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S. HRG. 109–249 

STEM CELLS RESEARCH, 2005 

HEARING 

BEFORE A

SUBCOMMITTEE OF THE 

COMMITTEE ON APPROPRIATIONS 

UNITED STATES SENATE 

ONE HUNDRED NINTH CONGRESS 

FIRST SESSION 

SPECIAL HEARING 

OCTOBER 19, 2005—WASHINGTON, DC 

Printed for the use of the Committee on Appropriations 

( 

Available via the World Wide Web: http://www.gpoaccess.gov/congress/index.html 
(II) 

COMMITTEE ON APPROPRIATIONS 

THAD COCHRAN, Mississippi, Chairman 

TED STEVENS, Alaska 

ARLEN SPECTER, Pennsylvania 

PETE V. DOMENICI, New Mexico 

CHRISTOPHER S. BOND, Missouri 

MITCH MCCONNELL, Kentucky 

CONRAD BURNS, Montana 

RICHARD C. SHELBY, Alabama 

JUDD GREGG, New Hampshire 

ROBERT F. BENNETT, Utah 

LARRY CRAIG, Idaho 

KAY BAILEY HUTCHISON, Texas 

MIKE DEWINE, Ohio 

SAM BROWNBACK, Kansas 

WAYNE ALLARD, Colorado 

ROBERT C. BYRD, West Virginia 

DANIEL K. INOUYE, Hawaii 

PATRICK J. LEAHY, Vermont 

TOM HARKIN, Iowa 

BARBARA A. MIKULSKI, Maryland 

HARRY REID, Nevada 

HERB KOHL, Wisconsin 

PATTY MURRAY, Washington 

BYRON L. DORGAN, North Dakota 

DIANNE FEINSTEIN, California 

RICHARD J. DURBIN, Illinois 

TIM JOHNSON, South Dakota 

MARY L. LANDRIEU, Louisiana 

J. KEITH KENNEDY, Staff Director 

TERRENCE E. SAUVAIN, Minority Staff Director 

SUBCOMMITTEE ON DEPARTMENTS OF LABOR, HEALTH AND HUMAN SERVICES, AND

EDUCATION, AND RELATED AGENCIES

ARLEN SPECTER, Pennsylvania, Chairman 

THAD COCHRAN, Mississippi 

JUDD GREGG, New Hampshire 

LARRY CRAIG, Idaho 

KAY BAILEY HUTCHISON, Texas 

TED STEVENS, Alaska 

MIKE DEWINE, Ohio 

RICHARD C. SHELBY, Alabama 

TOM HARKIN, Iowa 

DANIEL K. INOUYE, Hawaii 

HARRY REID, Nevada 

HERB KOHL, Wisconsin 

PATTY MURRAY, Washington 

MARY L. LANDRIEU, Louisiana 

RICHARD J. DURBIN, Illinois 

ROBERT C. BYRD, West Virginia (Ex officio) 

Professional Staff 

BETTILOU TAYLOR

JIM SOURWINE

MARK LAISCH

SUDIP SHRIKANT PARIKH

CANDICE ROGERS

LISA BERNHARDT

RACHEL JONES

ELLEN MURRAY (Minority) 

ERIK FATEMI (Minority) 

ADRIENNE HALLETT (Minority) 
(III) 

C O N T E N T S 

Page 

Opening statement of Senator Arlen Specter ........................................................ 1 

Statement of Senator Mary L. Landrieu ............................................................... 2 

Prepared statement .......................................................................................... 2 

Statement of Senator Tom Harkin ......................................................................... 4 

Statement of Senator Thad Cochran ...................................................................... 4 

Statement of Anthony Herrera, author and cancer survivor ............................... 5 

Prepared statement .......................................................................................... 7 

Statement of Judith Gasson, Ph.D., director, Jonsson Comprehensive Cancer 

Center .................................................................................................................... 8 

Prepared statement .......................................................................................... 9 

Statement of Rudolf Jaenisch, M.D., professor of biology, Massachusetts Institute of Technology ................................................................................................ 10 

Prepared statement .......................................................................................... 12 

Statement of Steven Teitelbaum, M.D., Wilma and Roswell Messing, professor 

of Pathology and Immunology, Washington University School of Medicine ... 14 

Prepared statement .......................................................................................... 16 

Statement of John Wagner, M.D., scientific director of clinical research, Blood 

and Marrow Transplant Program and Stem Cell Institute .............................. 17 

Prepared statement .......................................................................................... 19 

(1) 

STEM CELLS RESEARCH, 2005 

WEDNESDAY, OCTOBER 19, 2005 

U.S. SENATE,

SUBCOMMITTEE ON LABOR, HEALTH AND HUMAN

SERVICES, EDUCATION, AND RELATED AGENCIES, 

COMMITTEE ON APPROPRIATIONS, 

Washington, DC. 

The subcommittee met at 9:31 a.m., in room SD–138, Dirksen 

Senate Office Building, Hon. Arlen Specter (chairman) presiding. 

Present: Senators Specter, Cochran, Harkin, and Landrieu. 

OPENING STATEMENT OF SENATOR ARLEN SPECTER

Senator SPECTER. Good morning. 

Today our focus is on stem cells. Our hearing coincides with the 

announcement by the South Korean Nuclear Transplantation Consortium that they are starting an operation today on a world stem 

cell foundation which will be based in the Seoul National University in South Korea with satellite offices in San Francisco and England. I applaud what they are doing, but I regret that the United 

States is falling farther behind in world leadership on scientific research generally and specifically on stem cell research. 

Now, this is the 17th hearing which this subcommittee has held 

on this important subject since we first learned about stem cells in 

November 1998. It is well known that the stem cells have the possibility for curing or alleviating the problems of so many maladies. 

The House of Representatives has passed legislation which would 

remove the restriction by the Federal Government on funding stem 

cell research, and Senator Harkin and I have introduced identical 

legislation in the Senate, S. 471. The House bill received support 

from some 50 Republicans crossing party lines, and it is my view 

that with sufficient focus and sufficient attention, there could be 

amassed enough votes to override a presidential veto. I say that regretfully and reluctantly, but this is a matter of utmost importance 

and has a direct impact to some 110 million Americans. 

President Nixon declared war on cancer in 1970 and had the 

United States devoted the resources to that war which we devote 

to other wars, I think the war on cancer could have been won. I 

say that with special concern because I have had my own problems 

with Hodgkin’s lymphoma cancer this year, and cancer continues 

to claim deaths in the hundreds of thousands. 

This happens to be an especially busy day. Most days are busy 

on Capitol Hill, but we are in the midst of proceedings on the nomination of Ms. Miers for the Supreme Court, and I am going to have 
2 

to excuse myself at about 10:00, but I will have an opportunity before that occurs to hear all of the witnesses. 

Now I would like to yield to my distinguished ranking member, 

Senator Harkin. 

Senator HARKIN. Thank you very much, Mr. Chairman. Senator 

Landrieu I know has to leave right away. If I could just yield a couple minutes for her of my time, I would appreciate it. 

Senator SPECTER. Well, I was about to yield a couple minutes to 

Senator Cochran, but since you spoke first, Senator Landrieu. 

STATEMENT OF SENATOR MARY L. LANDRIEU

Senator LANDRIEU. Thank you, Mr. Chairman. I thank Senator 

Harkin just for one moment because I have got to leave for another 

meeting. It is a very busy day, as the chairman said. 

Let me thank the chairman and Senator Harkin for their pursuit 

of a solution to this dilemma and to this great challenge. I have 

a slightly different view that I will submit for the record in written 

testimony. 

For this morning, I will just say that as we pursue cures for the 

many diseases that challenge us and while I understand that embryonic stem cells hold promise for curing diseases, as the chairman and many others have pointed out, I think that we have to 

be very mindful of what many of our ethical leaders have said and 

the Catholic bishops in particular that it is important in the pursuit of progress to not undermine human dignity. And there is a 

line that can be drawn between progress and human dignity, and 

creating embryonic stem cells for the purpose of creating human 

beings for the purposes of destroying them for science crosses that 

line in my opinion. 

PREPARED STATEMENT

I will submit more to the record, but I understand that this will 

be a continued debate, and I thank the Senator for allowing me to 

express my views. 

Senator SPECTER. Thank you, Senator Landrieu. 

[The statement follows:] 

PREPARED STATEMENT OF SENATOR MARY L. LANDRIEU

Thank you, Mr. Chairman. I have had the privilege of serving as a member of 

this subcommittee for four years now. I think it is important to note that one of 

the very first hearings I attended was on this very issue. A lot has changed since 

then—both in the ethical debate and in the science. But what have not changed are 

the moral parameters that must guide us in these decisions. As Richard Doerflinger 

of the Catholic Conference of Bishops put it—‘‘We must be careful not to undermine 

human dignity in the pursuit of human progress.’’ 

Since that hearing four years ago, in August of 2001, the President issued an executive order, allowing for federal funding for stem cell research on the then existing stem cell lines. In November of that same year, he appointed a council to monitor stem cell research, to recommend appropriate guidelines and regulations, and 

to consider all of the medical and ethical ramifications of biomedical innovation. To 

date, this council has issued six hundred plus page reports on the bioethics issues 

involved in stem cell research. Meanwhile, the scientific community has moved forward in its advancements in knowledge and discovery. And everyday we, as members of Congress are faced with the questions of how far we should go in the name 

of science. 

There is no doubt that embryonic stem cell research holds the promise of curing 

diseases such as Parkinson’s, diabetes, Alzheimer’s and cancer. Even President 

Bush stressed the importance of federally funded research in approving the original 
3 

stem cell lines in 2001—he explicitly stated that federal dollars help attract the best 

and brightest scientists and help ensure that new discoveries are widely shared at 

the largest number of research facilities. Federal funding not only allows us to encourage and financially support this research, it allows us to use the power of the 

purse to be sure it is done in the most safe and ethical way possible. Mr. Chairman, 

I want to state clearly for the record, I support federal funding for embryonic stem 

cell research provided that the embryos used in these studies are those that are in 

excess from the fertility process and are knowingly donated for this purpose. 

