC has recommended and NIH has proposed. \9\
---------------------------------------------------------------------------
    \9\ See NBAC, Ethical Issues in Human Stem Cell Research, Vol. 1, 
pp. 68-69, Recommendation #1.
---------------------------------------------------------------------------
         another approach to public policy--the u.k. experience
    The United Kingdom has responded quite differently than the U.S. to 
human embryonic stem cell research, including ``therapeutic cloning.'' 
Following the 1984 Warnock committee report, the British government 
implemented most of that committee's recommendations in the 1990 Human 
Fertilisation and Embryology Act, which, among other things, 
established the Human Fertilisation and Embryology Authority (HFEA). 
Over the last decade, the HFEA, currently chaired by Ruth Deech, has 
had authority over in vitro fertilization, in policy and in practice. 
The Authority also licenses and monitors all human embryo research in 
the U.K., whatever the source of funding. In addition, it approves, in 
limited circumstances, the creation of embryos for research purposes. 
Over 53,000 embryos have been used in research, while 118 have been 
created specifically for research. In January, 2001, following vigorous 
public debate, the British Parliament approved regulations to enlarge 
the range of acceptable goals for human embryo research and also to 
permit the creation of embryos for research by nuclear transfer 
(``therapeutic cloning'').
    In the U.K., then, years prior to the recent debate about stem cell 
research, several substantive and procedural standards were established 
for embryo research, including the creation of embryos for research. 
Furthermore, the public appears to have considerable confidence in that 
framework, based on a decade's experience. As a result, the acceptance 
of ``therapeutic cloning'' required only an extension of the existing 
framework, rather than the invention of a new one.
    The U.K.'s strict regulation of reproductive technologies and 
authorization, but also tight control over, embryo research appears to 
have created a context for a positive response to the possibilities of 
human stem cell research. \10\ By contrast, in the U.S., regulation of 
reproductive technologies and fertility clinics, which is under the 
control of the states, is, at best, limited and uneven, and the Federal 
government has not allowed the use of Federal funds for embryo research 
(though, of course, privately funded research proceeds). As a result, 
the task of formulating public policy toward human embryonic stem cell 
research is much more challenging in the U.S.
---------------------------------------------------------------------------
    \10\ For helpful discussions of policy in the U.K., see LeRoy 
Walters, ``Human Embryo Research: Lessons from History,'' Science 293 
(August 24, 2001): 1401, and Nicholas Wade, ``Clearer Guidelines Help 
Britain to Advance Stem Cell Work,'' New York Times, August 14, 2001.
---------------------------------------------------------------------------
                               conclusion
    In conclusion, if President Bush's announced policy is ethically 
acceptable, as I believe it is, there is, in my judgment, no cogent 
ethical reason for stopping where that policy stops with the use of 
stem cell lines that were derived from embryos by August 9, 2001. 
Indeed, that temporal restriction is difficult to defend from an 
ethical standpoint. It is possible to use non-Federal funds (or even, I 
would argue, Federal funds) to derive stem cell lines from embryos 
within certain ethical requirements, and to provide Federal funds for 
research on those lines without sanctioning or encouraging the 
destruction of embryos or the creation of so-called ``extra'' or 
``surplus'' embryos in clinical IVF. I would support these other policy 
options (that is, derivation with non-Federal funds or with Federal 
funds) on the grounds that they will probably enable important research 
to proceed more rapidly, and will not breach crucial ethical 
boundaries. In addition, it is, in my judgment, ethically justifiable 
to provide Federal funds for deriving and conducting research on stem 
cell lines developed from aborted fetal tissue, in accord with the 
guidelines and regulations already established for human fetal tissue 
transplantation research.
    Whichever policies are finally adopted to enable important and 
promising stem cell research to go forward within ethical limits, we 
will need a strong public body to review protocols for deriving stem 
cells from embryos (and from fetal tissue) and to monitor this 
research.'' \11\ Perhaps the Council on Bioethics, which President Bush 
has announced, could fulfill these functions. If not, some other public 
body will be needed, as the U.K. experience suggests. In the UK, the 
Human Fertilisation and Embryology Authority is statutorily 
established, and that might be a model for Congress to consider. 
Indeed, Congress might consider whether we now need some oversight of 
human embryo research in the private arena.
---------------------------------------------------------------------------
    \11\ See NBAC, Ethical Issues in Human Stem Cell Research, Vol. 1, 
pp. 74-77, Recommendation #8.
---------------------------------------------------------------------------
    It is safe to assume that no policy currently under discussion will 
be the final one. We will need to revisit this research again and again 
as the science develops and as its ethical implications become clearer, 
particularly through a public body's review and oversight. Thus, no 
policy will end the national conversation about how to balance, over 
time, the relevant ethical considerations. Our public dialogue needs to 
continue, with the kind of reflective leadership that Congress, at its 
best, can provide. While this dialogue continues, we need a policy with 
greater flexibility than the one President Bush announced, but also 
with closer review and oversight than some would like.
    In a recent editorial in Science, ethicist LeRoy Walters stressed 
that ``Governments and their advisors will need to be humble and 
flexible, but also decisive and courageous.'' \12\ We must carefully 
scrutinize claims of scientific promise, being wary of unfounded 
optimism, but we must not neglect research that offers a significant 
prospect of major medical breakthroughs that may alleviate human 
suffering and reduce the number of premature deaths. As a society, we 
must provide clear and strong ethical guidelines, regulations, and 
safeguards in stem cell research, while avoiding unreasonably rigid 
rules that appear to be arbitrary and inconsistent.
---------------------------------------------------------------------------
    \12\ Walters, ``Human Embryo Research: Lessons from History.''
---------------------------------------------------------------------------
    Thank you--I will be glad to answer any questions. \13\
---------------------------------------------------------------------------
    \13\ 1 am grateful to Alta Charo and LeRoy Walters for their 
thoughtful and helpful comments on an earlier draft of this testimony; 
they are absolved of any responsibility for its content.

    The Chairman. Father FitzGerald?
    Father FitzGerald. I would like to thank the chair, Senator 
Kennedy, and the members of this distinguished community for 
this opportunity to come before you today to discuss this 
issue.
    I would like to bring to bear on this issue my background 
in molecular genetics, in bioethics and in religion to present 
a somewhat different perspective on this issue than I think we 
have heard yet today, and in doing so, I would like to offer a 
caveat. There is no way that I can do justice in the brief time 
allotted to me to the numerous serious, well-informed and 
thoughtful people who are deeply concerned about this research 
and the ethical and moral ramifications of pursuing it. These 
people, of course, include scientists who have no specific 
religious affiliation.
    Since I have limited time, let me focus on an issue that 
was raised earlier by Senator Frist and then again by Senator 
Dodd. That is the issue of what do we do when we do not know. 
There is much in this area that we do not know. We do not know 
much of the science, and we do not know much of what the 
ethical, moral, and social ramifications will be. How are we 
going to respond to that lack of knowledge, because in that 
lack of knowledge, we truly run the risk of overselling the 
promise and underemphasizing the problems associated with human 
embryonic stem cell research.
    As Senator Clinton stated, it is extremely important that 
we receive and create a factual base here on this issue, and 
that factual base will, of course, include more than just the 
scientific facts.
    In addressing this controversial issue, many groups and 
committees, such as the National Bioethics Advisory Committee 
as well as the proposed committee to be directed by Dr. Leon 
Kass, have been gathered together, bringing experts from 
various fields of inquiry and interest to propose how our 
pluralistic society should respond to this controversial area 
of biological research in pursuing progress while protecting 
justice.
    For instance, as Dr. Childress has mentioned, in trying to 
balance the concerns of many people in our Nation, the National 
Bioethics Advisory Commission came to the understanding that 
human embryos are not just tissue; they do have some moral 
status and some value to society. In deciding that, the 
National Bioethics Advisory Commission as well as others who 
have come to similar conclusions set the bar higher for us to 
pursue human embryonic stem cell research than it might be set 
for other avenues of research. So in order to clear that bar 
and justifiably pursue this research, one would have to meet a 
higher standard.
    Addressing a comment made earlier by Senator Specter--he 
said it is important that science may have the full range of 
opportunity--I think it is also important to recognize and 
remember that we already limit what science can do, especially 
in terms of human research.
    The two areas that are most often raised as important to 
address in meeting that higher standard for human embryonic 
stem cell research are need and the number of people who will 
benefit from this research. Let me first address the issue of 
need. Do we need human embryonic stem cell research?
    Granted, human embryonic stem cell research is 
scientifically quite interesting, fascinating, and of great 
interest to the people who do that research to pursue. However, 
oftentimes we talk about pursuing this in connection with the 
number of therapies and cures that will be discovered. In fact, 
I believe the phrases of countless numbers of people, and as 
Senator Specter said, it will touch virtually every family in 
America.
    I would argue that we could make the same claims for other 
avenues of research, certainly research being pursued in 
genetic therapies, research in proteomics, research in drug 
development, and of course, research in adult stem cells. All 
these other areas of research do not need to set the bar as 
high in being pursued. Embryos, human life, is not destroyed.
    Yet at the same time, scientists involved in those avenues 
of research could make similar claims as to the potential of 
their research and the number of diseases they are addressing.
    Second, when we talk about the number of people who will be 
treated and/or cured by any of these medical areas of research, 
we must be careful. We must again be careful not to oversell 
the promise, and remember that in fact there are many, even in 
our own Nation, who do not have access to these new 
technologies and cutting-edge medical products.
    Actually, Senator Murray brought up a very interesting 
example. She asked the question would we have to keep human 
embryonic stem cell products hostage to such things as male 
baldness. And the interesting parallel there is that in the 
early 1990's, a drug was developed with FDA approval to treat a 
disease that afflicts hundreds of thousands of people a year. 
It is called sleeping sickness. It happens in Africa. The drug 
was the most effective drug known against that disease. That 
drug was developed because it might be a cancer research tool 
or a cancer therapy. When it proved not to be so, the company 
that was producing the drug decided not to produce it anymore, 
even though it could cure or at least treat hundreds of 
thousands of people. However, the drug is back. It is back 
because it has a side effect--it causes hair loss. So they have 
turned it into a cream that women can use to treat excessive 
facial hair.
    When we say that we are going to promise people these 
wonderful potential therapies and cures, are we indeed giving 
them the accurate assessment of how our systems work? My issue 
is not necessarily specifically with the pharmaceutical 
industry but in promising the people of this country and 
perhaps the world a solution and a promise that we cannot 
fulfill.
    I very much appreciate the work of this committee, and I 
appreciate the reflection that has gone into the many proposals 
here and the many presentations, and I certainly hope that we 
continue in our reflection on this very serious issue.
    Thank you very much.
    The Chairman. Father, that drug that you are talking about 
is methyltrexate. My son, who had osteosarcoma, took that drug 
when he was in an NIH trial, and he survived. Now, there is 
about an 85 to 90 percent survival rate in children with that 
disease. So you would have a tough time convincing me on that 
particular one--I just picked that up.
    Father FitzGerald. Actually, the drug is not methyltrexate; 
it is F-fluorothene.
    The Chairman. OK. I stand to be corrected.
    [The prepared statement of Father FitzGerald follows:]
           Prepared Statement of Father Kevin FitzGerald, SJ
    Advances in medical research are happening at such a rapid rate 
that it seems new breakthroughs are announced every week in the media. 
These advances present the possibility for treatments and cures unheard 
of only a generation ago. However, advances in research can also create 
ethical problems seemingly so complex and convoluted that we wonder 
whether or not the research is worthwhile. In our society, which claims 
to value both progress and justice, we must find a balance between the 
Utopian hopes and Frankensteinian fears generated by these advances in 
medical research.
    One area of recent advance in medical research has raised new hopes 
for treating serious illnesses that result from the death or 
deterioration of cells and tissues we require for good health. These 
illnesses include terrible neurological disorders such as Parkinson's 
and Alzheimer's, as well as tragic injuries such as paralysis caused by 
damage to a person's brain or nervous tissue. The basis for these new 
hopes is a new understanding of human stem cells and their function.
    What do these stem cells do? The normal function of stem cells is 
to produce new cells and to replace cells we lose through the natural 
processes of cellular aging and death. From the time a human being 
begins as a fertilized egg, that human being grows and develops by 
cells continually dividing to make more cells. Early on in our 
development, when we are embryos, the primary function of most of the 
cells is to divide and rapidly make more cells. ``Embryonic'' stem 
cells have not yet become any particular type of cell, such as muscle 
or nerve, and so embryonic stem cells are thought to be capable of 
becoming any type of cell in the human body.
    As a human being continues to develop as a fetus, infant, child, 
and adult, the number of cells in the body increases. During these 
later stages, most of the cells stop dividing and take on specific 
duties and become brain cells, or liver cells, or skin cells, etc. 
However, some cells keep their ability to divide and replace other 
cells that have been lost due to damage or normal aging. These stem 
cells are generally called ``adult'' stem cells. Until just the past 
few years, adult stem cells were considered to be only in certain 
tissues, like blood and skin, and to have only the capacity to replace 
the cells of the particular tissue within which they were situated. 
Recent research has now indicated that adult stem cells are present in 
many, if not almost all, of the tissues of the body, including the 
brain. In addition, adult stem cells are not limited to replacing cells 
from only the type of tissue wherein they are found.
    New breakthroughs in stem cell research have involved both 
embryonic and adult stem cells. Researchers have discovered ways to 
isolate and grow embryonic stem cells such that they can be directed to 
become different types of cells that are found in the body. Since 
during embryonic development embryonic stem cells do become all the 
different types of cells in the body, researchers speculate that it 
should be possible, eventually, to direct embryonic stem cells to 
produce whichever kind of cell is needed.
    Though this research sounds very promising, there are both 
scientific and ethical troubles with embryonic stem cell research. One 
scientific obstacle is the problem of controlling the development of 
the embryonic stem cells into the type of tissue one might need to 
treat a disease or injury. Since these cells have such a great ability 
to make more cells of any kind, it is important that researchers know 
that there are no uncontrolled embryonic stem cells being implanted 
into a person. An embryonic stem cell that does not convert into the 
type of tissue desired could cause significant harm to a patient by 
making the wrong kind of cells in the treated tissue, or by just 
growing out of control and creating a tumor. Much research on embryonic 
stem cells would still be required in order to insure the safety of 
these proposed treatments. This research, in itself, raises a key moral 
issue.
    The key moral obstacle to embryonic stem cell research is that 
currently the only way embryonic stem cells are obtained is by 
destroying an embryo. Unlike tissue or organ transplantation, where the 
organs are removed after the death of a person, embryonic stem cells 
are not harvested after the embryo has died. The procedure for removing 
the embryonic stem cells from the embryo destroys the embryo.
