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[Senate Hearing 107-874]
[From the U.S. Government Publishing Office]

                                                        S. Hrg. 107-874
 
 STATUS OF THE IMPLEMENTATION OF THE FEDERAL STEM CELL RESEARCH POLICY
=======================================================================

                                HEARING

                                before a

                          SUBCOMMITTEE OF THE

            COMMITTEE ON APPROPRIATIONS UNITED STATES SENATE

                      ONE HUNDRED SEVENTH CONGRESS

                             SECOND SESSION

                               __________

                            SPECIAL HEARING

                   SEPTEMBER 25, 2002--WASHINGTON, DC

                               __________

         Printed for the use of the Committee on Appropriations

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

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                      COMMITTEE ON APPROPRIATIONS

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

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

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

                         Administrative Support
                             Carole Geagley

                            C O N T E N T S

                              ----------                              
                                                                   Page

Opening statement of Senator Arlen Specter.......................     1
Statement of Elias Zerhouni, M.D., Director, National Institutes 
  of Health, Department of Health and Human Services.............     2
    Prepared statement...........................................     5
Statement of Senator Deborah Ortiz, Sixth State District, 
  California State Senate........................................     8
Statement of Roger Pedersen, Ph.D., department of surgery, 
  Cambridge University...........................................    10
    Prepared statement...........................................    12
Opening statement of Senator Patty Murray........................    13
Statement of Gerald Schatten, Ph.D., professor of cell biology, 
  University of Pittsburgh, director, Pittsburgh Development 
  Center, and deputy director, Magee-Women's Research Institute..    14
    Prepared statement...........................................    16
Statement of Curt Civin, M.D., professor of cancer research, 
  Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins 
  University.....................................................    17
    Prepared statement...........................................    20
Statement of George Daley, M.D., Ph.D., assistant professor of 
  medicine, Harvard Medical School; and fellow, Whitehead 
  Institute for Biomedical Research..............................    22
    Prepared statement...........................................    24
Opening statement of Senator Kay Bailey Hutchison................    29
Prepared statement of Senator Larry Craig........................    32

 STATUS OF THE IMPLEMENTATION OF THE FEDERAL STEM CELL RESEARCH POLICY

                              ----------                              

                     WEDNESDAY, SEPTEMBER 25, 2002

                           U.S. Senate,    
    Subcommittee on Labor, Health and Human
     Services, and Education, and Related Agencies,
                               Committee on Appropriations,
                                                    Washington, DC.
    The subcommittee met at 9:30 a.m., in room SD-124, Dirksen 
Senate Office Building, Hon. Arlen Specter presiding.
    Present: Senators Murray, Specter, and Hutchison.