I have met with many constituents suffering from life altering and fatal diseases 

and they have told me the impact that this research may have on their lives. One 

such constituent who I will never forget is a nine year old girl, Sarah, who suffers 

from juvenile diabetes. Sarah told me of her daily routine of shots and blood tests. 

Her parents told me of some of the effects of diabetes such as vision loss, kidney 

failure, blindness, nerve damage, amputations, heart attack, and stroke. They 

begged me, on her behalf, not to block this important research that could mean a 

normal childhood for Sarah. Sarah is not alone in this hope, 35 children a day are 

diagnosed with Type One Diabetes. 

There are currently 400,000 frozen embryos at IVF clinics around the country— 

88.2 percent of which are used for implantation in the mother’s womb—2.8 percent 

are given up for adoption—the wonderful ‘‘snowflake babies’’ we all hear so much 

about. This translates to a total of 11,000 embryos that are not going to be implanted and are voluntarily donated. It is important to note, if these embryos were 

not donated then they would be destroyed—not for science—but thrown away with 

the rest of the medical waste for the day. We cannot allow these valuable embryos 

to be discarded when even according to the President’s Council on Bioethics, ‘‘stem 

cells and their derivatives may prove a valuable source of transplantable cells and 

tissues for repair and regeneration. If these healing powers could be harnessed, the 

medical benefits for humankind would be immense, perhaps ushering in an era of 

truly regenerative medicine.’’ 

Please do no let my views expressed today confuse your understanding of my support of legislation banning human cloning. Embryonic stem cell research using excess embryos from IVF treatments and creating cloned embryos for scientific purposes should not be confused. I believe that creating a human embryo for the sole 

purpose of its destruction through experimentation is wrong, unethical and should 

be illegal. Since I mentioned the hearing on stem cell research conducted by this 

committee four years ago at the beginning of my remarks, I think it is important 

to note that many members of this subcommittee also expressed concerns about the 

creation of human embryos for research. 

The human body is not a product to be mass-produced and stripped for parts, 

most especially in the earliest stages of its development—women’s eggs and wombs 

should never be commodities sold to the highest bidders. But this is a very real risk 

of so called ‘‘therapeutic cloning.’’ Experts estimate that over 800 million eggs would 

be needed to support one-sixteenth of the possible human cloning experiments. We 

are already getting reports that clinical researchers in Seoul, Korea, in England, 

and in San Francisco will be working with the South Korean veterinarian and stemcell biologist whose laboratory leads the world in the use of the somatic-cell nuclear 

transfer technique, to recruit women to donate eggs and patients to donate somatic 

cells. 

What’s more, regardless of what proponents of this research will tell you, there 

is only one kind of cloning. The only difference between what has come to be called 

‘‘reproductive cloning’’ and ‘‘therapeutic cloning’’ is what is done with the clones once 

they are created. Legislation that purports to ban the birth of a cloned human being 

does not ban its creation, only its implantation into a human uterus. Once we support and encourage the creation of millions of cloned human beings, do we really 

believe we would have the power to successfully monitor and ban their implantation? The only effective way to ban human cloning is to stop the process before it 

starts. 

Finally, Mr. Chairman, it is because I believe that there is immense potential in 

embryonic and adult stem cell research that I oppose federal support for human 

cloning. I believe that banning, even if only temporarily, this one procedure helps 

to focus science and funding for research to equally promising but less problematic 

areas such as embryonic and adult stem cell research. 

I look forward to hearing from the witnesses today and thank you, Chairman 

Specter for holding this important hearing. 

Senator SPECTER. Senator Harkin. 
4 

STATEMENT OF SENATOR TOM HARKIN

Senator HARKIN. Thank you, Mr. Chairman. Again, let me just 

compliment you on your great leadership on a lot of things, but especially on this issue since 1998, as you mentioned, and for calling 

this hearing. 

We have had a pretty busy year with hurricanes on the Gulf 

Coast, of course, and as the chairman knows better than anyone 

else, the Senate having to have hearings on two Supreme Court 

nominees, which the chairman chairs that committee. And yet, the 

need to continue our push for stem cell research is as critical as 

ever. 

I was privileged this summer to meet with some of the South Koreans, and you mentioned the article that was in the paper this 

morning, Mr. Chairman, that they are moving ahead on this, the 

whole area of somatic cell nuclear transfer, and the kind of promise 

that holds. 

I am just hopeful that we can move ahead on this. People are 

suffering and dying. They need hope. We know this holds great 

promise. We all know that medical research is not just done by one 

person, not done by two. The best research is when a lot of people 

are involved in it and it is spread around. That is what we need 

to do. We need to get more involved in this type of research. 

Yet, we have manacles put on our researchers today and we need 

to remove those. That is what the bill that Senator Specter and I 

have supported, the one that passed the House, does. That is why 

we hope today we can move ahead with a look at somatic cell nuclear transfer and what that means for the future of stem cell research. 

So, again, Mr. Chairman, I thank you for holding this hearing 

and, again, I thank you for your great leadership on this issue. 

Senator SPECTER. Senator Cochran, would you care to make an 

opening statement? 

STATEMENT OF SENATOR THAD COCHRAN

Senator COCHRAN. Mr. Chairman, thank you very much. I congratulate you on your continued strong leadership in helping to explore the possibilities that medical research holds for curing and 

preventing illness and disease. You have done more than anybody 

I can remember since I have been in the Senate to not only focus 

attention on these opportunities that we have to legislate and support research and to authorize programs to achieve these goals. So 

I congratulate you and thank you again for this hearing. 

I am really here to introduce Anthony Herrera who is a friend 

of mine since 1961, who is a member of this panel. So I will await 

your advice as to when that would be appropriate. 

Senator SPECTER. Well, thank you very much, Senator Cochran. 

We appreciate your continuing support for this subcommittee and 

your membership on the subcommittee, notwithstanding your very 

onerous duties as chairman of the full Appropriations Committee. 
5 

STATEMENT OF ANTHONY HERRERA, AUTHOR AND CANCER SURVIVOR 

Senator SPECTER. We now move to our panel of witnesses and 

our lead witness is Mr. Anthony Herrera. I yield to you again, Mr. 

Chairman, for the formal introduction. 

Senator COCHRAN. Thank you very much. It is a great pleasure 

for me to introduce to the committee Anthony Herrera, whom I 

have known since 1961. We met when I was entering my first year 

of law school and he was beginning his first year of undergraduate 

school at the University of Mississippi, and I happened to be in the 

same residence hall and became the dormitory manager, as we 

called it, back in those days. He was young, but energetic, full of 

an interest in all of the things that were going on at that campus. 

He became an excellent student and then went on to a successful 

career in the performing arts as an actor, writer, director. He has 

been on As the World Turns off and on for a long, long time. James 

Stenbeck is his stage name on that show. And James Stenbeck has 

been a survivor too of sorts. He would disappear. People would 

think he was a goner and then he would reappear sometime later 

full of life and enthusiasm. That is the story of Anthony Herrera 

as well. 

He has battled cancer and has survived. He has written a book 

about it, The Cancer War, which I recommend. I know the chairman has read it. I have read it. It is very instructive into the challenges that confront someone who is a victim of lymphoma or other 

forms of cancer. He had a rare kind of lymphoma. And transplants 

of bone marrow, stem cells, all of these things have been involved 

in his life. He has lived through it all and can help us understand 

the challenges that victims face and the possible successes there 

are in our effort to deal more successfully with some of these forms 

of cancer. 

So it is a great pleasure for me to welcome him and thank him 

for being here to help us understand the challenges. 

Senator SPECTER. Thank you very much, Mr. Chairman. 

Mr. Herrera, we do very much appreciate your being here today, 

especially since you came from Buenos Aires to participate in this 

hearing. I compliment you on the book which you have written, and 

I pay particular note to your references to stem cells as they relate 

to your situation. 

We now begin the customary 5-minute rounds for the witnesses 

and we start with Mr. Anthony Herrera. 

Mr. HERRERA. Thank you. 

In January 1997 at New York Hospital, I was diagnosed with 

mantle cell lymphoma and was told this disease will kill you. There 

is nothing we can do. You are going to die. 

Then without anesthesia, this oncologist drilled through my skin, 

through my periosteum and into the bone, and extracted marrow. 

The pain was incredible. 

That night I debated whether to put my .38 Smith & Wesson to 

my temple and pull the trigger or saddle up. I pondered each option. Then I pictured myself on a horse riding into a dark canyon. 

I found a poem by Tennessee Williams from the Night of the Iguana. I read it every day. 
6 

I then went to Sloan Kettering where I was told we are going to 

work hard and hope for the best. They had a new protocol for mantle cell developed with a hospital in Paris. I was the fifth patient 

in the United States to undergo this regimen, massive amounts of 

chemotherapy and total body irradiation to kill lymphoma cells and 

take my immune system to zero. 

On August 1, 1997, I received an autologous stem cell transplant, 

autologous meaning the stem cells were taken from my body. My 

mouth was full of sores. My skin was gray. I had no hair, no fingernails, no toenails, but I was found to be in remission. I lived under 

the belief that if the disease came back that I would die. 

In November 1998, I relapsed but during these 18 months, a new 

approach to the stem cell transplant for mantle cell had been developed at M.D. Anderson Cancer Center in Houston, Texas where I 

was admitted March 30, 1999. 

In April, I underwent an allogeneic stem cell transplant using a 

non-myeloablative regimen, allogeneic meaning the cells came from 

a donor, non-myeloablative meaning they did not burn my immune 

system to zero with chemo, hence less toxicity. 

Six weeks later, the lymphoma was still active. We tried a donor 

lymphocyte infusion, adding more of my brother’s cells. I then suffered from CMV. I bled internally and lost 30 pounds in 3 weeks, 

followed by a mild stroke and a seizure, but I was in remission. 

One year later, August 15, 2000, the CT-scan showed that I had 

relapsed. The disease was back. I was told without treatment you 

will die in less than 12 months and that another donor lymphocyte 

infusion could kill you. There was a small amount of disease, so I 

had time to think. 

