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Appendix A
Data Sources and Methods
The Committee on Review of the California Institute for Regenerative Medicine (CIRM) was
asked to assess CIRM’s initial processes, programmatic and scientific scope, organizational and
management systems, funding model, and intellectual property policies. To respond
comprehensively to its charge, the committee examined data from a variety of sources. These
sources included documents pertaining to the establishment and initial operation of CIRM and
numerous evaluation reports commissioned during the Institute’s initial 5- to 6-year period of
operation. Valuable input also was obtained through CIRM’s responses to the committee’s data
requests, three public meetings, two adjunct meetings, site visits to CIRM-funded research
facilities, telephone interviews, and written public comments in response to online questionnaires
targeting various stakeholders. The study was conducted over a 17-month period.
STUDY COMMITTEE
The study committee comprised 13 individuals with expertise in stem cell research,
developmental biology, bioethics, research administration, financial structures for biomedical
research, program evaluation, economics and finance, business administration, and intellectual
property. Appendix E provides biographical sketches of the committee members. The committee
convened for a total of 10 days on five different occasions in October 2011, January 2012, April
2012, June 2012, and August 2012.
DOCUMENT REVIEW
The committee reviewed background documents and evaluation reports pertaining to CIRM,
including Proposition 71 and Senate Bill (SB) 1064; 2004 Economic Impact Analysis reports;
the 2006 CIRM Scientific Strategic Plan; CIRM annual reports from 2007 through 2010; the
2008 Interim Economic Impact Review and addendum; the 2009 Strategic Plan Update; the 2009
Little Hoover Commission Review of CIRM; 2010 External Advisory Panel (EAP) evaluation
reports; the 2011 CIRM publication on the economic impact of research funded by CIRM; and
the 2012 CIRM transition plan. The committee also reviewed published reports on stem cell
research.
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A-1
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A-2 THE CALIFORNIA INSTITUTE FOR REGENERATIVE MEDICINE
DATA REQUESTS TO CIRM
The committee made a total of 79 requests for data, reports, and information from CIRM.
These requests were on topics related to CIRM’s initial processes and planning, grants and
programs, grant management and resources, reviews and evaluations, governance and
management, finances, collaborations, intellectual property policies, and conflict of interest
policies.
PUBLIC MEETINGS
The committee hosted three public meetings to obtain additional information on specific
aspects of the study charge. These meetings were held in conjunction with the committee’s
October (Washington, DC), January (San Francisco, California), and April (Irvine, California)
meetings. The committee determined the topics and speakers for these public meetings. As part
of the two public meetings in California, the committee held open forums at which members of
the public were invited to provide testimony on any topics related to the study charge.
At the first public meeting, CIRM delivered the charge to the committee and provided a
general overview of its structure and programs. At the second meeting, representatives from
CIRM’s leadership and governing board provided information on the Institute’s scientific
priorities and transition plan, standards working group, and intellectual property policies. In
addition, CIRM principal investigators provided their perspectives on the Institute. The third
public meeting included presentations from individuals with varying perspectives on CIRM—
investigators who applied for but did not receive funding from the Institute, current Disease
Team grantees, industry representatives, and a technology transfer officer. The agendas for the
public meetings are presented in Boxes A-1 through A-3.
ADJUNCT MEETINGS
A subgroup of the committee hosted two meetings with stem cell scientists outside of
California to obtain their perspectives on the work funded by CIRM and the Institute’s scientific
impact. Discussions centered on the scientists’ view of CIRM’s accomplishments and goals as
articulated in the 2012 Strategic Plan. The first meeting was held May 30, 2012, in Toronto,
Canada, and the second in Boston, Massachusetts, on June 26, 2012. These sites were selected as
the committee considered them to be major hubs of activity in stem cell research. Scientists
participating in these meeting are listed below.