               opening statement of senator arlen specter

    Senator Specter. Good morning, ladies and gentlemen. The 
hour of 9:30 having arrived, we will proceed with the hearing 
of the Appropriations Subcommittee on Labor, Health and Human 
Services, and Education.
    Our focus today is to examine the status and implementation 
of the President's policy on stem cell research. Shortly after 
stem cells came upon the scene, the subcommittee held hearings 
in December of 1998, and this is our 14th hearing to follow up 
on this very, very important field of medical research.
    The unique opportunities for the use of stem cells have 
been recognized in a wide variety of ailments. It has been a 
controversial matter because the stem cells are extracted from 
embryos. While there are other types of stem cells, our 
hearings have disclosed that embryonic stem cells are the most 
useful, and the opposition has focused on the possibility of 
life being produced by the embryos. If each embryo could 
produce life, that would obviously be the highest calling, but 
we know that thousands are thrown away. So it is my view that 
it is obviously preferable to use these embryos to save lives 
as opposed to discarding them.
    The President on August 9 of last year established a policy 
of limiting stem cells to 63 or 67 lines, or somewhere in that 
range, and a big issue arises as to whether that is adequate to 
carry on the research.
    During the course of the past year, we have had 
considerable controversy over nuclear transplantation which 
some people call therapeutic cloning, which is not cloning at 
all. This is a matter which is surrounded by controversy, and I 
think we have to find our way through because at least my view 
from the 14 hearings we have held is that it poses an enormous 
opportunity to conquer disease.
    That is a relatively short opening statement to set the 
parameters.
    The majority leader has scheduled two votes at 10:30, which 
means that the hearing will have to be adjourned for up to 30 
minutes. I am going to do my best to move through the hearing 
and conclude by 10:40. I will be a little late to the first 
vote, but I think that is preferable than to have witnesses and 
observers wait a half an hour. I know how busy the people are 
who are at the hearing as witnesses and also as observers.
STATEMENT OF ELIAS ZERHOUNI, M.D., DIRECTOR, NATIONAL 
            INSTITUTES OF HEALTH, DEPARTMENT OF HEALTH 
            AND HUMAN SERVICES
    Senator Specter. Our first witness is Dr. Elias Zerhouni, 
Director of the National Institutes of Health. He comes to this 
position with a very extraordinary record. He was executive 
vice dean of Johns Hopkins University School of Medicine, Chair 
of the Russell H. Morgan Department of Radiology and 
Radiological Science, and Martin Donner Professor of Radiology 
and Professor of Biomedical Engineering. Dr. Zerhouni received 
his medical degree from the University of Algiers School of 
Medicine, and he had his residency in diagnostic radiology at 
Johns Hopkins. He came to this country at the age of 24 and has 
had a really remarkable career.
    I have already had considerable contact with Dr. Zerhouni 
in his first 4 months on the job, and this is his first 
appearance before the subcommittee. We welcome you here, Dr. 
Zerhouni, and look forward to your testimony.
    Dr. Zerhouni. Thank you, Senator Specter. I am really 
pleased to be here this morning and testify about the role of 
NIH in advancing the field of stem cell research. We all know 
that if properly harnessed, adult and embryonic stem cells have 
the potential to replace cells that are damaged or diseased to 
restore vital functions of the human body.
    There are ample reasons for excitement. I personally 
thought this was a field that needed to grow when I was at 
Johns Hopkins. There is no question that there is huge 
potential and promise, and high expectations for the new 
treatments that are possible with this approach are 
understandable.
    But we should temper these expectations by the enormous 
challenges that must be addressed before the research evolves 
into proven therapy. I think we are at a very early stage of 
research in embryonic stem cell research and have a great deal 
of basic research to conduct before we can unlock the potential 
of these cells and fulfill their promise.
    What I would like to do, to go over my presentation, is to 
use some charts to my right to go over what the basic strategy 
for research and research development will be in stem cell 
research, whether adult or embryonic. We can divide that 
strategy into three phases. There is the early phase called the 
basic research phase, and then two follow-on phases called 
preclinical and clinical phase.
    The most important aspect of the basic research phase is 
for us to build the scientific capability of the field by 
creating career development pathways, training courses. The 
most important resource for any new field is trained 
investigators who are entering the field and advancing the 
field. We need to establish an infrastructure with cell culture 
methods, cell lines, expand the cell lines, characterize the 
cell line. The puzzle has to come together then in terms of us 
being able to both prove the long-term stability of these 
cells. We need to characterize them fully, and we need to make 
sure they are genetically stable. We need to understand the 
basic reason why we are so excited about stem cells is that 
they can differentiate and specialize into different cells in 
the human body. We need to understand that better at the most 
fundamental level, how is that done, what are the methods that 
we need to develop to understand this at the gene level and the 
molecular level. We need to understand how the cell cycle of 
the stem cell is controlled. One of the major risks in stem 
cell research is that these cells, once implanted, might revert 
to their more undifferentiated state and could grow into 
tumors. We need to understand that.
    Last but not least, we have to have a lot of research go on 
in understanding the interactions between the cell and the host 
and the immunology and the transplantation biology of these 
cells.
    As we progress, other elements of research will have to 
come into place. And this year we have had a lot of progress 
made. We have shown that in fact embryonic stem cells can 
differentiate into nerve tissue and insulin-producing cells. 
Adult stem cells have been shown to also be able to 
differentiate. And I believe that we should continue both 
embryonic stem cells and adult stem cell research at the same 
pace as fast as we can to go through our understanding of the 
puzzle that will then lead us to the preclinical phase where we 
need to have proof of concept experiments. We need to use the 
technology in animal models of disease. We need to prove what 
cell dosing we need to use, make sure that our understanding of 
tumor formation is complete, assess whether or not the cell is 
really functioning as we want to make it function, and 
eventually then, once we have accumulated that body of 
knowledge, go to the clinical phases of research, which are 
typically divided into three phases: to test whether or not 
there is any toxicity, what is the safety of these stem cells, 
and what is the efficacy of these stem cells to eventually go 
into therapies that will serve the public.
    Now, I would like to also cover with you the work that NIH 
has done over the past year in trying to advance the field. 
There are two important elements that we as enhancers of the 
research, as the institution at NIH that should look to 
implement the research agenda--there are two resources that I 
consider the most critical right now.
    One is the availability of researchers. So what we have 
done is try to develop training capabilities for researchers 
across the Nation. We have tried to decrease the shortage of 
researchers with expertise in stem cell research. We have 
extended additional grants to people who have expertise in stem 
cell research but not necessarily in human stem cell research. 
And we will strive to make stem cell research as attractive as 
possible to our most talented research scientists. So we are 
soliciting grant applications and I will give you some of the 
data related to that in a minute.
    One of the most important stumbling blocks is to make human 
stem cells more available for research. As you know, on 
November 7, 2001, NIH published the registry of derived stem 
cells that would be eligible for Federal funding. The registry 
consists of 14 sources across the world. The cells are in 
various stages of characterization and preparation for research 
applications. There are many steps required to develop 
embryonic stem cells from when they are first removed from an 
embryo and put into culture into an established, well-
characterized embryonic stem cell line ready for distribution 
to the research community.
    I tried to summarize this process right here on my chart on 
the left side to show you what is the exact process that we 
need to go through to make cell lines widely available for 
distribution. After the derivation and the placement into 
culture of cells from the inner cell mass, we obtain primary 
colonies, which takes 3 to 5 days, the first thing we have to 
do is expand the primary colonies, then put them into 
subculture wells. Now, we need to have enough expansion of 
these cell lines to be able to then have enough of them to be 
available for distribution. The success rate here is not very 
high. Only 10 percent of these subcultures eventually go on to 
establish lines that can be characterized as human embryonic 
stem cell lines.
    There are about 30 to 60 passages then, that have to occur 
to expand the number of cells within each line. As the cells 
divide, the total number of cells available to us for research 
increases. But at each passage, we need to make sure that these 
cells have not differentiated, and we need to have biomarkers 
for that purpose. We need to have ways of making sure that 
these cells still have the total potential of embryonic stem 
cells. That is done 30 to 60 times, and the expansion of these 
cultures is essentially the basis for the distribution that 
eventually occurs. It takes about 6 to 9 months to get from 
this stage to a stage where you have expanded these subcultures 
successfully, you have characterized them successfully, and one 
bank requires about 2 billion cells. To start to distribute 
these cells to the general public, you need approximately 2 
million cells per vial to do so.
    So the process obviously takes a while, and NIH has been 
very aggressive at, in fact, facilitating the availability of 
these cell lines from the derivations that were eligible for 
Federal funding. During the scaling-up process, investigators 
need to repeatedly check that the cells maintain their 
abilities, and once that is done, we can go forward with the 
distribution.
    So as a first step, Senator, toward overcoming this 
challenge, NIH announced five infrastructure grant awards 
totaling $4.3 million to five sources on the NIH registry, 
holding 23 of the eligible derivations. Two additional awards 
have passed peer review and await final approval in funding 
within the next few weeks. These awards will fund the 
expansion, the testing, the quality assurance, and the 
distribution of the cells through the process I just explained.
    We are also working with stem cell sources to address the 
complex issues that might limit widespread availability of 
these lines, in particular intellectual property issues. In the 
past year, NIH has negotiated agreements with four stem cell 
providers to allow both our intramural researchers access to 
their cells and also to allow extramural researchers to have 
access to these cells. Under these four agreements, six 
intramural laboratories at NIH have received cells to pursue 
research, and the agreements commit the four providers with 
whom we have signed agreements to offer these cells under 
similar terms to extramural investigators.
    WARF, for example, which is the source that has been the 
first to be able to provide cell lines has informed us that it 
has agreements in place with 111 researchers and has shipped 
cells to 74 of them. These researchers represent 61 
institutions, 12 of them in foreign countries.
    Another source, ES Cell International, informs us that it 
currently has a supply of cells that far exceeds current 
demand. We are still in active discussions with all sources to 
be able to provide additional cell lines.
    We are receiving investigator-initiated research grant 
applications from new investigators. So far five new grants, 
totaling $4.2 million, have been awarded. We have issued 32 
administrative supplements to existing grant awards that allow 
30 researchers from 25 different institutions to incorporate 
research on human embryonic stem cells as part of their ongoing 
federally supported research. This means that currently funded 
laboratories are extending their work to include human 
embryonic stem cells, which is a way for them to develop the 
skills and expertise needed in this field.
    Senator Specter. Dr. Zerhouni, I am reluctant to interrupt 
you, but if you could sum up now, we would appreciate it.
    Dr. Zerhouni. I will.
    I formed a stem cell task force as soon as I arrived at 
NIH, and the reason I did is because I felt that it was very 
important for NIH to promote this field as fast as we can both 
in terms of embryonic and adult stem cell research. I have 
appointed Dr. Jim Battey as head of the Stem Cell Task Force, 
and I am looking forward to continue, as aggressively as we 
can, the development of this work. Thank you.
    [The statement follows:]
                Prepared Statement of Dr. Elias Zerhouni
    Mr. Chairman, Senator Specter, and Members of the Subcommittee, I 
am pleased to appear before you today to testify about the role of NIH 
in advancing the field of stem cell research. Properly harnessed, adult 
and embryonic stem cells have the potential to replace cells that are 
damaged or diseased to restore vital functions of the human body. They 
offer the promise of curing disease and ending disabilities at some 
point in the future. So there are ample reasons for excitement about 
stem cell research, and high expectations for new treatments are 
understandable. But such expectations should be tempered by the 
enormous challenges that must be addressed before the research evolves 
into proven therapy.
    These challenges involve both human embryonic stem cell research 
and adult stem cell research. Human embryonic stem cells and adult stem 
cells have potential as future therapies. I believe that NIH should 
continue to fund research on both types of cells.
    We are at a very early stage of embryonic stem cell research, and 
have a great deal of basic research to conduct before we can unlock the 
potential of these cells and fulfill their promise. I will describe the 
pathway of discovery that I believe will unfold as the research evolves 
from stem cell lines to cell based therapy. In the basic research 
phase, which is the current focus of NIH-supported activities, we first 
need to build the scientific capacity. As is true for any area of 
research, progress depends on attracting outstanding scientists to 
design and perform the needed studies. NIH is providing opportunities 
for the scientific community to develop training courses for 
researchers to acquire the skills needed to culture embryonic stem 
cells, as well as opportunities to support stem cell research career 
pathways. NIH has already taken major steps to accomplish this goal by 
supporting infrastructure awards to expand cell lines, refine culture 
methods, and establish improved methods to select the most desirable 
embryonic stem cell populations.
    There have been significant scientific discoveries in the past year 
involving embryonic and adult stem cells. Scientists have recently 
shown that human embryonic stem cells can be directed to develop into 
cells resembling nerve cells, cardiac muscle cells and insulin 
producing cells. These are the cells that might someday be used to 
treat Parkinson's disease, heart disease and type I diabetes.
    In addition to the new research opportunity provided by the 
availability of human embryonic stem cells, NIH continues to 
aggressively support research on developing the therapeutic potential 
of adult stem cells. Scientists have recently discovered that adult 
stem cells in animals may be used to repair cartilage and bone damaged 
by injury and disease. In addition, research published this past summer 
showed that adult stem cells from the bone marrow of both rodents and 
humans can differentiate into multiple cell types, and can grow for 
long periods of time in culture. Understanding the molecular signals 
that direct adult stem cell differentiation may lead to new strategies 
for harnessing the power of a person's adult stem cells to replenish 
specialized cells destroyed by disease or aging. But it is clear that 
much more research needs to be done to explore the characteristics of 
adult stem cells, and to develop methods of expanding different 
populations of adult stem cells in the laboratory. In addition, for 
many types of adult stem cells, more research is needed to determine 
techniques to expand these cells in the laboratory, a capacity that 
will enable both basic and clinical studies using adult stem cells. NIH 
continues to believe that research on both embryonic stem cells and 
adult stem cells must be pursued simultaneously in order to learn as 
much as possible about the potential of these cells to treat human 
disease.
    These findings are important, but I continue to emphasize that we 
are at a very early stage. Much more basic research needs to be done. 
Stem cell researchers have shown that these cells have long term 
viability, with no evidence for genetic changes. However, human 
embryonic stem cells tend to be unstable and must be closely monitored 
to maintain them in their undifferentiated state. Much more basic 
research needs to be done to validate the long term stability of human 
embryonic stem cells, both in culture and after transplantation. 
Embryonic stem cells have the remarkable capacity to continue to grow 
indefinitely in an unspecialized state. In research involving other 
cell types, much has been learned about key regulators of cell 
division. Additional research is needed to determine how to harness the 
molecular systems that control this process, so that once transplanted, 
the specialized cells developed from embryonic stem cells do not revert 
back to their embryonic state and grow in an uncontrolled fashion 
leading to tumors or other unwelcome outcomes.
    If we are able to direct stem cells to develop into a specialized 
cell type, research will need to be done to determine that the 
specialized cells function appropriately in the context of an animal 
model system for human disease. NIH's long term commitment to 
developing such animal models for diseases such as diabetes, 
Parkinson's disease, and spinal cord injury will be an important factor 
in developing this aspect of embryonic stem cell research. As we 
proceed, NIH will also ensure that federal funds are used to support 
research that has scientific merit and demonstrates outstanding 
opportunities.
    Such basic research is only the first phase of the journey along 
the pathway of embryonic stem cell research. There are many pre-
clinical studies, which do not involve human subjects, that need to be 
performed before any new therapeutic modality advances to clinical 
trials on real patients. These studies include tests of the long term 
survival and fate of transplanted cells, cell dosing studies, as well 
as tests of the safety, toxicity, and effectiveness of the cells in 
treating animal models for disease.
    Trials using human subjects, the clinical research phase, will 
begin only after the basic and pre-clinical foundation has been laid. 
This foundation will minimize any chance of unpredictable harmful 
effects that stem cell based therapies might cause. These trials are 
usually phased, with the phase I trial focusing on safety, and phase II 
and III trials aimed at establishing optimal dose, providing additional 
assurance of safety, and determining efficacy. Only after these many 
important steps are taken will the promise of embryonic stem cells to 
treat disorders like diabetes, Parkinson's disease, spinal cord injury, 
and cardiac failure be realized.
    Having provided you with a strategic vision for research using 
human embryonic stem cells, I want to explain to you how NIH is 
addressing two immediate major issues that are essential for stem cell 
research community to move forward:
             increasing the number of stem cell researchers
    As is the case at the beginning of any new field of discovery, 
there is a shortage of researchers with expertise in stem cell 
research. This dearth is currently a rate-limiting step in advancing 
the progress of embryonic stem cell research. Simply growing embryonic 
stem cells to the state where they can be used for experimentation 
requires substantial knowledge, training and experience. NIH will 
strive to make stem cell research as attractive as possible to our most 
talented research scientists, whose creativity in developing 
investigator-initiated research will move the research agenda forward. 
NIH is soliciting grant applications to support training courses to 
teach investigators how best to grow stem cells into useful lines.
          making human stem cells more available for research
    On November 7, NIH published a registry of derived stem cells that 
would be eligible for federal funding. The registry consists of 14 
sources across the world. The cells are in various stages of 
characterization and preparation for research applications. There are 
many steps required to develop embryonic stem cells from when they are 
first removed from an embryo and put in culture into an established, 
well characterized embryonic stem cell line ready for distribution to 
the research community. After derivation, embryonic stem cells need to 
be expanded from a small cluster into hundreds of million of cells 
before they are ready for distribution. During the scaling up process, 
investigators need to repeatedly check that the cells maintain their 
ability to divide continuously and become all of the specialized cells 
required for research. This process of expanding a cell line requires 
time, resources, and expertise.
    As a first step toward overcoming this challenge, NIH has announced 
five infrastructure grant awards, totaling $4.3 million, to five 
sources on the NIH Registry holding 23 of the eligible derivations. Two 
additional awards passed peer review and await final approval and 
funding within the next few weeks. These awards will fund the 
expansion, testing, quality assurance and distribution of cells.
    We are also working with stem cell sources to address complex 
issues that might limit widespread availability of these eligible 
cells. In the past year, NIH has negotiated agreements with four stem 
cell providers to allow our intramural researchers access to their 
cells. These providers have also agreed to offer similar terms to our 
grantees, enabling them to obtain cells without developing their own 
agreements de novo. Under these four agreements, our intramural 
researchers are free to publish their findings and the NIH will own any 
inventions made in the course of its research. As a result of these 
agreements, six intramural laboratories have received stem cells and 
are pursuing research with them. The agreements commit the four 
providers to offering cells under similar terms to NIH's extramural 
investigators. In addition, the Wisconsin Alumni Research Foundation 
(WARF), which holds key patents on this technology, has agreed to 
provide a free license to non-profit researchers conducting academic 
research with cells from other providers. WARF has informed us that it 
has agreements in place with 111 researchers, and has shipped cells to 
74 of them. The researchers represent 61 institutions, 12 of them in 
foreign countries. Another source, ESI, informs us that it currently 
has a supply of cells that far exceeds current demand. Meanwhile, NIH 
is in active discussions with other sources listed on the NIH Registry 
in pursuit of additional agreements.
    NIH is beginning to receive investigator-initiated research grant 
applications from new investigators focusing on human embryonic stem 
cell research. So far, five new grants, totaling $4.2 million, have 
been awarded. Also, NIH has issued 32 administrative supplements to 
existing grant awards that will allow 30 researchers from 25 different 
institutions to rapidly incorporate research on human embryonic stem 
cells as part their ongoing federally-supported research. This means 
that currently funded laboratories are extending their work to include 
human embryonic stem cells, which is a way for them to develop their 
skills with these difficult cells and develop some preliminary data--
both key steps to success in future research. All told, over 40 
investigators are now funded by the NIH to work in this area.
    Much progress has occurred in the past year, including new 
discoveries, identifying sources of stem cells, negotiating access 
agreements, and creating a friendly environment to attract researchers. 
However, these are only initial steps. To move us into the next phase, 
I have created a new stem cell task force at NIH, led by Dr. James 
Battey, the Director of the National Institute on Deafness and Other 
Communication Disorders. The task force will provide direction for the 
future in the form of recommendations for NIH-supported research 
initiatives. Currently, the task force is reviewing the state of the 
science for all stem cell research, with the goal of using NIH 
resources to enable the scientific community to capitalize on this new 
and challenging opportunity.
    NIH would not be able to move forward in stem cell research, and 
for that matter, any other research, without the support of this 
Subcommittee. Thank you for your support. I look forward to working 
with you to advance this and all fields of biomedical research. I will 
be happy to answer any questions you might have.

