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32–808 PDF 2007

S. HRG. 110–208

CAN CONGRESS HELP FULFILL THE PROMISE

OF STEM CELL RESEARCH?

JOINT HEARING

BEFORE THE

COMMITTEE ON HEALTH, EDUCATION, 

LABOR, AND PENSIONS

AND THE

SUBCOMMITTEE ON LABOR, HEALTH AND HUMAN 

SERVICES, EDUCATION AND RELATED AGENCIES

OF THE

COMMITTEE ON APPROPRIATIONS

UNITED STATES SENATE

ONE HUNDRED TENTH CONGRESS

FIRST SESSION

ON

EXAMINING STEM CELL RESEARCH, FOCUSING ON ONGOING FEDERAL 

SUPPORT OF BOTH EMBRYONIC AND NON-EMBRYONIC STEM CELL 

RESEARCH AND SCIENTIFIC PROGRESS, INCLUDING RECENT FINDINGS ON AMNIOTIC FLUID STEM CELLS

JANUARY 19, 2007

Printed for the use of the Committee on Health, Education, Labor, and Pensions

(

Available via the World Wide Web: http://www.gpoaccess.gov 

COMMITTEE ON HEALTH, EDUCATION, LABOR, AND PENSIONS 

EDWARD M. KENNEDY, Massachusetts, Chairman 

CHRISTOPHER J. DODD, Connecticut 

TOM HARKIN, Iowa 

BARBARA A. MIKULSKI, Maryland 

JEFF BINGAMAN, New Mexico 

PATTY MURRAY, Washington 

JACK REED, Rhode Island 

HILLARY RODHAM CLINTON, New York 

BARACK OBAMA, Illinois 

BERNARD SANDERS (I), Vermont 

SHERROD BROWN, Ohio 

MICHAEL B. ENZI, Wyoming 

JUDD GREGG, New Hampshire 

LAMAR ALEXANDER, Tennessee 

RICHARD BURR, North Carolina 

JOHNNY ISAKSON, Georgia 

LISA MURKOWSKI, Alaska 

ORRIN G. HATCH, Utah 

PAT ROBERTS, Kansas 

WAYNE ALLARD, Colorado 

TOM COBURN, M.D., Oklahoma 

J. MICHAEL MYERS, Staff Director and Chief Counsel 

KATHERINE BRUNETT MCGUIRE, Minority Staff Director 

(II) 

(III)

COMMITTEE ON APPROPRIATIONS 

ROBERT C. BYRD, West Virginia, Chairman 

DANIEL K. INOUYE, Hawaii 

PATRICK J. LEAHY, Vermont 

TOM HARKIN, Iowa 

BARBARA A. MIKULSKI, Maryland 

HERB KOHL, Wisconsin 

PATTY MURRAY, Washington 

BYRON L. DORGAN, North Dakota 

DIANNE FEINSTEIN, California 

RICHARD J. DURBIN, Illinois 

TIM JOHNSON, South Dakota 

MARY L. LANDRIEU, Louisiana 

JACK REED, Rhode Island 

FRANK R. LAUTENBERG, New Jersey 

BEN NELSON, Nebraska 

THAD COCHRAN, Mississippi 

TED STEVENS, Alaska 

ARLEN SPECTER, Pennsylvania 

PETE V. DOMENICI, New Mexico 

CHRISTOPHER S. BOND, Missouri 

MITCH MCCONNELL, Kentucky 

RICHARD C. SHELBY, Alabama 

JUDD GREGG, New Hampshire 

ROBERT F. BENNETT, Utah 

LARRY CRAIG, Idaho 

KAY BAILEY HUTCHISON, Texas 

SAM BROWNBACK, Kansas 

WAYNE ALLARD, Colorado 

LAMAR ALEXANDER, Tennessee 

CHARLES KIEFFER, Staff Director 

BRUCE EVANS, Minority Staff Director 

SUBCOMMITTEE ON LABOR, HEALTH AND HUMAN SERVICES, EDUCATION, AND

RELATED AGENCIES

TOM HARKIN, Iowa, Chairman 

DANIEL K. INOUYE, Hawaii 

HERB KOHL, Wisconsin 

PATTY MURRAY, Washington 

MARY L. LANDRIEU, Louisiana 

RICHARD J. DURBIN, Illinois 

JACK REED, Rhode Island 

FRANK R. LAUTENBERG, New Jersey 

ROBERT C. BYRD, West Virginia (ex officio) 

ARLEN SPECTER, Pennsylvania 

THAD COCHRAN, Mississippi 

JUDD GREGG, New Hampshire 

LARRY CRAIG, Idaho 

KAY BAILEY HUTCHISON, Texas 

TED STEVENS, Alaska 

RICHARD C. SHELBY, Alabama

Professional Staff 

ELLEN MURRAY

ERIK FATEMI

MARK LAISCH

ADRIENNE HALLETT

LISA BERNHARDT

BETTILOU TAYLOR (Minority) 

SUDIP SHRIKANT PARIKH (Minority)

Administrative Support 

TERI CURTIN

JEFF KRATZ (Minority) 

C O N T E N T S 

STATEMENTS

FRIDAY, JANUARY 19, 2007

Page 

Kennedy, Hon. Edward M., Chairman, Committee on Health, Education, 

Labor, and Pensions, opening statement ........................................................... 1

Enzi, Hon. Michael B., a U.S. Senator from the State of Wyoming, opening 

statement .............................................................................................................. 3

Harkin, Hon. Tom, a U.S. Senator from the State of Iowa .................................. 4

Specter, Hon. Arlen, a U.S. Senator from the State of Pennsylvania ................. 6

Landis, Story C., Ph.D., Director, National Institute of Neurological Disorders 

and Stroke (NINDS), Department of Health and Human Services, Washington, DC ............................................................................................................. 7

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

Daley, George Q., M.D., Ph.D., Associate Professor of Pediatrics, Children’s 

Hospital, Boston, MA ........................................................................................... 13

Prepared statement .......................................................................................... 15

Stanford, Lauren, Juvenile Diabetes Patient, Plymouth, MA ............................. 16

Wagner, John E., Jr., M.D., Professor of Pediatrics, University of Minnesota 

Medical School, Minneapolis, MN ....................................................................... 19

Prepared statement .......................................................................................... 21

ADDITIONAL MATERIAL 

Statements, articles, publications, letters, etc.: 

RAND Research Brief ...................................................................................... 35

Letter from David A. Prentice and Gene Tarne with supporting materials ................................................................................................................ 51

Response to Questions of Senator Enzi by George Q. Daley, M.D., Ph.D. .. 76

Response to Question of Senator Cochran by George Q. Daley, M.D., 

Ph.D. .............................................................................................................. 77

Response to Questions of Senator Enzi by Lauren Stanford ........................ 77

Response to Questions of Senator Enzi by John E. Wagner, Jr., M.D. ........ 78

Response to Questions of Senator Cochran by John E. Wagner, Jr., M.D. . 79

(IV) 

(1)

CAN CONGRESS HELP FULFILL THE PROMISE 

OF STEM CELL RESEARCH? 

FRIDAY, JANUARY 19, 2007

U.S. SENATE, 

COMMITTEE ON HEALTH, EDUCATION, LABOR, AND PENSIONS, 

COMMITTEE ON APPROPRIATIONS, 

SUBCOMMITTEE ON LABOR, HEALTH AND HUMAN SERVICES, 

Washington, DC. 

The committees met, pursuant to notice, at 9:33 a.m. in Room 

SD–192, Dirksen Senate Office Building, Hon. Edward Kennedy, 

presiding. 

Present: Senators Kennedy, Harkin, Brown, Lautenberg, Reed, 

Sanders, Enzi, Specter, Stevens, Isakson, Murkowski, Coburn, 

Hatch, and Allard. 

OPENING STATEMENT OF SENATOR KENNEDY

The CHAIRMAN. Good morning. We’ll come to order. 

This is a very special day for not only the very important subject 

that we’re considering, stem cell research, but for those of us on 

these two committees: our Health, Education, Labor, and Pensions 

Committee, and the Appropriations Committee, which Senator 

Harkin and Senator Specter are leaders in the Senate and have 

done an extraordinary job over the period of these years, in terms 

of giving focus and life to this subject matter. We’re all partners, 

working closely together. We have a great admiration for the leadership that Senator Harkin and Senator Specter have provided in 

moving us all toward this place where we are today, really on the 

eve, almost, of Senate consideration of this legislation. And we’re 

enormously grateful to my colleague and friend Senator Enzi, who 

has been the chairman of our committee, and who is my partner 

in so many of these health issues and has been a valid and important ally in this undertaking. 

So, I’ll make a brief comment and ask my colleagues if they 

would be good enough to say a brief word, and we’ll move forward 

with a very, I think, distinguished panel that can be very helpful 

in bringing us up to date with the great sense of opportunity about 

stem cell research. 

Today’s hearing is really about hope. And hope is what stem cell 

research brings to millions of Americans who seek cures for cancer, 

diabetes, spinal cord injury, many other serious conditions—hope 

for those with Parkinson’s disease—the tremors of that disease can 

be cured; hope that spinal injury—spinal cord injuries can be 

healed; hope for children with diabetes. The constant worry and 

2

vigilance required to cope with their disease will be a thing of the 

past. 

A week ago, a solid bipartisan majority, in the House of Representatives, voted for hope and new progress in these battles 

against illness, by approving legislation to unlock the potential of 

stem cell research. Now the challenge is before the Senate, and we, 

too, must respond. Many of the Senate’s staunchest of supporters 

of stem cell research are here today. We represent diverse backgrounds and many faiths. We have come to our support of stem cell 

research by different paths, but we have all concluded that this research is one of the great potential breakthroughs of modern medicine, that it brings the potential of fuller, longer life for countless 

people who suffer from debilitating diseases. 

Many of those who oppose this research are here today, too, and 

we welcome their perspective. Those who oppose the research do so 

out of deeply held moral convictions, and we respect their views, 

even as we differ with them. Today, we’ll also hear from the leading scientists about recent advances in stem cell research, their potential to help Americans whose lives have been devastated by disease and injury. Some have suggested these new developments 

avoid the need for the use of stem cells derived from embryos, and 

we will hear the scientific community’s evaluation of that possibility. 

We welcome Dr. Story Landis, who will be the director of the—

she is the director of the National Institute of Neurological Diseases and Stroke, as well as Dr. George Daley, of the Children’s 

Hospital in Boston, Dr. George Wagner, the University of Minnesota. All of them are leaders in the field of stem cell research. 

But today’s hearing is not just a celebration of research, it’s a 

call for change in the search for new cures that have been severely 

limited by the restrictions that President Bush imposed on stem 

cell research 6 years ago when he limited the use of funds to the 

inadequate number of cell lines existing at the time. Last year, 

President Bush vetoed the bipartisan legislation to end those restrictions and offer the hope of fuller and longer lives to millions 

of our citizens. 

Today, we’ll hear of that hope from Lauren Stanford, of Plymouth, Massachusetts. We’ll hear of her courage and dignity in the 

face of diabetes. I was profoundly moved by the letter that she sent 

me during the stem cell debate, last year, describing her hope that 

stem cell research might allow her to live a future free of her illness. I’m sure our colleagues on our two committees will welcome 

the opportunity to hear her words, too. 

Lauren is not alone. She joins Nancy Reagan, dozens of Nobel 

laureates, thousands of scientists, millions of patients across the 

Nation, in calling for an end to the restrictions that have hobbled 

the search for new cures. The debates that we have held in recent 

years have already led many of our colleagues who opposed the research in the past to support it now. It may be too much to hope 

that President Bush will join those ranks, but if he could be here 

today to hear the hopes and dreams of patients like Lauren, surely 

he would have to re-examine his conscience and reconsider the restrictions imposed on the research. Let us all hope that, in a private moment, the President will undertake that re-examination 

3

and signal the acceptance of our new bipartisan stem cell legislation and the hopes of millions of Americans it represents. 

Time has come for Congress and the President to join together 

to unchain the creative energies of America’s scientists and allow 

them to pursue the promise of stem cell research. There could be 

fewer greater triumphs of bipartisan progress than to have the 

Stem Cell Research Enhancement Act signed into law. 

Senator Harkin. 

Senator Enzi. 

OPENING STATEMENT OF SENATOR ENZI

Senator ENZI. Thank you, Mr. Chairman, for holding this hearing. I want to thank the witnesses for coming. 

Throughout the history of our Nation, generations of American 

scientists have looked for ways to improve human condition and 

address the problem of disease and afflictions of old age. As they 

conducted their research, each scientist’s work built on the discoveries that preceded it, and the results they achieved over the years 

have enabled us to live longer, healthier, more productive lives. 

From time to time, there’s a breakthrough, or possible breakthrough, in medical science that has the potential to revolutionize 

not only our ability to diagnose or treat an affliction, but our basic 

understanding of how the human body operates. When that occurs, 

a debate ensures as society attempts to evaluate the new procedure’s potential to address the diseases that threaten our health, 

as well as the ethics of putting the new procedures into practice. 

Such a possible breakthrough is stem cell research. 

At present, its promise and potential for changing the way we 

view health and disease seems limitless. In theory, stem cells may 

be capable of doing everything we can possibly imagine, and more. 

Unfortunately, there’s often a wide gap between what’s possible in 

theory and what’s practical and possible in the real world. What 

the future of stem cells will be, no one knows for certain. Still, the 

possibilities are more than intriguing, and certainly worth an indepth look. 

The research that’s been conducted into stem cells so far has 

been so exciting because of the very nature of these cells. Stem 

cells have the capacity to renew themselves and then become specialized cells. Most of the cells that are in the body are created and 

committed to performing a specific function. The stem cell remains 

on the fence, uncommitted, until given a signal by the body to develop into a specialized cell. We’ve all heard the saying, ‘‘You don’t 

have to be a weatherman to know which way the wind’s blowing.’’ 

As for the research, however, you really do need a strong background in science to understand fully the specifics of stem cell research and its implications for the future. 

Fortunately, we’re not here to predict the impact stem cells will 

have on the healthcare system in the years to come, we’re here to 

discuss if it is appropriate to use Federal taxpayer dollars to finance additional work in this area, and there is a big difference. 

In discussing stem cell today, we’re not making a judgment about 

the science itself; rather, we’re considering what science should be 

supported by Federal taxpayer dollars. We’re considering the appropriate political oversight and public fiscal support of the work 

4

of those scientists in manipulating and possibly even destroying the 

basic building blocks of human life. We’re considering if we should 

pass legislation that will be vetoed by the President or legislation 

that will move this research field forward. We’re reaffirming how 

we, as a society, view the human embryo and its function. Without 

question, science must be guided by morality. There have been too 

many instances, over the course of human history, in which terrible 

things have been done in the name of science. 

In determining how to proceed, we, of course, must consider the 

promise of stem cell research generally and embryonic stem cell 

really specifically. But, in considering that promise, we must make 

it clear that, while stem cells may someday lead to therapeutic advancements for devastating diseases, like Alzheimer’s, diabetes, 

Parkinson’s, leukemia, and spinal cord injuries, that that day has 

not come. We must be careful not to oversell the promise of this 

research to the American people. While several nonembryonic stem 

cell therapies are now in practice, every reputable scientist will 

admit that possible cures or advanced treatment, using embryonic 

stem cells are many years away. So, while the research provides 

great hope for millions of Americans, at this point the full benefits 

have not been realized. They fire our imagination as we consider 

the possibilities that may or may not come to pass. If we truly trust 

science, then we should give science a chance to solve the dilemma 

before we reach the issue of public funding of embryonic stem cell 

research. 

As outlined by the report from the President’s Council on Bioethics, and is highlighted again with the recent announcement by 

Dr. Atala and others, related to amniotic stem cells—researchers 

are exploring a multitude of different ways by which we can create 

embryonic-like stem cell lines without harming or destroying embryos. Further, States and private research organizations are already plowing billions of dollars into human embryonic stem cell 

research that goes beyond the parameters of President Bush’s policy. Let those efforts continue while we continue working in Congress to support stem cell research that doesn’t involve harming or 

destroying an embryo, which is something that the vast majority 

of Americans could support. 

Thank you all for coming today. I look forward to the ongoing 

discussion. 

The CHAIRMAN. Thank you very much, Senator Enzi. 

And, as I mentioned earlier, Senator Harkin and Senator Specter 

played a special role in keeping this issue in the forefront here in 

the Senate, and we work very closely together. We’re delighted that 

we’ve been able to work so that the members of both committees 

could hear our excellent witnesses. 

Senator Harkin. 

OPENING STATEMENT OF SENATOR HARKIN

Senator HARKIN. Well, thank you very much, Mr. Chairman. 

You’ve been a leader on so many health issues for so many years, 

and I want to thank you for suggesting that we team up our two 

committees together on this joint hearing. 

This marks the 20th hearing that Senator Specter and I have 

held on human embryonic stem cells, dating back to December 

5

1998, 1 month after Dr. Jamie Thompson, from the University of 

Wisconsin, announced that he had isolated them, for the first time 

ever. Since that time, I’ve talked to hundreds of patients and their 

family members about their hopes for this research. I’ve visited laboratories and talked to scientists. I’ve heard from ethicists and religious leaders. And every day, I become more and more convinced 

that we, in Congress, need to do all we can to promote this possible 

life-saving, life-enhancing research. 

Meanwhile, the opponents have become more and more desperate. We saw that earlier this month during the hysteria over 

Dr. Anthony Atala’s new research on amniotic stem cells. Opponents breathlessly claimed that, on the basis of this one paper, embryonic stem cell research should be abandoned, even though Dr. 

Atala himself completely disagrees with that conclusion. Dr. Atala 

wrote, ‘‘It is essential that National Institutes of Health-funded researchers are able to fully pursue embryonic stem cell research as 

a complement to research into other forms of stem cells.’’ 

That’s a direct quote from Dr. Atala. 

A few days later, the White House released a 60-page polemic 

against embryonic stem cell research, in which it touted research 

by Dr. Kevin Eggan, of Harvard, who testified before our subcommittee last year. Here’s what Dr. Eggan wrote in response to 

that White House report. And Dr. Eggan was just in my office last 

week to substantiate it further. But here’s what Dr. Eggan wrote,

‘‘We are disappointed that the White House Office of Domestic Policy gave us 

no opportunity to correct the report’s clear misrepresentation of our work. On 

the contrary, we assert that human embryonic stem cells hold great promise to 

find new treatments and cures for diseases, and we support the Stem Cell Research Enhancement Act.’’

The House overwhelmingly passed that bill earlier this month, 

and the Senate will pass it soon. There’s no question about that. 

The only question is what the President will do when the bill 

reaches his desk. Most people assume that he’ll veto it. I’m not so 

sure. 

Earlier this month, White House spokeswoman Jeannie Mamo 

was quoted in a Gannett news story as saying this about stem cell 

research,

‘‘The President has said that, after careful and thoughtful deliberation with 

government and outside experts, there was only one moral line he said he would 

not cross, and that is that Federal taxpayer dollars should not be used in the 

destruction of embryos.’’

Well, this is a very interesting statement, because, if it’s true, 

the President should have no problem signing our bill. S. 5 would 

not allow Federal funding to be used for the destruction of embryos. That’s prohibited by what’s called the Dickey Amendment, 

which is included every year in our appropriations bill. Our stem 

cell bill doesn’t have anything to say about the Dickey Amendment. 

We’re only talking about using embryos that are going to be destroyed anyway. Every day, IVF clinics discard embryos that are no 

longer needed for fertility treatments. All we’re asking is to use 

stem cells from some of those excess embryos for research that 

would save people’s lives. No Federal tax dollars would be used to 

derive the stem cells. That work would be done using non-Federal 

funding. 

6

So, either this spokeswoman misrepresented the President’s position, in which case, I assume she’s been taken out to the woodshed, 

or the White House just opened the door to signing our bill. And 

I hope it’s the latter. I hope that President Bush will listen to the 

scientists at NIH and elsewhere, so many Nobel laureates all 

around this country and around the world, who want this research 

to proceed. Most important, I hope he’ll listen to millions of Americans who suffer from juvenile diabetes and spinal cord injuries and 

ALS and Parkinson’s and cancer, who view this stem cell research 

as their best hope for a treatment or a cure. 

I want to thank all the witnesses who have taken the time to 

give testimony before us today. We have an outstanding group of 

scientists, all of whom I’ve met before at some point over the years. 

I hadn’t met Lauren Stanford until this morning, but I feel like I 

know her, because Senator Kennedy and I talked about her a lot 

on the Senate floor last year, and I believe her story helped us pass 

H.R. 810, and will do so again. 

So, again, Mr. Chairman, thank you very much. I look forward 

to the testimony of our witnesses. 

The CHAIRMAN. Senator Specter. 

OPENING STATEMENT OF SENATOR SPECTER

Senator SPECTER. Thank you, Mr. Chairman. 

I believe that we are setting a record here this morning, on a Friday, in the U.S. Senate, to have 13 Senators present for a hearing. 

I believe that is solid testimony to the importance of this subject, 

and I believe it signifies a tremendous interest in utilizing Federal 

funds for embryonic stem cell research. 

We found out about stem cells when the scientists told us about 

them in November 1998, and, within a few days, the appropriations 

subcommittee held the first hearing, in this room, and this is our 

20th hearing. And I believe that sets something of a record, too. 

I agree with Senator Kennedy on his call for hope. I would supplement that with a call for political pressure. We are within close 

range of overriding a presidential veto. Sixty-three Senators voted 

in favor of use of Federal funds for embryonic stem cell research 

last year, and we’re within shouting distance, in the House, of having enough votes to override a presidential veto. 

Sometimes we forget that we live in a representative democracy, 

and that means that the people decide what the government is 

going to do. We had a clarion call on that, on the last election, 

where the American people spoke out on Iraq. I’m not sure that it’s 

been heard in all quarters, but the American people did speak out. 

And they also spoke out, in a number of States, on the issue of 

stem cell research. And I believe, if some of the Republican candidates, to put it candidly and bluntly, had been for stem cell research, I’d still be chairman of the Judiciary Committee. 

[Laughter.] 

Now, it’s a very basic matter of fact that there are 400,000 embryos that are frozen, and almost all of them will be thrown away 

if they’re not used. The subcommittee put up $2 million for an embryo adoption program, and, since that time, only about 100 have 

been adopted. So, it’s a simple matter of either to use them or lose 

them. If these embryos were going to create life, no one would be 

7

in favor of using them for research. And as Senator Harkin points 

out, this bill clearly does not allow Federal funding for the destruction of embryos. 

We have increased NIH funding on initiatives originating in this room with our subcommittee, raising the funding from $12 to $29 

billion, and it is scandalous that those funds are not available for 

embryonic stem cell research. In 1970, President Nixon declared 

war on cancer, and, had we pursued that war with the same intensity we pursue other wars, cancer would have been cured by now. 

And, frankly, I’m madder than hell about our failure to prosecute 

that war. I’m one of the victims of the failure to prosecute that 

war. I have urged the advocacy groups to organize a million-person 

march on the Mall loudly enough to be heard in the second floor 

of the living quarters of the White House. It’s close to the Mall. 

And it is really reprehensible that the National Cancer Institute 

was cut last year by $50 million, which goes to the NIH funding 

issue and the stem cell issue. But I think that, properly organized 

and with the pressure being put on the Members of Congress who 

have voted no, and ultimately on the White House, it’s a matter of 

when, not if, we’ll be using Federal funds for embryonic stem cell 

research. 

Thank you, Mr. Chairman. 

The CHAIRMAN. Thank you very much. 

Our first witness, Story Landis, who’s the director of the National Institute of Neurological Disorders and Stroke. Dr. Landis 

has been the director since September 1, 2003, and, as director of 

NINDS, Dr. Landis oversees an annual budget of a billion and a 

half dollars and a staff of more than 900 scientists—physicians, scientists, and administrators. The Institute supports research by investigators, public and private institutions across the country, as 

well as by scientists working in intramural laboratories and 

branches in Bethesda, Maryland. The Institutes’ mission is to reduce the burden—neurological disease, a burden borne by every 

age group, by every segment of society, by people all over the 

world. 

Ms. Landis, thank you very much for being here. 

Ms. LANDIS. Thank you very much——

The CHAIRMAN. We look forward——

Ms. LANDIS [continuing]. For inviting me. 

The CHAIRMAN [continuing]. And for your service. 

STATEMENT OF STORY C. LANDIS, PH.D., DIRECTOR, NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND 

STROKE (NINDS), DEPARTMENT OF HEALTH AND HUMAN 

SERVICES, WASHINGTON, DC 

Ms. LANDIS. Mr. Chairman, Senator Specter, Senator Enzi, and 

members of the subcommittee, I’m pleased to appear before you 

today to testify about the science of stem cell research. I look forward to discussing the compelling need to pursue both embryonic 

and nonembryonic stem cell research and the scientific challenges 

and progress, including a recently published scientific finding on 

amniotic-fluid stem cells. 

So, both embryonic and nonembryonic stem cells show promise 

for developing treatments for human diseases and injuries, and at 

8

the present time, we can’t predict which type of stem cell will be 

best for treating a given disease, nor, to be perfectly honest, is it 

likely that any one type of stem cell will be best for all uses of stem 

cells. Therefore, NIH should support research on stem cells from 

both embryonic and other sources. 

Now, the most obvious use of stem cells, which has captured the 

public’s attention, is to replace specific types of cells which are 

damaged by disease or injury, and my written testimony describes 

recent progress in preclinical animal studies using embryonic stem 

cells to replace dopamine-producing nerve cells that are lost in Parkinson’s, motor neurons, and supporting cells that are damaged in 

spinal cord injury, and liver cells that are affected by chronic liver 

diseases. 

However, beyond replacing cells or tissue, stem cell biology has 

many other potential applications. We could, for example, speed 

drug development by testing potential drugs in cell culture on specific kinds of human cells that are affected by disease, and stem 

cells represent a source of the necessary cells. 

Studies of human embryonic stem cells also yield information 

about the complex events that occur during human development, 

including the molecular mechanisms through which these 

pluripotent cells generate the hundreds and thousands of different 

kinds of cells that make up the human body. This knowledge will 

not only help us control stem cells from both embryonic and nonembryonic sources, but also will help us better understand the 

cause of many diseases, and that, in turn, will lead to more effective treatments. 

Finally, another potential application of stem cell biology is to 

learn how to encourage the stem cells that are present in even the 

adult human brain to repair damage. And this approach has recently shown promise in animal experiments in Parkinson’s disease 

and also stroke. 

But to realize the potential promise, the promise of stem cell biology for treating disease, scientists must learn how to reliably manipulate stem cells to have the characteristics necessary for each 

of these applications. We have to learn how to control stem cell proliferation to generate sufficient quantities of cells, we have to learn 

how to control their differentiation, create recipes for specific classes of cells. We also have to enable stem cells to survive after we 

transplant them, to integrate into the surrounding tissue, and to 

function for extended periods of time. Finally, we must control 

stem cell behavior to avoid harming the recipient, whether by generating tumors—and I’m sure this is an issue that will come up—

by forming faulty nerve cell connections, or in any other way. 

I’d like to speak briefly about amniotic-fluid-derived stem cells. 

This is a topic that’s received a great deal of attention recently. 

In a recently published article in Nature Biotechnology, Dr. Atala 

and his colleagues at Wake Forest University described how they 

isolated and characterized stem cells from the amniotic fluid that 

cushions the developing fetus in the uterus. This fluid is collected 

from pregnant women during amniocentesis, when they ask to be 

tested for a variety of congenital and developmental diseases and 

disorders. 

9

Now, scientists had previously shown that some of these cells 

could turn into fat cells, muscle cells, bone cells, and cells of the 

nervous system, but what Dr. Atala has done is to devise a method 

to select, from those multiple kinds of cells in the fluid, those cells 

which have the most stem-cell-like property, and then, also, he has 

extended our understanding of the kinds of properties of these 

cells, and what they can turn into. 

So, he and his colleagues have demonstrated that amniotic-fluidderived stem cells could produce several different adult cell types—

nerve cells, liver, cells, bone-forming cells—and, in the case of 

nerve cells, that they make proteins characteristic of nerve cells 

and that they can be integrated into the nervous system, that 

they’re self-renewing and maintain the normal number of chromosomes. But these cells are not equivalent to pluripotent embryonic stem cells. They have some of the properties, but not all. And, 

as we’ve already heard, he has concluded that these cells complement, but do not replace, human embryonic stem cell research. 

So, in conclusion, NIH places a very high priority on both embryonic and nonembryonic stem cell research that will be useful for 

basic, translational, and clinical studies. Science works best when 

scientists can pursue all avenues of research. And if I could be so 

presumptuous as to borrow a metaphor that Senator Harkin used 

in hearings in 1997 on the importance of funding basic science research, if the cure for Parkinson’s disease or juvenile diabetes lay 

behind one of four doors, wouldn’t you want the option to open all 

four doors at once instead of one door? And stem cell research is 

the same. 

Thank you very much. 

[The prepared statement of Ms. Landis follows:]

PREPARED STATEMENT OF STORY C. LANDIS, PH.D. 

OPENING REMARKS

I am pleased to appear before you today to testify about the science of stem cell 

research. I look forward to discussing ongoing Federal support of both embryonic 

and nonembryonic stem cell research and scientific progress, including the recently 

published findings on amniotic-fluid stem cells and other studies raising the possibility that nonembryonic stem cells have similar properties allowing them to differentiate into many different cell types. 