    Much of our health care tradition is based on the idea that healing 
is a benefit to be made available to all. Consequently, it is not 
acceptable that for some to be healed others must be sacrificed--no 
matter their State in life. In response to this perspective, some argue 
that frozen ``spare'' embryos, left over from in vitro fertilization 
treatments and not likely ever to be used to produce a pregnancy, might 
justifiably be destroyed in order to get embryonic stem cells. However, 
using a fundamental principle of health care which states that first of 
all one should not unnecessarily harm another, one can counter that no 
human life is ``spare.'' Who among us has the right to decide that 
another human life is a ``spare'' life, especially when that human life 
does not have the chance to contest the decision? We do not consider it 
appropriate to take organs from dying patients or prisoners on ``death 
row'' before they have died in order to increase someone else's chances 
for healing or cure. Neither, then, should we consider any embryos 
``spare'' so that we may destroy them for their stem cells.
    This defense of human life does not mean that all stem cell 
research must be rejected. Research on adult stem cells should be 
encouraged, especially in light of the new results that indicate its 
amazing promise for treatment and cure. For example, blood stem cells 
may be able to replace lost brain cells or liver cells, and vice versa. 
In the not-to-distant future, the issue of rejecting transplants may no 
longer be a problem because our own stem cells will be used to repair 
and replace our damaged cells and tissue.
    Even taking the promise of adult stem cells into consideration, 
some scientists argue that the most efficient way forward is to do 
research on all the various types of stem cells in order to see which 
will be best for treating the many different diseases we wish to cure. 
This approach of doing all the different types of experiment that can 
be done is often the best way for science to proceed. But is it always 
the best way for a society to proceed?
    We Americans know from our own history with eugenics and research 
on minorities or the mentally disabled the tragedies that can occur 
when public policies concerning human experimentation are shaped 
primarily by the dictates of science. More broadly, the history of 
humankind abounds with examples of how biological characteristics such 
as race, gender, intelligence, and age, have been used as justification 
for discrimination against certain groups within a society so that the 
dominant groups in a given society might take advantage of the 
vulnerable and those on the margins of the society. In response to 
these wrongs, our society has chosen to limit what experiments can be 
performed on human beings, even though these limits may slow scientific 
progress. Are we not repeating this same tragic pattern by declaring 
embryos of significantly lesser value than other human beings because 
of their developmental status? And if human embryos do have some 
significant value in our society, as the National Bioethics Advisory 
Committee concluded, then considering all the basic research that still 
can be done using animal models, human tissue culture, and adult stem 
cells, why is there a continuing clamor for the destruction of human 
embryos to fuel stem cell research?
    One reason often put forth as justification for the destruction of 
more and more human embryos is the need to bring healing and cures to 
the thousands, if not millions, who suffer from illnesses and diseases 
that may be amenable to treatment with embryonic stem cell therapies. 
Such an argument as this is of great significance for it connects to 
another fundamental principle of medicine: treat sickness and heal when 
you can. Yet, as the argument is stated, its significance rests in part 
on two assumptions: 1) that embryonic stem cells will be necessary, or 
superior to all other options, in the treatment of certain diseases, 
and 2) that the thousands and millions who need the treatments will 
have access to them.
    Addressing the first assumption, we need to recognize that the 
diseases suggested as likely targets for human embryonic stem cell 
research are also the targets of researchers using other approaches, 
such as genetic therapies, drug development, and adult stem cells. It 
may well be the case that for many patients the treatments for their 
illnesses may come more quickly from research avenues other than human 
embryonic stem cell research, and that these alternative treatments may 
even be better than any treatment derived from human embryonic stem 
cell research.
    Regarding the second assumption, we need to acknowledge that even 
if treatments from human embryonic stem cell research prove to be the 
best available and are developed first, many people who need these 
treatments will not have access to them. For example, the World Health 
Organization statistics show that approximately 900,000 children die 
each year of measles. Human embryonic stem cell research is not needed 
to prevent measles infections and the cost of the measles vaccine is 
dramatically less than any human embryonic stem cell treatments will be 
for a very long time, if they are ever produced. Hence, just because 
many people in the world tragically share a devastating disease, the 
greater tragedy is that only a relative few get to enjoy the benefits 
of cutting edge medical technology. This tragic reality should not 
prevent our pursuit of medical advances, but it should also not be 
forgotten when we decide as a society which medical research is 
justified and which is not.
    If the justification for proceeding with the destruction of human 
embryos for research rests even in part on these two assumptions of 
need and the number of those who will benefit, then this justification 
is flawed. Human embryos need not be destroyed in order that thousands 
or millions might be saved.
    Indeed, without the continual destruction of human embryos the 
future of medical advance will still be one of great hope. There are 
many avenues of medical research that can be pursued with broad ethical 
and societal support. As a people who value progress and justice, we 
can decide to pursue every avenue of medical research that is 
respectful of human life in all its stages, and brings care and healing 
to all those in need.

    The Chairman. Dr. Chute?
    Dr. Chute. First, I want to thank you, Senator Kennedy, and 
the committee for inviting me to appear today.
    I come to this issue as a clinician first, as a clinically 
trained hematologist and oncologist. For the past 5\1/2\ years, 
I have also been directing research in adult hematopoietic stem 
cell biology.
    I also want to say that it has been with great interest 
that I have followed the work of Dr. Thomson and those who have 
done embryonic stem cell line research. I have been fascinated 
that they have been able to propagate these cells in vitro as 
long as they have shown they can in peer-reviewed journals and 
that they can get these cells to differentiate into neuronal 
epithelial muscle and hematopoietic cells.
    It is very understandable that patients with very serious 
diseases are very excited and hopeful that there may be cures 
in the future for such things as diabetes, Parkinson's, and 
Alzheimer's derived from further research on these cells.
    But as the national discourse on embryonic stem cell 
research has progressed, there has been, ironically, a 
diminishing appreciation of the exceptional progress that has 
been ongoing and occurring in adult stem cell research, and I 
appear today to highlight the critical importance of the 
ongoing research in adult stem cell biology.I21In the year 
2000, there were 1.2 million new cases of cancer in the United 
States. Cancer is the second leading cause of death in the 
United States, and the incidence of cancer is increasing. Adult 
hematopoietic stem cells have been and are currently used 
successful in the treatment and cure of patients with leukemia, 
lymphoma and other hematologic malignancies.
    Also of great interest is that newly developed methods of 
transplanting adult hematopoietic stem cells have now been 
shown to effect major remissions in solid tumors. There is a 
group at NIH led by Dr. Barrett and Dr. Chiles that has 
recently shown that kidney cancer can be put into remission 
with the donation of normal peripheral blood stem cells, which 
is a fascinating new development in the use of these cells to 
treat cancer.
    Therefore, as this work moves forward, an even greater 
number of patients will benefit from adult stem cell 
transplantation. As further evidence, I submit to you that the 
two most important medications that we as oncologists give to 
patients in their cancer treatments are epagen and nupagen, 
which are both growth factors, both of which were developed and 
isolated through federally-funded adult stem cell research. 
These medications promote the recovery of red and white blood 
cells following chemotherapy and allow patients' quality of 
life to be significantly improved while also preventing life-
threatening infections.
    Adult hematopoietic stem cells are the ideal vehicle for 
gene therapy to treat such diseases as sickle cell anemia, 
hemophilia and immune deficiencies. More than 100,000 children 
in America are threatened with these life-threatening genetic 
diseases. For the first time in the past 2 years, two recent 
publications have shown the successful gene transfer into 
patients with disease. Both of these studies used adult 
hematopoietic stem cells.
    At the end of the year 2000, there were 70,000 Americans 
awaiting organ donation for kidney, liver, heart, or lung 
disease. Recent animal studies indicate that the co-
administration of hematopoietic stem cells along with organ 
transplantation may dramatically lessen the need for 
immunosuppression. If this work moves forward, thousands more 
patients will be able to be successfully transplanted with 
organs with much less morbidity.
    In addition to these current applications, even broader 
clinical therapies will derive from adult stem cells in the 
near future. Published reports in the last 5 years have shown 
that transplants of bone marrow stem cells can differentiate in 
vivo into functional liver cells, skeletal muscle, brain cells, 
and even functioning cardiac myocytes. These data indicate that 
a rare subset of hematopoietic stem cells is in fact 
pluripotent and possess at least limited plasticity. Adult cord 
blood stem cells can be maintained now in culture for up to 12 
weeks. Our laboratory has shown that bone marrow stem cells can 
be expanded 10-fold in just a week of culture under specialized 
conditions. Whether adult stem cells will match embryonic stem 
cells in the treatment of diseases like Parkinson's or 
Alzheimer's remains unknown, and in my opinion is probably 
unlikely. But given the dramatic progress in adult stem cell 
research in the last 5 years, I think continued funding is 
merited.
    To close, I would like to just make two points about 
embryonic stem cell research that are scientific concerns. 
First, immune barriers for embryonic stem cells to work in 
transplantation for treatments like Alzheimer's and Parkinson's 
will require immune barriers to be crossed. Scientists who are 
experts in this field, specifically, Dr. Thomson at Wisconsin, 
have argued that cloning is the way to get beyond this. In 
fact, I think the scientific community recognizes that if these 
immune barriers cannot be crossed with some method such as 
nuclear transfer, the translation into actual therapies for 
patients will be extremely difficult.
    Second, animal studies indicate that the transplantation of 
embryonic stem cells actually has a high incidence of teratoma, 
or tumor development, in small animal models. In vitro 
differentiation and genetic engineering of embryonic stem cells 
prior to transplant have been proposed as methods to circumvent 
this problem, but both of these approaches have further 
technical concerns. As Dr. Thomson himself has recently cited, 
the heterogeneous nature of embryonic stem cell development in 
culture has hampered the use of embryonic stem cell derivatives 
in transplantation.
    I close with the very brief following statement. The 
limitations of embryonic stem cells as a source of 
transplantable tissue should be openly and honestly presented 
to the public, since treatments and cures using these cells are 
certainly not imminent.
    In contrast, adult hematopoietic stem cells are 
successfully used in the treatment of patients with common 
diseases every day. Genetic engineering is not required for 
these cells to be safely applied. New treatments for diseases 
are imminent with adult stem cells and not hypothetical. While 
scientists continue to explore the basic biology of embryonic 
stem cell lines, the needs of thousands of patients who benefit 
from adult stem cell research should not be sacrificed.
    Thank you.
    [The prepared statement of Dr. Chute follows:]
               Prepared Statement of John P. Chute, M.D.
    1 would like to first thank Senator Kennedy and the Senate 
Committee for inviting me to appear at this important hearing. As a 
clinician who does research, it is an honor to be here. My specialty 
training is in Internal Medicine, Hematology and Medical Oncology. Upon 
completion of my clinical training in 1996, 1 joined the Stem Cell 
Biology Laboratory at the Naval Medical Research Institute. Since June 
1999, 1 have been the Head of the Hematopoietic Stem Cell Studies 
Section within the joint NIDDK/Navy Transplantation and Autoimmunity 
Branch. The focus of our lab has been the development of methods to 
cultivate and expand adult hematopoietic stem cells for medical 
therapies. Therefore, it has been with great interest that I have 
followed the remarkable progress which has been made in the development 
of human embryonic stem cell lines. I have admired the work of 
investigators like Dr. James Thomson at the University of Wisconsin in 
demonstrating that embryonic stem cell lines can be propogated in vitro 
through hundreds of cell divisions and that these cells can be induced 
to differentiate in vitro into neuronal, epithelial, muscle and 
hematopoietic cells, to name just a few (1,2). In contrast, the 
experience of investigators in attempting to propagate and induce the 
in vitro differentiation of adult hematopoietic stem cells into diverse 
tissues has been generally unsuccessful (3,4). Understandably, 
physicians, legislators, the media, and most importantly, patients, 
have become increasingly hopeful that the further development of human 
embryonic stem cell research might lead to treatments and even cures 
for diabetes mellitus, parkinson's disease, a1zheimers, muscular 
dystrophy, and other degenerative diseases. Unfortunately, the 
optimistic pronouncements by members of scientific community about the 
``imminent'' therapies which would be forthcoming from embryonic stem 
cell research has raised the innocent expectations of the most 
vulnerable members of our society (patients with debilitating diseases) 
to a level which medical researchers may never meet.
    Last month, President Bush set forth a policy to provide Federal 
funding to support ongoing research on specified human embryonic stem 
cell lines. During his address, he noted that the Federal government, 
via NIH, currently spends approximately 250 million dollars annually on 
``stem cell research''. The overwhelming majority of this money 
heretofore has been applied to basic and clinical research on adult-
source stem cells. In order to jump-start the Federal funding of 
embryonic stem cell research, it has been discussed that 100 million of 
the Federal dollars which the NIH would have appropriated toward adult 
stem cell research will be taken to support the fledgling embryonic 
stem cell initiative. Sacrificing 40% of the current adult stem cell 
research funding for the purpose of initiating further embryonic stem 
cell research would be a mistake of historical proportions and would 
risk harming hundreds of thousands of patients in the United States who 
currently benefit from adult stem cell based therapies.
    The American Cancer Society estimates that there were 1.2 million 
newly diagnosed patients with cancer in the U.S. in the year 2000 (5). 
Cancer is the second leading cause of death in the United States and it 
continues to gain on the leader, cardiovascular disease. Adult stem 
cells from bone marrow, cord blood, and peripheral blood have been and 
are currently used safely throughout the U.S. in the treatment and cure 
of tens of thousands of patients with leukemia, lymphoma, and certain 
types of solid cancers (6). Current research advanced by NIH 
researchers further indicates that, when given in a 
``nonmyeloablative'' manner (less toxic), patients with solid tumors 
(e.g. kidney cancer) for the first time are achieving complete 
remissions simply due to the anti-cancer effect of the infused bone 
marrow cells (7). These results indicate that an even greater number of 
cancer patients will benefit in the near future from adult stem cell 
transplantation. But more research funding will be needed to meet these 
goals. The success of adult stem cell research can also be measured by 
examining the two most important medications given to all patients who 
undergo chemotherapy and radiation: these medications, erythropoietin 
(epogen) and neupogen are natural growth factors which act within human 
bone marrow to stimulate red blood cell recovery and white blood cell 
recovery following highly toxic chemo- and radiation therapy. Without 
these 2 medications, thousands of cancer patients would require longer 
hospitalizations, suffer significant life-threatening infections, and 
patient quality of life would be dramatically worsened. Both of these 
medications were discovered via research on adult hematopoietic stem 
and progenitor cells.