    Senator Specter. Thank you very much, Dr. Zerhouni. A 
little more time was allowed for your presentation because of 
the importance of what NIH is doing in setting the stage for 
our other witnesses.
    Since there are two votes, as I had said earlier, at 10:30, 
we are going to proceed now--if you would keep your seat, Dr. 
Zerhouni--to hear from the other five panelists, and then we 
will proceed to questions. So if Senator Deborah Ortiz would 
step forward, along with Dr. Civin, Dr. Daley, Dr. Pedersen, 
and Dr. Schatten, we will hear your testimony.
STATEMENT OF SENATOR DEBORAH ORTIZ, SIXTH STATE 
            DISTRICT, CALIFORNIA STATE SENATE
    Senator Specter. Our first witness on this panel is Senator 
Deborah Ortiz, elected to the 6th State Senate District in 
California in November 1998. She is the Chair of the Health and 
Human Services Committee and a member of the Education, Budget, 
Public Employment and Retirement, and Natural Resources and 
Wildlife Committees. She received her undergraduate degree from 
the University of California at Davis and her law degree from 
McGeorge School of Law.
    As noted earlier, if we do not conclude by 10:40, we will 
have about a 30-minute break for the vote. So we are going to 
try to proceed to conclude at that time.
    Senator Harkin could not be here today. He is the chairman 
and I am ranking. We traded positions last year. Senator 
Jeffords arranged that.
    But we have had a very close collaboration, and as far as 
the operation of this subcommittee is concerned, it does not 
make any difference whether Senator Harkin is the Chair or I 
am. We have worked that closely.
    Senator Ortiz, thank you very much for coming all the way. 
We look forward to your testimony. This is a clock showing 5 
minutes, if you could please sum up and stop by the red light.
    Ms. Ortiz. Wonderful. Thank you, Senator Specter, as well 
as other members of the committee. I thank you for inviting me 
here. I am very conscious of running a committee on time, so I 
am going to pull out my watch and try to adhere to the 5-minute 
rule as well.
    Thank you for inviting me to join you today at today's 
hearing as you pursue the very important question and the task 
of examining the implementation of President Bush's stem cell 
research policy and the impact of that policy on the 
development of stem cell technologies.
    Let me begin by sharing with you why California found it 
imperative to move forward on stem cell research. In order to 
do so, let me share with you my personal history.
    I served as assemblywoman and was elected to the State 
Assembly in 1996. As I was transitioning in my newly elected 
position as assemblywoman, my mother had been diagnosed with 
ovarian cancer. I took very seriously the task of saving her 
life. As I did my research and as her disease progressed, I 
began to understand that as important as chemotherapy and 
treatments like chemotherapy are in the lives of millions of 
cancer patients and the families who take care of those 
individuals, I knew that the next level of cure for cancer, as 
well as all the other diseases that we are all absolutely 
committed to curing and improving the quality of life, that the 
real cure really resided at a very basic level in the research, 
and stem cell research was offering that promise.
    When President Bush declared the August 2001 64-line 
limitation for use in access to Federal dollars, I decided that 
I was going to try to have California move forward, and it 
became even more compelling this last spring as we began to see 
a couple of competing measures move through Congress. The 
Brownback bill posed the greatest concern to California, not 
just in its limitations and its criminalization of science and 
medicine, but also in the likelihood that there would be some 
success in his closing the door to science and technology and 
preventing the delivery of that promise to all of those 
Americans, over 100 million, who suffer from these diseases.
    We also saw Senator Feinstein's work, and I thank the 
members of this committee for having often a nonpartisan debate 
about a very important policy issue. We were hopeful that that 
bill would, indeed, become law and would preclude the Brownback 
bill from becoming law. That was not to happen.
    So as we moved forward in California, I hosted two 
significant hearings, one in Stanford with the brightest and 
most brilliant of minds, and Dr. Pedersen to my right here was 
good enough to videotape his testimony and welcome us from 
England and share with us why he left the United States in 
order to pursue the science that we all hope to achieve in 
California.
    Out of that hearing at Stanford, we decided to move forward 
and go to the Salk Institute and also have a hearing in which 
Hans Keirstead, who is doing some incredible research in Irvine 
in California, demonstrated the mice whose spinal columns had 
been severed in which the introduction of stem cells produced 
movement and function in the lower limbs of those mice. We also 
heard the testimony from Jerry Zucker, the father of the 14-
year-old daughter with juvenile diabetes, who shared with us 
his hope that his daughter would be able to live to see 
adulthood and not spend her life on dialysis and ultimately die 
at a very early age.
    California decided to move forward in this research. I 
introduced the bill that would legalize in California stem cell 
research with the appropriate ethical and IRB review, as well 
as prohibitions for sale and transfer of embryos.
    When we broadened that commitment to curing cancer, we 
acted decisively to pursue stem cell research in California. My 
law that the Governor has now signed has made, for all intents 
and purposes, the Bush policy on stem cell research irrelevant 
in the State of California. California will move forward to 
cure cancer, as well as Alzheimer's, as well as ALS, as well as 
Parkinson's, juvenile diabetes, address the spinal cord injury 
challenges and day-to-day realities of persons who live with 
those injuries. And we will move forward. We hope to share 
those therapies and that medical science and improvement with 
the rest of the country.
    We ask that Congress respect California's will to protect 
Californians and assure that that right will be protected and 
not preempted by any subsequent Federal law.
    California is moving forward because we understand our 
responsibility to pursue technology that promises to cure or 
effectively treat over 100 million Americans. To commit the 
necessary resources to deliver that hope, we have an 
unavoidable obligation to do everything we can do to realize 
the potential of stem cell research.
    Once again, California is moving forward. We ask you to 
respect that. We believe that the Bush policy is not only 
medically and scientifically unsound, it is simply irrelevant 
in the State of California.
    Thank you.
    Senator Specter. Thank you very much, Senator Ortiz.
STATEMENT OF ROGER PEDERSEN, Ph.D., DEPARTMENT OF 
            SURGERY, CAMBRIDGE UNIVERSITY
    Senator Specter. We turn now to Dr. Roger Pedersen, a 
leading stem cell researcher, who had been at the University of 
California in San Francisco until September of last year. At 
that time, Dr. Pedersen decided to relocate to the University 
of Cambridge where he could receive government funding for his 
research on human embryonic stem cells. Currently Dr. 
Pedersen's research is supported by the United Kingdom Medical 
Research Council and the Wellcome Trust. He received his Ph.D. 
in biology from Yale.
    We very much appreciate your coming such a long distance to 
join us to add your own views and insights. The necessity for 
your moving out of the United States is a matter of grave 
concern and is obviously a factor in determining what our 
policy should be as to stem cells. Dr. Pedersen, the floor is 
yours.
    Dr. Pedersen. Senator Specter, thank you very much for the 
opportunity to speak.
    As you know, until this time last year, I worked at the 
University of California, San Francisco, where I had been a 
faculty member for the previous 30 years and where we derived 
two of the novel embryonic stem cell lines on the NIH registry 
early last year. I now live and work in the United Kingdom 
where I am engaged in stem cell research at the University of 
Cambridge. In addition to having responsibilities for my own 
research team in the Department of Surgery there, I lead a 
consortium of 25 researchers who are focusing their individual 
groups on various aspects of stem cell biology and medicine. I 
also provide advice to other administrators and scientists in 
the United Kingdom who are guiding the development of the UK 
stem cell enterprise.
    I would like to add my enthusiasm for how exciting this is 
as the time for stem cell researchers. We are building on more 
than 20 years of experience using mouse embryonic stem cells 
for genetic studies and on even greater experience using human 
blood stem cells for clinical treatments. This has provided a 
foundation for the successful culturing of human embryonic stem 
cells and opened the opportunity to control the development of 
human cells in the laboratory into forming a variety of useful 
tissues.
    Importantly, we now have evidence that Dr. Zerhouni has 
mentioned from NIH researchers that mouse embryonic stem cells 
can be cultivated to produce insulin in mice and to alleviate, 
in other studies, the symptoms of Parkinson's in rats. These 
advances in stem cell biology raise our expectation for 
clinical benefits from stem cell medicine.
    All of us know of a courageous person like Christopher 
Reeve who could benefit from such novel therapies. For me, it 
was my mother who died of diabetes in 1989, yet still provides 
me with an enduring will to help people with that disease. How 
can we achieve the clinical promise of stem cell research on 
their behalf?
    Against these expectations, the pace of discovery of human 
embryonic stem cells seems painfully slow. The lack of any 
Federal support for research on human embryos, stretching all 
the way from 1978 to the present day, has undoubtedly delayed 
the benefits of research to infertile patients. And the long 
wait for Federal funding to support stem cell research has, I 
think, equally delayed the benefit of research to patients with 
degenerative diseases. I admit to having been frustrated myself 
with the length of time we had to wait for Federal funds for 
stem cell research. Admittedly, the establishment last summer 
of an NIH registry of human embryonic stem cells eligible for 
Federal funding was a significant first step in advancing such 
research.
    However, given the length of time required to build a 
successful research program, any concern on the part of 
researchers for a worsening in the present U.S. policy for stem 
cell funding would tend to keep prospective researchers on the 
sidelines. Such concerns would definitely undermine efforts to 
recruit additional researchers, particularly junior 
investigators, into the field. It would be particularly 
devastating if the U.S. Senate moved to criminalize the use of 
somatic cell nuclear transfer to generate immune-matched stem 
cells. And in this respect, it is very good to see my home 
State of California has made clear its position in support of 
this and all other aspects of stem cell research.
    How could the Federal Government do a better job of 
supporting stem cell research?
    First, let me offer my respect for the will and 
perseverance that the NIH has shown during the last decade in 
their desire to support the fields of human embryology and 
embryonic stem cells. I believe that their approach of building 
up the research infrastructure by supporting training of 
researchers and the standardization, characterization, and 
distribution of the human embryonic stem cell lines on the 
registry will prove to be a wise one for this country. I am not 
convinced that it is necessary to convert the present 
decentralized stem cell bank to a centralized repository. 
Rather, I think that such a move by the NIH would lead to 
additional delays in the accessibility of cell lines. 
Therefore, my advice to them is to hold their present course.
    But the truth is that the Federal Government as a whole 
must make a far larger commitment in order to realize the 
larger promise of stem cell medicine. New embryonic stem cell 
lines must be derived and characterized in order to meet 
current tissue standards for transplantation. Extensive studies 
are needed to define the conditions for generating large 
numbers of stem cell types from stem cell lines. Preclinical 
studies in animals, including not only rodents but also non-
human primates, will be essential. And finally, careful 
clinical trials in appropriate patient populations will be 
needed to prove the efficacy of stem cells as medicines. This 
will all take some years to achieve. I do not believe that 
miracles that endure happen overnight.
    To sum up my views, I believe what is needed is a long-term 
U.S. commitment to develop the public policies and to sustain 
the public funding that will make the stem cell dream come 
true. Why should we regard the ravages of disease as 
inevitable? If there is a war to be fought, surely it is 
against the presently untreatable diseases which kill thousands 
of people each day of the year. To mount an effective campaign 
against such diseases will require a coordinated international 
effort that harnesses the strength of each country. Any 
abdication on this front will likely cede the present U.S. 
leadership in the field of stem cells to Europe, Australia, or 
Asia, together with the economic benefits which will flow 
toward those countries that invest early and consistently in 
stem cell biology.

                           prepared statement

    In closing, Senator, I would like to take this opportunity 
to thank you and Senator Harkin for your enduring support for 
this field, not only for stem cell biology and medicine, but 
also for all those who suffer from diseases. Thank you for 
hearing my views.
    [The statement follows:]
                Prepared Statement of Roger A. Pedersen
    Honorable Senators Specter and Harkin, Distinguished Colleagues, 
and Guests: My name is Dr. Roger Arnold Pedersen. Until this time last 
year, I worked at the University of California, San Francisco, where I 
had been a faculty member for the previous 30 years, and where we 
derived two novel human embryonic stem cell lines early last year. I 
now live and work in the United Kingdom, where I am engaged in stem 
cell research at the University of Cambridge. In addition to having 
responsibilities in Cambridge for my own research team in the 
Department of Surgery, I lead a consortium of 25 researchers there who 
are focusing their individual groups on various aspects of stem cell 
biology and medicine. I also provide advice to other administrators and 
scientists in the United Kingdom who are guiding the development of the 
UK stem cell enterprise.
    Now is an exciting time for stem cell researchers. We are building 
on more than 20 years of experience using mouse embryonic stem cells 
for genetic studies and on even greater experience using human blood 
stem cells for clinical treatments. This has provided a foundation for 
the successful culturing of human embryonic stem cells, opening the 
opportunity to control the development of human cells in the laboratory 
into a variety of useful tissues. Importantly, we now have evidence 
that mouse embryonic stem cells can be cultivated to produce insulin in 
mice, thus alleviating the symptoms of diabetes, and to form cells that 
alleviate Parkinson's symptoms in rats. These advances in stem cell 
biology raise our expectations for clinical benefits from stem cell 
medicine. All of us know of a courageous person, like Christopher 
Reeves, who could benefit from such novel therapies. For me it was my 
mother, who died of diabetes in 1989, yet still provides me with an 
enduring will to help people with that disease. How can we achieve the 
clinical promise of stem cell research on their behalf?
    Against these expectations, the pace of discovery with human 
embryonic stem cells seems painfully slow. The lack of any federal 
support for research on human embryos--stretching all the way from 1978 
to the present day--has undoubtedly delayed the benefits of research to 
infertile patients. The long wait for federal funding to support stem 
cell research has delayed the benefits of research to patients with 
degenerative diseases. I admit to having been frustrated myself with 
the length of time we had to wait for federal funds for stem cell 
research. Admittedly, the establishment last summer of an NIH registry 
of human embryonic stem cells eligible for federal funding was a 
significant first step in advancing stem cell research. However, given 
the length of time required to build a successful research program, any 
concern for a worsening in the present U.S. policy for stem cell 
funding would tend to keep prospective researchers on the sidelines. 
Such concerns could definitely undermine efforts to recruit additional 
researchers--particularly junior investigators--into the field. It 
would be particularly devastating if the U.S. Senate moved to 
criminalize the use of somatic cell nuclear transfer to generate 
immune-matched stem cells. In this respect, it is good see that my home 
State of California has made clear its position in support of this and 
all other aspects of stem cell research.
    How could the federal government do a better job of encouraging 
stem cell research? First let me offer my respect for the will and 
perseverance that the NIH has shown during the last decade in their 
desire to support the fields of human embryology and embryonic stem 
cells. I believe that their approach to building up the research 
infrastructure by supporting training of researchers and the 
standardization, characterization and distribution of the human 
embryonic stem cell lines included on the stem cell registry will prove 
to be a wise one for this country. I am not convinced that it is 
necessary to convert the present ``decentralized'' stem cell bank to a 
centralized repository. Rather, I think such a move by the NIH would 
lead to additional delays in accessibility of cell lines. Therefore, my 
advice for them is to hold their present course.
    But the truth is, the federal government as a whole must make a far 
larger commitment in order to realize the larger promise of stem cell 
medicine. New embryonic stem cell lines must be derived and 
characterized in order to meet current tissue standards for 
transplantation. Extensive studies are needed to define the conditions 
for generating large numbers of specialized cell types. Pre-clinical 
studies in animals, including not only rodents but also non-human 
primates, will be essential. Finally, careful clinical trials in 
appropriate patient populations will be needed to prove the efficacy of 
stem cells as medicines. This will all take some years to achieve. 
Miracles that endure don't usually happen overnight.
    To sum up my views, I believe what is needed is a long-term United 
States commitment to development of public policies and sustenance of 
public funding that will make the stem cell dream come true. Why should 
we regard the ravages of disease as inevitable? If there is a war to be 
fought, surely it is against the presently untreatable diseases, which 
kill thousands of people each day of the year. To mount an effective 
campaign against such diseases will require a co-ordinated 
international effort that harnesses the strength of each country. Any 
abdication on this front will likely cede the present U.S. research 
leadership in the stem cell field to Europe, Australia or Asia. The 
economic benefits of stem cell medicine will flow towards those 
countries that invest early and consistently in stem cell biology.
    In closing, I would like to take this opportunity to extend my deep 
appreciation to both of you, Senator Specter and Senator Harkin, for 
your enduring and unequivocal support, not only for stem cell biology 
and medicine but for all those who suffer from diseases. Thank you for 
hearing my views.