THE NEED FOR RESEARCH TO EXPLORE THE POTENTIAL OF HUMAN STEM CELLS

Stem cells are cells that can multiply without changing, that is, self-renew, or can 

differentiate to produce specialized cell types. Stem cells have been derived from 

both embryonic and nonembryonic tissues, and these cells have different characteristics. Both embryonic and nonembryonic stem cells show potential for developing 

treatments for human diseases and injuries. Because of this, this Administration in 

2001 became the first to fund research on human embryonic as well as adult stem 

cells. There are many ways in which human stem cells might be used in basic and 

clinical research. However, only further research will overcome the technical hurdles 

between the potential of stem cells and the realization of these uses. 

The most obvious potential application of human stem cells would be the generation of cells and tissues for cell-based therapies. Stem cells, directed to differentiate 

into specific cell types, offer the possibility of a renewable source of replacement 

cells and tissues to treat a number of common diseases and disorders, including Parkinson’s disease, spinal cord injury, stroke, burns, heart disease, diabetes, osteoarthritis, and rheumatoid arthritis. 

To realize the potential of stem cell-based therapies for pervasive and debilitating 

diseases, scientists must learn to reliably manipulate stem cells so that they possess 

the necessary characteristics for successful differentiation, transplantation, and 

engraftment. Although scientists are making progress, we cannot yet control the difVerDate 0ct 09 2002 12:55 Dec 17, 2007 Jkt 000000 PO 00000 Frm 00013 Fmt 6633 Sfmt 6621 S:\DOCS\32808.TXT SLABOR1 PsN: DENISE
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ferentiation of stem cells adequately. To be useful for transplant purposes, stem 

cells must:

• Proliferate extensively and generate sufficient quantities of specialized cells. • Differentiate into the desired cell type(s). • Survive in the recipient after transplant. • Integrate into the surrounding tissue after transplant. • Function appropriately for extended periods of time. • Avoid harming the recipient in any way.

Stem cells have many other potential uses. Studies of human embryonic stem 

cells, for example, yield information about the complex events that occur during the 

initial stages of human development. A primary goal of this research is to identify 

the molecular mechanisms that allow undifferentiated stem cells to differentiate 

into one of the several hundred different cell types that make up the human body. 

Scientists know that turning genes on and off is central to this process. A significant 

challenge for stem cell research is that scientists do not yet fully understand the 

signals that turn specific genes on and off to influence the differentiation of the 

stem cell into a specialized cell with a specific function, like a nerve cell. This 

knowledge not only offers the opportunity to learn how to control stem cells from 

both embryonic and nonembryonic sources, but also to better understand the cause 

of a number of serious diseases, including those that affect infants and children, 

which in turn could lead to new and more effective intervention strategies and treatments. 

Among other applications, human stem cells could also be used to speed the development of new drugs. Initially testing thousands of potential drugs on cells in cell 

culture is potentially far more efficient than testing drugs in live animals. In vitro

systems are useful in predicting in vivo responses and provide the benefits of requiring fewer animals, requiring less test material, and enabling higher throughput. 

New medications could be tested for safety on the specific types of human cells that 

are affected in disease by deriving these cells from human stem cell lines. Other 

kinds of cell lines are already used in this way. Cancer cell lines, for example, are 

used to screen potential antitumor drugs. The availability of useful stem cell lines 

could allow drug testing in a wider range of cell types. However, scientists must 

learn to control the differentiation of stem cells into the specific cell type on which 

drugs will be tested. 

FEDERAL FUNDING OF STEM CELL RESEARCH

NIH has acted quickly and aggressively to provide support for this research in accordance with the President’s 2001 stem cell policy. Since 2001, NIH has invested 

nearly $3 billion on all forms of stem cell research. Within this total, NIH has contributed more than $130 million in research studying human embryonic stem cells, 

more than $1.1 billion on research using human nonembryonic stem cells, nearly 

$509 million on nonhuman embryonic, and more than $1.2 billion on nonhuman 

nonembryonic stem cells. 

Additionally, in fiscal year 2007, it is projected that NIH will spend more than 

$30 million on human embryonic stem cell research and about $200 million on 

human nonembryonic stem cell research, while also investing nearly $100 million 

on nonhuman embryonic stem cell research and more than $270 million on 

nonhuman nonembryonic stem cell research. 

In addition to this ample support, NIH has encouraged stem cell research through 

the establishment of an NIH Stem Cell Task Force, a Stem Cell Information Web 

Site, an Embryonic Stem Cell Characterization Unit, training courses in the culturing of human embryonic stem cells, support for multidisciplinary teams of stem 

cell investigators, and a National Stem Cell Bank and Centers of Excellence in 

Translational Human Stem Cell Research, as well as through extensive investigator 

initiated research. NIH determined that access to hESC lines listed on the NIH 

Stem Cell Registry and the lack of trained scientists with the ability to culture 

hESCs were obstacles to moving this field of research forward. To remove these potential barriers, the National Stem Cell Bank and the providers on the NIH Stem 

Cell Registry together have currently made over 700 shipments of the hESC cell 

lines that are eligible for Federal funding, as posted on the NIH Stem Cell Registry 

Web site. In addition, the NIH-supported hESC training courses have taught over 

200 scientists the techniques necessary to culture these cells. We plan to continue 

to aggressively fund this exciting area of science. 

NIH-supported scientists have developed efficient techniques to derive dopamineproducing nerve cells from human embryonic stem cells. The loss of dopamineproducing nerve cells is responsible for the movement problems of Parkinson’s disease. When grafted into the brain of a rat model for Parkinson’s disease, the stem 

11

cell-derived dopamine cells significantly improved the animals’ movement. However, 

after 3 months of transplantation, the scientists found that treated rats’ brains contained groups of undifferentiated cells that had become tumors. (Nature Medicine 

12:1259–1268, laboratory of S. Goldman). This had not been observed in other studies that transplanted neural stem cells and emphasizes the need for scientists to 

learn to better regulate cell division in transplanted pluripotent stem cells, whatever the source, before they may serve as a renewable source of replacement 

dopamine-producing nerve cells to treat Parkinson’s disease in humans. These results demonstrate both the potential and the challenge of stem cell research. 

In recent years, NIH-supported scientists have demonstrated that even the adult 

human brain can generate new nerve cells. Studies focused on encouraging the innate potential of stem cells that are normally present in the adult brain are another 

avenue of research that has also shown potential for treating Parkinson’s disease. 

In recent experiments, researchers used drugs to activate adult stem cells in the 

brains of adult rats with experimental Parkinson’s disease, which increased the proliferation of replacement cells and improved movement (The Journal of Neuroscience 

26:7272–7280, laboratory of C. Eckman). 

Currently, scientists are also using stem cells from a variety of sources to help 

animals with spinal cord injuries regain movement. Human embryonic stem cells 

have been coaxed into becoming a type of cell that repaired damaged nerve fiber 

insulation called myelin (The Journal of Neuroscience 25:4694–4705, laboratory of 

H.S. Keirstead). Human nonembryonic-neural stem cells helped replace damaged 

rat spinal cord nerve cells and myelin (Proceedings of the National Academy of 

Sciences of the USA 102:14069–14074, laboratory of A.J. Anderson). NIH-supported 

scientists now report that they can use mouse embryonic stem cells to make functional motor neurons, which are the spinal cord cells that send long nerve fibers 

called axons (the threadlike extensions on a neuron, or nerve cell, which conducts 

nerve impulses) to connect with leg muscles and other muscles used to move the 

body. The scientists combined several methods to coax the mouse embryonic stem 

cells to become motor neurons, to overcome molecules that restrain axon growth in 

adults, and to attract the motor neuron axons to the correct muscles. Previously paralyzed rats treated with the motor neurons were able to move their legs again, although they could not walk or grip with their feet as well as uninjured rats. This 

research gives scientists insight on how they might one day replace human motor 

neurons damaged by spinal cord injuries and motor neuron diseases such as Lou 

Gehrig’s disease (amyotrophic lateral sclerosis, or ALS) and spinal muscular atrophies. (Annals of Neurology 60(1)32–44, laboratory of D. Kerr) 

Japanese and NIH-funded scientists used mouse embryonic stem cells to make 

liver-like cells to create an implantable bioartificial liver. Chronic liver diseases such 

as cirrhosis and hepatitis affect 25 million Americans and scientists hope to overcome the shortage of organs available for transplants by using liver cells derived 

from stem cells to replace lost liver function. This implanted device uses liver cells 

to replace some liver function. Ninety percent of mice with liver failure that were 

implanted with the bioartificial liver survived at least three times longer than the 

untreated mice. If scientists can repeat these results with liver cells made from 

human stem cells, the technique offers potential both to individuals born with liver 

problems and to those who develop liver disease later in life. (Nature Biotechnology 

24:1412–1419, laboratory of I. Fox). 

AMNIOTIC-FLUID-DERIVED STEM CELLS

As you all know, there has been much interest in the recently published article 

in Nature Biotechnology by Dr. Anthony Atala and colleagues at Wake Forest University regarding stem cells isolated from the amniotic fluid that cushions the developing fetus in the uterus. Amniotic fluid is collected from pregnant women during 

amniocentesis to test for a variety of congenital and developmental diseases and disorders. Scientists have previously reported that some of these cells can differentiate 

into fat, muscle, bone, and nerve cells. Dr. Atala’s work extends our knowledge of 

the properties of these amniotic-fluid-derived stem cells (AFS). 

Dr. Atala and colleagues showed that AFS could produce cells that originate from 

each of the three embryonic germ layers that give rise to all of the cells in the body. 

More specifically, the scientists were able to develop in vitro conditions that produced nerve cells, liver cells, and bone-forming cells from AFS. The AFS-derived 

human nerve cells were able to make proteins typical of specialized nerve cells and 

were able to integrate into a mouse brain and survive for at least 2 months, although it is not yet clear whether these cells have all the properties of normal neurons. They also showed that AFS cells were also self renewing and maintained the 

normal number of chromosomes after a long time in culture over many cell diviVerDate 0ct 09 2002 12:55 Dec 17, 2007 Jkt 000000 PO 00000 Frm 00015 Fmt 6633 Sfmt 6621 S:\DOCS\32808.TXT SLABOR1 PsN: DENISE
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sions. However, undifferentiated AFS did not make all of the proteins expected in 

embryonic stem cells, and they were not shown to form a teratoma (a germ cell 

tumor), one of the essential characteristics of embryonic stem cells. Thus, given the 

characteristics of AFS, scientists conclude that these cells may be multipotent rather than pluripotent. Although scientists do not yet know how many different cell 

types AFS are capable of generating, banked AFS may one day enable the generation of tissue-matched cells for transplantation into humans. 

CONCLUSION

Since 2001, NIH has aggressively pursued research using embryonic and nonembryonic stem cells that will be useful for basic, translational, and clinical studies. 

We are continuing to move this research forward through training programs, the establishment of the NIH stem cell characterization unit, and the many grants that 

have been made to scientists to explore stem cell research. With NIH support, scientists have already made remarkable progress in understanding human embryonic 

stem cells, and we will provide continued support for these research efforts, consistent with Administration policy. 

I will be more than happy to answer any questions.

The CHAIRMAN. Thank you very much. 

I’m going to ask if you’d be joined by George Daley, who’s the associate professor at Harvard Medical School, President-elect of the 

International Society of Stem Cell Research; Lauren Stanford, to 

join you—Lauren Stanford, who has courageously fought juvenile 

diabetes and her letter has moved the entire Senate; and Dr. John 

Wagner, who’s a professor of pediatrics, University of Minnesota, 

an internationally respected researcher—stem cells. And we’ll hear 

from them, and then have the questions from the committees—for 

any of our panelists. 

We’ll start with Dr. Daley, if you’d be good enough—please——

Dr. DALEY. Thank you. 

The CHAIRMAN [continuing]. Welcome, from Boston. 

Dr. DALEY. Thank you, Senator Kennedy. I appreciate the opportunity to speak here. I was given the instructions that I could not 

read my testimony, so I will not. 

The CHAIRMAN. That’s right. 

Dr. DALEY. I just have some notes. 

The CHAIRMAN. Well, we’re trying something—there are a lot of 

people around that come up here and don’t know a great deal about 

it, and they spend a lot of time reading long dissertations on it that 

are rather dull and——

Dr. DALEY. Yeah. 

The CHAIRMAN [continuing]. Not terribly informational. 

[Laughter.] 

Dr. DALEY. I——

The CHAIRMAN. And our staffs could work out of that, so——

Dr. DALEY [continuing]. I’m worried——

The CHAIRMAN [continuing]. We have a rule on our committee——

Dr. DALEY. I’m certainly worried about being dull. 

The CHAIRMAN [continuing]. That unless you can speak on your 

subject for 5 minutes, we’re not going to spend the time. If you 

want to file other statements—but that is not——

Dr. DALEY. Thank you. 

The CHAIRMAN [continuing]. Guidance for any—we have a brilliant panel here. 

[Laughter.] 

And some of us have read extensively about your works, and——

13

Dr. DALEY. Well——

The CHAIRMAN [continuing]. Are enormously grateful. I just——

Dr. DALEY [continuing]. At the risk of being dull——

The CHAIRMAN. Well, I’d divert you for 1 minute. I do have to 

mention about getting testimony in on time. We are really going to 

try and do this. It makes a great deal of difference to our ability 

to prepare. And, in this instance, some of the material didn’t get 

in until 8:30 last evening, and we were in, ourselves, late. We’re 

really going to insist on that for our hearings in the future, and we 

are going to let people know, in the future, that we are going to 

insist on it. 

But I thank all of you. It was basically the NIH, and—I’ve been 

around here long enough—it isn’t NIH, it’s OMB clearing NIH, so 

I—we know who the culprits are. So——

[Laughter.] 

Culprits be warned. 

Dr. Daley——

Dr. DALEY. OK. 

The CHAIRMAN [continuing]. Carry on, please. 

Dr. DALEY. Thank you very much. 

STATEMENT OF GEORGE Q. DALEY, M.D., PH.D., ASSOCIATE

PROFESSOR OF PEDIATRICS, CHILDREN’S HOSPITAL,

BOSTON, MA 

Dr. DALEY. As you said, I’m here as a physician scientist from 

Children’s Hospital, Harvard Stem Cell Institute. I am the President-elect of the International Society for Stem Cell Research, and 

I’m here representing the American Society of Cell Biology, whose 

10,000 members include some of the world’s leading stem cell scientists. 

And I was asked to make some comments that would be pertinent to the current aspects of the stem cell debate, and to reinforce the need for expanded funding for embryonic stem cell research. 

The media has covered a remarkable array of supposed breakthroughs over the last many years that purport to announce cells 

that could replace the need for embryonic stem cells in research. 

A number of years ago, it was the multipotential adult progenitor 

cell from Catherine Verfaillie’s lab in Minnesota; we’ll hear more 

about that in the coming days. Then it was the fat stem cell, and 

clearly there’s a lot of fat stem cells around. And then it was umbilical-cord-blood stem cells, and then testis stem cells, and, only more 

recently, the amniotic-fluid stem cells. These are all fascinating 

and important tools for research, but none of them are embryonic 

stem cells. 

Stem cells, in fact, I want to point out, is really a category of 

cells, and the term ‘‘stem cells’’ is inexact. And it’s really more akin 

to the term ‘‘seeds.’’ We appreciate that not all seeds are alike. An 

apple seed makes apple trees, an orange seed makes orange seeds. 

And when we talk about apples and oranges, we don’t get them 

confused. Well, the distinctions between seeds are essential to the 

biologist, just as the distinctions among different stem cell types 

are essential. And yet, in the public debate, I think, we lose the 

14

sense of refinement about what different types of stem cells actually are. 

So, after many years of competing claims, embryonic stem cells 

remain the most versatile stem cell, they remain the gold standard 

of this fascinating biological concept of pluripotency. And, after 20 

years of research in the mouse, we know that embryonic stem cells 

can make any cell type in the body. Routinely, in my laboratory, 

we move from an embryonic stem cell in a petri dish to an entire 

mouse within a month. Those cells, we know, can make every cell 

type. 

So, embryonic stem cells have unique properties, and they will 

fulfill a unique purpose in research, a purpose that, I would argue, 

will not be replaced by all of these other types of stem cells. As was 

pointed out by Dr. Landis, human embryonic stem cells are important tools for basic research. We spend an enormous amount of 

time talking about their therapeutic value. All of the different stem 

cell types will have, we hope, 1 day, therapeutic value. But unique 

aspects of embryonic stem cells pertain to their ability to model the 

earliest steps of human development. If you really want to understand the genetic regulation and the diseases that set in during 

those first few days of human development, then studying 

amniotic-fluid stem cells and fat stem cells will not get you to those 

answers. Embryonic stem cells, therefore, are unique. 

Now, it’s often said, by opponents of embryonic stem cell research, that embryonic stem cells have never yielded a treatment, 

have never cured a patient. And that’s true, but I think it’s a patently unfair criticism, because human embryonic stem cells have 

only been around for 9 years. Actually, in the last year, if you just 

look at the medical literature, human embryonic stem cells have 

been used to generate a whole variety of human cell types—blood 

cells, heart muscle cells, nerve cells, and many, many more. So, 

they’re beginning to yield their fruits in basic research, and I think 

it’s only a matter of time before we see an impact on therapy. But 

to criticize embryonic stem cell value for medical research is to 

trivialize the enormous contribution of mouse ES cells for the past 

20 years. 

Scientists have generated literally thousands of strains of knockout mice, which all derive from mouse embryonic stem cells. And 

these have been used to model human cancer, neurodegenerative 

disease. And, just a few years ago, there was a publication that reported that knockout mouse strains validate the targets of the hundred best selling drugs. So, where it’s true that human embryonic 

stem cells have not yet yielded cures in the form of cell therapy, 

I think it’s clear that embryonic stem cells have already had a revolutionary effect on biology, and they have saved lives—not directly, through cell replacement, but indirectly, through insights 

into disease and the development of drugs. 

So, I want to close by saying that I believe there are no credible 

scientific arguments which say that we should be studying adult 

stem cells at the exclusion of embryonic stem cells. And I’m looking 

forward to answering questions pertaining to those issues. We 

must promote embryonic stem cell research and adult stem cell research with equal vigor. And Senate passage of S. 5 would be a 

very healthy start. 

15

Thank you very much. 

[The prepared statement of Dr. Daley follows:]

PREPARED STATEMENT OF GEORGE Q. DALEY, M.D., PH.D. 

My name is Dr. George Q. Daley and I’d like to begin by thanking the members 

of the committee for inviting me here today. I believe passionately in the scientific 

value of stem cell research, and I am eager to present my views to the committee. 

I am an Associate Professor at the Harvard Medical School based at the Boston 

Children’s Hospital. I am Associate Director of the Children’s Hospital Stem Cell 

Program and a founding member of the Harvard Stem Cell Institute. I serve on the 

Public Policy committee of the American Society for Cell Biology, which represents 

over 10,000 scientists, and I am President-Elect of the International Society for 

Stem Cell Research, the world’s leading organization of stem cell scientists, which 

has grown to over 2,500 members in just over 4 years. 

As a practicing physician-scientist, I run a busy research laboratory at the Children’s Hospital, where we study adult stem cells of the blood—both their normal 

regulation and their pathology, as in leukemia—and we study the formation of blood 

during embryonic development. For this, we use embryonic stem cells. I also care 

for adults and kids with malignant and genetic bone marrow conditions—diseases 

like leukemia and lymphoma, immune deficiency, and sickle cell anemia. Many of 

these diseases can be cured by bone marrow transplantation—a form of stem cell 

therapy that harnesses the power of adult blood stem cells, or as you will hear (or 

have heard) from Dr. Wagner, from stem cells in Umbilical Cord Blood. While transplants are effective for some, the reality is that marrow replacement represents a 

heroic attempt at a life saving therapy for fatal diseases. The transplantation regimen itself is highly toxic. I would not wish this therapy on anyone who was not otherwise facing a potentially terminal illness. As a direct response to these shortcomings of adult stem cell therapies, my lab investigates the formation of blood stem 

cells from embryonic stem cells, and is pursuing strategies for making rejection 

proof, autologous tissues for transplantation. Our current treatments for many blood 

diseases are stone age, and only through research can we hope to make progress. 

I believe that embryonic stem cell research holds the key to treating many blood 

diseases. 

Stem cells come in many varieties. Even the term ‘‘stem cell’’ is a very general 

term. It defines a generic category of cells that has many members with different 

properties. It’s about as specific as the category ‘‘seed.’’ Seeds of all types share 

many properties, but an apple seed makes apple trees and an orange seed makes 

oranges. When we compare apples and oranges no one confuses the two. To a biologist, the distinctions between seeds are crucial, as are the distinctions between different types of stem cells. No credible biologist would argue that one type of seed 

can teach you all you need to know about all seeds and all fruit. Yet somehow, when 

we speak about stem cells in the current debate, people tend not to appreciate the 

differences, and consider them all interchangeable. 

The media has covered a long list of ‘‘breakthroughs’’ that purportedly represent 

new sources of stem cells that substitute for embryonic stem cells. Initially, it was 

the Multipotential Adult Progenitor Cell from Catherine Verfaillie’s lab in Minnesota, later it was the fat stem cell, then umbilical cord blood stem cells, and stem 

cells from testes. Just last week we heard reports about stem cells from amniotic 

fluid. All of these new types of stem cells are important tools for research and may 

even one day yield new therapies. However, none of them is the equivalent of embryonic stem cells. Perhaps they can do some of the things that embryonic cells can 

do, but they cannot do all of them. The differences between these other stem cells 

and embryonic stem cells are very, very important. 

We have also heard that there are alternative means of generating embryonic 

stem cells without sacrificing embryos. There have been exciting recent developments that claim ‘‘reprogramming’’ of adult cells back to their primitive embryonic 

state, either by cell fusion with existing embryonic stem cells, or by introducing a 

small number of genes. Again, these achievements are noteworthy and fascinating, 

but they have not yet produced cells that faithfully mimic or replace the functions 

of true ES cells. 

After many years of competing claims, ES cells remain the most versatile of all 

stem cells. ES cells are the gold standard for the biological concept of pluripotency, 

and it has been known from over 20 years of research in the mouse that ES cells 

can make all the cells of the body. ES cells have unique properties and they fulfill 

a unique purpose in biological research. Human ES cells are irreplaceable tools for 

understanding the earliest stages of human development. They are unique precisely 

because they come from the earliest human embryos—before implantation into the 

16

womb, before even the most rudimentary human form has begun to take shape. Understanding how these primitive cells orchestrate the process of human development 

represents one of the greatest goals of modern biology. Figuring out how amniotic 

stem cells work or fat stem cells work will not teach us about the earliest days of 

human development. Many different types of stem cells—adult and embryonic—may 

prove useful for therapies. But embryonic stem cells are the only stem cells that 

have been proven to form all cells in the body, and this feature alone makes them 

worthy of study. 

With regards to medicine, it is sometimes said by opponents of ES cell research 

that ES cells have never cured anyone. This is a patently unfair assertion because 

human ES cells have only been around for 9 years, and even now cannot be considered routinely available to scientists in the United States. However, the detractors 

of ES cells are naı ̈ve in trivializing the contributions that ES cells have made to 

biomedical research. Mouse ES cells have been used extensively to model human 

disease and to study how gene variations influence cancer, heart disease, 

neurodegeneration, metabolic disease, and many, many others. Indeed, a paper published in 2003 reported that gene knock-out strains of mice, which derive from ES 

cells, provided key target validation for the effects of the 100 best selling drugs 

(Zambrowicz and Sands, Nature Reviews, 2003). It is therefore fair to say that ES 

cells have already saved lives—not directly through cell replacement therapies—but 

indirectly through key insights into human disease and the development of new 

drugs. 

In closing, I want to stress that there is no credible scientific argument that 

would justify studying only adult stem cells to the exclusion of embryonic stem cells. 

Medical science does not advance fastest by cutting off fruitful avenues of research 

that the overwhelming majority of scientists and leading scientific societies like the 

ASCB and the ISSCR believe are vital. We must promote embryonic and adult stem 

cell research with equal vigor. We need a more conducive Federal policy for human 

embryonic stem cell studies, and Senate passage of Stem Cell Bill would be a 

healthy start. This vital research should not be left up to the States to fund. We 

need to stop making pseudo-scientific arguments against embryonic stem cell research, and get on with the scientific challenges ahead. 

Thank you.

The CHAIRMAN. Thank you very much, Dr. Daley. Very, very 

helpful. 

We have Lauren Stanford, here, a freshman at North Plymouth 

High School. And we welcome her mother and father. I hear your 

grandfather was William Ohrenberger, who was the superintendent of schools in Boston, and a very enlightened and courageous one, for many, many years, one of the great educators in Boston, who also played professional football in the 1920s. He was 

quite a guy. And Lauren follows in a very wonderful tradition of 

public interest. She was good enough to write a very moving letter, 

a year or so ago, when we were—had these issues on the floor, and 

we’ve invited her back. We want to welcome her parents. 

This is the first day of school that she’s been absent in I don’t 

know how many years. But, Lauren, we—you’re among friends 

here, and so, we hope you’ll relax and, sort of, enjoy it, too. It might 

not seem that way, but we want——

[Laughter.] 

We want you to know that you’re among friends, and you’re very 

welcome here. You’ve got a very, very, very important message, and 

you’ve taken the time to give this a good deal of thought, and we’re 

very thankful for your being here. 

STATEMENT OF LAUREN STANFORD, JUVENILE DIABETES 

PATIENT, PLYMOUTH, MA 

Ms. STANFORD. Thank you. 

I’d like to thank Senators Kennedy, Harkin, Specter, and Enzi 

for inviting me to appear before your committees today. It’s wonVerDate 0ct 09 2002 12:55 Dec 17, 2007 Jkt 000000 PO 00000 Frm 00020 Fmt 6633 Sfmt 6601 S:\DOCS\32808.TXT SLABOR1 PsN: DENISE
17

derful to live in a Nation where the cares of a 15-year-old girl from 

a small town are heard in the U.S. Senate, and that is because of 

leaders like you. I admire and respect all of you so much. 

To see me sitting here, you’d think I’m just a normal American 

teenager. And, in most ways, I am. I play tennis and field hockey, 

I swim, and I ski. I’m pretty much addicted to Instant Messaging. 

[Laughter.] 

I cannot survive without my cell phone nearby. Yes, on the surface I’m just another American girl. 

But inside me, a battle has been raging for 10 years now, because, just after my 6th birthday, I was diagnosed with Type 1 diabetes. Type 1 isn’t something you do wrong to get, it isn’t something you can change your habits to avoid. In the past 10 years, 

diabetes has sent me to the hospital 14 times, twice to intensive 

care; it has pricked my fingertips over 30,000 times; it has injected 

needles in me tens of thousands of times; and it has forced me to 

learn to change my own pump catheter every 2 days, from the time 

I was just 7 years old. It has forced my mother to be a part of my 

life in a constant way, every hour of every day. Now, imagine accepting that, as a 15-year-old girl. 

Diabetes has, indeed, ruled every minute of my life. Every 2 

months, doctors peer deep into my eyes waiting for the time when 

they can tell me it’s begun to break down my eyesight. They poke 

at my feet and my hands to see if it’s robbed me of my circulation 

yet. They test my kidneys to see how far its assault on them has 

gone. And through all of this, I walk and talk and try to live in 

the world as just another American girl. 

But time is not on my side, and I know that my only hope for 

a cure lies in medical research. My parents—my family has helped 

me learn about research over the years. And my friends and I have 

raised a lot of money to help fund it. My group, called ‘‘Got Islets: 

Lauren’s League for a Cure,’’ has raised hundreds of thousands of 

dollars for the Juvenile Diabetes Research Foundation since I was 

diagnosed, and we are just a bunch of kids. But we kids can’t do 

it alone. We need the Government to help by allowing scientists to 

fully unleash the potential of embryonic stem cell research. This research could hold the key to a change in, not just my life, but in 

the life of so many Americans. 

Imagine if it can help get to the source of what causes diabetes 

and stop it before it starts. Imagine if it can find a way to create 

new islet cells so my destroyed ones can be replaced with working 

ones. I cannot imagine what it’s like to have 1 day—just 1 day 

when I was not sick. That’s because I’ve had diabetes for longer 

than I can remember. Now is the time to expand the current stem 

cell research policy, not just because I want to know firsthand what 

a healthy day feels like, but because scientists believe they can 

make real advances in the search for cures for diabetes and for 

other diseases, as well. 

While I wait for scientific advances, I really am doing all I can 

to help keep myself alive. Recently, I took a brave new step in 

fighting diabetes, and it has not been easy. I am now wearing what 

is called the Continuous Glucose Monitoring System, and I’m one 

of the first kids in the Nation to do it. This means I have a radio 

transmitter strapped to my side 24/7, in addition to my insulin 

18

pump. Attached to it is a wire probe that I insert under my skin 

every few days—again, on my own, doing something most would 

need medical staff for. It helps me see, more often, what my blood 

sugar is, and it helps me keep my blood sugar in better control. 

But it’s not a cure. It’s a step forward in helping me take better 

care of myself until scientists find a cure, because, even though I 

have more information from it, it’s not stopping diabetes from attacking my body. 

I still get high and low. I still need insulin. I still fear the future, 

because, you see, I have hopes and dreams for a future, like most 

other kids. I want to go to a great college, but I have to worry 

about how to balance my constant care with life in the dorms. I 

want to get married and have children, but I have to worry about: 

How will I make that happen? Women with diabetes can have children now, but only with a constant and very invasive care. I want 

to be a Senator, like you, but I have to worry if my body will hold 

up long enough to help me get the experience I need to even try 

for that. 

I have to admit, I am lucky in some ways. Living in Massachusetts and near Boston means I am very close to some of the best 

care in the world. At my diabetes camp and through my advocacy, 

I’ve met kids who are not that lucky. They don’t have a good team 

to help them take care of themselves and try new treatments, they 

don’t meet with great researchers like we have at Harvard. I worry 

for them, too. How will they achieve their dreams if I’m worried 

about mine? 

