STEM CELL
THERAPIES
Opportunities for Ensuring the Quality
and Safety of Clinical Offerings
SUMMARY OF A JOINT WORKSHOP by the Institute of Medicine, |
Adam C. Berger, Sarah H. Beachy, and Steve Olson, Rapporteurs
Board on Health Sciences Policy
Institute of Medicine
Board on Life Sciences
Division on Earth and Life Studies
INSTITUTE OF MEDICINE AND
NATIONAL RESEARCH COUNCIL
OF THE NATIONAL ACADEMIES
THE NATIONAL ACADEMIES PRESS
Washington, D.C.
THE NATIONAL ACADEMIES PRESS • 500 Fifth Street, NW • Washington, DC 20001
NOTICE: The workshop that is the subject of this workshop summary 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.
This project was supported by contracts between the National Academy of Sciences and the Alliance for Regenerative Medicine (unnumbered contract); California Institute for Regenerative Medicine (unnumbered contract); The Ellison Medical Foundation (Contract No. OP-CW-0405-13); Howard Hughes Medical Institute (unnumbered contract); International Society for Stem Cell Research (unnumbered contract); and U.K. Academy of Medical Sciences (unnumbered contract). The project was also sponsored by the Presidents’ Committee Funds of the National Academy of Sciences, the National Academy of Engineering, and the Institute of Medicine. The views presented in this publication do not necessarily reflect the views of the organizations or agencies that provided support for the activity.
International Standard Book Number-13: 978-0-309-30300-2
International Standard Book Number-10: 0-309-30300-1
Additional copies of this report are available for sale from the National Academies Press, 500 Fifth Street, NW, Keck 360, Washington, DC 20001; (800) 624-6242 or (202) 334-3313; http://www.nap.edu.
For more information about the Institute of Medicine, visit the IOM home page at: www.iom.edu. For more information about the National Academy of Sciences, visit the NAS home page at: http://www.nasonline.org.
Copyright 2014 by the National Academy of Sciences. All rights reserved.
Printed in the United States of America
COVER: Fluorescence microscopy of pluripotent mouse stem cells undergoing differentiation into a variety of cell lineages. Photo credit: Pablo Perez-Pinera, Jonathan Brunger, Farshid Guilak, and Charles Gersbach, Duke University.
Suggested citation: IOM (Institute of Medicine) and NAS (National Academy of Sciences). 2014. Stem cell therapies: Opportunities for ensuring the quality and safety of clinical offerings. Washington, DC: The National Academies Press.
THE NATIONAL ACADEMIES
Advisers to the Nation on Science, Engineering, and Medicine
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. Ralph J. Cicerone 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. C. D. Mote, Jr., 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. Harvey V. Fineberg 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. Ralph J. Cicerone and Dr. C. D. Mote, Jr., are chair and vice chair, respectively, of the National Research Council.
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PLANNING COMMITTEE1
R. ALTA CHARO (Chair), Warren P. Knowles Professor of Law and Bioethics, School of Law and Department of Medical History and Bioethics, School of Medicine and Public Health, University of Wisconsin–Madison
ROBIN ALI, Professor of Human Molecular Genetics, Institute of Ophthalmology, University College London, UK
I. GLENN COHEN, Professor of Law and Co-Director of the Petrie–Flom Center for Health Law Policy, Biotechnology and Bioethics, Harvard Law School, Cambridge, MA
ELLEN G. FEIGAL, Senior Vice President, Research and Development, California Institute for Regenerative Medicine, San Francisco, CA
FRED H. GAGE, Vi and John Adler Professor, Laboratory of Genetics, Salk Institute for Biological Studies, La Jolla, CA
LAWRENCE S. B. GOLDSTEIN, Distinguished Professor and Director of the University of California, San Diego, Stem Cell Program, Department of Cellular and Molecular Medicine, University of California, San Diego, School of Medicine, La Jolla, CA
GEOFF MACKAY, Chief Executive Officer, Organogenesis, Inc., Canton, MA
AMY COMSTOCK RICK, Chief Executive Officer, Parkinson’s Action Network, Washington, DC
IRVING L. WEISSMAN, Director, Institute of Stem Cell Biology and Regenerative Medicine; Director, Ludwig Center at Stanford; Professor, Departments of Pathology and Developmental Biology and Neurosurgery, Stanford University School of Medicine, Stanford, CA
Institute of Medicine and National Research Council Staff
ADAM C. BERGER, Project Director
KEEGAN SAWYER, Program Officer
SARAH H. BEACHY, Associate Program Officer
Y. CRYSTI PARK, Senior Program Assistant
MEREDITH L. HACKMANN, Senior Program Assistant (from January 2014)
__________________
1Planning committees are solely responsible for organizing the workshop, identifying topics, and choosing speakers. The responsibility for the published workshop summary rests with the workshop rapporteurs and the institution.