Six weeks later, I saddled up and requested a CT-scan. At this 

juncture, medical history was made. This scan showed less disease 

than 6 weeks before, which meant that my new immune system 

had started fighting the lymphoma without chemotherapy, without 

drugs, without radiation. My new immune system was taking out 

the cancer, my new immune system and my bone marrow created 

by donor stem cells. 

Throughout this journey, I listened to Willie Nelson, Louis Armstrong, and Agustin Lara of Mexico. I recited Tennessee Williams 

every day. I quoted from John Ford’s The Searchers. When asked 

if he wanted to quit, John Wayne retorted, that will be the day. 

I found dedicated and inspired doctors and nurses, such as Sergio 

Giralt and Joyce Newman, doctors and nurses with guts and vision. 

In 1950, William Faulkner won the Nobel Peace Prize for literature. He concluded his acceptance speech with the following. ‘‘I 

believe that man will not merely endure: he will prevail. He is immortal, not because he alone among creatures has an inexhaustible 

voice, but because he has a soul, a spirit capable of compassion and 

sacrifice. 

‘‘The writer’s duty is to write about these things. It is his privilege to help man endure by lifting his heart, by reminding him of 

the courage and honor and hope and pride and compassion and 

pity and sacrifice which have been the glory of his past. The poet’s 

voice need not merely be the record of man, it can be one of the 

props, the pillars to help him endure and prevail.’’ 
7 

I hope that you Senators and this Congress find it is your privilege and your duty to fight with your intelligence and pride and 

compassion to continue to build the pillars of man, the arts for the 

spirit, education for the mind, and medical research for the body. 

PREPARED STATEMENT

Ladies and gentlemen, let me leave you with this thought. The 

stem cell is the future of medicine and I am alive because of the 

progress in stem cell research. Thank you. 

[The statement follows:] 

PREPARED STATEMENT OF ANTHONY HERRERA

I was diagnosed with Mantle Cell Lymphoma in January of 1997 and was told 

at New York University Hospital, ‘‘This disease will kill you. There is nothing we 

can do. You are going to die.’’ 

That night I debated whether to put my .38 Smith & Wesson to my temple and 

pull the trigger or ‘‘saddle up.’’ I pondered each option. Then I saw myself on 

HORSE heading into a dark canyon. 

I found a poem by Tennessee Williams from the Night of the Iguana. I read it 

every day. 

I then went to Memorial Sloan-Kettering and was treated with a regimen of chop 

and ICE chemotherapy and total body irradiation. 

On August 1, 1997 I received an autologous stem cell transplant and was found 

to be in remission. 

In November 1998, I relapsed and received four cycles of chemotherapy. On March 

30, 1999 I was admitted to the University of Texas, M.D. Anderson Cancer Center 

in Houston Texas, under the care of Dr. Issa Khouri, M.D. 

I underwent an allogeneic stem cell transplantation using a non-myeloablative 

regimen. My brother John, was my donor. I required a boost of donor lymphocyte 

infusion after transplantation. 

I then suffered from CMV, a mild stroke and a seizure. 

I was found to be in remission August 15, 1999. 

This treatment was based on a concept developed at M.D. Anderson Cancer Center, that many neoplastic diseases can be treated by immune modulation only without the need for toxic high dose chemotherapy. 

Up until recently high dose chemotherapy was considered essential for marrow or 

stem cell transplantation. 

This new treatment offers new hope and new horizons for patients suffering from 

this otherwise fatal disease. 

I relapsed August 15, 2000. I was told without treatment ‘‘You will die in less 

than twelve months.’’ And that ‘‘another donor lymphocyte infusion—could kill you.’’ 

He then worked with Dr. Ira Braunschweig, formerly of MD Anderson Cancer 

Center, now medical director of Director of Bone Marrow Transplantation—The Albert Einstein College of Medicine. The plan at that time was to use Rituxan to control the lymphoma and then return to MD Anderson for a donor lymphocyte infusion. 

A CAT-scan from September 27, 2000 showed less disease without any treatment 

of any kind. This meant that his new immune system had started battling the disease. 

This was a medical history in the treatment of Mantle Cell Lymphoma in that 

the new immune system had started killing lymphoma cells and there by reducing 

the amount of disease without treatment of any kind. 

Dr. Braunschweig and I debated and then decided to proceed with four rounds 

of high dose Rituxan during the month of October, with the hope that the Rituxan 

would assist his new immune system in the battle. 

CAT scans and Gallium scans that followed from November through 30 January 

2001 showed a steady decrease in the amount of lymphoma and lymphoma related 

activity. 

Dr. Braunschweig and I have discussed several times whether there was a chance 

the rituxan aided his new immune system in the battle to control the Mantle Cell 

Lymphoma. 

We will never know. 
8 

What is concluded by Dr. Braunschweig, Dr. Andre Goy, Dr. James Gajewski and 

Dr. Sergio Giralt is that the donor infusion of my brother’s cells and the engrafting 

of this new immune system in his body that has kept me in remission for five years. 

This unexpected development of Graft vs. Lymphoma approach is positive news 

for fighting cancer and other life threatening diseases. 

Throughout this journey I listen to Willie Nelson, Louis Armstrong and Agustin 

Lara of Mexico. I quoted Tennessee Williams every day. I quoted from John Ford’s 

THE SEARCHERS... When was asked if he wanted to quit. John Wayne retorted, ‘‘That’ll be the day.’’ 

I am alive because of great Doctors and nurses with guts and vision. 1950 William 

Faulkner won the Nobel Prize for Literature . . . he concluded his speech with the 

following. 

I believe that man will not merely endure: he will prevail. He is immortal, not 

because he alone among creatures has an inexhaustible voice, but because he has 

a soul, a spirit capable of compassion and sacrifice and endurance. 

The poet’s, the writer’s, duty is to write about these things. It is his privilege to 

help man endure by lifting his heart, by reminding him of the courage and honor 

and hope and pride and compassion and pity and sacrifice which have been the 

glory of his past. The poet’s voice need not merely be the record of man, it can be 

one of the props, the pillars to help him endure and prevail . . . 

The stem cell is the future of medicine . . . 

I hope you senators and this congress find that it is your privilege and duty to 

fight with your intelligence and pride and compassion to continue to build the pillars of man—the arts for the spirit—education for the mind and medical research 

for the body. Stem cell research. All stem cell research. 

Thank you. 

Senator SPECTER. Thank you very much, Mr. Herrera, for that 

very poignant and emphatic testimony and for the authentication 

as to what stem cells can do, for what they have done for you. 

Our next witness is Dr. Judith Gasson, Director of Jonsson Comprehensive Cancer Center at UCLA. Dr. Gasson has a doctorate in 

physiology from the University of Colorado and post-doctoral work 

at Saulk Institute. Thank you very much for joining us today, Dr. 

Gasson, and the floor is yours. 

STATEMENT OF JUDITH GASSON, Ph.D., DIRECTOR, JONSSON COMPREHENSIVE CANCER CENTER 

Dr. GASSON. Thank you very much, Mr. Chairman. It is a great 

pleasure to continue the conversation that you and I began several 

years ago when you were visiting UCLA Medical School. At that 

time, we had a very serious discussion about how important it was 

that we continue to do this very important stem cell work, and I 

am happy to be here today. 

Cancer is now the leading cause of death in Americans under the 

age of 85. This year alone, 550,000 Americans will die from their 

disease. These numbers fail to account for the additional pain and 

suffering felt by their families and friends. 

Many scientists believe that stem cell research has the power to 

revolutionize cancer therapy in much the same way that targeted 

therapies have impacted cancer treatment over the past decade. 

There is now considerable evidence that many types of cancer, including breast cancer, prostate, brain, and certain leukemias, arise 

through mutations that occur in our adult stem cells. These socalled cancer stem cells retain the ability to self-renew, which is a 

signature feature of stem cells. However, they lose the ability to respond to the proper cues and to differentiate. 

Our current therapies are targeted to the bulk of the tumor and 

not to the cancer stem cell. How can we develop therapies that destroy the malignant stem cells, thereby eliminating both the tumor 
9 

and its chance to recur at a later time? Like all therapeutic advances, targeting cancer stem cells must be based on outstanding 

basic science. For this reason, embryonic stem cells must be studied to educate us on the fundamental processes and pathways that 

drive the growth of cancer stem cells. 

To be sure, studies are ongoing on adult stem cells, but these 

studies are incomplete and unable to answer all of the critical 

questions. Adult stem cells are rare in our bodies and cannot be induced to grow in the laboratory without also differentiating. 

We believe that characterizing the pathways that embryonic 

stem cells use to self-renew, using high-throughput screening technology, will allow us to develop small molecule inhibitors to those 

stem cell-specific pathways. If these chemical inhibitors of self-renewal of embryonic stem cells are isolated and characterized in the 

laboratory, they may actually provide the first benefit of stem cell 

research in patients. 

Paradoxically, as you just heard from Mr. Herrera, bone marrow 

stem cells are not only perhaps the source of some cancers, but 

they also have been used to treat certain cancers for the past 4 decades. Many patients are unable to benefit from this potentially lifesaving treatment because they either do not have a matched bone 

marrow donor or their own bone marrow has been compromised by 

treatment or invasion of cancer cells. The technique of somatic cell 

nuclear transfer would enable us to insert the DNA from a cancer 

patient’s skin cells into an egg and reprogram that DNA to become 

a pluripotent stem cell again. In this way, the patient’s blood and 

immune systems could be reconstituted and genetically identical to 

the patient. 

It has been estimated that there are currently 400,000 frozen 

embryos generated in in vitro fertilization clinics that will not be 

used. The vast majority of these frozen cells will be destroyed. 

The thousands of physicians and scientists, represented by the 

American Association of Cancer Research and the American Society 

of Clinical Oncology, issued public statements this year strongly 

endorsing the expansion of funding for embryonic stem cell research to improve the prevention, detection, and treatment of cancer. We estimate that this represents 30,000 physicians and scientists who believe that this important work will have an impact 

on the dreaded disease of cancer. 

PREPARED STATEMENT

To be sure, my commitment to this area of research is professional, but it is also personal. Three years ago next week I lost my 

own father to lymphoma. 

Thank you very much, Mr. Chairman. 

