Stem CELLS and the FUTURE OF REGENERATIVE MEDICINE

Committee on the Biological and Biomedical Applications of Stem Cell Research

Board on Life Sciences National Research Council

Board on Neuroscience and Behavioral Health Institute of Medicine

NATIONAL ACADEMY PRESS
Washington, D.C.



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Stem Cells and the Future of Regenerative Medicine Stem CELLS and the FUTURE OF REGENERATIVE MEDICINE Committee on the Biological and Biomedical Applications of Stem Cell Research Board on Life Sciences National Research Council Board on Neuroscience and Behavioral Health Institute of Medicine NATIONAL ACADEMY PRESS Washington, D.C.

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Stem Cells and the Future of Regenerative Medicine NATIONAL ACADEMY PRESS 2101 Constitution Avenue, N.W. Washington, DC 20418 NOTICE: The project that is the subject of this report was approved by the Governing Board of the National Research Council, whose members are drawn from the councils of the National Academy of Sciences, the National Academy of Engineering, and the Institute of Medicine. The members of the committee responsible for the report were chosen for their special competencies and with regard for appropriate balance. This study was supported by the National Research Council Fund and by the Ellison Medical Foundation under Agreement no. NI-CW-0007-01. Any opinions, findings, conclusions, or recommendations expressed in this publication are those of the authors and do not necessarily reflect the views of the organizations or agencies that provided support for the project. Library of Congress Cataloging-in-Publication Data Stem cells and the future of regenerative medicine / Committee on the Biological and Biomedical Applications of Stem Cell Research, Commission on Life Sciences National Research Council. p. cm. Includes bibliographical references and index. ISBN 0-309-07630-7 1. Stem cells—Research—Government policy—United States. I. National Research Council (U.S.). Committee on the Biological and Biomedical Applications of Stem Cell Research. QH587 .S726 2001 571.8'35—dc21 2001006360 Cover: Background image courtesy of Musee National du Chateau de Malmaison, Rueil-Malmaison/Lauros-Giraudon, Paris/SuperStock; stem cell photo courtesy of James Thomson Laboratory, University of Wisconsin Board of Regents. Additional copies of this report are available from National Academy Press, 2101 Constitution Avenue, NW, Lockbox 285, Washington, DC 20055; (800) 624-6242 or (202) 334-3313 (in the Washington metropolitan area); Internet, http://www.nap.edu Printed in the United States of America. Copyright 2002 by the National Academy of Sciences. All rights reserved.

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Stem Cells and the Future of Regenerative Medicine THE NATIONAL ACADEMIES National Academy of Sciences National Academy of Engineering Institute of Medicine National Research Council The National Academy of Sciences is a private, nonprofit, self-perpetuating society of distinguished scholars engaged in scientific and engineering research, dedicated to the furtherance of science and technology and to their use for the general welfare. Upon the authority of the charter granted to it by the Congress in 1863, the Academy has a mandate that requires it to advise the federal government on scientific and technical matters. Dr. Bruce M. Alberts is president of the National Academy of Sciences. The National Academy of Engineering was established in 1964, under the charter of the National Academy of Sciences, as a parallel organization of outstanding engineers. It is autonomous in its administration and in the selection of its members, sharing with the National Academy of Sciences the responsibility for advising the federal government. The National Academy of Engineering also sponsors engineering programs aimed at meeting national needs, encourages education and research, and recognizes the superior achievements of engineers. Dr. Wm. A. Wulf is president of the National Academy of Engineering. The Institute of Medicine was established in 1970 by the National Academy of Sciences to secure the services of eminent members of appropriate professions in the examination of policy matters pertaining to the health of the public. The Institute acts under the responsibility given to the National Academy of Sciences by its congressional charter to be an adviser to the federal government and, upon its own initiative, to identify issues of medical care, research, and education. Dr. Kenneth I. Shine is president of the Institute of Medicine. The National Research Council was organized by the National Academy of Sciences in 1916 to associate the broad community of science and technology with the Academy’s purposes of furthering knowledge and advising the federal government. Functioning in accordance with general policies determined by the Academy, the Council has become the principal operating agency of both the National Academy of Sciences and the National Academy of Engineering in providing services to the government, the public, and the scientific and engineering communities. The Council is administered jointly by both Academies and the Institute of Medicine. Dr. Bruce M. Alberts and Dr. Wm. A. Wulf are chairman and vice chairman, respectively, of the National Research Council.