I’m also very lucky that my parents are willing and able to work 

very hard to pay for all the things that diabetes demands, because, 

even with good insurance, it’s expensive. Pump supplies, needles, 

insulin, test strips, and more, it all adds up to tens of thousands 

of dollars my parents spend. What about the kids who are not 

lucky enough to afford that? 

Embryotic stem cell research could be a key answer to all of this. 

As I try my hardest to take the best care of myself I can, and those 

thousands of kids out there who are not as lucky as me do best in 

their situations, I hope that the Government will do its part by giving our best scientists the best tools to get a cure as soon as possible. 

One of the best tools out there is definitely embryonic stem cell 

research. With it, with our great Nation and brilliant scientists, I 

can go on and live the life that I dream of. Will I go to a good college? Maybe. Will I get married and have kids? Hopefully. Will I 

be a Senator? We shall see. But one thing is for sure—once stem 

cell research helps us cure diabetes, I’ll be that one thing I truly 

dream of being: just another American girl. 

The CHAIRMAN. Very good, Lauren. 

[Applause.] 

Very well done. 

John Wagner, professor of pediatrics, University of Minnesota, 

we welcome your testimony. 

19

STATEMENT OF JOHN E. WAGNER, JR., M.D., PROFESSOR OF 

PEDIATRICS, UNIVERSITY OF MINNESOTA MEDICAL 

SCHOOL, MINNEAPOLIS, MN 

Dr. WAGNER. Thank you, Senator Kennedy, Senator Enzi, Senator Specter——

The CHAIRMAN. Push the button. Push the button. 

Dr. WAGNER [continuing]. Thank you for allowing me to speak 

today. As was said, my name is John Wagner. I’m a professor of 

pediatrics. I’m a co-director of the Stem Cell Institute at our institution, as well as the head of the Bone Marrow Transplant Program. 

Over the past decade, there have been several major events. One 

is breaking down the genetic code, and the second is stem cell therapy. I take care of patients with incurable diseases, and I’m here 

to represent many of those patients, who are looking for cures, 

whether it be spinal cord injury, diabetes, Parkinson’s, whatever 

the disease. 

We’re looking for new strategies that give them hope, and, as 

others have already said, I think we’re on the cusp of seeing this 

become a reality. But I’m also here as a staunch advocate of adult 

stem cells. Clearly, there is a role for adult stem cells. We’ve seen 

a great deal of promise in all the publications that have been coming out over the past couple of years. These clearly need to be explored. But it needs to also be unequivocally clear that there is only 

one proven cure, that’s been documented, with stem cell therapies 

from adult or neonatal tissues, and that’s in the setting of bone 

marrow transplantation for the treatment of leukemia, lymphoma, 

sickle cell disease, immune deficiency. Clearly, what we’re doing 

there is, we’re replacing diseased bone marrow with bone-marrow 

stem cells or cord-blood-derived stem cells. Now, that’s the only 

proven cure. And as Dr. Daley said, you know, ‘‘Well, what have 

you shown us, in terms of cures, with embryonic stem cells?’’ Well, 

clearly lots of work needs to be done, both with embryonic stem 

cells, but with adult stem cells, as well. 

Many different trials are being conducted right now looking at 

the use of adult stem cells in the treatment of heart disease, in the 

treatment of spinal cord injury, bone disorders, genetic diseases. 

Clearly, they need to be explored. But have we proven any success 

yet? No, we haven’t. But I think that we need to also step back for 

a second, because I’m a clinical trialist, I’m the one who actually 

designs new therapies and tries them out for the first time. Some 

of the families of my patients are actually right here, because 

they’ve actually tried brand new things, because there is no cure 

for their underlying disease. So, we try new things, and it doesn’t 

always work. 

I think that there are a few obstacles and a few things about 

moving this field forward. One, is that—What are realistic expectations? When we do stem cell therapies, whether it be adult stem 

cells, as we’re doing very much today, or embryonic stem cells, perhaps in the future, you don’t expect home runs to occur the first 

time you test them out. 

Let me give you one example. What I did, and what I am a pioneer in, is the use of umbilical-cord blood as a source of stem cells 

for treating patients with leukemia. If you go back and look where 

20

we were in 1990, when I performed the first cord-blood transplant 

for a child with leukemia in the world, the patient didn’t survive. 

Did we give up? No, we continued. And, just a year ago, there—

with the Stem Cell Act, we were able to actually markedly expand 

the collection and storage of umbilical-cord blood. And why did we 

do that? It’s because we’ve actually been able to show, now, 

through our work at the University of Minnesota, that others are 

now replicating—we’ve tripled the survival in adult patients with 

leukemia and lymphoma. We’ve now been able to improve upon the 

overall survival rates with children. And we now are able to address the concerns of access to stem cell therapies to patients of 

ethnic and minority descent. We can find donors for almost everyone, which we could not previously do. So, we’ve made substantial 

progress, despite the fact that the first trials were failures. 

There are other obstacles, however, that you have to keep in 

mind. One of the things that’s touted as a benefit of adult stem 

cells, which is probably still a benefit of adult stem cells, is the fact 

that there could be tissue matching. I could collect stem cells from 

every one of you in this room and actually be able to create a 

multipotent adult stem cell that we could actually then re-infuse 

into your diseased heart or whatever the organ that needs to be 

fixed. Now, one thing we’ve also learned is that the immune system, unfortunately, attacks those cells, as well, even if it’s from 

your own body. There’s something peculiar about the stem cell

that we have to address. The fact that it’s matched, the fact that 

it’s from your own body, doesn’t mean that it won’t be immunerejected. 

Well, what we’ve also learned is strategies to make this work. 

And, in fact, over the next year, I hope I can say to you that we 

will have done the first trials with the multipotent adult stem cell 

that was discovered in our institution by Cath Verfaillie. It will 

then be tested in patients who are undergoing chemotherapy and 

radiation, as a way of tissue repair. The advantage of that setting 

is because of the fact that these patients will also be immune-suppressed and hopefully given the chance, the best chance, for these 

stem cells to engraft, to divide, to replicate, and to repair tissue. 

But what happens if it doesn’t work? Do we give up? No, we continue. And, in fact, probably the first trials won’t work. The first 

trials are actually a safety study. But this will be one of many generations of trials. Just like with cord blood, 16 years ago, when we 

did that first transplant, this will be an evolutionary process. 

The last thing, because one of the tasks today was—I was asked, 

Can Congress help fulfill the promise of stem cell research? And 

this goes back to Dr. Landis, is the fact that—you know, where are 

we, in terms of NIH dollars? We are still inhibited by—we have our 

hands tied by the amount of funding that’s available for this research. And, in fact, although I am an example of a successful candidate for getting research for clinical trials, unfortunately not a 

single trial has been designed by me, at least in the past, that has 

been substantial enough to be able to pay for the clinical trial 

itself. It requires multiple sources of funding for every clinical trial 

that we do, or I have to design the trial such that it’s a small trial 

that can be within the confines of what the NIH will allow me in 

their cap. 

21

But if we want to move these cell therapies forward, we have to 

invest in them, we have to recognize the obstacles, you have to understand the translational pipeline and its current problems. And 

how do we make this work? We have the tools to do that, and we’re 

here to help you, if you want us to. 

But every single one of us, in conclusion, will be faced with a disease that will be amenable to stem cell therapy. It might be our 

child, it might be our spouse, it could be us. Adult stem cells and 

cord-blood stem cells have benefits in the treatment of blood cancers. We know that. What we have to do is to be able to explore 

other diseases outside the context of bone marrow transplantation. 

It’s essential that Federal funds be devoted to this. I think that you 

support it. We have to make it move forward, but also have real 

expectations. We do this for ourselves, for the science, but, most 

importantly, for the children and our families. 

Thank you. 

[The prepared statement of Dr. Wagner follows:]

PREPARED STATEMENT OF PROFESSOR JOHN E. WAGNER, JR., M.D. 

EXECUTIVE SUMMARY

Over the past decade, two major events promise to revolutionize the practice of 

medicine—unraveling the genetic code and the isolation of the stem cell. Today, 

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

and marrow transplantation to treat diseases such as leukemia, lymphoma, sickle 

cell disease and various other blood and immune disorders. 

Accomplishments using stem cells from adult and neonatal tissues include: (1) our 

demonstration of their capacity to differentiate into cells of multiple tissues, (2) 

their safety and efficacy in laboratory models of disease, and (3) procedures for manufacturing stem cells for human testing. 

There are many new adult stem cell projects moving to clinical trials. It is unrealistic to expect that there will be home runs; and, it may take several generations 

of studies to make a new therapy work. The Stem Cell Therapeutic and Research 

Act of 2005 authorized substantial funds to be used to increase the Nation’s inventory of cord blood by 150,000 units. The NCI and NHLBI are supporting multi-institutional trials in children and adults to validate these results pioneered at the University of Minnesota. This is an example of what your support has accomplished and 

what it takes to move stem cell therapeutics from concept to clinical testing to 

standard of care. 

We are now ready to test the multipotent adult stem cell, the cells discovered by 

Dr. Verfaillie and colleagues. But, importantly we have also identified obstacles, 

reasons why these may fail to repair injured tissues. While it is touted as one more 

advantage of adult stem cells over ES cells, it is now clear that the most primitive 

adult stem cells, even those directly from the patient, are susceptible to immune attack. This serves as a clear example of why it is not enough to show that a cell 

can differentiate into a tissue, the right models need to be used to predict clinical 

outcome. 

Gap funding for Phase I clinical trials is an obstacle to our success. Currently, 

the Federal grants are too small to complete the trials and we must compile several 

funding sources to move forward. 

There are things we can do now that will speed the process of moving new laboratory discoveries to clinical trials. First, you need to understand the translational 

pipeline, its components, how it is funded, and the potential obstacles. Second, it 

is necessary to understand why there are disincentives for clinicians and basic scientists to engage in this translational research—as this will help identify solutions. 

Third, and perhaps most important, you must be able to differentiate speculation 

from fact, as it pertains to stem cells, as there is a considerable misinformation and 

misunderstanding out there on what adult stem cells can and cannot do. 

Senator Kennedy, Senator Harkin, Senator Enzi, and Senator Spector, thank you 

for the opportunity to speak today. My name is John Wagner. I am the Director of 

Hematology/Oncology and Blood and Marrow Transplantation Program and SciVerDate 0ct 09 2002 12:55 Dec 17, 2007 Jkt 000000 PO 00000 Frm 00025 Fmt 6633 Sfmt 6621 S:\DOCS\32808.TXT SLABOR1 PsN: DENISE
22

entific Director of Clinical Research for the Stem Cell Institute at the University 

of Minnesota. 

Over the past decade, two major events promise to revolutionize the practice of 

medicine—unraveling the genetic code and the isolation of the stem cell. The rate 

that new genes are discovered and their function understood have been extraordinary. Take for example, BRCA2—the breast cancer gene. In my own clinic in the 

treatment of children with rare life threatening disorders, we have learned that this 

genetic defect is not only associated with breast and ovarian cancer in adults but 

also leukemia, brain tumors and kidney tumors in very young children. In fact, detection of this genetic defect in young children has allowed me to predict with high 

certainty what cancers will develop and when. This is powerful information because 

it has allowed me the opportunity to pre-emptively intervene and alter the future 

predicted by these genes. One intervention has been the use of stem cells. 

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

of blood and marrow transplantation to treat diseases such as leukemia, lymphoma, 

sickle cell disease and various other blood and immune disorders. This has been 

known for 40 years. For these diseases, we infuse stem cells to repair marrow that 

has either been destroyed by the disease itself or by treatments, such as high doses 

of chemotherapy and radiation. These blood producing stem cells come from adult 

marrow or cord blood (the blood left in the placenta after a baby is born). 

A year and a half ago I presented before Senators Harkin and Spector to defend 

the vital importance of embryonic stem cell research. While I unequivocally support 

embryonic stem cell research, it must also be clear that adult stem cells have an 

important place in medicine as well. While adult stem cells do not replace the need 

for ES cells, they will likely complement it. 

The principal accomplishments over the past 5 years using stem cells from adult 

and neonatal tissues (such as cord blood, amniotic fluid and the cord itself) include: 

(1) our demonstration of their capacity to differentiate into cells of multiple tissues 

(e.g., mesenchymal cells into neurons; cord blood stem cells into cells of the lung), 

(2) their safety and efficacy in laboratory models of disease, and (3) procedures for 

manufacturing stem cells for human testing. In fact, the first clinical trials have already been initiated in acute heart disease (heart attacks) and chronic heart failure, 

acute brain injury and lung injury. In addition, clinical trials with organ-specific 

stem cells are already being studied in diabetes in addition to those in bone marrow 

transplantation. 

With National Institute of Health (NIH) research dollars and other governmental 

and nongovernmental support as well as philanthropic support, there are many new 

projects moving to clinical trials. At our own laboratory, we are collaborating with 

investigators at Johns Hopkins, helping to develop clinical manufacturing methods 

for testing cardiac stem cells; we are collaborating with investigators at Tulane, developing stem cell populations for treatment of genetic disease and bone repair; and, 

we are working with industry, such as Athersys, manufacturing multipotent adult 

stem cells for treatment of radiation and chemotherapy injury. Significant progress 

has been made. 

It is unrealistic to expect that there will be home runs; and, it may take several 

generations of studies to make a new therapy work. As an example, cord blood used 

to treat leukemia and lymphoma took years before it reached its current success. 

In 1990, I performed the first cord blood transplant in the world for a child with 

leukemia. While this child unfortunately died of his underlying disease, scientifically 

it was a success—thereby giving us a reason to push forward. Eight clinical trials 

later, we made modifications that have led to extraordinary survival rates in adults 

with leukemia. Now, patients from all over the world are now receiving this therapy. In addition, the ‘‘double cord blood’’ platform, has solved the problem of access—permitting us to find donors for more than 80 percent of patients, particularly 

important for patients of ethnic and racial minority descent. 

The Stem Cell Therapeutic and Research Act of 2005 authorized substantial funds 

to be used to increase the Nation’s inventory of cord blood by 150,000 units. The 

NCI and NHLBI are supporting multi-institutional trials in children and adults to 

validate these results pioneered at the University of Minnesota. This serves as just 

one example of what your support has accomplished and what it takes to move stem 

cell therapeutics from concept to clinical testing to standard of care. 

After 5 years of intense study, we are now ready to test the multipotent adult 

stem cell, the cells discovered by Dr. Verfaillie and colleagues. We are about to submit our first application to the U.S. Food and Drug Administration. Over the past 

2 years, we have compiled safety and efficacy data in laboratory models and developed the procedures for reliably producing these cells for individual patients. The 

first trials will take place in the setting of radiation and chemotherapy injury and 

the goal is to demonstrate safety and hopefully signs of tissue repair. Will it cure 

23

patients—may be not. Do we give up—no. As in the early trials with cord blood, 

we have to carefully design the right studies that will insure that we learn why the 

cells work or why they don’t work should that occur. We already know in laboratory 

models that multipotent adult stem cells will home preferentially to areas of tissue 

injury. 

But, importantly we have also identified obstacles, reasons why these may fail to 

repair injured tissues. While it is touted as one more advantage of adult stem cells 

over ES cells, it is now clear that the most primitive adult stem cells, even those 

directly from the patient, are susceptible to immune attack. This serves as a clear 

example of why it is not enough to show that a cell can differentiate into a tissue, 

the right models need to be used to predict clinical outcome. For this reason, our 

first trial with the multipotent adult stem cell will be in immune suppressed patients with tissue injury, giving every chance for these stem cells to engraft into 

damaged tissues and effect tissue repair. 

It is not enough to give hope based on the results from a Petri dish. We must 

have better models to move the science forward. It is exactly this stage of research 

that is sorely lacking in funding—this in between stage. Gap funding for Phase I 

clinical trials is an obstacle to our success. Currently, the Federal grants are too 

small to complete the trials and we must compile several funding sources to move 

forward. 

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

clinical treatment but rather—when? I receive hundreds of emails and letters 

monthly asking for direction, help and above all—hope. As a physician who sees patients for whom there is no known treatment, I explore the unknown. I have to keep 

trying. For the most part, I have made some good decisions and patients have benefits. While it will never be fast enough, there are things we can do now that will 

speed the process of moving new laboratory discoveries to clinical trials. First, you 

need to understand the translational pipeline, its components, how it is funded, and 

the potential obstacles. Second, it is necessary to understand why there are disincentives for clinicians and basic scientists to engage in this translational research—as this will help identify solutions. Third, and perhaps most important, you 

must be able to differentiate speculation from fact, as it pertains to stem cells, as 

there is a considerable misinformation and misunderstanding out there on what 

adult stem cells can and cannot do. 

It must be clear that no study with adult or cord blood stem cells outside the context of bone marrow transplantation has proven efficacy. While there are claims to 

suggest otherwise, the results are either contradictory or too preliminary. While I 

wish that I could tell you otherwise, speculation seems to get confused with fact. 

While promising, adult stem cells do not exhibit all the capacities of ES cells. For 

example, we have yet to see stem cells from cord blood or adult tissues (outside the 

heart) differentiate into heart muscle cells that spontaneous beat, as has been 

shown repeatedly with ES cells. 

Can Congress Help Fulfill the Promise of Stem Cell Research?—Absolutely. We are 

here today to help you understand what we know, what we think we know and how 

you might help translate this hope of stem cells into reality. In addition, it is important to know exactly how much is currently being spent on stem cell research. This 

involves separating how much is spent on adult/cord blood versus ES stem cells and 

separating adult/cord blood stem cells into hematopoietic (bone marrow transplant) 

and nonhematopoietic. In my opinion, this is not clear to the public. 

Every single one of us will be faced with a disease amenable to stem cell therapy. 

It may be our child, our spouse, our friend or even ourselves. Adult and cord blood 

stem cells have proven benefits in the treatment of blood cancers and other disorders and perhaps even in tissue repair that has yet to be clearly proven. It is essential that Federal funding be devoted to stem cell biology and therapeutics. All 

the required components to make this work already exist—we just need to bring 

them together. There are patients in this room today and parents of children who 

have passed away looking for a chance to see this hope move into a reality. The 

results are extraordinary; we have to make it happen now on their behalf. For them, 

the stakes are unimaginable.

The CHAIRMAN. Well, thank you. Thank you very much. Very 

good panel. 

We’ve got 12 members here. I thought we’d just do 4 minutes, 

so everybody gets—tries, basically, a question and a followup. 

That’s still 48 minutes, but at least we’ll give everybody, hopefully, 

an opportunity. And then, for those that want to—are able to reVerDate 0ct 09 2002 12:55 Dec 17, 2007 Jkt 000000 PO 00000 Frm 00027 Fmt 6633 Sfmt 6601 S:\DOCS\32808.TXT SLABOR1 PsN: DENISE
24

main, and our panel remain, then we’ll stay here afterwards, but 

permit everyone. 

I’d like to ask, Dr. Landis, Do you believe that restricting the 

NIH funding to the small number of cell lines included in the current policy allows the federally funded scientists to explore the full 

healing potential of a remarkable new field? Are we missing out on 

possible breakthroughs under the current policy? 

Ms. LANDIS. Yes, we are missing out on possible breakthroughs. 

From a purely scientific perspective, Federal funding of additional 

cell lines is necessary to advance the field. The cell lines that are 

eligible for NIH funding now have been shown to have genetic instabilities; in particular, with respect to epigenetic changes in 

methylation. NIH—scientists who are funded by NIH would also 

like to have access to cell lines that have been derived without the 

use of feeder cell lines or animal products. And, finally, there are 

a number of cell lines—many cell lines that have been generated 

since the President’s policy was put in place that have in them 

mutations specific to human diseases, like Huntington’s and ALS 

and Parkinson’s. That would be extraordinarily useful for learning 

about the progression of disease and testing drugs, and those are 

not available either. So, yes, more cell lines would be incredibly important. 

The CHAIRMAN. Dr. Daley, what happens to the best of the American researchers—give us—with the current policy? Where—has 

this research been going abroad? Tell us what’s happening to the 

young, ablest, most gifted researchers. Will they get into this field, 

or are——

Dr. DALEY. Yeah. 

The CHAIRMAN [continuing]. Or are they going into other areas? 

Dr. DALEY. Yeah, you know, we, in the United States, enjoy a remarkable luxury of support from the Federal Government for our 

research. And because we have, I think, some of the best research 

infrastructure, the best universities, we tend to attract the top 

young scientists from all over the world. Increasingly, though, 

when I interview researchers from Europe or from Asia, they ask 

whether or not there is a supportive enough environment in the 

United States that they should commit their careers to coming to 

the United States to do embryonic stem cell research. I can’t say—

or give you a number of the ones who decide against coming. The 

ones who do come to my lab are those intrepid few who are so 

caught up in the excitement of the science—and I say there are a 

remarkable number of scientists internationally who are voting 

with their feet to study these cells, they are fascinating cells. But 

I have every sense, every belief, that there are people who are 

being dissuaded from this very interesting new area of science because of the political climate here in the United States. 

The CHAIRMAN. Lauren, a young lady of courage and fearlessness, if you had the President here today, what do you think you’d 

tell him to encourage him to support this program? 

Ms. STANFORD. I think I’d probably just tell him about the struggles that I’ve gone through and about how passing this bill would 

be very important to me and all the other people with diabetes 

around the world, and it would be a big help if he just didn’t veto 

the bill. 

25

The CHAIRMAN. Very good to hear. 

My time is up. 

Senator Enzi. 

Senator ENZI. Thank you, Mr. Chairman. 

I recognize the message that everyone’s given, that more money 

needs to be spent on all kinds of stem cell research. And I don’t 

think there’s, probably, anybody that disagrees that if there was 

more spent, there would be more discoveries, and we’d know more. 

One of the difficulties is allocating more money to some things, 

when we’re requested to do it for everything. We have to recognize 

that there is a significant group of people out there that feel that 

embryonic stem cell research would be very similar to the other 

end of the spectrum, where people might go through nursing homes 

and find people who no longer can talk, probably close to death, 

they’re just going to be thrown away, and perhaps they could be 

used for research, but not with their permission. That group of people will object to spending taxpayers’ dollars on similar procedures 

for embryonic research. 

The more people that agree that there are moral ways to do this, 

the more support there’ll be for it. I want to congratulate Senator 

Isakson for the work that he’s done to try and come up with some 

compromises and expand the capability of research. A bill that, I 

think, has the capability of bringing more money into the system. 

Professor Wagner, I want to thank you for coming, and I want 

to thank the patients that joined with you today, as well. It does 

sound like you’re making great progress with the adult stem cell 

research. I know that you recognize embryonic stem cell research 

is important, as well. But do you think an expansion of adult stem 

cell research will lead to more therapies in the next 10 years, perhaps more quickly than embryonic stem cell research? Realistically, 

are there any particular treatments, other than the ones you’re 

working on, that you’re intrigued by and excited about seeing put 

into the clinical setting? 

Dr. WAGNER. Well, Senator Enzi, I mean, first off, you know, as 

you stated, I mean, I think that we should be exploring both embryonic stem cell research, as well as adult stem cell research. 

Clearly, more money in adult stem cell research will obviously help 

us advance that, perhaps more quickly, because of the very fact 

that there’s less money for embryonic. On the other hand, with 

that said, it clearly is a supporter for both, but yet, adult stem cell 

work is now being explored not only for, you know, correcting bone 

disease, liver disease, lung disease, it’s also now being explored for 

a way of treating patients with diabetes, as well. 

But we’re at the very earliest phases. The fact is, that it’s still 

quite speculative. You know, I’m not saying that it will ever 

achieve the same status as an embryonic stem-cell-derived therapy, 

but clearly we need to explore all the options, and everything is 

wide open. Many people are working on all these areas simultaneously. 

Senator ENZI. Very quickly, Dr. Landis, we’re operating under 

CR now, which limits the amount of money. There’s no expansion 

of money. Were we to end the embryonic stem cell Federal funding 

prohibition, what research would you cut in order to get the research done on that? 

26

Ms. LANDIS. That’s a very challenging question that we’re facing 

every day at my Institute. If we do this, what won’t we do? What 

NIH does, in general, is to not set aside specific pots of money for 

particular projects, but to fund the very best science. And, as we’ve 

heard, some of the very best scientists are incredibly excited about 

human embryonic stem cells, and expansion of the lines would enable them to write wonderful applications, which we would review 

and hopefully have the money to fund. 

Senator ENZI. Thank you. 

And my time is expired. 

The CHAIRMAN. Senator Harkin. 

Senator HARKIN. Thank you, Mr. Chairman. 

I just want to respond a little bit to what was just said. You 

know, this image is always brought up of old people, as if we’re 

going to use them for experimentation and stuff. Let’s just keep in 

mind what we’re talking about here. We’re talking about a blastocyst with about 150 cells, has absolutely no human form whatsoever. Does it contain all the genetic material and stuff? Yes, it 

does, just like a sperm and an egg does. That’s what we’re talking 

about here. 

So, I listened to the debate that was on the House floor last year 

on this, and one speaker got up and talked about destroying 

fetuses. This is the kind of misinformation that gets out all over 

America, that we’re going to destroy a fetus. And they said it clearly on the House floor, that that’s what this was about. So, you 

know we’ve got to continually combat this kind of misrepresentation of what we’re talking about here. 

One other thing we have to clear up is the fact that the Federal 

Government is already spending U.S. Federal tax dollars on embryonic stem cell research. We already are, on 21 outdated contaminated lines that were derived prior to 9 p.m., August 9, 2001. So, 

don’t tell me that we can’t spend U.S. Federal taxpayers’ dollars on 

stem cell research. We’re already doing it. The fact is, we’re only 

doing it on those that were derived prior to 9 p.m., August 9, 2001. 

Somehow, that’s moral. But to do it on those derived after August 

9, 2001, 9 p.m., is immoral. I don’t know why that is a dividing line 

of morality. I’ve often asked, ‘‘Why wasn’t it 9:05 p.m., 9:30 p.m.?’’ 

‘‘Midnight, 8 p.m.’’ Why was it 9 p.m., August 9, 2001, that somehow is the dividing line? 

Well, when that happened, I said, we thought 70-some lines were 

available—I thought that might be enough. But now we know it’s 

only 21, and every one’s been contaminated and will probably never 

lead to any kind of human therapies. So, we have to keep in mind 

that we already are spending taxpayer dollars on embryonic stem 

cell research. All we’re asking is, let’s expand that, and let’s get 

new lines, that are not contaminated, some that are healthy and 

vibrant, that have been derived already by private sources. That’s 

what we’re talking about. So, I continue to try to clear this up, to 

point out that NIH funds are already available for this. 

Now, I was just figuring out the budget here, Dr. Landis. Last 

year, about 2 percent—my figure—of the entire NIH budget went 

for all forms of stem cell research—adult, animal embryonic, animal nonembryonic, all of it. It was about 2 percent. 

27

I just think that that is woefully inadequate. And I just won’t 

buy this argument that somehow we’re so limited by the budget 

that we can’t do this, when we’re spending $8 billion a month on 

the war in Iraq. Eight billion a month? And we’re spending $637 

million, last year, total, on all stem cell research? Don’t tell me the 

budget’s limiting us. It’s the priorities we have as a Nation, and it’s 

the priorities we set as a Senate and a House, that determines how 

much we spend. There’s no magic thing out there that says you 

can’t spend more than this on research. 

Well, I’ve used up my time, and I didn’t even get to ask a question, but there is one I just want to ask. 

[Laughter.] 

Dr. Daley, I wanted to ask you this. Some people say we don’t 

need more Federal support of embryonic stem cell research because 

States and private resources are funding it. California’s jumped in. 

Wisconsin’s jumped in. New York’s jumped in. I don’t know if Massachusetts has. 

Dr. DALEY. Not yet, but I hope so. 

Senator HARKIN. Well, okay. So, maybe we don’t need more Federal funding. Let the States do it. 

Dr. DALEY. No, actually, this is not an issue for the States. I 

mean, who’s to say that the breakthroughs are going to come in 

California or New Jersey? And I have outstanding colleagues who 

are in other States—Michigan, Arkansas, Iowa. Those researchers 

in those States should be allowed to obtain Federal funding. This 

is a Federal issue. The Federal Government is the lifeblood of scientific research. Virtually all of my funding comes through the Federal Government. It’s a reliable source, it’s subject to peer review, 

it’s subject to ethical oversight. It’s very hard to raise private 

money, and it’s taken outside of those oversight processes when 

it’s—when the research is done privately. 

Senator HARKIN. Thank you, Doctor. 

Thank you, Mr. Chairman. 

The CHAIRMAN. Thank you. Thank you very much. 

Senator Specter. 

Senator SPECTER. Thank you, Mr. Chairman. 

Thank you very much for coming in, Ms. Stanford. I’m very interested to hear your desire to become a U.S. Senator. 

[Laughter.] 

You’re 15, you’ll be eligible to run in 15 years. I just want to offer 

a word of caution, that Senator Kennedy will still have a decade 

left——

[Laughter.] 

Under Senator Thurmond’s tenure. 

[Laughter.] 

So, be patient. 

[Laughter.] 

Lauren, when we have these hearings we wonder what their impact is. Speaking, perhaps, for many people in your situation, does 

this hearing give you more hope? Does it encourage you that something really may be done to deal with your diabetes problem? 

Ms. STANFORD. Well, just coming here and talking, I know that 

my voice is being heard, and that gives me a lot of hope that something may happen in the future and I may have impacted someVerDate 0ct 09 2002 12:55 Dec 17, 2007 Jkt 000000 PO 00000 Frm 00031 Fmt 6633 Sfmt 6601 S:\DOCS\32808.TXT SLABOR1 PsN: DENISE
28

one’s opinion on something else or a choice to make, and it makes 

me feel good about myself and good about the future. 

