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The California Institute for Regenerative
Medicine
Science, Governance, and the Pursuit of Cures
Committee on a Review of the California Institute for Regenerative Medicine
Board on Health Sciences Policy
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THE NATIONAL ACADEMIES PRESS 500 Fifth Street, NW Washington, DC 20001
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 competences and with regard
for appropriate balance.
This study was supported by Contract No. 2267 between the National Academy of Sciences and the
California Institute for Regenerative Medicine. Any opinions, findings, conclusions, or
recommendations expressed in this publication are those of the author(s) and do not necessarily
reflect the views of the organizations or agencies that provided support for the project.
International Standard Book Number 0-309-0XXXX-X
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.
Copyright 2013 by the National Academy of Sciences. All rights reserved.
Printed in the United States of America
The serpent has been a symbol of long life, healing, and knowledge among almost all cultures and
religions since the beginning of recorded history. The serpent adopted as a logotype by the Institute
of Medicine is a relief carving from ancient Greece, now held by the Staatliche Museen in Berlin.
Suggested citation: IOM (Institute of Medicine). 2013. The California Institute for Regenerative
Medicine: Science, governance, and the pursuit of cures. Washington, DC: The National Academies
Press.
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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. Charles M. Vest 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. Charles M. Vest are chair and vice chair, respectively, of the
National Research Council.
www.national-academies.org
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COMMITTEE ON A REVIEW OF THE CALIFORNIA INSTITUTE FOR
REGENERATIVE MEDICINE
HAROLD T. SHAPIRO (Chair), President Emeritus, Professor of Economics and Public Affairs, the
Woodrow Wilson School of Public and International Affairs, Princeton University, New Jersey
TERRY MAGNUSON (Vice Chair), Vice Dean for Research, School of Medicine, S.G. Kenan Professor
and Chair, Department of Genetics, University of North Carolina at Chapel Hill
RICHARD R. BEHRINGER, Professor, Department of Genetics, the University of Texas MD Anderson
Cancer Center, Houston
REBECCA S. EISENBERG, Robert and Barbara Luciano Professor, School of Law, University of
Michigan, Ann Arbor
INSOO HYUN, Associate Professor, Department of Bioethics, School of Medicine, Case Western Reserve
University, Cleveland, Ohio
GARY A. KORETZKY, Francis C. Wood Professor, Perelman School of Medicine at the University of
Pennsylvania, Philadelphia
CATO T. LAURENCIN, Albert and Wilda Van Dusen Distinguished Professor of Orthopaedic Surgery;
Professor of Chemical, Materials and Biomolecular Engineering; Chief Executive Officer, Connecticut
Institute for Clinical and Translational Science; and Director, Institute for Regenerative Engineering, the
University of Connecticut, Farmington
AARON D. LEVINE, Assistant Professor, School of Public Policy, Georgia Institute of Technology,
Atlanta
MICHAEL H. MAY, Chief Executive Officer, Center for Commercialization of Regenerative Medicine,
Toronto, Canada
CHERYL A. MOORE, Executive Vice President and Chief Operating Officer, Howard Hughes Medical
Institute, Chevy Chase, Maryland
STUART H. ORKIN, David G. Nathan Professor of Pediatrics; Chair, Department of Pediatric Oncology,
Dana-Farber Cancer Institute; and Investigator, Howard Hughes Medical Institute, Harvard Medical
School, Boston, Massachusetts
ALLEN M. SPIEGEL, Marilyn and Stanley M. Katz Dean, Albert Einstein College of Medicine of
Yeshiva University, Bronx, New York
SHARON TERRY, President and Chief Executive Officer, Genetic Alliance, Washington, DC
Consultant
RALPH SNYDERMAN, Chancellor Emeritus for Health Affairs; and James B. Duke Professor, School
of Medicine, Duke University, Durham, North Carolina
IOM Staff
ADRIENNE STITH BUTLER, Senior Program Officer
JING XI, Research Associate
THELMA L. COX, Senior Program Assistant
DONNA RANDALL, Administrative Assistant
LORA K. TAYLOR, Financial Associate
ANDREW M. POPE, Director, Board on Health Sciences Policy
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REVIEWERS
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 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 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:
Laurence Baker, Stanford University
Christopher H. Colecchi, Broadview Ventures, Inc
James Fossett, University of Albany
Joseph G. Hacia, University of Southern California
Zach Hall, Retired
Mark S. Humayun, University of Southern California
Edison Liu, The Jackson Laboratory
Mary Majumder, Baylor College of Medicine
Robert M. Nerem, Georgia Institute of Technology
Mahendra Rao, The National Institutes of Health
David Resnick, Nixon Peabody
Janet Rossant, University of Toronto
William A. Sahlman, Harvard Business School
Bernard Siegel, Genetics Policy Institute
Nancy S. Wexler, Columbia University
Although the reviewers listed above provided many constructive comments and suggestions, they