    In our laboratory, we have utilized Federal research funds to 
search for the genes and proteins which induce the self-renewal of 
adult hematopoietic stem cells (8). We are currently participating in a 
cooperative research agreement with Large Scale Biology Corporation 
(Vacaville, CA and Rockville, MD) to apply high level protein 
identification techniques to achieve this end. We anticipate 
identification of specific human stem cell growth factors within the 
next 2 years. This research may lead to new therapeutic growth factors 
which could be administered to patients with cancer to allow earlier 
recovery from the adverse effects of chemotherapy and radiation. Yet, 
despite its potent effects in the elimination of disease, bone marrow 
transplantation is a morbid procedure for donors, requiring one liter 
of marrow to be harvested for each adult transplantation. Umbilical 
cord blood is an alternative and attractive source of hematopoietic 
stem cells for this purpose, and the ease of collection and banking of 
cord blood stem cells could allow for a universal bank of stem cells so 
that the majority, not the minority, of Americans might have an 
immunologically matched stem cell graft available for them should they 
need it. But more Federal funding, not less would be needed to develop 
such a national cord blood bank.
    Adult stem cells from the bone marrow or peripheral blood are also 
the ideal vehicle for gene therapy to treat such common and 
debilitating diseases as sickle cell anemia, hemophilia, thallasemia, 
and immune deficiencies (9). More than 100,000 children are afflicted 
with life-threatening forms of these genetic diseases in the United 
States (10). For the first time, 2 recent reports have demonstrated 
successful gene transfer and gene expression of functional proteins in 
patients transplanted with adult stem cells. As adult stem cell 
researchers continue to provide new methods to successfully introduce 
normal genes into adult stem cells, major diseases such as sickle cell 
anemia and hemophilia will become treatable for the first time. In 
addition, with the advent of the human genome database, an increasing 
number of treatable genetic diseases will be identified. But more 
research funding will be required to reach these goals.
    As of the end of the year 2000, there were 70,373 Americans 
awaiting organ donation for end stage kidney, liver, heart, or lung 
disease (11). Organ transplant recipients face major morbidities and 
life-threatening complications due to the immunosuppression which they 
require in order to prevent organ rejection. Recent research in animal 
models has indicated that concomitant infusion of donor bone marrow 
stem cells can induce an ``immune tolerant'' State in the recipient 
which may allow long-term acceptance of the donated organ without the 
requirement for prolonged immunosuppression (12). The standardized 
clinical transplantation of donor bone marrow coupled with organ 
transplantation could allow tens of thousands of more patients to be 
successfully cured of their end stage liver or kidney disease with much 
less morbidity and complications.
    In addition to these current clinical applications, ongoing 
research indicates that even broader clinical therapies will derive 
from adult stem cells in the near future. Investigators have 
demonstrated in animal models that transplanted bone marrow stem cells 
have the capacity to differentiate in vivo into liver cells, skeletal 
muscle cells, brain cells, and even functioning cardiac muscle cells 
(13-17). These data indicate that a rare subset of adult hematopoietic 
stem cells are pluripotent and possess at least limited plasticity. 
Scientists in the U.S. and Italy have recently demonstrated that cord 
blood stem cells could be maintained and expanded in number in culture 
for up to 12 weeks and our laboratory has recently shown that bone 
marrow stem cells can be increased 10-fold in 1 week of culture under 
specialized conditions (18-20). The capacity to expand the numbers of 
human adult stem cells in vitro is a critical first step toward 
harnessing these cells for future medical treatments. Whether adult 
stem cells will be useful in the future clinical treatment of diseases 
such as diabetes, parkinson's or a1zheimer's remains unknown and 
perhaps unlikely. But given the dramatic discoveries of the past 5 
years, along with the fact that adult stem cells are a proven safe and 
genetically stable source of tissue for transplantation, continued 
research on adult stem cells is merited.
    Embryonic stem cells hold great promise as a potential source of 
tissues for the treatment of many debilitating and degenerative 
diseases. However, that potential faces several important scientific 
hurdles prior to the realization of patient application. First, immune-
mediated rejection of transplanted embryonic stem cell grafts will 
occur in recipients unless the individuals are heavily 
immunosuppressed. This immunosuppression would result in major 
morbidities and life threatening complications for patients which might 
outweigh the potential benefits of the transplant (1). Alternatively, a 
patient's DNA could be introduced into an enucleated egg, for the 
purposes of generating blastocytsts (embryos) which contain embryonic 
stem cells which would be immunologically identical to the patient. 
Even if this form of cloning was not controversial, it is highly 
inefficient and would likely require large resources of eggs for the 
purposes of developing genetically matched tissue grafts. Second, 
animal studies indicate that the transplantation of embryonic stem 
cells leads to a high incidence of teratoma (tumor) development. This 
risk is clearly incompatible with patient transplantation and requires 
that these cells would have to be induced to differentiate in vitro 
into desired tissue lineage or genetically engineered prior to 
transplantation (1). Such transplanted cells would likely have a 
limited in vivo life expectancy and might require additional 
transplantations over time. These limitations of embryonic stem cells 
as a source of tissue for human transplantation should be openly and 
honestly presented to the public, since treatments and cures from these 
cells are not imminent. Most importantly, the requisite studies in 
small animals and primates to prove efficacy and safety are far from 
completed. In contrast, adult stem cells are successfully used in the 
treatment and cure of many diseases every day and genetic engineering 
is not required for these cells to be safely applied. More importantly, 
new treatments are imminent, not hypothetical. While scientists 
continue to explore the basic biology of embryonic stem cell lines, the 
needs of thousands of patients who benefit from adult stem cell 
research should not be sacrificed.

                               References

    1. Odorico J, Kaufman D, Thomson J. Multilineage differentiation 
from human embryonic stem cell lines. Stem Cells 19:193, 2001.
    Thomson J, Itskovitz-Eldor J, Shapiro S et al. Embryonic stem cell 
lines derived from human blastocysts. Science 282:1145, 1998.
    Traycoff C, Cornetta K, Yoder M et al. Ex vivo expansion of murine 
hematopoietic progenitor cells generates classes of expanded cells 
possessing different levels of bone marrow repopulating potential. Exp 
Hematol 24:299, 1996.
    Antoniou M. Embryonic stem cell research: The case against.... Nat 
Med 7:397, 2001.
    American Cancer Society Report: Facts and Figures 2000. Cancer: 
Basic Facts. Chapter One, page 1, 2000.
    International Bone Marrow Transplant Registry. Information 
Services, Summary 2000.
    Childs R, Chernoff A, Contentin N et al. Regression of metastatic 
renal cell carcinoma after nonmyeloablative allogeneic peripheral blood 
stem cell transplantation. N Engl J Med 343: 750, 2000.
    Chute J, Saini A, Kampen R, et al. A comparative study of the cell 
cycle status and primitive cell adhesion molecule profile of human 
CD34+ cells cultured in stroma-free versus porcine microvascular 
endothelial cell cultures. Exp Hematol 27:370, 1999.
    Dick J. Gene therapy turns the comer. Nat Med 6:652, 2000.
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Questions: How common is sickle cell anemia? 2001; National Hemophilia 
Foundation. Information Center, page 1, 2001.
    UNOS Transplant Patient Datasource. Statistics 2000, National Data.
    Hale D, Gottschalk R, Umemura A, et al. Establishment of stable 
multilineage hematopoietic chimerism. and donor-specific tolerance 
without irradiation. Transplantation 69:7, 2000.
    Lagasse E, Connors H, Al Dhalimy M, et al. Purified hematopoietic 
stem cells can differentiate into hepatocytes in vivo. Nat Med 6:1229, 
2000.
    1. Ferrari G, Cusella-De Angelis G, Coletta M et al. Muscle 
regeneration by bone marrow derived myogenic progenitors. Science 279: 
1528, 1998.
    Mezey E, Chandross K, Harta G et al. Turning blood into brain: 
cells bearing neuronal antigens generated in vivo from bone marrow. 
Science 290:1779, 2000.
    Brazelton T, Rossi F, Keshet G et al. From marrow to brain: 
expression of neuronal phenotypes in adult mice. Science 290:1775, 
2000.
    Orlic D, Kajstura J, Chimenti S et al. Bone marrow cells regenerate 
infracted myocardium. Nature 410:701, 2001.
    Piacibello W, Sanavio F, Severino A, et al. Engraftment in nonobese 
diabetic severe combined immunodeficient mice of human CD34+ cord blood 
cells after ex vivo expansion: evidence for the amplification and self 
renewal of repopulating stem cells. Blood 93:3736, 1999.
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cell monolayers
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CD34+CD38progenitor cells. Hu Gen Tber 11: 2515, 2000.
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Submitted 2001.

    The Chairman. Thank you very much.
    I thank all the members of the panel. We are going to 
recess, and I would like to come back with some questions at 
2:15. I know this causes some concern, perhaps, for your 
schedules, but if you can come back--if you cannot, I can 
understand, and we will submit some written questions. I do not 
think it will take long; hopefully, we will have you out by 3 
o'clock. So if we could do that, we would very much appreciate 
it. We have had a very full morning, and the two parties have 
caucuses that start at 12:30, where I see most of our members 
have gone. I think that by 2:15, they will have ended, and 
hopefully, we will have some additional participation.
    I am very, very grateful to all of our panelists.
    Senator Frist. Mr. Chairman.
    The Chairman. Yes, Senator Frist.
    Senator Frist. Let me just add that this is fascinating 
based on the discussions this morning. If some of the panelists 
cannot stay, if we could just submit to them written questions, 
because we do not know what their schedules are. I do have 
questions, and I do not think we can spend a long time this 
afternoon, so if we could be permitted to submit written 
questions.
    The Chairman. Certainly.
    Thank you. We will meet again at 2:15.
    The committee stands in recess.
    [Whereupon, at 1 p.m., the committee recessed, to reconvene 
at 2:25 p.m. this same day.]

                           AFTERNOON SESSION

    The Chairman. The committee will come to order.
    I will invite our witnesses to return to the table if they 
would, please. Thank you very much for adjusting your 
schedules.
    I would like to go through a few questions if I might, and 
I will start with Dr. Melton. You expressed several concerns 
about the restrictions placed on stem cell research under the 
President's proposal. Do you believe that the restrictions 
could significantly impede scientific progress and slow the 
development of new cures using these cells?
    Mr. Melton. Yes, I do. I do think they will impede 
progress.
    The Chairman. Do you want to elaborate on that? We talked 
this morning about the issues of accessibility and reliability 
and safety, contamination. As a researcher, what are your 
concerns?
    Mr. Melton. I could give one example. You could imagine 
that with the cells that are available, scientists discover a 
means by which they can turn human embryonic stem cells into 
pancreatic beta cells for the treatment of diabetes. You would 
then want to use human embryonic stem cells that have been 
freshly derived in the absence of these potentially 
contaminating mouse feeders and use those for therapies, 
because that would eliminate concerns about known and unknown 
viruses. As I understand it, the new cell lines which would be 
derived would be ineligible for Federal researchers. That is 
one example of the possible problems that will likely arise.
    The Chairman. How concerned should we be about 
contamination? Have you used products from other countries, and 
what is your own sense or knowledge as to how high you think 
the risk might be?
    Mr. Melton. I am not really qualified to comment on that, 
but I would say that the FDA, as I understand it, will treat 
this as a xenotransplant, which means that the hurdles and the 
requirements for using a therapy will be much more onerous.
    Dr. Chute can probably comment more on the difficulties 
with using animal products in treating humans.
    The Chairman. Dr. Chute?
    Dr. Chute. Thank you, Senator Kennedy.
    In our laboratory, we worked with a feeder layer that was a 
porcine feeder to grow adult hematopoietic stem cells. I have 
had iterations with the FDA both on using a porcine and a human 
feeder layer, and without question, there is a long series of 
safety tests that you have to do on the human cells that are 
cultivated with xeno feeder layers such as mouse/porcine. And 
even with a human feeder layer, there is still a very, very 
long list of safety tests that have to be done.
    But I would make the comment that I do not think it is 
impossible if you use a mouse feeder layer that the human cells 
derived from those cultures could ever be used in the clinic. I 
think it just makes it more arduous, without question. It might 
add a year or a two potentially to the time before you get to 
the clinic.
    The Chairman. Well, you are certainly restricted now, under 
the August 9 date, so you probably would not get there.
    Dr. Chute. Correct.
    The Chairman. Dr. Melton, after reviewing the agreement 
signed by NIH and the University of Wisconsin, I see that it 
expressly forbids use of the cells for therapeutic or 
diagnostic purposes. What are the implications of restrictions 
of this kind for the work you do in trying to find new 
treatments for juvenile diabetes?
    Mr. Melton. Well, first, I am sort of surprised by that 
news, because I have not seen the agreement. But if it is the 
case as you say that the cells could not be used for 
treatments, I would think that would be a damning condition. 
Clearly, there is no point in doing the research that I am 
doing if it were not for the possibility of treating people. So 
I would have to see the conditions to comment further.
    The Chairman. Ms. Hersey, the President's plan gives a 
handful of suppliers control over all the federally-approved 
stem cell lines. Based on your experience negotiating 
agreements with private companies, wouldn't you agree that this 
type of monopoly is likely to make it difficult for NIH-funded 
researchers to get prompt access to these cells?
    Ms. Hersey. Yes, I think it is going to make it very 
difficult, Senator.
    The Chairman. Are you familiar with the other types of 
situations that you know of from the past that are similar to 
this kind of a monopoly?
    Ms. Hersey. There have been several. Especially where the 
company holds patent right, we have seen a number of them. In 
some cases, the NIH has been able to step in as they have this 
time and try to make a difference for the university 
researchers. But I would say that at least 20 percent of the 
time, we cannot get access to the materials we want because of 
the encumbrances.
    The Chairman. Dr. Childress, I want to underline one point 
you made in your testimony. Is it fair to say that you see no 
ethical differences between a stem cell derived from a 
discarded embryo on August 9 and one derived at a later date?
    Mr. Childress. As long as they meet the kinds of ethical 
standards that President Bush laid out, and I think those are 
important ones, about the embryo being left over following 
efforts at reproduction, that the donors give voluntary 
informed consent, and that there be no financial inducements. 
If we go forward and apply the same ethical standards to the 
derivation of stem cells from embryos in the future, I cannot 
see an ethical difference. I cannot see that what happened 
before August 9 and what happens after is ethically different.
