[Senate Hearing 106-413]
[From the U.S. Government Printing Office]





                                                        S. Hrg. 106-413

 
                       STEM CELL RESEARCH, PART 3

=======================================================================

                                HEARINGS

                                before a

                          SUBCOMMITTEE OF THE

            COMMITTEE ON APPROPRIATIONS UNITED STATES SENATE

                       ONE HUNDRED SIXTH CONGRESS

                             SECOND SESSION

                               __________

                            SPECIAL HEARINGS

                     APRIL 26, 2000--WASHINGTON, DC
                   SEPTEMBER 7, 2000--WASHINGTON, DC
                   SEPTEMBER 14, 2000--WASHINGTON, DC

                               __________

         Printed for the use of the Committee on Appropriations


 Available via the World Wide Web: http://www.access.gpo.gov/congress/
                                 senate



                    U.S. GOVERNMENT PRINTING OFFICE
66-482 cc                   WASHINGTON : 2001

                                 ______

_______________________________________________________________________
            For sale by the U.S. Government Printing Office
Superintendent of Documents, Congressional Sales Office, Washington, DC 
                                 20402




                      COMMITTEE ON APPROPRIATIONS

                     TED STEVENS, Alaska, Chairman
THAD COCHRAN, Mississippi            ROBERT C. BYRD, West Virginia
ARLEN SPECTER, Pennsylvania          DANIEL K. INOUYE, Hawaii
PETE V. DOMENICI, New Mexico         ERNEST F. HOLLINGS, South Carolina
CHRISTOPHER S. BOND, Missouri        PATRICK J. LEAHY, Vermont
SLADE GORTON, Washington             FRANK R. LAUTENBERG, New Jersey
MITCH McCONNELL, Kentucky            TOM HARKIN, Iowa
CONRAD BURNS, Montana                BARBARA A. MIKULSKI, Maryland
RICHARD C. SHELBY, Alabama           HARRY REID, Nevada
JUDD GREGG, New Hampshire            HERB KOHL, Wisconsin
ROBERT F. BENNETT, Utah              PATTY MURRAY, Washington
BEN NIGHTHORSE CAMPBELL, Colorado    BYRON L. DORGAN, North Dakota
LARRY CRAIG, Idaho                   DIANNE FEINSTEIN, California
KAY BAILEY HUTCHISON, Texas          RICHARD J. DURBIN, Illinois
JON KYL, Arizona
                   Steven J. Cortese, Staff Director
                 Lisa Sutherland, Deputy Staff Director
               James H. English, Minority Staff Director
                                 ------                                

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

                 ARLEN SPECTER, Pennsylvania, Chairman
THAD COCHRAN, Mississippi            TOM HARKIN, Iowa
SLADE GORTON, Washington             ERNEST F. HOLLINGS, South Carolina
JUDD GREGG, New Hampshire            DANIEL K. INOUYE, Hawaii
LARRY CRAIG, Idaho                   HARRY REID, Nevada
KAY BAILEY HUTCHISON, Texas          HERB KOHL, Wisconsin
TED STEVENS, Alaska                  PATTY MURRAY, Washington
JON KYL, Arizona                     DIANNE FEINSTEIN, California
                                     ROBERT C. BYRD, West Virginia
                                       (Ex officio)

                           Professional Staff

                            Bettilou Taylor
                             Mary Dietrich
                              Jim Sourwine
                        Ellen Murray (Minority)

                         Administrative Support

                             Kevin Johnson
                       Carole Geagley (Minority)




                            C O N T E N T S

                              ----------                              

                       Wednesday, April 26, 2000

                                                                   Page

Opening statement of Senator Arlen Specter.......................     1
Statement of Gerald Fischbach, M.D., Director, National Institute 
  of Neurological Disorders and Stroke, National Institutes of 
  Health, Department of Health and Human Services................     2
    Prepared statement...........................................     5
Statement of Allen M. Spiegel, M.D., Director, National Institute 
  of Diabetes and Digestive and Kidney Diseases, National 
  Institutes of Health, Department of Health and Human Services..     4
    Prepared statement...........................................     5
Opening statement of Senator Tom Harkin..........................    10
Opening statement of Senator Harry Reid..........................    11
Opening statement of Senator Patty Murray........................    13
Statement of Hon. Sam Brownback, U.S. Senator from Kansas........    15
    Prepared statement...........................................    19
Statement of Frank Young, M.D., Ph.D., Former Commissioner, Food 
  and Drug Administration, Department of Agriculture.............    21
Statement of Mary Jane Owen, M.S.W., executive director, National 
  Catholic Office for Persons With Disabilities..................    23
    Prepared statement...........................................    26
Statement of Christopher Reeve, actor/director; chairman, 
  Christopher Reeve Paralysis Foundation.........................    35
    Prepared statement...........................................    38
Statement of Jennifer Estess, actor/producer.....................    41
Statement of Lawrence B. Goldstein, Ph.D., professor, Division of 
  Cellular and Molecular Medicine, University of California, San 
  Diego School of Medicine; investigator, Howard Hughes Medical 
  Institute......................................................    42
    Prepared statement...........................................    45

                      Thursday, September 7, 2000

Opening statement of Senator Arlen Specter.......................    49
Opening statement of Senator Tom Harkin..........................    51
Statement of Gerald D. Fischbach, M.D., Director, National 
  Institute of Neurological Disorders and Stroke, National 
  Institutes of Health, Department of Health and Human Services..    52
    Prepared statement...........................................    55
Statement of Allen M. Spiegel, M.D., Director, National Institute 
  of Diabetes and Digestive and Kidney Diseases, National 
  Institutes of Health, Department of Health and Human Services..    54
    Prepared statement...........................................    55
Statement of David A. Prentice, M.D., Ph.D., professor of life 
  sciences, Indiana State University.............................    61
    Prepared statement...........................................    65
Statement of Micheline M. Mathews-Roth, M.D., associate professor 
  of medicine, Harvard Medical School............................    67
    Prepared statement...........................................    70

                      Thursday, September 14, 2000

Opening statement of Senator Arlen Specter.......................    81
Statement of Senator Tom Harkin..................................    82
Statement of Senator Paul D. Wellstone...........................    83
Statement of Richard O. Hynes, Ph.D, director, Center for Cancer 
  Research, Massachusetts Institute of Technology................    83
    Prepared statement...........................................    85
Statement of Darwin J. Prockop, M.D., Ph.D, director, Center for 
  Gene Therapy, Tulane University Medical Center.................    87
    Prepared statement...........................................    89
Statement of Ron Heagy, president and founder of the Life is an 
  Attitude Foundation............................................    93
Statement of Russell Saltzman, pastor, Ruskin Heights Lutheran 
  Church, Kansas City, MO........................................    95
    Prepared statement...........................................    96
Statement of Anton-Lewis Usala, M.D., chairman/chief technical 
  officer, Encelle, Inc., Greenville, NC.........................    98
    Prepared statement...........................................   100
Statement of Gina Gershon, actress...............................   110
Statement of Jennifer Estess, actor/producer.....................   111
Statement of Mary Tyler Moore, international chairman, Juvenile 
  Diabetes Foundation............................................   113
    Prepared statement...........................................   115
Statement of Michael J. Fox, Foundation for Parkinson's Research.   117
  


                 FEDERAL FUNDING FOR STEM CELL RESEARCH

                              ----------                              


                       WEDNESDAY, APRIL 26, 2000

                           U.S. Senate,    
    Subcommittee on Labor, Health and Human
     Services, and Education, and Related Agencies,
                               Committee on Appropriations,
                                                    Washington, DC.
    The subcommittee met at 11:02 a.m., in room SH-216, Hart 
Senate Office Building, Hon. Arlen Specter (chairman) 
presiding.
    Present: Senators Specter, Harkin, Reid, and Murray.


               OPENING STATEMENT OF SENATOR ARLEN SPECTER


    Senator Specter. Good morning, ladies and gentlemen. The 
hearing of the Appropriations Subcommittee on Labor, Health and 
Human Services, and Education will now proceed.
    We are taking up a very important subject today, and that 
is the issue of Federal funding for stem cell research. This is 
the fifth hearing which the subcommittee is holding on this 
very important subject, and we do it as a preliminary to action 
on the Senate floor on this subject, which will be taken up in 
the course of the next several weeks.
    When the medical possibilities of stem cells was noted in 
November of 1998, this subcommittee very promptly scheduled a 
hearing in December of 1998 to take up the issue of the private 
research which had been done showing that stem cells had 
enormous potential for many diseases, Parkinson's, Alzheimer's, 
amyotrophic lateral sclerosis, known as Lou Gehrig's disease, 
possibly implications for heart disease.
    And subsequent hearings were held, one on the subject of 
the proprietary interest in these patents. A very major 
question arises as to whether this is something which ought to 
be in the private domain or ought to be in the public domain. 
And we have had hearings which have taken up the ethical and 
moral considerations. And there is a very profound debate which 
has been undertaken on this very, very important subject.
    My analysis has been that the use of discarded embryos 
would not affect human life, that in in vitro fertilization, 
there are many embryos taken, and if there is any possibility 
of human life, I would be the first to oppose any use for 
medical research if potential human life were to be involved. 
My analysis and study has demonstrated that the discarded 
embryos are not going to be used for human life, so it is not a 
question of taking human life or the risk of taking human life, 
but the potential for saving life. I believe that stem cell 
research has the potential for a veritable fountain of youth.
    And there are other views and other views must be taken 
into account and will be considered as the Congress considers 
the issue of whether the current ban on use of Federal funding 
for embryonic research will take place.
    The General Counsel for the Department of Health and Human 
Services has rendered a ruling that Federal funding may be used 
on the stem cells once extracted from the embryos, but not on 
the embryos themselves. And there is a substantial body of 
medical evidence which says that is not sufficient, that the 
embryos really need to be used.
    This issue has a corollary on fetal tissue which had been 
banned for many years, and after extensive consideration, fetal 
tissue is now used for medical research. There had been a 
concern that the use of fetal tissue would encourage abortions, 
and I think those fears were finally allayed. Senator Thurmond 
was a key vote on that matter, and I think persuaded many in 
the Senate when Senator Thurmond voted for the use of fetal 
tissue for medical purposes in a very close personal matter 
which his daughter having juvenile diabetes, given his deep 
respect for human life, as I also have that deep respect and I 
think we all do, but now fetal tissue is used for medical 
research because the conclusion was reached that it does not 
promote or encourage abortions.
    We had inserted a provision in the appropriations bill last 
fall to eliminate the ban on Federal funding, and when it 
appeared that that would tie up the appropriations bill, we 
removed that provision with the understanding reached with our 
distinguished Majority Leader Senator Lott that the Senate 
would take up the bill as a freestanding bill, which was 
introduced in January by Senator Harkin and myself on 
bipartisan support.
    We will now proceed with our first panel: Dr. Gerald 
Fischbach and Dr. Allen Spiegel. If you gentlemen would step 
forward.
STATEMENT OF GERALD FISCHBACH, M.D., DIRECTOR, NATIONAL 
            INSTITUTE OF NEUROLOGICAL DISORDERS AND 
            STROKE, NATIONAL INSTITUTES OF HEALTH, 
            DEPARTMENT OF HEALTH AND HUMAN SERVICES
    Senator Specter. Dr. Fischbach was appointed Director of 
the National Institute of Neurological Disorders and Stroke in 
1998. From 1990 until 1998, he served as Director of the 
Neurobiology Departments at the Harvard Medical School and 
Massachusetts General Hospital. He has also been Professor of 
Neurobiology and head of the Department of Anatomy and 
Neurobiology at Washington University School of Medicine.
    Thank you for joining us, Dr. Fischbach, and we look 
forward to your testimony.
    Dr. Fischbach. Thank you, Mr. Chairman.
    In the next 2 or 3 minutes I would like to give you a brief 
statement with a definition of stem cells, their general 
promise, and then their promise in the area of my expertise in 
brain science.
    As you know, stem cells are unique in that they have a 
capability of self-renewal. They can give rise to many cells of 
the same type, but they also have the very special property of 
giving rise to unique highly specialized cells, such as heart 
cells, muscle cells, nerve cells, and pancreas cells under the 
right conditions.
    There is a hierarchy of stem cells. Some stem cells are 
more limited in their capacity to proliferate and to give rise 
to different types of cells than others. Much like seeds in the 
woods, some seeds can give rise to a few trees with a limited 
number of branches and potential avenues of growth, whereas 
other seeds can populate a whole forest and can give rise to 
trees with an enormously elaborate set of branches and arbors.
    The pluripotent human stem cells, which we will discuss 
further, are in the latter class according to current 
scientific information. They have the broadest potential for 
renewal and the broadest potential for specialization at the 
same time.
    Stem cells have enormous promise in four major areas. The 
one that really brings us here today, that has ignited patient 
advocacy communities and inspired scientists, is the ability of 
stem cells to repair damaged tissue, to replace cells that have 
become dysfunctional. And while Dr. Spiegel and I are here 
representing two organs, the pancreas and the brain, I know you 
realize that virtually every institute at the NIH has a deep 
interest in stem cell research for replacing various tissues 
throughout the body. The potential in this regard is exciting 
and unlimited, and I think it is the reason Science magazine 
named stem cells as the breakthrough of the year last year in 
all fields, chemistry, physics, and biology.
    Stem cells are also promising as a means for discovering 
new drugs with modern new assays. They are very important for 
understanding the mechanism of disease, and very recently it 
has been found that they have enormous potential for delivering 
medicines. They seem to track the path of disease cells and 
deliver medicines right to the source.
    There is nowhere, I believe, a more urgent need for the use 
of stem cells than in these devastating neurodegenerative 
disorders of the brain. Nerve cells in the brain, by and large, 
are a non-renewable resource. When they are damaged or lost, 
they cannot, with very, very rare exception, be replaced. In 
previous years, there has been hope only for symptomatic, not 
for therapeutic interventions. And there are cases now where we 
can pinpoint deficits where stem cells have already had great 
promise in animal models of human disease, replacing as you 
have mentioned, dopamine neurons in Parkinson's disease, motor 
neurons in ALS and spinal muscular atrophy, cholinergic neurons 
in Alzheimer's disease.
    But you have to realize that the brain is probably the most 
complex structure in the known universe with over 100 billion 
cells of great diversity. So, it is important to choose stem 
cells that themselves have the potential for great diversity 
and that can repopulate the needed environment. The complexity 
of the brain requires team work, so in other disorders, in 
repair after stroke, in repair after spinal cord injury, or in 
general diseases such as multiple sclerosis, the full diversity 
of the stem cell phenotype is needed.
    Now, there are problems and there is a need to do 
additional research. And I will end with this. These are very 
important challenges that many of us feel are on the horizon. 
We must understand how to regulate the proliferation of stem 
cells. We must learn how to steer them into one branch of that 
tree versus another, and we must really learn how to promote 
their long-term survival once they are implanted. The hope is 
that with Federal funding of the use of stem cells, we can 
bring one of the the world's treasures, which I believe is the 
American scientific community, funded by the NIH, to bear on 
these problems.
    Senator Specter. Thank you very much, Dr. Fischbach.
STATEMENT OF ALLEN M. SPIEGEL, M.D., DIRECTOR, NATIONAL 
            INSTITUTE OF DIABETES AND DIGESTIVE AND 
            KIDNEY DISEASES, NATIONAL INSTITUTES OF 
            HEALTH, DEPARTMENT OF HEALTH AND HUMAN 
            SERVICES
    Senator Specter. We turn now to Dr. Allen Spiegel, 
appointed Director of the National Institute of Diabetes and 
Digestive and Kidney Diseases last year. Prior to his 
appointment as Director, Dr. Spiegel had a number of positions 
at the Institute, including Director of Intramural Research and 
Chief of Metabolic Disease Branch. He holds an M.D. from 
Harvard, a bachelors from Columbia.
    Thank you for joining us, Dr. Spiegel, and we look forward 
to your testimony.
    Dr. Spiegel. Thank you, Mr. Chairman. I appreciate the 
opportunity to appear before you today to discuss the promise 
of research on human pluripotent stem cells.
    The research holds great potential for treatment of many of 
the diseases within the research mission of the National 
Institute of Diabetes and Digestive and Kidney Diseases, but 
I'd like to focus my remarks today on the treatment of type 1, 
or juvenile, diabetes.
    In type 1 diabetes, there is a lack of insulin due to 
destruction by the body's own immune system of the insulin-
producing cells in the pancreatic islets. While treatment with 
insulin is life-saving, it is not a cure. Our research has 
shown that tight control of the blood sugar with insulin 
treatment can delay or prevent the complications of diabetes, 
such as blindness, and kidney failure, but tight control is 
extremely difficult to achieve. In this regard, nothing I could 
say would be as eloquent as the testimony you yourself heard 
last year from the children who came to Washington as part of 
the Children's Congress on Diabetes. The frequent needle sticks 
and constant danger of low blood sugar they described are only 
some of the difficulties they and their parents must endure 
every day and night of their lives.
    This is why scientists are working so diligently to find 
ways to cure type 1 diabetes. Of the many approaches we are 
pursuing toward this goal, the most promising, in my view, is 
transplantation of the insulin-producing islet cells. For many 
years, this approach had minimal success, but recent advances 
in immunology research have led to innovative treatments that 
effectively block islet transplant rejection. While the results 
in humans are still preliminary, and there is certainly need 
for wide replication before we can be sure, islet 
transplantation offers the prospect of a real cure for type 1 
diabetes.


                           PREPARED STATEMENT


    The very real problem, though, is that the available supply 
of pancreases for harvesting islets is completely inadequate 
for the hundreds of thousands of patients with type 1 diabetes 
who would be candidates for such transplants. It is here that 
research on human pluripotent stem cells with their theoretical 
ability to provide a limitless source of islet cells is so 
important. Some have argued that we could achieve the same goal 
by using adult pancreatic stem cells, and I agree that this 
line of research must be vigorously pursued. But at this point, 
we have no certainty that adult pancreatic stem cells can be 
isolated in a practical way, nor that they can replicate to 
provide sufficient numbers of islet cells for transplantation. 
For this reason, it is vital that we simultaneously pursue 
research on human pluripotent stem cells, which offer the 
greatest promise of providing an adequate supply of islet cells 
for treating and ultimately curing type 1 diabetes.
    Thank you for your attention.
    Senator Specter. Thank you very much, Dr. Spiegel.
    [The joint statement follows:]
   Joint Prepared Statement of Allen M. Spiegel, M.D. and Gerald D. 
                            Fischbach, M.D.
    Mr. Chairman and Members of the Subcommittee, we are pleased to 
appear before you to discuss the promise of human pluripotent stem cell 
research. Recent published reports on the isolation and successful 
culturing of the first human pluripotent stem cell lines have generated 
great excitement among scientists, patients and their families. 
Research using human pluripotent stem cells holds enormous promise for 
advances in the prevention, treatment, and diagnosis of a vast array of 
diseases. Virtually every realm of medicine might be touched by this 
innovation. Because of this enormous promise, NIH believes that this 
research must proceed, as long as it is conducted ethically and 
legally.

                          WHAT ARE STEM CELLS?
    Stem cells are self-renewing and can give rise to the more 
specialized cells of the human body, such as muscle cells, blood cells 
and brain cells. They are best described in the context of normal human 
development. When a sperm fertilizes an egg, the product is a single 
cell that has the potential to form an entire organism. This fertilized 
egg is a totipotent stem cell, which has the potential to develop into 
a complete organism. In the first hours and days after fertilization, 
this cell begins to divide into identical totipotent stem cells. Then, 
approximately four days after fertilization, these totipotent stem 
cells begin to specialize, forming a hollow sphere of cells called a 
blastocyst. One part of the blastocyst is a cluster of cells called the 
inner cell mass, which are the stem cells that will go on to form most 
of the cells and tissues of the human body. These are pluripotent stem 
cells, which are different than totipotent stem cells. Pluripotent stem 
cells do not develop into a complete organism.
    Recently, human pluripotent stem cells have been isolated from two 
sources: the inner cell mass of human embryos at the blastocyst stage 
and from fetal tissue obtained from terminated pregnancies. Because 
these cells are capable of limitless division and self-renewal, they 
can be maintained indefinitely in tissue culture, making them a vital 
resource for research.

            WHY ARE HUMAN PLURIPOTENT STEM CELLS IMPORTANT?
    There are several reasons why the isolation of human pluripotent 
stem cells might lead to better treatment, even cures, of many 
diseases. At the most fundamental level, pluripotent stem cells could 
help us to understand the complex events that occur during normal human 
development. By identifying the mechanisms underlying routine cell 
differentiation we hope to understand how disease-causing aberrations 
occur. Another goal of this research would be the identification of the 
factors involved in the cellular decision-making process that results 
in cell specialization--why do some cells become heart cells, for 
example, while other cells become liver cells? We know that turning 
genes on and off is central to this process, but we do not know much 
about these ``decision-making'' genes or what turns them on or off. 
Some of our most serious medical conditions, such as cancer and birth 
defects, are due to abnormal cell differentiation and cell division. A 
better understanding of normal cell processes will allow us to further 
delineate the fundamental errors that cause these often deadly 
illnesses.
    Human pluripotent stem cell research could also dramatically change 
the way we develop drugs and test them for safety. While a limited 
number of cultivated cell lines are currently available and provide 
invaluable tools for drug development and testing, pluripotent stem 
cells would allow expansion of this testing to more varied cell types. 
For example, drugs could be tested first on particular cell lines to 
determine toxicity, before they are tested in either animals or humans. 
Although this would not replace testing in animals and in human beings, 
it would streamline the process of drug development, and reduce 
potential for harm in humans and animals. Only the drugs that are both 
safe and appear to have a beneficial effect in cell line testing would 
graduate to further testing in laboratory animals and human subjects.
    Perhaps the most far-reaching potential application of human 
pluripotent stem cells is the generation of cells and tissue that could 
be used for ``cell transplantation therapies,'' which are aimed at 
diseases and disorders resulting from the destruction or dysfunction of 
specific cells and tissue. Although donated organs and tissues can 
sometimes be used to replace diseased or destroyed tissue, the number 
of people suffering from such disorders far outstrips the number of 
organs and tissues available for transplantation. Pluripotent stem 
cells, stimulated to develop into specialized cells and tissue, offer 
real hope for the possibility of a renewable source of replacement 
cells and tissue to treat a myriad of diseases, conditions, and 
disabilities for which replacement tissue is in short supply. Examples 
of these include neurological disorders, burns, heart disease, 
osteoarthritis and rheumatoid arthritis.

           HUMAN PLURIPOTENT STEM CELLS AND DIABETES RESEARCH
    One of the best examples of the promise of this line of research is 
in the treatment of Type 1 diabetes. Research on islet cell 
transplantation and stem-cell biology offers compelling opportunities 
for the development of new, innovative approaches for treating and 
ultimately curing this disease.
    Type 1 diabetes, often referred to as Juvenile Diabetes, is 
characterized by the inability of the body to produce insulin, a 
hormone necessary for glucose metabolism. This form of diabetes occurs 
when the body's immune system attacks and destroys its own insulin-
producing beta cells in the islets of the pancreas. As a result of 
inadequate insulin production, glucose does not enter cells as readily 
as when insulin levels are normal. The standard treatment is to try to 
control the glucose level with insulin injections. Insulin treatment 
can sustain the patient's life, but not necessarily prevent the 
devastating complications of type I diabetes, which include kidney 
failure, blindness, amputation, heart attack and stroke. Clinical 
trials have shown that these complications can be prevented or 
significantly delayed by maintaining blood glucose levels as close to 
normal as possible. However, precise blood glucose control is difficult 
to achieve and requires multiple daily injections of insulin or use of 
an insulin pump. These regimens are extremely challenging to follow, 
especially for children and teenagers. In addition, one risk of such 
precise blood glucose control is the development of dangerously low 
blood sugars which could cause loss of consciousness, seizures or other 
complications.
    To address these problems, researchers are investigating 
alternative approaches to restoring insulin-producing capacity, 
including attempts to develop an artificial pancreas, whole pancreas 
transplantation, and islet cell transplantation. Formidable 
bioengineering problems attend development of an artificial pancreas, 
and while researchers are working diligently to overcome them, a time 
frame for success cannot be predicted. Whole pancreas transplantation, 
while successful in some patients, is an extremely difficult surgical 
procedure and it requires lifelong treatment with immunosuppressive 
drugs that can have toxic side effects. This surgery is typically 
performed only in adults, often in conjunction with a needed kidney 
transplant for which immunosuppressive drugs would already be required. 
But, the success rate for the survival of the transplanted pancreas is 
much lower than the survival rate for the transplanted kidney.
    Islet cell transplantation is a much simpler procedure than whole 
pancreatic transplantation and has several potential advantages. Until 
very recently, serious technical problems have been a major impediment 
to rapid progress in islet transplantation research. These key 
challenges have been: (1) to keep the body's immune defense system from 
rejecting the transplanted islets; and (2) to ensure that there is a 
sufficient supply of islet cells for transplantation. To date, only 
about five percent of people with diabetes who have received 
transplanted islets along with immunosuppressive drugs have been able 
to stay off insulin longer than one year. Stem cell research offers the 
potential to overcome these obstacles.
    The renewed promise of islet cell transplantation derives from two 
complementary research opportunities. The first is the development of 
new methods for adjusting the immune system to keep the body from 
rejecting transplanted islet cells. The second is the prospect that 
stem cell research could ensure the needed supply of islet cells for 
transplantation. Human pluripotent stem cells offer the greatest 
promise of providing a limitless source of islet cells for treating and 
curing type 1 diabetes. Together, these opportunities offer 
unprecedented hope for curing type 1 diabetes, especially for children 
and young adults whose disease has not yet progressed to the point of 
debilitating complications.

      HUMAN PLURIPOTENT STEM CELL RESEARCH AND THE NERVOUS SYSTEM
    As significant as the promise of stem cells is for the treatment of 
diabetes, the potential of stem cells for treating diseases of the 
nervous system is equally impressive. It is startling to consider the 
range of neurological disorders for which scientists are actively 
investigating stem cell therapies in animal models. A partial list 
would include classic neurodegenerative diseases such as Parkinson's 
disease, Alzheimer's disease and amyotrophic lateral sclerosis (ALS); 
acute insults of stroke, brain trauma and spinal cord injury; multiple 
sclerosis and other demyelinating disorders; and inherited disorders 
such as Tay-Sachs disease and Duchenne muscular dystrophy. It might be 
possible to use stem cells to treat epilepsy and brain tumors. We have 
only begun to understand the extraordinary range of possibilities that 
stem cells present for treatment of these maladies.
    The most obvious and exciting use of stem cells in neurological 
disorders is to replace lost nerve cells. Many diseases destroy 
particular types of nerve cells, and mature nerve cells cannot produce 
new cells to replace those that are lost. Animal experiments have 
demonstrated that the potential exists for coaxing stem cells to 
specialize and replace the dopamine cells that are lost in the brain in 
Parkinson's disease. A similar approach might apply to several other 
neurological disorders. Stem cells, given appropriate control signals, 
might specialize to replace the lost acetylcholine producing nerve 
cells in Alzheimer's disease, to restore lost motor neurons in ALS, or 
to produce inhibitory cells to help restrain electrical activity in 
epilepsy.
    Replacing lost nerve cells is only the beginning of the list of 
possible therapeutic applications for stem cells. For some disorders, 
such as multiple sclerosis, stem cells might replace supporting cells--
such as the glial cells, which provide the insulation necessary to 
allow some nerves to conduct electrical impulses rapidly. Stem cell 
strategies might be useful for correcting inherited defects. For 
example, in disorders that devastate children's brains we might rely on 
the ability of stem cells to migrate widely in the brain and supply the 
vital missing enzyme that leads to early and tragic death from Tay-
Sachs disease. In addition, stem cells might regenerate the many 
different kinds of complex brain tissue that are damaged as a result of 
brain trauma or stroke. Transplanted stem cells might also supply 
natural growth and survival chemicals to pave the way for regeneration 
of remaining healthy neural tissue following spinal cord injury. Recent 
findings suggest that stem cells might be harnessed to seek out and 
destroy brain tumor cells that evade surgery or radiotherapy. The list 
of possible applications of stem cells continues to grow as we learn 
more about these cells.

                           FUTURE CHALLENGES
    There is much to be done before these discoveries can be 
incorporated into clinical practice. First, we must do the basic 
research to understand the process by which human cells become 
specialized, so that we can direct pluripotent stem cells to become the 
type(s) of tissue needed for transplantation. For example, applying 
basic knowledge obtained from research in developmental and stem cell 
biology will enable the production of progenitor stem cells and the 
rational design of cellular therapies for human diseases such as 
diabetes. It is essential to underscore that studies of stem cells and 
the genes that regulate their development could be important for the 
development of ways to intervene in type 1 diabetes, and various 
neurological conditions, even beyond their use in transplantation.
    Second, before these cells can be used for transplantation, the 
well-known problem of immune rejection must be overcome. Because human 
pluripotent stem cells derived from embryos or fetal tissue would be 
genetically different from the recipient, future research would need to 
focus on modifying human pluripotent stem cells to minimize tissue 
incompatibility or to create tissue banks with the most common tissue-
type profiles. In addition, just delivering cells to the appropriate 
sites within the human body is an extremely difficult task. All of 
these factors argue for intensified efforts to understand the basic 
biology of pluripotent stem cells and, with due caution, to apply what 
is learned towards the treatment of disease.

             WHAT ARE THE LIMITATIONS OF ADULT STEM CELLS?
    Recent findings have shown that even the adult human brain harbors 
neural stem cells, and that these adult stem cells can respond to a 
wide range of external and internal influences, such as learning, 
stress, exercise, seizures, and trauma. In addition, if pancreatic stem 
cells are ever isolated from adult tissue, it might be possible to 
direct these cells to differentiate into islet cells. The 
identification of adult pancreatic stem cells would open up entirely 
new prospects, beyond transplantation strategies, for encouraging the 
body's own stem cells to help repair damage.
    It is important to note that scientists who are leading the way in 
studying adult stem cells present compelling arguments why we must 
pursue research on both pluripotent and adult stem cells. While some 
stem cells are present in adults, there may not be an adult stem cell 
for every type of cell in the body, and they may be present in only 
minute numbers. In addition, they may be very difficult to isolate; for 
example, in the case of adult neural stem cells, they may be confined 
to certain regions of the brain that are not easily accessible. More 
importantly, pluripotent and adult stem cells are not qualitatively 
alike. Pluripotent stem cells have truly amazing abilities to self-
renew and to form many different cell types, even complex tissues, but 
in contrast the full potential of adult stem cells is uncertain, and, 
in fact, there is evidence to suggest they may be more limited. Unlike 
pluripotent stem cells, the adult stem cells may be able to divide only 
a limited number of times, which would limit their usefulness in the 
production of adequate numbers of well characterized cells for reliable 
therapies. Another issue is the question of how robust transplanted 
adult cells may be or how vulnerable to disease processes. In light of 
these limitations, it is important that we pursue research on both 
pluripotent and adult stem cells simultaneously.

                             NIH GUIDELINES
    Given the enormous promise of human pluripotent stem cells to the 
development of new therapies for the most devastating diseases, it is 
important that both privately and federally funded researchers have the 
opportunity to pursue this promise. To this end, on December 2, 1999, 
NIH published draft Guidelines in the Federal Register. NIH is 
currently in the process of analyzing public comments and will publish 
final Guidelines in the Federal Register. NIH will not fund human 
pluripotent stem cell research until final Guidelines have been 
published and an oversight process is in place.

                               CONCLUSION
    Mr. Chairman, we appreciate the opportunity to discuss this 
promising and extraordinary science and are pleased to respond to any 
questions you may have.

    Senator Specter. Dr. Fischbach, could you describe in lay 
terms exactly what a stem cell is and how it works to cure or 
prospectively cure, say, Parkinson's disease?
    Dr. Fischbach. A stem cell is a cell that has the 
capability of giving rise to many other types of cells. It is 
like the stem at the root of a tree. It also can renew itself. 
It can reproduce many times, but it also can, under appropriate 
cues, give rise to one or another specialized cell.
    Senator Specter. And illustratively, how would that cell 
reproduce a cell which is deficient, causing someone to suffer 
from Parkinson's?
    Dr. Fischbach. The environment and cues, many of which are 
known, can steer that cell into a pathway of differentiation, 
as it is called, to produce a nerve cell that is deficient in 
Parkinson's disease.
    Senator Specter. So that it produces a nerve cell to 
replace a deficient nerve cell in a person's body.
    Dr. Fischbach. Yes.
    Senator Specter. And it is the deficiency of that nerve 
cell, for example, which causes Parkinson's.
    Dr. Fischbach. That is exactly right.
    Senator Specter. I have asked for a list of the ailments 
where there is the potential for cure by stem cells. I would 
like you to tell me if this is complete. Diabetes, Alzheimer's, 
heart disease, muscular dystrophy, Parkinson's, spinal cord, 
amyotrophic lateral sclerosis, stroke, multiple sclerosis, and 
Tay-Sachs. Is there potential for curing all of those ailments 
with stem cells?
    Dr. Fischbach. I think there is great potential for curing 
or certainly reducing the burden of those diseases to an 
enormous degree.
    Senator Specter. Taking Parkinson's, again for illustrative 
purposes, we have had testimony that we may be within 5 to 10 
years, 5 to 7 years of a cure of Parkinson's. What is the 
expectation of accelerating, speeding up that process through 
the use of stem cells?
    Dr. Fischbach. I think that estimate, which I have also 
heard and been very involved with, is very dependent on the use 
of stem cells. That is certainly one of the main efforts that 
will be undertaken to make the Parkinson's strategic research 
plan successful.
    Senator Specter. Dr. Fischbach--and, Dr. Spiegel, you may 
want to comment on this as well--there have been suggestions 
that there are alternative sources for stem cells besides 
embryos: umbilical cords, a variety of other sources. Are there 
other ways to get an adequate number of stem cells to do the 
medical research which you have described?
    Dr. Fischbach. I think this is a very important question. I 
think we both would like to comment.
    My strong feeling is there are other sources. The key 
question is are they adequate? That is a matter for future 
research. Stem cells have been discovered in many tissues. 
Specifically, certain types of stem cells have been discovered 
in the brain, but we do not know as much about them in terms of 
how renewable they are and what the limits, and range of their 
capabilities are. And as I said, we need to maximize both of 
those.
    My current reading of the literature is by neither one of 
those criteria are they as adequate as embryonic stem cells.

                          SUPPLY OF STEM CELLS

    Senator Specter. Dr. Spiegel, you had made the comment 
about diabetes. What is your professional judgment as to 
whether there would be an adequate supply of stem cells to work 
on all of these terrible ailments if we do not utilize 
discarded embryos?
    Dr. Spiegel. As I indicated in my comments, I think this 
would be tying one hand behind our back and tying the hands of 
our most superb scientists who are eager to pursue research in 
this area.
    I indicated that I absolutely agree that we do need to 
pursue vigorously the adult stem cell area. We convened 
scientists and experts, from all over the country just this 
past couple of weeks at the NIH to review stem cell research. 
They were essentially unanimous in their feeling that we 
vigorously need to pursue the human pluripotent stem cell in 
addition to the adult stem cell question. This is particularly 
true in areas such as the pancreas where, unlike the blood 
system and unlike the intestinal system, for example, there is 
as yet no definitive evidence for adult pancreatic stem cells.
    Senator Specter. How many scientists were convened at the 
meeting which you referred to?
    Dr. Spiegel. On the order of 200, including some of the 
really most superb scientists in the area of developmental 
biology and stem cells.
    Senator Specter. Well, my red light just went on, so I am 
going to turn to our very distinguished ranking member, Senator 
Harkin.

                Opening Statement of Senator Tom Harkin

    Senator Harkin. Thank you very much, Mr. Chairman. I 
apologize for being a little late. I will just make a 
statement. I know other people have been here before me, and I 
will defer to them for their questions. Then I will come back 
and ask my questions after them.
    As you know, Chairman Specter and I have a very close 
bipartisan partnership when it comes to medical research. We 
both strongly believe that medical research holds great hope 
for improving the health and well-being of millions of our 
fellow Americans. I want to compliment and congratulate our 
chairman, Senator Specter, for his very bold leadership on this 
issue of biomedical research. It is a welcome breath of fresh 
air amid some of the confusion and I think some of the 
misinformation that has been bandied about on different aspects 
of biomedical research, but especially the one that we are here 
today to talk about and that is stem cell research. So, I just 
want to thank you, Mr. Chairman, for your leadership on this 
issue.
    I am late because I was just at another hearing on defense. 
You were there briefly and I knew you had to leave before I 
did. We were talking about our defense structure, and I was 
struck by the fact that here we are putting enormous amounts of 
money in to protecting our country, keeping our country strong. 
It occurred to me, as I was listening to the amount of money 
that we are now asking for more missile defense and smart bombs 
and things like that, that it came back to me a little known 
fact, and that is in the last 5 years, we have spent more money 
on military research and development than we have spent on all 
biomedical research since the turn of the century. That shocks 
a lot of people when I tell them that, but it is true. You can 
add it up. I mean everything. I mean smallpox, diphtheria, 
polio, cancer, everything that we have spent all this money on 
since the turn of the century, we have spent more than that on 
military R&D in the last 5 years. So, we have smart bombs and 
we have smart missiles and we have a strong defense, and I am 
thankful for that.
    We need the smart bombs and the smart missiles to knock out 
Parkinson's disease and ALS and spinal cord injuries and 
juvenile diabetes, all of the ailments that I believe can be 
overcome if we just really focus on research. So, when people 
tell us under your leadership that we are putting too much 
money in NIH, I always roll out that fact of what we have done 
in the military and how little we are doing in medical 
research.
    In this area of stem cell research, it holds so much 
promise. Of course, there are different areas of getting stem 
cells: adult stem cells, embryonic, fetal. To say we are only 
going to use one source is, as you said, Dr. Spiegel, to tie 
one hand behind our backs. We need to unleash an army, unleash 
an army of researchers all over this country to use this new 
resource and to bring us forward in developing stem cell 
research to the point where I believe it is going to take us. 
No one can guarantee it. No one can guarantee what it is going 
to lead to, but to blindly stop it now or to tie one hand 
behind our back I think is basically to doom millions of 
Americans and people around the world to perhaps lives that 
they might not otherwise be leading.
    So, I am grateful that you are here. I just wanted to make 
that statement to say that we need to push the boundaries.
    Again, we need the Federal guidelines according to the 
Bioethics Commission and their findings and the statement they 
came out with. As you probably have pointed out in your 
testimony, private research is going on in this area. But to 
the extent that it is done privately and done chaotically, it 
is going to push back the time when we have the kind of 
discoveries that would come if we did it in a regulated, 
ethical manner. If we did it that way and we were able to guide 
and direct this research, I believe the fulfillment of the 
promise would come much more rapidly than if we just leave it 
in a chaotic system out there with no supervision and no 
ethical controls.
    Mr. Chairman, thank you for affording me this time.
    Senator Specter. Well, thank you very much, Senator Harkin. 
Thank you for your good words and for our bipartisan 
partnership.
    Our practice is to take Senators in order except for the 
ranking member because of the structure of the Senate. We turn 
now to Senator Reid.

                Opening Statement of Senator Harry Reid

    Senator Reid. Mr. Chairman, I appreciate very much the 
bipartisan tone that this subcommittee has set. It is easy to 
talk in a bipartisan tone in a subcommittee hearing, but you 
and Senator Harkin have done that on the Senate floor. An 
example was the budget battle that we won on the Senate floor 
and lost in conference, but I admire and respect the work that 
you did on the budget.
    I had, Dr. Fischbach, Dr. Spiegel, the good fortune this 
morning to spend some time with Dr. Ruth Kirschstein, the 
acting Director of NIH. We talked about a number of things, but 
one of the things that we spoke about is her experience. She 
talked about her work in the early 1950's when they knew they 
were getting close to finding a cure--I do not know if ``cure'' 
is the right word, but a way to prevent polio. And growing up 
in that era, I can remember that was the thing that we were 
most afraid of. It was not the nuclear holocaust. We were 
afraid of getting polio, all the young boys and girls. And she 
said that they knew that they had in their sight a way to cure 
polio.
    We have talked this morning about diabetes, Alzheimer's, 
Parkinson's, and a number of other things. Do either of you 
gentlemen think that we have in sight a cure for some of these 
diseases that we are so afraid of today: Parkinson's, 
Alzheimer's? Half the money we spend in nursing homes in 
America today is spent on those two diseases.
    Dr. Fischbach. I think there is enormous hope for real 
advances and in some of the cases, real cures in the sense of 
stopping the progression of a disease. The analogy with polio 
is an interesting one and quite stimulating to think about. 
That was due to the infection by a specific organism and a 
vaccine worked a miracle.
    I think in the disorders I am most familiar with, the 
complexity of the brain is a hurdle to overcome.
    Senator Reid. And you made that very clear in your 
testimony.
    Dr. Fischbach. But I am as optimistic now as I have been 
throughout my scientific career in the last 30 years that we 
will be able to stop and in some cases reverse disorders of the 
brain that are associated with loss of nerve cells.
    Senator Reid. Dr. Spiegel, do you agree with Dr. Fischbach?
    Dr. Spiegel. I would like to make two points in response to 
your question.
    First, I want to indicate, outside of the context of the 
immediate subject, that we are not focused only on cures. Those 
are incredibly important, but you mentioned the polio vaccine. 
Prevention is critical, and we are very heavily focused on 
prevention of many diseases, including type 1 diabetes.

                            TRANSPLANTATION

    In terms of cures, the situation for some of the diseases--
I would say type 1 diabetes and liver failure--the situation is 
particularly interesting with respect to transplantation. 
Transplantation is a potential cure, as I indicated, for both 
of these diseases, and with immunology advances, we have the 
possibility of not just substituting one disease, complications 
of treatment with immunosuppressive drugs, for another, but 
rather of allowing these individuals to lead a relatively 
normal life.
    The problem, though, is that despite the best efforts of 
increasing organ donation, there simply is an inadequate supply 
to provide these cures. And this is where stem cell research is 
so important. I do not want to over promise, and I think we 
always have to be very cautious. I think Senator Harkin made 
the point we cannot guarantee it. But certainly, if we do not 
do the research, it will not happen. That is guaranteed.
    Senator Harkin. That is guaranteed.
    Senator Reid. Let me say that in biomedical research I 
certainly do not want anyone's hands tied behind them. We have 
scientists all over the United States and the direction that 
they take and the grants that they write that are okayed by you 
folks at NIH and the work that you do at NIH--I want no hands 
tied behind anyone's back. I can speak, as someone who has a 
pro-life voting record here in the Senate, that as far as I am 
concerned, there are no holds barred on what you should be able 
to do using embryos, any other place that you feel that you can 
get stem cells. I am all behind you and I think that the work 
that you have done has been exemplary and I look forward to 
joining hands with the members of this subcommittee, the 
members of the full committee, and the Senate in giving you all 
the resources that you need to help people that really need 
help.
    Dr. Spiegel. I am certainly grateful for your support and 
for that of the chairman and Senator Harkin. It has been 
incredibly important throughout. We appreciate the support for 
NIH in general and on this issue. I want to just articulate 
that.
    Senator Reid. Mr. Chairman, I have other obligations. I 
wanted to come and make this brief statement. Again, it is a 
great panel. I am sorry I cannot be here for the rest of it.
    Senator Specter. Well, thank you very much, Senator Reid, 
and thank you for your comments about the funding. I think it 
is worth just a sentence or two to note that in the past 3 
years, this subcommittee has taken the lead on increasing NIH 
funding by more than $5 billion. It was at $13 billion, and in 
the last 3 years, we have added some $5 billion. And we have 
done that over the votes which we have not gotten extra funding 
for, but we have established priorities. One of my frequent 
statements is that the National Institutes of Health are the 
crown jewels of the Federal Government. Some say the only 
jewels of the Federal Government.
    But there is nothing more important than health. Health is 
number one. We have a very large group here today, as we have 
had with juvenile diabetes and as we have had with cancer and 
as we will have later in a couple of weeks with amyotrophic 
lateral sclerosis. The public is very concerned about medical 
research and about curing Parkinson's or Alzheimer's, these 
dreaded diseases, or heart disease. And this subcommittee 
intends to do what it can to get that done.
    Senator Murray.

