[Senate Hearing 108-959]
[From the U.S. Government Publishing Office]




                                                        S. Hrg. 108-959
 
                       ADULT STEM CELL RESEARCH: 
                        SUCCESSES FROM THE FIELD 

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

                                HEARING

                               before the

                 SUBCOMMITTEE ON SCIENCE, TECHNOLOGY, 
                               AND SPACE

                                 of the

                         COMMITTEE ON COMMERCE,
                      SCIENCE, AND TRANSPORTATION
                          UNITED STATES SENATE

                      ONE HUNDRED EIGHTH CONGRESS

                             SECOND SESSION

                               __________

                             JULY 14, 2004

                               __________

    Printed for the use of the Committee on Commerce, Science, and 
                             Transportation


                         U.S. GOVERNMENT PRINTING OFFICE 

81-637 PDF                       WASHINGTON : 2013

For sale by the Superintendent of Documents, U.S. Government Printing 
Office, Internet: bookstore.gpo.gov Phone: toll free (866) 512-1800;
DC area (202) 512-1800 Fax: (202) 512-2104 Mail: Stop IDCC, 
Washington, DC 20402-0001 



       SENATE COMMITTEE ON COMMERCE, SCIENCE, AND TRANSPORTATION

                      ONE HUNDRED EIGHTH CONGRESS

                             SECOND SESSION

                     JOHN McCAIN, Arizona, Chairman
TED STEVENS, Alaska                  ERNEST F. HOLLINGS, South 
CONRAD BURNS, Montana                    Carolina, Ranking
TRENT LOTT, Mississippi              DANIEL K. INOUYE, Hawaii
KAY BAILEY HUTCHISON, Texas          JOHN D. ROCKEFELLER IV, West 
OLYMPIA J. SNOWE, Maine                  Virginia
SAM BROWNBACK, Kansas                JOHN F. KERRY, Massachusetts
GORDON H. SMITH, Oregon              JOHN B. BREAUX, Louisiana
PETER G. FITZGERALD, Illinois        BYRON L. DORGAN, North Dakota
JOHN ENSIGN, Nevada                  RON WYDEN, Oregon
GEORGE ALLEN, Virginia               BARBARA BOXER, California
JOHN E. SUNUNU, New Hampshire        BILL NELSON, Florida
                                     MARIA CANTWELL, Washington
                                     FRANK R. LAUTENBERG, New Jersey
      Jeanne Bumpus, Republican Staff Director and General Counsel
             Robert W. Chamberlin, Republican Chief Counsel
      Kevin D. Kayes, Democratic Staff Director and Chief Counsel
                Gregg Elias, Democratic General Counsel
                                 ------                                

             SUBCOMMITTEE ON SCIENCE, TECHNOLOGY, AND SPACE

                    SAM BROWNBACK, Kansas, Chairman
TED STEVENS, Alaska                  JOHN B. BREAUX, Louisiana, Ranking
CONRAD BURNS, Montana                JOHN D. ROCKEFELLER IV, West 
TRENT LOTT, Mississippi                  Virginia
KAY BAILEY HUTCHISON, Texas          JOHN F. KERRY, Massachusetts
JOHN ENSIGN, Nevada                  BYRON L. DORGAN, North Dakota
GEORGE ALLEN, Virginia               RON WYDEN, Oregon
JOHN E. SUNUNU, New Hampshire        BILL NELSON, Florida
                                     FRANK R. LAUTENBERG, New Jersey



                            C O N T E N T S

                              ----------                              
                                                                   Page
Hearing held on July 14, 2004....................................     1
Statement of Senator Brownback...................................     1
Statement of Senator Lautenberg..................................     3
Statement of Senator Nelson......................................     5
Statement of Senator Wyden.......................................     2

                               Witnesses

Dominguez, Laura.................................................    49
    Prepared statement...........................................    51
Fajt, Susan R., Spinal Cord Injured Recipient of Olfactory Mucosa 
  Transplantation................................................    52
    Prepared statement...........................................    54
Goldstein, Robert, Chief Scientific Officer, Juvenile Diabetes 
  Research Foundation International (JDRF).......................    58
    Prepared statement...........................................    60
Gonzalez, Hon. Charles A., U.S. Representative from Texas........    48
Levesque, M.D., Michel F., FRCS(C), FACS, Cedars-Sinai Medical 
  Center, Los Angeles, California; Associate Clinical Professor, 
  UCLA School of Medicine and Member of UCLA Brain Institute; 
  Chairman, Foundation for Neural Repair.........................     5
    Prepared statement...........................................     8
Peduzzi-Nelson, Ph.D., Jean D., Department of Physiological 
  Optics, University of Alabama at Birmingham....................    17
    Prepared statement...........................................    19
Turner, Dennis...................................................    56
    Prepared statement...........................................    57
Weissman, M.D., Irving, Karel and Avice Beekhuis Professor of 
  Cancer Biology, Director of the Institute of Cancer and Stem 
  Cell Biology, and Professor in the Departments of Pathology, 
  Developmental Biology, and Biology, Stanford University School 
  of Medicine....................................................    30
    Prepared statement...........................................    32

                                Appendix

Armitage, Faye, Jacksonville, FL, prepared statement.............    73


                       ADULT STEM CELL RESEARCH: 
                        SUCCESSES FROM THE FIELD

                              ----------                              


                        WEDNESDAY, JULY 14, 2004

                               U.S. Senate,
    Subcommittee on Science, Technology, and Space,
        Committee on Commerce, Science, and Transportation,
                                                    Washington, DC.
    The Subcommittee met, pursuant to notice, at 2:35 p.m. in 
room SR-253, Russell Senate Office Building, Hon. Sam 
Brownback, Chairman of the Subcommittee, presiding.

           OPENING STATEMENT OF HON. SAM BROWNBACK, 
                    U.S. SENATOR FROM KANSAS

    Senator Brownback. This hearing will come to order. Thank 
you all for joining us today in an exciting hearing.
    Today's hearing is about miracles and answered prayers. 
People have prayed for cures to lives destroyed by accidents 
and ravaged by diseases. You'll see, on video, paraplegics 
walk--with aid, but walking, nonetheless. You will see 
Parkinson's dealt with--still with difficulty, but being dealt 
with. Today's hearing's about miracles, prayers answered, 
prayers yet to be answered, because we have much yet to do.
    I'm delighted to be joined by my colleagues on this 
discussion about adult stem cell research. That's what the 
hearing will be about, that's what it will focus on. This is a 
noncontroversial area in stem cells. We've all heard a lot 
about stem cells. The adult stem cell area, umbilical cord-
blood stem cells are ones that nobody disagrees with, that 
everybody is supportive of. Everybody is supportive of the 
scientific research, and the things that are taking place are 
absolutely profound--I would put in the category of miraculous 
and answers to prayer. And you will see some of that on display 
today.
    But there is much to be done. I think we need to continue 
the funding aggressively in the field adult stem cell research, 
in pushing that forward so that we can find more cures for more 
types of diseases that ravage the body and that hurt us all.
    We will not be dealing with the issue of embryonic stem 
cells today in the hearing. The hearing's focused on adult stem 
cells. That's what we've intended to put forward and to try to 
address at this hearing. There is a controversial area on stem 
cells, in the embryonic field. That has been the subject of a 
number of different hearings, particularly in the 
Appropriations Committee, and we'll not be addressing that 
issue today.
    My hope is that at this hearing we will be able to engage 
people in a dialog of hope; and be able to show people with 
devastating diseases or injuries promise and hope to move 
forward in life. We'll have a panel of experts and a panel of 
patients--experts to tell us what is taking place in the field; 
the patients to show us what's taking place in their lives.
    And I look forward to having this good news hearing. There 
are always many controversial subjects; we face many difficult 
subjects. This is a good news hearing. We've got much yet to 
do, but there is some good news to be celebrated here today.
    With that, I look forward to the presentations, and I want 
to turn to the Ranking Member, Senator Wyden, for his opening 
statement.

                 STATEMENT OF HON. RON WYDEN, 
                    U.S. SENATOR FROM OREGON

    Senator Wyden. Thank you very much, Mr. Chairman. And, as 
always, you know how much I enjoy working with you. And having 
chaired this Subcommittee in the past, I'm acutely aware that 
this issue generates such passions that it is almost 
physiologically impossible to be unaware of the politics of 
stem cell research. No Senator who participates in a hearing on 
this subject could possibly feel that they're being recast in 
an updated version of the movie Casablanca, and then pipe up 
that they're just shocked and absolutely amazed about the 
presence of politics.
    Now, that having been said, I'm hopeful--as I think your 
opening statement indicated, Mr. Chairman--I'm hopeful that 
this hearing can help inject, if ever so slightly, a bit more 
nonpartisanship with respect to this issue. And I come to this 
issue in a nonpartisan way with the view that I think while the 
research shows that using adult stem cells can help some 
people, there are millions of Americans who suffer from a host 
of devastating diseases, and their valiantly supportive 
families, who I believe deserve more.
    And my concerns with respect to this issue and the science, 
Mr. Chairman, can be summed up in just one paragraph that I 
pulled off the NIH website a few minutes ago. On the NIH 
website, there's a section called ``Facts on Stem Cells.'' And 
I would just like to read into the record one paragraph with 
respect to what is on the government's official website with 
respect to how adult stem cells are used and the opportunities 
that they present to the American people.
    I quote here, ``There are currently several limitations to 
using adult stem cells. Although many different kinds of 
multipotent stem cells have been identified, adult stem cells 
that could give rise to all cell and tissue types have not yet 
been found. Adult stem cells are often present in only minute 
quantities, and they can, therefore, be difficult to isolate 
and purify. There is also evidence that they may not have the 
same capacity to multiple as embryonic stem cells do. Finally, 
adult stem cells may contain more DNA abnormalities caused by 
sunlight, toxins, and errors in making more DNA copies during 
the course of a lifetime. These potential weaknesses might 
limit the usefulness of adult stem cells.''
    Mr. Chairman, I would just ask that the ``Facts on Stem 
Cells,'' the portion of which I've written, could be entered 
into the record at this point.
    Senator Brownback. Without objection.
    [The information referred to follows:]

    An excerpt from the NIH website (http://stemcells.nih.gov/info/
faqs.asp)

    There are currently several limitations to using adult stem cells. 
Although many different kinds of multipotent stem cells have been 
identified, adult stem cells that could give rise to all cell and 
tissue types have not yet been found. Adult stem cells are often 
present in only minute quantities and can therefore be difficult to 
isolate and purify. There is also evidence that they may not have the 
same capacity to multiply as embryonic stem cells do. Finally, adult 
stem cells may contain more DNA abnormalities--caused by sunlight, 
toxins, and errors in making more DNA copies during the course of a 
lifetime. These potential weaknesses might limit the usefulness of 
adult stem cells.

    Senator Wyden. Mr. Chairman, let me just close by way of 
saying I think we are going to have a chance to explore this 
issue in some detail. There are a lot of ramifications to it. 
I'm interested in talking to the scientists, for example, with 
respect to how this is going to affect private-sector research. 
Given the government's limitations on funding this research, I 
think it's going to have debilitating effects, in terms of 
generating the dollars that are going to be needed for private-
sector research into other areas. But, more than anything, I 
come today--and you and I have worked together on so many 
areas--I come to say that I'm very much aware of the passions 
on this issue, and--all sides--and I am hopeful that we can use 
this hearing to try to find a bit more common ground, because 
that's what the American people, I think, are calling for in 
this area, so that we can find the cures and therapies that you 
correctly stated in your opening statement would give families 
hope. And I look forward to working with you on this.
    Senator Brownback. Thank you, Senator Wyden.
    Senator Lautenberg?

            STATEMENT OF HON. FRANK R. LAUTENBERG, 
                  U.S. SENATOR FROM NEW JERSEY

    Senator Lautenberg. Thanks, Mr. Chairman.
    I think it's fair to say that we'll all agree that stem 
cell research is critical to our mission to fight and cure 
disease in this country and throughout the world. 
Unfortunately, this research continues to become embroiled in a 
political controversy. And I listened very carefully to the 
Chairman's delineation of the ground that we're going to cover, 
and I think it's, sort of, akin to a discussion on research on 
cancer that we say, ``Well, we can only look at one type of 
treatment. We can only look at radiation. We can only look at 
diet, or we can only look at chemotherapy or something.'' And 
if you want to cure cancer, I don't think you can put out some 
of those methods that work. Some need one another to work well. 
And the debate over whether we should pursue adult stem cell 
research or embryonic stem cell research sets up an 
unreasonable choice.
    Both types of stem cell research should be pursued 
simultaneously. Each offers the potential for cures. Neither is 
a substitute for the other. No promising stem cell research 
should be stopped. Stem cell research, particularly the 
burgeoning field of embryonic stem cell research, has 
tremendous potential to help us better understand, treat, and 
even cure deadly and disabling diseases like diabetes and 
cancer, Parkinson's, Alzheimer's, and Multiple Sclerosis. Stem 
cell research could help us cut the incidence of heart disease, 
the Nation's leading killer.
    Most Americans support stem cell research, as do Members of 
Congress from both sides of the political aisle. And former 
First Lady Nancy Reagan, who spent 10 years watching her 
husband suffer from Alzheimer's, is a stringent advocate. 
Virtually every major medical, scientific, and patient advocacy 
groups support embryonic cell research. And I'm talking about 
the American Medical Association, the Federation of American 
Societies for Experimental Biology, the Juvenile Diabetes 
Research Foundation, the Parkinson's Action Network.
    In my view, President Bush's stem cell research policy does 
sacrifice some sound science, and I wish it weren't so. 
President Bush's stem cell research policy is, in effect, 
denying millions of people suffering from physically and 
mentally debilitating diseases, illnesses, and injuries from 
being cured.
    And I know that the views of those--and I have great 
respect for the Chairman--of those who oppose embryonic stem 
cell research are sincere. But I've met with too many diabetic 
children and their families. I've see how much they suffer, and 
I simply can't tell these children or their parents that, in 
the hierarchy of rights, a week-old undifferentiated cell is 
more important than they are and cannot be used in researching, 
treating, or possibly curing their terrible disease.
    The millions of men, women, and children who are suffering 
from diabetes and other life threatening diseases, illnesses, 
and injuries are engaged in a race against time. Talk to these 
children, and understand how uncomfortable life is, even as 
they live it precariously. And it's our responsibility to make 
sure that they benefit as quickly as possible from the wonders 
that modern science, medicine, and technology have to offer.
    Now, Mr. Chairman, one of the things that I am very proud 
of in my lifetime is a facility called the Lautenberg Cancer 
Research Center, named for my father, who died when he was 43 
years old, and was a health faddist, as in those days. But when 
cancer overtook, there was no way to overcome.
    And one of our outstanding witnesses here, Dr. Weissman, is 
going to be testifying, and he's just come back from a one week 
lecture at the Lautenberg Cancer Research Institute, which is 
in Jerusalem, where a friend of mine moved many years ago and 
asked if I would help in establishing a Lautenberg Cancer 
Research Center, and I, fortunately, was able to provide the 
funding for it, and I look forward to his testimony. And I 
review the work that we do at the Lautenberg Cancer Research 
Center, and stem cells are an important part of the agenda. And 
I hope that we'll be able to move the debate along so that we 
don't engage in a political difference and permit science to 
run its own course.
    Thank you.
    Senator Brownback. Senator Nelson?

                STATEMENT OF HON. BILL NELSON, 
                   U.S. SENATOR FROM FLORIDA

    Senator Nelson. Mr. Chairman, ever since I had the 
privilege of conducting the experiment proposed by the 
Comprehensive Cancer Center at the University of Alabama at 
Birmingham onboard the 24th flight of the Space Shuttle, and 
where I had one little opportunity to glimpse into the work of 
scientists, my admiration and appreciation and conclusion is, 
let's don't hold them back.
    Clearly, when we get into the question of life, it's going 
to be an emotional consideration. But here, we're talking about 
research on stem cells that are not as a result of a fertilized 
egg, but, rather, stem cells that have been artificially 
created, implanted, and produced. For us to get this into the 
realm of saying we're going to stop this, with all of its 
potential of saving life, seems to me not to be the place to 
draw the line.
    So I'm looking forward to the testimony today. Thank you, 
Mr. Chairman.
    Senator Brownback. Thank you very much.
    Gentlemen, we'll call up the first panel. And if you could 
come up, we would appreciate that.
    It will be a panel of experts, Dr. Michel Levesque, of 
Beverly Hills, California; Dr. Jean Peduzzi-Nelson, of 
University of Alabama at Birmingham; and Dr. Irv Weissman, of 
Stanford University Medical School, in California.
    I want to thank the panel for coming forward, and I thank 
you in advance for your testimony.
    I would note that your entire written statement will be put 
in the record at the outset, and so you're welcome to just 
summarize, if you would like to, or you can present your 
statements, as well. I would appreciate it if you could keep 
them as concise as possible so we could have plenty of time for 
exchange and interchange.
    Dr. Levesque, we appreciate your testimony.

     STATEMENT OF MICHEL F. LEVESQUE, M.D, FRCS(C), FACS, 
CEDARS-SINAI MEDICAL CENTER, LOS ANGELES, CALIFORNIA; ASSOCIATE 
CLINICAL PROFESSOR, UCLA SCHOOL OF MEDICINE AND MEMBER OF UCLA 
    BRAIN INSTITUTE; CHAIRMAN, FOUNDATION FOR NEURAL REPAIR

    Dr. Levesque. Thank you, Senator. Good afternoon.
    Senator Brownback. Move your--you're confusing us here, 
your signs are off a person. So, Dr. Weissman, if you'd pull 
yours in----
    [Laughter.]
    Senator Brownback. Thank you.
    Dr. Levesque. So, good afternoon. My name is Michel 
Levesque, and I'm a physician, scientist, and neurosurgeon 
based at Cedars-Sinai Medical Center in Los Angeles. I'm also 
an Associate Clinical Professor of Neurosurgery at the UCLA 
School of Medicine, and member of the UCLA Brain Research 
Institute. I'm also the founder of NeuroGeneration, a biotech 
company pioneering neural stem cell therapies, and Chairman of 
the Foundation for Neural Repair, a not-for-profit foundation 
sponsoring free clinical research to accelerate human trials 
using neural stem cells.
    Mr. Chairman and Members of the Subcommittee, I want to 
thank you for the opportunity to testify today on our current 
experience with the use of stem cells in humans, and, more 
specifically, adult neural stem cells for neurological 
disorders like Parkinson's disease.
    Although nonpartisan, my testimony attempts to provide a 
realistic perspective on the promises and limitations of cell 
therapy for neurological disorders, either from embryonic or 
adult-derived stem cells.
    As a scientist and physician treating patients with 
irreversible neurological disorders, it is of utmost importance 
to understand both the fact and the fiction of cell therapy, 
and the hopes it generates in our patients and their families.
    What is stem cell therapy? Stem cell research and therapy 
are some of new several tools, like vaccines, genes, or small 
molecules, targeting diseases not treated by traditional 
medication therapies.
    Stem cell research looks at basic mechanism of cell cycle 
at sequential expression of different genes during the 
formation of the embryo and cellular specialization and 
differentiation into different tissues. Stem cell research also 
explore the causes of disease, the mechanisms of cell 
degeneration and cell death.
    Stem cell therapy attempts to replace the cell loss and 
induce repair mechanism in models of disease. Clinical research 
and therapeutic trials, on the other hand, study the safety and 
efficacy of stem cells in patient with certain disorders.
    Neural repair and neural transplantation using cell therapy 
aim at introducing cellular products to replace the deficient 
cells, or induce local neural repair in the central nervous 
system.
    What are human adult neural stem cells? Since 1996, our 
laboratories have been involved with the isolation of human 
adult-derived neural stem cells obtained from patients 
undergoing neurosurgical procedures. In the adult brain, these 
cells cannot, on their own, trigger repair responses. However, 
if placed in experimental conditions, stimulating certain 
genes, these neural stem cells can be awakened and begin to 
divide and regenerate along similar steps of normal 
development.
    These newly created neural stem cells can grow for several 
months in laboratory conditions, reaching several millions in 
number. The ability to self-replicate and form all types of 
cells found in the central nervous system can be verified in 
vitro under controlled conditions.
    Prior to transplantation, neural stem cells are then 
differentiated, stopping the replication process to produce 
mature neuron of different types, including dopamine-secreting 
neurons, which are deficient in Parkinson's disease.
    These newly formed cells are unadulterated, having not been 
exposed to years of chronic oxidative stress or other 
predisposing environmental factors leading to cell damage and 
cell death.
    Adult neural stem cells represent a new source of cell 
replacement with identical genetic material to the patient, and 
mitigate the risk of immune rejections and transmittable 
disease.
    Can stem cell therapy help neurodegenerative disorders such 
as Parkinson's disease? Parkinson's disease is associated with 
a progressive cell loss of midbrain dopamine-secreting neurons. 
The causes of Parkinson's disease remain unknown. Like 
Alzheimer's disease, there is evidence showing that a 
combination of environmental factors and genetic predisposition 
are precursors of the disease. Current animal models derived 
from toxic exposure or transgenic manipulations do not 
replicate all changes found in human brain.
    In fact, Parkinson's disease is much more complex in human 
patients because of secondary chemical changes throughout the 
rest of the brain superimposed on long-term medical therapy.
    Embryonic stem cells have the potential, virtual potential, 
to generate any type of cell in the body. One of the key 
problem, however, is to elucidate the proper steps along the 
formation of neural stem cells, and then to achieve proper 
differentiation.
    In addition, there remain risks of unstable phenotypic 
expression, possible transdifferentiation into other types of 
tissue causing tumors, immune reactions in the host brain, and 
questionable functional benefits.
    Currently available embryonic cell lines are not 
appropriate to answer these scientific questions. Embryonic 
cell has yet to be scientifically proven as safe and even 
effective in human patients. On the other hand, mature neurons 
derived from the patient's own brain can be transplanted back 
safely and improve symptoms.
    We recently presented a clinical outcome of this autologous 
method at the meeting of the International Congress of 
Parkinson's Disease in Rome. We previously transplanted the 
patient with advanced Parkinson's disease with differentiated 
neurons derived from an initial biopsy. At 3 years post-
operatively, the UPDRS score improved by 81 percent while on 
medication, and 83 percent while off medication. We demonstrate 
here the long-term clinical improvement of Parkinson's disease 
symptoms in a single patient.
    To conclude this presentation, adult human neural stem 
cells derived from a patient's own tissue can become a source 
of replacing neurons useful for grafting in the treatment of 
neural degenerative disorder.
    Degenerative and traumatic disorders of the brain represent 
an enormous challenge to the patient, their family, and 
healthcare provider. The ethical debate between the embryonic 
stem cell proponents and those who are opponents, opposed to 
their use, distracts from other avenues with promising 
outcomes. It also overlooks other ethical issues of resource 
allocation between basic research, clinical research, patient 
care, and health insurance.
    Scientific knowledge has rapidly progressed in the last 5 
years, and stem cell research remains a very promising field 
for neurological disorders. The ethical debate we are facing 
today is to access proper funding to proceed with human 
clinical trials using neural stem cells. Our challenge is to 
build the proper infrastructures committed to these long-term 
goals. For a fraction of the price of a B-1 bomber, millions of 
lives can be improved, if not saved, with the use of these 
neural stem cells.
    Thank you.
    [The prepared statement of Dr. Levesque follows:]

