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STEM CELL SCIENCE: THE FOUNDATION FOR 

FUTURE CURES 

HEARING 

BEFORE THE

SUBCOMMITTEE ON HEALTH 

OF THE

COMMITTEE ON ENERGY AND 

COMMERCE 

HOUSE OF REPRESENTATIVES 

ONE HUNDRED TENTH CONGRESS 

SECOND SESSION 

MAY 8, 2008 

Serial No. 110–115 

( 

Printed for the use of the Committee on Energy and Commerce 

energycommerce.house.gov 

COMMITTEE ON ENERGY AND COMMERCE 

JOHN D. DINGELL, Michigan, Chairman 

HENRY A. WAXMAN, California 

EDWARD J. MARKEY, Massachusetts 

RICK BOUCHER, Virginia 

EDOLPHUS TOWNS, New York 

FRANK PALLONE, JR., New Jersey 

BART GORDON, Tennessee 

BOBBY L. RUSH, Illinois 

ANNA G. ESHOO, California 

BART STUPAK, Michigan 

ELIOT L. ENGEL, New York 

ALBERT R. WYNN, Maryland 

GENE GREEN, Texas 

DIANA DEGETTE, Colorado 

Vice Chairman 

LOIS CAPPS, California 

MIKE DOYLE, Pennsylvania 

JANE HARMAN, California 

TOM ALLEN, Maine 

JAN SCHAKOWSKY, Illinois 

HILDA L. SOLIS, California 

CHARLES A. GONZALEZ, Texas 

JAY INSLEE, Washington 

TAMMY BALDWIN, Wisconsin 

MIKE ROSS, Arkansas 

DARLENE HOOLEY, Oregon 

ANTHONY D. WEINER, New York 

JIM MATHESON, Utah 

G.K. BUTTERFIELD, North Carolina 

CHARLIE MELANCON, Louisiana 

JOHN BARROW, Georgia 

BARON P. HILL, Indiana 

JOE BARTON, Texas 

Ranking Member 

RALPH M. HALL, Texas 

FRED UPTON, Michigan 

CLIFF STEARNS, Florida 

NATHAN DEAL, Georgia 

ED WHITFIELD, Kentucky 

BARBARA CUBIN, Wyoming 

JOHN SHIMKUS, Illinois 

HEATHER WILSON, New Mexico 

JOHN B. SHADEGG, Arizona 

CHARLES W. ‘‘CHIP’’ PICKERING, 

Mississippi 

VITO FOSSELLA, New York 

STEVE BUYER, Indiana 

GEORGE RADANOVICH, California 

JOSEPH R. PITTS, Pennsylvania 

MARY BONO MACK, California 

GREG WALDEN, Oregon 

LEE TERRY, Nebraska 

MIKE FERGUSON, New Jersey 

MIKE ROGERS, Michigan 

SUE WILKINS MYRICK, North Carolina 

JOHN SULLIVAN, Oklahoma 

TIM MURPHY, Pennsylvania 

MICHAEL C. BURGESS, Texas 

MARSHA BLACKBURN, Tennessee 

PROFESSIONAL STAFF

DENNIS B. FITZGIBBONS, Chief of Staff 

GREGG A. ROTHSCHILD, Chief Counsel 

SHARON E. DAVIS, Chief Clerk 

DAVID CAVICKE, Minority Staff Director 

(II)

(III) 

SUBCOMMITTEE ON HEALTH

FRANK PALLONE, JR., New Jersey, Chairman 

HENRY A. WAXMAN, California 

EDOLPHUS TOWNS, New York 

BART GORDON, Tennessee 

ANNA G. ESHOO, California 

GENE GREEN, Texas 

Vice Chairman 

DIANA DEGETTE, Colorado 

LOIS CAPPS, California 

TOM ALLEN, Maine 

TAMMY BALDWIN, Wisconsin 

ELIOT L. ENGEL, New York 

JAN SCHAKOWSKY, Illinois 

HILDA L. SOLIS, California 

MIKE ROSS, Arkansas 

DARLENE HOOLEY, Oregon 

ANTHONY D. WEINER, New York 

JIM MATHESON, Utah 

JOHN D. DINGELL, Michigan (ex officio) 

NATHAN DEAL, Georgia, 

Ranking Member 

RALPH M. HALL, Texas 

BARBARA CUBIN, Wyoming 

HEATHER WILSON, New Mexico 

JOHN B. SHADEGG, Arizona 

STEVE BUYER, Indiana 

JOSEPH R. PITTS, Pennsylvania 

MIKE FERGUSON, New Jersey 

MIKE ROGERS, Michigan 

SUE WILKINS MYRICK, North Carolina 

JOHN SULLIVAN, Oklahoma 

TIM MURPHY, Pennsylvania 

MICHAEL C. BURGESS, Texas 

MARSHA BLACKBURN, Tennessee 

JOE BARTON, Texas (ex officio) 

(V) 

C O N T E N T S 

Page 

Hon. Frank Pallone, Jr., a Representative in Congress from the State of 

New Jersey, opening statement .......................................................................... 1 

Hon. Nathan Deal, a Representative in Congress from the State ofGeorgia, 

opening statement ................................................................................................ 3 

Hon. Henry A. Waxman, a Representative in Congress from the State of 

California, opening statement ............................................................................. 4 

Hon. Joseph R Pitts, a Representative in Congress from the Commonwealth 

of Pennsylvania, opening statement ................................................................... 5 

Hon. John D. Dingell, a Representative in Congress from the State of Michigan, opening statement ....................................................................................... 6 

Hon. Tim Murphy, a Representative in Congress from the Commonwealth 

of Pennsylvania, opening statement ................................................................... 8 

Hon. Diana D. DeGette, a Representative in Congress from the State of 

Colorado, opening statement ............................................................................... 8 

Prepared statement .......................................................................................... 10 

Hon. Marsha Blackburn, a Representative in Congress from the State of 

Tennessee, opening statement ............................................................................ 11 

Hon. Tammy Baldwin, a Representative in Congress from the State of Wisconsin, opening statement ................................................................................... 12 

Hon. Mike Ferguson, a Representative in Congress from the State of New 

Jersey, opening statement ................................................................................... 13 

Hon. Gene Green, a Representative in Congress from the State of Texas, 

opening statement ................................................................................................ 14 

Hon. Anna G. Eshoo, a Representative in Congress from the State of California, prepared statement .................................................................................. 107 

Hon. Barbara Cubin, a Representative in Congress from the State of Wyoming, prepared statement ................................................................................... 107 

Hon. Lois Capps, a Representative in Congress from the State of California, 

prepared statement .............................................................................................. 108 

Hon. Edolphus Towns, a Representative in Congress from the State of New 

York, prepared statement .................................................................................... 109 

Hon. John Sullivan, a Representative in Congress from the State of New 

Oklahoma, prepared statement .......................................................................... 110 

WITNESSES

Elias A. Zerhouni, M.D., Director, National Institutes of Health ....................... 15 

Prepared statement .......................................................................................... 18 

Submitted questions ......................................................................................... 112 

John D. Gearhart, Ph.D., C. Michael Armstrong professor of medicine, Institute for Cell Engineering, Johns Hopkins University ....................................... 55 

Prepared statement .......................................................................................... 57 

Answers to submitted questions 1 ...................................................................

Amit N. Patel, M.D., M.S., director of cardiac cell therapy, The Heart, Lung 

and Esophageal Institute, UPMC Presbyterian, McGowan Institute of Regenerative Medicine ............................................................................................. 62 

Prepared statement .......................................................................................... 65 

Answers to submitted questions ...................................................................... 121 

Douglas T. Rice, Spokane Valley, Washington ...................................................... 69 

Prepared statement .......................................................................................... 71 

Answers to submitted questions ...................................................................... 125 

George Q. Daley, M.D., Ph.D., president, International Society for Stem Cell 

Research; and associate professor of pediatrics, Children’s Hospital Boston . 75 

Prepared statement .......................................................................................... 76 

Answers to submitted questions ...................................................................... 127 

Page

VI 

Weyman Johnson, Jr., J.D., chairman, National Multiple Sclerosis Society ...... 78 

Prepared statement .......................................................................................... 79 

Answers to submitted questions ...................................................................... 133 

Joseph R. Bertino, M.D., interim director and chief scientific officer, The 

Cancer Institute of New Jersey .......................................................................... 82 

Prepared statement .......................................................................................... 84 

Answers to submitted questions ...................................................................... 185 

John K. Fraser, Ph.D., principal scientist, Cytori Therapeutics .......................... 88 

Prepared statement .......................................................................................... 90 

Answers to submitted questions ...................................................................... 136 

SUBMITTED MATERIAL

‘‘Autologous Nonmyeloablative Hematopoietic Stem Cell Transplantation in 

Newly Diagnosed Type 1 Diabetes Mellitus,’’ Journal of the American Medical Association, April 11, 2007 ........................................................................... 40 

‘‘Stem Cell Vindication,’’ Charles Krauthammer, Washington Post, November 

30, 2007 ................................................................................................................. 49 

1Mr. Gearhart did not answer submitted questions for the record. 

(1) 

STEM CELL SCIENCE: THE FOUNDATION FOR 

FUTURE CURES 

THURSDAY, MAY 8, 2008 

HOUSE OF REPRESENTATIVES, SUBCOMMITTEE ON HEALTH, COMMITTEE ON ENERGY AND COMMERCE, Washington, DC. 

The subcommittee met, pursuant to call, at 10:00 a.m., in room 

2322 of the Rayburn House Office Building, Hon. Frank Pallone, 

Jr. (chairman) presiding. 

Members present: Representatives Pallone, Waxman, Towns, 

Green, DeGette, Capps, Baldwin, Dingell (ex officio), Deal, Hall, 

Pitts, Ferguson, Myrick, Sullivan, Murphy, Burgess, Blackburn, 

and Barton (ex officio). 

Staff present: Jessica McNiece, Katherine Martin, Melissa 

Sidman, Chad Grant, and Robert Clark. 

Mr. PALLONE. I call the meeting of the subcommittee to order. 

First of all, let me say good morning to everybody, and explain 

that today the subcommittee is meeting to hear about stem cell 

science and the potential it holds, and I will recognize myself for 

an opening statement initially. 

OPENING STATEMENT OF HON. FRANK PALLONE, JR., A REPRESENTATIVE IN CONGRESS FROM THE STATE OF NEW JERSEY 

Mr. PALLONE. In terms of the potential for stem cell science to 

develop new treatments, therapies, and cures for a myriad of diseases, conditions, and disabilities, there is obviously a lot of potential and could impact so many people in their lives. There are few 

areas of scientific inquiry that hold the same level of promise to 

revolutionize the practice of medicine. Stem cells offer the possibility of replacing damaged or diseased cells inside the body with 

healthy ones. They could make it possible to strengthen failing 

heart muscle, regenerate severed spinal cord nerves, replace damaged brain cells, and cure many other currently incurable disorders. 

Through my service on the subcommittee, I have had the opportunity to meet and hear from people from communities across the 

country and they have come to share their stories or the stories of 

their loved ones; just as an example, a young child with diabetes 

who requires daily medical attention, an adult who has left her job 

to care for a father whose mind has been ravaged by the effects of 

Alzheimer’s disease, a husband who watched his wife’s motor function deteriorate with the onset of Parkinson’s disease. Their stories 

2 

vary tremendously and range from the heartbreaking to the 

harrowing yet they all share one common theme, and that is the 

message of hope, hope that someday stem cell research will unlock 

the door and reveal a new discovery that will cure them of their 

ailments. 

I believe it is our obligation as legislators to enact a Federal policy that will help advance all types of stem cell research and provide the opportunity for such discoveries to take place. Unfortunately, the current Federal policy on stem cell research is falling 

short of that goal. The President’s 2001 Executive order limits the 

use of Federal funds for research on the few lines of stem cells that 

had already been harvested. At the time he said that, stem cell research offered great promise. Almost 7 years later, it is clear to me 

that the President’s policy has placed arbitrary constraints on stem 

cell research and has put patients in great peril. 

Since the President issued his Executive order, we have undoubtedly lost valuable time and resources that could have been devoted 

to advancing stem cell research. While there have been important 

advancements in certain fields such as stem cells harvested from 

cord blood and adult stem cells, the scientific community appears 

to be in agreement that it is embryonic stem cell research that 

holds the greatest promise for the development of new cures and 

treatments. Unfortunately, the Administration’s current policy on 

embryonic stem cell research has tied the hands of researchers, impeding scientific progress and inhibiting America’s ability to compete with scientists around the world. Thankfully, the private sector and individual States have decided to forge ahead, paving the 

way without any Federal funding. 

In 2005, my home State of New Jersey became the first State to 

provide for the public funding of embryonic stem cell research. 

Since then, plans for construction have begun on a new state-ofthe-art facility that will house the Stem Cell Institute of New Jersey, a joint initiative undertaken by the University of Medicine and 

Dentistry of New Jersey and Rutgers, the State University of New 

Jersey, and I want to welcome Dr. Bertino, the interim director of 

the Stem Cell Institute of New Jersey, who will be testifying on our 

second panel today. 

But New Jersey is not the only State taking the lead. A number 

of other States have either enacted their own measures that would 

fund various forms of stem cell research or have bills pending before their legislatures. While I am thankful for these efforts, I believe that in order to truly propel the advancement of stem cell research, we need a Federal policy that builds upon the advancements being funded in the private sector and at the State level. 

Last year, the House and Senate passed such a policy with overwhelming bipartisan majorities. The Stem Cell Research Enhancement Act, sponsored by Ms. DeGette, would have allowed Federal 

funding for stem cell research to be conducted on embryos that 

would otherwise have been discarded from fertility clinics and with 

the consent of the embryos’ donors. Unfortunately, this commonsense policy was met swiftly with the President’s veto pen, the very 

first of his presidency. I know this is a controversial issue for many 

Americans, including many members who serve on this subcommittee, and I can respect that. However, I still have trouble unVerDate Nov 24 2008 16:13 Mar 16, 2010 Jkt 037690 PO 00000 Frm 00008 Fmt 6633 Sfmt 6633 C:\DOCUMENTS AND SETTINGS\CWELLS1\HEARINGS\110-115 SCOM1 PsN: JIMC
3 

derstanding the opposition that exists to such a commonsense approach that would allow for the progression of stem cell science in 

what I view as a careful, ethical, and respectful fashion. 

The fact is that Americans want stem cell science to advance. An 

overwhelming majority of Americans support embryonic stem cell 

research and their representatives in Congress do so as well, and 

they want us as legislators to do everything we can to help unlock 

the potential of embryonic stem cells in the quickest fashion possible and bring new life-saving therapies to the patients who need 

them. 

With millions of Americans dying each year from diseases that 

might be cured by stem cell therapies, we can’t wait any longer. 

The time has come to enact a new Federal policy, and I know that 

Ms. DeGette in particular is concerned about that. She asked that 

we have this hearing today. 

Mr. PALLONE. I now recognize our ranking member, Mr. Deal, for 

5 minutes for an opening statement. 

OPENING STATEMENT OF HON. NATHAN DEAL, A REPRESENTATIVE IN CONGRESS FROM THE STATE OF GEORGIA 

Mr. DEAL. Thank you, Mr. Chairman. 

With individuals with degenerative life-altering diseases or lifechanging events resulting in paralysis, the possibilities presented 

by embryonic stem cell research represent a glimmer of hope to 

heal a loved one or reverse the damage caused by debilitating disease. For others, this issue seems just as personal as they struggle 

to reconcile the possibilities presented by research and science with 

their own personal convictions about the sanctity of any human 

life. It is at this intersection where we find ourselves this morning. 

My hope is that we could explore the possibilities presented by all 

types of stem cell research and willingly confront the ethical and 

scientific questions raised by this issue. 

To my knowledge, adult stem cell research, which does not raise 

the ethical questions surrounding the destruction of a human embryo, has resulted in many new and exciting discoveries. I would 

hope that our witnesses could further elaborate on the potential of 

research conducted with adult stem cells and other cells that are 

capable of producing all or almost all of the cell types of the developing body. We must consider whether we should be taking funding 

away from the areas of research which have been proven to work 

and the promising adult stem cell therapies which have already improved patient health. Specifically, I hope our witnesses can tell us 

about the existing track record of adult stem cell research as compared to embryonic stem cell research. 

I think the question we should be trying to answer here is 

whether or not there is a middle ground which allows scientists to 

continue their cutting-edge research while respecting the sanctity 

of every human life. Hopefully our witnesses today can describe the 

variety of research being done with all types of stem cells today. 

It would be very useful to learn more about the future of embryonic 

stem cell research and the time frame in which researchers expect 

to develop these treatments, which are often cited by supporters of 

embryonic stem cell research. 

4 

I think this should be a good hearing on the issue and certainly 

one that warrants our complete attention, and I thank all of our 

witnesses for coming and I look forward to your testimony. I yield 

back. 

Mr. PALLONE. I yield 5 minutes to the gentleman from California—I am sorry—3 minutes to Mr. Waxman. 

OPENING STATEMENT OF HON. HENRY A. WAXMAN, A REPRESENTATIVE IN CONGRESS FROM THE STATE OF CALIFORNIA 

Mr. WAXMAN. Thank you, Mr. Chairman, for recognizing me and 

for holding this hearing today. 

Stem cell research is truly exciting scientific research. Stem cells, 

both embryonic and adult, hold great potential. For example, we 

will hear today about how adult stem cells may be used to treat 

potentially deadly heart conditions and embryonic cells have the 

potential to become any cell in the body. There is great hope that 

these cells will help us understand more about such devastating 

diseases as Parkinson’s and diabetes and perhaps some day lead to 

treatments. And in a fascinating advance announced last year, several labs have been able to reprogram adult cells to develop into 

multiple kinds of cells, much as embryonic stem cells can. 

What I think will become clear as we hear from scientific experts 

today is it doesn’t make any sense to pit one type of stem cell research against another. Each line of research holds distinct promise. They function differently as research models and may function 

differently as potential routes to therapies. It makes sense to encourage the growth of all of these types of research, not to sit here 

and argue about which is more promising than another and why. 

Unfortunately, all too frequently, discussions of stem cell issues 

are based more on politics than on science. As we have seen in too 

many areas, from stacked advisory committees to the deletion of 

accurate scientific information from government Web sites, the 

science around stem cells has at times been distorted to justify a 

particular political or ethical view. We are given inaccurate accounts of the availability of embryonic stem cell lines derived from 

the President’s moratorium and in certain cases, misleading claims 

about adult stem cells have been used to argue that there is no scientific need whatsoever for embryonic stem cell research. Of 

course, ethical, political, and other considerations affect policy decisions, but distorting science is wrong. 

I think we are going to hear from a number of experts who will 

tell us that there is a consensus among scientists that we should 

support embryonic stem cell research. New methods of creating 

stem cells are promising. Without funding embryonic stem cell research, we are guaranteed to learn nothing from it. We will leave 

the field behind in the United States and we will lose the opportunity to develop a meaningful Federal framework of oversight and 

ethical guidelines. 

I hope today’s hearing creates a better understanding in Congress and America of why support for all kinds of stem cell research continues to be so important. 

Mr. PALLONE. Thank you, Mr. Waxman. 

Mr. Pitts. 

5 

OPENING STATEMENT OF HON. JOSEPH R. PITTS, A REPRESENTATIVE IN CONGRESS FROM THE COMMONWEALTH 

OF PENNSYLVANIA 

Mr. PITTS. Thank you, Mr. Chairman. I would like to thank you 

for convening this hearing today to discuss the future of stem cells, 

and I am grateful for this rare platform to highlight the incredible 

developments in stem cell research that are being used to successfully treat people for several dozen different conditions. These conditions include heart disease, juvenile diabetes, Parkinson’s, liver 

failure, lupus, sickle cell anemia, and spinal cord injuries to name 

a few. 

Over the last decade, there has been contentious debate over the 

issue of taxpayer funding for stem cell research that results in the 

destruction of a human embryo. At the center of this debate has 

been the hope for treatment and cures for patients across the world 

who suffer from a host of different diseases. So I would like to talk 

about just that, the patients. 

We have here on the left a picture of three patients. The first one 

here on the left is Amy Daniels. Amy was diagnosed with systemic 

scleroderma, a rare autoimmune disease that affects connective tissue in the body. Next to Amy is Barry Gowdy, who suffered from 

multiple sclerosis. And last is Joe Rosen, a patient with 

antiphospholipid syndrome, an autoimmune disorder that causes 

blood clots. These three patients endured vastly different experiences but share two things in common. First, all three of them had 

lost hope that they could ever live a normal life, and second, all 

three of them found hope in the form of adult stem cell treatments, 

which have successfully mitigated their symptoms. 

Another patient is seated here with us today. In 2003, Carol 

Franz was diagnosed with multiple myeloma. Myeloma is a blood 

cancer that eats away at the bones. X-rays of Carol’s bones made 

them look like target practice. Faced with the daunting fears of a 

deadly form of cancer, Carol found hope as she was told about a 

treatment that could help her by using her own stem cells, and 

now Carol sits before us having survived two bouts with cancer 

after receiving two stem cell transplants, and she wears a bright 

green tee shirt that says ‘‘Survivor: adult stem cell transplant.’’ 

And this mantra is based not on ideology but on science. It is based 

on what works. It is based on what saved Carol’s life twice. Adult 

stem cells are doing what we have all hoped for and wished for: 

they are successfully treating patients. 

I look forward to hearing the testimony of yet another patient 

and witness on this panel, Doug Rice, who has been treated for 

heart disease using adult stem cells. Unfortunately, the political 

agenda for taxpayer-funded research that destroys human embryos 

and has failed to treat any patients has diverted the focus away 

from the success of adult stem cells. In fact, it was just 1 year ago 

that Dr. Richard Burt, along with Brazilian researcher Dr. Julio 

Voltarelli, conducted a study that used stem cells from patients’ 

own bodies to successfully reverse type 1 juvenile diabetes in 13 

out of 15 patients over a several-year period. It was regrettable 

that this remarkable research had to be conducted in Brazil due to 

a lack of interest in the United States. 

6 

Thankfully, last fall, the contentious and heated debate surrounding stem cell research was quieted by a scientific breakthrough which has shown the ability to create embryonic-like stem 

cells. This research will face all of the same hurdles as embryonic 

stem cells, including tumors and rejection. However, it holds all the 

potential touted by proponents of embryonic stem cell research but 

without any of the ethical concerns. Dr. Rudolph Jaenisch of the 

Whitehead Institute confirmed that, ‘‘Biologically, there is no difference’’ between iPS and embryonic stem cells. Dr. James Thomson, University of Wisconsin—— 

Mr. PALLONE. Mr. Pitts, if you could just wrap it up. You are a 

minute and 26 seconds over. 

Mr. PITTS. I am sorry. Dr. Thomson, the pioneer of embryonic destructive stem cell research, was one of the scientists to discover 

this new method and he described significant advantages of iPS 

cells because they don’t pose the same ethical challenges as destroying embryos, cloning or harvesting eggs. So the topic of this 

hearing is the future of stem cells. 

Thank you, Mr. Chairman, for holding this important hearing. 

Mr. PALLONE. Let me just mention to members that we are going 

to have, I believe, five votes in another 15 minutes but we will continue and try to get a couple more opening statements in before 

then and then we will come back. 

I now recognize the chairman of the full committee, Mr. Dingell. 

OPENING STATEMENT OF HON. JOHN D. DINGELL, A REPRESENTATIVE IN CONGRESS FROM THE STATE OF MICHIGAN 

Mr. DINGELL. Mr. Chairman, I thank you for your courtesy. I 

thank you for the recognition and I commend you for this hearing 

this morning. 

Stem cell research holds great promise for a better understanding and treatment of a broad range of debilitating and deadly 

diseases and conditions including Parkinson’s disease, cancer, Alzheimer’s disease, diabetes, and multiple sclerosis, amongst others, 

yet a significant problem is created by politics and the promise is 

being somewhat imperiled or indeed seriously threatened by politics. 

This committee is engaged in a practice that is very important: 

oversight, the gathering of information to understand what our national policies should be and what our actions should be here in the 

Congress in the way of legislation, what we should do in the way 

of expenditure of monies and national efforts to achieve great national purposes. 