    The concern that some have expressed, that this might well 
sanction and encourage the destruction of embryos, I think is 
also problematic as a concern, because after all, to this 
point, people may well have made decisions about destruction of 
embryos and the possibility of privately-funded research, since 
that research has been going forward.
    Furthermore, we really do not have evidence from fertility 
clinics that people deciding to discard embryos make this a 
major factor in decisionmaking. So I would be inclined to say 
that we can build the ethical safeguards to prevent the kind of 
scenario that has concerned many.
    The Chairman. You have followed the fetal transplantation 
issue closely.
    Mr. Childress. Yes.
    The Chairman. Have you formed any impression--and maybe I 
could ask others on the panel as well--in that debate, we had 
the agreement for the use of certain fetal tissue, but we also 
established guidelines for utilization in the private sector--
have you thought about that issue as well, and can you tell us 
what your thinking has been, what the advantages or concerns 
would be, or perhaps the disadvantages?
    Mr. Childress. First, I think there are significant 
parallels between the kinds of ethical safeguards one would try 
to set up, which appear to have been effective, in the area of 
human fetal tissue transplantation research, and the kinds of 
guidelines that would be appropriate in two different settings. 
One would be the use of embryos left over after in vitro 
fertilization, but also the research that we have not really 
talked about today, the derivation of embryonic germ cells from 
aborted fetuses. One could draw a parallel there also, and that 
area of research has been omitted from much of the recent 
discussion, and I am not sure whether it merits further 
attention or not. I have not followed the scientific 
developments on that side, but one could perhaps make a case 
for paying some attention to that as well.
    The Chairman. Throughout your distinguished career, you 
have shown a deep reverence for life. Do you think that 
allowing federally-funded doctors both to use and derive stem 
cells from discarded embryos is consistent with those deeply-
held beliefs?
    Mr. Childress. I believe so, first of all, if we work with 
the notion which the National Bioethics Advisory Commission 
also tried to articulate, that it is very important to 
recognize that the embryo deserves an appropriate form of 
respect, appropriate to that stage of developmental life. Now, 
there will be widespread disagreement in society, as I have 
mentioned earlier, about the moral status of the embryo and 
exactly what kind of respect it deserves, what kind of 
protection is appropriate.
    On the National Bioethics Advisory Commission, we drew 
several implications from the notion of respect for the early 
embryo. One certainly was that in this area, we should not be 
buying and selling embryos or, if we go in the direction of 
fetal tissue, we should not be buying and selling fetal tissue.
    One could also argue that we should not use left over 
embryos unless they are necessary.
    Now, there would be considerable debate. I happen to be on 
the side of those who think that, given the promise of this 
research for alleviating suffering and reducing the incidence 
of premature death, we ought to be exploring all sources of 
stem cells at this point. And I certainly agree with my 
colleagues who stress that we should not neglect adult stem 
cells in this process, and just seeing which ones will be most 
important in developing the kinds of treatments and, we hope, 
cures that might be possible.
    One implication that we drew from this principle of respect 
for the early embryo was that at this point, we should not 
deliberately create embryos for purposes of use in research, 
whether it is through IVF or so-called therapeutic cloning.
    We emphasized--and I would agree with this emphasis--that 
the point we were making was ``at this time,'' because it may 
well be necessary to revisit this question of so-called 
therapeutic cloning if the basic research ends up producing 
some clinically effective treatments that may well best be 
done, or perhaps only be done, with matched tissue. So it may 
be necessary to revisit that at some point. But we have heard 
from many colleagues, and we still have a long way to go before 
that becomes a critical question again and then might raise the 
issue of the necessity of going in that direction.
    I would be opposed, though, at this point to a ban on 
therapeutic cloning, worrying that that would indeed set an 
inappropriate limit for future developments.
    The Chairman. Thank you.
    We will submit the questions of our colleagues.
    [Response to questions from committee members were not 
available at time of printing.]
    The Chairman. I am grateful to the panel, and I thank all 
the witnesses for their excellent testimony. It has been an 
extremely informative and important hearing. It is clear that 
stem cell research offers a virtually unprecedented opportunity 
to find cures for a host of dread afflictions from cancer to 
heart disease, from diabetes to spinal cord injury to 
Parkinson's disease to Alzheimer's disease.
    It is also clear that there are serious concerns in the 
scientific community about whether the restrictive rules 
currently imposed by the Bush Administration will allow this 
research to proceed speedily and effectively. These concerns 
range from the number, safety and durability of the existing 
cell lines to whether they will be truly widely available to 
researchers. The memorandum of understanding that Secretary 
Thompson announced this morning specifically prohibits the use 
of the cell lines in clinical research, the research that is 
done to actually test possible treatments for illnesses.
    Millions of patients and their families expect that stem 
cell research will move forward as rapidly as possible. It will 
be unacceptable to offer these patients and families the 
promise of effective stem cell research but deny them the 
reality of it. We will continue to examine the questions raised 
at the hearing, and I am optimistic that Congress will take 
whatever steps are necessary to ensure that stem cell research 
proceeds effectively and ethically.
    The committee stands in recess. Thank you all very much.
    [Additional material follows:]

                          ADDITIONAL MATERIAL

                  Statement of the Alpha-1 Foundation
    Alpha-1 Foundation supports S. 723: Embryonic Stem Cell Research.
    The Alpha-1 Foundation is a national not-for-profit organization 
dedicated to providing the leadership and resources that will result in 
increased research, improved health, worldwide detection and a cure for 
Alpha1-Antitrypsin Deficiency (Alpha-1). Alpha-1 is a 
genetic disorder that results in devastating and fatal lung and liver 
disease. Alpha-1 is a major cause for lung transplantation in adults 
and a leading cause for pediatric liver transplants. Diagnosed patients 
can engage in preventative health measures. Treatment of the lung 
disease associated with Alpha-1 consists of a sole therapy that is 
derived from human plasma, infused weekly and is in critically short 
supply. Individuals with the pulmonary destruction associated with 
Alpha-1 often require supplemental oxygen and suffer the pain known 
only to those unable to catch their breath.
    The Alpha-1 Foundation has joined the Coalition for the Advancement 
of Medical Research whose membership includes voluntary health 
organizations, scientific and academic societies, industry, 
universities, medical organizations, and others to add our voice in 
support of the initiative to reinstate the Federal guidelines for 
embryonic stem cell research. The Foundation supports the NIH 
guidelines in the belief that they were developed with stringent 
oversight and that Federally funded research will allow for this 
research to move forward in an ethical manner.
    Further the Alpha-1 Foundation Medical and Scientific Advisory 
Committee (MASAC) passed a resolution supporting S. 723 because 
embryonic stem cell research may hold great promise in the search for a 
cure for Alpha-1. The list of Alpha 1 Foundation MASAC members and a 
copy of the resolution in support of S. 723 is attached.
alpha-1 foundation medical and scientific advisory committee resolution 
                      regarding stem cell research
    Whereas, the Medical and Scientific Advisory Committee of the 
Alpha-1 Foundation (MASAC) is aware of the many issues surrounding the 
use of stems cells in biomedical research and the debate regarding the 
National Institutes of Health Stem Cell Guidelines, and,
    Whereas, MASAC appreciates the potential role of stem cell research 
in identifying a cure for individuals with the genetic deficiency of 
Alpha1-Antitrypsin,
    Therefore, MASAC resolves the following:
    1. MASAC endorses the current Federal Stem Cell Guidelines as set 
forth by the National Institutes of Health.
    1. MASAC endorses and recommends passage of US Senate bill S. 723, 
sponsored by Senators Specter and Harkin.
    Further, MASAC recommends these resolutions be included in a letter 
addressed to both Senators Specter and Harkin and recommends that 
individuals address letters to their Congressional members requesting 
support of S. 723.
           medical and scientific advisory committee members
    James K. Stoller, M.D., Chair, Cleveland Clinic Foundation
    Mark L. Brantly, M.D., University of Florida College of Medicine
    Manuel G. Cosio, M.D., Royal Victoria Hospital, McGill University
    Frederick deSerres, Ph.D.*, Chapel Hill, NC
    Robert J. Fallat, M.D., California Pacific Medical Center
    Ann R. Knebel, R.N., D.N.Sc., NIH, National Institute of Nursing 
Research
    Joe Reidy*, Waldwick, NJ
    Caroline Riely, M.D., University of Tennessee, Memphis
    Robert M. Senior, M.D., Washington University School of Medicine
    Edwin K. Silverman, M.D., Ph.D., University of Maryland School of 
Medicine
    Gordon L. Snider, M.D., Boston VA Medical Center
    Charlie Strange, M.D., Medical University of South Carolina
    Bruce C. Trapnell, M.D., Children's Hospital and Medical Center
    Gerard M. Turino, M.D., Columbia University College of Physicians & 
Surgeons
    Catherine A. Valenti*, Meridian, ID
    Debbie Waldrop, M.S.N., R.N., C.C.R.C., University of Texas at 
Tyler
    Thomas B. Witt*, Severn, MD
    Bioethicist Consultant, Evan DeRenzo, Ph.D., Center for Ethics/ 
Washington Hospital Center
    Ex-Officio, Robert A. Sandhaus, M.D., PhD, FCCP, Alpha-1 Foundation
    John W. Walsh*, President and CEO, Alpha-1 Foundation
    *Diagnosed with Alpha1-Antitrypsin Deficiency
                        Statement of Don C. Reed
    Dear Senator Kennedy, honorable Committee Members: Seven years ago, 
my son Roman Reed suffered an accident while playing college football. 
His neck was broken; he became paralyzed from the shoulders down. Since 
then, our family has become involved in the struggle to find a cure for 
paralysis. We were fortunate to have a new law passed in California, 
the Roman Reed Spinal Cord Injury Research Act, setting aside a small 
amount of money each year for paralysis research.
    Imagine our joy, therefore, to hear about the amazing possibilities 
of embryonic stem cells. If new nerve cells could be therapeutically 
cloned, and imprinted with Roman's DNA pattern, his own body could 
regrow nerves to heal the damaged spine. Our son might be able to close 
the fingers of his hands again, maybe even rise and walk.
    Unfortunately, despite President Bush's public commitment to allow 
embryonic stem cell research, steps are being taken which will 
effectively kill that research.
    1. The President supports and has promised to sign House Resolution 
2505. Under this terrifying anti-science law, it will be a Federal 
crime to make embryonic stem cells: a felony, punishable by a ten-year 
jail sentence, and a one million dollar fine. H.R. 2505 treats 
therapeutic cloning of cells as if it was the reproductive cloning of 
humans. Obviously, to multiply infant copies of ourselves is wrong, and 
should be illegal. But therapeutic cloning? That is about cure: making 
cells, healing people, saving lives. Comparing therapeutic and 
reproductive cloning is like comparing a surgeon's scalpel to a 
criminal's switchblade. Their purposes are completely different.
    Under H.R. 2505, if a researcher found the answer to cancer, 
paralysis, or AIDS--but cloned just one embryo to make stem cells--he 
or she would have to receive the Nobel Peace Prize in jail.
    2. The President is also shutting off the only other source of 
embryonic stem cells; he will not allow the scientific use of embryos 
left over from fertility procedures. Under the Bush guidelines, no more 
new embryonic stem cell lines can be made. Ever.
    3. The Administration's list of 64 viable stem cell lines is 
neither sufficient nor even accurate. Sweden, for example, is credited 
with seventeen ``robust, vital'' lines. The Swedes made a phone call to 
correct this, stating they have three usable lines, not seventeen. Only 
ten laboratories in the world even have viable embryonic stem cell 
lines. America has four.
    4. Even these extremely few stem cell lines can never be used to 
help people. As is the case with all new science, experimental 
animals--(laboratory mice)--were used to make the lines. Food and Drug 
Administration guidelines on inter-species experiments disqualifies 
them for human cure. If we could use therapeutic cloning, this would 
not be a problem. We could just manufacture some more--but H.R. 2505 
makes that illegal.
    The President's proposal, then, leaves us with nothing but the 
promise of what might have been.
    This decision will hurt every American. For those who suffer 
crippling and life-threateninq diseases now, and the families who watch 
them suffer, our most promising possibility of cure has been denied.
    For sheer financial self-interest alone, the quest for cure must be 
allowed. Our country faces an increasingly unpayable mountain of 
medical debt, public and private. It is overwhelmingly expensive to 
provide longterm hospitalization and attendant care.
    Example: spinal cord injury, which my son Roman has, costs America 
approximately $20 billion a year in medical costs and lost wages. 
That's about $170 per taxpayer, for just one medical condition. And the 
cost in suffering to 450,000 paralyzed Americans and their families? 
That terrible price can never be calculated.
    Why would anyone want to deny cure to the injured and critically 
ill? The problem, conservatives point out, is that when we dissect an 
embryo to obtain stem cells, we are destroying living tissue. That 
near-microscopic dot is technically alive.
    And there is our choice. Like the battlefield medic who decides 
which soldier's life to try and save, because he cannot save them all, 
we too must choose; a 5-7 day old collection of cells in a glass petri 
dish--or a hundred million suffering people.
    Think of folks you know. Like President Ronald Reagan, who has 
Ahlzheimer's disease. Or Michael J. Fox, with Parkinson's. Mary Tyler 
Moore, juvenile diabetes. Magic Johnson, HIV. Elizabeth Montgomery, who 
died of cancer. Vice President Dick Cheney, heart disease. Christopher 
Reeve, spinal cord injury. Annette Funicello, multiple sclerosis. And 
other folks, out of the public eye, like a soldier terribly burned on 
the battlefield and living in continual pain, or my sister Patty, who 
died of leukemia at age 24. Perhaps, God forbid, even someone in your 
own family.
    Whose rights shall we protect--our loved ones, the living people of 
the world--or a dot in a dish, a collection of cells which can neither 
think nor feel?
    Honored committee members, you who will make this momentuous 
decision: do not feel rushed. Give us your best. For in your hands are 
the hopes and dreams of those imprisoned by infirmity, confined to a 
lifetime of wheelchairs and hospital beds, and the endless humiliations 
of helplessness.
    As my paralyzed son Roman puts it: ``Take a stand with us today, in 
favor of research for cure. Take a stand--so 1 day, everybody can.''
    Thank you.
    Don C.Reed is the father of Roman Reed, and the sponsor of 
California's Roman Reed Spinal Cord Injury Research Act.
                   Statement of Christopher C. Straub
    I am the Executive Director of the Culture of Life Foundation, an 
educational foundation dedicated to finding and spreading the 
scientific truths that confirm the dignity and inviolability of every 
human life from the moment of conception until natural death. I am 
submitting testimony today in favor of holding to the course set by 
President Bush in his August 9th statement. In other words, I urge this 
Committee and the Senate to support robust funding for adult stem cells 
and stem cells derived from umbilical cord blood and placentas, and to 
reject any increase in the number or sources of embryonic stem cell 
lines beyond those already authorized by the President.