International Society for Stem Cell Research Staff
NANCY WITTY, Executive Director
HEATHER ROOKE, Scientific Director
BOARD ON HEALTH SCIENCES POLICY
JEFFREY KAHN (Chair), Levi Professor of Bioethics and Public Policy, Berman Institute of Bioethics, Johns Hopkins University, Baltimore, MD
ELI ADASHI, Professor of Medical Science, The Warren Alpert Medical School, Brown University, Providence, RI
WYLIE BURKE, Professor and Chair, Department of Bioethics and Humanities, University of Washington, Seattle
ROBERT M. CALIFF, Director, Duke Translational Medicine Institute; Professor of Medicine, Vice Chancellor for Clinical and Translational Research, Duke University Medical Center, Durham, NC
KATHLEEN A. DRACUP, Professor, School of Nursing, University of California, San Francisco
MICHAEL EHLERS, Senior Vice President, Chief Scientific Officer of Neuroscience, Pfizer Inc., Cambridge, MA
NAOMI L. GERBER, University Professor, Center for the Study of Chronic Illness and Disability, George Mason University, Fairfax, VA
LEWIS R. GOLDFRANK, Herbert W. Adams Professor and Chair, Department of Emergency Medicine, New York University School of Medicine; Director of Emergency Medicine, Bellevue Hospital Center and New York University Langone Medical Center
STEVEN E. HYMAN, Director, Stanley Center, Broad Insitute of Harvard University and the Massachusetts Institute of Technology, Cambridge, MA
HARRY T. ORR, Director, Institute of Human Genetics; Tulloch Professor of Genetics, Department of Laboratory Medicine and Pathology, University of Minnesota, Minneapolis
SHARON TERRY, President and Chief Executive Officer, Genetic Alliance, Washington, DC
REED V. TUCKSON, Managing Director, Tuckson Health Connections, LLC, Edina, MN
CLYDE YANCY, Magerstadt Professor of Medicine and Chief, Division of Cardiology, Northwestern University Feinberg School of Medicine; Associate Director, Bluhm Cardiovascular Insitute, Northwestern Memorial Hospital, Chicago, IL
Board on Health Sciences Policy Staff
ANDREW M. POPE, Director
ADAM C. BERGER, Senior Program Officer
SARAH H. BEACHY, Associate Program Officer
Y. CRYSTI PARK, Senior Program Assistant
MEREDITH L. HACKMANN, Senior Program Assistant (from January 2014)
DONNA RANDALL, Administrative Assistant
BOARD ON LIFE SCIENCES
JAMES P. COLLINS (Chair), Virginia M. Ullman Professor of Natural History and the Environment, Arizona State University, Tempe
ENRIQUETA C. BOND, Former President, Burroughs Wellcome Fund, Marshall, VA
ROGER D. CONE, Professor and Chairman, Department of Molecular Physiology and Biophysics; Director, Vanderbilt Institute for Obesity and Metabolism; Vanderbilt University Medical Center, Nashville, TN
SEAN EDDY, Group Leader, Howard Hughes Medical Institute, Janelia Farm Research Campus, Ashburn, VA
SARAH C. R. ELGIN, Viktor Hamburger Professor of Arts & Sciences, Department of Biology, Washington University, St. Louis, MO
DAVID R. FRANZ, Former Commander, U.S. Army Medical Research Institute of Infectious Diseases, Frederick, MD
LOUIS J. GROSS, James R. Cox and Alvan and Sally Beaman Distinguished Professor of Ecology and Evolutionary Biology and Mathematics; Director, National Institute for Mathematical and Biological Synthesis; Director, Institute for Environmental Modeling; University of Tennessee, Knoxville
ELIZABETH HEITMAN, Associate Professor of Medicine and Anesthesiology, Vanderbilt University Medical Center, Nashville, TN
JOHN G. HILDEBRAND, Regents Professor, University of Arizona, Tucson
RICHARD A. JOHNSON, Retired Partner, Arnold & Porter, LLC, Washington, DC
JUDITH KIMBLE, Vilas Professor, Howard Hughes Medical Institute Investigator, University of Wisconsin–Madison
CATO T. LAURENCIN, University Professor, University of Connecticut Health Center, Farmington
ALAN I. LESHNER, Chief Executive Officer, American Association for the Advancement of Science, Washington, DC
KAREN E. NELSON, President, J. Craig Venter Institute, Rockville, MD
ROBERT M. NEREM, Parker H. Petit Distinguished Chair for Engineering in Medicine; Institute Professor Emeritus, Georgia Institute of Technology, Atlanta
CAMILLE PARMESAN, Professor, University of Texas, Austin; National Marine Aquarium Chair, Plymouth University, Plymouth, UK
ALISON G. POWER, Professor, Cornell University, Ithaca, NY
