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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 

( 

(II) 

SENATE COMMITTEE ON COMMERCE, SCIENCE, AND TRANSPORTATION 

ONE HUNDRED EIGHTH CONGRESS 

SECOND SESSION 

JOHN MCCAIN, Arizona, Chairman 

TED STEVENS, Alaska 

CONRAD BURNS, Montana 

TRENT LOTT, Mississippi 

KAY BAILEY HUTCHISON, Texas 

OLYMPIA J. SNOWE, Maine 

SAM BROWNBACK, Kansas 

GORDON H. SMITH, Oregon 

PETER G. FITZGERALD, Illinois 

JOHN ENSIGN, Nevada 

GEORGE ALLEN, Virginia 

JOHN E. SUNUNU, New Hampshire 

ERNEST F. HOLLINGS, South Carolina, 

Ranking 

DANIEL K. INOUYE, Hawaii 

JOHN D. ROCKEFELLER IV, West Virginia 

JOHN F. KERRY, Massachusetts 

JOHN B. BREAUX, Louisiana 

BYRON L. DORGAN, North Dakota 

RON WYDEN, Oregon 

BARBARA BOXER, California 

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 

CONRAD BURNS, Montana 

TRENT LOTT, Mississippi 

KAY BAILEY HUTCHISON, Texas 

JOHN ENSIGN, Nevada 

GEORGE ALLEN, Virginia 

JOHN E. SUNUNU, New Hampshire 

JOHN B. BREAUX, Louisiana, Ranking 

JOHN D. ROCKEFELLER IV, West Virginia 

JOHN F. KERRY, Massachusetts 

BYRON L. DORGAN, North Dakota 

RON WYDEN, Oregon 

BILL NELSON, Florida 

FRANK R. LAUTENBERG, New Jersey 

(III) 

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 

(1) 

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. 

2 

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.’’ 

3 

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 

4 

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? 

5 

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 CedarsSinai 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 expeVerDate Nov 24 2008 08:48 Jun 27, 2013 Jkt 075679 PO 00000 Frm 00009 Fmt 6633 Sfmt 6601 S:\GPO\DOCS\81637.TXT JACKIE
6 

rience 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 environVerDate Nov 24 2008 08:48 Jun 27, 2013 Jkt 075679 PO 00000 Frm 00010 Fmt 6633 Sfmt 6601 S:\GPO\DOCS\81637.TXT JACKIE
7 

mental 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:] 

8 

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 Le ́vesque, 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 

9 

and transmittable diseases generally associated with tissue transplants from a 

source external to the patient such as HIV, Encephalitis, Hepatitis and CreutzfeldJacobs 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 

10 

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. 


714LEVES1.eps
11 


714LEVES2.eps

Parkinson's disease 

Parldnson's Disease is the most common 

neurodcgcnemtive movement disorder and affW;ts 

more than 1 million pelliOns in the US (with an 

estimated prevalence of 1% of the population 

over 50 years). 

Parlcinson's disease is associated with a 

progressive loss of midbmin dopamine (DA) 

neurons (estimated nonnally at 500,000 in the 

substantia nigm compacta(SNc» 

Neurotransp1antation 

Transplantation of dopamioe neuroblasts from fetal 

mesencephalon restore DA release and ameliorate 

motor deficits . 

Major limitations: ethical. technical variability. poor 

tissue survival. infection. standardization, quality 

control ... Results of 2 double blind slUdies:failures 

Alternative sources of celJs under investigation: 

Porcine oeuroblasts. retinal cell. embryonic 

stem (ES) cells and 

adult newal stem cells CNSQ. 

Palhophysiology of Parkinson's Disease 

Exact cause remains unknown. 

Lewy bodies are foand in 

pigmented bmin stem neurons. 

80% of cell loss before clinical 

symptoms occur. 

Long tenn medical therapy with !e~v==I!~~ severe secondary motor complications and striaJaI 

dysfunction. Newa1 transplantation aims at 

replacing the loss of nigrostriatal neurons within the 

striatum itself. 

Neural Stem Cd! 
12 


714LEVES3.eps

Background: Hmnan Adult NSC 

l.MultiJK*Dt __ dcm cdII .c Coad aloaa 1hc .,adriatlar DCIII'OaXia .... Ko,..I99Z 

Rey.olclsad Wciaa,1991 

Loia aDd AlYIftZ-BaOa.l993 

2.Pro1ifr:ralc"'CI.~iDJ1!:IPODIeto:mFaocIbFGF 

Monbcadd "'1994, Wciaaetat.l996 

3.CoutiJIDOaS ac:amacaaia oa:an in add: rodall cIc:atI&e I)'I"QI 

AItJum ad n.. 1965 

""' ....... 1981,""""' 

4.Ccrical DeOJOICII- debIIe Notllcbolm. 1986, GoaIdetal,l999, Palma'tal., 2000: 

"'Wbac, Oh WIMn: a'C b11 SIan Cdkl ..... 