Senator SPECTER. Dr. Wagner, you testified that you infuse stem 

cells into the heart or other organs which need to be repaired. 

Could you be specific as to what prospects there are—I know it’s 

subject to experimentation, but what prospects there are to deal 

with Lauren Stanford’s diabetes, or what the prospects are to deal 

with Arlen Specter’s Hodgkins disease? 

Dr. WAGNER. Well, you’ve asked quite a few questions right 

there. I mean, but in terms of the prospects for——

Senator SPECTER. I’ve only got 4 minutes. 

[Laughter.] 

Dr. WAGNER. Well, clearly, the one we know is actually Arlen 

Specter’s cancer, because if it’s a lymphoma, then clearly we have 

tried-and-true therapies, though I don’t know the details. But, on 

the other hand, we have—we do have therapeutic—proven results 

in patients with lymphoma leukemia. 

The question then is, is that—Where do we go outside the context of the classic bone marrow transplantation, such as in diabetes 

or in heart disease? What I can specifically address right now is 

that there are a number of studies, both at our institution and others, where we’re specifically taking stem cell populations, whether 

it be cardiac stem-cell-derived, or whether it be mesenchymal stemcell-derived, and actually inputting them into patients with heart 

failure. Have we proven benefit? The results are mixed. But this—

again, this is a step one. But we are moving these cell therapies 

into clinical testing. It will be only time before we know the true 

benefit in that speculation, at this point. 

Senator SPECTER. Dr. Daley, this is your fourth appearance before this subcommittee—in 2002, in 2005, in 2006. Are you getting 

a little tired of coming here without better results? Can you be a 

little more persuasive on this thing? 

Dr. DALEY. Yes, I——

[Laughter.] 

Well, I have to say that I think it’s been a bit frustrating that 

the political—or the—let me say it directly—I think the politicians 

have been lagging somewhat behind the American public. We’ve 

been out there, as a scientific community, trying to speak out on 

these issues, trying to educate, trying to justify the importance of 

stem cell research. 

Senator SPECTER. Dr. Daley, let me interrupt you——

Dr. DALEY. And——

Senator Specter [continuing]. Because I’ve only got a few seconds 

left, and I want to ask Dr. Landis a question. 

You are the vice chair of the NIH Stem Cell Task Force? 

Ms. LANDIS. Yes. 

Senator SPECTER. The subcommittee has polled all of the institutes, and it’s gotten responses almost everywhere, ‘‘Please give us 

embryonic stem cells.’’ The question is raised, Where are you going 

to get the money? As a matter of priorities, isn’t it true that many 

of the institutes would put embryonic stem cell research at a higher priority and make some funding available? 

Ms. LANDIS. Yes, we absolutely would. In fact, in NINDS we 

have several program announcements with setasides specifically for 

29

enhancing the likelihood of investigators working in this area that 

they would get funded. 

Senator SPECTER. Thank you, Mr. Chairman. 

The CHAIRMAN. Thank you. 

Senator Brown. 

Senator BROWN. Thank you, Mr. Chairman. And thank you for 

calling this hearing, the joint hearing. 

I am——

The CHAIRMAN. I have Senator Brown, Coburn, Lautenberg, and 

Isakson. If there’s a difference in that, or someone has a particular 

schedule, if they want to just have their staff let us know, and we’ll 

try. 

Thank you. 

Senator BROWN. Thank you. 

I sat on the House Subcommittee on Health for many years and 

it’s heard several hearings on this whole issue of embryonic stem 

cell research. Of all the issues we discuss in healthcare and in 

other issues in the Senate and in the House when I was there, few 

issues seem so clear cut to me as this one. From all the substantive 

questions, we talk about how the position of our Federal Government on embryonic stem cell is almost for sure causing some very 

young—for some young, very bright scientists to look elsewhere. 

We lose that potential. Some evidence of some scientists going overseas to Singapore, others—perhaps overstated, but some evidence 

of that. I hear Senator Harkin and then Dr. Landis repeating the 

four-door metaphor, if you will. All this is—as I said, of all the 

issues that come in front of this committee and other committees, 

this one seems so clear cut to me, and it’s so discouraging, having 

just gone through a campaign talking about this issue and seeing 

overwhelming public support for it, that we can’t get further along 

than we have. 

Dr. Daley, would you give us some very specific, understandableto-the-public, recent breakthroughs or about-to-happen breakthroughs that can help us along with this, to go home and talk 

about in continuing to educate the public, who will then continue 

to educate the President and other policymakers about the importance of stem cell, if you would. 

Dr. DALEY. Yeah, I would point to the papers, just in the last 

year, that have highlighted the formation of specific cells from 

human embryonic stem cells. It’s the beginning. You know, are 

they breakthroughs? This is the hard work of basic science. But the 

fact that you can make human skin cells, tendon cells, bone cells, 

liver cells, muscle cells, dopaminergic neurons, motor neurons—I 

have a list here. This is a graph that shows the publications, by 

year, for different stem cells. Look at the inflection point here. It’s 

just growing exponentially, the number of publications around embryonic stem cells. And there are breakthroughs among them. I 

would point to any number of diseases where understanding the 

cellular basis is really advanced by these types of publications. 

Senator BROWN. Thank you. 

Thank you, Mr. Chairman. 

The CHAIRMAN. Senator Coburn. 

Senator COBURN. Thank you. Senator Kennedy, if I might, I 

might yield to Senator Isakson. I believe he has a plane to catch. 

30

The CHAIRMAN. Fine. 

Senator ISAKSON. Good, absolutely. Thanks, Senator Coburn. 

First of all, I thank all the—I thank the Chairman for calling the 

hearing today, and all the other chairmen and ranking members——

[Laughter.] 

Their testimony. I don’t want to make any of them mad. And 

thank all the panelists. Lauren, you were terrific. I’m glad you are 

in Massachusetts and Senator Kennedy has to worry about you. 

[Laughter.] 

If you move to Georgia, I’m in big trouble, and I know that. 

[Laughter.] 

And I have to say, personally, to Dr. Wagner, on a personal note, 

my sister’s life was saved because of bone marrow transplant therapy that was developed in the 1990s and tried at the University 

of Nebraska Medical Center in Omaha, where I spent 3 weeks staying with her when that happened. So, I’m very grateful for the 

work that you do, and the advancement of the work that you do. 

Dr. Landis, it’s my understanding that one of the benefits of any 

NIH investment is, it takes the information that is gained from the 

research and puts it in the public domain; whereas, if it’s done 

strictly privately or overseas, that information remains proprietary. 

Is that correct? 

Ms. LANDIS. So—there are differences in publication strategies 

used by people funded by NIH, versus people who work at companies, so that’s true. And NIH investigators, more and more, are 

being asked to put information in the public domain. So, in—as a 

generalization, that is true. 

Senator ISAKSON. Dr. Daley, not—I appreciate your comments on 

the recent articles on ‘‘amniotonic’’—if that’s the right——

Dr. DALEY. Amniotic. 

Senator ISAKSON [continuing]. Pronunciation. This question does 

not relate to that. But on deriving embryonic stem cells for research purposes, which is something everybody, I think, is for, 

there are—the differences come down on the destruction-of-embryos 

question that Senator Harkin, Senator Kennedy, and Senator Enzi 

have all made very quality statements, on both sides of that particular issue. 

At the University of Georgia, three lines, which do receive NIH 

funding, were developed—or embryonic stem cell lines done specifically in diabetes research with eminent scholars—were derived 

from the extraction of embryonic stem cells from level-three gardener-principle grading in the in vitro fertilization process. Do you 

have—that’s one known alternative that does not involve the destruction of an embryo that can be implanted or frozen. Do you 

know of others? 

Dr. DALEY. Yeah. I mean, I know of Dr. Stice’s work, in Georgia. 

In our own lab, we have actually derived five new lines from embryos that were considered such poor quality that they would even 

be discarded before freezing, they’re just not even part of the IVF 

process. We have derived lines. 

There are a number of issues. The efficiency with which you can 

derive those lines is significantly lower than using the embryos 

that are frozen, the embryos that have been judged to be of suffiVerDate 0ct 09 2002 12:55 Dec 17, 2007 Jkt 000000 PO 00000 Frm 00034 Fmt 6633 Sfmt 6601 S:\DOCS\32808.TXT SLABOR1 PsN: DENISE
31

cient quality for clinical use, but which would otherwise be discarded. I think our preference would be to take advantage of the 

hundreds of thousands of embryos that are destined for medical 

waste. We can use those and make good lines. 

We need new lines. I just had our—our lab had a meeting yesterday, where we talked about the crazy H9 cells. My lab now has 

been growing this one NIH line, H9, for 6 years. It’s now so distorted that we call it ‘‘crazy H9.’’ I mean, we really—we really—

you know, we’re handcuffed if we can’t continue to innovate in the 

area of stem cells. There are many, many new lines—and the lines 

that model genetic disease, as Dr. Landis spoke of, these are enormously valuable, and, Why can’t we use our Federal dollars to 

study them? 

The CHAIRMAN. Could I ask—if the Senator would yield—could 

you expand? You said that the efficiency is not as good. As I got 

the thrust of the question, does not destroy the embryo, but that 

was the—as I understood what—the Senator has spoken to me 

about this. I’m interested in your responses. I wrote down ‘‘the efficiency is not as good, and we ought to’’——

Dr. DALEY. Yeah. 

The CHAIRMAN [continuing]. ‘‘Be able to deal with the others.’’ 

Can you still do it? 

Dr. DALEY. Well, they——

The CHAIRMAN. I mean, is it a way of proceeding [inaudible]? 

Dr. DALEY. So, one——

The CHAIRMAN. And what would be the disadvantage? 

Dr. DALEY. Right. One——

The CHAIRMAN. Excuse me. 

Dr. DALEY [continuing]. Strategy that’s been put forth as a—and 

considered as possibly ethically more acceptable than using viable 

embryos is to use the embryos that are deemed of poor clinical 

quality. So, at day three in an in vitro fertilization lab, the 

embryologists will look at the embryos and they’ll judge whether 

the cells are intact or whether they’re fragmented or not. And if 

they’re given a choice, they’ll pick ones that look viable, and the 

ones that have fragmented will be discarded. We get those embryos, and we use them. 

Now, we believe that they will allow us to make normal stem cell 

lines, but I’m not necessarily certain that there aren’t hidden genetic defects in those cells. For some reason, those embryos didn’t 

form. 

The CHAIRMAN. Yeah. 

Dr. DALEY. And so, not only is it much less efficient—it’s about 

1 percent of those poor-quality embryos that we can make yes-cells 

from. I’m not only concerned about the efficiency, I’m really concerned about the integrity of those lines. 

The CHAIRMAN. Senator Lautenberg. 

Senator LAUTENBERG. Thanks, Mr. Chairman. 

I listened with interest to the testimony of each one of you, and 

I congratulate you for doing it. 

But, in particular, Lauren, your story will be listened to by lots 

of people. They’ll hear your voice, and we’re very proud of you. And 

I reach out to you, because I’m a grandfather of 10 grandchildren, 

32

and I realize how lucky we are that they’re without any difficulty 

like the one you have. 

And, to Mr. and Mrs. Stanford, I want you to know this. We’ve 

heard the discussion about money, about resources, and how, ‘‘We 

just don’t have the money.’’ What we’re saying to you, in body, is 

that your priority for Lauren doesn’t compare to the priority of 

making a war that over two-thirds of the American people reject. 

That would represent 200 million people in our society who don’t 

want us to carry on the war as it is. But we can’t afford to spend 

more than $130 million on embryonic stem cell research? I find it 

difficult to understand, and I find it shocking. And I defy any member of the U.S. Senate to tell you that that priority, with that beautiful young woman, that intelligent young woman, who can make 

such a contribution to our society, doesn’t rank with a war that’s 

really distasteful to most of the people in the country. 

Mr. Daley, the lack of the proper investment in stem cell research has slowed progress. Is there any judgment, any guess, 

about how much we’ve lost by not paying attention to this, by not 

making the proper investments to find out what’s there? 

Dr. DALEY. It’s always difficult to answer a question about what 

might have been, what could have been, if we had had more resources. I can speak very personally, that this has led to countless 

hours of delay working through various institutional review boards 

to get approval to do nonpresidential—what we call nonpresidential 

work. Raising private money takes enormous, enormous amounts of 

time. 

We have to set aside, in our laboratory, behind a black curtain, 

a room exclusively for privately funded embryonic stem cell research, where every single pipette, every single bottle, every single 

piece of equipment is labeled with a big sign that says, ‘‘NP,’’ which 

classifies as the nonpresidential resources. It’s an enormous obstruction to progress in this very vital area of research. 

Senator LAUTENBERG. Mr. Chairman, as we sit here, we hear 

about the possibilities that might be there, about relieving young 

people, like Lauren, from having to stick their fingers and so forth. 

We also know, or we believe, that we’re going to be facing a request 

for $100 billion for a supplemental for the war in Iraq, primarily. 

Yet still, out of $3 billion invested in stem cell research, only 4 percent was allowed to be invested in embryonic stem cell research. 

Thank you. 

And thank you, Mr. Chairman, for holding this hearing. 

The CHAIRMAN. Thank you. Thank you very much, Frank. Thank 

you. 

Senator Coburn. 

Senator COBURN. Thank you. 

First of all, let me say how much I appreciate each of you, in 

terms of your dedication to what you’re doing. The area of expertise 

that you’re in today is going to be critical for our future. 

And, Lauren, I want to tell you, I’ve delivered 4,000 babies—I diagnosed a 6-month-old with type-1 diabetes before, and cared for 

her until she graduated from college. You have a great future in 

front of you, and you can have all the children you want, with today’s management techniques. So, don’t worry about that. And I’d 

33

love to see you up there, instead of Senator Kennedy, I promise 

you. 

[Laughter.] 

Besides being a lot better to look at—

[Laughter.] 

A couple of points I want to make, and then I want to ask a couple of questions. 

First of all, let’s make sure we understand the dividing line on 

this debate. Some of us very earnestly believe life begins at conception. At the moment that sperm and egg divide—combine, we believe there’s life there. And so, that’s where the ethical problem is. 

And we want to work as hard as we can to get around that and 

not rationalize away the fact that we believe that’s life. And that 

has to be respected. That position is not taken lightly. Everything 

about our life revolves around some of those critically held beliefs. 

And so, I won’t demean anybody who disagrees with that, but 

you—we can’t be demeaned because we believe that. And I hope 

we’ll respect that opinion. That says nothing about us not wanting 

to get everywhere you all want to get, in terms of cures. 

I’m a two-time cancer survivor. I may be a two-time cancer survivor, we don’t know yet. But the point is—and I have family—sister-in-law and sister with breast cancer—I mean, you know, I don’t 

have a very good set of genes, quite frankly. But the point is, we 

have hope, too, even those that oppose this on this very ideal and 

heartfelt thinking of the value of the initiation of life. 

And I think Senator Isakson is really on to something. And I 

think we have a way that we can move a President to sign money 

for research, even though it might be harder, but the idea of nonviable, nonlife-giving embryos to be used to develop stem lines. 

And, as you said, Dr. Daley, you don’t know yet whether or not 

they’re a compromised cell line. Well, let’s find out. 

I can tell you that there’s—you’re going to get a veto. That’s No. 

1. So, let’s send him something that he won’t veto that helps move 

us down the track. What I would hope is that you all would agree 

to work with us to try to come to that point. Senator Isakson and 

Senator Coleman have worked hard on what looks like a great 

compromise, which we’ll be discussing with people. I can certainly 

live with it, given my beliefs, and I’d hope you all would. 

The other thing that I want to talk about, and I guess I’d better 

ask my question—let me ask my question, and then, if I get a 

chance to talk about it again, I will. 

Autologous transfer. Dr. Wagner’s talked about rejection with 

what they’ve seen so far. But there is no question, there is more 

rejection, within the body, of foreign protein than there is 

autologous protein. Is that not true? 

Dr. WAGNER. That’s generally correct. However, it may be different for stem cells. 

Senator COBURN. Right. But the fact is, everything we know 

about immunology today is, if you put foreign protein into the body, 

you’re going to have a greater reaction than if you put your own 

protein into your body. And so, we have to believe, until your research proves otherwise, that there’s less likelihood to be a rejection if you were using autologous cells, if, in fact, we can use 

autologous cells. And I mean cells that come from your own body. 

34

And the reason I’m a big believer in the research that’s going—

I don’t discount, for a minute, the great work that’s going to come 

from biochemical studies, drug studies, disease-treatment studies, 

and disease treatment from embryonic stem cell research. And I 

wouldn’t discount that for a minute. But the real cures, in my belief, based on rejection and the potential for rejection being less 

with autologous cells, I believe, is going to come from some type of 

nonembryonic stem cells, but maybe more potent or pluripotent, 

not totipotent cells. 

And I’d just like your comments on what you know in the literature, in terms of rejection, in terms of mitochondrial DNA that’s 

going to be a factor in anything that we do, in terms of embryonic 

stem cells, in terms of transplantation. And just a comment on 

that, for a minute, if you would. 

Dr. WAGNER. To whom? 

Senator COBURN. Either one. 

Dr. WAGNER. Well, can I start first? 

Well, first off, you know, although I didn’t get into the details, 

the one thing that we also know, based on our work, is that, because stem cells lack class-one antigens—and I know that’s—

doesn’t really matter to the majority of you——

[Laughter.] 

However, it will be eradicated by natural killer cells. So, even if 

it’s autologous, it will have an immune reaction. 

The second thing is, it’s a misconception that if you believe 

that—the future is going to be, you know, individual autologous 

products, we—it’s too expensive and too difficult to do for each individual patient. Yes, proof of principles can be established using 

that, but, in the great future, we’re going to have to do an off-theshelf product that will not be completely matched, even with 

autologous stem cells. 

Dr. DALEY. Yeah, I would just second that. I would agree with 

that. 

Senator COBURN. Thank you. 

The CHAIRMAN. Senator Sanders. 

Senator COBURN. Senator Kennedy, I would also like to submit 

for the record the RAND study on the availability of embryos——

The CHAIRMAN. Be so included. 

Senator COBURN [continuing]. That are out there. 

The CHAIRMAN. Be so included. 

[The information previously referred to follows:]

35


32808-1.eps
36

Senator SANDERS. Thank you, Mr. Chairman, for holding this important hearing. Let me concur with colleagues in congratulating 

this extraordinarily wonderful panel for your testimony. 

The House of Representatives recently voted, 253 to 174, to lift 

the current limits on Federal funding for embryonic stem cell research. And I have every reason to believe that the U.S. Senate is 

also going to vote in that direction. I certainly will vote for that. 

Unfortunately, we have a situation—and Senator Coburn just 

told you what I suspect is the truth—that we have a President of 

the United States who will likely veto this legislation. I think that 

that is a tragedy, but that is the apparent reality. 

The President regards this issue as murder. I, myself, have a little bit of difficulty understanding that. And the question that I 


32808-2.eps
37

wanted to ask Dr. Daley is, Isn’t it simply true that embryonic 

stem cells which are not implanted are simply discarded and 

thrown into the trash? Is that the case, or is that not the case? 

Dr. DALEY. Well, I mean, there—one has to figure out what to 

do with the many, many tens of thousands—some would say hundreds of thousands—of embryos that are frozen. They’re—a very, 

very tiny percentage would be adopted by others, a small number 

will be used by the couples themselves in future pregnancies, but 

the vast majority will be essentially destined for medical waste, 

discarded. 

Senator SANDERS. And these are cells which you are telling us, 

today, could possibly lead to huge breakthroughs in a whole host 

of diseases which plague millions of Americans and people throughout the world, is that the case? 

Dr. DALEY. Well, I mean, I think the extension is that there are 

enormous opportunities for using those embryos in medical research, whether it’s for deriving stem cells, which is only one aspect of embryo research, these are enormously valuable tools and 

objects for study. 

Senator SANDERS. So, on one hand, we are looking at these cells 

being discarded, destroyed; on the other hand, we are looking at 

these cells being used for research which can make major breakthroughs in some of the most terrible diseases facing humanity. Is 

that really the equation that we’re looking at? 

Dr. DALEY. I believe that is the direct——

Senator SANDERS. Well, you know——

Dr. DALEY [continuing]. Equation. 

Senator SANDERS [continuing]. On many issues, the United 

States is being seen in a lower and lower light all over the world. 

And I have to say that when people around the world—when serious people are trying to deal with some of the worst illnesses and 

diseases facing humanity, they are wondering what is going on in 

our great Nation. And I would hope very much that all over our 

country people begin to stand up and express the long-held faith 

that we, as Americans, have had in basic science; that we try to 

continue the traditions that we have had as being a nation leading 

the world in breakthrough scientific research; and that we give the 

President of the United States all of the reasons in the world, scientific and political, that he should not veto this legislation. 

Thank you very much, Mr. Chairman 

The CHAIRMAN. Thank you very much. 

Senator Hatch. 

Senator HATCH. Well, thank you, Mr. Chairman. 

A lot of my questions have been asked, but I’d like to just take 

this time to make some points. 

I want to thank all of our distinguished scientists for taking the 

time to join us. And I especially want to thank you, Lauren, for 

being here—your testimony is very important to me, and, I think, 

to all of us here—for gracing us with your presence. 

Mr. Chairman, this hearing is important, because opponents of 

embryonic stem cell research point to the fact that there are no 

treatments with embryonic stem cells—they say that it’s a failed 

science. I say it’s a handcuff science. And I’ve brought——

[Laughter.] 

38

These handcuffs, from one of my Secret Service buddies, to make 

that point. 

[Laughter.] 

Now, think of these handcuffs while you listen to the expert scientists assembled here. They will tell us that they and their colleagues are holding the line against spinal cord injuries, Parkinson’s disease, diabetes, and other illnesses. They are exploring the 

potential of stem cells from umbilical-cord blood, of stem cells from 

amniotic fluid, and, of course, of stem cells taken from adults, all 

of which we think is crucial and important, but they are not advancing as rapidly against these afflictions as they could by ethically using frozen cells from—stem cells from frozen and unused 

embryos, because their hands are bound. 

While all forms of stem cell research should be aggressively pursued, scientists see great potential in the use of embryonic stem 

cells because they have the unique ability to become any kind of 

cell in the body, yet we are placing unnecessary and potentially disastrous obstacles in the way of scientists who wish to pursue this 

research to develop breakthrough treatments. 

Let me give you just a few examples. 

Dr. Marie Csete is an anesthesiologist and cell biologist from 

Emory University who works with embryonic stem cells. She tells 

us that the restrictions that current Federal policy places upon her 

and her colleagues are, in her words, ‘‘so odious that many scientists just do not try.’’ I’ll bet you agree with that. 

Dr. DALEY. Yeah. 

Senator HATCH. OK. We are wasting researchers’ time, we are 

wasting their resources, and, in the final analysis, we are wasting 

the lives of many people who could be saved. 

Now, I commend President Bush for authorizing Federal funds to 

study approved human embryonic stem cell lines isolated before 

August 2001. He’s the only President who has allowed this. But 

this was hardly the key to unlock the treatments of the future. In 

2001, there were 71 approved stem cell lines, that has since dwindled to 21 usable lines. And an NIH-funded stem cell researcher at 

the University of Texas, Dr. Ping Wu, told me that, in reality, 

there are only 12 usable lines, the others will not grow. Dr. Wu 

says the few usable cell lines are not enough to represent the general population in any way. Furthermore, these lines are contaminated with animal cells, mouse feeder cells, if you will, and, therefore, can never be placed in humans. 

Dr. Linda Kelley, who happens to be here today, is an associate 

professor of medicine at the University of Utah, somebody I greatly 

admire. She told me that the approved cell lines are so unstable 

that—I know I’m taking a little more time. Is that all right, Mr. 

Chairman? 

The CHAIRMAN. That’s—you’re always——

[Laughter.] 

You always have something useful to say. 

[Laughter.] 

And so, we’re glad to——

Senator HATCH. He doesn’t dare prohibit me, I’ll tell you. I know 

how to get to him. 

The CHAIRMAN. Yeah. 

39

[Laughter.] 

Senator HATCH. But Dr. Kelley is an associate professor of medicine at the University of Utah. She told me that the approved cell 

lines are so unstable that, in her words, ‘‘You are lucky if you can 

recover 10 percent of the cells they send to you.’’ Now, she said the 

cells have been reused for so long that they have degraded and no 

longer represent the human population at all. And I’ll bet you 

agree with that. 

Another unintended consequence of the President’s policy is the 

creation of monopolies. Many owners of these few approved stem 

cell lines have used their monopoly to make the cells very expensive and difficult to obtain. 

Dr. Rick Wetsel, at the University of Texas Health Center, told me about paying $5,000 for one approved cell line, only to find that 

the cells were worthless, forcing him to pay another $5,000 and 

wait 6 months for a new batch. Another scientist, Dr. Csete, who 

I mentioned before, was charged $20,000 for what should have 

been a $500 cell line. The cells they’ve purchased have been reproduced so many times that they do not live very long and cannot be 

used with normal laboratory techniques. So, they are spending 

more money for less valuable material. 

These restrictions also waste time and effort. NIH funds are the 

bedrock of every university’s research program. Hardly a piece of 

equipment or a technician in a medical school, is not in some way, 

supported by NIH. You agree with that, don’t you? You bet your 

life. 

Ms. LANDIS. Yes. 

[Laughter.] 

Senator HATCH. Medical school deans and scientists are afraid of 

violating Federal law by allowing equipment and personnel funded 

by the NIH to touch a nonapproved cell line. You all agree with 

that. 

Dr. Wetsel, in Houston, spent several years obtaining enough 

funds from a private donor to work with a fresh cell line derived 

from a discarded frozen embryo. He was forced to use most of the 

precious funds to buy duplicate equipment and then place it in a 

small laboratory that was isolated from the rest of his NIH-funded 

facility. 

Dr. Csete told me that she is unable to send her doctors-in-training to study stem cell techniques in expert laboratories that work 

with nonapproved lines, because their salaries were funded by 

NIH. 

Scientists in the United States are either walking away from embryonic stem cell research or they’re walking away from the United 

States. 

Now, Mr. Chairman, let me just cite one more proof of how our 

current policy is handcuffing this promising research. 

In the first 6 years after human embryonic stem cells were discovered, at the University of Wisconsin in 1998, half of the 20 most 

quoted publications on this research came from the United States. 

But a closer look at these publications is troubling. Seven of those 

ten U.S. publications came from the University of Wisconsin and 

Geron Corporation, both heavily endowed with private funding. 

Only 3 of the 125 U.S. academic medical centers contributed a top 

40

human embryonic stem cell publication in the 6 years after their 

discovery. If the United States is to remain among the world’s leaders in this research, that simply must change. We must give scientists who want to work with embryonic stem cells a chance, just 

like we do for stem cells from cord blood, amniotic fluid, and from 

adults, all of which I support strongly. 

As Professor Kelley told me, ‘‘There is so much to be learned, and 

it is terribly frustrating.’’ It shouldn’t be that way. It doesn’t have 

to be that way. And I think we’ve got to unlock these handcuffs and 

let our scientists find these treatments and cures that’ll help 

Lauren and others similarly situated. And that’s all you want, is 

a chance to really make these things go. 

And last, but not least, I said, after we had won this debate on 

the floor of the Senate—in the press conference afterwards, I said, 

‘‘Look, there are at least 300 embryonic stem cell lines that are fertile and working in our society today. Why can’t we, since the Government had not participated in the destruction of the embryo, 

allow NIH to partner with those 300—with those companies and 

those 300 lines that would partner with them’’—and I think they 

all would—‘‘so that we can push this research forward?’’ That’s 

what was the theory behind the original 71 stem cell lines that the 

President said we could have. Why not do that? And I don’t think 

it’s a good answer to say, ‘‘Well, that would encourage them to continue to destroy embryos.’’

I just want to tell you how much your testimonies, all of you, 

have meant. And the leadership of these fellows sitting up in front 

here—and we’re happy to have Bernie Sanders with us. I’ve got to 

admit, I was worried about that, but I——

[Laughter.] 

I appreciated his comments this morning. 

But I want to thank each of you. I think you’ve made excellent 

statements. They’re straightforward, they’re honest, and, frankly, 

accurate and true. 

Sorry, Mr. Chairman, I took too long, I know that. 

The CHAIRMAN. Well, thank you. We’ll forgive that extra time, as 

long as you get the SCHIP out of the Finance Committee to look 

at our health insurance for the——

[Laughter.] 

Senator HATCH. Well, we got it out before. 

The CHAIRMAN. That’s right. 

Senator Allard, thank you for your patience and for joining our 

committee. I look forward to hearing from you. 

Senator ALLARD. Well, thank you, Mr. Chairman. And I appreciate the fact that you’re holding this hearing. It’s the first opportunity I’ve had to listen to testimony from the NIH. 

And I’m going to, kind of, steer away from the political arguments and focus a little bit on the science and kind of look at it 

from a practical aspect. 

It’s obvious that we’ve got a problem with the number of dollars 

that you can use for research; you have to set priorities. The other 

challenge that I see are that we have an ethical concern raised by 

some members of this committee. I know, in the scientific community, we also have those ethical concerns. 

41

So, the question that I see before us is, How do we take a patient 

like Lauren—by the way, I’m a veterinarian, so I’ve had some medical training—but how do you take a patient like Lauren, and how 

do you most quickly get a remedy for her juvenile diabetes? 

And I’d also bring out another axiom, in veterinary medicine, 

when we use a more specific treatment, the fewer side effects we’re 

going to deal with. That’s true in immunology, too. We’re developing vaccines whose antigens are more specific, so you have fewer 

reactions to it. 