Scientists, it should be observed, work with two kinds of stem 

cells: adult stem cells and embryonic stem cells. Current science indicates that adult and embryonic stem cells differ in significant 

ways and therefore we need to examine both. Yet despite well-documented benefits of embryonic stem cell research and pleas from the 

scientific community, the Administration has regrettably adopted 

research restrictions that inhibit the ability of scientists to fully explore the potential of embryonic stem cells. In this Congress, the 

House and Senate have sent the President not once but twice biVerDate Nov 24 2008 16:13 Mar 16, 2010 Jkt 037690 PO 00000 Frm 00012 Fmt 6633 Sfmt 6633 C:\DOCUMENTS AND SETTINGS\CWELLS1\HEARINGS\110-115 SCOM1 PsN: JIMC
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partisan legislation that would limit and lift these restrictions, and 

both times the President has vetoed this legislation. 

Researchers in my own State of Michigan have been doubly hamstrung by Federal constraints and by State limitations. The University of Michigan has an impressive Life Sciences Institute, focusing on stem cell research and a prominent University Center for 

Stem Cell Biology. In 2003, under the capable leadership of Dr. 

Max Wicha, who directs the Comprehensive Cancer Center at the 

University of Michigan, scientists there discovered breast cancer 

stem cells, and last year found stem cells in pancreatic cancer. 

These are especially noteworthy and impressive accomplishments 

and give us knowledge and warnings that are important to us in 

our concerns about these matters. Given the limited funding available to the university with State and Federal dollars unavailable 

for research, the university scrambles to support this 

groundbreaking research with private funds. 

I do not profess to know which stem cell lines are most valuable 

or which ones offer the most promise or which can give the greatest 

hope to those living with debilitating conditions and diseases. I 

defer to the experts on such questions such as Dr. Zerhouni, the 

director of NIH, who is here today, and Doctor, by the way, welcome to you. Your comments in 2007, I will quote: ‘‘It is in the best 

interests of our scientists, our science, and our country that we find 

ways and that the Nation find a way to go full speed across adult 

and embryonic stem cells equally.’’ From my standpoint, it is clear 

today that the American science will be better served and the Nation better served if we let our scientists have access to more cell 

lines. 

I defer to the Institute of Medicine, IOM, which stated in 2002, 

and I quote, ‘‘Studies of both embryonic and adult human stem 

cells will be required to most efficiently advance the scientific and 

therapeutic potential of regenerative medicine.’’ 

Research on both adult and embryonic human stem cells should 

be pursued. None of us can guarantee to those suffering from Parkinson’s disease, spinal cord injuries or multiple sclerosis or any 

other condition that embryonic stem cell research will bring success 

but we can assure and we can guarantee that if we don’t and if we 

let politics, not science, guide our efforts, we are consigning ourselves to failure and to suffering. 

I thank the chairman, Mr. Pallone, for holding today’s hearing, 

and I commend our colleague, Ms. DeGette, for her dedication and 

commitment on this issue. Finally again, I thank our friend, the 

NIH director, Dr. Zerhouni, for rearranging his schedule to be here 

with us today. I look forward to the testimony of our expert witnesses on the current state of stem cell research and science, and 

I thank you, Mr. Chairman; I thank my colleagues and I thank our 

witnesses. 

Mr. PALLONE. Thank you, Chairman Dingell. 

I would like to take one more opening statement but let me just 

mention, we have five votes, 15 and then four fives, 10 minutes of 

debate on a motion to recommit, a 15-minute vote on that and then 

another five, so we are probably talking close to an hour once we 

go into recess. But I would like to have Mr. Murphy recognized for 

an opening statement and then after that we will go vote. 

8 

OPENING STATEMENT OF HON. TIM MURPHY, A REPRESENTATIVE IN CONGRESS FROM THE COMMONWEALTH OF PENNSYLVANIA 

Mr. MURPHY. Thank you, Mr. Chairman, and let me begin by 

welcoming one of our witnesses here today, Dr. Patel, who is Director of Cardiovascular Cell Therapies at the University of Pittsburgh Medical Center. I look forward to hearing his testimony. 

While we are talking about science and research, I think it is important to understand that ethics cannot be diminished by relabeling it as political and dismissing the value of ethical review 

through polling or politics. The life of a human embryo is not insignificant and not immaterial to scientific research, and one cannot 

perform scientific medical research without including medical ethics. A couple years ago, at the time that Congress was voting on 

embryonic stem cell research, a study came out out of South Korea, 

Seoul International University, I believe, and many were so eager 

to find the results they wanted to see that they failed to see that 

the results were not what was really found. 

We need to continue stem cell research but to also review its scientific merit and outcome and to always, always review each finding under the lamp of careful scientific and ethical scrutiny. 

The Federal Government does not prohibit any private individual 

or business from carrying out embryonic stem cell research but we 

have chosen to hold off taxpayers’ dollars for this, and it is not just 

a matter of deciding on a poll. We have to acknowledge that years 

from now, perhaps this very subcommittee will be debating and 

holding hearings on what we may now consider as the unthinkable: 

cloning replicas of ourselves to be used as organ gardens waiting 

to be harvested. Indeed, that may come in the future. But let us 

understand when it comes to stem cell research, dozens and dozens 

of great scientific breakthroughs have come from using adult stem 

cells, placenta, umbilical cord, muscle, skin, other issues, and that 

is important, but the number of studies that have come out that 

have shown significant scientific results from embryonic stem cells 

is zero. 

So I hope that this panel will look at these issues as ones that 

are important to review and that we cannot, no matter how hard 

we might use tactics to dismiss it as political, we cannot dismiss 

ourselves from the obligation of carefully, carefully reviewing each 

thing we do. Life does have value, saving lives has value, and scientific research cannot be made distinct from ethical oversight of 

that same research. 

I yield back. 

Mr. PALLONE. I think we have time for one more, so I recognize 

Ms. DeGette for an opening statement. 

Ms. DEGETTE. Thank you, Mr. Chairman. I would ask unanimous consent to put my full opening statement in the record. 

Mr. PALLONE. Without objection, so ordered. 

OPENING STATEMENT OF HON. DIANA D. DEGETTE, A REPRESENTATIVE IN CONGRESS FROM THE STATE OF COLORADO 

Ms. DEGETTE. Thank you, and I want to thank you for holding 

this very first ever hearing on stem cell research in the Energy and 

9 

Commerce Committee. It is an incredibly important topic, and I 

want to thank Dr. Zerhouni for coming and rearranging his schedule today. 

It is particularly important that we have this hearing because 

over the past year we have had many developments in the field of 

cell-based scientific research. We saw breakthroughs and accomplishments that could not have been predicted even months before 

they happened: insulin-producing islet cells created from embryonic 

stem cells, induced pluripotent stem cells developed from adult 

skin cells, and primate embryonic stem cells generated through somatic cell nuclear transfer. All of this proves that one can rarely 

predict the outcomes of scientific research and it underscores what 

the other members have been saying, that it is crucial to make the 

investment in all ethical forms of research to begin with. 

That is what we are going to explore during this hearing: where 

we are now and where we are going with stem cell research. Every 

time there has been some new discovery in some other type of research besides embryonic stem cells, the Bush Administration and 

opponents of this research try to claim it is a substitute for embryonic stem cell research, yet as every researcher tells me, all of 

these forms of cell-based research are complementary and they all 

aid future developments of cures for patients, which we see so eloquently here in the front row. It simply does not make sense to remove one avenue of research from the equation, especially one that 

is relatively well developed. We should continue pursuing all forms 

of ethical research. 

It makes me particularly angry when people try to claim that 

adult stem cells can substitute cures for diseases for which adult 

stem cells have shown no clinical promise whatsoever. I know that 

these wonderful patients who are here today who have been cured 

by adult stem cells, mostly for blood-related diseases, would never 

say that somebody with diabetes or somebody with Parkinson’s or 

somebody with nerve damage or somebody with macular degeneration, all diseases for which embryonic stem cell research has shown 

promise and adult stem cells have shown no clinical promise, no 

one would say those people should not be cured, and that is the 

whole issue here today. I pray every day that my 14-year-old 

daughter will be cured of diabetes and I frankly don’t care if she 

is cured by embryonic stem cell research or adult stem cell research or ethical somatic cell nuclear transfer. I don’t really care 

and I don’t think the rest of the parents in this country care either. 

But what we do need to do as a government is we need to take 

our responsibility seriously and we need to say we are going to expand this research in an ethical way, we are going to make a national commitment to doing it, and we are not going to play politics 

with it. That is why I want to introduce and congratulate my 

friend, Mike Castle, who has snuck into the back of the room, who 

has been my compadre and fellow fighter on this issue. Mike and 

I are developing new legislation which I hope this hearing will help 

us begin to get evidence for, and what we believe our new legislation should do is obviously lift the ban on Federal funding for research on embryonic stem cell lines developed after August 2001, 

construct a framework for ethical oversight of all cell-based research developed by the National Institutes of Health and with the 

10 

NIH as a key player, and make the national commitment to this 

research that we should have had for the last 10 years. We expect 

to be introducing this legislation soon and are looking forward to 

input from the experts in the field. 

And just one last note, Mr. Chairman. Absent in this whole discussion today and absent in the Bush Administration’s national 

discussion is the fact that there is no Federal ethical oversight over 

the research that is going on either among the States with the limited Federal dollars that are available right now or perhaps most 

disturbing to me, with private entities that are doing the research. 

We need to both make the commitment to all ethical cell-based research but we also need to make the commitment to ethical oversight because some of this research is on the edge of bioethics and 

we need to make sure that we get it right for the patients of tomorrow. 

With that, Mr. Chairman, I appreciate your comity and I yield 

back. 

[The prepared statement of Ms. DeGette follows:] 

STATEMENT OF DIANA DEGETTE

Mr. Chairman, I want to thank you for holding today’s hearing on the future of 

stem cell research. Over the past year there have been many important developments in the field of cell-based scientific research. We saw breakthroughs and accomplishments that couldn’t have been predicted even months before they happened—insulin producing islet cells created from embryonic stem cells, induced 

pluripotent stem cells (IPS) developed from adult skin cells, and primate embryonic 

stem cells generated through somatic cell nuclear transfer (SCNT). All of this proves 

that one can rarely predict the outcomes of scientific research and underscores why 

it is crucial to make the investment in all ethical forms of research to begin with. 

This is what we are going to explore during this hearing: where we are now and 

where we are going with stem cell research. 

Everytime there has been a new discovery in some type of research besides embryonic stem cells, the Bush Administration tries to claim that it is a substitute for 

embryonic stem cell research. Yet, in actuality the numerous types of cell-based research are all complementary—they aide future developments or provide the background necessary for some yet-to-be-discovered breakthrough. It simply does not 

make sense to remove one avenue of research from the equation—we should continue pursuing all forms of ethical research and see where the science takes us. 

It is important that we still pursue embryonic stem cell research, for example, 

since it remains the most promising avenue of research for certain debilitating diseases like diabetes, Parkinson’s, and Multiple Sclerosis. However, there is still plenty to learn about both embryonic and induced pluripotent stem cells. Embryonic 

stem cells, as the vast majority of scientists agree, are currently the gold-standard 

for stem-cell research, and are the basis upon which to measure the success of IPS 

cells. The goal of IPS cell research is to make them mimic embryonic stem cells. 

But, how are we ever going to know whether the IPS cells are acting like embryonic 

stem cells if we haven’t done enough research on embryonic cells to even know what 

we are looking for? 

None of the recent progress in the adult stem cell field would have even been possible without the original embryonic stem cell research. Looking forward, we simply 

do not know where the advances will come from for each of the many diseases that 

we need to address-we do not know which will come from embryonic stem cell research and which will come from IPS research. We need to support both embryonic 

stem cell research and IPS research and let the science decide which is more promising over the long-run. 

We do not yet fully know what the recent IPS stem cell breakthrough means in 

terms of application. It seems as though it will likely prove to be a significant scientific advance. However, we do not yet know whether it will prove to be a significant medical advance. For example, IPS cells currently remain far too dangerous for 

actual treatment, and we do not know whether they will ever be safe for humans. 

Cutting off funding for other promising avenues of research in the meantime would 

be about the most short-sighted things we can do. When we develop new tools, we 

11 

don’t throw out the old ones that still serve a valuable and unique purpose. Why 

should it be any different when it comes to medical research? 

Although we are making great progress in the field of stem cell research, it has 

not progressed as far as it might have had the Administration instituted a cohesive 

federal policy for ethical oversight of stem cell research, rather than simply banning 

the use of federal funding for research on embryonic stem cell lines developed after 

August 9, 2001. Progress has been even further hindered because of inadequate resources for all research at NIH. 

With all the new research coming down the pipeline, much of which we have yet 

to even imagine, it is clear to me that we need a comprehensive, ethical oversight 

framework for all cell-based research, as well as a national commitment to a robust 

research program in the United States. 

So, in light of these issues, I have been working to develop new stem cell legislation with my dear friend Mr. Castle, who was kind enough to join us here today. 

We know that NIH is best-suited to overseeing and coordinating all forms of ethical 

stem cell research. It is best positioned to ensure that all research meets high ethical standards, as it has long experience overseeing cutting edge research and establishing regulations that ensure the research is done ethically. So, the new legislation 

will: 

• Construct a framework for ethical oversight of all cell-based research, with NIH 

as a key player; 

• Ban certain unethical activities, 

•Lift the ban on federal funding for research on embryonic stem cell lines developed after August, 2001. 

Input from the experts in the fields is key to crafting quality legislation, which 

is also part of the reason we are holding this hearing. I look forward to a vigorous 

discussion here today with our witnesses about where the science is currently, 

where the science is likely to go in the future, and what we, as federal lawmakers, 

should do in order to best support and promote all the promising new research that 

our scientists are working on. 

Thank you, Mr. Chairman. I yield back the balance of my time. 

Mr. PALLONE. Thank you. 

The subcommittee will stand in recess until the votes are completed, about an hour, maybe a little less. 

[Recess.] 

Mr. PALLONE. The subcommittee will reconvene. We were I guess 

longer than we expected. We left off with Congresswoman DeGette, 

and next I recognize the gentlewoman from Tennessee, Ms. Blackburn. 

OPENING STATEMENT OF HON. MARSHA BLACKBURN, A REPRESENTATIVE IN CONGRESS FROM THE STATE OF TENNESSEE 

Ms. BLACKBURN. Thank you, Mr. Chairman. I thank our witnesses for their patience today. As the chairman said, we were a 

little longer than we had anticipated being, but we do appreciate 

that you are here. We are looking forward to what you have to say. 

We all know that embryonic stem cell research continues to be 

a controversial issue. In my opinion, it does implicate ethical and 

moral standards within scientific progress and has the potential to 

offend millions of our constituents. It is my understanding that no 

journals have shown any treatment trials in human beings to have 

been successful using embryonic stem cells but there has been successful stem cell research, most definitely yes, from adult stem 

cells. In almost all cases, adult stem cells are equivalent or superior to embryonic stem cells and there are plenty of sources of adult 

stem cells, amniotic and placental fluid, cord blood, bone marrow— 

and none of these sources require any destruction of precious 

human embryos. 

12 

But many organizations continue to push for funding for embryonic stem cell research, claiming that it is the holy grail for cures 

of many diseases. One particular disease that is touted for support 

of embryonic stem cell research is diabetes, but since 2002, published studies in stem cells in diabetes journals concluded that 

trials using these cells showed no cures, and most of the time the 

treatments resulted in tumors, and I hope we will hear a little 

more about that. 

Significant progress, however, has been made on treating diabetes with adult stem cells, and since 2003, studies in the same journal showed adult stem cells successfully treated diabetes in mice, 

and when human trials conducted in Brazil and Europe began to 

use adult stem cells for treatment, many of the patients were insulin free after the stem cell transplant. The Federal Government 

should not be funding research that is showing no results and forcing Americans to pay for research that requires the destruction of 

human embryos, research that offends their moral and ethical sensibilities. Adult stem cells have a proven track record, and the NIH 

should be focusing, in my opinion, much of their research effort on 

this. I urge my colleagues to consider what is laid before us today, 

to ask good questions and to inquire about science that actually 

works and shows results. 

Thank you, Mr. Chairman. I yield back the balance of my time. 

Mr. PALLONE. Thank you. 

Next is the gentlewoman from Wisconsin, Ms. Baldwin, recognized for an opening statement. 

OPENING STATEMENT OF HON. TAMMY BALDWIN, A REPRESENTATIVE IN CONGRESS FROM THE STATE OF WISCONSIN 

Ms. BALDWIN. Thank you, Mr. Chairman. I really appreciate the 

fact that you are holding this very important hearing today. 

I am a strong supporter of embryonic stem cell research. I am 

fortunate to represent the University of Wisconsin, Madison campus, where Dr. Jamie Thomson and his team were the first to derive and culture human embryonic stem cells in a lab, and I have 

had the opportunity to tour Dr. Thomson’s lab and review the work 

that happens in that lab, and the field is truly groundbreaking. 

Embryonic stem cells open the possibility of dramatic new medical treatments, transplantation therapies, and cures, but at 9 p.m. 

on August 9, 2001, the hope and promise of this embryonic stem 

cell research was greatly curtailed by this Administration’s restrictions on the Federal research dollars for embryonic stem cells. The 

President’s policy that limits Federal funding for embryonic stem 

cell research to those stem cell lines that were created before a certain time and date is arbitrary and irrational, and it needlessly ties 

the hands of our scientists as they search for cures and treatments 

to diseases and conditions like diabetes, Parkinson’s disease, Alzheimer’s disease, and spinal cord injury. It also sends a very negative message to young, upcoming scientists that this is not the field 

to enter if you hope to secure Federal grant funding to support 

your research efforts. 

But despite the President turning his back on the promise of embryonic stem cell research, I am pleased that many States, univerVerDate Nov 24 2008 16:13 Mar 16, 2010 Jkt 037690 PO 00000 Frm 00018 Fmt 6633 Sfmt 6633 C:\DOCUMENTS AND SETTINGS\CWELLS1\HEARINGS\110-115 SCOM1 PsN: JIMC
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sities and private research foundations have stepped in to fill that 

role and the research has continued. Late last year, the same Dr. 

Thomson that I referenced earlier announced that he had discovered a way to reprogram skin cells into stem cells that seem to act 

like embryonic stem cells. While this development is very exciting, 

we must continue to support embryonic stem cell research and explore all the possibilities that this science holds. Whether we are 

talking about embryonic stem cells, adult stem cells, cord blood 

stem cells, or these new reprogrammed cells, we must explore all 

avenues of research. We owe it to the millions of Americans who 

suffer from diabetes, Parkinson’s disease, paralysis, and countless 

other conditions to realize the potential of all of this research. 

And I just want to close by associating myself with Congresswoman DeGette’s frustration, she said anger, over the confusion between adult and embryonic stem cells and the arguments that have 

been proffered. These stem cells have different properties. I can’t 

say with scientific accuracy that it is like comparing apples to oranges but I can say that we need to clarify the properties and why 

we need to pursue both lines of research, and I hope that our expert witnesses will help educate the members of Congress on this 

committee on the different properties that those stem cells have. 

Thank you, Mr. Chairman, and I yield back. 

Mr. PALLONE. Thank you. 

Mr. Ferguson of New Jersey. 

OPENING STATEMENT OF HON. MIKE FERGUSON, A REPRESENTATIVE IN CONGRESS FROM THE STATE OF NEW JERSEY 

Mr. FERGUSON. Thank you, Mr. Chairman. Thanks for holding 

this hearing. 

I am sure many people are aware, as we have heard already, 

that there have been great strides that scientists have been making 

in the past several years in stem cell research, in treating and even 

curing patients that have life-altering diseases. Research has produced very exciting developments such as the development that 

Ms. Baldwin was talking about, the induced pluripotent stem, or 

iPS, cells, which are derived from nonpluripotent cells by inserting 

genes to create the pluripotent stem cell. In 2006, Shinya 

Yamanaka of Kyoto University published the first article concerning iPS cells in mice, and 16 months later, his group and a 

group led by, as was said, Dr. James Thomson at the University 

of Wisconsin-Madison, reported the creation of human iPS cells. 

But I have to say, as Dr. Thomson himself has said, if human 

embryonic—I quote, ‘‘If human embryonic stem cell research does 

not make you at least a little bit uncomfortable, you have not 

thought about it enough.’’ He is right. And fortunately, there are 

better alternatives. There are more promising alternatives. There 

are alternatives that are showing treatments and progress in 

human beings today. Carol France is sitting in front of us. She suffers from multiple myeloma. Five years ago, my mother died from 

multiple myeloma. When she was first diagnosed at age 52, she 

was told she probably had a year to live. She lived 6 years because 

she had a similar treatment that is extending Carol’s life today. 

One of our children was able to—when my mother was first diagVerDate Nov 24 2008 16:13 Mar 16, 2010 Jkt 037690 PO 00000 Frm 00019 Fmt 6633 Sfmt 6633 C:\DOCUMENTS AND SETTINGS\CWELLS1\HEARINGS\110-115 SCOM1 PsN: JIMC
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nosed, she had no grandchildren. Three of our kids were born in 

the 6 years that her life was extended because of this stem cell 

treatment, not an embryonic stem cell treatment where there are 

no treatments, no humans that are benefiting from that today, but 

a treatment that is benefiting Carol and countless other people 

today, not just in cancers, but yes, there is progress in Parkinson’s 

disease. Yes, there is progress in diabetes as was shown in the Brazilian study. It is true. 

So when we are looking at where we spend scarce taxpayer dollars on Federal research, let us look at what is working, where the 

promise is, and not spinning our wheels going elsewhere. You 

know, I think citizens are rightly concerned about where their tax 

dollars are going, and in fact, my home State, the chairman and 

my home State of New Jersey, just last year, in New Jersey, embryonic stem cell research is done privately. We don’t even have a 

law against human cloning in New Jersey so we are pretty socalled progressive State when it comes to scientific research. But 

last year, voters in our State rejected a $450 million embryonic 

stem cell research center. Now, in the State of New Jersey, a ballot 

test hasn’t been defeated in 17 years, and in fact, there was another ballot question on the ballot at the same time that would 

have funded something else that passed. This is the only one in 17 

years that failed. I think voters and citizens as they look at the scientific evidence, I think as they look at the progress and they see 

the great progress of adult stem cell research and the people that 

it is benefiting today and they look at the alternatives, I think they 

are seeing that our—the question is not what is legal, the question 

is, where should we be spending taxpayer money? Where are we 

going to get the most bang from our buck? And I think people are 

beginning to see more and more clearly, particularly because of the 

research of Dr. Thomson and others, that there are very promising, 

very ethical opportunities for this research and we don’t have to go 

down a route that frankly has a lot of the ethical baggage that embryonic stem cell research has. 

Thank you, Mr. Chairman. I yield back. 

Mr. PALLONE. Thank you, Mr. Ferguson. 

Our vice chair, Mr. Green, recognized for an opening statement. 

OPENING STATEMENT OF HON. GENE GREEN, A 

REPRESENTATIVE IN CONGRESS FROM THE STATE OF TEXAS 

Mr. GREEN. Thank you, Mr. Chairman. I am shocked that New 

Jersey doesn’t have a ban on human cloning. 

Mr. FERGUSON. Me too. 

Mr. GREEN. My concern is that some of my colleagues may want 

to put that ban in effect in Texas and we would have no Texans 

that sound like me here. 

But be that as it may, there is not anyone in this room or in our 

country who has a friend or family member or a neighbor that 

hasn’t suffered from diabetes, Alzheimer’s, or Parkinson’s disease 

or a spinal cord injury, and how difficult that struggle is. But the 

issue, and you hear it today, the diversity of opinion is we can do 

what we need to do with adult stem cell research and there has 

been some great strides, but there is a substantial difference between adult stem cells and embryonic stem cells, and that is why 

15 

we need both. We don’t need to say we can only do it with adult, 

and that is what frustrating about this debate. 

Embryonic stem cells can actually divide indefinitely and evolve 

into any cell type in our body, and that is the big difference. We 

need to research it all and not just artificially say we are not going 

to do something, and that is what is frustrating. I have seen poll 

after poll the last number of years since the President set his criteria that 70 to 80 percent of the people support embryonic stem 

cell research, just because why we would put our head in the 

ground when we shouldn’t—when there is some potential for that. 

And I would hope the next Congress, if not this one, would pass 

the legislation again because it has been overwhelmingly passed in 

the House and the Senate, obviously not enough to override a veto, 

but hopefully we will pass it during the next Congress, if not this 

one. 

Mr. Chairman, I would like to ask my statement be placed in the 

record, and thank our witnesses for their patience for all our votes 

we had on the floor. 

[Mr. Green did not submit a prepared statement for the record.] 

Mr. PALLONE. Without objection, so ordered, and I think we have 

completed our opening statements, so we will turn to our witness, 

who has been waiting patiently here for 2 hours or so. 