[The statement follows:] 

PREPARED STATEMENT OF JUDITH GASSON

Cancer is now the leading cause of death in Americans under the age of 75. This 

year alone 550,000 Americans will die from their disease. These numbers fail to account for the additional pain and suffering felt by their family and friends. 

Many scientists believe that stem cell research has the power to revolutionize cancer therapy in much the same way that ‘‘targeted’’ therapies have impacted cancer 

treatment over the past decade. There is now considerable evidence that many types 

of cancer including breast, prostate, brain and leukemias arise through mutations 
10 

acquired in our adult stem cells. These so-called ‘‘cancer stem cells’’ retain the ability to self-renew, which is the signature feature of stem cells. However they lose the 

ability to respond to normal differentiation signals. 

Our current therapies are targeted to the bulk of the tumor, but not to the cancer 

stem cells. How can we develop therapies that destroy the cancer stem cells, thereby 

eliminating the tumor and its chances to recur? Like all therapeutic advances targeting cancer stem cells must be based upon outstanding basic science. For this reason embryonic stem cells must be studied to educate us on the fundamental processes and pathways that drive the growth of cancer stem cells. To be sure studies 

are ongoing with adult stem cells, but these studies are incomplete and unable to 

answer all of the critical questions. Adult stem cells are rare and cannot be induced 

to grow in the laboratory without also differentiating. We believe that characterizing 

the pathways that embryonic stem cells use to self-renew, using high-throughput 

screens, will lead to the development of small molecule inhibitors. It is these chemical inhibitors of self-renewal of embryonic stem cells that may provide the first benefits of stem cell research in patients. 

Paradoxically bone marrow stem cells have been used to treat certain cancers for 

the past four decades. Many patients are unable to benefit from this potentially lifesaving treatment because they don’t have a matched bone marrow donor and their 

own bone marrow has been comprised by treatment or invaded by cancer cells. The 

technique of somatic cell nuclear transfer would enable us to insert DNA from a 

cancer patient’s skin cell into an egg and re-program it from a skin cell to a 

pluripotent stem cell. In this way, the patient’s blood and immune systems could 

be reconstituted and genetically identical to the patient. 

It’s been estimated that there are currently 400,000 frozen embryos generated 

from in vitro fertilization that will not be used. The vast majority of these will be 

destroyed. The thousands of physicians and scientists represented by the American 

Association of Cancer Research and the American Society of Clinical Oncology 

issued public statements this year strongly endorsing the expansion of funding for 

embryonic stem cell research to improve the prevention, detection and treatment of 

cancer. 

Senator SPECTER. Thank you very much, Dr. Gasson. 

Our next witness is Dr. Rudolf Jaenisch, Professor of Biology at 

Massachusetts Institute of Technology and a member of the Whitehead Institute for Biomedical Research. He received his doctorate 

in medicine from the University of Munich. Thank you for joining 

us today, Dr. Jaenisch, and we look forward to your testimony. 

STATEMENT OF RUDOLF JAENISCH, M.D., PROFESSOR OF BIOLOGY, 

MASSACHUSETTS INSTITUTE OF TECHNOLOGY 

Dr. JAENISCH. Thank you, Mr. Chairman. 

So I am a founding member of the Whitehead Institute and a 

professor of biology at MIT. My main research interest is epigenetic 

regulation, embryonic stem cells, and to understand the mechanisms of nuclear transfer and the reprogramming of the genome 

following nuclear transfer. We have studied this in mice, and the 

conclusion from all work was that reproductive cloning in humans 

is unsafe and should be banned. 

Our work was also of relevance for the therapeutic application of 

somatic cell nuclear transfer. We have done this in a mouse model 

again of severe combined immune deficiency, SCID. This condition 

exists in humans. And we have used this technique to restore the 

immune system in these mice. And I believe that this proof of principle experiment is directly relevant for treatment of human blood 

diseases, such as leukemia as we heard. 

The recent success by the Korean group indicates that nuclear 

transfer in humans is much more efficient than we assumed before, 

and they believe the treatment of bone marrow diseases will likely 

be the first human disease that will be treated by SCNT. 
11 

Embryonic stem cells clearly—and we heard this—are of great 

potential value to treat diseases, and I am confident that if we are 

allowed to derive new stem cells from in vitro fertilized embryos, 

that would enormously help us to understand the system. But I 

want to talk about nuclear transfer today. 

The proof of principle experiments are clear. In principle, this 

technology will work in humans to treat diseases such as blood diseases, Parkinson’s, and diabetes. We have to learn technology, but 

this I think is only technology. 

So what are the concerns of those who oppose nuclear cloning in 

humans for the purpose of generating customized embryonic stem 

cells for therapy or for research? 

I believe the key concern is that the derivation of an embryonic 

stem cell from a cloned construct would necessarily involve the destruction of the blastocyst and thus destruction of potential normal 

human life. The crucial question is: does the cloned blastocyst really represent potential normal human life? And that is what I want 

to concentrate on. 

From all experience with cloned animals, I would argue that the 

cloned blastocyst has little, if any, potential to ever develop into a 

normal baby. Most will die in development and the few that survive to birth will develop severe defects with age because of the reprogramming faults following nuclear transplantation. 

For these reasons, it has been suggested, because a cloned blastocyst is so different from the normal blastocyst which is derived 

from a fertilized egg, that it should not be designated as an embryo. And I agree with this notion. However, we have to admit that 

the cloned blastocyst has a chance, although an exceedingly small 

chance, to develop into cloned animals such as Dolly. But Dolly 

died because she suffered from major ailments, as most clones do. 

But it is this statistically small chance of a clone to develop to birth 

and beyond what troubles most who oppose the technology. 

The altered nuclear transfer approach has been proposed by Dr. 

Hurlbut as a potential solution. This approach would cause the 

product of nuclear transfer to be inherently unable to ever develop 

into a fetus or a baby because of its inability to establish the very 

first step of embryonic organization and the inability to establish 

that fetal/maternal connection. The procedure, as proposed by 

Hurlbut, involves the genetic manipulation of the donor cell, not of 

the embryo, with the goal to generate a construct which can still 

generate embryonic stem cells but cannot implant and generate a 

fetus. So the goal is, therefore, to generate what he calls a biological construct or biological artifact that lacks the essential attributes of an embryo and has no potential whatsoever to develop 

into a fetus but still could proliferate and give rise to ES cells. 

ANT, altered nuclear transfer, was last year proposed as a 

thought experiment. We have now performed the proof of principle 

experiment in the mouse, published this week in Nature, that validates this proposal. So let me explain. 

In our experiment, we introduced an RNAi construct into the 

skin cells prior to nuclear transfer. The RNAi was directed against 

Cdx2. This is a gene which is crucial for the establishment of the 

very first lineage in embryonic development which is established at 

the 16-cell stage. The genetically altered skin cells do not express 
12 

Cdx2, but once the nucleus is transferred to the egg, the cloned 

product cannot establish this key lineage. It will develop still to an 

abnormal blastocyst which collapses because the trophectoderm lineage, which will give rise to the placenta, cannot form. 

The embryonic stem cells derived from this construct are indistinguishable in their potential from a normal embryonic stem cell. 

So the key question for the debate here is: does it generate embryos 

and how abnormal are they? 

So I would argue that the ANT, altered nuclear transfer, embryo 

is already abnormal at the 4- to 8-cell stage molecularly because 

the gene is then expressed. It is not expressed then. But it becomes 

morphologically only abnormal within 2 cell divisions. 

Senator SPECTER. Dr. Jaenisch, could you summarize your testimony at this point? Your full statement will be made a part of the 

record. 

Dr. JAENISCH. So I will then summarize that the question is can 

we designate these ANT embryos as normal, these ANT blastocysts 

as normal embryos. And I would think they are a mass of differentiating cells, but they definitely lack the intricate organization of 

the embryo and its potential. 

PREPARED STATEMENT

I want to emphasize that ANT is a modification, not an alternative, to nuclear transplantation. It requires additional manipulation of the donor cells that may complicate the logistics of an already complex procedure, and this has concerned many scientists. 

However, our procedure has shown that the procedure is so simple 

and straightforward that it may be acceptable as a requirement if 

it would resolve the ethical objections against somatic cell nuclear 

transfer and allow this research to go ahead. 

[The statement follows:] 

PREPARED STATEMENT OF DR. RUDOLF JAENISCH

Mr. Chairman and members of the Subcommittee, my name is Rudolf Jaenisch. 

I am a founding Member of the Whitehead Institute and Professor of Biology at 

MIT. Before coming to the Whitehead Institute I was the head of the Department 

of Tumor Virology at the Heinrich-Pette Institute of the University of Hamburg in 

Germany. I am privileged to have helped establish the field of transgenic science. 

Transgenic science deals with the transfer of genes to create mouse models of 

human disease. My present research focuses on epigenetic gene regulation, on embryonic stem cells, and on nuclear cloning. Our focus is understanding the mechanisms that bring about reprogramming of a somatic nucleus to an embryonic one 

after its transfer into the egg. I work with mice and our results have demonstrated 

that nuclear cloning is inefficient, that most clones die at an early embryonic stage 

and that the few that survive to birth and beyond harbor serious defects and are 

not normal. The conclusion from this work is that reproductive cloning of humans 

is an unsafe technology that should be banned. 

Our work has shown that somatic cell nuclear transfer (SCNT) can generate ‘‘customized’’ embryonic stem cells that can be used for the treatment of genetic diseases. We have performed a ‘‘proof of principle’’ experiment in mice that carry a specific mutation which causes a defective immune system. Human patients with a corresponding mutation (designated as Severe Combined Immune Deficiency or SCID) 

are unable to fight infections and have a grim prognosis. In our proof of principle 

experiment the nuclei of SCID mouse skin cells were transplanted into enucleated 

eggs to generate cloned blastocysts (NT-blastocysts) that were then placed into tissue culture to derive ‘‘customized’’ cloned embryonic stem cells (NT-ES cells). The 

genetic mutation was corrected by gene targeting, the ‘‘repaired’’ NT-ES cells were 

then induced to differentiate into blood stem cells and, when transplanted back into 

the mutant mouse, restored immune function. I believe that this proof of principle 
13 

experiment is directly relevant for the treatment of human blood diseases such as 

thalassemia, sickle cell anemia or leukemia. The recent success by the Korean group 

(Hwang et al.) indicates that nuclear transfer in humans is more efficient than was 

assumed before and I believe that treatment of bone marrow diseases will likely be 

one of the first human diseases that will be treated with SCNT. 

