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
INSTITUTE OF MEDICINE
OF THE NATIONAL ACADEMIES
THE NATIONAL ACADEMIES PRESS
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.
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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.
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. 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.
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, 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
RALPH SNYDERMAN, Chancellor Emeritus for Health Affairs; and James B. Duke Professor, School of Medicine, Duke University, Durham, North Carolina
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, National Institutes of Health
David Resnick, Nixon Peabody
Janet Rossant, University of Toronto
William A. Sahlman, Harvard Business School
Bernard Seigel, 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.
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. Although 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 per 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 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
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 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.
|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|
|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|
|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|