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Suggested Citation:"Front Matter." Institute of Medicine. 2013. The California Institute for Regenerative Medicine: Science, Governance, and the Pursuit of Cures. Washington, DC: The National Academies Press. doi: 10.17226/13523.
×

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

Washington, D.C.

www.nap.edu

Suggested Citation:"Front Matter." Institute of Medicine. 2013. The California Institute for Regenerative Medicine: Science, Governance, and the Pursuit of Cures. Washington, DC: The National Academies Press. doi: 10.17226/13523.
×

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-13: 978-0-309-26590-4
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Copyright 2013 by the National Academy of Sciences. All rights reserved.

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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.

Suggested Citation:"Front Matter." Institute of Medicine. 2013. The California Institute for Regenerative Medicine: Science, Governance, and the Pursuit of Cures. Washington, DC: The National Academies Press. doi: 10.17226/13523.
×

Knowing is not enough; we must apply.
Willing is not enough; we must do.
”      

                                                —Goethe

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INSTITUTE OF MEDICINE
              OF THE NATIONAL ACADEMIES

Advising the Nation. Improving Health.

Suggested Citation:"Front Matter." Institute of Medicine. 2013. The California Institute for Regenerative Medicine: Science, Governance, and the Pursuit of Cures. Washington, DC: The National Academies Press. doi: 10.17226/13523.
×

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.

www.national-academies.org

Suggested Citation:"Front Matter." Institute of Medicine. 2013. The California Institute for Regenerative Medicine: Science, Governance, and the Pursuit of Cures. Washington, DC: The National Academies Press. doi: 10.17226/13523.
×

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

Consultant

RALPH SNYDERMAN, Chancellor Emeritus for Health Affairs; and James B. Duke Professor, School of Medicine, Duke University, Durham, North Carolina

Suggested Citation:"Front Matter." Institute of Medicine. 2013. The California Institute for Regenerative Medicine: Science, Governance, and the Pursuit of Cures. Washington, DC: The National Academies Press. doi: 10.17226/13523.
×

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

Suggested Citation:"Front Matter." Institute of Medicine. 2013. The California Institute for Regenerative Medicine: Science, Governance, and the Pursuit of Cures. Washington, DC: The National Academies Press. doi: 10.17226/13523.
×

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, National Institutes of Health

David Resnick, Nixon Peabody

Janet Rossant, University of Toronto

Page viii Cite
Suggested Citation:"Front Matter." Institute of Medicine. 2013. The California Institute for Regenerative Medicine: Science, Governance, and the Pursuit of Cures. Washington, DC: The National Academies Press. doi: 10.17226/13523.
×

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.

Suggested Citation:"Front Matter." Institute of Medicine. 2013. The California Institute for Regenerative Medicine: Science, Governance, and the Pursuit of Cures. Washington, DC: The National Academies Press. doi: 10.17226/13523.
×

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. 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

Suggested Citation:"Front Matter." Institute of Medicine. 2013. The California Institute for Regenerative Medicine: Science, Governance, and the Pursuit of Cures. Washington, DC: The National Academies Press. doi: 10.17226/13523.
×

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

Suggested Citation:"Front Matter." Institute of Medicine. 2013. The California Institute for Regenerative Medicine: Science, Governance, and the Pursuit of Cures. Washington, DC: The National Academies Press. doi: 10.17226/13523.
×

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 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.

Suggested Citation:"Front Matter." Institute of Medicine. 2013. The California Institute for Regenerative Medicine: Science, Governance, and the Pursuit of Cures. Washington, DC: The National Academies Press. doi: 10.17226/13523.
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Suggested Citation:"Front Matter." Institute of Medicine. 2013. The California Institute for Regenerative Medicine: Science, Governance, and the Pursuit of Cures. Washington, DC: The National Academies Press. doi: 10.17226/13523.
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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
Suggested Citation:"Front Matter." Institute of Medicine. 2013. The California Institute for Regenerative Medicine: Science, Governance, and the Pursuit of Cures. Washington, DC: The National Academies Press. doi: 10.17226/13523.
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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
Suggested Citation:"Front Matter." Institute of Medicine. 2013. The California Institute for Regenerative Medicine: Science, Governance, and the Pursuit of Cures. Washington, DC: The National Academies Press. doi: 10.17226/13523.
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Suggested Citation:"Front Matter." Institute of Medicine. 2013. The California Institute for Regenerative Medicine: Science, Governance, and the Pursuit of Cures. Washington, DC: The National Academies Press. doi: 10.17226/13523.
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Page xiii Cite
Suggested Citation:"Front Matter." Institute of Medicine. 2013. The California Institute for Regenerative Medicine: Science, Governance, and the Pursuit of Cures. Washington, DC: The National Academies Press. doi: 10.17226/13523.
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The California Institute for Regenerative Medicine (CIRM) was created in 2005 by The California Stem Cell Research and Cures Act (Proposition 71) to distribute $3 billion in state funds for stem cell research. The passage of Proposition 71 by the voters of California occurred at a time when federal funding for research involving human embryonic stem cells was uncertain, given the ethical questions raised by such research. During its initial period of operations, CIRM has successfully and thoughtfully provided more than $1.3 billion in awards to 59 California institutions, consistent with its stated mission.

As it transitions to a broadened portfolio of grants to stimulate progress toward its translational goals, the Institute should obtain cohesive, longitudinal, and integrated advice; restructure its grant application review process; and enhance industry epresentation in aspects of its operations. CIRM's unique governance structure, while seful in its initial stages, might diminish its effectiveness moving forward. The California Institute for Regenerative Medicine: Science, Governance, and the Pursuit of Cures recommends specific steps to enhance CIRM's organization and management, as well as its scientific policies and processes, as it transitions to the critical next stages of its research and development program.

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