Embryonic stem cells have an enormous potential for therapy of debilitating diseases such as cancer, diabetes, Parkinson’s or other degenerative diseases. To realize this therapeutic potential much research is needed to learn how to differentiate 

the embryonic cells into cells used for transplantation. I am confident that the possibility to derive new ES cell lines from IVF embryos as debated in Congress would 

enormously help this research. 

I will focus on nuclear transfer (NT). In addition to its potential for customized 

therapy, nuclear transfer derived ES cells would be an extraordinary important tool 

to study complex diseases such as ALS or Alzheimers in the test tube since ‘‘customized’’ ES cells derived from a patient would carry all the genetic alterations that 

caused the disease in the patient. The exciting prospect is that differentiation of the 

ES cells in the culture dish may provide clues to what goes wrong with the cells 

and how to establish therapies. This is not a future promise but this could be done 

today using the technology established by the Korean group that was the first to 

successfully derive human stem cells from cloned blastocysts. 

What are the concerns of those who oppose nuclear cloning in humans for the purpose of generating ‘‘customized’’ embryonic stem cells for research or for therapy? 

I believe the key concern is that the derivation of an embryonic stem cell would necessarily involve the destruction of the blastocyst and thus the destruction of potential human life. The crucial question is: does the cloned blastocyst really represent 

potential normal human life? 

From all experience with cloned animals I would argue that the cloned blastocyst 

has little if any potential to develop into a normal baby as most would die in development and the few that survive will be abnormal and will develop severe defects 

with age. This is because reprogramming of the somatic cell’s genome after nuclear 

transplantation is a faulty process causing the great majority of clones to have hundreds of genes incorrectly expressed. For these reasons it has been suggested that, 

because the cloned blastocyst is so different from the normal blastocyst derived from 

a fertilized egg, it should not be designated as an ‘‘embryo’’—and I agree with this 

notion. However, the cloned blastocyst has some chance, an exceedingly small 

chance, to ever develop into a cloned animal such as Dolly. And Dolly died early 

because she suffered from major ailments due to faulty reprogramming as most if 

not all cloned animals do. It is this statistically small chance of a clone to develop 

to birth and beyond that troubles, I believe, those who are opposed to the NT technology. 

The Altered Nuclear Transfer (ANT) approach has been proposed by Dr. Hurlbut 

from Stanford as a potential solution for the ethical dilemma. This approach would 

cause the product of nuclear transfer to be inherently unable to ever develop into 

a fetus or a baby, because of its inability to establish the very first step of embryonic 

organization and its inability to establish a fetal-maternal connection. With other 

words, the ANT procedure would reduce the statistically low chance of an NT blastocyst to develop to birth to zero. The procedure, as proposed by Hurlbut, involves 

the genetic manipulation of the donor skin cell with the goal to inactivate a gene 

that is required for embryo development if the nucleus of the manipulated cell 

would be transplanted into an enucleated egg as in SCNT. The manipulation would, 

however, have no ill effect on the derivation of embryonic stem cells from the product of SCNT. Thus, the alteration causes the somatic nucleus to function in such 

a way that no embryo is generated but embryonic stem cells can be produced. The 

goal of ANT is to generate a nuclear transfer product that lacks the essential attributes of an embryo and has no potential whatsoever to develop into a fetus but 

still could proliferate and give rise to embryonic stem cells. ANT was suggested last 

year as a thought experiment. We have now performed a proof of principle experiment in the mouse (published this week in Nature) that validates the proposal. 

In our experiment an RNAi construct that inactivates the Cdx2 gene was introduced into skin cells. Cdx2 has a crucial function in the establishment of the first 

embryonic lineage, the trophectoderm that is established at the 16-cell stage and 

forms the placenta of the embryo. Skin cells normally do not express the Cdx2 gene. 

But when used as donors for nuclear transplantation, the ANT product is unable 

to activate the gene and therefore unable to establish the trophectoderm lineage. 

However, the product of nuclear transfer did proliferate and formed an abnormal 

NT-blastocyst. The normal blastocyst consists of the inner cell mass (which will form 

the embryo proper) and a cavity which is surrounded by trophectoderm cells (which 

will form part of the placenta). In contrast to the normal embryo, the ANT blasto-
14 

cyst collapses because the trophectoderm cells are lacking. Importantly, when placed 

into tissue culture, the ANT blastocyst generates embryonic stem cells that have the 

full potential for differentiation and therapy and thus are indistinguishable from 

embryonic stem cells that are derived from a fertilized embryo. 

Does the ANT procedure generate ‘‘embryos’’, even if only abnormal ones? Our experiments clearly show that the Cdx2 deficient blastocyst has no potential to implant and to ever develop into a fetus because it lacks the trophectoderm lineage 

that gives rise to the placenta. Cdx2 is activated at the 8-cell stage and activation 

of this key gene is prevented in the ANT product. Thus, the product of ANT-SCNT 

is already molecularly different from the normal embryo at the 8-cell stage and becomes morphologically abnormal within the next two cell divisions. The placenta is 

an integral part of the embryo and not some component that could be separated 

from the embryo. It is like the engine of a car: one cannot separate the engine from 

the car and still call it a car. Because the ANT product lacks essential properties 

of the fertilized embryo, it is not justified to call it an ‘‘embryo’’. 

It is important to emphasize that ANT is not an alternative to nuclear transplantation but a modification of an experimentally highly demanding process. It requires 

additional manipulations of the donor cells that will complicate the logistics of an 

already complex procedure, and this has raised concerns among many scientists. 

Also, it has not been determined whether Cdx2 has a similar function on human 

placentation as in mouse. Because the effect of gene inhibition on human placentation cannot be directly tested, surrogate assays such as in vitro differentiation of 

human ES cells are required to assess the effect of CDX2 deficiency on human placental development. The experiments in mice have shown a proof of concept of the 

ANT procedure. It would be unfortunate, however, if the implementation of this approach would delay the research on human SCNT. 

Senator SPECTER. Thank you very much, Dr. Jaenisch. 

Our next witness is Dr. Steven Teitelbaum, Professor of Pathology at Washington University School of Medicine, an M.D. from 

Washington University, residency at New York University. Thank 

you very much for coming in today, Dr. Teitelbaum, and we are interested in hearing your testimony. 

STATEMENT OF STEVEN TEITELBAUM, M.D., WILMA AND ROSWELL 

MESSING, PROFESSOR OF PATHOLOGY AND IMMUNOLOGY, 

WASHINGTON UNIVERSITY SCHOOL OF MEDICINE 

Dr. TEITELBAUM. Mr. Chairman, I thank the committee for the 

honor of speaking to you today. 

Mr. Chairman, I have been a physician scientist for more than 

30 years. I have authored in excess of 300 scientific papers, and I 

am here to tell you that in my estimation we are facing a unique 

opportunity in the form of embryonic stem cell research to potentially alleviate the misery of our fellow Americans with a number 

of presently incurable diseases. But to get there, we must do the 

science. 

Opponents of embryonic stem cell research often articulate their 

position as a contest between adult and embryonic stem cells. Mr. 

Chairman, this is not a contest between various types of stem cells. 

It is a contest between us as a society and disease. We should be 

moving forward on all fronts, adult, embryonic, and umbilical cord 

stem cells to win the battle. The tool is not important. What counts 

is curing our neighbors. 

That said, because of their flexibility, embryonic stem cells hold 

more promise to ameliorate presently incurable diseases than any 

other approach. I stress the word ‘‘promise’’ because we are not 

there yet, and it is my belief that it will be some time before we 

are positioned to safely use these cells for therapy. But if scientists 

are prevented from exploring the biology of human embryonic stem 

cells, we will never get there. 
15 

Mr. Chairman, as you know, human embryonic stem cells can 

presently be obtained from two sources; namely, the spare products 

of in vitro fertilization, which ultimately would be destroyed, and 

by somatic cell nuclear transfer, also known as SCNT or therapeutic cloning. Although both approaches hold enormous therapeutic potential, I am particularly taken with the promise of SCNT 

because it may alleviate the major complication of tissue and cell 

transplantation, namely rejection and its attendant life-threatening 

consequences. 

Mr. Chairman, I am a bone biologist and physician, and as such, 

I see many patients who have received organ and cell transplants. 

These patients typically develop severe osteoporosis and often have 

many fractures because of the harsh medications they must take 

to prevent rejection of their transplant. It is my hope that embryonic stem cells, generated by SCNT, which contain the transplant 

recipient’s own DNA will reduce the necessity for these devastating 

anti-rejection drugs. 

But, Mr. Chairman, my hopes for SCNT are more personal and 

harken back more than 20 years when I was a young assistant professor. At that time, I became interested in a genetic disease of the 

skeleton known as osteopetrosis, or marble bone disease, and I 

want to tell you a story about a child who profoundly impacted my 

life. 

Osteopetrosis is a disease in which kids make too much bone. 

Consequently, their skulls become very thick and compress their 

brains and nerves, such as those leading to the eye. Bone also overgrows the bone marrow, preventing formation of blood cells. Until 

the story I am about to tell you, all kids with the malignant form 

of osteopetrosis developed fatal neurological complications, including blindness, and infections due to bone marrow suppression. 

These children invariably died in the first decade, most before the 

age of 5. 

In the early 1980’s, our team thought we had identified the abnormal cell causing osteopetrosis and concluded it resided in the 

bone marrow. We reasoned, therefore, that if we gave an 

osteopetrotic infant a bone marrow transplant which contains adult 

stem cells, we might cure the disease. We realized the enormous 

risk of rejection, so we waited until we had a perfect immunological 

match between the donor and recipient, in this instance the 3- 

month-old little girl you see in the top picture. So we gave this 

baby a bone marrow transplant and achieved the first cure of this 

disease. The middle panel shows her at 3 years of age, and the bottom picture, which is recent, was taken upon her graduation from 

college. Senators, being part of a team which was first to cure a 

fatal disease, particularly that of children, is a doctor’s dream. It 

does not get any better. 