               Opening Statement of Senator Patty Murray

    Senator Murray. Well, Mr. Chairman, thank you very much for 
having this hearing. I want to just personally thank you, Mr. 
Chairman, for all the time and effort you have spent on this 
particular issue. I think the work you have done has really 
built a balanced and a fair hearing record on stem cell 
research and I think has moved us in a very positive direction.
    And I want to thank Senator Harkin as well for his work on 
this and his excellent statement as well.
    You know, my father had multiple sclerosis. He was 
diagnosed when I was 15. He lived most of my life in a 
wheelchair, and I know the personal hope that he had many times 
and the disappointments he had throughout his lifetime.
    This research, obviously, will not help him. He passed away 
several years ago, but I know of thousands and thousands and 
thousands of families who are counting on us to do the right 
thing so that there is hope in their future.
    So, I really want to thank you, Mr. Chairman, Senator 
Harkin, for your work on this, and hope that we can continue to 
push the limits on this and find some resolution not only for 
MS, for Parkinson's, Alzheimer's, for so many other exciting 
opportunities that are out there.
    I have listened to the people who oppose this, and I think 
part of what we are hearing is that they have little 
understanding of some of the bioethical requirements that NIH 
has regarding stem cell research. If you could just take a 
minute and outline for us what ethical standards you do adhere 
to in this research, I would appreciate it.
    Dr. Fischbach. Well, we can both take a shot at that. I 
think the guidelines being proposed by the NIH address these 
really awesome ethical issues in terms of ensuring informed 
consent on the part of those who donate embryos, ensuring the 
utmost care in the scientific use and complete dedication to 
the full informed use of the data. They are complex and they 
involve the use of the tissue and the circumstances under which 
it can be used. It involves oversight to make sure that these 
guidelines are followed. It involves review by scientists, lay 
people on council, and NIH officials. So, I think they are a 
rather complete examination of the legal, the ethical, and the 
social issues, as well as the scientific issues involved.
    Senator Murray. Dr. Spiegel?
    Dr. Spiegel. The only point I would add is very much in 
line with what the chairman had to say, and that is the 
guidelines try to ensure to totally dissociate the issue of the 
availability of embryos from the issue of the use of the stem 
cells. Informed consent, as we have heard, no monetary 
remuneration, nothing that would encourage this, in other 
words, to totally dissociate the two issues and to allow real 
public oversight and scrutiny of the way that these stem cells 
would be used. I think that is what is vitally important.
    Senator Murray. Thank you.
    We all understand that without Federal standards and 
guidelines the potential for abuse of this research is 
significant. I think the recent front-page article in the 
Washington Post made that point very clear.
    From your opinion, if there is no Federal involvement in 
there, what would the ramifications be?
    Dr. Fischbach. My own opinion is that the Federal 
involvement is critical for the ethical conduct of this type of 
science. Science conducted behind closed doors is behind closed 
doors, and we cannot regulate that at all or bring the most 
disinterested parties to the table to think about the 
ramifications.
    Dr. Spiegel. Certainly the ethical issues are paramount, 
but the other point we have alluded to several times. There is 
a lot of work going on in the private sector, and at some 
level, that is fine. But only with Federal funding, and in 
addition, the oversight, are we going to really see all the 
talented scientists who want to work in this area contribute 
and give us their capabilities.
    Senator Murray. Would there also be ramifications for who 
would have access to the research and the results of that 
research if the Federal Government is not involved?
    Dr. Fischbach. At the present time, we do not have any 
reason to believe there would be limited access. But this is a 
very dynamic, rapidly moving field, and there is always the 
potential for restriction of access. But we do not--perhaps Dr. 
Spiegel would like to add--see any evidence at the present time 
for restriction of access.
    Dr. Spiegel. I will just leave it at that.
    Senator Murray. All right. Thank you very much, Mr. 
Chairman.
    Senator Specter. Thank you, Senator Murray.
    Before moving on to the next panel, just a comment about 
the time table. It was on January 15 of last year that the 
Health and Human Services General Counsel determined that the 
use of Federal funds to support stem cell research did not 
violate the appropriations ban. It was not until April 8 that a 
meeting was held with public and private groups to discuss the 
guidelines. On December 2, there were draft guidelines 
published with the comment period for 60 days, which would have 
run out in early February. That was extended to February 22.
    I know there are a great many comments, but there needs to 
be a sense of urgency to getting this done because every day is 
a day lost which could be saving lives. This subcommittee has 
placed a very high priority on this subject, having five 
hearings, more than we have had on any other subject, because 
of the importance of focusing public attention and getting it 
moving. So, we would urge you to apply a sense of urgency here 
and get it done.
    Dr. Fischbach. I think we all absolutely agree with that.
    Senator Specter. We will not hold you any longer, so you 
can go back to your offices to get this done.
    Thank you very much.
    We will now turn to our second panel: Senator Sam 
Brownback, Dr. Frank Young, and Ms. Mary Jane Owen.
    In our previous hearings, we have heard from witnesses who 
have been opposed to eliminating the Federal ban. In our last 
hearing, we heard from Congressman Jay Dickey who opposes 
Federal funding on embryos in stem cell research. We consider 
it very important to have balance in the presentation of the 
views so that all of the information can be available to the 
Senate when we debate this matter and render our judgments and 
views can be available to the American people.
STATEMENT OF HON. SAM BROWNBACK, U.S. SENATOR FROM 
            KANSAS
    Senator Specter. Our lead witness here is Senator Sam 
Brownback, elected to the Senate in 1996. It says to take the 
place of Senator Dole. I do not know that anybody can take the 
place of Senator Dole.
    Senator Brownback. That is not possible.
    Senator Specter. Senator Brownback and I agree on many, 
many matters. We disagree on this one.
    We share common roots. I was born and raised in Kansas and 
sometimes like to think of myself as Kansas' third Senator.
    Senator Brownback. We do too.
    Senator Specter. I would have to put myself fourth behind 
Senator Dole whose presence is still on the scene.
    Senator Brownback serves on the Commerce, Science, and 
Transportation Committee, as well as the Health, Education, 
Labor, and Pensions Committee. He chairs the Subcommittee on 
the Middle East for the Foreign Relations Committee where he 
has been very active.
    He grew up in Parker, Kansas, has a law degree from the 
University of Kansas and his bachelor's from Kansas State.
    Welcome, Senator Brownback, and we look forward to your 
testimony.
    Senator Brownback. Thank you, Senator Specter, Senator 
Harkin, Senator Murray. Thank you for allowing me the 
opportunity to speak in front of you this morning to testify. I 
do not know that I would consider myself the lead witness of 
this panel. You have a former FDA Commissioner and I think you 
will hear from Ms. Owen a very clear statement as well.
    Senator Specter. In the Senate, Senator Brownback, you are 
the lead witness.
    Senator Brownback. Well, you are kind.
    I appreciate this opportunity. I have great respect for 
your thoughts. I have a great respect for your heart on these 
matters and on all matters, but on this one, we do disagree. I 
will be highlighting why I believe this issue should be handled 
another way, that we should be funding aggressively research in 
the area of adult stem cell research.
    I have supported this panel's efforts to double NIH funding 
over a 5-year time period. I think that is critically 
important, very important, that we fund the science.
    I also think it is very important that we have a critical 
ethical view as to what we are doing. At the center of this 
debate, at the very center of this debate, is the real 
question: Is the young human person or property? And that is at 
the very center of what this debate is about, and I want to 
articulate that further. But that is the center of what this 
question is about.
    We are here today to discuss some of the issues that have 
been raised regarding Federal funding for human embryonic stem 
cell research. My position on this is that Federal funding of 
human embryonic stem cell research is illegal, is immoral, and 
it is unnecessary. And I want to go into those three issues.
    As this subcommittee is well aware, Congress outlawed 
Federal funding for harmful human embryo research in 1996 and 
has maintained that prohibition ever since. The ban is broad 
based and specific. Funds cannot be used for--and I quote now 
from the act--``research in which a human embryo or embryos are 
destroyed, discarded, or knowingly subjected to risk of injury 
or death.'' The intent of Congress is clear. If a research 
project requires the destruction of human embryos, no Federal 
funds should be used for this project.
    NIH recently published its proposed guidelines to 
circumvent this language. Marcy Wilder, who I might add was a 
former legal director for the National Abortion Rights Action 
League and now Associate General Counsel at the Department of 
Health and Human Services, wrote a legal opinion that sought to 
justify the research being proposed by the NIH.
    Yet, despite this fig leaf of the HHS legal opinion, the 
fact remains that this research is illegal. It is illegal for 
this reason. The deliberate killing of a human embryo is an 
essential component of the contemplated research, and without 
the destruction of the embryo, the proposed research would be 
impossible.
    Now, despite the legal gyrations of HHS, this is a point 
that is not lost on the National Bioethics Advisory Commission. 
Although their conclusions are wrong in my estimation, NBAC 
observes in their recommendations to the President the 
inconsistencies of the HHS legal opinion and the NIH 
recommendations. Accordingly, NBAC recommends an approach that 
is at once both more honest and more heinous.
    Which brings us to a discussion of the morality of this 
legislation. The NBAC position is given legislative form in the 
Senate bill that we are discussing today, S. 2015, currently 
referred to the Health, Education, Labor, and Pensions 
Committee on which I sit. Among other and perhaps more serious 
policy changes, it would constitute a lifting of the ban on 
human embryo research. In brief, the Stem Cell Research Act of 
2000 seeks to allow Federal funding for researchers to kill 
living human embryos. Under this bill, Federal researchers 
would be allowed to obtain their own supply of living human 
embryos which they would then be allowed to kill for research 
purposes.
    Now, the very act of harvesting cells from live human 
embryos results in the death of the embryo. Therefore, if 
enacted, this bill would result in the deliberate destruction 
of human embryos.
    This bill will even violate current Federal policy on fetal 
tissue in my estimation, which allows harvesting of tissue only 
after an abortion was performed for other reasons and the 
unborn child is already dead. Under this bill, the Federal 
Government will use tax dollars to incentivize the killing of 
live embryos for the immediate and direct purpose of using 
their parts for research.
    Taxpayer funding of this research is problematic for a 
variety of reasons. First among those concerns is that if 
Congress were to approve this legislation, it would officially 
declare for the first time in our Nation's history that 
Government may exploit and destroy human life for its own or 
somebody else's purposes.
    Now, this research is also problematic because it would use 
Federal tax dollars to allow the Government to procure and 
therefore own a vast supply of living human embryos. Now, this 
notion of ownership, particularly by the Federal Government, of 
other human beings I believe is deeply disturbing.
    The bill even allows Federal funding for destructive 
research using embryos created by cloning so long as that does 
not result in--quote from the legislation--``the reproductive 
cloning of a human being.'' On the one hand, this is an 
attempt, it seems to me, to authorize the critical issue of 
human cloning when what is really needed is the continuation of 
the full public debate on this point. On the other hand, this 
approach recognizes that for the purposes of possible clinical 
applications, particularly to avoid possible tissue rejection, 
human cloning is the logical next step, or so-called 
therapeutic cloning. This means that live embryos created by 
researchers can be experimented on and destroyed but cannot be 
allowed to survive to live birth. That is prohibited in the 
legislation, which seems to create a new class of human beings 
who under the law will simply not be allowed to live.
    I think history has already taught us some important 
lessons on separating human beings into different classes. The 
Dred Scott case issue was on that point where Dred Scott held 
that African Americans at that time ``had no rights which the 
white man was bound to respect.''
    Now, I am not suggesting at all that that is the intent of 
the chairman or of the ranking member of this committee, but if 
you look into what the effect of what would happen with this 
legislation, I think we get terribly close to those same issues 
that we have seen throughout our history, and it is deeply 
troubling to me.
    My final point is that the human embryonic stem cell 
research is unnecessary, and this is a key point because I want 
to see people healed, which is what the chairman is after, 
which is what the ranking member is after. We want to see these 
diseases no more hit our people or anybody else across the 
planet. That is our heart and that is our objective, and on 
that we all agree. That is why I am saying this is not 
necessary. We can go on the areas of legitimate research into 
adult stem cells which do not create the moral and ethical 
difficulties that we do in human embryo stem cell research.
    Dr. Young will testify more about what is taking place in 
this area in the research now.
    In the past, Congress has increased funding for NIH. New 
advances in adult stem cell research, being reported almost 
weekly, show more promise than destructive embryo research. And 
I want to give just a few of these.
    Just this past February, writing in the journal Neurons, 
scientists at Children's Hospital in Boston announced that they 
had successfully generated new brain cells in birds using adult 
neural stem cells.
    Writing in the March 20 issue of Nature Medicine, 
University of Florida scientists reported that they reversed 
insulin-dependent diabetes in mice by using adult pancreatic 
stem cells. Their quote, ``The next step is to take this into 
humans, they say.'' They have extracted and cultivated viable 
adult brain stem cells from eight living human patients 
undergoing surgery for other reasons.
    Writing in the March 17 issue of Science, University of 
Toronto researchers reported they found retinal stem cells in 
the eyes of adult mice, cows, and humans, and have shown that 
they can be used to produce new neurons presenting the 
prospects of repairing or regenerating damaged retinas and 
restored sight.
    In April, Dr. Karen Obote and colleagues at Children's 
Hospital in Boston reported at a meeting of the American 
Association of Neurological Surgeons in San Francisco that they 
can use adult neural stem cells to target brain tumors in mice. 
The cells could be used to reduce or kill the tumors by 
delivering new genes or carrying cancer drugs to where they are 
needed.
    Clearly we must continue to fight and help cure diseases 
and to eliminate suffering. I have got a chart over here on 
some of these areas that are just now coming out in the adult 
stem cell area.
    The other issue that they--we do not have the ethical/moral 
problems. We do not have the problems of the immunity system 
rejecting cells, if we use our own adult stem cells.
    Mr. Chairman, ranking member of this committee, I have 
great respect for your heart and your desire in here, and I 
think on that we completely agree. And I have no question about 
your motives in doing this. I just think we are crossing an 
enormous issue here of looking at this human life as a piece of 
property rather than as a person.

                           PREPARED STATEMENT

    And we do not need to go there. We can address these issues 
with adult stem cells. We can address these issues with 
increased funding in NIH and increased funding in these key 
areas that are showing so much promise right now to address 
these terrible diseases. That is the way we can go together. 
That is where our hearts can be pure and we can do the right 
thing and feel good about it rather than accepting the 
deliberate killing of one human innocent life in order to help 
another, which has never been right throughout human history.
    I thank you, Mr. Chairman, for your patience and your 
willingness to hear me out on this point.
    [The statement follows:]
              Prepared Statement of Senator Sam Brownback
    Thank you Mr. Chairman and members of the Subcommittee for the 
opportunity to testify on this very important issue.
    We are here today to discuss some of the issues that have been 
raised regarding federal funding of human embryonic stem cell research. 
My position is that federally funded human embryonic stem cell research 
is illegal, immoral and unnecessary.
    As this subcommittee is well aware, Congress outlawed federal 
funding for harmful embryo research in 1996 and has maintained that 
prohibition ever since. The ban is broad-based and specific; funds 
cannot be used for ``research in which a human embryo or embryos are 
destroyed, discarded or knowingly subjected to risk of injury or 
death.'' The intent of Congress is clearif a research project requires 
the destruction of human embryos no federal funds should be used for 
that project.
    The language of the ban is clear--as are the lessons from history. 
The Nuremberg Code (from the trials of Nazi war criminals) states, ``No 
experiment should be conducted where there is an a priori reason to 
believe that death or disabling injury will occur.'' Also, the World 
Medical Association asserts in the Declaration of Helsinki that, ``In 
research on man, the interest of science and society should never take 
precedence over considerations related to the well-being of the 
subject.'' Further, ``concern for the interests of the subject must 
always prevail over the interest of science and society.'' As well, it 
is hardly necessary to note that it has already been proven 
biologically that an embryo constitutes human life (MERCK MANUAL and 
NBAC recommendation to the President, ``Ethical Issues in Human Stem 
Cell Research'').
    Pope John Paul II, quite independent of government law, and 
particularly of our own ban on embryo research states, in his 
encyclical, Evangelium Vitae, ``[The] evaluation of the morality of 
abortion is to be applied also to the recent forms of intervention on 
human embryos which, although carried out for purposes legitimate in 
themselves, inevitably involve the killing of those embryos. This is 
the case with experimentation on embryos, which is becoming 
increasingly widespread in the field of biomedical research and is 
legally permitted in some countries. Although ``one must uphold as 
licit procedures carried out on the human embryo which respect the life 
and integrity of the embryo and do not involve disproportionate risks 
for it, but rather are directed to its healing, the improvement of its 
condition of health, or its individual survival'', it must nonetheless 
be stated that the use of human embryos or fetuses as an object of 
experimentation constitutes a crime against their dignity as human 
beings who have a right to the same respect owed to a child once born, 
just as to every person.
    ``This moral condemnation also regards procedures that exploit 
living human embryos and fetuses--sometimes specifically ``produced'' 
for this purpose by in vitro fertilization--either to be used as 
``biological material'' or as providers of organs or tissue for 
transplants in the treatment of certain diseases. The killing of 
innocent human creatures, even if carried out to help others, 
constitutes an absolutely unacceptable act.''

             THE NATIONAL INSTITUTES OF HEALTH AND THE HHS
    NIH recently published its proposed guidelines to circumvent the 
congressionally imposed ban on destructive human embryo research.
    Marcy Wilder, former Legal Director of the National Abortion Rights 
Action League, and now Associate General Counsel at the Department of 
Health and Human Services wrote a legal opinion that sought to justify 
the research being proposed by the NIH. Yet despite the fig leaf of the 
HHS legal opinion, the fact remains that this research is illegal. It 
is illegal for this reason: the deliberate killing of a human embryo is 
an essential component of the contemplated research; and without the 
destruction of the embryo the proposed research would be impossible.
    Despite the legal sophistry of HHS, this is a point that is not 
lost on the National Bioethics Advisory Commission. Although there 
conclusions are wrong, NBAC observes in their recommendation to the 
President the inconsistency of the HHS legal opinion and the NIH 
recommendations. Accordingly, NBAC recommends an approach that is at 
once both more honest--and more heinous.

    WHICH BRINGS US TO A DISCUSSION OF THE MORALITY OF THIS RESEARCH
    The NBAC position is given legislative form in Senate Bill 2015. S. 
2015, currently referred to the Health Education Labor and Pensions 
Committee, on which I sit would, among other and perhaps more serious 
policy changes, constitute a lifting of the ban on human embryo 
research.
    In brief, the ``Stem Cell Research Act of 2000'' seeks to allow 
federal funding for researchers to kill living human embryos.
    Under this bill federal researchers would be allowed to obtain 
their own supply of living human embryos, which they would then be 
allowed to kill for research purposes.
    The very act of harvesting stem cells--or perhaps more accurately 
constructing so-called embryonic stem cells--from live human embryos 
results in the death of the embryo. Therefore, if enacted, this bill 
would result in the deliberate destruction of human embryos.
    This bill even violates current federal policy on fetal tissue, 
which allows harvesting of tissue only after an abortion was performed 
for other reasons and the unborn child is already dead. Under this 
bill, the federal government will use tax dollars to kill live embryos 
for the immediate and direct purpose of using their parts for research.
    Taxpayer funding of this research is problematic for a variety of 
reasons. First among those concerns is that, if Congress were to 
approve S. 2015, it would officially declare for the first time in our 
nation's history that government may exploit and destroy human life for 
its own or somebody else's purposes.
    This research is also problematic because it would use federal tax 
dollars to allow the government to procure, and therefore ``own,'' a 
vast supply of living human embryos. The notion of ``ownership,'' 
particularly by the Federal government, of other human beings is deeply 
disturbing.
    The bill even allows federal funding for destructive research using 
embryos created by cloning, so long as this does not result in ``the 
reproductive cloning of a human being.'' On the one hand, this is an 
attempt to authorize the critical issue of human cloning by stealth; 
when what is really needed is the continuation of the full public 
debate. On the other hand, this approach recognizes that for the 
purposes of possible clinical applications, particularly to avoid 
possible tissue rejection, human cloning is the logical next step--so-
called, ``therapeutic cloning.'' This means that live embryos created 
by researchers can be experimented on and destroyed, but allowing them 
to survive to live birth is prohibited. The bill defines a new class of 
human beings who, under the law, will simply not be allowed to live.
    History has already taught us the lessons of separating human 
beings into different classes. The Dred Scott case held that African-
Americans, ``had no rights which the white man was bound to respect.'' 
Today, 143 years later, it is precisely this same argument which is now 
being used to legitimate the destruction of human embryos. It is the 
contention of S. 2015, as well as the NIH, that human embryos do not 
have rights which people, already born, are bound to respect.

         HUMAN EMBRYONIC STEM CELL RESEARCH IS ALSO UNNECESSARY
    There are legitimate areas of research which are showing more 
promise than embryonic stem cell research and which do not create moral 
and ethical difficulties. Dr. Frank Young, former FDA commissioner, 
under Ronald Reagan has detailed some of the alternatives in his 
testimony which you will hear later.
    In the past, Congress has increased funding for NIH. New advances 
in adult stem cell research, being reported almost weekly, show more 
promise than destructive embryo research.
    Just this past February, writing in the journal Neuron, scientists 
at Children's Hospital in Boston announced that they successfully 
generated new brain cells in birds using adult neural stem cells 
(MSNBC, Feb. 23).
    Also, writing in the March 2000 issue of Nature Medicine, 
University of Florida scientists reported that they reversed insulin-
dependent diabetes in mice by using adult pancreatic stem cells. ``The 
next step is take this into humans,'' they say. They add that they have 
extracted and cultured viable brain stem cells from the hippocampus of 
eight living human patients undergoing surgery for other reasons 
(Reuters, February 28).
    Writing in the March 17 issue of Science, University of Toronto 
researchers reported that they found retinal stem cells in the eyes of 
adult mice, cows and humans and have shown that they can be used to 
produce new neurons, presenting the prospect of repairing or 
regenerating damaged retinas and restoring sight (UniSci, March 17).
    In April, Dr. Karen Aboody and colleagues at Children's Hospital in 
Boston report at a meeting of the American Association of Neurological 
Surgeons in San Francisco that they can use adult neural stem cells to 
target brain tumors in mice. The cells could be used to reduce or kill 
the tumors, by delivering new genes or carrying cancer drugs to where 
they are needed (Reuters, April 10).
    Clearly we must continue to fight to help cure disease and to 
alleviate suffering. However, it is never acceptable to deliberately 
kill one innocent human being in order to help another. When did it 
become acceptable to use an evil means to pursue a good end, even a 
great one? Doesn't the so-called good end actually become bad by using 
bad means? If we manage the cure of some diseases and the betterment of 
some aspects of bodily health by means that involve the killing of the 
most defenseless and innocent of human beings, we will rightfully be 
judged harshly by history as having sought some benefits at the expense 
of our humanity and moral being. The twentieth century has already 
taught these lessons--are we to ignore them at the beginning of this 
century? Or to put it another way, as George Santayana once said, 
``Those who cannot remember the past are condemned to repeat it.''
    Thank you, Mr. Chairman.

    Senator Specter. Well, thank you very much, Senator 
Brownback for your testimony. We appreciate your sincerity and 
your position, and we will have some questions on the morality 
and the alternatives to human stem cells. But first we are 
going to go to our other two panelists.

STATEMENT OF FRANK YOUNG, M.D., Ph.D., FORMER 
            COMMISSIONER, FOOD AND DRUG ADMINISTRATION, 
            DEPARTMENT OF AGRICULTURE
    Senator Specter. I would like to call now on Dr. Frank 
Young, Commissioner of the Food and Drug Administration from 
1984 to 1989. Previously served as Deputy Assistant Secretary 
for Health in the Bush administration and as Director of the 
Department of Health and Human Services of Emergency 
Preparedness. He was Dean of the University of Rochester 
Medical School, a microbiologist by training. He now serves 
also as pastor of adult ministries at the Fourth Presbyterian 
Church in Bethesda, MD.
    It is a 1-year anniversary, Dr. Young, since you testified 
on April 26 last year. So, welcome back.
    Dr. Young. Thank you very much, Mr. Chairman and the 
distinguished Senator Harkin. I thank you both from the bottom 
of my heart for your years of steadfast support of the National 
Institutes of Health and biomedical sciences. I have had a 
chance to work with both of you in the past and I know of your 
genuine commitment. I only urge you on in the support of this.
    As a pastor, it would be remiss of me not to remind all of 
us that we are totally biodegradable. I have conducted more 
memorial services than I would like to admit now. So, as we 
look at curing disease, we have to recognize that we each at 
one day will ourselves die. And it is with that perspective of 
the role of research that I would like to put my comments 
before you today.
    I also want to thank you, Mr. Chairman, for the excellent 
way in which you have held these hearings. You have brought 
forth witnesses of all persuasions, a rare event in 
Washingtonville. And I thank you for what you have done in that 
way.
    Two quotes might frame this, and I have brought for the 
press table the copy of the Science issue, which I am sure you 
have, if not, I can provide you with more of these.
    One is cited in there by Rabelais in 1532 who stated, 
``Science without conscience is but death of the soul.'' So, we 
have to look at where and how we develop our advances.
    And more recently Robertson stated in a California Law 
Review--and I quote--``Science is not an unmitigated blessing. 
It is expensive and its discoveries, like the Tree of Knowledge 
in Eden, expands man's capacity for evil as well as good. More 
knowledge is not a good in itself, nor is it necessarily 
productive of net good. Society as the provider of resources, 
the bearer of costs, the reaper of the benefits has an 
overriding interest in the consequences of science and hence 
the direction and routes that research takes,'' which you are 
doing today.
    I will just summarize my statement.
    Senator Specter. That is fine, Dr. Young.
    Dr. Young. There are these issues that I think are 
critical.
    First, we are dealing with utilitarian ethics. We are 
focusing on the decision of whether or not to use the promise 
of potential good for the end of a life that we know is 
essentially here. And that decision, as the Senator said, is 
not one to be taken lightly. So, as we focus our energy, it is 
at what price is humanness in the 21st century.
    My worry, as both a physician and a pastor, is the degree 
of violence that we see on human beings. We just saw it the 
other day at the National Zoo. We see it in Bosnia. We have 
seen it in Kosovo. We have seen it in many places. And I 
believe the sacredness of human life is at the very heart of 
what we will be in the 21st century.
    I would suggest that as we do research, we take research in 
a cautious fashion and first of all do no harm. As the Senator 
summarized, we are blessed with the capacity to have stem cells 
in most every organ in the body, and it was also stated in the 
former panel. These enable us to regenerate our skin, to be 
able to have liver regeneration at times. These are very 
important parts that are just being discovered now.
    When I last testified before you, Mr. Chairman, we had very 
little knowledge of adult stem cells. One of the most 
interesting ones was one in mice recently where they were able 
to take from bone marrow, the adult bone marrow, and isolate 
stem cells in as few as 50 stem cells added to the animal with 
liver disease was able to produce a change in symptomatology.
    The stem cells from an adult have these advantages. One, it 
does not carry the moral baggage. Two, they are readily 
isolated particularly from bone marrow. Three, they can be 
isolated and preserved from cord blood, and in fact, one 
company now is advocating at a small fee the preservation of 
cord blood stem cells for use of the individual later on. 
Therefore, it is not a need to destroy embryos that are 
``spare'' at this time.
    Four, I would like to say that we have been lax in the 
Federal Government in regulating in vitro fertilization 
clinics. Some of the margins that we see now are related to 
this very expensive therapy unregulated that does generate 
these ``excess'' individuals. And if we are not careful, they 
can easily regenerate and develop more of ``spare'' embryos.
    Next, it is critical to realize that the immunological 
capacity and also the relative ease of differentiating the 
adult stem cell vis-a-vis the pluripotent stem cell from 
embryos may provide an advantage. As the first panel said, 
these are promises. Remember President Nixon led a war on 
cancer in 1968. Unfortunately, we are still a ways from there.
    Research is critical but research crossing a line of 
destroying human life I believe borders on not only the 
unnecessary but the immoral at this time.
    I thank you, sir, for the privilege of testifying again.
    Senator Specter. Thank you very much, Dr. Young, for your 
testimony. Thank you.

STATEMENT OF MARY JANE OWEN, M.S.W., EXECUTIVE 
            DIRECTOR, NATIONAL CATHOLIC OFFICE FOR 
            PERSONS WITH DISABILITIES
    Senator Specter. We now turn to Ms. Mary Jane Owen, 
Executive Director for the National Catholic Office for Persons 
with Disabilities since 1991. In 1986, Ms. Owen established the 
Disability Focus, Incorporated which is an organization 
promoting a disability perspective on all social policy and 
advocating for the appointment of qualified people with 
disabilities at all levels of Government and business. She 
received her master's degree in social work from the University 
of California at Berkeley.
    We welcome you here, Ms. Owen, and if we may be of any 
assistance to you on the hearing or anything else, let us know. 
We look forward to your testimony.
    Ms. Owen. Thank you. That is very kind of you to offer your 
assistance.
    I am so gratified to be here to be able to participate in 
the testimony that you are hearing about this very critical 
issue.
    If I overstay my time in terms of talking, please forgive 
me. It is not out of disrespect for the lights. As a blind, 
partially hearing person, it is very hard to stop me when I get 
going. So, I simply in advance say please give me warning if I 
have extended----
    Senator Specter. Ms. Owen, we will give you a little slack 
here.
    Ms. Owen. OK.
    I am very concerned about this topic. I think that it is 
completely unnecessary and immoral for us to use live embryos, 
to use living human beings for research which is unnecessary 
because as my two compatriots here on this panel have stated so 
clearly, we have alternatives.
    I hope that you will review my written testimony which has 
been submitted to you. I think that so much of what I was 
planning to say, in terms of the moral concerns, as well as the 
alternatives, have already been very, very adequately covered 
by them. But I do think that as a disabled person, as a person 
who has been a national leader in this disability rights 
movement since 1972, that I do want to share some of my 
concerns as it relates to disability and cure.
    The title that I gave my testimony involves the word 
``frenzied.'' I think that we are involved in a frenzied 
pursuit based on fear. As a matter of fact, I think one of the 
members of the committee even referred to the fact that we fear 
our own vulnerability.
    You know, it was over 20 years ago that I first developed a 
definition that has been accepted rather widely, and that is 
that disabilities are the normal, expected, anticipated outcome 
of the risks and stresses of the living process.
    Now, I think that many of us do not accept that reality. We 
are fragile creatures. I do not think that it is an error in 
our Creator's planning. I think that our vulnerability is one 
of the factors that causes us to be more civilized.
    I see fear in my society almost every day. I give some 
examples in my written testimony where people approach me and 
say, I would rather be dead than live like you. I am blind. I 
am partially hearing. I am a wheelchair user because of 
neurological and spinal injuries. But I live a very successful 
life because of the advances that we have made in treatment and 
in rehabilitation.
    Sometimes as an outpatient at the National Rehabilitation 
Hospital, I am amazed at the lack of those services that are 
available to young people currently entering that facility. 
When I was there most recently, I was there for 6 months. That 
does not happen anymore. Young people today are being turned 
out of that facility in a matter of weeks. We need to fund the 
kind of things that allow people with disabilities to truly 
fulfill their potential.
    Yes, I think we are terrified of disabilities. We are 
terrified of disease.
    It was suggested that possibly we were looking for a cure. 
I think that realistically what we can expect is a postponement 
of our mortality, and Dr. Young referred to that. We do not 
cure our vulnerability because it exists, and I would submit 
that it is a positive, not a negative.
    When I travel--and I often travel alone. And it is obvious 
to everyone, including you, Mr. Chairman, that I might need 
assistance. It is amazing to me how, as I travel alone, as I 
need help, as I need assistance, that people thank me when they 
have been given the excuse to be civilized, to be interactive, 
to be a part of a larger community.
    I would further submit that this fear, this frenzy to 
escape our destiny, which truly is what Dr. Young was referring 
to--we are biodegradable. I would submit that our fear, our 
frenzied fear, our dread, our abhorrence of our shared 
vulnerability is what drives this pursuit of some way to 
escape.
    You know, as a child, I mentioned in my written testimony 
that there was a Navajo rug that hung on the family home. There 
is always a flaw in those beautiful Navajo rugs. Any of you 
from New Mexico know this. And I remember as a child recovering 
from meningitis, running my finger over the break in the 
pattern and thinking this is profound. This is profound.
    We are not manufactured like Ken and Barbie. We are not 
uniform. You know, Ken and Barbie, if they come down the 
production line and there is a flaw, they are pulled out. They 
are no longer a part of the Mattel family. They are cast aside 
into the bin for discards. Their essence is regenerated and 
becomes a part of another Ken or Barbie. We, in contrast, are 
created unique, separate from all else. We have gifts, we have 
weaknesses. My weaknesses, your weaknesses, my strengths, your 
strengths, each one of you, I would suggest that those 
intertwined weavings create the strongest social fabric.
    So, I would say I would beg you to stop and think. Is the 
fear of disability so great in this Nation that we must choose 
an immoral and unnecessary strategy to avoid recognition that--
yes, we do need to cure, we do need to treat, we do need to 
find ways to prolong life. And I am fully in support of stem 
cell research, and I would hope that NIH would continue to be 
funded to the extent that it can pursue adult stem cell 
research. Adult stem cell research.
    You have heard and I read some of the same articles that 
Senator Brownback referred to. They are so inspiring. Do I want 
to see again? Do I want to hear as well as I used to? Do I want 
to dance again? Yes, it would be okay. But please know that I 
do not want those things at the cost of any living person. And 
I consider live embryos to be people.
    I want to just say one more thing in terms of my daughter. 
My daughter was born with a 1 percent chance of survival. I had 
heard as a young woman during the height of the push for 
abortion that children the size of my daughter were simply 
collections of cells. I watched her. I watched her tenacity. I 
watched her desire to live. I watched every breath as she was 
breathing it. If she had not had a pro-life doctor there who 
spent 24 hours promoting her life, she would not have survived. 
She struggled to stay alive. Every time I hear people talk 
about the ease of utilization of live embryos, I am reminded of 
the beauty of that tiny, tiny little body of my daughter. She 
is a wonderful, brilliant, young woman today. I am so thankful 
that there was no one in the corridor outside the delivery room 
who might have grabbed her.
    Senator Specter. Ms. Owen, your red light has been on for 
quite a while now. If you could----
    Ms. Owen. I am sorry.
    Senator Specter. I say your red light has been on for a 
while now.
    Ms. Owen. Thank you for letting me go on. I appreciate your 
indulgence.
    Senator Specter. I know you have not observed, you cannot 
see it, but we did want to hear you out. If you could wrap up.
    Ms. Owen. I am finished.

                           PREPARED STATEMENT

    I just want to say that immoral? Yes. Unnecessary? Yes. The 
results from adult stem cell research is very exciting and I 
hope that each one of you are keeping up with that research 
possibility.
    Senator Specter. Thank you very much, Ms. Owen.
    [The statement follows:]

                  Prepared Statement of Mary Jane Owen

                              INTRODUCTION
    First, I wish to thank all who have welcomed me to appear before 
this important Committee to offer testimony on an issue with profound 
social, medical, and most of all, moral implications.
    I am Mary Jane Owen, the Executive Director of the National 
Catholic Office for Persons with Disabilities, a national organization 
charged with creating welcome and justice, within the church and the 
total fabric of society, for over 12 million Catholics with 
disabilities. This mission requires extensive travel, speaking and 
writing, which I have been able to do despite--or possibly better--
because of being a blind, partially hearing woman with neurological and 
spinal impairments which require use of a wheelchair.
    Over a half century of my working life, I have been a psychiatric 
social worker, a professor of social research, a federal administrator, 
a free-lance consultant and a businesswoman. More importantly, I have 
been a participant observer in the difficult decades of change and 
progress as our society has created new options and opportunities for 
people with disabilities. Remarkable changes have taken place, 
including passage of the Americans with Disabilities Act, an effort in 
which I worked, along with thousands of other disabled people in our 
search for fuller participation within our society.
    I am here to urge you: Do not, in the name of progress for disabled 
people, certify or justify the destructive harvesting of human embryos 
for stem cell research, a practice both immoral and unnecessary.
    While disabled people are interested in cures, as well as better 
tools for living, greater inclusion in society and other possibilities 
which will improve our quality of life, we are not so desperate for 
cures that moral considerations disappear.
    Do not use our struggles and aspirations to justify an immoral 
policy that will encourage the destruction of unborn babies. Instead, 
direct researchers to use the many alternative sources of stem cells so 
that this promising area of research may be developed in an ethically 
defensible manner!

                          MORAL CONSIDERATIONS
    Congress determined once, and I believe rightly, that harvesting 
fetuses is wrong. There are many moral reasons for this. In addition to 
the opposition to abortion itself, shared by many Americans, there is 
also the conviction that human embryos and fetuses should not be 
harvested lest they come to be seen as products for sale.
    This is not an insignificant issue. The medical and biotechnology 
revolution will be even more powerful in its implications than our 
internet/information technology revolution. Medical and biological 
technology can change our very identity as human beings. I do not 
propose that we stop this revolution, but I am confident we must 
carefully consider where we want to go and where we are being carried 
by frenzied attempts to deny our shared vulnerability.
    Technology, commerce and science are all pushing us inexorably in 
the direction of regarding human beings as products. We need to 
consider if we continue down this slippery slope, are we ready to 
justify the creation of human beings for spare parts? We are forced to 
ask this question today, for the possibilities lie just ahead. 
Researchers are already harvesting fetuses, and their so-called utility 
has already become part of the moral calculus of abortion. Now the 
demand for live embryos for stem cell harvesting threatens to become 
part of the moral calculus of fertility treatments.
    Yes, medical progress is desirable. Yes, new research vistas 
require new ethical guidelines--But I pray they will NOT involve a 
repudiation of our past moral stance and move toward an exclusively 
utilitarian set of ``ethical'' rules. We must carefully calculate what 
is essential to human decency and then defend that essence, even 
hedging it about with an extra margin of caution. We Americans disagree 
about many things, but most of us consider the idea of harvesting 
fellow human beings for the advantage of the few as abhorrent. Let us 
respect that moral intuition and the traditional values upon which it 
rests.
    There are medical experts who will assure us we need not sacrifice 
scientific progress because of our abhorrence of a utilitarian approach 
toward human life.

                 THE ALTERNATIVE SOURCES OF STEM CELLS
    It is clear that researchers should have access to stem cells. The 
issue is: why embryonic stem cells? Some would claim that refusing to 
subsidize this particular kind of stem cell research would unduly 
hamper medical progress. However, there are many other sources of these 
vital human tissues which give clear indication of their potential for 
positive results. Some would even avoid the possibilities of rejection 
which are inherent in the use of tissue not recognized by the host 
body.
    Exciting possibilities lie ahead in making use of self-contributed 
stem cells, as well as those harvested as a result of necessary 
surgical interventions, tissue contributed by consenting adults and the 
by-products of natural births.

                     THE PUSH TO BETRAY THE UNBORN
    Congress has already attempted to prevent federal funding for 
harmful human embryo research, but now the issue has arisen again, and 
some say it is urgent to allow and encourage the use of embryonic cells 
in research. What has changed since that earlier decision? Yes, 
research possibilities have opened up but every day we are learning 
more about alternative sources of stem cells.
    And so we are forced to ask ourselves why a civilized society would 
seek to overcome its moral compunctions about harvesting the unborn. Is 
it that our fear of disease, disability and mortality is so 
overwhelming that we forget or deny what we know is morally correct? I 
am suggesting the frenzied rush to harvest embryonic humans is based 
upon an undue fear of human fragility and disabilities. We fear 
anticipated pain. Yet we know that management of pain has made 
tremendous strides in the past few years.
    I witness society's fear and anxiety about disability every day. I 
have often been amazed by strangers who approach me to confide that 
they would rather be dead than become blind or use a wheelchair. Aside 
from the bluntness of these remarks, what surprises me is that many 
people can not imagine that I have a happy life. And yet I am as happy, 
as successful and productive as anyone I know. It seems obvious to me 
that their fears about their own future disabilities keep them from 
seeing the reality of my life--and the possibilities for all people 
with assorted disabilities.
    In my lifetime I have also observed our medical system negate the 
idealism of the Hippocratic Oath and move from viewing its services as 
having ties to charity. Within a for-profit enterprise, the view that 
some human lives are potential ``product'' may be tempting but it is 
still immoral. The sacrifice of some human lives for the benefit of 
others must be defined as illegal, as it has been in the past.
    My view can be summarized thus: Many of my fellow citizens suffer 
unduly from fears and frenzied anxiety about assorted disabilities and 
fragilities.

          A NEW UNDERSTANDING OF DISABILITY AND VULNERABILITY
    Twenty years ago I proposed a new and positive definition of 
disabilities which has been used by advocates in the intervening years:
    Disabilities are the normal, expected and anticipated outcome of 
the risks, stresses and strains of the living process itself.
    The eventual outcome of the shared fragility of our bodies is the 
development of physiological glitches at some point in the normal life 
cycle. Disabilities are not something which happens only to the unlucky 
few but is an event which takes place for us all. We may face this 
eventuality before birth, in early life, during the height of our 
productive years or at the end of life. When we view our shared 
vulnerability with these conceptual lenses, we understand that the 
principles of universal design should be considered as we modify our 
environments, programs and institutions.
    Broadly available medical services, rehabilitation techniques and 
technology and the evolving expectations of millions of American 
citizens with disabilities can revolutionize the future status of those 
millions of Americans who are destined to experience physical, 
cognitive and sensory limitations. Public recognition and support of 
these alternatives can relieve the rampant anxiety evident in the 
frenzied pursuit of ``cures'' at any moral cost.
    Unfortunately, the picture of life with disabilities often seen in 
the popular media and skillfully utilized by those segments of our 
population seeking to deny the essential dignity and value of all human 
life, are generating the current pressure to authorize destructive 
harvesting of stem cells from embryos as essential in our struggle to 
prolong our productive lives.
    We are not manufactured like Ken and Barbie, expected to fit a 
model of physical perfection; required to be uniform in all those 
characteristics by which we might be judged as having value. We are not 
fabricated of high impact plastic. When a single Ken or Barbie appears 
on the production line with even a tiny flaw, the results are thrown 
into the recycle bin. That member of the Mattel family will be broken 
down into its component parts to be used again in the ongoing effort to 
reach uniform perfection.
    In contrast to that process, each of us was uniquely and 
individually created. For those of us who are Christian, we believe 
that this variety in abilities and strengths, disabilities and 
weaknesses, in some mysterious way which we can not fathom, reveal some 
essential part of our Heavenly Father. Other faith and cultural groups 
confirm the normality of assorted ``imperfections.'' The beautiful 
Navaho rug that hung on the wall of my childhood home was woven with 
the traditional ``flaw'' which marked it as reflective of the 
traditional belief that only the ``Creator'' was perfect. As I traced 
that defect in perfection, it comforted me as I dealt with the minor 
disabilities caused by an early bout of meningitis.
    Not only are we created to be unique, we are created of extremely 
fragile material. We may be born without disabilities, but we must 
anticipate that at some point in our lives we will be forced to 
recognize our shared vulnerability. That recognition can inspire us to 
acknowledge our need for each other and for the Creator. It may also 
move us to cherish those of our community who are in need of 
assistance, medical services or rehabilitation.
    Many of us unfortunately believe that disabilities are a cosmic 
accident which we must correct. I agree that we must work to do what we 
can to reduce or prevent disabilities, using morally defensible means. 
Much progress has already been made in prevention, treating or even 
curing a variety of disabling conditions. However, I would affirm we 
would never completely eliminate the vulnerability of the human 
organism nor would it be such a great blessing if we could. My personal 
experience convinces me that it is by God's wisdom that the gift of 
life comes in fragile earthen vessels. Many of the virtues we feel are 
the best that humanity has to offer, such as love, faith, hope, mercy, 
and courage, are associated directly or indirectly with our 
vulnerability.
    I suggest that we, as a nation, need to use these alternative 
lenses through which to view human vulnerability and disabilities as we 
re-compute our outmoded ``scientific'' formulae for assessing other 
people's quality of life. We need you, members of the United States 
Senate, to call for a nation-wide calming of the frenzied research 
efforts based upon destroying future citizens, rather than endorsing 
this national anxiety. The potential quality of any human life can not 
be judged by outside authority.

              THE POSITIVE ASPECTS OF HUMAN VULNERABILITY
    I propose that the catalytic effect of our mutual need for each 
other is a social positive which must not be lost for it fosters a 
sense of the need for mutual aid and interaction within our 
communities.
    We no longer need to respond in the same way our ancient ancestors 
did to the dangers rampant in their primitive world. Fearful avoidance, 
the patterns of behavior called forth by fear that the weak and 
vulnerable would fall prey to the wooly mammoth, is no longer 
appropriate. Superior brawn, eyesight, hearing and speed are not the 
only human characteristics essential today. Many of us sit at our 
computers which meet the needs of those of our brothers and sisters 
with the palsied movements of Parkinson disease; the paralyzed arms of 
the quadriplegic, or which speak to the blind or accommodate the 
confused efforts of the poor speller. Today these ``imperfect'' 
characteristics no longer need throw us into a panic or deep 
depression. Our quality of life can remain quite satisfactory in spite 
of those impairments which would have severely limited the lives of our 
grandparents.
    In my travels, I have observed that the intertwining threads of the 
interaction between people are enhanced when we acknowledge our need 
for assistance or help. Apparently, at this point in our history, we 
seek ``excuses'' to be interactive and to discard our sense of complete 
autonomy. This serves to refresh our sense of being an essential part 
of a vital community. This interaction is a potent antidote to the 
rampant alienation which threatens so many of our neighborhoods today.