    Senator Specter. Thank you very much, Dr. Pedersen.
    Senator Murray.

               OPENING STATEMENT OF SENATOR PATTY MURRAY

    Senator Murray. Mr. Chairman, I am sorry to interrupt, and 
I have to get to a markup. If I could just ask Dr. Zerhouni one 
really critical question.
    Senator Specter. Of course, Senator Murray. Proceed.
    Senator Murray. I really appreciate your holding this 
hearing. I think many of us were very concerned about the 
President's decision to limit stem cell lines a year ago and 
are watching with interest that California has now move ahead 
on this and are very concerned what will happen in our States 
with perhaps a drain of researchers and where that will go.
    But I just wanted to quickly ask Dr. Zerhouni if State 
funds are used for embryonic stem cell research, will 
researchers in California or any other States that enact laws 
like this receive NIH funds in the future, or will they be 
prohibited from receiving those funds?
    Dr. Zerhouni. No. They can receive NIH funds if they work 
on the eligible cell lines that President's policy has 
identified as eligible for Federal funding. There will be no 
problem, and we have put in place the appropriate steps so that 
an investigator could work with Federal funding on eligible 
cell lines and work with State funding on other cell lines as 
desired, as currently is allowed. There is no change from what 
we have today.
    Senator Murray. Thank you, Mr. Chairman. I have a number of 
other questions I would like to submit for the record.
    Senator Specter. Of course, Senator Murray, they will be 
accepted for the record and responses will be made.
    Dr. Pedersen, let me thank you for your good words for 
Senator Harkin and myself.
    We had set upon a program to double NIH funding. We have 
moved it from $12 billion to $23 billion. This year we have in 
our budget $3.7 billion in addition. But there has to be a 
bill. So far we have not had any legislation come out of the 
appropriations process, and if we are to have a continuing 
resolution, that means that the funding will probably stay 
level. That will be very, very bad for many projects, but 
especially for NIH where we will have done more than the 
doubling which we had anticipated. From $12 billion, it would 
put us at $26.7 billion.
    I make that comment at this time so that all of those here 
can use your lobbying influences to help us get a bill, and if 
you want a more particular road map, I would be glad to talk to 
you later.
STATEMENT OF GERALD SCHATTEN, Ph.D., PROFESSOR OF CELL 
            BIOLOGY, UNIVERSITY OF PITTSBURGH, 
            DIRECTOR, PITTSBURGH DEVELOPMENT CENTER, 
            AND DEPUTY DIRECTOR, MAGEE-WOMEN'S RESEARCH 
            INSTITUTE
    Senator Specter. We will turn now to Dr. Gerald Schatten, 
deputy director of Magee-Women's Research Institute and 
director of the Pittsburgh Development Center. He is a 
professor and vice chair of obstetrics, gynecology and 
reproductive sciences and cell biology at the University of 
Pittsburgh School of Medicine. He received his Ph.D. from the 
University of California at Berkeley.
    I have worked with you and, we are glad to have you in 
Pennsylvania, Dr. Schatten. It seems to me we have got a very 
heavy California influence here today.
    The floor is yours.
    Dr. Schatten. Thank you, Senator Specter, and it is a great 
pleasure for me to have this opportunity to speak with you.
    The NIH deserves tremendous commendations for their efforts 
in rapid implementation this past year, but serious and 
substantial work remains. From my own experiences, I need to 
voice grave concerns about the current Federal stem cell 
policies because it is already hindering invaluable research, 
undermining the wisest investments, and delaying the day when 
we will know for sure whether human embryonic stem cells can be 
used to treat diseases.
    The NIH's registry lists 71 lines. Science reports only 16 
are available. My search has identified just a handful. As of 
last Thursday, we have just two.
    We need accuracy and clarity. Perhaps 71 lines do meet 
eligibility criteria, but just being eligible is not the same 
as available.
    To obtain approved lines, I have traveled to Europe and 
Asia to collaborate with scientists in Korea, Singapore, 
Australia, Sweden, and the UK. They are willing and motivated. 
But should American science not also be conducted on American 
soil?
    NIH has sponsored my research for the last 25 years, and we 
investigate how fertilization succeeds and how the embryo 
develops. Last November, we were among the first to apply to 
investigate how human embryonic stem cells divide and 
proliferate. When cells lose chromosomes, they can develop into 
cancers. If human embryonic stem cells lose chromosomes when 
they are put into a patient's body, as Dr. Zerhouni mentioned, 
they could develop into cancers. Chromosome movements in human 
embryonic stem cells must be accurate and that is just what we 
are doing in our laboratory.
    Researchers at the Pittsburgh Development Center of Magee-
Women's are discovering that embryos form very differently 
between the mammals cloned successfully by somatic cell nuclear 
transfer and primates, as investigated in monkeys. Cloned cows 
and mice can develop without any sperm contributions, whereas 
primates, in which all somatic cell cloning attempts have 
failed so far, appear to depend on a unique complementation 
between the egg's machinery and the sperm's special structures.
    Reproductive cloning in humans is dangerous, unethical, 
unjustified, and for biological purposes we would predict that 
it will fail.
    Therapeutic cloning, on the other hand, in which embryonic 
stem cells are produced in a plastic dish in the absence of any 
sperm or any fertilization event, promises unique methods to 
overcome our body's own immune rejection systems. The editorial 
this week in Science entitled ``Harmful Moratorium on Stem Cell 
Research'' is authored by some of the hand-picked members of 
the President's own Bioethics Panel.
    Last April, NIH modestly funded our proposal for just a 
year. These supplements are insufficient in time or amount for 
the best research programs to justify redirection. The NIH must 
be more aggressive in supplementing investigator-initiated 
grants with significant funding. Cooperative agreements would 
enlarge the talent pool. New equipment is necessary to ensure 
the separate of stem cell research from ongoing activities. 
Labs selected for multi-year awards should also be responsible 
for research training.
    Commercial-academic cooperations also need to be encouraged 
further.
    On Sunday, I met with Lans Taylor, who is CEO of the 
Pittsburgh-based company Cellomics. He has mocked up human 
embryonic stem cell pluripotency kits and assays to determine 
whether these lines will develop into neurons. Other companies 
could further reduce the hurdles very swiftly if they were 
encouraged to jump into this field and reduce the research 
hurdles.
    During this hearing, we have discussed national policy and 
contrasted it with stem cell rules elsewhere. We are the United 
States and each State has its own laws and restrictions that 
may prove enabling or restrictive. Senator Specter, you know 
well that our Commonwealth of Pennsylvania has language 
restricting human embryonic stem cell research. The Abortion 
Control Act of 1989, written long before stem cells were 
discovered, prohibits embryonic research.
    Homeland Security Director Tom Ridge, while still Governor 
of Pennsylvania, decided that cells derived outside of 
Pennsylvania were eligible for research within our 
commonwealth.
    We have heard just now from Senator Ortiz that California 
is enacting laws to enable human embryonic stem cell studies.
    It may be within this subcommittee's purview that in 
addition to witnessing American scientists emigrating, we may 
soon see U.S. scientists relocating from States with ambiguous 
laws to other States.
    Senator Specter, subcommittee members, I applaud you and 
others in Congress for your unwavering support of the NIH. Your 
sponsorship and encouragement of healthy Federal and private 
sector competition produced the human genome sequence under 
budget and far sooner than expected. When we decided to 
decipher the genome, which also generated controversies, we 
deliberated thoughtfully and invested adequately.
    More and better lines are needed now and current policies 
are already delaying stem cell research, forcing it off shore 
or into inaccessible reaches in the private sector.

                           prepared statement

    Would Galileo have been satisfied if he could have looked 
at 65 or 71 stars? Maybe, but he would not have discovered our 
place in the solar system unless Jupiter traveled through that 
narrow field.
    In today's terms, the cost of the Hubble telescope and all 
of NASA is the same if our focus is restricted or if we are 
permitted to explore the heavens.
    Thank you.
    [The statement follows:]
               Prepared Statement of Dr. Gerald Schatten
    Good morning, Chairman Harkin, Senator Specter, and other 
distinguished Subcommittee Members. I'm Gerald Schatten, Professor of 
Cell Biology at the University of Pittsburgh and Director of the 
Pittsburgh Development Center.
    The NIH deserves commendation for their efforts and rapid 
implementation this past year, but serious and substantial work 
remains. From my experiences, I need to voice grave concerns about the 
current federal stem cell policy because it's already hindering 
invaluable research, undermining the wisest expenditures and delaying 
the day when we'll know whether stem cells can be used to treat 
diseases.
    The NIH's Human Embryonic Stem Cell Registry lists 71 lines. 
SCIENCE reports only 16 are available. My search has identified just a 
handful right now--as of last Thursday, we've received two.
    We need accuracy and clarity. Perhaps 71 lines meet eligibility 
criteria, but just being eligible isn't the same as available.
    To obtain approved lines, I've traveled to Europe and Asia to 
collaborate with scientists in Korea, Singapore, Australia, Sweden and 
the UK. They're willing and motivated . . . but American science should 
also be conducted on American soil.
    NIH has sponsored my team's research for the past twenty-five years 
and we investigate how fertilization succeeds and how the embryo forms. 
Last November, we were among the first to apply to investigate how 
human embryonic stem cells divide and proliferate. When cells lose 
chromosomes, they can develop into cancers. Chromosome movements must 
be accurate in HESCs for safe transfer to patients, or new cancers 
might arise--and that's what we're working to understand and prevent.
    Researchers at the Pittsburgh Development Center are discovering 
that embryos form very differently between the mammals cloned 
successfully by somatic cell nuclear transfer (SCNT) and primates, as 
investigated with monkeys. Cloned cows and mice can develop without any 
sperm contributions, whereas primates, in which all SCNT cloning 
attempts have failed so far, appear to depend on the unique 
complementation of the egg's essential machinery with special sperm's 
structures.
    Reproductive cloning in humans is dangerous, unjustified and 
unethical.
    Therapeutic cloning, in which embryonic stem cells are produced in 
a plastic dish in the absence of any sperm or fertilization event, 
promises unique methods to overcome our body's natural immune rejection 
systems. The editorial this week in SCIENCE entitled ``Harmful 
Moratorium on Stem Cell Research'' is authored by some of the 
handpicked members of the President's Bioethics Panel.
    Last April NIH modestly funded our proposal for just one year. 
These supplements are insufficient in time or amount for the best 
research programs to justify redirection. The NIH must be more 
aggressive in supplementing investigator-initiated grants with 
significant, not token, funding. Cooperative agreements would enlarge 
the pool of talented labs. New equipment is necessary both to ensure 
the separation of HESC research from on-going lab activities and also 
because the tests for pluripotency and differentiation are specialized. 
Labs selected for multi-year awards could be responsible for research 
training.
    Commercial-academic cooperation needs to be encouraged further. As 
one example, I asked Lans Taylor, CEO of Cellomics to mock up 
pluripotency, growth and differentiation assays. Pittsburgh-based 
Cellomics and other companies could further reduce research hurdles 
quickly.
    During this hearing, we've discussed our national policy and 
contrasted it with stem cell rules elsewhere. We are united States--and 
each state has its own laws and regulations that may prove enabling or 
restrictive.
    Senator Specter knows well that our Commonwealth of Pennsylvania 
has language restricting HESC research. The Abortion Control Act of 
1989, written long before HESCs were discovered, prohibits embryonic 
research.
    Homeland Security Director Tom Ridge, while still Governor of 
Pennsylvania, decided that cells derived outside of Pennsylvania were 
eligible for research within our commonwealth.
    We've all read that California is enacting laws to enable HESC 
studies.
    It is may be within this subcommittee's purview that in addition to 
witnessing American scientists emigrating, we may soon also see U.S. 
scientists relocating from States with ambiguous laws to other States.
    Mr. Chairman, Senator Specter, Subcommittee Members, I applaud you 
and others in Congress for your unwavering support of NIH--your 
sponsorship and encouragement of healthy federal and private sector 
competition produced the human genome sequence under budget and sooner 
than predicted. When we decided to decipher the Genome, which also 
generated controversies, we deliberated thoughtfully and invested 
adequately.
    More and better lines are needed now and current policies are 
already delaying stem cell research, forcing it offshore or into 
inaccessible reaches in the private sector.
    Would Galileo have been satisfied if he could have looked at 59 or 
71 stars? Maybe, but he wouldn't have discovered our place in the Solar 
System unless Jupiter traveled through that narrow field.
    In today's terms, the cost of the Hubble telescope and all of NASA 
is the same if our focus is restricted, or if we're permitted to 
explore the heavens.
    Thank you.