As I picked up from your testimony, we have a huge immunological problem here. It seems to me that we would do best to focus 

on stem cells for islet cells than we would to focus our efforts on 

a pluripotent type of cell, that, in the long run, the chances of coming up with a treatment that would have fewer immunological 

problems would be to take a specific approach like that. 

My question to both of the physicians that are here is, In your 

research, in trying to set priorities, have you thought about taking 

this type of approach, as opposed to an omnipotent approach and 

if you have, how far along are you in this? I mean, have we identified—the questions have come up—have we identified stem cells for 

islet cells, or have we identified stem cells for pancreatic cells, in 

general? Just how far along are we in that? And I think that would 

help us in our debate. 

Dr. DALEY. Yeah. So, if we speak about type-1 diabetes, which 

is the loss of insulin-producing beta cells, and the best hope for 

cure for Lauren is to replace those beta cells. It is currently highly 

controversial as to whether or not her body, or any of our bodies, 

actually possess stem cells that regenerate insulin-producing beta 

cells. Highly controversial. It is, however——

Senator ALLARD. So, some are saying that they believe there is 

that——

Dr. DALEY. Some are saying it’s——

Senator Allard [continuing]. Possibility, some say that it——

Dr. DALEY. And it’s——

Senator Allard [continuing]. Doesn’t. 

Dr. DALEY. And so——

Senator ALLARD. Yeah. 

Mr. DALEY [continuing]. The way to balance the priorities of research are to let expert scientists make those decisions. I don’t 

think those priorities are well decided here in the Halls of Congress. And that’s done through a very extensive and rigorous peerreview system that the NIH has pioneered. I think that’s where the 

decisions should be made. 

Dr. WAGNER. But just to further that, when stuff—basically, you 

know, all those avenues are being pursued, perhaps at one institution, or many institutions. But I can tell you, even at our own institution, we’re exploring not only islets, as a form of therapy gotten 

from the patient—him or herself—we’re also exploring the use of 

sibling donors, also exploring unrelated donors for islets, as well as 

porcine donors, the pig donors, for islets, as a strategy for treating 

human patients with diabetes, in addition to multipotent adult 

stem cells, in addition to embryonic stem cells. But going back to 

the analogy that Mr. Harkin had, you know, stated years ago, Why 

would we ever want to close any of those doors, when we don’t 

42

know which one will be the true therapy that will have the most 

benefit for the patients with diabetes? We don’t want to close any 

door. And that’s what the scientists are asking for, that ability. 

Senator ALLARD. Well, the problem I have with that is you know, 

maybe not close the door, but what you need to do is, you need to 

look at where you’re going to most likely get the best results from 

the taxpayer dollars that you’re spending. I mean, that’s——

Dr. WAGNER. But we don’t——

Senator Allard [continuing]. That’s the challenge we have. And 

I think it’s——

Dr. DALEY. Right. 

Senator Allard [continuing]. Your challenge, as researchers——

Dr. DALEY. And that’s the challenge——

Senator Allard [continuing]. To convince us——

Mr. DALEY [continuing]. Of the peer-review process——

Senator ALLARD. Yeah—is to convince us——

Mr. DALEY [continuing]. To determine the right——

Senator ALLARD [continuing]. As scientists——

Dr. DALEY [continuing]. Priority. 

Senator ALLARD [continuing]. That you have that plan and you’ve 

given that some thought. And that’s the reason I bring up the arguments in the way I did, because the challenge I think you have, 

as scientists, to present to us, as policymakers, Where you are 

going to get the best results that’ll get the quickest cure for Lauren 

while realizing we have a limited resource in taxpayer dollars. We 

just can’t—we can’t open every door——

Dr. WAGNER. Sure. 

Senator ALLARD [continuing]. So we have to take a look at those 

doors that most likely will open up to a quicker solution. 

Dr. WAGNER. Well, in response to that, I think that, you know, 

we’re already doing it. All the doors are open. It’s just some of the 

doors are being markedly slowed down. 

But the fact is, is that you’re asking for the answer before the 

researchers know what the answer is. We don’t know what the best 

therapy will be. Of course, in the meantime, we explore what we 

can do, and that is, we can look at islets as a form of cellular therapy. But I would believe, based on the results that we have so far, 

that islet transplants themselves are a short-term fix, they don’t 

reproduce themselves for the life of the patient. Maybe we’ll figure 

out a way of doing that in the future, but right now we don’t know. 

And how can I speculate what I don’t know? So, we have to explore 

all the options. And I think that we’re doing that. 

Ms. LANDIS. So, if I could just add, for nervous-system diseases, 

the evidence is pretty clear that adult stem cells, and even, most 

recently, the amniotic-fluid-derived stem cells, really aren’t going to 

provide us with the tools that we need. We now have recipes to create dopamine neurons for Parkinson’s, motor neurons for spinal 

cord injury, oligodendrocytes, or ensheathing cells, for spinal cord 

injury, retinal progenitor cells. And that’s been done within the 5 

years since the President’s policy was put——

Senator ALLARD. Yeah. 

Ms. LANDIS [continuing]. In place. 

Senator ALLARD. Yeah. 

43

Ms. LANDIS. And if, with Senator Hatch’s handcuffs, we’ve been 

able to do that, imagine what the opportunities are without the 

handcuffs. 

Senator ALLARD. Thank you. 

The CHAIRMAN. Thank you. 

Senator Hatch. 

Senator HATCH. Mr. Chairman, could I just ask the whole 

panel—the three doctors one question? 

The CHAIRMAN. Certainly. 

Senator HATCH. Sorry to go out of turn, but——

Dr. Landis, we’ll start with you. In your opinion—and I think 

this is an important question—if NIH funds were made available 

for research, you know, on all ethically obtained embryos from in 

vitro fertilization, would the probability of finding treatments and/

or cures, we’ll put it that way—for human diseases, increase or decrease? 

Ms. LANDIS. Absolutely it would increase. There’s no question 

about that. 

Senator HATCH. Just barely, or would you have a real opportunity to——

Ms. LANDIS. We would have a real opportunity. I can give you 

one specific example. Huntington’s disease is an inherited disease, 

triplet repeat disease, causes a particular kind of death of neuron 

in the brain. We have no good animal models. We don’t know why 

those cells die. We don’t know how to stop that process. If we had 

embryonic stem cells derived from discarded embryos that were not 

implanted, we would be able to make extraordinary inroads into 

therapeutics for that disease. 

Senator HATCH. Dr. Daley. 

Dr. DALEY. I would say, in general, any investment in basic biomedical research is an incredibly important investment for this 

country. It pays off handsomely, in terms of human health. It’s now 

an issue of national security, given the issues around bioterrorism. 

And we, in general, will derive enormous economic benefit, longterm economic benefit, from raising the overall NIH budget, not 

just stem cells. 

Senator HATCH. For embryonic stem cells. 

Mr. Wagner. 

Dr. WAGNER. The first thing that I’d do, like Dr. Kelley, would 

be to actually derive cell lines that would be perfect for use in clinical settings and for patients, which—none of them currently exist 

today. Second thing I’d do is, I would actually then make you also 

think about that this is more than just a cell therapy. As Dr. Daley 

previously mentioned, these cells also give us an unprecedented 

ability to look at new drugs. We can look at molecular events and 

better understand why diseases occur. So, it’s much more than just 

a cell therapy. 

So, it would have a profound effect. But, also, from a practical 

point of view, right now the restrictions that we have are, as you’ve 

heard, just getting the ability to be able to take the cell that’s 

newly derived and be able to give it to our neighbor in Iowa or to 

be able to then give it to the lab next door, outside the confounds 

of what the Government will allow us, because these are NIH-fundVerDate 0ct 09 2002 12:55 Dec 17, 2007 Jkt 000000 PO 00000 Frm 00047 Fmt 6633 Sfmt 6601 S:\DOCS\32808.TXT SLABOR1 PsN: DENISE
44

ed labs, would make it just extraordinarily easy for us to make advances rapidly. 

Senator HATCH. Lauren, we want to help all people like you. And 

these great scientists can do it, if their hands are not handcuffed. 

And we’ve just got to make sure they’re not handcuffed. We’re 

going to win on this, but it’s a shame that it’s been 3, 4 years since 

we really started putting a drive on it. And, for the life of me, I 

can’t understand how some of my friends believe that discarding, 

as hospital waste, 7,000 to 20,000 embryos a year is the right thing 

to do. We ought to be utilizing them for Lauren and people who 

similarly suffer. We’ve just got to wake up on this; untie your 

hands and allow you to really do the research that has to be done. 

This group—many in this hearing today are really dedicated to 

trying to do that. And others are very sincerely on the other side, 

but it’s just a matter of time. We’ve just got to move this forward. 

And I particularly appreciated your testimony in front of NIH 

today. I know it took a lot of courage for you to do that. 

The CHAIRMAN. Thank you. 

Senator HATCH. Thank you. 

The CHAIRMAN. Senator Hatch, like our colleagues Senators Harkin, Specter, and others, has been a real leader in this whole——

Senator HATCH. Thanks. 

The CHAIRMAN [continuing]. And I want to pay tribute to his 

leadership. It’s been very, very important. 

And I had just one final question, if I could. All of you have been 

extremely patient. And that is, Dr. Landis, could we talk about 

those ethical restrictions that we have in the research now that 

guide Federal research, not necessarily applicable to other research? One of the powerful arguments that can be made is, with 

the Federal research, on that, there are going to be the appropriate 

kind of ethical guidelines which are so——

Ms. LANDIS. Right. 

The CHAIRMAN [continuing]. Important, in a major——

Ms. LANDIS. Right. 

The CHAIRMAN [continuing]. New area——

Ms. LANDIS. Right. 

The CHAIRMAN [continuing]. Of research. 

Ms. LANDIS. So, I——

The CHAIRMAN. Just——

Ms. LANDIS [continuing]. I think it’s very——

The CHAIRMAN [continuing]. Just briefly——

Ms. LANDIS [continuing]. Clear that federally funded research 

has monitoring, oversight, and transparency that privately funded 

research will not necessarily have, and that, to the extent that embryonic stem cell research is funded by Federal dollars, then that 

research will benefit from those oversight procedures. 

The CHAIRMAN. I think it’s very important that it’s been included 

in the legislation from the beginning. 

Tom. 

Senator HARKIN. Mr. Chairman, again, I want to thank everyone 

for being here and for hanging in there on this and continuing to 

inform us and enlighten us on the various aspects of all the different forms of stem cell research. And I just want to make it clear 

for the record, from my standpoint, this Senator’s standpoint, I’m 

45

a strong supporter of all stem cell research. I made that clear from 

the very beginning, whether it’s adult stem cell, amniotic, cord 

blood—I think these are all worthy of the most profound research 

that we can do in our society. And they can all lead to different 

things. Some may be good here. You used the analogy of the seeds. 

Some may be good here, Dr. Wagner, for what you’re doing out 

there; some may be good some other place. 

But I think what Senator Hatch said was really very important 

and we’ve got to keep it in mind. I had asked Dr. Daley this question before, about why don’t we just leave it to the States and private entities? Senator Hatch, I think you’ve really hit upon a key 

part of that, and that is—and I’ve heard this from scientists around 

the country that, because NIH funding is so pervasive through universities and academic research centers and everything else, that 

if they want to do this kind of research, the hoops they’ve got to 

go through. If California moves ahead on what they’re doing, they 

will be building separate buildings, separate research centers just 

to do this. What a waste of money and resources. Scientists right 

now have to set up different rooms and different labs, and they 

can’t use their computers at night, because those computers are 

used also for NIH-funded research, so they can’t get online and do 

that. 

Last, one of the analogies I would make on why this is so important for Federal research, is that this committee funded the first 

money into the human genome research in 1989. Dr. Watson came 

to see us. We started putting money in it. And out of that came 

the human genome center at NIH and the mapping and sequencing 

of the human gene. What’s so wonderful about that is not only the 

knowledge that we’ve gained from that, but the fact that it’s free 

for everybody. It’s out there. Anyone, anywhere in the world, can 

go in there and find all that information. Now, if we had left that 

just to the private sector—and, believe me, I love Craig Venter, he’s 

been a friend of mine, he’s done great research, but the fact is that 

we would have had snippets, perhaps, of different parts of the genome that would have been available, at a great, high price that 

Senator Hatch was talking about, and others, but we might not 

have had the whole genome mapped and sequenced. But the fact 

is, you can go online right now, and you can find any one of those 

3 billion pairs anywhere, and it’s available for research. It seems 

to me, stem cell research lends itself to this kind of thing. And I 

don’t mean just embryonic stem cells, I mean all stem cell research, 

that if it’s done by NIH, and funded by NIH, you get it done ethically, you have monitoring, you don’t have duplication, it’s much 

more efficient and effective, you have scientists talking to one another in free form, and all the results of that information is available to the public. It’s available to anyone. 

And, who knows, there might be a young Lauren someplace 

who’s just a budding young scientist, not bound by old concepts and 

old ways of doing things, that sees some research being done there 

and say, ‘‘I think I can do something different with that.’’ It’s one 

of those young scientists that’s going to find how to take some of 

these cells and move them in different directions. That, to me, is 

the promise of all this. 

46

I love the handcuffs. I mean, I think that was really illustrative 

of what we’re talking about here, Senator Hatch, and getting the 

handcuffs off of the Federal Reserve. 

Dr. DALEY. Senator Harkin. 

Senator HARKIN. Who said something? 

Dr. DALEY. I’m sorry, if I could just return to your issue of the 

ethical oversight of stem cell research, I want to make the point 

that scientists are very motivated to do the research in a climate 

of rigorous and ethical oversight, and the International Society for 

Stem Cell Research is about to announce a set of guidelines to govern the conduct, to establish rules of play for scientists. These are 

guidelines that have been vetted through an international committee. And what we’re hoping is that scientists all over the world, 

well beyond the reach of U.S. oversight, will agree to the same 

common set of ethical principles so that the public, worldwide, can 

really embrace this science. 

Senator HARKIN. Well, I pointed out before that the bill that 

we’re talking about here, that we passed last year, the same one 

we’re talking about now, has stronger ethical guidelines and strictures on it than what’s in existing law right now. Very strong ethical guidelines. As you know, the research can only be done on 

those embryos left over in in vitro fertilization clinics and has to 

have the fully informed written consent of the donors. No money 

can change hands, so there can’t be any farming and that kind of 

stuff; it has to be done voluntarily with fully informed written consent and only with those embryos that would be discarded anyway. 

And last, they can only be used for stem cell research—can’t be 

used for implantation and other things, only be used for stem cell 

research. To me, these are pretty tough ethical guidelines, right 

there. Tougher than what we have in existing law right now. I always say to my friends that if they want more ethical standards, 

well, we have them in our bill, and they’re there. 

Well, Mr. Chairman, again, thank you very much for this joint 

hearing. We will continue to pursue this on both levels, on yours 

and on the Appropriations Committee. And, of course, we are hoping, Dr. Landis, with your great leadership, and the whole NIH, 

that we can have a better budget for NIH, this next year. It’s unconscionable to me, and I know it is to my good friend Arlen Specter, that we are fighting, right now, just to get funding for NIH at 

the 2005 level. It’s not that we’re asking for a big increase, we’re 

just trying to get back to the 2005 level in the budget. Hopefully 

this year we can move it ahead, and more aggressively. And again, 

if we do that and open up these doors, perhaps we can make some 

really, really significant progress so that Lauren can become that 

Senator in Massachusetts. 

[Laughter.] 

The CHAIRMAN. That’s good. 

Senator HARKIN. So, thank you very much. 

The CHAIRMAN. Tom, thanks so much. You can feel Senator Harkin’s passion about this issue, and can see why he’s such a leader 

in this undertaking. 

Senator Hatch just has a final few questions and—like to address 

the panel. 

47

Senator HATCH. I’m sorry to keep you a little bit longer, but this 

is an important question. We’ll start with you, Dr. Wagner, and 

then have the three doctors give a crack at this. 

I don’t know if you’re all aware of it, but the Web site for the 

organization called Do No Harm lists 71 diseases that are treatable 

by adult stem cells. Among the diseases listed spinal cord injury, 

stroke, and Parkinson’s disease. Now, three clinical scientists from 

three separate academic medical centers reviewed this Web site 

and concluded that adult stem cells were clinically proven, and 

FDA approved to treat only 9 diseases, not 71. 

So, my question on that is this. Which is the correct number? 

How many diseases are you aware of that are treatable by adult 

stem cells currently? I’m for pushing adult stem cell research as 

fast as we can, as far as we can, but I don’t want to have misrepresentations if these doctors are right, that there are only 9 diseases 

and not 71. 

Dr. WAGNER. Well, I think that within the context, as I presented, the context of using adult stem cells, the only proven use 

of adult stem cells is in the setting of bone marrow transplant to 

treat leukemia——

Senator HATCH. Right. 

Dr. WAGNER [continuing]. Bone-marrow failure. As far as I know, 

there is no proven use of adult stem cells. However, are there FDAsponsored or, you know, monitored trials that are ongoing to ask 

the question, you know, Would this be useful therapy? Yes, there’s 

quite a few different therapies currently being explored, but are not 

yet definitive. 

Senator HATCH. But to say that there are 71 currently in use 

would not be a good representation by a scientist. 

Dr. WAGNER. No, it’s misleading. Seventy-one may be under 

study, but certainly have not been proven. 

Senator HATCH. Dr. Daley. 

Dr. DALEY. Yeah, I mean, with all due respect to Dr. Wagner’s 

fabulous contributions to bone marrow transplantation, this is not 

a panacea, this is a heroic, highly toxic form of therapy which is 

really used in an attempt to save people with fatal diseases. To say 

that we don’t need embryonic stem cells, because look at all this 

success with adult stem cells, is really to deny the fact that the 

current therapies are inadequate. They are just grossly inadequate. 

The future is in pushing research so that we can truly have curative therapies for those 71 diseases. 

Senator HATCH. Do you agree with that? 

Ms. LANDIS. So, I actually have the letter to Science—it was in 

the July 13 issue—in front of me, from Smith, Neaves, and 

Teitelbaum, which lists the evidence indicated that there are 9 and 

not 71, and you might want to have it introduced into the record. 

Senator HATCH. I’ll ask the Chairman to introduce it in the 

record.

[Editor’s Note: The information previously referred to can be found at 

www.sciencemag.org.] 

[Response by Prentice and Tarne to the above letter can be found in Additional Material.]

Ms. LANDIS. And it’s very clear that there are nine approved—

FDA-approved, clinically tested treatments, but that is all that 

48

presently exist. NIH believes it’s critical to continue to fund all 

kinds of stem cell research so that, again, we can move that 9 closer to the 71. 

Senator HATCH. Dr. Landis, are you aware of any young scientists who have redirected their research interest because of the 

lack of Federal funding for embryonic stem cell research? 

Ms. LANDIS. I know of many young scientists who are finishing 

their training who are very reluctant to move into, or retain activities in, human embryonic stem cell research, because of all of the 

complexities that you’ve heard to date. 

Senator HATCH. Well, now, Dr. Daley, let me just finish with 

these questions. Really, this panel has been one of the best panels 

I’ve ever heard on healthcare. And I’ve heard a lot of them over the 

last 30 years with my friends on my left here. And we’ve sat 

through a lot of hearings, and this has been a great panel. But, Dr. 

Daley, opponents of embryonic stem cell research have made much 

of the fact that these cells are reported to produce tumors in experimental animals. What are scientists’ views on this problem? And 

how do you think the potential for amniotic stem cells may prove 

to stack up against embryonic stem cells as potential therapies, or 

is it too early to tell? 

Dr. DALEY. We know about this issue, that embryonic stem cells 

form a type of benign encapsulated mass, which is called a tumor. 

We have strategies for dealing with that. No one is thinking about 

any kind of cell therapy that would involve transplanting the undifferentiated stem cells. You predifferentiate, you make the tissue of 

interest, the highly specialized insulin-producing cell or blood cell—

those are not tumorigenic cells. It’s an issue of safety, it’s an issue 

of clinical testing. We’re at the earliest stages, we know about it, 

and we’re going to anticipate it, and we’re going to look very, very 

hard for ways to get around it. 

Senator HATCH. Then you believe you can solve that problem. 

Dr. DALEY. We do believe we can solve that problem, yes. 

Senator HATCH. OK. Now, what about the potential for amniotic 

stem cells that—how may they prove to stack up against embryonic 

stem cells as potential therapies or—again, is it too——

Dr. DALEY. Yeah. 

Senator HATCH [continuing]. Early to tell? 

Dr. DALEY. Well, amniotic stem cells are fascinating. They are 

not embryonic stem cells. They will not do everything that embryonic stem cells do. But we have work, at the Children’s Hospital, 

with kids who are diagnosed with diaphragmatic hernias, in 

ultrasound, in utero. You can take the amniotic cells, you can grow 

a patch to be used in that child. This is a very exciting application 

of this work. In no way should one choose embryonic over amniotic. 

They both need to be studied, and we need more funding to do it. 

Senator HATCH. So, they’re complementary. 

Dr. DALEY. They’re complementary, not competitive. 

Senator HATCH. Do either of the other two doctors care to comment about that? 

Dr. Wagner? 

Ms. LANDIS. So, if I could just say, there’s a paper recently that’s 

received a lot of attention. Cells differentiated into dopamine neurons transplanted in human embryonic stem cells, differentiated 

49

into dopamine neurons transplanted into a rat model of Parkinson’s, and they did, in fact, see tumors. I would say that this is one 

of a number of studies, and, in the other studies, there was no evidence of a teratoma or a tumor. And the point that Dr. Daley has 

made is that almost certainly undifferentiated cells were transplanted, and, by selecting the differentiated from the undifferentiated, you can prevent tumor formation. So, this is——

Senator HATCH. OK. 

Ms. LANDIS [continuing]. An unusual——

Senator HATCH. But you can use embryonic stem cells to reach 

the differentiated status, is——

Ms. LANDIS. Right. 

Senator HATCH [continuing]. What I——

Ms. LANDIS. Right. 

Senator HATCH [continuing]. Understand——

Ms. LANDIS. Right. Right. 

Senator HATCH [continuing]. Dr. Daley said. 

Dr. Wagner, we’ll finish with you. 

Dr. WAGNER. My only comment was really to emphasize that, 

you know, all the data suggests, exactly as Dr. Daley had indicated, that there are strategies that we can employ to overcome 

this issue of teratomas from the embryonic stem cells. Furthermore, you know, the idea of using amniotic stem cells as a future 

therapy currently is—certainly needs to be explored, but it’s currently all speculative, at this point, where it will go. Again, we 

need to explore all the options. 

Senator HATCH. But without exploring it, we will never——

Dr. WAGNER. We’ll never know. 

Senator HATCH [continuing]. Be able to know whether we can arrive at these treatments or cures that could save us trillions of dollars in healthcare costs and maybe make a lot of the current surgical procedures and other procedures not necessary. I see this as 

the only way we’re going to stop our healthcare budget from just 

consuming the whole Federal budget. But it’s going to take years. 

That’s what science is all about. It’s not something you snap your 

fingers about, it’s something that takes years and years, by brilliant people, who basically never give up, and who can get around 

these so-called ‘‘problems’’ by continued research. 

And I just want to compliment all of you here today. And, 

Lauren, thank you for taking time to be here. You’re the most sophisticated—and you’re in ninth grade, you say? 

Ms. STANFORD. Uh-huh. 

Senator HATCH. You’re the most sophisticated ninth grader I’ve 

ever met in the Senate, so I just want you to know that. 

[Laughter.] 

The CHAIRMAN. So, thank you. 

Just concluding, I’m a great believer that we are in the lifescience century, and we have—the opportunities for breakthroughs 

are unlimited, and the impact that it can have on the quality of 

life in our families and for our country, and, as was pointed out, 

for our economy, in—an innovative new economy with all the implications that that has in—for people that are working in this and 

for leading the world, and demonstrate our interest in helping to 

solve the human condition in other parts of the world. We have—

50

all of this is a breathtaking possibility. And this is an aspect of it, 

in terms of the stem cell research that is just an indispensable aspect of it. So, we are very strong supporters. 

What I want to just say is, we have every intention, for those 

that are here today, of having this on the Senate floor. This is a—

priorities in the House, and we have talked to the leadership, our 

Democratic leader, hopefully with our Republican leader, as—that 

I mentioned, Senator Hatch has been a strong leader on this issue. 

We’ve had a very strong bipartisan commitment. But this is going 

to be on the—this is a high priority, and we expect this to be considered—the earlier the better, but certainly in February. And so, 

this is moving ahead, and this hearing will be really instrumental. 

And we want to just conclude by congratulating Lauren’s parents, too, for doing a great job. 

The committee stands in recess. 

[Additional material follows:]

51

ADDITIONAL MATERIAL


32808-3.eps
52

SUPPORTING MATERIAL FOR TREATING DISEASES WITH ADULT STEM CELLS

DAVID A. PRENTICE AND GENE TARNE

TABLE S1. DISEASE AND CONDITION CHART 

List of conditions in which adult stem cell use produced therapeutic benefit for human patients (1) 

Disease or Condition Quotes from Smith, Neaves, Teitelbaum (emphasis 

added) (2) Additional Comments 

Brain Tumors ..........................................

(Medulloblastoma and Glioma) (3–5) ...

Two clinical studies and one literature review 

indicated that some patients who have 

their brain cancers treated with high-dose 

chemotherapy show improved long-term 

survival rates when transplants of adult 

stem cells from bone marrow or blood are 

used to alleviate side effects of the chemotherapy.

Clinical improvement shown by 

peer-reviewed reference. 

Retinoblastoma (6–7) ............................ Two clinical reports indicated that a small 

group of patients with malignant 

retinoblastoma show improved survival 

rates when transplants of adult stem cells 

from bone marrow or blood are used to alleviate side effects of chemotherapy.

Clinical improvement shown by 

peer-reviewed reference. 

Ovarian Cancer (8–9) ............................ One clinical study and one literature review 

indicated that a subset of ovarian cancer 

patients responds better to high-dose 

chemotherapy when treatment is followed 

by adult stem cell transplants.

Clinical improvement shown by 

peer-reviewed reference. 

Merkel Cell Carcinoma (10) ................... A case study reporting that a single Merkel 

cell carcinoma patient showed a longerthan expected survival time when given 

an adult stem cell transplant after chemotherapy.

Clinical improvement shown by 

peer-reviewed reference. 

Testicular Cancer (11) ........................... Bhatia et al. described a clinical evaluation 

showing improved long-term survival of 

relapsed testicular cancer patients following a radical therapy that included a 

transplant of adult stem cells from bone 

marrow or blood.

One technical reference removed. 

Remaining reference not mentioned by Smith et al. in letter. 

Clinical improvement shown by 

peer-reviewed reference. 

Lymphoma (12–14) ................................ Three clinical reports of various lymphoma 

types and patient numbers indicated that 

some patients show improved long-term 

survival when adult stem cell transplants 

follow high-dose chemotherapy.

Clinical improvement shown by 

peer-reviewed reference. 

Non-Hodgkin’s Lymphoma (15–18) ....... Three clinical studies reported that some nonHodgkin’s lymphoma patients show improved long-term survival when adult 

stem cell transplants follow high-dose 

chemotherapy.

One technical reference removed. 

Three references not mentioned 

by Smith et al. in letter. 

Clinical improvement shown by 

peer-reviewed reference. 

Hodgkin’s Lymphoma (19–20) ............... Two clinical studies indicated that some patients with Hodgkin’s lymphoma show 

overall improved survival rates when 

transplanted with adult stem cells from 

blood.

Clinical improvement shown by 

peer-reviewed reference. 

Acute Lymphoblastic Leukemia (21–23) Two clinical studies, each incorporating multiple leukemia types, indicated that adult 

stem cell transplants from bone marrow or 

umbilical cord blood improve the survival

of children with leukemia when the transplants are performed during the early 

phase of disease.

Adult stem cell transplants from bone marrow 

or blood can induce lasting remission

when leukemias are diagnosed early.

Clinical improvement shown by 

peer-reviewed reference. 

FDA-approved through phase IV 

clinical trials according to 

Smith et al. 

53

TABLE S1. DISEASE AND CONDITION CHART—Continued

List of conditions in which adult stem cell use produced therapeutic benefit for human patients (1) 

Disease or Condition Quotes from Smith, Neaves, Teitelbaum (emphasis 

added) (2) Additional Comments 

Acute Myelogenous Leukemia (24–27) .. Three clinical studies indicated that AML patients who receive adult stem cell transplants after initial disease remission demonstrate improved overall survival.

Adult stem cell transplants from bone marrow 

or blood can accomplish significant improvements in the survival of early-stage 

AML.

Clinical improvement shown by 

peer-reviewed reference. 

FDA-approved through phase IV 

clinical trials according to 

Smith et al. 

Chronic Myelogenous Leukemia (28–29) Two clinical studies, each incorporating multiple leukemia types, indicated that adult 

stem cell transplants from bone marrow or 

umbilical cord blood improve the survival

of children with leukemia when the transplants are performed during the early 

phase of disease.

Adult stem cell transplants from bone marrow 

or blood can induce lasting remission

when leukemias are diagnosed early.

Clinical improvement shown by 

peer-reviewed reference. 

FDA-approved through phase IV 

clinical trials according to 

Smith et al. 

Juvenile Myelomonocytic Leukemia (30) Two clinical studies, each incorporating multiple leukemia types, indicated that adult 

stem cell transplants from bone marrow or 

umbilical cord blood improve the survival

of children with leukemia when the transplants are performed during the early 

phase of disease.

Adult stem cell transplants from bone marrow 

or blood can induce lasting remission

when leukemias are diagnosed early.

Clinical improvement shown by 

peer-reviewed reference. 

FDA-approved through phase IV 

clinical trials according to 

Smith et al.

Angioimmunoblastic Lymphadenopathy 

(31).