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Stem Cells and the Future of Regenerative Medicine COMMITTEE ON THE BIOLOGICAL AND BIOMEDICAL APPLICATIONS OF STEM CELL RESEARCH BERT VOGELSTEIN (Chair), Johns Hopkins Oncology Center, Baltimore, and Howard Hughes Medical Institute BARRY R. BLOOM, Harvard School of Public Health, Cambridge, Massachusetts COREY S. GOODMAN, University of California, Berkeley, and Howard Hughes Medical Institute PATRICIA A. KING, Georgetown University Law Center, Washington, D.C. GUY M. MCKHANN, Johns Hopkins University School of Medicine, Baltimore MYRON L. WEISFELDT, Columbia University College of Physicians and Surgeons, New York KATHLEEN R. MERIKANGAS (liaison, Board on Neuroscience and Behavioral Health), Yale University, New Haven, Connecticut Project Staff FRANCES E. SHARPLES, Director, Board on Life Sciences TERRY C. PELLMAR, Director, Board on Neuroscience and Behavioral Health ROBIN A. SCHOEN, Program Officer JANET E. JOY, Senior Program Officer BRIDGET K. B. AVILA, Senior Project Assistant LAURA T. HOLLIDAY, Research Assistant DEREK M. SWEATT, Research Assistant NORMAN GROSSBLATT, Senior Editor

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Stem Cells and the Future of Regenerative Medicine BOARD ON LIFE SCIENCES NATIONAL RESEARCH COUNCIL COREY S. GOODMAN (Chair), University of California, Berkeley MICHAEL T. CLEGG, University of California, Riverside DAVID S. EISENBERG, University of California, Los Angeles DAVID J. GALAS, Keck Graduate Institute of Applied Life Science, Claremont, California BARBARA GASTEL, Texas A&M University, College Station JAMES M. GENTILE, Hope College, Holland, Michigan DAVID V. GOEDDEL, Tularik, Inc., South San Francisco, California ELLIOT M. MEYEROWITZ, California Institute of Technology, Pasadena ROBERT T. PAINE, University of Washington, Seattle STUART L. PIMM, Columbia University, New York JOAN B. ROSE, University of South Florida, St. Petersburg GERALD M. RUBIN, Howard Hughes Medical Institute, Chevy Chase, Maryland RONALD R. SEDEROFF, North Carolina State University, Raleigh ROBERT R. SOKAL, State University of New York, Stony Brook SHIRLEY M. TILGHMAN, Princeton University, New Jersey RAYMOND L. WHITE, DNA Sciences, Inc., Fremont, California Staff FRANCES E. SHARPLES, Director JENNIFER KUZMA, Senior Program Officer KERRY A. BRENNER, Program Officer JOAN G. ESNAYRA, Program Officer ROBIN A. SCHOEN, Program Officer MARILEE K. SHELTON, Program Officer ROBERT T. YUAN, Program Officer LAURA T. HOLLIDAY, Research Assistant DEREK M. SWEATT, Research Assistant BRIDGET K.B. AVILA, Senior Project Assistant DENISE D. GROSSHANS, Project Assistant