 Prepared Statement of Michel F. Levesque, M.D., FRCS(C), FACS, Cedars-
   Sinai Medical Center, Los Angeles, California; Associate Clinical 
      Professor, UCLA School of Medicine and Member of UCLA Brain 
           Institute; Chairman, Foundation for Neural Repair
    My name is Michel Levesque, and I am a physician, neuroscientist 
and neurosurgeon based at Cedars-Sinai Medical Center in Los Angeles. I 
am Associate Clinical Professor of Neurosurgery at the UCLA School of 
Medicine and member of the UCLA Brain Research Institute. I am also the 
founder of NeuroGeneration, a biotechnology company pioneering 
autologous neural stem cell therapies, and Chairman of the Foundation 
for Neural Repair, a not-for-profit foundation, sponsoring 
translational research to accelerate human trials using neural stem 
cells.
    Mr. Chairman and members of the Subcommittee, I want to thank you 
for the opportunity to testify today on our current experience with the 
use of stem cells in humans, and more specifically, adult neural stem 
cell-derived neurons, for neurodegenerative disorders like Parkinson's 
disease.
    Although non-partisan, my testimony attempts to provide a realistic 
perspective on the promises and limitations of cell therapy for 
neurological disorders, either from embryonic-or adult-derived stem 
cells.
    As a scientist and physician treating patients with irreversible 
neurological disorders, it is of utmost importance to understand both 
the fact and fiction of cell therapy and the hopes it generates in our 
patients and their families.
What Is Stem Cell Therapy?
    Stem cell research and therapy are some of several new tools, like 
vaccines, genes or small molecules, targeting diseases not treated by 
traditional medication therapies.
    Stem cell research looks at basic mechanisms of the cell cycle, at 
sequential expression of different genes during the formation of the 
embryo, and at cellular specialization and differentiation into 
different tissues. Stem cell research can also explore the causes of 
diseases, cell degeneration and cell death.
    Stem cell therapy attempts to replace the cell loss and induce 
repair mechanisms in models of disease. Clinical research and 
therapeutic trials, on the other hand, study the safety and efficacy of 
stem cell therapy in patients with certain disorders.
    Neural repair and neural transplantation using cell therapy aim at 
introducing cellular products, or biological modifiers, to replace the 
deficient cells and/or induce local neural repair in the central 
nervous system.
What Are Human Adult Neural Stem Cells?
    In nature, neural stem cells are formed after a cascade of 
sequential events activates genes within embryonic cells during 
development. They are derived from a specific layer of the embryo and 
can only become, under normal conditions, precursors of cells found 
only in the central nervous system.
    Since 1996, our laboratories have been involved with the isolation 
and characterization of human adult-derived neural stem cells, obtained 
from patients undergoing neurosurgical procedures. In the adult brain, 
these cells cannot on their own trigger repair responses. However, if 
placed in experimental laboratory conditions stimulating certain genes, 
these neural stem cells can be ``awakened'' and begin to divide and 
replicate events of normal development.
    These newly created neural stem cells can grow for several months 
in laboratory conditions reaching several millions in number, a process 
called cell expansion. Their ability to self-replicate and form all 
types of cells found in the central nervous system can be verified in 
vitro under controlled conditions. They can be placed in storage or 
maintained in sterile incubators until ready for use.
    Prior to transplantation, neural stem cells are then exposed to a 
modified environment triggering differentiation, stopping the 
replication process to produce mature neurons of different types, 
including dopamine-secreting neurons, which are deficient in 
Parkinson's disease. In the laboratory, differentiated neurons can be 
characterized with specific markers, and their function demonstrated by 
the increased production of dopamine.
    These cells have survived transplantation and corrected motor 
deficits in a rat model of Parkinson's disease. Our animal studies 
showed that human adult neural stem cells do not divide once 
differentiated, do not form aberrant tissue or tumors after chronic 
transplantation, and have normal karyotypes (number of chromosomes). 
Sterility is documented throughout the expansion phases.
    These newly formed cells are unadulterated, having not been exposed 
to years of chronic oxidative stress and other predisposing factors 
leading to neurodegeneration. Autologous adult neural stem cells 
represent a new source of cell replacement with identical genetic 
material to the patient, and mitigate the risks of immune rejections 
and transmittable diseases generally associated with tissue transplants 
from a source external to the patient such as HIV, Encephalitis, 
Hepatitis and Creutzfeld-Jacobs Disease.
Can Stem Cell Therapy Help Neurodegenerative Diseases Such as 
        Parkinson's Disease?
    Parkinson's disease is associated with a progressive cell loss of 
midbrain dopamine-secreting neurons. Dopamine is an essential brain 
chemical for proper modulation and execution of motor function. Because 
of the limited spatial involvement and biochemical specificity, this 
disease may seem relatively easy to repair. Dopamine neurons delivered 
by fetal transplantation previously were shown to help certain patients 
with Parkinson's disease, but had significant risk factors, 
complications, and ethical issues.
    The causes of Parkinson's disease remain unknown. Like Alzheimer's 
disease, there is evidence showing that a combination of environmental 
factors and genetic predisposition are precursors to the disease. 
Current animal models, derived from toxic exposure or transgenic 
manipulation, do no replicate all changes found in the human brain.
    In fact, Parkinson's disease is much more complex in human patients 
because of secondary physiological and chemical changes throughout the 
rest of the brain, superimposed on long-term medical therapy. Indeed 
one of the major complications of dopamine drug therapy is the 
paradoxical creation of dyskinesia, another movement disorder involving 
uncontrollable thrashing movements.
    This complication was also found in some patients receiving fetal 
transplantation, suggesting that an uncontrolled delivery of excessive 
dopamine may not be beneficial. Stem cell-derived products have the 
advantages of being produced under controlled environment and 
characterized both in their types and function prior to 
transplantation.
    Embryonic stem cells have the potential to generate any type of 
cells and presumably can be guided in their differentiation to generate 
an unlimited number of dopamine neurons. One of the problems is to 
understand the proper steps to guide the gene expression along the 
formation of neural stem cells and then to achieve proper 
differentiation.
    In addition there remain risks of unstable phenotypic expression, 
possible transdifferentiation into other types of tissue causing 
tumors, immune reactions in the host brain and questionable functional 
benefits. Several additional studies are needed in order to answer 
these questions and objectively compare these ``off the shelf'' cell 
lines to our customized approach using autologous adult neural stem 
cells.
    While the use of somatic nuclear cell transfer (SNCT) technology 
could decrease risks of immune reactions, this area of research 
minimizes the importance of ``imprinting'', or influences of the extra-
nuclear material on normal cellular development.
    Currently available embryonic cell lines are not appropriate to 
answer these scientific questions. Embryonic cell therapy has yet to be 
scientifically proven as safe, if even effective, in human patients.
Mature Neurons Derived from the Patient's Own Brain Can Be 
        Transplanted Back Safely and Improve Symptoms
    We recently presented the clinical outcome of our autologous method 
at the International Congress of Parkinson's disease and Movement 
Disorders in Rome. In accordance with our institutional review board, 
we transplanted a patient with advanced Parkinson's disease with 
differentiated neurons derived from an initial needle biopsy. At three 
years post-operatively, the overall Unified Parkinson's Disease Rating 
Scale (UPDRS) improved by 81 percent while ``on'' medication and 83 
percent while ``off'' medication. We demonstrated here the long-term 
clinical remission of Parkinson's disease symptoms in a single patient.
    Because of their biocompatibility, safety and potential integration 
into the host striatum, autologous adult neural stem cells and stem 
cell-derived neurons represent an effective alternative to current cell 
therapy aimed at the restoration of dopamine neuronal loss in 
Parkinson's disease. Under the guidance and supervision of the Food and 
Drug Administration (FDA) office of Cellular, Tissues and Gene 
Therapies and the Center for Biologics Evaluation and Treatment (CBER) 
we are about to begin Phase II trials using this promising cell 
therapy.
Conclusion
    Degenerative and traumatic disorders of the brain represent an 
enormous burden to the patient, their family and health care providers. 
The current debate between the embryonic stem cell proponents and those 
who are opposed to their use distracts from other avenues with 
promising outcome, such as adult stem cell therapy. It also overlooks 
other important issues of resource allocation between basic and 
clinical research, health insurance, and patient care.
    Scientific knowledge has rapidly progressed in the last five years 
and stem cell research and therapy remains a very promising field for 
treatment of neurological disorders. In a recent biotechnology industry 
meeting, a presentation had the approximate title: ``Businesses are 
from Mars, Academics are from Venus''. What was forgotten there is that 
patients are from planet Earth and this is what should guide our 
efforts.
    Adult human neural stem cells derived from a patient's own tissue 
can become a source of replacement neurons, useful for grafting in the 
treatment of neurodegenerative disorders. With time and adequate 
support this approach has the potential of making neural stem cell 
therapy acceptable and available to a large number of patients.
    Dear members of the Committee, I appreciate the opportunity to 
present our results with the use of human adult neural stem cell-
derived neurons and to contribute to an honest and objective debate on 
these important issues.

[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]

    Senator Brownback. Thank you, Dr. Levesque. And I look 
forward, in questioning, to asking you about some of your 
patients that you've treated. We will have--we will have one 
patient of yours, I believe, on the second panel.
    Dr. Levesque. That's----
    Senator Brownback. Is that correct?
    Dr. Levesque.--correct.
    Senator Brownback. Look forward to that testimony.
    Dr. Nelson?

          STATEMENT OF JEAN D. PEDUZZI-NELSON, Ph.D.,

              DEPARTMENT OF PHYSIOLOGICAL OPTICS,

              UNIVERSITY OF ALABAMA AT BIRMINGHAM

    Dr. Peduzzi-Nelson. Thank you, Senator Brownback and 
Members of the Subcommittee.
    Senator Brownback. Why don't you get that microphone a 
little closer to you, if you would? Thanks.
    Dr. Peduzzi-Nelson. It's a pleasure to be here today.
    I'd like to tell you about the spectacular results in my 
patients. Despite what you read in the lab, my patients are all 
very short and fuzzy, and also commonly known as rats.
    [Laughter.]
    Dr. Peduzzi-Nelson. I'm--also have been asked to present 
the results of Dr. Carlos Lima, in Portugal. He came to my labs 
and showed me the techniques that he is using in patients, and 
he asked me to present these results.
    For the last 12 years of my life, at the University of 
Alabama at Birmingham, I have been searching for an effective 
treatment for spinal cord injury--not just some types of spinal 
cord injury, but a particular type, severe spinal cord injury, 
and the chronic condition where, after a year, there is 
virtually no--there's nothing available for these patients, and 
there's no further improvement after a year after spinal cord 
injury.
    So I have tried just about everything that's out there. I 
would try anything that seemed reasonable. And the advantages 
of the adult stem cells is that you avoid the problems of 
rejection, you avoid the problems of overgrowth, or tumors, and 
you avoid all ethical concerns in using adult stem cells. 
You've heard, from Dr. Levesque, some of his amazing findings 
in Parkinson's disease. And recently there have been really 
amazing findings in spinal cord injuries using Dr. Lima's 
procedure.
    It all began--I'm, sort of, going to tell you all this as a 
story--it all, sort of, started in 1991. Dr. Lima got the idea 
that maybe, for spinal cord treatment, an effective way of 
approaching this problem would be the olfactory mucosa. The 
olfactory mucosa is part of the tissue that lines the inside of 
the nose. And we knew, at that time, that it has lifelong 
regenerative capacity, but we didn't know a lot about that 
tissue. So he started to investigate this tissue. He got 
autopsy material from 300 patients and actually studied the 
tissue in different-aged people. He also started an animal 
trial.
    Now, he was at the Hospital Egas Moniz in Lisbon, Portugal, 
and what he did was a study in guinea pigs. He actually cut the 
spinal cord. He went back a week later and put some of the 
animal's olfactory mucosa in this area of the cut spinal cord. 
And what he found was that some of these animals that received 
the transplant began to move, and the ones that did not have 
the transplant continued to drag their legs.
    Now, he had very limited facilities at this hospital. As a 
matter of fact, he took the animals home with him so that he 
could take care of them. And based on these results, he 
investigated the possibility of looking into a clinical trial. 
And what he did was, first he assembled a team of physicians. 
He's a neurologist and a pathologist. He began working with two 
neurosurgeons and an ENT doctor. And they formed a team to try 
to repeat this procedure, but repeat it in patients with 
chronic severe spinal cord injuries.
    And he started with seven Portuguese patients. Some of 
these patients are two and a half years out right now. And 
before he started this procedure, they did the procedure in 
cadavers so they could go through the procedure and work out 
the details. And he was working with people that have a 
complete spinal cord injury. That means most of these patients 
had no sensation, no feeling below the site of injury. They 
also had no movement. None of the muscles below the site of 
injury would--had any response, any activity.
    And what he found was that all of the patients, the first 
seven patients that he used this treatment, that there was 
improvement. Some of them had very dramatic improvement, some 
of them have limited improvement, but all of them showed 
improvement--some gain in sensation, some gain in motor 
activity. One woman, 6 months after the surgery, regained 
bladder control. There was another woman who, if she had proper 
facilities, would probably be walking today.
    The problem in Portugal was that there was very limited 
rehab facilities available. So as a next step, he began--he 
accepted some patients who were interested in this treatment in 
the U.S. He accepted them to come to Portugal, and some very 
brave Americans flew over to Portugal and had this surgical 
procedure done. He had hoped that because there are better 
rehabilitative facilities in the U.S., he might see even better 
improvement in these patients.
    Now, two of these very brave young women are here today, 
and you're going to hear their testimony, Laura Dominguez and 
Susan Fajt. And both of these patients had the surgery about 2 
years after their severe spinal cord injury. So, at that period 
of time, for everyone else, there was no hope of any 
improvement. Usually patients, after a year, have gotten back 
any improvement that they would see in their lifetime. And 
these brave women and their families went to Portugal to have 
this procedure. And both of them have seen some improvement.
    They have--Laura had no feeling and no movement below the 
level of--in her legs. And after the treatment, now she's able 
to walk with braces, she's able to point her toes, and has 
regained some sensation.
    Susan, another brave soul who's here today to talk to you, 
has also been able to do things that the U.S. physicians told 
her would never happen in her lifetime. She is able to walk 
with braces. She has regained a certain degree of bladder 
control. And she has regained feeling in her legs.
    So these are very--no, they're not walking into the 
courtroom unaided, but these are very dramatic findings for 
someone who, at the time of injury, the doctors told them, 
``There's going to be no further improvement. There are no 
treatments available.'' This is a very hard thing to hear at 
their young age.
    Now, where do we go from here? Obviously, there is further 
to go in terms of improvement with adult stem cells. In my own 
research lab, what we've found using this technique, using the 
olfactory mucosa, was that--in these rats with severe chronic 
injuries, I got the best improvement that I've seen, trying 
everything that was available in the last 12 years that I could 
try. And so what we need to do next is to either have further 
improvements--my set of experiments, I'm going to use the 
olfactory mucosa treatment, and combine it with other 
treatments so that we can get an even better improvement.
    And the other thing that's lacking is that we need a better 
rehabilitative program in the U.S. The rehabilitation in the 
U.S. was not designed to handle patients who all of a sudden 
gained functional connections after several years. And this has 
to be done very carefully so that there is not injuries. And 
both of these women have been tremendously helped by their 
family in going even all over the world to get the best 
rehabilitative programs developed. And especially Susan and her 
father, they have developed devices and patented devices, in 
hopes of getting further improvements that are effective.
    Senator Brownback. OK.
    Dr. Peduzzi-Nelson. I hope----
    Senator Brownback. Let's--we'll kind of wrap this up, if 
you can here, very quickly.
    Dr. Peduzzi-Nelson. I'd just--that about summarizes it, 
that we're hoping to go forward from this point and even have 
better improvement with adult stem cells.
    [The prepared statement of Dr. Peduzzi-Nelson follows:]

  Prepared Statement of Jean D. Peduzzi-Nelson, Ph.D., Department of 
       Physiological Optics, University of Alabama at Birmingham
``The Truth is not Being Presented''
    Thank you Senator Brownback and distinguished Senators of the 
Subcommittee for the invitation to present to you today. First of all, 
I would like to commend your subcommittee for bringing to light some of 
the remarkable advances in adult stem cell research. I have long 
admired the work of Dr. Michel Levesque in Parkinson's disease and I'm 
glad that the subcommittee had the opportunity to see the remarkable 
improvement of his patient with Parkinson's disease who had received a 
treatment derived from the adult stem cells in his own brain. I am 
thrilled to hear Dr. Levesque's plan to expand the clinical trials at 
Cedars Sinai Hospital in California. I know that actually seeing and 
hearing patients that improved is the strongest evidence of the 
potential of adult stem cells. This evidence provides strong refutation 
to claims about the limited usefulness of adult stem cells and other 
sources of cells such as umbilical cord cells. Hearing from patients 
that actually improved using adult stem cells is more interesting than 
scientific data and discussions about the stem cell/cloning 
controversy, but I need your indulgence to present the truth about stem 
cells and cloning.

   1.  Some people naively think that the stem cell controversy is just 
        related to the abortion issues, political party alignment, 
        religious beliefs, or scientific freedom. However, none of 
        these are the driving force in the effort to promote Federal 
        funding of human embryonic stem cells or human cloning. The 
        most profitable, not the best, treatment for people is being 
        promoted. The main reason for the current controversy regarding 
        human embryonic stem cells & cloning is money. The old 
        statement of `follow the money' explains many of the opposing 
        statements made regarding this controversy. It is a superior 
        business plan to have a mass-produced product such as 
        embryonic/fetal/cloned stem cells that can be sold nationwide 
        and has patentable intellectual property.\1\ Cloned stem cells 
        derived from embryos with genetic defects represent the 
        possibility of millions in patentable stem cell lines. Adult 
        stem cell therapies are much better for people with diseases or 
        injuries but generate an inferior business plan. In the case of 
        adult stem cells where, in most cases, a person's own cells can 
        be used, one can only develop a procedure that is generally not 
        patentable according to new patent laws. However, the 
        embryonic/fetal/cloned stem cells can lead to tremendous 
        profits in the short run. Proof of this is the millions of 
        dollars furnished by venture capitalists to help pass a measure 
        that would provide $3 billion for stem cell research in 
        California.\2\
---------------------------------------------------------------------------
    \1\ Marshall, E.(2000) The Business of Stem Cells, Science, 
287:1419-1421.
    \2\ The Ledger.com, Lakeland, FL, Published Thursday, May 20, 2004, 
``Venture capital money backs California stem cell measure'', PAUL 
ELIAS

   2.  Checks and balances in the form of public policy are needed in 
        society to control greed, especially in those cases where the 
        greater good of the people will be served. Embryonic/fetal stem 
        cells have the problems of overgrowth, rejection, possible 
        disease transmission, and ethical issues. Tumors have been 
        found in experimental animals \3\ \4\ and disastrous results 
        have been reported in 2 separate clinical trials \5\ \6\ using 
        embryonic/fetal tissue/cells. The government should not finance 
        an area of research that is not only dangerous, but also many 
        people view as unethical. Many Americans are against the 
        deliberate destruction of human life. The ban against Federal 
        funding of human stem cells (except for the 67 human stem cell 
        lines) provides a small hope that the financially unprofitable 
        adult stem cell (that are better for people with diseases or 
        injuries) might go forward.
---------------------------------------------------------------------------
    \3\ L.M. Bjorklund et al.; ``Embryonic stem cells develop into 
functional dopaminergic neurons after transplantation in a Parkinson 
rat model,'' Proc. Natl. Acad. Sci. USA 99, 2344-2349; 19 Feb 2002.
    \4\ F Nishimura et al.; ``Potential use of embryonic stem cells for 
the treatment of mouse Parkinsonian models: improved behavior by 
transplantation of in vitro differentiated dopaminergic neurons from 
embryonic stem cells''; Stem Cells 21, 171-180; March 2003.
    \5\ Freed CR, Greene PE, Breeze RE, Tsai WY, DuMouchel W, Kao R, 
Dillon S, Winfield H, Culver S, Trojanowski JQ, Eidelberg D, Fahn S 
(2001) Transplantation of embryonic dopamine neurons for severe 
Parkinson's disease. New Engl. J. Med. 344:710-9.
    \6\ Olanow CW. Goetz CG. Kordower JH. Stoessl AJ. Sossi V. Brin MF. 
Shannon KM. Nauert GM. Perl DP. Godbold J. Freeman TB. A double-blind 
controlled trial of bilateral fetal nigral transplantation in 
Parkinson's disease. [Clinical Trial. Journal Article. Randomized 
Controlled Trial] Annals of Neurology. 54(3):403-14, 2003
---------------------------------------------------------------------------
   3.  The myth of the availability of countless frozen embryos in 
        fertility clinics is just not true. To use even one of these 
        embryos would require legal release from the parents that in 
        most states is not easily accomplished. In many cases, it is 
        not that easy to locate the parents especially in the cases of 
        divorce or separation. It is generally assumed that it would 
        not be hard to get parents to agree. However, when it comes to 
        make the final decision, many parents are unsure that they want 
        these potential lives destroyed. Many of the frozen embryos are 
        also not viable. Despite the impressive results with in vitro 
        fertilization, recent studies suggest that these children have 
        a higher rate of congenital anomalies and human overgrowth 
        syndrome.\7\ \8\
---------------------------------------------------------------------------
    \7\ Sutcliffe, A.G, D'Souza SW, Cadman J, Richards, B, McKinlay IA, 
Liberman B (1995) Minor congenital anomalies, major congenital 
malformation and development in children conceived from cryopreserved 
embryos. Hum Reprol. 10: 3332-3337.
    \8\ DeBaun, E.L Niemitz and A. P. Feinberg (2003) Association of In 
Vitro fertilization with Beckwith-Wiedemann syndrome and epigenetic 
alterations of LIT1 and H19. Am. J. Hum. Genet. 72: 156-160.

   4.  The best way to honor the memory and work of President Reagan is 
        to not provide Federal funding for something that President 
        Reagan, if alive today, would vehemently oppose. There is no 
        doubt that President Reagan would not favor Federal support of 
        research using human embryos. This is very clear from an 
        address given by President Reagan \9\:
---------------------------------------------------------------------------
    \9\ PERSONHOOD PROCLAMATION, National Sanctity of Human Life 
Day,1988, By the President Reagan, A Proclamation:

      ``I, Ronald Reagan, President of the United States of America, by 
            virtue of the authority vested in me by the Constitution 
            and the laws of the United States, do hereby proclaim and 
            declare the unalienable personhood of every American, from 
            the moment of conception until natural death, and I do 
            proclaim, ordain, and declare that I will take care that 
            the Constitution and laws of the United States are 
            faithfully executed for the protection of America's unborn 
---------------------------------------------------------------------------
            children.''

   5.  The often stated advantage that embryonic stem cells can make 
        every cell in body is not an advantage for people with diseases 
        or injuries. This is only important in terms of a business 
        plan. Science has not worked out all the requirements needed to 
        direct them properly on their path and make sure that they do 
        not develop improperly or become tumors. There are many sources 
        of stem cells in the adult body. Whether each type of adult 
        stem cells can make every different cell type in the body is a 
        mute issue. For example, neurons (nerve cells) can be derived 
        from cells in the adult brain \10\ \11\, bone marrow \12\, 
        muscle \13\ or skin cells \14\. Also there is evidence by Dr. 
        Verfaillie and colleagues at University of Minnesota that stem 
        cells from adults are able to form any cell type in the 
        body.\15\
---------------------------------------------------------------------------
    \10\ Vescovi AL. Parati EA. Gritti A. Poulin P. Ferrario M. Wanke 
E. Frolichsthal-Schoeller P. Cova L. Arcellana-Panlilio M. Colombo A. 
Galli R. Isolation and cloning of multipotential stem cells from the 
embryonic human CNS and establishment of transplantable human neural 
stem cell lines by epigenetic stimulation. Experimental Neurology. 
156(1):71-83, 1999.
    \11\ Song HJ. Stevens CF. Gage FH. Neural stem cells from adult 
hippocampus develop essential properties of functional CNS neurons. 
Nature Neuroscience. 5(5):438-45, 2002.
    \12\ Keene CD. Ortiz-Gonzalez XR. Jiang Y. Largaespada DA. 
Verfaillie CM. Low WC. Neural differentiation and incorporation of bone 
marrow-derived multipotent adult progenitor cells after single cell 
transplantation into blastocyst stage mouse embryos. [Journal Article] 
Cell Transplantation. 12(3):201-13, 2003.
    \13\ Romero-Ramos M. Vourc'h P. Young HE. Lucas PA. Wu Y. 
Chivatakarn O. Zaman R. Dunkelman N. el-Kalay MA. Chesselet MF. 
Neuronal differentiation of stem cells isolated from adult muscle. 
Journal of Neuroscience Research. 69(6):894-907, 2002.
    \14\ Toma JG. Akhavan M. Fernandes KJ. Barnabe-Heider F. Sadikot A. 
Kaplan DR. Miller FD. Isolation of multipotent adult stem cells from 
the dermis of mammalian skin. Nature Cell Biology. 3(9):778-84, 2001.
    \15\ Jiang Y. Henderson D. Blackstad M. Chen A. Miller RF. 
Verfaillie CM. Neuroectodermal differentiation from mouse multipotent 
adult progenitor cells. Proceedings of the National Academy of Sciences 
of the United States of America. 100 Suppl 1:11854-60, 2003.

   6.  Several clinical disasters have occurred using embryonic cells/
        tissue that contain stem cells. The clinical trials in 
        Parkinson's disease had dramatic differences in their findings 
        depending on the original source of the cells: fetuses or the 
        person's own cells. You've already heard and seen the 
        spectacular results of Dr. Levesque. However, you may not have 
        heard about the clinical trial disasters using embryonic/fetal 
        tissue. When a transplant consists of embryonic/fetal tissue, 
        the stem/progenitor cells are the only cells that survive. A 
        clinical trial was done by Dr. Freed and colleagues \16\ in 
        which 19 patients received cells derived from 4 different 
        fetuses from abortions at 7-8 weeks after conception. The 
        patients that were under 60 years showed about a 28 percent 
        improvement in the Unified Parkinson's Disease Rating Scale 
        (UPDRS). However, about 15 percent of these patients showed 
        devastating deterioration at 1 year after treatment that was 
        believed to result from cellular overgrowth. In another 
        clinical trial for Parkinson's disease using embryonic tissue 
        (kept in cold media until transplant), similar results were 
        obtained but the rapid deterioration in some patients was 
        believed to be from rejection of the foreign cells/tissue 
        derived from embryo or fetus.\17\
---------------------------------------------------------------------------
    \16\ Freed CR, Greene PE, Breeze RE, Tsai WY, DuMouchel W, Kao R, 
Dillon S, Winfield H, Culver S, Trojanowski JQ, Eidelberg D, Fahn S 
(2001) Transplantation of embryonic dopamine neurons for severe 
Parkinson's disease. New Engl. J. Med. 344:710-9.
    \17\ Olanow, C.W., Goetz, C.G., Kordower, J.H., Stoessl, A.J., 
Sossi, V., Brin, M.F., Shannon, K.M., Nauert, G.M., Perl, D.P., 
Godbold, J., et al., 2003. A double-blind controlled trial of bilateral 
fetal nigral transplantation in Parkinson's disease. Annals of 
Neurology 54:403-414.

   7.  Terrible catastrophes using embryonic/fetal stem cells are also 
        observed in animal experiments. In an animal model of 
        Parkinson's disease, rats injected with embryonic stem cells 
        showed a slight benefit in about 50 percent of the rats, but 
        one-fifth (20 percent) of the rats died of brain tumors caused 
        by the embryonic stem cells.\18\ This was confirmed in another 
        similar study conducted by a different group of researchers who 
        also found tumor formation in about 20 percent of the rats.\19\ 
        In yet another study it was reported that keeping embryonic or 
        fetal stem cells in culture for long periods of time cause 
        genetic mutations and tumor formation when these cells are 
        transplanted.\20\
---------------------------------------------------------------------------
    \18\ L.M. Bjorklund et al.; ``Embryonic stem cells develop into 
functional dopaminergic neurons after transplantation in a Parkinson 
rat model,'' Proc. Natl. Acad. Sci.USA 99, 2344-2349; 2002.
    \19\ F Nishimura et al.; ``Potential use of embryonic stem cells 
for the treatment of mouse Parkinsonian models: improved behavior by 
transplantation of in vitro differentiated dopaminergic neurons from 
embryonic stem cells''; Stem Cells 21, 171-180; March 2003.
    \20\ Morshead, C.M., P. Benveniste, N.N. Iscove and D. van der Kooy 
(2002) Hemapoietic competence is a rare property of neural stem cells 
that may depend on genetic and epigenetic alterations. Nature Medicine 
8:268-273.