Background: Hmnan Adult NSC 

Our Neural Repair Research laboratories isolated 

human NSC from 

• Selective bippocampectomy spectlDCD (Epi1epai:l199B) 

-Prefrontal neocortex (Soc ~ At..I9!19). as 

previously found in rodents, birds and prima .... 

-Adult NSC are similar to Embiyonic NSC in their. 

l.odf_"""""",, 

2. abiIi1y to diffeftllUte ill __ CII" JWi 

cdl 3. __ iItcapa(MJIIIIIIdri.c:a .Ii .. ri ... ) 

Neural Stem Cells (NSC> remsent a renewable source ofh1UJW) NeUJal cells. 

Growth of Neunol Stem Cells 

_ .. -

Background: Hmnan Adult NSC 

5. Neand 8CaD cdb iIolated from cmbIyotric: ... ..wt ..... 1nia 

S.ellllsa:!. eta(. lW1, <ltaimeD-Rmmaa et Ii, 1997, 

VCIIOOYidll,1999, Kukc:ko.,c:t",1999 

6. HamIIII NSC mnlve aad dilTezadi3rcimo ...... .ad Jfia is 

..... rodem inia. 

V.-oridal.l999.Fric:bI'"ctlll ,l999,Carpc:lllk:rctll,l999 

7.Ditferad:popabIiOll aCNSC..m. rep,.r liffert:llCaJDa 

."..,...,._oi ........ _(Somm« ..... I ... ) 

~-' ...,wamo< ..... aodJ.,..,. 1998) 

RegeneratioD P11rallels OntogeDesis 

Studies of cell division aad ceU fate detDODSb'a&e 

panillels _. developmclllllleeU biology aad 

rqencrative processes 

Expression of Regulatory Genes 

NSC gene anay 

profiling using eDNA 

microarrays 

Both Adulland Fetal _ .. t:: 

NSC express neurogenic 

tIanscription factors 

(HLH. HD,zF) 
13 


714LEVES4.eps

Experimental Results: 

Aduh Human Neural Stem Cells 

Adoh NSC survive after 

stritaI transplaotatioa ill 6-

OH DOPA ... poztittsooWI 

modeL expR8S stable 

pbenotypc8, mignote within 

striatom. 

Aduh NSCdiffen:Jtti_ 

DA neurons in rat model 

can produce DA aod 

comet rotIOionaI bebavior. 

Experimental Results: Functional 

Human Neural Stem ceDs 

3.A'teI'qeFaDcticealRecoYe:Q'after~: 

........." .... _--_ ..... 

--

Results: Isolation 

Epigenetic stimulation 

with bFGF (basic 

fibroblast growth 

13ctor) and EGF 

(epidennal growth 

fuctor) 

bFGF. EGF. other 

fuctolS Induce Iarg ... 

number ofNSC 1D 

smvIve 

Experimental Results: Survival 

Human Neural Stem ceDs 

L Anraaeioq: tam ani .... «.....: 311i 

2.. Aw-aaplbdioaC1B(+)Nc:III..:: 

__ lSCIr ._aII 

i~'~~ " M .. 0 a m __ ....... CllUIIII 

Methods: From Biopsy to Transplantation 

Patient ---_ t · Harvesting biopsy 

T=~OO 1 :=:'~NSC t Propagation of NSC 

DilfeRntiation of 

u~ ~ 

5-10 .... "IO'-IO'fold iucn:ase. Safety. sterility 

testings, genetic profde, potency assays 

4-6 PlOtlIbs 

Methods: Cell Expansion 

1. Stln'iving cells grown in 

serum-free media 

supplemented with 

growth fac1Drs 

2 Newal Stem cdls 

grown as neurospheres 

in tissue culture flasks 

3. Spheres dissociated by 

mechanical trituration 

evezy 2-5 days 
14 


714LEVES5.eps

Results: CeU Expansion 

NSC propagated as 

neurospberes in presence of 

EGF and bFGF for6 months 

Cell. reproducibly express 

Nestin and Musabi mRNA, 

typical marlrersof NSC 

After 6 months. NSC 

exceeded 2S millions 

Results: Neural Stem Cell 

Differentiation 

NEURAL STEM CELL ACl 

LINE 

ASTROC\'TES(GFAP+) 49% 

OLIGODENDROC\'TES 

(GALC+) 