First of all, welcome. Dr. Elias Zerhouni is director of the National Institutes of Health, and we appreciate your being here 

today. We have 5-minute opening statements. They become part of 

the hearing record, and you may in the discretion of the Committee 

submit additional statements in writing for inclusion in the record, 

and I now recognize you for 5 minutes. Thank you for being here. 

STATEMENT OF ELIAS A. ZERHOUNI, M.D., DIRECTOR, 

NATIONAL INSTITUTES OF HEALTH 

Dr. ZERHOUNI. Well, thank you, Mr. Chairman. It is worth waiting 2 hours to discuss stem cell research, and thank you, members 

of the subcommittee. 

I am really pleased to appear before you today to testify about 

the current state of stem cell research science and its significance, 

its current prospects, and its likely future. But let me start by saying that from the scientific standpoint, this is one of the most important, if not the most important, areas of medical research today. 

It has the potential to not only treat millions of individuals but also 

allow us to discover some of the fundamental findings and discoveries that we need to make in this century if we are going to be 

effective as a society in lessening the burden of disease. 

The central issue which I would like to go over in my oral statement and submit my total written statement for the record is the 

significance of this research from the standpoint of science. Why is 

it important that stem cell research be pursued very aggressively? 

I have a panel that I would like to share with you and I think we 

have distributed copies of that to each member. But let me just tell 

you what the real mystery is for us as doctors or scientists. It is 

the mystery of how DNA, which is exactly the same in every one 

cell of our body, goes from what we call a totipotent cell with the 

exact same DNA, to then form a complete organism with over 260 

different cell types in what we know as ourselves. This is a fundaVerDate Nov 24 2008 16:13 Mar 16, 2010 Jkt 037690 PO 00000 Frm 00021 Fmt 6633 Sfmt 6633 C:\DOCUMENTS AND SETTINGS\CWELLS1\HEARINGS\110-115 SCOM1 PsN: JIMC
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mental mystery that we need to unravel in this century. Why? Because we know also that DNA has been sequenced. We know how 

the DNA code is written. We know all the letters of the DNA code. 

What we do not know is how it is played, how it is programmed. 

So we know the hardware of how cells do this; we don’t know the 

software. And the whole field of stem cell research cannot be separated from our standpoint into components of adult or intermediate 

because they are all part of the same continuum, and let me explain that for you. Clearly, when a totipotent cell evolves, it plays 

a program, a program of molecular factors that are timed to change 

the characteristics of the DNA and how the DNA is played out. 

That then leads to a pluripotent cell. That pluripotent cell has a 

very interesting characteristic. It can self-renew. It can stay, in 

other words, idle until it goes forward in development and then can 

create through a second set of programs a program to create three 

precursors of our body systems. One is a line called the endodermal 

line. The internal organs, the guts, for example, arise from that 

line. The second is the mesodermal mid layer which really gives 

rise to muscles and bones and heart and blood. And then there is 

the ectodermal line, the epilayer, the outer layer, which gives rise 

to the nervous system and all of the neurons and all of the superficial layers of the skin. 

Now, we know that we can evolve a pluripotent cell into one of 

these, and this is the discovery that Jamie Thomson was credited 

for, finding that in fact you can cultivate these pluripotent cells, 

these embryonic stem cell lines, and then program them in different directions. This is where the research has been very active. 

Now, as we also learned, this is not the only program that is 

played. You still need to go from this line, from this cell, for example, the mesodermal precursor, and then you go through a different 

series of what we call adult stem cells. So you may have adult stem 

cells through multiple programs, many of which are completely unknown to us. We know some; we don’t know many of them. And 

then these will then give elements of the blood, for example, the 

white blood cells or the muscle, the deep layers of the skin, the 

skin fibroblast. 

Now, why is it important to understand that when we talk about 

adult stem cells, embryonic stem cells, committed precursors, it is 

very important to understand that this is a whole, that in fact, 

when we look at embryonic stem cell research, what we are looking 

at is to look forward in the programming from a totally 

unprogrammed cell to a fully programmed cell. Now, adult stem 

cells are partially programmed cells, which are able to evolve into 

different end points. Now, the therapies in adult stem cells have 

been developed for over 40 years, and the first one to be developed 

was the idea of replacing the bone marrow in patients who had 

blood cancers like myeloma or leukemias and so on, and the idea 

was to eradicate the cancer cells and then fish from the bone marrow some of these stem cell precursors to replace the bone marrow 

in a healthy way. 

So for most of the past 40 years, we have used that therapy to 

treat many cancers, and over the past 10 years there has been another line of research, which is to replace the immune system. We 

have many autoimmune diseases—multiple sclerosis, type 1 diabeVerDate Nov 24 2008 16:13 Mar 16, 2010 Jkt 037690 PO 00000 Frm 00022 Fmt 6633 Sfmt 6633 C:\DOCUMENTS AND SETTINGS\CWELLS1\HEARINGS\110-115 SCOM1 PsN: JIMC
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tes, lupus, scleroderma—and so doctors have had the idea of using 

the technique that was developed for cancer to use it to treat autoimmune disease where your own immune system goes awry and attacks your own tissues. So the idea there is to change that immune 

system, actually destroy it with radiation and chemotherapy, and 

replace it with a healthy bone marrow precursor that would then 

replace that. So fundamentally, if you think about the central 

issue, the central issue is, how is the software of DNA organized? 

We know the hardware; we don’t now the software. How do we discover how that is organized in health and disease is the central scientific question. 

Now, when you look at this, as you know, scientists have been 

looking at all angles of this research, and two things happened between 2001 and today as we were able to fund for the first time 

embryonic stem cell research. Researchers tried to look for what is 

it that makes a pluripotent cell a stem cell, and what they started 

to describe are DNA factors, genes, that were active at that time 

and then they defined culture conditions which allowed those cells 

to expand. Now, the thing that is very important to understand is 

that embryonic stem cells can be expanded many times and adult 

stem cells, up to today are not something that is frequent in the 

body and that we can expand as well as we do embryonic stem 

cells. So researchers have been thinking, can we create a new 

source of pluripotent stem cells, and this is the discovery that Dr. 

Yamanaka made, Dr. Thomson. Dr. Daley, who is one of your witnesses today, also showed the same thing, and that is that you can 

take a skin fibroblast and with these same factors that were discovered during embryonic stem cell research, apply them to a fully 

programmed cell, and lo and behold, you can deprogram the cell, 

erase the program, the software that was there and bring that cell 

back to what seems to be the exact same potency as the stem cell. 

It looks very similar but we know already they are not identical. 

But they have the same potential of being reprogrammed into the 

first three precursors. Now, here is another important issue, and 

that is that if you were able to cross-program these cells from a 

blood cell to a neural cell to a pancreatic cell, you would have made 

a great breakthrough. To this date, we have absolutely no evidence 

that once you have a precursor, you can reprogram it. 

So in summary, what I would like to say is that from the scientific standpoint, adult stem cell research, embryonic stem cell research, and induced pluripotent stem cell research are the faces of 

the same coin. They are intrinsically interrelated. They are related 

to the fundamental program of learning how to program, reprogram, deprogram DNA so that we can use these cells for therapies. 

So I will stop here, Mr. Chairman, and I would be happy to take 

your questions. 

[The prepared statement of Dr. Zerhouni follows:] 

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Mr. PALLONE. Thank you, Doctor, and we have questions now. I 

will start and recognize myself for 5 minutes. 

My colleague—this is just a quick one. My colleague, Representative Blackburn, commented in her opening statement that embryonic stem cells have not produced any results and that adult stem 

cells have shown more promise. You know, can you just respond to 

that? I mean, just in general. 

Dr. ZERHOUNI. I think it is correct that if you look at clinical applications, because we started in adult stem cells a long time ago, 

1956 was the first animal bone marrow transplant—we have 

learned a lot more about this and how to use that in many other 

diseases, primarily in two conditions: cancer and autoimmune diseases. Most of the diseases that are today helped by adult stem 

cells fit into these two categories. So it is absolutely clear that it 

takes about 17 years for the development of an idea to the first 

trial. We have had a lot more time in adult stem cells, a lot more 

funding—— 

Mr. PALLONE. But what about the promise of the embryonic? In 

other words, she said they haven’t produced any results but is 

there still promise out there for embryonic? 

Dr. ZERHOUNI. I think absolutely. I think that it is true that if 

you look at the snapshot of today, that we have made more clinical 

applications available. If you look at the scientific question, as I described, discovering the program that will make those things happen, it is very premature to say that one has promise and the other 

one doesn’t. 

Mr. PALLONE. Now, one of the witnesses—I hate to do this when 

I ask you about something the next panel is going to say before 

they have said it, but one of our witnesses on the next panel, Dr. 

John Fraser, asserts, and I quote, ‘‘that increasing funding to embryonic stem cell research means a decrease in funding to other 

stem cell research. Increasing funding to embryonic stem cell research at the expense of funding adult stem cell research means 

that valuable clinical opportunities that are serving patients today 

and others that appear on the cusp of doing so will be sacrificed 

for a technology and approach that while scientifically interesting 

contains enormous obstacles before responsible clinical application 

can be contemplated.’’ Did you want to comment on that as well? 

Dr. FRASER. That has been removed from my testimony. That is 

an old version that is not part of my testimony today. 

Mr. PALLONE. All right. Let me say for the record that I appreciate what you said, but his comments are not part of the record 

until he gets up here and testifies later. But if you would just— 

all right. Let me—it is a little bizarre. You are saying you didn’t 

say this? 

Dr. FRASER. I am saying that I amended—the document that I 

sent was amended, and you have an older version. 

Mr. PALLONE. Oh, OK. Well, you can comment on the older 

version then. 

Dr. ZERHOUNI. I have to tell you, I think it is premature to make 

statements as to the ultimate potential of one or another. It is all 

interconnected. It is all the same problem. I don’t know where the 

breakthroughs are going to come from, and if I don’t know, then 

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29 

sequences of doing that. There are ways of doing it ethically, and 

I think we need to really think about those. There is no doubt that 

our scientists are just as concerned as anybody else in finding solutions that are ethical, but I think we can’t just completely shut a 

door with the knowledge that we have today. As the director of 

NIH, we do not know enough to know where to stop, when to stop 

one kind of research or another. 

Mr. PALLONE. OK. If we could just stop the clock a minute, I just 

don’t want the reporter to have difficulty. We have never had that 

before in my experience where somebody talked who wasn’t part of 

the panel. Are you able to handle that? 

The REPORTER. Yes, sir. 

Mr. PALLONE. OK. So you have his comment, both of his comments? 

The REPORTER. Yes, sir. 

Mr. PALLONE. Then let me ask you, let us go back, Dr. Zerhouni. 

Can you explain to me the significance of this date, August 9, 2001, 

that the President has chosen? You know, he says no Federal funding for research on stem cell lines derived after August 9, 2001. 

What is the significance of the date? I mean, does it relate in any 

way to research or the scientific evidence? 

Dr. ZERHOUNI. I remember that the Federal Government could 

not fund any research deriving embryonic stem cells because of the 

Dickey-Wicker amendment. There is an amendment on the books 

which prevented NIH to fund any embryonic stem cell research deriving embryonic stem cells. The President made a decision to allow 

research to proceed and be funded for cell lines that had already 

been derived so that there would be no further destruction of embryos. That is what I understand the logic of the decision to be. I 

wasn’t involved in the decision. But the 2001 date was a date 

which the President made a decision to fund what was developed 

prior to this, including Dr. Thomson’s lines and so on and many 

others, but not any further. 

Mr. PALLONE. But there wasn’t any scientific significance to the 

date? 

Dr. ZERHOUNI. No, I don’t think that the decision was based on 

purely scientific considerations. 

Mr. PALLONE. OK. Now, do you believe that NIH is in danger of 

falling behind other countries with respect to biomedical research 

due to the restrictions that are based on that August 9, 2001, date? 

Dr. ZERHOUNI. It is very difficult to state categorically one way 

or the other. There is no doubt that about 50 percent of all the research that is published is currently published with results that 

are coming from NIH funding of this research. But there is no 

doubt that the rest of the world is also advancing. Fifty percent is 

published by the rest of the world. So I don’t think that it would 

be—it is hard to predict but I don’t think it would be in our best 

interests, if you will, to not continue to proceed in understanding 

the DNA programming, reprogramming issue that I think is core 

to biology in the 21st century. 

Mr. PALLONE. Thank you. 

Mr. Pitts. 

Mr. PITTS. Thank you, Mr. Chairman. 

30 

Thank you, Dr. Zerhouni, for your testimony. First, how much 

NIH funding has gone toward human adult stem cell clinical trials 

beyond bone marrow transplants? 

Dr. ZERHOUNI. Total funding for human non-embryonic stem cells 

is $203 million. 

Mr. PITTS. Does that include the bone marrow transplants? 

Dr. ZERHOUNI. I would think it does on all applications of adult 

stem cells. 

Mr. PITTS. How much funding has NIH provided for human embryonic stem cell research and animal embryonic stem cell research? 

Dr. ZERHOUNI. On a yearly basis, $203 million is a yearly number. We have been funding human non-embryonic stem cells at 

about $203 million, human embryonic stem cells at about $41 million a year, and non-human embryonic stem cells probably $150 

million but I will check that number for you. 

Mr. PITTS. How much NIH funding has gone toward the new 

human iPS research? 

Dr. ZERHOUNI. The new iPS research, if you looked at many of 

the funding, for example, Dr. Daley, who is here, was funded by 

NIH as a Pioneer Award winner from the NIH, but the total before 

the discoveries were made is about $4 million. But we have, as you 

know, launched a program to encourage this area of research. and 

we are currently looking at proposals. It is a recent discovery, so 

you couldn’t fund it as much until it was discovered. 

Mr. PITTS. Now, how many vials of stem cells does NIH have 

available? 

Dr. ZERHOUNI. I don’t know the exact number but I can tell you 

that we have shipped about 1,400 vials of human embryonic stem 

cells from our stem cell bank. I don’t know how many are available 

in the stem cell bank. 

Mr. PITTS. Have you ever turned down requests for a sample of 

the approved lines due to lack of availability? 

Dr. ZERHOUNI. I am not aware of that, but I know that scientists 

will tell you that there are lines that they wish not to use because 

there have been changes in the quality of those lines. So they tend 

to use fewer lines than all 21 lines because some of them don’t necessarily function as they wish. 

Mr. PITTS. Of the approved lines, how many have not yet been 

developed for research? 

Dr. ZERHOUNI. So we had initially 71 unique derivations, and 

about 21 have been developed and expanded and are available for 

research. About the same number were attempted to be developed 

but failed. The failure rate is quite high in developing these lines. 

And there are about 25 or 30 which have not been developed, have 

not been expanded for various reasons. 

Mr. PITTS. Is it possible that some of those lines were not developed on mouse feeder cells? 

Dr. ZERHOUNI. It is possible. Most of the—all the lines we have 

currently expanded have been developed on mouse feeder cells, 

which was the technology at the time. 

Mr. PITTS. Do you have any idea of how many were not developed on mouse feeder cells? 

31 

Dr. ZERHOUNI. I think we know that the Goteborg University in 

Sweden has 16 derivations which have not been developed at all 

and are attempted to be developed on human—on non-mouse feeder cells. 

Mr. PITTS. Now, you have stated before that the Bush-approved 

human embryonic stem cells are contaminated. However, Dr. 

James Thomson has stated that these cells can be washed and the 

contamination is not a problem. Are you aware of the study published by Dr. Thomson? 

Dr. ZERHOUNI. So we looked at that several years—I don’t know 

I declared that but we did look at this very carefully, and we have 

pointed out in testimony as well as in written statements that the 

fact that something is grown in mouse feeder cells makes applications much more difficult and FDA approval more difficult but not 

impossible. We do have other products like vaccines that have been 

developed in that way. So our testimony does not say it cannot be 

done, but it is a lot more difficult to do. 

Mr. PITTS. Now, you said you weren’t aware of any patient being 

successfully treated with embryonic stem cells. When is the soonest 

that you would anticipate clinical applications using embryonic 

stem cells? 

Dr. ZERHOUNI. The one current clinical application at the FDA 

is one by a company, Geron I think is the name, G-e-r-o-n, for 

using human embryonic stem cells for spinal cord injuries. That is 

the only one that is near clinical application, has not yet been approved by FDA for trials. 

Mr. PITTS. And how would treatments be affected by their propensity for tumor formation? 

Dr. ZERHOUNI. That is a problem you need to resolve before you 

can implant human embryonic stem cells. This is why most of the 

researchers working with human embryonic stem cells need to continue to work on these programs so that they can move the cell to 

a point where it will no longer develop a tumor. 

Mr. PITTS. Thank you, Mr. Chairman. I think my time is up. It 

is hard to see. 

Mr. PALLONE. You still have another 25 seconds if you want to 

use them. 

Mr. PITTS. Well, I will ask one more. Why did NIH not fund clinical trials for Harvard researcher Denise Faustman even though 

she reversed diabetes in mice and was FDA approved to start 

trials? 

Dr. ZERHOUNI. I am a little stumped on this one. I don’t know 

the details of this particular researcher and the particular trial. so 

I will get back to you on the record for that. 

Mr. PITTS. All right. Thank you, Mr. Chairman. 

Mr. PALLONE. Ms. DeGette. 

Ms. DEGETTE. Thank you very much, Mr. Chairman. 

Dr. Zerhouni, I want to ask you, you said in your opening statement that this type of research, the general category of cell-based 

research, is one of the most important, if not the most important, 

forms of research we can do going forward in the future. What is 

the entire NIH budget? 

Dr. ZERHOUNI. About $29 billion. 

32 

Ms. DEGETTE. Twenty-nine billion dollars. What is the total 

budget for the cell-based research including embryonic and non-embryonic? 

Dr. ZERHOUNI. About $655 million a year. 

Ms. DEGETTE. Six hundred and fifty-five million dollars a year. 

So I think probably if Congress were willing to authorize and appropriate a substantially higher research budget for all of these 

types of research, the NIH could probably find some people who 

would—some researchers who would be willing to take those grants 

and to make them into some promising discoveries, don’t you? 

Dr. ZERHOUNI. Definitely. 

Ms. DEGETTE. Do you think the NIH would need to have more 

research to really make this kind of—— 

Dr. ZERHOUNI. If we could have more resources, we could accelerate this research much faster. 

Ms. DEGETTE. And if we accelerated the research faster without 

predicting specific advances, what kinds of things do you think 

could happen? 

Dr. ZERHOUNI. Well, clearly, as I said, the scientific community 

is making rapid progress in understanding these factors, these molecular programming factors. Every week, every 2 weeks, we get a 

report of scientists, for example, developing a very potent capable 

line, both in humans as well as in animal systems. The question 

though is going to be, how fast can you do this. Now, this 

deprogramming advance, this breakthrough, happened because we 

learned of the factors that were in this first program. Now, we are 

going to learn more and go forward into this route, as we can fund 

scientists to do that. 

Ms. DEGETTE. Will the current embryonic stem cell lines, the 21, 

give or take a little, lines that still are allowed to be used with Federal dollars by federally-funded labs be sufficient to sustain this 

type of future research? 

Dr. ZERHOUNI. Scientists will tell you that they need access to 

more cell lines that are earlier in their history. What happens is, 

as you cultivate a cell line, over time it accumulates changes, both 

genetic changes and software changes, program changes, which 

makes a lot of scientists say I would rather have a cell which is 

early in this development right here so I can understand the—— 

Ms. DEGETTE. Not to cut you off but what you are saying is these 

cell lines that existed as of August 2001 are now getting old from 

a research standpoint and the researchers would like to have 

newer stem cell lines? 

Dr. ZERHOUNI. Many researchers can use them, they are using 

them, but many cannot. 

Ms. DEGETTE. Now, the way it works, both with approved lines 

at the NIH and also private researchers is, they take cell lines that 

are developed from embryos which were created for in vitro fertilization clinics and not used by the patients and then slated to 

be thrown away, correct? I mean, these embryos are—— 

Dr. ZERHOUNI. I would assume that is true, but I don’t know all 

the details of every case. 

Ms. DEGETTE. OK. I will ask the researchers. I wanted to ask 

you if you are familiar with this Brazilian diabetes study that some 

have referred to today, and whether or not in fact that study 

33 

showed U.S. researchers that diabetes was curable by adult stem 

cells. 

Dr. ZERHOUNI. I am familiar with that study, and this is the 

study that I think Congressman Pitts was mentioning. Actually the 

study was conceived by a researcher at Northwestern University 

and the idea there was this: type 1 diabetes is probably an autoimmune disease where your own immune system is destroying your 

own cells. So again, along the line of what I described where you 

use bone marrow stem cells, adult stem cells to replace the immune 

system. The idea then was, why don’t we use the treatment that 

was developed for cancer patients into young type 1 diabetes patients to prevent the destruction of their stem cells. When that 

science was reviewed by our ethics experts and by experts in bone 

marrow transplants, it was felt that this would be unethical because the mortality rate is 5 percent in these diseases. Now, you 

can take that risk when you are dealing with a cancer that has a 

life expectancy of a year, like leukemia, but the problem is, type 

1 diabetes is manageable today. We have patients who live almost 

normal lives. So the risk-benefit ratio as assessed by the ethical 

boards, the institutional review board said this isn’t something that 

should be started in children, we should start it in adults perhaps 

or with a different risk ratio and not go forward with—— 

Ms. DEGETTE. So that was never really in clinical trials in the 

United States, correct? 

Dr. ZERHOUNI. Not that I know of because of the ethics issues. 

Ms. DEGETTE. I have one last question. Right now does the NIH 

have ethical oversight over the embryonic stem cell research that 

is conducted at the State level or by private firms? 

Dr. ZERHOUNI. Well, as you know, we can only use Federal funds 

for the approved uses of embryonic stem cells so we cannot really 

have that oversight responsibility. I think that this is something 

that I wish common ground could be found over time. I think NIH 

has always played the harmonizing role and prevented in fact unethical uses as well as promoted the good use of science, so I would 

say that no, we do not, and I wish we did. 

Ms. DEGETTE. And do you think that the NIH would have the 

capability of developing such ethical oversight over cell-based research? 

Dr. ZERHOUNI. Definitely I think NIH should have an enhanced 

role in that. I think we have shown over the years that we can do 

this. We have regulated, for example, gene therapy through the Recombinant DNAAdvisory Committee for over 30 years. It has 

worked very well. And the same thing is true now with biosecurity 

issues. I think we have the talent and frankly, I don’t know of any 

other organization in the world that could do a better job than 

NIH. 

Ms. DEGETTE. Thank you very much, and thank you for joining 

us, Dr. Zerhouni. 

Dr. ZERHOUNI. Thank you. 

Mr. PALLONE. Mr. Deal. 

Mr. DEAL. Thank you. 

Dr. Zerhouni, let me first of all begin by thanking you for the excellent job you do in managing and directing NIH. I think political 

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34 

fortable with your leadership and your knowledge of issues as you 

expressed on one of the more difficult issue that all of us are confronted with, this one that this hearing is about today, and I continue to be impressed by your leadership and thank you for that. 

Dr. ZERHOUNI. Thank you. 

Mr. DEAL. Let me ask you about one aspect. I wasn’t here but 

I was listening to you over my computer in my office as I was doing 

some other things, and one of the things that is interesting, at 

least to me, and I wish you would expound upon it a little more, 

and that is the new human iPS research. Would you expound on 

that a little bit more? What is the degree of enthusiasm about this 

at NIH? Is it something that really has great potential, do you 

think? 

Dr. ZERHOUNI. I think it is one of the biggest breakthroughs in 

stem cell research in recent years. We are very excited about it. We 

want to explore it. Because the idea that you can take a cell that 

has gone through full programming and then using four factors, 

you can deprogram it to be able to do other things, that is a venue 

that is extraordinarily exciting. We are putting out requests for 

proposals. I know we have received 29 proposals just in the first 

submission, the majority of which are on iPS cells. And remember 

that iPS cells are not just to replace cells in your body. They are 

also tools to make progress in other areas. For example, if you have 

a patient with a disease and you developed a pluripotent cell from 

that patient, think about what you can do to discover new treatments, new drugs, new therapies. Pharmaceutical companies are 

very excited about this potential. You could reduce the toxicity of 

drugs that today hurt patients because of heart toxicity or liver 

toxicity. So you could create liver cells or you can create heart cells 

and test the drug in vitro and prevent the toxicity. So there are 

many more uses than just the typical we are going to replace neurons or we are going to replace diabetic cells, much more exciting 

than—and I have to commend the scientists. Remember that what 

they did is, they learned from embryonic stem cells and immediately applied it in a way that will allow us to all go forward without the concerns that many of us have about this research. 