                          ON A PERSONAL LEVEL
    I carry the genes for the blindness which knocked me from my ivory 
tower. And I may have passed that pattern of visual impairment to my 
daughter. Growing up I was surrounded by elderly blind women, all of 
whom were active in their communities, well educated for their time and 
place and recognized for their abilities more than their limitations. I 
was unprepared for the loss of my sight at the height of my rapid climb 
up the academic ladder.
    The two women closest to me both died with Alzheimer's disease. I 
may be programmed for a similar experience. Awareness of that 
possibility makes each day of intellectual exploration more precious 
and pushes me to greater achievement.
    The women who built the base upon which I exist would never have 
wished for a single life of an unborn baby to be sacrificed so that 
their physical or cognitive challenges might have been eliminated or 
postponed. They were strong feminists in the model of Susan B. Anthony. 
They saw abortion as anti-woman and recognized that overcoming 
challenges as a characteristic to be admired, not avoided.
    When I visited with my aunt, Naomi Harward, during the period when 
her Alzheimer's disease was being diagnosed, one morning I found her in 
tears. She had been widely recognized as a political force, having 
headed up a campaign to recall Governor Meecham. She was the oldest 
woman to appear on the cover of MS Magazine.
    She said, ``I don't want to have Alzheimer's.'' She had watched her 
sister, my mother, another widely recognized and strong feminist, live 
with that condition for long years. We embraced as I reminded her that 
none of us know what the future holds. I told her that at each step in 
her life she had showed me the way to live gracefully and successfully, 
no matter the challenges faced. That was a powerful moment in my life. 
She died quietly and peacefully during the few moments when her beloved 
grandson and his wife stepped out of her room. We felt she had chosen 
that moment to slip away and join those parts of herself which had 
already gone before.
    She was so strongly opposed to abortion; the idea of harvesting 
stem cells from defenseless unborn children would have aroused loud and 
political protest, I am sure.
    When my daughter was born, she had less than 1 percent chance of 
survival based upon her prematurity. I'd been told that babies at her 
stage of development were merely blobs of cells, not unique and 
individually crafted tiny people. I shall never forget the power and 
beauty of her tiny body; the fragility of life and her stubborn hold on 
it. I could see the intensity of her desire to live as each breath was 
aided; each beat of her heart monitored. That image continues to 
inspire me and that tiny essence of human life comes to mind every time 
I hear reference to the harvesting of stem cells from unborn children 
as an essential medical practice.
    My daughter graduated cum laude from Harvard twenty years later and 
continues to be a joy and support. Thank God no one was lurking in 
those sterile halls, awaiting the emergence of a frail and fresh 
``product'' to be marketed for research or ``therapeutic'' purposes.
    I have neurological impairments and have sustained spinal injuries, 
and while I enjoy dancing in my wheelchair, it might be pleasant to do 
so in what is considered a normal way. But be very clear in this: I am 
deeply opposed to any gain in my sight, mobility, or even my hearing if 
it was purchased at the cost of a single human life.
    As I've already noted, I may face Alzheimer's disease at some point 
in my future. But that eventuality is less frightening than a world in 
which, as a matter of medical intervention, one life can be casually 
eliminated in order to offer a few additional months of ``normality'' 
to another.
    I've talked with and cried with hundreds of my colleagues within 
the disability movement. We seek better funding for rehabilitation, not 
a quick fix. I challenge you, members of the United States Senate and 
all my fellow citizens, to create environments, fund current 
rehabilitation programs and alter perceptions of human vulnerability 
which frighten those who fear what their future might hold. We can 
escape from the ancient demons which haunt nightmares.
    The worse thing in life is not disability, or pain, or even death. 
The worse thing I can imagine is to create a society which sees itself 
as justified in treating other people as objects to be used or 
discarded, as best fits the desires of the moment. I would not wish to 
live in such a world. And the moral choices we make today will surely 
shape our future. Even a ``small thing'' like the fate of an unborn 
child can have great implications as we create that future for 
ourselves and our children.

    Senator Specter. Senator Brownback, when we deal with the 
morality issue, I totally agree with you that that is the 
principal concern, that we proceed in an ethical and in a moral 
way. I would join you totally in your objection, abhorrence, 
disdain for interfering with human life. But the embryos which 
are to be used here for extracting stem cells are discarded. 
They are not to be used. They have been prepared for in vitro 
fertilization, and if they are not used for the research, they 
will not be used at all. So, how do you deal with that fact 
that no human life is to be taken?
    Senator Brownback. Well, I think the issue you raise here, 
what you are talking about is strangely familiar from our past 
where we say these are going to discarded and die anyway, so 
therefore, why not get some benefit out of them. That sounds 
strangely like some of the things that happened in World War 
II.
    Senator Specter. Well, how so? These have been discarded. 
It is not that they are going to be discarded. How would that 
run afoul or be analogous to what happened in World War II?
    Senator Brownback. Well, you are taking live human embryos 
in this case and they will be extracted--their stem cells from 
them. You had the Nazis in World War II saying, of these 
people, they are going to be killed. Why do we not experiment 
on them and find out what happens with these experiments? They 
are going to die anyway.
    Senator Specter. But they were living people unlike the 
embryos.
    Senator Brownback. These are living embryos. These are 
living embryos. And they are being treated in this case as 
property.
    Senator Specter. But the people whom the Nazis experimented 
with in the abhorrent way in the holocaust were living.
    Senator Brownback. These are living embryos. You are taking 
living embryos.
    Senator Specter. Let me ask you, Senator Brownback, do you 
oppose in vitro fertilization?
    Senator Brownback. I have not thought through that one, and 
I am not prepared here today to talk about that particular 
issue. The issue in front of us is you have a live embryo.
    Senator Specter. I raise the in vitro fertilization issue--
and you are correct. It is not before us. We are exploring the 
matter today, but we are going to have an opportunity to 
discuss it on the Senate floor at greater length.
    But I raise the in vitro fertilization issue because there 
are some who do object to that, and it is a consequence of in 
vitro fertilization that these embryos are created. There might 
be an argument that every one of these embryos is entitled to 
life. But the process of in vitro fertilization is to have a 
large group and then to use some but not to use others. So, 
this is something we will be getting into.
    But I understand your point of view.
    Let me shift for just a moment to the question of----
    Senator Brownback. If I could on that very point.
    Senator Specter. Go ahead, sir.
    Senator Brownback. Because you are seeing the courts now 
across the country starting to discuss this issue of are these 
embryos then person or property. And that is at the core of 
what we are talking about here. We will hopefully have some of 
the legal opinions of what are developing on this, but does 
that not sound dangerously close to things that we have skirted 
around in the past and dealt with in this body a number of 
years ago?
    And my point on all this is we do not have to go this way. 
Let us go the way that I will join you--and I do not know if 
you can ask for a level of funding on adult stem cell research 
that I would not support because I think it is so promising for 
its potential and ethical. But let us not go into this one that 
we have so many of these questions that mankind has been around 
for a long time.
    Senator Specter. Well, I am going to come to that in a 
moment.
    Ms. Owen. Could I respond to your question?
    Senator Specter. Not yet, Ms. Owen. You can but not quite 
yet.
    When you talk about a person--you cannot, Ms. Owen, because 
I want to develop this thought with Senator Brownback which we 
are discussing, but I will come back to you.
    When you talk about a person and we have these concerns 
about when life begins and you have conception and then 
abortion--and I am personally very much opposed to abortion, 
although I think it has to be a decision for the woman.
    But when you talk about conception and whether there is a 
person, that conceived entity is on its way to life. But that 
is not the situation--just to pursue this for another step, 
Senator Brownback--with the discarded embryo. A discarded 
embryo is not on its way to life. I do not like the concept of 
property as opposed to person. As we consider the issue of a 
person where there is conception, that entity is on its way to 
life. But I do have that distinction which I would be 
interested in your comment about with respect to the discarded 
embryo.
    Senator Brownback. If it is a live embryo and it is 
destroyed for this purpose, it is killed. And you have that 
destruction that is taking place. And then in this case we are 
even saying that destruction we will then do this with the 
parts out of it. I cannot see that that is something that this 
great Nation really wants to move along when we have another 
option that is so promising in front of us. We just do not have 
to go this way.
    Senator Specter. Well, let us take up that option for just 
a moment with you, Dr. Young. I note, in one of the 
commentaries from your magazine that you made available to us, 
the following statement with respect to adult stem cells. I had 
a concern from other materials which I have studied that the 
stem cells from adult tissue, matured cells are already 
differentiated into a narrow type, range of stem cells. Those 
cells cannot support the kind of research on a broad basis.
    Quoting from the publication which you have made available 
to us on page 1419, ``Adult stem cells have a drawback, 
however, in that some seem to lose their ability to divide and 
differentiate after a time in culture. This short life-span 
might make them unsuitable for some medical applications.'' Do 
you disagree with that limitation on adult stem cells?
    Dr. Young. I think the jury is out in general. This is a 
very well-balanced set of articles, and there are other 
articles there which state that one of the liabilities of the 
embryonic stem cells is that they differentiate in a more 
uncontrolled fashion, whereas it is better to use adult 
embryonic cells which would be able to differentiate more 
unidirectionally. I think that further research has to be 
undertaken.
    But in the interim, I do believe that it is critical to 
focus on the animal work with embryonic stem cells which can 
yield a great deal of information and the work on human adult 
stem cells, and then pause for a while.
    If I could add one answer to the question that you asked 
the Senator, I think it could be very enlightening. There was a 
case a short time ago where one of the in vitro fertilization 
clinics--it was about a year and a half ago--found that they 
had an embryo, ``a spare embryo,'' that was left from a woman's 
in vitro fertilization about 6 to 8 years ago. It was reported 
in the Los Angeles Times that that woman then desired to have 
that embryo implanted and gave birth to a twin, if you would, 6 
to 8 years down the line. It goes in accordance with what the 
Senator was saying.
    My remark that I really think the Senate needs to address 
is that of the regulation of in vitro fertilization. I have 
supported in vitro fertilization, have counseled couples in our 
church, and have worked with them on this area. But there does 
not need to be ``the development of excess embryos.'' And there 
does not need to be the concept of spare or embryos that are 
about to be destroyed. I think that is one the Senate might 
like to look into.
    Senator Specter. Before turning to Senator Harkin, Ms. 
Owen, you had a comment you wanted to make. Ms. Owen?
    Ms. Owen. Yes. I would like to respond to your question 
about the viability.
    Senator Specter. Fine. We would be pleased to hear you.
    Ms. Owen. You see, I think that one of the things that 
possibly needs to be considered is that fact that we are in a 
society where we are beginning to talk about the sale of body 
parts. I think that when we begin to say it is all right to 
have excessive numbers of embryos created, we are saying go 
ahead, private industry, go ahead, NIH, go ahead and create 
excess embryos not just for in vitro fertilization, but with 
the idea--maybe it is behind closed doors--but with the idea we 
can make a profit by selling these embryos. So, I do not think 
it is just simply a few embryos that we are talking about 
today. We are talking about the potential of creating hundreds 
of embryos for research purposes.
    Senator Specter. Thank you, Ms. Owen.
    Senator Harkin.
    Senator Harkin. Thank you very much, Mr. Chairman. I too 
want to thank especially my colleague, Senator Brownback, with 
whom I have a great working relationship and friendship. I know 
how deeply you feel about this issue, and I respect that. I 
respect it greatly. I respect all of you who have perhaps come 
at this from a different viewpoint perhaps than I come at it 
from.
    But I believe that by working together in a rational, 
reasonable way, I think that we can find pathways out of this. 
I just want to make sure that we are all talking about the same 
thing and that we are all knowing that the words we use--we 
kind of agree upon what they mean. I think a lot of times maybe 
both sides of an issue talk to themselves a lot and they do not 
talk to each other enough to try to figure out some pathways 
that we can move ahead.
    To that extent, I just again wanted to reiterate what our 
chairman said, that we are looking at the--without getting into 
whether adult stem cells will do the job or not. No one knows. 
OK, we do not know. We do not know. But we do know and 
scientists do have a belief that at least in the pluripotent 
stage of embryonic stem cells that they do hold great promise. 
Again, there are no guarantees out there. The only guarantee is 
that if we do not do it, possible cures, interventions and 
preventions for a whole host of illnesses will be put off for 
longer periods of time. That guarantee we do have.
    Second, with regard to the issue of embryos--again, Sam, we 
get in this whole debate about the Nazi Germany and that kind 
of thing. But I think we do have to keep some perspective on 
this. I put something on this piece of paper, and I will bet no 
one out there can tell me what is on that piece of paper 
because you cannot see it. But I took my pencil and I put a dot 
on it. That is how big we are talking about. That is how big 
the human embryo is that we are talking about. In fact, in most 
cases you cannot even see it with the naked eye.
    Now, again I am not a theologian, but to equate that with 
the individual person that the Nazis were experimenting upon I 
think is to stretch the meaning of humanness and what a human 
being is.
    Now, with regard to that little dot that is on the piece of 
paper, there are, as the chairman said, leftover--and this is 
where it gets to you, Dr. Young. There are leftover embryos of 
this size, frozen, about 100,000, Sam. We estimate about 
100,000 of these are frozen in liquid nitrogen right now that 
are left over. I have been told by scientists that we probably 
do not need any more than that. I mean, we do not need to go 
out and get more stem cells and that. We have 100,000 embryonic 
stem cells right now left over from in vitro fertilization. 
Regardless of what you may think about in vitro fertilization, 
they are there. They are frozen. The question is, what is going 
to happen to them?
    Dr. Young, you pointed you--and I read that article about 
the woman that can back 6 years later. Well, again, that is 
fine.
    Now, the guidelines that we set up--I am going to just jump 
ahead here a little bit. The guidelines that were proposed said 
this. And this gets to you, Ms. Owen, about the sale. You were 
talking about selling human parts and stuff. The guidelines 
stated three things. We could only use embryonic stem cells 
from excess in vitro embryos, ones that are there right now. 
OK? Second, there must be no financial compensation. No one can 
sell these. No one can pay for them. And third, they can only 
be used with the informed consent of the donors, the two 
parents, whoever that is. So, if there is someone who donated 
that in vitro fertilization and they are deceased now, they 
cannot give that informed consent. Only those who can give 
their informed consent could use it. Those are the guidelines.
    So, we have no need to create more than the 100,000 that we 
have now, regardless of what you may think about whether we 
should go on with in vitro fertilization or not. Those are 
already frozen. They are there. The question for us I think, 
Sam, and others is they are there. Are we going to keep them in 
liquid nitrogen forever and ever and ever? I doubt it.
    Senator Brownback. Well, if I could answer that.
    Senator Harkin. Yes.
    Senator Brownback. Because you are seeing now court cases 
of people coming back that these have been frozen and kept. The 
couple is divorced.
    Senator Harkin. Right.
    Senator Brownback. One wants to implant the frozen embryo, 
the other does not.
    Senator Harkin. Yes.
    Senator Brownback. And the court is then wrestling with the 
issue, who gets to decide, and are they person or property. The 
court is wrestling with this now. Should we in legislation 
say--because as I read this, it would appear to me we are 
saying these are property.
    Senator Harkin. We are saying that only with the informed 
consent of the two donors, Sam.
    Senator Brownback. Well, but I am saying what the court is 
wrestling right now.
    Senator Harkin. But you have two donors that are wrestling 
with each other.
    Senator Brownback. You do but the central question, the 
central legal question is, is it person or property? The court, 
in a line of decisions that I think is probably going to start 
developing here, is wrestling with the same sort of thing they 
did 100 years ago in this country.
    And there is a Boston case where they are saying, well, it 
is not really a person, it is not really property, it is an 
entity with the potential for life is one lower court ruling. 
Now, does that sound like saying somebody is three-fifths of a 
person and not a whole as we have in our prior history as a 
country? I think in this legislation, we are saying these are 
property to be decided upon by these two people what happens to 
this.
    I do not think we are there at deciding that issue at this 
point, and I do not think it is wise for us to, particularly 
since we have got an option that we do not have to do this.
    Senator Harkin. The option I think, Sam, as others have 
pointed out, and as Science magazine, as scientist, after 
scientist, after scientist will tell you there may be some 
benefit to adult stem cells, but as far as they can determine 
now, their life is very short. They are not eternal like the 
pluripotent stem cells of embryos.
    Senator Brownback. Pluripotent are not eternal. I do not 
think you would say that those would be eternal masses either.
    Senator Harkin. No. They can regenerate. They can 
regenerate.
    Dr. Young. They can also differentiate very rapidly.
    Senator Harkin. But they can also regenerate too.
    So, you can use adult stem cells. I am not saying we should 
not use them. I am saying why close off all these doors that 
researchers could use.
    Again, Sam, keep in mind there are private entities out 
there right now conducting this kind of stem cell research. It 
is being done chaotically. It is being done without guidelines. 
Who knows what is happening out there?
    I think we have taken the responsible, reasonable approach. 
We set up a Bioethics Commission. We said only from excess in 
vitro embryos, no financial compensation, informed consent of 
the donors. To me, you know, you are talking about these as 
being spare and products for sale. They are not for sale. These 
are not spare. They are there.
    It seems to me the height of morality to say that in order 
to help someone's life to prevent Alzheimer's or ALS or to 
regenerate neurons, to help people with juvenile diabetes, it 
seems to me the moral thing to do would be to use what we have 
there in these in vitro cells that are left over, the 100,000, 
to permit the kind of ethical guideline structure that we set 
up so that scientists can use those to help make lives better. 
To me that seems to me--again, I know we differ on this but I 
hope you give me as much benefit as I give you approaching this 
from a moral standpoint.
    Senator Brownback. And I have stated that repeatedly. I do. 
I do not think our hearts are any different of what we want to 
get done.
    Senator Harkin. But I think our position is just as moral 
and as morally defensible as your position.
    Senator Brownback. Well, I would question whether we want 
to destroy one human life for another. And there is private 
work going on. There are also 10 States that have banned the 
destructive embryo research even privately funded. 10 States 
have already done that. So, this would have a Federal stepping 
in on top of what States are doing as well.
    We do not have to go this route. I listed the research that 
is taking place in the very areas that you are talking about 
solving and the promising work. Would it be not only moral but 
also wise to invest more in that area and focus our efforts 
there that we can get there faster?
    Plus, we have not talked about the immunosuppressant 
problem that you are going to have if we develop these off of 
bodies that are not the same as the body that you want to put 
this new entity in. You do not have that issue that you have to 
wrestle with. If you develop pancreatic cells out of an embryo, 
you can have, and in likelihood will have, immunosuppressant 
problems with the body rejecting that. Whereas, if you pull it 
out of your own adult stem cells, you do not have that issue.
    I think we have got a wise and moral route that we can all 
go together on.
    Senator Harkin. With adult stem cells, you have one door to 
open. Maybe it will work. But using other embryonic stem cells, 
you open up a whole host of other doors that may lead you to 
the right path for the prevention and the cure of many of our 
common ailments. That is all I am saying.
    Thank you, Mr. Chairman.
    Senator Specter. Thank you very much, Senator Harkin.
    Thank you very much, Senator Brownback, Ms. Owen, Dr. 
Young. Dr. Young, you remind us that we are biodegradable. 
Maybe now be can be bio-regeneratable.
    Dr. Young. I think for a short time that is possible.
    Senator Specter. One of the heros of my youth was Ponce de 
Leon who searched for the fountain of youth, and if we have it 
here, let us look at it.
    Stay tuned, ladies and gentlemen. This debate is not over. 
We will be on the Senate floor where we have unlimited time to 
speak, and on this issue, we will probably be using it.
    Thank you very much, Senator Brownback.
    Ms. Owen. Thank you.
    Dr. Young. Thank you very much.

STATEMENT OF CHRISTOPHER REEVE, ACTOR/DIRECTOR; 
            CHAIRMAN, CHRISTOPHER REEVE PARALYSIS 
            FOUNDATION
    Senator Specter. We now turn to our third panel. We have 
Mr. Christopher Reeve, Ms. Jennifer Estess, and Dr. Lawrence 
Goldstein.
    We thank you very much, Mr. Reeve, specially for your 
personal crusade on stem cells. We noted with America generally 
and the world the traumatic experience you had with your 
accident on horseback and the severing of your spinal column, 
but you have come back to lead this crusade in a very 
inspirational way. The current issue of Time magazine has your 
comments and pays special tribute to what you have done. I know 
that this is a matter of--well, it is of life concern to you. 
It is that important. Possible regeneration of your spinal 
column to enable you to fly again. So, we thank you for all of 
your extraordinary work here, and we thank you for being 
available to testify. We now turn the floor to you.
    Mr. Reeve. Well, thank you very much, Mr. Chairman. I 
appreciate the opportunity to be here to testify.
    I would start by saying that in vitro fertility clinics 
have been around since the 1950's, and as far as I am aware, 
even though the Pope, of course, stated a position against it 
and it is a sin for a Catholic to use in vitro fertilization, 
there has not been an enormous outcry or protest. In other 
words, these clinics have existed in relative peace for nearly 
40 years, and I do not understand why all of a sudden there is 
a huge issue about it, now that discarded embryos will be used 
for research instead of just being thrown into the garbage.
    A critical factor in the quality of life for present and 
future generations will be what we do with human embryonic stem 
cells. These cells have the potential to cure disease and 
conditions ranging from Parkinson's and MS to diabetes, heart 
disease, to Alzheimer's, Lou Gehrig's, even spinal cord 
injuries like my own. They have been called the body's self-
repair kit.
    Their extraordinary potential is a recent discovery. And 
much basic research needs to be done before they can be sent to 
the front lines in the battle against diseases. But no obstacle 
should stand in the way of responsible investigation of their 
possibilities. To that end, the work should be turned over to 
the Federal Government through the National Institutes of 
Health. That will avoid abuses by for-profit corporations, 
avoid secrecy and destructive competition between laboratories, 
and ensure the widest possible dissemination of scientific 
breakthroughs. Human trials should be conducted either on the 
NIH campus or in carefully monitored clinical facilities.
    Now, fortunately, stem cells are readily available and 
easily harvested. In fertility clinics, women are given a 
choice of what to do with unused fertilized embryos: they can 
be discarded, donated to research, or frozen for future use. 
Under NIH supervision, scientists should be allowed to take 
cells only from women who freely consent to their use for 
research. One very important factor is that this process would 
not be open-ended. Within 1 to 2 years, a sufficient number 
could be gathered and made available to investigators. So, for 
those reasons, the ban on federally funded human embryonic stem 
cell research should be lifted as quickly as possible.
    Again, why has the use of discarded embryos for research 
suddenly become such an issue? Is it more ethical for a woman 
to donate unused embryos that will never become human beings or 
to let them be tossed away as so much garbage when they could 
help save thousands of lives?
    Now, treatment with stem cells has already begun. They have 
been taken from umbilical cords and become healthy red blood 
cells used to cure sickle-cell anemia. Stem cell therapy is 
also being used against certain types of cancer. But those are 
cells that have significantly differentiated; that is, they are 
no longer pluripotent or capable of transforming into other 
cell types. For the true biological miracles that researchers 
have only begun to foresee, medical science must turn to 
undifferentiated stem cells. We need to clear the path for them 
as quickly as possible.
    Now, controversy over the treatment of certain diseases is 
nothing new in this country. Witness the overwhelming 
opposition to Government funding of AIDS research in the early 
1980's. For years, the Government tossed this issue around as a 
political football until a massive grassroots effort forced 
legislators to respond. And today NIH is authorized to spend 
approximately $1.8 billion annually on new protocols, and the 
virus is largely under control in the United States.
    Now, at this point I wish to submit a letter of support 
from four theologians representing the Protestant, Catholic, 
Jewish, and Islamic faiths. They say:

    Our opinions about embryonic stem cell research reflect 
several different religious perspectives: Jewish, Catholic, 
Protestant and Islam. While they do not represent a single 
voice from these religious communities, they do offer a 
collective belief that is based on similar views about certain 
moral and ethical questions.
    According to our religious beliefs, all human life must be 
protected. However, they also indicate that there is a 
significant difference between an embryo suspended in liquid 
nitrogen that will never be implanted inside a womb and an 
unborn child who is already in the womb.
    Our religious beliefs also stress the importance of 
compassion. Thus, we support embryonic stem cell research 
because it would use these frozen or otherwise discarded 
embryos to help ease the suffering of those with catastrophic 
diseases such as diabetes, Parkinson's, cancer, Alzheimer's, 
heart disease, osteoporosis and arthritis.
    As theologians, we put a great deal of importance upon the 
need for ethical standards in biomedicine. Currently, stem cell 
research is being conducted solely in the private sector, where 
it is not subject to the important guidelines developed by the 
NIH, parameters that reflect critical input from ethicists and 
theologians. However, the guidelines would only come into 
effect with Federal funding, which is another reason we support 
moving forward with the research under NIH. In addition, 
Federal funding would not only speed the discovery of cures, 
but ensure infrastructure is in place that will guarantee 
ethical conduct and maximize the benefit to society.
    Our religious beliefs tells us that embryonic stem cell 
research is worthy of Federal support for millions across the 
country who are suffering from diseases. We hope you will join 
us and support stem cell research through the NIH.
    We look forward to your support for this research, as the 
lives of millions are counting on you.

    And this is signed by Rabbi Elliot Dorff, Ph.D., who is 
Professor of Philosophy at the University of Judaism. It is 
signed by Nancy J. Duff, Ph.D., Associate Professor of 
Theological Ethics at Princeton Theological Seminary. It is 
signed by a name I cannot pronounce: Abdulaziz Sachedina. I 
cannot say this. Anyway, Islamic Ph.D., Department of Religious 
Studies at the University of Virginia. And in my opinion, most 
importantly, by Margaret Farley, Ph.D., who is Professor of 
Christian Ethics at the Yale University Divinity School, and 
she is a Catholic.

                           PREPARED STATEMENT

    Finally, I wish to enter into the record a list of over 100 
disease groups, clinicians, foundations, universities and 
medical schools, all of whom are supportive of me and my 
advocacy for stem cell research. You will see that in 
attachment 2.
    But while we prolong the stem cell debate, millions 
continue to suffer. It is time to harness the power of 
Government and go forward. Thank you.
    [The statement follows:]

                Prepared Statement of Christopher Reeve

    WE MUST PURSUE RESEARCH ON EMBRYONIC STEM CELLS.
    With the life expectancy of average Americans heading as high as 85 
to 90 years, it is our responsibility to do everything possible to 
protect the quality of life of the present and future generations. A 
critical factor will be what we do with human embryonic stem cells. 
These cells have the potential to cure diseases and conditions ranging 
from Parkinson's and multiple sclerosis to diabetes and heart disease, 
Alzheimer's, Lou Gehrig's disease, even spinal-cord injuries like my 
own. They have been called the body's self-repair kit.
    Their extraordinary potential is a recent discovery. And much basic 
research needs to be done before they can be sent to the front lines in 
the battle against disease. But no obstacle should stand in the way of 
responsible investigation of their possibilities. To that end, the work 
should be funded and supervised by the Federal Government through the 
National Institutes of Health (NIH). That will avoid abuses by for-
profit corporations, avoid secrecy and destructive competition between 
laboratories and ensure the widest possible dissemination of scientific 
breakthroughs. Human trials should be conducted either on the NIH 
campus or in carefully monitored clinical facilities.
    Fortunately, stem cells are readily available and easily harvested. 
In fertility clinics, women are given a choice of what to do with 
unused fertilized embryos: they can be discarded, donated to research 
or frozen for future use. Under NIH supervision, scientists should be 
allowed to take cells only from women who freely consent to their use 
for research. This process would not be open ended; within one to two 
years a sufficient number could be gathered and made available to 
investigators. For those reasons, the ban on federally funded human 
embryonic stem cell research should be lifted as quickly as possible.
    But why has the use of discarded embryos for research suddenly 
become such an issue? Is it more ethical for a woman to donate unused 
embryos that will never become human beings, or to let them be tossed 
away as so much garbage when they could help save thousands of lives?
    Treatment with stem cells has already begun. They have been taken 
from umbilical cords and become healthy red cells used to cure sickle-
cell anemia. Stem cell therapy is also being used against certain types 
of cancer. But those are cells that have significantly differentiated; 
that is, they are no longer pluripotent, or capable of transforming 
into other cell types. For the true biological miracles that 
researchers have only begun to foresee, medical science must turn to 
undifferentiated stem cells. We need to clear the path for them as 
rapidly as possible.
    Controversy over the treatment of certain diseases is nothing new 
in this country: witness the overwhelming opposition to government 
funding of AIDS research in the early 1980's. For years the issue was a 
political football until a massive grass-roots effort forced 
legislators to respond. Today, the NIH is authorized to spend 
approximately $1.8 billion annually on new protocols, and the virus is 
largely under control in the United States.
    In conclusion, I wish to submit a letter of support from four 
theologians representing the Protestant, Catholic, Jewish and Islamic 
faiths (see Attachment 1).
    And finally, I wish to enter into the record a list of over 90 
disease groups, clinicians, foundations, universities and medical 
schools, all of whom have endorsed my testimony (see Attachment 2).
    While we prolong the stem cell debate, millions continue to suffer. 
It is time to harness the power of government and go forward.

Attachments.

                              Attachment 1

                                                  October 12, 1999.
Hon. J. Dennis Hastert,
House of Representatives,
Washington, DC.
    Dear Representative Hastert: Our opinions about embryonic stem cell 
research reflect several different religious perspectives: Jewish, 
Catholic, Protestant and Islam. While they do not represent a single 
voice from these religious communities, they do offer a collective 
belief that is based on similar views about certain moral and ethical 
questions.
    According to our religious beliefs, all human life must be 
protected. However, they also indicate that there is a significant 
difference between an embryo suspended in liquid nitrogen that will 
never be implanted inside a womb, and an unborn child who is already in 
the womb.
    Our religious beliefs also stress the importance of compassion. 
Thus, we support embryonic stem cell research because it would use 
these frozen or otherwise discarded embryos to help ease the suffering 
of those with catastrophic diseases such as diabetes, Parkinson's, 
cancer, Alzheimer's, heart disease, osteoporosis and arthritis.
    As theologians, we put a great deal of importance upon the need for 
ethical standards in biomedicine. Currently, stem cell research is 
being conducted solely in the private sector, where it is not subject 
to the important guidelines developed by the National Institutes of 
Health (NIH)--parameters that reflect critical input from ethicists and 
theologians. However, the guidelines would only come into effect with 
federal funding, which is another reason we support moving forward with 
the research under NIH. In addition, federal funding would not only 
speed the discovery of cures, but ensure infrastructure is in place 
that will guarantee ethical conduct and maximize benefit to society.
    Our religious beliefs tell us that federal funding of embryonic 
stem cell research is worthy of federal support for millions across the 
country who are suffering from diseases. We hope you will join us and 
support stem cell research through the NIH.
    We look forward to your support for this research, as the lives of 
millions are counting on you.
            Sincerely,
                                   Rabbi Elliot Dorff, Ph.D.,
                                           Professor of Philosophy, 
                                               University of Judaism
                                   Margaret Farley, Ph.D.,
                                           Professor of Christian 
                                               Ethics, Yale University 
                                               Divinity School
                                   Nancy J. Duff, Ph.D.,
                                           Associate Professor of 
                                               Theological Ethics, 
                                               Princeton Theological 
                                               Seminary
                                   Abdulaziz Sachedina, Ph.D.,
                                           Department of Religious 
                                               Studies, University of 
                                               Virginia
                                 ______
                                 

                              Attachment 2

    Editor's note: This is a list of over 100 disease groups, 
clinicians, foundations, universities and medical schools, all of whom 
are supportive of Christopher Reeve and his advocacy for stem cell 
research. This group of supporters was gathered with the assistance of 
the American Society for Cell Biology. For more information, please 
contact Tim Leshan at 301-530-7153 or at [email protected]

AIDS Action
Alliance for Aging Research
Alpha One Foundation
ALS Association
American Association for Dental Research
American Association for the Advancement of Science
American Association for the Study of Liver Diseases
American Association of Anatomists
American Association of Immunologists
American Autoimmune Related Diseases Association
American College of Obstetricians and Gynecologists
American Dental Education Association
American Foundation for AIDS Research
American Gastroenterological Association
American Medical Association
American Parkinson Disease Association
American Pediatric Society
American Physiological Society
American Society for Biochemistry and Molecular Biology
American Society for Cell Biology
American Society for Clinical Nutrition
American Society for Microbiology
American Society for Pharmacology and Experimental Therapeutics
American Society for Reproductive Medicine
American Society of Hematology
American Society of Human Genetics
American Society of Pediatric Hernatology/Oncology
Americans for Medical Progress
Association for Research in Vision and Ophthalmology
Association of American Medical Colleges
Association of American Universities
Association of Independent Research Institutes
Association of Medical School Pediatric Department Chairs
Association of Professors of Medicine
Association of Subspecialty Professors
Bay Area Bioscience Center
Biophysical Society
Boston University Medical Center
Canavan Research Fund
Cancer Treatment Research Foundation
Candlelighters Childhood Cancer Foundation
Cedars-Sinai Medical Center
Christopher Reeve Paralysis Foundation
Coalition of Advocates for Research on the Eye (CARE)
College of American Pathologists
Cooley's Anemia Foundation
Coriell Institute for Medical Research
Council of Graduate Schools
Endocrine Society
Federation of American Societies for Experimental Biology
Foundation Fighting Blindness
FRAXA Research Foundation
Friends of the National Institute of Dental and Craniofacial Research
Genetics Society of America
Hadassah
Harvard University
International Foundation for Anticancer Drug Discovery
International Longevity Center--USA, Ltd.
International Myeloma Foundation
Interstitial Cystitis Association
Jeffrey Modell Foundation
Johns Hopkins University
Joint Steering Committee for Public Policy
Juvenile Diabetes Foundation International
Kidney Cancer Association
Lankenau Medical Research Center
Lombardi Cancer Center
Medical College of Wisconsin
Medical University of South Carolina
Memorial Sloan-Kettering Cancer Center
Mount Sinai School of Medicine
Myasthenia Gravis Foundation of America
National Alliance for Eye and Vision Research
National Alopecia Areata Foundation
National Association for Biomedical Research
National Association of State Universities and Land-Grant Colleges
National Childhood Cancer Foundation
National Coalition for Cancer Research
National Health Council
National Psoriasis Foundation
Oncology Nursing Society
Paralyzed Veterans of America
Parkinson's Action Network
Parkinson's Disease Foundation
Patients' Coalition for Urgent Research (CURe)
Prevent Blindness America
Project A.L.S.
The Protein Society
PXE International
Radiation Research Society
Research!America
Research Society on Alcoholism
Research to Prevent Blindness
RESOLVE, National Infertility Association
Sjogren's Syndrome Foundation
Society for Pediatric Research
Society for Women's Health Research
The Genome Action Coalition (TGAC)
Treatment Action Group (TAG)
University of California, San Diego School of Medicine
University of Minnesota
University of Rochester Medical Center
University of Washington
University of Wisconsin-Madison
UPMC Health System
Washington University School of Medicine
Weill Medical College of Cornell University

    Senator Specter. Thank you very much, Mr. Reeve, for that 
very impressive testimony. We see your enthusiasm and your 
determination, notwithstanding your own personal situation, and 
we will work with you to try to get the research necessary to 
revitalize the spinal column.
    We will make a part of the record, Mr. Reeve, the long list 
of groups, clinicians, foundations, universities, and medical 
schools which support stem cell research, which you have 
provided to us, together with the letter which you made 
available, and the record will be able to handle all those 
names we cannot pronounce.
    Mr. Reeve. Thank you.
STATEMENT OF JENNIFER ESTESS, ACTOR/PRODUCER
    Senator Specter. We turn now to Ms. Jennifer Estess, who 
helped form the off-Broadway theater company, Naked Angels. She 
is the Executive Producer of a CBS original movie based on her 
life, scheduled to air early next year. She received her 
bachelor's degree in fine arts from the New York University and 
is now President of the Amyotrophic Lateral Sclerosis 
Foundation which she organized in 1998 after being diagnosed 
with ALS in 1997 at the age of 35.
    We thank you very much for joining us, Ms. Estess, and look 
forward to your testimony.
    Ms. Estess. Thank you.
    Good morning, Chairman Specter, Senator Harkin, and members 
of the committee. I also want to thank Christopher Reeve for 
inviting me here today to speak with him. It is an honor. And I 
want to thank you for inviting me to speak today.
    I am Jennifer Estess. I have ALS, also known as Lou 
Gehrig's disease, which is always fatal.
    As a little girl growing up in America, I had great plans. 
I dreamed of falling in love, getting married, and raising a 
family. I dreamed of making a difference.
    As a teenager, I learned from my sisters that hard work, 
commitment, and family would make my dreams a reality.
    As a woman of 35, my American dream was starting to come 
true. I was a producer living in New York City. I was working 
12-hour days and loving it. I was a healthy and strong young 
woman looking for Mr. Right and ready to take the next step.
    Then one day I started to feel tired and run down. I 
thought it was the flu. But weeks passed and the feeling did 
not go away. A city block seemed like a mile. Stairs were like 
mountains. And suddenly I was having trouble holding my 3-year-
old nephew in my arms.
    It was like a scene out of a bad movie when the neurologist 
turned to me and said, you have ALS. In 2 to 5 years, you will 
lose the ability to walk, to talk, to swallow, to breathe. You 
will die. There is no medicine I can give you.
    That first year ALS took my legs, the second my hands, my 
arms. Now I sit before you unable to breathe freely, but my 
mind is fine and it is alert. And I am still dreaming.
    The day of my diagnosis, I asked the neurologist why, if we 
could transplant hearts and lungs, could we not also replace 
damaged motor neurons, the cells destroyed in ALS? The 
neurologist shook his head. He told me, the cell transplants 
were science fiction.
    Three years later I am here to say that we may have 
discovered a way to replace the cells that carry messages from 
the brain to the muscles.
    Project ALS, which I built with my sisters and my friends, 
has assembled and funded a Dream Team of scientists. In the 
last 9 months, these scientists have produced stunning evidence 
that neural stem cell replacement can replace damaged motor 
neurons. These new cells may one day allow me to do the things 
I miss so much like brushing my hair, laughing out loud, and 
holding a cup of coffee.
    The evidence now shows that neural stem cell replacement is 
our best hope for treatment and cures, not only for ALS, but 
for Alzheimer's, Parkinson's, stroke, multiple sclerosis, and 
spinal cord injury affecting over 10 million Americans today. I 
am sure each of you will be touched by one of these devastating 
diseases.
    I believe it is upon us as brothers, sisters, fathers, 
mothers, Americans to join us in this growing family that will 
solve these problems. I also believe that stem cell research 
will be the answer for millions of people, babies, children, 
and adults, who will be blind-sided as I was. And I am here 
because I do not want anyone else to have to go through this.
    Make no mistake. ALS is a national disaster. We must land 
at the heart of this disaster. I hope that Congress, the 
National Institutes of Health, and the Food and Drug 
Administration will join Project ALS and me in pursuing the 
safest, shortest distance between stem cells and the patients 
who desperately need them.
    Each day I speak from inside my body which has now become a 
prison. I am still here and dreaming of an America that will 
protect the right to life, liberty, and the pursuit of 
happiness for all.
    Thank you so much.
    Senator Specter. Ms. Estess, we are very grateful to you 
for appearing here today. We understand the difficulties that 
you have described. Your voice is loud and clear and we hear 
the message. When you appear and Mr. Reeve appears and we have 
your circumstances in mind and know that you are representative 
of many, many people in America and in the world whose lives 
may be saved by stem cell research, it provides the kind of 
public understanding that I think is going to be necessary to 
change the law and activate this kind of National Institutes of 
Health funding which is the source of the real hope to deal 
with ALS, to deal with spinal cords, and to deal with heart 
disease, and to deal with Parkinson's.
    When you look at Senator Harkin and me and say that we will 
have the same problems, we could tell you some stories of our 
own. So, we thank you.
    Ms. Estess. Thank you.

STATEMENT OF LAWRENCE B. GOLDSTEIN, Ph.D., PROFESSOR, 
            DIVISION OF CELLULAR AND MOLECULAR 
            MEDICINE, UNIVERSITY OF CALIFORNIA, SAN 
            DIEGO SCHOOL OF MEDICINE; INVESTIGATOR, 
            HOWARD HUGHES MEDICAL INSTITUTE
    Senator Specter. Dr. Goldstein, we now turn to you, 
Professor of Pharmacology at the Division of Cellular and 
Molecular Medicine, investigator of the Howard Hughes Medical 
Institute at the University of California, Ph.D. from the 
University of Washington. You testified before the subcommittee 
at one of our early hearings in January of last year. We thank 
you for joining us and we appreciate your focus on the issue as 
to adult stem cells versus embryonic stem cells.
    Dr. Goldstein. And related topics, yes.
    Mr. Chairman, members of the subcommittee, I am Lawrence 
Goldstein. I am here today as a representative of the American 
Society for Cell Biology. The society represents 10,000 basic 
biomedical researchers most of whom work in our Nation's 
leading research universities and institutes.
    I am a professor now in the Department of Cellular and 
Molecular Medicine at the University of California, San Diego 
School of Medicine and an investigator in the Howard Hughes 
Medical Institute. Before moving to San Diego, I was a 
professor in the Department of Cellular and Developmental 
Biology at Harvard University for 10 years. My research focuses 
on the roles of protein motors in neuronal function and 
neurodegenerative disease.
    It is a pleasure to be here again today and it is a 
particular honor to be here with Ms. Estess and Mr. Reeve who 
have been such articulate advocates for biomedical research. My 
oral remarks will be a shorter version of my written remarks, 
so they will not be identical.
    Senator Specter. Your full written statement will be in the 
record, Dr. Goldstein.
    Dr. Goldstein. Thank you, Senator.
    I want to thank you, Senator Specter and Senator Harkin, 
for your leadership in ensuring that the NIH is funded 
sufficiently to pursue the most promising basic and clinical 
research opportunities. One such opportunity is human embryonic 
stem cell research. S. 2015, the Stem Cell Research Act of 
2000, which you have introduced, would allow federally funded 
scientists to not only use, but also to derive human embryonic 
stem cell lines from embryos that are in excess of clinical 
need and that would be donated with appropriate informed 
consent from in vitro fertilization clinics. These cell lines 
would be used for research purposes with the goal of developing 
new therapeutic strategies to treat devastating human disease.
    The American Society for Cell Biology stands firmly in 
support of this bill. We believe that permitting peer-reviewed, 
Federal funds to be used for this research is our best 
assurance that the research will be of the highest quality and 
benefit and performed with proper ethical oversight and public 
input.
    There are five supporting points I want to make.
    First, as you have heard, human embryonic stem cells have 
enormous potential research and therapeutic value because they 
appear to be capable of generating many, if not all, of the 
cell types that make up a human organism. Research work over 
the past 20 years using mouse embryonic stem cells has 
demonstrated that these cells by themselves cannot form an 
adult organism, but they can differentiate into any adult cell 
type. Most important, mouse embryonic stem cells have been used 
in a variety of ``proof of therapeutic principle'' experiments 
in several animal models of human disease. For example, these 
cells appear to be able to produce neural progenitors that can 
repair spinal cord damage and reconstitute various types of 
brain cells and neurons. If reproducible with human embryonic 
stem cells, we may be able to treat Parkinson's disease, 
Alzheimer's disease, and other diseases such as ALS. We may be 
able to produce bone marrow cells to treat cancer and other 
diseases and pancreatic cells to alleviate diabetes. In fact, 
we may be on the verge of a new era of medicine, one in which 
cell therapy could help restore normal function to a variety of 
affected tissues.
    Second, some have argued that so-called adult stem cells 
derived from adult tissues are of equivalent promise, less 
ethically compromised, and should therefore be pursued 
exclusively. But it is far too early to know if adult stem 
cells have the same potential as embryonic stem cells, whether 
they can be harvested in sufficient quantities to treat 
disease, and whether they can grow indefinitely as can 
embryonic stem cells. In fact, it is likely to take years to 
find out if adult stem cells will be useful for treating many 
diseases that may be treatable sooner with embryonic stem 
cells.
    Senator Specter. When you say years to find out, Dr. 
Goldstein, could you give us your judgment on approximately how 
long? Because we are really looking at an alternative here and 
the duration is a relevant factor in so many people not being 
treatable in the interim.
    Dr. Goldstein. There are many different claims about how 
long it will take to do these things. Adult stem cells could 
give us something in a year. It could be 10 years. Embryonic 
stem cells have shown, I think, greater promise in some of the 
animal models, and we could be looking at something in 2 to 3 
years.
    But we cannot work with one hand tied behind our back, as 
was so eloquently pointed out by Senator Harkin and by others. 
And, of course, if we delay with diseases that prove to be 
treatable with adult stem cells, we risk unnecessary delays for 
patients such as Ms. Estess who may die or endure needless 
suffering while the promise is being tested. Thus, it is 
critical that we not prohibit or hinder research in any of 
these areas.
    The third point I want to make is that the Stem Cell 
Research Act ensures that important avenues of medical research 
will not be restricted to the private sector. Precluding 
federally funded scientists from using human embryonic stem 
cells would effectively bar the majority of the Nation's most 
qualified researchers from pursuing scientific opportunities 
most likely to lead to rapid advances.
    In fact, past experience has proven that broad access to 
breakthrough discoveries increases the likelihood of novel and 
innovative extensions. The molecular biology revolution, as I 
am sure you know, which is responsible for enormous social, 
economic, and medical advances is the product of innovations 
and discoveries made by thousands of highly qualified and 
creative federally funded scientists who developed new 
techniques and applications.
    Fourth, an important feature of S. 2015 is that it will 
allow federally funded investigators to derive human embryonic 
stem cell lines with appropriate ethical safeguards. This is 
important because we know that a variety of poorly understood 
factors cause embryonic stem cells to lose their capacity to 
differentiate into all possible cell types. This loss of 
capacity may be caused by growth conditions, derivation 
conditions, or other variables of handling or storage. Enabling 
individual publicly funded investigators to derive cell lines 
using a variety of conditions is the best way to learn what 
conditions are critical to generate therapeutically useful 
cells.
    Fifth and finally, there are serious ethical implications 
to not proceeding. Thus, we have heard many well-meaning 
ethical concerns about the sacrifice of embryos to prepare stem 
cells. However, the embryos in question will be legally 
destroyed in any case, and I have to ask, is it ethical to 
literally throw away one of our best opportunities to help our 
friends, our parents, and our children, many of whom will 
suffer and die if we do not find suitable therapies?