were not asked to endorse the report’s conclusions or recommendations, nor did they see the final draft of
the report before its release. The review of this report was overseen by Ellen Wright Clayton, Vanderbilt
University and Huda Akil, University of Michigan. Appointed by the National Research Council and the
Institute of Medicine, 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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Preface
The energetic, imaginative, and committed coalition of California citizens and others
responsible for the passage of Proposition 71 in the 2004 general election produced a social
innovation. While state initiatives in research and development are not new, this initiative, in
both scope and design, broke new ground. In essence, the voters of California expressed a strong
desire to move ahead in the field of regenerative medicine, including research using human
embryonic stem cells, despite the ongoing near paralysis of the federal government in aspects of
this arena. In the globalized world of biomedical research, they grasped the possibility that by
building on California’s already strong and deep biomedical research and biotechnology
community and by structuring a distinctive model of finance, they could not only dramatically
advance the field of regenerative medicine, but also establish California as one of the worldwide
hubs in this promising area of biomedical research and development. At the time, this was also a
courageous initiative given that certain aspects of regenerative medicine, especially work using
embryonic stem cells derived from human embryos, were highly controversial in ethical terms. It
is worth remembering that in 2004, there had been little demonstration of the potential for
reprogramming somatic cells to bring them to a pluripotent state.
The California Institute for Regenerative Medicine (CIRM) was the organization charged
with responsibility for thoughtfully expending the $3 billion set aside by voters through the
passage of Proposition 71 to advance critical aspects of the field of regenerative medicine in
California. Indeed, one of the Institute’s principal aims was to help create in California an
international hub of research and development in regenerative medicine. It is the committee’s
judgment that overall, CIRM has done a very good job of initially establishing and then updating
the strategic plans that have set priorities for and guided its programs, and of taking advantage of
its guaranteed flow of $300 million a year for 10 years to establish a sustainable position in
regenerative medicine for California. The challenge of moving its research programs closer to
the clinic and California’s large biotechnology sector is certainly on CIRM’s agenda, but
substantial achievements in this arena remain to be made.
Despite its demonstrable achievements to date, as well as the largely positive independent
reports covering various aspects of its operations, no one would claim that CIRM is a perfect
organization or that it should adhere slavishly to its initial form of organization, set of
regulations, or pattern of priorities. The field of regenerative medicine has advanced rapidly
since November 2004, and CIRM itself has seen the need to alter its activities and approaches in
some areas. The committee believes the same should be true of its governance structure, some of
its administrative practices, and its use of external perspectives on strategic scientific priorities
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x THE CALIFORNIA INSTITUTE FOR REGENERATIVE MEDICINE
and on the evaluation of other key policies, such as Intellectual Property, to ensure that they
continue to encourage the development and deployment of new treatments. Experience has
shown that Proposition 71 can, in partnership with the California Legislature and the governor,
be amended in a manner that would optimize CIRM’s functionality and best serve the interests of
the citizens of California.
In this report, the committee has endeavored to evaluate various aspects of CIRM’s programs
and experiences with the aim of acknowledging both its successes and remaining challenges. The
committee also has considered the lessons of CIRM’s experience for other states, or even the
federal government, that might wish to use CIRM’s experience to inform some of their
initiatives.
Finally, we wish to thank our colleagues on the committee for their tireless devotion to this
task. We also wish to express our appreciation to CIRM for its openness and responsiveness to
the committee’s many requests for information during the course of this study.
Harold T. Shapiro, Chair
Terry Magnuson, Vice Chair
Committee on a Review of the California
Institute for Regenerative Medicine
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Acknowledgments
Several individuals and organizations made important contributions to the study committee’s
process and to this report. The committee wishes to thank these individuals, but recognizes that
attempts to identify all and acknowledge their contributions would require more space than is
available in this brief section.
To begin, the committee would like to thank the sponsor of this study. Funds for the
committee’s work were provided by the California Institute for Regenerative Medicine (CIRM).