   8.  Cloned human stem cells will not be useful as long as the cloned 
        human embryos are incapable of forming a person. It often 
        stated that there is no chance of human reproductive cloning 
        because 99.2 percent of cloned embryos can not survive. 
        However, these same faulty cloned embryos are being praised as 
        being a source of valuable stem cells that will advance the 
        cure of genetic disorders. If these cloned human embryos are so 
        abnormal that they almost never can survive in the womb then 
        stem cells derived from them would also abnormal and not useful 
        for research. The big push for cloned stem cells is the 
        possibility of patenting stem cell lines derived from these 
---------------------------------------------------------------------------
        cloned embryos.

   9.  If human cloning is funded to produce cloned stem cells, 
        reproductive cloning could not be prohibited. Eventually if 
        scientists continue to produce cloned human embryos, it will be 
        possible to form cloned human embryos without defects that will 
        readily give develop to a fully mature person. Although it is 
        often stated that no one would risk the million dollar penalty, 
        the amount invested that resulted in a cloned cat in Texas was 
        3.7 million dollars. A lot of Americans have less of a moral 
        dilemma with the birth of an individual derived from a clone 
        than creating human life then destroying it for some vague 
        scientific purpose . To my knowledge, there have been no 
        genetic diseases in animals cured with stem cells from clones 
        even though there is no current bans regarding cloning. 
        However, patents of these human stem cells from cloned embryos 
        are likely to bring millions to biotech companies.

  10.  Adult stem cells have been shown to make insulin. Although there 
        are many claims to the contrary, recent studies have shown that 
        stem cells from adults can make insulin. At the University of 
        Florida in Gainsville, Dr. Tang and associates were successful 
        in getting insulin-producing cells from adult bone marrow stem 
        cells. These cell secreted insulin in a controlled manner and 
        reversed diabetes in mice.\21\ Also a cell type isolated from 
        bone marrow called MIAMI cells were shown to produce insulin. 
        Insulin producing cells are also produced from embryonic stem 
        cells.\22\ However, the stem cells from embryos were inferior 
        to the stem cells from adults because the insulin producing 
        cells from the embryos were not responsive to changing levels 
        of glucose.\23\
---------------------------------------------------------------------------
    \21\ Tang, D-Q, L-Z Cao, B.R. Burkhardt, C-Q Xia, S.A. Litherland, 
M.A. Atkinson, and L-J Yang (2004) In Vivo and In Vitro 
Characterization of Insulin-Producing Cells Obtained From Murine Bone 
Marrow Diabetes 53:1721-1732.
    \22\ D'Ippolito G. Diabira S. Howard GA. Menei P. Roos BA. Schiller 
PC. Marrow-isolated adult multilineage inducible (MIAMI) cells, a 
unique population of postnatal young and old human cells with extensive 
expansion and differentiation potential. Journal of Cell Science. 
117(Pt 14):2971-81, 2004.
    \23\ Soria B. Roche E. Berna G. Leon-Quinto T. Reig JA. Martin F. 
Insulin-secreting cells derived from embryonic stem cells normalize 
glycemia in streptozotocin-induced diabetic mice. [Journal Article] 
Diabetes. 49(2):157-62, 2000.

  11.  Research is not being slowed by the current ban on Federal 
        funding of human embryonic/fetal stem cells. Every clinical 
        trial, new drug, new treatment is based on animal studies. 
        There is no ban on animal embryonic or fetal stem cells or 
        animal cloned cells. There is only a ban on Federal funding of 
        human embryonic or fetal stem cells. As a matter of fact, this 
        ban will bring balance so that adult stem cell research will be 
        further explored even though it is less profitable. There is no 
        ban on using embryonic or fetal stem from animals or private 
---------------------------------------------------------------------------
        funding of research using human stem cells.

  12.   Many alternative treatments besides stem cells are showing 
        progress for treating diseases and injuries. Before I talk 
        about the progress in adult stem, I would like to mention that 
        in terms of injuries or diseases such as Alzheimer's disease, 
        spinal cord injury, head injury, diabetes, ALS (Lou Gehrig's 
        disease), liver or heart damage and Parkinson's disease, there 
        are many other alternatives therapies being scientifically or 
        clinically explored. As a prominent stem cell researcher named 
        Dr. Ron McKay said recently that it was a fairy tale to think 
        that stem cells could help Alzheimer's disease.\24\ In the case 
        of diabetes, there is an exciting new drug called liraglutide 
        that seems promising in type 2 diabetes.\25\ In recent study 
        using a mouse model of Parkinson's disease, therapeutic 
        immunization using immune cells prevented nerve cells from 
        dying.\26\ Progress is also being made in diabetes across the 
        country using islet cell transplants. Recently at my 
        university, University of Alabama at Birmingham, Professor 
        Devin Eckhoff performed an islet cell transplant into a young 
        woman who was totally dependent on insulin shots since age 2. 
        The transplanted cells were obtained from a pancreas of a 
        patient who died in an accident. These transplanted cells 
        immediately began to function and it is hoped that this patient 
        will never have to take insulin shots again.\27\ Unfortunately 
        Dr. Eckhoff and his patient were unable to join us today but 
        may testify later in the year.
---------------------------------------------------------------------------
    \24\ Stem Cells An Unlikely Therapy for Alzheimer's Reagan-Inspired 
Zeal For Study Continues By Rick Weiss, Washington Post, June 10, 2004; 
Page A03.
    \25\ http://www.glucagon.com/liraglutide.htm.
    \26\ Benner, E.J., R. L. Mosley, C.J. Destache, T.B. Lewis, V. 
Jackson-Lewis, S. Gorantla, C. Nemachek, S. R. Green, S. Przedborski, 
and H.E. Gendelman Therapeutic immunization protects dopaminergic 
neurons in a mouse model of Parkinson's disease PNAS, 2004.
    \27\ Black, H. UAB's first islet-cell transplant a success, UAB 
reporter, vol. 28(27), April 26, 2004.

  13.  There has been tremendous progress in adult stem cell research 
        in the last few years. In another study, adult stem cells 
        transplanted into mice with liver injuries helped restore liver 
        function within two to seven days.\28\ Transplantation of stem 
        cells from adult human brain causes myelination to occur in a 
        focally demyelinated spinal cord of the rat.\29\ Demyelination 
        is common in spinal cord injury and disease states such as 
        Multiple Sclerosis, and interferes with signal conduction 
        between the neurons. Human cells from adult have been used to 
        treat animal models of disease states. For example, human cells 
        led to functional improvement in animal models of Parkinson's 
        disease using human bone cells \30\ or using neural stem 
        cells.\31\ Human brain adult stem cells can even be obtained 
        after death \32\ so if a person's own stem cells are not used; 
        there are other less objectionable alternatives. Another 
        alternative to the use of embryonic stem cells is human 
        umbilical cord blood. Human umbilical cord blood has the 
        potential to form neurons \33\ \34\ as well as other cell 
        types.\35\ Human umbilical cord blood injected IV caused a 
        functional improvement when injected into experimental animals 
        with traumatic brain injury or stroke.\36\ \37\ Bone marrow 
        stromal cells from adult rats promote functional recovery after 
        spinal cord injury in rats when given 1 week after injury,\38\ 
        even when the cells are injected intravenously.\39\ Bone marrow 
        stromal cells also will migrate to site of a head injury when 
        given IV and caused a functional improvement.\40\
---------------------------------------------------------------------------
    \28\ Y.-Y. Jang, M.I. Collector, S.B. Baylin, A.M. Diehl, S.J. 
Sharkis, Hematopoietic stem cells convert into liver cells within days 
without fusion. Nature Cell Biology: 6, 532-539, 2004.
    \29\ Akiyama Y; Honmou O; Kato T; Uede T; Hashi K; Kocsis JD: 
Transplantation of clonal neural precursor cells derived from adult 
human brain establishes functional peripheral myelin in the rat spinal 
cord. Exp Neurol 167:27-39, 2001.
    \30\ Hou LL. Zheng M. Wang DM. Yuan HF. Li HM. Chen L. Bai CX. 
Zhang Y. Pei XT.[Migration and differentiation of human bone marrow 
mesenchymal stem cells in the rat brain].Sheng Li Hsueh Pao--Acta 
Physiologica Sinica. 55(2):153-9, 2003.
    \31\ Liker MA. Petzinger GM. Nixon K. McNeill T. Jakowec MW.Human 
neural stem cell transplantation in the MPTP-lesioned mouse. Brain 
Research. 971(2):168-77, 2003.
    \32\ Palmer TD. Schwartz PH. Taupin P. Kaspar B. Stein SA. Gage FH. 
Cell culture. Progenitor cells from human brain after death. Nature. 
411(6833):42-3, 2001.
    \33\ Sanchez-Ramos JR. Song S. Kamath SG. Zigova T. Willing A. 
Cardozo-Pelaez F. Stedeford T. Chopp M. Sanberg PR. Expression of 
neural markers in human umbilical cord blood. Experimental Neurology. 
171(1):109-15, 2001.
    \34\ BuzaAska L. Stachowiak E. Stachowiak M. DomaAska-Janik 
K.Neural stem cell line derived from human umbilical cord blood--
morphological and functional properties. Journal of Neurochemistry. 85 
Suppl 2:33, 2003.
    \35\ Goodwin HS. Bicknese AR. Chien SN. Bogucki BD. Quinn CO. Wall 
DA. Multilineage differentiation activity by cells isolated from 
umbilical cord blood: expression of bone, fat, and neural markers. 
Biology of Blood & Marrow Transplantation. 7(11):581-8, 2001.
    \36\ Lu D. Sanberg PR. Mahmood A. Li Y. Wang L. Sanchez-Ramos J. 
Chopp M. Intravenous administration of human umbilical cord blood 
reduces neurological deficit in the rat after traumatic brain injury. 
Cell Transplantation. 11(3):275-81, 2002.
    \37\ Sanberg PR. Chopp M. Willing AE. Zigova T. Saporta S. Song S. 
Bickford P. Garbuzova-Davis S. Newman M. Cameron DF. Sanchez-Ramos 
J.Potential of umbilical cord blood cells for brain repair. Journal of 
Neurochemistry. 81 Suppl 1:83, 2002.
    \38\ Hofstetter CP. Schwarz EJ. Hess D. Widenfalk J. El Manira A. 
Prockop DJ. Olson L. Marrow stromal cells form guiding strands in the 
injured spinal cord and promote recovery. Proceedings of the National 
Academy of Sciences of the United States of America. 99(4):2199-204, 
2002.
    \39\ Akiyama Y. Radtke C. Honmou O. Kocsis JD. Remyelination of the 
spinal cord following intravenous delivery of bone marrow cells. 
[Journal Article] GLIA. 39(3):229-36, 2002.
    \40\ Lu D, Mahmood A, Wang L, Li Y, Lu M, Chopp M.(2001) Adult bone 
marrow stromal cells administered intravenously to rats after traumatic 
brain injury migrate into brain and improve neurological outcome. 
Neuroreport 12:559-63.

  14.  There has been progress in treating genetic disorders using 
        adult stem cells or viruses in animal studies but no progress 
        using cloned stem cells to treat genetic disorders in animals. 
        In the case of genetic defects, there are several other 
        alternatives to cloning. One is gene therapy that has been 
        successfully used in mice \41\ and humans. More recently stem 
        cells have been used as vehicle to deliver genes to the 
        brain.\42\ \43\ \44\ \45\ Another valuable source of research 
        into genetic disorders is adult stem cells that can obtained 
        from patients with genetic defects or strong genetic background 
        to develop particular diseases.
---------------------------------------------------------------------------
    \41\ Shen JS. Watabe K. Ohashi T. Eto Y. Intraventricular 
administration of recombinant adenovirus to neonatal twitcher mouse 
leads to clinicopathological improvements. Gene Therapy. 8(14):1081-7, 
2001.
    \42\ Schwarz EJ. Reger RL. Alexander GM. Class R. Azizi SA. Prockop 
DJ. Rat marrow stromal cells rapidly transduced with a self-
inactivating retrovirus synthesize L-DOPA in vitro. Gene Therapy. 
8(16):1214-23, 2001.
    \43\ Nakano K. Migita M. Mochizuki H. Shimada T. Differentiation of 
transplanted bone marrow cells in the adult mouse brain. 
Transplantation. 71(12):1735-40, 2001.
    \44\ Park KW. Eglitis MA. Mouradian MM. Protection of nigral 
neurons by GDNF-engineered marrow cell transplantation. Neuroscience 
Research. 40(4):315-23, 2001.
    \45\ Ehtesham M. Kabos P. Gutierrez MA. Chung NH. Griffith TS. 
Black KL. Yu JS. Induction of glioblastoma apoptosis using neural stem 
cell-mediated delivery of tumor necrosis factor-related apoptosis-
inducing ligand. Cancer Research. 62(24):7170-4, 2002.

  15.  Tremendous progress has been made using adult stem cells in 
        clinical trials in treating diseases and injuries. You have 
        already heard about the wonderful results of Dr. Levesque at 
        Cedars-Sinai in treating Parkinson's disease using a person's 
        own stem cells. I would now like to describe the use of 
        olfactory mucosa in the treatment of spinal cord injury.
Olfactory Mucosa
    The olfactory mucosa lines the upper nasal cavity. It all starts 
with a brilliant neurologist from Portugal named Dr. Carlos Lima. He is 
also a pathologist that has published on the olfactory system and 
studied a collection of hundreds of olfactory mucosas from cadavers. In 
1991 which is the year before stem cells were first discovered in the 
brain, he decided to explore the potential of olfactory mucosa in the 
treatment of spinal cord injury because the olfactory system was the 
only system in the adult nervous system that regenerates. With very 
limited facilities, Dr. Lima began a study using 14 guinea pigs in 
which the spinal cord was completely cut (transected). A week later, he 
implanted a piece of olfactory mucosa from the nose of that animal. He 
noticed that the guinea pigs that received the transplant were able to 
walk much better than the guinea pigs without the transplant. When he 
examined the spinal cords, the guinea pigs that improved showed tissue 
bridging between the 2 cut ends.
    We now know that there are several advantages to the olfactory 
mucosa. The major advantage of the olfactory mucosa is its lifelong 
continual regenerative capacity including the production of nerve 
cells. It is also accessible with minimally invasive techniques. The 
olfactory mucosa contains 2 cells types that we know help repair the 
nervous system: stem cells and olfactory ensheathing cells. The 
olfactory ensheathing cells encourage the growth of nerve cell 
processes (axons) and promote the myelination (covering on nerve cell 
processes that speed up the signal between neurons). Removal of part of 
the mucosa causes no permanent damage to olfaction (smelling). Problems 
of rejection, overgrowth, disease transmission, and ethical issues can 
be avoided because a person's own olfactory mucosa can be used.
    When Dr. Lima visited my lab, he showed me and my collaborator, Dr. 
Jay Meythaler, his procedure. I began a rat study with that was 
supported by the Foundation for Neural Repair. In this study, we 
compared a wide variety of treatments in rats with chronic, severe 
spinal cord injury. The person doing the functional testing was unaware 
of the treatment that the rat received. The average functional scores 
of the 6 weeks prior to the treatment period were compared to the 
average functional scores of weeks 5-10 after treatment. The 
improvement was greatest in the rats with the olfactory mucosa 
transplants. Also improvement was found in the rats that received bone 
stromal cells IV injections. This improvement with the olfactory mucosa 
cells is the greatest improvement that I have found in the 12 years of 
evaluating treatments for severe spinal cord injury. Below is the graph 
of the results:

[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]

    Excellent graft integration and reduction in lesion size were 
observed in the spinal cords of rats receiving the olfactory mucosa 
transplants.
Clinical Trials by Dr. Carlos Lima and Colleagues in Portugal
    Based on the animal results, Dr. Lima proposed a clinic trial in 
Portugal. A team of physicians was formed that was headed by the 
neurologist and pathologist, Dr. Carlos Lima and included the 
Neurosurgeon, Dr. Jose Pratas-Vital, an Otolaryngologist, Dr. Pedro 
Escada; and a Neurosurgeon, Dr. Armando Hasse-Ferreira. As a first step 
in this procedure, the team of doctors did numerous sham operations on 
cadavers to master the technique. The whole procedure was reviewed and 
approved by the Ethical Committee and Administration of the Hospital 
Egas Moniz-Lisbon. Dr. Lima and his team of doctors have requested that 
I present the results of the study. All of the people were treated in 
Portugal between 6 months and 6 years after their injury. The normal 
improvement, if any, that occurs after spinal cord injury takes place 
in the 6 months to a year after injury so these patients were treated 
at a time when no further improvements are expected. In this procedure, 
the area of the spinal cord damage is exposed surgically in patients 
with severe spinal cord injuries. Then a small piece of olfactory 
mucosa in the upper part of nose is removed from that same patient. The 
olfactory mucosa is then rinsed, cut in small pieces and placed in the 
spinal cord. Below are the MRIs of one of the patients from Portugal 
named Ana: The area that the arrow is pointing at on the left is the 
MRI before the treatment. There is a cystic cavity that appears white. 
On the right is the MRI after the treatment, the arrow points to the 
same area that is almost completely filled.

[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]

It appeared that as in the animal studies, there was bridging of the 
injury. However, it is impossible to tell that there was tissue in a 
living individual but it is probable.
    All of the patients tolerated well the surgery. Olfaction returned 
to normal by 3 months after the surgery. All of the patients showed 
improvements. One of the patients regained bladder control at 15 months 
after the surgery. Regaining bladder control is extremely important to 
those patients with spinal cord injury. All but one of the patients 
gained feeling in some areas of their body where they previously had no 
feeling. All of the patients gained the ability to move certain muscles 
that they could not move before the olfactory mucosa treatment.
    In order to quantify the changes as a result of the treatment, an 
evaluation called the ASIA neurological exam is used. As you can see 
from this diagram below, points are given for each part of the body 
that has sensation or movement. A normal person has 112 on the sensory 
scale and 100 on the motor scale. The results of his first seven 
Portuguese patients that were treated from 6 months to 6 years after 
injury are presented using the ASIA neurological exam.

[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]

    The beginning score (Pre-Op) is the score before receiving the 
olfactory mucosa treatment and is shown on the far left. The results 
after the olfactory mucosa treatment by Dr. Carlos Lima and colleagues 
are recorded at every six months after surgery. The patients were 
operated at different time so some of the patients only have a few 
scores so far. An increase in score means that there is an increase in 
sensory or motor function.

[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]

    In summary, all of his patients that were treated with the 
olfactory mucosa showed some improvement. However, most of the patients 
did not have access to the best rehab facilities. This was very 
frustrating because it appeared that the patients would improve further 
if only better rehab facilities were available.
    In hopes of the patients being able to have access to better rehab 
facilities, several American patients that had requested the treatment 
were enrolled in the clinical trial. Some of these patients were 
carefully evaluated by physicians in the U.S. before and after the 
olfactory mucosa treatment in Portugal. Two of these brave young women 
are here today to tell about their experiences.
Results in Two Americans after Olfactory Mucosa Treatment by Dr. Lima
    Laura Dominguez had her accident on July 3, 2001. She had no 
movement of her legs or hips and no feeling below her collarbone. 
Laura, was 18 years old, tetraplegic with a lesion at the 6th cervical 
level that was 2 cms long. The lesion was mixed glial and connective 
tissue produced by a contusion and laceration. She went to a variety of 
excellent rehabilitation centers including Dr. John McDonald's in St. 
Louis and Project Walk in California. These centers helped her improve 
her upper body strength but still she could no move her hips, legs or 
feet and she had no feeling in these areas. In the U.S., Dr. Steve 
Hinderer and The Rehabilitation Institute of Michigan (currently headed 
by Dr. Jay Meythaler, associated with Detroit Medical Center and Wayne 
State University) began to look into the potential of Dr. Lima's 
procedures on the encouragement of Fred Nader whose daughter had a 
spinal cord injury. After almost 2 years after her accident, Laura and 
her family decided to go to Portugal to have the olfactory mucosa 
surgery performed by Dr. Lima and his team of doctors in March of last 
year. After her surgery, she regained some sensation and motor control 
of certain muscles. She is now able to point her toes. With braces, she 
is able to walk some distance. Although she has made remarkable 
improvements, a rehabilitation program that is actually tailored to 
these types of patients needs to be developed. Laura has received some 
help in developing a vigorous rehabilitation program from a talented 
karate instructor named Ivan Ujeta. Aquatherapy (water therapy) has 
proven to be particularly helpful. However, Laura and her family feel 
that rehabilitation programs need to be developed.
    Susan Fijt was in a car accident on Nov. 17, 2001. The spinal cord 
lesion was at thoracic level 7 and 8 and was about 3 cms long. Susan 
was an ASIA A (complete). She had no voluntary or sensory below her 
level of injury. Susan had no sensory or motor on S4-S5 segments. At 
about 21/2 years after her injury, Susan went to Portugal to have the 
surgery performed by Dr. Lima and his team in June of last year (2003). 
She started to have real gains around 6 months after the olfactory 
mucosa treatment with increased bladder control, sensory recovery and 
first movements of her thigh muscles. Susan and her father looked for 
the best rehab program; however, it seemed that optimal rehabilitation 
program has yet to be designed. Her father, John Fijt with her help 
began to develop and patent devices such as a cross-trainer, standing 
wheel-chair (Venus craft), and camel wheel-chair (lowers or raises to 
facilitate going into and out of the pool) that would help her 
progress. She gained voluntary movements on thigh muscles. In May at 
Dr. Albert Bohbot in France, Susan got more strength on these muscles 
and began walking on a walker with braces on legs. The graph below 
shows the changes in her ASIA scores.

[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]

    The story of these 2 courageous young women dramatically shows the 
progress of adult stem cells and tissue and the need for further 
research into the less profitable, but more beneficial, direction of 
adult stem cells. Further work is needed to improve this technique, 
with the addition of other treatments including a rehabilitation 
program that will maximize the functional improvement.
    My statements represent my scientific viewpoint and not the opinion 
of The University of Alabama at Birmingham which has no official 
opinion on this topic. A special note of thanks to Dr. Joseph Horton at 
The University of Alabama at Birmingham who arranged for the 
digitization of some of the MRIs on very notice.

    Senator Brownback. Very good.
    Dr. Weissman, you've testified many times, and I'm 
delighted to have you back again.