                           PREPARED STATEMENT

    In closing, we have before us an unprecedented scientific 
opportunity to engage in a noble effort to develop new forms of 
medicine. That opportunity offers hope to the many millions of 
our citizens who rely on the shared stewardship of our 
scientists and our political leaders to enable science's 
achievements to relieve all people of the burden of serious 
disease.
    I want to thank you for your courage in providing 
leadership on this most important, indeed, life or death issue 
and for inviting my testimony today.
    [The statement follows:]

              Prepared Statement Lawrence S. B. Goldstein
    Mr. Chairman and members of the Subcommittee: I am Lawrence 
Goldstein. I am here today as a representative of the American Society 
for Cell Biology. The Society represents 10,000 basic biomedical 
researchers throughout the United States and the world, most of whom 
work in our Nation's leading research universities and institutes. It 
is my pleasure to appear before you again and it is a particular honor 
to be here with Mr. Reeve who has been such an articulate and effective 
spokesperson on behalf of biomedical research.
    I am a Professor in the Department of Cellular and Molecular 
Medicine at the University of California, San Diego School of Medicine. 
I am also an Investigator of the Howard Hughes Medical Institute. 
Before moving to San Diego I was a Professor in the Department of 
Cellular and Developmental Biology at Harvard University for 10 years. 
My research focuses on the molecular and genetic analysis of protein 
motors and their roles in neuronal function and neurodegenerative 
disease.
    I want to thank you, Senator Specter and Senator Harkin, for 
ensuring through your leadership of this important subcommittee that 
the NIH is funded sufficiently to pursue the most promising basic and 
clinical research opportunities. One such opportunity is embryonic stem 
cell research. S.2015, ``The Stem Cell Research Act Of 2000'' which you 
have introduced, would allow federally-funded scientists to not only 
use, but also to derive embryonic stem cell lines for research purposes 
with the goal of developing new therapeutic strategies to treat 
devastating human disease. The American Society for Cell Biology stands 
firmly in support of this bill.
    Just over a year ago, a milestone in biomedical research was 
achieved when human embryonic stem cell lines were obtained by growing 
cells from the inner cell mass of early stage human embryos. This 
discovery catalyzed a serious debate on Capitol Hill about whether 
federal funds should be used to support further research in this area. 
At issue is whether the merits of public funding and the dreadful 
burden of disease balance concerns about the origin of these special 
cells. Because of its great potential to treat disease and alleviate 
human suffering, the American Society for Cell Biology along with many 
other scientific organizations and societies have expressed strong 
support for Federal funding of this important research.
    The American Society for Cell Biology supports The Stem Cell 
Research Act Of 2000 for four major reasons:
    First, research work over the past 20 years using mouse embryonic 
stem cells has demonstrated the promise of embryonic stem, or ES, cells 
for basic research and potential disease therapy. These cells by 
themselves cannot form a mouse, but they can differentiate into any of 
the cell types that comprise a mouse. Mouse ES cells have been used to 
elucidate many important aspects of normal mouse embryology and 
development, but, most important, mouse ES cells are currently being 
used in a variety of ``proof of therapeutic principle'' experiments in 
several animal models of human disease. For example, these cells appear 
to be able to produce neural progenitors that can repair spinal cord 
damage and reconstitute brain cells that produce the chemicals that 
control cognition, motion and sensory perception. If reproducible with 
human ES cells we may be able to treat Parkinson's disease and 
Alzheimer's disease. We may be able to produce bone marrow cells to 
treat cancer and other hematopoietic diseases, and pancreatic cells to 
alleviate diabetes. In fact, we may be on the cusp of a new era of 
medicine, one in which cell therapy could restore normal function to a 
variety of affected tissues.
    It is important to note that some have argued that so-called 
``adult stem cells'', derived from adult tissues are of equivalent 
promise, less ethically compromised, and should therefore be pursued 
exclusively. But, notwithstanding important advances in the field of 
adult stem cell research, it is far too early to know if adult stem 
cells have the same potential as embryonic stem cells, whether they can 
be harvested in sufficient quantities to treat or cure disease, and 
whether they can grow indefinitely as can ES cells. For example, for 
juvenile diabetes, there is little reliable evidence at present that 
adult stem cells could be used for treatment, and thus embryonic stem 
cells are the best near-term candidates for therapy. Furthermore, 
embryonic, fetal and adult stem cells are very different from each 
other. It is not at all clear that they will prove to be 
interchangeable or equally receptive to manipulations that would make 
them useful for therapy. Thus, it is likely to take years to find out 
if adult stem cells will be useful for treating many diseases that may 
be treatable sooner with embryonic stem cells. For diseases that prove 
not to be treatable with adult stem cells, we risk unnecessary delays 
for patients who may die or endure needless suffering while the promise 
of adult stem cells is being tested. It is critical that we not 
prohibit or hinder research in any of these areas.
    Second, there is great medical need and urgency for stem cell 
research. To understand the need for rapid research progress with human 
pluripotent stem cells, one need look no further than many common, and 
often fatal, diseases such as cancer, heart disease and kidney disease. 
These diseases are treatable in whole or in part by tissue or organ 
transplants, but there are persistent and deadly problems of rejection 
and a woefully inadequate supply of suitable donor organs and tissues. 
In addition, the grim arithmetic of most organ transplants requires 
those who are seriously ill to wait for the tragic accidental death of 
another person so that they may live. Worse, for juvenile diabetes and 
many other diseases, there is not even a suitable transplantation 
therapy or other cure. Unless we use federal funds for all aspects of 
human pluripotent stem cell research new treatments for these 
conditions may be delayed by years, and many who might otherwise have 
been saved will surely die or endure needless suffering.
    Third, The Stem Cell Research Act of 2000 ensures that important 
avenues of medical research will not be restricted to the private 
sector. We agree with the National Bioethics Advisory Commission that, 
``relying on cell lines that might be derived exclusively by a subset 
of privately funded researchers who are interested in this area could 
severely limit scientific and clinical progress.'' The effect of 
precluding federally-funded biomedical from such work will effectively 
bar the majority of the Nation's most prominent and most-qualified 
researchers from engaging in this critical research. Excluding these 
publicly funded investigators will close off scientific opportunities 
to those most qualified to make rapid and dramatic advances towards 
using stem cells for the treatment of disease.
    We also know that a variety of poorly understood factors cause 
embryonic stem cells to lose their capacity to differentiate into all 
possible cell types. This loss of capacity may be caused by growth 
conditions, derivation conditions, or other variables of handling. 
Enabling individual publicly-funded investigators to derive cell lines 
using a variety of conditions in their own laboratories is the best 
route to finding out what conditions are critical to generate useful 
cells for therapeutic purposes. In the future, it is likely that cells 
prepared in one's own laboratory will have been derived, stored, and 
maintained in ways that maximize their potency for particular uses, 
whereas cells obtained from commercial sources are likely to be of 
unknown genetic background and history and therefore less useful for 
some important studies.
    Federal funding is also the best way to guarantee that stem cell 
therapies are developed with the greatest consideration of the public 
good. Left to the private sector alone, stem-cell derived treatments 
may only be pursued for diseases that commercial companies project to 
yield the largest profit if treated. Thus, market forces could create a 
situation where deadly, but less widespread, diseases are ignored.
    In fact, past experience has proven that allowing greater and more 
diverse access to breakthrough discoveries increases the likelihood of 
novel and innovative extensions. The Molecular Biology Revolution, 
which is responsible for enormous social, economic, and medical 
advances is the product of innovations and discoveries made by 
thousands of highly qualified and creative individual scientists who 
developed new techniques and applications. Our world would be very 
different today had this vital technology been restricted to just a few 
investigators.
    Fourth and finally, the American Society for Cell Biology supports 
The Stem Cell Research Act of 2000 because it guarantees that this 
important biomedical research will proceed with the highest possible 
ethical standards, and with sensitivity to the public concern about the 
origins of these cells. The National Biomedical Ethics Advisory 
Commission held numerous hearings and debates on this issue and 
solicited input from the public, scientific experts, and religious 
leaders from different faiths. After careful deliberation, the National 
Biomedical Ethics Advisory Commission concluded that it would be 
ethically permissible to prepare stem cell lines from embryos that had 
been obtained in the course of in vitro fertilization procedures, but 
were deemed by the donors and the physician to be in excess of the 
clinical need for the intended procedure: that is, those destined to be 
ethically and legally discarded.
    The National Biomedical Ethics Advisory Commission also recommended 
specific regulatory and oversight procedures to ensure that the 
creation and use of human stem cells would meet the highest ethical 
standards. In fact, the draft guidelines developed by the NIH to govern 
use of embryonic stem cells specify similar criteria to those suggested 
by the National Biomedical Ethics Advisory Commission. Analogous 
procedures are wisely specified in the Stem Cell Act of 2000. These 
guidelines include complete separation between those who conduct 
research and those who donate stem cells, thus preventing any potential 
effort to develop embryos for the purpose of research. For federally 
funded research, this bill would also prohibit the use of embryos that 
are purchased or sold, and will continue to ensure that human embryos 
are not created for research purposes.
    It is important to keep in mind that banning federal funding for 
human pluripotent stem cell research will not eliminate it. Such 
research will proceed in private industry and in other countries. This 
fact prompts serious concern that such work could be conducted in 
secret, without benefit of ethical regulation or public debate. Thus, 
prohibiting Federal funding will de facto prohibit public involvement 
and input into the future of this important field. Permitting peer-
reviewed federal funds to be used for this research, combined with 
public oversight, is our best assurance that research will be of the 
highest quality and performed with proper ethical oversight and public 
input. Federal funding of this research will require the scientific 
community and the government to work together to establish an 
appropriate set of rules for this research. These rules will ensure the 
advancement of critical medical research and maintain respect for 
public sensibilities.
    There are also serious ethical implications to not proceeding. 
Thus, while I am acutely and personally aware of the well meaning 
ethical concerns that have been expressed about the sacrifice of 
embryos to prepare stem cells, I am also cognizant that the embryos in 
question will be legally and ethically destroyed in any case. We must 
then ask: Is it ethical to literally throw away the opportunity to 
allow all people to benefit from the demise of these embryos? How can 
we justify not pursuing every reasonable means of finding cures for our 
friends, our parents, and our children, who will suffer and die if we 
do not find suitable therapies?
    In thinking about the answers to these questions, I am reminded of 
the many parallels between our debate today about the potential use of 
human embryonic stem cells to treat disease and past debates about 
organ transplants and the proper treatment of donor families. The 
famous heart transplant surgeon Dr. Christian Barnard, stated it best 
in response to questions he received about the ethics of heart 
transplantation. He said: ``Would it not be immoral to bury a heart 
when we have the ability to save a life?'' We submit that the answer is 
the same for human embryonic stem cells. Ethics, scientific 
opportunity, and medical need can surely be balanced.
    In closing, we have before us an unprecedented scientific 
opportunity to engage in a noble effort to develop new forms of 
medicine. That opportunity offers hope to the many millions of our 
citizens who rely on the shared stewardship of our scientists and our 
political leaders to enable science's achievements to relieve all 
people of the burden of serious disease.
    Thank you for your courage in providing leadership on this most 
important--indeed, life-or-death--issue, and for inviting my testimony 
today.

    Senator Specter. Thank you very much, Dr. Goldstein.
    It is now 1 o'clock and we have a news conference scheduled 
at 1:00 so that there may be broader coverage to what Mr. 
Christopher Reeve and Ms. Jennifer Estess have to say to 
America and the world. Your testimony has been eloquent, and I 
do not think that any questions would lead to embellishing it.
    I have never done this before, but I am going to invite 
people--I understand this is being carried live on C-SPAN I--to 
write to me and/or your own Senators as to whether you think 
there ought to be Federal funding used for stem cell research. 
You can write to me, Arlen Specter, United States Senate, in 
Washington, D.C., and I will get it. Or if you choose to write 
to your own Senator or Senators, send me a copy. I think it 
would be interesting to see what the response is.
    This is a very controversial issue and there are many 
people who feel very strongly about the factors which have been 
testified to here today. Like the issue of fetal tissue, it has 
become embroiled in another debate. We have heard three 
witnesses testify in opposition to your views, Mr. Reeve, Ms. 
Estess, and Dr. Goldstein, in opposition to my views. I think 
the balance of authority is that the embryos are discarded and 
will not be used, as Dr. Goldstein puts it, an ethical question 
in discarding them and in not using them.
    But this matter is going to be on the Senate floor and 
Senators pay attention to constituents. So, let us hear from 
you because we will post the tally on the website.
    Dr. Goldstein, do you want the last word?
    Dr. Goldstein. I would, if that is OK with you, Senator.
    Senator Specter. I am not guaranteeing you will have the 
very last word, but go ahead.
    Dr. Goldstein. I would not presume.
    I just want us to pose the scenario that Senator Brownback 
and others have suggested where we stop embryonic stem cell 
work and work exclusively on adult stem cells for 5 years, say. 
So, what happens if in 5 years we find out that adult stem 
cells are not going to do the job? What do we tell Mr. Reeve 
and Ms. Estess at that time? Sorry, we have to start over? 
There may not be another opportunity. I cannot accept that 
conclusion.
    Senator Specter. Well, you raise a good point. You know 
that stem cells from embryos will work, and that is what we 
have to make happen.
    Ladies and gentlemen, we thank you for coming today. It is 
a very large group here in the hearing room. Would you keep 
your seats please? Keep your seats until Mr. Reeve and Ms. 
Estess have a chance to move out of the room. Then we will be 
assembling in the outer corridor for the news conference.
    Thank you all very much, Mr. Christopher Reeve, Ms. 
Jennifer Estess, Dr. Lawrence Goldstein.

                         CONCLUSION OF HEARING

    Thank you all very much for being here. That concludes our 
hearing. The subcommittee will stand in recess subject to the 
call of the Chair.
    [Whereupon, at 1:04 p.m., Wednesday, April 26, the hearing 
was concluded, and the subcommittee was recessed, to reconvene 
subject to the call of the Chair.]


                           STEM CELL RESEARCH

                              ----------                              


                      THURSDAY, SEPTEMBER 7, 2000

                           U.S. Senate,    
    Subcommittee on Labor, Health and Human
     Services, and Education, and Related Agencies,
                               Committee on Appropriations,
                                                    Washington, DC.
    The subcommittee met at 9:35 a.m. in room SD-124, Dirksen 
Senate Office Building, Hon. Arlen Specter (chairman) 
presiding.
    Present: Senators Specter, Gorton, and Harkin.

               Opening statement of Senator Arlen Specter

    Senator Specter. Good morning, ladies and gentlemen. We 
will be proceeding with this appropriations Subcommittee on 
Labor, Health and Human Services and Education, and I am 
delighted to see our witnesses arriving. Traffic in Washington 
today is unusually bad, I am advised.
    Do not pause in the audience, Dr. Fischbach and Dr. 
Spiegel. Move right up into the chairs which were occupied 
yesterday by executives from Firestone and Ford, where the 
Appropriations Transportation Subcommittee, which I am a member 
of, had a hearing, and I want you to know that our question for 
you today will be much more difficult and tough than they were 
for the officials from Ford and Firestone.
    We have convened this session of the subcommittee to 
further our inquiry into stem cells. This is the sixth hearing 
which the subcommittee will have held on this very important 
subject going back to December of 1998, within a few weeks 
after the stem cell opportunities were disclosed. It was 
apparent at that time that stem cells had enormous potential as 
a veritable fountain of youth to replace cells in the human 
body which were diseased, and to solve many serious problems, 
many serious diseases.
    With some of the results now showing enormous progress on 
Parkinson's and progress on Alzheimer's and on spinal cord and 
on a wide range of medical problems, this sub committee felt a 
special responsibility to undertake this issue because it was 
in an appropriations bill in the mid-nineties where there was a 
prohibition against using Federal funds for stem cell research.
    There has been an interpretation by the General Counsel for 
the Department of Health and Human Services that Federal funds 
may be made on stem cell research once stem cells have been 
extracted from embryos, but Federal funds may not be used to 
extract these stem cells from embryos, and that still leaves a 
very wide area of restriction on Federal research.
    Let me note the arrival of my distinguished colleague from 
Iowa, Senator Harkin. It is nice to see you. We have an ongoing 
partnership which has survived the political process, as he 
says from time to time. When Democrats control the Senate he 
chairs this subcommittee. When Republicans now control, I 
chair, and we both learned a long time ago that if you want to 
get something in Washington you have to be willing to cross 
party lines.
    We have maintained our approach on a nonpartisan, 
bipartisan basis, and I think the results show, such as our 
funding for the National Institutes of Health, where we have 
increased the funding very, very materially.
    The three times of stem cells which are possible for 
extraction are embryonic stem cells, fetal tissue stem cells, 
and adult stem cells. Without going into the details, it is 
apparent scientifically, and we will hear from the experts on 
this today, that adult stem cells do not have the potential 
which is present with the embryonic stem cells.
    The issue has been raised as to the propriety of using 
embryos, and we have had in our hearings the leading opponents 
from both the House and the Senate who have testified, and we 
propose to present the full picture, and in addition to the 
hearing today we are going to be having a hearing next week, 
because we expect this issue to come to the Senate floor for a 
vote.
    The subcommittee report had included last year a change in 
this prohibition on Federal funding for stem cell research, and 
we removed it at full committee last year because it had the 
potential for tying up our appropriations bill and our Majority 
Leader, Senator Lott, agreed to bring the issue to the floor as 
a freestanding bill, which now will be taken up sometime this 
month, so we have had the full range of hearings.
    In my opinion the issue on stem cells is very much like the 
issue on fetal tissue, where there was a lot of controversy, 
pro-choice versus pro-life, and then it was finally established 
to the satisfaction of, I think, almost everyone that use of 
fetal tissue would not encourage abortions, but it would only 
be discarded fetal tissue.
    The matter with stem cells I think is highly analogous. 
These are embryos which are not going to be used. They have 
been created for in vitro fertilization, but they are to be 
discarded, so the issue boils down to whether they will be 
discarded and have no use, or whether they can be used to save 
lives, and I think that is a balance which comes out in favor 
of savings lives.
    But there is serious debate on this subject, and this 
subcommittee will pursue it as far as we can to set the stage 
at having a very meaningful floor debate, and I think the 
Senate vote will be very, very important not only on this 
subject but on the future of medical research as we have this 
opportunity for a veritable fountain of youth.
    Simultaneously with my red light going on--I have a little 
trouble understanding, frankly, why there are lights on for the 
chairman, but there were, so I will abide by them.
    Now I would yield to my distinguished colleague, the 
Ranking Member, Senator Harkin.

                Opening Statement of Senator Tom Harkin

    Senator Harkin. Thank you very much, Senator Specter. It is 
good to be back with you here in this subcommittee and back in 
the Senate after our brief break period. I want to thank you 
for your determined and continued leadership on a broad variety 
of health-related issues, especially, as you mentioned, 
leadership on the continued funding for the National Institutes 
of Health.
    I am pleased and honored to be a partner with you in those 
efforts. As Senator Specter said, we have a close bipartisan 
relationship in regards to medical research. We both belief in 
the strong potential that medical research holds for improving 
the lives of our fellow Americans and again I want to thank 
Senator Specter for his leadership on those issues, but more 
specifically on this issue that confronts us today.
    I am pleased that we have the opportunity to hear from our 
panelists on the issue of the final NIH guidelines for the 
conduct of embryonic stem cell research. These guidelines are a 
critical step forward in the pursuit of medical breakthroughs, 
and I believe they will help lead to new therapies and 
treatments that could save or improve the lives of countless 
Americans.
    I look forward to discussing both the scientific and the 
ethical impact of this research with our panelists today. Quite 
frankly, we are in a war, a war against cancer, spinal cord 
injury, and Parkinson's and diabetes and Alzheimer's and a host 
of other diseases and disorders which rob so many people and 
their loved ones of health.
    These enemies are in many ways more fierce than those 
encountered in any military operation in our Nation's history. 
The death toll from just one of these, cancer, over the past 10 
years exceeds the casualties for all of the military wars in 
our history. In fact, there have been nearly five times as many 
American casualties in the war against cancer in the past 10 
years than all of the military wars fought since the American 
Revolution.
    In World War II we left no stone unturned to gain victory. 
In the Persian Gulf War we developed the smart bombs that could 
go down Main Street and take a left on Elm and go into the 
window of Apartment Number 25. We developed these smart bombs. 
Well, we should have the same attitude when it comes to our 
wars against disease.
    We should leave no stone unturned, and that is why the NIH 
guidelines and the ability to move forward with this research 
are so important. I believe we are at the crossroads of what 
could be a smart bomb against many diseases, and the key to 
victory, and we should pursue it.
    Now, we will hear, I know, testimony as to whether we 
should use embryonic stem cells or just use adult-derived stem 
cells. Well, again my feeling on this is that all avenues 
should be pursued. I have often said that basic research is 
like having a number of doors that are closed. If you have 10 
doors closed and you open one, chances are, your odds are, what 
about 10 to 1 that you are going to find something, but you 
open all 10 doors your odds are pretty good that you are going 
to make the breakthrough, and so rather than just going down 
one pathway I think we need to go down a number of different 
pathways.
    I believe the NIH guidelines are an important step forward 
so that scientists have a procedural and ethical framework to 
pursue this research in an ethical and sound manner.
    Again, I just would close by saying that stem cell research 
holds a lot of hope, a lot of potential for millions of people 
around the world who are sick and in pain, and I think it is 
wrong for us to prevent or delay our world-class scientists 
from building on the progress that has been made. If we are 
going to win this war, we have to pursue every path.
    Thank you very much, Mr. Chairman.
    Senator Specter. Thank you, Senator Harkin, and thank you 
for your cooperation and your leadership on this subcommittee 
on these very important issues.

STATEMENT OF GERALD D. FISCHBACH, M.D., DIRECTOR, 
            NATIONAL INSTITUTE OF NEUROLOGICAL 
            DISORDERS AND STROKE, NATIONAL INSTITUTES 
            OF HEALTH, DEPARTMENT OF HEALTH AND HUMAN 
            SERVICES
    Senator Specter. The focus of today's hearing is to be on 
the National Institutes of Health guidelines which were 
published very recently, August 25, for research involving 
human embryonic stem cells, and our first witness is the 
distinguished Director of the National Institute of 
Neurological Disorders and Stroke, a position which Dr. Gerald 
Fischbach has held since 1998.
    Prior to that time, he served as chairman of the 
Neurobiology Department at the Harvard Medical School and the 
Massachusetts General Hospital, past president of the Society 
for Neuroscience, a member of the National Academy of Science, 
a very, very distinguished research scientist, representative 
of the caliber of the National Institutes of Health.
    Dr. Fischbach, welcome. You can skip the traffic problems 
and go right to the guidelines.
    Dr. Fischbach. Thank you, Mr. Chairman and Senator Harkin. 
I am pleased to be here today, and actually relieved to be here 
on time, but pleased to be here to discuss the use the medical 
promise of human pluripotent stem cells and the new NIH 
guidelines released a couple of weeks ago, which are designed 
to ensure that such cells are used in an ethical and legal 
manner.
    Stem cells indeed are revolutionary tools, perhaps the most 
exciting development in biomedical science in my career, at 
least dealing with the nervous system. They are exciting for 
many reasons but they do, as very few other therapies do, offer 
the possibility of reversing the course of disease, of actually 
curing disease in addition to controlling symptoms.
    This for the first time offers the possibility of reversing 
disease processes and replacing damaged parts in the body, and 
the promise is due to two properties of stem cells. One, they 
can proliferate, and they can multiply and produce a large 
supply of these tools and cells. Second, they can be, under 
proper coaxing, forced to differentiate. That is, to develop 
into mature cells that actually can function as missing parts 
in various organs.
    Now, there are many, many uses of stem cells. One of them 
certainly will be in the area of novel tools for drug 
discovery. There is some evidence that they will be the vehicle 
that will enable breakthroughs in gene therapy, and they 
certainly will teach us a great deal about the fundamental 
biology of development and the pathogenesis of disease.
    But perhaps the most striking aspect of step cell biology 
which has attracted most attention is the ability to transplant 
them into the body, and this is what has caused such great 
excitement, and if I can for a moment speak about the nervous 
system of the brain, with which I have been particularly 
involved, this is a vital, vital property of these cells and 
the nervous system.
    With rare exception our nerve cells are nonrenewable 
resources. When a nerve cell dies in our brains it is simply 
not replaced, or not replaced easily. With rare exceptions 
cells do not divide in the brain, so when they degenerate they 
are lost. Therefore, the ability to replace cells is extremely 
important and major advances have been made, as you said, 
Senator Specter, in Parkinson's disease and a number of other 
disorders.
    In Parkinson's disease, we know exactly where the lesion 
starts. We know the type of nerve cell that degenerates 
initially, and it has been possible, using tissue, fetal 
tissue, to replace these degenerating cells and to reverse the 
symptoms of Parkinson's disease in animal models and in first 
studies in human trials. This offers great promise, and 
challenges us to do more research on stem cells and to replace 
the fetal tissue implants with a much more controllable and 
useful source.
    But it does not stop there. Stem cells have been used 
recently in treatment of sequellae of stroke, ALS, spinal cord 
injury, tumors of the nervous system, and strangely enough, 
because it is widely disseminated, even in multiple sclerosis.
    Now, there is a hierarchy of stem cells. I think the 
consensus of scientific opinion is that cells proliferate and 
differentiate when isolated from the adult nervous system, but 
there is a common view that cells proliferate more plentifully, 
and they have a wider range of choices, if taken from the fetal 
or embryonic tissue, so we would like to discuss that 
hierarchy.
    Now, much remains to be learned. Although I have emphasized 
the promise, much research needs to be done about how we can 
control the proliferation of these cells and how we can urge 
them to differentiate in one half or another, and how we can 
prevent overproduction of cells.
    Now, we recognize that there are difficult ethical issues 
involved in formulating policies in dealing with human 
embryonic stem cells, which in my view are the most promising 
at the present moment of all the stem cells isolated to date, 
and therefore the NIH has formulated guidelines. They have 
proceeded slowly and in public.
    This committee has had several public hearings. The NIH 
developed a working group from the Advisory Council to the 
Director which included scientists, ethicists, patients, 
patient advocates, lawyers, and laymen, and that resulted in 
the guidelines which we are prepared to discuss, and I want to 
thank you for the opportunity to present this statement, and I 
would be prepared to answer questions that may arise.
    Senator Specter. Thank you very much, Dr. Fischbach. Before 
calling on Dr. Spiegel, I would like to yield to our 
distinguished colleague, Senator Slade Gorton, for an opening 
statement if he has one.
    Senator Gorton. Please go ahead.

STATEMENT OF ALLEN M. SPIEGEL, M.D., DIRECTOR, NATIONAL 
            INSTITUTE OF DIABETES AND DIGESTIVE AND 
            KIDNEY DISEASES, NATIONAL INSTITUTES OF 
            HEALTH, DEPARTMENT OF HEALTH AND HUMAN 
            SERVICES
    Senator Specter. All right, then. We turn to Dr. Allen M. 
Spiegel, appointed Director of the National Institute of 
Diabetes and Digestive Kidney Diseases last year, and prior to 
that appointment Dr. Spiegel held significant positions in the 
Institute, including Director of Intramural Research, and Chief 
of Metabolic Disease, an M.D. from Harvard and a B.A. from 
Columbia. Thank you for joining us, Dr. Spiegel, and we look 
forward to your testimony.
    Dr. Spiegel. Thank you very much, Mr. Chairman, and 
Senators Harkin and Gorton. I appreciate the opportunity to 
appear before you today with Dr. Fischbach on behalf of my 
colleagues at the National Institutes of Health to discuss the 
promise of research on human pluripotent stem cells, research 
that will be funded under the recently released NIH guidelines.
    The guidelines prescribe procedures to enhance both the 
scientific and ethical oversight of this important area of 
research. As described in our written statement, the guidelines 
specify the documentation and assurances that must accompany 
requests for NIH funding for research utilizing human 
pluripotent stem cells. These include cells that were derived 
in the private sector from human embryos that were created for 
fertility treatment, were frozen, and are in excess of clinical 
need. They also set out specific conditions for the use of 
pluripotent stem cells derived from fetal tissue.
    These guidelines will encourage openness, help make certain 
that researchers can make use of these critical research tools, 
and help assure public access to the practical medical benefits 
of research using these tools.
    You have heard from Dr. Fischbach of the potential 
applications of research on human pluripotent stem cells to 
developing treatments for neurological disorders. In fact, this 
research holds great potential for leading to new cell 
therapies for many other types of disease, such as those 
involving the heart and the liver.
    Perhaps one of the best examples of the promise of this 
line of research is in the treatment of Type 1 diabetes, or 
juvenile onset diabetes. This form of diabetes is characterized 
by the inability to produce insulin, the hormone necessary for 
sugar metabolism. Type 1 diabetes occurs when the body's immune 
system attacks and destroys its own insulin-producing islet 
cells in the pancreas. The standard treatment is to try to 
control the sugar level with insulin injections. This is 
exceedingly difficult even in adults, and much more difficult 
in children with the disease. Transplantation of insulin-
producing islet cells, in theory, offers a much better approach 
to controlling sugar levels.
    In a recent published study, seven patients receiving islet 
transplants became completely independent of the need for 
insulin injections. NIH is currently funding a multi-center 
trial of the protocol used in this study to determine if the 
same high rate of success can be achieved in a larger number of 
patients. A successful outcome to this trial, as exciting as it 
would be for patients with Type 1 diabetes, and for their 
families, will only serve to underscore the limitation in the 
supply of islets available for transplantation compared with 
the need.
    While many approaches to address the islet supply problem, 
including research on adult stem cells, should be and are being 
vigorously pursued, human pluripotent stem cells offer the 
greatest promise of providing a limitless source of islet cells 
for transplantation in patients with Type 1 diabetes.

                           PREPARED STATEMENT

    The release of the NIH guidelines now allows us to fund 
this research that could lead to a cure for Type 1 diabetes and 
for other serious diseases for which there is no satisfactory 
current treatment.
    Thank you, Mr. Chairman. Dr. Fischbach and I would be 
pleased to respond to questions you and the other members may 
have.
    [The joint statement follows:]
  Prepared Joint Statement of Gerald D. Fischbach and Allen M. Spiegel
    Mr. Chairman, Senator Harkin, and Members of the Subcommittee, I am 
Dr. Gerald Fischbach, Director of the National Institute of 
Neurological Disorders and Stroke. I am accompanied by Dr. Allen 
Spiegel, Director of the National Institute of Diabetes and Digestive 
and Kidney Diseases. We are before you again to discuss one of the most 
exciting areas of biomedical research: the enormous potential of human 
pluripotent stem cells to treat and cure debilitating and deadly 
diseases.
    Only two years ago, researchers discovered and isolated these 
primordial cells--whose existence in humans had been theorized but 
never proven--the precursors of most of the other cells in the body. 
Their unique properties of self-renewal and the ability to 
differentiate into the full spectrum of other cell types make them 
ideal candidates for repairing and replacing tissues and organs ravaged 
by disease. New more effective treatments, and maybe even cures, might 
be developed for juvenile-onset diabetes, Parkinson's Disease, spinal 
cord injury, ALS or Lou Gehrig's disease, Alzheimer's Disease and many 
other brain disorders. There is similar potential for the treatment of 
cancer and heart disease. Virtually every realm of medicine and human 
health might benefit from this innovation. Stem cell research could 
alleviate a great deal of human suffering.
    Chairman Specter, you and Senator Harkin, in particular, have 
encouraged the National Institutes of Health (NIH) to invest our 
resources in stem cell research in order to pursue its enormous 
opportunities. Patients who are suffering from the most deadly and 
disabling diseases have also asked that NIH fund this promising arena 
of research. We believe that federal funding would encourage openness, 
stimulate more discoveries and translate the promise of this research 
into practical use more quickly, efficiently, and effectively--and with 
procedural safeguards.
    Recognizing that the ethical issues related to this research 
required careful consideration, NIH and the Department of Health and 
Human Services were committed to developing Guidelines to help ensure 
that pluripotent stem cell research funded by NIH would be conducted in 
a legal and ethical manner. In drafting the Guidelines, the NIH sought 
the advice of scientists, patients and patient advocates, ethicists, 
clinicians, lawyers, the National Bioethics Advisory Commission, and 
members of Congress. Draft Guidelines were published in the Federal 
Register last December. The NIH reviewed and considered all comments in 
preparing the final Guidelines.
    We are pleased to inform you that, on August 25, NIH published 
final Guidelines for research using human pluripotent stem cells. NIH 
is prepared to begin receiving applications immediately. As soon as the 
oversight process is in place, NIH will be in a position to fund such 
research. We expect broad interest from researchers seeking funding for 
pluripotent stem cell research, and we are hoping to begin funding this 
research as soon as possible. For procedural reasons, the soonest that 
awards could likely be made is early next year.

                          WHAT ARE STEM CELLS?
    Human pluripotent stem cells are a unique scientific and medical 
resource. They can develop into most of the specialized cells and 
tissues of the body, such as muscle cells, nerve cells, liver cells, 
and blood cells, and they are self-renewing, making them readily 
available for research, and potentially, for treatment purposes. 
Scientists derived these unique cells from human embryos and from fetal 
tissue.

            WHY ARE HUMAN PLURIPOTENT STEM CELLS IMPORTANT?
    There are three reasons why the isolation of human pluripotent stem 
cells is so important to science and the future of public health.
    First, pluripotent stem cells could help us to understand the 
complex events that occur during human development.
    Second, human pluripotent stem cell research could also 
dramatically change the way we develop drugs and test them for safety 
and efficacy. Rather than evaluating safety and efficacy of a candidate 
drug in an animal model of a human disease, these drugs could be tested 
against a human cell line that had been developed to mimic the disease 
processes. This would not replace whole animal and human testing, but 
it would streamline the road to discovery, and ensure that only the 
safest drugs are tested in humans.
    Third, and perhaps the most far-reaching potential application of 
human pluripotent stem cells, the generation of cells and tissue could 
be used for ``cell transplantation therapies.'' Such therapies are 
aimed at diseases and disorders resulting from the destruction or 
dysfunction of specific cells and tissue. Although donated organs and 
tissues can sometimes be used to replace diseased or destroyed tissue, 
the number of people suffering from such disorders far outstrips the 
number of organs and tissues available for transplantation. Pluripotent 
stem cells, stimulated to develop into specialized cells and tissue, 
offer real hope for the possibility of a renewable source of 
replacement cells and tissue to treat a myriad of diseases, conditions, 
and disabilities for which replacement tissue is in short supply. 
Examples of these include neurological disorders (including spinal cord 
injuries and ALS), diabetes, burns, heart disease, and arthritis.

                     REQUIREMENTS OF THE GUIDELINES
    The Guidelines prescribe procedures to ensure that NIH-funded 
research in this important arena is conducted in an ethical and legal 
manner. They specify the documentation and assurances that must 
accompany requests for NIH funding for research utilizing human 
pluripotent stem cells. These Guidelines will encourage openness, help 
make certain that researchers can make use of these critical research 
tools, and help assure public access to the practical medical benefits 
of research using these cells. The Guidelines accomplish these goals in 
the following ways.
    First, the Guidelines help ensure that embryos will not be created 
for the purpose of deriving human pluripotent stem cells to be used in 
NIH-supported research. Investigators seeking NIH funds are required to 
provide documentation that the human pluripotent stem cells were 
derived from frozen embryos that were created for the purpose of 
fertility treatment and that were in excess of clinical need. They 
require a clear separation between the fertility treatment and the 
decision to donate embryos for this research. In addition, the donation 
of the human embryos must be made without any restriction regarding the 
individual who may be the ultimate recipient of the cells for 
transplantation. Similarly, researchers wishing to use human fetal 
tissue to derive stem cells must demonstrate that they are in 
compliance with all applicable laws and regulations. The Federal 
statute applicable to NIH-funded fetal tissue transplantation research 
also includes provisions creating a separation between the decision to 
terminate a pregnancy and the decision to donate fetal tissue for 
research.
    Second, the Guidelines ensure that individual choosing to donate 
embryos cannot receive any inducements, monetary or otherwise. The 
Guidelines detail specific elements that must be included in the 
informed consent to help ensure that potential donors receive 
sufficient information to allow them to decide whether or not to donate 
human embryos for this type of research. The Guidelines require review 
and approval by an Institutional Review Board (IRB) to ensure that 
consent was informed, voluntary, and meaningful.
    Third, the Guidelines require accountability on the part of the 
researcher. Detailed documentation must be submitted to NIH to 
demonstrate compliance. For example, the grantee institution must sign 
an assurance that the research to be conducted is in compliance with 
the Guidelines, and that the institution will maintain documentation to 
support the assurance. The researcher/grantee institution must submit a 
sample informed consent document, with patient identifier information 
removed, a description of the informed consent process, and 
documentation of IRB review.
    Fourth, the Guidelines specify types of research that the NIH will 
not fund. For example, NIH will not fund any research that seeks to 
derive pluripotent stem cells from human embryos, research utilizing 
pluripotent stem cells that were derived from human embryos created for 
research purposes, or any research that seeks to derive or utilize stem 
cells from embryos that were created using somatic cell nuclear 
transfer (cloning technology).
    Fifth, the NIH has designed an oversight process that will provide 
an extra level of protection, above and beyond standard peer review of 
grant applications, to ensure that researchers have complied with the 
Guidelines. A newly-created NIH working group called the Human 
Pluripotent Stem Cell Review Group (HPSCRG) will review documentation 
submitted by researchers demonstrating that they are in compliance with 
the Guidelines. Members of the HPSCRG will subsequently make 
recommendations to its parent committee, the Center for Scientific 
Review Advisory Committee. NIH will not fund research or allow existing 
funds to be used for research using human pluripotent stem cells 
derived from human embryos or human fetal tissue until the required 
compliance documentation receives HPSCRG review and approval of the NIH 
Center for Scientific Review Advisory Committee. Continued compliance 
with the Guidelines will be a term and condition of the NIH award.

           HUMAN PLURIPOTENT STEM CELLS AND DIABETES RESEARCH
    One of the best examples of the promise of this line of research is 
in the treatment of Type 1 diabetes. Research on islet cell 
transplantation and stem-cell biology offers compelling opportunities 
for the development of new, innovative approaches for treating and 
ultimately curing this disease.
    Type 1 diabetes, often referred to as juvenile diabetes, is 
characterized by the inability of the body to produce insulin, a 
hormone necessary for glucose metabolism. This form of diabetes occurs 
when the body's immune system attacks and destroys its own insulin-
producing islet cells in the pancreas. As a result of inadequate 
insulin production, glucose does not enter cells as readily as when 
insulin levels are normal. The standard treatment is to try to control 
the glucose level with insulin injections.
    Transplantation of insulin-producing islet cells is an alternative 
approach to controlling glucose levels. In a recent study, seven 
patients receiving islet transplants became completely independent of 
the need for insulin injections. NIH is currently funding a multi-
center trial of the protocol used in this study to determine if the 
same success can be achieved in a larger number of patients. A 
successful outcome to this trial, as exciting as it would be for 
patients with Type 1 diabetes, will only serve to underscore the 
limitation in the supply of islets available for transplantation 
compared to demand. While many approaches to address the islet supply 
problem, including work on cell bioengineering and adult stem cells are 
being vigorously pursued, human pluripotent stem cells offer the 
greatest promise of providing a limitless source of islet cells for 
treating and curing Type 1 diabetes.

      HUMAN PLURIPOTENT STEM CELL RESEARCH AND THE NERVOUS SYSTEM
    As significant as the promise of stem cells is for the treatment of 
diabetes, the potential of stem cells for treating diseases of the 
nervous system is equally impressive. The most obvious and exciting use 
of stem cells in neurological disorders is to replace nerve cells lost 
to disease or injury. Many diseases destroy particular types of nerve 
cells, and mature nerve cells cannot produce new cells to replace those 
that are lost. Animal experiments have demonstrated that the potential 
exists for coaxing stem cells to specialize and replace the dopamine 
cells that are lost in the brain through Parkinson's disease. A similar 
approach might also apply to several other neurological disorders. 
Human pluripotent stem cells, given appropriate control signals, might 
specialize to replace the lost acetylcholine producing nerve cells in 
Alzheimer's disease, to restore lost motor neurons in ALS, or to 
produce inhibitory cells to help restrain electrical activity in 
epilepsy.
    Replacing lost nerve cells is only the beginning of the list of 
possible therapeutic applications for stem cells. For some disorders, 
such as multiple sclerosis, stem cells might replace supporting cells, 
such as the glial cells, which provide the insulation necessary to 
allow some nerves to conduct electrical impulses rapidly. In addition 
to their potential in replacement therapy, stem cells can provided 
nutritive factors that might prevent the loss of nerve cells in the 
first place. Stem cell strategies might be useful for correcting 
inherited defects. For example, in disorders that devastate children's 
brains, we might rely on the ability of stem cells to migrate widely in 
the brain and supply the vital missing enzyme that leads to early and 
tragic death from Tay-Sach's disease. In addition, stem cells might 
regenerate the many different kinds of complex brain tissue that are 
damaged as a result of brain trauma or stroke. Transplanted stem cells 
might also supply natural growth and survival chemicals to pave the way 
for regeneration of remaining healthy neural tissue following spinal 
cord injury. Recent findings suggest that stem cells might be harnessed 
to seek out and destroy brain tumor cells that evade surgery or 
radiotherapy. The list of possible applications of stem cells continues 
to grow as we learn more about these cells.

                               CONCLUSION
    Mr. Chairman, we appreciate the opportunity to discuss this 
promising and extraordinary science and are pleased to respond to any 
questions you may have.