    Senator Specter. Thank you, Dr. Schatten. Dr. Schatten, you 
are safe in Pennsylvania. Do not move.
STATEMENT OF CURT CIVIN, M.D., PROFESSOR OF CANCER 
            RESEARCH, SIDNEY KIMMEL COMPREHENSIVE 
            CANCER CENTER, JOHNS HOPKINS UNIVERSITY
    Senator Specter. We turn now to Dr. Curt Civin, King Fahd 
Professor of Oncology at Johns Hopkins University where he 
developed a stem cell selection process which has led to the 
development of more effective and less toxic cancer therapies. 
Dr. Civin holds nine patents for biomedical inventions related 
to stem cell research. He received his M.D. from Harvard 
Medical School.
    Thanks for joining us, Dr. Civin, and we look forward to 
your testimony.
    Dr. Civin. Thank you, Senator Specter, Senator Hutchison, 
Senator Ortiz. Thank you for the honor of testifying before you 
today.
    I am very grateful for this committee's strong and 
consistent support for lifesaving biomedical research. It is 
also a special privilege for me to testify today with my friend 
and former Johns Hopkins colleague, Elias Zerhouni. Our Nation 
is indeed privileged to have a scientist of his distinction and 
capability serve as NIH Director.
    I am professor of Cancer Research at the Johns Hopkins 
University School of Medicine, where I hope to stay. My 
clinical specialty is caring for children with cancer and this 
motivates my research. For the past 23 years, I have studied 
adult stem cells, mainly human bone marrow stem cells that can 
reconstitute our blood and immune systems. I discovered the 
CD34 stem cell molecule that allows identification and 
isolation of these rare blood-forming stem cells. The discovery 
is widely used in stem cell research and in clinical bone 
marrow transplantation, and two companies have licensed related 
inventions. And so I want to disclose to you that Johns Hopkins 
University and I have a financial interest in certain stem cell 
inventions and medical therapies.
    Today my research continues to focus on adult stem cells. 
We need to figure out how to grow these stem cells easily and 
in large numbers so that, for example, a bone marrow donation 
from one single donor can provide enough stem cells for 
multiple transplant patients.
    The 1998 discovery of human embryonic stem cells 
significantly raised our hopes of solving this therapeutic 
problem. By studying these cells, we hope to discover the 
molecular pathways by which they can proliferate without 
differentiating and then figure out how, in effect, to push the 
same molecular buttons in adult stem cells. Such discoveries 
would enhance the treatment of my cancer patients and might 
also help in the development of stem cell regenerative medical 
therapies for the range of other diseases.
    President Bush's decision to allow federally funded 
research on a qualified number of human ES stem cell lines 
increased our hopes of advancing this research. The decision 
has, however, proved much more limited than we anticipated. 
More than a year after the President's announcement, I am still 
waiting to receive my very first stem cell line. In fact, 
embryonic stem cell research is crawling like a caterpillar. 
Few human embryonic stem cell lines exist and most are not 
truly available. A number of the lines on the NIH stem cell 
registry have been tied up in questions of ownership. Many of 
the owners of the not-in-dispute cell lines are not anxious to 
share them with other researchers. Those that are willing to 
share their lines expect a piece of the profits on future 
discoveries. The terms of material transfer agreements are 
often difficult and time consuming to negotiate. The owners 
also expect an up-front fee. The going rate is $5,000, an 
amount 50 to 100 times greater than what we are accustomed to 
paying for a cell line.
    Besides these administrative burdens, there are significant 
technical challenges as well. Little is known about the cell 
lines themselves. Without this information, individual 
researchers are essentially flying blind. We must characterize 
the cell lines ourselves, an extraordinarily inefficient use of 
limited resources.
    An example. Last fall, a colleague applied to receive the 
best studied of the cell lines on the initial NIH list, the H1 
cell line from Wisconsin. Six months later he received the 
cells. It took him more than 4 months to grow enough ES cells 
to perform even preliminary experiments. These cells grow 
exceedingly slowly, one-tenth the rate of the cells we usually 
work with.
    For my research, I need several ES cell lines since I am 
sure that not all will form blood cells or will grow rapidly. 
Last fall I was contacted by a company in India which owns 
seven of the embryonic stem cell lines on the NIH registry. 
They wanted to collaborate with my lab to explore the blood-
forming potential of these lines. I spent several months 
negotiating collaboration and materials transfer, but the 
imminent agreement was abruptly canceled in May. The company 
told me that the Indian Government had put an indefinite hold 
on sending ES cell lines out of their country.
    In July I applied for a different ES cell line from 
Wisconsin that is reported to grow somewhat faster than H1 and 
to form some blood cells. I have been told that because of 
technical problems with these cells, I will not receive them 
until October at the earliest.
    Stem cell research has tremendous potential to deliver 
treatments and cures. With research we can make stem cells that 
are self-renewing, that are less likely to be rejected by the 
recipient's immune system and that regenerate tissues and 
organs fully.
    Today the United States of America is the best place in the 
world to do all biomedical research. I do not want us to lose 
that lead in stem cell research. And we are really in danger of 
doing so. Without our vigorous leadership in federally 
supported research in this country, the worldwide pace of 
discoveries will be much slower than necessary. Instead of 
being the first in line to benefit from new treatments as they 
are now, our patients in America will have to wait. We will 
lose talent to other nations, as you have heard, and new jobs 
and industries will be spawned elsewhere.
    Every week we read about exciting new stem cell research 
underway in other countries. Prime Minister Tony Blair of the 
UK recently said he wants Britain to be the best place in the 
world for stem cell research. Singapore has invested $1.7 
billion. I am heartened by Dr. Zerhouni's recent creation of 
the NIH Stem Cell Task Force and look forward to its 
contributions. Much work needs to be done.
    Mr. Chairman, I am also, again, grateful to the 
subcommittee for including language here in your fiscal year 
2003 committee report directing NIH to take positive steps to 
stimulate research. Specifically I would strongly endorse your 
language, urging NIH to develop a stem cell repository. A 
repository would promote research and lower the barriers to 
obtaining stem cell lines for investigators like me. Under this 
arrangement, NIH would characterize the lines and then act as a 
technical resource and distribution center. This would 
eliminate duplication of effort and provide an invaluable 
technical resource for growing the cells.