You may be asking yourselves why this guy, who is here as an 

advocate of embryonic stem cell research, is telling us about his 

victory with adult stem cells. Senators, I am recounting the story 

to underscore the importance of moving forward on all fronts because, regrettably, there is a down side to my tale. You will remember that this was a perfect immunologic match, and therefore there 

was little chance of rejection. Unfortunately, such matches are extremely rare and therefore, we presently cure less than 10 percent 
16 

of kids with osteopetrosis. The use of SCNT, in which embryonic 

stem cells contain the patient’s own DNA, if successful, would 

markedly increase the cure rate of this disease. 

PREPARED STATEMENT

Mr. Chairman, because of my familiarity with osteopetrosis, I am 

frequently contacted by parents with afflicted children. I have to 

tell them that the chances of curing your child is no more than 10 

percent. I want to tell them it is greater than 90 percent. SCNT, 

if we pursue it, may get us there. 

Thank you. 

[The statement follows:] 

PREPARED STATEMENT OF DR. STEVEN TEITELBAUM

Thank you Mr. Chairman. My name is Steven Teitelbaum. I’m the Wilma and 

Roswell Messing Professor of Pathology and Immunology at Washington University 

School of Medicine and I thank the committee for the honor of speaking to you 

today. 

Mr. Chairman, I’ve been a physician-scientist for more than 30 years. I’ve authored in excess of 300 scientific papers and I’m here to tell you that, in my estimation, we are facing a unique opportunity in the form of embryonic stem cell research, to potentially alleviate the misery of our fellow Americans with a number 

of presently incurable diseases. But to get there, we must do the science. 

Opponents of human embryonic stem cell research often articulate their position 

as a contest between adult an embryonic stem cells. Mr. Chairman, this is not a 

contest between various types of stem cells. It is a contest between us as a society 

and disease. We should be moving forward on all fronts, adult, embryonic and umbilical cord stem cells, to win the battle. The tool is not important. What counts is 

curing our neighbors. 

That said, because of their flexibility, embryonic stem cells hold more promise to 

ameliorate presently incurable diseases than any other approach. I stress the word 

‘‘promise’’ because we are not there yet and it is my belief that it will be some time 

before we are positioned to safely use these cells for therapy. But if scientists are 

prevented from exploring the biology of human embryonic stem cells, we will never 

get there. 

Mr. Chairman, as you know, human embryonic stem cells can presently be obtained from two sources, namely the spare products of in vitro fertilization, which 

ultimately would be destroyed, and by somatic cell nuclear transfer, also known as 

SCNT or therapeutic cloning. Although both approaches hold enormous therapeutic 

potential, I’m particularly taken with the promise of SCNT because it may alleviate 

the major complication of tissue and cell transplantation, namely rejection and its 

attendant life threatening consequences. 

Mr. Chairman, I’m a bone biologist and physician and as such I see many patients 

who have received organ and cell transplants. These patients typically develop severe osteoporosis and often have many fractures because of the harsh medications 

they must take to prevent rejection of their transplant. It is my hope that embryonic 

stem cells, generated by SCNT, which contain the transplant recipient’s own DNA, 

will reduce the necessity for these devastating anti-rejection drugs. 

But Mr. Chairman, my hopes for SCNT are more personal and hearken back more 

than 20 years when I was a young assistant professor. At that time I became interested in a genetic disease of the skeleton known as osteopetrosis or marble bone disease and I want to tell you a story about an afflicted child who profoundly impacted 

my life. Osteopetrosis is a disease in which kids make too much bone. Consequently, 

their skulls become very thick and compress their brains and nerves, such as those 

leading to the eye. Bone also overgrows the bone marrow preventing formation of 

blood cells. Until the story I’m about to tell you, all kids with the malignant form 

of osteopetrosis developed fatal neurological complications, including blindness, and 

infections due to bone marrow suppression. These children invariably died in the 

first decade, most before the age of five. 

In the early 80s, our team thought we had identified the abnormal cell causing 

osteopetrosis and concluded it resided in the bone marrow. We reasoned, therefore, 

that if we gave an osteopetrotic infant a bone marrow transplant, which contains 

adult stem cells, we might cure the disease. We realized the enormous risk of rejection so we waited until we had a perfect immunological match between the donor 
17 

and recipient, in this case a 3 month old little girl you see in the top picture. So 

we gave this baby a bone marrow transplant and achieved the first cure of this disease. The middle panel shows her at 3 years of age and the bottom picture, which 

is recent, was taken upon her graduation from college. Senators, being part of a 

team which is first to cure a fatal disease, particularly of children, is a doctor’s 

dream. It doesn’t get any better. 

You may be asking yourselves why this guy, who is here as an advocate of embryonic stem cell research, is telling us about his victory with adult stem cells. Senators, I’m recounting the story to underscore the importance of moving forward on 

all fronts because regrettably there is a downside to my tale. You’ll remember that 

this was a perfect immunological match and therefore there was little chance of rejection. Unfortunately, such matches are extremely rare and therefore we presently 

cure less than 10 percent of kids with osteopetrosis. The use of SCNT, in which embryonic stem cells contain the patient’s own DNA, if successful, would markedly increase the cure rate of this disease. Mr. Chairman, because of my familiarity with 

osteopetrosis I’m frequently contacted by parents with afflicted children. I have to 

tell them the chances of curing your child is no more than 10 percent. I want to 

tell them it’s greater than 90 percent. SCNT, if we pursue it, may get us there. 

Senator SPECTER. Thank you very much, Dr. Teitelbaum. 

Our final witness is Dr. John Wagner, Professor of Pediatrics and 

Scientific Director of the Stem Cell Institute at the University of 

Minnesota. An M.D. at Jefferson Medical College in Philadelphia 

and internship and residency at Duke University School of Medicine. Thank you for coming to Washington today, Dr. Wagner, and 

we look forward to your testimony. 

STATEMENT OF JOHN WAGNER, M.D., SCIENTIFIC DIRECTOR OF CLINICAL RESEARCH, BLOOD AND MARROW TRANSPLANT PROGRAM 

AND STEM CELL INSTITUTE 

Dr. WAGNER. Mr. Chairman and committee members, I am coming here as a clinician, as a stem cell researcher. It is not a question of whether or not this knowledge is going to be translated into 

something clinically useful. The real question is, when is that going 

to happen? 

The work should not be restricted to private industry. Stem cell 

research should be taking place in academic institutions, supported 

by Federal dollars with guaranteed oversight, peer review, and 

transparency. 

Right now, as we have heard already in testimony this morning, 

there is only one proven use of stem cells and that is in the context 

of blood and marrow transplantation to treat diseases like leukemia, lymphoma, sickle cell disease, and a variety of other blood 

and immune disorders. In these instances, we need to infuse stem 

cells to repair the marrow that has been destroyed either by the 

disease itself or by the therapy we use to treat that disease, such 

as with chemotherapy and irradiation. These blood-producing stem 

cells are found in marrow and they are found in cord blood, which 

is the blood that is left in the placenta after a baby is born. 

Tremendous achievements have already been made in these 

areas, particularly in the area of cord blood most recently, and in 

fact, the Institute of Medicine last April made recommendations 

that we significantly augment the Nation’s inventory of cord blood 

to help take care of our patients around the country. 

While my own work is focused on the development of stem cell 

therapies from cord blood or adult tissues and, perhaps surprisingly, not embryonic stem cells, I am here today really to defend 

ES cell work. It must be unequivocally clear that our work in cord 

blood and adult stem cells does not eliminate the need for work in 
18 

ES cells. Yes, it is true that stem cells and cord blood and adult 

tissues can differentiate into perhaps the lining cells of the gut or 

the liver or neural tissue, but they do not exhibit all the capacities 

of ES cells. For example, we have yet to see stem cells from cord 

blood or adult tissues differentiate into heart muscle that spontaneously beats in the petri dish. That has been shown repetitively by 

people working on ES cells. 

The University of Minnesota is well known for its work in adult 

stem cells in umbilical cord blood, and with Catherine Verfaille, we 

have pioneered that work in cord blood and multipotent adult stem 

cells and we see great promise in those areas. But we recognize, 

although there is tremendous potential, there are also limitations. 

It is critical that you also know that every discovery that has occurred with ES cells has really benefitted us working on adult stem 

cells and cord blood. 

But speaking as a clinician who creates these new stem cell 

therapies for treating children and adults with a variety of ‘‘incurable’’ diseases, it not only gives us significant hope, but it also 

comes with risk. This winter we hope to be able to try our first 

stem cell transplants in the treatment of patients damaged by 

chemotherapy and irradiation, not just for bone marrow recovery, 

but also the other tissues that are involved in the treatment and 

damaged by it. We have to go through the ethics committees, we 

have to go through human subjects committee, and the FDA. But 

we are going to move this therapy forward, obvious, with all the 

proper oversight. 

But it is incomprehensible that we do otherwise, that we restrict 

ourselves to one type of stem cell. Like others in this room, I feel 

compelled to move this forward on behalf of the thousands of patients that write to me every week asking to allow them to be the 

first stem cell recipient. In fact, this is just one e-mail that I received yesterday from a woman who is 39 years old saying, I had 

a stroke several years ago. What can you do for me? Let me be the 

first. Why can I not be a healthy wife to my husband, a mother 

to my young child? 

Is this all hype? Where are the first trials with ES cells? Certainly the lack of funding and restricted access to suitable stem cell 

lines has been a major barrier in our research efforts. We need to 

address those barriers where possible. For example, can we separate reproductive cloning from nuclear transfer? If you desire rapid 

translation of ES cells into real clinical therapies, let us not restrict 

it. We need to be able to use nuclear transfer because it is likely 

to be instrumental in moving that therapy forward as quickly as 

possible. 

Again, this is not some scientist’s dream. It has been done with 

human cells, at least in South Korea. Every single one of us will 

be faced with a disease amenable to stem cell therapy. It may be 

our child, our spouse, our friend, or even ourselves, and you can 

ask Mr. Herrera and you can ask Ms. Carolyn Kohn, who is in the 

audience, who had a child die of aplastic anemia. 