MARGARET RILEY, Professor, University of Massachusetts; President and Founder, Massachusetts Academy of Sciences, Amherst
JANIS C. WEEKS, Professor, University of Oregon, Eugene
MARY WOOLLEY, President, Research!America, Alexandria, VA
Board on Life Sciences Staff
FRANCES E. SHARPLES, Director
KEEGAN SAWYER, Program Officer
Reviewers
This workshop summary has been reviewed in draft form by individuals chosen for their diverse perspectives and technical expertise, in accordance with procedures approved by the National Research Council’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 workshop summary 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 process. We wish to thank the following individuals for their review of this workshop summary:
I. Glenn Cohen, Harvard Law School
Susan Howley, Christopher & Dana Reeve Foundation
Mahendra Rao, National Institutes of Health
Celia Witten, U.S. Food and Drug Administration
Although the reviewers listed above have provided many constructive comments and suggestions, they did not see the final draft of the workshop summary before its release. The review of this workshop summary was overseen by Caswell Evans, University of Illinois at Chicago, and Harold J. Fallon, University of Alabama at Birmingham School of Medicine. Appointed by the National Research Council and the Institute of Medicine, they were responsible for making certain that an independent examination of this workshop summary was carried out in accordance with institutional procedures and that all review comments
were carefully considered. Responsibility for the final content of this workshop summary rests entirely with the rapporteurs and the institution.
Acknowledgments
The support of the sponsors of Stem Cell Therapies: Opportunities for Ensuring the Quality and Safety of Clinical Offerings was crucial to the planning and conduct of the workshop and the development of the workshop summary report. Sponsorship was provided by the Alliance for Regenerative Medicine; California Institute for Regenerative Medicine; The Ellison Medical Foundation; Howard Hughes Medical Institute; International Society for Stem Cell Research; the Presidents’ Committee Funds of the National Academy of Sciences, the National Academy of Engineering, and the Institute of Medicine; and U.K. Academy of Medical Sciences.
The planning committee and staff wish to express their gratitude to the expert speakers whose presentations helped outline the challenges and opportunities for ensuring the quality and safety of stem cell therapies. The staff also wish to thank the members of the planning committee for their work in developing an excellent workshop agenda. The project director would like to thank project staff, who worked diligently to develop both the workshop and the resulting summary.
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Contents
1 INTRODUCTION AND THEMES OF THE WORKSHOP
2 STEM CELL THERAPIES—KNOWNS AND UNKNOWNS
Stem Cells as Regulated Therapeutics
Clinical Offerings Using Stem Cells
Medical Tourism: Patients Seeking Stem Cell Treatments
Patients Seeking Unproven or Unregulated Treatments
4 COMPARATIVE REGULATORY AND LEGAL FRAMEWORKS
Regulatory and Legal Frameworks in the United States
Regulatory and Legal Frameworks in Italy
Regulatory and Legal Frameworks in Japan
Regulatory and Legal Frameworks in Mexico
AIDS |
acquired immunodeficiency syndrome |
ASRM |
American Society for Reproductive Medicine |
CAS |
Chinese Academy of Sciences |
CEO |
chief executive officer |
CIRM |
California Institute for Regenerative Medicine |
COFEPRIS |
Comisión Federal para la Protección contra Riesgos Sanitarios (Mexico) |
EMA |
European Medicines Agency |
FDA |
U.S. Food and Drug Administration |
FTC |
Federal Trade Commission |
GMP |
good manufacturing process |
IOM |
Institute of Medicine |
IRB |
institutional review board |
ISCT |
International Society for Cellular Therapy |
ISSCR |
International Society for Stem Cell Research |
MHLW |
Ministry of Health, Labor, and Welfare (Japan) |
MS |
multiple sclerosis |
NAS |
National Academy of Sciences |
PMDA |
Pharmaceuticals and Medical Devices Agency (Japan) |
SART |
Society for Assisted Reproductive Technologies |
U.S. |
United States |