 STATEMENT OF IRVING WEISSMAN, M.D., KAREL AND AVICE BEEKHUIS 
   PROFESSOR OF CANCER BIOLOGY, DIRECTOR OF THE INSTITUTE OF 
CANCER AND STEM CELL BIOLOGY, AND PROFESSOR IN THE DEPARTMENTS 
                         OF PATHOLOGY, 
         DEVELOPMENTAL BIOLOGY, AND BIOLOGY, STANFORD 
                 UNIVERSITY SCHOOL OF MEDICINE

    Dr. Weissman. Thank you.
    So my name is Irv Weissman. I'm an M.D. I'm Director of the 
Stanford Institute of Cancer and Stem Cell Biology and 
Medicine. I'm a stem cell biologist.
    We purified blood-forming stem cells first from mouse, and 
later isolated human brain-forming stem cells. Blood-forming 
stem cells regenerate the blood in the immune systems after 
radiation exposure or after high-dose cancer therapies.
    I cofounded Cellerant, Inc., to transplant human blood-
forming stem cells to regenerate the blood in these patients, 
and also to replace genetically defective blood systems with 
healthy stem cells in diseases such as sickle cell anemia and 
the autoimmune diseases. We have shown, in diabetic mice, that 
a blood-forming stem cell transplant from a genetically 
resistant donor permanently blocks the autoimmune reaction that 
kills the insulin-producing cells.
    Such stem cell transplants also block autoimmune reactions 
in mouse models of Multiple Sclerosis and Lupus, to name a few. 
And the hosts whose immune systems come from a stem cell donor 
can, for life, accept the tissue, organ, or cell transplants 
from that donor without any anti-rejection drugs. That is, the 
donor system repopulates the body. It won't reject the host, 
and it won't reject itself.
    I also cofounded a company called Stem Cells, Inc., to 
treat neurodegenerative diseases, the kind that Dr. Levesque 
was just talking about, by transplanting brain stem cells, 
adult-type tissue brain stem cells. The company has promising 
data in treating mice that have a mouse model of a human fatal 
childhood neurodegenerative disease--that one's called Batten's 
disease--and mice with spinal cord injury, and a variety of 
demyelinating diseases. We are also currently testing these 
cells in a mouse model of human Alzheimer's disease with a 
group in Montana. In all of these tests, only small numbers of 
purified stem cells are required to give lifelong and robust 
tissue regeneration.
    I do not have any connection with any commercial entity in 
the area of embryonic stem cells or nuclear-transfer-produced 
pluripotent stem cells. While I'm probably the strongest 
advocate of adult-tissue stem-cell approaches, I'm also the 
strongest critic of unproven stem cell discoveries.
    You may have heard that one kind of adult-tissue stem cell 
can easily, robustly turn into any adult tissue. I was 
especially excited with claims that the blood-forming stem 
cells, the ones that we discovered, could regenerate injured 
hearts or brains or muscles or insulin-producing cells. But 
when we tested these notions directly and experimentally with 
purified blood-forming stem cells, or any bone marrow cells, 
the blood-forming stem cells only made blood. They did not 
regenerate the heart, the brain, the muscle, or insulin-
producing islets. So we were very disappointed.
    What about embryonic stem cells from in vitro fertilization 
clinics, and nuclear-transfer stem cells? I'll call them NT 
stem cells. The current embryonic stem cells allowed by 
President Bush to be studied with government funding are 
important in studying human developmental biology, but cannot 
tell us about human inherited diseases or be used in transplant 
therapies.
    NT stem cells are made, for example, by taking a skin cell, 
putting it into an egg that lacks chromosomes--they had it 
removed--stimulating it to divide to form a stage at which you 
can make these pluripotent stem cell lines, so they come from 
the donor nucleus. These stem cell lines develop in a test tube 
into every cell type in the body. We can do it in mice.
    If the skin cell comes from a donor with a Bubble Boy 
immunodeficiency--you remember John Travolta in the movie?--the 
mouse donor of that stem cell gives rise to a stem cell line 
that redevelops that disease. If it comes from a cancer stem 
cell--say, a melanoma--the stem cell line redevelops the 
melanoma, whether it's in a mouse or in a test tube. Perhaps 
even cells from a complex inherited disorder, like Lou Gehrig's 
disease, will someday make stem cell lines that undergo 
motoneuron degeneration in the lab. These are scientific 
discoveries now present in mouse labs.
    There's something in common between virtually all human 
genetic diseases and all human cancers. That is, although we 
are finding out which genes seem to be involved, thanks to the 
Human Genome Project mapping genes that correlate with the 
disease, but we don't know in which cells and how the disease 
develops. And to find treatments and cures, that is just what 
we must know. We must understand how genetic defects in humans 
that lead to disease cause that disease if we're going to get 
anywhere to try to cure these diseases.
    There's a promising field publicly called therapeutic 
cloning, where you start with a cell from you to make a stem 
cell line transplantable to you. That field is just at the 
beginning, but if we can make--if the scientific community 
could make progress, it has enormous therapeutical potential 
also.
    So we come to the problem. It would be certainly of great 
medical benefit to open these platform technologies to produce 
predefined stem cell lines. Imagine if we had, and could 
distribute to the best and the brightest, a juvenile diabetes 
stem cell line from a juvenile diabetes patient, or a Lou 
Gehrig's disease stem cell line that recapitulates that 
disease. Today, the best and brightest biomedical experts in 
the U.S. cannot receive or use such cell lines because they 
would have been made after August 9, 2001. It doesn't make 
sense to me. What makes even less sense is the bill proposed to 
criminalize all aspects of producing, studying, and even 
developing treatments using NT stem cell technology.
    If this turns out to be like the recombinant DNA example 25 
years ago, which we regulated rather than banned, tens of 
thousands of born human lives are at stake. In my view, whoever 
of you acts to ban this research is responsible for the lives 
it could save. I know that's a hard statement, but I believe 
that. As an M.D., I took an oath to try to save those kinds of 
lives.
    Banning research for an ideology is just not the American 
way. It's more like Russia, which, in the 1930s, banned 
Darwinian genetics, Darwin's genetics, in favor of Lamarckian 
approaches espoused by Stalin's advisor, Lysenko. We all know 
what happened there. Some scientists were fired, others jailed, 
and others emigrated to the U.S. to set up the U.S. as the 
world leader in genetic and biological research. For at least 
50 years, Russia didn't produce any advances in genetics. Their 
crops failed, and few premier Russian geneticists were trained. 
The biotechnology industry passed them by, and Russian patients 
suffered. Fifty years.
    I beg you to think hard about what you do before you enact 
the first ideological ban of biomedical research in the history 
of the U.S. Separate the issues and ban reproductive cloning of 
humans, because that needs to be done to protect patients. 
That's a whole 'nother subject.
    There has to be a stem cell research bill that funds and 
regulates this kind of research. We want it regulated. We want 
it regulated like recombinant DNA. We want to make sure no 
rogue labs take advantage of it. Don't put us on the sidelines 
while we read of advances in South Korea, the UK, Singapore, 
Israel, or China. Remember, nearly every American family has a 
family member or a close friend with one of the diseases this 
technology could help.
    I thank you.
    [The prepared statement of Dr. Weissman follows:]

 Prepared Statement of Irving Weissman, M.D., Karel and Avice Beekhuis 
 Professor of Cancer Biology, Director of the Institute of Cancer and 
   Stem Cell Biology, and Professor in the Departments of Pathology, 
   Developmental Biology, and Biology, Stanford University School of 
                                Medicine
    My name is Irv Weissman. I received my MD degree in 1965 from 
Stanford, where I am now the Karel and Avice Beekhuis Professor of 
Cancer Biology, Director of the Institute of Cancer and Stem Cell 
Biology and Medicine, and Professor in the Departments of Pathology, 
Developmental Biology, and by courtesy, Biology; I attach my full CV 
for your information. I was also Chairman of the National Academies 
(NAS, NAE, IOM, NRC) Panel on the Scientific and Medical Aspects of 
Human Reproductive Cloning, which also dealt with the issue of human 
pluripotent and human embryonic stem cell research.
    My field of research is adult tissue stem cell biology. We were 
first to isolate any adult (or tissue) stem cell--the mouse 
hematopoietic (blood-forming) stem cell (HSC), followed by the human 
HSC, the human CNS (brain cell forming) stem cell, and most or all 
blood system committed progenitors in mouse and man.
    I am cofounder of the following adult or tissue stem cell 
companies--Stem Cells, Inc (mainly human CNS stem cells) and SyStemix, 
Inc (human HSC). SyStemix released the stem cell service transplant 
functions to Celtrans, now Cellerant, Inc, to deliver human HSC and 
blood system progenitors to patient populations. I own stock in Stem 
Cells, Inc, and Cellerant, Inc and am a Director of both companies. 
These relationships have been disclosed to Stanford, and subjected to 
extensive review to assure avoidance of conflicts of interest, 
including the establishment of oversight committees, when indicated . I 
have no commercial or advising relationship with any for profit entity 
in the fields of human embryonic stem cells or nuclear transfer (NT) to 
produce human pluripotent stem cells .
    As a scientist in adult tissue stem cell research I have played a 
role in helping define the field, and in that role pointing out errors 
or misstatements or less than rigorous research. Stem cells are defined 
as cells that can divide to give rise to new stem cells, by a process 
we call self-renewal; and also progenitors and mature tissue cells, in 
a process called differentiation. The clonal progeny of a single HSC 
include HSC and all blood cells . The progeny of brain stem cells 
include more brain stem cells, as well as the differentiated brain cell 
types.
    Any use of the term stem cell must have the characterization of the 
cell (at the single cell level), include a proof of the capacity for 
self-renewal and differentiation. These are generally accepted 
definitions of the field by the leaders in the field of stem cell 
biology. However, much of the testimony you have heard in the past and 
will continue to hear have fallen short of this standard. All those who 
have testified, are testifying and will testify should be held to that 
standard. So far, isolated tissue stem cells upon transplantation to 
appropriate hosts results in robust regeneration of all the kinds of 
cells in the tissue from which the stem cells were isolated, almost 
always requiring only small cell numbers.
    Many clinically important avenues have been opened by this type of 
adult tissue stem cell research. For example, human HSC (blood-forming 
stem cells) have been isolated from patients with widespread cancers, 
in which the cancer cells and the HSC are intermixed in blood and bone 
marrow; the isolated blood-forming stem cells are no longer 
contaminated with cancer cells. In 3 early phase clinical trials it was 
shown that these pure HSC's regenerate the blood forming system of 
patients treated with massive doses of chemotherapy; the chemotherapy 
is used to kill as many cancer cells in the body as possible, and the 
HSC transplants (that are not cancer cell contaminated) restore blood 
formation as efficiently as any bone marrow transplant, but without 
giving back cancer cells to the patient. In mouse models of human 
disease we have been able to replace the disease-causing blood forming 
system with a blood-forming system that resists that disease; an 
example is mouse type 1 (juvenile) diabetes, where a timely transplant 
permanently stops the autoimmune attack on the insulin-producing 
pancreatic cells. Other such blood diseases include sickle cell anemia, 
thalassemia, severe-combined immunodeficiency (the so-called bubble boy 
disease), and the mouse model of lupus, among many others.
    In addition, the replacement of the blood forming system of mouse 
strain A with HSC from mouse strain B has allowed the permanent 
transplantation of heart, or skin, or insulin-producing islet cells 
from B donors to A hosts without any subsequent immunosuppression. 
Nuclear accidents and exposure to other blood-destroying agents can 
only be treated with HSC or blood progenitors.
    We are now exploring in mouse models the utility of brain-forming 
stem cells in various neurodegenerative disorders, including spinal 
cord injury, inborn errors such as Batten's Disease, Niemann-Pick, etc, 
as well as Alzheimer's, Lou Gehrig's, Parkinson's, and Huntington's 
Diseases, and even cerebral palsy. All of this research is at an early 
stage, and we cannot predict which, if any indications will be 
ameliorated or cured. You might think that I am biased. But science is 
a field that demands independent replication, so any bias I have will 
be tested empirically. For all of these reasons, you should know that I 
am the strongest possible advocate for tissue (adult) stem cell 
research and therapies, but I am also the strongest critic of 
inappropriate extrapolations and inadequate claims from 'stem cell' 
therapies that are unproven. We have only found adult tissue stem cells 
so far for a few tissues, and much discovery research will be needed to 
find others, if they exist.
    A central issue in this hearing is whether adult or tissue stem 
cells of one type, can change their fate to that of another tissue, for 
example blood-forming stem cells to brain, or heart, or skeletal 
muscle, by transdifferentiation. When the first reports of HSC 
transdifferentiation to regenerating heart cells, or brain cells, or 
liver cells, or skeletal muscle cells were reported I was excited that 
the HSC we had isolated might have much broader clinical uses than we 
had initially envisioned. So we embarked on experiments to repeat the 
original findings., hoping to make them better understood and easier 
and more efficient by improving the processes involved. But we found 
that we could not confirm blood-forming stem cells giving rise to 
brain, or heart, or liver, or skeletal muscle in a robust fashion. I 
attach several of our papers that represent attempts to reveal normal 
tissue regeneration using stem cells from distinct tissues. In brief, 
we could not substantiate the claims; only rare (less than 0.1 percent) 
of any damaged and repairing tissues (heart, leg muscles, brain) had 
donor cell markers in regenerating host tissues. All of these rare 
cases of donor markers in host cells turned out to be due to a very 
rare event that can occur in tissue damage--the fusion of donor blood 
cells used in mopping up damaged areas with resident tissue cells that 
survived the damage, not the transdifferentiation of blood-forming stem 
cells to brain, or heart, or muscle, or liver. These findings, like 
many in biomedical sciences, turned out to be due to different 
interpretations of similar findings, or due to some consistent 
misleading methods to reveal the underlying phenomena. All of us in the 
life sciences have experienced the disappointment that what we thought 
was a major finding turned out to be due to something other than we 
suspected at the time. Luckily, the practice of studying particular 
subjects in several independent labs provides a continually self-
correcting aspect to our field. Moreover these cell fusions were rare 
and not robust events leading to massive tissue regeneration. While 
with added experiments these rare cell fusion events may turn out to be 
of some biological interest, none of us should expect that such cells 
provide a means to regenerate different tissues and organs. On the 
other hand, adult tissue stem cells (as described above) can lead to 
robust regeneration, but only of the tissue from which they came.
    These findings (and others) have led us to posit several 
requirements, all of which should be met before one begins clinical 
trials in stem cell research, and of course before the press should 
pronounce preliminary results as conclusions and before legislative 
bodies should base their decisions on these findings as facts. These 
are:

  (1)  The original research finding must be published in a peer-
        reviewed journal . . . but that is not enough.

  (2)  The experiments as reported must be replicated in several 
        independent laboratories . . . but that is not enough.

  (3)  Any way you investigate the phenomenon you should be able to 
        come to the original conclusions . . . but that is not enough.

  (4)  Preclinical (i.e., animal) experiments should show that the 
        injected cells can robustly regenerate the damaged tissues in a 
        timely fashion before they should be considered for human 
        clinical trials.

    About 3 years ago I was asked by the Presidents of the National 
Academies (National Academy of Sciences, National Academy of 
Engineering, National Research Council, Institute of Medicine of the 
National Academies) to lead a panel to gather information and provide a 
thorough, objective report on two related issues, the scientific and 
medical aspects of human reproductive cloning, and the use of nuclear 
transfer technology to produce human pluripotent stem cell lines. They 
chose the panel to provide experts in the related fields of life 
sciences, medicine; and medical ethics as it applies to human 
participants in medical research trials or experiments. We all agreed 
that we had not made up our minds on these subjects beforehand; that we 
would gather as much data as could be obtained; that we would have a 
public meeting of experts and would-be practitioners of both fields; 
and that we would keep our deliberations and thoughts confidential 
until we had heard and read all of the relevant data and had discussed 
them thoroughly, and prepared our consensus report for public 
disclosure. I have appended the executive summary of that report.
    In brief, we found from very extensive animal studies that a clonal 
embryoid blastocyst (I call it embryoid because it was not generated by 
sperm-egg fertilization, but by transfer of a body cell nucleus into an 
egg whose own nucleus had been removed) implanted into the uterus of a 
hormonally prepared female of the same species only results in a live 
birth in 0.8 percent of the cases, and even in those cases most died 
soon after birth. More ominously, unlike a miscarriage that is over in 
the first trimester without measurable morbidity or mortality, these 
reproductive clones aberrantly died throughout pregnancy, often taking 
the mother with them. This would clearly be an unacceptable risk for 
humans, as codified in the medical ethics literature, e.g., the 
Nuremburg code. Accordingly, we concluded, unanimously, that there 
should be a legally enforceable ban on human reproductive cloning, 
defining human reproductive cloning as placement in a uterus of a human 
blastocyst derived by nuclear transplantation. As you know, Congress 
has not chosen to separate the issues and provide for such a ban by 
itself.
    From a scientific, medical, and medical ethical perspective, there 
was not considered to be a similar justification for a ban on nuclear 
transfer (NT) to produce human pluripotent stem cells. In order to 
judge the potential scientific and medical value of such research, we 
considered all experiments published in animal systems, and unanimously 
recommended that biomedical research using nuclear transfer to produce 
stem cells be permitted, and called for a broad national dialogue on 
the societal, religious, and ethical issues on this matter. I am here 
today to bring you up to date on these issues so that you can enlarge 
the debate on societal grounds.
    Let me remind you of the process of producing such lines in mice, 
which presumably would be the blueprint for the production of human 
pluripotent cells. A somatic cell (from skin, or other adult tissues) 
is placed into an enucleated egg, the cell resulting from that NT is 
stimulated to divide, resulting in an embryoid 'blastocyst'. That 
'blastocyst' contains about 40 pluripotent (many potentialities) cells 
inside a hollow sphere of so-called trophoblastic cells. The 
trophoblast cells are necessary for the blastocyst to implant in the 
uterus, the trophoblast cells contributing to the placenta. The 
blastocyst cannot proceed to even the next stage of development unless 
it implants and receives signals and nutrition from the uterus. A 
blastocyst lacking the trophoblast cells cannot implant. The 
pluripotent cells of the preimplantation embryoid blastocyst can then 
be removed and cultured to produce the pluripotent stem cell line. 
These pluripotent cells lack the capacity to make reproductive clones, 
and only make all tissue types in a disorganized fashion. Neither these 
nor true embryonic stem cells can make embryos, or fetuses, and 
therefore it would be a misnomer to claim they can be used to generate' 
embryo farms'.
    It has been shown in mice that the genome of the donor body cell is 
what is retained in the pluripotent stem cell line. Nuclei taken from 
mice with severe combined immunodeficiency (the so-called bubble boy 
disease) give rise to pluripotent stem cells that also have that 
disease, seen most graphically if the tissue HSC from these lines are 
transplanted into suitable radiated mice. The pluripotent cells can 
contribute to every other tissue, but can't make immune lymphocytes. 
Correction of the defective gene in the cell line corrects the disease 
even when transplanted into appropriate hosts. This suggests that one 
might be able to develop similar cell lines derived from humans with 
genetically determined diseases such as immunodeficiency, or adult or 
juvenile diabetes, or immune disorders such as lupus, rheumatoid 
arthritis, or multiple sclerosis, or neurodegenerative diseases like 
Lou Gehrig's, some Parkinson's, some Alzheimer's, Huntington's Disease, 
and all lysosmal storage diseases--just to name a few such diseases--to 
try to elucidate how certain genes lead to the disease, whether studied 
in test tubes or in immunodeficient mice. And this is only a short list 
of human genetically determined disorders.
    There are now several experiments in mice that show at least some 
cancers can be used in NT to produce pluripotent cells, so not all of 
the mutations that lead to these cancers prohibit them from being 
reprogrammed to make pluripotent cells. At least one of these, 
malignant melanoma, has been shown by Rudi Jaenisch to redevelop 
melanomas if put into appropriate mice.
    One could also begin to figure out how to develop tissue stem cells 
from a particular person that might be transplanted back into that 
person--perhaps after fixing the defective disease genes--a process 
called therapeutic cloning in the popular press. (This is the only NT 
application that is appropriately called therapeutic cloning). So you 
might think we would be encouraged at the potential medical advances in 
adult stem cell research, in embryonic stem cell research, and in NT 
stem cell research to expand our efforts for new discoveries and new 
therapies in these exciting areas. However, the bills put forward by 
Senator Brownback and Representative Weldon call for banning NT 
research, with criminal penalties at every stage of research as well as 
therapies derived from that research. Before one enacts the first (that 
I know) ban on biomedical research in U.S. history based on ideology, 
not safety, we should realize what will be lost, and think deeply about 
the political, medical, societal, commercial, and moral consequences of 
such a ban. To do so we need to know what experiments and therapies 
today, cannot be accomplished with adult tissue stem cells or the 
allowed human embryonic stem cell lines. These can be summarized in 4 
areas:

  (1)  Genetic diversity of embryonic and pluripotent stem cell lines. 
        The genetic diversity of the usable 9-64 lines currently 
        available is that of the population that in the U.S. undergoes 
        in vitro fertilization; they are largely white, well to do, and 
        always infertile. There is no doubt that the wide variety of 
        racial and ethnic populations that characterize America are not 
        represented in these cell lines, and of course, it would be 
        extremely unlikely if any had the genetically determined 
        diseases such as sickle cell anemia, thalassemia, and adult 
        onset diabetes, to name a few, prevalent in black, 
        Mediterranean, and native American populations resident in the 
        U.S. There are probably tens to hundreds of genetic disorders, 
        and none will be represented in this limited number of cell 
        lines. NT is a method to make sure they are represented.

  (2)  Genetically determined human diseases. The NT technology might 
        give us cell lines important to understand how simple (one gene 
        defect) or multigenic disorders are caused, and how they might 
        be approached and treated. For example, Lou Gehrig's Disease 
        (LGD) is multigenic, resulting in a loss of motor neurons with 
        tragic consequences for reasons we don't understand. If one 
        could have a pluripotent LGD cell line, one might be able to 
        repair one gene at a time, and determine if in test tubes, or 
        in immunodeficient mice (systems wherein mouse embryonic stem 
        cell-derived tissue stem cells can give rise to motor neurons 
        and the muscle cells they serve)whether the disease development 
        is halted. Knowing those genes as validated targets should be 
        useful for medical scientists, gene therapists, stem cell 
        transplanters, and even small molecule pharmaceutical 
        companies.

  (3)  Cancer cells. All cancers differ from other cells in the body in 
        that they have suffered several, if not many genetic mutations 
        or alterations that play a role in their progress from a normal 
        cell to a cancer cell that can spread and kill a person. There 
        are, to date, no exceptions. It is therefore likely that NT 
        research could make available pluripotent cell lines made from 
        real patients' cancers capable of evolving the particular 
        cancer, and these lines should be susceptible to the same kinds 
        of research to define the dangerous genes, and how to attack 
        them. For both reasons (2) and (3) shown above it should be 
        clear that we are hoping for a chance to learn about how these 
        terrible life-shortening diseases develop, how we can 
        intervene, and eventually, how we might cure them. No other 
        methods that I know of and that are presently available allow 
        these kinds of approaches. It is hard for me as an MD and 
        medical researcher to ignore such promising lines of inquiry.

  (4)  Therapeutic cloning. The possibility that we will someday be 
        able to make NT stem cells from us for us could open the way 
        for a broad scale development regenerative medicine. While it 
        is undetermined whether these approaches will replace the few 
        known adult tissue stem cell therapies, it would be foolish to 
        bet the health of the American people that they will not; and 
        in addition, there are many, many tissues that we do not have 
        replacement stem or progenitor cells yet. And even the approved 
        human embryonic stem cell lines will likely not be useful or 
        allowed for direct transplantation therapies, as they are 
        compatible with few or no persons, and they are all grown in a 
        way that they could be contaminated with leukemia viruses from 
        the mouse feeder layers they are grown on. At the same time one 
        should not be susceptible to the hype that tomorrow, or even 5 
        years from now we will have transplantable cells from NT lines 
        for therapies, as these cells are developed from early stage 
        cells, and will need to undergo the changes all of our stem 
        cells naturally undergo to give rise to mature tissue stem 
        cells. We should remember that high quality research takes 
        time, and we must not overestimate how quickly the work will 
        go. But if we don't start, we'll never get there.

    This last point deserves some comment. Congress has been wise 
enough to understand that the support of basic medical research 
eventually leads to medical breakthroughs and medical therapies. No 
line of fundamental biomedical research at the beginning results in 
short-term therapies. One hears often that embryonic stem cell research 
or pluripotent stem cell research must be lacking in possibilities as 
no cures have yet been found. Using that logic, funding NIH and NSF 
should be abandoned. Human embryonic stem cells were first reported in 
1998, first distributed beyond the founder lab a couple of years later, 
and first allowed for NIH funding in 2002, following the President's 
executive order. Any clinical trial with cells takes at least 1-2 years 
to get the cells properly established to be safe and nontoxic, and of 
course several years of preclinical animal experiments to show there is 
an indication for a trial. It is frankly impossibly premature to 
conclude they will not work. And NT pluripotent stem cell lines have 
only been reported once, this year, in a preliminary report from South 
Korea.
    Twice in the 20th century governments approached biomedical 
genetics research with the intent to regulate or ban it (albeit not 
criminalize it). In the late 1970s, and early 1980s the Cambridge Mass 
city council and the Berkeley CA city council considered prohibiting 
recombinant DNA research in their jurisdictions, and the issue of 
safety was raised in the U.S. Congress. Recombinant DNA is spliced 
together DNA segments, and the issue at that time was putting human 
genes like insulin into bacteria to produce human insulin for 
diabetics. Many thought such genetic manipulations could be dangerous, 
and others wished it banned because it offended them, or because they 
reserved to God the right to ``create life''. But instead of banning 
the research, the NIH regulated such research. Even today to carry out 
a recombinant DNA experiment with new methods or possibly dangerous 
genes it is required to seek and obtain approval from these regulatory 
bodies. What was the result? Only the birth of biotechnology, the 
expansion of these research techniques to every branch of biomedical 
research, and the annual saving or making better of >100,000 lives per 
year. Had this recombinant DNA research been banned those lives would 
be saddled with disease or lost. The lost or impaired lives of those 
people would, in my view, be the moral responsibility of those who 
advocated or helped enact the ban. In addition biotech firms were 
started in the U.S., and U.S. citizens were first to get the treatment 
benefits, By now U.S. biotechnology companies rival classical Pharma 
companies for value and world leadership. The U.S. is the world leader 
in these advances, advances that were slow in coming, but undoubtedly 
have changed the lives of diseased patients for the better.
    The second example occurred in the 1920s and 1930s in Russia. At 
that time Russia and the U.S. lead the world in genetics research. But 
in Russia a maverick geneticist named Trofim Lysenko became a science 
advisor to Joseph Stalin, and persuaded Stalin that Darwin and Mendel's 
views on natural selection were wrong. By Darwinism, for example, 
spontaneous variants could occur rarely, and might affect, for example, 
resistance to cold or dark in only about 1 in 1 million seeds. Another 
view, proposed by Lamarck, stated that gradual changes in light and 
temperature over the growing season would cause all plants to undergo 
adaptations, and that all germ cells would transfer such changes. In 
that view one could change the response to cold in a single plant, only 
requiring that winner adaptations would be inherited in seeds; such a 
result would have shaken up American genetics. Unfortunately for Russia 
Stalin chose Lysenko's proposed methods and potential results, a choice 
that proved to be wrong. The tried and true method of painstaking 
determination of the rare cold-resistant ``mutants'' and their 
selection for next generation's produce was left high and dry. So 
Lysenko was revered and Darwinists reviled. The Russian crops failed, 
and the next generation of Russian scientists were untrained in 
genetics. Several important Russian geneticists were blackballed and 
some jailed. Others, migrated to the U.S., or if already in the U.S., 
stayed, where they helped lead the U.S. to unquestioned leadership in 
the field. As a result for the next 50 years Russia produced no great 
geneticists and no great genetics. The biomedical revolution bypassed 
the Russians, as did medical treatments and the economic benefits that 
would have accrued.
    I urge you to think hard whether you wish to overrule good science 
and medicine and ban some kinds of biomedical research and therapies 
for the first time in American history. In my own personal moral view, 
those in a position of advice or authority who participate in the 
banning or enforced delays of biomedical research that could lead to 
the saving of lives and the amelioration of suffering are directly and 
morally responsible for the lives made worse or lost due the ban, or 
even of a moratorium that would deny such treatments in that short 
window of time when it could help or save them. I recognize that for 
some there are strong religious and/or other moral bases for beliefs 
that the NT 'blastocyst' has the same rights as born friends and 
family. In our pluralistic society they have the sovereign right to act 
on their beliefs for their own conduct. But my reading of the oath I 
took upon receiving my MD that the health of the patients are my first 
priority. This supersedes any personal moral, political, ethnic, and 
religious beliefs that would block the treatment of current or future 
patients; and that oath has guided my career. If you have real concerns 
about our economy, or our ability to recruit and train the best and 
brightest for biomedicine, or our ability to develop and prescribe the 
best therapies for our patients, I believe you will choose the American 
way of sensible actions, and when appropriate, regulation, not 
abolition.
    In summary, adult tissue stem cells, embryonic stem cells, and NT 
stem cells each have important and unique properties to allow the 
biomedical and clinical community the opportunity to pursue the 
understanding of human development, the regeneration of damaged 
tissues, the development of human genetic diseases, and the broadest 
possible approaches of translating those discoveries to the treatment 
of patients with grievous diseases. In my view it is irresponsible to 
fail to pursue all such avenues in parallel to stop or ameliorate the 
tragedies our families endure because of these diseases. And of course, 
in my view it is worse than irresponsible to ban these pursuits.
    Thank you for your attention.