Mr. DEAL. Well, thank you. I can see you have enthusiasm on 

this. 

Dr. ZERHOUNI. I surely do. 

Mr. DEAL. I think rightfully so, apparently. Although you do not 

control all of the research that is being done, especially on embryonic stem cell research, could you give us some idea from your perspective the magnitude of research that is being done that is not 

NIH-funded in this entire area? 

Dr. ZERHOUNI. If you are referring to embryonic stem cell research alone, we feel that, because of initiatives in several States 

that the rest of the country spends more than we do at NIH for 

the $40 million that we spend. If you look at the totality though 

of what we do in this entire spectrum that I described, which is 

really a continuum, it is all sides of the same coin. When we look 

at that, we spend $655 million total, which is higher than any 

other actor out there. California just this week announced a $225 

million investment in this type of research. So I would say that if 

you look at non-Federal sources, it probably equals the Federal inVerDate Nov 24 2008 16:13 Mar 16, 2010 Jkt 037690 PO 00000 Frm 00040 Fmt 6633 Sfmt 6633 C:\DOCUMENTS AND SETTINGS\CWELLS1\HEARINGS\110-115 SCOM1 PsN: JIMC
35 

vestment, but I can’t really tell you because I don’t know what is 

happening in industry or in private entities. 

Mr. DEAL. Well, Mr. Chairman, I am going to yield back my 

time, but again, thank you very much, Dr. Zerhouni, for being here. 

Mr. PALLONE. Thank you, Mr. Deal. 

Ms. Capps is recognized for questions. 

Ms. CAPPS. Thank you very much for your patience with our proceedings in the House, and I want to first of all say since I didn’t 

get to make an opening statement, how proud I am of the National 

Institutes of Health. I am bragging about all the—people know all 

the things that are wrong about Congress and I say but there is 

at least one good thing that is happening that really impacts lives 

in this country but it is also our biggest gift to the world that we 

are able to do all of that. That happens on the campus and other 

places as well. 

I have a lot of questions I would like to ask you but I want to 

start with one that was touched upon in the opening remarks, and 

someone else may have asked you this. One of the things we are 

clearly missing in the national policy on stem cell research is a 

standard that we need that can be provided for us in the way of 

ethical standards, an ethical framework. We have now seen in my 

State of California and other States and other private entities a lot 

of push forward because of the lack of support from the Federal 

government. That is in one of the best natures of our country as 

well. But what is clearly missing from all of this from my perspective, but I would like to learn from you, how do you regard the importance of an ethical framework to guide both private and public 

research and endeavors into all stem cell research? 

Dr. ZERHOUNI. Without harmonious and coherent oversight, 

which historically NIH has provided and is the best organization 

in the world to provide, you can see a world where different standards are going to be used. FDA will have real trouble finding out 

whether the research in California is more valid than the research 

in Washington or somewhere else. It will slow down progress for 

all stem cell research, not just embryonic stem cell research, because we need to characterize exactly what those cells do. There is 

the risk of tumor development. We need to control that. You cannot 

do that well at the speed you need to do it. It is hard enough when 

it is well overseen. It is, in my view, very shortsighted not to oversee it at the Federal level. 

Ms. CAPPS. So if we were able to pass legislation that authorized 

Federal involvement, it wouldn’t just be funding for research 

through NIH, it would also be to provide that ethical framework 

and guidelines for all of the research that is going on? 

Dr. ZERHOUNI. I think some common ground has to be found. I 

really believe it is in the best interests of our millions of patients 

and the best interest of our country to act in unison when it comes 

to ethical oversight of any area of medical research. 

Ms. CAPPS. OK. In whatever time I have left, and you may have 

touched on this before, but if there has been anything left out, 

there were efforts underway before 2001 and advances have been 

outside the Federal government’s purview, both through States and 

through private enterprise. What is missing apart from that ethical 

framework? What could be the contribution of providing funds for 

36 

research specifically through the Federal government? What would 

you do? 

Dr. ZERHOUNI. There was no Federal funding of human embryonic stem cell research before 2001. 

Ms. CAPPS. Oh, I know that. 

Dr. ZERHOUNI. And so we have had this 6 years experience of 

how to do this. I think what in my view would be very important 

is to get over in some fashion or another in a good way the issue 

of providing scientists with avenues of exploration with strong safeguards, strong ability for us to prevent some of the rightly scary 

scenarios that could develop. So we need to have that now because 

it wasn’t that important in 2001 since the science wasn’t advanced, 

but I can tell you, it is advancing at an enormous speed, and I 

think we owe it to ourselves to create a new framework to oversee 

this research over time. Now, it could be that you can separate 

funding from oversight. I mean, there are many ways that can be 

done, but we cannot just say stop this and do this and no oversight. 

Ms. CAPPS. So in this vacuum, you say that some dangerous or 

unintended consequence could be developing, putting some of our 

citizens at risk? 

Dr. ZERHOUNI. Well, let me just be frank here. 

Ms. CAPPS. Yes. 

Dr. ZERHOUNI. I get e-mails from clinics in various countries that 

do not have the oversight structure we do about promising treatments for stroke patients in the Dominican Republic, other treatments in countries that just don’t have the oversight infrastructure 

we have. I am very concerned. As a physician I am concerned. I 

know the despair of patients who need treatment, and that can be 

abused and used. We have this in cancer therapies, and we are seeing it in stem cell therapies. Why would we let our citizens go in 

an unregulated, not-overseen environment with the risks we know 

about this research and say, go ahead, it is much better there than 

it is here? It is not correct to say that, and I am very worried that 

there will be people harmed by this. 

Ms. CAPPS. So there is a moral component to this in terms of our 

leadership, and these are our citizens, many of them who are flocking to places because they have been promised certain things? 

Dr. ZERHOUNI. Absolutely. I mean, look, hope is hope, and as you 

know, we need to really understand that. 

Ms. CAPPS. Thank you. 

Mr. PALLONE. Thank you. 

Our ranking member, Mr. Barton, recognized for questions. 

Mr. BARTON. Thank you, Mr. Chairman. I will be very brief. 

I want to welcome you, Dr. Zerhouni. It is good to see you. I 

haven’t seen you in person in a while though we have talked by 

telephone several times. I know the purpose of today’s hearing is 

an update on stem cell research, but I can’t pass up the opportunity to ask you to give us a brief review of the NIH reform bill 

that this committee passed on a bipartisan basis at the end of the 

last Congress. Could you kind of tell us where that is and what, 

if anything, we need to do to help you implement it? 

Dr. ZERHOUNI. Well, first of all, let me thank you, Mr. Chairman. 

I think you have accomplished what NIH needed to have for many, 

many years. As you know, there had not been a reauthorization of 

37 

NIH for many years, and you have been able to do this with your 

colleagues on a bipartisan basis and I am very, very pleased and 

proud of the fact that both sides came together in authorizing the 

NIH Reform Act of 2006. 

The main impact of the reauthorization, in my view, is that it 

has institutionalized the concept that as science is becoming more 

complex, as science is also converging between different Institutes, 

the NIH Reform Act has allowed us to have cross-collaborations 

with a Common Fund so that no one is being taxed, if you will, for 

doing the right thing across diseases. Now, we know, as you just 

heard, that many diseases, for example, multiple sclerosis or diabetes, are treated with the same approach because they are all autoimmune diseases. Well, those diseases obviously are taken care of 

by multiple Institutes. So I would say that the fact that also in the 

same year, the bill passed in 2006 and the Joint Resolution of Congress, the appropriators then decided to fund the Common Fund as 

a separate entity so that Institutes will no longer have to contribute to that, I thought that was a great statement of support. 

We appreciate it, and I think that you will see results of that on 

a going-forward basis that I think you would be surprised at the 

change in the ability of NIH to address cross-cutting issues that go 

beyond any one Institute’s mission or Institute’s focus. 

Mr. BARTON. Well, I have asked Chairman Dingell to hold an 

oversight hearing where we could go into detail on it, so hopefully 

he will do that in the near future. 

Ms. DEGETTE. Will the ranking member yield? 

Mr. BARTON. Yes. 

Ms. DEGETTE. As the Vice Chairman of the Committee, I will tell 

you, and a big supporter of that bill, I also want to thank you, Mr. 

Barton, for that legislation. I think it has been great. And I have 

also spoken with Mr. Dingell about doing oversight hearings and 

I expect we will be doing that this year. 

Mr. BARTON. Anyway, it is good to see you, Doctor, and we will 

hopefully welcome you back soon to talk on some other issues. 

I yield back, Mr. Chairman. 

Mr. PALLONE. Thank you, Mr. Barton. I was going to say that 

now that you asked that question, we didn’t need to have the hearing, but I guess—I am just kidding. 

Next we have Ms. Baldwin recognized for questions. 

Ms. BALDWIN. Thank you, Mr. Chairman, and I want to add my 

words of support for the incredible and unique role that the NIH 

plays in the world and in the United States. Following on Ranking 

Member Barton’s comments, I feel like my constituents are beneficiaries in so many different ways, whether it is the results of the 

research that is funded, the funding that comes into research universities like the University of Wisconsin-Madison or in my own 

case having been raised by my grandparents, my grandfather was 

an NIH-funded scientist at the university, so I am a beneficiary in 

yet another unique way of NIH funding. 

I want to talk about a couple of things sort of pivoting off the 

questions that you have gotten about ethical concerns and needing 

to have a harmonious oversight process. There is another role that 

NIH plays, which is priority setting through the process of reviewing the grant proposals, and because a part of the overall stem cell 

38 

research that is not being conducted through NIH, I am wondering 

what comments you might have of the role that NIH plays in priority setting in this overall endeavor. 

Dr. ZERHOUNI. Well, again, I think as you have seen through history, NIH since 1945 has basically been the tempo maker for 

science in many ways. The first treatments, for example, for leukemia that changed the mortality in children from 95 percent to 5 

percent were done because of that process, and we need to continue 

to do that. So my sense is that the more we have an open understanding of how to run this forward, given the fact that it is getting 

closer to clinical applications, needs to be enhanced. 

Ms. BALDWIN. Now, I mentioned in my opening statement one of 

many concerns I have about the current funding policy for embryonic stem cell research is the message it sends to young, upcoming 

scientists in terms of what direction they should go in, but I am 

additionally concerned about the consequence of the current Federal policy because in many ways, it seems like we are maintaining 

two separate structures. I know in many research institutions, they 

have to build and equip two sets of labs, one that conducts NIHfunded research, a parallel, oftentimes a whole building is constructed and lab equipment is acquired. Do you have any sense of 

what sort of costs are involved in this sort of dual structure that 

is occurring all over the country? 

Dr. ZERHOUNI. In all fairness, NIH does not impose separation 

of physical facilities. We have been extremely clear that you cannot 

use Federal funds for unapproved uses but you can account separately within your own laboratory for that. It is difficult to do 

though. Most of our researchers say, you know, I don’t want to get 

in trouble, I would rather separate the two completely. 

Ms. BALDWIN. That has certainly been my reflection in my home 

community. 

Dr. ZERHOUNI. Right. So it is an impediment to the researchers, 

who really want to do risk management in the institutions. From 

our standpoint, we are satisfied with the accounting procedures 

that we have put in place, and we haven’t had a case where there 

has been a significant issue that we have been concerned about. 

But I think at the end, the institutions, our concern about that— 

in California, I know that the first $225 million are actually dedicated to building separate facilities. I know that this is a concern 

out there. 

Ms. BALDWIN. Lastly, we have had some discussion about how 

much funding has been devoted to the new iPS findings. Going forward, what sort of growth do you expect in terms of contributing 

funding to that? 

Dr. ZERHOUNI. I cannot be precise, but I can see exponential 

growth in the field of induced pluripotent stem cells for the reasons 

that I mentioned. One, it is much more practical, easier to do. It 

also highlights different ways of programming DNA, as I said at 

the beginning. It has multiple uses other than just the clinical use, 

because right now these cells are not ready for clinical use. They 

are generated using viruses that carry these factors, so we have to 

do more research on them to find a way to use them safely in the 

environment. But my sense is that already we know of many researchers—you are going to hear from Dr. Daley, who is a leader 

39 

in that field—many researchers, many applications, and researchers who are currently funded by NIH, redirecting their research to 

that area. So you will see—I think you will see major growth in 

that field. 

Ms. BALDWIN. Dr. Zerhouni, thank you very much for your time. 

Mr. PALLONE. Thank you. 

The gentleman from New Jersey, Mr. Ferguson. 

Mr. FERGUSON. Thank you, Mr. Chairman. 

Dr. Zerhouni, thank you again for being here today. We very 

much appreciate your testimony and your leadership at NIH over 

the years. I think you know that I personally am an admirer of 

yours and appreciate the dialogs that we have had over these 

years. 

Mr. Chairman, I just wanted to submit two things for the record 

that I referenced in my opening statement. One is the autologous— 

this is a Journal of the American Medical Association study published April 11, 2007, documenting the progress that has been 

made with adult stem cell research in type 1 diabetes. I can give 

this to you. I would like to submit it for the record, please. 

Mr. PALLONE. Without objection. 

Mr. FERGUSON. And the other was the quotation that I mentioned from Dr. James Thomson that was in an article in the 

Washington Post from November 30, 2007. I would like to submit 

that. 

Mr. PALLONE. Without objection, so ordered. 

[The information follows:] 

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Mr. FERGUSON. Thank you. 

Dr. Zerhouni, I think you are familiar with Celgene Cellular 

Therapeutics. They are a biotech company in my district in New 

Jersey. They do—really one of the leaders in stem cell research. 

They do really extraordinary work and they have developed a clinical application to create blood stem cells by using human placentaderived stem cells along with umbilical cord blood cells. The first 

application of this particular technology was completed at Louisiana State University Health Sciences Center, the Health 

Sciences Center at Children’s Hospital on March 28, 2008, just not 

very long ago. It was big news. They treated a pediatric patient 

who was suffering from acute lymphoblastic leukemia, which is a 

cancer of the bone marrow and the blood. 

Mr. BURGESS. I think he knows. 

Mr. FERGUSON. Yes, I am sure he knows, but I want to have it 

on the record. Thanks, Mike. 

I think I began my statement by saying I am sure he knows, but 

in any event, yet another example of remarkable progress and potential treatments that are coming from again not an embryonic 

stem cell source but a different source, in this case placental stem 

cells and cord blood stem cells. It just further highlights for me, we 

were talking about these essentially two different things and I 

mentioned before some of the sort of ethical questions obviously 

that are still out there, and you have referenced many times in 

your discussion about the ethical considerations as we look at these 

different types of research, and clearly I think we would all agree 

that there are things that we can do but ought not do in life, right? 

But that is really not the question that we are talking about here 

today. The question that we are talking about today is, should all 

things that we can do be funded using taxpayer money? That is 

really the question that we are getting at here today, and voters 

in New Jersey just last fall decided embryonic stem cell research 

was not something they wanted their taxpayer dollars to go to 

fund. So that was one opportunity for voters to be heard. But we 

have to have that conversation all the time certainly in this committee and this subcommittee. 

I wanted to pursue something you had talked about before, and 

clearly for many people, even obviously Dr. Thomson and others 

who have raised questions, certainly ethical considerations about 

embryonic stem cell research, and I am genuinely curious about 

this because I don’t know where this goes. If there are ethical concerns that some people, many people have about embryonic stem 

cell research, the nature of that research today, my question is, I 

guess I don’t have a good enough imagination or certainly not scientific expert enough to know, where does it go when perhaps, as 

you said, perhaps years down the road if some treatment or 

progress comes from embryonic research—I talked to the researchers in my district and in New Jersey about placental and stem cell 

and cord blood research, and one of the things they love about that 

kind of research is, when they come up with an application, as they 

seem to have, there is a virtually limitless supply of—I don’t know 

what you would call it—raw material, you know. How many children are born every day? How many placentas and cord blood, you 

know, we have a virtually limitless source of these cells. If we were 

51 

to get to that point some years in the future, as you have said, with 

embryonic research, where does the raw material come from? 

Dr. ZERHOUNI. You are asking extremely important and difficult 

questions. I think we absolutely do not want to obviate the need 

for a deep conversation. Likewise, scientists, as I described, believed that progress will come from our understanding at the deepest level of the molecular program that is timed to create the cells 

or create the appropriate neurons or that lead to understanding 

disease and eventually cure it. Most scientists when you talk to 

them, in my conversations with them, including Dr. Thomson, 

would say the picture 20 years down the line is that as we discover 

these programming factors, we probably won’t need any particular 

one source. We will program, if you will, the software of any cell. 

Am I talking about science fiction? No, it has already happened in 

front of us; with four factors we have reprogrammed skin fibroblasts. So I think the discussion will evolve, and you are right. Is 

there anything that we could do that should not be done? I just 

gave you an example of the autologous bone marrow transplants in 

young type 1 diabetes patients where we know that 1 in 20 will die 

within 100 days of having received that transplant. That is just as 

important a consideration as the other consideration that you referred to, which is what is the limit, what is the barrier here. We 

clearly as scientists—now, I am talking from the scientific point of 

view—if you understand that the problem for us is to truly advance 

the cures that we need to implement which are dependent on our 

understanding of DNA programming and reprogramming and how 

do you modulate that, the embryonic stem cell is just unique in the 

sense that it can self-propagate. If it wasn’t for that, I don’t think 

scientists would be as excited about it. The fact is, you cannot get 

cord blood cells to multiply the way you get an embryonic stem cell 

to, but that doesn’t mean it is not possible because we also are 

showing that it is doable. So, Congressman Ferguson, I know you 

have thought about this, and we have had these conversations. I 

don’t know where the happy medium is, but I know that we cannot 

close our eyes to the fact that the progress may come from any one 

of these sources. So NIH wants to fund all of those areas of research, whether it be cord blood or placenta, and we do. 

Mr. PALLONE. We are out of time, 3 minutes over. 

Mr. FERGUSON. If I could just close on that and respond, I think 

that is a very thoughtful answer. I appreciate it. The researchers 

that I have talked to in our district and in—— 

Mr. PALLONE. Mr. Ferguson, not for anything but we are 2 minutes over so we have to move on. 

Mr. FERGUSON. Sorry. 

Mr. PALLONE. All right. Ms. Baldwin? Oh, she is not here. Mr. 

Burgess. 

Mr. BURGESS. Thank you, Mr. Chairman. Let me just yield 30 

seconds to my friend from New Jersey to finish his thought. 

Mr. FERGUSON. That is extremely courteous, Dr. Burgess. Thank 

you. 

I would just say that the researchers that I have talked to in our 

district have raised that question with me, and these are all folks 

who agree, and I think everyone would agree that all types of research, particularly as we have seen embryonic stem cell research 

52 

and these other types of research, are extremely interesting, extremely interesting and potentially valuable. I think anybody who 

is being honest would have to acknowledge that they are potentially valuable. The question that we are struggling with here is 

not whether it should exist, not whether the embryonic stem cell 

research should exist. That is not the question that we are dealing 

with today or have been dealing with. The question is, where are 

we going to spend scarce taxpayer dollars? On the most promising, 

immediately beneficial examples and research or are we going to 

roll the dice on other forms? That is really the question that we are 

after today, so I appreciate it. Thank you, Dr. Burgess. 

Mr. BURGESS. You are very welcome. That is a rhetorical question. It doesn’t require a response. 

Let me ask you—Mr. Ferguson raised another very good point. 

What about just the volume of material that is going to be required 

to do the type of research or to provide the therapeutic benefit? 

There is a virtually unlimited supply of cells from amniotic fluid 

and cord blood and a relatively finite supply of human embryonic 

stem cells, regardless of whether or not any funding source is lifted. 

Dr. ZERHOUNI. Well, as you know, because of the new discoveries, 

we have sort of bypassed this issue of being able to expand the 

cells that we have through the induced pluripotent stem cells. 

There is no doubt that when you look at placenta or cord blood, we 

have—we are unable to take a cord blood sample and expand it to 

use in patients other than young children. 

Mr. BURGESS. But if I could interrupt you, what about the 

pluripotent cell from amniotic fluid? 

Dr. ZERHOUNI. Well, there is one documented work from Dr. 

Atala there and we are looking forward to see what the expansion 

potential of that is there, but there is no doubt that scientists will 

explore every door. The one thing that we don’t know is where the 

magic answer is. So everybody is really going to explore all of those 

avenues. We want to support them all. 

Mr. BURGESS. And we talked a little bit about funding, and I 

wish I had a great deal of time to spend on that, but as far as Dr. 

Anthony Atala’s work is concerned, is any of that supported by 

NIH funding? 

Dr. ZERHOUNI. Oh, definitely, yes. 

Mr. BURGESS. So he has an ongoing grant from NIH? 

Dr. ZERHOUNI. Oh, yes, I think he has had it for a long time. 

Mr. BURGESS. And reports are coming back to you so you are able 

to evaluate the work that is going on down in North Carolina? 

Dr. ZERHOUNI. Absolutely. We are keeping a close eye on our investments. 

Mr. BURGESS. And I am happy that you do. Let me just ask too 

on this, since Mr. Barton brought up the issue of the reauthorization and the $29.5 billion that was the baseline funding in the reauthorization bill and the increases were slated to be 5 percent per 

year. Were you able to get to that amount last year in the appropriations cycle through Congress? 

Dr. ZERHOUNI. No, and it is something that we will have to consider in the long term and look at the long-term impacts. I think 

the increases have been below inflation, and we have managed and 

53 

tried to reorganize our priorities, but they have not been at the authorization level. 

Mr. BURGESS. Correct, and the reason for that bipartisan reauthorization was to give you the certainty of that funding stream so 

that when you go out and hire young scientists to start new labs, 

you will know that you will be able to continue to fund that. I won’t 

ask you to be a prophet here but what do you intuit about this 

year’s appropriations cycle as far as the NIH is concerned? 

Dr. ZERHOUNI. I appreciate your point, Dr. Burgess. I think you 

are very aware. I know from our conversations that you know, 

based on your own experience, that young scientists make decisions 

not on the basis of today but on the basis of what they see coming, 

and as we send a message that is discouraging, there is a definite 

sense out there of young scientists deciding not to go into science. 

Mr. BURGESS. I would just remind those who are in the party in 

control that control now the appropriations process, we were criticized when we were in control for leveling off the funding for several years after a doubling and now it appear that even in spite 

of the hard work that was done by both sides of the dais in this 

committee in the last Congress that that doesn’t seem to be reaching the level that any of us had intended. 

Let me just ask one last line of questions, and I mean to get a 

response to this in writing. Currently, as far as the treatment of 

diabetes, the ability to implant an islet cell from a cadaveric source 

currently exists. Is that correct? 

Dr. ZERHOUNI. That is correct. 

Mr. BURGESS. And NIH is using that and that is successful, but 

those patients will have to take a drug to inhibit rejection from 

that point on. And I don’t think the human embryonic stem cell 

has ever been able to produce insulin that would impact blood 

sugar, but if it did, and if that cell were then implanted like other 

islet cells have been implanted from a cadaveric source, would that 

same requirement for taking anti-rejection drugs be required for 

that individual? 

Dr. ZERHOUNI. So it all depends on where the cell comes from. 

If it comes from the patient himself or herself, no. 

Mr. BURGESS. Which is why the reprogramming activities—— 

Mr. PALLONE. We are going to have to—— 

Mr. BURGESS [continuing]. Are so exciting. 

Mr. PALLONE. One more question and that is it because you are 

over too. 

Mr. BURGESS. For the anti-rejection medication. 

Dr. ZERHOUNI. It would make sense but I would be very careful, 

Dr. Burgess, because when you reprogram a cell with outside factors and viruses and so on, it is not clear that you won’t have an 

immune response. This needs study, but in theory you are correct. 

Mr. BURGESS. Very well. I will yield back, Mr. Chairman. 

Mr. PALLONE. Thank you. 

The gentlewoman from North Carolina, Ms. Myrick. 

Ms. MYRICK. Thank you, Mr. Chairman, and I echo all the accolades that others have said. We appreciate all the hard work you 

do. Thank you for that. 

I wanted to ask you, I was really astounded when I learned that 

the scientists at Wake Forest and Rutgers had actually—this was 

54 

funded by the Pentagon, by the way—but they managed to grow 

a human ear and it was generated from the stem cells of a badly 

wounded Marine, and they grew it on the back of a mouse, as I am 

sure you probably know, to be transplanted onto the Marine, and 

my question is twofold. One, can you comment on the promise of 

such research, and do you think that a bill like H.R. 810 alone 

would allow for this same sort of breakthrough, even though the 

embryo lines eligible for Federal funding under the bill may not actually come from the patient, and would scientists need to create 

or clone embryos in order to tailor-make therapies like this in the 

near term, I am talking; not 30 years from now but in the near 

term? 