A case study reported that a single AILD patient experienced an extended diseasefree period after receiving high-dose 

chemotherapy and a transplant of stem 

cells derived from blood.

Clinical improvement shown by 

peer-reviewed reference. 

Multiple Myeloma (32–33) ..................... Vesole et al. showed that a high-dose chemotherapy regimen followed by transplanting 

adult stem cells from blood resulted in 

modest survival improvements in half of 

study participants.

Clinical improvement shown by 

peer-reviewed reference; updated. 

FDA-approved through phase IV 

clinical trials according to 

Smith et al. 

Myelodysplasia (34–35) ......................... Two clinical studies, each incorporating a 

small number of patients with 

myelodysplasia, suggested that high-dose 

chemotherapy in combination with adult 

stem cell transplants from bone marrow or 

umbilical cord blood improve the survival 

of myelodysplasia patients, particularly 

when this treatment is performed during 

the early phase of disease.

Adult stem cell transplants from bone marrow 

or blood enable myelodysplasia patients to 

withstand a higher dose of chemotherapy, 

thereby increasing the chances of the 

treatment inducing lasting remission.

Clinical improvement shown by 

peer-reviewed reference. 

FDA-approved through phase IV 

clinical trials according to 

Smith et al. 

Breast Cancer (36–39) .......................... Four clinical studies reported that patients 

with high-risk or advanced breast cancer 

had improved survival rates when intensive radiation and/or chemotherapy was 

followed by a transplant of adult stem 

cells derived from bone marrow or blood.

Clinical improvement shown by 

peer-reviewed reference. 

54

TABLE S1. DISEASE AND CONDITION CHART—Continued

List of conditions in which adult stem cell use produced therapeutic benefit for human patients (1) 

Disease or Condition Quotes from Smith, Neaves, Teitelbaum (emphasis 

added) (2) Additional Comments 

Neuroblastoma (40) ............................... A clinical study indicated that transplantation 

of adult stem cells derived from blood is 

associated with improved survival rates

for a specific kind of high-risk neuroblastoma.

Clinical improvement shown by 

peer-reviewed reference. 

Renal Cell Carcinoma (41–44) .............. One clinical study and one case report indicated that, in patients with metastatic 

renal cell carcinoma, transplants of donated adult stem cells from blood delayed 

cancer spread and resulted in overall increase in long-term survival of some patients.

Clinical improvement shown by 

peer-reviewed reference; updated. 

Soft Tissue Sarcoma (45) ...................... One clinical study indicated that some STC 

patients exhibited higher survival rates

when treated with adult stem cells from 

blood after high-dose chemotherapy.

Clinical improvement shown by 

peer-reviewed reference.

Various Solid Tumors (46–50) ............... Four clinical studies evaluating the safety 

and/or efficacy of adult stem cell transplants as a treatment for various solid tumors (inc. breast, ovarian, pediatric brain 

cancers) showed that adult stem cell 

transplants may reduce chemotherapy-related side effects for some cancer patients.

Clinical improvement shown by 

peer-reviewed reference. 

Waldenstrom’s Macroglobulinemia (51) One clinical study indicated that some WM 

patients receiving both high-dose chemotherapy and a transplant of bloodforming 

stem cells showed improved survival rates.

Clinical improvement shown by 

peer-reviewed reference. 

Hemophagocytic Lymphohistiocytosis 

(52).

A case study reported that a child with HLH 

received a transplant of stem cells donated by the patient’s mother 2 months 

after a transplant of liver tissue from the 

same parent. The patient was disease-free 

for 4 months post-stem cell transplant.

Clinical improvement shown by 

peer-reviewed reference. 

POEMS Syndrome (Osteosclerotic 

Myeloma) (53).

An initial clinical study indicated that transplants of adult stem cells from blood alleviated some of the symptoms of POEMS.

Clinical improvement shown by 

peer-reviewed reference.

Systemic Lupus (54–62) ........................ Early reports suggest that immune reconstitution by adult stem cell transplants 

may induce an extended disease-free period in some lupus patients who have 

failed conventional therapies.

Clinical improvement shown by 

peer-reviewed reference. 

Sjogren’s Syndrome (63) ........................ ‘‘Resetting’’ the immune system with chemotherapy and an adult stem cell transplant 

may induce an extended disease-free 

state in some patients with Sjorgen’s syndrome.

Clinical improvement shown by 

peer-reviewed reference. 

Myasthenia (64) ..................................... ........................................................................... Clinical improvement shown by 

peer-reviewed reference. 

Autoimmune Cytopenia (65–66) ............ ‘‘Resetting’’ the immune system with chemotherapy and an adult stem cell transplant 

may induce an extended disease-free 

state in some patients with this disease, 

and a more recent clinical study suggests that such treatment can confer 

benefit to some patients in spite of a 

risk of severe side effects.

One technical reference removed. 

Clinical improvement shown by 

peer-reviewed reference; updated. 

55

TABLE S1. DISEASE AND CONDITION CHART—Continued

List of conditions in which adult stem cell use produced therapeutic benefit for human patients (1) 

Disease or Condition Quotes from Smith, Neaves, Teitelbaum (emphasis 

added) (2) Additional Comments 

Scleromyxedema (67) ............................. More recent evidence indicates that high-dose 

chemotherapy followed by transplants of 

blood-forming stem cells reverse many 

disease symptoms for an extended period, but this treatment is not curative.

Clinical improvement shown by 

peer-reviewed reference. 

Scleroderma (68–69) ............................. Two literature reviews written by the same 

first author described early clinical studies 

of adult stem cell transplants as a treatment for various autoimmune diseases. 

The authors propose that these transplants 

can cause disease remission in some patients.

Clinical improvement shown by 

peer-reviewed reference. 

Crohn’s Disease (70–73) ....................... Initial, small-scale clinical evaluations suggest that this combination approach can 

suppress disease in some patients who 

fail standard treatments, but the adult 

stem cell transplants are intended to help 

patients survive the immune suppressive 

regimen, not directly treat the disease.

Clinical improvement shown by 

peer-reviewed reference. 

Behcet’s Disease (74) ............................ ‘‘Resetting’’ the immune system with chemotherapy and an adult stem cell transplant 

has been observed to induce an extended 

disease-free state in some patients with 

Behcet’s disease.

Clinical improvement shown by 

peer-reviewed reference. 

Rheumatoid Arthritis (75–81) ................ Five early clinical studies and two literature 

reviews indicated that transplants of adult 

stem cells, either donated or from the patient him/herself, in combination with radical use of conventional therapies (e.g., 

immune suppression, chemotherapy and/or 

radiation) delay the course of rheumatoid 

arthritis in some patients with advanced 

disease. More recent evidence suggests 

that some patients with severe rheumatoid 

arthritis who have failed conventional 

therapies can experience an extended 

disease-free period when adult stem cell 

transplants are used as part of a radical 

treatment protocol.

Clinical improvement shown by 

peer-reviewed reference. 

Juvenile Arthritis (82–84) ...................... More recently, adult stem cell transplants 

have been used in combination with immune suppression or radiation treatment. 

Results indicate that about half the patients show disease remission following 

this treatment.

Clinical improvement shown by 

peer-reviewed reference; updated. 

Multiple Sclerosis (85–90) ..................... The combination of adult stem cell transplantation and radical therapy decreased the 

number of observable MS lesions, but following the extent of disease-free remission 

would have required further study. More recent research indicates that radical treatments that include adult stem cell transplants can improve the overall quality of 

life of patients with severe multiple sclerosis (for whom there are no effective 

alternative treatments).

However, the transplant’s ability to reverse 

the onset of MS remains unproven, and in 

most cases the transplant is intended to 

help alleviate the side effects of harsh 

chemotherapy and/or immune suppression.

Clinical improvement shown by 

peer-reviewed reference. 

56

TABLE S1. DISEASE AND CONDITION CHART—Continued

List of conditions in which adult stem cell use produced therapeutic benefit for human patients (1) 

Disease or Condition Quotes from Smith, Neaves, Teitelbaum (emphasis 

added) (2) Additional Comments 

Polychondritis (91) ................................. The single patient included in the cited study 

was reported to have achieved an extended disease-free period.

Clinical improvement shown by 

peer-reviewed reference. 

Systemic Vasculitis (92) ........................ ‘‘Resetting’’ the immune system with chemotherapy and an adult stem cell transplant 

has been observed to induce an extended 

disease-free state in some patients with 

systemic vasculitis.

Clinical improvement shown by 

peer-reviewed reference. 

Alopecia Universalis (93) ....................... This reference was a case study reporting 

that a lymphoma patient who received a 

bone marrow transplant also experienced 

hair regrowth.

Clinical improvement shown by 

peer-reviewed reference.

Severe Combined Immunodeficiency 

Syndrome-X1 (94–95).

In some patients, this therapy is curative,

though immune rejection concerns persist 

throughout the life of the patient.

Clinical improvement shown by 

peer-reviewed reference. 

FDA-approved through phase IV 

clinical trials according to 

Smith et al. 

X-Linked Lymphoproliferative Syndrome 

And X-Linked Hyperimmunoglobulin 

M Syndrome (96–97).

In some patients, this therapy is curative, 

though it remains experimental. Immune 

rejection concerns persist throughout the 

life of the patient, and it is not a suitable 

treatment option for all patients.

Clinical improvement shown by 

peer-reviewed reference. 

Sickle Cell Anemia (98–103) ................. One case report and one observational clinical study (totaling experience with 5 patients) indicated that adult stem cell 

transplants from bone marrow or umbilical 

cord blood can provide some benefit to 

sickle cell patients. A third literature review proposed that adult stem cell transplants hold the potential to treat sickle 

cell anemia because sickle cell results 

from a defect in blood-forming stem cells 

in bone marrow, restoring healthy stem 

cells to a patient’s bone marrow can reverse the disease.

Clinical improvement shown by 

peer-reviewed reference; updated. 

Sideroblastic Anemia (104–105) ........... These references were two small clinical 

studies suggesting that transplants of 

adult stem cells from bone marrow or 

blood can reverse sideroblastic anemia 

for an extended period.

Clinical improvement shown by 

peer-reviewed reference. 

Aplastic Anemia (106–107) ................... Combinations of immune suppression and 

adult stem cell transplantation can improve the long-term survival of aplastic 

anemia patients.

Clinical improvement shown by 

peer-reviewed reference 

FDA-approved through phase IV 

clinical trials according to 

Smith et al. 

Red Cell Aplasia (108) .......................... Transplants of donated blood-forming stem 

cells in combination of chemotherapy may 

improve the long-term survival of some 

patients.

Clinical improvement shown by 

peer-reviewed reference; updated. 

Amegokaryocytic Thrombocytopenia 

(109).

Combinations of chemotherapy, immune suppression and adult stem cell transplants 

have been proposed as a potentially curative treatment. However, due to the 

small number of patients affected by this 

disease, this treatment protocol remains 

experimental.

Clinical improvement shown by 

peer-reviewed reference. 

57

TABLE S1. DISEASE AND CONDITION CHART—Continued

List of conditions in which adult stem cell use produced therapeutic benefit for human patients (1) 

Disease or Condition Quotes from Smith, Neaves, Teitelbaum (emphasis 

added) (2) Additional Comments 

Thalassemia major (110) ....................... This reference is a case report indicating that 

a transplant of donated blood-forming 

stem cells suppressed disease in two 

thalassemia patients. Severe thalassemia 

is often treated by bone marrow transplantation, although this procedure carries considerable risk and is not suitable for all 

patients.

Clinical improvement shown by 

peer-reviewed reference. FDAapproved through phase IV 

clinical trials according to 

Smith et al. 

Primary Amyloidosis (111) ..................... This reference is a literature review proposing 

that transplants of adult stem cells from 

blood and high-dose chemotherapy provide 

an improved treatment for primary amyloidosis. On a small scale, adult stem cell 

transplants have been shown to benefit 

patients with advanced disease, though 

significant treatment-related side effects 

were reported.

Clinical improvement shown by 

peer-reviewed reference. 

Diamond Blackfan Anemia (112) .......... Adult stem cell transplants can reverse bone 

marrow failure in some patients, but they 

do not alter the genetic defect underlying 

the disease and so are not curative.

Clinical improvement shown by 

peer-reviewed reference. 

Fanconi’s Anemia (113–115) ................. Adult stem cell transplants can reverse bone 

marrow failure in some patients, but they 

do not alter the genetic defect underlying 

the disease and so are not curative.

Clinical improvement shown by 

peer-reviewed reference; updated. 

Chronic Epstein-Barr Infection (116–

117).

High-dose chemotherapy and bone marrow replenishment has been reported to reduce 

the amount of active virus in the body 

and can improve survival of some patients.

Clinical improvement shown by 

peer-reviewed reference. 

Hurler’s Syndrome (118–120) ................ One retrospective analysis and one small 

clinical study indicated that adult stem 

cell transplants protected some of the tissues attacked by Hurler’s syndrome but 

provided little relief to other tissues. Longterm survival was improved, with the 

greatest benefit seen in children transplanted early in life.

Clinical improvement shown by 

peer-reviewed reference; updated. 

Osteogenesis Imperfecta (121–123) ...... Three clinical studies, all from the same first 

author, suggested that transplants of 

bone-forming stem cells from bone marrow 

are feasible and can improve the bone 

growth of children suffering from 

osteogenesis imperfecta.

Clinical improvement shown by 

peer-reviewed reference. 

Krabbe Leukodystrophy (124–125) ........ Two early clinical studies reported that cognitive impairments from Krabbe’s disease 

are reduced when children are treated 

with transplants of donated umbilical cord 

blood stem cells.

Clinical improvement shown by 

peer-reviewed reference. 

Osteopetrosis (126–128) ........................ These references were one retrospective analysis and one small clinical study indicating that transplants of adult stem cells 

from bone marrow (either donated or from 

the patient him/herself) improve the longterm survival of some children with a 

certain kind of osteopetrosis.

Clinical improvement shown by 

peer-reviewed reference; updated. 

58

TABLE S1. DISEASE AND CONDITION CHART—Continued

List of conditions in which adult stem cell use produced therapeutic benefit for human patients (1) 

Disease or Condition Quotes from Smith, Neaves, Teitelbaum (emphasis 

added) (2) Additional Comments 

Cerebral X-Linked Adrenoleukodystrophy 

(129).

This reference was one retrospective analysis 

indicating that transplants of adult stem 

cells from blood improve the long-term 

survival of some patients with early-stage 

cerebral Xlinked adrenoleukodystrophy. 

Roughly half of study subjects ultimately 

succumbed to the disease, and the transplant therapy was shown to be significantly less effective for children with advanced disease.

Clinical improvement shown by 

peer-reviewed reference. 

Sandhoff Disease ................................... ........................................................................... Removed from list awaiting 

peer-reviewed report. 

Corneal Regeneration (130–138) ........... All papers reported regeneration of the cornea and improved vision in a subset of 

patients.

Clinical improvement shown by 

peer-reviewed reference. 

Smith et al. misstate repetition 

of reports. 

Limb Gangrene (139) ............................. One pilot study reported that implantation of 

bone marrow stem cells into non-healing 

skin ulcers restored some blood flow to 

the affected area and accomplished 

moderate repair.

Clinical improvement shown by 

peer-reviewed reference. 

Surface Wound Healing (140) ................ ........................................................................... Switched animal & clinical 

paper. 

Clinical improvement shown by 

peer-reviewed reference; updated. 

Jaw Bone Replacement (141) ................ A case report detailed a tissue engineering 

approach to making a new jaw for a patient who had lost his to cancer. By this 

technique, a jaw-shaped metal frame is 

seeded with bone marrow stem cells and 

growth-promoting drugs before implantation in the patient’s shoulder. After 7 

weeks bone grew over the frame and 

was then removed from the shoulder and 

installed as the patient’s new jaw.

Clinical improvement shown by 

peer-reviewed reference. 

Skull Bone Repair (142) ........................ A case report described a tissue engineering 

approach to closing a large skull fracture. 

The open portion of the patient’s skull was 

covered with a protein-based glue that had 

fat stem cells seeded within it. New bone 

growth was observed 3 months after this 

procedure.

Clinical improvement shown by 

peer-reviewed reference. 

Acute Heart Damage (143–159) ............ Seven experimental or early phase clinical 

studies, including one placebo-controlled 

clinical trial, indicated that transfusion of 

a patient’s own bone marrow-derived stem 

cells into the heart shortly after heart attack is relatively safe and is associated 

with regeneration of heart tissue and improved heart function.

The cited studies suggest that transplantation of adult stem cells from bone marrow is associated with improved recovery 

after heart attack.

Clinical improvement shown by 

peer-reviewed reference.

Stroke (160–163) ................................... Three experimental studies reported that implantation of brain stem cells into the 

brains of long-term stroke patients was 

feasible and relatively safe.

Clinical improvement shown by 

peer-reviewed reference; updated. 

59

TABLE S1. DISEASE AND CONDITION CHART—Continued

List of conditions in which adult stem cell use produced therapeutic benefit for human patients (1) 

Disease or Condition Quotes from Smith, Neaves, Teitelbaum (emphasis 

added) (2) Additional Comments 

Parkinson’s Disease (164–166) ............. ........................................................................... Removed abstract & 2 Congressional testimonies. Valid 

stimulation of endogenous 

stem cells not mentioned by 

letter authors. 

Clinical improvement shown by 

peer-reviewed reference; updated. 

Spinal Cord Injury (167) ........................ ........................................................................... Removed 3 Congressional testimonies. 

Clinical improvement shown by 

peer-reviewed reference. 

NOTES: Column 1 shows the disease or condition listed as treated, with peer-reviewed sample references. Column 2 lists comments validating patient improvement from the supplement of Smith, Neaves and Teitelbaum. Column 3 provides additional information regarding listed 

references. 

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adults with malignant primary brain tumors’’; J. Neurooncol. 44, 147–153; Sept., 

1999. 

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87-95; 1999. 

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2001. 

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60

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2000. 

18. Kirita T et al.; ‘‘Primary non-Hodgkin’s lymphoma of the mandible treated with 

radiotherapy, chemotherapy, and autologous peripheral blood stem cell transplantation’’; Oral Surg Oral Med Oral Pathol Oral Radiol Endod. 90, 450–455; Oct. 

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19. Peggs KS et al., ‘‘Clinical evidence of a graft-versus-Hodgkin’s-lymphoma effect 

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27. Bruserud 0 et al.; ‘‘New strategies in the treatment of acute myelogenous leukemia: mobilization and transplantation of autologous peripheral blood stem cells 

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29. Ohnuma K et al.; ‘‘Cord blood transplantation from HLA-mismatched unrelated 

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30. Ohnuma K et al.; ‘‘Cord blood transplantation from HLA-mismatched unrelated 

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61

39. Koc, ON et al.; ‘‘Rapid Hematopoietic Recovery After Coinfusion of AutologousBlood Stem Cells and Culture-Expanded Marrow Mesenchymal Stem Cells in Advanced Breast Cancer Patients Receiving High-Dose Chemotherapy’’; J Clin Oncol 

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48. Lafay-Cousin L et al.; ‘‘High-dose thiotepa and hematopoietic stem cell transplantation in pediatric malignant mesenchymal tumors: a phase II study’’; Bone 

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51. Anagnostopoulos A et al.; ‘‘High-dose chemotherapy followed by stem cell transplantation in patients with resistant Waldenstrom’s macroglobulinemia’’; Bone 

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54. Burt RK et al., Nonmyeloablative hematopoietic stem cell transplantation for 

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55. Burt RK et al., ‘‘Induction of tolerance in autoimmune diseases by hematopoietic 

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56. Wulffraat NM et al.; ‘‘Prolonged remission without treatment after autologous 

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57. Rosen O et al.; ‘‘Autologous stem-cell transplantation in refractory autoimmune 

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58. Traynor AE et al.; ‘‘Treatment of severe systemic lupus erythematosus with 

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59. Burt, RK and Traynor, AE; ‘‘Hematopoietic Stem Cell Transplantation: A New 

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60. Burt RK et al.; ‘‘Hematopoietic stem cell transplantation of multiple sclerosis, 

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62

61. Traynor A and Burt RK; ‘‘Haematopoietic stem cell transplantation for active 

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62. Martini A et al.; ‘‘Marked and sustained improvement 2 years after autologous 

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63. Rabusin M et al.; ‘‘Immunoablation followed by autologous hematopoietic stem 

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64. Rabusin M et al.; ‘‘Immunoablation followed by autologous hematopoietic stem 

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65. Passweg, JR et al., Haematopoetic stem cell transplantation for refractory autoimmune cytopenia, British Journal of Haematology 125, 749–755, June 2004. 

66. Rabusin M et al.; ‘‘Immunoablation followed by autologous hematopoietic stem 

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67. A.M. Feasel et al., ‘‘Complete remission of scleromyxedema following autologous 

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68. Burt RK et al., ‘‘Induction of tolerance in autoimmune diseases by hematopoietic 

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69. Burt, RK and Traynor, AE; ‘‘Hematopoietic Stem Cell Transplantation: A New 

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70. Kreisel W et al., Complete remission of Crohn’s disease after high-dose 

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72. Rabusin M et al.; ‘‘Immunoablation followed by autologous hematopoietic stem 

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85(11 Suppl), 81–85; Nov. 2000. 

73. Hawkey CJ et al.; ‘‘Stem cell transplantation for inflammatory bowel disease: 

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74. Rabusin M et al.; ‘‘Immunoablation followed by autologous hematopoietic stem 

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85(11 Suppl), 81–85; Nov. 2000. 

75. Burt RK et al., ‘‘Induction of tolerance in autoimmune diseases by hematopoietic 

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77. Verburg RJ et al.; ‘‘High-dose chemotherapy and autologous hematopoietic stem 

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78. Rabusin M et al.; ‘‘Immunoablation followed by autologous hematopoietic stem 

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85(11 Suppl), 81–85; Nov. 2000. 

79. Burt, RK and Traynor, AE; ‘‘Hematopoietic Stem Cell Transplantation: A New 

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80. Burt RK et al.; ‘‘Hematopoietic stem cell transplantation of multiple sclerosis, 

rheumatoid arthritis, and systemic lupus erythematosus’’; Cancer Treat. Res. 101, 

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81. Burt, RK et al., ‘‘Autologous hematopoietic stem cell transplantation in refractory rheumatoid arthritis: sustained response in two of four patients,’’ Arthritis 

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83. Rabusin M et al.; ‘‘Immunoablation followed by autologous hematopoietic stem 

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63

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88. Rabusin M et al.; ‘‘Immunoablation followed by autologous hematopoietic stem 

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89. Burt, RK and Traynor, AE; ‘‘Hematopoietic Stem Cell Transplantation: A New 

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90. Burt RK et al.; ‘‘Hematopoietic stem cell transplantation of multiple sclerosis, 

rheumatoid arthritis, and systemic lupus erythematosus’’; Cancer Treat. Res. 101, 

157–184; 1999. 

91. Rosen O et al.; ‘‘Autologous stem-cell transplantation in refractory autoimmune 

diseases after in vivo immunoablation and ex vivo depletion of mononuclear cells’’; 

Arthritis res. 2, 327–336; 2000. 

92. Rabusin M et al.; ‘‘Immunoablation followed by autologous hematopoietic stem 

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97. Amrolia, P. et al., ‘‘Nonmyeloablative stem cell transplantation for congenital 

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98. Klein A et al., Hematopoietic stem cell transplantation for severe sickle cell disease, Rev Med Brux. 2005;26 Spec no:Sp23–5. 

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104. Ayas M et al.; ‘‘Congenital sideroblastic anaemia successfully treated using 

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105. Gonzalez MI et al.; ‘‘Allogeneic peripheral stem cell transplantation in a case 

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106. Gurman G et al.; ‘‘Allogeneic peripheral blood stem cell transplantation for severe aplastic anemia’’; Ther Apher 5(1), 54–57; Feb. 2001. 

107. Kook H et al.; ‘‘Rubella-associated aplastic anemia treated by syngeneic stem 

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108. Rabusin M et al.; ‘‘Immunoablation followed by autologous hematopoietic stem 

cell infusion for the treatment of severe autoimmune disease’’; Haematologica 

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109. Yesilipek et al.; ‘‘Peripheral stem cell transplantation in a child with 

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2000. 

110. Tan PH et al., ‘‘Unrelated peripheral blood and cord blood hematopoietic stem 

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64

111. Sezer O et al.; ‘‘Novel approaches to the treatment of primary amyloidosis’’; 

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112. Ostronoff M et al., ‘‘Successful nonmyeloablative bone marrow transplantation 

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113. Bitan M et al., Fludarabine-based reduced intensity conditioning for stem cell 

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114. Tan PL et al., Successful engraftment without radiation after fludarabine-based 

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115. Kohli-Kumar M et al., ‘‘Haemopoietic stem/progenitor cell transplant in Fanconi 

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116. Fujii N et al.; ‘‘Allogeneic peripheral blood stem cell transplantation for the 

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26, 805–808; Oct. 2000. 

117. Okamura T et al.; ‘‘Blood stem-cell transplantation for chronic active EpsteinBarr virus with lymphoproliferation’’; Lancet 356, 223–224; July 2000. 

118. Cox-Brinkman J et al., Haematopoietic cell transplantation (HCT) in combination with enzyme replacement therapy (ERT) in patients with Hurler syndrome, 

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119. Staba SL et al., Cord-blood transplants from unrelated donors in patients with 

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120. Koc ON et al., Allogeneic mesenchymal stem cell infusion for treatment of 

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121. Horwitz EM et al., ‘‘Isolated allogeneic bone marrow-derived mesenchymal cells 

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122. Horwitz EM et al., ‘‘Clinical responses to bone marrow transplantation in children with severe osteogenesis imperfecta,’’ Blood 97, 1227–1231; 1 March 2001. 

123. Horwitz, EM et al.; ‘‘Transplantability and therapeutic effects of bone marrowderived mesenchymal cells in children with osteogenesis imperfecta’’; Nat. Med. 

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124. Escolar ML et al., ‘‘Transplantation of umbilical cord-blood in babies with infantile Krabbe’s disease,’’ New England Journal of Medicine 352, 2069–2081, 19 

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125. Krivit W et al., ‘‘Hematopoietic Stem-Cell Transplantation in Globoid-Cell 

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126. Tsuji Y et al., Successful nonmyeloablative cord blood transplantation for an 

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127. Driessen GJ et al., Long-term outcome of haematopoietic stem cell transplantation in autosomal recessive osteopetrosis: an EBMT report, Bone Marrow Transplantation 32, 657–663, October 2003. 

128. Schulz et al., HLA-haploidentical blood progenitor cell transplantation in 

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129. Peters C et al., Cerebral X-linked adrenoleukodystrophy: the international 

hematopoietic cell transplantation experience from 1982 to 1999, Blood 104, 881–

888, 1 August 2004. 

130. Inatomi T et al., Midterm results on ocular surface reconstruction using cultivated autologous oral mucosal epithelial transplantation, American Journal of 

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131. Nishida K et al., Corneal reconstruction with tissue-engineered cell sheets composed of autologous oral mucosal epithelium, New England Journal of Medicine

351, 1187–1196, 16 September 2004. 

132. Anderson DF et al.; ‘‘Amniotic Membrane Transplantation After the Primary 

Surgical Management of Band Keratopathy’’; Cornea 20(4), 354–361; May 2001. 

133. Anderson DF et al.; ‘‘Amniotic membrane transplantation for partial limbal 

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134. Henderson TR et al.; ‘‘The long term outcome of limbal allografts: the search 

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65

135. Daya SM, Ilari FA; ‘‘Living related conjuctival limbal allograft for the treatment of stem cell deficiency’’; Opthalmology 180, 126–133; January 2001. 

136. Schwab IR et al.; ‘‘Successful transplantation of bioengineered tissue replacements in patients with ocular surface disease’’; Cornea 19, 421–426; July 2000. 

137. Tsai et al.; ‘‘Reconstruction of damaged corneas by transplantation of 

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2000. 

138. Tsubota K et al.; ‘‘Treatment of severe ocular-surface disorders with corneal 

epithelial stem-cell transplantation’’; New England Journal of Medicine 340, 

1697–1703; June 3, 1999. 

139. Tateishi-Yuyama E et al.; ‘‘Therapeutic angiogenesis for patients with limb 

ischaemia by autologous transplantation of bone-marrow cells: a pilot study and 

a randomised controlled trial’’; Lancet 360, 427–435; 10 August 2002. 

140. Badiavas EV and Falanga V, ‘‘Treatment of chronic wounds with bone marrowderived cells,’’ Archives of Dermatology 139, 510–516, 2003. 

141. Warnke PH et al., Growth and transplantation of a custom vascularised bone 

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142. Lendeckel S et al., Autologous stem cells (adipose) and fibrin glue used to treat 

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143. Joseph J et al., Safety and effectiveness of granulocyte-colony stimulating factor 

in mobilizing stem cells and improving cytokine profile in advanced chronic heart 

failure, American Journal of Cardiology 97, 681–684, 1 March 2006. 

144. Blocklet D et al., Myocardial homing of nonmobilized peripheral-blood CD34+ 

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145. Janssens S et al., Autologous bone marrow-derived stem-cell transfer in patients with ST-segment elevation myocardial infarction: double-blind, randomised 

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146. Patel AN et al., Surgical treatment for congestive heart failure with autologous 

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147. Ince H et al., Preservation from left ventricular remodeling by front-integrated 

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148. Ince H et al., Prevention of left ventricular remodeling with granulocyte colonystimulating after acute myocardial infarction, Circulation 112, I-73-1-80, 30 August 2005. 