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Stem Cells and the Future of Regenerative Medicine BOARD ON NEUROSCIENCE AND BEHAVIORAL HEALTH INSTITUTE OF MEDICINE ANN M. GRAYBIEL (Chair), Massachusetts Institute of Technology, Cambridge KENNETH B. WELLS (Vice-Chair), Neuropsychiatric Institute, University of California, Los Angeles NANCY E. ADLER, University of California, San Francisco RICHARD J. BONNIE, University of Virginia School of Law, Charlottesville WILLIAM E. BUNNEY, University of California, Irvine RICHARD G. FRANK, Harvard Medical School, Boston, Massachusetts JEROME KAGAN, Harvard University, Cambridge, Massachusetts HERBERT D. KLEBER, Columbia University and New York State Psychiatric Institute, New York BEVERLY B. LONG, World Federation for Mental Health, Atlanta, Georgia KATHLEEN R. MERIKANGAS, Yale University, New Haven, Connecticut STEVEN M. MIRIN, American Psychiatric Association, Washington, D.C. STEVEN M. PAUL, Lilly Research Laboratories, Indianapolis, Indiana DAVID REISS, George Washington University Medical Center, Washington, D.C. RHONDA J. ROBINSON-BEALE, Blue Cross/Blue Shield of Michigan, Southfield STEPHEN WAXMAN, Yale Medical School, New Haven, Connecticut NANCY S. WEXLER, Columbia University, New York ANNE B. YOUNG, Massachusetts General Hospital, Boston Staff TERRY C. PELLMAR, Director SARA K. GOLDSMITH, Senior Program Officer JANET E. JOY, Senior Program Officer ALLISON L. FRIEDMAN, Research Assistant LORA K. TAYLOR, Administrative Assistant ALLISON M. PANZER, Project Assistant

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Stem Cells and the Future of Regenerative Medicine Acknowledgments This report is the product of many individuals. First, we would like to thank all the speakers who attended our workshop, Stem Cells and the Future of Regenerative Medicine, on June 22, 2001. Without the input of each of these speakers, this report would not have been possible. Iqbal Ahmad, University of Nebraska Medical Center George Annas, Boston University Schools of Medicine and Public Health Ernest Beutler, Scripps Research Institute Kevin FitzGerald, Georgetown University Fred Gage, Salk Institute Margaret Goodell, Taylor College of Medicine Marcus Grompe, Oregon Health Sciences University Ihor Lemischka, Princeton University Olle Lindvall, Lund University Ron McKay, National Institute of Neurological Disorders and Stroke Thomas Okarma, Geron Corporation David Prentice, Indiana State University Arti Rai, University of Pennsylvania School of Law Jay Siegel, Food and Drug Administration James Thomson, University of Wisconsin LeRoy Walters, Georgetown University Irving Weissman, Stanford University

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Stem Cells and the Future of Regenerative Medicine Second, this report has been reviewed in draft form by individuals chosen for their diverse perspectives and technical expertise, in accordance with procedures approved by the NRC’s Report Review Committee. The purpose of this independent review is to provide candid and critical comments that will assist the institution in making its published report as sound as possible and to ensure that the report meets institutional standards for objectivity, evidence, and responsiveness to the study charge. The review comments and draft manuscript remain confidential to protect the integrity of the deliberative process. We wish to thank the following individuals for their review of this report: Fred Alt, Howard Hughes Medical Institute, Harvard Medical School Fred Appelbaum, Fred Hutchinson Cancer Research Center Daniel Callahan, The Hastings Center R. Alta Charo, University of Wisconsin Law School Carolyn Compton, McGill University William Danforth, Washington University Neal First, University of Wisconsin Barbara Gastel, Texas A&M University John Gerhart, University of California, Berkeley Paul Gilman, Celera Genomics Micheline Mathews-Roth, Harvard Medical School Martin Raff, University College London Nathan Rosenberg, Stanford University Evan Snyder, Boston Children’s Hospital Virginia Weldon, Monsanto Company Although the reviewers listed above have provided many constructive comments and suggestions, they were not asked to endorse the conclusions or recommendations nor did they see the final draft of the report before its release. The review of this report was overseen by Ronald Estabrook of the University of Texas Southwestern Medical Center and Floyd Bloom of the Scripps Research Institute. Appointed by the

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Stem Cells and the Future of Regenerative Medicine National Research Council, they were responsible for making certain that an independent examination of this report was carried out in accordance with institutional procedures and that all review comments were carefully considered. Responsibility for the final content of this report rests entirely with the authoring committee and the institution.