Dr. ZERHOUNI. As I said, I think currently the difficulty of using 

adult stem cells and using them clinically is less because we have 

had a lot more experience. We have had 40 years of experience. 

What you are referring to is tissue engineering, which is the specialty that you are mentioning that you are aware of, and in tissue 

engineering, we have learned how to grow cells, for example, vessels or skin cells, on a 3-dimensional basis. That is currently available. We have grown skin, for example, for burn patients for many, 

many years already. The real issue, though, is how do you change 

the destiny of a cell to become an islet cell? So we know how to 

make the same cell expand into the same cell. We don’t necessarily 

know how to take that cell, even though it is pluripotent, into replacing a neuron. That is the prospect of what we are doing, Congresswoman. 

Ms. MYRICK. I appreciate it. And Mr. Chairman, if you would 

allow me, I have two articles by Dr. Atala at Wake Forest that I 

would like to submit for the record, if I may. 

Mr. PALLONE. I looked at the one Mr. Ferguson gave me and it 

was not easily understood, so I will ask that you give me those copies and then we will take a look at it again, if that is all right, 

and—— 

Ms. MYRICK. No, it is fine. 

Mr. PALLONE. Let me take a look. 

Ms. MYRICK. Thank you. 

Mr. PALLONE. The gentleman from Oklahoma? No? OK. I think 

that completes our questions, and thank you, Dr. Zerhouni. 

Dr. ZERHOUNI. You are welcome. 

Mr. PALLONE. We really appreciate your testimony and all that 

you did and all that you continue to do. Thank you. 

Dr. ZERHOUNI. Thank you very much. 

Mr. PALLONE. I would ask our second panel to come forward. I 

want to welcome our second panel, and let me introduce everyone 

from left to right once we have the signs posted here. Welcome. I 

will start with, on my left is Dr. John Gearhart, who is the C. Michael Armstrong professor of medicine at the Institute for Cell Engineering at the Johns Hopkins University. And then we have Dr. 

Amit Patel, who is director of cardiac cell therapy, the Heart, Lung 

and Esophageal Surgery Institute Surgery at UPMC Presbyterian 

in Pittsburgh—I am sorry—UPMC Presbyterian, McGowan Institute of Regenerative Medicine in Pittsburgh, Pennsylvania. And 

then Mr. Douglas T. Rice from Spokane Valley, Washington. Dr. 

George Daley, associate professor of pediatrics for the Karp Family 

55 

Research—I guess that is your address, I am sorry—associate professor of pediatrics at the Children’s Hospital in Boston. And then 

we have Mr. Weyman Johnson, Jr., who is chairman of the National Multiple Sclerosis Society, and from my own State of New 

Jersey, Dr. Joseph Bertino, who is interim director and chief scientific officer for the Cancer Institute of New Jersey. Good to see 

you again. And then we have Dr. John K. Fraser, who is principal 

scientist with Cytori Therapeutics—I hope I got that right—in San 

Diego, California. 

And as I said before, we have 5-minute opening statements. They 

become part of the record, and you may, in the discretion of the 

committee, be asked to submit additional written statements for inclusion in the record, depending on the questions that we get to. 

We will start with an opening statement by Dr. Gearhart. 

STATEMENT OF JOHN D. GEARHART, PH.D., C. MICHAEL ARMSTRONG PROFESSOR OF MEDICINE, INSTITUTE FOR CELL 

ENGINEERING, JOHNS HOPKINS UNIVERSITY 

Mr. GEARHART. Thank you, Mr. Chairman and members of the 

Committee. Ten years ago, we had our first hearing in Congress on 

embryonic stem cells. This was the result of the publications from 

two laboratories of the discovery of these cells. I had the privilege 

of being part of that, and Dr. Harold Varmus was here at the time 

as the director of the NIH and he at that time put forward what 

these cells could be used for, the potential of these cells, and I 

thought it would be interesting to the Committee to review his 

comments and then to tell you where I see as an active researcher 

in this field where our science is with embryonic stem cell research. 

So the initial comment that he made was that these cells could 

be a boon to basic science, to understanding human biology and 

human development. And indeed, we see that one of the primary 

uses to date of these cells is to understand some of these very early 

events in embryogenesis for which there is no other avenue of research to understand how we go from a single cell egg up to an individual that has 200 trillion cells. What are the processes involved? And so we and others have used these cells and culture to 

discover new genes, new genes that are involved in the formation 

of the central nervous system, of the heart, and recently in our laboratory we discovered 40 new genes in the very earliest stages of 

the development of the circulatory system, which happens within 

the first few weeks of our development. There is no other way that 

we could have gotten this information, and these are critical genes. 

We can demonstrate by shutting them down, manipulating them as 

we do, that they are important in development. This is just but one 

example of the use of these cells that are going to be made in understanding how our program, the genetic program that Dr. 

Zerhouni mentioned, is played out so that we can get a handle on 

birth defects and ultimately on some of the disease processes that 

occur in our bodies. And this has been an extremely exciting development. 

Secondly, he mentioned that these cells could be used in the testing of drugs and factors directly on human cells without having to 

subject patients to them, and we see this happening now, of culturing a variety of different cell types, having them in culture and 

56 

subjecting them to different types of toxins, drugs of different 

kinds, and see the response of the cells without going through either animal models, which sometimes aren’t important, or directly 

a variety of different human genotypes. This is occurring. 

Also, we see remarkable work being done on figuring out how we 

go through these lineages, how a single cell can become one of the 

260 different cell types. This isn’t trivial. We have a cell in culture 

that can form all of those cell types. How do we get it to form just 

a liver cell or a dopaminergic neuron? And we are figuring out 

these processes by trying to mimic what is occurring in an embryo 

and then using that information to direct the specialization of that 

cell. This is enormous from the standpoint of saying, well, if we are 

going to develop some kinds of therapies, we are going to have to 

get a homogenous population of cells that we know what they are 

that we can put into a patient. This is extremely important. 

Another avenue he said would be the use of these cells in transplantation research for diseased or damaged tissue, and we now 

see in the published literature dozens and dozens of examples of 

where cells derived from human embryonic stem cells have been 

placed into animal models either for disease or injury. Yes, there 

are variable outcomes to this but it shows a great deal of promise. 

So in all of these avenues, we are seeing this in research, and 

I just want to tell you that is going to take a while. This is something else that came out of this initial meeting, was this projection 

of how long it would take for us to develop these kinds of therapies 

for patients. It is going to take years, and much of it is safety. We 

don’t want to place cells into a patient without knowing what their 

fate is going to be and how we can regulate it, and we are getting 

a handle on that in the modes of delivery, the types of cells we put 

in, whole new—we have made radiologists even richer from the 

standpoint that when we first went to the FDA, we were asked, if 

you are putting in 300,000 cells, we want to know where every one 

of those cells is going. We want live-time tracking of these cells. So 

we are delighted at the progress of this. 

Now, let me tell you how I—— 

Mr. PALLONE. I hate to interrupt you all because what you are 

saying is so important but we have a long panel, so you have to 

wrap it up. 

Mr. GEARHART. That is fine. Well, I have recently seen how policy issues can trump science and I am very disappointed. Reference 

was made recently to the Army’s Institute of Regenerative Medicine announcement of $250 million. I think you should be aware 

that what was not permitted in those studies was anything dealing 

with embryonic stem cells, and I just feel that we are shooting ourselves in the foot by not also having that avenue explored for some 

of this very important regenerative medicine. 

[The prepared statement of Mr. Gearhart follows:] 

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Mr. PALLONE. Thank you, Doctor. 

Dr. Patel. 

STATEMENT OF AMIT N. PATEL, M.D., M.S., DIRECTOR OF CARDIAC CELL THERAPY, THE HEART, LUNG AND ESOPHAGEAL 

INSTITUTE, UPMC PRESBYTERIAN, MCGOWAN INSTITUTE OF 

REGENERATIVE MEDICINE 

Dr. PATEL. I would like to thank the Chairman and the members 

of the Committee for giving me this opportunity to testify before 

you. I just have to make a quick note that the testimony that I am 

giving today is of my own opinion and not necessarily that of the 

institution that I am currently employed by. 

My career has really been developed and based on the treatment 

for cardiovascular disease. I am a cardiac surgeon and a 

translational scientist, meaning my goal is to take the science that 

many of the panelists here have been doing for longer than I have 

been alive that they first started and how can we most safely and 

efficiently help the patients who have the disease today, and based 

on that, cardiovascular disease, as we know, is the greatest cause 

of death in America. There are millions of patients every year who 

die from new heart attacks, limb ischemia, not getting enough 

blood supply to their legs, and the most end stage, which is about 

5 million patients with heart failure. Fifty percent of those patients 

will die within 5 years of their diagnosis in the most severe forms. 

So the question that I have and I try to help my patients with is, 

I do bypass surgeries, I do valves, I do heart transplants, but with 

our limited organs, the risks of complications of anti-rejection medications, I have to find other solutions that safely can help these patients just because I can’t help all of them, and every day I get 

calls from patients from within the United States and around the 

world, can you provide me a therapy, just as Dr. Zerhouni said. 

But the key is, how we can do it safe and effective here in the 

United States. And so there are two problems that we have tried 

to solve and by no means have an answer to but have some early 

treatments for is, for heart failure, our basic problem is, we have 

a pump that just cannot supply enough blood by delivering enough 

oxygen to the entire body, and in patients with limb ischemia, 

these are patients that due to lack of enough oxygen and blood supply to their legs, these patients end up with amputations. So when 

you combine those together, the total loss that was reported by the 

American Heart Association in 2005 was $394 billion, $242 billion 

from the healthcare expenditures and $152 billion from loss in productivity from death and disability. So as we know how dramatic 

of an impact this has on not just the capital resources but human 

resources, that is the two things that we have really focused on. 

So our role of stem cell therapy really has been, well, what do 

we want to do. It is great for these very complex diseases and disorders such as Parkinson’s and other neurological or immunological 

problems, but our goal is very simple. We need a heart that has 

more ability to pump by either providing more cardiac myocytes, or 

heart muscle, and increasing the blood supply, developing new 

blood vessels. And in patients with limb ischemia, how can we develop more blood cells that will prevent these patients from getting 

amputation. So it sounds like a simple solution that we need to adVerDate Nov 24 2008 16:13 Mar 16, 2010 Jkt 037690 PO 00000 Frm 00068 Fmt 6633 Sfmt 6633 C:\DOCUMENTS AND SETTINGS\CWELLS1\HEARINGS\110-115 SCOM1 PsN: JIMC
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dress, so our goal has been, how can we help the patients today 

with the cells that we have available that have been safe, and the 

question of safety is always an issue. 

Five years ago, when we first started some of the earlier clinical 

work, that was a very significant concern and we received one of 

the first FDA approvals to do human trials here in the United 

States, and a similar group in Texas also received this approval 

using bone marrow-derived cells, and it is not to say that that was 

the perfect answer or solution because before that, in France, patients had received biopsies of their muscle from their thigh, they 

expanded them in culture and injected them into their heart and 

caused significant irregular heartbeats. So translating too early 

from the science without knowing a lot of the answers is also not 

the right answer, so there has to be a safe and ethical balance. But 

now, when those same myoblasts in the United States were taken 

in a safer fashion, delivered with a catheter in heart failure patients, that is now expanded to a phase II 390-patient clinical study 

that is funded by industry. So it is not that the cells are bad, it 

is knowing the right indications for the patients and the right way 

to culture them. 

We have been able to take bone marrow in varied forms. The 

earliest science and animal work showed great potential that these 

bone marrow cells magically will become all these different cell 

types. The reality is that this may happen in the dish but it is very 

unlikely in our patients that this will happen, but the key is, how 

can we most safely, effectively do this, not only for our adult cells 

but all the other multipotent cells that we are hoping to deliver, 

such as the adipose cells, which you will hear about, amniotic cells, 

placental, menstrual, and even the embryonic, so it is the whole litany of cells. It doesn’t matter where the cell comes from, we still 

need to go through the same questions to how to provide the most 

safe, reliable delivery of cells, also issues of dosing. It is very similar to pharmacological therapies that we need to know doses, 

toxicities, where are all these cells going to go. I could put them 

in the heart. If I flush them down the arteries in the heart, greater 

than 90 percent of the cells end up in the lungs, liver, or spleen. 

The question is, what are they doing there. So when it is their own 

cells, there has been a level of safety now after about 8 years of 

treatments throughout the world in registered trials. There is probably about four times as many unregistered patients who are—— 

Mr. PALLONE. I am going to ask you to wrap up. 

Dr. PATEL. Sure. That in the 1,000 patients that have been treated in registered trials, there has been definite safety shown with 

bone marrow-derived cells. There has been a modest improvement 

in cardiac function, and in the right selected patients, there has 

been a very significant improvement that has shown decreased 

death, decreased re-admission, and up to 5 years now the safety 

along with sustained improvement. There is the possibility that 

these patients may need redosing, but the biggest issue is, we have 

been benefited by the NIH. There is the center of cell therapy, center of heart failure and cardiac surgery where we could further answer a lot of these scientific questions along with providing clinical 

therapies for patients here in the United States today so they don’t 

64 

have to go overseas and get unregulated and unscrupulous therapies where they have to pay a lot of their own money. Thank you. 

The prepared statement of Dr. Patel follows:] 

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Mr. PALLONE. Thank you, Dr. Patel. 

Mr. Rice. 

STATEMENT OF DOUGLAS T. RICE, SPOKANE VALLEY, 

WASHINGTON 

Mr. RICE. My name is Doug Rice and I am 62 years old. I have 

congestive heart disease and diabetes. I could be one of over 

750,000 people that die in the United States yearly, but I am not 

dead, not because I shouldn’t be, but there is a resolution to this 

problem. I am not a miracle, a phenomenon, but a living person 

that by the grace of God was saved from a disease that kills approximately 2,000 people daily. However, I had to travel to Bangkok, Thailand, and go in debt to do something that should be readily available in the United States. I used my own adult stem cells 

and a simple angioplasty procedure to have my life given back to 

me. Your own adult stem cells have so much more to give than we 

give them credit for. A lot of other diseases are being treated successfully by just using the adult stem cells. 

My story is simple. In 1992, I had my first heart attack and was 

also diagnosed with diabetes. That same year, my mother died of 

congestive heart failure and diabetes, just like what I have. Also 

just last year, my sister died of what I have. I have had numerous 

heart attacks and diabetes episodes as well as having to be jumpstarted at least three times. I have had a TMR—that is a 

transmyocardial revascularization procedure—that uses a laser to 

drill holes in the left ventricle to get better blood flow. This did not 

help. In 1998, I was given only 2 years to live unless I received a 

heart transplant. Because of my diabetes, I did not quality for it. 

We tried different things that helped, and then in November of 

2005 I could not walk but a few feet. I had to sleep sitting up and 

was just worn out. My ejection fraction, the amount of blood my 

heart pumps out each beat, was around 11 percent. The average is 

over 50 percent. My cardiologist, Dr. Canaday, said at best I had 

4 months without a mechanical heart pump to survive. It was battery operated, and I decided that I did not want to be battery powered. 

That night my best friend, Sheba Rice, went on the Internet looking for new heart treatments. She found Thera Vita, a company in 

Bangkok, Thailand, that had been having success using the adult 

stem cells. We contacted them, went to Bangkok in January of 

2006, and other than drawing blood, shipping to Israel, then having the adult stem cells sent back and implanted in me via 

angioplasty, it was simple. The hardest part was the 20-hour flight. 

When I returned to Spokane, within a month my ejection fraction 

was tested. It was 28 percent and going up. I felt better than I had 

felt in years. I was motivated to tell the world, and that is when 

I found out that over 750,000 people a year die from heart disease. 

These 750,000 heart patients that will die do not make the mainstream press, no newspaper articles of any significance, and certainly most politicians in Washington don’t even like to discuss it. 

Sadly, it is a fact, if a family dies in a car wreck, children are 

gunned down in a school or a disgruntled person shoots or maims 

his or her coworkers, it is big news. But 750,000 people die at a 

rate of over 2,000 people a day and no one takes time to talk for 

70 

them. Not all are old. Some are very young and with families and 

friends to care about. Most people just don’t realize that they die 

although almost everyone knows someone that has died or will die 

from this disease. 

The Federal Government has spent millions of dollars on embryonic stem cells but not one person has been treated and the animals tested often get tumors. 

By some estimates, over 400,000 people with various cancers and 

other diseases have been successfully treated and most are alive to 

talk about the adult stem cell treatment using their own blood cells 

or ones from cord blood cells. The honest experts say maybe in 10 

or 20 years embryonic stem cells might have potential to treat 

someone, but not now, and there is something that works now. The 

adult stem cells work. What does it take to make people realize 

that a bird in the hand is worth two in the bush, especially when 

it comes to people’s lives? 

If you ask most people about stem cells, they only heard about 

embryonic because that is all you hear about. Education, education, 

education and the facts regarding adult stem cells are the only way 

to succeed in moving this issue to the forefront for funding and actual treatments now. 

I get a lot of calls on a daily basis because I have been treated 

with my adult stem cells, and the most frequent question is, why 

did you have to go to Thailand? Because there is no treatment 

available in the United States. I had to pay for it myself. My insurance did not cover the costs of this treatment, though I heard that 

in Germany, insurance covers stem cell treatment for heart disease. I also know that much of the stem cell debate in recent years 

has been drastically increased funding for embryonic stem cells despite the fact they have not treated patients for any disease. Patients are being increasingly treated with adult stem cells but we 

need drastically more Federal funding for adult stem cell treatments. These cells aren’t patentable, so private investment is far 

behind. The government should spend more on clinical trials so 

Americans like myself can have the same chance at a treatment 

that I had. I am just one man, and all I can do is talk to everybody 

I know, and it is a fact, you ask anybody what a stem cell is, and 

the first word out of their mouth is embryonic because that is all 

you ever hear. I listen to every TV station, news station and you 

never hear the word ‘‘adult stem cells.’’ I am alive because of it. 

Thank you. 

[The prepared statement of Mr. Rice follows:] 

71 

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73 

74 

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Mr. PALLONE. Thank you, Mr. Rice. 

Dr. Daley. 

STATEMENT OF GEORGE Q. DALEY, M.D., PH.D., PRESIDENT, 

INTERNATIONAL SOCIETY FOR STEM CELL RESEARCH AND 

ASSOCIATE PROFESSOR OF PEDIATRICS, CHILDREN’S HOSPITAL BOSTON 

Dr. DALEY. Thank you, Mr. Chairman and members for the 

chance to testify. It is difficult to add much to what Dr. Zerhouni 

talked about. He really gave a very spirited and compelling argument in support of an integrated approach to stem cell research. 

I am here to give the perspective of a physician scientist. I am 

from Children’s Hospital and Harvard Medical School and I am 

also the current president of the International Society for Stem 

Cell Research. My laboratory studies blood development, blood cancers, and various experimental transplantation therapies, and in 

my clinical duties at the Children’s Hospital I take care of kids 

with a variety of blood diseases and so I see firsthand the advantages and the limitations of the current therapies such as adult 

stem cell therapies. 

All stem cells, whether they are embryonic, fetal, neonatal, adult, 

have great promise for medicine. However, I am concerned because 

the recent breakthroughs in the reprogramming of adult skin cells 

have renewed the calls for limitations on embryonic stem cell research, and I wish to testify unequivocally that enacting such limitations would be unwise. My organization, the International Society for Stem Cell Research, continues to assert, as do I think the 

vast majority of scientists, that only through an expanded support 

for all avenues of stem cell research can we ensure the most rapid 

pace of discovery. 

Much excitement in stem cell research has focused on this remarkable property of embryonic stem cells, a property we call 

pluripotency, that was described by Dr. Zerhouni. This is the capacity for a cell to generate any tissue in the body. It is an enormously valuable property. Recently several laboratories, including 

my own, reported that a small set of genes which were originally 

discovered because of their link to pluripotency in embryonic stem 

cells, can be inserted into human skin cells to convert them to a 

cell which is like a seed for all tissues in the body, a cell that very 

closely resembles but may not be identical to embryonic stem cells. 

I can show you the scar on my forearm. We can do this with any 

patient, and in a matter of weeks take skin cells and turn them 

into pluripotent stem cells. 

This is no doubt a major breakthrough in medical research and 

it is going to have important implications for modeling disease, and 

I certainly hope that one day it is going to usher in new cellular 

therapies. But I have to caution and reiterate the caution of Dr. 

Zerhouni that realizing this promise is going to take time. A major 

concern for this new methodology is the viruses that we use to 

carry the reprogramming genes. They themselves are linked to cancer. And even if we can remove viruses from this process, the genes 

and pathways that are activated in the cells are also associated 

with cancer and we don’t know how these cells are going to reVerDate Nov 24 2008 16:13 Mar 16, 2010 Jkt 037690 PO 00000 Frm 00081 Fmt 6633 Sfmt 6633 C:\DOCUMENTS AND SETTINGS\CWELLS1\HEARINGS\110-115 SCOM1 PsN: JIMC
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spond. We don’t know what their long-term predispositions to abnormal growth or even cancer might be. 

Furthermore, I want to say that even though my lab has generated these induced pluripotent stem cells, my lab will continue 

to vigorously study embryonic stem cells. First, we need to directly 

compare the properties of our embryonic stem cells against the 

properties of our induced pluripotent, or iPS, cells. And there are 

already some whispers in the community and some preliminary 

data that iPS cells are not as robust as embryonic stem cells for 

the formation of certain tissues, but it is going to take years for scientists to understand the similarities and differences. 

I would also mention that even though we have iPS cells, my laboratory will continue to vigorously pursue somatic cell nuclear 

transfer. Reprogramming by nuclear transfer is faster than genebased reprogramming and may entail very different mechanisms 

that will teach us a lot about how to make pluripotent tissues better. The iPS breakthrough is being heralded by opponents of stem 

cell research as a solution to the long-smoldering debate over the 

necessity for embryonic stem cells, and we have heard the arguments before. We heard them in 2002 when multi-potential adult 

progenitor stem cells were announced. We heard them later in 

2004 and 2006 when fat and amniotic fluid stem cells were announced and again we are hearing them today. Congress has been 

wise not to yield to these arguments. I remind you that it was basic 

stem cell research that really led to the breakthroughs in iPS cell 

research. 

Yesterday I gave an address to the Congressional Biomedical Research Caucus and I answered the question, ‘‘Do we still need embryonic stem cell research?’’ with a resounding ‘‘yes.’’ And I would 

say that embryonic stem cells remain the gold standard, will remain so for the foreseeable future, and there is still real value in 

passing H.R. 810, the original bill put forth by Members Castle and 

DeGette. 

I look forward to answering your questions in the Q&A period. 

Thank you. 

[The prepared statement of Dr. Daley follows:] 

STATEMENT OF GEORGE Q. DALEY

Thank you for the invitation to speak today on the subject of stem cell science. 

My name is George Daley and I am an Associate Professor of Biological Chemistry, 

Medicine, and Pediatrics at Children’s Hospital Boston and Harvard Medical School, 

a core faculty member of the Harvard Stem Cell Institute, an investigator of the 

Howard Hughes Medical Institute, and the current President of the International 

Society for Stem Cell Research (ISSCR), the major professional organization of stem 

cell scientists worldwide. My laboratory studies blood development, blood cancer, 

and experimental transplant therapies for diseases like sickle cell anemia, immune 

deficiency, and leukemia. In my clinical duties at Children’s Hospital, I care for patients with these devastating blood diseases, and see first hand the need for better 

treatments. Stem cell research offers hope. 

Let me recount the stories of two patients I cared for recently at Children’s Hospital that illustrate the shortcomings of current therapies. One was a young AfricanAmerican boy with sickle cell anemia, suddenly struck down by what we call a pain 

crisis. When I saw him in the emergency room, he was writhing on the gurney and 

whimpering in pain. Despite powerful, high doses of intravenous morphine, I was 

unable to give that child adequate relief from his pain and suffering for several 

days. A second case was an infant who suffered repeated infections and had spent 

half his young life in the hospital hooked up to intravenous antibiotics. His disease 

77 

was immune-deficiency, and unfortunately he had no sibling donors for a potentially 

curative adult stem cell transplant. Stem cell research is laying the foundation for 

improved treatments for these kids and countless other children and adults with debilitating, life-threatening diseases. 