    Our Foundation was deeply disappointed in the portion of the 
President's August 9th statement which permitted Federal funds to be 
expended on research on stem cell lines which are the fruits of the 
prior killing of human embryos. We also recognize that the President 
drew clear lines of limitation, both in the number of embryonic stem 
cell lines authorized for Federally funded research, and in the 
prohibition on Federally funded embryo destruction.
    In support of maintaining those lines, I will review for you some 
facts not well covered in the news media: the tremendous advances 
already made by researchers and clinicians using adult stem cells. But 
even if adult stem cells were worthless and embryonic stem cells 
guaranteed immortality, I would still urge you to reject the funding of 
research on embryonic stem cells.
    The embryo is a human being, fully equipped with the 46 chromosomes 
of a human being and with the complete genetic data that mark its 
unique humanity. All it needs to develop into a person as apparently 
independent as the rest of us is to be appropriately protected and 
provided with food and oxygen. I will not mention the embryo's size or 
weight or appearance--to reject its humanity on those superficial 
grounds is not a serious argument in this age of science. The argument 
that its humanity is based on its location (e.g., a uterus) rather than 
its nature is likewise absurd; consider whether or not an embryo would 
be human if it were placed in some artificial womb of the future. To 
deny the embryo's humanity based on the location of the embryo is to 
determine its humanity based on the intentions of the person who has 
power over it. In this view, if the embryo is intended to survive and 
grow, then it is human; if it is intended to be killed, then it is not 
human. By this reasoning, there are no human persons on the death rows 
of our penitentiaries.
    The embryo's humanity from fertilization onward is not a matter of 
religious faith, but of reason applied to scientific data. ``The 
genetic information (DNA), which will determine a person's physical 
characteristics and much of his intelligence and personality is present 
at fertilization. Fertilization is the process during which a male 
gamete or sperm . . . unites with a female gamete or oocyte . . . to 
form a single cell called a zygote. This highly specialized, totipotent 
cell marks the beginning of each of us as a unique individual'' 
(William J. Larsen, Essentials in Human Embryology, New York: Churchill 
Livingstone, 1998, p. 17). To determine the embryo human at any point 
subsequent to fertilization is arbitrary, subjective, unscientific, and 
may coincide with the personal interests of those making the 
determination. All the talk about ``treating the embryo with dignity'' 
is nothing more than a nervous tiptoeing around the awesome and simple 
truth of its human personhood.
    To obtain stem cells from an embryo, the embryo--this person--must 
be killed. Killing innocent people is considered evil in Judeo-
Christian morality and in the laws that derive from that morality, the 
laws by which we govern ourselves. Throughout human history, it has 
been demonstrated repeatedly that we humans cannot obtain a good 
outcome from an evil action. This rule operates with mathematical 
certitude. That is why embryonic stem cell research is objectionable 
and why tax dollars should not be used to pay for it.
    The President, of course, did not authorize the killing of any 
embryos, and I am grateful for that. He did authorize Federal funding 
for research on 64 stem cell lines derived from embryos that had 
previously been killed, and that was a mistake on his part. I ask you 
to imagine you are a medical researcher in Germany sixty years ago and 
the German Army contracts with your laboratory to research a diet and 
medications to help soldiers resist extremes of cold. The data you are 
given to work with comes from experiments on condemned prisoners who 
were forced to participate and who in most cases froze to death in the 
course of the experiment. If you know the source of the data, it is 
wrong for you to work on this contract. This is the situation with the 
stem cell lines upon which Federally funded research is permitted: 
although the killing was done elsewhere, by another, it is wrong to 
seek the fruits of it. That is the basis of our Foundation's objection.
    The work on the 64 stem cell lines is also the seed of more 
killing, as scientists State their requirement for more and better 
embryonic stem cells than those authorized by the Administration. The 
authorized cell lines may not be sufficient. Many of them may be 
tainted by contact with feeder cells from mice. More work reportedly 
remains to be done on some of the cell lines to be sure of their 
viability. There may be fewer than 64 viable lines among those the 
Administration has authorized; the August 29 New York Times reported 
that a hospital in Sweden, which the Administration asserts has 
nineteen lines, by its own account has only three established lines, 
four more that are being studied, and twelve that are in early stages. 
In addition, the ownership of patents and rights to the process by 
which the cells were derived and developed is not completely clear, and 
none of the corporate and academic laboratories seem disposed at this 
point to imitate Dr. Jonas Salk's generosity with his epochal 
discovery.
    If more cell lines are demanded, the in vitro fertilization (IVF) 
industry reportedly has an overstock of at least 100,000 frozen embryos 
that were supposedly a rich source for stem cell research. But that 
source may have been a mirage: the August 26 New York Times reported 
that most parents of frozen embryos created in the IVF process do not 
want to contribute their embryos to science. The only other source may 
therefore be embryos specifically created to be destroyed in research. 
Many of the lines President Bush has authorized for federally funded 
research are probably in this category, and laboratories like the Jones 
Institute for Reproductive Medicine are ready to make many more. 
Against this possibility I again recall to you the human personhood of 
every embryo, even one destined for destruction within days of 
creation. No potential good can justify such a killing.
    With the destruction of embryos for medical research we see the 
division of humankind into two parts: those who are destined to be 
killed for the benefit of others, and those who benefit from the death 
of another. This is as profound an inequality as has ever existed in 
human history.
    Meanwhile, research on stem cells from postnatal human tissue and 
from umbilical cords and placentas continues to make spectacular 
breakthroughs in the treatment of human patients as well as laboratory 
animals. With exception of the news this summer that adult stem cells 
reside in one of our Nation's most bountiful resources, human fat, 
there has been little coverage of the spectacular breakthroughs made 
using adult stem cells against some of the very conditions and diseases 
which are used to justify the killing of embryos. Our foundation tracks 
these discoveries and issues a report each week, and I submit a list of 
those discoveries. You should know that adult stem cells have already 
been used to create knee cartilage, heart muscle, liver tissue, kidney 
cells, and bone mass in real human patients. Adult stem cells have been 
used to grow restorative tissue around spinal cord injuries, to restore 
vision, and to help people suffering from diabetes to become insulin-
free. Adult stem cells have been used to treat Crohn's disease, to 
overcome immune deficiency in children, to restore bone marrow in 
people with leukemia, and to fight a wide variety of cancers. Fresh 
successes are reported almost daily. For example, the August edition of 
Archives of Dermatology reported that doctors at the M.D. Anderson 
Cancer Center in Houston, Texas, used adult stem cells to cure a man 
with rare but devastating skin disease, scleromyxedema. This disease 
had stiffened and thickened the patient's skin to the point he could 
not close his eyelids or eat. His doctors collected adult stem cells 
from his own bone marrow, then destroyed his immune system with 
chemotherapy and transplanted his stem cells back into his body to 
permit them to rebuild his immune system. As a result, the 
scleromyxedema is now in remission.
    There are also spectacular nonstem cell treatments being developed 
to fight the diseases and disorders most often cited as a rationale for 
embryonic stem cell research. Against Alzheimer's, for example, a 
university in Tokyo has discovered a protein with the potential to 
prevent or reverse the disease, and researchers in Dublin and at New 
York University have separately discovered what may be an Alzheimer's 
vaccine. Against cancer, nonstem cell developments include new toxins 
to be directed against cancer cells, using red blood cells guided by 
ultrasound to deliver anti-cancer drugs directly to tumors, and 
developing antibodies to deliver lethal radiation to a tumor.
    Adult stem cells have another advantage: when taken from the 
patient the possibility of tissue rejection is greatly reduced. For 
this reason, many Americans are now banking their infant's umbilical 
cords.
    The performance and the promise of adult stem cells are so great, 
and the news blackout and scant attention paid them by elite opinion is 
so marked, that there may be some motive at work besides concern for 
health. Whether it is a desire to derive a supposed benefit from the 
death of an embryo, the attraction of huge grants and concomitant fame, 
or the urge to smash another traditional human taboo, the embryonic 
stem cell is supposedly the new hope of mankind and the adult stem 
cell, which has already accomplished much more, is ignored. Unless we 
wake up to the facts and respond to them by reinforcing the success of 
adult stem cells, we will delay the day we conquer some of the cruelest 
diseases and conditions that afflict us, while at the same time 
becoming a nation that kills out of utilitarian motives.
              Statement of the Culture of Life Foundation
                                diabetes
Adult Stem Cell Research
     Adult Stem Cells Cure Type I Diabetes in Mice: Dr. Denise 
Faustman of Harvard University Medical School has discovered an 
exciting potential new therapy that could possibly cure type I 
diabetics. The treatment effectively cured diabetes in mice, and human 
trials are currently being set up. Patients with type I diabetes have a 
defect in their immune system wherein their white blood cells (WBC's) 
inappropriately attack their own ``pancreatic islet cells'' in their 
pancreas. These cells are the primary producers of insulin for the 
body. Dr. Faustman's experiments entailed killing the defective WBC's 
in mice with diabetes by giving them certain drugs. She then planned to 
somehow ``reprogram'' the new WBC's that would be produced, so that 
they wouldn't attack the pancreatic islet cells again, and then to 
transplant new islet cells into the mice. However, she found that there 
was no need to do so. After the defective WBC's were gone, adult stem 
cells in the mice ``took over,'' and started producing new pancreatic 
islet cells within 40 days. If the human clinical trials prove 
effective, this treatment would be helpful for not only diabetics, but 
also for other diseases that involve similar ``autoimmune'' symptoms, 
like lupus, rheumatoid arthritis, multiple sclerosis, and more than 50 
other illnesses. All without need of embryonic stem cells.
    Source: Cromie, William; ``Adult Stem Cells Effect a Cure: Diabetes 
cure may eliminate need for embryo cells''; Harvard University Gazette, 
July 19, 2001, at http://www.hno.harvard.edu/gazette/index.html/01-
stemcells.html.
Non-ES/Fetal Cell Research
     Formation of Insulin-Producing Pancreatic Islet Cells from 
Adult Pancreatic Tissue: A major goal with scientists studying diabetes 
is to come up with a good source of pancreatic cells that produce 
insulin. One of the primary insulin-producing cells in pancreases are 
``islet cells'', which secrete insulin in response to glucose 
stimulation. In patients with type I and type 2 diabetes, a transplant 
of such cells would be tremendously helpful, since not nearly enough 
cadaver pancreas transplants for such patients become available on 
time. In July 2000, scientists at Harvard Medical School announced that 
they had found that by specially-preparing and nurturing adult 
pancreatic ductal tissue (which normally does not produce much 
insulin), they were able to coax the ductal cells to form pancreatic 
islet cells. These pancreatic islet cells were then further found to 
produce insulin, with increased amounts produced when stimulated with 
glucose. If this method of pancreatic cell differentiation and growth 
can be optimized to produce large amounts of cells, it could mean an 
important new type of cell replacement therapy for type 1 and type 2 
diabetics.
    Source: Bonner-Weir, et al.; ``In vitro cultivation of human islets 
from expanded ductal tissue''; Proceedings of the National Academy of 
Sciences USA; July 5, 2000; 97 (410); 7999-8004.
     Insulin-Producing Pig Cells Are Helping Diabetic Baboons: 
Scientists at Duke University have demonstrated that specially-coated, 
insulin-producing pancreas cells from pigs are keeping a diabetic 
baboon off of insulin. The pig pancreas cells were encapsulated within 
a complex carbohydrate known as alginate. This coating had pores that 
were large enough to allow insulin to be released, but small enough 
that the baboon's immune system antibodies and cells were not triggered 
to give a noticeable response to the foreign cells. The encapsulated 
pig cells were injected into the baboon's peritoneal cavity. The baboon 
has not needed extra insulin for 9 months, since the injection. With 
the success of this treatment, it is estimated by the researchers that 
human clinical trials could begin within the year. Some organizations 
dedicated to the treatment of diabetes are supporters of embryonic stem 
(ES) cell research. However, there have been no such successful studies 
to date using ES cells to treat diabetes; likewise, no ES cell studies 
have shown such promise or possibility to move to human clinical trials 
so quickly.
    More information can be found at: http://www.dukenews.duke.edu/Med/
baboon.htm. (From 6/15/01 Duke University News Release)
     Fat Cell Hormone Controls Diabetes: A hormone naturally 
produced by fat cells shows promise for treating diabetes. The hormone, 
called adiponectin, was recently found to be able to reduce the 
severity of diabetes in mice. It was able to normalize elevated levels 
of glucose in mice, and was also able to regulate the production of 
glucose by rat liver cells. Both of these abilities could help to treat 
diabetes in humans. One biotech company, Genset in Paris, France, is so 
encouraged by the findings that they are already planning clinical 
trials in humans using the hormone. There is so much potential with 
such nonembryonic/nonfetal research, that there is no need to delve 
into research on diabetes using human embryonic stem cells.
    Sources: Science http://www.sciencemag.org/ (need subscription); 
article entitled ``Hormone Keeps Diabetes in Check'' by Josh Chamot in 
ScienceNow section of journal); also Berg, Anders et al.; ``The 
adipocyte-secreted protein Acrp30 enhances hepatic insulin action''; 
Nature Medicine; August 2001; Vol. 7, No. 8; pp. 947-953.
   alzheimer's disease/parkinson's disease/brain & spinal cord injury
Adult Stem Cell Research
     Neural cells isolated from adult cadaver human brains were 
found to be able to grow into different types of neural cells, 
including neurons, astrocytes, and oligodendrocytes. (Palmer, et al.; 
``Cell culture: Progenitor cells from human brain after death''; 
Nature; May 3, 2001 (411); 42-43.)
     Human umbilical cord blood treated with growth factors 
express markers that indicate that they can give rise to neurons. 
(University of South Florida Health Science Center Press Release, May 
9, 2001 and February 18, 2001; also presented at May 6, 2001 American 
Academy of Neurology Meeting)
Adult Neural Stem Cells Purified From Mouse Brain Can Differentiate
     Scientists in Australia report in this week's journal 
Nature that they were able to purify adult stem cells from mouse brain. 
This is normally somewhat difficult, since it has been estimated that 
stem cells number only 1 for every 300 cells in the adult brain. But 
they were able to do so via modem cell separation techniques, and have 
also been able to stimulate their collected neural stem cells to 
differentiate into muscle cells by growing them together with other 
muscle cells. If it is found that human neural stem cells can do the 
same, such adult stem cells could possibly be used to treat spinal cord 
or muscle (including cardiac) injury and many brain diseases (such as 
Alzheimer's or Parkinson's), without the use of a single embryonic stem 
cell.