Toronto
Mick Bhatia, Stem Cell and Cancer Research Institute, McMaster University (by phone)
James Ellis, Department of Molecular Genetics, University of Toronto
Andras Nagy, Samuel Lunenfeld Research Institute, Mount Sinai Hospital
Janet Rossant, Department of Medical Genetics and Microbiology, University of Toronto
Molly Shoichet, Department of Chemical Engineering and Applied Chemistry, University of
Toronto (by phone)
William Stanford, Department of Cellular and Molecular Medicine, University of Ottawa
Peter Zandstra, Institute of Biomaterials and Biomedical Engineering, University of Toronto
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DATA SOURCES AND METHODS A-3
Boston
Fernando Camargo, Stem Cell and Regenerative Biology, Harvard University
Konrad Hochedlinger, Stem Cell and Regenerative Biology, Harvard University
Jerome Ritz, Connell O’Reilly Cell Manipulation and Gene Transfer Laboratory, Dana-Farber
Cancer Institute, Harvard Medical School
Anthony Rosenzweig, Beth Israel Deaconess Medical Center, Harvard Medical School
David Scadden, Stem Cell and Regenerative Biology, Harvard University
Ramesh Shivdasani, School of Medicine, and Dana-Farber Cancer Institute, Harvard Medical
School
Les Silberstein, Joint Program in Transfusion Medicine, Harvard Medical School
Amy Wagers, Stem Cell and Regenerative Biology, Harvard University
Clifford Woolf, FM Kirby Neurobiology Center, Children’s Hospital, Harvard Medical School
Leonard Zon, Stem Cell and Regenerative Biology, Harvard University
SITE VISITS
Individual members of the committee conducted three site visits to CIRM-funded research
facilities to obtain information about stem cell programs funded by the Institute. The committee
selected one private university, one medical school, and one public university to visit. In January
2012, committee members visited CIRM facilities at the University of California at Davis (UC
Davis), the University of California at San Francisco (UCSF), and Stanford University. The
visits included tours of the stem cell facilities and discussions with principal investigators about
the CIRM-funded work being conducted at the institutions. Investigators and university
leadership who participated in each site visit are listed below. In addition, in August 2012, the
committee chair and vice chair visited Celgene, a biopharmaceutical company in Summit, New
Jersey. The purpose of this visit was to help the committee better understand the process of
discovery, development, and commercialization of products to treat disease.
UC Davis
Gerhard Bauer, Stem Cell Research Program, UC Davis Institute for Regenerative Cures (IRC)
Paul Knoepfler, Department of Cell Biology and Human Anatomy, School of Medicine
Kit Lam, Department of Biochemistry and Molecular Medicine, UC Davis Comprehensive
Cancer Center
Claire Pomeroy, CEO, UC Davis Health System and Dean of School of Medicine
Mark Zern, Transplant Research Institute
Min Zhao, Department of Dermatology, School of Medicine
UCSF
Arturo Alvarez-Bullya, Department of Neurological Surgery, Eli and Edythe Broad Center of
Regenerative Medicine and Stem Cell Research
Sam Hawgood, Dean of the UCSF School of Medicine and Vice Chancellor for Medical Affairs
Diana Laird, Department of Obstetrics, Gynecology and Reproductive Sciences, UCSF Medical
Center
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A-4 THE CALIFORNIA INSTITUTE FOR REGENERATIVE MEDICINE
Daniel Lim, Department of Neurological Surgery, Eli and Edythe Broad Center of Regenerative
Medicine and Stem Cell Research
Emmanuelle Passegue, Division of Hematology/Oncology, Department of Medicine, Eli and
Edythe Broad Center of Regenerative Medicine and Stem Cell Research
Holger Willenbring, Department of Surgery, Eli and Edythe Broad Center of Regenerative
Medicine and Stem Cell Research
Stanford University
Michael Longaker, Department of Surgery
Ravi Majeti, Division of Hematology, School of Medicine
Norma Neff, Research Technical Manager
Renee A. Reijo-Pera, Center for Human Embryonic Stem Cell Research and Education
Yuval Rinkevich, Institute for Stem Cell Biology and Regenerative Medicine
Chris Shay, Space and Renovations Project Management
Judy Shizuru, Department of Blood & Marrow Transplantation, School of Medicine
Irving Weissman, Department of Pathology, Institute for Stem Cell Biology and Regenerative
Medicine, School of Medicine
Marius Wernig, Department of Pathology, Institute for Stem Cell Biology and Regenerative
Medicine, School of Medicine
Joanna Wysocka, Department of Chemical and System Biology
INTERVIEWS
Committee members spoke with many individuals during the course of this study. These
formal and informal conversations, which took place by phone between the committee’s in-
person meetings, were intended to gather information to inform the committee’s deliberations
and to clarify questions. The individuals who provided this information are listed below.