149. Bartunek J et al., Intracoronary injection of CD133-positive enriched bone marrow progenitor cells promotes cardiac recovery after recent myocardial infarction, 

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150. Dohmann HFR et al., Transendocardial autologous bone marrow mononuclear 

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151. Wollert KC et al., ‘‘Intracoronary autologous bone-marrow cell transfer after 

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152. Britten MB et al., ‘‘Infarct remodeling after intracoronary progenitor cell treatment in patients with acute myocardial infarction’’; Circulation 108, 2212–2218; 

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153. Perin EC et al.; ‘‘Transendocardial, autologous bone marrow cell transplantation for severe, chronic ischemic heart failure’’; Circulation 107, r75-r83; published online May 2003. 

154. Stamm C et al.; ‘‘Autologous bone-marrow stem-cell transplantation for myocardial regeneration’’; The Lancet 361, 45–46; 4 January 2003. 

155. Tse H-F et al.; ‘‘Angiogenesis in ischaemic myocardium by intramyocardial 

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4 January 2003. 

156. Strauer BE et al.; ‘‘Repair of infarcted myocardium by autologous intracoronary 

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157. Strauer BE et al.; ‘‘Myocardial regeneration after intracoronary transplantation 

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158. Menasche ́ P et al. ‘‘Myoblast transplantation for heart failure.’’ Lancet 357, 

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159. Menasche ́ P et al. [‘‘Autologous skeletal myoblast transplantation for cardiac 

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66

160. Shyu W-C et al., Granulocyte colony-stimulating factor for acute ischemic 

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161. Stilley CS et al., Changes in cognitive function after neuronal cell transplantation for basal ganglia stroke, Neurology 63, 1320–1322, October 2004. 

162. Meltzer CC et al.; ‘‘Serial [18F]Fluorodeoxyglucose Positron Emission Tomography after Human Neuronal Implantation for Stroke’’; Neurosurgery 49, 586–

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163. Kondziolka D et al.; ‘‘Transplantation of cultured human neuronal cells for patients with stroke’’; Neurology 55, 565–569; August 2000. 

164. Love S et al., Glial cell line-derived neurotrophic factor induces neuronal 

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165. Slevin JT et al., Improvement of bilateral motor functions in patients with Parkinson disease through the unilateral intraputaminal infusion of glial cell line-derived neurotrophic factor, Journal of Neurosurgery 102, 216–222, February 2005. 

166. Gill SS et al.; ‘‘Direct brain infusion of glial cell line-derived neurotrophic factor 

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PEER-REVIEWED REFERENCES SHOWING APPLICATIONS OF ADULT STEM CELLS THAT

PRODUCE THERAPEUTIC BENEFIT FOR HUMAN PATIENTS

(NOT A COMPLETE LISTING, SAMPLE REFERENCES)

ADULT STEM CELLS—HEMATOPOIETIC REPLACEMENT

CANCERS

BRAIN TUMORS—medulloblastoma and glioma 

Dunkel, IJ; ‘‘High-dose chemotherapy with autologous stem cell rescue for malignant 

brain tumors’’; Cancer Invest. 18, 492–493; 2000. 

Abrey, LE et al.; ‘‘High dose chemotherapy with autologous stem cell rescue in 

adults with malignant primary brain tumors’’; J. Neurooncol. 44, 14: -153; Sept., 

1999. 

Finlay, JL; ‘‘The role of high-dose chemotherapy and stem cell rescue in the treatment of malignant brain tumors: a reappraisal’’; Pediatr. Transplant 3 Suppl. 1, 

8: -95; 1999. 

RETINOBLASTOMA 

Hertzberg H et al.; ‘‘Recurrent disseminated retinoblastoma in a : -year-old girl 

treated successfully by high-dose chemotherapy and CD34-selected autologous peripheral blood stem cell transplantation’’; Bone Marrow Transplant 2: (6), 653–

655; March 2001. 

Dunkel IJ et al.; ‘‘Successful treatment of metastatic retinoblastoma’’; Cancer 89, 

211: -2121; Nov 15 2000. 

OVARIAN CANCER 

Stiff PJ et al.; ‘‘High-dose chemotherapy and autologous stem-cell transplantation 

for ovarian cancer: An autologous blood and marrow transplant registry report’’; 

Ann. Intern. Med. 133, 504–515; Oct. 3, 2000. 

Schilder, RJ and Shea, TC; ‘‘Multiple cycles of high-dose chemotherapy for ovarian 

cancer’’; Semin. Oncol. 25, 349–355; June 1998. 

MERKEL CELL CARCINOMA 

Waldmann V et al.; ‘‘Transient complete remission of metastasized merkel cell carcinoma by high-dose polychemotherapy and autologous peripheral blood stem cell 

transplantation’’; Br. J. Dermatol. 143, 83: -839; Oct 2000. 

TESTICULAR CANCER 

Bhatia S et al.; ‘‘High-dose chemotherapy as initial salvage chemotherapy in patients with relapsed testicular cancer’’; J. Clin. Oncol. 18, 3346–3351; Oct. 19, 

2000. 

LYMPHOMA 

Tabata M et al.; ‘‘Peripheral blood stem cell transplantation in patients over 65 

years old with malignant lymphoma—possibility of early completion of chemotherapy and improvement of performance status’’; Intern Med 40, 4: 1–4: 4; June 

2001. 

67

Josting, A; ‘‘Treatment of Primary Progressive Hodgkin’s and Aggressive Non-Hodgkin’s Lymphoma: Is There a Chance for Cure?’’; J Clin Oncol 18, 332–339; 2000. 

Koizumi M et al.; ‘‘Successful treatment of intravascular malignant lymphomatosis 

with high-dose chemotherapy and autologous peripheral blood stem cell transplantation’’; Bone Marrow Transplant 2: 1101–1103; May 2001. 

NON-HODGKIN’S LYMPHOMA 

Buadi FK et al., Autologous hematopoietic stem cell transplantation for older patients with relapsed non-Hodgkin’s lymphoma, Bone Marrow Transplant 3: , 101: 

-1022, June 2006. 

Tabata M et al.; ‘‘Peripheral blood stem cell transplantation in patients over 65 

years old with malignant lymphoma—possibility of early completion of chemotherapy and improvement of performance status’’; Intern Med 40, 4: 1-4: 4; June 

2001. 

Josting, A; ‘‘Treatment of Primary Progressive Hodgkin’s and Aggressive Non-Hodgkin’s Lymphoma: Is There a Chance for Cure?’’; J Clin Oncol 18, 332–339; 2000. 

Kirita T et al.; ‘‘Primary non-Hodgkin’s lymphoma of the mandible treated with radiotherapy, chemotherapy, and autologous peripheral blood stem cell transplantation’’; Oral Surg Oral Med Oral Pathol Oral Radiol Endod. 90, 450–455; Oct. 

2000. 

HODGKIN’S LYMPHOMA 

Peggs KS et al., ‘‘Clinical evidence of a graft-versus-Hodgkin’s-lymphoma effect after 

reduced-intensity allogeneic transplantion,’’ Lancet 365, 1934–1941, 4 June 2005. 

Josting, A; ‘‘Treatment of Primary Progressive Hodgkin’s and Aggressive Non-Hodgkin’s Lymphoma: Is There a Chance for Cure?’’; J Clin Oncol 18, 332–339; 2000. 

ACUTE LYMPHOBLASTIC LEUKEMIA 

Laughlin MJ et al.; ‘‘Hematopoietic engraftment and survival in adult recipients of 

umbilical-cord blood from unrelated donors,’’ New England Journal of Medicine 

344, 1815–1822; June 14, 2001. 

Ohnuma K et al.; ‘‘Cord blood transplantation from HLA-mismatched unrelated donors as a treatment for children with haematological malignancies’’; Br J 

Haematol 112(4), 981–98: ; March 2001. 

Marco F et al.; ‘‘High Survival Rate in Infant Acute Leukemia Treated With Early 

High-Dose Chemotherapy and Stem-Cell Support’’; J Clin Oncol 18, 3256–3261; 

Sept. 15 2000. 

ACUTE MYELOGENOUS LEUKEMIA 

Laughlin MJ et al.; ‘‘Hematopoietic engraftment and survival in adult recipients of 

umbilical-cord blood from unrelated donors,’’ New England Journal of Medicine 

344, 1815–1822; June 14, 2001. 

Ohnuma K et al.; ‘‘Cord blood transplantation from HLA-mismatched unrelated donors as a treatment for children with haematological malignancies’’; Br J 

Haematol 112(4), 981–98: ; March 2001. 

Gorin NC et al.; ‘‘Feasibility and recent improvement of autologous stem cell transplantation for acute myelocytic leukaemia in patients over 60 years of age: importance of the source of stem cells’’; Br. J. Haematol. 110, 88: -893; Sept 2000. 

Bruserud 0 et al.; ‘‘New strategies in the treatment of acute myelogenous leukemia: 

mobilization and transplantation of autologous peripheral blood stem cells in 

adult patients’’; Stem Cells 18, 343–351; 2000. 

CHRONIC MYELOGENOUS LEUKEMIA 

Laughlin MJ et al.; ‘‘Hematopoietic engraftment and survival in adult recipients of 

umbilical-cord blood from unrelated donors,’’ New England Journal of Medicine 

344, 1815–1822; June 14, 2001. 

Ohnuma K et al.; ‘‘Cord blood transplantation from HLA-mismatched unrelated donors as a treatment for children with haematological malignancies’’; Br J 

Haematol 112(4), 981–98: ; March 2001. 

JUVENILE MYELOMONOCYTIC LEUKEMIA 

Ohnuma K et al.; ‘‘Cord blood transplantation from HLA-mismatched unrelated donors as a treatment for children with haematological malignancies’’; Br J 

Haematol 112(4), 981–98: ; March 2001. 

CHRONIC MYELOMONOCYTIC LEUKEMIA 

Elliott MA et al., Allogeneic stem cell transplantation and donor lymphocyte infusions for chronic myelomonocytic leukemia, Bone Marrow Transplantation 3: , 

1003–1008, 2006. 

68

ANGIOIMMUNOBLASTIC LYMPHADENOPATHY with DYSPROTEINEMIA 

Lindahl J et al.; ‘‘High-dose chemotherapy and APSCT as a potential cure for relapsing hemolysing AILD’’; Leuk Res 25(3), 26: -2: 0; March 2001. 

MULTIPLE MYELOMA 

Aviles A et al., Biological modifiers as cytoreductive therapy before stem cell transplant in previously untreated patients with multiple myeloma, Annals of Oncology

16, 219–221, 2005. 

Vesole, DH et al.; ‘‘High-Dose Melphalan With Autotransplantation for Refractory 

Multiple Myeloma: Results of a Southwest Oncology Group Phase II Trial’’; J Clin 

Oncol 1: , 21: 3–21: 9; July 1999. 

MYELODYSPLASIA 

Ohnuma K et al.; ‘‘Cord blood transplantation from HLA-mismatched unrelated donors as a treatment for children with haematological malignancies’’; Br J 

Haematol 112(4), 981–98: ; March 2001. 

Bensinger WI et al.; ‘‘Transplantation of bone marrow as compared with peripheralblood cells from HLA-identical relatives in patients with hematologic cancers’’; 

New England Journal of Medicine 344, 1: 5–181; Jan 18 2001. 

BREAST CANCER 

Damon LE et al.; ‘‘High-dose chemotherapy and hematopoietic stem cell rescue for 

breast cancer: experience in California’’; Biol. Blood Marrow Transplant 6, 496–

505; 2000. 

Paquette, RL et al., ‘‘Ex vivo expanded unselected peripheral blood: progenitor cells 

reduce posttransplantation neutropenia, thrombocytopenia, and anemia in patients with breast cancer,’’ Blood 96, 2385–2390; October, 2000. 

Stiff P et al.; ‘‘Autologous transplantation of ex vivo expanded bone marrow cells 

grown from small aliquots after high-dose chemotherapy for breast cancer’’; Blood 

95, 2169-21: 4; March 15, 2000. 

Koc, ON et al.; ‘‘Rapid Hematopoietic Recovery After Coinfusion of Autologous-Blood 

Stem Cells and Culture-Expanded Marrow Mesenchymal Stem Cells in Advanced 

Breast Cancer Patients Receiving High-Dose Chemotherapy’’; J Clin Oncol 18, 30: 

-316; January 2000. 

NEUROBLASTOMA 

Kawa, K et al.; ‘‘Long-Term Survivors of Advanced Neuroblastoma With MYCN Amplification: A Report of 19 Patients Surviving Disease-Free for More Than 66 

Months’’; J Clin Oncol 1: :3216–3220; October 1999. 

RENAL CELL CARCINOMA 

Barkholt L et al., Allogeneic haematopoietic stem cell transplantation for metastatic 

renal carcinoma in Europe, Annals of Oncology published online 28 April 2006. 

Arya M et al., Allogeneic hematopoietic stem-cell transplantation: the next generation of therapy for metastatic renal cell cancer, Nat Clin Pract Oncol. 1, 32–38, 

Nov 2004. 

Childs R et al., ‘‘Regression of Metastatic Renal-Cell Carcinoma after 

Nonmyeloablative Allogeneic Peripheral-Blood Stem-Cell Transplantation,’’ New 

England Journal of Medicine 343, : 50-: 58; Sept. 14, 2000. 

Childs, RW; ‘‘Successful Treatment of Metastatic Renal Cell Carcinoma With a 

Nonmyeloablative Allogeneic Peripheral-Blood Progenitor-Cell Transplant: Evidence for a Graft-Versus-Tumor Effect’’:; J Clin Oncol 1: , 2044–2049; July 1999. 

SOFT TISSUE SARCOMA 

Blay JY et al.; ‘‘High-dose chemotherapy with autologous hematopoietic stem-cell 

transplantation for advanced soft tissue sarcoma in adults’’; J. Clin. Oncol. 18, 

3643–3650; Nov 1 2000. 

EWING’S SARCOMA 

Drabko K et al., Megachemotherapy followed by autologous stem cell transplantation in children with Ewing’s sarcoma, Pediatric Transplantation 9, 618–621, 

2005. 

VARIOUS SOLID TUMORS 

Pedrazolli P et al., High dose chemotherapy with autologous hematopoietic stem cell 

support for solid tumors other than breast cancer in adults, Annals of Oncology

published online 1: March 2006. 

Nieboer P et al.; ‘‘Long-term haematological recovery following high-dose chemotherapy with autologous bone marrow transplantation or peripheral stem cell 

69

transplantation in patients with solid tumours’’; Bone Marrow Transplant 2: , 

959–966; May 2001. 

Lafay-Cousin L et al.; ‘‘High-dose thiotepa and hematopoietic stem cell transplantation in pediatric malignant mesenchymal tumors: a phase II study’’; Bone Marrow Transplant 26, 62: -632; Sept. 2000. 

Michon, J and Schleiermacher, G. ‘‘Autologous haematopoietic stem cell transplantation for paediatric solid tumors,’’ Baillieres Best Practice Research in Clinical 

Haematology 12, 24: -259, March–June, 1999. 

Schilder, RJ et al.; ‘‘Phase I trial of multiple cycles of high-dose chemotherapy supported by autologous peripheral-blood stem cells’’; J. Clin. Oncol. 1: , 2198–220: 

; July 1999. 

WALDENSTROM’S MACROGLOBULINEMIA 

Anagnostopoulos A et al.; ‘‘High-dose chemotherapy followed by stem cell transplantation in patients with resistant Waldenstrom’s macroglobulinemia’’; Bone Marrow Transplant 2: , 102: -1029; May 2001. 

HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS 

Matthes-Martin S et al.; ‘‘Successful stem cell transplantation following orthotopic 

liver transplantation from the same haploidentical family donor in a girl with 

hemophagocytic lymphohistiocytosis’’; Blood 96, 399: -3999; Dec 1, 2000. 

POEMS SYNDROME (OSTEOSCLEROTIC MYELOMA) 

Dispenzieri A et al., Peripheral blood stem cell transplantation in 16 patients with 

POEMS syndrome, and a review of the literature, Blood 104, 3400-340: , 15 November 2004. 

MYELOFIBROSIS 

Cornetta K et al., Umbilical cord blood transplantation in adults: results of the prospective Cord Blood Transplantation (COBLT), Biol Blood Marrow Transplant 11, 

149–160, February 2005. 

Cervantes F, Modern management of myelofibrosis, Br J Haematol 128, 583–592, 

March 2005. 

Kroger N et al., Pilot study of reduced-intensity conditioning followed by allogeneic 

stem cell transplantation from related and unrelated donors in patients with 

myelofibrosis, Br J Haematol 128, 690-69: , March 2005. 

Thiele J et al., Dynamics of bone marrow changes in patients with chronic idiopathic 

myelofibrosis following allogeneic stem cell transplantation, Histol Histopathol 20, 

8: -89, July 2005. 

Rondelli D et al., Allogeneic hematopoietic stem-cell transplantation with reducedintensity conditioning in intermediate- or high-risk patients with myelofibrosis 

with myeloid metaplasia, Blood 105, 4115–4119, 15 May 2005. 

Benesova P et al., [Complete regression of bone marrow fibrosis following allogeneic 

peripheral blood stem cell transplantation in a patient with idiopathic 

myelofibrosis] [Article in Czech], Cesk Patol 40, 16: -1: 1, October 2004. 

ADULT STEM CELLS-IMMUNE SYSTEM REPLACEMENT 

AUTOIMMUNE DISEASES

SYSTEMIC LUPUS 

Burt RK et al., Nonmyeloablative hematopoietic stem cell transplantation for systemic lupus erythematosus, Journal of the American Medical Association 295, 52: 

-535, February 1, 2006. 

Burt RK et al., ‘‘Induction of tolerance in autoimmune diseases by hematopoietic 

stem cell transplantation: getting closer to a cure?,’’ Blood 99, : 68-: 84, 1 February 2002. 

Wulffraat NM et al.; ‘‘Prolonged remission without treatment after autologous stem 

cell transplantation for refractory childhood systemic lupus erythematosus’’; Arthritis Rheum 44(3), : 28-: 31; March 2001. 

Rosen O et al.; ‘‘Autologous stem-cell transplantation in refractory autoimmune diseases after in vivo immunoablation and ex vivo depletion of mononuclear cells’’; 

Arthritis res. 2, 32: -336; 2000. 

Traynor AE et al.; ‘‘Treatment of severe systemic lupus erythematosus with highdose chemotherapy and haemopoietic stem-cell transplantation: a phase I study’’; 

Lancet 356, : 01-: 0: ; August 26, 2000. 

Burt, RK and Traynor, AE; ‘‘Hematopoietic Stem Cell Transplantation: A New Therapy for Autoimmune Disease’’; Stem Cells 1: , 366-3: 2; 1999. 

70

Burt RK et al.; ‘‘Hematopoietic stem cell transplantation of multiple sclerosis, rheumatoid arthritis, and systemic lupus erythematosus’’; Cancer Treat. Res. 101, 15: 

-184; 1999. 

Traynor A and Burt RK; ‘‘Haematopoietic stem cell transplantation for active systemic lupus erythematosus’’; Rheumatology 38, : 6: -: : 2; August 1999. 

Martini A et al.; ‘‘Marked and sustained improvement 2 years after autologous stem 

cell transplant in a girl with system sclerosis’’; Rheumatology 38, : : 3; August 

1999. 

SJOGREN’ S SYNDROME 

Rabusin M et al.; ‘‘Immunoablation followed by autologous hematopoietic stem cell 

infusion for the treatment of severe autoimmune disease’’; Haematologica 85(11 

Suppl), 81–85; Nov. 2000. 

MYASTHENIA 

Rabusin M et al.; ‘‘Immunoablation followed by autologous hematopoietic stem cell 

infusion for the treatment of severe autoimmune disease’’; Haematologica 85(11 

Suppl), 81–85; Nov. 2000. 

AUTOIMMUNE CYTOPENIA 

Passweg, JR et al., Haematopoetic stem cell transplantation for refractory autoimmune cytopenia, British Journal of Haematology 125, : 49-: 55, June 2004. 

Rabusin M et al.; ‘‘Immunoablation followed by autologous hematopoietic stem cell 

infusion for the treatment of severe autoimmune disease’’; Haematologica 85(11 

Suppl), 81–85; Nov. 2000. 

SCLEROMYXEDEMA 

A.M. Feasel et al., ‘‘Complete remission of scleromyxedema following autologous 

stem cell transplantation,’’ Archives of Dermatology 13: , 10: 1-10: 2; Aug. 2001. 

SCLERODERMA 

Burt RK et al., ‘‘Induction of tolerance in autoimmune diseases by hematopoietic 

stem cell transplantation: getting closer to a cure?,’’ Blood 99, : 68-: 84, 1 February 2002. 

Burt, RK and Traynor, AE; ‘‘Hematopoietic Stem Cell Transplantation: A New Therapy for Autoimmune Disease’’; Stem Cellsl: , 366-3: 2; 1999. 

CROHN’S DISEASE 

Kreisel W et al., Complete remission of Crohn’s disease after high-dose 

cyclophosphamide and autologous stem cell transplantation, Bone Marrow Transplantation 32, 33: -340, 2003. 

Burt RK et al., ‘‘High-dose immune suppression and autologous hematopoietic stem 

cell transplantation in refractory Crohn disease,’’ Blood 101, 2064–2066, March 

2003. 

Rabusin M et al.; ‘‘Immunoablation followed by autologous hematopoietic stem cell 

infusion for the treatment of severe autoimmune disease’’; Haematologica 85(11 

Suppl), 81–85; Nov. 2000. 

Hawkey CJ et al.; ‘‘Stem cell transplantation for inflammatory bowel disease: practical and ethical issues’’; Gut 46, 869-8: 2; June 2000. 

BEHCET’S DISEASE 

Rabusin M et al.; ‘‘Immunoablation followed by autologous hematopoietic stem cell 

infusion for the treatment of severe autoimmune disease’’; Haematologica 85(11 

Suppl), 81–85; Nov. 2000. 

RHEUMATOID ARTHRITIS 

Burt RK et al., ‘‘Induction of tolerance in autoimmune diseases by hematopoietic 

stem cell transplantation: getting closer to a cure?,’’ Blood 99, : 68-: 84, 1 February 2002. 

Burt RK et al., ‘‘Induction of remission of severe and refractory rheumatoid arthritis 

by allogeneic mixed chimerism,’’ Arthritis & Rheumatism 50, 2466-24: 0, August 

2004. 

Verburg RJ et al.; ‘‘High-dose chemotherapy and autologous hematopoietic stem cell 

transplantation in patients with rheumatoid arthritis: results of an open study to 

assess feasibility, safety, and efficacy’’; Arthritis Rheum 44(4), : 54-: 60; April 

2001. 

Rabusin M et al.; ‘‘Immunoablation followed by autologous hematopoietic stem cell 

infusion for the treatment of severe autoimmune disease’’; Haematologica 85(11 

Suppl), 81–85; Nov. 2000. 

71

Burt, RK and Traynor, AE; ‘‘Hematopoietic Stem Cell Transplantation: A New Therapy for Autoimmune Disease’’; Stem Cellsl: , 366-3: 2; 1999. 

Burt RK et al.; ‘‘Hematopoietic stem cell transplantation of multiple sclerosis, rheumatoid arthritis, and systemic lupus erythematosus’’; Cancer Treat. Res. 101, 15: 

-184; 1999. 

Burt, RK et al., ‘‘Autologous hematopoietic stem cell transplantation in refractory 

rheumatoid arthritis: sustained response in two of four patients,’’ Arthritis & 

Rheumatology 42, 2281–2285, November, 1999. 

JUVENILE ARTHRITIS 

I M de Kleer et al., Autologous stem cell transplantation for refractory juvenile idiopathic arthritis: analysis of clinical effects, mortality, and transplant related morbidity, Ann Rheum Dis 63, 1318–1326, 2004. 

Rabusin M et al.; ‘‘Immunoablation followed by autologous hematopoietic stem cell 

infusion for the treatment of severe autoimmune disease’’; Haematologica 85(11 

Suppl), 81–85; Nov. 2000. 

Burt, RK and Traynor, AE; ‘‘Hematopoietic Stem Cell Transplantation: A New Therapy for Autoimmune Disease’’; Stem Cellsl: , 366-3: 2; 1999. 

MULTIPLE SCLEROSIS 

Saccardi R et al., Autologous HSCT for severe progressive multiple sclerosis in a 

multicenter trial: impact on disease activity and quality of life, Blood 105, 2601-

260: , 15 March 2005. 

Burt RK et al., ‘‘Induction of tolerance in autoimmune diseases by hematopoietic 

stem cell transplantation: getting closer to a cure?,’’ Blood 99, : 68-: 84, 1 February 2002. 

Mancardi GL et al.; ‘‘Autologous hematopoietic stem cell transplantation suppresses 

Gd-enhanced MRI activity in MS’’; Neurology 5: , 62–68; July 10, 2001. 

Rabusin M et al.; ‘‘Immunoablation followed by autologous hematopoietic stem cell 

infusion for the treatment of severe autoimmune disease’’; Haematologica 85(11 

Suppl), 81–85; Nov. 2000. 

Burt, RK and Traynor, AE; ′′Hematopoietic Stem Cell Transplantation: A New Therapy for Autoimmune Disease’’; Stem Cellsl: , 366-3: 2; 1999. 

Burt RK et al.; ‘‘Hematopoietic stem cell transplantation of multiple sclerosis, rheumatoid arthritis, and systemic lupus erythematosus’’; Cancer Treat. Res. 101, 15: 

-184; 1999. 

POLYCHONDRITIS 

Rosen O et al.; ‘‘Autologous stem-cell transplantation in refractory autoimmune diseases after in vivo immunoablation and ex vivo depletion of mononuclear cells’’; 

Arthritis res. 2, 32: -336; 2000. 

SYSTEMIC VASCULITIS 

Rabusin M et al.; ‘‘Immunoablation followed by autologous hematopoietic stem cell 

infusion for the treatment of severe autoimmune disease’’; Haematologica 85(11 

Suppl), 81–85; Nov. 2000. 

ALOPECIA UNIVERSAL 

Seifert B et al., Complete remission of alopecia universalis after allogeneic 

hematopoietic stem cell transplantion, Blood 105, 426-42: , 1 January 2005. 

BUERGER’S DISEASE 

Kim D-I et al., Angiogenesis facilitated by autologous whole bone marrow stem cell 

transplantation for Buerger’s disease, Stem Cells 24, 1194–1200, 2006. 

IMMUNODEFICIENCIES

SEVERE COMBINED IMMUNODEFICIENCY SYNDROME 

Grunebaum E et al., Bone marrow transplantation for severe combined immune deficiency, Journal of the American Medical Association 295, 508–518, 1 February 

2006. 

Cavazzana-Calvo M et al.; ‘‘Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease’’; Science 288, 669-6: 2; April 28, 2000. (NOTE: gene 

therapy using bone marrow adult stem cells as gene vehicle) 

X-LINKED LYMPHOPROLIFERATIVE SYNDROME and 

X-LINKED HYPERIMMUNOGLOBULIN M SYNDROME 

Banked unrelated umbilical cord blood was used to reconstitute the immune system 

in 2 brothers with X-linked lymphoproliferative syndrome and 1 boy with X-linked 

72

hyperimmunoglobulin-M syndrome. Two years after transplantation, all 3 patients 

have normal immune systems. These reports support the wider use of banked partially matched cord blood for transplantation in primary immunodeficiencies. 

Reference: Ziegner UH et al.; ‘‘Unrelated umbilical cord stem cell transplantation for 

X-linked immunodeficiencies’’; J Pediatr 138(4), 5: 0-5: 3; April 2001. 

Eight children with severe immunodeficiencies treated by adult bone marrow stem 

cell transplants. Six of eight showed relatively normal immune systems after 1 

year. 

Reference: Amrolia, P. et al., ‘‘Nonmyeloablative stem cell transplantation for congenital immunodeficiencies,’’ Blood 96, 1239–1246, Aug. 15, 2000. 

ANEMIAS AND OTHER BLOOD CONDITIONS

SICKLE CELL ANEMIA 

Klein A et al., Hematopoietic stem cell transplantation for severe sickle cell disease, 

Rev Med Brux. 2005;26 Spec no:Sp23-5. 

Adamkiewicz TV et al., Transplantation of unrelated placental blood cells in children with high-risk sickle cell disease, Bone Marrow Transplant. 34, 405–411, 

Sept 2004. 

Wu CJ et al., Molecular assessment of erythroid lineage chimerism following 

nonmyeloablative allogeneic stem cell transplantation, Exp Hematol. 31, 924–933, 

Oct 2003. 

Gore L. et al.; ‘‘Successful cord blood transplantation for sickle cell anemia from a 

sibling who is human leukocyte antigen-identical: implications for comprehensive 

care,’’ J Pediatr Hematol Oncol 22(5):43: -440; Sep–Oct 2000. 

Steen RG et al.; ‘‘Improved cerebrovascular patency following therapy in patients 

with sickle cell disease: initial results in 4 patients who received HLA-identical 

hematopoietic stem cell allografts’’; Ann Neurol 49(2), 222–229; Feb. 2001. 

Wethers DL; ‘‘Sickle cell disease in childhood: Part II. Diagnosis and treatment of 

major complications and recent advances in treatment’’; Am. Fam. Physician 62, 

1309–1314; Sept. 15, 2000. 

SIDEROBLASTIC ANEMIA 

Ayas M et al.; ‘‘Congenital sideroblastic anaemia successfully treated using 

allogeneic stem cell transplantation’’; Br J Haematol 113, 938–939; June 2001. 

Gonzalez MI et al.; ‘‘Allogeneic peripheral stem cell transplantation in a case of hereditary sideroblastic anaemia’’; British Journal of Haematology 109, 658–660; 

2000. 

APLASTIC ANEMIA 

Gurman G et al.; ‘‘Allogeneic peripheral blood stem cell transplantation for severe 

aplastic anemia’’; Ther Apher 5(1), 54-5: ; Feb. 2001. 