    Senator Specter. Thank you very much, Dr. Spiegel. As you 
men have testified, I have made notes of the kinds of diseases 
which could be cured by stem cells and I have noted the 
following, and I would like to ask you if I am correct and if 
there are others, and the follow-up question is going to be, 
how many Americans, in ball park figures, are affected by these 
diseases?
    Parkinson's, amyotrophic lateral sclerosis, strokes, spinal 
cord, tumor, multiple sclerosis, heart, liver, diabetes? Is 
that the total? How about the impact on cancer, if any?
    Dr. Fischbach. Cancer should be included in that list. 
There is evidence for stem cells homing to tumors to deliver 
toxins to tumor cells.
    Senator Specter. Could you give a ball park figure as to 
how many Americans suffer from these ailments?
    Dr Spiegel. I will address that with an example. For liver 
disease, we need to understand that liver transplantation is a 
cure for liver failure caused by hepatitis viruses, by 
autoimmune diseases, by many other conditions.
    Thousands of Americans die every year awaiting liver 
transplants. Therefore, an alternative to the insufficient 
supply of cadaveric livers is vital, and thousands of American 
lives could be saved if cell therapy for liver disease could be 
developed as an alternative to liver transplantation.
    Senator Specter. We may have a short answer. Senator Harkin 
has anticipated this issue and has listed these categories, and 
I would be interested in your response to put it into the 
record beyond the hearsay document which Senator Harkin has 
handed me: cardiovascular disease, number of persons affected, 
58 million; autoimmune, 30 million; diabetes, 16 million; 
osteoporosis, 10 million; cancer, 8.2 million; Alzheimer's, 4 
million; Parkinson's, 1.5 million; severe burns, .3 million, 
spinal cord injuries, .25 million; birth defects, 150,000 per 
year, total of 28,400,000.
    Does that sound about right to you, Dr. Fischbach?
    Dr. Fischbach. Yes, it does, and if you are listing the 
number of people affected by those disorders, that does sound 
right, certainly.
    Senator Specter. That is about half of America.
    Senator Harkin. Well, heart disease gets about half the 
people in America.
    Senator Specter. Well, that is quite an impressive list. 
Thank you, Senator Harkin.
    With respect to the opportunities for adults themselves, I 
know you have already testified that the adults themselves are 
not as well-directed, and we are going to have another panel in 
opposition to your views, but could you elaborate, Dr. 
Fischbach, on why adult stem cells do not eliminate the need 
for stem cells from embryos, as you earlier generalized?
    Dr. Fischbach. That is actually the perfect way to state 
it, and I think we can both respond to that. I believe that 
stem cells have been discovered in adult tissues, actually 
quite surprisingly in the brain, and that they do have the 
capacity to proliferate, and to differentiate. Indeed, they may 
even switch identities, and cells from other organs, from the 
blood, have some promise of forming nerve cells in tissue 
culture, but I think these are early reports and the conversion 
to a neuronal phenotype is not complete, and the overwhelming 
evidence is that they do not generate sufficient numbers of 
nerve cells to be useful in the brain.
    So I think the way you phrased it is exactly right. It 
would not be responsible of us to use these cells in place of 
embryonic stem cells, which at the present time most of the 
advances I described are using embryonic stem cells, and that 
is where the lead effort is right now, and the most promise.
    Senator Specter. Well, could you specify which of these 
enumerated diseases have promise with the embryonic stem cells 
and which do not with the adult stem cells?
    Dr. Fischbach. It is hard to say do not, because the 
research is such that there can be, and we are hoping for 
further breakthroughs in the use of stem cells from adult 
tissues, but the embryonic stem cells have already been used in 
the treatment of Parkinson's disease, stroke and some aspects 
of spinal cord injury, and adult stem cells have not.
    Senator Specter. My red light has just gone on. I will 
yield to Senator Harkin.
    Senator Harkin. Thank you, Mr. Chairman. Just a couple of 
follow-ups. I expect many scientists have been anxious to get 
started on this research, and waiting for these guidelines for 
sometime now. Do you have any idea how many researchers have 
already told NIH that they want to apply for funding?
    Dr. Fischbach. I do not think we have those numbers in yet. 
I have seen one report that one of the developers of embryonic 
stem cells has had 150 requests the day after the stem cells 
were published, but I do not have the numbers for NIH receipt.
    Senator Harkin. A number of privately funded researchers 
are already working on stem cells. What will the impact on the 
field be once NIH starts funding this research, since it is 
already being done in the private sector? Tell us what, once we 
move ahead with these guidelines, assuming we do, what will be 
the impact on the field?
    Dr. Spiegel. I can comment in part relating to the previous 
question. Our institute now funds several dozen investigators 
who have expressed interest in working on these human 
pluripotent stem cells, particularly with reference to the 
pancreatic islets and the liver cells.
    We support a very large portfolio of research currently on 
adult stem cells. We are very vigorously supporting research in 
that area. These same researchers, recognizing the incredible 
potential of the human embryonic stem cells and pluripotent 
stem cells from fetal tissue, are eager to be able to do this. 
I think the impact will be enormous, because--although some 
private sector investigators are working in that field--we need 
to harness the entire brain power the NIH is capable of 
mobilizing.
    Dr. Fischbach. I think it will make the research more open, 
more public, more published, and more diverse, and better.
    Senator Harkin. And since there are guidelines now for the 
private sector, I was told one day it was sort of like the Wild 
West, there is no real guidelines, and while the guidelines 
promulgated by NIH are not forced on the private sector, these 
are not mandatory on private researchers, is it your 
understanding that once NIH publishes guidelines like this it 
sort of--because NIH is so big and the funding comes to NIH 
that the private researchers then would follow and sort of come 
under the umbrella of these guidelines? Would that be your 
understanding?
    Dr. Fischbach. Senator, that is one reason I am proud to be 
at the NIH. I think the NIH sets the standard for the field and 
for the world, and I think you behave differently at some risk, 
and I think the guidelines will influence industry. Certainly 
the guidelines specify we will fund research using embryonic 
stem cells derived from human embryos only according to strict 
regulations and rules, and that will have a big influence.
    Senator Harkin. Again, for the lay person's mind like mine, 
would you again, as much as you can in lay person's terms, 
discuss the news that came out over the summer about the 
finding that they could use adult bone marrow cells, stem 
cells, that could differentiate into other cell lines, and once 
that came out some people said to me, well then, you do not 
need to use embryonic any more now we have this, and I think 
there is some confusion out there, because I talked to a lot of 
people and they said, well, you do not need the embryonic. We 
now know that we can take bone marrow cells, adult stem cells, 
and they can differentiate into, I think it was liver--
neurological cells.
    So please, explain what this is all about.
    Dr. Fischbach. Again, we can both take a shot at that.
    My understanding of that paper is that it is an important 
observation in that it does show that bone marrow, which we 
have known for 40 or 50 years has diverse developmental 
potential, and some of those cells--remember, bone cells are 
turning over all the time in our body, unlike nerve cells.
    So there are stem cells in bone marrow to replace the 
marrow, especially during the war on radiation injury and 
marrow transplants, and this report pointed out that when the 
cells are treated in a certain way in tissue culture, that a 
few of the cells would begin to express--in other words, begin 
to manifest certain proteins that are characteristic of nerve 
cells, and they look a little bit like nerve cells under the 
microscope, but there is no demonstration yet that this will be 
a realistic supply of cells yet, and there is no demonstration 
that these cells will become the type of nerve cells that are 
needed. It is like an existence proof. Something can happen, 
but we do not know how far down the road they will go to be 
useful.
    So while it is an interesting first step, I cannot tell 
whether it is 1 year, 5 years, 10 years, or a generation before 
those cells will be truly clinically useful, so I think it 
should not preclude--I do not agree with the statement that we 
have shown this, therefore you do not need human embryonic stem 
cells.
    Dr. Spiegel. Since you asked in lay terms, let me try a 
sports analogy. We are talking in terms of human pluripotent 
stem cells capable of playing any position on a team, and 
having a limitless supply, a bench strength, that has 
incredible depth.
    In terms of the adult stem cells, there is new evidence 
that they may not have the high degree of versatility of human 
pluripotent stem cells. If we use a baseball analogy, adult 
stem cells may be like a utility infielder who can play a 
couple of positions in the field, but it is not necessarily 
good at all positions. Much more research needs to be done to 
see whether adult stem cells can really play every position in 
the sense of combating mutiple diseases. They do not have the 
same bench strength.
    In embryonic stem cells from mice, there are several cell 
lines that are replicating, that is, dividing endlessly in many 
labs and being used in limitless supply. I think that is a 
critical difference.
    When we think in terms of our goal, which is the treatment 
of disease and alleviating suffering, we have to think in terms 
of the range of disease possibility. The embryonic human 
pluripotent stem cells can address every one of these diseases. 
The adult stem cell research may, and we hope it will, be 
successful in certain areas, but not necessarily every area.
    Senator Specter. There is a vote in process right now on 
the Senate floor, to be followed by another vote, and we are 
going to take a very brief recess. We will return as promptly 
as we can, and at that time we will turn to the next panel of 
distinguished witnesses, Dr. Prentice and Dr. Roth, and we 
would ask you, Dr. Fischbach and you, Dr. Spiegel, to stay, and 
after we hear the testimony from those expert witnesses we may 
want to have some interchange, and so we stand in recess for 
just a few minutes.
    The subcommittee will resume.
    Although both Senator Harkin and I have expressed our views 
on this subject by introducing legislation to remove the 
prohibition so that there may be funding on stem cell research, 
we have both had many contacts from constituents who have a 
different point of view, and whatever our personal views may 
be, the subcommittee considers it indispensable to hear all 
sides, and earlier in our hearings we had heard the leading 
opponents of stem cell research both in the Senate and in the 
House as witnesses before this subcommittee, and now we turn to 
two distinguished witnesses who are opposed to stem cell 
research, Dr. David A. Prentice and Micheline M. Mathews-Roth. 
Dr. Mathews-Roth, if you would step forward.

STATEMENT OF DAVID A. PRENTICE, M.D., Ph.D., PROFESSOR 
            OF LIFE SCIENCES, INDIANA STATE UNIVERSITY
    Senator Specter. We will hear first from Dr. Prentice, 
professor of life sciences, Indiana State University, and an 
adjunct professor of medicaid and molecular genetics for 
Indiana University School of Medicine. He also serves as 
science fellow for United States Senator Sam Brownback, who had 
testified earlier in opposition to stem cell research at one of 
our prior hearings.
    Dr. Prentice is a founding member of Do No Harm, the 
Coalition of Americans for Research Ethics. He received his 
bachelor of science in cellular biology and his Ph.D in 
biochemistry from the University of Kansas, parenthetically, 
Dr. Prentice, my home State. We welcome you here and look 
forward to your testimony.
    Dr. Prentice. Mr. Chairman, thanks for this opportunity to 
appear before you today to talk about this extremely important 
issue, stem cell research. Your colleague, Senator Brownback, 
has stated previously his position that federally funded human 
embryonic stem cell research is illegal, immoral, and 
unnecessary, and others have discussed at length the arguments 
related to illegal and immoral.
    My point today would be to talk at some length about the 
unnecessary aspect, whether we should actually be doing human 
embryonic stem cell research, so I would like to spend some 
time talking about that, because I feel at times the statements 
that have been made are somewhat misleading.
    Mr. Chairman, because embryonic stem cells are derived from 
very early embryos in the continuum of human development, they 
should have the ability to form virtually every tissue needed 
in the body. It is for this reason that many are anxious to see 
these cells used for possible clinical applications.
    You are also well aware that production of human embryonic 
stem cells does require the destruction of human embryos. There 
are some scientific reasons why human embryonic stem cells may 
not be as desirable as adult stem cells. One is simply the fact 
that tissues generated by these cells will cause transplant 
rejection, the same as any normal organ transplant will require 
the use of toxic immunosuppressive drugs, possibly for the 
lifetime of the patient.
    Now, one possible solution might be to clone the patient, 
destroy that embryo, essentially the patient's own twin to 
divide the cells, but this complicates the debate even more 
with an additional controversial technique.
    Another possible problem may be control of differentiation 
of embryonic stem cells to form the desired tissue, rather than 
other tissues, or possibly even a tumor.
    Senator Specter. What was it you just said, Dr. Prentice, 
your last point?
    Dr. Prentice. Another problem might be precise control of 
the differentiation of the embryonic stem cell so that we get 
the desired tissue.
    Now, one other concern that has come up in terms of 
embryonic stem cells, these have been termed pluripotent, able 
to form most, not necessarily all stem cell types, but actually 
published reports indicate that they are totipotent, able to 
form all tissues of the developing human, or possibly even an 
integral human embryo itself.
    The publications regarding human or primate embryonic stem 
cells and their derivation note that, unlike mass embryonic 
stem cells, human and primate embryonic stem cells can form not 
only the tissues that become part of the human body, but also 
trophoblast tissue.
    Now, you are probably well aware that it is the trophoblast 
tissue that surrounds what become the embryonic stem cells that 
allows the embryo to implant into the uterine wall and nurtures 
early development. This is the tissue layer of the embryo that 
is destroyed when the embryonic stem cells are derived.
    Now, reformation of this tissue layer in cultures of 
embryonic stem cells could actually lead to reformation of 
complete human embryos in culture, able to survive if implanted 
into a womb. This means actually the possible production and 
destruction of thousands of new humans in culture created with 
Federal funds, which would be a direct violation of Federal law 
and NIH's own guidelines. Image of a virtual embryo body shop 
springs to mind.
    If we could turn to my main goal here, talking about the 
ethical alternative of adult stem cells, there have been a 
number of statements critical of this alternative and their 
abilities, and I am somewhat mystified by these statements, Mr. 
Chairman, because they seem to have been made without a 
thorough reading of the scientific literature, especially 
within the last 1 to 2 years.
    For example, it has been stated that embryonic stem cells 
will be in clinical use before adult stem cells, but to date 
there have been no publications regarding the current clinical 
use of human embryonic stem cells. The statement was made 
earlier about clinical use for Parkinson's and stroke, but 
those particular uses used transplants of fetal brain tissue, 
not embryonic stem cells.
    I have, and I will be submitting for the record, a number 
of references regarding current clinical uses, including 
cancer, lupus, various other immune diseases, corneal scarring.
    Another criticism that has been leveled is that there are 
only a few types, but more and more we are finding there are 
many different types of adult stem cells, and these essentially 
are pluripotent, just as has been claimed for the embryonic 
stem cells.
    In fact, in June of this year, a group in Sweden performed 
an experiment with mice using adult neural stem cells, showing 
these cells are pluripotent. They repeated an experiment done 
in 1984 using mouse embryonic stem cells, an experiment, I 
might add, which has not been done and ethically should not be 
done with human embryonic stem cells.
    The group stated that these studies suggest stem cells in 
different adult tissues may be more similar than previously 
thought, and perhaps in some cases have a developmental 
repertoire close to embryonic stem cells.
    I realize I am short on time, Senator, and I will be 
introducing----
    Senator Specter. Your red light is on, but you may proceed.
    Dr. Prentice. Thank you, sir. If I could just hit a few of 
the high points, as we say, others have said adult neural stem 
cells can be stimulated to grow even while still within the 
brain if given sufficient signal. One report notes adult neural 
stem cells can be multiplied in culture and are, quote, similar 
to human embryonic stem cells, end quote.
    Other adult stem cells have been identified in muscle, and 
just 2 days ago a report in the Journal of Cell Biology 
announced purification of adult stem cells from the muscle of a 
mouse model of Duchenne's muscular dystrophy. These cells were 
introduced intravenously injected back into the dystrophic 
mice, and resulted in muscle regeneration and partial 
restoration of dystrophon expression in the mice.
    Diabetes, one of the leading killers in the United States, 
previous witnesses mentioned the use of adult stem cells from 
cadavers, and this is a very promising technique, but obviously 
the patient could use their own stem cells. This would be a 
distinct advantage.
    In March of this year, adult pancreatic stem cells from 
mice were used to reverse diabetes in these animals. These 
animals are a model of Type I, or juvenile diabetes. After 
treatment the mice no longer needed insulin shots to survive.
    Other criticisms are that the adult stem cells are hard to 
access. They do not multiply well in culture. In August, 
researchers showed that human adult bone marrow stem cells can 
take up a new job description, if you will, and be changed into 
neurons. The report notes that these grow readily in culture, 
are readily accessible, and provide a renewable population.
    In July, two groups, one in the United States and one in 
the U.K., found human adult bone marrow stem cells could form 
liver and they noted, we could avoid problems with current 
liver transplants where the patient's body rejects the foreign 
organ. Another one said, this would suggest maybe we do not 
need any type of fetal stem cell at all, that our adult bodies 
continue to have stem cells that can do this stuff, perhaps not 
precise scientific language, Senator, but I think they make the 
point that we do seem to have within our bodies these cells, 
pluripotent adult stem cells, which can take care of organ and 
tissue regeneration.
    Some have urged research on both types of stem cell should 
go forth, but the President's own National Bioethics Advisory 
Commission has said that because human embryos deserve respect 
as a developing form of human life, destroying them is, quote, 
justifiable only if no less morally problematic alternatives 
are available for advancing research, close quote.
    Senators, the scientific literature overwhelmingly 
demonstrates that adult stem cells are already fulfilling the 
goals only hoped for with embryonic stem cells, making 
destruction of human embryos completely unjustifiable.
    Mr. Chairman, essentially, and we are really talking about 
a human rights issue, it has been said that these human embryos 
targeted for destruction to derive embryonic stem cells will 
die anyway, so we should get some good out of them, possibly to 
benefit those with life-threatening diseases.

                           PREPARED STATEMENT

    If we follow this path of devaluing human life and 
sacrificing one set of lives for the potential benefit of 
others, how long might it be before those patients with life-
threatening diseases will hear the same phrase? They are going 
to die anyway. Let us get some good out of them. Whom will we 
choose to assign value, and who will dare to make those 
choices? I implore you not to follow this path.
    I thank you once again for this opportunity to discuss this 
extraordinarily important topic with you, and I will be pleased 
to respond to any questions you might have.
    [The statement follows:]

                Prepared Statement of David A. Prentice
    Mr. Chairman and Members of the Subcommittee, I thank you for this 
opportunity to appear before you today to talk about this extremely 
important issue of stem cell research. Your colleague, Senator 
Brownback, has stated previously his position that federally funded 
human embryonic stem cell research is ``illegal, immoral, and 
unnecessary''. Others have discussed at length the arguments related to 
the ``illegal'' and ``immoral'' aspects, but not enough has been said 
regarding the reasons that it is ``unnecessary'', and in fact it would 
appear at times that some of the information which has been brought 
forth is misleading. Therefore, I would like to spend some time with 
you today discussing this aspect in terms of an ethical alternative to 
embryonic stem cells, the so-called ``adult stem cells'', and the 
scientific information which indicates that this alternative actually 
does make destruction of human embryos completely unnecessary.
    Mr. Chairman, while I am certain that you and the other Members of 
this Subcommittee are by now well aware of the basic information 
regarding stem cells, for the benefit of those in the audience who are 
relatively new to this debate I would like to give just a brief 
background. A stem cell is a cell that can proliferate with almost 
unlimited potential, maintaining a pool of growing and dividing cells, 
with the added ability that some of the daughter cells can 
differentiate into specific cell types. Thus, a stem cell allows for 
replenishment of itself while providing for specific functional tissues 
needed by the body. Stem cells have been known for many years, such as 
the blood stem cells in our bone marrow which continually replenish the 
red and white cells and platelets in our bloodstream. Embryonic stem 
cells (ES cells) have been isolated from mice for 20 years. In the fall 
of 1998, there were two reports on isolation of human embryonic stem 
cells; in Dr. John Gearhart's laboratory at Johns Hopkins, the workers 
actually isolated embryonic germ cells from early human fetuses after 
elective abortion, while Dr. James Thomson's laboratory in Wisconsin 
isolated true embryonic stem cells from human embryos a few days old. 
Because embryonic stem cells are derived from embryos quite early in 
the continuum of human development, they should have the ability to 
form virtually any tissue needed in the body. It is for this reason 
that many are anxious to use these cells for possible clinical 
applications in repair of damaged or diseased tissues. As you are also 
well aware, part of the debate revolves around the requisite 
destruction of the human embryos for derivation of these embryonic stem 
cells.
    There are, however, some scientific reasons why use of these human 
embryonic stem cells is less desirable than use of adult stem cells. 
One is simply the fact that tissues generated by these cells will face 
transplant rejection, as would any normal organ transplant, and require 
use of toxic immunosuppressive drugs, perhaps for the lifetime of the 
patient. While one possible solution to this problem would be to clone 
the patient and destroy that embryo, the patient's ``twin'', to derive 
the cells, this complicates the debate even more with an additional 
controversial technique. Another possible problem may be control of the 
differentiation of these embryonic stem cells such that they form the 
desired tissue, and not other tissues or even a tumor.
    One concern and potential problem is that while these embryonic 
stem cells have been termed ``pluripotent'', able to form most but not 
all cell types, published reports indicate that they are actually 
``totipotent'', able to form all tissues of the developing human, or 
even the integral human embryo itself. The publications regarding human 
or primate embryonic stem cell derivation note that, unlike mouse 
embryonic stem cells, human and primate embryonic stem cells can form 
not only tissues which become part of the human body, but also 
trophoblast tissue. Trophoblast is the tissue layer of the early embryo 
that allows it to implant into the uterine wall, forms part of the 
placenta, and essentially nurtures early development. It is this layer 
which is removed in the destruction of the early embryo. Re-formation 
of this tissue layer in cultures of embryonic stem cells could lead to 
re-formation of complete human embryos in culture, able to survive if 
implanted into a womb. This means the possible production and 
destruction of thousands of new human embryos in culture, created with 
Federal funds, a direct violation of Federal law and NIH's own 
guidelines. The image of a virtual ``embryo body shop'' springs to 
mind. This concern certainly impacts the debate again in terms of the 
legal and moral aspects.
    But now turning to the main topic, the ethical alternative of adult 
stem cells. Let me note for those unfamiliar with the terminology that 
these cells reside not only in adults but in all of our bodies from 
birth, one rich source being cord blood from the umbilical cords of 
newborns. There have been a number of statements critical of this 
alternative and the abilities of adult stem cells to fulfill the 
promise of tissue repair. I am somewhat mystified by these statements 
which malign the capabilities of adult stem cells, and can only assume 
that the statements have been made without a thorough reading of the 
scientific literature, especially within the last 1-2 years.
    For example, it has been stated that embryonic stem cells will be 
in clinical use before adult stem cells. To date, there have been no 
publications regarding the current clinical use of human embryonic stem 
cells. However, human adult stem cells have already been used 
successfully for several clinical treatments. There is a wealth of 
references on the use of stem cells as an adjunct in the treatment of 
numerous types of cancer, including brain tumors, ovarian cancer, 
various solid tumors, multiple myeloma, breast cancer, and non-
Hodgkin's lymphoma. The adult stem cells are purified from bone marrow 
or circulating blood, preferably from the patient, and after treatment 
to destroy the cancer cells the patient is given back their own 
purified stem cells (an ``autologous'' transplant.)
    Adult stem cells have also been used successfully to treat several 
autoimmune diseases such as multiple sclerosis, systemic lupus, 
juvenile rheumatoid arthritis, and rheumatoid arthritis, as well as 
anemias. A report in August noted one of the first uses of an adult 
neural stem cell line for treatment of stroke. This summer there were 
two reports of the use of corneal stem cells to restore vision to 
patients with corneal scarring in which normal corneal transplants 
would not work. Again, in most of the cases, the patient's own adult 
stem cells were used. Adult stem cells have been used to treat children 
with a condition which leads to bone and cartilage deformities. And, in 
April of this year, ``adult'' bone marrow stem cells were used in what 
seems to be the first successful example of human gene therapy, in 
which infants with a severe immunodeficiency seem to have been cured by 
giving them their own stem cells with the defective gene replaced.
    One of the common criticisms leveled against adult stem cells is 
that there are only a few types, and they are not pluripotent, lacking 
the range of ability to differentiate into all tissues which is claimed 
for embryonic stem cells. In point of fact, human embryonic stem cells 
also have not been shown at this time to be able to generate all 
tissues in the body; nonetheless, it is presumed that they have 
pluripotent potential.
    For adult stem cells, however, in June of this year a group in 
Sweden performed an experiment with mice using adult neural stem cells 
which shows that these adult cells are pluripotent. The study 
replicates an experiment done in 1984 using mouse embryonic stem cells, 
an experiment which has not been done-and ethically should not be done-
with human embryonic stem cells. The Swedish group's results confirm 
that adult neural stem cells are pluripotent-the cells were able to 
participate in formation of heart, lung, intestine, liver, nervous 
system, muscle, and other tissues. The authors state that ``--these 
studies suggest that stem cells in different adult tissues may be more 
similar than previously thought and perhaps in some cases have a 
developmental repertoire close to that of ES cells.''
    Other reports note that adult neural stem cells can be stimulated 
to grow even while still within the brain if given sufficient signal. 
Several sites with the human brain have been found to contain neural 
stem cells, and that these cells can be used in animals to repair 
spinal cord damage and other neural damage. One report notes that these 
human adult neural stem cells can be multiplied in culture, established 
as continuous cell lines, and are ``similar to human embryonic stem 
cells.'' The authors of this study also note that ``The fact that this 
revolutionary strategy uses autologous neuronal material means that it 
has all of the advantages of biosafety, histocompatibility, and 
neurophysiological efficiency. Furthermore, it does not raise the 
ethical and moral questions associated with the use of embryonic or 
heterologous material.''
    Other adult stem cells identified include those in muscle, and 
these cells also appear capable of reprogramming to form different cell 
types including blood, bone, and cartilage. Just 2 days ago a report in 
the Journal of Cell Biology announced purification of adult stem cells 
from muscle of a mouse model of Duchenne's muscular dystrophy. The 
cells were intravenously injected back into the dystrophic mice, and 
resulted in muscle regeneration and partial restoration of dystrophin 
expression in the mice. Transplantation of these cells engineered to 
secrete a bone protein resulted in the cells changing from muscle to 
bone cells, and accelerated healing of a skull defect in mice.
    Diabetes is one of the leading killers in the United States. There 
have recently been reports of successful treatment using adult 
pancreatic stem cells from cadavers. Again, if a patient could use 
their own stem cells, there would be a distinct advantage. In this 
respect, in March of this year adult pancreatic stem cells from mice 
were used to reverse diabetes in these animals, employing their own 
stem cells. After treatment, the mice no longer needed insulin shots to 
survive.
    Other criticisms of adult stem cells are that they are hard to 
access, and that they do not multiply well in culture. One of the most 
versatile and accessible adult stem cell populations is in bone marrow. 
In August researchers showed that these human adult bone marrow stem 
cells can take up a ``new job description'' and be changed into 
neurons. The authors noted that this source could provide ``a virtually 
limitless supply'' of nerve cells. According to reports, the cells 
``grow rapidly in culture'', ``are readily accessible'', and ``provide 
a renewable population.'' They also add that ``Autologous 
transplantation overcomes the ethical and immunological concerns 
associated with the use of fetal tissue.''
    In July, two groups, one in the U.S. and one in the U.K., found 
that human adult bone marrow stem cells could form liver. In 
considering the promising potential of these stem cells, one researcher 
noted, ``We could avoid problems with current liver transplants where 
the patient's body rejects the foreign organ.'' Another said ``This 
would suggest that maybe you don't need any type of fetal stem cell at 
all-that our adult bodies continue to have stem cells that can do this 
stuff.''
    Others have found that human adult bone marrow stem cells can be 
reprogrammed to form bone, cartilage, muscle, and fat cells. You might 
ask, ``Why would I want fat cells?'' Such tissues have significant 
applications in reconstructive surgery. Numerous reports in the last 
year demonstrate the significant proliferative capacity of adult stem 
cells in culture when given the proper signals.
    And one more note on the tremendous potential of adult stem cells 
in terms of tissue engineering and reconstructive repair. There are now 
numerous reports where adult stem cells have been seeded onto polymer 
matrices. Testing these constructs in a sheep model system, autologous 
adult heart cells have been used to form heart valves and aorta.
    Some have urged that research on both adult and embryonic stem 
cells should go forth. However, the President's own National Bioethics 
Advisory Commission has said that because human embryos deserve respect 
as a developing form of human life, destroying them ``is justifiable 
only if no less morally problematic alternatives are available for 
advancing research.'' Senators, the scientific literature 
overwhelmingly demonstrates that adult stem cells are already 
fulfilling the goals only hoped for with embryonic stem cells, making 
destruction of human embryos completely unjustifiable.
    Mr. Chairman, respected Members of this Subcommittee, it has been 
said that these human embryos targeted for destruction to derive 
embryonic stem cells will die anyway, and so we should get some good 
out of them, possibly to benefit those with life-threatening diseases. 
If we follow this path, devaluing human life and sacrificing one set of 
lives for the potential benefit of others, how long might it be before 
those patients with life-threatening diseases will hear the same 
phrase-"they're going to die anyway, let's get some good out of them?'' 
To whom will we choose to assign value, and who will dare to make those 
choices? I implore you not to follow this path.
    Thank you once again for this opportunity to discuss this 
extraordinarily important topic with you, and I would be pleased to 
respond to any questions you might have.

STATEMENT OF MICHELINE M. MATHEWS-ROTH, M.D., ASSOCIATE 
            PROFESSOR OF MEDICINE, HARVARD MEDICAL 
            SCHOOL
    Senator Specter. Thank you very much, Dr. Prentice. We will 
come to questions as soon as we hear from Dr. Mathews-Roth, who 
is an associate professor of medicine at Harvard Medical 
School, and an associate physician at Brigham Women's Hospital. 
She performs basic science and clinical research and 
photobiology. Her clinical studies focus on developing 
photoprotective treatments for photosensitivity diseases. Dr. 
Mathews-Roth received her M.D. from New York University.
    Thank you for joining us, Dr. Mathews-Roth, and we look 
forward to your testimony.
    Dr. Mathews-Roth. I just want to say I am not here as a 
representative of either the Brigham or Harvard Medical School, 
but as a physician who deals with patients with a genetic 
disease that is called erythropoietic protoporphyria. We call 
it EPP because nobody wants to say erythropoietic 
protoporphyria, and I am also concerned about the ethics 
involved in embryonic and fetal stem cell research.
    I did leave a copy of my full testimony. I will try and 
summarize it. I also would like to leave two sets of clippings 
that describe some of the adult stem cell work as an appendix 
to my testimony, if that is okay.
    Senator Specter. Dr. Roth, your full statement will be made 
a part of the record, as will the articles you referred to, 
without objection.
    Dr. Mathews-Roth. I think it is crucial for people and 
legislators alike to realize the only way to obtain embryonic 
stem cells is, as Dr. Prentice was saying, is to literally tear 
apart growing human embryos, and I brought a picture. this is 
the zona palusa, which sort of holds everybody together. This 
is the trophoblast layer that Dr. Prentice was talking about. 
These little guys here are the inner cell mass, or stem cells 
that people are interested in getting, so literally you have to 
break these guys apart to get it.
    Now, what we all know is that the embryological fact is 
that we do know when a new life begins, and that is at 
fertilization, when egg and sperm join. Now, stem cells are not 
embryos, but as Dr. Prentice was pointing out, the human ones 
seem to be a little bit more totipotent than mouse embryo 
cells, but remember, you have to break apart an embryo to get 
the stem cells, and this is really the ethical objection, so I 
think we all have to ask ourselves, do we really want to allow 
the deliberate killing of even one of the youngest members of 
our species to help sick members of our own species also?
    It is a tough ethical question, but again, can we really do 
this because we know ethically that a good end can never 
justify using a bad means, or an evil means to attain it.
    The fact that the embryos which would be used for stem cell 
harvest are spares and would die or be destroyed anyway still 
does not justify killing them for their stem cells. I think 
there is a big difference between that and between people who 
sign organ donation cards. They make the informed decision. I 
have decided after I die a natural death, or am killed in an 
accident, or whatever, then I will donate my organs.
    There is a heck of a lot of difference between that and 
deliberately killing a growing human to get organs, and again 
also I am concerned with the problem that Dr. Prentice 
mentioned of rejection, immunological rejections. What do we 
suggest? Adult stem cells, really emphasizing research on adult 
stem cells.
    And also I would add, which I sort of forget to put in my 
testimony, the use of embryonic tissues from spontaneous 
abortions or miscarriages, I was talking to a physician, or a 
Ph.D. who does this kind of work, and yes, if you establish a 
network and let hospitals know that you are interested in doing 
this work, and there is no ethical problem here, these babies 
have died a natural death, their parents can say yeah, just 
like to a child or somebody who is killed in an accident, you 
may use the organs.
    These are potential forms, or potential sources also of 
adult, quote-unquote, if you want to call them that, stem 
cells. Well, I guess the term is really fetal stem cells, so 
no, we are not leaving patients in the lurch. We are saying, 
look, there is so much promise for adult stem cells that really 
the research should be emphasizing those.
    There is mounting evidence of the versatility of these 
adult stem cells that may make the use, as Dr. Prentice was 
saying, of fetal cells unnecessary, and really, I agree with 
this statement--well, actually it is the NBAC statement, if 
there was another morally and scientifically acceptable 
alternative, use it. Do not use the embryological stem cells.
    I want to quote--I was going to, and I have in my testimony 
several examples, as Dr. Prentice did, of the use of adult stem 
cells. I just really want to quote one of those in a little bit 
more detail. ``Adult bone marrow stem cells such as the marrow 
stromal cells have proven to be extremely versatile, as 
Woodberry, et al., in a paper describing the transformation of 
marrow stromal cells into nerve cells''--and I believe Dr. 
Fischbach referred to that, and so did Dr. Prentice.
    He says the marrow cells are readily accessible, overcoming 
the risk of obtaining neural stem cells from the brain, and 
provide a renewable population. Autologous transplantation, 
that is, using the patient's own cells, overcomes the ethical 
and immunological concerns associated with the use of fetal 
tissue.
    Moreover, marrow stromal cells grow rapidly in culture, 
precluding the need for immortalization, and immortalization, I 
believe, adds a little foreign virus to the cells, and 
differentiates into neurons exclusively with the use of a 
simple protocol, and other scientists, Allison et al. reports 
that human hematopoietic stem cells can differentiate into 
liver cells in the tissues, in the livers of people receiving 
bone marrow transplant, so if they can do it in people 
receiving bone marrow transplants, they can also do it in 
people who need help with their liver.
    The exciting thing about these bone marrow stem cells is 
that they are able to be transformed in all of the three 
original embryonic layers, the ectoderm--and this is the 
example for that, is the nervous tissue--mesoderm, muscle, fat, 
and bone cells, and endoderm, example is the liver cells, and 
it is just a matter of time that more things will be developed 
about them.
    In my testimony I was going through the four objections 
that the NIH guidelines had suggested to liver cells, or to use 
of adult cells, so I am sort of briefly going through that. One 
of their objections is valid. They said that in diseases caused 
by genetic defect, the defect would also be present in the 
patient's stem cell.
    However, this problem can be solved by ex vivo gene 
therapy. That is gene therapy to cells that you take out of the 
person's body, then you put them back in, adding the normal 
gene to bone marrow stem cells which are then treated to get 
them to develop into, say, whatever needed cells you need, like 
liver cells or muscle, fat, or bone, or nervous tissue.
    Successful gene therapy to bone marrow cells has also been 
reported by French workers, who cured a severe combined 
immunodeficiency disease in children with gene therapy to the 
bone marrow, and I am happy to tell you my colleagues and I are 
also studying gene therapy using bone marrow cells. We have 
cured the mouse model of the genetic disease that I am 
studying, erythropoietic protoporphyria with ex vivo gene 
therapy.
    Again, the advantage of that is that you--and obviously we 
are hoping to eventually develop it for people. The advantage 
of that is, if you take the person's own stem cells, treat 
them, cure them with gene therapy, put them back into the 
patient, they are not going to get rejection and it is going to 
work.
    So in summary, I just want to say the most ethical and 
scientifically appropriate steps for the Government to take 
concerning stem cell research are first to revoke the 
guidelines and to continue the ban on research on embryonic 
stem cells, and two, I would say also to impose a ban on 
research on stem cells obtained from induced aborted fetuses, 
because there is a danger of their potential of encouraging 
abortion to obtain these cells, and we have heard stories about 
doctors, well, sort of suggesting to patients, why don't you 
wait a week or two, or something like that.
    I think there are problems here. However, on the other 
hand, approving research on fetal cells derived from 
miscarriages, and I think it is somewhat of a myth to say that 
this is not practical, having done this the Government must 
strongly encourage and fund research in adult stem cells as 
well as research on umbilical cord blood and placental stem 
cells. Although these latter ones may cause immunological 
problems they are probably still worth looking at.
    So that the full potential of adult stem cells and possibly 
these other forms, certainly the fetal stem cells from aborted, 
from miscarried fetuses can be really looked at and explored to 
find out just how versatile these cells really are, and this 
way I think we are not deserting our patients, but we are doing 
things that would be ethically acceptable to everyone.

                           PREPARED STATEMENT

    I would hate to have to tell my patients that, boy, I have 
to destroy a human life before I can treat you, and I do not 
think we have to do that. I think work on adult stem cells will 
avoid that awful problem.
    Thank you, sir.
    [The statement follows:]
            Prepared Statement of Micheline M. Mathews-Roth
    Dr. Mathews-Roth is an Associate Professor of Medicine at the 
Harvard Medical School, and a Physician at the Brigham & Women's 
Hospital, in Boston, Massachusetts. She performs clinical and basic 
research on the genetic disease, erythropoietic protoporphyria.

    [NOTE.--The views presented here are Dr. Mathews-Roth's--she is NOT 
acting as a spokesperson for either the Harvard Medical School or the 
Brigham & Women's Hospital.]

    The stated purpose of the Guidelines for Research Using Human 
Pluripotent Stem Cells issued by the National Institutes of Health is 
to ``establish procedures to help ensure that NIH-funded research in 
this area is conducted in an ethical and legal manner''. However these 
guidelines, as presently written, are ethically very questionable. 
Since the guidelines deal with the beginnings of individual human 
lives, and thus an issue of great importance, American society cannot 
tolerate ethical and scientific errors concerning this issue. It is 
crucial for people to understand that the only way to obtain embryonic 
stem cells is to literally tear apart growing human embryos. Although 
the Guidelines state that ``NIH funds may not be used to derive human 
pluropotent stem cells from human embryos'' (though funds can be used 
to derive stem cells from fetal tissue), nevertheless, these guidelines 
are allowing scientists to let the youngest members of our species, 
even though their bodies consist of just a few cells, to grow for a few 
days, and then deliberately kill them by breaking them apart to obtain 
their stem cells. My understanding is that the bill, S. 2015 is similar 
in intent to the Guidelines, but would remove the restriction about 
deriving the stem cells from embryos. Good ethics hold that a good end 
can never justify evil means to attain it. Certainly killing a growing 
human (no matter what its age) is not an ethical means to obtain even a 
most desirable end such as curing disease. The fact that the embryos 
would die or be destroyed anyway does not justify the act of killing 
those whom we all know are the youngest members of our species. The 
Guidelines and S. 2015 condone the attitude of ``they are going to die 
anyway, so why not use them?'': this attitude applies to the use of 
stem cells derived from fetal tissue as well as from embryos. This 
willingness to destroy the youngest of our species is a logical 
continuation of the ``slippery slope'' this country got on when it 
condoned killing of somewhat older members of our species by allowing 
abortion on demand. It is doubtful that the ``safeguards'' proposed in 
the Guidelines will totally prevent the abuses they are meant to 
prevent. The more ethical thing to do is to prohibit the production of 
embryonic or fetal stem cells.
    In addition to the basic ethical problem with the Guidelines (and 
with S. 2015), there are scientific problems with the use of both 
embryonic and fetal pluripotent stem cells which may make their use 
unnecessary, which the Guidelines do not sufficiently address: the 
first is the use of adult stem cells instead of embryonic or fetal stem 
cells, and the second is the problem of immunological rejection of 
fetal/embryonic stem cell grafts by their recipients.
    The Guidelines, in their responses to public comments that the ban 
on the use of embryonic stem cells remain in force and emphasis be 
placed on research of adult, placental and umbilical cord stem cells 
instead, seem to ignore the mounting evidence in the current scientific 
literature of the versatility of adult stem cells, which may make the 
use of embryonic stem cells unnecessary, and avoid the ethical problems 
of working with embryonic/fetal stem cells. Rather than simultaneously 
pursuing all lines of research, the most ethical thing to do is to 
thoroughly study adult/placental/umbilical cord stem cells to determine 
their full potential, and continue the ban on the study of embryo/fetal 
stem cells until this work is done.
    The Guidelines state four reasons for not banning embryonic stem 
cell research in favor of work on adult stem cells (``Scope of 
Guidelines and General Issues'' section, Paragraph 4; ``Respondents . . 
. adult tissues.'') These reasons are not totally accurate in view of 
what is presently known about adult stem cells. Following are some 
examples from the recent medical literature to illustrate this point.
    (1) The Guidelines state that adult stem cells may have more 
limited potential than embryonic stem cells, and that stem cells for 
all cell and tissue types have not yet been found in the adult human. 
On the contrary, after reviewing some other studies, Clarke et al. 
state that ``. . . these studies suggest that stem cells in different 
adult tissues may be more similar than previously thought and perhaps 
in some cases have a developmental repertoire close to that of ES 
[embryonic stem] cells'' (Science, vol. 288 page 1660, June 2, 2000). 
It is important to remember that studies on adult stem cells have not 
been going on for a particularly long time--much further progress can 
be expected with future studies. The guidelines mentioned that cardiac 
or pancreatic islet stem cells had not been found. It may not be 
necessary to find specific organ stem cells--there is evidence that 
bone marrow stronal cells or cells like them may be present in the 
heart and could be converted into heart muscle-like cells (see page 233 
of Kessler & Byrne, Annual Review of Physiology, vol. 61, page 219, 
1999) and also that ductal structures of the adult pancreas contain 
stem cells that differentiate into insulin-producing cells in an animal 
model (Ramiya et al., Nature Medicine, vol. 6, page 278, 2000).
    (2) The Guidelines claim that stem cells of adults are only present 
in minute quantities, are difficult to purify and may decrease with 
age. Such a generalization should not be made. Bone-marrow stem cells 
such as marrow stromal cells have proved to be extremely versatile: 
recent research has demonstrated their ability to develop into neurons 
as well as other tissue cell types such as muscle, fat, bone, and even 
liver cells: and marrow stromal cells are easily obtainable and can be 
made to multiply in large numbers. As Woodbury et al., in a paper 
describing the transformation of marrow stromal cells into nerve cells, 
point out: ``The marrow cells are readily accessible, overcoming the 
risks of obtaining neural stem cells from the brain, and provide a 
renewable population. Autologous transplantation [i.e. using the 
patient's own cells] overcomes the ethical and immunological concerns 
associated with the use of fetal tissue. Moreover, marrow stromal cells 
grow rapidly in culture, precluding the need for immortalization, and 
differentiate into neurons exclusively with use of a simple protocol'' 
(Journal of Neuroscience Research, vol. 61, page 364, 2000). These 
authors' statement seems to refute the Guidelines' expressed concerns 
about ease of isolation and obtaining sufficient quantities of cells. 
Additionally, Allison et al. report that human hematopoietic stem cells 
can differentiate into hepatocytes (liver cells) in the livers of 
people receiving bone marrow transplants (Nature vol. 406, page 6793, 
2000). The exciting thing about these bone-marrow stem cells is that 
they are able to be transformed into all of the three original 
embryonic layers, ectoderm (represented by nervous tissue), mesoderom 
(muscle, fat and bone cells) and endoderm, (liver cells).
    (3) The Guidelines correctly state that in diseases caused by a 
genetic defect, the defect would also be present in the patient's stem 
cells. This is true, but this problem can be solved by ex vivo [outside 
the body] gene therapy, adding the normal gene to bone marrow stem 
cells, which are then treated to get them to develop into the needed 
cells. Successful gene therapy to marrow cells has already been 
reported by French workers who have cured severe combined 
immunodeficiency disease wirth gene therapy to the bone marrow 
(Cavazzana-Calvo et al., Science, vol. 288, page 669, 2000). My 
colleagues and I are also studying gene therapy using bone marrow stem 
cells for the treatment of a genetic disease: we have cured the mouse 
model of a human disease called erythropoietic protoporphyria with 
``ex-vivo'' gene therapy (Pawliuk et al., Nature Medicine, vol. 5, page 
768, 1999).
    (4) The Guidelines also claim that there is evidence that the stem 
cells from adults may not have the same capacity to multiply as do 
younger cells. This does not seem to be true, since at least in the 
case of bone-marrow stem cells, adults can be successful bone marrow 
donors (Anderlini et al., British Journal of Hematology, vol. 97, 
p.485, 1997).
    The Guidelines do not mention the main problem with the use of 
embryonic and fetal stem cells, that is the rejection of the grafts by 
the recipients. This serious immunological problem is the same as is 
encountered when people receive lung, kidney, heart or liver 
transplants from non-identical twin donors, necessitating the use of 
immunosuppressive drugs for the rest of the recipient's life. This 
immunological problem will not occur if stem cells from the person's 
own body are used--an important value of adult stem cells, derived from 
the recipient, and a strong reason in favor of exploring the full 
potential of adult stem cells.
    In summary, the most ethical and scientifically appropriate steps 
for the Government to take concerning stem cell research are to: (1) 
revoke the ``Guidelines'' and to continue the ban on research on 
embryonic stem cells, and (2) impose a ban on research on stem cells 
obtained from aborted fetuses (in the latter case because of the 
potential of encouraging abortion to obtain these cells). Having done 
this, the Government must strongly encourage and fund research on adult 
stem cells, as well as research on umbilical cord blood and placental 
stem cells (although these latter two groups may cause immunological 
problems), so that the full potential of all of these other kinds of 
stem cells for generating tissue cells of various kinds can be fully 
detennined.