Cindy Bell, Genome Canada and former Director of Cancer stem Cell Consortium
Alan Bernstein, former Executive Director, Global HIV Vaccine Enterprise
Genc Bülent, Bundesministerium für Bildung und Forschung (BMBF) (Federal Ministry of
Education and Research), Germany
George Daley, Stem Cell Transplantation at the Children’s Hospital
Kristen Doyle, Cancer Prevention and Research Institute of Texas
Bill Gimson, Cancer Prevention and Research Institute of Texas
Dan Gincel, Maryland Stem Cell Research Fund
Zach W. Hall, former president, CIRM
Marianne Horn, Connecticut Department of Public Health
Richard Klausner, The Column Group
Bob Klein, former board chair, CIRM
Bernard Lo, UCSF School of Medicine, CIRM Standards Working Group
Jeff Lomax, CIRM
Bert Lubin, Children’s Hospital and Research Center Foundation
David Magnus, Stanford University School of Medicine
Ed Penhoet, Alta Partners, former vice chair of CIRM’s Governing Board
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DATA SOURCES AND METHODS A-5
Mahendra S. Rao, National Institutes of Health (NIH) Intramural Center for Regenerative
Medicine
Beth Roxland, New York State Task Force on Life and the Law
Christopher Scott, Stanford University School of Medicine
Michael Stöcker, BMBF
Lawrence S. Sturman, New York State Stem Cell Science
Alan Trounson, CIRM
ONLINE QUESTIONNAIRES
The committee used online questionnaires to obtain public input from a variety of
stakeholders on topics related to the study charge. A total of seven questionnaires in different
topic areas targeting different stakeholders were placed on the Institute of Medicine (IOM)
website. These questionnaires were intended to help the committee understand perspectives of a
variety of stakeholders on CIRM and its work. The questionnaires were constructed for CIRM
Independent Citizens Oversight Committee (ICOC) members, leadership from CIRM-funded
institutions, CIRM principal investigators, university technology transfer professionals,
investigators who applied for but did not receive funding from CIRM, CIRM for-profit industry
partners, CIRM international partners, and stakeholders and members of the general public. A
summary of themes from the responses to the questionnaires can be found in Appendix B.
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A-6 THE CALIFORNIA INSTITUTE FOR REGENERATIVE MEDICINE
BOX A-1
Committee on a Review of the California Institute for Regenerative Medicine (CIRM)
The National Academies Keck Building
500 Fifth Street, NW
Washington, DC 20001
Room 110
AGENDA FOR PUBLIC SESSION
October 19, 2011
11:15 a.m. WELCOME AND COMMITTEE INTRODUCTIONS
Harold T. Shapiro, Ph.D.
OM Committee Chair
11:25 a.m. OVERVIEW OF CIRM: INITIAL PROCESSES, FUNDING MODEL,
ORGANIZATION, AND MANAGEMENT SYSTEMS
Robert Klein II, J.D.
Chair Emeritus, CIRM Governing Board
12:30 p.m. LUNCH
1:15 p.m. OVERVIEW OF THE SCIENCE AND CIRM’S PROGRAMMATIC AND
SCIENTIFIC SCOPE
Ellen G. Feigal, M.D.
Senior Vice President, Research and Development, CIRM
2:00 p.m. INTELLECTUAL PROPERTY AND BUSINESS DEVELOPMENT
Elona Baum, J.D.
General Counsel and Vice President, Business Development, CIRM
2:20 p.m. CHARGE TO THE COMMITTEE
Jonathan Thomas, J.D., D.Phil.
Chair, CIRM Governing Board
2:35 p.m. QUESTIONS AND DISCUSSION OF STUDY CHARGE
3:30 p.m. ADJOURN OPEN SESSION
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DATA SOURCES AND METHODS A-7
BOX A-2
Committee on a Review of the California Institute for Regenerative Medicine (CIRM)
South San Francisco Conference Center
255 South Airport Boulevard
South San Francisco, CA 94080
AGENDA FOR PUBLIC SESSION
January 24, 2012
9:00 a.m. WELCOME AND COMMITTEE INTRODUCTIONS
Harold T. Shapiro, Ph.D.