    Senator Brownback. Thank you, Dr. Weissman.
    We will focus the hearing on adult stem cells, as I have 
stated that we were focusing on. We've had a series of hearings 
on many other types of stem cells where Dr. Weissman and others 
have testified at many times, and on cloning, so we want to 
focus on adult stem cell research, and that's the focus of the 
hearing.
    Dr. Levesque, thank you very much for being here. Do you 
have some patients that you've treated, of Parkinson's? I 
believe on the next panel, one of your patients will testify, 
is that correct?
    Dr. Levesque. So far, we've transplanted one patient with 
his own neurons derived from his neural stem cells. We are to 
begin a phase two trial in the next few months with possibly an 
additional 15 patients.
    Now, we've harvested many more patients to look at the 
presence of the adult neural stem cells in their brain, and 
this has been an ongoing effort for several years.
    Senator Brownback. Will you describe that one patient, I 
believe, that's going to testify next--Mr. Dennis Turner was 
your first patient, is that correct?
    Dr. Levesque. Yes, that's correct. Well----
    Senator Brownback. Describe his condition, if you would. 
And, actually, if Dennis could step up, that might help, if you 
don't mind doing that, Dr. Levesque.
    Dr. Levesque. Well, it's OK. I would have brought some 
videotape, you know, of medication before surgery and of 
medication after surgery. But Mr. Turner can speak for himself. 
Essentially, he had reached an advanced stage where he would 
have met criteria for implantation using any other type of cell 
therapy. As you know, previously the NIH funded a double-blind 
study using fetal tissue for Parkinson's disease. So using this 
same criteria, he would have been a candidate for this type of 
transplantation. But, instead, we used a population of cells 
derived from his biopsy of a cortex, which regenerated millions 
of his own neural stem cells.
    Senator Brownback. Will you break that down for me? Where 
did you get the stem cells from?
    Dr. Levesque. From his own brain.
    Senator Brownback. From his own brain. Where in the brain?
    Dr. Levesque. Nondominant prefrontal region.
    Senator Brownback. OK. And what did you do with the cells?
    Dr. Levesque. We placed these cells in the media--culture 
media in laboratory, and we isolated maybe 50 of these, what we 
call stem cells. And these cells began to divide for several 
months, until we had over 20 million of his neural stem cells. 
Then these cells were characterized. That is, we were able to 
prove that they can become neurons, glial cells, and other type 
of techniques to demonstrate they are stem cells. And then we 
induced these cells to mature prior to transplantation. And 
then he received an injection on one side of his brain. At that 
time, it was--the criteria was a unilateral implantation--at 
that time, he was extremely afflicted, and more severely on the 
right than on the left, so he received an implantation on the 
left hemisphere, which controls the right side.
    Subsequent to this implantation, it took several months to 
see any significant improvement. In fact, it took over 6 months 
to see a benefit of this type of cell therapy. As you can 
imagine, we're dealing with a biological organ, which is the 
human body and the human brain, and this is not like a switch 
that you turn on and off to reverse the course of a disease or 
transform symptoms overnight. But, overall, the biological 
process took possibly 9 months to have a beneficial effect on 
his symptoms, and his symptoms then progressed and improved 
over the next 3 years, where his symptoms disappeared 
completely on the side that received implantation.
    Senator Brownback. Symptoms disappeared completely on the 
side that received it. So you're going to move this forward 
then to an additional scale of clinical trials?
    Dr. Levesque. Right. We need to study more patients. This 
is just one patient. We need to have a larger series of 
patients, and also evaluate the dosage to know exactly what the 
number of cells that needs to be transplanted to produce the 
most efficacious effect on the patient.
    Senator Brownback. Advanced Parkinson's disease, to the 
point of symptoms disappeared completely----
    Dr. Levesque. Right.
    Senator Brownback.--for this patient.
    Dr. Levesque. You have to realize that only one side was 
operated. The other side was left alone, and he has progressed 
significantly now on the opposite side. And, at this point, 
he'd like to be implanted on the opposite side, and this is 
something we will hopefully offer him in the near future.
    Senator Brownback. That's fantastic. And I'll look forward 
to his testimony.
    Dr. Nelson, you've got a couple of patients from the 
gentleman that you've worked with in Portugal. They'll be here 
and testify. As this procedure has been developed--we'll hear 
from the two ladies--how has it been perfected? What else has 
been done to deal with these massive spinal cord injuries?
    Dr. Peduzzi-Nelson. I've tested a variety of things in the 
lab--different types of growth factors, pumping in growth 
factors, different types of matrices, different cell types, 
different types of stem cells and support cells--and all of 
them have, at one time or other, shown a small benefit. And 
what I've seen in the animal studies--and the reason why the 
animal studies are important is that when you're working with 
people, every injury is different; when you're working with 
animals, you can produce a large group of animals with the same 
type of injury, and then divide the animals up and give them 
different treatments, and have someone who's completely unaware 
of what treatment the animal received test these animals. And 
what we do is, we test them weekly over a course of a year and 
a half. And the surprising results were that the olfactory 
mucosa, as a source of adult stem cells, worked the best.
    But there are also other cell types in the olfactory 
mucosa. There's a support cell called the olfactory ensheathing 
cell. And that cell, when it's purified by itself, others have 
found improvement just using that cell type.
    So we think that olfactory mucosa definitely is something 
that's very promising. And if we can add some other 
combinations to this, we may see even further benefit. And 
there's a real lacking, in terms of rehab methods, that we need 
to perfect the rehab methods so we could see the maximum 
improvement in these patients.
    Senator Brownback. That's fantastic. I'll look forward to 
these witnesses testifying.
    Senator Wyden?
    Senator Wyden. Thank you, Mr. Chairman.
    Dr. Nelson and Dr. Levesque, I think you heard me go 
through the NIH website that outlines, in their view--the 
government's view--the limitations today on adult stem cell 
research. And I just want to ask you if you share the views of 
the government, because it seems to me that you all are taking 
a very different approach.
    For example, on the NIH website, it says, and I'll just 
quote here, ``Adult stem cells are often present in only minute 
quantities, and can, therefore, be difficult to isolate and to 
purify.'' That's a pretty significant limitation----
    Dr. Levesque. That is correct.
    Mr. Hutchinson.--that the Federal Government finds, and I'd 
be curious whether you two share the view of the National 
Institutes of Health on that point.
    Dr. Levesque?
    Dr. Levesque. I agree. This is a difficult proposition, to 
isolate an adult neural stem cell. It's not easy. This is a 
known limitation of the adult stem cells. I mean, there a lot 
of unknown limitations from the embryonic stem cell point of 
view that cannot be understood because we don't have enough 
data or knowledge about this. So I think, as anything in 
science, we need to seek new aspect and new causation factors 
of disease, and we need to address the benefit, pros and cons, 
of all type of tools to treat the disorders.
    So, yes, adult stem cells can be limited in their 
isolation. However, they have benefits, also, in the way that, 
with an autologous approach, you don't have to deal with the 
immune rejections of any implanted tissue. And----
    Senator Wyden. My time's going to be short, and I 
appreciate your saying you agree with the National Institutes 
of Health on that point. They also say that there's evidence 
that they don't have the same capacity to multiply as embryonic 
stem cells do. Dr. Nelson, is----
    Dr. Peduzzi-Nelson. Yes.
    Senator Wyden.--the NIH right, or what do you think?
    Dr. Peduzzi-Nelson. I would say that what--the paragraph 
you read probably represents some of the scientific view, but I 
think in the case of stem cells, less is more. OK? And the 
reason is, there's a problem with embryonic and fetal stem 
cells, is they grow too well. When you put them in animals, 
they grow too well and form tumors, and occasionally kill the 
animal. Adult stem cells exhibit a more controlled growth. 
They're not impossible to grow. I'm not the famous stem cell 
biologist here next to me, but, even in my own lab at the 
University of Alabama at Birmingham, they're fairly simple to 
grow, and they grow in a controlled manner, and it is possible 
to get enough stem cells to use as a treatment. And this 
controlled growth, I think, is the best option, because there 
is not these other problems, such as rejection.
    Senator Wyden. The government states that adult stem cells 
have more DNA abnormalities. Do you share that view or find it 
troubling?
    Dr. Peduzzi-Nelson. What I have found is that, using adult 
stem cells, we haven't found any abnormalities in the animals. 
And I think that statement--it takes me back to a story in my 
childhood where the neighbor said she would make--that Duncan 
Hines had developed a cake mix, and obviously they put a lot of 
research into it, and it has to be better than anything 
homemade. Well, I think that if you talk about abnormalities 
that develop in ourself, in the adult stem cells from our own 
body, and you're saying that growing them in a very artificial 
culture situation leads to more abnormalities, I think at least 
some people would think that a better way to protect the stem 
cells is in your own body.
    Senator Wyden. Again, I think--you say some scientists 
share this view. This is the official position of the Federal 
Government, folks. Federal Government, on its website, is 
talking about the limitations on adult stem cell research that 
obviously you all don't see in the same way.
    Let me ask you about an issue that I think goes to the 
heart, for me, of how we evaluate your views. You, in 
particular, Dr. Levesque, at page 3 of your testimony, are 
quite critical of embryonic stem cell research. It's in the 
third to last paragraph, talking about the ramifications of 
tumors and possible ramifications for the brain, and the like. 
My question to you is, have you done research involving 
embryonic stem cell lines so that you can make that comment on 
the basis of comparing research involving embryonic stem cells 
to adult stem cell----
    Dr. Levesque. I believe you refer to the paragraph where I 
state that the--there's a strong potential for the embryonic 
stem cells to generate any type of cells. The problems we 
have----
    Senator Wyden. My question, Doctor, is, have you done 
research involving embryonic stem cell research? That's a yes 
or no answer.
    Dr. Levesque. The answer is no.
    Senator Wyden. Thank you.
    Dr. Levesque. However----
    Senator Wyden. Dr. Nelson, have you?
    Dr. Peduzzi-Nelson. Yes, I have. I've done--used animal--
there's no ban on animal research using embryonic or fetal stem 
cells, and I have. And I've also used adult stem cells that 
were similarly prepared. These experiments were done at 
slightly different times, but the results were very similar. 
They were slightly better with the adult stem cells. But if 
they're not done at the same time, you can't make a direct 
comparison.
    Senator Wyden. Let me ask you about the ethical concerns, 
Dr. Nelson, that you mentioned. You said you had ethical 
concerns with respect to this research, and that certainly is 
something that crops up again and again in this Committee. What 
exactly are your ethical concerns with respect to embryonic 
stem cell research? And, again, how are those concerns 
alleviated by looking to adult stem cell lines instead?
    Dr. Peduzzi-Nelson. I think there's no ethical controversy 
with regard to adult stem cells. I----
    Senator Wyden. I asked about yours.
    Dr. Peduzzi-Nelson. Oh.
    Senator Wyden. I'd like to know about your ethical 
concerns, as a scientist----
    Dr. Peduzzi-Nelson. OK.
    Senator Wyden.--with respect to embryonic stem cell 
research, so I can factor that in to your argument that the 
focus should be, by the government, on adult stem cells.
    Dr. Peduzzi-Nelson. I didn't present my ethical concerns. I 
do believe that, if given the option of a treatment, a 
direction of treatment, that has not led to tumors and death 
and overgrowth, and you have an option of a treatment, that all 
of the science that I've looked at says that this is a better 
option for the treatment, and the fact that there are no 
ethical concerns using adult stem cells.
    I am here, not on the basis of ethics or politics or 
anything else; I'm here because, in the last 12 years of my 
life, I have been obsessed with finding the best treatment for 
spinal cord injury. This has been my focus, and I haven't 
looked in either direction. And what I see out there is that 
there is a possibility that adult stem cell treatment might not 
go forward because it's a very difficult area to bring forward. 
It's difficult because, commercially, a lot of companies are 
less interested, at this point, in adult stem cells.
    So my main reason for being here today has to do with 
trying to get the best treatment out there for spinal cord 
injury. Whether I'm pro-life or pro-choice, I wish that all 
these types of things could be kept out of the discussion, and 
we can just concentrate on the families and the patients and 
the people suffering out there.
    Senator Wyden. My time is up. And I would only say, ma'am, 
if we look to the patients and the families, they're making it 
clear they want Federal policy to change in this area. That's 
what my constituents come to town meetings and say, that's what 
public opinion polls say, that's what we hear again and again.
    And I will only tell you that what I think is the reason 
that adult stem cell lines aren't being pursued by a lot of 
private companies is, I think that they agree with the National 
Institutes of Health. I mean, I thought it was very important, 
when we come to this hearing, as I think you have, to your 
credit, suggested, that we stick to the facts. And today, on 
the Federal Government's website, they outline at least four 
significant limitations with respect to using adult stem cell 
lines. And that's what's motivating the companies of this 
country. The reason the companies are hesitant to make 
investments in this area, not because any Member of the U.S. 
Senate wants to limit this research, the companies are 
reluctant to make the investments because they share the view 
of the National Institutes of Health that our research 
possibilities in this area are limited.
    Dr. Peduzzi-Nelson. I'd just like to mention----
    Senator Wyden. Mr. Chairman, I know my time is up, but I 
think----
    Senator Brownback. Just go ahead and--please go ahead and 
respond.
    Dr. Peduzzi-Nelson. Just one--two sentences. I'll make it 
two sentence. The procedure that I talked mainly about was the 
olfactory mucosa. It cannot be patented. OK? There is no 
patent. There is no way to get companies interested in there 
because there's no profit in there. That's why the struggle is 
for adult stem cells to go forward, is--if you have techniques 
that are a patent, you don't have intellectual property.
    Senator Wyden. Yes, I----
    Dr. Peduzzi-Nelson. And NIH doesn't tell researchers what 
direction to go, or they think this or think that. People go 
into their research labs and go forward with the best----
    Senator Wyden. I've made it clear that there are areas 
where clearly adult stem cell research can be useful. But what 
is even more clear is that the preponderance of scientific 
evidence is--and I think this is what is guiding these private 
companies looking to investments--is that there are vastly more 
possibilities using embryonic stem cells.
    Dr. Peduzzi-Nelson. I think they might be interested in the 
money, too.
    [Laughter.]
    Senator Brownback. Yes, I think that would be a fair point.
    Senator Lautenberg?
    Senator Lautenberg. Thank you very much, Mr. Chairman.
    Dr. Nelson, you brought up the subject, and I have to 
followup. And everybody is under oath automatically when you're 
testifying before a Committee, even if the hand isn't raised 
and you don't take it. Do you--are you a member of a pro-life 
group in any way?
    Senator Brownback. Is that a relevant----
    Senator Lautenberg. Yes, it's----
    Senator Brownback.--question----
    Senator Lautenberg.--relevant to me, Mr. Chairman. And I do 
have my right----
    Senator Brownback.--for an adult stem cell hearing?
    Senator Lautenberg.--as a Member of the Committee, to ask 
the questions. And I can't--you can't give me a stacked deck 
unless I know what the cards are. And you'll forgive me, Mr. 
Chairman.
    Senator Brownback. Well, but I really----
    Senator Lautenberg. Mr. Chairman----
    Senator Brownback. Is that a relevant question?
    Senator Lautenberg.--I want you to look at the testimony, 
if I must, when it talks about, ``I, Ronald Reagan,'' and she 
quotes the President of the United States, and then denies any 
ethical connection to this. And I want to challenge this, and I 
want to find out the truth, if I may, Mr. Chairman, pursue the 
course of----
    Senator Brownback. Then I guess we should have Dr. 
Weissman, too, say that?
    Senator Lautenberg. Sure. I'd ask--you can ask it on your 
time, Mr. Chairman.
    [Laughter.]
    Senator Brownback. If the witness chooses to answer.
    Senator Lautenberg. Well, if the witness doesn't choose to 
answer, then it can be contempt.
    And are you a member?
    Senator Brownback. No, it cannot.
    Senator Lautenberg. Mr. Chairman, there's an obligation to 
answer the question that's put to you when you sit in the 
witness chair. Why are we having this--why are we having this 
debate? All I--let me defer and give you a chance to think 
about it.
    Dr. Levesque----
    Dr. Levesque: Yes?
    Senator Lautenberg.--does a single case of an outstanding 
reaction to a process make a scientific enterprise a valid one 
to say that that's the way we ought to go?
    Dr. Levesque. Well, it's just one step. I mean, we need to 
evaluate, as I said, more studies.
    Senator Lautenberg. So that single case that you talked 
about is not really indicative of a sense of an appropriate 
scientific course of study.
    Dr. Levesque. Well, I'd disagree with that. I think science 
has to further the evaluation of----
    Senator Lautenberg. Right.
    Dr. Levesque.--this therapy, or not. Science is built on 
several steps, and this is just one step----
    Senator Lautenberg. Just one step, all right. Thank you.
    Dr. Nelson, are you now a member of a pro-life committee in 
any way?
    Dr. Peduzzi-Nelson. In an attempt to not be in contempt of 
court, I honestly say I don't remember joining any of the 
groups that--you know, that are----
    Senator Lautenberg. OK, but you are concerned about the 
ethics of science, because----
    Dr. Peduzzi-Nelson. I think a lot of Americans do not 
believe in creating life to destroy it. You know, I----
    Senator Lautenberg. Yes, OK.
    Dr. Peduzzi-Nelson.--think that's a concern----
    Senator Lautenberg. Fine. That----
    Dr. Peduzzi-Nelson.--of some Americans.
    Senator Lautenberg.--that summarizes an attitude, that you 
are more concerned about the ethic, in my view, because you 
hear, quote, the revered President Ronald Reagan, ``By virtue 
of the authority''--you quote him--``vested in me by the 
Constitution and laws of the United States do hereby proclaim 
and declare the unalienable personhood of every American from 
the moment of conception til natural death.'' So you use that 
as a reference. So that, then, tells me that that's where your 
studies are focused, that you're hewing to a line of morality, 
as you see it, that governs your scientific behavior. Is that a 
fair statement?
    Dr. Peduzzi-Nelson. I'd say I included that in my testimony 
because----
    Senator Lautenberg. Yes.
    Dr. Peduzzi-Nelson.--there are people out there who 
respected--very much respected Ronald Reagan as a President, 
and----
    Senator Lautenberg. As a scientist?
    Dr. Peduzzi-Nelson. They respected him--no, I'm saying 
that, in the U.S., that many people respected President Reagan, 
and recently the reason why there is so much attention right 
now to this area is because of the suggestion that this might 
have helped President Reagan or that----
    Senator Lautenberg. No, but didn't Mrs. Reagan say that she 
would hope that we'd pursue stem cell--embryonic stem cell 
research?
    Dr. Peduzzi-Nelson. I'm just--I presented that as a 
statement. I didn't make this up. I think----
    Senator Lautenberg. No, I know. But----
    Dr. Peduzzi-Nelson.--this is a----
    Senator Lautenberg. But this----
    Dr. Peduzzi-Nelson.--correct quote that--you know, if we 
wanted to----
    Senator Lautenberg. If you'll forgive me----
    Dr. Peduzzi-Nelson.--honor someone's memory----
    Senator Lautenberg. It became--yes, it became, however, an 
anchor for your testimony on science. And, therefore, it has to 
have some relevance, or you wouldn't have put it in there. And 
I just wonder whether we're now going through a political 
discussion or a scientific discussion.
    Dr. Weissman, as a researcher who works mainly with adult 
stem cells, do you support the Federal funding of embryonic 
stem cell research?
    Dr. Weissman. Oh, of course. But I don't think it goes far 
enough. So I support what's happened. I think it was a brave 
move forward to at least make those 64 or so cell lines 
available for study and government funding. But now, on 
reflection, I think it just doesn't go far enough.
    Could I clarify one very small point----
    Senator Lautenberg. Please do.
    Dr. Weissman.--just so everybody understands? Embryonic 
stem cells, mouse or human, can cause tumors if put in the body 
while they're still just embryonic stem cells. But once you 
generate a mature tissue cell, they do not cause tumors. That's 
scientifically accurate.
    Senator Lautenberg. Thank you very much.
    Because in Dr. Nelson's testimony it says, ``the oft-stated 
advantage that embryonic stem cells can make every cell in the 
body is not an advantage for people with disease or injuries,'' 
is that a statement that can reliably be made, Dr. Weissman?
    Dr. Weissman. Well, not in my view. And I think I've made 
the point that what we need to do to be able to understand each 
of these various diseases is not just think of the cells that 
you get out as therapies, but as tools or engines of 
discovering what caused that disease. That's where we really 
need to be able to move to move this field forward for the long 
term. So I disagree with that point of view.
    Senator Lautenberg. Thank you very much.
    Senator Brownback. Senator Nelson?
    Senator Nelson. Thank you, Mr. Chairman.
    Senator Brownback. Thank you.
    Senator Nelson. Dr. Weissman, I'm quoting from--let's see, 
this is your article in the New England Journal of Medicine, 
``I believe that new lines of human embryonic stem cells will 
be needed.'' And then you go on to say, ``One way is by 
transferring somatic cell nuclei into enucleated eggs, nuclear 
transplantation.'' Would you describe that procedure?
    Dr. Weissman. Sure. And this has been done successfully 
many times in mice, maybe once or two times in humans. So you 
take the nucleus from a body cell, let's say a skin cell. That 
cell has genetically been programmed, at that point, to be a 
skin cell, and has the genes on to be skin, but not the genes 
on to be early pluripotent cells. When you put that into an 
egg, by injection, that had its own genetic material removed, 
what's left in the egg, remarkably, stimulates the nucleus of 
that skin cell to reprogram itself, to shut down skin cell 
genes, to open up genes that would make it very early stage.
    Then you have to do something to make it divide. Normally, 
when a sperm/egg fuse, there's no question, because what's 
stimulates division. But you stimulate it to divide. And after, 
if you're lucky--and this is rare--after about seven or eight 
or nine cell divisions, you have a ball of cells, the outside 
of which could, if implanted, start to form a placenta, and the 
inner cells are these pluripotent cells, the ones from which 
you make a cell line. It has no nerve cells, it has no hard 
cells, it has no determined cells at all.
    Senator Nelson. So you're taking the nucleus out of a cell, 
you're transplanting a nucleus in that you want to multiply.
    Dr. Weissman. Right.
    Senator Nelson. And so you're not dealing from a fertilized 
egg.
    Dr. Weissman. That's right.
    Senator Nelson. You're taking the nucleus out.
    Dr. Weissman. At no point do you have fertilization 
occurring in the natural way of making an embryo or a fetus.
    Senator Nelson. Is there progress between adult stem cells 
and the embryonic stem cells?
    Dr. Weissman. There is tremendous progress going on in both 
fields. I hope you understand that my own chosen field, and my 
own consulting with industry, is all on adult stem cells. I 
believe in them deeply. The kinds of experiments that we've 
done with blood-forming stem cells, we've already treated 70 to 
80 patients, I think successfully, in clinical trials. So I 
believe in it. But what we want, what we need, out of nuclear 
transfer to produce these pluripotent stem cell lines is 
entirely different. We cannot possibly do that with adult stem 
cells. We cannot possibly do that with the approved lines from 
in vitro fertilization clinics. In vitro fertilization clinics, 
we must admit, attract people in the United States who are 
mainly white, middle-class to well-to-do, always infertile. 
There are no diseased cell lines coming out of an in vitro 
fertilization clinic, so we can't learn about the diseases that 
we promised the American people we want the help do. The whole 
reason for a National Institutes for Health--H for Health--is 
that we would commit ourselves to carry out research on all 
diseases, that we wouldn't look for barriers in the way not to 
do it.
    Senator Nelson. Dr. Nelson, in the procedure that Dr. 
Weissman has just described, transferring somatic cell nuclei 
into enucleated eggs, nuclear transplantation, do you have an 
ethical problem with that procedure?
    Dr. Peduzzi-Nelson. I think one of the things we have to 
remember--I'd just like to clarify that this is the same 
procedure that was used to produce Dolly, the sheep, which is a 
real sheep. The problem I have with this procedure is that I 
see that millions of dollars can be made from these patent-able 
cell lines. And right now, you could get adult stem cells from 
people with these various diseases, or a strong genetic 
background to develop these various diseases. You could get 
these cells, and not have one cell line from one patient that 
has, for example, juvenile diabetes, but get a large number of 
stem cells in culture so that they can be evaluated. So I think 
this is a better research direction.
    Again, the problem is, the biotech industry expects that 
these patents on these cloned stem cell lines are going to be 
worth millions of dollars when you patent these. If you just 
got an adult stem cell from a child or an adult, and got it 
from a wide variety of people that either have the disease or 
have a strong likelihood of getting the disease, I think this 
is a much better research direction.
    Senator Nelson. You certainly answered that that's your 
preference in the research direction. The question was, Do you 
see an ethical problem with nuclear transplantation?
    Dr. Peduzzi-Nelson. The ethical problem that I see is that 
I don't believe that it's--that you should create human life 
just to destroy it for some vague scientific purpose. I think 
that there is a better research direction that the people out 
there, the patients, deserve to have pursued. It's a less 
profitable direction. And because it's less profitable, I don't 
think it is going to be pursued.
    Senator Nelson. What was the verb or the adjective that you 
used--did you say ``destroy'' human life? What was the verb you 
used?
    Dr. Peduzzi-Nelson. I'm sorry, I guess I have problems with 
verbs here. I don't remember my exact statement. In this 
process, you would create a cell that, if it was implanted in 
the uterus, could develop into a person. And so, I do consider 
this human life.
    Senator Nelson. Even though that cell was set to be 
discarded.
    Dr. Peduzzi-Nelson. It would be lots of cells that would be 
set to be discarded. But if you did take that cell--and we're 
calling it somatic nuclear transplantation--transplant, rather 
than cloning--that cell, if you put it into the uterus, could 
develop into a human being.
    Senator Nelson. And so--I'm trying to understand your 
reasoning--so the fact that that cell could develop into a 
human being is your objection to using nuclear transplantation 
for the purpose of research.
    Dr. Peduzzi-Nelson. My objection to it is, one, I think 
there are better options that--one, there are better options 
that will make less money, that will never be pursued; and, 
two, I do consider it needless destruction of human life.
    Senator Nelson. OK, that's what I was trying to get at----
    Senator Brownback. Your time is----
    Senator Nelson.--to get your----
    Senator Brownback.--up, Senator Nelson. We need to head on. 
Do you have one quick one so we can get the next panel up?
    Senator Nelson. I'm legitimately trying to understand this 
issue, Mr. Chairman, as to why this gets to be such a cat fight 
over ethics when we've got such tremendous promise for research 
and advancing toward the cure of diseases. So I'll continue my 
questioning later.
    Senator Brownback. Thank you.
    Senator Wyden. Mr. Chairman?
    Senator Brownback. Yes?
    Senator Wyden. Mr. Chairman, thank you. Just one quick one. 
And this goes to--again, to the NIH website, Dr. Weissman.
    [Laughter.]
    Senator Wyden. The government says that adult stem cells 
don't give rise to a lot of the tissue types that would be 
important to people. My question to you is--embryonic stem 
cells can be used to grow a variety of tissues. Are adult stem 
cell lines more limited?
    Dr. Weissman. Yes.
    Senator Wyden. So----
    Dr. Weissman. And this, we've tested directly. I work with 
both mouse embryonic stem cells and mouse and human 
hematopoietic, blood-forming, and neural. So the neural stem 
cells only make brain cells. They do it beautifully, they grow 
well. Michel was absolutely right, they are something we want 
to test and we hope will work very well.
    The embryonic stem cells, being pluripotent, can give rise 
to every tissue in the body. We don't yet have adult stem cells 
for every tissue of the body.
    Senator Wyden. Mr. Chairman, thank you.
    Senator Brownback. Thank you.
    We'll have our--I want to thank this panel of witnesses. I 
think they've been quite illuminating, and this science has 
developed significantly, and I appreciate all of your 
testimony, even if it can be difficult to do. Thank you very 
much for being here. And, more importantly, thank you for your 
work. That's extremely important.
    Now I have a panel of patients that'll be coming forward. 
Ms. Laura Dominguez, from San Antonio, Texas; Ms. Susan Fajt, 
from Austin, Texas--both had massive spinal cord injuries and 
will now be here to testify; Mr. Dennis Turner, we've heard 
spoken of already, was a Parkinson's patient, has had 
treatment--are the three patients that we will have on this 
panel. And then Dr. Robert Goldstein, from the Juvenile 
Diabetes Research Foundation in New York, will also testify.
    [Pause.]
    Senator Brownback. I'm excited to hear from this panel, and 
I want to encourage all of you to be calm; nothing to be 
nervous about. I know several of you--OK, that's easy for me to 
say, I apologize. But I do hope you can be calm and just enjoy 
this, because I really think you're an inspiration to a number 
of people that are struggling with horrific difficulties. You 
represent the tip of the spear, going forward.
    Congressman Gonzalez, I understand one of the witnesses is 
a constituent of yours that you would like to introduce. And 
then, once we do that, I would like to go, immediately to a 
video of Laura Dominguez and Susan Fajt. It's a short video, 
showing some of their progress, if that would be acceptable.
    Congressman?