All stem cells—whether from embryonic, fetal, neonatal, or adult sources—hold 

great promise. The crowning scientific achievement of the twentieth century was the 

sequencing of the human genome, and the dominant mission of twenty-first century 

science is to discover how that blueprint drives the formation of tissues and organs, 

and how tissues are sustained, repaired, and rejuvenated over time. Stem cell research goes to the core of human biology and medicine. 

Much excitement in stem cell research has focused on a property of embryonic 

cells called pluripotency—the capacity to generate all of the tissues in an organism. 

Recently, several laboratories, including my own, reported that a small set of genes 

linked to pluripotency in embryonic stem (ES) cells can be inserted into human skin 

cells to induce pluripotency—to endow skin cells with this same remarkable capacity 

to become a seed for all tissues in the body. By using gene-based reprogramming 

to make these so-called induced pluripotent stem cells (called ‘‘iPS cells’’), scientists 

can now produce customized, patient-specific stem cells in the Petri dish. In a matter of weeks, we can take cells from a patient’s forearm and transform them into 

pluripotent stem cells that we believe closely approximate embryonic stem cells. 

This is a major breakthrough in medical research, empowering scientists to create 

cellular models of human disease. It may also mean that one day we will treat patients with rejuvenated and repaired versions of their own tissues. 

Realizing this promise will take time. A key concern is that the viruses used to 

carry the reprogramming genes into human skin cells can cause cancer. Moreover, 

the genes and pathways the viruses stimulate are themselves associated with cancer, raising the concern that even if viruses can be eliminated from the process, the 

reprogrammed cells might remain predisposed to cancer. For these reasons, iPS cells 

may never be suitable for use in patients. I sincerely hope that iPS cells are the 

long-sought-after customized patient-specific stem cell, but much more research 

must be done. 

Even with iPS cells in hand, my laboratory will continue to study embryonic stem 

cells. First, we need to directly compare the capacity of these two types of stem cells 

to generate specific tissues. Some very preliminary data has suggested that iPS cells 

may be less potent than embryonic stem cells in making blood, while others are noting a deficiency in making heart muscle cells. It will take years for scientists to understand the similarities and differences between these two valuable classes of 

pluripotent stem cells. Even with iPS cells in hand, my laboratory will continue to 

investigate somatic cell nuclear transfer as a means of generating pluripotent stem 

cells. Reprogramming by nuclear transfer is faster and may entail very different 

mechanisms than gene-based reprogramming. Learning why may lead to better 

methods for making iPS cells. 

The iPS breakthrough is being heralded by opponents of embryonic stem cell research as a solution to the long-smoldering debate over the necessity of embryonic 

stem cell research. We have heard the arguments for many years, first made when 

multi-potential adult progenitor cells (MAPCs) were reported in 2002, and later 

when stem cells were isolated from Fat and Amniotic fluid; we are told that alternatives are available that preclude the need for embryonic stem cell research. Congress has been wise to not yield to such arguments. Indeed, it was embryonic stem 

cell research that led directly to the breakthrough in iPS cells, and my own laboratory was poised to generate iPS cells in large part because of our experience and 

expertise in deriving and culturing human embryonic stem cells. Today, it would 

again be a mistake to place limits on the tools available to biomedical scientists to 

pursue the next medical breakthroughs. The right course for biomedical science and 

ultimately the right decision for patients and our health care system, is to expand 

the scope of federal funding for all forms of stem cell research, including the many 

lines of embryonic stem cells created after the President’s artificial deadline of August 9th, 2001. 

Yesterday, in my address to the Congressional Biomedical Research Caucus, I was 

asked the question: ‘‘Do we still need research on embryonic stem cells?’’ to which 

I replied a resounding ′′Yes.′′ Embryonic stem cells remain the gold standard today 

and will remain so for the foreseeable future. If we are to maximize the pace of scientific discovery and accelerate development of new treatments for disease, we must 

continue to vigorously pursue all forms of stem cell research, using ES cells derived 

from embryos, pluripotent stem cells generated by nuclear transfer and gene-based 

reprogramming, and adult stem cells. Passage of the bill H.R. 810 originally proposed by members Castle and Degette remains a worthy goal. 

78 

Mr. PALLONE. Thank you, Dr. Daley. 

Mr. Johnson. 

STATEMENT OF WEYMAN JOHNSON, JR., J.D., CHAIRMAN, 

NATIONAL MULTIPLE SCLEROSIS SOCIETY 

Mr. JOHNSON. Thank you, Chairman Pallone and Ranking Member Deal. Thank you, all the members of the Committee. I am honored to be invited to speak here today among many distinguished 

panelists and to represent individuals who live with chronic disease. 

Expanded embryonic stem cell research will advance our 

progress in many diseases, but today I will focus on one, multiple 

sclerosis, and it is not because it is more important than any disease, it is because it is the disease I know about. It is the disease 

that comprises my story. 

I first learned close-up about multiple sclerosis when I was just 

a kid, 12 years old, and my father was diagnosed with multiple 

sclerosis. He is no longer living, but late in his life, MS affected 

him severely. His own sister, Allene was the first person I met with 

MS. She was diagnosed in the mid-1950s. I never knew Allene, unless she was in a bed or in a wheelchair. When I was a child, I 

was told that incidence of MS in our family was merely a coincidence. Today, through genetic research, we know that it is simply 

not true. 

In 1989, my own sister, Lanay, who is only a few years older 

than I am, was diagnosed with multiple sclerosis. Today, she uses 

a power wheelchair to move everywhere she goes. Her hands don’t 

work well anymore. She can no longer teach the way she did in the 

public school systems in Georgia for many years. She can no longer 

play the piano the way she did so beautifully. When I think about 

the sanctity of life, I include my sister’s life in those thoughts. 

A few years after she was diagnosed, I was diagnosed. In our 

family, we hate this disease. We hate its impact on our family and 

other families. We hate the threat it poses to future generations. 

While I have not been severely disabled by multiple sclerosis, I 

have seen its severe effects up close. 

The scientific community is making progress into the genetic factors involved in multiple sclerosis. There are still more questions 

than answers, however. All kinds of research must continue. 

I remember being told that multiple sclerosis is a disease that 

doesn’t affect my friends in the African-American community, that 

it is only for white people. With scientific advance, we have found 

that is not true. We also used to hear that this disease did not happen to children, but that is not true either. We know now that 

there are thousands of children in the United States and thousands 

of children throughout the world who live with this disease. All 

kinds of research must continue. 

Before 1993, there were no treatments at all for multiple sclerosis. Now we have six. But there is a wide spectrum of disability 

among people living with multiple sclerosis. Most of the available 

therapies work only for those on the lucky end of the spectrum, like 

me. For people like my sister on the more unlucky end, there are 

still few remedies. All kinds of research must continue. 

79 

Every hour, somebody new is diagnosed with multiple sclerosis. 

It is an unpredictable, often disabling disease of the central nervous system. The progress, the severity, the specific symptoms of 

MS in any one person still cannot be predicted. The cause is unknown, and there is no cure. But embryonic stem cell research 

holds unique promise to repair nerve cells to slow the progression 

of MS and to find a cure. I am just one person living with a chronic 

disease, but I am also privileged to serve as the chair of the board 

of the National Multiple Sclerosis Society. At the National MS Society, we believe that all promising avenues of research that could 

lead to new ways to prevent, repair, slow the progression or cure 

MS must be pursued with adherence to the strictest legal and procedural guidelines. 

I salute Congresswoman Capps. She was chosen last year as our 

organization’s legislator of the year. We thank her for her support 

for people with MS. I salute in absentia Dr. Burgess, who is a 

member of the MS Caucus of the House of Representatives. He and 

I might not agree categorically on every issue but I appreciate his 

support and the support of other Congresspersons for people with 

MS. 

I am asking you today to expand Federal policy in embryonic 

stem cell research and to ensure that research continues for the 

more than 400,000 other Americans who live with MS and the 100 

million Americans with other diseases and conditions. Research on 

all kinds of stem cells is critical because we have no way of knowing now which kind of stem cell will be of the most value for MS, 

for Parkinson’s, for Alzheimer’s, for cancer, for heart disease, for 

many other conditions. Just as with genetics and race and age, 

there is much left to learn about how to treat and cure MS, about 

how to treat and cure other diseases. Expanding our embryonic 

stem cell research is just one avenue. 

As I close, I will note one side note. Our organization in January 

of 2007, along with our sister organization, the MS International 

Federation, sponsored an embryonic stem cell symposium in San 

Francisco. The heartening part of that symposium was that there 

was new research about repair that was available. The disheartening part was that there were not very many American scientists 

leading on the cutting edges. I think that is a shame that we may 

have abdicated our leadership role in the intellectual and scientific 

progress in the world. We ask for your commitment not to give up 

on legislation like the Stem Cell Research Enhancement Act. We 

don’t have the luxury of time. Like many others who live with a 

chronic disease, I know that maybe not today, maybe not next week 

but I pray soon with patience and continued research, there will be 

a world without multiple sclerosis and a world of decreased disease. 

Thank you very much for helping move us closer, and thank you 

for your time. 

[The prepared statement of Mr. Johnson follows:] 

STATEMENT OF WEYMAN JOHNSON

SUMMARY

• Summary of my personal and family experiences with a chronic, disabling disease. 

80 

• Speak to a patient perspective on my own diagnosis with multiple sclerosis. 

• Speak to the position of a national voluntary health organization, as chairman 

of the board of the National Multiple Sclerosis Society. • Speak to the need for continued research and the hope it brings for people living 

with chronic diseases and conditions nationwide. • Support the need for the Committee and Congress to remain committed to legislation like the Stem Cell Research Enhancement Act. • Embryonic stem cell research holds an incredibly unique promise for people living with chronic diseases and conditions, and the progress made to date on embryonic stem cell lines should not be abandoned. 

TESTIMONY

Thank you Chairman Pallone and Ranking Member Deal. Thank you members of 

the Committee. I am honored to be invited to speak here today among many distinguished panelists and to represent patients who live with chronic disease. 

Many diseases could benefit from expanded embryonic stem cell research. But 

today I will focus on one—multiple sclerosis. Not because it is more important than 

others, but because I know multiple sclerosis. 

I remember multiple sclerosis and how it entered my life as a child, in 1964, just 

barely 13 years old. My father received a diagnosis of MS suddenly. He died in 2001. 

His sister, my aunt Allene, also had MS. Research into this disease, into genetics 

was just starting to evolve in the 1960s. 

There were good doctors then, but they did not recognize a genetic connection. 

They said MS in my family was a mere coincidence. Because of research, we now 

know that is not true. 

My own sister, who’s only a few years older than I, lives with MS. She uses a 

power wheelchair, her hands don’t work well anymore, she can no longer teach the 

way she did, or play the piano the way she did. A few years after she was diagnosed, so was I. We hate this disease, its impact on our family, and the threat it 

poses to our future generations. 

We are making progress into the genetic factors involved in multiple sclerosis. 

However there are still more questions than answers. The research must continue. 

I remember being told that MS is a disease that doesn’t affect my friends in the 

African American community. This is only for white people from Minnesota. With 

good science, we have found that’s not true. The research must continue. 

We also used to hear that this disease does not happen to children. But that is 

not true either. We now know there are thousands of children in the United States, 

thousands of children throughout the world, who live with this disease. The research must continue. 

Before 1993, there were no treatments at all for multiple sclerosis. Now we have 

six. But there is a wide spectrum among people living with MS. Most of the therapies will only work for those of us on the lucky end of the spectrum like me. But 

for people like my sister, on the more unlucky end, there’s still not much out there 

that provides effective treatment. So the research must continue. 

Every hour, someone new is diagnosed with MS. It’s an unpredictable, often disabling disease of the central nervous system. The progress, severity, and specific 

symptoms of MS in any one person still cannot be predicted. The cause is unknown, 

and there is no cure. But embryonic stem cell research holds an incredibly unique 

promise to repair nerve cells, to slow the progression of MS, to help find a cure. 

One area that holds great promise, but is often misunderstood, is Somatic Cell 

Nuclear Transfer. We have seen some exciting breakthroughs. But as with all 

science, this research takes time. We are still exploring this avenue for medical research. I have hope that SCNT will succeed because of its promise to repair nerve 

cells, creating new tissues, and more. I know that researchers are focused on the 

idea of creating cells and tissues for transplantation and research. They are trying 

to understand how different genes are turned on and off. They are not focused on 

cloning. I know that as we explore somatic cell nuclear transfer research more, we 

will see greater potential for developing individualized cell and tissue therapies. 

That holds great promise for people living with MS like me, whose body’s own defense system is attacking the myelin surrounding and protecting our central nervous 

system. 

I am but one person living with a chronic disease. But I am also fortunate to serve 

as chairman of the board of the National Multiple Sclerosis Society. We believe that 

all promising avenues of research that could lead to new ways to prevent, repair, 

slow the progression, or cure MS must be explored, with adherence to the strictest 

ethical and procedural guidelines. The National Multiple Sclerosis Society believes 

that all promising avenues of research that could lead to the cure or prevention of 

81 

multiple sclerosis or relieve its symptoms must be explored. The Society supports 

the Stem Cell Research Enhancement Act to expand the number of approved stem 

cell lines that are available for federally funded research. The Society supports the 

conduct of scientifically meritorious medical research, including research using 

human cells, in accordance with Federal, State, and local laws and with adherence 

to the strictest ethical and procedural guidelines. Research on all types of stem cells 

is critical because we have no way of knowing which type of stem cell will be of 

the most value in MS research. Stem cells—adult or embryonic—could have the potential to be used to protect and rebuild tissues that are damaged by MS, and to 

deliver molecules that foster repair or protect vulnerable tissues from further injury. 

So I ask you to expand the federal policy on embryonic stem cell research and 

ensure that research continues —for the more than 400,000 other Americans who 

live with MS and 100 million Americans with other diseases and conditions. Research on all types of stem cells is critical because we have no way of knowing at 

this point which type of stem cell will be of the most value—for multiple sclerosis, 

for Parkinson’s, for Alzheimer’s, for cancer, for heart disease, for spinal cord and 

brain injuries, for many other conditions. 

Just like with genetics and race and age, there is so much left to learn about how 

to treat and cure MS, about how to treat and cure other diseases. Expanding our 

embryonic stem cell research is just one avenue. But it is an avenue of research that 

must continue. Federal barriers must be lifted. 

You might see that I am not the only person living with MS on Capitol Hill today. 

Hundreds of MS activists are visiting with their legislators on the Hill right now, 

talking about the need to advance medical research. 

Embryonic stem cell research remains one of the most promising avenues of research to cure diseases and end suffering. I am not a scientist, but I am an observer 

of science. And I know that science is a matter that requires some patience. That’s 

why we must expand the important work done to date with embryonic stem cell 

lines. The research must continue. So we can improve the lives of people with chronic diseases and conditions. So we can improve the lives of families for generations 

to come. For my grandchildren and for yours. 

We need your commitment to not give up on legislation like the Stem Cell Research Enhancement Act. We don’t have the luxury of time. Like many others who 

live with a chronic disease, I know, maybe not today, maybe not next week, but I 

pray soon, with patience and continued research, that there will be no more disease. 

Thank you for helping us move closer, and thank you for your time. 

NATIONAL MULTIPLE SCLEROSIS SOCIETY

POLICY POSITION

EMBRYONIC STEM CELL LINES AVAILABLE FOR FEDERALLY-FUNDED RESEARCH

Position: The National Multiple Sclerosis Society believes that all promising avenues of research that could lead to the cure or prevention of multiple sclerosis or 

relieve its symptoms must be explored. The Society supports the Stem Cell Research 

Enhancement Act (H.R. 3 and S. 5) to expand the number of approved stem cell 

lines that are available for federally funded research. 

The Society supports the conduct of scientifically meritorious medical research, including research using human cells, in accordance with federal, state, and local laws 

and with adherence to the strictest ethical and procedural guidelines. Research on 

all types of stem cells is critical because we have no way of knowing which type 

of stem cell will be of the most value in MS research. Stem cells—adult or embryonic—could have the potential to be used to protect and rebuild tissues that are 

damaged by MS, and to deliver molecules that foster repair or protect vulnerable 

tissues from further injury. 

Request: We urge Congress to support the Stem Cell Research Enhancement Act 

of 2007 (H.R. 3 and S. 5) at all levels of the legislative process. This legislation 

would increase the number of approved embryonic stem cell lines that can be used 

in federally-funded research by allowing new lines to be generated from embryos 

that have been donated for research purposes by people using the services of in vitro 

fertilization clinics, while establishing important ethical protections. 

Supporting Rationale: There is broad agreement that the policy limiting the number of stem cell lines available for federally funded research is flawed. 

• An insufficient supply of stem cell lines currently exists, as only 22 of the 70 

approved lines are available to researchers. In addition, all of the available lines are 

contaminated by nutrients from mouse feeder cells. Many in the scientific community believe that these stem cell lines are unsuitable for research and hinder U.S. 

82 

scientists’ ability to capitalize on the potential breakthroughs from embryonic stem 

cell research. • At the same time, it has become increasingly clear that stem cell research holds 

tremendous promise for MS and many other diseases and disorders. Research suggests that stem cells might have many uses: for delivery of growth factors and 

drugs, for tissue culture systems for drug and gene discovery, for understanding and 

modeling MS, and for repairing or protecting brain tissue. • However, our scientific advisors have told us that we still don’t know which type 

of stem cells will be most valuable for MS research, and thus we must support policies that promote the conduct of research using all types of stem cells. 

Mr. PALLONE. Thank you, Mr. Johnson. 

Dr. Bertino. 

STATEMENT OF JOSEPH R. BERTINO, M.D., INTERIM DIRECTOR AND CHIEF SCIENTIFIC OFFICER, THE CANCER INSTITUTE OF NEW JERSEY 

Dr. BERTINO. Mr. Chairman, members of the Committee, thank 

you for inviting me to present my testimony today. 

New Jersey has been a leader in supporting stem cell research. 

In 2004, the Stem Cell Institute of New Jersey was created by a 

memorandum of understanding between Rutgers, the State University of New Jersey, and UMDNJ-Robert Wood Johnson Medical 

School. The State then committed $8.5 million to support work at 

the Stem Cell Institute, including $5.5 million in capital funds to 

Robert Wood Johnson Medical School and Rutgers University for 

laboratory renovations and GMP facilities. 

In December 2005, New Jersey became the first State to finance 

stem cell research that included research on human embryonic 

stem cells. The Commission on Science and Technology awarded a 

total of $5 million to 17 research teams. 

In 2006, the finance committee of the General Assembly passed a $250 million bill to build stem cell research facilities in New 

Brunswick, Camden, and Newark. One hundred fifty million dollars of this was for a joint Rutgers-Robert Wood Johnson Stem Cell 

Institute in New Brunswick. And just last year, New Jersey awarded grants totaling $10 million to stem cell researchers, including 

two grants to fund core laboratories for embryonic stem cell research. 

Despite polls that show that the majority of New Jerseyans were 

in favor of supporting embryonic stem cell research, a referendum 

was defeated in 2007 that would have provided $450 million over 

10 years to support all stem cell research, not only embryonic stem 

cell research. The major reasons for defeat of the referendum were 

believed to be the off-year election, with fewer than 30 percent of 

voters coming to the polls, and the concern that this would add to 

the public’s tax burden. 

Governor Corzine continues to be a strong supporter of stem cell 

research and the building of the joint Robert Wood Johnson-Rutgers Stem Cell Institute in New Brunswick. Key members of the 

New Jersey legislature also continue to strongly support stem cell 

research. 

For the past 2 years, over 50 investigators from academia and 

pharmaceutical companies in New Jersey have been meeting 

monthly to report their work in stem cell research, to discuss 

progress in the field and to plan collaborative experiments. Two 

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types of stem cells are found in the bone marrow: hematopoietic 

stem cells, that form blood cells; and mesenchymal stem cells, capable of differentiating or forming, for example, bone or cartilage or 

nerve cells. Hematopoietic stem cells are now used at Robert Wood 

Johnson Hospital and throughout the world to treat patients with 

cancer following chemotherapy or immune diseases. Mesenchymal 

stem cells from bone marrow or cord blood are being tested for 

their ability to prevent graft vs. host disease after marrow transplantation, and other uses under study by New Jersey investigators 

include targeting tumors with mesenchymal stem cells carrying 

toxins, and use in regenerative medicine, in particular spinal cord 

injury and damaged hearts. 

Researchers at both Rutgers and UMDNJ have special expertise 

and interest in neural stem cells that have the potential for treatment of brain disorders as well as to serve as models to promote 

drug discovery. 

We know that cord blood, placenta, and amniotic fluid are also 

a rich source of stem cells. Clinical trials are in progress, for example, by Wise Young from Rutgers, with collaboration of investigators in China using a subset of cord blood cells to treat spinal cord 

injury. The characterization of stem cells from placenta is under 

study by Robert Wood Johnson Medical School investigators in collaboration with Celgene, a New Jersey-based biotech company. 

Work on human embryonic stem cells, as you heard, has been 

hampered by Federal guidelines that limit studies to 20 cell lines 

that have been around for several years and have limitations. Rutgers and Robert Wood Johnson Medical School stem cell researchers with New Jersey State funding have been able to expand research activities using newly established embryonic stem cell lines, 

and importantly, the completion of a GMP facility at the Cancer Institute/Stem Cell Institute which allow stem cells to be produced in 

quantities necessary for clinical studies. 

The funding provided by the State of New Jersey has provided 

key support for both the research outlined above and additional research focused on a variety of important disease conditions including multiple sclerosis, Parkinson’s disease, Alzheimer’s disease, and 

diabetes, and a key part of our efforts has been the establishment 

of stem cell banking of umbilical cord blood and other stem cells. 

In New Jersey, stem cell banks are leaders in this field. 

I would be happy to answer any of the committee’s questions. 

Thank you very much. 

[The prepared statement of Dr. Bertino follows:] 

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Mr. PALLONE. Thank you, Dr. Bertino. 

Dr. Fraser. 

STATEMENT OF JOHN K. FRASER, PH.D., PRINCIPAL 

SCIENTIST, CYTORI THERAPEUTICS 

Dr. FRASER. Good morning, Mr. Chairman, members of the Committee. Thank you for this opportunity. 

My name is John Fraser, and I am principal scientist at Cytori 

Therapeutics, Inc., a publicly traded adult stem cell company based 

in San Diego, California. Cytori is at the forefront of bringing adult 

stem cells to patients as we are currently selling a stem cell-based 

product in Europe, conducting three separate clinical trials and 

have a technology which has now been used in over 200 patient 

procedures. From my graduate studies in New Zealand through to 

a post-doctoral and faculty appointment to UCLA, my entire research career has been in the field of adult stem cells. 

The topic of today’s meeting is consideration of stem cells as the 

future of medicine, and indeed, stem cells will be an important part 

of the clinical armamentarium going forward. But as we have 

heard, this is nothing new. Hematopoietic stem cells have been 

used in medicine for at least 50 years, and we referred earlier to 

the pioneering work performed in the late 1950s by Dr. E. Donnall 

Thomas, who performed bone marrow transplant studies that ultimately led to his award of the Nobel Prize for Medicine in 1990. 

Like many, I consider 1961 as the birth date of the stem cell field 

as that was the year that James Till and Ernest McCulloch published research that led to the description of the very first stem 

cell, the hematopoietic stem cell, still widely considered to be the 

model for all adult stem cell types. 

Hematopoietic stem cells make bone marrow transplants possible. This is because they have the ability to regenerate the entire 

blood system of the recipient for the rest of that person’s life. Simply put, hematopoietic stem cells are the regenerative engine of the 

blood system. In my opinion, this is a key point of distinction between adult stem cells and embryonic stem cells. Embryonic stem 

cells are capable of immense proliferation and essentially universal 

plasticity. This is because they are, first and foremost, developmental cells. They are derived from a cell mass from which the entire organism develops. 

By contrast, adult stem cells are, first and foremost, regenerative 

cells responsible for maintaining and healing organs and tissues in 

the face of daily wear and tear, injury and disease. They are, by 

their nature, repair cells. They act in response to a need and they 

shut off once that need is completed. One way to look at this is to 

view embryonic stem cells as responsible for generating all the tissues of an organism while adult stem cells are responsible for 

maintaining and healing them. 

The natural role of adult stem cells in repair and regeneration 

makes them ideally suited to clinical use. This has been proven in 

tens of thousands of bone marrow transplant patients over the last 

40 years. This paradigm, as you have heard, is now increasingly 

being repeated as other adult cell types associated with repair and 

regeneration are being applied in different diseases. 

