```

[Senate Hearing 109-249]
[From the U.S. Government Publishing Office]

                                                        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

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                               __________
                      COMMITTEE ON APPROPRIATIONS

                  THAD COCHRAN, Mississippi, Chairman
TED STEVENS, Alaska                  ROBERT C. BYRD, West Virginia
ARLEN SPECTER, Pennsylvania          DANIEL K. INOUYE, Hawaii
PETE V. DOMENICI, New Mexico         PATRICK J. LEAHY, Vermont
CHRISTOPHER S. BOND, Missouri        TOM HARKIN, Iowa
MITCH McCONNELL, Kentucky            BARBARA A. MIKULSKI, Maryland
CONRAD BURNS, Montana                HARRY REID, Nevada
RICHARD C. SHELBY, Alabama           HERB KOHL, Wisconsin
JUDD GREGG, New Hampshire            PATTY MURRAY, Washington
ROBERT F. BENNETT, Utah              BYRON L. DORGAN, North Dakota
LARRY CRAIG, Idaho                   DIANNE FEINSTEIN, California
KAY BAILEY HUTCHISON, Texas          RICHARD J. DURBIN, Illinois
MIKE DeWINE, Ohio                    TIM JOHNSON, South Dakota
SAM BROWNBACK, Kansas                MARY L. LANDRIEU, Louisiana
WAYNE ALLARD, Colorado
                    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            TOM HARKIN, Iowa
JUDD GREGG, New Hampshire            DANIEL K. INOUYE, Hawaii
LARRY CRAIG, Idaho                   HARRY REID, Nevada
KAY BAILEY HUTCHISON, Texas          HERB KOHL, Wisconsin
TED STEVENS, Alaska                  PATTY MURRAY, Washington
MIKE DeWINE, Ohio                    MARY L. LANDRIEU, Louisiana
RICHARD C. SHELBY, Alabama           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)

                            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

                       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 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 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 stem-cell 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.

                    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.

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.
    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.''
    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.
    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 so-called 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 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 life-saving 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 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 life-saving 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.
    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 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 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 blastocyst 
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.
    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 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 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 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 dollars 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, 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 over-produce. 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.
    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 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 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 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 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 follow-up 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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