    Senator Specter. Thank you very much, Dr. Mathews-Roth.
    Dr. Prentice, Dr. Mathews-Roth, supported the use of stem 
cells from fetal tissue except where there were induced aborted 
fetuses. Am I citing your testimony correctly, Dr. Roth?
    Dr. Mathews-Roth. Yes, and emphasize no induced abortions, 
just spontaneous.
    Senator Specter. I said that, you excluded that, but 
otherwise fetal tissue. Dr. Roth has confirmed my statement 
that it is appropriate to use those stem cells. Do you agree 
with that, Dr. Prentice?
    Dr. Mathews-Roth. Excuse me, could I say one thing? I think 
that that research has been restricted to marrow stromal cells. 
I would not be in favor of using the germ line cells from the 
germ line fetal tissue.
    Senator Specter. But there is fetal tissue you would 
approve?
    Dr. Mathews-Roth. I would say bone marrow stromal cells or 
other organ cells from the spontaneously aborted fetuses.
    Senator Specter. But there is a form of stem cell from 
fetal tissue that you would approve of?
    Dr. Mathews-Roth. Yes.
    Senator Specter. Dr. Prentice, do you agree with that?
    Dr. Prentice. As far as I understand it, Senator, that 
would be an ethically as well as already legally acceptable 
choice, to use these spontaneously aborted fetuses.
    Senator Specter. Well, I raise the issue because for a long 
time there had been a total objection to the use of fetal 
tissue at all, and that was on the basis that once you open the 
door, a slippery slope, you start to use fetal tissue even if 
they are from circumstances where the abortion was not made, it 
was going to happen anyway, so that at least so many people who 
had objected to the use of fetal tissue for some time have 
really reversed their position.
    Senator Thurmond, interestingly, a very strong pro-life 
Senator, a model of conservatism, was against the use of fetal 
tissue, and then in his own family his daughter had juvenile 
diabetes, and Senator Thurmond testified many years ago before 
stem cells came up on the juvenile diabetes issue, and then he 
has testified now in favor of stem cells, but on the use of 
fetal tissue, when Senator Thurmond changed his vote it went 
from about 40 Senators in favor of using fetal tissue to about 
80, and I raise that issue because of the potential 
similarities, where there had been an objection raised to fetal 
tissue, which has abated at least in some circumstances, as Dr. 
Roth would approve, and you concur, Dr. Prentice.
    Dr. Prentice. Senator, if I might comment, that source, 
even though it might be acceptable, might still face some of 
the problems of the embryonic stem cell sources that we have 
mentioned, because unless we are using cells from the patient 
themselves, their own adult stem cells, we are still going to 
run this risk of immunological rejection.
    The other difference between those arguments is, if we are 
using fetal tissue at that point, by current Federal 
regulations the embryo is already dead, to derive embryonic 
stem cells, we must actually wilfully destroy the embryo, so 
there is a difference.
    Senator Specter. Well, let us take that point up, because 
that is really a core argument which has been made here, and 
Dr. Roth is very emphatic about destroying a human life, but 
where you have these embryos which have been created for in 
vitro fertilization and they are to be discarded, and the 
choice is just having them discarded, or using stem cells which 
may be derived from them to save lives, what is the balance of 
argument on destroying a human life when the issue arises as to 
what is the quality of a human life in the embryo, if any.
    I understand your point about aging people. Are you going 
to kill them to help others? I do not think that follows, and 
the testimony that Senator Brownback advanced about the analogy 
to the Holocaust, aside from the issues of sensitivity, is just 
not, at least in my judgment, very sound, but come to grips 
with the destruction of a human life. When these embryos--how 
do you categorize them as a human life, and how do you justify 
letting them be destroyed collaterally by being discarded when 
there could be a use made?
    I notice you nodding, Dr. Prentice, and we will turn to you 
first. Well, we can go to the ladies first. Dr. Roth, you go 
first.
    Dr. Mathews-Roth. Again, it was what I was trying to say 
before, between the difference of organ donation and taking a 
life of an embryo. I think there are some things that we just 
ethically should not do, and I think that these extra embryos, 
if you cannot find adoptive parents for them that are willing 
to use them because they cannot have children--and there have 
been some reports of women applying to IVF clinics and saying, 
hey, have you got any spare embryos, if you can get permission 
from the original parents I would like them because my husband 
and I cannot do it, or whatever--I thing ethically the best 
thing to do is let them die a natural death if you want to put 
it that way.
    In spite of the fact that a good end would occur, I still 
think the means of destroying these embryos, of letting them 
grow for a few days and ripping them apart to take these stem 
cells, that is a bad means. I do not think ethically you can 
justify it.
    Senator Specter. Dr. Roth, you referred to consent from the 
parents. Are you suggesting that if you had consent from the 
parents it would be appropriate to use the embryos?
    Dr. Mathews-Roth. I still would have a big problem with 
that, because it is direct killing. There is a difference when 
a child dies in an accident or of diabetes, or of overwhelming 
infection that we cannot cure, and this child dies a natural 
death, then I do not think there is a problem, if the organs 
obviously are suitable, for a parent to say, sure, take my 
child as an organ donor, or if there is a miscarriage, take my 
child as an organ donor, but with these embryos you have got to 
kill them, literally destroy them.
    Senator Specter. Dr. Prentice, let us turn to you on the 
issue of human life, quality of human life contrasted with 
saving so many other lives.
    Dr. Prentice. Actually, Senator, I am not sure that, from 
my own perspective, I could justify even discarding those 
embryos. I know that is a very--it is a sticky issue.
    Senator Specter. You cannot justify what, sir?
    Dr. Prentice. That I could justify discarding the embryos, 
either, and I realize, so what do we do with all of those 
embryos, and perhaps that is something that Congress itself 
should be looking at in terms of the IVF industry.
    Senator Specter. Well, what would the alternative be?
    Dr. Prentice. I am not certain, sir, until we can get them 
adopted, or what the answer might be.
    Senator Specter. Well, they have to be carried to term. Are 
you suggesting that the Government would compel someone, a 
woman to carry them to term?
    Dr. Prentice. I do not think we should be in the business 
of compelling anyone to carry these embryos to term or destroy 
them, either way.
    Senator Specter. Well then, what would you do?
    Dr. Prentice. I think it would have to be perhaps some sort 
of an adoption program, but that is not really the focus of the 
issue. The focus----
    Senator Specter. I agree with you, but you brought it up, 
so I wanted to explore it if you care to. What would you do 
with them, if not discard them? If there were another 
alternative to discarding them, I think you have a very valid 
point.
    This is a very sensitive and very difficult issue, when 
human life begins, and I understand your sincerity in saying 
that human life begins when you have a human embryo, and the 
next step up is if you have human life, what is the quality of 
life, but if you can structure having them carried to term so 
that you have a baby after birth, then I think we would be in a 
very different realm. I would have a very different view.
    I would not in any way suggest sacrificing a child who has 
been born for what benefit there may be to others from the 
child's body, but where you have an embryo which is going to be 
discarded, then it is a different matter. If you would care to 
pursue an alternative to what you do as opposed to discarding, 
I would be interested in hearing you.
    Dr. Prentice. I have not really thought through the issue, 
Senator, but as we mentioned, one possibility might be some 
sort of adoption program.
    Senator Specter. How would you structure an adoption 
program?
    Dr. Prentice. I am not really certain. I have not really 
had time to think through the issue. It has really just come 
up.
    Senator Specter. Well, in order to get to adoption, you 
have to have a child born.
    Dr. Prentice. Well, no, sir.
    Senator Specter. Excuse me, Dr. Roth.
    Dr. Mathews-Roth. You have to find a mother to carry it.
    Senator Specter. That is what I was saying. You and I agree 
on this now, I want it noted.
    Dr. Mathews-Roth. One way you could avoid--through the 
adoption business is for IVF clinics to publicize the fact that 
they have got extra embryos up, quotes, for adoption, and if 
infertile couples, neither of whom are able to--the eggs of the 
women, she does not have enough eggs, or the man has no sperm, 
if they want to adopt one of these embryos fully made, 
implanted into a uterus, then this is possible. This is a way 
to go, and it is a matter of getting the news out there that 
these are available, but I personally find no way of justifying 
destroying an embryo even to help another person, I think 
ethically.
    Senator Specter. I am advised by staff there are 100,000 
frozen embryos in excess of what people propose to use by 
carrying to term.
    Dr. Mathews-Roth. I have also heard from IVF people that 
the tendency has been in the past, up until now, to make many 
more than actually needed. They seem now to want not to do this 
so much, so in the future there may not be that many extra 
embryos. I do not know what the figure is of infertile people 
who would like to adopt embryos.
    Senator Specter. Dr. Roth, I wrote down one of your 
statements, that if there is a moral or ethical alternative 
then we should not use embryos. Do I quote you accurately?
    Dr. Mathews-Roth. I guess that was my paraphrase of what 
the NBAC was trying to say, that if there was a morally 
acceptable alternative to using fetal embryo, or fetal stem 
cells, then we should use it.
    What we are both proposing, of course, is adult stem cells, 
and I am going one step further and saying hemopoietic stem 
cells, or possibly solid organ stem cells from spontaneously 
miscarried fetuses.
    Senator Specter. The contention has been raised about adult 
stem cells being adequate to handle these issues, and if adult 
stem cells are not adequate, would you then be willing to use 
embryonic stem cells?
    Dr. Mathews-Roth. I do not think so, not if it requires 
killing. That is not good. I want to get away from killing 
people, even little, eensy-weensy tiny people. That is one of 
the reasons I do not like abortion, either. Abortion is direct 
killing.
    Senator Specter. I want to be sure we have an adequate 
exploration of your views, and these green, yellow, and red 
lights are only guideposts, and the red light for Dr. Prentice 
was on, he noted, and I said go ahead, and yours was on a 
while, and I have questioned you fairly extensively, far beyond 
my customary time.
    I would like now to have Dr. Fischbach and Dr. Spiegel come 
back with you, and I would like to have a discussion among the 
four of you scientists, and keep your seat, Dr. Prentice and 
Dr. Roth. They will surround you.
    Dr. Fischbach, you have heard the testimony of Dr. 
Prentice, then Dr. Roth, about the potential applications for 
adult stem cells, and Dr. Prentice has been considerate enough 
to bring a chart along to illustrate his point. What is your 
view of what Dr. Prentice has said about the use of adult stem 
cells?
    Dr. Fischbach. There were several things mentioned, and I 
think Dr. Spiegel and I both might comment. I think the notion 
that stem cells in bone marrow might be useful for replacing 
nerve cells is a hope we all share, and I think it is in the 
future.
    The publication referred to demonstrated a very first step, 
a small first step, and that for a few hours in tissue culture 
cells from bone marrow appear to have some of the properties of 
nerve cells. It will take years to translate that into a useful 
therapy. I believe that studies of human, of embryonic stem 
cells are much closer to that goal now, but I think both 
efforts should be pursued vigorously.
    Senator Specter. Are there things embryonic stem cells can 
do that adult stem cells cannot do?
    Dr. Fischbach. I do not know any case where an adult stem 
cell derived from an adult animal has formed a nerve cell that 
can make and release dopamine, and can reverse the behavioral 
defect of Parkinson's disease. That may change with time, with 
years, but I feel differently about patients dying with 
devastating neurological disorders. I do not think we have that 
time to wait.
    Senator Specter. Dr. Prentice, would you care to comment on 
Dr. Fischbach's statement?
    Dr. Prentice. Mr. Chairman, I am not aware of currently a 
dopaminergic adult neural stem line, but there was a report in 
this month's Experimental Neurology where neurons were 
generated from adult stem cells, neural stem cells which formed 
functional glutamenergic and gatamenergic synapses in culture, 
so there is evidence the adult stem cells can form functional 
nerve connections and functional types of synapses in culture.
    There have also been examples where in rat and mouse models 
the adult stem cells have been transplanted back into the 
animal and actually formed normal synapses in the animal.
    Senator Specter. Dr. Spiegel, would you care to comment on 
this issue, as to overall, but in part responding to what Dr. 
Prentice has said?
    Dr. Spiegel. I will not comment on the aspect of these 
cells because I am not an expert in that area.
    Senator Specter. Well, before you leave it, would you care 
to comment on the issue of neuronal cells, Dr. Fischbach?
    Dr. Fischbach. I think we shifted grounds in midstream from 
bone marrow to neural stem cells. I think the adult neural stem 
cells have promise, but I do not think they have yet reformed 
function to the extent of correcting a neurological defect.
    Senator Specter. Dr. Spiegel, and Dr. Mathews-Roth, if you 
want to make a comment, go ahead.
    Dr. Mathews-Roth. I just want to say I think the bone 
marrow stromal cells are much more plastic than the bone marrow 
hemopoietic regenerative stem cells.
    Senator Specter. Dr. Fischbach, for the record, would you 
respond or care to respond? I will study this later and try to 
figure it out.
    Dr. Fischbach. I think one way to approach this is, there 
are many types of cells in bone marrow. Some cells are more 
germinal in the sense that they can give rise to many other 
types of cells. I agree that there are different types of cells 
in the bone marrow, and some have potential as stem cells for 
therapy.
    Senator Specter. Do you think we should impose a 
requirement on Senators before being permitted to vote on this 
issue, that they know something about it, like these complex 
matters you are advancing here today, and you do not have to 
answer that, doctor. Dr. Spiegel, you were right in the middle 
of a response when we had a couple of interruptions. You have 
the floor.
    Dr. Spiegel. Thank you, Mr. Chairman. I am not aware of a 
single report in which either bone marrow stromal cells or 
hematopoietic cells formed pancreatic islet cells. I am clearly 
aware of a report, for example, in ``Nature Medicine'' of mouse 
embryonic stem cells forming pancreatic beta cells and curing 
diabetes.
    There are also reports of a diabetes breakthrough using 
adult stem cells from the pancreas, and this is what Dr. 
Mathews-Roth was showing as a clip. This is work we vigorously 
support, from the University of Florida, in which adult 
pancreatic stem cells were transplanted into mice with 
diabetes, with some positive effect.
    There are also efforts to isolate human pancreatic ductile 
cells at the Joslin Diabetes Clinic. However, the numbers 
generated were clinically insufficient by two orders of 
magnitude, that is, they were only one-hundredth of the amount 
necessary for any kind of clinical utility. In the final 
analysis, as Senator Harkin said, we really need to pursue each 
of these avenues of embryonic and adult stem cell research to 
have the greatest possibility of success.
    Dr. Fischbach. I want to underline that, if I can, that 
these involve quantitative statements. The question is not, can 
this happen, but the real issue is, can this happen in ways 
that we can control and make clinical use of, and I think that 
sets embryonic stem cells apart at the current time.
    Senator Specter. Dr. Mathews-Roth, would you like to make a 
concluding statement?
    Dr. Mathews-Roth. I would just say----
    Senator Specter. I am going to give each of you an 
opportunity to conclude here. Your turn comes first, Dr. 
Mathews-Roth.
    Dr. Mathews-Roth. I guess my concern is that there are some 
things that ethically we should not do. I am not convinced that 
it is going to take a terribly long time to get adult stem 
cells up to speed, because they have not been worked on as long 
as fetal stem cells, and I really think for the ethics 
involved, for the idea of yeah, it is okay to kill these little 
guys, even if they are very teeny, tiny humans, I think that is 
wrong, and that is why I think that ban should continue.
    Look at adult stem cells. Look at stem cells, organ stem 
cells, maybe pancreatic ductile stem cells from spontaneously--
spontaneously aborted, that is the medical terminology for 
miscarriages, miscarried babies may be a way to go, from 
stillborn children may be a way to go, but do not kill to do 
this.
    Senator Specter. Dr. Spiegel, would you care to make a 
concluding statement?
    Dr. Spiegel. The issue of transplant rejection and immune 
suppression has been raised, and this is a vigorous area of 
research that we are pursuing. This research is essential 
because of the many organ transplants we are already doing, and 
I am happy to say that there is really significant progress and 
potential success, which could address this issue.
    As a physician, scientist, and public servant, I have tried 
to illuminate the terms of the debate, and to offer an 
objective view about the science. I hope I have contributed to 
that.
    Senator Specter. Thank you, Dr. Spiegel.
    Dr. Prentice, would you care to make a concluding 
statement?
    Dr. Prentice. Thank you, Mr. Chairman. In the end, in a 
real sense, the question we are grappling with here is not a 
scientific question, whether adult or whether embryonic is the 
correct way to go, the only way to go and so on. I think you 
have heard evidence on both sides of the promise of both 
particular types of stem cells, but in the end this is really a 
moral and ethical question which you and the other Senators are 
going to have to grapple with, and it is my opinion that it has 
never been acceptable to sacrifice one set of human lives for 
the benefit of others.
    Thank you, sir.
    Senator Specter. Thank you, Dr. Prentice. Dr. Fischbach, 
would you like to sum up?
    Dr. Fischbach. Senator, I think these are exciting times. 
Everything we have talked about today has been published or 
discussed for the first time in the last 2 years, most of it in 
the last year, and we are faced with issues of extraordinary 
promise, and revolutionary times.
    I think these ethical issues are profound, and I think that 
is the very purpose of the NIH guidelines is to allow work to 
go forward under strict regulation and debate, and to allow it 
to proceed.
    I would say that every time society has closed down avenues 
of investigation people have suffered, and I think the 
guidelines try to address that issue.
    Senator Specter. Thank you very much, Dr. Fischbach, Dr.
Mathews-Roth, Dr. Prentice, Dr. Spiegel. I believe this has 
been a very productive hearing, unusually so. I think it is 
always useful when we have people who are knowledgeable 
advocate their positions, and especially when there is the kind 
of friendly but adversarial opposition that we have had here 
today.
    I think it has been very constructive, and I know my 
colleagues in the Senate are very much concerned about this, 
and we will be studying the record so we can understand it, 
because a good bit of what has been testified to here today 
requires some definitions to work it through, but the exchanges 
I think have been very helpful in that regard.

                         CONCLUSION OF HEARING

    Thank you all very much for being here, that concludes our 
hearing. The subcommittee will stand in recess subject to the 
call of the Chair.
    [Whereupon, at 11:20 a.m., Thursday, September 7, the 
hearing was concluded, and the subcommittee was recessed, to 
reconvene subject to the call of the Chair.]


                           STEM CELL RESEARCH

                              ----------                              


                      THURSDAY, SEPTEMBER 14, 2000

                           U.S. Senate,    
    Subcommittee on Labor, Health and Human
     Services, and Education, and Related Agencies,
                               Committee on Appropriations,
                                                    Washington, DC.
    The subcommittee met at 9:33 a.m., in room SH-216, Hart 
Senate Office Building, Hon. Arlen Specter (chairman) 
presiding.
    Present: Senators Specter, Harkin, and Reid.
    Also present: Senator Wellstone.

               Opening statement of Senator Arlen Specter

    Senator Specter. Good morning, ladies and gentlemen. The 
hour of 9:30 having arrived, the Subcommittee on Labor, Health 
and Human Services, and Education for the Appropriations 
Committee will now proceed with this hearing, which is the 
seventh in a series of hearings on stem cells.
    When the dramatic breakthrough occurred on stem cell 
research in November of 1998, this subcommittee undertook these 
hearings to focus on the prohibition which exists in the law 
saying that the Federal Government, the National Institutes of 
Health, may not engage in research to derive stem cells from 
embryos.
    The potential for stem cells is virtually limitless, so we 
are told by the research community and by the people who are 
studying this subject. The stem cells pose a veritable fountain 
of youth, with already some tremendous inroads on Parkinson's 
Disease, prospects on Alzheimer's and stroke, potential for 
heart ailments and very vast potential in what we have found in 
a series of hearings which we have already conducted.
    There are those who object to stem cell research on 
ethical, moral, perhaps religious grounds, with the contention 
that the embryo is a living being. We have invited people on 
both sides of the issue to testify so that we can have all of 
the positions presented, and that the Senate can be in the best 
place to make an informed judgment.
    We have had leading opponents from the Senate and the House 
of Representatives testify before this subcommittee, and today 
we have a very distinguished panel of witnesses. Dr. Richard 
Hynes, director of the Center for Cancer Research from 
Massachusetts Institute of Technology, Dr. Darwin Prockop, 
director of the Center for Gene Therapy from Tulane University; 
in opposition we have Mr. Ron Heagy, president and founder of, 
quote, Life is an Attitude Foundation, Mr. Ron Saltzman, pastor 
of the Ruskin Heights Lutheran Church in Kansas City, Missouri, 
and Dr. Anton-Lewis Usala, chairman and chief technical officer 
for Encelle.
    We have had assurances from the Majority Leader that the 
legislation which Senator Harkin and I have introduced will be 
called for a vote this month, so we are accelerating our 
efforts to present all facets of the issue.
    We also have for our third panel Mr. Michael J. Fox, Ms. 
Mary Tyler Moore, Ms. Jennifer Estess, and Ms. Gina Gershon, 
and as frequently is the case, we have called on people who are 
well-known to the public, because when, candidly Michael J. Fox 
and Mary Tyler Moore testify about their ailments and the 
opportunity for stem cell research illustratively to cure 
Michael J. Fox from Parkinson's, that is a point of really 
great emphasis.
    That is a brief summary of my formal opening statement 
which will be put in the record without objection, and now I 
yield to my distinguished colleague, Senator Harkin, Ranking 
Democrat for the subcommittee.

                    Statement of Senator Tom Harkin

    Senator Harkin. Thank you very much, Mr. Chairman, and my 
thanks to our distinguished panelists who are here today. As 
you said, Mr. Chairman, this is our seventh hearing on the 
issue of stem cell research. I want to commend you, Mr. 
Chairman, for the seriousness with which you take our 
responsibility over this important controversial issue. These 
hearings are part of a necessary dialogue on both the promise 
and the ethical implications of stem cell research.
    Two years ago, researchers discovered and isolated the 
human pluripotent stem cells, the precursor of almost all of 
the cells in the human body, and demonstrated the ability to 
self-renew and differentiate into many different cell types.
    As we have learned, they hold unprecedented potential for 
the treatment and cure of the terrible diseases and conditions 
that strike too many of our family members and friends, and so 
we are on, I think, the cusp of some really exciting research 
and discovery. No one knows what the potential for this is, but 
almost all of the scientists I have spoken to indicate that 
there could be tremendous, tremendous potential for the 
interventions and cures of some of our most disabling diseases.
    There have been, obviously, ethical and moral 
considerations involving the use of these cells. For a couple 
of years the NIH, along with the commission, the President's 
Commission on Ethical Standards have been working to set up 
guidelines.
    On August 23, NIH issued the final guidelines, and I 
believe these guidelines will permit the research to advance 
and move ahead within, I believe, fairly stringent and 
comprehensive ethical guidelines, and for the record I want to 
once again say what those are, because I keep hearing about how 
people are going to sell these cells, and they are going to 
patent, they are going to make money, and all that kind of 
stuff. Let me just read again what the guidelines are.
    NIH will not fund the actual derivation of pluripotent stem 
cells.
    NIH funds can be used only if the stem cells were derived 
from embryos in excess of need that were created as part of in 
vitro fertilization procedures.
    Providers of stem cells cannot make a profit from supplying 
them to federally funded researchers.
    And last, providers of stem cells must provide 
documentation of informed consent by the donors.
    Those are the ethical guidelines. NIH will begin accepting 
grant applications for projects sometime early next year. Our 
legislation is pending and, as the chairman said, we hope that 
we can get it up before we leave here this year.
    So that is just again the shortened part of my statement, 
Mr. Chairman, and again I want to thank you for your tremendous 
leadership on this very, very important issue.
    Senator Specter. Senator Wellstone has joined us this 
morning. We welcome him here and would invite him to make 
whatever opening comments he makes here today.

                 Statement of Senator Paul D. Wellstone

    Senator Wellstone. Mr. Chairman, I appreciate your 
courtesy, and I know we need to get going with this hearing, a 
very important hearing. Let me just say to both Senator Specter 
and Senator Harkin that I come here to just show my strong 
interest and support for your work. I have done a lot of work 
with the Parkinson's community, but this legislation and your 
effort extends way beyond just one community. It is terribly 
important to many, many people, many, many families, many 
Americans, and I am here just as an indication of the strong 
support for your work.
    I thank you.
    Senator Specter. Senator Wellstone, at a previous hearing 
we made a tabulation, and stem cell research could affect 125 
million Americans, just about half of the people in this 
country with a variety of ailments, which it may be able to 
deal with.

STATEMENT OF RICHARD O. HYNES, Ph.D, DIRECTOR, CENTER 
            FOR CANCER RESEARCH, MASSACHUSETTS 
            INSTITUTE OF TECHNOLOGY
    Senator Specter. We turn now to Dr. Richard Hynes, director 
of the Center for Cancer Research at Massachusetts Institute of 
Technology since 1991. He serves as president of the American 
Society for Cell Biology, and is a member of the National 
Academy of Sciences. Dr. Hynes received his M.A. in 
biochemistry from Cambridge University in England, and his Ph.D 
in biology from MIT. Welcome, Dr. Hynes, and in accordance with 
our standard practice the green light starts when you do, and 
it signifies 5 minutes, the yellow light gives you a minute, 
and the red light says stop.
    The floor is yours.
    Dr. Hynes. Thank you, Senator. I appreciate greatly the 
invitation to appear before you today, and I am here 
representing the American Society of Cell Biologists, 10,000 
biomedical researchers, and many of our other colleagues in the 
biomedical research community, and I am here to explain to you 
and to your colleagues why our organization feels strongly that 
the full potential of human embryonic stem cell research must 
be realized.
    On behalf of the society I want first to extend my deepest 
appreciation to you, Chairman Specter, to Ranking Member 
Harkin, and to members of the subcommittee for your visionary 
and courageous commitment to investment in biomedical research 
at the NIH. This funding is vitally important to allow our 
Nation's scientists and clinicians to exploit the tremendous 
opportunities offered by the current revolution in biomedical 
science in order to enhance public health.
    I want to stress today that embryonic stem cell research is 
among the most promising of these new opportunities. We 
understand the ethical concerns that some have raised about 
this research, but we respectfully submit that appropriately 
regulated research on human embryonic stem cells can be 
conducted while taking these concerns into account.
    We believe that this research will undoubtedly lead to new 
ways to treat disease and disability. Embryonic stem cells will 
allow creation of new healthy tissue to replace damaged or dead 
tissue. Examples include bone marrow for the treatment of 
cancer and sickle cell anemia, pancreatic cells for the 
treatment of diabetes, neuronal cells for the treatment of 
Parkinson's, Alzheimer's, and various brain and spinal 
disorders, and I could make a much longer list, but we do not 
have the time.
    We are not alone in our conviction that this invaluable 
research must go forward. Attached to my testimony is a 
statement signed by 17 organizations around the country, 
including the American Medical Association, the Michael J. Fox 
Foundation for Parkinson's Research, the Juvenile Diabetes 
Foundation, the Christopher Reeve Paralysis Foundation, and the 
American Association for Cancer Research, among many others.
    I also want to reiterate the support of the American 
Society for Cell Biology for the Stem cell Research Act of 2000 
which you have introduced, that would allow not only the user 
but also the derivation of embryonic stem cell lines.
    Some have argued that human embryonic stem cell research is 
illegal, unnecessary, and immoral. We respectfully disagree on 
all counts. On the contrary, we believe that it would be 
immoral not to pursue this great opportunity to improve the 
quality of life.
    The charge of illegality is unfounded. As you know, various 
appropriations bills have restricted embryo research for 
several years, but these bills are silent on the use of 
embryonic stem cells. Such cells are not embryos, and they 
cannot independently develop into embryos.
    The NIH guidelines set specific criteria governing the 
derivation of the stem cells, as Senator Harkin just pointed 
out, requiring informed consent, precluding payment, and 
prohibiting the creation of embryos for research purposes. 
Cells used for research must be derived solely from embryos 
generated for fertility treatments and in excess of clinical 
need. Such embryos would otherwise be discarded.
    Previously, the use of embryonic stem cells was restricted 
to private and commercial entities which were not accountable 
to the public. NIH funding of stem cell research will allow 
Federal Government to exercise control over the standards for 
the use of stem cells and will encourage public input.
    Second, critics argue that embryonic stem cell research is 
unnecessary because stem cells from adult tissues may be 
equally effective. I regret that this claim is ill-informed and 
misleading. Recent reports on adult stem cells are indeed 
encouraging, but this line of research is in its very early 
stages, and far from definitive at this point. We know little 
about the availability of adult stem cells, their 
differentiation, or their potential for prolonged maintenance 
outside the body.
    While we strongly support continued research on adult stem 
cells, it is far too early to conclude that they will be as 
effective in treating and preventing disease as embryonic stem 
cells seem certain to be. If we hold up the progress on 
embryonic stem cells we may be very much at a loss in a few 
years time.

                           PREPARED STATEMENT

    Finally, we believe it would be immoral not to pursue this 
embryonic stem cell research within the appropriate regulatory 
oversight, because it has such enormous potential to save human 
lives and to mitigate human suffering. I submit that if the 
issue is morality, using embryonic stem cells for life-saving 
research is greatly preferable to discarding them. I, along 
with many others, believe we should take advantage of the 
enormous life-saving potential of the thousands of embryos 
currently frozen and destined for destruction.
    So I thank you for the opportunity to testify before you, 
Mr. Chairman, and I would be pleased to answer any questions.
    [The statement follows:]
                 Prepared Statement of Richard O. Hynes
    Mr. Chairman and members of the Subcommittee: I am Richard Hynes, 
Professor of Biology at the Massachusetts Institute of Technology, 
where I am also an investigator of the Howard Hughes Medical Institute 
and Director of the Center for Cancer Research. I am a member of the US 
National Academy of Sciences and of the Institute of Medicine. I am 
here today as President of the American Society for Cell Biology. The 
Society represents 10,000 basic biomedical researchers, most of whom 
work in our Nation's leading research universities and institutes. It 
is my great pleasure to appear before you to explain why our 
organization feels that it is so important that the full potential of 
human embryonic stem cell research be realized.
    On behalf of the American Society for Cell Biology, I wish first to 
extend my deepest appreciation to you, Chairman Specter, to Ranking 
Member Harkin and to the members of the Subcommittee for the visionary 
and courageous commitment you have made through your investment in 
biomedical research at the NIH. We are extremely grateful that you have 
embraced the goal of doubling the NIH budget over five years and that 
we are halfway towards reaching that goal. This funding is vitally 
important to allow our nation's scientists and clinicians to exploit 
the tremendous opportunities offered by the current revolution in 
biomedical research to enhance the health of the American public.
    We believe deeply that this investment in biomedical research will 
be most effectively used if embryonic stem cell research is included 
among the innovative methods used to develop treatments and preventions 
for disease. We understand the ethical concerns that some have raised 
about this research but we respectfully submit that appropriately 
regulated research on human embryonic stem cells can be conducted while 
taking into account those concerns. The Guidelines recently released by 
the National Institutes of Health enable federally funded scientists to 
conduct research using pluripotent human embryonic stem cell lines. We 
believe that this research will undoubtedly lead to new ways to treat 
disease and disability. Embryonic stem cells will allow the creation of 
new, healthy tissue to replace damaged or dead tissue, such as bone 
marrow for the treatment of cancers, sickle cell anemia and 
thalassemis; pancreatic cells for the treatment of diabetes, and 
neuronal cells for the treatment of Parkinson's disease, Alzheimer's 
and various brain and spinal cord injuries and disorders. The prospects 
offered by this research are analogous to, but will likely far surpass, 
the benefits realized by organ transplantation over recent decades.
    We do not stand alone in our determination that this invaluable 
research must go forward. Attached to my testimony is a statement 
signed by 70 American organizations, including the American Medical 
Association, the Michael J. Fox Foundation for Parkinson's Research, 
the Juvenile Diabetes Foundation International, the Christopher Reeve 
Paralysis Foundation, the American Association for Cancer Research, and 
the Federation of American Societies of Experimental Biology which I 
respectfully request be submitted for the record.
    I also want to reiterate the support of the American Society for 
Cell Biology for S. 2015, ``The Stem Cell Research Act of 2000'' which 
you have introduced, that would allow federally-funded scientists not 
only to use, but also to derive embryonic stem cell lines for research 
purposes.
    Some have argued that human embryonic stem cell research is 
``illegal, unnecessary and immoral.'' We respectfully disagree on all 
counts. On the contrary, we believe that it would be immoral not to 
pursue this great opportunity to improve the quality of human life.
    First, the charge that the NIH has acted illegally is unfounded. As 
you well know, the Labor, Health & Human Services and Education 
Appropriations bills have restricted embryo research for the last 
several years, but these bills are silent on the use of embryonic stem 
cells. These cells are not embryos and they cannot independently 
develop into embryos. The NIH Guidelines prohibit the use of NIH funds 
to create embryos for experimental purposes and they set specific 
criteria governing the sources from which embryonic stem cells can be 
obtained. These guidelines require the informed consent of the donors, 
preclude any possible direct benefit to such donors, and prohibit the 
creation of embryos for research purposes. Cells used for research must 
be derived solely from embryos generated for fertility treatments and 
in excess of clinical need. Such embryos would otherwise be discarded. 
A critical element of the NIH Guidelines is that the federal government 
will oversee the use of embryonic stem cells. Heretofore, this valuable 
resource was available exclusively to private and commercial entities, 
which were not accountable to the public. By funding human embryonic 
stem cell research, the federal government may exercise control over 
standards for use of stem cells. This provision will facilitate open 
debate and encourage public input into the appropriate uses of this 
important scientific opportunity.
    Second, critics argue that embryonic stem cell research is 
unnecessary because stem cells derived from adult tissues may be used 
with equal effectiveness. I regret that this claim is ill-informed and 
misleading. Scientists are indeed guardedly encouraged by recent 
reports of plasticity of some adult stem cells, but this line of 
research is in its very early stages and far from definitive. We know 
little about the availability of adult stem cells, their 
differentiation, or their potential for prolonged maintenance outside 
the body. While we strongly support continued research on adult stem 
cells, it is far too early to conclude that they will be as effective 
in treating and preventing disease as embryonic stem cells seem certain 
to be. If embryonic stem cell research were to be halted based on that 
hope, it is entirely possible that years would pass before scientists 
determine whether or not adult stem cells are of equivalent value. 
During those years embryonic stem cell research can an should be 
pursued in parallel, to the great benefit of many of our fellow 
citizens. This possibility was emphasized in a letter to Chairman 
Specter in May from some of this Nation's leading researchers 
investigating adult stem cells who stated: ``We are . . . dismayed that 
our research . . . is being used as a justification to hinder or 
prohibit research with embryonic stem cells. It is simply incorrect to 
use the future promise of adult stem cell research as an argument that 
embryonic stem cell research is not critical and essential.'' Again, I 
respectfully request that you submit their letters for the record.
    Finally, given these facts, we believe it would be immoral not to 
pursue embryonic stem cell research, within the appropriate regulatory 
oversight mandated by the NIH Guidelines, because this research has 
enormous potential to save human lives and to mitigate human suffering. 
The embryos in question would be obtained from in vitro fertilization 
clinics only from those in excess of clinical need. I submit that, if 
the issue is morality, using embryonic cells for potentially life-
saving research is greatly preferable to discarding them. Surely we 
should take advantage of the enormous life-saving potential of the 
thousands of embryos that are currently frozen and destined for 
destruction?
    Great Britain has recognized the value of stem cell research, 
having strongly recommend the use of embryonic stem cells, and is now 
considering enabling publicly funded researchers to establish new 
embryonic stem cell lines. Other European countries are moving in the 
same direction. I do not believe that the Europeans are less moral or 
ethical than we. I also do not believe that they are less sensitive to 
the sanctity of life. I do believe that they have acted appropriately 
to enact by law the generation of new sources for stem cells in order 
to save lives and reduce suffering of the living and I believe we 
should do the same in this country.
    In conclusion, The American Society for Cell Biology strongly 
endorses the NIH Guidelines which will enable federally-funded 
scientists to pursue embryonic stem cell research. We also endorse S. 
2015, ``The Stem Cell Research Act of 2000''. We feel that, for the 
sake of humanity, studies using all forms of stem cells--embryonic, 
fetal and adult--should be pursued vigorously. We owe it to all those 
who are suffering to explore all possible avenues that could lead to 
the prevention of, and remedies for, disease.
    I thank you for the opportunity to testify before you, Mr. 
Chairman. I would be pleased to answer any questions.

    Senator Specter. Well, thank you very much, Dr. Hynes. You 
point out that there is no law prohibiting Federal funding on 
the stem cells once they have been extracted from the embryos, 
which I think is an important distinction to make clear, as you 
have, but the existing prohibition is on the use of Federal 
funding to take the embryos themselves and to extract the stem 
cells.
    Once they have been extracted by somebody else, then 
Federal funding may be used, and I think the point you make on 
the discarding of the embryos is very important. The embryos 
were created for in vitro fertilization, and those which are 
not used will be discarded.
    When we dealt with the issue of fetal tissue many years 
ago, objections were raised that fetal tissue would promote 
abortions, and finally that argument was discarded, but I think 
it is important to focus on those couple of additional factors 
at this juncture.

STATEMENT OF DARWIN J. PROCKOP, M.D., Ph.D, DIRECTOR, 
            CENTER FOR GENE THERAPY, TULANE UNIVERSITY 
            MEDICAL CENTER
    Senator Specter. Now we would like to turn to the testimony 
of Dr. Darwin Prockop, who as of March of this year was 
appointed director of the Center for Gene Therapy at Tulane 
University Medical Center. Prior to that time, he was director 
of gene therapy at Hanneman University School of Medicine.
    Dr. Prockop discovered the gene defect that causes brittle 
bone disease, and his most recent work involves successfully 
converting stem cells taken from adult bone marrow into nerve 
cells. He received his M.D. from the University of Pennsylvania 
and his Ph.D in biochemistry from George Washington University, 
so I guess we have to call you doctor, Dr. Prockop.
    The floor is yours, sir.
    Dr. Prockop. Thank you very much, Senator Specter. It is a 
real privilege to appear before this subcommittee.
    I think I have been asked to speak to you today because 
Tulane Center for Gene Therapy is focusing on using adult stem 
cells, and developing these cells as a way of treating a number 
of diseases.
    These adult stem cells were discovered over 20 years ago, 
the ones we are dealing with, and have been shown to have a 
number of remarkable qualities. One is that they can be easily 
obtained from a patient, and that means that we can use these 
cells from the same patient who is going to be treated without 
the use of embryonic tissues or cells. Another feature is that 
we can grow them very rapidly in the laboratory. The technical 
consequence of that is, we do not need viruses to work with 
these cells.
    The third and most remarkable feature of these cells is 
that they can become many of the hundreds of kinds of cells one 
finds in the human body, and they seem to be part of us, as a 
kind of repairing tissue, so for example, a few years ago we 
discovered that if one takes these cells from an experimental 
animal and puts them into a vein of the same animal, the cells 
go to many, many tissues, and they seem to take part in the 
repair of those tissues. They form the new cells of those 
tissues.
    So, for example, some of the cells go to bone and become 
new bone cells. The implication of that observation for therapy 
of bone disease is very broad. In fact, Drs. Edwin Horowitz and 
Malcolm Brenner and others at St. Jude's Hospital, with whom we 
are collaborating now, have recently reported some remarkable 
results in children with very brittle bones, children whose 
bones are so brittle they break a rib rolling over in bed.
    Some of the cells, after infusion in animals, go to lungs, 
and so the possibility is there of using these cells to repair 
the lung in cystic fibrosis.
    Some of the cells go to cartilage and joints. The 
possibility is there of using these cells to treat the damaged 
cartilage and joints that occurs as a result of arthritis.
    Some of the cells go to muscle, particularly if the muscle 
is damaged, so the possibility is there of using the cells to 
treat muscular dystrophy.
    In fact, at this point we are not quite sure what the whole 
spectrum is of diseases that could be treated. We cannot rule 
out the possibility of using these cells for diabetes, for 
heart disease, and kidneys. We simply do not know.
    But one of the most remarkable features of these cells is 
that when you put them in the brain of an experimental animal, 
they become new brain cells. The discovery was made in our 
laboratory by Dr. Asum Azizi and Donald Phinney some 2, 3 years 
ago. It is extended now by the work of Ira Black at Robert Wood 
Johnson Medical School, and two other investigators at other 
medical schools.
    They will become, in fact, some new brain cells, not all 
possible brain cells, and those observations have led us and 
others now to pursue the possibility of using these cells to 
treat Parkinsonism. In fact, we published a few months ago some 
very promising results in a rat model for Parkinson's.
    We are also thinking about using the cells to treat 
Alzheimer's disease, and stroke, even multiple sclerosis and 
spinal cord repair, so the prospects here are really very 
exciting.
    But I would like to close by making two points. Yes, all 
these things have been documented, the things I have just said. 
The possibility is there of using adult stem cells to treat 
these diseases, and we do not need therefore to use fetal 
tissues, and we do not need to put any foreign cells into a 
patient, and also we probably do not need to use a virus.
    But the second point I would like to stress is, we are not 
there yet. We have a very long way to go. For example, we are 
working very hard in using these cells, adult stem cells for 
Parkinsonism, but we are at least 2 years, maybe 3 years away 
from the first trial in patients.

                           PREPARED STATEMENT

    We need a lot of further work, we need continuing support 
from the National Institutes of Health, other sources such as 
the Louisiana State Consortium for Gene Therapy. We have to 
build up the data we need from animal experiments before we can 
try that. I think it would be a grave mistake to say, because 
of the work we have done and others have done with adult stem 
cells, one should now stop the kind of work with fetal tissues 
and cells as called for under the NIH guidelines.
    Thank you very much.
    [The statement follows:]
                Prepared Statement of Darwin J. Prockop
    Good Morning: My name is Darwin Prockop. I am currently the 
Director of the Center for Gene Therapy of Tulane University Health 
Sciences Center in New Orleans, Louisiana. I have an M.D. degree, a 
Ph.D. degree and two honorary doctoral degrees. In addition, I am a 
member of the National Academy of Sciences and the Institute of 
Medicine. I have published over 400 scientific articles. My work has 
been continuously supported by the National Institutes of Health. In 
addition, the Tulane Center for Gene Therapy that I now head is 
supported by funds from Tulane University, the Columbia Healthcare 
Association and the State of Louisiana Consortium for Gene Therapy.
    Our Tulane Center for Gene Therapy is carrying out research on a 
special class of adult stem cells that we think can potentially be used 
for the treatment of a number of devastating diseases. The adult stem 
cells we are using were first discovered 20 years ago, and we now know 
they have a number of truly remarkable features. One remarkable feature 
is that they can easily be obtained from a small sample of bone marrow 
from the same patient who is to be treated. Therefore if our therapies 
work, we will not need fetal tissues nor will we need to introduce 
foreign cells into a patient. Another important feature of the cells is 
that we can grow them rapidly in the laboratory. A technical 
consequence of this fact is that we can introduce new genes without the 
use of a virus. But the cells are of special interest primarily because 
they can change into a large number of the many different types of 
cells found in the human body. For example, we discovered several years 
ago that if the cells are isolated from the bone marrow of an animal, 
and then injected back into the blood stream of the same experimental 
animal from which the cells were obtained, they travel to a number of 
different organs and tissues. Moreover, when they reach a given organ 
or tissue they become new cells of the same kind that are normally 
found in that organ or tissue. In effect, the cells can repair and 
rejuvenate the organ or tissue.
    These features of the cells raise some exciting possibilities for 
using them in the therapy of diseases. For example, after infusion into 
a vein, some of the cells go to bone and become new bone cells. 
Therefore, the cells can potentially be used to treat diseases of bone 
such as osteoporosis. In fact, Drs. Edwin Horowitz, Malcolm Brenner and 
others at St. Jude's Children's Hospital In Memphis have recently 
reported promising results with the cells in treating children with 
severe brittle bone disease. Some of the cells go to lung and become 
lung cells. Therefore, they can potentially be useful in treating 
diseases of the lung such as cystic fibrosis. Some of the cells go to 
joints and cartilage and become cells of joints and cartilage. 
Therefore, they can potentially be used to treat diseases of the joint 
such as arthritis. Some preliminary data suggest that in the future the 
cells may also be useful in treating additional diseases such as 
diabetes, heart disease and kidney disease. One of the most dramatic 
observations about such cells is that they can become new cells of the 
brain. The first observations here were made one and two years ago in 
my laboratory primarily by Dr. Asum Azizi and Dr. Donald Phinney. In 
the past several months these observations have been extended by Dr. 
Ira Black and his associates at the Robert Wood Johnson Medical School 
and by research groups at two other medical schools. Because the cells 
can become some of the cells of the central nervous system, my 
laboratory and others are currently trying to see if they can be used 
to treat disease of the central nervous system such as Parkinsonism, 
Alzheimer's disease, stroke, spinal cord injury and multiple sclerosis. 
In fact, we have recently published some promising results using the 
cells in a rat model of Parkinsonism.
    In closing I would like to make two general statements. One is, 
yes, it is true that if the work that my laboratory and many others are 
now doing continues to be successful, it will open the possibility of 
using stem cells from adults, or even from the patient himself or 
herself, to treat a large number of terrible diseases. If successful, 
the therapies will not use any fetal tissues and probably will not use 
any viruses.
    However, I would like to stress the second very important point: we 
are not there yet. We have a long way to go. In my estimation, it will 
be at least two years before the first adult stem cells can be tested 
in patients with diseases such as Parkinsonism. It will probably be 
much longer for testing the cells in patients with the other diseases I 
have mentioned. We simply cannot be certain in advance which therapies 
will work and which will not. We need several years of hard work and we 
need continuing support from sources such as the National Institutes of 
Health and other sources such as the Louisiana State Consortium for 
Gene Therapy and the Columbia Healthcare Association that are currently 
supporting the Tulane Center for Gene Therapy. In my opinion, it would 
be a serious mistake to stop all research on human embryonic cells and 
tissues because of the exciting discoveries my laboratory and others 
have recently made about adult stem cells. We are simply not ready for 
a moon shot-like strategy in which we place all our bets on adult stem 
cells. I think it would be a mistake to tell the millions and millions 
of people whose lives are being destroyed by these terrible diseases 
that they to wait three, four, five years or even longer until we see 
the results obtained with adult stem cells before we even begin doing 
research on the other kinds of tissues and cells that may cure their 
diseases. I myself would be extremely sorry to see this sub committee 
or any other group decide that because of the work we and others have 
done on marrow stem cells, the kinds of research called for in the new 
NIH guidelines should be stopped.