            STATEMENT OF HON. CHARLES A. GONZALEZ, 
                 U.S. REPRESENTATIVE FROM TEXAS

    Mr. Gonzalez. Thank you.
    Mr. Chairman, Members of the Committee, thank you very much 
for giving me this opportunity. Of course, I represent the area 
from which both, of course, Laura and the Dominguez family 
would--actually reside. I have known her father for a number of 
years, having been a state district judge, and we used to refer 
to in those days as a ``baby lawyer,'' he used to appear before 
my court.
    I do want to preface my own statement, and it's going to be 
very short, but that it had been some years since I had seen 
Laura, and the truth is, it had been maybe 6 years. I guess she 
was about 12 or 13 years of age. And, at that time, I saw her 
at a wonderful dance, a celebration that we have in San 
Antonio. And she had the most beautiful dress on. And I 
remember, she may have been a member of someone's court, which 
they have this big celebration. But she was not only walking, 
but she was dancing. And so I would hope that we all join 
forces and hands and open every possible door to research that 
is out there so that one day again I can go to another dance, 
see the Dominguez family, and see Laura dance.
    I am very honored to introduce to this distinguished 
Committee, Laura Dominguez. Laura is a smart and fun-loving 19-
year-old. She enjoys traveling and, just like many other 
teenagers today, spending time on her computer.
    Laura is also one of 200,000 Americans living with a spinal 
cord injury. At the age of 16, Laura was in a car accident that 
caused her neck to break. Doctors said she would never walk 
again. This young woman was undaunted by the prognosis, and has 
since exhibited amazing courage to prove these doctors wrong.
    Laura is here today to put a face with the often abstract 
debates in which we policymakers often engage. She is an 
example of the miraculous strides that can be made in 
overcoming severe spinal cord injuries, if only we concentrated 
more resources to such goals, despite our differences on 
approach.
    Again, it's with great honor, as her representative and a 
friend of the Dominguez family, to introduce to this Committee, 
Laura Dominguez. And thank you very much.
    Senator Brownback. Thank you, Congressman, and that is a 
beautiful image--a dance, and to see that happen again.
    We have a short video showing some of the start and a point 
where we are now for both Laura Dominguez and Susan Fajt. And 
if you'd turn that video on, please.
    Ladies, if either of you want to describe the status of 
where you are in these, please speak up.
    Ms. Fajt. That would be myself, Susan Fajt. I am swimming 
in Texas at Joy Braun's house, one of my dear friends. And I'm 
also speaking, at the present time, about my quest for the 
cure.
    Senator Brownback. I don't know if we have sound with that 
video.
    [Video presentation.]
    Senator Brownback. So this is a PBS special. That is one 
big smile, Laura. Wait til you see her dance.
    Laura Dominguez, you're welcome to testify, and I'm 
pleased--we are honored to have you here.

                  STATEMENT OF LAURA DOMINGUEZ

    Ms. Dominguez. All right, thanks.
    Okay, so 3 years ago, while on my way home from summer 
school, my brother and I were involved in a car accident that 
left me paralyzed from the neck down. The accident was caused 
by an oil spill on the highway, an oil spill that we had 
nothing to do with, but, by chance, was on the road in our 
lane. I suffered a C6 vertebrae burst, and my spinal cord was 
severely damaged. In addition to the C6 burst, I also had a C1 
and C4 fracture. So I came close to being gone.
    Anyways, at the time, the doctors gave me absolutely no 
chance of ever walking again. I refused to accept their 
prognosis, and began searching for other options.
    After being hospitalized in several hospitals for almost 1 
year, my mother and I relocated to San Diego, California, so 
that I could undergo extensive physical therapy. While in 
California, we met a family whose daughter was also suffering 
from a similar spinal cord injury. They were also looking for 
other alternatives to deal with spinal cord injuries.
    After extensive research and consultations with medical 
experts in the medical field of spinal cord injuries, we also--
we all decided the best procedure that exists today was being 
performed in Portugal. We teamed up with the Nader family, 
which was the family from San Diego, and also a group of 
doctors from the Detroit Medical Center, and flew to Portugal 
to undergo this new surgical procedure.
    The surgery involved the removal of tissue from my 
olfactory mucosa, and transplanting it into my spinal cord at 
the injury site. Those procedures--the harvesting of the tissue 
and the transplant--were done at the same time. I was the tenth 
person in the world, and the second American, to have this 
procedure done.
    After the surgery, I returned to California to continue 
physical therapy. I stayed there until July 2003, and then 
returned back home. At that time, an MRI was taken, and it 
revealed that my spinal cord had begun to heal. Approximately 
70 percent of the lesion now looked like normal spinal cord 
tissue. I was also starting to regain feeling in my upper body, 
and, within 6 months, I had regained feeling down to my 
abdomen.
    Improvements in my sensory have continued until the present 
time. I can now feel down to my hip level, and have started to 
regain feeling and some movement down to my legs. My upper body 
has gained more strength and balance.
    Another one of the most evident improvements has been my 
ability to stand, and remain standing, using a walker, with 
minimal assistance. When I stand, I can contract my quadriceps 
and hamstring muscles. I can also stand on my toes when I am on 
my feet. And, more importantly--oh, when laying down in a prone 
position, I am able to move my feet.
    My training has continued to this day, and I am able to 
better use the muscles in my hips. I am able, with assistance, 
to walk, with braces, a distance of 114 feet. It takes 
approximately 30 minutes to walk this distance, and it is 
extremely tiring, but it can be done.
    I will continue to challenge myself until I can fully walk 
again with little or no assistance from braces or the help of a 
physical therapist. I know this will be possible by my 21st 
birthday.
    It is my understanding that the nervous system is one of 
the most difficult and complex to repair after an injury or 
trauma, but, in my case, the procedure that was performed in 
Portugal is working, as I have regained more feeling and 
movement. Some of the movements that I am able to do is a 
function that is controlled by the very tip of the spinal cord. 
Although the intensive physical training that I have has 
enhanced my ability to regain strength and movement, I did not 
have the type of function and feeling I have now prior to the 
surgery.
    It only stands to reason that if stem cells can repair the 
complex functions of the spinal cord, they can be used to 
repair other injured internal organs or other body parts, 
whether an injury is caused by trauma or disease. The way I see 
it, scientists have been given the knowledge and tools to 
develop and make use of adult stem cells. This knowledge should 
be taken full advantage of to help people overcome injuries or 
terminal illness. At the very least, people can benefit from 
the possibility of a better quality of life.
    My life changed from one minute to the next. A catastrophic 
injury can happen to any person under any circumstance, whether 
it be a car accident or some other innocent event or 
occurrence. The U.S. has been the world leader in science and 
health, and its citizens should not be forced to go to other 
countries to look for help or cures. The tools to help 
Americans should be made available in this country.
    Thanks.
    [The prepared statement of Ms. Dominguez follows:]

                 Prepared Statement of Laura Dominguez
    My name is Laura Dominguez. I am 19 years old and live in San 
Antonio, TX. Three years ago, while on the way home from summer school, 
my brother and I were involved in a car accident that left me paralyzed 
from the neck down. The accident was caused by an oil spill on the 
highway. An oil spill that we had nothing to do with, but by chance was 
on the roadway in our lane. I suffered a C6 vertebrae burst fracture 
and my spinal cord was severely damaged. At that time doctors gave me 
absolutely no chance of ever walking again. I refused to accept their 
prognosis and began searching for other options.
    After being hospitalized (in several hospitals) for almost a year, 
my mother and I relocated to San Diego, CA so that I could undergo 
extensive physical therapy. While in California, we met a family whose 
daughter was suffering from a similar spinal cord injury. They were 
also looking for other alternatives to deal with spinal cord injuries. 
After extensive research and consultations with medical experts in the 
field of spinal cord injuries, we decided the best procedure, that 
exists today, was being performed in Portugal. We teamed up with the 
Nader family, a group of Doctors from the Detroit Medical Center, and 
flew to Portugal to undergo this new surgical procedure.
    The surgery involved the removal of tissue from my olfactory sinus 
area and transplanting it into my spinal cord at the injury site. Both 
procedures, the harvesting of the tissue and the transplant were done 
at the same time. I was the tenth person in the world and the second 
American to have this procedure done.
    After the surgery, I returned to California to continue physical 
therapy. I stayed there until July of 2003 and then returned back to 
San Antonio, TX. At that time an MRI was taken and it revealed my 
spinal cord had begun to heal. Approximately 70 percent of the lesion 
now looked like normal spinal cord tissue.
    I was also starting to regain feeling in my upper body and within 
six months I had regained feeling down to my abdomen. Improvements in 
my sensory feelings have continued until the present time. I can now 
feel down to my hip level and have started to regain feeling and some 
movement down to my legs. My upper body has gained more strength and 
balance. Another one of the most evident improvements has been my 
ability to stand and remain standing, using a walker, and with minimal 
assistance. When I stand I can contract my quadriceps and hamstring 
muscles. I can also stand on my toes when I am on my feet. And more 
importantly, while lying down in a prone position, I am able to move my 
feet.
    My training has continued to this day and I am able to better use 
the muscles in my hip area. I am able, with assistance and the use of 
braces, to walk a distance of over 1,400 feet. It takes approximately 
thirty minutes to walk this distance and it is extremely tiring, but it 
can be done. I will continue to challenge myself until I can fully walk 
again with little or no assistance from braces or the help of a 
therapist. I hope . . . no, I know . . . this will be possible by my 
21st birthday.
    It is my understanding that the nervous system is one of the most 
difficult and complex to repair after an injury or trauma. But in my 
case, the procedure that was performed in Portugal is working as I have 
regained more feeling and movement. Some of the movements that I am 
able to make are functions that are controlled by the very tip of my 
spinal cord. Although the intensive physical training that I had 
enhanced my ability to regain strength and movement, prior to surgery I 
did not have the type of function and feeling that I have now.
    It only stands to reason that if adult stem cells can repair the 
complex functions of the spinal cord, they can repair and help other 
injured internal organs or other parts of the body, whether an injury 
is caused by trauma or disease. The way I see it, scientists have been 
given the knowledge and tools to develop and make use of adult stem 
cells, whether they are derived from tissue removed from the olfactory 
mucosa or otherwise. This knowledge should be taken full advantage of 
to help people overcome injuries that can be helped by stem cells or 
people that suffer from some terminal or debilitating diseases. At the 
very least, some people can benefit from the possibility of a better 
quality of life.
    My life changed from one minute to the next. A catastrophic injury 
can happen to any person under any circumstance, whether it be a car 
accident such as mine or some other innocent event or occurrence. The 
U.S. has been the world leader in science and health and its citizens 
should not be forced to go to other countries to look for help or 
cures. The tools to help Americans should be made available in this 
country.

    Senator Brownback. Thank you. Wow, that's beautiful.
    Susan Fajt, which--I love that last name.
    Ms. Fajt. Thank you, Senator.
    Senator Brownback. Let's hear your testimony.

 STATEMENT OF SUSAN R. FAJT, SPINAL CORD INJURED RECIPIENT OF 
                OLFACTORY MUCOSA TRANSPLANTATION

    Ms. Fajt. Please bear with me, mine is much longer than 
Laura's.
    OK. Hello, my name is Susan Fajt, and I would like to thank 
Chairman Brownback and Members of this Committee for this 
opportunity to tell you of adult stem cell treatment I received 
for spinal cord injury in Portugal, by Carlos Lima, and its 
results to date. But, first, allow me to share with you some 
basic facts about spinal cord injury to explain why I chose Dr. 
Lima's procedure.
    On November 17, 2001, I suffered a spinal cord and became 
paralyzed in an auto accident. My life is changed in ways 
unfathomable. Emotions run strong, and decisions must be made 
to end needless suffering. I chose to live and fight for a 
cure. Perhaps paralysis has robbed me of my freedom, but it can 
never take away my belief that a cure is attainable through 
research. There are currently no effective treatments available 
for spinal cord injury in the United States.
    When I was injured, I was 24 years of age, and I loved life 
more than you can imagine. Today, I have been given a great 
honor to tell you the story of my quest for a cure for this 
catastrophic condition.
    Once realizing that my injury was no longer a nightmare, 
but a devastating reality, I set out to find the best possible 
treatment in hopes I would be cured and recover everything in 
which I had lost. After tears of pain and years of searching, I 
found, through my own research, Dr. Carlos Lima in Portugal. My 
treatment with Dr. Lima took place on June 17, 2003. I was the 
eleventh patient in the world, and the third in the United 
States, to receive this treatment.
    Dr. Lima used adult stem cell treatment that uses an 
olfactory mucosa graft to promote axons to bridge the site of 
contusion, in hopes that my functional recovery would help me 
to once again walk, run, dance, and do everything I love, not 
to mention normal daily activities which are so easily taken 
for granted, such as bowel and bladder control.
    Only part of my dreams have been attained, but I've come 
farther than any of my American doctors have ever thought. My 
most recent MRI took place 5 days ago. The doctors were in 
disbelief at the improvement they saw where my spinal cord had 
been injured.
    I have recovered some functional improvement through Dr. 
Lima's procedure, such as the ability to hold my bladder and, 
at times, even void on my own. Sensation has been restored, 
though it is not completely normal. When concentrating, I am 
now able to contract my thighs; once again, this was also 
impossible before my surgery in Portugal. But, most important 
on my way to recovery, is that I can now walk with the aid of 
braces. I am now preparing to shed the shell of this 
wheelchair, which has confined me for over 2 years, to more 
often use my braces and walker for mobility purposes. This is 
something my doctors here in American told me would never be 
possible with my level of injury, and to accept my fate. With 
Dr. Lima's adult stem cell-based therapy, I have accomplished 
much more than the U.S. doctors said was possible, but this is 
only the first step to a complete cure.
    The next step is to find a combinational treatment, as well 
as an excellent rehabilitation program that will complement the 
results of Dr. Lima's surgery so that a complete recovery can 
be obtained from a spinal cord injury. I have literally gone 
all over the world in a quest for a program that will allow me 
to benefit as much as possible. Unfortunately, no program 
exists as of to date.
    Through love and faith, my father and I have taken upon an 
endeavor of creating new devices that assist me in working out 
2 to 3 hours each day to reach my maximum potential. In the 
near future, I hope to open a rehabilitation program so that 
others can benefit from our innovative equipment.
    Spinal cord injury is one of the cruelest injuries to 
affect the human condition. It causes extreme neurological pain 
and excruciating psychological trauma, amongst other things. 
Fortunately, I am not built to accept failure, so I plead with 
you to hear my cry for funding and other support for therapies, 
such as the one I received, that will free me and millions of 
others who also suffer in this primitive wheelchair.
    A cure for spinal cord injury will not be an easy task; 
however, when there is a will, there is a way. In addition to 
increasing funding to record levels, increasing public 
awareness about spinal cord injury and about treatments such as 
Dr. Lima's which are showing real results is imperative and 
desperately needed.
    The U.S. taxpayers pay over 30 million per day on care for 
spinal cord injury, and only 68 million per year in a search 
for a cure. Common sense tells me that by taking away 2 days of 
our care and, in its place, using this money for a cure, time 
will inevitably be on our side.
    Medical research in the U.S. is more advanced and far 
superior to any other country in this world, yet citizens such 
as myself risk their lives and are forced to seek treatment 
from foreign countries.
    Researchers need to be held accountable by the U.S. 
Government to design and implement research that results in 
human clinical trials. No more research for the sake of 
research. Furthermore, funding needs to be invested in 
staggering amounts for rehabilitation programs, as we have 
nothing of substance to help us recover after sustaining a 
spinal cord injury.
    I ask you for just one moment to imagine if I were your 
daughter, wife, or loved one. Would you help me in my quest, 
and take the opportunity you have before you to promote and 
publicize this research, which has already helped me, so that 1 
day I may dance the dance of life again, or would you allow me 
to suffer needlessly? The matter of funding medical research is 
of great importance, and I plead with you to do what your heart 
tells you. Please redirect the research in this country so that 
no--more resources and public awareness are given to treatments 
such as the one I received in Portugal. Free us from paralysis, 
and, in return, at the end of your life, you will know you have 
left this world a better place than what you have found it.
    In closing, I will echo the words that the Honorable 
President Ronald Reagan spoke to Gorbachev, ``If you seek 
peace, tear down these walls.'' Members of the Committee, if 
you seek cures for the millions of Americans currently 
suffering from spinal cord injuries and diseases, tear down 
these walls and free us from our wheelchairs.
    Thank you, and godspeed.
    [The prepared statement of Ms. Fajt follows:]

 Prepared Statement of Susan R. Fajt, Spinal Cord Injured Recipient of 
                    Olfactory Mucosa Transplantation
    Thank you Chairman Brownback and members of Committee, for this 
opportunity to tell you of the treatment I received for spinal cord 
injury in Portugal and its results to date. But first, allow me to 
share with you some basic facts about spinal cord injury to explain why 
I chose Dr. Lima's procedure.
    On November 17, 2001, I suffered a spinal cord injury and became 
paralyzed in an auto accident. My life has changed in ways 
unfathomable. Emotions run strong and decisions must be made to end 
needless suffering. I chose to live and fight for a cure. Perhaps 
paralysis has robbed me of my freedom, but, it can never take away my 
belief that a cure is attainable through research. There are currently 
no treatments available for spinal cord injury in the U.S.
    When I was injured I was twenty four years old, I loved life more 
than you can imagine! Today, I have been given a great honor to tell 
you the story of my quest for a cure for this catastrophic condition. 
Once realizing that my injury was no longer a nightmare but devastating 
reality, I set out to find the best possible treatment in hopes I would 
be cured, and recover everything in which I had lost.
    After tears of pain and years of searching, I found Dr. Carlos Lima 
in Portugal. He used a Olfactory Muscosa graft to promote axons to 
bridge the site of contusion in my hopes that functional recovery would 
help me to once again walk, run, dance, and do everything I would love 
not to mention--normal daily activities which are so easily taken for 
granted, such as, bowel and bladder control.
    Sadly, only part of my dreams has been attained. I have recovered 
some functional improvement through Dr. Lima's procedure such as, the 
ability to hold my bladder and at times even void on my own. Sensation 
has been restored, though it is not completely normal. When 
concentrating I am now able to contract my thighs, once again this was 
also impossible before my surgery in Portugal. I can now walk with the 
aid of braces, which my doctors here in America told me would never be 
possible with my level of injury and to accept my fate. With Dr. Lima's 
surgery, I have accomplished much more than my U.S. doctors said was 
possible but this is only the first step to a complete cure. The next 
step is to find combination treatments as well as an excellent 
rehabilitation program that will complement the results of Dr. Lima's 
surgery so that a complete recovery can be obtained from a spinal cord 
injury. I have literally gone all over the world in the quest for a 
program that will allow me to benefit as much as possible. 
Unfortunately, no such program exists to date. Through love and faith, 
my father and I have taken upon an endeavor of creating new devices 
that assist me in working out to my maximum potential. In the near 
future, I hope to open a rehabilitation program so that others can 
benefit from our innovative equipment. Spinal cord injury is one of the 
cruelest injuries to affect the human condition, most occur to young 
people who are just beginning to embrace their lives as adults. It is 
an injury that destroys the human body, causes extreme neurological 
pain, and extreme psychological trauma.
    Fortunately, I am not built to accept failure, so I plead with you 
to hear my cry for funding for research that will me and millions of 
others who also suffer from this primitive wheelchair.
    A cure for spinal cord injury will not be an easy task. However, 
when there is a will there is a way! Increasing funding to record 
levels that is specifically directed at injuries and diseases is 
imperative and desperately needed.
    The U.S. taxpayer pays over 30 million dollars per day on care for 
spinal cord injury and only 68 million per year in a search for a cure. 
Common sense tells me that by taking away two days of our care and in 
its place use this money for a cure, time will inevitably be on our 
side.
    Medical research in the United States is more advanced and far more 
superior to any other country in the world. Yet citizens, such as 
myself, risk their lives and are forced to seek treatment in foreign 
countries because treatments are not available in the U.S. Researchers 
need to be held accountable by the U.S. government to design and 
implement research that results in human clinical trials. Furthermore, 
more research dollars need to be invested in clinical trials and 
rehabilitation. I ask you for just one moment to imagine if I where 
your daughter, wife or loved one. Would you help me with my quest and 
take the opportunity you have to fund research so that I may one day 
dance the dance of life again, or would you allow me to suffer 
needlessly?
    The matter of funding medical research is before you and I plead 
with you to do what your heart tells you. Please re-direct the research 
in this country so more money is directed at curing injuries and 
diseases instead of the majority of the money going to basic research 
that is centuries away from applied applications. In this way, people 
do not have to go to other countries for treatments. Free us so that at 
the end of your life you will know you have left this world a better 
place than what you have found it.
    In closing, I will echo the words that the Honorable President 
Ronald Regan spoke to Gorbachev, ``if you seek peace. . .tear down this 
wall!'' Members of the Committee, if you seek cures for the millions of 
Americans currently suffering from spinal cord injuries and diseases, 
tear down these walls and free us from our wheelchairs!
    Thank you.