89 

In our own case, Cytori has initiated several clinical studies 

using cells obtained from the patient’s own fat, adipose tissue, 

which is recognized as one of the richest and most accessible 

sources of adult stem cells. The goal of these studies is to bring 

forth new treatments for the millions of patients suffering from 

heart disease as well as other issues such as reconstructing the 

breast following partial mastectomy. We also intend to start studies in intervertebral disc repair. 

Other researchers have published case reports and small clinical 

studies using fat tissue-derived stem cells and treating certain 

kinds of wound complications with bone marrow, GHVD, and in 

bone defects. Published preclinical studies have indicated potential 

in treating renal damage associated with chemotherapy, preserving 

dopaminergic neurons in a Parkinson’s disease model, treatment of 

liver damage, ischemic, and hemorrhagic stroke, and in tissues as 

disparate as the cornea, the lung, and the vocal fold. 

Published clinical studies with other types of adult stem cells 

have shown improvement in cardiac function, in inherited brittle 

bone disease, liver disease, and peripheral vascular disease, to 

name but a few. 

However, as you have heard, there are still many unanswered 

questions, and clearly, additional science is needed. In certain settings, the mechanism through which adult stem cells provide benefit is not well understood. It is also not yet clear which adult stem 

cells provide greatest efficacy in which diseases. These are important questions that companies such as Cytori have neither the resources nor oftentimes the incentive to address. 

For example, certain potentially beneficial populations fall outside of patent protections, providing limited incentive for companies 

to invest their resources in proving a technology that may then be 

applied without their participation. Without Federal support, much 

of this promise could be left to wither on the vine. 

Cytori believes that ultimately science and the marketplace will 

determine which technologies will succeed. We have looked at the 

field of regenerative medicine, performed our own basic science, 

preclinical, and now clinical research, and we are optimistic regarding the ability of our approach to harness the natural role of adult 

stem and regenerative cells to provide clinically effective and costeffective treatments for a range of human diseases in the near future. 

We urge your continuing support of adult stem cell research. 

Thank you. 

[The prepared statement of Dr. Fraser follows:] 

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Mr. PALLONE. Thank you, Dr. Fraser. 

We will take questions now, and I will recognize myself for 5 

minutes initially. 

I have to start with you, Dr. Fraser, because of what you said 

originally, and I noticed that you didn’t make any reference in your 

statement now to the fact that—and I will go back to what you said 

in the previous one, that increasing funding to embryonic stem cell 

research means a decrease in funding to other stem cell research. 

I don’t want to get into it, but basically we had one statement earlier in the evening and then it was revised, you know, based on 

what you said today, and I don’t see any more reference to this 

idea that increasing funding to embryonic means a decrease in 

funding to others. So why did you take that out? 

Dr. FRASER. Sir, I received my formal invitation to attend this 

meeting while I was at the airport in San Diego on my way here. 

At that time there was a version of my testimony which was under 

review and was sent to committee staff before it had been completed. I contacted committee staff—— 

Mr. PALLONE. Well, no, you are more than welcome to change it. 

I am just asking why. Why is it no longer—— 

Dr. FRASER. I think the initial comments that I made overstated 

the position. I think the point that was made there and which is 

no longer is that you have to make difficult decisions. You can’t 

find everything, and for every dollar you take away—sorry—every 

dollar you add somewhere else, you have to take it away from 

somewhere else, and I—— 

Mr. PALLONE. Well, not necessarily, but—— 

Dr. FRASER. Well, that would be nice, but we all know the realities of the current fiscal and economic situation. I am simply encouraging you not to take away funding from adult stem cell research. 

Mr. PALLONE. Well, I don’t think we are suggesting that, but I 

mean, do you support embryonic stem cell research? 

Dr. FRASER. The company has an official position which says we 

have no official position regarding embryonic stem cell research. 

Mr. PALLONE. What about you personally? 

Dr. FRASER. I am not here as an individual, I am here representing the company. 

Mr. PALLONE. So you just basically have no response to that 

question? 

Dr. FRASER. Well, sir, I have spent my entire career in adult 

stem cells. That was not a conscious decision. That was pretty 

much an accident when I was in graduate school. I am very happy 

with where I am. I am certainly not saying—— 

Mr. PALLONE. I am just trying to find out whether you support—— 

Dr. FRASER. I am not—— 

Mr. PALLONE [continuing]. Support embryonic stem cell, and you 

don’t want to answer that? 

Dr. FRASER. Embryonic stem cells are valuable, and research 

that has been performed under the NIH funding with the current 

situation has produced valuable insights. 

Mr. PALLONE. All right. I will leave it at that. Thank you. 

97 

Let me ask Dr. Bertino a couple of questions, and thank you 

again for being here today. I am obviously proud of my home State 

in that we were the first to publicly finance embryonic stem cell research, and of course, the new Stem Cell Institute, which is going 

to be in my district in New Brunswick, but given what our State 

and many other States are doing in terms of taking the initiative 

on their own to advance embryonic stem cell research, some have 

argued that there is no need for additional Federal funding, you 

know, the States and private sector can do it on their own. But can 

you speak to this? Do you believe that New Jersey and other States 

with similar initiatives have enough financial resources to achieve 

the full potential that stem cells may hold, or do you think there 

is a need for additional Federal funds? 

Dr. BERTINO. I think there is clearly a need for additional Federal funds. 

Mr. PALLONE. I think the mic is not on, Doctor. There you go. 

Dr. BERTINO. What we are seeing already is that more and more 

investigators are becoming interested in stem cell research because 

of the tremendous impact this paradigm shift is having on medicine, and as we attract the youngest and most talented researchers 

in this area, we have to provide them with funds, and the State 

at this level cannot take care of all the exciting research that is 

possible. I think if the stem cell bill was approved and we did get 

the $450 million over 10 years, I think that would have been a 

major step in supporting all the good research in the State, but 

that didn’t happen. 

Mr. PALLONE. And what about the money that is being spent in 

stem cell research in the United States versus, you know, in other 

parts of the world? Is the United States on par with other countries; are we falling behind? Is that going to imperil our ability to 

recruit top researchers unless we spend more money by comparison 

to other countries? 

Dr. BERTINO. I don’t know the details and I can’t really answer 

that question. There are pockets of good research money for stem 

cell research from different States,—I think Connecticut, New Jersey, California—but there are many States that have not stepped 

up to the plate. 

Mr. PALLONE. Well, let me ask Dr. Daley, if you don’t mind, in 

terms of United States versus other countries and whether we are 

doing enough and may fall behind and not maybe get researchers 

to come here. 

Dr. DALEY. I think one of the real issues is the supply and demand. The real question is, how many—we have a huge number 

of very, very gifted scientists here in the United States, many of 

whom I think have been scared off from the embryonic stem cell 

field because of the political concerns and the lack of funding. In 

other parts of the world, and I think about Singapore and China, 

they have specifically invested in this area because of the vacuum 

left by the Federal policy in the United States. I have heard that 

directly from representatives of the Economic Development Board 

of Singapore. They want to know what we are not able to do because they want to invest in that, because that gives them a competitive advantage. So I think, you know, it is always hard to say 

what might have been but I can tell you that had we had a more 

98 

expansive Federal policy, the kinds of breakthroughs we are seeing 

today might have happened years ago. We might have been even 

further along. I think the United States—I am still very, very bullish on what the United States can do and contribute in stem cells 

and I hope that the Federal policy will get behind the scientists because we enjoy the greatest community of scientists in the world. 

Mr. PALLONE. Thank you. Thank you to all of you. 

Mr. Deal. 

Mr. DEAL. Thank you. 

I thank all of you for being here today. You certainly have some 

varied points of view here. As I listened to all of you, though, I 

think I detected at least three examples of successful clinical applications of adult stem cells, I think Dr. Patel, Mr. Rice, and Dr. Fraser specifically. Maybe I missed it, but did any of you suggest that 

there are successful clinical applications of embryonic stem cells? 

Dr. DALEY. This is really an interesting question that keeps coming up. There is no way that a cell which was discovered only 10 

years ago would be able to compete with the clinical results of 

hematopoietic stem cells, which were introduced into therapy in the 

1950s. It took 30 years before the discoverer of bone marrow transplant, E. Donnall Thomas, was actually recognized with the Nobel 

Prize for that. I think it is really unfair to hold embryonic stem 

cells to the same kind of standard. They are new. This is a new 

technology. 

Mr. DEAL. I wasn’t questioning whether it was fair or not. I was 

questioning about what the facts are. 

Dr. DALEY. Well, the facts are that this is a fresh, new technology which is finding its way into the laboratories and will ultimately find its way into having a clinical impact. I think we have 

a responsibility to educate the public that scientific cures don’t 

happen overnight, that this is a very long and tedious path and it 

involves basic investments. The NIH has been tremendous for supporting basic research and we enjoy the tremendous benefits in our 

healthcare system, we enjoy the tremendous benefits in our biotechnology industry, but we are at risk of not taking advantage of 

the tremendous possibility of embryonic research because of a Federal policy which has limited investments in that very exciting 

area. 

Mr. DEAL. Well, I think the answer was, I did not hear any, and 

the second question then, Dr. Daley, since you have taken it on in 

the context of—— 

Dr. DALEY. I think you need to ask that question in another 10 

years. 

Mr. DEAL. All right. Well, that is my next question—— 

Dr. DALEY. And then we will see how things stand. 

Mr. DEAL. —if you will let me ask it. How soon do you expect 

clinical applications from embryonic stem cell research to be used? 

Dr. DALEY. So I want to say that it is very important that we 

educate the public about the nature of medical discovery. After I 

leave this hearing, I am flying to Chicago where the International 

Society for Stem Cell Research is convening its clinical translation 

task force. We have a group of 30 scientists, and bioethicists from 

all over the world who are tackling the question of what is a prudent approach to translate this new science of stem cells into real 

99 

clinical therapies. We already know there are companies that are 

attempting to commercialize both adult and embryonic stem cells. 

You heard reference to the Geron Corporation, which may in fact 

introduce the first clinical trial of an embryonic stem cell-derived 

cell to treat spinal cord injury. There is a big difference and a delay 

between the first introduction of a treatment into human patients 

and realizing real clinical benefit. If you look back at the history 

of medical technology, whether we are thinking about therapeutic 

antibodies or drugs, there is often a 20-year time lag. I would anticipate that we have to take another 10 years, so 20 years after 

the original introduction of embryonic stem cells, before we start to 

see therapies based on stem cells. 

Now, in the much nearer term, we are already benefiting from 

25 years of understanding mouse embryonic stem cells. In 1981, 

mouse embryonic stem cells were first isolated, won the Nobel 

Prize for Martin Evans this past year. There have been countless 

numbers of mouse models of human disease that have been generated, funded by the NIH which have revolutionized our understanding of cardiovascular disease, neurodegenerative disease, cancer and the like. So in indirect ways, that investment in basic research is translating into cures. 

Mr. DEAL. Well, you are not suggesting, though, that we should 

not continue research and investment in adult stem cell research, 

are you? 

Dr. DALEY. I think my testimony clearly stipulates that we need 

a vigorous and increased support for all forms of stem cell research. 

We are having a very difficult time as scientists right now through 

the NIH because the budget has been kept flat. We had a doubling, 

and it created a tremendous infusion of talent, great, high-caliber 

talent into American science, and now we are seeing a receding because we can’t support all that momentum. 

Mr. DEAL. We are very proud of our side for being able to double 

that budget on our side, so join with us to get some pressure on 

these folks to make sure we keep that 5 percent as a minimum increase every year. 

I think my time is probably expired. Thank you, Mr. Chairman. 

Mr. PALLONE. Thank you, Mr. Deal. 

I recognize the gentlewoman from Colorado, Ms. DeGette. 

Ms. DEGETTE. Thank you very much, Mr. Chairman. Before I 

question, I have two unanimous-consent requests. The first one is 

that I be allowed to submit testimony of Dr. Debra Mathews on the 

ethics of stem cell research. We have cleared this with the Minority. For the record, we tried to get Dr. Mathews to come but because of the short notice, we were unable to. And my second UC 

request is to submit Dr. Zerhouni’s chart that he referred to in his 

testimony for the record. 

Mr. PALLONE. Let me also mention that I have the copies of the 

documents that the gentlewoman from North Carolina gave me. I 

am no less knowledgeable on the subject after having glanced at 

them than I was before but I would also unanimous consent that 

they be submitted as part of the record. 

Without objection, all four documents will be submitted. So ordered. 

[This information was unavailable at time of printing.] 

100 

Ms. DEGETTE. Thank you very much, Mr. Chairman. 

I only have 5 minutes so I am going to ask the panel if they 

would mind giving short answers to my questions if possible because I have a lot of ground to cover. 

I wanted to ask you first Dr. Gearhart, as a researcher, has the 

research community found that the restrictions on Federal funding 

for embryonic stem cell research that were enacted in 2001 affected 

research in the area of embryonic stem cell research? 

Mr. GEARHART. Well, it has. 

Ms. DEGETTE. And briefly, how has that—— 

Mr. GEARHART. In several ways. One Dr. Daley referred to is students and post-docs and fellows coming to the lab and looking at 

long-term support in this area, very problematic in this country as 

we look back in 2001, we didn’t know where it was going, and this 

was before there was a big—— 

Ms. DEGETTE. And so it is limiting the number of people who 

want to go into that type of research? 

Mr. GEARHART. Well, yes. They have to be practical and look to 

see what kind of a future there is. 

Ms. DEGETTE. And do you think that the research itself would 

benefit if a greater number of embryonic stem cell lines were allowed under the Federal—— 

Mr. GEARHART. Oh, absolutely. I think we have arguments for 

utility, performance and safety that trump all of that, and there 

are many experiments that we don’t want to do with some of the 

existing lines. It is not worth the effort. 

Ms. DEGETTE. And someone, I think Ms. Capps, asked Dr. 

Zerhouni about the Federal funding for facilities and how people 

were having to build parallel labs. Are you finding that also happening in the research community where private universities or 

other groups are feeling like they can’t use anything that has had 

Federal funding involved with it? 

Mr. GEARHART. Well, we do. It varies from institution to institution. At Hopkins, the decision was made, not by us, that we could 

use the same facility but the bookkeeping from where someone’s 

funding is coming from as either salary or supplies, we have to 

mark all of this as to which one is federally approved, which is not 

federally approved. It becomes a bookkeeping and practical nightmare under those conditions. 

Ms. DEGETTE. And at other facilities, they have determined that 

if there is any Federal funding in those labs—— 

Mr. GEARHART. That is correct. They will build a separate lab. 

Ms. DEGETTE. They are building separate labs. 

Mr. GEARHART. Absolutely. 

Dr. BERTINO. In New Jersey, we have built separate labs because 

it is too much of a hassle. 

Ms. DEGETTE. And also in Colorado, by the way. 

Dr. Daley, I wanted to ask you, you are the president of the 

International Society for Stem Cell Research, and someone asked 

you briefly about the international implications, but I have learned 

through talking to researchers at the international level that the 

U.S. restrictions are also hurting the international research because of collaboration issues. If a scientist in Singapore, for examVerDate Nov 24 2008 16:13 Mar 16, 2010 Jkt 037690 PO 00000 Frm 00106 Fmt 6633 Sfmt 6633 C:\DOCUMENTS AND SETTINGS\CWELLS1\HEARINGS\110-115 SCOM1 PsN: JIMC
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ple, wants to collaborate with a U.S. scientist, the restrictions are 

having an impact on that. Is that correct? 

Dr. DALEY. Oh, absolutely. 

Ms. DEGETTE. Could you explain briefly why that is so? 

Dr. DALEY. Yes, well, it is not only international, it is interstate 

concerns. I mean, I have a colleague, a very respected colleague, 

Sean Morrison in Michigan, who can’t do the kinds of research that 

I do in my own lab because it is restricted in Michigan so that limits the kinds of collaborations that we can have. Science is increasingly a global activity. We are about to have our international 

meeting, we will have 2,500 scientists from all over the world, and 

we have this patchwork quilt of regulations. It is not good for 

science. 

Ms. DEGETTE. And would it also be fair to say that it would be 

helpful to have a national ethics oversight system for the research 

that is being done here, much like—— 

Dr. DALEY. No doubt. 

Ms. DEGETTE. —in the United Kingdom and in other countries? 

Dr. DALEY. No doubt. 

Ms. DEGETTE. Now, it sounds like it was actually your skin cells 

that were used in this iPS experiment. Is that right? 

Dr. DALEY. Well, I tried, but my skin cells didn’t yield an iPS 

line. 

Ms. DEGETTE. Well, the iPS research, I am assuming that hasn’t 

led to any kind of clinical cures for anything, even though it has 

been touted by some as the alternative to embryonic stem cell research, has it? 

Dr. DALEY. No, it hasn’t. 

Ms. DEGETTE. And I would also expect that since that research 

is 10 years behind human embryonic stem cell research and 20 or 

30 years behind mouse embryonic stem cell research, the clinical 

applications for iPS are going to be that much farther out down the 

road from now, correct? 

Dr. DALEY. Well, we are hopeful that we can piggyback on some 

of the embryonic stem cell research and accelerate that. 

Ms. DEGETTE. If we expand embryonic stem cell research lines 

that Federal funding can be used for, would you expect that that 

would also help your iPS research then? 

Dr. DALEY. Yes. 

Ms. DEGETTE. Why is that? 

Dr. DALEY. Well, I mean, we still don’t know enough about these 

iPS cells to even know and predict with confidence we will ever be 

able to use them in patients. I am confident that they will be valuable for modeling disease. We are already doing that in our own 

laboratory, and I think it is a very important point that so much 

of the debate has focused on whether or not stem cells will directly 

cure disease, but I want to reiterate the value of basic research and 

the fact that these stem cells are really changing the paradigm of 

that research. 

Ms. DEGETTE. And this is exactly what Dr. Zerhouni was talking 

about, isn’t it? 

Dr. DALEY. Actually, what Dr. Zerhouni was arguing, and it is 

the first time I have really heard it argued so compellingly—in fact, 

I would love to have him come and give that speech to my stem 

102 

cell research laboratory—is that all of the questions asked by scientists about stem cells are really the same. It is about programming of cell fates, and so we never have these kinds of disagreements at our scientific meetings about embryonic versus adult. 

This is a debate that happens in Congress. 

Ms. DEGETTE. Thank you very much. 

Mr. PALLONE. Thank you. 

Mr. Pitts. 

Mr. PITTS. Thank you, Mr. Chairman. 

Dr. Gearhart, do you support gestating human children to later 

fetal stages to harvest issues to treat disease? 

Mr. GEARHART. Absolutely not. 

Mr. PITTS. Does anyone in the panel support that? OK. 

Dr. Daley, do you think that the Federal government should fund 

somatic cell nuclear transfer or cloning for research? 

Dr. DALEY. I do support it because I think it has enormous medical implications. The study of somatic cell nuclear transfer research, I do support that, yes. 

Mr. PITTS. And so you think that should be legal? 

Dr. DALEY. It is legal. 

Mr. PITTS. And you think it should remain legal. Do you think 

that the Federal government should fund research in which animal 

eggs and human cells are mixed to create embryos that are part 

animal, part human? 

Dr. DALEY. I believe that this range of experiments that you are 

defining are best left to the experts in the scientific community to 

set the priorities. I do believe that there are scientific arguments 

to support that area of research as has been supported by the 

United Kingdom. So, yes, I do believe that that is a potentially valuable area of research and it should be under the purview of the 

scientific community. 

Mr. PITTS. And that should be legal? 

Dr. DALEY. It is legal. 

Mr. PITTS. It is legal and should remain legal? 

Dr. DALEY. Yes. 

Mr. PITTS. Dr. Patel, how many patients have you treated for 

heart disease with adult stem cells? 

Dr. PATEL. In our team, we have treated over 100 here in the 

United States but we have had over 30 groups from around the 

world come and train and try the different techniques. The key is, 

we do it in a very regulated and ultimately our goal is to have it 

as safe as possible so now that some of the trials have evolved to 

phase III trials, both in Germany and in Brazil, where they are all 

federally-funded trials since they are mostly bone marrow-derived 

treatments. The problem is that even though we treat patients as 

still experimental, there are people who try to do these as approved 

or unregulated therapies, and that is our biggest concern irrespective of the cell, and we do worry that when you take the more 

multipotent cells, that we are going to see severe adverse events 

which could potentially shut down our entire field just due to the 

fact that patients are going to these countries and having these unregulated therapies. So we are actually very happy that the NIH 

has created these centers for at least cardiovascular disease where 

103 

we could offer these type of treatments in controlled trials here in 

the United States today. 

Mr. PITTS. Do you agree that adult stem cells show promise only 

for blood diseases or autoimmune diseases and that they don’t 

show as much promise as embryonic stem cells for things like Parkinson’s or spinal cord injury or macular degeneration or diabetes? 

Dr. PATEL. Well, my expertise is cardiovascular disease. 

Mr. PITTS. What about the heart? 

Dr. PATEL. So in the heart, adult stem cells show great promise 

and there are many different types that we need to continue to 

work that actually can differentiate in the lab to new heart muscle 

and blood vessels. The key is safely translating those therapies into 

patients. So in terms of other diseases, there are clinical trials for 

type 2 diabetes, also for Parkinson’s and also for spinal cord disorders but currently they are not ongoing in the United States. 

These are all trials that are either in Europe or in South America 

that are funded by their governments, and hopefully as some of 

these posters and presentations are presented at the ISSCR and 

the ISCT, that as the academic community goes through these 

trials, we can hopefully bring these back to the United States and 

see if we can replicate them, just as the iPS cells were originally 

created in Japan and Dr. Daley’s group along with others were able 

to reproduce that so that will advance the field and also keep it a 

very safe therapy. 

Mr. PITTS. Dr. Daley, you support human cloning. You stated, I 

think, yesterday that human cloning is necessary to do iPS research. Since there are no human cloned embryonic stem cell lines, 

yet there are 124 human iPS lines including at least 15 human iPS 

cell lines that you have developed according to your publication online in Nature at the end of 2007, how do you justify that statement? 

Dr. DALEY. Mr. Pitts, I am very pleased that you are reading my 

paper in Nature. 

Mr. PITTS. My staff did. 

Dr. DALEY. Oh, OK. Well, if you read that paper or your staff and 

some of my other publications, I think you would see the justification, and that I have written that there is a strong distinction between your use of cloning and the legitimate medical applications 

of copying cells, copying cells so that we can learn about this reprogramming process that Dr. Zerhouni described. It is a fascinating and important fundamental question in biology. We still 

don’t know whether the reprogramming we are inducing with these 

candidate genes is the same process of the reprogramming that 

happens with nuclear transfer. We think this is a frontier of medicine with enormous potential, and I think that we should allow the 

scientists to explore and use all of the tools available to them subject to very rigorous and very scrupulous scientific and ethical review, and that has been done for my own experiments through at 

least four different institutional review committees. 

Ms. DEGETTE [presiding]. The gentleman’s time has expired. 

The gentlelady from California. 

Ms. CAPPS. Thank you, Madam Chairwoman. 

I want to continue this line of thought. We need to have several 

more things on this, Madam Chairwoman. This is a very important 

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issue. To follow along the previous questioner, Dr. Daley, we are 

confused often here and I think the media is too, which influences 

us a lot. Do you support reproductive cloning? 

Dr. DALEY. No, I don’t. 

Ms. CAPPS. And maybe you want to take a minute, this is a big 

issue. When you talk about human cloning, people get really scared 

and react with sort of blanket prohibitions. Could you just expand 

a little bit on that so we understand clearly? And then this harkens 

back to me, this need for ethical oversight, even with respect to 

how other countries are dealing with it and how they are filling in 

the vacuum, as you have said, because we have created one. 

Dr. DALEY. There has been an enormous amount of public debate 

and some scientific discussion about the value versus the risks to 

society of using nuclear transfer. Nuclear transfer is the method 

that has been used in animal biology to perform reproductive 

cloning for many different mammalian types—mice and dogs—and 

there are legitimate scientific reasons to do this and there are 

issues of animal husbandry which have supported this. There is 

also one methodology for using nuclear transfer to establish stem 

cell lines. That has been enormously productive in mice. My own 

laboratory, together with Rudy Jaenisch, has published using nuclear transfer to treat a genetic disease in a mouse. Recently these 

nuclear transfer lines have been produced from primates. It has 

not been done from humans. And I think that much of the enthusiasm is now going to be diverted to producing these stem cell lines 

using the iPS methodology. So my own laboratory is performing an 

enormous amount of experiments on the iPS methodology, but because of the scientific value, the intrinsic scientific value of the nuclear reprogramming, we continue to pursue that. But it is very important to draw the distinction between copying cells and copying 

babies. No one in the scientific community—and I chaired last year 

the International Society’s guidelines on human stem cell research, 

and there was a clear prohibition against productive cloning. So no 

legitimate scientists think that this is an area of great interest, but 

many scientists feel that understanding nuclear transfer so that we 

can reprogram individual cells is highly, highly valuable. And so 

you will see, I think, broad consensus for studying the various 

ways of reprogramming because no one knows yet which way is ultimately going to be the most valuable. 