Cord blood certainly has its proven benefits in the treatment of 

a variety of diseases. It has great potential perhaps in the future 

for tissue repair that yet has clearly to be identified. Federal dol-
19 

lars should be devoted to the work of all these stem cell sources, 

including ES cells. ES cell work must continue in parallel. 

PREPARED STATEMENT

As a clinician that treats these patients who are defined as incurable, I feel obligated to be here on their behalf. I am sure that 

many of them are anxiously waiting to hear what happens today. 

For them, the stakes must be simply unimaginable. 

[The statement follows:] 

PREPARED STATEMENT OF DR. JOHN E. WAGNER

Stem cell therapy will revolutionize the practice of medicine. For the first time 

there will be treatments for spinal cord injury, diabetes, cancer, stroke, and heart 

disease with potentially long term benefits. The proof of principle already exists. 

It is not a question of whether this new knowledge will translate’ into clinical 

therapies but rather how long. Will clinical trials in diabetes or stroke be soon or 

decades away? Will this work be driven by private industry without any oversight 

or in academic environments using federal support; conducted in university settings 

which guarantee requisite oversight, publication, peer review and transparency? 

So what do we know about stem cells today? 

There is only one proven established use of stem cells and that is in the setting 

of bone marrow transplantation. For decades it has been known that marrow stem 

cells can be transplanted from one individual to another in order to replace the 

blood and marrow of patients with leukemia/lymphoma/multiple myeloma/other diseases after their own marrow has been destroyed by disease or treatment with high 

doses of chemotherapy and radiation. These stem cells come from adult marrow or 

umbilical cord blood. 

My own work is focused on umbilical cord blood and development of novel phase 

I clinical trials. In this discussion, we cannot forget that cord blood is already an 

established treatment with tremendous potential. Recently, the Institute of Medicine summarized its findings on the benefits of cord blood and the urgent need to 

expand the useable inventory. Cord blood is rapidly becoming the standard of care 

in children. We have recently reported outcomes in adults with results that are unprecedented. However, it must be clear that cord blood stem cells are not the stem 

cells found in embryonic stem cell lines. The stem cells in adult tissues and umbilical cord blood have different properties and may or may not have unlimited differentiation capacity. While it is hoped that one day we will be able to take adult 

tissue or cord blood stem cells and trick it to become ‘‘ES-like’’, this is not yet possible. Despite what the opponents to ES cell work would suggest, it is simply not 

true. 

The University of Minnesota is well known in the field of stem cell research. We 

have the longest standing Stem Cell Institute in the country. My work in umbilical 

cord blood stem cell research and Catherine Verfaillie’s work on the multipotent 

adult stem cell clearly demonstrate our hope to maximize the potential of cord blood 

and adult tissue stem cells but we recognize that there are limitations. Of course 

we are excited about the future potential of these stem cells but never have we suggested that they obviate the need for ES cell research. For example, never have the 

stem cells from cord blood or adult tissues ever produced heart muscle cells that 

spontaneously beat or formed islets that secrete insulin, as has been shown repeatedly with ES. 

It is critical for the public to know that if we are ever to make cord blood and 

adult tissue stem cells function like ES cells, we need to study ES cells. Every discovery with ES cells has furthered our work with stem cells from umbilical cord 

blood or adult tissues. 

Now speaking as a clinician who actually performs new therapies with stems cells 

in humans, we are indeed planning to perform the first clinical trial with multipotent adult stem cells this winter in an attempt to repair tissues damaged by radiation and chemotherapy. My goal is to move stem cell therapy forward in numerous 

areas as the clinical director of the Stem Cell Institute. Once we meet the requirements of the Human Subjects Committee, FDA, Ethics committees, we plan to move 

stem cell therapies forward regardless of whether they are ES, cord blood or adult 

tissue-derived. It is incomprehensible to do otherwise. Like others, I receive thousands of letters, emails, phone calls per month asking me to allow them to be the 

first to receive stem cell treatments—these people have cancer, spinal cord injury, 
20 

diabetes, strokes, Parkinson’s disease, and other genetic diseases. (Show sample 

emails from this week). 

You ask, what is the future of ES cells to cure a disease—the answer is simply 

‘‘breathtaking’’. Clearly there are risks as ES cells if left undifferentiated have a 

propensity to cause tumors. But still, many are working to make these cells therapeutically valuable. In addition to the development of novel strategies for treating 

Parkinson’s, diabetes, stroke and spinal cord injury, some like Daniel Kaufman at 

the University of Minnesota are focused on manufacturing red blood cells in massive 

scale thus reducing our dependence upon volunteer donors or developing nature killer cells as anticancer agents-both derived from ES cells. So why has there not been 

a single trial thus far with ES cell—funding, access to suitable cells lines, and research on the immune response to these stem cells. Nuclear transfer will be crucial 

to this success—‘‘tailor made’’ stem cells lines for individuals will be required to 

counter likely immune responses. Again, this is not futuristic, the South Korean scientists have clearly demonstrated that this is not just desirable but possible. 

To restrict work with ES cells or bar SCNT would cripple our capacity to move 

all stem cell therapies forward ES cells are the gold standard and research with 

them will maximize the potential of cord blood and adult stem cells and pursuit of 

multiple approaches will permit the most rapid translation of stem cells possible 

into efficacious clinical therapies. Every single one of us will be faced with a child, 

friend, loved one, or even ourselves with a disease amenable to stem cell therapy 

in the not too distance future. Umbilical cord blood has proven benefits in the treatment of leukemia, lymphoma, blood disorders, immune deficiencies and metabolic 

diseases today. Banking of cord blood is in the nation’s interest and federal dollars 

should continue to be spent to determine the breadth of what it can offer well beyond the confines of blood and marrow diseases. At the same time in parallel, we 

must also push ES and adult stem cells to the limits of what they can offer. And 

for ES cells, banning SCNT could prevent its future success as SCNT is likely to 

be the key that will make ES cell therapies more widely available more rapidly. I 

am here as an advocate for the thousands of people who have asked me to push 

this forward. 

Senator SPECTER. Thank you very much, Dr. Wagner. 

Mr. Herrera, you have testified about your situation being a 

medical breakthrough in medical history. Was the aspect of using 

stem cells on your lymphoma the unique breakthrough that you referred to? 

Mr. HERRERA. Without question. The difference between the first 

transplant and the second transplant was at the first transplant, 

they took stem cells out of my body. I injected myself with a drug 

called Neupogen every day. This causes the bone marrow to overproduce. Little baby stem cells are floating around in the blood. 

They stick a pipe in here, run it through a machine, and they take 

out the little baby stem cells. 

The problem with that transplant for mantle cell lymphoma, 

which they were not aware of at the time, was this is my immune 

system. 

So the theory of the allogeneic stem cell transplant—I go back to 

my Mississippi roots—we are having civil disorder in Wiggins, Mississippi, so we call out the Stone County National Guard. I am told 

not to let anybody cross this line. Winfield Alexander wants to 

cross the line. I cannot stick Winfield in the gut with a bayonet because he was my Boy Scout leader in the rattlesnake patrol. But 

if you bring in the National Guard from Montana, they speak the 

same language, they can read the signs, and they are not going to 

have a problem sticking Winfield with a bayonet. So the foreign immune system is going to be tougher on the lymphoma, on the blood 

cancer, because it does not know it that well. That is kind of a 

basic comment, but that is how I had to understand it. 

Senator SPECTER. Thank you, Mr. Herrera. 
21 

I very much regret that I am going to have excuse myself at this 

point. I turn the gavel over to Senator Cochran. 

What I would appreciate your doing, each of you, is to write a 

memo or a letter to the subcommittee as to what you could do if 

Federal funding were available for your stem cell research. Dr. 

Gasson is from UCLA where I visited several years ago. Without 

the particulars at hand, I know UCLA is the beneficiary of very 

substantial NIH grants. 

This subcommittee, Senator Harkin, Senator Cochran, and then 

the full committee has taken the lead in increasing Federal funding 

from $12 billion to $28 billion. And we are now on the cutting edge. 

Dr. Wagner, you talked about use of Federal funds. 

I regret that there are not more Senators available, but this is 

the third time it will be said. This is a very, very busy place, but 

your testimony is transcribed. Staff are here and Senators will review it. If you would supplement what you have testified to, Dr. 

Teitelbaum, Dr. Jaenisch, Dr. Wagner, Dr. Gasson, with what the 

Federal funding could do. We are going to have a vote on this one 

day soon, without going into all the technicalities. And the evidence 

that you will present will be very helpful when we fight it out on 

the Senate floor. Things are quiet here today, but we are going to 

have a pretty heavy debate on this subject and your participation 

and your evidence will be very, very helpful in achieving a very, 

very important goal for medical science. 

Senator Cochran, let me thank you for taking the gavel. It belongs to you anyway. 

Senator COCHRAN [presiding]. Thank you for your patience with 

our change of command and responsibility. 

I appreciate so much each of your efforts to be here today, to 

take time to prepare a presentation for our committee so that we 

can better understand the challenges and the responsibilities that 

we have for identifying ways that we can continue to support medical research, to take those actions that will help find cures for diseases, prevent diseases. So this is all very serious business, and I 

appreciate very much the fact that you have taken time and devoted your efforts and energies to this hearing today. 

Senator Specter, as I said in my opening comments, has been a 

champion for medical research, and the figures that he cites, the 

increase in the funding that we have been able to provide or to recommend—we do not get to decide. We recommend to the full committee. The full committee approves and recommends to the Senate, and we have to work out differences between the Senate and 

the House. But it has been a successful campaign to more than 

double the amount of money that is available for researchers and 

those providing treatment in our battle to find cures and to prevent 

disease, particularly cancer. 

Let me ask a few questions. I understand, Dr. Teitelbaum, you 

are at the Washington University School of Medicine and have 

completed a residency at New York University. Let me ask you. 

What would you say is the overwhelming opinion of scientists regarding the need to expand the current stem cell policy? Is there 

any disagreement within the community? 