    Senator Specter. Thank you, Dr. Prockop.
    Picking up on one of your last statements about the 
prospects for having stem cells utilized in humans in 
Parkinson's, would you elaborate first of all why--2 to 3 years 
is pretty good. We press with some frequency the people who 
appear here, especially those from NIH, where Senator Harkin 
and I have led the way for very material increases. In the last 
four appropriations periods we have increased the funding by 
some $7 billion, so that the current projection for next year 
will be in excess of $20 billion. What do you anticipate in 2 
to 3 years with respect to stem cells and Parkinson's?
    Dr. Prockop. Well, I can give you the best case scenario. 
If our experiments now being conducted in animals come out 
right in terms of efficacy, in toxicity, we think we may be 
able to begin the very first clinical trials in patients with 
Parkinsonism in 2 or 3 years. That is our best case scenario.
    Senator Specter. Why not sooner, Dr. Prockop?
    Dr. Prockop. Senator Specter, we have to be safe about 
this. We have to be sure we are not going to do more damage 
than we are going to help the patients.
    Senator Specter. What sort of experimentation do you 
anticipate between now and 2 to 3 years, when you are using the 
stem cells on people who have Parkinson's?
    Dr. Prockop. First we have to go to animal models of the 
disease, and there are models in rats and monkeys that are 
pretty close to what people suffer from in this disease, and we 
have to do the tests there. It is not a test one does for a day 
or a week. One has to do the treatment and then wait out 3 
months, or 6 months, or 9 months to be certain there is not 
some unfortunate consequence of what you are doing. It takes 
that, and one does not go from small animals to the more 
expensive tests in monkeys until one has those.
    Senator Specter. All right. You move to 2 or 3 years. You 
make the use of stem cells with people who have Parkinson's. Do 
you have a projection as to what your expectation is once that 
treatment starts, how long it would take from there, how long 
from there to cure Parkinson's?
    Dr. Prockop. Well, we have to go through the FDA 
regulations on this, and it turns out that is a new chapter. 
The stage of stem cells in people is a new chapter for the 
kinds of regulations the Federal Drug Administration should set 
up, and a beginning dialogue has already started between people 
working with stem cells and the FDA.
    What do you need in the way of safety and tests of efficacy 
before it is appropriate to go to a patient? It would just be 
terrible, and there are many examples in medicine, as you know, 
where a drug that looks very good on preliminary tests has some 
disastrous results in the first few patients.
    Senator Specter. Well, you are going to have to qualify 
with the Food and Drug Administration, understandably so, 
important precautions, but my question goes to, do you have a 
projection as to how long it will take to cure Parkinson's?
    Dr. Prockop. Senator Specter, I am an optimist, all right. 
One has to be an optimist to do research. I think it is 
somewhere in the order of 4 or 5 years. I am saying it is 2 or 
3 years for the first few patients, carefully controlled 
studies and a very carefully controlled environment. I am 
hoping it goes fast after that.
    Senator Specter. I appreciate your optimism, and there are 
many people hanging on, really hanging on your every word, 
people who have Parkinson's. One of my constituents from 
Pittsburgh has an hourglass. Whenever I see him he turns it 
upside down to remind me that every hour is slipping away, and 
that if something is not done soon it will be too late for him, 
so these time parameters are closely watched.
    Dr. Prockop. These, as you know, are very difficult 
judgments, when you can make the jump from the laboratory to 
the patient. It is not an easy decision, particularly when 
introducing something so new as stem cells into people.
    Senator Specter. Dr. Hynes, you had started to say that 
there would be a longer list than the itemized diseases which 
you thought could be dealt with by stem cells. Let me move to 
the issue of cancer, which is your specialty. What is your 
thinking about the potential for stem cells on cancer?
    Dr. Hynes. Well, a third of the people in this room are 
going to get cancer at some point in their lives, and so a 
large proportion of the American public needs treatment for 
this disease.
    Senator Specter. How many of the people in the room did you 
say would get cancer?
    Dr. Hynes. 1 in 3. We are making some progress on that, but 
we desperately need new approaches, and the application of stem 
cells to variations on bone marrow stem cell transportation is 
certainly one of the opportunities that I think is going to be 
very valuable--the replenishment of tissues that are damaged 
during therapy, the replacement of tissues that have to be 
removed.
    These stem cells are likely to be able to give rise to a 
large proportion of the tissues of the body, as are adult stem 
cells. I think that we need to work on both of them, and I 
think the opportunities from both of them approaching cancer 
are considerable.
    Senator Specter. You said it would take a long time to list 
all of the ailments which might be affected by stem cells. I 
think it is worth the time. Tell us what you have in mind.
    Dr. Hynes. Well, let me give you several more. For 
instance, replenishment of heart muscle tissue. We know from 
studies of embryonic stem cells in mice that one of the easy 
cells to derive from those is functional beating heart muscle 
cells, so that is a source of cells that could be used to 
repair damaged heart tissue. That will take a while, obviously. 
It is a complicated issue, but I think it is a definite 
prospect.
    Multiple sclerosis, the loss of the cells that ensheathe 
neurons, glial cells. Glial cells can be derived from embryonic 
stem cells, as can neurons, and so I already mentioned the 
neuronal diseases that affect the neurons themselves, but the 
accessory cells such as glia are also damaged in many diseases.
    Juvenile diabetes, the ability to make islet cells that 
make insulin, that one we are not so close to, I do not think, 
because I do not think there is so much evidence yet as to how 
to derive those cells from any source, but I think there is a 
very good chance that that will come about, so that is three 
more examples for you.
    Senator Specter. OK. Thank you very much.
    I yield now to my distinguished colleague, Senator Harkin.
    Senator Harkin. Mr. Chairman, you have covered all the 
points that I think need to be covered here. I just want to 
thank you two witnesses for being here. I just want to make it 
clear as to sort of the summation of what your testimony is, 
and that first of all, am I right in assuming that you both 
feel that the ethical guidelines that are published by NIH are 
sound and will permit the Federal funding of stem cell research 
within good, ethical and moral guidelines? Do you feel that 
they are strong guidelines?
    Dr. Hynes. I firmly believe that, that they separate the 
decisions about the embryos themselves from the science that 
goes forward from them, and I think that is a clear, clean 
separation. I think the ethical concerns are important and have 
been addressed by these guidelines.
    Senator Harkin. Dr. Prockop.
    Dr. Prockop. I think the guidelines are really a very good 
plan for us to move ahead.
    Senator Harkin. Second, it is both your positions that--
especially you, Dr. Prockop. You said that in my opinion it 
would be a serious mistake to stop all research on human 
embryonic cells and tissues because of the exciting discoveries 
in my laboratory, in other words, recently made about adult 
stem cells. We are simply not ready for a moon-shot-like 
strategy in which we place all our bets on adult stem cells.
    So your position is that the research ought to go forward 
on all the fronts. Is that basically what you are saying?
    Dr. Prockop. Absolutely. Absolutely, Senator Harkin. I 
really think it would be very unwise to do anything else at 
this time.
    Senator Harkin. Is that your feeling, too, doctor?
    Dr. Hynes. That is absolutely my opinion, too. Each of 
these approaches is likely to produce useful insights. They 
will not be identical, they will be complementary, and we 
should proceed on all fronts, adult, fetal, and embryonic stem 
cells.
    Senator Harkin. Thank you both very much.
    Senator Specter. Well, thank you very much, Dr. Hynes and 
Dr. Prockop. We appreciate the work you are doing. It is avant 
garde. This subcommittee and the full Congress is committed to 
research help. We have put in this year's budget $2.7 billion 
increase in NIH funding, which was on top of $2.3 billion last 
year, which had been reduced to $2.2 billion on an across-the-
board cut. We put in $2 billion the year before. We put in 
almost a billion the year before that, so the total is right at 
$8 billion in increases because of our determination to see 
this kind of phenomenal research advance, and the scientific 
community has performed magnificently, and the Congress would 
like to, if not perform magnificently, at least perform a 
supporting role, so we thank you.
    Dr. Hynes. Well, Senator, we thank you very much for your 
support, and we will do our best to make good use of it.
    Senator Specter. Thank you. I call our next panel, Dr. 
Ronald Heagy, Mr. Russell Saltzman, Dr. Anton-Lewis Usala.

STATEMENT OF RON HEAGY, PRESIDENT AND FOUNDER OF THE 
            LIFE IS AN ATTITUDE FOUNDATION
    Senator Specter. Our first witness, Dr. Ronald Heagy, is 
president and founder of the Life is an Attitude Foundation. He 
became a quadriplegic at the age of 17 when he was injured 
while surfing. He is a motivational speaker and the author of 
his autobiography, Life is an Attitude.
    In 1995, he received the Disabled Oregonian of the Year 
Award, and he received his master's degree in social work from 
San Diego State University.
    Mr. Heagy, we welcome you here. Let the record show that 
Mr. Heagy appears in a wheelchair with a microphone close to 
his lips and his hands.
    Mr. Heagy. A chin-operated wheelchair.
    Senator Specter. Thank you very much, and the floor is 
yours.
    Mr. Heagy. Thank you very much, and I appreciate this 
opportunity as a citizen of America and a resident of Oregon. I 
went through quite an adventure to get here, and the natives 
from the West Coast bring greetings to you.
    I have accomplished a number of things. My disability 
occurred as a result of my choice. We know that choices and 
consequences occur, and I ended up at 17 in a wheelchair, 
paralyzed from the neck down, a quadriplegic on a ventilator 
machine. Thank God I am no longer on a ventilator machine. I 
had a choice to make, whether I go on in life or I give up, and 
I chose to go on. I learned how to write with a pencil in my 
mouth, a stick in my teeth.
    I type, and I entered college. I was placed on welfare, 
started out with no income, had no support in college. I worked 
hard without the State support a lot of people assume that 
folks like myself get. I am interested in seeing some of those 
programs be developed, of course.
    I accomplished a master's degree at Seneca State 
University, and went out to try to get a job. I went through 20 
different interviews, did not get a job, so I started my own 
job, became a speaker. I spoke at my own high school, my first 
speech, and I have spoken to 2 million high school students in 
this Nation. I just finished speaking yesterday to 1,000 middle 
school children, and my emphasis is on life, and here I think 
we have an issue that focuses not on life.
    Of course, I hear everyone make assumptions, and there are 
opinions, and mine is an opinion, but we have no idea what this 
can do, we have no idea the ramifications that this sends out 
to our country.
    Who determines the quality of life? My life did not begin 
until I ended up in a wheelchair. Prior to this, I had a 
scholarship to play at Oregon State, I was a proud and arrogant 
person, lived for myself. I am now building camps across 
America for kids with disabilities to go out and enjoy the 
forest and the trees. I encourage them to be all that they can 
be, and I have seen the effect that a disabled person can have 
on others, including myself.
    You look at my web site, go over on .com, read my 
guestbook, I have thousands of entries from students, high 
school students across the Nation who have been encouraged not 
to commit suicide. I could read them here, but for the sake of 
time--if someone chooses to live as a result of my disability, 
I think it is encouraging to me to continue on.
    Life happens. Sometimes life dumps on you. You have got a 
couple of choices, get bitter or better, get negative or 
positive, and there is more than just Parkinson's here, and I 
know that we are all talking about some celebrity status, and I 
do not hold a celebrity status, but I do hold an opinion that a 
lot of my little disabled friends also hold.
    I am opposed to this on moral grounds, No. 1. No. 2, it 
forces financial support from taxpayers that may have 
opposition to this, who they would have no choice but for their 
dollars to go towards this.
    No. 3, what happens if we do find a cure using this type of 
procedure? Am I going to be forced to take the cure? If you say 
to me, well, we are not going to support you through medicare 
or medicaid any more because we have a cure for you, and I say, 
I cannot take it because it came through this procedure that I 
do not believe in, that is not just for me. That could be 
millions of Americans. There are 61 million Americans with 
disabilities that I am aware of, and it sounds like the numbers 
have changed, and not every one of them are on Prozac.
    There is life after disability, and I visited last week 
here to speak before some of the Federal Government leaders 
encouraging them. They asked me, they paid me to come speak to 
encourage them. I visited the Holocaust--I think this is the 
beginning of the end, and I am opposed to it.
    The other day I had a young man tell me at a school that he 
appreciated me talking about life, because there was so much 
shooting in America. What am I going to tell the kids--I am 
going to Denver, Colorado next week. What am I going to tell 
them, that we are deciding to take life, any form of life for 
the purpose of bettering somebody else's life?
    Isn't that exactly what these students are doing? I am 
trying to talk against that. If you get in my way, I am going 
to take you out, because my life would be better if you were 
not here. This is just a--it is appalling to myself, and I 
would appreciate this country considering other means in order 
for folks like myself to be bettered, and I believe that my 
quality of life is determined by my attitude and not my 
circumstance.
    Thank you.
    Senator Specter. Well, thank you very much, Mr. Heagy, for 
your comments, your testimony, your approach to the subject. We 
turn now to Pastor Russell Saltzman of the Ruskin Heights 
Lutheran Church in Kansas City, Missouri. Pastor Saltzman has 
served in other parishes in Omaha, Chicago, Charleston, South 
Carolina. In 1995 he was diagnosed with diabetes, so has some 
special insights. He received his master's in divinity from 
Trinity Lutheran Seminary in Columbus, Ohio.
    Welcome, Pastor Saltzman, and we look forward to your 
testimony.

STATEMENT OF RUSSELL SALTZMAN, PASTOR, RUSKIN HEIGHTS 
            LUTHERAN CHURCH, KANSAS CITY, MO
    Mr. Saltzman. Thank you, Mr. Chairman, Senator, for the 
opportunity to appear before this subcommittee. I count it as a 
privilege. I once worked for a Member of Congress and I know 
the energy and the time you bring to this work, and how 
difficult your decisions sometimes are, and you are to be 
thanked for your efforts.
    This is my second visit to the Capitol grounds since I left 
Washington, and I left January 20, 1973, and that was the day 
Richard Nixon was inaugurated for his second term. I like 
pointing this out. I left Washington and he had to resign 
later. My wife hates that joke. Evidently she is right.
    Mr. Saltzman. I am here appearing as a person with diabetes 
to testify against the use of human embryonic stem cell 
research. First, though, I will reveal something of myself. I 
am the adopted child of Harry and Lola Saltzman. My parents, 
who live yet in the house my father built, and where I was 
raised in Olathe, Kansas, and since I am an adopted child you 
might guess accurately that the circumstances of my conception 
were not ideal.
    When I was conceived in the summer of 1946 I was an 
unplanned and an unwanted pregnancy. My parents, birth parents, 
were members of the same family. In fact, they were step-
siblings, and very possibly my conception was the result not 
only of step-sibling incest but step-sibling rape.
    There is no question in my mind, given the circumstances 
today, that my birth mother would have been urged to accept 
abortion, and very likely would have sought one as the means of 
solving the dilemma I represented. I am unable to look at these 
issues in any light except that of my origin, and when I say 
that appearing here is a privilege, I hope I also convey my 
sense of the miraculous, for had my conception occurred after 
1972 I would not be here at all.
    And suddenly it comes to mind that, having been aborted, 
the fetal parts that were once me might have become research 
material for somebody's investigation into the very disease I 
have come here to discuss, so I say at the outset, this is a 
terrible thing we undertake in these discussions, not only 
because the matter touches me so personally, but also because I 
know our common origin, our core humanity that links us one to 
another, whatever our stage of development of maturity. We all 
spring once from an act of union between egg and sperm. We all 
once were human embryos. We all once were fetuses, quickening 
in our mother's wombs. We are all each human life.
    We may hope that all of us were conceived in love, but in 
my case that matters to me not at all whether I was conceived 
in love or in violence. What is important for me is the fact 
that I am here in the first place, and my existence, by itself, 
has some considerable consequence for other people, not least 
for my seven children, two of whom are adopted.
    I suffer from diabetes, and since my diagnosis I have 
learned that the burden of chronic illness is a genuine burden, 
and I have experienced the progression of this illness from a 
time when simple diet alterations controlled it to the point 
now where I require oral and insulin injections, and it is the 
chronic part that constitutes the real burden, knowing I will 
never be rid of it, and yet I cannot consent to stem cell 
therapy, and what I find disturbing about this is that no 
technique at present has produced a pancreatic cell.
    It comes to a question, is the human embryo human life, or 
is it a mere bit of research material, and if it is mere 
research material, then why should any human life at any stage 
of development, yours or mine, carry any special privilege?
    But if the embryo is human life, then we should have in 
place some restraint that cautions the strong against using the 
weak for their own purposes.

                           PREPARED STATEMENT

    If a cure for diabetes and a host of other ailments require 
the production and destruction of human embryos, then I beg you 
to consider the possibility that some diseases are better than 
their cure.
    Thank you.
    [The statement follows:]

               Prepared Statement of Russell E. Saltzman
    Thank you, Mr. Chairman and Senators, for the opportunity to appear 
before this subcommittee this morning. I count it as a privilege, I 
once worked for a Member of Congress and I know the energy and the time 
you bring to this work and how difficult your decisions sometimes are, 
and you are to be thanked for your efforts. This is my first visit to 
the Capitol grounds since I left Washington on January 20, 1973. That 
was the day Richard Nixon was inaugurated for his second term. I don't 
want to make too much of it, but I do enjoy pointing out that I left 
Washington and the president had to resign.
    I am here as a person with diabetes to testify against the use of 
human embryonic stem cell research. But I shall first reveal something 
of myself. I am the adopted child of Harry and Lola Saltzman, my 
parents who live yet in the home where I was raised in Olathe, Kansas.
    Since I am an adopted child, you might guess, accurately, that the 
circumstances of my conception were not ideal. In the summer of 1946, I 
was an unplanned, unwanted pregnancy. My birth parents were members of 
the same family. In fact they were step-siblings. Very possibly my 
conception was the result not only of step-sibling incest, but step-
sibling rape.
    There is no question in my mind--given the circumstances current 
these days--that my birth mother would have been urged to accept 
abortion and very likely would have sought one as the means of solving 
the dilemma I represented. I am unable to look at abortion in any light 
except those of my origin. When I say that appearing here is a 
privilege, I hope I also convey my sense of the miraculous, for had my 
conception occurred after 1972, I would not be here at all.
    And suddenly it comes to mind that--having been aborted--the fetal 
parts that were once me might have become research material for 
somebody's investigation into the very disease I have come here to 
discuss.
    So at the outset, I say it is a terrible thing we undertake in 
these discussions, not only because the matter touches me so 
personally, but also because I know our common origin, the base 
humanity that links us one to another, whatever our stage of 
development or maturity. We all once sprang from an act of union 
between egg and sperm. We all once were human embryos. We all once were 
fetuses quickening in our mothers' wombs. We are all, each, human life. 
We may hope that all of us were conceived in love, but in my case that 
matters not at all. Whether I was conceived in love or in violence, 
what is important for me is the fact that I am here in the first place. 
My existence by itself has some considerable consequence for other 
people, not least for my seven children, two of whom are adopted.
    I suffer from diabetes. Since my diagnosis in 1995, I have learned 
that the burden of a chronic illness is a real burden. I have 
experienced the progression of this illness from a time when simple 
diet alterations controlled it, to the point now where I require both 
oral medication and insulin injections. It is the chronic part that 
constitutes the real burden, knowing I shall never be rid of it, 
knowing my life will always be governed by diet and injection 
schedules, and knowing, too, that my death probably will be the result 
of some diabetic complication. When I say I wish I did not have it, I 
am saying there is almost anything I would do to get rid of it.
    Almost.
    The prospect of stem cell therapy derived from human embryonic 
research--involving the destruction of a human embryo--touches me in a 
most profound way. I would never consent to any treatment for my 
diabetes that directly or indirectly came about as the result of 
destroying a human embryo. What I find disturbing about this incessant 
rush to harvest stem cells from embryos is the fact that no researcher 
to date has been able to develop a pancreatic cell from the techniques 
presently used, this while there are several promising avenues of 
research that do not involve destruction of a human embryo.
    Most recently, I have learned about investigations by Canadian 
researchers that employed pancreatic islet cells from cadavers. The 
technique successfully eliminated insulin-dependence of several 
diabetics who received the procedure. The procedure is subject to 
further trials and it must be nuanced in application. But this holds 
greater promise for a diabetic cure than anything else I have heard 
about--and islet cell transplant is ethically neutral. It has no moral 
implications associated with it. Yet, we here in the United States seem 
in a rush to use what is arguably the most ethically objectionable 
method available, while other morally neutral medical technologies 
virtually are at hand. The President's own National Bioethics Advisory 
Commission has said that because human embryos deserve respect as a 
developing form of human life, destroying them ``is justifiable only if 
no less morally problematic alternatives are available for advancing 
research.'' The fact is, those alternatives exist.
    It comes to a question. Is the human embryo human life, or is it a 
mere bit of research material? If it is mere research material, then 
why should any human life at any stage of development--yours or mine--
carry any special privilege? But if the embryo is human life, then we 
should have in place some restraint that cautions the strong against 
using the weak for their own purposes.
    I would commend to your reading Aldous Huxley's Brave New World. 
Writing in 1933 Huxley, with astonishing prophetic foresight, created a 
world of genetic clones and what he called ``decanted babies.'' All 
this arose because in the world of his novel, the human embryo was 
merely research material. He worried that science was being twisted all 
around. Where once, as with the sabbath, science was made for Man, he 
foresaw a time when Man would be made for science. In Huxley's 
fictionalized world the process that turned science around was 
methodical and deliberate, and without moral regard. In our own world, 
the process going on is less tidy but no less deliberate, and, I fear, 
with equally little moral regard.
    If a cure for diabetes and a host of other ailments require the 
production and destruction of human embryos, then I beg you to consider 
the possibility that some diseases are better than their cure.

    Senator Specter. Thank you very much, Pastor Saltzman.
    We will have questions when we conclude the full panel, and 
we now turn to the third and final member of the panel, Dr. 
Anton-Lewis Usala, founder, chairman, and chief scientific 
officer for Encelle, a biomedical device transplantation 
company located in Greenville, North Carolina. He received his 
medical degree from Jefferson Medical College in Philadelphia. 
Welcome, Dr. Usala. The floor is yours.

STATEMENT OF ANTON-LEWIS USALA, M.D., CHAIRMAN/CHIEF 
            TECHNICAL OFFICER, ENCELLE, INC., 
            GREENVILLE, NC
    Dr. Usala. Senators, I come as a medical scientist, a 
person who has had diabetes 41 of my 42 years, and a concerned 
citizen of the United States. I was asked to present the 
approach my company has developed for the treatment of type I 
diabetes and other diseases states that require replacement of 
nonfunctioning tissues.
    The premise of our approach is to replace as 
physiologically as possible the insulin-producing cells that 
have been destroyed in children with diabetes. This effort 
requires large numbers of replacement cells, as there are 
approximately 1 million patients with type I diabetes in the 
United States, and since only several thousand human pancreases 
are available to provide a resource for these cells, developing 
a method for preventing rejection of animal-derived cells has 
become the focus of our effort.
    Our most promising approach to solving the problem of 
cross-species transplantation has involved culturing tissue 
from pig pancreases and proprietary hydrogel material derived 
from pig skin. The cells in this material are then injected in 
a diabetic animal without using any immunosuppression. The 
material is a copolymer designed to simulate the structure of 
connective tissue present during the first few months of fetal 
development.
    Connective tissue is the scaffolding the cells attach to, 
and during phytogenesis the connective tissue is quite 
different in appearance than later in life.
    Stem cells are attractive as a therapy because they are 
responsive to embryonic signals and are thereby differentiable. 
That is, they can differentiate into many different kinds of 
tissue. Our biopolymer material has been used for its ability 
to directly simulate host tissue differentiation, regeneration 
and repair without the need for stem cell transplantation.
    Last Thursday, the Food and Drug Administration authorized 
the commencement of human clinical trials utilizing our 
material in the treatment of diabetic foot ulcers. We have 
demonstrated that a single injection of the material induces 
complete reconstruction of chronic and acute skin wounds in 
rabbits, dogs, and pigs. This includes reconstruction of skin 
architecture that is normally only made during fetal 
development. Blood vessels, hair follicles, and dermal 
structures are integrated in a scarless healing after a single 
injection of the material.
    Replication of specific tissue requires cells to receive an 
enormous number of specific signals. What defines a human life 
is cellular mass able to produce and integrate this enormous 
number of sequences, and this occurs shortly after 
fertilization. The cascade of specific cellular differentiation 
begins and continues throughout the adult life of the person. 
It can be argued when the reasoning of the fetal organism 
begins. It cannot be argued when it is human.
    Cells from a human embryonic donor can be induced to this 
degree. Severed from the intricate and specific environment in 
which they are made, totipotent or pluripotent cells require 
the signals from thousands of sources. While knowledge would 
undoubtedly be gained from this research, this is different 
from development of a treatment, and there are different 
approaches that do not require using human embryonic cells.
    Whether or not our approach will induce recreation of 
complex tissue structures in human patients will be known very 
shortly. I hope what we have observed in preclinical studies 
will also be the case in patients with diabetic foot ulcers. If 
so, variations on this approach will be researched by others 
for the ability to induce other types of tissue repair such as 
nerves, utilizing the patient's own regenerative capabilities.
    I am 42 years old, and have had diabetes 41 years. I have 
seen the therapy for diabetes dramatically change over this 
time. Prior to the manufacture of disposable needles, which 
appeared when I was 8 years old, I recall my mother sharpening 
reusable hypodermic needles on a stone to make the injections 
less painful. During my childhood, adolescence and young 
adulthood, the medical community nearly unanimously concurred 
that blood sugar control made no difference in the development 
of diabetic complications, and thousands of children with 
diabetes died prematurely as a result.
    Clearly, embryonic stem cell research will require enormous 
resources to define a usable path to treatment, if such a path 
exists. While many respected scientists are understandably 
enthused about the possibilities, human embryonic stem cells 
may not provide a viable path, and as the path adopted for 
treatment of diabetes for 40 years, just as this treatment was 
also not viable.
    Senators, I am not sure that my experience gives me any 
more legitimacy in my views than any of the other 1 million 
patients with diabetes or any other conditions, but the more 
fundamental issue is not whether embryonic stem cells will 
provide a better path for a cure. It is whether or not this 
line of research should be entertained by society.
    All societies are based on law, even unjust societies. The 
difference between a just and an unjust society is the premises 
it accepts to begin establishment of legal precedents. The 
United States, in my view, is a uniquely just society because 
of the first 10 amendments to its Constitution. These 
amendments for the first time in human history established a 
system where the rights of the individual supersede the 
perceived right of the State, implicitly defining the 
individual as the most valued entity within the society.
    History has amply demonstrated the ghastly consequences 
when Government arbitrarily defines what constitutes human 
life. The law is based on precedent, and once the United States 
allows the individual human embryo to be sacrificed for a 
perceived greater good, the greatest defense for the rights of 
individuals will have been eroded. For the first time, the 
perceived right of the Government will supersede the right of 
the individual.

                           PREPARED STATEMENT

    The pain and suffering endured by those with disease must 
be addressed, and the means to providing solutions encouraged. 
The decision to allow human embryonic stem cell research, 
however, must not be made solely on the basis of its utility in 
curing disease. It must be considered in the context of its 
potential to initiate an insidious disease in our society's 
values and its potential to erode the foundation that the 
United States provides for all individuals. Alternative methods 
and cures must be pursued on both scientific and societal 
grounds.
    Thank you.
    [The statement follows:]

                Prepared Statement of Anton-Lewis Usala
    Senators: I come as a medical scientist, a person who has had 
diabetes 41 of my 42 years, and a concerned citizen of the United 
States. I was asked to present the approach my company has developed 
for the treatment of Type I diabetes, and other disease states that 
require replacement of non-functioning tissues. The premise of our 
approach is to replace, as naturally and physiologically as possible, 
the insulin producing cells that are destroyed in children with 
diabetes. This effort would require large numbers of replacement cells, 
as there are approximately one million patients with Type I diabetes in 
the United States. Since only several thousand human pancreases are 
available to provide a source of these cells, developing a method of 
preventing rejection of animal derived insulin producing cells has 
become the focus of our effort.
    Our most promising approach to solving the problem of cross species 
transplantation has involved culturing tissue from pig pancreases in a 
proprietary hydrogel material derived from pig skin. The cells in this 
material are then injected in a diabetic animal, without using any 
immunosuppression. The material is a co-polymer designed to simulate 
the structure of connective tissue present during the first few months 
of fetal development. Connective tissue is the scaffolding that cells 
attach to, and during fetogenesis, the connective tissue is quite 
different in appearance than later in life.
    My hypothesis was that during embryogenesis, undifferentiated cells 
must first bind to this connective tissue in order to properly 
communicate, integrate, and mature with surrounding cells. If this were 
true, then perhaps allowing mature cells to bind to a similar 
connective structure would induce de-differentiation, allowing them to 
receive local signals from the area in which they were transplanted. 
This de-differentiation apparently reduces the ability of the patient 
to recognize the tissue as foreign. When we have placed pig pancreatic 
cells in our biopolymer material, and injected them into either the 
muscle or liver of diabetic dogs, the animals' diabetes control is 
greatly improved. Furthermore, we have demonstrated that the pig tissue 
survives, and integrates within the dog tissue, without the need for 
immunosuppressive drugs. This is not yet a cure, but does demonstrate 
the ability to keep foreign tissue alive and partially functional 
without the need for either human embryonic tissues or 
immunosuppression.
    Stem cells are attractive as a therapy because they are responsive 
to embryonic signals and are thereby ``differentiable'' into many 
different kinds of tissue. Our biopolymer material has been used for 
its ability to directly stimulate host tissue de-differentiation, 
regeneration and repair, without the need for stem cell 
transplantation. Last Thursday, the Food and Drug Administration 
authorized the commencement of human clinical trials utilizing our 
material in the treatment of diabetic foot ulcers. We have demonstrated 
that a single injection of the material induces complete reconstruction 
of chronic and acute skin wounds in rabbits, dogs, and pigs. This 
includes reconstruction of skin architecture that is non-nally only 
made during fetal development. Blood vessels, hair follicles, and 
dermal structures are integrated in a scarless, rapid healing after a 
single injection of the material. While the complete mechanism of 
action is not known, we believe these findings result from the 
following:
    Upon injection, the biopolymer binds to the patient tissue with 
which it comes in contact. Upon binding to this fetal-like connective 
tissue biopolymer, the host cells de-differentiate into a fetal shape, 
resembling pluripotent mesenchymal tissue. Over time, we have observed 
these de-differentiated cells apparently differentiating into specific 
structures required in the wound areas. Thus, the host cells are again 
receptive to local signals that enable re-creation of specialized 
structures after binding to the biopolymer. Thus far, the material is 
limited in its ability to induce recreation to skin and related 
structures.
    Replication of specific tissue requires cells to receive an 
enormous number of specific signals. What defines a human life is the 
cellular mass that is able to produce and integrate this enormous 
number of sequences, that occurs shortly after fertilization. The 
cascade specific cellular differentiation begins, and continues 
throughout the adult life of the person. It can be argued when the 
reasoning of the fetal organism begins; it cannot be argued when it is 
human.
    While the developing embryo is equipped to orchestrate the timing 
of these millions of signals, it is not at all clear how taking 
totipotent or pluripotent cells from a human embryonic donor can be 
induced to this degree. Severed from the intricate and specific 
environment in which they are made, totipotent or pluripotent cells 
require the signals from thousands of sources. While knowledge would 
undoubtedly be gained from this research, this is different from 
development of a treatment, and there are different approaches that do 
not require using human embryonic cells.
    Whether or not our approach will induce recreation of complex 
tissue structures in human patients will be known shortly. I hope what 
we have observed in animal studies will also be the case in patients 
with diabetic foot ulcers. If so, variations on this approach will be 
researched by others for the ability to induce other types of tissue 
repair, such as nerves, utilizing the patient's own regenerative 
abilities.
    Often, the highly regarded scientific and medical community will 
take a position based on their collective interpretation of data that 
is incorrect. Differing opinions are not heard, because they are 
outside the mainstream thinking. In the treatment of diabetes, this has 
had chilling results. I am 42 years old, and have had diabetes 41 
years. Prior to the manufacturing of disposable needles when I was 8 
years old, I recall my mother sharpening the re-useable hypodermic 
needles on a stone to make the insulin injection less painful. During 
my childhood, adolescence, and young adulthood, the medical community 
nearly unanimously concurred that blood sugar control made no 
difference in the development of diabetic complications. In 1968, the 
life expectancy of a child who developed diabetes before the age of ten 
was approximately 30 years, as most would die of kidney failure. When I 
was 10, it seemed to me that nature keeps blood sugars within a very 
tight range for some reason, and it accomplishes this by releasing 
insulin from the pancreas whenever a person eats food. Shortly 
thereafter, I began to surreptitiously take injections of fast acting 
insulin before or immediately after eating. My intent was to try to 
replicate nature by providing insulin when it was required to normalize 
blood sugar after eating.
    I sit here today able to talk about the mistakes. Most of the 
children I grew up with that have diabetes are no longer alive, as 
their physicians practiced the state of the art medical doctrine, which 
was that control of blood sugar was not important. Throughout my 
medical school training, this is what was taught. It wasn't until the 
early 1990's that the medical community finally reversed its view, 
after hundreds of thousands of children had died from complications of 
poorly controlled diabetes. Clearly, embryonic stem cell research will 
require enormous resources to define a useable path to treatment--if 
such a path exists. While many respected scientists are understandably 
enthused about their possibility, human embryonic stem cells may not 
provide a viable path, as the path adopted for treatment of diabetes 
for forty years was also not viable.
    Senators, I am not sure that my experience gives me any more 
legitimacy in my views than any of the other one million patients with 
diabetes. But the more fundamental issue is not whether embryonic stem 
cells will provide a better path for a cure or not; it is whether or 
not this line of research should be entertained in our society.
    All societies are based on law, even unjust societies. The 
difference between a just and an unjust society is the premises it 
accepts to begin establishment of legal precedents. The United States, 
in my view, is a uniquely just society because of the First Ten 
Amendments to its Constitution. These Amendments, for the first time in 
human history, established a system where the rights of the individual 
supercede the perceived right of the State, implicitly defining the 
individual as the most valued entity within the society.
    History has amply demonstrated the ghastly consequences when 
government arbitrarily defines what constitutes human life. I am not 
suggesting that those who want to use human embryonic tissue are of the 
same mind. However, the law is based on precedent, and once the United 
States allows the individual human embryo to be sacrificed for a 
perceived greater good, the greatest defense for the rights of 
individuals will have been eroded. For the first time, the perceived 
right of the government will supercede the right of the individual. The 
pain and suffering endured by those with disease must be addressed, and 
the means to providing solutions encouraged. The decision to allow 
human embryonic stem cell research, however, must not be made solely on 
the basis of its utility in curing disease. It must be considered in 
the context of its potential to initiate an insidious disease in our 
society's values, and its potential to erode the foundation of the 
United States' protection of all individuals. Alternative methods and 
cures must be pursued, on both scientific and societal grounds.