Ms. CAPPS. And doesn’t this also speak to a federally-established 

set of guidelines that could direct the way this kind of research is 

done so that we can be proud and confident that our scientists will 

clearly be able to distinguish between the various levels of research 

to safeguard the threats that many people are concerned about? 

Dr. DALEY. The NIH has enormous respect from all of the scientists in this country and it has always played a critical role in 

scientific peer review and scientific oversight, and I think it has 

been unfortunate that it has not been able to play its routine leadership role in this critical area of this exploding biology. 

Ms. CAPPS. I want to try to get one other question in, if I can. 

With the description of adult stem cells coming on to the scene and 

they are being lauded as the end-all, then there are many, even 

among our colleagues, who say that well, we don’t need embryonic 

stem cell research then, and I know you have been around this, but 

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clearly for the record. Also in terms of the long-term effects of it, 

do we really know—I think Dr. Patel has alluded to this. It is very 

new technology that we really don’t know the end results. Maybe 

you would use the remaining time to distinguish there. 

Dr. DALEY. It is just—it is far too premature to imagine how we 

are going to use embryonic, neonatal, adult in the many different 

indications. I am confident that we are going to find very, very valuable applications for adult stem cells and that is why we need to 

continue to work in those areas, but why close any doors? 

Ms. CAPPS. Thank you very much. 

Ms. DEGETTE. The gentleman from Texas, Mr. Hall. 

Mr. HALL. I am sorry that I didn’t get to hear all the testimony, 

and I am more sorry than that that I don’t really know how to ask 

what I want to know. I have an illness in my family for which 

there is no cure, and it was illness that was treated for some time 

for Parkinson’s 18 months to 2 years, and on my way back up here 

one time, I asked my wife to give me her file, and I like to read 

everything I can read about Parkinson’s, and by the time I got up 

here, I wanted to go directly to a hospital or doctor’s office because 

I had almost every symptom, but I am 85 years old. I am the oldest 

guy in the Congress. I am the dean of the United States Congress, 

and people think that is bad but it is not nearly as bad as somebody saying don’t he look natural. 

So I ask you this question, and it is a very important question 

to me and I don’t know how to ask it properly, but I think Mr. Rice 

went overseas to have his treatment and it has been suggested that 

we go to India, that that was where the best available treatment 

was. I don’t think we could stand that. Another to Mexico. I am not 

inclined to do that; another to Seattle, that there were some treatments there that was available. And as most acknowledge, it is not 

paid by insurance, and I have had price estimates all the way from 

$25,000 to $40,000 to $60,000, and none of those are too great if 

I thought it would help her for 15 minutes. The decision was made 

that she didn’t have Parkinson’s because the week I read all that, 

I went back and said we will go to Mayo and know what we have, 

and we went to Mayo, stayed 4 days, didn’t want to know if she 

needed an appendectomy or ingrown toenail or anything else. The 

question was, did she have Parkinson’s, and the answer after 41⁄2

days was absolutely not. Three weeks later, a letter back saying 

that, however, she could have peri-Parkinson’s. 

Now, that would be distressing to some but it was hope to me 

because I understand stem cells one day might eradicate Parkinson’s. I have heard that said and that may be an overstatement, 

but what are the facts with the effect of stem cells on Parkinson’s? 

Who should I ask that? 

Mr. GEARHART. We have done some work on this. The stem cell 

therapies for Parkinson’s actually began by using portions of fetal 

brains that were obtained through abortion in northern Europe. 

This was a standard measure of care. Patients receiving these cells 

did improve over a period of time and then they lost that improvement and came back to what they were before. These cells really 

weren’t stem cells. These were fully formed dopaminergic neurons, 

the cells that are lost here, and they just don’t hook up appropriately when they are fully formed. There was a clinical trial in 

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the early 1990s here in Denver that reported the same thing pretty 

much. The newer technologies that are being worked on in the laboratory, and this is all through now animal modeling of Parkinson’s 

disease, in which we can grow in great abundance and derive and 

grow dopaminergic neurons from embryonic stem cells. It is one of 

the most robust sources of these cells. These cells have been introduced into various animal models from rats to mice to monkeys in 

which we see very much the same thing. There was a very interesting series of experiments, summary of experiments published in 

Nature recently in which the evidence showed that these cells can 

go in, they can integrate, they can function for a long period of 

time. 

Now, this brings up another issue. Some of these cells that were 

grafted in are beginning to show the cellular basis of Parkinson’s 

disease. We know that there is a certain morphology associated and 

subcellular components that indicate Parkinson’s disease, something we have not mentioned here. We have mentioned only that 

we are growing cells to replace those that are lost. We have said 

very little about the companion compartment of this that is so critical. We have got to learn more about the pathogenesis of disease 

and how to shut it down. We mentioned autoimmune for many of 

the diseases that are at the basis of this. If we don’t learn what 

that is about, putting new cells in isn’t necessarily going to help 

you. 

So what we are seeing, and a short answer here, is that there 

is an improvement in patients, well, at least in animals and in the 

patients that had the fetal tissue grafts, but it is not of long standing. 

Mr. HALL. Let me ask you this, and I note that some asked 

whether or not we were aware that the leading experts on embryonic stem cell research now says treatments from that source may 

be one or two decades or more away. Is that what you are saying? 

Mr. GEARHART. Yes. At the moment, we are going through proof 

of concept experiments. These are laboratory-based animals. 

Mr. HALL. I am getting close to my 5 minutes. 

Mr. GEARHART. Right. So—— 

Ms. DEGETTE. You are over 5 minutes, so if Dr. Gearhart 

could—— 

Mr. HALL. May I ask one more question? 

Ms. DEGETTE. Sure. 

Mr. HALL. If we do avail ourselves of this thrust for stem cells, 

and it has been told to me so simple that you put two stem cells 

in, one finds and destroys and the other takes it place, well, I am 

willing to accept that but I know it is much more than that. But 

is there any danger if the stem cells do not help? 

Mr. GEARHART. Oh, absolutely. 

Mr. HALL. That they will do damage? 

Mr. GEARHART. Yes, absolutely. There is—— 

Mr. HALL. Briefly tell me yes or no. 

Mr. GEARHART. Yes. I would be happy to give you lots of data on 

that. 

Mr. HALL. And I will take that up with the folks that I am talking to. Thank you for that. 

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Ms. DEGETTE. Thank you very much. I really want to thank this 

panel for coming on very short notice. It was an excellent panel, 

and every single witness added to our knowledge. As I mentioned 

at the beginning, this is the first hearing that we have had in the 

Energy and Commerce Committee ever on all of these cell therapies, so it has been very useful and I know on behalf of Mr. Pallone, I want to thank all of you for the Committee. This concludes 

all questioning. 

In conclusion, I want to remind the members that you may submit additional questions for the record to be answered by the relevant witnesses. The questions should be submitted to the committee clerk within 10 days, and the clerk will notify the offices of 

the procedures. 

Without objection, this meeting is adjourned. 

[Whereupon, at 3:15 p.m., the subcommittee was adjourned.] 

[Material submitted for inclusion in the record follows:] 

STATEMENT OF HON. ANNA G. ESHOO

Thank you, Chairman Pallone, for convening another hearing on the important 

topic of stem cell research. Congress has clearly demonstrated our commitment to 

expanding stem cell research in our country. We’ve held hearings, we’ve debated, 

and both chambers passed legislation. Unfortunately, the Administration does not 

share our view. 

The very first veto of President Bush’s was stem cell research and the expansion 

of Federal funding for it. We cannot overlook the necessity and potential of this research and the new treatments and discoveries that will invariably come from this 

exciting area of science, saving lives, and eradicating the pain and suffering of so 

many. I have cosponsored legislation to provide federal funds for stem cell research 

and continue to be a strong advocate on this issue. 

We cannot continue to allow the United States to fall behind our international 

counterparts because of the current restrictions. Our scientists are hamstrung, able 

to only use federal funds on human stem cell lines derived prior to the President’s 

ban in August 2001. As those cells lines age, they undergo biological changes that 

reduce their scientific potential. To be the world’s leader, researchers in our country 

should not be reduced to using old stem cell lines that are of limited value. Our constituents who suffer from diabetes, spinal cord injuries, Parkinson’s, and many other 

diseases are relying on us to give American researchers the tools and resources they 

need to develop new treatments. Stem cell research has far too much potential for 

us to restrict federal funding which limits the hopes and dreams of the American 

people. 

The result of our Federal policy on stem cells today is sending our best scientists 

to research facilities overseas. Those who are still in the U.S. are watching from the 

sidelines and it is only a matter of time when the breakthroughs will occur. 

Stem cells and the treatments and discoveries locked within them represent the 

future of health and medicine. I’m pleased that we are once again bringing attention 

to the issue of stem cell research. I thank the witnesses for being here today and 

I look forward to their testimony. My hope is that we can reverse the current federal 

policy and lift up the million of Americans who will benefit from an enlightened policy. 

STATEMENT OF HON. BARBARA CUBIN

Thank you Mr. Chairman. 

Today’s hearing gives this committee a valuable opportunity to examine recent 

breaththroughs in stem cell science. Stem cells are literally building blocks of 

human life. They hold the promise of curing or treating a host of serious diseases, 

from Parkinson’s and Alzheimer’s to heart disease and diabetes. 

There are several accounts of stem cell therapies that are working right now to 

treat disease. Doug Rice of Washington State, who will be sitting on our second 

panel today, will share the improvements he has experienced with his heart condition using stem cells isolated in his own bloodstream. 

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Blood stem cells have also been used by researchers from Northwestern University and Brazil to successfully treat type 1 diabetes. Thirteen of the fifteen patients 

involved in the trial became insulin-free according to the Journal of American Medical Association. 

As a strong supporter of Alzheimer’s research, I am particularly encouraged by 

research at the University of California, Irvine, in which scientists are using stem 

cells to restore the memory of mice. The research could lead to breakthroughs not 

just for Alzheimer’s, but also stroke and traumatic brain injury. 

Perhaps one of the more exciting stem cell advances is the development of induced 

pluripotent stem cells. In this astonishing process, genes are added to ordinary skin 

cells in order to create stems cells with potentially therapeutic applications. While 

the science and its application to humans is still developing, the cells are believed 

to be pluripotent, that is, capable of differentiating into any cell type. 

All of these treatments and potential treatments have one vital characteristic in 

common. Their stem cells were derived in ways that did not involve the destruction 

of a human embryo. The induced pluripotent cells in particular hold the promise to 

be just as versatile as embryonic stem cells, both in treatment and for research purposes. 

I cannot support Federal funding for embryonic stem cell research that harms or 

destroys any human life. As we work tirelessly to improve the health of the ill, this 

is still no justification for taking another human life. Moreover, no embryonic stem 

cell has been used to treat disease or injury, while adult stem cells are being used 

clinically at this very moment. 

This hearing is entitled, ‘‘Stem Cell Science: The Foundation for Future Cures.’’ 

With induced pluripotent stem cells, we have an ethical foundation for future treatments. With other adult stem cells, the future is now. The Federal Government 

owes it to millions of disease suffering Americans to support the development of 

these therapies. 

With that, I welcome our panelists. Thank you Mr. Chairman. I reserve the balance of my time. 

STATEMENT OF HON. LOIS CAPPS

Thank you, Chairman Pallone, for holding this hearing. 

Even though our current Administration has prohibited federally funded embryonic stem cell research, America’s biomedical research community has continued on 

with this important work. 

Our Nation’s leading scientists know the facts. 

They know that both adult and embryonic stem cell research hold the potential 

to cure some of humanity’s most devastating diseases: 

Cancer, Diabetes, Parkinson’s, Alzheimer’s and I’m sure, many more. 

I’m so pleased to have some of those leading scientists with us here today. 

To share with us the truth about stem cell research. 

About the nature of embryonic stem cell research and about the promise of adult 

stem cell research. 

Adult stem cell research is crucial. 

We need it. 

But we need embryonic stem cell research, too, because one is not a replacement 

for the other. 

They are two pieces of a large puzzle. 

I am proud that my own state of California has been a leader in this field and 

filled in some gaps where the federal government has been absent. 

But state and private funding are only pieces of the puzzle. 

Federal dollars, predominantly through the NIH, are the primary source of funding for basic research— 

The kind of research that identifies the fundamentals for future research that will 

eventually lead to cures. 

It is quite frankly embarrassing to have taken this big step backward over the 

past few years as the rest of the world has soared ahead. 

But again, I’m so thankful that we have scientists, health care professionals, patients and other advocates who have found ways to keep research going so that we 

won’t waste any more time in our quest for those cures. 

Finally, I’d like to thank my colleague, Diana DeGette, for her tireless leadership 

on this issue. 

I look forward to hearing from today’s witnesses. 

I yield back. 

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STATEMENT OF HON. EDOLPHUS TOWNS

Let me thank you Chairman Pallone and Ranking Member Deal for holding this 

timely hearing on ‘‘Stem Cell Science: The Foundation for Future Cures.’’ 

Embryonic stem cells may hold the key to curing a host of debilitating conditions 

that affect millions of people around the globe. These diseases include Parkinson’s 

disease, diabetes, traumatic spinal cord injury, Purkinje cell degeneration, heart disease, cancer, multiple sclerosis, vision and hearing loss, and others. 

Given advancements in research, it is appropriate that we convene at this time 

to assess current developments in stem cell research, discuss the use of adult stem 

cells versus embryonic stem cells, and explore a new method known as ‘‘somatic cell 

nuclear transfer’’. 

In 2007, I co-sponsored and voted in favor of Representative DeGette’s bill to authorize embryonic stem cell research, and am proud of it passage in Congress. It 

was a dark day for all people who suffer from diseases that may be cured by this 

research when the President vetoed the bill, H.R. 3. 

When the administration imposed additional restrictions on embryonic stem cell 

research with its 2001 embryonic stem cell policy and 2007 executive order, it crippled U.S. research efforts in these areas. Thankfully, Japan and Europe continued 

with their embryonic stem cell research and moved the world forward in the quest 

for cures. It is time that the U.S. resume its place as a preeminent contributor to 

this critical effort. 

To this end, I welcome efforts to create a record of the work of NIH, FDA, the 

private sector, and other countries in the area of stem cell research. This database 

is critical to our coordinated efforts to advance stem cell research as efficiently and 

effectively as possible. 

I wholeheartedly believe that such research can be conducted in an ethical manner. As a God-fearing man of faith, I humbly appreciate it is God who is responsible 

for both diseases and cures. Cures can only come about upon God’s command. I believe he wants us to move forward on research and that we should let him shepherd 

us on this quest, and bring relief to those who suffer from terrible diseases unnecessarily. 

Thank you Mr. Chairman. I respectfully yield back the remainder of my time. 

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DOUGLAS T. RICE, RESPONSES TO QUESTIONS FROM HON. JOSEPH R. 

PITTS

June 19, 2008 

U.S. House of Representatives 

316 Ford House Office Building 

Washington, D.C. 20515 

Congressman John D. Dingell 

Ref: The Honorable Joseph R. Pitts questions regarding testimony on May 8th, 

2008 

Question: Could you please tell us about your experience and difficulties 

in obtaining adult stem cell treatment for your heart condition? What were 

the costs involved? Was insurance coverage available? Was FDA approval 

available at that time? 

Answer: As I testified at the hearing, I was given 3-4 months to live without a 

Heart Transplant, since I was diabetic, I was not eligible and did not want to have 

the Mechanical Heart transplant as I have seen the results and have never seen 

anyone get better. After verifying that no solution was available in the U.S., my exwife went on line looking for new technology and found that in Thailand, a company 

named Theravitae was doing Adult Stem Cell transplants that were successful. 

After meeting with my cardiologist, it was decided I had no other chance to live and 

the risk versus reward was worthwhile. 

The costs were $40,000 plus airfare and you had to take someone with you, total cost was approximately $50,000. I had to borrow the money and move quickly to 

get there in time. 

My insurance including the V.A. would not cover any of it, though there was nothing available in the U.S., luckily I had friends and family that wanted me around 

or I would be dead by now. 

FDA did not allow the use of the Adult Stem Cell in this type of treatment though 

they allowed the use of the ASC in Cancer and other illnesses. They would let you 

draw the blood to send to Israel but not the cath procedure to insert the stem cells. 

There are now successful trials being done in the U.S. using the Adult Stem Cells 

on the heart with tremendous success stories. Also, there is a new clinic using the 

same procedure as Theravitae in the Dominican Republic by an American doctor 

and has been very successful. He has recently saved the hands and feet of a young 

athlete that had lost all circulation there and after ASC treatment has saved them. 

Question: To your knowledge, how many other patients have been treated for heart disease using adult stem cells by the doctors who treated you? 

Answer: I believe that in Thailand, over 200 patients have been successfully treated for end stage heart disease and other heart related diseases. Me being one of 

them, also numerous other ones I have met and speak with. I have been in contact 

with other countries and most are using the Adult Stem Cell treatment to save 

many lives. 

To cover some very valid issues about the Adult Stem Cell treatments that are 

being used in the U.S. 

Almost a million people die every year in America from Heart Disease, there has 

been a valid treatment for years using ASC and yet Billions have been spent on researching the Embryonic Stem Cells ( with no success ) when those funds could 

have used for treatment rather than just research. Over 700,000 Cancer patients 

and other illnesses have been treated since 1959 and every day new success’s are 

being tried and used. 

Why, when a single celebrity dies does the news media cover it for weeks and 

months, yet when almost a million Americans die of a treatable disease, you never 

hear of them? I have tried to get on national media to tell the facts with no success, 

yet Embryonic are discussed all the time and they don’t work at this time if ever. 

Why does Congress have hearings about it and the only thing really discussed is 

how well ESC is progressing, yet not one human treated! What will it take to convince anyone that with more treatment using existing Adult Stem Cells, Americans 

could live with treatment? 

I was privileged to be there to introduce The Patients First Bill last year and yet 

it still hasn’t passed. What is the real reason that a true success in medicine is set 

aside for something that doesn’t work and even scientist say may never? 

I travel as much as I can to educate Americans on the Adult Stem Cell and the 

difference with ′′Fact and Fiction′′ regarding Embryonic Stem Cells. And, believe me 

there is a lot of fiction going on about ESC. 

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Though I try, I am not financially strong enough to really make a difference, but 

I try as best as I can. But how can we let millions of people die every year when 

there is a possible treatment that works now? How do we face the families of the 

ones that could have been treated knowing that we are not doing all we can to help. 

How can you, as their representatives not stand up for them and fight for their right 

to live a better life. How do you sit in meetings and basically just talk about how 

great a job the funds you have allotted for research with ESC has not saved one 

life while the funds you did not fund for ASC could have saved millions? As an 

American, and one that was allowed to live, but had to go to another country using 

American technology to do so, I question the FDA’s line of thinking and to be honest 

our government. 

I hope and pray that this will help move the Adult Stem Cell Story into the news 

and the facts will speak for themselves. 

Respectfully, 

Douglas T. Rice 

Adult Stem Cell Recipient 

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WEYMAN JOHNSON, JR., RESPONSES TO QUESTIONS FROM HON. 

JOSEPH R. PITTS

June 23, 2008 

Honorable Joseph Pitts 

Committee on Energy and Commerce 

2125 Rayburn House Office Building 

Washington, D.C. 20515 

Dear Congressman Pitts, 

Thank you for the opportunity to appear before the Subcommittee on Health on 

Thursday, May 8, 2008 at the hearing entitled ′′Stem Cell Science: The Foundation 

for Future Cures.′′ 

The field of stem cell research brings hope to millions of Americans who are affected by chronic diseases including more than 400,000 who are living with multiple 

sclerosis (MS). The National Multiple Sclerosis Society believes all promising avenues of research must be explored and remains committed to ensuring all types of 

stem cell research is pursued under strict ethical guidelines and in accordance with 

the law. 

Enclosed in this correspondence is my response to the questions Chairman John 

Dingell sent to me on your behalf. I am happy to provide further detail if necessary. 

The National MS Society stands by to serve as a resource to you and any Member 

of the Committee. 

Sincerely, 

Weyman Johnson, Jr., J.D. 

Chairman of the Board 

Enclosure 

Cc:The Honorable John Dingell, Chairman 

Committee on Energy and Commerce 

The Honorable Joe Barton, Ranking Members 

Committee on Energy and Commerce 

The Honorable Frank Pallone, Chairman 

Subcommittee on Health 

The Honorable Nathan Deal, Ranking Member 

Subcommittee on Health 

1) Question: Is the National Multiple Sclerosis Society spending any research funds on ′′somatic cell nuclear transfer′′ or human embryonic stem 

cell research? If so, how much is being spent and what percentage of your 

research budget is allocated for these types of research? 

Response: From the beginning, the National MS Society has funded research seeking clues to the cause, treatment and cure of MS, and to spark research efforts 

around the world. Although MS is not hereditary or contagious, it is believed to 

occur in genetically susceptible people who are exposed to an infectious agent, such 

as a virus or bacterium. These factors combine to cause the person’s immune system 

to attack myelin insulation on nerve fibers. 

The National MS Society is a driving force of MS research, and as such, our research efforts support studies in many different areas of scientific studies from immunology to genetics to understanding ways to repair the damage to myelin. The 

Society is expending nearly $45 million this year alone to propel MS research forward, including funding over 440 new and ongoing MS investigations in the U.S. 

and abroad, across all areas of research. 

Today the most exciting area of research, and one that holds true promise for 

those individuals with MS, is in the area of repair and protection of the nervous 

system. The Society is currently not funding any projects using SCNT. Of the 440 

projects which we are currently supporting, 80 (18%) are focused on repair using 

both human and animal cells. Of the 440 awards, 7 (1.6%) projects are using human 

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embryonic stem cells at an annual cost of $1.24 million (2.8% of our overall annual 

research budget). 

2) Question: Has the National Multiple Sclerosis Society funded any adult 

stem cell research? If so, how much is being spent and what percentage of 

your research budget is allocated for these types of research? 

Response: Some tissues and organs have little capacity for self-repair. One such 

organ is the brain; and nerve cells or neurons are known to be very restricted in 

their capacity to regenerate following damage or disease. The adult brain and spinal 

cord appear to have only a limited ability to produce new neurons. This is one reason why recovery is often limited when the nervous system is injured. 

One of the most exciting frontiers in medicine is the potential use of stem cells 

for treating diseases for which there are no cures. One strategy is by replacing cells 

using embryonic cells, and another strategy is using adult cells - either from a donor 

or by using the patient’s own cells. It is important that both of these avenues are 

pursued. 

Of the 440 projects which the National MS Society is currently supporting, 6 

(1.4%) projects are using human adult stem cells at an annual cost of $2.0 million 

(4.4% of our overall annual research budget). With regards to the use of adult stem 

cells, it is important to clarify the two different approaches which are being studied: 

one is to repair the damage in MS, and the other is to use bone marrow adult stem 

cells in transplantation to reconstitute the immune system. To date, it is the latter 

research which has shown some promise as a treatment in some individuals with 

aggressive MS. MS investigators are currently studying whether bone marrow 

transplantation is an effective treatment in a group of closely matched people with 

MS. Since the immune system is misdirected in MS, the hope is that by transplanting these adult bone marrow stem cells, one can reconstitute a naive immune 

system that will not attack myelin and thereby, will correct itself. 

The second use of stem cells is to repair the damage in MS. We know that the 

damage is occurring in the central nervous system, namely, the brain and the spinal 

cord and the optic nerves. So we need to figure out a way that repair, actually, occurs at the site of the injury. We can broadly divide the research efforts into two 

categories. One is, can we promote the cells that are already there, what we call 

the adult endogenous progenitor cells, to function more effectively, or is the challenge going to be do we have to provide the cells from outside? We know that during 

an attack of MS, the myelin is injured and; we also know that there is an element 

of repair. But how do we stimulate the repair in the body and what is the best 

source of cells to use? If we had ways of directing the function of these adult stem 

cells, then these are the cells that would, actually, be the ones used in the disease 

repair. 

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