Dr. TEITELBAUM. I think not, Senator. I think that the overwhelming opinion of scientists is to move forward on all fronts, that 
22 

there is potential in adult stem cell research, embryonic stem cell 

research, and umbilical cord blood stem cell research. We cannot 

determine which technique will yield what results until we do the 

science. 

Senator COCHRAN. Dr. Gasson, I heard your comments, before I 

had to step out of the hearing room, in your opening statements. 

What forms of cancer do you think will be the most responsive to 

drugs developed using stem cells? 

Dr. GASSON. We believe that those types of cancer that have been 

shown experimentally to be derived from a mutated adult stem cell 

would be the very best targets for those small molecules. Fortunately or unfortunately for us, they include some of the most common forms of cancer, such as breast cancer, prostate, colon, some 

of the leukemias, and as you are probably quite well aware, brain 

tumors which are truly devastating. 

We think that the concept of the cancer stem cell explains a lot 

about the natural history of the disease. The patient develops cancer and is treated with surgery, radiation, perhaps chemotherapy, 

and the bulk of the tumor, the large mass of the tumor goes away. 

But for some patients over the next 2, 3, 4, or 5 years, the tumor 

comes back and the tumor that recurs is typically even more aggressive. And we think this is because the treatments that we have 

now do not kill the tumor stem cell, and so slowly it begins to divide and it recreates the tumor cells in the patient. And now those 

cells are even more resistant to the treatments that we have. So 

until and unless we are able to either destroy the cancer stem cell, 

or at least keep it under control, we will be continuing to face the 

possibility of recurrence in these very common and very deadly 

cancers. 

Senator COCHRAN. Dr. Jaenisch, there was some indication in a 

Washington Post article that the altered nuclear transplantation 

technique that your lab has achieved may be a way around the objections of some who oppose embryonic stem cell research. Would 

you have pursued this line of research if not for the restrictions in 

place on stem cell research? 

Dr. JAENISCH. I think our research had two goals. One is a scientific one. We wanted to see whether these cells can do what we 

thought they could do. But I think the major goal was to find a potential compromise which could compromise between the concerns 

of those who object to the nuclear transfer procedure and those who 

think that is really important to do. 

So I think the altered nuclear transfer procedure is a modification of the nuclear transfer procedure. It is an additional step 

which complicates an already complex procedure. But from our experience with mice at least, it is such a straightforward and simple 

modification that it may be acceptable as a compromise if that 

would allow then this research to go on and to provide the funding 

for this type of research. 

Senator COCHRAN. Is the kind of research you are doing susceptible to funding by the National Institutes of Health? 

Dr. JAENISCH. All my research is funded by the National Institutes of Health, but I work with mice, so it is not controversial. But 

we would like to move into human cells. We would like to work 

with the new human stem cells. We would like to understand how 
23 

the human cells compare with the mouse cells, and we are very 

limited. We do not have funding for that. 

Senator COCHRAN. Well, do you think that Federal funding 

should be diverted from other forms of stem cell research to support alternative methods to derive stem cells? 

Dr. JAENISCH. Well, I am not sure if it should be diverted. As 

was said by all the speakers here, we really need to pursue all 

these avenues. So alternative methods—several have been proposed. 

I think the final goal of the field in my opinion is to understand 

what reprogramming means. How does the egg reprogram a somatic nucleus and eventually do it without the egg. In order to get 

there, we need the egg. We need human eggs to learn how the 

human egg does reprogramming. So I think it is counterproductive 

for this goal at this point that research is not allowed to use 

human eggs. 

Senator COCHRAN. Anthony, I am so pleased that you were able 

to be here today to put in perspective from a patient’s point of view 

how important research in the development of new treatments, 

finding new ways of dealing with these medical problems will be, 

and the role that we can play. I know if you had a vote, you would 

probably vote to quadruple, double, exponentially increase funding. 

But where in the area of research from your experience do we 

need to supplement and try to provide more incentives through 

Federal research appropriations to achieve the goals of curing cancers like yours? 

Mr. HERRERA. What these ladies and gentlemen up here have 

said is that there should be massive amounts, billions of dollars, 

and no restrictions on any of this research because I have met with 

the doctor at M.D. Anderson—Andreyev I believe is his name—and 

we were talking about the embryonic versus the adult. He said we 

need lots of room to experiment. 

The drug that helped save my life, which has probably saved 

hundreds of thousands of lives, Neupogen, was developed by Janice 

Gabrilove and two other doctors. She was in charge of my first 

bone marrow transplant. I said, how did you develop this drug? 

She said we did not have a straight line. We were in there in that 

region working, and all of a sudden there was a path we could follow. 

So there needs to be no restriction. There needs to be massive 

amounts of money. South Korea, Singapore 2 years ago were ahead 

of us. China just put billions of dollars into research. So there 

should be no restrictions and massive amounts of money put behind this in my opinion. 

Senator COCHRAN. From your experience, could you tell us in 

your own words what the difference is from a patient’s point of 

view in a bone marrow transplant therapy and a stem cell transplant therapy? 

Mr. HERRERA. Actually—someone please correct me if I am 

wrong—they are the same thing except the way you get the cell. 

The reason it is called a bone marrow transplant is before this drug 

Neupogen and before the apheresis machine, they had to drill into 

the bone marrow to suck out marrow and then get the stem cell 

out of that. Am I correct on that? So it evolved into simply being 
24 

called the stem cell transplant. Some hospitals still drill into the 

bone and suck out the marrow to get the stem cell, but they are 

ultimately the same thing. 

Senator COCHRAN. It does not sound like much fun. 

Mr. HERRERA. There was not a lot of fun through the whole process, Senator. 

Senator COCHRAN. I can remember reading your description of 

the pain that you suffered in that first effort to get some of your 

bone marrow. No anesthetic. 

Mr. HERRERA. That was not good medicine. 

Senator COCHRAN. I hope that is not a widespread practice now. 

Mr. HERRERA. I have learned that it is not. 

Senator COCHRAN. Good. 

Dr. Wagner, we appreciate your being here as well. Since your 

primary interest appears to be cord blood stem cell research, as I 

understand it, why are you so supportive of embryonic stem cell 

and nuclear transplantation research? 

Dr. WAGNER. My interest is, obviously, in cord blood as one avenue. As the clinical director of the Stem Cell Institute, I am really 

interested in all aspects of stem cell therapies, whether it comes 

from embryonic stem cells, adult tissues, or umbilical cord blood. 

So we are exploring all avenues. 

However, my own personal research area in the laboratory is 

with umbilical cord blood and trying to figure out what really the 

breadth of applicability will be. So we are investigating not only in 

the context of classical bone marrow transplantation, which has 

proven to be of great use, but also looking at what its differentiation potential is, can it differentiate into various tissues. 

But remember that what we said over and over again is that ES 

cells are the gold standard by which everything is compared. And 

everything that we have learned with embryonic stem cells, in 

terms of the mechanisms of what makes them able to become liver 

or lung or brain, or whatever it is, has given us clues or techniques 

that allow us to see if we can get adult tissues or cord blood tissues 

to do the same thing. So without having that research move forward with embryonic stem cells, we have no hope to make adult 

tissues or cord blood stem cells become you would all like it to become as the stem cell source. 

Senator COCHRAN. Does it surprise you that the NCI funds less than $5 million worth of embryonic stem cell research? And why 

do you think the level of stem cell research is so low in the context 

of a $5 billion budget? 

Dr. WAGNER. You are asking my opinion now. 

Senator COCHRAN. Yes. 

Dr. WAGNER. Well, clearly, I think the reason why the budget is 

so low is, in part, related to the ethical issues associated with embryonic stem cell work. However, there is considerable funding for 

adult tissue stem cells, as well as umbilical cord blood. However, 

what we need to be doing is working on embryonic stem cells. Unfortunately, the budget is low and it has actually been extraordinarily restrictive in what we are able to do. 

Right now as the clinician that hopes to move some of these cell 

therapies forward, we have no hope of using the existing stem cell 

lines that are currently approved because of the fact that many of 
25 

them were developed on murine feeder layers or they have cytogenetic abnormalities having been passaged in a culture. And as 

someone who manufactures cells for clinical use, they would never 

fulfill our criteria. So certainly we need to markedly expand the 

amount of resources or else we will never be able to move it forward. 

Senator COCHRAN. Dr. Gasson, you also lead an NCI-designated 

comprehensive cancer center and support research on embryonic 

stem cells. Do you have an opinion about the disparity in terms of 

the $5 million for stem cell research compared with a $5 billion 

budget? 

Dr. GASSON. I have two additional thoughts to add to Dr. Wagner’s comments. 

First of all, this notion that cancer arises from a cancer stem cell 

is fairly new. If you are trying to study the cancer stem cell, that 

is an adult stem cell, and so that particular type of work has just 

recently been done and probably would not be counted under the 

rubric of embryonic stem cell research. 

But the main reason is the reason that Dr. Jaenisch articulated, 

which is most of the people that are trying to work in this field are 

working with mouse ES cells and mouse models because of the restrictions on the use of human ES cells. And Dr. Jaenisch is a perfect example. These people are extraordinarily talented. They have 

devoted their careers to understanding these things. If we could 

channel them from the mouse to working on human ES cells, we 

could accelerate the pace of progress enormously. So it is a followup on Dr. Wagner’s answer, which is that the restrictions are pushing people to work in the mouse system. 

CONCLUSION OF HEARING

Senator COCHRAN. Let me thank all of you for your generous contribution of time and effort to this hearing. We appreciate it very 

much, and I am sure we will benefit from your observations and 

your wisdom as we proceed through the appropriations process for 

writing a bill that actually is going to come to the floor next week 

possibly. This will be the last appropriations bill considered by the 

Senate this year. So we want to be sure we have our facts and arguments available to describe the reasons why we think funding of 

additional medical research is so important to the future of our 

country and mankind. Thank you for the contribution you have 

made to that effort. 

The hearing is recessed. 

[Whereupon, at 10:31 a.m., Wednesday, October 19, the hearing 

was concluded, and the subcommittee was recessed, to reconvene 

subject to the call of the Chair.] 

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