    Source: Rietze, Rodney, et al.; ``Purification of a pluripotent 
neural stem cell from the adult mouse brain''; Nature; Vol. 412, Aug. 
16, 2001; pp. 736-739; also Pearson, Helen; ``No stemming the tide''; 
Nature; Vol. 412, Aug. 16, 2001.
Adult Skin Stem Cells Can Turn Into Brain Tissue
     Researchers at McGill University in Montreal, Canada have 
found that they can repeatably isolate adult stem cells from the skin 
of mice. Repeatability is a necessary objective for researchers with 
stem cells, and so is versatility--the ability to become more than one 
type of cell. These skin stem cells, called SKPs (for SKin Precursor 
cells) have both going for them. Over and over, the scientists were 
able to coax the isolated stem cells to form other types of cells, from 
neurons to muscle cells to fat cells. The scientists' studies with 
adult human scalp tissue have found that similar stem cells can be 
found in human epidermis. This is extremely encouraging, both from an 
ethical point of view and from an immunological point of view. If taken 
from a patient's own skin, SKP adult stem cells would likely provoke 
little or no immunological response, and would not necessitate the use 
of strong drugs to suppress the patient's immune system. In contrast, 
the use of embryonic or fetal stem cells would both be immoral and 
would likely necessitate the use of such drugs. SKP stem cells appear 
to have much potential for use in diseases or disorders of the central 
nervous system.
    Sources: Toma, Jean, et al.; ``Isolation of multipotent adult stem 
cells from the dermis of mammalian skin;'' pp. 778-784; Sept. 2001, 
Vol. 3, issue 9 (published online on 13 Aug. 2001), also http://
www.washtimes.com/national/20010814-73539816.htm (8/14/01 Washington 
Times article by Carolyn Abraham entitled, ``Stem Cells from Skin Grow 
Into Brain Tissue''); also, http://www.sciencedaily.com/releases/2001/
08/010814063557.htm (ScienceDaily 8/14/01 report) and http://
www.mcgill.ca/public/releases/2001/august/stemcells/ (McGill University 
8/13/01 Media Release)
Other Non-ES/Fetal Cell Research
     At Keio University in Tokyo, scientists have discovered a 
protein that stops the death of brain cells that occurs in Alzheimer's 
patients. The findings were reported in the May 22, 2001 edition of the 
Proceedings of the National Academy of Sciences. This protein, called 
humanin by its discoverers, may not only prevent further deterioration 
from Alzheimer's, but could be the first step in curing the disease. 
Alzheimer's is one of the diseases most often mentioned in 
justifications of embryonic stem cell research.
     NYU Researchers Design Successful Vaccine Against 
Alzheimer's Disease: Scientists at New York University have created a 
vaccine against Alzheimer's disease which has proven effective in mouse 
studies at removing amyloid plaques in the brain. These plaques are 
aggregated clumps of the protein amyloid in the brains of Alzheimer's 
patients, which are thought to contribute to the characteristic memory 
loss, confusion, and mood disturbances in such patients. Like the 
recent report of an Alzheimer's vaccine produced by the Elan 
Corporation in Dublin (Adult Stem Cell Research Weekly Highlights #3) 
this vaccine is also made of a synthetic protein. However, the NYU 
group believes that their vaccine has less chance of causing later 
potential plaque formation than the vaccine from the Dublin group. 
Their experimental results were very promising in mice with plaques: 7 
months after injection with the vaccine, a bad form of the amyloid was 
reduced in their brains by 89 percent in the cortex (the seat of higher 
thought), and by 81 percent in the hippocampus (the brains memory 
center), compared to the control, nonvaccinated groups. In addition, 
the vaccinated mice had 57 percent less soluble amyloid present. This 
is yet another promising discovery in treatment options for Alzheimer's 
disease which does not resort to utilizing embryonic or fetal stem 
cells.
    Source: http://www.eurekalert.org/ (from 8/2/01 report)
     Alzheimer's Drug Being Used to Treat Brain Injury: The 
University of Florida and 14 other centers will soon participate in 
clinical trials testing a drug, donepezil (more commonly known by the 
trade name Aricept, and manufactured by Eisai Co. Ltd.), in patients 
with brain trauma. The drug is currently used to slow memory decline in 
Alzheimer's disease. It is thought that because patients with brain 
trauma often have trouble with memory and cognition as with Alzheimer's 
disease, an effective treatment for Alzheimer's could be helpful for 
treating brain injury. Likewise, people that have received brain trauma 
have been found to be more likely to develop Alzheimer's later in life. 
Thus, a treatment that alleviates the symptoms of brain trauma could 
also have the effect of stemming the onset of Alzheimer's. This is the 
first randomized clinical trial ever conducted that treats chronic 
traumatic brain injury. It does not necessitate the usage of embryonic 
or fetal stem cells.
    Source: http://unisci.com/stories/20013/0801012.htm (from 8/l/01 
report)
     Manipulation of a Single Gene in Neurons Can Lead to 
Profuse Cell Regeneration: Could Lead to New Treatments for Brain and 
Spinal Cord Patients: A July 1, 2001 News Release from the National 
Institutes of Health/National Institute of Neurological Disorders and 
Stroke focuses on the recent work of researcher Maureen L. Condic, 
Ph.D. (University of Utah School of Medicine in Salt Lake City). She 
has shown that by genetically modifying adult rat neurons to produce 
more of a protein called integrin, a dramatic increase in nerve fiber 
growth could be achieved. The amount of growth was more than 10 times 
greater than in any other published study of adult neuron regeneration, 
and was indistinguishable from neuron growth in newborn adult animals. 
Previous studies had primarily focused on somehow changing the 
environment around neurons to stimulate them to grow, but this study 
shows that a key to neuronal growth may be as simple as changing the 
performance of one gene. This could lead to better treatment strategies 
(and nonembryonic stem cell research ones) for brain and spinal cord 
injury, as well as for Parkinson's and Alzheimer's diseases.
    Sources: July 1, 2001 News Release from the National Institutes of 
Health/National Institute of Neurological Disorders and Stroke 
(original source) and http://www.sciencedaily.com/releases/2001/07/
010702084939.htm
     Anti-Alzheimer's Antibody Gives Clues to Why it Decreases 
Development of Plagues in Brain: Researchers at Washington University 
School of Medicine studying the function of an anti-Alzheimer's 
antibody have found that the antibody draws a protein called amyloid-
beta out of the brain in mice and diverts it to the bloodstream. 
Amyloid-beta contributes to the formation of ``amyloid plaques'' in the 
brains of Alzheimer's disease patients. Scientist David M. Holtzman, 
M.D. says, ``Within hours of injecting the antibody into mice, the 
concentration of amyloid-beta in the bloodstream rose approximately 
1,000 times higher than it had been before the injection.'' In 
addition, animals injected with the antibody over a period of months 
developed less amyloid plaques than control animals did. The difference 
between the groups was statistically significant. Utilization of such 
an anti-Alzheimer's antibody could be a useful and nonmorally 
problematic therapy for Alzheimer's patients.
    Sources: News Release from Washington University School of Medicine 
(original source) and http://www.sciencedaily.com/releases/2001/07/
010704093453.htm (7/3/01)
     New Hope in Spinal Cord Treatment Utilizing Patients' Own 
White Blood Cells: Doctors at Proneuron Biotechnologies in Tel Aviv, 
Israel, are using a promising new technique to treat spinal cord 
injuries. They inject a patient's own white blood cells directly into 
their spinal cord, after activating the cells by incubating them with 
skin cells from the patient's body. So far, results are encouraging. A 
19-year old girl who was paralyzed from her chest down by a car 
accident was treated with the procedure, and a year later, has regained 
pain sensation in one of her legs, can contract some leg muscles, and 
can curl her toes. It's not clear right now whether the activated white 
blood cells are promoting regrowth of the neurons or are simply keeping 
more neurons from dying. The treatment tries to utilize the knowledge 
that white blood cells are known to usually play a big role in the 
healing of injuries. Normally, however, white blood cells do not heal 
injuries to the spinal cord, because of the ``blood-brain barrier.'' 
This is a term for the tight cell junctions in the blood vessels in the 
brain and spinal cord areas, which isolate the brain and spinal cord 
from the blood cells and potential toxins in the blood. With the new 
procedure, the doctors get past this barrier by injecting the cells 
directly into the spinal column. Three other people have been treated 
by Proneuron with this and other new techniques for paralyzed patients, 
including a 19-year-old man from Colorado who was injured while using a 
snowboard. The procedure described here can only be performed within 14 
days of the spinal cord injury, and Proneuron is seeking more patients 
from around the world that are interested in trying the procedure. They 
are willing to pick up transportation and living expenses, and the cost 
of the treatment itself for the patient. This type of treatment is 
especially significant in light of the fact that people who advocate 
embryonic stem cell research believe that spinal cord injuries can be 
helped by such research. This white blood cell procedure (also known as 
``autologous macrophage procedure'') is already showing promise in 
humans, and does not face the ethical or immunological problems that 
would come from human embryonic stem cell or fetal cell research.
    Source:http://www.cnn.com/2001/HEALTH/conditions/07/20/
spinal.cord.injuries.ap/index.html (from 7/20/01 report); also 
www.proneuron.com
     Alzheimer's Vaccine Holds Promise and Appears Safe in 
Human Tests: Scientists at the Elan Corporation, a pharmaceutical 
company headquartered in Dublin, have found that a vaccine they have 
been testing to treat Alzheimer's disease has shown promise in mice and 
appears safe in humans. The vaccine, called AN-1792, is made of a 
synthetic protein which is a form of the protein beta amyloid that is 
found in the characteristic ``plaques'' (protein aggregates) in the 
brains of Alzheimer's patients. These plaques are thought to be what 
causes the memory loss and behavior changes which are symptoms of 
Alzheimer's disease. In mouse experiments, the synthetic protein 
vaccine was injected into mice that had plaques (i.e., they were a 
mouse ``model'' of the human disease). Two groups of mice were studied 
after such immunization: older mice that had already formed brain 
plaques and younger mice that had not yet formed plaques. With the 
older mice, it was found that there had been a significant curbing of 
plaque formation. With the younger mice, it was found that the 
immunization had kept virtually all of the mice from even developing 
plaques. It is thought that the vaccine somehow clears plaques out of 
the brain. In more recent testing of the vaccine in humans to determine 
its safety, it was found that the vaccine appeared to have no 
significant adverse effects. In addition, the patients had developed an 
immune response to it, which is what should happen if the protein is 
having any effect in their bodies. Although the patients did not 
exhibit any cognitive or memory improvements, the scientists hope that 
the next phase of upcoming human trials, which test for vaccine 
efficacy, will show positive results as did the mice experiments. This 
research appears to be much safer than the research reported in the 
March 8, 2001 New England Journal of Medicine involving fetal and 
possibly embryonic cells injected into the brains of Parkinson's 
patients (another neural disease). That treatment had disastrous 
results, such as wild, aberrant movements in the patients. In addition, 
the research involving this vaccine is an example of one of the moral 
research routes that can be taken as an alternative to research 
involving the destruction of embryonic human beings.
    Sources: http://www.cnn.com/2000/HEALTH/aging/07/11/
alzheimer.vaccine/ (from 7/11/01 report); also http://www.cnn.com/2001/
HEALTH/07/23/alzheimers.vaccine/index.html (from 7/23/01 report); also 
http://www.elan.ie/alzheimer/pressrelease.asp; also http://www.elan.ie/
alzheimer/research.asp
Promising New Treatment for Partial Spinal Cord Injury
     Researchers at the Weizmann Institute of Science in Israel 
are experimenting with a new therapy intended for patients with partial 
spinal cord injury. With partial spinal cord injury, the several days 
immediately following the injury are crucial, because during this time, 
there is usually further neural damage that occurs in a wave that 
spreads from the injury site, and can be even more destructive than the 
initial damage. The goal of the researchers is to find a treatment that 
will effectively stop this postinjury degeneration. In their recent 
studies on rats with partial spinal cord injury, they injected the rats 
postinjury with specific proteins which they had derived from the 
central nervous system (and which were selected to be able to boost the 
rats' immune system). The treated rats showed significant recovery of 
movement, and their spinal cords contained nerve fibers that appeared 
substantially healthier than those of nontreated rats (which suggests 
that the proteins halted further neural degeneration). These results 
are very encouraging, and counteract (with hard data) the hype from 
pro-embryonic stem (ES) cell research organizations that ES cells are 
needed for healing of spinal cord injury. This nonES therapy is only 
one of several we've reported on in the last few weeks which treat 
neural injury and disease in a completely moral manner.
    Sources:http://wis-wander.weizmann.ac.il/weizmann/doa iis.dll/
Serve/item/English/1.200.7.5.html and http://www.sciencedaily.com/
releases/2001/08/010817081453.htm (8/17/01 report)
                        heart disease and repair
Adult Stem Cell Research
     Bone marrow cells were found to regenerate heart cells 
after induced heart attack-type damage in mice. (Orlic, et al.; ``Bone 
marrow cells regenerate infarcted myocardium''; Nature; April 5, 2001 
(410); 701-705.)
     The June 7 edition of the NEJM also reported on research 
that found that human heart muscle cells regenerate after a heart 
attack. These researchers, at the National Institutes of Health and New 
York Medical College, now seek to determine why heart muscle cells are 
dividing. One hypothesis is that these cells originate from stem cells 
present in the heart. Members of the research team had already shown in 
mice that adult stem cells from bone can become functioning heart 
muscle.
     A team at University of California at Los Angeles 
announced in May of this year that they had injected adult stem cells 
from a heart attack victim's bicep to replace heart muscle tissue 
damaged in a heart attack. Separately, also in May, a team at Baylor 
University announced their discovery that stem cells from the bone 
marrow of an adult mouse had the capability to transform into blood 
vessels and cardiac muscle.
Other Non-ES/Fetal Cell Research
     Newly-Discovered Heart Cell Protein Could be Used in 
Cardiac Treatment or for Conversion of Other Cells to Cardiac Cells: In 
the June 29, 2001 issue of Cell, researchers at the UT Southwestern 
Medical Center at Dallas report that they have discovered a protein 
called myocardin in heart cells which is responsible for turning on 
cardiac genes. Dr. Eric Olson, chairman of molecular biology, says, 
``The Holy Grail in the heart field is finding the gene or genes that 
can convert noncardiac cells into cardiac cells.'' The scientists 
studied the function of the protein in frog embryos and found that when 
myocardin is absent, the heart does not develop. It is possible that 
the gene that controls myocardin is a type of ``master gene'' for the 
heart. If so, then it could bode well for all sorts of possible heart 
treatments, such as changing nonheart cells into heart cells (an 
alternative to embryonic stem cells), or utilization in heart repair 
therapies in all ages (babies born with heart defects to adults with 
heart defects or heart attacks).