NEURONS (I m 35% 

TUBULIN+) 

Results: Dopamine Synlhesis 

111 VUm l>opIrI'Dc 8Ca'dioII--.ed tria BPLC"',.. Bc:fon _ Aftm-SOmM KO CarlO miatce 

lU- "' '" >0 '" NO> 

_ .......... c.lUIM '" ~ ..... NtelSl:IIIIIMtianI " 

Melhods: Ceu Differentiation! Immunostaining 

LCeUs dissociated 011 cufturc dishes in serum·.free medium 

Growth £acton aDd RdiDoi<: Acid. cAMP .... GDNF 

2.lmmunohi-'ochemic.l saaiaiag; 

Sm-tubuliD antibodies 

ADIi-GAD(GABA) 

AII1iL-g1_ 

AII1i glycine 

AII1i (CHAT) 

ADIi tyrosiae hydroxylase (111) 

AII1i dopa decarboxylase (DOC) 

Results: Neural Stem Ceu Differentiation II 

_IIoW NEURONS r..- ACl LINE 35% 

T,...-wJlydI'O.,-(+)_ 15% 

Dopa Doca......,-(+) __ 13% 

GABA-.- GI ___ 60% 

ZO% 

CboIJaerpc .ea ..... 2% 

Glydaer&l< ........ 3% 

Phase I Results: Clinical Presentation 

Age: 51-year Right H.MaIe 

Disease presenlation: Asymmelrical-Tremor 

Rigidity-Hypolcinesia 

Levodopa response: Positive 

Molor Ructuations: Some wearing off 

Dyskinesia: None 

Disease dlmllion: 11 years 

Hoehn and Yahr SIage: "off" meds:4 

"on"meds:2S 

Levodopa daily intake: 600 mglday 
15 


714LEVES6.eps

Phase I Results: Clinical Presentation 

oQinical Study approved by CedarsSinai Medical Center IRB 

-Initial surgery. 

Left Thalamic Stimulator: Sept 

1998 Cortical Biopsy(9Omm') 

oCell expansion-Differentiation 

• Transplantation procedure: 

7 months Poot Biopsy 

Unilateral Left Putamen 

Results: Fnnctional Imaging 

Clinical Evaluation 

Results: Stereotactic Microtransplantation 

• UnilateJal Putamen Targets and T"'jectories: 

Six micro-injections, 4mm apart, using modified 

microsyringe on bydraulic microdrive. 

Delivery ",Ie of 5 J'l/min of cell suspension 

(lmiUion cellsl1Omioisite) 

Estimated sUlvival of 600,000 to 1,800,000 cells: 

630,000 oeDIODS 

93 000 DA netIIODS 

42,OOOGlutamale 

130,000 GABA De1U01IS 

Clinical Evaluation 

Long Term Outcome Results:UPDRS 

• Tobl UPDRS: 81" impoyClDf:ld ....... , B31iJ ""ofr 

T ......... 

~ i c I ~ 

l le::::.:1 

: 

r--. r-. ~ I 

-- ~.- ~- ~-
16 

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? 


714LEVES7.eps
17 

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 docVerDate Nov 24 2008 08:48 Jun 27, 2013 Jkt 075679 PO 00000 Frm 00021 Fmt 6633 Sfmt 6601 S:\GPO\DOCS\81637.TXT JACKIE
18 

tor. 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 

19 

1Marshall, E.(2000) The Business of Stem Cells, Science, 287:1419–1421. 

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 embryVerDate Nov 24 2008 08:48 Jun 27, 2013 Jkt 075679 PO 00000 Frm 00023 Fmt 6633 Sfmt 6621 S:\GPO\DOCS\81637.TXT JACKIE
20 

2The Ledger.com, Lakeland, FL, Published Thursday, May 20, 2004, ‘‘Venture capital money 

backs California stem cell measure’’, PAUL ELIAS 3L.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. 4F 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. 5Freed 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. 6Olanow 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 7Sutcliffe, 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. 8DeBaun, 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. 9PERSONHOOD PROCLAMATION, National Sanctity of Human Life Day,1988, By the 

President Reagan, A Proclamation: 

onic/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

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. 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

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: 

‘‘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 

21 

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. 11Song 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. 12Keene 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. 14Toma 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. 16Freed 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. 17Olanow, 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. 18L.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. 19F 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. 20Morshead, 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. 