The committee thanks Lynn Harwell, who served as project officer, and CIRM staff for their
assistance during the study process.
The committee gratefully acknowledges the contributions of the many individuals who
assisted in the conduct of this study. The perspectives of many individuals and organizations
were valuable in understanding CIRM and its work. The committee thanks those who provided
important oral testimony at its open workshops. Appendix A lists each of these individuals and
their affiliations. As part of its review, the committee also visited three sites that receive CIRM
funding to gather information about the role of that support in their work. In addition, many
individuals with knowledge of CIRM, as well as analogous programs in other states, participated
in interviews with committee members (see Appendix A). The committee also received written
testimony through several questionnaires targeting various stakeholder groups. The committee
greatly appreciates the time, effort, and information provided by all of these knowledgeable and
dedicated individuals.
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Contents
ACRONYMS xv
SUMMARY S-1
Abstract S-1
Charge to the Committee and Study Approach S-2
CIRM’s Origins and Transition S-2
Governance of CIRM S-4
The Scientific Program S-7
Intellectual Property Policies S-10
Conclusion S-12
1 INTRODUCTION AND CONTEXT 1-1
Statement of Task and Study Approach 1-2
Character and Potential of Stem Cell Research 1-4
The Stem Cell Controversy 1-5
Organization of This Report 1-7
References 1-9
2 CIRM’S INITIAL PROCESSES AND FUNDING MODEL 2-1
Origins of CIRM 2-1
Funding of CIRM and Its Economic Impact 2-4
CIRM in the Context of Other State-Based Science and Technology Initiatives 2-8
Conclusions and Recommendation 2-13
References 2-16
3 CIRM’S GOVERNANCE STRUCTURE 3-1
Organization of CIRM’s Governance Structure 3-1
Governance Structure of Programs Comparable to CIRM 3-3
Prior Assessments of CIRM’S Governance Structure 3-5
Conclusions and Recommendations 3-6
References 3-17
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xiv THE CALIFORNIA INSTITUTE FOR REGENERATIVE MEDICINE
4 NATURE, SCOPE, AND ACCOMPLISHMENTS OF THE CIRM
SCIENTIFIC PROGRAM 4-1
Strategic Planning 4-1
Grant Review and Funding Process 4-7
Bioethics 4-10
Industry Engagement 4-11
Conclusions and Recommendations 4-12
References 4-22
5 CIRM’S INTELLECTUAL PROPERTY POLICIES 5-1
Description of CIRM Intellectual Property Policies 5-2
The Legal Framework and Reach of CIRM’s Intellectual Property Policies 5-4
Revenue Sharing 5-5
Access Plans 5-8
March-in Rights 5-10
Dissemination of Biomedical Material Within California 5-11
Conclusions and Recommendations 5-13
References 5-15
GLOSSARY GL-1
APPENDIXES
A Data Sources and Methods A-1
B Summary of Questionnaires B-1
C Proposition 71 C-1
D Senate Bill 1064 D-1
E Committee Biographies E-1
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Acronyms
CCR5 C-C chemokine receptor type 5
CEO chief executive officer
CFO chief financial officer
CIRM California Institute for Regenerative Medicine
CPRIT Cancer Prevention Research Institute of Texas
CRADA cooperative research and development agreement
CTRC clinical translational research center
CTSA Clinical and Translational Science Award
EAP External Advisory Panel
FDA Food and Drug Administration
FTE full-time equivalent
GSP gross state domestic product
GWG Grants Working Group
hES human embryonic stem (cell)
HHS Department of Health and Human Services
ICOC Independent Citizens Oversight Committee
IND Investigational New Drug
IOM Institute of Medicine
iPS induced pluripotent stem (cell)
IRB institutional review board
ISSCR International Society for Stem Cell Research
MSCRF Maryland Stem Cell Research Fund
NACD National Association of Corporate Directors
NAS National Academy of Sciences
NCSL National Conference of State Legislatures
NGA National Governors Association
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xvi THE CALIFORNIA INSTITUTE FOR REGENERATIVE MEDICINE
NIH National Institutes of Health
NRC National Research Council
NYSTEM New York State Stem Cell Science Research Fund
OTA Office of Technology Assessment
R&D research and development
RFA request for applications
RNAi ribonucleic acid interference
SAB Scientific Advisory Board
SVP senior vice president
SWG Standards Working Group
TGR The Guttmacher Report
UCLA University of California, Los Angeles
UCSD University of California, San Diego
UCSF University of California, San Francisco
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