    Senator Brownback. Thank you, Susan.
    With the indulgence of Senator Wyden, could--are either of 
you willing to demonstrate and physically say, ``Here's where I 
was, and here's what I can do now,'' and show us? Or, I don't 
want to put you on the spot and in a nervous position, if 
you're not--but if you are, we would obviously appreciate that.
    Ms. Fajt. Well, last time I came, I was prepared to walk, 
and I brought my braces, and I did do that in front of one of 
the other Senators. And this time, being that I decided to 
focus more on the mental, instead of the physical. Otherwise, I 
would have brought my braces, and would have proudly walked 
across this floor in front of you.
    Senator Brownback. Laura?
    Ms. Dominguez. I just prefer to show what's on the video.
    Senator Brownback. All right, that's fine. And we'll get to 
some questions----
    Ms. Fajt. And I do have a video of me walking in France, 
just a month ago, and I believe you've seen it. And it was--I 
asked for it to be burned onto a DVD so that others could see 
it.
    Senator Brownback. We'll circulate that.
    Ms. Fajt. So--but, obviously, I can do much better now than 
I could then.
    Senator Brownback. Very good.
    Mr. Turner, Dennis Turner, was a Parkinson's patient. We 
heard from his doctor, Dr. Levesque, in the first panel. Mr. 
Turner, we're pleased to have you here at the hearing, and we'd 
look forward to your testimony.

                  STATEMENT OF DENNIS TURNER, 
                    SAN CLEMENTE, CALIFORNIA

    Mr. Turner. Thank you, Senator.
    Thank you, Chairman Brownback, for your interest in 
Parkinson's disease, in my treatment by Dr. Levesque, and my 
hopes and concerns for the future.
    For 14 years, I've had Parkinson's disease. This 
irreversible disease involves the slow destruction of 
specialized cells in the brain, called dopamine neurons. By 
early 1991, I suffered extreme shaking on the right side of my 
body, stiffness in my gait and movements. After some years of 
medication, I developed fluctuation and poor response to 
Sinemet. This made daily activities needing the coordinated use 
of both my hands hard or impossible, such as putting on my 
contact lenses. My disability prevented me from using my right 
arm.
    Other than my Parkinson's symptoms, I was physically very 
active and fit. Because of this, Dr. Levesque felt that I'd be 
a good candidate for experimental treatment. He explained that 
he would take a very small tissue sample from my brain, 
removing its adult neural stem cells. He would then multiply 
and mature these cells into dopamine neurons, and then inject 
these cells back into the left side of my brain, which controls 
the right side of my body. He proposed treating only the left 
side because it controls the right side of my body, the side 
with the most severe Parkinson's symptoms.
    Dr. Levesque did not tell me that this treatment would 
permanently cure my condition. Science has yet to learn what 
causes Parkinson's disease, much less how to remove it. 
However, since this cell-replacement approach had never been 
tried in a human patient, we hoped for the best. And since my 
only other realistic alternative was to continue growing worse 
til I eventually died, I decided to have the surgical procedure 
in 1999, one to remove the tissue, and another to inject the 
cells. I was awake for both procedures, under local anesthesia.
    Soon after having the cells injected, my Parkinson's 
symptoms began to dramatically improve. My trembling grew less 
and less, until, to all appearances, it was gone, only slightly 
reappearing if I became upset or nervous.
    [Laughter.]
    Mr. Turner. Which I am. I don't have as nice a name as you.
    [Laughter.]
    Mr. Turner. Dr. Levesque had me tested by a neurologist, 
who said that he wouldn't have known that I had Parkinson's if 
he met me on the street. I was once again able to use my right 
hand and arm, and enjoy normal activities that I had given up 
hope of ever doing.
    Since being diagnosed with Parkinson's disease, my 
condition has slowly, but continuously worsened. I can't say 
with certainty what my condition would have become if Dr. 
Levesque had not used my own adult stem cells to treat me, but 
I have no doubt that, because of this treatment, I have enjoyed 
5 years of quality of life that I feared had passed me by.
    Last year, after 4 years of being virtually symptom free, 
my Parkinson's symptoms began reappearing in my body's left 
side. Today, I have various degrees of trembling in both hands, 
although I feel that the left is slightly worse. Nevertheless, 
I would not hesitate for a second to have Dr. Levesque use my 
stem cells to treat me for a second time, since, in my case, 
they were safe, effective, and involved no risk of rejection.
    Because of my improvements through Dr. Levesque's 
treatment, I've been able to indulge in my passion of big-game 
photography these past 5 years. While on safari in 2001 in 
Africa, I scrambled up a tree to avoid being run over by a 
black rhinoceros. And you've got to be fast for those babies.
    [Laughter.]
    Mr. Turner. I swam in the South Atlantic--that's off South 
Africa--with great white sharks. This was not cage diving; this 
was snorkeling with them. Two weeks ago, I returned from Africa 
after photographing cheetahs and leopards in the wild. Here are 
a few examples of the pictures I took--which I forgot to bring 
them, but they're nice pictures.
    [Laughter.]
    Mr. Turner. Pictures I took. They represent memories and 
experiences I feel I have Dr. Levesque to thank for.
    I came here to offer him my sincere gratitude, and to offer 
others with Parkinson's disease a concrete hope for--reason for 
hope.
    This summarizes my history with Parkinson's, and the 
positive effects I experienced through a treatment that used my 
adult stem cells. I am very happy with the results, and would 
dearly love to have a second treatment. And, Mike, you can do 
it tomorrow. I'll be there.
    Thank you, sir.
    [The prepared statement of Mr. Turner follows:]

                  Prepared Statement of Dennis Turner
    Thank you, Chairman Brownback, for your interest in Parkinson's 
Disease, in my treatment by Dr. Levesque, and in my hopes and concerns 
for the future.
    For fourteen years I've had Parkinson's Disease. This irreversible 
disease involves the slow destruction of specialized cells in the 
brain, called Dopamine Neurons. By early 1991 I suffered extreme 
shaking of the right side of my body, stiffness in my gait and 
movements. After some years of medication, I developed fluctuation and 
poor response to Sinemet. This made daily activities needing the 
coordinated use of both hands hard or impossible, such as putting in 
contact lenses. My disability prevented me from using my right arm.
    Other than my Parkinson's symptoms I was physically very active and 
fit. Because of this Dr. Levesque felt that I'd be a good candidate for 
an experimental treatment. He explained that he would take a very small 
tissue sample from my brain, removing its adult neural stem cells. He 
would then multiply and mature these cells into Dopamine Neurons, then 
inject these cells back into the left side of my brain. He proposed 
treating only the left side because it controls the right side of the 
body, the side with the most severe Parkinson's symptoms.
    Dr. Levesque did not tell me that this treatment would permanently 
cure my condition. Science has yet to learn what causes Parkinson's 
Disease, much less how to remove it. However, since this cell-
replacement approach had never been tried in a human patient we hoped 
for the best. And since my only other realistic alternative was to 
continue growing worse until I eventually died, I decided to have the 
surgical procedures in 1999, one to remove the tissue and another to 
inject the cells. I was awake for both procedures, under local 
anesthesia.
    Soon after having the cells injected my Parkinson's symptoms began 
to improve. My trembling grew less and less, until to all appearances 
it was gone, only slightly reappearing if I became upset. Dr. Levesque 
had me tested by a Neurologist, who said he wouldn't have known I had 
Parkinson's if he had met me on the street. I was once again able to 
use my right hand and arm normally, enjoying activities that I had 
given up hope of ever doing.
    Since being diagnosed with Parkinson's Disease my condition had 
slowly, but continuously worsened. I can't say with certainty what my 
condition would have become if Dr. Levesque had not used my own adult 
stem cells to treat me. But I have no doubt that because of this 
treatment I've enjoyed 5 years of quality life that I feared had passed 
me by.
    Last year, after 4 years of being virtually symptom free, my 
Parkinson's symptoms began reappearing in my body's left side. Today I 
have various degrees of trembling in both hands, although I feel that 
the left is slightly worse. Nevertheless, I wouldn't hesitate for a 
second to have Dr. Levesque use my adult stem cells to treat me a 
second time, since in my case they were safe, effective, and involved 
no risk of rejection.
    Because of my improvements through Dr. Levesque's treatment I've 
been able to indulge in my passion for big game photography these past 
5 years. While on safari in 2001 I scrambled up a tree to avoid being 
run over by a Rhino. I swam in the South Atlantic with Great White 
Sharks. Two weeks ago I returned from Africa after photographing 
Cheetahs and Leopards in the wild. Here are a few examples of the 
pictures I took. They represent memories and experiences I feel I have 
Dr. Levesque to thank for. I came here to offer him my sincere 
gratitude, and to offer others with Parkinson's a concrete reason for 
hope.
    This summarizes my history with Parkinson's and the positive 
effects I experienced through a treatment that used my own adult stem 
cells. I'm very happy with its results and would dearly love to have a 
second treatment.

    Senator Brownback. Thank you, Mr. Turner.
    Dr. Goldstein is Chief Science Officer for the Juvenile 
Diabetes Research Foundation. This is a patient panel, but had 
requested to testify on this panel, so we wanted to accommodate 
you, Dr. Goldstein, on this panel.

        STATEMENT OF ROBERT GOLDSTEIN, CHIEF SCIENTIFIC 
        OFFICER, JUVENILE DIABETES RESEARCH FOUNDATION 
                      INTERNATIONAL (JDRF)

    Dr. Goldstein. Chairman Brownback and Members of the 
Subcommittee, thank you very much for the opportunity to appear 
before you today. I'm Robert Goldstein, Chief Scientific 
Officer.
    I am joined today by the Langbein family. I'd like to ask 
them to stand, please. Jamie was diagnosed with juvenile 
diabetes at the age of one. She worries about being different 
from her friends in school, and parents worry about the long-
term complications of diabetes and their daughter's future, and 
whether their other children will be diagnosed with the 
disease. Jamie represents just one of the nearly two million 
people who battle juvenile diabetes each and every day.
    Thank you so much for being here today.
    Senator Brownback. Yes, thank you for joining us.
    Dr. Goldstein. JDRF is the leading charitable funder of 
juvenile diabetes research worldwide. Established over 30 years 
ago by parents of children with juvenile diabetes, our mission 
is to find a cure.
    Over the years, JDRF has provided some $800 million in 
grants for diabetes research. To fund that science, JDRF 
volunteers do their part every day to raise money in their 
communities, and we are proud of the strong partnership that we 
have developed with the Federal Government.
    JDRF aggressively pursues all avenues of promising 
research. In Fiscal Year 2004, our commitments in the area of 
stem cell research totaled $8.2 million. Of this amount, $6.3 
million is spent in the area of embryonic stem cell research, 
and less than $2 million on other areas, including adult stem 
cells. JDRF will continue to support both adult and embryonic 
stem cell research.
    We appreciate that no one can predict what area will 
produce new therapies or a cure, and JDRF views adult and 
embryonic stem cell research as complementary pathways to our 
goal. Let me explain why, using pancreatic islet cell 
transplantation as an example.
    In islet transplantation, the beta or insulin-producing 
cells are isolated from a cadaveric pancreas, and then infused 
into a person with juvenile diabetes. Once transplanted, these 
new islets begin to produce and release insulin into the 
patient's body. Since the year 2000, nearly 300 people have 
received islet transplants, and the majority of them lead 
significantly better and healthier lives.
    These results are very exciting, but there are significant 
hurdles in moving this from an experimental procedure to a 
standard therapy. One such hurdle is the severe shortage of 
donated pancreases. In 2001, approximately 400 were available 
for islet transplantation and research, compared to the almost 
two million Americans with juvenile diabetes.
    Here, then, is one reason why we are so excited about 
recent advances in embryonic stem cell research. Recent studies 
have demonstrated the ability to coax embryonic stem cells into 
insulin-producing cells in the lab. We have good reason to 
believe that embryonic stem cells will 1 day be able to grow 
large amounts of insulin-producing beta cells for transplant, 
but more work needs to be done.
    Unfortunately, adult stem cells have not shown the same 
promise for diabetes. In a recent report, Harvard University 
researcher Doug Melton published a paper in Nature pointing out 
that, in mice, new beta cells in the pancreas are formed 
through the replication of existing beta cells, rather than 
through the differentiation of adult stem cells. This finding 
has important implications, especially if confirmed in humans.
    In Type 1 diabetes, the autoimmune response destroys the 
beta cells. This means that in order to cure Type 1 diabetes, 
we'll have to rely on an external source of beta cells. 
Embryonic stem cells may well prove to be the main source for 
generating beta cells.
    JDRF funds research to develop beta cells from adult stem 
cells, or to regenerate beta cells from existing precursor 
cells. Researchers have reported that human adult duct tissue 
might have the potential to develop into beta cells. Other 
groups have results that indicate that transplanted bone marrow 
cells may be able to show insulin production. Some have used 
these findings to argue that adult stem cells may be the answer 
for curing juvenile diabetes. JDRF takes the position that 
research using both embryonic and adult stem cells, perhaps in 
side-by-side comparisons, will get us to our goal fastest.
    Mr. Chairman, JDRF recognizes that the science of producing 
insulin-secreting cells from either adult or embryonic stem 
cells is at an early stage. Given this reality, how can we 
adequately compare the effectiveness of adult and embryonic 
stem cell research unless both avenues are pursued 
simultaneously and with equal rigor? While we have made great 
strides toward our goal of a cure, more needs to be done, and 
we don't have time to wait. In the battle against diabetes, we 
are in a race against time.
    To put the urgency of finding a cure into perspective, I'd 
like to share some words from Mary Tyler Moore, JDRF's 
international chairman, that she shared with Members of the 
House. Mary states, ``In the nearly 6 years since human 
embryonic stem cells were first successfully cultured in a lab, 
diabetes has contributed to the deaths of as many as three 
million people and cost our Nation over $750 billion. It has 
caused nearly 500,000 amputations, rendered over 100,000 people 
blind, forced a quarter of a million people to require kidney 
transplants or dialysis. And 120,000 moms have been told that 
their child has Type 1 diabetes, a disease which, during that 
time period, would require each of these children to have 8,700 
injections of insulin and 17,500 pricks of their fingers to 
check blood sugar levels--just for that child to survive.''
    Thank you, again, for the opportunity to appear before you 
today. I'm happy to answer any questions.
    [The prepared statement of Dr. Goldstein follows:]

   Prepared Statement of Robert Goldstein, Chief Scientific Officer, 
       Juvenile Diabetes Research Foundation International (JDRF)
    Chairman Brownback and members of this Subcommittee, thank you for 
the opportunity to appear before you today to participate in this 
important hearing on adult stem cell research. I am Robert Goldstein, 
Chief Scientific Officer of the Juvenile Diabetes Research Foundation 
(JDRF). I am joined today by the Langbein family who represent the 
millions of families who struggle with the daily challenges and fears 
of caring for a loved one with juvenile diabetes. Jamie was diagnosed 
at the age of one, and she has been on an insulin pump since the age of 
four. Jamie's diabetes affects her life every day, all day. Her parents 
must test her blood sugar eight times a day, and every time she eats, 
exercises, or goes to a birthday party, Jamie must account for what she 
eats or how much exercise she does and adjust her dose of insulin 
accordingly so she doesn't end up in the hospital or in a coma. Her mom 
gave up her career as an attorney so that she could always be nearby if 
Jamie had problems with her pump or blood sugar while at school, and 
her parents get up frequently during the night to check her blood sugar 
level. Jamie worries about being different from her friends in school, 
and her parents worry about the long-term complications of diabetes and 
their daughter's future and whether their other children will be 
diagnosed with the disease. This is just one child of the nearly two 
million people who battle juvenile diabetes each and every day.
    JDRF is the leading charitable funder of juvenile diabetes research 
worldwide. Established more than 30 years ago by parents of children 
with juvenile diabetes, our mission is to find a cure for juvenile 
diabetes and its complications. Over the years, JDRF has provided some 
$800 million in grants for diabetes research at most of the world's 
leading universities, laboratories, and hospitals. To fund that 
science, JDRF volunteers do their part every day to raise money in our 
communities across the country--through walks, galas, and other 
events--and we are proud of the strong partnership for funding research 
that we have developed with the Federal government.
    JDRF, as the world's leading charitable funder of diabetes 
research, aggressively pursues all avenues of promising research and 
makes its funding decisions based upon vigorous scientific review 
based, in many ways, upon the NIH model. In the area of stem cell 
science, JDRF funds scientists exploring the opportunities created by 
both adult and embryonic stem cell research. In Fiscal Year 2004, JDRF 
commitments in the area of stem cell research total $8.2 million. Of 
this amount, $6.3 million is spent in the area of embryonic stem cell 
research and less than $2 million is spent on other areas of stem cell 
research, including adult stem cells. We focus on both areas--as well 
as dozens of other avenues of scientific investigation--because no one 
can predict what area of research will produce new therapies or a cure 
for juvenile diabetes.
    Adult stem cell research has been pursued for more than 35 years, 
and as you know, embryonic stem cells were just discovered in 1998. 
JDRF will continue to support both adult and embryonic stem cell 
research so that we can pursue a cure as strongly as possible. However, 
the research community believes that embryonic stem cells offer more 
promise in the area of diabetes. Let me explain why, using pancreatic 
islet cell transplantation as an example. Islet transplantation has 
been a spectacular breakthrough in diabetes research. In islet 
transplantation, the beta--or insulin-producing--cells are isolated 
from a cadaver pancreas and then infused into a person with juvenile 
diabetes through a catheter inserted into the portal vein of their 
liver. Once transplanted, these new islets recognize blood sugar levels 
and begin to produce and release insulin into the patient's body. Islet 
transplantation had been attempted since the 1970s with limited 
success. However, in the year 2000, researchers made a breakthrough in 
the procedure, and since that time nearly 300 people have received 
islet transplants and the majority of them lead significantly better 
and healthier lives. In most of these individuals, therapeutic control 
of their diabetes has improved remarkably, and in many instances they 
do not even have to take insulin injections. Furthermore, many of the 
patients have reported a reversal in some of their complications, 
especially hypoglycemia unawareness but also improvement in vision and 
less pain from neuropathy.
    These results are very exciting, but there are significant hurdles 
in moving this from an experimental procedure to a standard therapy 
that could benefit the millions of people with diabetes--many of them 
children. One such hurdle is the severe shortage of donated pancreases. 
In 2001, approximately 400 pancreata were available for islet 
transplantation and research, compared to the almost two million 
Americans with juvenile diabetes.
    Here, then, is one reason why we are so excited about recent 
advances in embryonic stem cell research. Recent studies have 
demonstrated the ability to coax embryonic stem cells into insulin-
producing cells in the lab. We have good reason to believe that 
embryonic stem cells will one day be able to grow large amounts of 
insulin-producing beta cells for transplant, but more work needs to be 
done. Unfortunately, adult stem cells have not shown the same promise 
when it comes to diabetes. Last month, Harvard University researcher 
Douglas Melton published a paper in Nature pointing out that in mice, 
new beta cells in the pancreas are formed through the replication of 
existing beta cells rather than through the differentiation of adult 
stem cells. This finding indicates that adult stem cells in the 
pancreas do not contribute to beta cell formation, and that embryonic 
stem cells may prove to be the only stem cells that will be useful to 
generate beta cells for the treatment of Type 1 diabetes. Other studies 
indicate that mouse embryonic stem cells can be differentiated into 
insulin-producing cells, and several studies suggest that this can be 
done using human embryonic stem cells.
    JDRF funds research to develop beta cells from adult stem cells, or 
to regenerate beta cells from existing precursor cells. Researchers 
have reported that human adult duct tissue might have the potential to 
develop into beta cells. Other groups have results that indicate that 
transplanted bone marrow cells may be able to show insulin production. 
Some have used these findings to argue that adult stem cells may be the 
answer for curing juvenile diabetes. JDRF takes the position that 
research using both embryonic and adult stem cells, perhaps even in 
side-by-side comparisons, will get us to our goal fastest.
    Mr. Chairman, adult stem cells may one day prove to be the answer 
to alleviating the pain and suffering caused by certain diseases--I 
certainly hope that is the case. We have heard some remarkable stories 
from some of the witnesses today. But we have no idea of knowing which 
diseases those may be, and unfortunately we are not certain of the 
widespread application of these treatments. We do know that to date, 
adult stem cells have not been shown to hold as much promise for 
juvenile diabetes as embryonic stem cells. Given this reality, how can 
we turn our backs on other exciting research opportunities, such as 
embryonic stem cell research, thereby potentially delaying life-saving 
therapies and cures for millions of people? And how can we adequately 
compare the effectiveness of adult and embryonic stem cell research 
unless both avenues are pursued simultaneously and with equal rigor?
    We are in an extraordinary time of opportunity in the area of 
medical research, and this country is leading the way. Scientists 
around the world agree that stem cell research holds tremendous promise 
for hundreds of millions of people. I applaud you for continuing to 
monitor advances in the area of adult stem cell research, and I 
encourage you to do the same for embryonic stem cell research. For 
certain diseases such as juvenile diabetes, embryonic stem cells hold 
the most promise, and we can't afford to lose any more time.
    While we have made great strides towards our goal of a cure, more 
needs to be done, and we don't have time to wait. Insulin is not a cure 
for juvenile diabetes, nor does it prevent the onset of complications 
such as kidney failure, blindness, heart disease and amputations. 
Diabetic retinopathy is the leading cause of adult blindness in the 
United States; ninety percent of patients have evidence of retinopathy 
after fifteen years of diabetes with approximately 25,000 new cases of 
blindness per year. Diabetes is also the leading cause of renal failure 
in the United States, accounting for forty percent of new cases per 
year. Greater than half of all patients with diabetes develop 
neuropathy, making diabetic neuropathy the most common cause of non-
traumatic amputations and autonomic failure. In his or her lifetime, a 
diabetic patient with neuropathy has a fifteen percent chance to 
undergo one or more amputations. Mr. Chairman, in the battle against 
diabetes, we are in a race against time.
    Not a day goes by that JDRF doesn't receive calls or letters or e-
mail messages from mothers or fathers of children with type 1 diabetes 
asking ``When will my child be cured?'' On the one hand, it is 
extremely difficult to explain the pace of science, particularly to a 
mother whose five-year-old has to prick his finger six or seven times a 
day to test his blood sugar, who needs three or four injections of 
insulin every day, who is afraid to go to sleepovers or summer camp for 
fear of falling into a coma, and who is at constant risk of developing 
a host of complications that could cut short his life. But on the other 
hand, it is downright tragic to have to explain how the pace of science 
could be slowed even further by focusing on one area of research and 
excluding another.
    To put the urgency of finding a cure into perspective, I'd like to 
share some words from Mary Tyler Moore, JDRF's International Chairman, 
that she shared with Members of the House. Mary states that ``in the 
nearly six years since human embryonic stem cells were first 
successfully cultured in a lab, . . . diabetes has contributed to the 
deaths of as many as 3 million people and cost our Nation over $750 
billion. It has caused nearly 500,000 amputations, rendered over 
100,000 people blind, and forced a quarter million people to require 
kidney transplants or dialysis. And 120,000 moms have been told that 
their child has Type 1 diabetes--a disease which during that time 
period would require each of these children to have 8,700 injections of 
insulin and 17,500 pricks of their fingers to check blood sugar 
levels--just for that child to survive.''
    Thank you again for the opportunity to appear before you today. I 
am happy to answer any questions you may have.