(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

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

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

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 

22 

21Tang, 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. 22D’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. 23Soria 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. 24Stem Cells An Unlikely Therapy for Alzheimer’s Reagan-Inspired Zeal For Study Continues 

By Rick Weiss, Washington Post, June 10, 2004; Page A03. 25http://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. 

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

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 UnfortuVerDate Nov 24 2008 08:48 Jun 27, 2013 Jkt 075679 PO 00000 Frm 00026 Fmt 6633 Sfmt 6621 S:\GPO\DOCS\81637.TXT JACKIE
23 

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. 31Liker 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. 32Palmer 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. 33Sanchez-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. 35Goodwin 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. 36Lu 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. 37Sanberg PR. Chopp M. Willing AE. Zigova T. Saporta S. Song S. Bickford P. GarbuzovaDavis 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. 40Lu 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. 41Shen 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. 

nately Dr. Eckhoff and his patient were unable to join us today but may testify later in the year. 

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

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 vehiVerDate Nov 24 2008 08:48 Jun 27, 2013 Jkt 075679 PO 00000 Frm 00027 Fmt 6633 Sfmt 6621 S:\GPO\DOCS\81637.TXT JACKIE
24 

42Schwarz 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. 44Park 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. 

cle 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. 

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: 

25 

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 HasseFerreira. 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. 


714PED1.eps
26 

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. 


714PED2.eps
27 

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. 


714PED3.eps 714PED4.eps
28 

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 


714PED5.eps 714PED6.eps 714PED7.eps

80 

ASIA Scores (Sensory/Pinprick) 

70 • 

60 ......... R. 1 

......... R.2 

50 

R. 3 

.....,..... R. 4 

......... R. 5 

40 ......... R. 6 

-+- R. 7 

30 

R"e-Op 6m 12m 18m 24 m 

ASIA Score (Motor Arms) 

50 

45 • • • • • 

40 ......... R.1 

35 R . 3 

30 

25 ......... R . 6 

20 

15 

10 

5 

before 6m 12m 18m 24 m 

ASIA Scores (Motor Legs) 

8 

7 

6 ---+- A. 1 

5 --A.2 

A. 3 

4 -M- A. 4 

3 ........ A. 5 ........ A.6 

2 ......... A. 7 

0 

Pre-Op 6m 12m 18m 24 m 
29 

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. 


714PED8.eps
30 

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 

31 

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 

32 

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 overVerDate Nov 24 2008 08:48 Jun 27, 2013 Jkt 075679 PO 00000 Frm 00036 Fmt 6633 Sfmt 6621 S:\GPO\DOCS\81637.TXT JACKIE
33 

sight 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 

34 

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 

35 

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 

36 

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 lifeshortening 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 

37 

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? 

38 

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. 

39 

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 

40 

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 

41 

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 

42 

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 

43 

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. 

44 

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—— 

45 

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 stimuVerDate Nov 24 2008 08:48 Jun 27, 2013 Jkt 075679 PO 00000 Frm 00049 Fmt 6633 Sfmt 6601 S:\GPO\DOCS\81637.TXT JACKIE
46 

lates 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 

47 

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. 

48 

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 

49 

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. 

50 

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 inVerDate Nov 24 2008 08:48 Jun 27, 2013 Jkt 075679 PO 00000 Frm 00054 Fmt 6633 Sfmt 6601 S:\GPO\DOCS\81637.TXT JACKIE
51 

jured 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 

52 

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 

53 

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, 

54 

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. 

55 

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. 

56 

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 

57 

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 

58 

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 

59 

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 insulinproducing 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. 

60 

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 

61 

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 disVerDate Nov 24 2008 08:48 Jun 27, 2013 Jkt 075679 PO 00000 Frm 00065 Fmt 6633 Sfmt 6621 S:\GPO\DOCS\81637.TXT JACKIE
62 

eases 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. 

63 

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. 

64 

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? 

65 

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 addiVerDate Nov 24 2008 08:48 Jun 27, 2013 Jkt 075679 PO 00000 Frm 00069 Fmt 6633 Sfmt 6601 S:\GPO\DOCS\81637.TXT JACKIE
66 

tional 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—— 

67 

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. 

68 

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 alreadyliving 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. 

69 

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 

70 

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? 

71 

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 

72 

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.] 

(73) 

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. 

Æ