    Sources: Wang, Da-Zhi, et al.; ``Activation of Cardiac Gene 
Expression by Myocardin, a Transcriptional Cofactor for Serum Response 
Factor''; Cell, Vol. 105, 851-862, June 29, 2001; also, http://
unisci.com/stories/20012/0629013.htm
Heart Cells Divide, Enlarge, and Live Longer With Special Enzyme
     Scientists at Baylor College of Medicine in Houston have 
found a way to increase heart cell division, increase heart cell size, 
and make heart cells live longer. This is great news for people prone 
to heart attacks or other heart ailments. The researchers genetically 
engineered their laboratory mice to produce a specific enzyme called 
TERT (Telomerase Reverse Transcriptase) in their heart muscles. This 
enzyme is normally only produced in very young mice, and is involved in 
the process of chromosome duplication during cell division. The 
engineered mice continued to produce the enzyme even after they grew 
older. At first, the mice produced many more heart cells than normal 
mice. Several weeks later, cell division ceased and the cells grew 
larger (hypertrophy). In addition, the cells appeared to be more 
resistant to cell death than normal cells. Usually, the condition of 
hypertrophy confers a weakness to the heart. With these mice, however, 
their hearts did not have impaired functioning. One of the scientists 
in the group, Dr. Michael Schneider, said that the scientists involved 
believe that adding TERT to an adult heart could offer protection 
against the type of cell death experienced during a heart attack. In 
addition, TERT could possibly be added to cells that have been grafted 
onto an injured heart. This could probably aid in cell growth and 
protection from cell death. It is just one more example of the exciting 
research being done without using embryonic stem cells.
    Source: http://www.sciencedaily.com/releases/2001/08/
010821074944.htm (8/22/01 report) and Oh, et al., ``Telomerase reverse 
transcriptase promotes cardiac muscle cell proliferation, hypertrophy, 
and survival'', Proceedings of the National Academy of Sciences, 
published online August 21, 2001 www.pnas.org/cgi/doi/10.1073/
pnas.191169098 [Need subscription].
                      kidney disease/renal failure
Adult Stem Cell Research
     Bone Marrow Cells Can Become Kidney Cells: Scientists at 
the Imperial Cancer Research Fund and Imperial College School of 
Medicine in the UK have recently discovered that bone marrow stem cells 
(which are adult stem cells) can change into kidney cells in both mice 
and humans. For the mice that they studied, they examined the kidneys 
of female mice which had been given bone marrow transplants from male 
mouse bone marrow. For the humans, they analyzed kidneys of male 
patients who had been given transplants of female kidneys. The 
researchers utilized a special DNA probe technique on the kidneys, 
which can detect if a cell originated from a male or female. For both 
groups, it was found that the mouse and the human kidneys contained 
male kidney cells as well as female kidney cells. It was determined 
that these male cells had come from the mouse's or patient's own bone 
marrow, had implanted into the new kidneys, and had literally become 
kidney cells. This finding opens up many promising possibilities. 
People whose kidneys are damaged or failing because of disease or 
injury could be treated with their own bone marrow cells to form new 
kidney cells and possibly whole new kidneys (which would help solve the 
desperate current shortage of kidney transplants). This is especially 
beneficial for the realm of kidney treatment, because as organs, they 
are particularly bad at repairing themselves. Also, there would be less 
immune rejection, because the repairing cells would be coming from the 
patients' own bone marrow. Professor Nick Wright, one of the authors of 
the paper in the Journal of Pathology Online which describes the 
experiments, says that an additional possibility using this knowledge 
could be to genetically engineer bone marrow stem cells to contain 
genes ``resistant to cancer or other disease, to protect the kidneys 
from further damage.'' Says Dr. Poulsom, lead author on the paper, 
``The potential for advances in medicine from using adult stem cells is 
enormous. They can give rise to many different types of cells so any 
organ may 1 day be repaired. Using adult stem cells also avoids the 
ethical dilemmas associated with embryonic stem cell work.''
    Sources: Poulsom, et al., Journal of Pathology Online; ``Bone 
marrow contributes to renal parenchymal turnover and regeneration''; 25 
July 2001; also Pro-Life Infonet 7/25/01 #2486 (contact: 
[email protected]); also http://unisci.com/stories/20013/
0725011.htm (from 7/25/01 report)
                               hemophilia
Non-ES/Fetal Cell Research
     Research showing that the genetic modification of cells 
could fight hemophilia, announced in the June 7, 2001 edition of the 
New England Journal of Medicine (NEJM), used adult cells taken from the 
skin of the patients' arms. The cells were then modified to add the 
gene for a missing clotting factor, grown in the laboratory, and 
injected in to the patient.
                                 cancer
Placental Stem Cell Research
     Placental blood was found to be a useful source of stem 
cells for bone marrow reconstitution in patients with leukemia and 
other diseases. (Rubinstein, et al.; ``Outcomes among 562 recipients of 
placental-blood transplants from unrelated donors''; New England 
Journal of Medicine; November 26, 1998; 339 (22); 1565-1577.)
Non-ES/Fetal Cell Research
     Cancer Treatment Made Better Through ``Sticky'' 
Antibodies: A team of scientists at the University of California, 
Davis, has developed a new method for altering antibodies for targeting 
the destruction of tumors. Antibodies are molecules within the body's 
immune system which normally hone in on foreign elements in the body, 
to fight infections. It has been found that they can also be directed 
to fight cancerous tumors. When they are connected with a radioactive 
element (which is joined to a small carrier molecule), the special 
antibodies not only hone in on the tumor, but also deliver lethal 
radiation to the tumor. Radiation is a commonly-used therapy to fight 
cancer. When used in this way, the radiation goes straight to the 
cancerous cells, rather than to other parts of the body as well (which 
is a frequent side-effect of usual radiation therapy). Previous studies 
have combined the antibody-radiation complex in a rather temporary 
manner, using methods that unfortunately allow the radiation carrier 
molecule to unhinge from the antibody. The scientists at UC Davis have 
come up with a new method which engineers the binding region of the 
antibody to adhere permanently to the radiation carrier molecule. In 
effect, the antibody-radiation juncture is ``stickier''. This should 
allow for a cancer treatment regimen that is more precise, delivering 
the radiation directly to the tumor site without potential radiation 
leakage in other parts of the body. This is yet another cancer therapy 
which does not utilize embryonic stem cells (which have been touted as 
being a possibility for use in cancer research).
    Source: Chmura, et al.; ``Antibodies with infinite affinity;'' 
Proc. Natl. Acad. Sci. USA, Vol. 98, Issue 15, 84808484, July 17, 2001; 
also, http://unisci.com/stories/20013/0717015.htm
     Drug Capable of Destroying Only Oxygen-Starved Cells 
Within Tumors: Researchers at the University of Ulster in the UK have 
developed a promising new drug for cancer treatment. It is different 
from other current drugs on the market in that it targets hypoxic 
cancerous cells, which are cells deep within tumors that are not 
receiving much oxygen. These types of cells have proven to be 
particularly difficult to treat with the normal range of cancer drugs 
and radiation therapy, which are generally only good at killing cancer 
cells on the exterior of a tumor (and which do receive oxygen). The 
hypoxic cancerous cells within tumors are also known to be especially 
malignant, and have been found to be one of the prime instigators of 
secondary bouts of cancer, after the external tumor cells have been 
killed off. The new drug, called AQ4N, is also different from standard 
chemotherapy drugs, in that when administered, it is not toxic to the 
body. It only becomes toxic when it reaches the tumor cells deep inside 
the tumor, and is only toxic to those particular cells. This would 
avoid the common side-effect of the current chemotherapy drugs to kill 
other, noncancerous cells in the body (in doing so, they cause hair 
loss and extreme nausea to cancer patients). It is thought that perhaps 
this new drug can eventually be used in combination with current modes 
of drug and radiation therapy to be an effective cancer treatment 
regimen. The development of drugs such as this is a moral and effective 
alternative to cancer research involving embryonic stem cells.
    Source: http://unisci.com/stories/20013/0719012.htm
     Making Deadly Acute Myeloid Leukemia (AML) Treatable: 
Scientists at Johns Hopkins University have found that drug called 
AG1295 can treat acute myeloid leukemia (AML), which is the most common 
form of adult leukemia. At least 40% of patients with AML have a 
genetic mutation wherein the gene called ``FLT3'' is altered. This gene 
is normally involved in the growth and maturation of blood cells, and 
when it is altered, uncontrolled division (causing leukemia) can occur. 
The researchers discovered that the drug AG1295 specifically interferes 
with the action of the altered FLT3 protein, blocking the uncontrolled 
growth and causing the leukemia cells to die. Safety and efficacy 
trials in animals and humans will need to be done, but it is a big step 
that the investigators have been able to pin down the genetic 
abnormalities related to this type of leukemia. This is good news, and 
another exciting prospect that doesn't involve embryonic stem cell 
research.
    Sources: http://unisci.com/stories/20013/0727012.htm (from 7/27/01 
report) and http://www.sciencedaily.com/releases/2001/07/
010726102007.htm (from 7/26/01 report)
     Teaching the Immune System to Fight Cancer and Other 
Diseases: Researchers at Oxford University's Department of Clinical 
Medicine have developed a modified adenovirus that can teach the body's 
immune system how to fight tumors, malaria, and hepatitis. The vaccine-
type method uses two sequential types of immunization. The first, 
called the ``prime,'' introduces bits of the tumor or pathogen into the 
body. This allows the body to be able to identify and recognize the 
antigen. Later, a second immunization, called the ``boost,'' involves 
injecting a modified adenovirus. This adenovirus elicits a larger 
immune response which includes production of special ``CD8+ killer T 
cells'' by the body. Because of the first ``prime'' injection, these 
killer T-cells are specifically made to hone in on the tumor or 
pathogen. Clinical trials of such vaccines are currently being started 
by Oxford's spinout company, Oxxon Pharmaccines. Such research is 
encouraging and is yet another type of cancer research that doesn't 
involve human embryonic stem cells or fetal cells.
    Source: http://unisci.com/stories/20013/0724011.htm (from 7/24/01 
report)
     Red Blood Cells Can Deliver Anti-Cancer Drugs With Great 
Accuracy: A scientist at the University of Ulster, Professor Tony 
McHale, has developed an ingenious way to deliver drugs to target 
tumors with precision. He uses normal red blood cells (RBC's). RBC's, 
which routinely carry oxygen throughout the body, have been used in 
past studies in an attempt to deliver drugs throughout the body before, 
without much success. This is because it is difficult to get the cells 
to deliver the drugs to the exact point in the body that one is 
interested in targeting. Professor McHale, however, has discovered a 
way to get around this. He found a way to sensitize red blood cells so 
that they will explode when they come in contact with an ultrasound 
beam. After sensitizing the RBC's and loading them up with the desired 
drug, doctors focus an ultrasound beam on the targeted tumor. Once the 
RBC's reach this area, they explode, delivering the drugs in a precise 
manner. In this way, surrounding tissue is left unaffected, which is 
also a key benefit to his procedure, since most current cancer drugs 
have a way of affecting other, noncancerous cells in the body (which 
can cause hair loss and nausea in cancer patients). This is yet another 
great cancer research discovery that does not use a single embryonic 
stem cell.
    Source: http://unisci.com/stories/20013/0724012.htm (from 7/24/01 
report)
     Inactivating Cancer Cells: Scientists at the Lautenberg 
Center for General and Tumor Immunology of the Hebrew University--
Hadassah Medical School say that they have a new and safer way to 
battle cancer. They say that doctors should induce cancer to remain in 
a dormant State, rather than treat it by massive chemotherapy treatment 
(which normally kills both cancerous and normal cells). The researchers 
have developed a technique whereby they link a toxic molecule with an 
antibody directed against specific receptors on cancer cells. After 
injecting this complex into the blood stream, the cancer cells can be 
induced to internalize the complex. The toxin portion of the complex 
then inactivates the cancer cell, and prevents it from replicating. In 
mouse experiments, the scientists caused a human tumor to grow on an 
immunologically-deficient strain of mice. When treated with the human 
antibody-toxin complex, the cancer cells in the mice became dormant. 
The mice stayed healthy for as long as they were being treated. If this 
treatment proves effective in human clinical trials, it could be used 
as a therapy for many different kinds of cancer. Importantly, it does 
not necessitate the use of human embryonic stem cells.
    Source: http://www.eurekalert.org/ (8/7/01 report entitled ``Hebrew 
University researchers seek to `inactivate' cancer cells'')
                            crohn's disease
Adult Stem Cell Research
     Adult Stem Cells Used to Treat Crohn's Disease: Crohn's 
disease, which affects more than 50,000 Americans, is an autoimmune 
disorder wherein a patient's white blood cells attack their own 
stomach. It can cause extreme pain, chronic diarrhea, and an inability 
to digest food. Ten weeks ago, Northwestern Memorial Hospital in 
Chicago, Illinois performed the first stem-cell transplant to treat 
Crohn's disease, with successful results. The outcome was so 
encouraging that they performed a second transplant on a 16-year-old 
boy on Monday (8/6/01). The first transplant involved a 22-year-old 
woman who had suffered from Crohn's disease since childhood. The 
doctors first collected some of her own blood stem cells. They then 
used powerful chemotherapeutic drugs to destroy her faulty immune 
system, and afterwards injected the blood stem cells back into her 
circulatory system. The stem cells traveled to her bone marrow and 
immediately began producing new immune system cells. After previously 
failing all other treatments, the patient now has no diarrhea or 
abdominal pain. Similar procedures have been used successfully to treat 
lupus (another autoimmune disease) and with promising results to treat 
multiple sclerosis. All with the use of a patient's own stem cells, not 
embryonic stem cells.
    Source: http://www.suntimes.com/output/news/cst-nws-stem07.html 
(Golab, Art; Chicago-Sun Times; 8/7/01; ``Stem Cells Used to Treat 
Crohn's'')
                             skin disorder
Adult Stem Cells Successful in Treating Rare Skin Disorder
     A team of doctors at the University of Texas M.D. Anderson 
Cancer Center in Houston recently performed the first known adult stem 
cell transplant to treat a rare skin disorder called scleromyxedema. 