    Senator Specter. Thank you very much, Dr. Usala.
    I will begin the questioning with you, Mr. Heagy, and you 
obviously have undergone great trauma, and interestingly make 
the comment that your life began on the day when you were 
disabled, and you talk about the quality of life, and Dr. Usala 
talks about sacrificing human embryos.
    What we are considering here in a sense is the availability 
of stem cells from embryos to cure very horrible illnesses, 
Parkinson's, Alzheimer's, stroke, perhaps cancer, and we have 
the embryo, which Dr. Usala defines as human embryos, but what 
is your evaluation of the quality of life, on the assumption 
that the human embryo is living, or with the conclusion that 
the human embryo is living, with the ability of those stem 
cells to cure so many people from dreaded diseases?
    Mr. Heagy. I think the difference in my opinion would be 
federally funding programs and the ramifications of the future 
that we do not have control of. At this point we all are here, 
but eventually we will all die and new people will come 
forward, and this I think is the beginning of some serious 
decay in respect to human life.
    Senator Specter. The embryos are going to be destroyed, as 
we all know, if they are not used in in vitro fertilization, 
and being discarded, they will not live, again assuming life, 
and how do you evaluate their being discarded and the quality 
of life which they have as embryos contrasted with the good 
that may be done to people who have Parkinson's or other 
diseases?
    Mr. Heagy. I believe that that is another issue that this 
Nation has determined was an okay procedure, and it is a 
different subject. We are talking about a new set of laws, a 
new set of procedures, and utilizing that and funding that 
through taxpayer's dollars. I am not opposed to research. I am 
not opposed to walking again. I am just opposed to the process 
that we are discussing here and the funding source, and the 
good people in this Nation that have serious negative 
implications to this process. Forcing them to fund this is--it 
goes against what I know about America.
    Senator Specter. Let me ask the same essential question, 
Dr. Usala. You talk about sacrificing human embryos. To what 
extent would you say that you have a human embryo, given the 
status of the embryo in in vitro fertilization and the 
consequence of its being discarded, so it either--it ceases to 
exist, or cannot help people who have horrible illness, what do 
you think?
    Dr. Usala. I think you have crystallized, Senator, the 
dilemma. I think that some of what I have learned is from the 
result of not elective, but research that was done on 
abortuses, and so some of what we were able to do to stimulate 
the regeneration, and I am talking total regeneration and 
reconstruction, was coming from that kind of, taking an infant 
that was prematurely born and died, we were able to get some 
knowledge from that.
    But Senator, what I am concerned about is the paradigm 
shift that is occurring here. We all know what happens once the 
precedent is that, well, taking a human embryo and using it for 
research is okay. What I am very much concerned about is the 
paradigm shift and its effect on the United States 
Constitution. I am very, very concerned about that.
    Senator Specter. You say you think it is okay to use the 
human embryo? You are concerned about what that may lead to?
    Dr. Usala. What we know it will lead to, Senator.
    Senator Specter. Well, what do you know it will lead to?
    Dr. Usala. Well, for instance, for in vitro fertilization 
they can create embryos and----
    Senator Specter. I am going to go a little over my 5 
minutes here, because I want you to have a full opportunity to 
set forth your viewpoints here.
    Dr. Usala. Thank you, Senator. What we know will happen is 
that with in vitro fertilization, if we have these embryos, 
science is an all-consuming fire. What will happen is, maybe a 
few more embryos will be fertilized so that we could give the 
embryos to justifiably sound science for development.
    Senator Specter. So you think they will be creating embryos 
for scientific research.
    Dr. Usala. It will lead to that, if we state as the 
precedent that the use of these embryos in this way is in and 
of itself okay.
    Senator Specter. But if we could certify, guarantee that 
embryos would not be created except for genuine purposes of in 
vitro fertilization, and it is a medical decision as to how 
many you need to create and what you will do with them, but you 
do not create them specifically for scientific research, if 
that could be done, would that satisfy you?
    Dr. Usala. It cannot be. I am a medical scientist, and I 
know what happens. What happens is, as I say, science is an 
all-consuming fire, and the need to know is all-consuming, and 
what will happen is, well, let us say we need to do, 
arbitrarily, 10 embryos to have a reasonable success at in 
vitro fertilization. What will happen, I assure you, is that 
for medical reasons is they will say, well, we should probably 
do 20, because that way we can get a better feel for this. It 
will happen, Senator, there is no doubt. It cannot be 
legislated.
    Senator Specter. Well, the guidelines prohibit it. 
Specifically, quote, the ban on Federal funding involves the 
prohibition of Federal funding for creation of a human embryo, 
or embryos for research purposes, but I understand your point 
of view, and we are very interested in it.
    Pastor Saltzman, you and I have the same home State. I grew 
up in Russell, Kansas.
    Mr. Saltzman. We were at opposite ends.
    Senator Specter. Not quite. Russell is about half-way 
across the State, and Olathe, of course, is in the eastern part 
of the State. When you were 1 year old I was graduating from 
high school, so I have seniority on you. I want you to 
understand that.
    Mr. Saltzman. I am happy to hear it.
    Senator Specter. Pastor Saltzman, you talk about, if 
aborted you might have had your fetal tissue as part of someone 
else. Do you oppose the use of fetal tissue for medical 
purposes?
    Mr. Saltzman. By and large, yes. I think the present system 
we have is subject to far too much abuse. We have had examples 
in the Kansas City area of tissue extraction companies playing 
things with the fees and such as that.
    Senator Specter. Tissue extraction companies, fees?
    Mr. Saltzman. Yes.
    Senator Specter. Well, there had been the concern expressed 
by many that use of fetal tissue would promote abortions. That 
concern has pretty much receded. We had an interesting vote in 
the Senate when Senator Thurmond, who is a very strong 
conservative and strong right-to-life, had opposed the use of 
fetal tissue, and then his daughter had juvenile diabetes, so 
he testified in favor of use of fetal tissue, and instead of 40 
Senators favoring fetal tissue, the number jumped to 80, and 
parenthetically it is worth observing that Senator Thurmond 
believes in the use of stem cells.
    Mr. Saltzman. If you are speaking of fetal material derived 
innocently----
    Senator Specter. Yes.
    Mr. Saltzman. Spontaneous miscarriage, that removes the 
moral dilemma for me.
    Senator Specter. Well, but suppose you have fetal tissue, 
or however you designate it, terminology, from an abortion 
which is being done not to create fetal tissue but for many 
other reasons why they are performed, do you object to the use 
of that tissue?
    Mr. Saltzman. I do.
    Mr. Heagy. Could I make a comment on that, Senator?
    Senator Specter. We would be interested in your views on 
the life or quality of life of embryos. You said we were all 
once embryos, but the general pattern is that we are embryos 
having been created by in most cases by a wife and a husband 
and then carried to term, as you and I were, but what about 
these embryos which are frozen, created for purposes of in 
vitro fertilization and then----
    Mr. Saltzman. I think if you are asking about their 
disposition----
    Senator Specter. Disposition, quality of life----
    Mr. Saltzman. I think that is a separate issue, and it 
needs to be addressed separately. What I do know is that we 
should not use human life for the benefit of ourselves. If 
these embryos are going to be discarded anyway--I know the 
argument goes--why not go ahead and use them? We have prisoners 
on death row that are going to be executed anyway. Why not get 
some use out of them before they are dead?
    All of this cheapens, I think, human life and our respect 
for it. We have decided that some may be used by others for 
their own purposes. Is this what we are saying?
    If the embryo was human life, as the President's own 
advisory bioethics commission said, if the embryo is human 
life, then it deserves the respect owing to human life, and the 
commission also said then that when it comes to research in 
these areas we need to find the least morally objectionable 
method to advance our research, and that is the President's own 
commission, and this is all I am saying, too.
    Senator Specter. Well, it is a matter of where you draw the 
line. If you have convicts who are serving life sentences----
    Mr. Saltzman. Where do you draw the line? Where do you make 
the distinction? The convict is bigger and the embryo is not?
    Senator Specter. Well, I would draw the line that a convict 
is a living person, and although he may have the death penalty, 
or she may have the death penalty, they may have a DNA test 
tomorrow and they may be exonerated, but at any rate, they are 
living people, and this comes back to the issue of the quality 
of life which is articulated of the embryo and the contrast as 
to what can be done by way of saving a lot of people from 
greater diseases.
    Senator Harkin--Pastor Saltzman, I did not want to cut you 
off if you want to say something more.
    Senator Harkin. Thank you, Mr. Chairman. I have listened 
closely to your line of questioning and the answers and I 
again--I do not think anyone should--I know Senator Specter 
well, we know each other well. I do not think anyone should get 
the misimpression that we do not wrestle with the ethical and 
moral implications of what this is all about.
    We push very hard a couple of years ago, 2, 3 years ago to 
get the bioethics commission. We had them here to testify to 
get them to move on this, to examine in depth the ethical and 
moral implications of this, and to give us some 
recommendations--I am not an ethicist. There are people that 
understand these things much better than I do. You, for 
example--but try to get as much of an input as possible into 
this to advise us on what would be--if there is an ethical and 
moral way of doing this, and if there is, how? Well, they came 
back and said yes there is, and here is how, and they put these 
guidelines out, and I guess we get back to the point that--the 
dividing point on this seems to me to be whether or not one--
you see, I think if one is opposed to in vitro fertilization as 
a procedure, just totally opposed to that.
    Then I can understand how logically from that one might say 
that the use of any of these embryos from that is immoral. If 
the first act is immoral, the other acts are immoral, so I 
guess we have to first find out whether or not the person 
thinks in vitro fertilization is an ethical or moral act.
    Second, then it seems to me that once you have in vitro 
fertilization, you have got all these frozen embryos--I guess 
we have got 100,000, I guess, that are frozen now in nitrogen, 
liquid nitrogen--that they are going to be discarded. They are 
just not going to be kept forever and ever and ever and ever. 
They are going to be discarded.
    So if they are going to be discarded, is it more ethical 
and moral, then, to use these in a method that might reduce 
human suffering, prolong life, make our quality of life better? 
Is it more ethical and moral to do that than it is to discard 
them? That seems to me where we are coming, where we are sort 
of at the balance point here.
    Mr. Saltzman. Let me pose a question to you, Senator, if I 
may. We have 100,000 frozen embryos at present on hand, 
roughly.
    Senator Harkin. Yes.
    Mr. Saltzman. We use those up. We go ahead with our stem 
cell research, and we use those up. We need more. Are we not 
stimulating the production of human embryos for the purpose of 
experimentation? We are not going to stop at 100,000.
    Senator Harkin. Well, let me just say this. No, under the 
guidelines, again, unless you just say the guidelines are 
worthless, but I do not think they are worthless. They are 
saying that----
    Mr. Saltzman. Can they be amended? They will be.
    Senator Harkin. Well, it says here that providers of stem 
cells cannot make a profit. You cannot sell them, and you have 
to have informed consent.
    For example, if I am a donor through in vitro 
fertilization, I say no, you cannot use those embryos for that, 
you cannot use them. You have to have the informed consent of 
the donors themselves, and they have to be derived in excess of 
those needed for in vitro fertilization, so if someone goes in 
for in vitro fertilization, embryos are created, one is 
implanted, and that embryo develops into a human being and a 
child, then you have the other embryos that are frozen, I mean, 
how can you say you are producing the embryos for research when 
in fact it is a couple going in for in vitro fertilization? 
They are not going in saying, we want to donate for research. 
They are donating it for in vitro fertilization.
    Mr. Saltzman. Why would the fertility clinic--I presume the 
Federal money would be spent paying the fertility clinic for 
the embryos that have been donated, is that not so?
    Senator Harkin. I am not--say that again. What is that?
    Mr. Saltzman. When a couple goes in, they make 10 embryos, 
they use one, there is 9 left over, the Federal Government will 
pay the fertility clinic for the 9 embryos, will they not?
    Senator Harkin. I do not think so.
    Mr. Saltzman. No?
    Senator Harkin. Two things. Under the guidelines, they 
cannot pay for them. They can pay for the cost of 
transportation, storage and transportation, but they cannot 
make a profit from them. They cannot sell them. Second, the 
guidelines specifically require the separation of the donation 
and the generation.
    Mr. Saltzman. I think that is a bookkeeping thing, you 
know.
    Senator Harkin. Well, let me ask you this. Are you opposed 
to in vitro fertilization?
    Mr. Saltzman. Personally?
    Senator Harkin. Well, I do not know, I mean personally, 
philosophically, ethically, morally, are you opposed to it?
    Mr. Saltzman. No. I believe that is morally neutral. It 
raises a host of questions afterwards, though, and that is why 
we are having these discussions today. What do you do with 
these embryos that are left?
    Senator Harkin. Well, that is right. I keep saying that is 
the balance point.
    Mr. Saltzman. I do not know what to do with them. I know 
what not to do.
    Senator Harkin. Well, do you think they should--I mean, if 
they are going to be destroyed, I mean, again, what do you do?
    Mr. Saltzman. I cannot--we are going to die, all of us.
    Senator Harkin. We are all going to die, that is true.
    Mr. Saltzman. But you know, I once said, just because we 
say this thing is going to happen anyway does not allow us to 
do another thing first.
    Senator Harkin. Well, again I think I can understand if you 
were opposed to in vitro fertilization, then I can see it, but 
if you are for it, then you have got to say, okay, what do you 
do with these left-over--and if you have strict guidelines that 
separate it out, you cannot pay for them, you cannot generate 
it for that purpose, it seems to me that--to me, anyway, that 
the morally and ethically responsible thing would be to say how 
can--if there is a promise, in terms of research under ethical 
guidelines, to use them to alleviate human suffering, that is 
where I come down. I come down on that side of it, I guess.
    Mr. Saltzman. You are familiar with the old argument, does 
good actually derive from evil means? It does not. I believe 
that human embryonic research, the production and use of human 
embryos for those purposes----
    Senator Harkin. But we are not doing that. These are in 
excess of in vitro fertilization. I keep saying that we are not 
producing them for this purpose. The guidelines are very, very 
specific on that.
    Well, obviously we can go on and discuss this for a long 
time.
    Mr. Saltzman. Have you ever read Brave New World, by Aldous 
Huxley?
    Senator Harkin. Sure, when I was in college, and I read it 
once again later on.
    Mr. Saltzman. Read it again--three times. He wrote it in 
1933. He first saw genetic cloning in what he called decanted 
babies.
    Senator Harkin. Yes.
    Mr. Saltzman. What he worried about then is what we need to 
worry about today. He foresaw a time, as with the Sabbath, when 
science was made for man and instead we have come to a day when 
man is being made for science, and somehow things have been 
twisted all around.
    Senator Harkin. I do not agree with that. I mean, I do not 
think that is where we are at this point. I happen to think 
that our scientists are working to try to find interventions 
and cures for diseases and illnesses that affect humankind. 
There may be other new illnesses and diseases that arise in the 
future that we do not even know about, and we are going to 
continually need to do the research necessary to try to 
intervene on those.
    I guess I am not a fatalist. I do not believe in, I guess--
maybe because of my religious background, I do not believe in 
predestination. I do not believe in--I am not a fatalist. I do 
not believe that just because a person has had an accident, or 
because they were born a certain way, or have an illness, that 
that is how it must be forever and ever. If there are medical 
ways of intervening to relieve suffering or to make a life more 
whole, I think that is what we ought to be about.
    I think that is the humane, moral approach that we ought to 
be about as a society. It is not demeaning human life. I think 
it is enhancing human life and making human life better. Of 
course we are all going to die, but I do not think that it is a 
necessary lot in life that people have to suffer from 
debilitating diseases. If that is so, we never would have cured 
polio. If that is so, we would not have called smallpox, or the 
other things that we have overcome. Now we are looking at other 
things that we can overcome.
    Mr. Saltzman. No one seated here is opposed to research. 
All we are saying is there are less ethically objectionable 
methods to do the same kind of research.
    Senator Harkin. Well, I do not know. I mean, obviously I 
think--I agree we ought to do the adult stem cell research, as 
Dr. Hynes and Dr. Prockop said before, but that does not mean 
we should do that and not do the other ones. I think we ought 
to advance on all these fronts.
    But I am taking a lot of time to engage in a polemic 
exercise here, and I apologize to you for it, but it is an 
important aspect, and it is something that we wrestle with all 
the time.
    Mr. Saltzman. I have enjoyed the exchange. Thank you.
    Senator Specter. Thank you very much, Senator Harkin. I 
think it is very useful to spend extra time on those who are 
opposed to stem cell research, we have a complete record and 
give every opportunity to state your position, because we put 
on the record that we have introduced legislation in favor of 
research, but in so doing we want to be sure that these 
hearings are conducted in a very fair and evenhanded way to 
give you a full opportunity to express all of the opposing 
views.
    Senator Harkin. I am sorry, I have one other thing if you 
do not mind, Mr. Chairman. I was so involved with Dr. Saltzman.
    Mr. Heagy said something that I wanted to respond to. If 
you know anything about my background, you know I worked for a 
long time in the area of disability rights, American 
Disabilities Act, which I was the chief sponsor of. You made a 
mention of something about, does this mean that we are going to 
back off on support for community services and things for 
people with disabilities. I just want to answer that, and the 
answer is absolutely not.
    In fact, Senator Specter and I are both sponsor and 
cosponsor of the MCASSA bill--I can never remember what it 
means--Medicaid Community Attendance Services and Supports Act, 
which reforms medicaid to enable and empower people with 
disabilities to decide exactly how they want their support 
services delivered, in a community setting, at home, with 
personal attendance services, or in an institutional setting.
    Mr. Heagy. In my State I--yes. For 18 years of my life I 
received in-home support services so that I did not have to be 
in a nursing home, that I could go out and try to make a life 
for myself, but it has been recently that we have implemented 
an initiative for disabled folks to go to work and continue to 
get those benefits, because I require 24-hour care, which I 
would have to make $100,000 a year just to be able to afford my 
caretakers.
    Senator Harkin. I just wanted to respond that we are not 
backing off of that.
    Mr. Heagy. I appreciate that, but there is a whole lot that 
we needed to do to get here. There was hardly any 
transportation. I mean, I had to roll from the hotel to here, 
and the fact that these services are not accessible, it is just 
difficult. There are issues in life that we need to address and 
spend more time, and the money that we are spending.
    And I just wanted to ask concerning, real quick, the frozen 
embryos are potential human life right now. If you farm them 
out and let them die, then they are dead, correct, and 
prisoners, and like--what we were discussing, yes, they have a 
voice right now, but once they are executed they are dead, so 
why can we not take their parts. It is the same thought process 
in my mind, and do we seek permission from the folks that gave 
up those 100,000 frozen embryos to see if they morally agree 
with this? You are talking about the future, but what about 
where those came from? Are those donors okay with this?
    Senator Specter. Mr. Heagy, I am advised that consent has 
been obtained from the donors, that we have some 100,000 unused 
embryos, and the NIH guideline requires informed consent from 
the donors, including the statement that embryos or fetal 
tissue will be used to derive stem cells, so that that has been 
obtained.
    We have been joined by Senator Reid, who is a member of the 
subcommittee. Senator Wellstone has rejoined us, but let us 
turn to Senator Reid now, and Senator Wellstone, we will come 
back to you if you wish to ask questions.
    Senator Reid. Mr. Chairman, thank you very much. I have to 
be at the Senate when it opens, so I thank you very much for 
allowing me to say just a few words. I first of all am 
continuing to be the biggest cheerleaders that you and Senator 
Harkin have. I greatly appreciate the work that you have done 
in this area. This hearing is also part of the ongoing work 
that you are doing, and I cannot give you enough accolades for 
the work that you are doing and have done.
    Let me just say, briefly, that I come at this hearing and 
the work that you have done in this area not because of 
statistics and philosophy, but because people that I know that 
I have hope that work that we are doing here and work being 
done in the scientific community will help them.
    Steve Ragazio is a young man who has Lou Gehrig's disease. 
He is the president of the largest utility we have in Nevada, 
and every day he gets up his condition is worse. I hope that 
the work that is being done is going to help Steve, that he can 
play with his children and do things that he could do a few 
months ago. He cannot do it now.
    I hope that Molly Singer, who is a little 11-year-old girl 
that I have known since she was a baby, who is a twin, we can 
do something about her diabetic condition as a result of the 
work that is being done because of the emphasis that you, 
Senators Specter and Harkin, have focused on this issue, and 
the money that has come from this subcommittee to allow 
research to continue.
    So we have made progress. Again, I am not here to 
philosophize. I am here to help Molly and Steve get well, and I 
hope that we can do that.
    Senator Harkin. Thank you very much.
    Senator Reid. I will ask permission of the committee to be 
excused to go to the Senate. We are opening at 11 a.m.
    Senator Specter. Thank you very much for joining us, 
Senator Reid, and thank you very much for those very 
complimentary----
    Senator Reid. The one thing I did want to say, also I want 
to thank Michael J. Fox, Jennifer Estess, and Mary Tyler Moore. 
It is wonderful that they are willing to put their celebrity 
status on the line to help us with this project.
    Senator Specter. I agree with you, Senator Reid.
    Senator Wellstone.
    Senator Wellstone. Fine, Mr. Chairman. I thank the panel. I 
came in at the very end, so I do not have the context. I am 
just here right now. Thank you.
    Senator Specter. We are going to take a very brief recess. 
Thank you very much for coming in, Mr. Heagy, Pastor Saltzman, 
and Dr. Usala. Your testimony is very helpful in putting on the 
record the articulated views in opposition to stem cell 
research, which have been very cogently stated, so thank you.
    The hearing will now resume, and we call Ms. Gina Gershon, 
Ms. Jennifer Estess, Ms. Mary Tyler Moore, Mr. Michael J. Fox. 
We very much appreciate your coming in, and to comment briefly 
on a statement I made at the outset of the hearing, when people 
understand the impact of these illnesses, when Americans 
understand the impact of these illnesses on people they know, 
there tends to be a greater public awareness and more public 
support, so after hearing from the scientists, and after 
hearing from people who were in opposition on a variety of 
grounds to stem cell research, we do thank you for joining us.
    We have Jennifer Estess who is coming in, but in the 
interim we will proceed with the testimony of Ms. Gina Gershon, 
who has enjoyed a wide-ranging career in film, television, and 
on the stage. She most recently was seen in the Academy Award-
nominated film, The Insider with Al Pacino, and for the past 20 
years Ms. Gershon has been a close friend to Ms. Estess.
    Ms. Gershon met Ms. Estess while studying for her bachelor 
of arts degree in New York University. We are going to be 
joined here by Ms. Estess momentarily. Let us wait just a 
moment or two. She is coming through the main door because of 
the logistics of the situation.
    While Ms. Jennifer Estess is coming in, and we thank her 
for joining us again today, she is president of the project, 
amyotrophic lateral sclerosis, which she founded in 1998 after 
being diagnosed with the disease in 1997 at the age of 35. 
Since that time, she has been working with the corporate 
entertainment communities to help raise funds to find a cure 
for ALS, also known as Lou Gehrig's disease. She was the 
producing director of the off-stage Broadway theater company 
Naked Angels, and is currently the executive producer of CBS 
movie based on her life, scheduled to air early next year.
    This is Ms. Estess' second appearance before the 
subcommittee, having testified last time with Mr. Christopher 
Reeves back in April 26 of this year.
    Ms. Gershon, we turn to you. We have lights, with the green 
one indicating 5 minutes, the yellow 1 minute, and the red, 
stop, so you may proceed.

STATEMENT OF GINA GERSHON, ACTRESS
    Ms. Gershon. Honored chairman, distinguished members of the 
committee, I want to first thank you for giving me the 
opportunity to speak to you today on behalf of my friend, 
Jennifer Estess, and her work with Project ALS.
    Jennifer and I met as freshmen in college, and we both 
spent our twenties in New York City planning our futures and 
building a theater company together. In a group of passionately 
independent young people, it was always inevitably Jennifer who 
took it upon herself to focus our work, who, despite our 
energetic and often conflicting ambitions always managed to 
keep the bigger picture in sight for us.
    So it was no surprise to me, or any of us who knew her, 
that when she was diagnosed with ALS, when she was told that 
she had, at most, 5 years to live, Jennifer instinctively 
turned her attention from her own condition to the larger goal. 
She was scared. We were all scared, but once again she asked us 
to look beyond ourselves to help her find a cure.
    That search led her and her sisters to the stem cell 
research being conducted independently at some of the Nation's 
leading research institutions. She brought these doctors 
together, encouraged them to share their research, and asked 
how she could help. When they said they needed more money, she 
and her sisters and friends created Project ALS and began 
raising money, over $4 million so far, almost all of which has 
gone directly to equipment and research, and that research has 
already produced amazing results.
    As you know, ALS is a singularly destructive disease, 
striking randomly, methodically paralyzing its victims muscle 
by muscle until they can no longer breathe. It now seems 
possible that embryonic stem cells have the potential to stop 
or even reverse that process. We do not know for certain, but 
to not find out, to not get every possibility of finding out, 
seems like an agonizingly cruel decision.
    While there are some people here today who feel that some 
diseases are better than the cure, I am positive that many 
people would feel that a cure is much preferable to having the 
disease. People should be allowed to have that choice.
    Mr. Chairman, members of the committee, I cannot pretend to 
be an expert on this subject. It is complex and controversial. 
I know that. My friend here is dying, and I have to do 
everything I can to help her, and to help her find a cure, if 
not in her lifetime, perhaps in our own.
    Thank you.
    Senator Specter. Well, thank you very much, Ms. Gershon. 
Amyotrophic lateral sclerosis is certainly a dreaded disease. 
It came into very sharp public view with Lou Gehrig, who was 
stricken with amyotrophic lateral sclerosis and disabled, and 
died shortly thereafter.
    Ms. Estess, we appreciate your being with us again, and we 
look forward to your testimony.

STATEMENT OF JENNIFER ESTESS, ACTOR/PRODUCER
    Ms. Estess. Thank you so much. I also wanted to thank Gina, 
and I feel honored to be on the panel with Michael and Mary, 
and good afternoon to Chairman Specter and the members of this 
committee. I want to thank you for inviting me to come back to 
speak to you again.
    My name is Jennifer Estess, and I have ALS, a disease which 
is always fatal. 5 months ago I came here to tell you about my 
story, my work, and to ask for your help. Since that time, 
while we wait, I have come to rely on this ventilator. That is 
because ALS is destroying the muscles I use to breathe.
    Three years ago I was a healthy woman who worked hard and 
who dreamed of starting a family. Today, I cannot walk, brush 
my own hair, or hold my nieces or nephews. But although my body 
has deteriorated, I am proud to report that stem cell research 
funded by Project ALS has progressed with stunning results.
    When I was first diagnosed, I turned to the medical 
community for help. They told me that I was beyond the reach of 
science and medicine. There was not one drug to slow or stop my 
disease. A cure was unimaginable. But we now know about stem 
cells and their amazing medical potential.
    We know that they instinctively migrate to the area of 
injury in the brain and spine. We know that they transform into 
healthy new cells. We know that they may eventually repair the 
damaged nervous system. Every indication from our distinguished 
team of doctors is that there will be a treatment, even a cure 
for ALS, that stem cell therapy will work on humans, but we 
need your help.
    We need your funding. We need your funding to capitalize on 
the tremendous success that Project ALS and other organizations 
have realized in the private sector. We need Government control 
so that no one will abuse stem cell research, and we need the 
Federal Government to ensure that this life-saving work is 
carried out in the safest, most responsible way.
    Since we last met, I have taken what would have previously 
been a vacation to the beaches of Southern California. As you 
might imagine, it was quite a trick for me to get to the shore. 
It took literally a team of people, my sisters, my friends, my 
health aides, to carry me to the water's edge. I lay on a beach 
chair as they lifted me up, and we all moved as a group into 
the water, and it struck me that the only way we will ever beat 
ALS is to work together like this to further the promise of 
stem cells, because we are running out of time.
    Millions of Americans with ALS, Parkinson's, Alzheimer's, 
are running out of time. The children who love them are running 
out of time.
    Mr. Chairman, I know that good science makes no promises 
and I am under no illusion that the potential rewards of this 
research will save my life, but while I am still able to 
breathe, I cannot sit silently as the hope for millions of 
other Americans stalls or stagnates or dies, and so although I 
am still unaccustomed to asking for help, I am urging you today 
to pass the Stem Cell Research Act of 2000.
    On behalf of Project ALS and the millions of Americans 
living and dying with ALS, Parkinson's, Alzheimer's, and 
childhood brain diseases, I implore you to move this 
legislation forward with a renewed sense of urgency, to let 
this opportunity not slip away, to let us save our friends, our 
families, and ourselves now, because we can.
    Thank you very much.
    Senator Specter. Thank you very much, Ms. Estess. We 
appreciate you coming back today. We know it is very difficult 
for you to do that, and since you were here in April our 
subcommittee came forward with a recommendation of $2.7 billion 
of additional NIH funding. That is the largest amount it 
increased. The year before we recommended $2.3 billion, the 
year before $2 billion, and the year before that, a billion, 
which are vastly increased sums over what had been done.
    So within the last four appropriations cycles we have added 
some $8 billion to NIH funding on top of $12 billion, so you 
can see that kind of an increase, we are determined to double 
the research funding, and when the stem cell issue was 
disclosed in November of 1998, Senator Harkin and I introduced 
legislation so that we could use the Federal funding to extract 
stem cells from embryos. There has been a ruling that Federal 
funding may be used on the stem cells once they are extracted, 
but it is very important not to have the limitation on the 
Federal funding to extract the stem cells, so we are moving 
forward and anticipate a vote on it in the Senate yet this 
month.
    So we appreciate your coming in and providing this 
testimony, and we not only hear you, but we have acted on it.
    Ms. Estess. Thank you, sir.
    Senator Specter. Our next witness is the distinguished star 
of radio, stage and screen, Ms. Mary Tyler Moore, who has been 
an active advocate for more than a decade, to my personal 
knowledge, about the time Senator Harkin was made a 
subcommittee member, honorary subcommittee member at least, 
probably best known for her television roles in the Dick Van 
Dyke Show and the Mary Tyler Moore Show, received an Emmy in 
1992 for her role in Stolen Babies, and was nominated for an 
Oscar in Ordinary People. Broadway honored Ms. Moore with a 
special Tony for Whose Life Is It Anyway.
    She has lived with diabetes for over 30 years, and has 
worked to raise public and congressional awareness for this 
disease. For the last 16 years she has served as the 
international chairman of the Juvenile Diabetes Foundation, and 
last week I saw her leading an entourage on the first floor of 
the Hart Senate Office Building on her lobbying ways, and--did 
she come to see you then, Tom? Me neither.
    Ms. Moore, you have the floor.

STATEMENT OF MARY TYLER MOORE, INTERNATIONAL CHAIRMAN, 
            JUVENILE DIABETES FOUNDATION
    Ms. Moore. Thank you, Mr. Chairman. Senator Harkin, Mr. 
Chairman, members of the subcommittee, I thank you for the 
opportunity today to discuss stem cell research, an issue that 
could have enormous impact in the fight for a cure for 
diabetes.
    I am here today as the international chairman of the 
Juvenile Diabetes Foundation, JDF, an organization whose 
mission is to find a cure for diabetes and its complications, a 
disease that affects a total of 16 million Americans, and costs 
more than $100 billion per year.
    Since its founding in 1970, JDF has grown to become the 
largest private not-for-profit supporter of diabetes research 
in the world. This year, JDF will fund up to $120 million worth 
of diabetes research, with all of this funding coming from 
privately raised resources.
    I want to thank the subcommittee for its extraordinarily 
strong support for Federal funding for the National Institutes 
of Health. I vividly recall several years ago my joining many 
of you in a kick-off press conference for the bipartisan effort 
to double the budget of the NIH, and that would be over 5 
years. Well, I am so appreciative of that work, and been so 
glad to see that you have brought us to the almost end of that 
5-year period.
    However, despite the increases in research funding, our 
mission to find a cure for diabetes may not happen unless 
scientists are free to use funds to explore some of the most 
promising avenues leading to a cure. Stem cell research is an 
integral part of finding a cure for this disease.
    Many of you know that I have had juvenile diabetes for more 
than 30 years. The disease alone is always difficult to live 
with. Trying to balance blood sugar levels and insulin 
injections is hard enough, but adding to that the severe 
complications of diabetes makes things frightening. I know too 
well the fears that arise from this part of the disease, and I 
know as well as anyone that insulin is not a cure.
    In many ways I have been more fortunate than others who 
have diabetes. While I have almost faced blindness due to the 
diabetic retinopathy, I am still able to see. While I have 
faced the possibility of amputation, I am still able to walk. 
But things could have turned the other way, as they have for 
millions of Americans, and the future of any person with 
diabetes is always uncertain.
    But Mr. Chairman, there is evidence of a cure that is 
within our grasp. Earlier this year, in a study at the 
University of Alberta, Canada, 12 individuals with juvenile 
diabetes received a successful transplantation of insulin-
producing cells, and they no longer require insulin.
    The transplants involve a minimally invasive injection 
procedure which does not involve surgery. The cells are placed 
into the portal vein. They then migrate to the liver, where, 
even though they are not in the pancreas, they take root and 
produce sufficient insulin, and almost perfect control of blood 
sugar.
    However, one of the major drawbacks of this study is that 
two cadaver pancreases are needed for each transplantation, and 
fewer than 2,000 are available each year. With millions of 
Americans having diabetes, including nearly 2 million with 
juvenile diabetes, you can see that even if this procedure is 
perfected, as we hope it will be, there are not nearly enough 
organs to cure everybody.
    Stem cell research offers a tremendous amount of hope for 
this research. Scientists believe that one day stem cells will 
develop into any human tissue or organ. When scientists are 
able to specialize these cells to become insulin-producing 
islet cells, lines could be developed to produce an unlimited 
number of insulin-producing cells. This would effectively solve 
the supply issue.
    Well, I do understand that some people have heartfelt 
concerns about this research. I believe that, given the 
safeguards developed within the National Institutes of Health, 
and considering that the embryos and fetal tissue used in this 
life-saving science would be destroyed, I believe that stem 
cell research should be pursued vigorously and is appropriate 
for Federal funding.

                           PREPARED STATEMENT

    Mr. Chairman, every year thousands upon thousands of 
children in this country are diagnosed with juvenile diabetes, 
and one American dies from diabetes every 3 minutes. We owe it 
to these children and the 16 million Americans with the disease 
to pursue all promising research avenues, including stem cell 
research, within the ethical framework established by the 
Federal Government.
    I thank you for hearing this plea, and I will to the best 
of my ability answer any questions that you might have.
    [The statement follows:]

                 Prepared Statement of Mary Tyler Moore
    Mr. Chairman, Senator Harkin, and members of the Subcommittee, 
thank you for the opportunity today to discuss stem cell research, an 
issue that could have an enormous impact in the fight to find a cure 
for diabetes.
    I am here today as International Chairman of the Juvenile Diabetes 
Foundation (JDF), an organization whose mission is to find a cure for 
diabetes and its complications, a disease that affects a total of 16 
million Americans and costs more than $100 billion per year. Since its 
founding in 1970, JDF has grown to become the largest private, non-
profit supporter of diabetes research in the world. This year, JDF will 
fund up to $120 million worth of diabetes research, with all of this 
funding coming from privately raised resources.
    And I want to thank the Subcommittee for its extraordinarily strong 
support for federal funding for the National Institutes of Health. I 
vividly recall several years ago joining many of you in a kick off 
press conference for the bipartisan effort to double the budget of the 
NIH over five years. I am so appreciative of the work that you have 
done so far to get us almost through year three of this effort.
    However, despite these increases in research funding, our mission 
to find a cure for diabetes may not happen unless scientists are free 
to use federal funds to explore some of the most promising avenues 
toward a cure. And stem cell research is an integral part of finding a 
cure for the disease.
    Many of you know that I have had juvenile diabetes for more than 
thirty years. The disease alone is always difficult to live with--
trying to balance blood sugar levels and insulin injections is hard 
enough--but adding to that the severe complications of diabetes makes 
things much scarier. I know too well the fears that arise from this 
part of the disease. And, I know as well as anyone that insulin is not 
a cure.
    In many ways I have been more fortunate than others who have 
diabetes. While I have almost faced blindness from diabetic 
retinopathy, I am still able to see. While I have faced the possibility 
of amputation, I am still able to walk. But, things could have turned 
out differently for me as they have for millions of Americans, and the 
future of any person with diabetes is always uncertain.
    However, Mr. Chairman, there is hope for a cure. In fact, earlier 
this year, in a study at the University of Alberta, Canada, seven 
individuals with juvenile diabetes received a successful 
transplantation of insulin producing cells and no longer require 
injections of insulin.
    The transplants involve a minimally invasive injection procedure 
which does not require surgery. The cells are placed into the liver 
through the portal vein. The cells then migrate to the liver where, 
even though they are not in the pancreas, take root and produce 
sufficient insulin and almost perfect control of blood sugar. However, 
one of the major drawbacks of this study is that two cadaver pancreata 
are needed for each transplantation, and less than 2,000 are available 
each year. With millions of Americans with diabetes, including nearly 
two million with juvenile diabetes, you can see that even if this 
procedure is perfected--as we hope it will--there are not nearly enough 
organs to cure everyone.
    However, stem cell research offers a tremendous amount of hope for 
this research. Scientists believe that one day stem cells will develop 
into any human tissue or organ. When scientists are able to specialize 
these cells to become insulin-producing islet cells, cell lines could 
be developed to produce an unlimited number of insulin-producing cells. 
This would effectively solve the supply problem.
    While I do understand that some people have heartfelt concerns 
about this research, I believe that given the safeguards developed 
within the National Institutes of Health's recently released 
guidelines, and considering that the embryos and fetal tissue used in 
this research would be destroyed if not donated to this life-saving 
science, I believe that stem cell research should be pursued vigorously 
and is appropriate for federal funding.
    Mr. Chairman, every day, 35 children in this country are diagnosed 
with juvenile diabetes, and one American dies from diabetes every three 
minutes. We owe it to these children and the 16 million Americans with 
the disease to pursue all promising research avenues, including stem 
cell research, within the ethical framework established by the federal 
government.
    I would be happy to answer any questions, and I would like to 
introduce Dr. Marc Hurlbert from the Juvenile Diabetes Foundation, if 
you have any scientific questions about this research.

    Senator Specter. Thank you very much, Ms. Moore, for your 
very poignant and moving testimony. When you talk about what 
could happen to you with diabetes, blindness or amputation, 
that is really laying it on the line, and when you talk about 
somebody dying every 3 minutes, 2 people have died since we 
started your introduction, and that is why there are many of us 
who are so determined to make use of embryos which will be 
discarded anyway----
    Ms. Moore. Exactly.
    Senator Specter [continuing]. To save lives and stop human 
suffering.
    We now turn to Mr. Michael J. Fox, who has had a 
spectacular career, first as Alex B. Keaton on the television 
series, Family Ties, later in a number of movies, including 
Back to the Future, and most recently on television again in 
the highly acclaimed Spin City. This past week, Mr. Fox won an 
Emmy award for best actor in a comedy series for his work in 
Spin City.
    He was diagnosed with Parkinson's in 1991 at the age of 30, 
and since announcing his retirement from Spin City, he has been 
devoting his time to the Michael J. Fox Foundation for 
Parkinson's Research, and I might comment that he even came to 
the Republican convention in Philadelphia.
    Mr. Fox. I was in Los Angeles as well, sir.
    Senator Specter. Well, when you--Senator Harkin mentions 
that you hit them both. I was not at the other one. What was 
the other one, did you say?
    Mr. Fox. As you said, sir, it is a nonpartisan problem that 
will take a bipartisan solution.
    Senator Specter. Well, we are bipartisan here, as the 
Democrat and Republican representation on the podium suggests, 
but here again, spectacular work, and from time to time 
questions are raised about celebrities appearing at 
congressional hearings.
    In fact, it is even more than questions. There is some 
pretty severe criticism about it, but we make no apologies, 
because we need public awareness of issues like stem cells, 
which could cure Parkinson's, or amyotrophic lateral sclerosis, 
or Alzheimer's, and when public attention is focused on Michael 
J. Fox because the American people know you, Michael, it is a 
great use of your talent and celebrity status to let people 
know what is going on and get public support for this kind of a 
measure.
    So we thank your for your time and your efforts, and we 
know how debilitating the illness is, and we are just very 
hopeful that soon--we had the testimony from the scientists 
this morning--in 2 or 3 years there will be ready for use with 
people, and that you will be back on ABC TV--I do not want to 
say that in derogation of the other networks, which are here, 
but back--well, you choose where you go back. We just want you 
back.
    Mr. Fox. I will be open to all offers.
    Senator Specter. The floor is yours, Mr. Fox.

STATEMENT OF MICHAEL J. FOX, FOUNDATION FOR PARKINSON'S 
            RESEARCH
    Mr. Fox. Thank you, Mr. Chairman and Senator Harkin. Thank 
you for inviting me to testify, and good morning.
    It is hard to believe that--I should say thank you to my 
fellow panelists. I am honored to be in your company, and let 
us hope it is for the good.
    It is hard to believe an entire year has passed since I 
first appeared before the subcommittee and addressed the need 
to increase Federal funding for Parkinson's research. I am 
grateful for that opportunity to speak on behalf of the 
Parkinson's community about the reality of living with this 
disease.
    It was during a hearing in September 1999 that Senators 
provided exciting testimony as to just how close we may be to a 
cure for Parkinson's. In pursuit of that cure, I am back this 
year to lend my voice and that of the Michael J. Fox Foundation 
for Parkinson's Research to support Federal funding for 
pluripotent stem cell research, including that research covered 
by the NIH guidelines announced on August 23.
    I do not intend to become a professional witness. I am not 
a politician, nor I am a doctor, nor a research scientist. You 
do not need me to explain embryonic stem cell research or its 
medical applications, so what does qualify me to be at this 
table?
    The answer is simple. I am one of a million involuntary 
experts on Parkinson's disease in the United States battling 
its destructive nature as we wait for a cure. We need a rescue, 
and the country should know it.
    I am also here because I am a guy with PD who happens to be 
on TV. Because of that, many people have felt comfortable 
reaching out to me. By now, many of you have heard my story, 
but you have not heard this story, about a 38-year-old senior 
editor whose PD caused her to lose her job at a publishing 
house, plunging her from New York's middle class into poverty. 
She is now forced to exist on medicare and SSDI benefits, which 
are nearly consumed by her monthly medication costs alone.
    Nor have you heard about my new friend, a former lawyer, 
now living on disability, who corresponds with me regularly. 
Two weeks ago his friends and family watched in horror as he 
disappeared into stony immobility while waiting for a 
prescription delivery that had been delayed. This demonstrated 
dramatically just how tenuous normalcy is.
    And you have never heard about Brenda, a 53-year-old former 
computer specialist. Recently her drugs failed to kick in, and 
she found herself frozen in the bathtub with no one to help 
her. She remained there for hours until enough medication had 
reached her brain to allow her to crawl out of the tub. By this 
time she was suffering a panic attack and could not speak. She 
could only reach her computer to contact friends for help. Her 
biggest regret, she says now, was that CNN was not there to 
provide live, up-to-the-minute coverage of her predicament.
    None of these people mind that I get more attention than 
they do. They simply say that if I get a shot at this 
microphone, that I start talking, so here I am again.
    For 2 years, you have had a parade of witnesses, 
scientists, ethicists, theologians of every school, and some 
celebrities, discussing every nuance of stem cell research. You 
have given time to all sides of the issue, including a few very 
vocal opponents, but the consistent and inescapable conclusion 
is that this research offers the potential to eliminate 
diseases, literally save millions of lives.
    So while I applaud your fairness, I cannot help but say 
respectfully, enough. It is time to act on what we have 
learned. Sadly, we have lost 2 years' progress towards a cure. 
Further delay will come at a high price. That is why I am back 
before this committee today. Every day funding is delayed means 
that a person with Parkinson's is getting closer to total loss 
of independence, or slipping slowly toward the progressive 
inevitability of this disease. These delays have real human 
consequences that are measured in human suffering and loss of 
life.
    The pioneers in stem cell research, Drs. Gerhardt, 
Thompson, and West, told this committee in December 1998 that 
Parkinson's would be one of the first diseases to benefit from 
the use of stem cells. Still researchers have testified that it 
will take at least 3 years from the time they received funding 
to the development of the first stem cell therapies for 
Parkinson's.
    Even at the fastest possible pace under the newly released 
NIH guidelines, the first scientists to receive Federal funding 
will not begin working until late next year, which all means 
that we are still years away from our rescue. Please, help us 
to not wait any longer than we have to.
    I am not here solely to represent the benefits of stem cell 
research for Parkinson's patients. There are many other 
promising applications, from heart disease, to blindness, to 
Alzheimer's, to burn victims, to cancer, to HIV/AIDS, to 
stroke, to autism, to deafness, to schizophrenia, to diabetes, 
to MS and ALS.
    Stem cell research could also help those with spinal cord 
injuries. My friend Christopher Reeve sends his regrets that he 
could not be here today to emphasize the urgency that we both 
feel. His testimony has been submitted for the record, but I 
would like to share a few of his words with you now.
    Since its inception, a fundamental principle of our 
Government has been to respond to the needs of people. Now, a 
major scientific breakthrough has given us the opportunity to 
uphold another principle of our Government, to do the greatest 
good for the greatest number of people. I am referring, of 
course, to the recent discovery of the miraculous discovery of 
stem cells.
    Now, those of us who are privileged to testify before our 
representatives, though suffering individually from specific 
conditions, have the unique opportunity to speak on behalf of 
an entire population, both at home and in every corner of the 
global village, who suffer from almost every conceivable form 
of debilitation.
    Thank you, Christopher.
    The NIH is ready. Extensive and meticulous guidelines for 
stem cell research have been written and approved. We urge you 
to not let politics interfere and needlessly delay this 
critical research. For 2 years, I have been watching this 
debate. To me, this is not theoretical. There are real 
consequences here. As I said earlier, I do not profess to be an 
ethicist, but I do consider myself a good and decent man, a 
loving father and husband. I would not claim any benefit that I 
believe was made possible by harm done to another person. That 
is not the reality here.
    I am not a political scientist, either, but I have an 
immigrant's love for my country, and I am humbled to 
participate in this process. I see a need for our politicians 
to act on our behalf, and for them not to politicize a 
wonderful medical advance.
    So that is what I ask of the people who are touched by this 
issue. Those afflicted and affected, every patient, as well as 
every mother, father, brother, sister, grandchild, uncle, aunt, 
teacher, coach, friend, or neighbor of a person with 
Parkinson's, in addition to the even wider circle of those with 
related illnesses and their families and loved ones, which 
ultimately include every person in this country, you all have a 
stake in the outcome, and I ask those of you who are watching 
today to join me in the conversation, study the record, visit 
the NIH web site, add your voice. These good men and women are 
your representatives. Ask them to tell you where they stand.
    I am confident that the vast majority of you would want 
this research funded, and quickly. I see in these cells a 
chance for a medical miracle. The Government has done its work. 
We ask you now to release our tax dollars so the scientists can 
do theirs.
    I thank you.
    Senator Specter. Thank you very much, Mr. Fox, for that 
very compelling testimony.
    Mr. Fox. That red light is very----
    Senator Specter. The red light was on, but it has been on 
on other occasions for longer periods of time with people not 
stopping talking, and it is OK. It is a parameter and a 
guideline. It is not absolute.
    You made a statement, Mr. Fox, saying, quote, you would not 
claim any benefit that did any harm to another person, and that 
leads me to the critical question on this issue, which will be 
up for a vote, and the Congress, the Senate, decides questions 
for public policy, and we have to make decisions weighing the 
arguments on one side, and weighing the arguments on the other 
side, and the argument on one side in opposition to Federal 
funding for taking stem cells from embryos is that we are 
dealing with a human entity, which is alive.
    At the same time, it is acknowledged that the 100,000 stem 
cells which are now being discarded with the consent of the 
donors will not be used for any purpose, but will be discarded, 
and any semblance of life will be ended.
    On the other side, those argue that with 125 million 
Americans being affected by the long list of diseases, 
thousands with Parkinson's, thousands with ALS, what would your 
message be to the United States Senate as to how you balance 
the contention of destroying an embryo, which is living but 
soon to be discarded, with the benefit to people who suffer 
from Parkinson's, as you do.
    Mr. Fox. Well, I think obviously that aspect of it is part 
of a grave debate, which there are passionate opinions and 
feelings on either side, and it is a debate that in a sense 
does not relate specifically to this, but I will address it. As 
you said, those cells that are undifferentiated, pluripotent in 
the sense that they have not been assigned to be things, they 
do not know what they want to be, they are very early in 
development, they are pluripotent in the sense that they can be 
anything, are being destroyed.
    It is a separate argument, why they are being destroyed, 
whether they should be destroyed, and like I said, there are 
finer minds than mine that can debate that. The fact is, they 
are being destroyed, they are being wasted, and the potential 
to change the health of this country and this world is 
something that it just seems to me the loss of that in the face 
of this potential is great, and it is a chance to do good and 
to do well.
    Senator Specter. Thank you very much, Mr. Fox.
    Let me turn to you next, Ms. Estess, since you suffer from 
amyotrophic lateral sclerosis, and as you commented, and as is 
well-known, it is always fatal, a question of how long, medical 
research I have seen, 2 to 7 years. What would your message be 
to the Senators who will be saying aye or nay on the contrast 
between the contention of embryos being alive, but soon to be 
discarded, contrasted with what might be done to save your 
life?
    Ms. Estess. I think that the focus is not that, it is our 
responsibility as Americans to protect each other, and Michael 
said while he was speaking with you that every single person in 
this room is going to be touched by one of these illnesses, and 
I also think that often we do not want to look at things that 
are destroying lives, such as ALS and Parkinson's and diabetes 
that Mary spoke about.
    I think it is our responsibility as Americans to push this 
forward so we can get treatments to these people so they can be 
with their families and loved ones.
    Senator Specter. Ms. Moore, same question. It is the 
critical issue, the choice between the embryos, where the 
contention is made that they are living, although there is 
agreement they will be discarded, contrasted with healing and 
saving lives.
    Ms. Moore. The embryos that are being discussed, according 
to science, bears as much resemblance to a human being as a 
goldfish, I think makes the answer clear. We are dealing with 
flesh and blood people now who feel pain, feel fear, feel 
debilitation, and our obligation is to those who are here.
    Senator Specter. Very cogently and movingly stated.
    Ms. Gershon, we will give you the last word. It is a pretty 
hard act to follow.
    Ms. Gershon. Well, I think Mary just said it. I mean, that 
is perfect. All I know is, when I look at, you know, my friend 
and people that I know, suffering, to have the choice to help 
them or not help them, there should be no question.
    Senator Specter. Easy choice.
    Ms. Gershon. Easy choice. To me that is pro-life. Let these 
people live.
    Senator Specter. The pro-life position is to let Ms. Estess 
live.
    Ms. Gershon. Yes.
    Senator Specter. And others.
    Senator Harkin. And others. Thank you, Mr. Chairman. I just 
want to thank all of you for not only being here today, but for 
your continuing efforts to get the public more knowledgeable 
about what we are about.
    I could not help, Mary, when you were talking about 
resembling--I did this once before. I held up a piece of paper. 
Can you tell me what is on that piece of paper?
    Ms. Moore. You have got to be joking. No.
    Senator Harkin. There is a little, teeny little pencil dot 
that I put on there that you cannot even see. That is the size 
of the embryos we are talking about. I think a lot of people 
get confused, thinking an embryo is something, almost like a 
fetus or something like that, fully developed fetus. We are 
talking about something less than the size of a pencil dot----
    Ms. Moore. Yes.
    Senator Harkin [continuing]. That contains the cells, the 
undifferentiated cells that Michael Fox was talking about, so 
somehow to equate this with a fully developed human being I 
think is stretching credulity quite a bit.
    Anyway, I just thank you, because we need your help. I 
think the bill that Senator Specter has authored, I am 
cosponsor of, that we have the assurances of the Majority 
Leader that we will have a vote on it----
    Senator Specter. That is correct, this month.
    Senator Harkin [continuing]. Before we leave here this 
month. I do not know the outcome of that vote. I believe we 
have the votes. I hope we have the votes. But your being here 
today and your continued efforts in the public realm to just 
focus attention on this, to bring it out of the shadows and put 
the sunshine on it, does more to help us get this through than 
anything else. I am consciously optimistic that we can get it 
passed, and I am even more than consciously optimistic that in 
the next few years we are going to see tremendous breakthroughs 
using stem cells in addressing these illnesses and diseases, so 
again I just thank you very much for your efforts.
    Senator Specter. Thank you, Senator Harkin, and thank you, 
Ms. Gershon, Ms. Estess, Ms. Moore, and Mr. Fox.
    Mr. Fox. Thank you.

                         CONCLUSION OF HEARING

    Senator Specter. Thank you for being here. That concludes 
our hearing. The subcommittee will stand in recess subject to 
the call of the Chair.
    [Whereupon, at 11:43 a.m., Thursday, September 14, the 
hearing was concluded, and the subcommittee was recessed, to 
reconvene subject to the call of the Chair.]

                                   -