    Senator Brownback. Thank you. And I want to thank the 
Juvenile Diabetes Foundation for all the funding and the 
advocacy work that they're doing. A number of families in my 
state, constituents and friends, have children with juvenile 
diabetes, and I appreciate the work that you're doing.
    I've got some questions I want to ask. Let me start, if we 
could, with Mr. Turner, if you don't mind. Please describe 
where you were before your treatment, the adult stem cell 
treatment. Just describe to me, in your words, what you were 
functioning like.
    Mr. Turner. I had extreme shaking of the right hand. I had 
difficulty writing my name. I had difficulty drawing concentric 
circles. Dr. Levesque took films of me before and after the 
surgery.
    Senator Brownback. Could you do the safaris and the 
traveling such as you have been doing?
    Mr. Turner. They were getting more difficult to do. And the 
second safari that I took was in 1999, after the surgery was 
done.
    Senator Brownback. You've said you were having a lot of 
difficulty, and then you had the transplant of your own adult 
stem cells from--grown outside of your body and then put back 
in.
    Mr. Turner. Yes, sir.
    Senator Brownback. What happened--what were you like after, 
and at the best point----
    Mr. Turner. I was functioning normally.
    My walk was OK. My right and left hand were fine. Before, 
my right hand wouldn't swing when I walked. Then it started 
swinging after the surgery. I could put my contacts in. I could 
function normally. In fact, the neurologist that examined me 
said that everything--he wouldn't even have known I had it, if 
he had of not been told.
    Senator Brownback. And when did you get to that point in 
time after your surgery? How much time?
    Mr. Turner. About 6 months, Mike? Six months.
    Senator Brownback. And then--now, you've said that you've 
had some regression now that's taken place recently.
    Mr. Turner. Lately, about--oh, about 9 months ago, the 
symptoms started appearing in my left hand, my right hand 
started developing the symptoms again. And I just--it just 
regressed.
    Senator Brownback. And so you would like to be a candidate 
for a second round of this stem cell therapy.
    Mr. Turner. You bet your life. You bet my life, actually.
    [Laughter.]
    Mr. Turner. You could bet your life, too.
    Senator Brownback. We're betting a lot of lives, because we 
want to get cures for this taking place, and you're showing 
some of the greatest promise of anything I've seen or heard 
about.
    Mr. Turner. I mean, when I was running from the black rhino 
in Zimbabwe, and, you know, you've got to head for a tree, 
you've got to be awful fast, because those things can really 
move. They don't look like they're fast, but they are. And when 
you photograph them, you always look for a tree or a rock to 
get up on. And I don't think I'd be here today if it wasn't for 
Michel, because that rhino would have caught me. So you've got 
to dive, you've got to do a lot of things awful fast. My 
physical abilities got better. I could do a lot more. And, just 
lately, I went to Africa, about 2 months ago, and I could feel 
a difference in my abilities than what I had before. I hope 
it's just not all age.
    Senator Brownback. Laura--and if your dad wants to answer 
these, that's fine, if you don't want to. And, Mrs. Dominguez 
is here with us, as well. Tell me, in your words, your 
progression, where you are now, and where you see yourself 
going. Where were you after the spinal cord injury at its worst 
situation, just in your words?
    Ms. Dominguez. Well, right after my injury, I had regained, 
I guess some arm movement and some hand movement, and also some 
muscles in my abdomen. And I had no movement, like, in my lower 
body. So, I mean, as far as where I see myself, I mean, I'm 
going to get out of this chair.
    Senator Brownback. And you now have walked 114 feet, did 
you tell me that, in the testimony?
    Ms. Dominguez. Oh, yes, that was a mistake. It was actually 
1,400.
    Senator Brownback. Fourteen hundred feet?
    Ms. Dominguez. Yes.
    Senator Brownback. Over a period of 30 minutes.
    Ms. Dominguez. Yes.
    Senator Brownback. Is that correct?
    Ms. Dominguez. Yes. With the braces.
    Senator Brownback. With braces. Were other people assisting 
you, or were you----
    Ms. Dominguez. Yes.
    Senator Brownback. Is that--was that you doing most----
    Ms. Dominguez. I had some assistance. Like, my dad will 
help, because the braces are really heavy. So----
    Senator Brownback. And with physical therapy--on the 
physical therapy you've emphasized a great deal. So apparently 
the stem cells can reunite and start the process, but then 
you've got to retrain the body----
    Ms. Dominguez. Right.
    Senator Brownback.--is that the process, basically, you're 
in now, is retraining the body to move? And I see your muscles 
have atrophied, obviously, extensively from lack of use. You've 
got to build the strength back up in those, as well.
    Susan, how about yourself. Now, again, where were you when 
you hit the lowest point, and where are you now?
    Ms. Fajt. Well, the lowest point was obviously when I went 
flying through the roof of a house and got paralyzed.
    Senator Brownback. From where down?
    Ms. Fajt. From level T7, thoracic 7, down.
    Senator Brownback. OK, describe that.
    Ms. Fajt. The chest, right where you're at--chest level, 
down. Right here----
    Senator Brownback. OK.
    Ms. Fajt.--down. And, as of now, I have sensation all the 
way down to my toes, and I have my abdominals, I have my hip 
flexers, which help me to walk with my braces, which I can do 
unassisted with my braces and a walker, for approximately an 
eighth of a mile, or longer and without--nonstop.
    Senator Brownback. Nonstop----
    Ms. Fajt. Nonstop.
    Senator Brownback.--and nobody assisting you.
    Ms. Fajt. Nobody assisting me. And I have my thigh muscles, 
my calves, my toe--if I concentrate while right in my bathtub 
and just stretching and trying to get my toes to move, I can do 
that, as well. Sensation is a major factor here. That comes 
first. It also comes with bad. There's--you know, I can 
obviously feel pain in my lower back at times, and especially 
after I exercise for extensive periods of time.
    What was your other question?
    Senator Brownback. You've pretty well hit it. Where the 
two--where you were and where you are now. I understand where 
we're going. This is full-scale----
    Ms. Fajt. Yes, and----
    Senator Brownback.--full mobility----
    Ms. Fajt. Right.
    Senator Brownback.--no assistance----
    Ms. Fajt. Right.
    Senator Brownback.--where we're headed to, and I have no 
doubt you're going to make it.
    Ms. Fajt. Thank you.
    Senator Brownback. I want to go another round if we could 
afterwards, but Senator Wyden?
    Senator Wyden. Thank you, Mr. Chairman.
    And Laura and Susan and Dennis, it is just a thrill to see 
your tenacity. I mean, I think that you just, you know, summed 
it up, Laura. You just put it out bluntly, you're going to get 
out of the chair, and that's, of course, exactly the kind of 
inspiration that people are looking for. And I'd just say God 
bless to each of you.
    And I have only one comment. I mean, the three of you, you 
know, aren't politicians. I mean, you're people who just want 
cures. You want cures. You want your government to get serious 
about it. And I think, to me, Susan, it really comes down to 
the point you made at the end of your testimony where you talk 
about what legislators would do if it were our family.
    Ms. Fajt. Yes.
    Senator Wyden. I've got a 15-year-old daughter, Lily.
    Ms. Fajt. Right.
    Senator Wyden. I think the reasons that I'm here--and you 
haven't heard me say boo about Democrats and Republicans. 
That's not the way I approach this.
    Ms. Fajt. Right.
    Senator Wyden. I'm here because I think to give more people 
a chance at success, the kind of accomplishments that you're 
talking about, we've just got to take the shackles off our 
scientists.
    Ms. Fajt. And you can----
    Senator Wyden. We've got to----
    Ms. Fajt.--do that.
    Senator Wyden. You get we can. And that's what a lot of us 
are trying to do in the Congress. And, unfortunately, that's 
what's been seen by some as political. But, to me, it's about 
science, and it's about responding to your question. You've 
laid it out. The Congress can do it. It's a question of 
political will, it's a question of the right policies, and it's 
a question of the right funding. I want you to know I'm going 
to do everything I can, not as a Democrat or somebody in a 
political party, but because I think it's right, because I 
think that's the answer to the very appropriate question you 
gave.
    Dr. Goldstein, one question for you for this round. You 
heard the three patients. And I think, again, Susan, you put it 
very well, this whole spectacle of having to traipse all over 
the globe in order to get care, that's a disgrace. That's just 
wrong.
    Ms. Fajt. Yes.
    Senator Wyden. In a country as strong and rich as ours, 
that shouldn't happen. My question to you, Dr. Goldstein, is, 
Don't we run the risk, as a nation, of having more patients and 
families having to make those around-the-world journeys with 
these policies that restrict research and work in this area?
    Dr. Goldstein. The research is very actively going on 
outside the United States. We would prefer--JDRF would prefer 
that it geared up in the traditional fashion that NIH functions 
so that much more of the research could go on here. That would 
produce therapies that will be acceptable and regulated and 
useful within this country. And this is one of the few examples 
where outside the United States seems to be stimulated in more 
ways.
    Senator Wyden. Well, I think that--the reason I asked, I 
think--again, I don't want to approach this in a political 
fashion--is that I do think that the research restrictions will 
contribute to additional cases of the kind of problem that 
Susan described, and I want to see that changed.
    A question I also want to ask for you, Dr. Goldstein, I 
asked Dr. Weissman, that embryonic stem cells can be used in a 
variety of tissues, and he indicated to me that adult stem 
cells end up being more restrictive, in terms of the tissues in 
which it can be used. Do you essentially share that view, as 
well?
    Dr. Goldstein. Yes. People have been studying adult stem 
cells for more than 30 years. They've been trying to create 
insulin-secreting cells. That has not occurred. Embryonic stem 
cell research has already demonstrated that at a proof-of-
concept level. So we would argue that--let's pursue everything 
that's promising as quickly and as urgently as possible.
    Senator Wyden. Well, I think--and I'm going to have to go 
in a second--I think sums up how I approach it. Let's pursue 
everything, and with exactly the kind of urgency you're 
describing. And I think that's what the American people 
deserve, and that's certainly what the people in my state are 
saying. And I'd just say God bless to each of you.
    Thank you, Mr. Chairman.
    Senator Brownback. Thank you.
    If I could, Dr. Goldstein, I was just given a note that 
Diane Faustman, Harvard, used adult cells, recently, from the 
spleen in diabetic mice, and the researchers in their paper 
noted permanent reversal of the disease. They're attempting to 
get some funding for clinical trials. Are you familiar with 
this work that they're doing? And----
    Dr. Goldstein. Yes, sir.
    Senator Brownback.--what do you think of it?
    Dr. Goldstein. Oh, it's terrific. It's proof of something 
in animals that needs to be translated to people. We hope it 
works.
    Senator Brownback. Are you doing some of the funding on 
this work, too, or do you know--maybe you don't----
    Dr. Goldstein. I can't publicly respond.
    Senator Brownback. OK. It just seems to me that it's one of 
those promising areas. And what I'm trying to do is find areas 
that we can have successes that we don't have the controversy 
surrounding so that it's easy to move forward with.
    Dr. Goldstein. Well, let me respond without saying any 
investigator's name. We're funding research to prove, confirm, 
reduplicate, and study those findings because we think they're 
important, as we are funding an encouraging research, as I said 
to Senator Wyden, in every area that seems promising. We think 
that you don't know the answer to a research question until you 
do the work. And so we don't want to stop, or not do, work 
until we get some answers, and that why we're--you know, just 
to repeat it, we're ecumenical, we want to support all 
promising avenues of research.
    Senator Brownback. You noted the need for donated spleens, 
is that correct, that you were--or, excuse me, pancreas 
donations----
    Dr. Goldstein. Yes.
    Senator Brownback.--that some of that is occurring, but not 
near enough or as far as current knowledge and ability to get 
to the islet----
    Dr. Goldstein. Right.
    Senator Brownback.--cells. Is that something that we should 
be pressing more from here?
    Dr. Goldstein. Yes. Approximately 6,000-plus people die in 
the United States each year and donate organs. Now, many more 
people die and do not donate organs. But we're only able to 
acquire pancreases from maybe two thirds of that group.
    Senator Brownback. That donate organs.
    Dr. Goldstein. That donate organs.
    Senator Brownback. Whereas, if everybody----
    Dr. Goldstein. Well, if everybody--if everybody who died 
donated organs, there would be a lot of improvement for people 
to receive kidney transplants, heart transplants, lung 
transplants. There's a big, big need, very long waiting lists. 
So the organ donation issue has been around for a while, and 
the relative donation rate has been fairly flat, as opposed to 
increasing by 10 percent or 20 percent a year. So we'd love to 
have help to alert people to the benefits of donating more 
organs for transplant. That would help all kinds of people with 
all organs.
    Senator Brownback. And on juvenile diabetes, age is not an 
issue for--as far as the donated pancreas--because of the cells 
you're pulling out, or----
    Dr. Goldstein. Well, there are some limitations, in terms 
of--once you get past 65 or 70, if you're an organ donor, 
sometimes those organs are less able to provide good quality 
islets. But, in general, there's a wide range of potential 
donors that the islets could be prepared from.
    My point in the testimony was that, at our best, this week 
could maybe help 400 people with islet transplantations, and we 
have 30-40,000 people each year getting diagnosed with the 
disease, so that--that's a big disconnect, and we're probably 
not going to solve that problem by increasing organ donation, 
per se. That's why we need alternative sources for islets and 
beta cells, cells that secrete insulin.
    Senator Brownback. OK.
    Senator Wyden. Can I make sure I got that number straight? 
We can do three or four hundred now, and what's really needed, 
you said, are 30- or 40,000?
    Dr. Goldstein. Right.
    Dr. Goldstein. Newly diagnosed people with the disease, 
that number runs 30- or 40,000 a year. There are close to two 
million people with the disease today. The common feature is, 
they lack insulin-secreting cells. So if transplantation of 
insulin-secreting cells is to be a solution, 400 or 500 is not 
going to help that many people.
    Senator Wyden. I want to make sure I get the enormity of 
this, because I think what you're saying is just staggering. 
There are already two million individuals who are going to need 
this assistance, and every year we widen the gap dramatically. 
There are 300 people----
    Dr. Goldstein. That's correct.
    Senator Wyden.--for whom you can get assistance, and it's 
30- or 40,000 who need it on an annual basis.
    Dr. Goldstein. That's correct.
    Senator Wyden. Thank you.
    Senator Brownback. Dr. Goldstein, let me ask you, are you 
familiar with the areas of work going on in the adult stem cell 
area on juvenile diabetes treatment?
    Dr. Goldstein. I have some, yes.
    Senator Brownback. What do you--do you see some promising 
technologies developing there?
    Dr. Goldstein. There's a lot of work going on to coax 
already-living cells to reduplicate. That's some form of 
regeneration or neogenesis from already-mature cells. That work 
is going on in encouraging pancreatic ductal cells to 
reduplicate, et cetera. The numbers that they're able to 
reduplicate into tend to be small, not huge.
    Senator Brownback. That's--the nature of the adult stem 
cell----
    Dr. Goldstein. Right.
    Senator Brownback.--is, generally it's more controllable, 
but it's a slower-growing cell. Is that----
    Dr. Goldstein. Well, the----
    Senator Brownback.--in multiplying?
    Dr. Goldstein. It just doesn't create the numbers in 
experimental models that would be needed for widespread 
therapies. Today. Maybe tomorrow the data will look better. So 
like we support researchers to proliferate already-mature 
ductal cells, we support research to proliferate the few cells 
that are left, and we support work for an alternative source, a 
fresh source.
    Senator Brownback. I understand.
    Dr. Goldstein. That's the idea.
    Senator Brownback. Mr. Turner--if I could, with the 
permission of my colleague, could I have Dr. Levesque come up 
and answer--what's Mr. Turner's status now? You did the 
treatment on him. And is there something that you would expect 
that he would get an additional treatment on? If you don't mind 
coming back up, Dr. Levesque, I would really like to pursue 
this, where you think Mr. Turner is now in his treatment, and 
what we need to do or to learn from Mr. Turner's treatment.
    Dr. Levesque. Well, what we've learned is that the 
treatment we gave Mr. Turner worked for 4 years, and his 
symptoms progressed more significantly on the side that was not 
operated than on the side he has been operated. We need to work 
on the dosage of cells to be implanted, the type of cells that 
need to be implanted. And that's why we need to do more 
clinical studies.
    Senator Brownback. In your clinical study that's coming up 
now that you're going, what are you doing different on this one 
that you didn't do on Mr. Turner's?
    Dr. Levesque. Nothing. It's going to be identical in the 
type of procedure and the type of implantation. There will be 
different dosage, however, in the next group of patients. There 
will be four different groups of patient receiving incremental 
dosage.
    Senator Brownback. Of different dosages, so you'll be able 
to----
    Dr. Levesque. Different number of cells, yes.
    Senator Brownback. Are you discouraged that, after a period 
of 4 years or so, some of the symptoms are returning? What do 
you give of that?
    Dr. Levesque. Well, you know, I think this is an 
observation that we need to evaluate scientifically, and I 
think we have to do more studies to understand the progression 
of the disease and the cause of the disease. Is it the number 
of cells we implanted initially? The cell survival? We don't 
know. There are factors that we did have a significant 
improvement of his symptoms at 6 months after the injection, 
and this persisted for several years. So I think we're on a 
right path to find some type of therapy and cellular therapy 
for this disorder. We still have a lot to find out on the 
course of the disease and the evolution of the disease with 
this type of therapy.
    Definitely this type of therapy is--appears, anyway, to be 
better than the daily medication that are required by the 
hundreds of patient that have this disorder, and we are seeking 
alternative method to improve these patients. So we need to do 
more evaluation with this future group of patients.
    Senator Brownback. Are other people doing this same 
treatment regime and testing it through clinical trials?
    Dr. Levesque. Not this type of approach, as far as I know. 
There are other type of trials going on for Parkinson's 
disease, one of them using cells derived from the retina. And 
this type of cell secretes dopamine. But it's the same type of 
cells encapsulated to eliminate the rejection. At this point, I 
don't know the status of this clinical study.
    So, at this point, this is the only study going on using 
neural stem cells for Parkinson's disease.
    Senator Brownback. Good.
    Ron?
    Senator Wyden. Thank you, Mr. Chairman.
    Again, Mr. Turner, we're just thrilled to hear about your 
success.
    Mr. Turner. Could I add one thing----
    Senator Wyden. Absolutely. Why don't you, and then I have 
questions for your doctor. Go ahead, please.
    Mr. Turner. This is just to you, in general, sir. The thing 
that amazes the most is, he injected the cells into the left 
side of my brain that controls the right side of my body; and 
my left side, at that time, was fine. My left side, right now, 
is far worse than my right side, and my right side showed the 
symptoms first. So something must be going on there. I don't 
know. If he doesn't know, no one knows.
    Senator Wyden. Well, I just think when patients fight and 
have the kind of tenacity that you've shown, that that's a big 
part of what treatment's all about and why you're such a good 
role model, and why we're glad and thrilled that you're here 
and you tell us about your progress.
    Dr. Levesque, I want to ask you, though, a question with 
respect--you know, Parkinson's and this whole matter of 
embryonic stem cells being used in the research--when I asked 
you earlier about the comparison of adult stem cells and 
embryonic stem cells, you said--and I appreciate your candor--
that you had not done work with respect to embryonic stem 
cells. And that, to me, was important. May not be important to 
others, but that was important to me, in terms of the 
comparison, and particularly given the fact that you had been 
pretty critical, in your testimony, of embryonic stem cell 
research. My question to you is--a lot of advocates for cures 
to Parkinson's, a lot of the organizations, would like to see 
embryonic stem cells used to help pursue cures in this area. At 
least that's my understanding. Given that, do you, at any 
point, plan to try, even for the purpose of comparison--since 
you're making these statements about adult stem cells lines 
versus embryonic stem cells--do you, at any point, plan to even 
try to have some patients assisted with embryonic stem cells so 
that at least you could back up the kind of statement you made 
in your testimony today?
    Dr. Levesque. All right, let me comment to the first 
statement. My testimony is critical, from our aspect, of the 
perception and benefit of cell therapy in general, not only 
embryonic, but also adult. We have to understand where this 
type of therapy is in relation with other type of therapy, and 
I've mentioned that in my testimony, as well. And, as I 
mentioned, other avenues are potentially viable and successful 
beyond the cell therapy. So I was critical not only of this 
type of embryonic stem cell therapy, the are things that we 
don't know. There's a lot of noise and push to move on to 
embryonic stem cell therapy, but it's unknown what are the 
safety issues and benefit issue using these stem cells, the 
embryonic stem cell. I agree, we need to do more research, we 
need to compare both type of cells. Because the bottom line is 
that the embryonic stem cells will use the same pathways that 
the adult neural stem cell line will use to become 
differentiated neuron. So the embryonic stem cell can be used 
to become all kind of tissue. But you have to understand that 
the pathway to create the bottom neurons will be the same that 
we use for the adult neural stem cells. The risk and benefit of 
each of these type of therapy has to be evaluated.
    The approach I use has the benefit to be autologous. It's 
the same tissue than the patient. Whereas, the embryonic cells, 
these are cells derived from other patients. We don't know the 
risk of immune rejections using this tissue. From other type of 
research, we know that there's an immune reactions when we 
implant cells or foreign tissue in the brain. So there are ways 
to evaluate and minimize these type of rejections. One company 
is encapsulating these cells with some type of substance, 
supposedly to minimize this immune reaction. Perhaps the 
nuclear cell transplant technology will also minimize the risk 
of rejection. We don't know. We need to do more research.
    Senator Wyden. Well, again, with all due respect, your 
testimony is quite critical on embryonic stem cell research, 
and it is not critical of adult stem cell research, and the 
reason I asked you the question about whether, at some point, 
you would be willing to look at embryonic stem cells for the 
treatment of Parkinson's is, I think that would certainly, in 
my own view--as a legislator who has spent a lot of time on 
science issues, that would be relevant to me.
    Dr. Levesque. I think--if I can answer--I quite think the 
current approved cell lines are inadequate to study these 
questions. And, obviously----
    Senator Wyden. So you favor changing the Federal 
Government's policy?
    Dr. Levesque. I think if the Federal Government----
    Senator Wyden. That's a yes or no question.
    Dr. Levesque. It is, yes. Because----
    Senator Wyden. You favor changing the Administration's 
policy----
    Dr. Levesque. Yes.
    Senator Wyden.--on stem cell research?
    Dr. Levesque. Well, no, don't change the policy. The policy 
is approving these cell--we have cell lines that have been 
approved for research; however, these cell lines are 
inadequate. We need new cell lines. So, yes, we need to add 
more cell lines to the current cell lines to answer these 
specific questions.
    Senator Wyden. Where would they come from?
    Dr. Levesque. Well, these have to be obtained from either 
embryonic IVF clinic that discard the tissue, or with the 
somatic nuclear transfer using an ovum from a donor.
    Senator Wyden. And you think all this can be done without 
changing Federal policy?
    Dr. Levesque. No, and we need to change the restriction on 
these cell lines, definitely.
    Ms. Fajt. I agree.
    Senator Wyden. All right. So I think we're hearing 
something significant here. Dr. Levesque, you want to change 
Federal policy on stem cell research.
    Susan, you want to change Federal policy on stem cell 
research?
    Mr. Turner, do you think Federal policy ought to be 
changed? Just based on what you know.
    Mr. Turner. I would base my opinion on what Dr. Levesque 
said, because of his education. I'm educated as a mechanical/
electrical engineer, so I don't have the basis of the research 
to make that kind of decision.
    Senator Wyden. Extra points for candor.
    Laura, you want to say anything on this?
    Ms. Dominguez. No.
    Senator Wyden. All right, you're off the hook, and spared.
    I thank you all, and all of you have been very helpful. 
And, look, this is a field where there are differences of 
opinion. That's what I tried to talk about at the beginning. 
And, Dr. Levesque, you know, understand that I'm just thrilled 
with all that you've done for Mr. Turner. I mean, to have him 
come and to say what he said about you is thrilling.
    I'm here because I want to change Federal policy, because I 
think there can a lot more people like these three wonderful 
witnesses that are at the table. And to do it, we've got to 
change Federal policy, take the shackles off our scientists, 
and let them do what they were trained to do, which is to be 
scientific advocates. They weren't trained to do politics, they 
don't have election certificates. They were trained to be 
scientists. And the Federal Government has held them back, and 
it's wrong, and that's why I and, I think, a lot of legislators 
of both political parties want to change it.
    And I want to conclude by way of saying, to my friend Sam 
Brownback, who feels strongly about this subject and sees it 
differently, that I commend him for his fairness. He has always 
gone out of his way to make sure that all viewpoints are aired 
on this. And to my friend Chairman Brownback, I say thank you.
    Senator Brownback. Thank you.
    I want to thank the panelists for being here today on a 
panel discussing adult stem cells. And there are a variety of 
opinions on other topics which we've had numerous hearings in 
the Congress that could go into. And I've stayed completely 
away from and not engaged in this discussion because we really 
did want to focus on this particular area of adult stem cell 
research that I think we've had insufficient hearings on. What 
do we need to do to make further progress on these areas that 
are actually producing results and working today? What do we 
need to do on areas that we need to further get research on in 
the adult stem cell area that we all agree on and that we all 
agree is producing results? What do we need to do in therapies 
so that patients can get the treatments here rather than in 
Portugal? What do we need to do in therapy so that, once you 
get the treatments, you get the follow-up afterward, in care? 
And we could engage in the broader ethical debate, which has 
been raging for some period of time, and that would be fine, 
but it's not the point of what we've really tried to focus on 
today, of what can we do to support the real cures that we're 
seeing in front of us, and I want to see these treatments 
advance as much as we possibly can.
    This discussion has been constructive. Sorry to have 
engaged some of you in a political debate when we were really 
just trying to look at the scientific treatments and issues 
that you've been dealing with. And I do hope, with all my 
heart, we're going to continue to move forward and make some 
real advances in areas that you're helping to frontier, and I 
believe, with all my heart, we will.
    Thanks for joining us, all, very much. Thank you all, as an 
audience that has passions on this, for being a listening and 
not-participating audience. I appreciate that greatly.
    The hearing's adjourned.
    [Whereupon, at 4:55 p.m., the hearing was adjourned.]
                            A P P E N D I X

         Prepared Statement of Faye Armitage, Jacksonville, FL
    To All Members of the U.S. Senate:

    Thank you for the opportunity to express the extreme urgency of the 
matter before you.
    Since there undoubtedly are millions of others who will want to 
enter their testimonies into the Congressional Record, I will be direct 
so that I do not take up too much of your time.
    Currently 3,000 people a day are dying from medical conditions 
which could benefit from stem cell research. Not rescinding the 
restrictions on stem cell research would be unconscionable.
    Since transdifferentiation of adult stem cells has never been 
proven (according to Dr. Irv Weissman, leading adult stem cell 
researcher and many others), it is our moral obligation to expand the 
stem cell policy to accelerate embryonic stem cell research.
    The suggestion that stem cell research is ``killing human embryos'' 
is misleading. The embryonic stem cells come from frozen blastocysts 
(fertilized eggs) from in vitro fertilization clinics, eggs that will 
be thrown away anyway by the IVF clinic (with donor consent) because 
they can no longer be used to create babies. Many of these fertilized 
eggs are defective/old, and so were not chosen for implantation--but 
can still be used to create stem cells. Is it better to trash them or 
use them to save lives? I'm sure that most sensible and rational people 
would agree that the latter option is moral.
    Another frequently quoted adult stem cell researcher, Dr. Wise 
Young recently said this: ``. . . at the present, embryonic stem cells 
are the only cells that have been shown to produce neurons when 
transplanted into the brain and spinal cord.
    Can we do the above with adult stem cells? Maybe, but it will take 
longer to do this research with adult stem cells. Waiting is not an 
acceptable option for people with severe disabilities, particularly 
terminal neurodegenerative diseases such as Alzheimer's, Parkinson's 
disease, and ALS.''
    Now I'll explain my personal interest to accelerate ESC research: 
My 14 year old son Jason wants to be freed from paralysis and resume 
his life. Our family suffered the most unimaginable devastation when, 
at the age of seven, Jason became quadriplegic after a collision with 
another player on the soccer field.
    Since the essence of life for a child is physical activity, Jason 
has all but lost his childhood. Every day, Jason is confronted with the 
unbearable pain of watching his four sisters' lives move forward, while 
he feels so terribly left behind.
    Please don't condemn Jason and others like him to this continued 
suffering by not immediately expanding funding for ESC research. Jason 
desperately wants his life back!
    Every day that ESC research funding is held up, 3,000 more people 
die from conditions that could benefit from stem cell research.
    Thank you again for this opportunity to speak to you on the urgency 
of increased funding for stem cell research.

                                  