                           prepared statement

    Once again I want to thank you for your commitment to 
biomedical research and for your assistance in clearing 
unnecessary impediments to progress. You have really made a 
difference. Thank you.
    [The statement follows:]
                Prepared Statement of Dr. Curt I. Civin
    Chairman Harkin, Ranking Member Specter, and members of the 
Subcommittee thank you for the honor of testifying before you today 
about the hurdles that I, and many other, scientists have experienced 
in attempting to conduct embryonic stem cell research in the wake of 
President Bush's decision last year to permit research on a qualified 
number of stem cell lines. I am very grateful for your strong and 
consistent support of biomedical research and your interest in 
promoting life-saving stem cell research. I am also grateful for the 
language included in your fiscal year 2003 Committee Report directing 
NIH to take a number of positive steps to stimulate stem cell research.
    I am Professor of Cancer Research at the Sidney Kimmel 
Comprehensive Cancer Center of the Johns Hopkins University School of 
Medicine. My clinical specialty is caring for children with cancer, and 
this motivates my laboratory research on normal and leukemic stem 
cells. For the past 23 years, I have studied adult stem cells, mainly 
the stem cells from human bone marrow that can reconstitute our blood 
and immune systems after intensive radio-chemotherapy in a bone marrow 
transplant. I am best known scientifically for discovery of the CD34 
stem cell molecule that allows identification and isolation of these 
rare blood-forming stem cells. The CD34 monoclonal antibody is widely 
used in stem cell research as well as clinical bone marrow 
transplantation, and for this I received the National Inventor of the 
Year Award in 1999. Thousands of patients have received successful bone 
marrow stem cell transplants, mainly to mediate the toxic effects of 
their cancer therapy, but also for diseases such as immune 
deficiencies, autoimmune disorders, and aplastic anemia. Two companies 
have licensed related inventions, and so I must disclose to you that 
Johns Hopkins University and I have a financial interest in certain 
stem cell research and medical therapies.
    Today, my research continues to focus on adult stem cells. Despite 
our successes, over 15 years of intense investigations on adult blood-
forming stem cells has not taught us all we need to know about the 
biology of these adult stem cells. For example, we need to figure out 
ways to grow these cells easily and in large numbers so that like yeast 
in a fermenter a marrow donation from one donor could be expanded to 
provide stem cells for multiple bone marrow transplant recipient 
patients. The problem is that, outside of the body, these blood-forming 
stem cells rarely proliferate without differentiating. That is, the 
stem cells divide into more mature progeny that are no longer stem 
cells.
    So I was excited by the 1998 discovery of human embryonic stem 
cells (hES) that can expand indefinitely in tissue culture without 
losing their capacity to generate stem cells of many types of organs 
and tissues. Our hope is to study these embryonic stem cells, discover 
the molecular pathways by which they can proliferate without 
differentiating, and then figure out how, in effect, to push the same 
molecular buttons in adult stem cells. Such discoveries would enhance 
the treatment of my patients with cancer, by using transplants of adult 
stem cells taken from bone marrow. In addition, the lessons from this 
research might also help in the development of stem cell regenerative 
medical therapies for a range of other diseases. Note that the ultimate 
goal of my research is to facilitate the use of adult stem cells in the 
clinic by studying embryonic stem cells in the laboratory.
    In the years 1998-99, I was able to study a single line of human 
embryonic germ cells that was derived at Johns Hopkins. I had to be 
exceedingly careful not to use any federal funds to do these studies. 
Corporate agreements slowed and limited extensive experiments. 
Unfortunately, our research did not find the key to unlock the 
mechanisms that could turn this cell line into blood-forming cells. One 
possible reason is that this cell line seemed to have a predilection to 
develop into nerve, not blood cells. I then needed to obtain several 
other cell lines for further studies. Federal policy decisions in 2000/
2001 appeared to allow me to do this. However, these guidelines were 
put on hold in early 2001, until President Bush announced the current 
guidelines in August 2001, more than a year ago.
    The President's decision renewed our hopes of pursuing this 
therapeutic research. NIH's initial list of stem cell lines that could 
be used in federally funded research seemed like a straightforward 
source of available resources. However, we quickly found out that none 
of these cell lines was available readily to us. That remains true 
today.
    In fact, embryonic stem cell research is crawling like a 
caterpillar. While NIH has listed more eligible lines on its registry 
(http://escr.nih.gov/), only a tiny fraction of these lines are 
accessible--and only to those persistent and patient enough to jump 
through a series of hoops and endure lengthy waits. I am still waiting 
to receive my first stem cell line.
    The difficulties are numerous. As recent news articles have 
reported, and my experience has shown, some of the lines have been 
tied-up in questions of ownership. Many of the owners of lines, not in 
dispute, are not anxious to share them with other researchers. Those 
that are willing to share the lines, are not willing to do so without 
getting a piece of the profits of future discoveries made using the 
lines. The terms of material transfer agreements are often difficult 
and time-consuming to negotiate. The owners also expect an upfront fee. 
The going rate is $5,000--an amount 50-100 times greater than the $50-
$100 we are accustomed to paying for a cell line.
    While the administrative burdens necessary to obtain stem cells 
from NIH's list of eligible lines are tremendous and the costs 
significant, little is known about the lines themselves. Without this 
information, individual researchers are essentially flying blind. They 
must characterize the lines themselves and determine through a painful 
process of trial and error whether any line will advance their 
research. This is an extraordinarily inefficient use of limited 
resources.
    The best studied of the cell lines on the initial NIH-approved list 
was the H1 cell line from Wisconsin. A colleague of mine applied to 
receive these cells in fall, 2001. Finally six months later, after 
complex material transfer negotiations and a $5,000 payment, he 
received the cells. Having cleared the administrative and financial 
hurdles, the next problem he confronted was technical. These cells grow 
exceedingly slowly, one-tenth the rate of the cells we usually work 
with. So it has taken my colleague more than four additional months of 
incremental steps until he has been able to grow enough ES cells to 
perform even preliminary experiments.
    I need several ES cell lines, since I suspect from prior experience 
that not all will form blood cells, or grow rapidly. In Fall 2001, 
Reliance Life Sciences, a company from India contacted me. One of 
Reliance's scientists had been the Ph.D. mentor for a current 
postdoctoral fellow in my lab, and he knew our work well. Seven of the 
ES cell lines on NIH's current list of 81 approved hES cell lines are 
owned by Reliance, and they wanted to collaborate with my lab to 
explore the blood-forming potential of these cell lines. I spent 
several months negotiating collaboration and materials transfer, but 
the imminent agreement was abruptly cancelled in May 2002. The company 
told me that the Indian government had put an indefinite hold on 
sending human ES cells out of their country.
    Another ES cell line from Wisconsin, called H9, is reported to grow 
somewhat faster than H1, and to form some blood cells. Having learned 
in June 2002, that H9 cells would be available in July, I applied for 
this cell line. I completed the now simpler material transfer forms, 
and paid my $5,000, but I have been told that because of some technical 
problems with the H9 cells, I will not receive them until October, at 
the earliest. I look forward to these experiments, but despair of being 
able, in the near future, to obtain or afford multiple ES cell lines 
for the research I would like to do.
    My experience obtaining stem cells from NIH's approved list is not 
unique. This paper chase for stem cell lines has stunted the field of 
stem cell research. Most investigators need multiple hES cell lines. 
Few hES cell lines exist, and most are not truly available. In fact, it 
is my understanding that only the cells from Wisconsin and, as of just 
last week, one cell line from the University of California, San 
Francisco are available to scientists who are neither collaborators of 
the companies nor investigators who derived the lines. Only a few 
federal grants are trickling out for stem cell research. The review of 
a grant application is always rigorous. Scientist peer-reviewers demand 
that the applicant demonstrate experience with the cells and model 
systems proposed, and some strong preliminary results showing that the 
concepts proposed for investigation are not just wishful thinking. This 
has served as a Catch-22 for many scientists who want to study human ES 
cells, since the human ES cell lines they need simply to begin their 
research are few and costly, they grow very slowly, and the available 
cell lines may not be able to function as needed, as in my case to 
develop blood, or other specific tissue types.
    Many scientists have similar stories to tell, and you will hear 
from several eminent ones today. We all believe that stem cell research 
has tremendous potential to deliver treatments and cures. I believe 
that the pressure should be on us, as stem cell researchers to turn 
that potential into treatments for our patients. With research, we can 
make stem cells that are self-renewing, that are less likely to be 
rejected by the recipient's immune system, and which regenerate a 
variety of engineered tissues and organs that might even perform better 
than the originals. As a scientist, I want to get started. I want to 
bring these benefits to my patients and others. I do not want to limp 
along. I want other scientists to enter this field. I want to be 
spurred on by their advances.
    Today, the USA is the best place in the world for every field of 
biomedical research. I do not want us to lose that lead in stem cell 
research--and we are in danger of doing so. Without our vigorous 
leadership in federally supported stem cell research, the pace of 
discoveries will be much slower than necessary. Instead of being the 
first in line to benefit from new treatments developed at home, 
Americans will have to wait. We will lose talent to other nations. And, 
new jobs and industries will be spawned elsewhere.
    Every week, we read about exciting new stem cell research underway 
in countries, many of which have not been known, historically, as 
leaders in biomedical research. The list includes China, Singapore, 
Australia, and the U.K. The Financial Times reports that Prime Minister 
Tony Blair wants to make Britain the ``best place in the world'' for 
stem cell research, so that ``in time our scientists, together with 
those we are attracting from overseas, can develop new therapies to 
tackle brain and spinal cord repair, Alzheimer's disease, and other 
degenerative diseases such as Parkinson's.'' \1\ Singapore shares these 
ambitions. The Economist reports that the Asian nation seeks to become 
a magnet for stem cell research. In the last two years alone, it has 
invested $1.7 billion in efforts to attract global talent and industry 
and build its infrastructure to support stem cell research.\2\
---------------------------------------------------------------------------
    \1\ ``The stem of competitiveness,'' The Financial Times, August 
30, 2002.
    \2\ ``Send in the clones,'' The Economist, August 24, 2002 U.S. 
Edition.
---------------------------------------------------------------------------
    I believe it is our nation's responsibility and indeed in our 
interest not to let the discoveries, the treatments and cures, and the 
jobs that stem cell research will someday provide move overseas. We 
need to pursue all promising avenues of stem cell discovery. U.S. 
scientists need better access to human embryonic stem cells to continue 
to lead the field of stem cell research.
    I am heartened by NIH Director Elias Zerhouni's recent creation of 
a Stem Cell Task Force and look forward to its contributions to the 
field. Much work needs to be done to reduce administrative and 
technical barriers and to encourage more scientists to pursue this 
vital research.
    I also strongly endorse your fiscal year 2003 Committee Report 
language on stem cell research, urging NIH to develop a stem cell 
repository. Such an initiative would promote research by lowering the 
costs--in both time and money--of obtaining stem cell lines. Under this 
arrangement, NIH would characterize the cell lines, and then act as a 
technical resource and distribution center for scientists seeking to 
obtain them. This would eliminate duplication of effort and provide an 
invaluable technical resource for growing the cells. Vesting one 
organization with the ability to distribute all cell lines would also 
produce economies of scale.
    Once again, Mr. Chairman and members of the subcommittee, I want to 
thank you for your commitment to biomedical research and for your 
assistance in clearing unnecessary impediments to progress.

    Thank you very much, Dr. Civin.
STATEMENT OF GEORGE DALEY, M.D., Ph.D., ASSISTANT 
            PROFESSOR OF MEDICINE, HARVARD MEDICAL 
            SCHOOL; AND FELLOW, WHITEHEAD INSTITUTE FOR 
            BIOMEDICAL RESEARCH
    Senator Specter. Our next witness is Dr. George Daley, 
assistant professor of Medicine at Harvard and a fellow at the 
MIT-affiliated Whitehead Institute where he studies stem cells 
of the blood. His research has helped define the molecular 
basis for human leukemia and provided insights into normal 
blood development. Prior to his appointment at Harvard, Dr. 
Daley served as chief resident in medicine at Massachusetts 
General Hospital. His Ph.D. is from MIT and his M.D. is from 
the Harvard Medical School.
    Welcome, Dr. Daley. We look forward to your testimony.
    Dr. Daley. Thank you, Chairman Specter, distinguished 
members of the subcommittee.
    My name is George Daley. I am a faculty member at Harvard 
Medical School and I run a research laboratory at the MIT-
affiliated Whitehead Institute that studies stem cells that the 
body uses to form blood. This has prompted our intense interest 
in using human embryonic stem cells for our research. My 
laboratory currently holds NIH grants to support research on 
both mouse and human embryonic stem cell biology.
    My laboratory has spent the last 7 years using mouse ES 
cells to investigate how blood cells develop in the Petri dish. 
Recently our group has taken a step forward. We successfully 
transplanted mice with blood stem cells derived entirely from 
mouse embryonic stem cells. Then, in collaboration with my 
colleague, Rudolf Jaenisch, we performed an important first 
demonstration of therapeutic cloning to treat a mouse with a 
genetic immunodeficiency, similar to the Bubble-boy disease. 
Our team plucked a cell from the tail of an afflicted mouse, 
used nuclear transfer to create an ES cell line, used gene 
therapy to correct the genetic defect, and then performed blood 
stem cell transplants into diseased mice. The repaired ES cells 
provided a source of immune cells and antibodies in the treated 
mice.
    Encouraged by this first proof of principle in an animal 
model, my team is eager to apply the same strategy to human ES 
cells. Our hope is that one day the process will be efficient, 
safe, and effective for treating patients with a variety of 
genetic and malignant bone marrow diseases.
    However, over the past year, the progress of my own team 
and I would say that of the research community in general has 
been palpably slowed, in part because of the frustrating lack 
of access to human ES cells and in part due to the restrictive 
nature of the President's funding policy as mandated in his 
address of August 9, 2001.
    I wish to make three points.
    First, the biomedical research community needs more cell 
lines. While the President announced that over 60-odd lines 
were available, it has become increasingly clear over the past 
year that far fewer lines have been characterized adequately, 
perhaps only a handful.
    Second, the research community needs a central repository 
for ES cell lines, preferably in a facility funded by NIH, that 
would provide free access to a comprehensive set of carefully 
maintained and documented lines for research.
    Third, I wish to emphasize that the Federal funding 
guidelines are currently so restrictive that they are already 
threatening this fledgling, yet highly promising field of 
research.
    First, my personal experience. My team was one of the first 
in the United States to gain access to the ES cells that Jamie 
Thomson and his colleagues derived at the University of 
Wisconsin. However, since obtaining that single cell line in 
mid-2000, we have been frustrated in attempts to obtain 
another. One week ago, after nearly 2 years of inquiries with a 
number of other research groups, we finally received our second 
line.
    Why did it take so long? Well, the number of laboratories 
interested in working with these cells is increasing 
explosively. This dictates that a more effective means must be 
established for the distribution of these valuable reagents. I 
believe that a central warehouse and processing facility should 
be established and funded by the NIH. A central repository 
would maintain consistent, standard operating procedures for 
the culture and maintenance of the cell lines.
    Finally, I want to comment on the state of research on 
human embryonic stem cells after a year under the policy 
announced by President Bush last August. I would applaud 
President Bush for his principled stance in favor of human ES 
cell research. Having access to even a few well-characterized 
human ES cell lines enables us in the research community to 
begin to address generic questions about ES cell biology. 
However, this is only the beginning and the current policy will 
not enable the research community to follow through with the 
work needed to treat patients. President Bush made the right 
call in allowing Federal funding for research, but his policy 
excludes some of the most important and promising new avenues.
    As I have stated, it is unclear precisely how many cell 
lines exist, but I strongly believe that the number is far 
fewer than listed on the NIH registry.
    Second, the President's policy does not allow support for 
deriving new cell lines which is of tremendous scientific 
interest.
    My last point, the President's policy does not allow for 
studies of ES cell lines derived by nuclear transfer. This is 
currently the most appealing avenue for creating ES cells from 
patients with specific diseases and for creating ES cell lines 
that are genetically matched to patients. Our research team 
showed that nuclear transfer methods can be applied in the 
practice of therapeutic cloning in mice. I have no doubt that 
legitimate and successful medical treatments in real patients 
will be developed sooner if the Federal Government funds 
nuclear transfer studies with human ES cells starting today. 
The sad and undeniable truth is that the existing restrictions 
are keeping these advances from being realized.