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Stem Cells and the Future of Regenerative Medicine Preface Stem cell research has the potential to affect the lives of millions of people in the United States and around the world. This research is now regularly front-page news because of the controversy surrounding the derivation of stem cells from human embryos. Realizing the promise of stem cells for yielding new medical therapies will require us to grapple with more than just scientific uncertainties. The stem cell debate has led scientists and nonscientists alike to contemplate profound issues, such as who we are and what makes us human beings. The excitement and controversy surrounding stem cells caused the National Research Council’s Board on Life Sciences and the Institute of Medicine’s Board on Neuroscience and Behavioral Health to recommend that the National Academies sponsor a workshop to assess the scientific and therapeutic value of stem cells. The presidents of the National Academies agreed and provided most of the funding that supported the production of this report. The Ellison Foundation provided additional funding. In a collaboration of the two boards, the Committee on the Biological and Biomedical Applications of Stem Cell Research was formed. The persons appointed to serve on the committee have a wealth of expertise in the basic and clinical biomedical sciences but do not themselves perform stem cell

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Stem Cells and the Future of Regenerative Medicine research. The latter characteristic was intended to ensure that none of the committee members had a vested interest in any form of stem cell research. Expertise represented on the committee includes molecular biology, immunology, cell biology, cardiology, hematology, neurosciences, developmental biology, infectious disease, cancer, and bioethics, all of which are integrally related to stem cell research and its potential for developing tissue-replacement therapies that will restore lost function in damaged organs. At the committee’s workshop, held on June 22, 2001, scientists, philosophers, ethicists, and legal experts presented their views in two general categories. First, leading scientific investigators addressed the following scientific questions: What are stem cells? What are their sources, and what biological differences exist among cells of different origins? How do these differences translate into advantages or disadvantages for research and medical applications? What is the potential of stem cells for regenerative medicine, and what obstacles must be overcome to make them useful for new medical therapies? Second, experts in philosophy, law, and ethics presented a variety of ethical and other arguments relevant to public-policy considerations on stem cells. Audio files of the speakers’ presentations are available until December 31, 2002, at the workshop Web site: www.nationalacademies.org/stemcells. This report presents the committee’s findings and recommendations. It is based on careful consideration of information presented at the workshop and on data and opinions found in the scientific and other scholarly literature. The committee is extremely respectful of all perspectives in this debate and has taken them into account in forming its recommendations. I wish to thank all the members of the committee for their valuable contributions and especially for their insights into both the scientific and the societal issues. In particular, Corey Goodman, chair of the Board on Life Sciences, was responsible for much of the initial impetus for the workshop. I also wish to acknowledge the staff of the National Research Council (Robin Schoen, Bridget Avila, and Fran Sharples) and the

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Stem Cells and the Future of Regenerative Medicine Institute of Medicine (Janet Joy and Terry Pellmar) for their thorough, thoughtful, and efficient assistance with all aspects of the workshop and report preparation. This report would have been impossible without them. Bert Vogelstein, Chair Committee on the Biological and Biomedical Applications of Stem Cell Research

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Stem Cells and the Future of Regenerative Medicine Contents     EXECUTIVE SUMMARY   1 1   PROJECT OVERVIEW AND DEFINITIONS   7 2   ADULT STEM CELLS   19 3   EMBRYONIC STEM CELLS   31 4   OPPORTUNITIES FOR AND BARRIERS TO PROGRESS IN STEM CELL RESEARCH FOR REGENERATIVE MEDICINE   41 5   FINDINGS AND RECOMMENDATIONS   55     REFERENCES   61     GLOSSARY   67     APPENDIXES         A Committee Biographies   73     B Workshop Agenda and Speaker Biographies   79     INDEX   89

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Stem Cells and the Future of Regenerative Medicine Stem CELLS and the FUTURE OF REGENERATIVE MEDICINE

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