The disorder is characterized by waxy, stiff, thickened skin. In the 
Houston patient, it had progressed so far that the skin on his face had 
a ``cobblestone appearance,'' and he could not close his eyelids 
completely or eat. The doctors first collected stem cells from the 
patient's own bone marrow, and then purposely destroyed his immune 
system with chemotherapy. They then transplanted his stem cells back 
into his system in order to allow the cells to reconstruct his immune 
system. Three months after the transplant, the patient's face does not 
have the cobblestone appearance anymore, and he can now close his eyes 
and open his mouth. This is another example of highly encouraging 
results from adult stem cells in clinical work on human patients.
    Sources: Prolife Infonet 8/20/01, #2509; Reuters Health, Aug. 17, 
2001; also Feasel, et al., ``Complete Remission of Scleromyxedema 
Following Autologous Stem Cell Transplantation'', Archives of 
Dermatology; August 2001, Vol. 137, No. 8, pp. 1071-1072.
                              thalassemia
Umbilical Cord Cell Transplant Successfully Used To Treat Thalassemia
     Doctors from Singapore's National University Hospital and 
Singapore General Hospital have succeeded in the first known case of an 
umbilical cord cell transplant into a patient with Thalassemia Major. 
Thalassemia is a rare genetic blood disorder wherein the patient's red 
blood cells are unusually small and contain defective hemoglobin, and 
can lead to anemia. If left untreated, it is fatal for children. 
Thalassemia Major is one of the most severe forms of the disease. 
Usually, thalassemia is treated with bone marrow transplants. If a 
matching bone marrow donor cannot be found, the patient can be given 
numerous blood transfusions (which carry with them the risk of 
infections or development of diabetes or liver cirrhosis due to 
overdose of iron). In this recent case, the patient was given the 
umbilical cord blood of an unrelated donor. 28 days after the 
transplant, the patient was producing normal red blood cells, and was 
released 8 days after that. Umbilical cord cell transplants offer 
patients an opportunity for an alternative source to bone marrow 
transplants; it offers more versatility as well, since the umbilical 
cord donor does not appear to need to even be related.
    Source: Agence France Press; August 14, 2001; International News 
Section; ``Singapore scores medical first in treatment of 
thalassaemia.''
                     applicable to various diseases
Non-ES/Fetal Cell Research
     Blocking Programmed Cell Death and Engulfment of Cells 
Could Have Potential for Treating Various Diseases: Researchers at Cold 
Spring Harbor Laboratory have found methods for decreasing cell death 
in the developing nervous system of the nematode worm known as C. 
elegans. They have been studying the cell survival and cell death 
processes in the worm, since it has a transparent body and such cell 
activities can be monitored with a microscope. Like in other animals, 
the normal process of cell death involves a signal inside of cells that 
are about to die, which causes a programmed death response in the 
cells. Soon afterwards, neighboring cells ``engulf'' the dying cell to 
complete the process. The scientists have found that when they were 
able to weaken the ``death signal'' within the cell slated to die, they 
were able to save a percentage of the cells. In addition, when they 
were able to weaken the ``engulfment machinery'' in the engulfing 
neighbor cells at the same time, an even greater number of cells were 
saved from death. The researchers suggest that similarly modifying the 
engulfment machinery in some cells in humans could prove to be an 
effective therapy for patients with neurodegenerative disease, stroke, 
and cancer.
    Sources: Daniel J. Hoeppner, et al.; ``Engulfment genes cooperate 
with ced-3 to promote cell death in Caenorhabditis elegans''; Vol. 412; 
July 12, 2001; pp. 202-206; also News Release from Cold Spring Harbor 
Laboratory (original source) and http://www.sciencedaily.com/releases/
2001/07/010712080935.htm
     Some Mouse Tissue Capable Of Regeneration: A special 
strain of mouse may contain secrets for rapid regeneration of injured 
bodily tissue. These laboratory mice, called ``MRL mice,'' have been 
noted to have a special ability to heal themselves with minimal 
scarring. Recent studies by researchers at the Wister Institute in 
Philadelphia have shown that even after serious heart injury, these 
mice show a fascinating ability to heal their own heart tissue without 
the use of drugs, injections of stem cells, or other intervention. 
Their experiments showed that in control mice, only 1-3% of the heart 
cells in the region of cardiac injury were capable of dividing. In the 
MRL mice, however, up to 20% of cells in the damaged heart area were 
capable of dividing after the injury. Further studies on these mice 
will possibly be aimed at identifying specific genetic and molecular 
markers which are the cause of such rapid regeneration ability. In 
doing so, scientists can potentially design drugs and treatments that 
can lead to improved healing in many types of injuries and illnesses. 
All without necessitating the use of embryonic stem cells.
    Source: http://unisci.com/stories/20013/0807014.htm (8/7/01 report)
     Platelets Have Ability to Produce Proteins In Response To 
Signals: Scientists at the University of Utah have found out some new 
things about platelets--they are much more versatile than once thought. 
Platelets are one of the components of blood (besides red blood cells 
and plasma), and are known to be involved in wound healing, by sticking 
together to form plugs at wound sites to help close the wounds up. They 
have been long thought to have a relatively limited ability to produce 
proteins in response to cellular signals as normal cells do, because 
they lack a cell nucleus (which is usually the interpreter of cellular 
signals). However, the Utah scientists have found that platelets really 
do have the capacity for significant production of proteins for wound 
repair, in response to cellular signals. They found that platelets 
contain RNA transcripts inside them. These portions of cellular 
machinery are involved in the production of proteins. Specifically, it 
was discovered that with these RNA transcripts, platelets can produce 
``interleukin -1 -beta'', which is a protein that signals the cells in 
damaged blood vessels to display receptors for white blood cells (so 
that the white blood cells can hone in on the injury). These 
discoveries offer new insights into ways that blood vessels are 
repaired, and could lead to the development of drugs and treatments 
that can be more specifically-targeted. Platelets are involved in many 
illnesses (heart attacks, circulation problems, strokes, gangrene). 
This new knowledge could be utilized to treat all of them, with no need 
for embryonic stem cells.
    Source: http://unisci.com/stories/20013/0806011.htm (8/6/01 report)
More Umbilical Cord Cell Possibilities
     ViaCell Inc., an Umbilical Cord Blood Research and Banking 
Company, Comments on Bush Decision to Fund Alternatives. ViaCell, Inc., 
commenting on a portion of the recent Bush Administration's decision 
regarding stem cells, expressed approval at the Administration's 
decision to increase funding for alternatives to human embryonic stem 
cells such as adult and umbilical cord cells. ViaCell has developed a 
new technology which they call ``Selective 
Amplification'', which allows the expansion of stem 
cell populations from umbilical cords. In addition, they are 
``preparing and Investigational New Drug (IND) application to initiate 
human clinical trials designed to demonstrate the safety, efficacy, and 
clinical benefits of selectively amplified stem cells from umbilical 
cord blood.'' They mention that some key advantages of umbilical cord 
stem cells include their use in the therapy of over 45 diseases; their 
ability to not cause as many immunological problems as other types of 
cells when transplanted into donors; and the fact that they can 
differentiate into various cell types (including hematopoetic, 
mesenchymal, and neural cells). Of course, one of the primary 
advantages is that they do not involve the ethical and moral concerns 
that the use of human embryonic stem cells do.
    Source: http://www.eurekalert.org/ (8/10/01 report); Contacts 
include Ernie Knewitz (Media Contact for Noonan/Russo Communications; 
[email protected] or 212-696-4455 ext. 204) or Marc Beer (Chief 
Executive Officer of ViaCell, Inc.; [email protected] or 800-766-
0966 ext. 4650)
   adult stem cells' ability to change into many other types of cells
Adult Stem Cell Research
     A group of researchers at Johns Hopkins University has 
found that mouse bone marrow cells can change in vivo into the cells 
which line the lungs, intestines, and skin. Dr. Sharkis, the leader of 
the research team, was quoted in the May 4, 2001, issue of Cell as 
saying that ``an infusion of stem cells after toxic cancer treatments 
may be able to repair damaged tissue throughout the body, and serve as 
treatments for a variety of diseases characterized by tissue and organ 
damage, such as diabetes and cystic fibrosis.'' (Krause, et al.; 
``Multi-Organ, Multi-Lineage Engraftment by a Single Bone Marrow-
Derived Stem Cell''; Cell; May 4, 2001: 105 (3); 369-377.)
     Fat cells taken from liposuction were able to change into 
cells resembling cartilage, bone, and muscle. (Zuk, et al.; 
``Multilineage cells from human adipose tissue: implications for cell-
based therapies''; Tissue Engineering; 97 (410) 2001; 211-228.)
     Skin Of Adult Mammals Contains Multipotent Stem Cells (1/
26/01 www.unisci.com article). A French research team published in the 
January 26, 2001 issue of Cell that the skin of adult mammals contains 
stem cells which can give rise to the epidermis, sebaceous glands, and 
hair follicles. They found that the stem cells are mainly located 
within the hair follicles. This discovery can aid in research and 
treatment in dermatology, oncology, and cosmetology. In dermatology, 
these adult stem cells have the potential to help in the areas of wound 
healing and in improving the results of skin grafts in burn patients. 
In oncology, the adult stem cells show promise in helping to understand 
the origins of some skin cancers. In cosmetology, it is thought that 
research on the cells could help to treat hair loss or excess body 
hair.
    Sources: Oshima, et al.; ``Morphogenesis and Renewal of Hair 
Follicles from Adult Multipotent Stem Cells;'' Cell; Vol. 104 (2), pp. 
233-245, January 26, 2001; also, http://unisci.com/stories/20011/
0126015.htm.
               alternatives to using embryonic stem cells
Adult Stem Cells
     Numerous recent studies have revealed the potential of 
adult stem cells to become several different types of tissue. This 
capability defeats the accepted scientific knowledge of decades past; 
it was formerly thought that once a stem cell within a developing 
embryo had differentiated and committed itself into one cell type, it 
couldn't re-differentiate later in life from an adult cell into another 
cell type. It has now been found that adult neural murine stem cells 
can transform themselves into blood cells, \1\ adult muscle stem cells 
can convert into large quantities of blood cells, \2\ adult bone marrow 
cells can become liver cells. \3\
---------------------------------------------------------------------------
    \1\ Bjornson, et al., ``Turning Brain into Blood: A Hematopoietic 
Fate Adopted by Adult Neural Stem Cells in Vivo,'' Science [January 22, 
1999] 534-7.
    \2\ Jackson, Mi and Goodell, Proceedings of the National Academy of 
the Sciences [December 7, 1999] 14193-5.
    \3\ Thiese, Krause, et al., Hepatology [January, 2000] 235-40.
---------------------------------------------------------------------------
     This progress on adult stem cells is wonderful for 
potential patient recipients, because there would not be the problem of 
immune rejection of the cells that one would find with embryonic stem 
cells. Adult stem cell lines could be grown from the patients' own 
body, thus overcoming any potential graft-versus-host disease and any 
need for strong immunosuppressive drug therapy.
     Likewise, adult stem cells are much more accessible than 
embryonic stem cells. They are located throughout the human body, even 
in adipose (fat) tissue.
Umbilical Cord and Placental Cells
     Umbilical cord blood banks and placenta banks are in 
operation in several states and are being organized in others. These 
cells have shown promise in being used to combat leukemia. \4\
---------------------------------------------------------------------------
    \4\ Rubinstein, et al., ``Outcomes among 562 Recipients of 
Placental-Blood Transplants from Unrelated Donors,'' New England 
Journal of Medicine, 339 [Nov. 26, 1998) 1565-1577.
---------------------------------------------------------------------------
          detrimental findings with es and fetal cell research
     A study in Science (Humpherys, et al.; ``Epigenetic 
Instability in ES Cells and Cloned Mice''; Volume 293 (5527); July 6, 
2001; pp. 95-97; or http://www.sciencemag.org/ [subscription 
necessary]) shows that embryonic stem cells and cloned cells are 
extremely genetically unstable. The researchers examined the gene 
expression (the production of proteins from genes) of the cells of mice 
obtained by cloning. The clones were obtained by performing a transfer 
of an embryonic stem cell into a de-nucleated egg and then implanting 
the embryo into a surrogate mother. The original donor embryonic stem 
cells were also examined. It was found that gene expression by both 
sets of cells was very different when compared from cell to cell. This 
implies that ``even apparently normal cloned animals may have subtle 
abnormalities in gene expression.'' (p. 95) It also implies that if 
embryonic stem cells would 1 day be able to be coaxed adequately into 
cells of interest (i.e., cardiac, neuronal, etc.) for transplant 
purposes, the cells may exhibit aberrant behavior due to each cell 
being wildly different in the proteins they produce.
     Injection of fetal cells into the brains of Parkinson's 
disease patients was found to cause wild, aberrant movements such as 
arm swinging and head-jerking. It is thought that these fetal cells 
produced too much dopamine, and were difficult to control. (Freed, et 
al.; ``Transplantation of Embryonic Dopamine Neurons for Severe 
Parkinson's Disease''; New England Journal of Medicine; March 8, 2001; 
Vol. 344 (10); pp. 710-719).
     Unstable Gene Expression Found in Both Clones and 
Embryonic Stem Cells: Scientists have just published a study in Science 
in which they have found that embryonic stem cells and cloned cells are 
extremely genetically unstable. The researchers examined the gene 
expression (the production of proteins from genes) of the cells of mice 
obtained by cloning. The clones were obtained by performing a transfer 
of an embryonic stem cell into a de-nucleated egg and then implanting 
the embryo into a surrogate mother. The original donor embryonic stem 
cells were also examined. It was found that gene expression by both 
sets of cells was very different when compared from cell to cell. This 
implies that ``even apparently normal cloned animals may have subtle 
abnormalities in gene expression.'' (p. 95) It also implies that if 
embryonic stem cells would 1 day be able to be coaxed adequately into 
cells of interest (i.e., cardiac, neuronal, etc.) for transplant 
purposes, the cells may exhibit aberrant behavior due to each cell 
being wildly different in the proteins they produce.
    This is a further reason for not utilizing human embryonic stem 
cells or human clones in research. There are alternative cells which 
are morally, and as this new finding shows, scientifically and 
medically, more acceptable: adult stem cells, placental cells, and 
umbilical cord cells.
    Sources: Humpherys, et al.; ``Epigenetic Instability in ES Cells 
and Cloned Mice''; Volume 293 (5527); July 6, 2001; pp. 95-97; or 
http://www.sciencemag.org/ [subscription necessary]

    [Whereupon, at 2:40 p.m., the committee was adjourned.]

                                    

      
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