                           prepared statement

    In conclusion, I would say that the field of ES cell 
research is in a fragile state at best under the current 
presidential policy. The current policy represents a half-
hearted effort to support this revolution in biology and 
threatens to starve the field at a time when greater 
nourishment is critical. It is a testimonial to the passion of 
the young scientists that come to my lab who are so driven by 
the enormous potential of ES cells that they are willing to 
work diligently despite the uncertainties intrinsic to the 
current policy. It is the spirit of scientific passion and 
enthusiasm, combined with a truly generous financial commitment 
to health care research by our Federal Government that has made 
American science and our health care system the envy of the 
world. As a Nation, we should not miss the opportunity to 
nurture and invigorate this exciting field of medical research.
    Thank you.
    [The statement follows:]
               Prepared Statement of Dr. George Q. Daley
    Distinguished members of the subcommittee. Thank you for the 
opportunity to address you this morning.
    My name is George Daley. I am an Assistant Professor of Medicine at 
Harvard Medical School, a Research Fellow at the MIT-affiliated 
Whitehead Institute, and a staff hematologist at the Massachusetts 
General Hospital and the Children's Hospital in Boston. I run a 
research laboratory that studies stem cells that the body uses to form 
blood. We aim to understand how blood stem cells become deranged in 
diseases such as leukemia as well as to understand how normal blood 
cells develop within the embryo. This has prompted our intense interest 
in using human embryonic stem cells--ES cells--in our research.
    My laboratory has spent the last seven years using mouse ES cells 
to investigate how blood cells develop in the Petri dish. Recently our 
group has taken a step forward. We successfully transplanted mice with 
blood stem cells derived entirely from mouse ES cells. Then, in 
collaboration with my colleague Rudolf Jaenisch, we performed an 
important first demonstration of therapeutic cloning to treat a mouse 
with a genetic immune deficiency similar to the Bubble-boy disease. Our 
team plucked a cell from the tail of an afflicted mouse, used nuclear 
transfer to generate an ES cell line, used gene therapy to correct the 
genetic defect, and then performed blood stem cell transplants into 
disease mice. The repaired ES cells provided a source of immune cells 
and antibodies in the treated mice. Encouraged by this first proof-of-
principle in mice, my team is eager to apply the same strategies to 
human ES cells. Our hope is that one day the process will be efficient, 
safe, and effective for treating patients with a variety of genetic and 
malignant bone marrow diseases.
    However, over the past year the progress of my own team and of that 
of the research community has been palpably slowed, in part because of 
the frustrating lack of access to human ES cells, and in part due to 
the restrictive nature of the President's funding policy as mandated in 
his address of August 9, 2001.
    I will make three points. First, the biomedical research community 
needs more cell lines. While the President announced that over 60-odd 
cell lines were available as of August 9, 2001, it has become 
increasingly clear that far fewer lines have been characterized 
adequately. Some two thirds of the cell lines are located outside of 
the United States, and most are controlled by commercial entities. 
Currently, only a handful of lines are available to U.S. scientists.
    Second, the biomedical research community needs a central 
repository for embryonic stem cell lines, preferably in a facility 
funded by NIH, that would serve the needs of the research community as 
a whole, providing free access to a comprehensive set of carefully 
maintained and documented human ES lines for research.
    And third, I wish to emphasize that the federal funding guidelines 
are currently so restrictive that they are already threatening this 
fledgling yet highly promising field of biomedical research.
    First, my personal experience. My team was one of the first in the 
United States to gain access to the human ES cells that Jamie Thomson 
and his colleagues derived at the University of Wisconsin. However, 
since obtaining a single line of cells in mid-2000, we have been 
frustrated in attempts to obtain additional lines. Many experiments 
require comparing the behavior of several different embryonic stem cell 
lines, since the behavior of any single line may be atypical and 
therefore highly misleading. One week ago, after nearly two years of 
inquiries with number of other research groups, we finally received a 
second cell line.
    Why did it take so long? What are the hurdles that hinder sharing 
of these critically important research tools? What might be done to 
remedy the situation? The issues are complex. It is human nature that 
some scientists might wish to preserve their monopoly over these 
valuable cell lines. However, an accepted norm within our field, and a 
stipulation for publishing in most if not all journals, is that all 
research reagents will be made readily available to the research 
community to enable research results to be replicated and extended. 
Typically, when we request a research tool from a colleague in the 
research community, we receive it immediately, especially in this era 
of email and FEDEX. For all but a small handful of human ES cell lines 
listed on the NIH registry, this simply is not happening.
    There is the perception that human ES cells hold significant 
commercial value given their potential for yielding products. Companies 
control most of the lines, and they have worked aggressively to 
dominate the intellectual property that flows from these cells. Thus, 
protracted negotiations over Material Transfer Agreements--called 
MTAs--have slowed the sharing of these lines with the wider research 
community. One of the most valuable initiatives performed by the U.S. 
Public Health Service and the NIH since August 9, 2001, was to 
negotiate with the University of Wisconsin a Memorandum of 
Understanding that provides a common set of terms for all federally 
funded researchers.
    We have spent significant effort over the last two years 
negotiating MTAs with four different groups. Our most recent experience 
is a testimonial on how these negotiations should and can work. One of 
my post-doctoral scientists met Dr. Meri Firpo of the University of 
California, San Francisco, at a scientific meeting in August. Dr. Firpo 
had received a grant from the NIH to build an infrastructure to 
disseminate the lines, and graciously agreed to expedite our request. 
Our MTA negotiations were simplified by the prior agreements that had 
been hammered out by the NIH. Exactly 8 days ago, only some 6 weeks 
after originally speaking with Dr. Firpo, we received a second strain 
of human embryonic stem cells. We also received a handbook of detailed 
recipes for growing and maintaining the cells, and data on the 
characterization of the cell line. Clearly, Dr. Firpo is providing an 
exceptional service and her behavior should become a standard for how 
valuable embryonic stem cell lines are distributed to the scientific 
community.
    The number of laboratories interested in working with these lines 
is already large and is increasingly explosively. This dictates that a 
more effective means must be established for the distribution of these 
valuable research reagents. I believe that a central warehouse and 
processing laboratory should be established and funded by the NIH to 
facilitate greater access to human ES cell lines for the general 
community of biomedical researchers. A central repository would 
maintain consistent standard operating procedures for the culture and 
maintenance of the cell lines. Strict quality control parameters would 
be established, and cell lines would be faithfully characterized under 
a uniform set of conditions. The Medical Research Council of the UK has 
just announced that it would fund such an effort in that country.
    Finally, I wish to comment on the state of research on human ES 
cells after a year under the policy announced by President Bush last 
August 9, 2001. I would applaud President Bush for his principled 
stance in favor of human ES cell research. Having access to even a few 
well-characterized human ES cell lines enables many of us in the 
research community to begin to address generic questions about ES cell 
biology. We can make advances in cultivation of the cells, in coaxing 
the cells to become blood cells, neurons, insulin-producing cells, and 
the like. But this work is only the beginning, and the current policy 
will not enable the research community to follow-through with the work 
needed to treat patients. President Bush made the right call in 
allowing Federal funding for research, but his policy excludes some of 
the most important and promising avenues, and critical features of the 
policy are tying the hands of the research community.
    First, the President announced that research support would be 
tendered only for cell lines that pre-existed before August 9, 2001. As 
I have stated, it is unclear precisely how many cell lines exist but I 
strongly believe that the number is far fewer than listed on the NIH 
registry. The President's policy prevents U.S. scientists from 
exploiting new cell lines as they become available. Scientists are by 
their very nature innovators, and hungry for the latest, most up-to-
date technology and tools. All human ES cells listed on the NIH 
registry were derived in contact with mouse feeder cells. Scientists 
throughout the world are actively seeking to develop new cell lines 
that avoid this contamination, and would therefore be more valuable for 
generating human therapies. Indeed, scientists from Singapore have 
published the derivation of lines free of mouse cell contamination, but 
under the current policy, U.S. scientists can not study these cells 
using federal funds.
    Second, the President's policy does not allow support for deriving 
new cell lines, which is of tremendous scientific interest. This is in 
stark contrast to the United Kingdom, whose scientists have made many 
of the seminal discoveries in ES cell biology and given their greater 
freedom are poised to dominate further. Our research community, hobbled 
by current restrictions, is falling behind researchers in other 
countries that are racing ahead to take full advantage of the 
possibilities that embryonic stem cells offer.
    Third, the President's policy does not allow for studies of ES cell 
lines derived by nuclear transfer. This is currently the most appealing 
avenue for creating ES cells from patients with specific diseases, and 
for creating ES cell lines that are genetically matched to patients, 
which would overcome immune rejection of transplanted ES cell products. 
Our research team showed that nuclear transfer methods can be applied 
in the practice of therapeutic cloning in mice. I have no doubt that 
legitimate and successful medical treatments in real patients will be 
developed sooner if the Federal government funded nuclear transfer 
studies with human cells today. The sad and undeniable truth is that 
the existing restrictions are keeping these advances from being 
realized.
    I realize that funding for some of these initiatives is currently 
prohibited by federal statute and that a change in legislation to 
specifically allow this work is needed. I applaud you Senator Specter, 
in your efforts to propose such legislation.
    In conclusion, I would say that the field of human ES cell research 
is in a fragile state at best under the current Presidential policy. 
The current policy represents a half-hearted effort, and threatens to 
starve the field at a time when greater nourishment is critical. The 
scientists who come to train in my lab voice concerns that they might 
face inadequate research support in the future. It is a testimonial to 
the passion of these young scientists, who are so driven by the 
enormous potential of ES cells that they are willing to work diligently 
despite the uncertainties inherent under the current policy. It is that 
spirit of scientific passion and enthusiasm--combined with a truly 
generous financial commitment to health care research by our Federal 
government, that has made American biomedical science and our health 
care system the envy of the world.
    Here at the beginning of the 21st century, we stand at the 
threshold of a new era in biomedicine, when cells will be harnessed to 
treat a wide array of degenerative conditions in an aging society. As a 
nation, we should not miss the opportunity to nurture and invigorate 
this exciting field of biomedical research.
    Thank you for your time and interest in this matter.

    Senator Specter. Thank you very much, Dr. Daley.
    Dr. Zerhouni, we start with you on the questioning. You 
heard the testimony about the sharp limitations on the 
availability of stem cell research lines. In the fall of last 
year when NIH assembled the stem cell registry, it listed 78 
stem cell lines from 14 sources around the world. According to 
the information which our subcommittee has been able to glean, 
only five of these lines are available to stem cell 
researchers. NIH has awarded, as you testified, $4.3 million in 
infrastructure grants to five companies and institutions with 
23 eligible stem cell lines. Of these five companies, we are 
told only four have signed material transfer agreements with 
NIH, and these four companies have only 17 eligible stem cell 
lines. Of these 17 stem cell lines, only 5 have been shipped 
and are available to researchers.
    Would you start the clock? We are going to have 5-minute 
rounds.
    The question is, is there a sufficient number of stem cell 
lines available for the required research?
    Dr. Zerhouni. Well, in terms of the number of eligible 
lines which is, as you said, 78, and the number of lines 
available for wide distribution, we would agree that over the 
year there has been an increasing number of lines. Last spring 
there was one. This month there are five. As far as we can tell 
from talking to all the suppliers, there are 10. I went over in 
my opening statement the time line that it takes to go from an 
eligible line that is just derived to a widely available 
distributable line.
    So I think that progress is being made and we are, as 
mentioned by some of the researchers, diligently working with 
as many sources as we can to make more lines available.
    Senator Specter. You are working with the sources of the 78 
lines which were approved by the President as of August 9.
    Dr. Zerhouni. Correct.
    Senator Specter. And the question is, is that adequate? I 
know you are bound by the administration's decision, but the 
Congress has the authority to legislate in the field. The 
President may veto it but we can override a veto by two-thirds. 
In the spring of 2000, when NIH funding was not available on 
all of the stem cell research, Senator Harkin, myself, and 
others started a move, and we had letters signed by 64 Senators 
disagreeing with the Federal policy on stem cells. I had 
personal commitments from 12 more who were unwilling to put it 
in writing, but assured me that they would support legislation 
in the field.
    Then the President came out with his compromise position. 
In essence, on August 9, and immediately after that, there was 
a flurry of publicity as to whether those lines were adequate. 
And the indications were at that time that they were not.
    After September 11, all of the oxygen has been sucked out 
of Washington on virtually every other subject until Iraq came 
along to take some precedence. And we have been waiting to see 
the developments and have deferred this hearing, but now we are 
going to be faced with a decision as to what to do next.
    So it is a pretty blunt question as to whether the existing 
stem cell lines are adequate. You have heard your colleagues at 
the table. What do you think?
    Dr. Zerhouni. My feeling is that we are at a very early 
stage. It is actually not knowable how many lines you will need 
to advance the field. In other comparable fields, very often 
researchers want to limit the number of well-characterized 
lines that are used for experimentation.
    So I would say at this moment, I do not think we know the 
answer. We need to work and develop more researchers and more 
laboratories that are going to experiment with the lines that 
we want to make available to find that answer. I really do not 
know the answer.
    Senator Specter. Well, Dr. Zerhouni, when will we know? Can 
you give me a time line as to when we will know?
    Dr. Zerhouni. We will work as diligently as we can to make 
as many lines available to as many laboratories. There is no 
limit to the funding that we can direct to the laboratories 
that present good, solid research proposals.
    Senator Specter. Dr. Pedersen, to what extent do you think 
your example will be followed by others, in leaving the United 
States to go to places like Great Britain where you can get 
funding on stem cell research?
    Dr. Pedersen. I cannot answer that exactly, Senator, but we 
are working as diligently as possible to recruit them.
    Senator Specter. Have you had any success?
    Dr. Pedersen. How I would like to answer that is in part a 
response to your prior question, which is how many cell lines 
are available to do the necessary work and how many are needed. 
There are, I think, adequate numbers of existing cell lines to 
do a portion of the work, the portion that will be focused on 
in the United States with Federal funding, namely, the 
characterization of the steps needed to get useful, specialized 
cells. So the dozen or so that are currently available, 
actually available probably are sufficient to do that.
    I think this number issue is a red herring, though, because 
to go into patient care, the existing lines are not really 
useful because they have all been grown with a combination of 
mouse cells which makes them unsuitable for transplantation. So 
the actual number of suitable lines for transplantation is zero 
in the current set, and new lines must be generated. So the 
clinical delivery very likely will take place elsewhere and 
people who are interested in participating in that part of it 
will probably have to do so elsewhere.
    Senator Specter. So you are saying the number of stem cell 
lines for clinical delivery, transplantation is zero.
    Dr. Pedersen. On the registry is zero. There is one line 
that was developed recently in Singapore.
    Senator Specter. Well, that could hardly be characterized 
as an adequate number.
    Dr. Pedersen. And so the development of such lines in the 
U.S. would require a change in policy.
    Senator Specter. My red light is on, plus 39 seconds from 
your last answer.
    Senator Hutchison.