Kook H et al.; ‘‘Rubella-associated aplastic anemia treated by syngeneic stem cell 

transplantations’’; Am. J. Hematol. 64, 303–305; August 2000. 

RED CELL APLASIA 

Rabusin M et al.; ‘‘Immunoablation followed by autologous hematopoietic stem cell 

infusion for the treatment of severe autoimmune disease’’; Haematologica 85(11 

Suppl), 81–85; Nov. 2000. 

AMEGAKARYOCYTIC THROMBOCYTOPENIA 

Yesilipek et al.; ‘‘Peripheral stem cell transplantation in a child with 

amegakaryocytic thrombocytopenia’’; Bone Marrow Transplant 26, 5: 1-5: 2; Sept. 

2000. 

THALASSEMIA 

Tan PH et al., ‘‘Unrelated peripheral blood and cord blood hematopoietic stem cell 

transplants for thalassemia major,’’ Am J Hematol : 5, 209–212, April 2004. 

PRIMARY AMYLOIDOSIS 

Sezer 0 et al.; ‘‘Novel approaches to the treatment of primary amyloidosis’’; Exper 

Opin. Investig. Drugs 9, 2343–2350; Oct 2000. 

DIAMOND BLACKFAN ANEMIA 

Ostronoff M et al., ‘‘Successful nonmyeloablative bone marrow transplantation in a 

corticosteroid-resistant infant with Diamond-Blackfan anemia,’’ Bone Marrow 

Transplant. 34, 3: 1-3: 2, August 2004. 

73

FANCONI’S ANEMIA 

Bitan M et al., Fludarabine-based reduced intensity conditioning for stem cell transplantation of fanconi anemia patients from fully matched related and unrelated 

donors, Biol Blood Marrow Transplant. 12, : 12-: 18, July 2006. 

Tan PL et al., Successful engraftment without radiation after fludarabine-based regimen in Fanconi anemia patients undergoing genotypically identical donor 

hematopoietic cell transplantation, Pediatr Blood Cancer, 46, 630-636, May 1, 

2006. 

Kohli-Kumar M et al., ‘‘Haemopoietic stem/progenitor cell transplant in Fanconi 

anaemia using HLA-matched sibling umbilical cord blood cells,’’ British Journal 

of Haematology 85, 419–422, October 1993. 

CHRONIC EPSTEIN-BARR INFECTION 

Fujii N et al.; ‘‘Allogeneic peripheral blood stem cell transplantation for the treatment of chronic active epstein-barr virus infection’’; Bone Marrow Transplant 26, 

805–808; Oct. 2000. 

Okamura T et al.; ‘‘Blood stem-cell transplantation for chronic active Epstein-Barr 

virus with lymphoproliferation’’; Lancet 356, 223–224; July 2000. 

ADULT STEM CELLS—REPAIR/REPLACEMENT OF SOLID TISSUES

METABOLIC DISORDERS

HURLER’S SYNDROME 

Cox-Brinkman J et al., Haematopoietic cell transplantation (HCT) in combination 

with enzyme replacement therapy (ERT) in patients with Hurler syndrome, Bone 

Marrow Transplantation 38, 1: -21, 2006. 

Staba SL et al., Cord-blood transplants from unrelated donors in patients with 

Hurler’s syndrome,’’ New England Journal of Medicine 350, 1960–1969, 6 May 

2004. 

Koc ON et al., Allogeneic mesenchymal stem cell infusion for treatment of 

metachromatic leukodystrophy (MLD) and Hurler syndrome (MPS-IH), Bone Marrow Transplant 215–222; Aug 2002. 

OSTEOGENESIS IMPERFECTA 

Horwitz EM et al., ‘‘Isolated allogeneic bone marrow-derived mesenchymal cells 

engraft and stimulate growth in children with osteogenesis imperfecta: Implications for cell therapy of bone,’’ Proceedings of the National Academy of Sciences 

USA 99, 8932-893: ; 25 June 2002. 

Horwitz EM et al., ‘‘Clinical responses to bone marrow transplantation in children 

with severe osteogenesis imperfecta,’’ Blood 9: , 122: -1231; 1 March 2001. 

Horwitz, EM et al.; ‘‘Transplantability and therapeutic effects of bone marrow-derived mesenchymal cells in children with osteogenesis imperfecta’’; Nat. Med. 5, 

309–313; March 1999. 

KRABBE LEUKODYSTROPHY 

Escolar ML et al., ‘‘Transplantation of umbilical cord-blood in babies with infantile 

Krabbe’s disease,’’ New England Journal of Medicine 352, 2069–2081, 19 May 

2005. 

Krivit W et al., ‘‘Hematopoietic Stem-Cell Transplantation in Globoid-Cell 

Leukodystrophy,’’ New England Journal of Medicine 338, 1119-112: , Apr 16, 

1998. 

OSTEOPETROSIS 

Tsuji Y et al., Successful nonmyeloablative cord blood transplantation for an infant 

with malignant infantile osteopetrosis, J Pediatr Hematol Oncol. 2: , 495–498, 

Sept. 2005. 

Driessen GJ et al., Long-term outcome of haematopoietic stem cell transplantation 

in autosomal recessive osteopetrosis: an EBMT report, Bone Marrow Transplantation 32, 65: -663, October 2003. 

Schulz et al., HLA-haploidentical blood progenitor cell transplantation in 

osteopetrosis, Blood 99, 3458–3460, 1 May 2002. 

CEREBRAL X-LINKED ADRENOLEUKODYSTROPHY 

Peters C et al., Cerebral X-linked adrenoleukodystrophy: the international 

hematopoietic cell transplantation experience from 1982 to 1999, Blood 104, 881–

888, 1 August 2004. 

74

OCULAR

CORNEAL REGENERATION 

Inatomi T et al., Midterm results on ocular surface reconstruction using cultivated 

autologous oral mucosal epithelial transplantation, American Journal of Ophthalmology 141, 26: -2: 5, February 2006. 

Nishida K et al., Corneal reconstruction with tissue-engineered cell sheets composed 

of autologous oral mucosal epithelium, New England Journal of Medicine 351, 

118: -1196, 16 September 2004. 

Anderson DF et al.; ‘‘Amniotic Membrane Transplantation After the Primary Surgical Management of Band Keratopathy’’; Cornea 20(4), 354–361; May 2001. 

Anderson DF et al.; ‘‘Amniotic membrane transplantation for partial limbal stem 

cell deficiency’’; Br J Ophthalmol 85(5), 56: -5: 5; May 2001. 

Henderson TR et al.; ‘‘The long term outcome of limbal allografts: the search for surviving cells’’; Br J Ophthalmol 85(5), 604–609; May 2001. 

Daya SM, Ilari FA; ‘‘Living related conjuctival limbal allograft for the treatment of 

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Schwab IR et al.; ‘‘Successful transplantation of bioengineered tissue replacements 

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Tsai et al.; ‘‘Reconstruction of damaged corneas by transplantation of autologous 

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Tsubota K et al.; ‘‘Treatment of severe ocular-surface disorders with corneal 

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-1: 03; June 3, 1999. 

WOUNDS & INJURIES

LIMB GANGRENE 

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ischaemia by autologous transplantation of bone-marrow cells: a pilot study and 

a randomised controlled trial’’; Lancet 360, 42: -435; 10 August 2002. 

SURFACE WOUND HEALING 

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JAWBONE REPLACEMENT 

Warnke PH et al., Growth and transplantation of a custom vascularised bone graft 

in a man, Lancet 364, : 66-: : 0, 28 August 2004. 

SKULL BONE REPAIR 

Lendeckel S et al., Autologous stem cells (adipose) and fibrin glue used to treat 

widespread traumatic calvarial defects: case report, Journal of Cranio-Maxillofacial Surgery 32, 3: 0-3: 3, 2004. 

HEART DAMAGE

ACUTE HEART DAMAGE 

Joseph J et al., Safety and effectiveness of granulocyte-colony stimulating factor in 

mobilizing stem cells and improving cytokine profile in advanced chronic heart 

failure, American Journal of Cardiology 9: , 681–684, 1 March 2006. 

Blocklet D et al., Myocardial homing of nonmobilized peripheral-blood CD34+ cells 

after intracoronary injection, Stem Cells 24, 333–336, February 2006. 

Janssens S et al., Autologous bone marrow-derived stem-cell transfer in patients 

with ST-segment elevation myocardial infarction: double-blind, randomised controlled trial, Lancet 36: , 113–121, 14 January 2006. 

Patel AN et al., Surgical treatment for congestive heart failure with autologous 

adult stem cell transplantation: a prospective randomized study, Journal Thoracic 

Cardiovascular Surgery 130, 1631–1638, December 2005. 

Ince H et al., Preservation from left ventricular remodeling by front-integrated 

revascularization and stem cell liberation in evolving acute myocardial infarction 

by use of granulocyte-colony-stimulating factor (FIRSTLINE-AMI), Circulation

112, 309: -3106, 15 November 2005. 

Ince H et al., Prevention of left ventricular remodeling with granulocyte colony-stimulating after acute myocardial infarction, Circulation 112, I-: 3-I-80, 30 August 

2005. 

Bartunek J et al., Intracoronary injection of CD133-positive enriched bone marrow 

progenitor cells promotes cardiac recovery after recent myocardial infarction, Circulation 112, I-1: 8-I-183, 30 August 2005. 

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Dohmann HFR et al., Transendocardial autologous bone marrow mononuclear cell 

injection in ischemic heart failure, Circulation 112, 121–126, 26 July 2005. 

Wollert KC et al., ‘‘Intracoronary autologous bone-marrow cell transfer after myocardial infarction: the BOOST randomised controlled clinical trial,’’ Lancet 364, 141–

148, 10 July 2004. 

Britten MB et al., ‘‘Infarct remodeling after intracoronary progenitor cell treatment 

in patients with acute myocardial infarction’’; Circulation 108, 2212–2218; Nov 

2003. 

Perin EC et al.; ‘‘Transendocardial, autologous bone marrow cell transplantation for 

severe, chronic ischemic heart failure’’; Circulation 10: , r: 5-r83; published online 

May 2003. 

Stamm C et al.; ‘‘Autologous bone-marrow stem-cell transplantation for myocardial 

regeneration’’; The Lancet 361, 45–46; 4 January 2003. 

Tse H-F et al.; ‘‘Angiogenesis in ischaemic myocardium by intramyocardial 

autologous bone marrow mononuclear cell implantation’’; The Lancet 361, 4: -49; 

4 January 2003. 

Strauer BE et al.; ‘‘Repair of infarcted myocardium by autologous intracoronary 

mononuclear bone marrow cell transplantation in humans’’; Circulation 106, 

1913–1918; 8 October 2002. 

Strauer BE et al.; ‘‘Myocardial regeneration after intracoronary transplantation of 

human autologous stem cells following acute myocardial infarction’’; Dtsch Med 

Wochenschr 126, 932–938; Aug 24, 2001. 

Menasche ́ P et al. ‘‘Myoblast transplantation for heart failure.’’ Lancet 35: , 2: 9-280; 

Jan 2: , 2001. 

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182; March 2001. 

CHRONIC CORONARY ARTERY DISEASE 

Strauer BE et al., Regeneration of human infarcted heart muscle by intracoronary 

autologous bone marrow cell transplantation in chronic coronary artery disease, 

Journal of the American College of Cardiology 46, 1651–1658, 1 November 2005. 

NEURAL DEGENERATIVE DISEASES & INJURIES

STROKE

Shyu W-C et al., Granulocyte colony-stimulating factor for acute ischemic stroke: a 

randomized controlled trial, Canadian Medical Association Journal 1: 4, 92: -933, 

28 March 2006. 

Stilley CS et al., Changes in cognitive function after neuronal cell transplantation 

for basal ganglia stroke, Neurology 63, 1320–1322, October 2004. 

Meltzer CC et al.; ‘‘Serial [18F]Fluorodeoxyglucose Positron Emission Tomography 

after Human Neuronal Implantation for Stroke’’; Neurosurgery 49, 586–592; 2001. 

Kondziolka D et al.; ‘‘Transplantation of cultured human neuronal cells for patients 

with stroke’’; Neurology 55, 565–569; August 2000. 

PARKINSON’S DISEASE 

Using Direct Stimulation of Patients’ Endogenous Adult Neural Stem Cells 

Love S et al., Glial cell line-derived neurotrophic factor induces neuronal sprouting 

in human brain, Nature Medicine 11, : 03-: 04, July 2005. 

Slevin JT et al., Improvement of bilateral motor functions in patients with Parkinson disease through the unilateral intraputaminal infusion of glial cell line-derived neurotrophic factor, Journal of Neurosurgery 102, 216–222, February 2005. 

Gill SS et al.; ‘‘Direct brain infusion of glial cell line-derived neurotrophic factor in 

Parkinson disease’’; Nature Medicine 9, 589–595; May 2003 (published online 31 

March 2003). 

SPINAL CORD INJURY 

Lima C et al., Olfactory mucosa autografts in human spinal cord injury: A pilot clinical study, Journal of Spinal Cord Medicine 29, 191–203, July 2006. 

LIVER DISEASE

CHRONIC LIVER DISEASE 

Gordon MY et al., Characterisation and clinical application of human CD34+ stem/

progenitor cell populations mobilised into the blood by G-CSF, Stem Cells 24, 

1822–1830, July 2006; published online March 30, 2006. 

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LIVER CIRRHOSIS 

Terai S et al., Improved liver function in liver cirrhosis patients after autologous 

bone marrow cell fusion therapy, Stem Cells published online 15 June 2006; DOI: 

10.1634/stemcells.2005-0542. 

BLADDER DISEASE

END-STAGE BLADDER DISEASE 

Atala A et al., Tissue-engineered autologous bladders for patients needing 

cytoplasty, The Lancet 36: , 1241–1246, 15 April 2006. 

RESPONSE TO QUESTIONS OF SENATOR ENZI BY GEORGE Q. DALEY, M.D., PH.D. 

Question 1. Professor Daley, sometimes I wonder if we are being realistic about 

the short foreseeable advances from stem cell research, in terms of it always seems 

medical research is a slow endeavor. For instance, some adult stem cell therapies 

have been in commercial investigation for the past 10 years and not yet reached the 

market. My staff reports that there are 1,229 publicly available adult stem cell 

trials of which 614 are currently enrolling patients. Are there any embryonic stem 

cell therapies that are in early clinical trials now? Do you have a realistic best case 

thought when an embryonic stem cell therapy might be widely used? 

Answer 1. Medical research is a slow, methodical, step wise endeavor to ferret out 

the biological basis of disease, and to translate those basic insights into new forms 

of diagnosis or treatment. All new medical technologies take years, sometimes decades to realize their full clinical potential. 

Embryonic stem cell research will pay off both in the near term and the longterm. Near-term, scientists are already using embryonic stem cells to unravel the 

secrets of early human development and cell differentiation—to learn how the cells 

of the human embryo first become specialized into nerves, muscle, blood, and more. 

Such basic research is certain to yield insights into miscarriage and infertility, chromosomal abnormalities, intra-uterine growth defects, tissue generation and regeneration, and cancer. Drugs that are currently being used clinically are being tested 

in various in vitro assays that employ embryonic stem cells, and we might learn 

that existing drugs can stimulate stem cell function and encourage tissue repair. In 

the long-term, we hope that scientists will learn how to coax embryonic stem cells 

to become specific tissues for therapy—skin cells, blood cells, nerve cells and many 

others. 

It is impossible to predict with any certainty when embryonic stem cells will 

themselves serve as a source of specialized cells for cell replacement therapy. Geron, 

a leading biotechnology company that has pioneered embryonic stem cell research, 

has stated publicly that they wish to begin human trials of specialized tissues from 

human embryonic stem cells for the treatment of spinal cord injury within the next 

2 years. My best estimate is that products derived from embryonic stem cells will 

be tested in patients within the next 5 to 7 years, but that effective cell-based therapies cannot be expected for at least a decade or more. 

The history of biomedicine teaches us that most new forms of therapy take many 

years to evolve and bear fruit. This was true for the translation of recombinant DNA 

into new protein-based drugs (insulin, interferon, erythropoietin), for monoclonal 

antibodies, and I believe will be true for embryonic stem cell-based therapies.

Question 2. Professor Daley, the Administrations current stem cell policy does not 

prevent any embryonic stem cell research. Accordingly, States and private foundations are supporting some research. State funding alone is expected to add up to 

several billions of dollars of funding in the next few years. Accordingly, I noticed 

some of your work is supported by private foundations. It seems to me that many 

of these foundations that help fund disease research are wonderful drivers of innovation. Is there anything we can do in Congress to encourage foundations like those 

to be created? 

Answer 2. Unfortunately, the Administration’s current stem cell policy does indeed effectively prevent an enormous amount of embryonic stem cell research. Federal funds are essential to virtually every major biomedical research laboratory, and 

are used to purchase equipment and supplies and to pay scientists’ salaries. Because 

no Federal money can be used for any embryonic stem cell research that does not 

narrowly conform to the administration’s policy (that is, purely in vitro research on 

a small number of lines created prior to August 9, 2001), any so-called nonconforming ‘‘nonPresidential’’ research must be performed with entirely separate equipment and supplies by personnel whose salary comes from private sources. Because 

only a few laboratories and institutions have the resources to duplicate equipment, 

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space, supplies, and personnel, the vast majority of American scientists cannot and 

do not act on the creative experimental ideas they might have for working in nonconforming areas of research. The power of the purse is extremely strong, and 

serves to limit innovation and intellectual creativity. 

State funds and private foundations are not a solution for funding areas of research that are in the vital national scientific interest. For scientists outside of a 

few exclusive States like New Jersey, Connecticut, and California, no significant alternative funds exist. Thus the scientists of States like Wyoming and many others 

are largely excluded from new and exciting areas of embryonic stem cell science that 

fall outside the narrow funding guidelines of the administration policy. All but a few 

foundations have endowments large enough to have a substantive impact on biomedical research, and altogether will not be able to make up for the absence of 

funding through the National Institutes of Health.

Question 3. Professor Daley, as a physician-scientist, do you think we are doing 

enough to develop adequate numbers of physician scientists to fulfill the promise of 

stem cell research? Can you suggest any changes we might make to encourage more 

doctors to pursue innovative research like you are doing? 

Answer 3. The dark storm-clouds over the current NIH funding climate serve as 

the greatest hindrance to developing more physician-scientists. Physician-scientists 

must train for many years before achieving independence, and they depend upon 

Federal grant dollars to initiate their new research programs. The doubling of the 

NIH budget created an enormous new flow of research and allowed for the development of many new scientists. But given that the NIH budget is no longer even 

matching the rising costs due to inflation, everyone’s budgets are being cut across 

the board, and junior investigators are being hit the hardest. Moreover, the political 

controversy over stem cell research dissuades all but the most idealistic and motivated scientists from pursuing stem cell research.

Question 4. Dr. Daley, in your research efforts at the Boston Children’s Hospital, 

you are using embryonic stem cells to replace problematic genes in certain diseases, 

such as sickle cell disease and leukemia. In your estimation, how close are you to 

a breakthrough that will improve the health of patients with these diseases? 

Answer 4. Our research aims to combine gene therapy and cell therapy, so that 

patients with genetic diseases can be treated with their own genetically-repaired 

cells in a way that is safe and effective. We are working on technology platforms 

that could be applied to any one of dozens of bone marrow diseases, with sickle cell 

anemia, immune deficiency, and leukemia are but a few. We are working diligently 

in hopes of achieving breakthroughs that will help improve the health of my patients. No one knows for certain when breakthroughs will happen, but in my estimation, I expect to see such advances within my career, and hopefully within the 

next decade or two. Basic research is a long-term investment, but such investments 

have paid off handsomely for the United States. 

RESPONSE TO QUESTION OF SENATOR COCHRAN BY GEORGE Q. DALEY, M.D., PH.D. 

Question. I would address this question to any of our panel members today, has 

new scientific research emerged to support or negate the need for additional embryonic stem cell lines to further your research efforts? 

Answer. I would argue that no research has emerged that would negate the need 

for additional embryonic stem cell lines, as the many so-called ‘‘alternatives’’ to embryonic stem cells are not perfect substitutes. Ample evidence exists and has been 

published that many of the current NIH-approved ‘‘Presidential’’ embryonic stem 

cell lines develop genetic defects when cultured for prolonged periods. This fact 

alone argues that a new supply of lines is needed. Moreover, there have been many 

new lines established since the administration’s policy of August 9, 2001 was put 

in place, and many of these new lines have advantageous properties for stem cell 

research: for instance, they carry specific gene defects for human disease, making 

them extremely valuable for medical research, or they have been derived under improved, animal-free conditions that make them particularly favorable for clinical 

use.

REPSONSE TO QUESTION OF SENATOR ENZI BY LAUREN STANFORD

Question. I want to join the other Senators and thank you for coming here to testify. You are saluted for active participation in the political process and for raising 

money for diabetes research. I also wish you the best of luck in becoming a Senator. 

What advice would you have for other teenagers who have been diagnosed with Diabetes? 

78

Answer. I think I would tell other teens with diabetes two things. First, don’t lie 

to your parents about things like your blood sugars. If you feel like it’s all too much 

you are better off just telling them the truth so they can help you. I made that mistake 2 years ago of not telling them that I was sick of it and not giving myself the 

insulin I needed and I almost died. It’s better to just be honest and get some help. 

Second, I would tell them that their voices are important too. When we are little 

kids we all do the diabetes walks and speak up and all that, but it seems like as 

soon as a lot of us get to be teens we stop speaking out. It’s hard to be different 

when you are a teen, but I think people really listen to us. So keep speaking up 

about needing a cure and then you won’t have to be sick of diabetes anymore, once 

it is cured.

RESPONSE TO QUESTIONS OF SENATOR ENZI BY JOHN E. WAGNER, JR., M.D. 

Question 1. Professor Wagner, thank you for coming and I wanted to thank your 

patients that joined you today. It sounds like you are making great progress with 

adult stem cell research. I know you recognize embryonic stem cell research is important, but do you think an expansion of adult stem cell research will lead to more 

therapies in the next 10 years? Realistically, is there any particular treatments, 

other than the ones you are working on, that you are intrigued by and excited about 

seeing tried in a clinical setting? 

Answer 1. Without question, there are a number of adult stem cell therapies outside the ones I am working on that are either in the planning stage or in progress 

that bear close monitoring. For example, adult stem cell therapies are being planned 

for spinal cord injury, neurodegenerative disease, type I diabetes and vascular injury. Trials are underway evaluating adult stem cell therapies for acute and chronic 

heart failure, bone and joint cartilage repair, and acute brain injury. However, it 

must be unequivocally clear that these therapies are as yet unproven in terms of 

efficacy. Certainly, the field of adult stem cell research is extraordinarily exciting 

and deserves heightened funding in order to capitalize on its full potential. But, this 

in no way deters from the pressing need for greater embryonic stem cell research. 

ES cells offer opportunities that are either not possible with adult stem cells, such 

as the development of disease specific ES cell lines with which to identify pathological mechanisms sensitive to novel pharmacologic agents, or may later prove to be 

better than adult stem cells for specific diseases. It is possible, for instance, that 

ES cells have a greater capacity to make heart muscle than adult stem cells. Or, 

have a greater capacity to make islets for treatment of diabetes. Also, there may 

be circumstances that adult stem cells may be less prone to immune attack, making 

that a better source for other diseases. Based on work done at the University of 

Minnesota and elsewhere, I believe that we will see major breakthroughs in adult 

and embryonic stem cell research over the few years that will lead to an increasing 

number of clinical trials.

Question 2. Professor Wagner, we all saw Professor Atala announce his breakthrough regarding amniotic stem cells and that advance got an awful lot of press. 

In addition to your own work, should we expect other announcements that also have 

potential to generate new methods of generating stem cells in the not to distant future? 

Answer 2. Professor Atala’s report several weeks ago did get a great deal of press. 

I know of other research here at the University of Minnesota and elsewhere specifically evaluating other potential stem cell sources as well as improved methodologies 

for isolating and expanding stem cell populations from umbilical cord blood, the umbilical cord itself and various adult tissues. However, it is important to recognize 

that we do not yet know how the various stem cell populations compare with each 

other or whether one source or isolation/expansion methodology offers a true advantage in terms of treating patients with disease. All these announcements are exciting but be very careful about what is in the press as it may not accurately reflect 

what is known versus what is pure speculation. As a scientist, I am asked to speculate as to the meaning of a particular discovery, but it’s just that—speculation. 

Sometimes, the press may interpret speculation as fact inappropriately. In my own 

experience, some have taken our own discoveries on adult stem cells and used them 

to nullify the critical importance of ES cells. 

There is purposeful misinformation that scientists, focused on ‘‘finding truth,’’ 

often find themselves too ill-equipped to respond to. So, while these new discoveries 

with adult stem cells are promising, such as that reported by Professor Atala, it is 

way too premature to suggest that they replace ES cells in our collective efforts to 

reduce suffering and disease.

79

Question 3. Professor Wagner, I’d like to ask you the same question I asked Professor Daley, it seems like in order to deliver on the promise of stem cell research, 

we need more physician investigators like yourself. Can you suggest anything Congress should consider to encourage physicians to pursue the development of innovative therapies like you have? 

Answer 3. It should be clear that we are all devoted to reducing suffering and 

minimizing the impact of debilitating diseases, such as cancer, diabetes, and heart 

failure. The list of diseases is long and the number of affected people asking for a 

chance to live healthy, productive lives, is enormous. I know that you and others 

genuinely want to help us to do just that—the goals are clear and the rewards will 

be immeasurable. 

First, allow us to pursue all promising avenues. We did this with cancer and we 

did this with AIDS. Today, survival rates are the highest ever. Is there more work 

to do—obviously, yes. But, the achievements to date can be attributed to the 

breadth of the attack. Rather than limit science, the science was embraced. Rather 

than limit funding, the funding was dramatically augmented. 

In my opinion, the stem cell and its impact upon science and medicine will be revolutionary. It promises to fundamentally change the way we understand disease and 

the practice of medicine. Just as the Nation dreamed about the limits of space five 

decades ago, we dream about the limits of stem cells today. Could President Kennedy have imagined the gains we have made in telecommunications when he proposed the development of NASA? Could he know that there would be a phone at 

every ear and GPS device in every car? No. But he did know that space was worth 

the investment because the returns 1 day could be spectacular.

Question 4. What can Congress do to encourage physicians to develop innovative 

stem cell therapies? 

Answer 4. As a physician, I would start by eliminating the barriers and provide 

incentives for collaborations between basic scientists and clinical investigators and 

invest in such translational research. Piecemeal approaches will beget mediocre 

progress. Substantial and strategically focused efforts, free of politics, and supportive of both adult and ES cell basic, translational and clinical research are required. Right now, the effort is diluted between industry and the various institutes 

of the NIH. Just as Congress needs to be united, so does the NIH. The potential 

impact of this research is unprecedented and for that reason, a focused effort is required. Whether this should manifest itself as a new Institute for Stem Cell Research or as an additional line item allocation, Congress needs to address the gaps 

in the stem cell translational pipeline. 

RESPONSE TO QUESTION OF SENATOR COCHRAN BY JOHN E. WAGNER, JR., M.D. 

Question. I would address this question to any of our panel members today, has 

new scientific research emerged to support or negate the need for additional embryonic stem cell lines to further your research efforts? 

Answer. Without a doubt, all stem cell science benefits from the work we do in 

any one area. We will never know the full potential of adult stem cells unless we 

have the chance to compare them side by side with embryonic stem cells. Just as 

we have tested the limits and possibilities of bone marrow versus cord blood for 

transplantation, we must allow the full vetting of the scientific potential of this 

science. Indeed, while embryonic stem cells are clearly the ‘‘gold standard’’ against 

which all other stem cell sources are compared, today’s researchers’ hands are tied 

as the population of embryonic stem cells available for Federal funding are less than 

optimal either for basic research or clinical testing. We need to manufacture new 

cell lines that will (1) broaden genetic, racial and ethnic diversity, (2) be free of animal tissues, and (3) have a defined history both in terms of proven gamete donor 

consents as well as reagent exposures and number of passages. Embryonic stem cell 

lines and its derivates are more than simple sources of tissues for repair; they provide us with an unprecedented resource for understanding mechanisms of disease 

and development of targeted treatment strategies to modify or prevent disease. We 

have the knowledge and know-how now. While there may be political reasons, there 

is absolutely no objective, scientific research that negates the need for more embryonic stem cell research and the development of new cell lines. Without question, the 

‘‘Presidential’’ stem cell lines are suboptimal. Acquisition of genetic defects after 

years of passaging in culture and their derivation on murine feeder layers are two 

reasons that make them suboptimal. Yet, these are the only cell lines for which Federal dollars can be used. Without question, no adult stem cell population, outside 

the context of bone marrow transplantation, has any proven efficacy. Certainly, 

work is on-going to determine the place of adult stem cells. It is important to underVerDate 0ct 09 2002 12:55 Dec 17, 2007 Jkt 000000 PO 00000 Frm 00083 Fmt 6633 Sfmt 6621 S:\DOCS\32808.TXT SLABOR1 PsN: DENISE
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stand that every discovery with embryonic stem cells has only enhanced our own 

work with adult stem cells. These facts are uncontestable! 

As proposed by some, why not first determine the true capacity of adult stem cells 

and potentially eliminate the ethical issues associated with embryonic stem cells? 

The argument has three major drawbacks: (1) without ES research, how will we 

ever determine how far adult stem cells can go in the treatment of disease?; (2) deleterious impact upon our efficiency in moving stem cell therapies forward; and (3) 

without the unbiased pursuit of knowledge, how can science move forward?

[Whereupon, at 11:41 a.m., the hearing was adjourned.] 

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