           OPENING STATEMENT OF SENATOR KAY BAILEY HUTCHISON

    Senator Hutchison. Well, thank you, Mr. Chairman, for 
holding this hearing, and I thank all of you for coming.
    I am very concerned about the real-life stories. I always 
am interested when policies are set, and I think the 
President's policy was meant to be one that would allow for 
real use of the lines that are available so that we could do 
the testing that is necessary. But then I hear your stories 
about trying to get lines and then India does not let them out, 
and then you try to get a line in another way from Wisconsin 
and, yes, it is coming, well, no it is not, well, yes, it is 
later. That does not seem to be working in the practical sense.
    I have researchers at UT Southwestern who are very 
concerned about availability. They are now doing work with 
animal nerve and pancreatic stem cells. They want to further 
that study, and they are very concerned about access.
    So I would just ask you this question. I think that Senator 
Specter asked the big question, what can we do to make it more 
practically available? I think all of us are concerned about 
that.
    On top of that, though, I now have another concern, and 
that is I have some great research institutions, M.D. Anderson, 
Baylor, UT Southwestern, UT San Antonio, UT Galveston. They are 
really on the cutting edge of research. Now California has 
stepped forward I think in a way that could start luring some 
of our good scientists to California, and I would like to ask 
you this question, Dr. Zerhouni.
    Now that California is looking at giving State help in this 
regard and so many of our institutions are public institutions 
that do get private help but also need government help, what 
can we do to keep everything balanced so that all of a sudden 
we do not see people migrating from Harvard and Johns Hopkins 
and all these other great research institutions, including 
mine, all to the west coast? I am not saying I do not wish the 
west coast well. I do, but I do not want to, all of a sudden, 
throw a big kink in the research world. So tell me how we can 
deal with that effectively.
    Dr. Zerhouni. Well, clearly I think, in terms of Federal 
funding, we need to look at all the aspects of facilitation of 
the research, and that is what we are doing. We have 
established the Stem cell Task Force where we can get input 
from the scientific community from all States as to exactly how 
we can, in fact, enhance the ability of these institutions to 
do the research. As Dr. Pedersen said, we need to do the basic 
research before we can go to clinical research. It will take 
years, but we need to do that.
    In terms of State support and private support, there is 
nothing in the current policy that prevents that from 
happening. In California, for example, the UCSF lines were 
developed in part with State help.
    Senator Hutchison. I understand that, Dr. Zerhouni. I know 
it is possible, but the amount that is available from private 
and State funds is not comparable to what could be available 
from Federal funds. So you are now looking at a potential 
problem I think even though private funds can be used, but it 
does become complicated, especially if you are doing a project 
that has Federal funds and then you want to take the next step. 
You want to go to the stem cell part that would actually show 
results. Do you have to stop doing the federally funded 
research?
    Dr. Zerhouni. Currently we allow researchers to do side-by-
side federally funded research and non-federally funded 
research in parallel. There is no NIH policy that prevents that 
as long as appropriate accounting mechanisms are in place. So I 
think that can still be done.
    Senator Hutchison. And it can be the same, exact project. 
So you are on a project and the next thing you want to do is 
test it with embryonic stem cells, and you have a federally 
funded project. Can you use embryonic stem cells under the 
present law or the President's policy to do that research?
    Dr. Zerhouni. As long as there is strict accounting of what 
is used. The present policy is very clear. You cannot use 
Federal funds for non-eligible lines, and you have to have in 
place accounting mechanisms which are clearly spelled out in 
our policies to be able to do that, but it is doable.
    Senator Hutchison. I would like to ask other members of the 
panel if they feel that that differentiation is enough to allow 
people to go forward.
    Dr. Civin. Senator Hutchison, I was thinking of a slightly 
different analogy, in terms of California, when I read this in 
the news, and I was hoping that Maryland would do the same 
thing and that other States would follow Justice Brandeis' 
suggestion I believe, that the States should be the 
laboratories of democracy in this area of stem cell research 
and that we should experiment with different solutions. I 
happen to think that the solution from the State of California 
is outstanding and would like to see that be the example for 
our Federal solution that is followed by the entire Government 
because this is where the money that will make the rubber hit 
the road will happen.
    Very little funding realistically will come from the States 
for very much research. It has to be Federal funding for it to 
work. Ultimately it has to be a coherent Federal policy so that 
we can collaborate across the country on our research. But I 
see instead California as an example of a laboratory for 
democracy here.
    Senator Hutchison. Dr. Schatten.
    Dr. Schatten. Senator Hutchison, thank you so much for 
these probing questions. I think we need to acknowledge that 
Dr. Zerhouni and the entire NIH is doing absolutely everything 
within their power, but it is not within their power. I know 
from my own experiences that in order for us to work on eggs 
that have failed to be fertilized from an IVF clinic, we need 
absolutely everything to be privately funded, even million 
dollar microscopes that we might use for just 10 minutes.
    Many university administrators are terrified that their 
full Federal funding could be withdrawn if one investigator 
enters into an area that might pose a risk because of a 
confusion in how funds are either mingled or not commingled. I 
think really it is at this level, as Senator Specter has 
mentioned, that we could benefit from a clear national policy.
    Thank you.
    Senator Hutchison. Thank you.
    I have seen the difference that the doubling of the NIH 
budget has made in the area of research, particularly into 
diseases that are not prolific diseases but nevertheless need 
to have research into them. I know that the availability of 
Federal funding is making a huge difference in the knowledge 
base that we have in medical research into so many diseases 
that have been ignored in the past.
    So I would just say that I think Dr. Schatten's point is 
very good. I think NIH is doing everything they can. I think 
they are moving forward in every possible way with the policies 
that we have, but I just want to see what more we can do and 
also determine if something works in practicality after it is 
set forward.
    I even think the President meant to do that. I think he 
said we are going to try this. This is the best way to approach 
it, in a very careful way, and he wanted to be careful because 
he values life so much. So I know his intentions were right, 
but I also think he left it open for us to come back and say--
not me, but you, the research community and you, Dr. Zerhouni--
he is going to look to you for advice to say did it work. How 
can we continue progress in a way that also gives value to 
human life? I think we just have to keep working on input from 
the research community and creativity, which I think Senator 
Specter is showing in trying to create a bank at NIH perhaps 
with some of these lines to make sure that they do not get into 
legalistic delays and bureaucratic stumbling that stop progress 
in America.
    I want this research to be done in America, and I want you 
to come home.
    Senator Specter. Thank you very much, Senator Hutchison.
    We are now about 9 minutes into a 15-minute vote, so we are 
going to have to adjourn, as I had mentioned earlier.
    What I would like you to do, Dr. Schatten, Dr. Civin, Dr. 
Daley, Senator Ortiz, and Dr. Pedersen, is to provide in 
writing what you would like to have available. You, Dr. Daley, 
talked about nuclear transfer studies. I would like to get the 
specifics as to what you have in mind. Dr. Civin commented 
about waiting a year for lines and he has not gotten them yet. 
Dr. Schatten talked about not enough lines available and 
worried about therapeutic cloning, as Senator Ortiz said.
    I believe that the legislation passed by the House imposing 
criminal penalties on nuclear transplantation, or so-called 
therapeutic cloning, will not come to pass. We have stopped it 
in the Senate. We may have 60 votes if there is time on the 
calendar to pass a bill which would permit nuclear 
transplantation.
    I think Senator Hutchison summarized the matter well. The 
President took a significant step on August the 9 in permitting 
some Federal funding. He was subjected to a lot of criticism. 
It is pretty hard not to be criticized on virtually anything 
the President or any of us in elective office do, so we are 
used to that. But then we have to see what has happened.
    I would like you to respond specifically to what you would 
like to see done. Senator Ortiz, you have special insights into 
legislation. Give me your insights as to what legislation you 
would like to see done.
    My own sense at the moment is that Congress is going to 
have to legislate on the subject. We had waited for a year-plus 
to see the experience.
    Dr. Zerhouni, if you can supplement what you have said with 
a time line as to when you think you might know, we would be 
interested to know that.
    But I think the time has come to legislate in the field. As 
I said, last year we had 64 Senators in writing and commitments 
from 12 more, and 12 and 64 are 76, which is 9 more than 67. So 
we are in a position to move where the need is sufficiently 
great.
    I think this testimony has been very, very helpful. I think 
we have gotten the kernel of it, and your written answers will 
give us the balance. So thank you all very much for coming in.

                     ADDITIONAL SUBMITTED STATEMENT

    We have received the prepared statement of Senator Larry 
Craig which will be placed in the record.
    [The statement follows:]
               Prepared Statement of Senator Larry Craig
    Mr. Chairman, I would like to thank you for holding this hearing 
today to spotlight stem cell research. I would also like to thank all 
of our witnesses here today for taking the opportunity to address this 
very complex issue.
    Stem cell research continues to offer a great deal of promise. This 
research could lead to exponential improvements in the treatment of 
many terminal and debilitating conditions. In many cases, researchers 
are already beginning to see the promise of this research.
    With the President's decision last year to allow federal funds to 
be used to support research on existing stem cell lines, researchers 
have begun to make progress. Many scientists in this field of research 
confirm that there are ample number of cell lines available to fully 
understand how these cells work. We must encourage more researchers to 
take advantage of this historic opportunity.
    Researchers are in the early stages of this process. It is a 
difficult science and should be dealt with at an appropriate pace. Many 
will say that this process is moving too slowly. However, there are 
many scientific hurdles that will have to be surmounted before we get 
to the point of actually replacing damaged tissues in the body and 
understanding the potential for clinical applications. This could take 
years. But we should invest the time and resources into doing what it 
takes to get to that point.
    I understand that NIH has made a good-faith effort to facilitate 
the use of the existing stem cell lines and has created a framework to 
enable researchers to begin stem cell research in the United States. 
Understanding that there are many obstacles that must be overcome 
before major strides are made, we must be as supportive of this 
research as possible.
    Again, I thank the Chairman for holding this hearing and look 
forward to learning more about the developments in this important 
research.

                         CONCLUSION OF HEARING

    Senator Specter. Thank you all very much for being here, 
that concludes our hearing.
    [Whereupon, at 10:45 a.m., Wednesday, September 25, the 
hearing was concluded, and the subcommittee was recessed, to 
reconvene subject to the call of the Chair.]

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