IOM Committee Chair
9:10 a.m. EVOLVING SCIENTIFIC PRIORITIES AT CIRM
Alan Trounson, Ph.D.
CIRM President
10:10 a.m. OVERVIEW OF CIRM SCIENTIFIC AND MEDICAL ACCOUNTABILITY
STANDARDS WORKING GROUP
Bernard Lo, M.D.
Standards Working Group Co-Chair
10:50 a.m. BREAK
11:00 a.m. CIRM INTELLECTUAL PROPERTY POLICIES
Duane J. Roth
CIRM Governing Board Vice Chair and Intellectual Property/Industry
Subcommittee Member
11:40 a.m. OVERVIEW OF CIRM TRANSITION PLAN
Jonathan Thomas, J.D., D.Phil.
Chair, CIRM Governing Board
12:20 p.m. LUNCH
1:15 p.m. PERSPECTIVES ON CIRM FROM THE PATIENT ADVOCACY COMMUNITY
Jeff Sheehy
Co-Vice Chair of the Scientific and Medical Research Funding Working Group
and CIRM Patient Advocate Board Member
Director of Communications
AIDS Research Institute
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A-8 THE CALIFORNIA INSTITUTE FOR REGENERATIVE MEDICINE
University of California, San Francisco
1:50 p.m. PERSPECTIVES ON CIRM FROM PRINCIPAL INVESTIGATORS
Irina Conboy, Ph.D.
Assistant Professor, Department of Bioengineering
University of California, Berkeley
John P. Murnane, Ph.D.
Professor, Department of Radiation Oncology
University of California, San Francisco
Howard Y. Chang, M.D., Ph.D.
Stanford University School of Medicine
Howard Hughes Medical Institute
Helen M. Blau, Ph.D.
Donald E. and Delia B. Baxter Professor
Director, Baxter Laboratory for Stem Cell Biology
Stanford University School of Medicine
Frederick J. Meyers, M.D., M.A.C.P. (by phone- invited)
Executive Associate Dean, University of California, Davis, School of Medicine
Alice F. Tarantal, Ph.D. (by phone)
Professor and Vice-Chair for Research, Department of Pediatrics
University of California, Davis, School of Medicine
3:15 p.m. BREAK
3:30 p.m. RECAP/DISCUSSION OF MORNING AND AFTERNOON PRESENTATIONS
4:00 p.m. PUBLIC COMMENT
Individuals register for a slot and have 5 minutes for comments on any topic
related to the study charge.
5:00 p.m. ADJOURN OPEN SESSION
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DATA SOURCES AND METHODS A-9
BOX A-3
Committee on a Review of the California Institute for Regenerative Medicine (CIRM)
Arnold and Mabel Beckman Center
of the National Academies of Sciences and Engineering
Huntington Room
100 Academy
Irvine, CA 92617
AGENDA FOR PUBLIC SESSION
April 10, 2012
9:00 a.m. WELCOME AND COMMITTEE INTRODUCTIONS
Harold T. Shapiro, Ph.D.
IOM Committee Chair
9:10 a.m. OVERVIEW OF LITTLE HOOVER COMMISSION REPORT
Stuart Drown (by phone)
Executive Director, Little Hoover Commission
10:00 a.m. PERSPECTIVES ON CIRM FROM STAKEHOLDERS
Ruth Holton-Hodson
California Deputy State Controller
Ken Taymor, J.D. (by phone)
Executive Director, Berkeley Center for Law, Business and Economy
John Simpson (by phone)
Director, Stem Cell Oversight and Accountability Project
Consumer Watchdog
Marcy Darnovsky, PhD (by phone)
Associate Executive Director, Center for Genetics and Society
David Jensen
Publisher-Editor, California Stem Cell Report
11:30 a.m. LUNCH
12:00 p.m. PERSPECTIVES FROM CIRM APPLICANTS
Leonard H. Rome, Ph.D. (by phone)
Senior Associate Dean for Research and Professor of Biological Chemistry,
David Geffen School of Medicine
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A-10 THE CALIFORNIA INSTITUTE FOR REGENERATIVE MEDICINE
University of California, Los Angeles
Xuejun H. Parsons, Ph.D.
Associate Professor of Regenerative Medicine and Scientific Director in
Cardiovascular and Neural Regeneration
San Diego Regenerative Medicine Institute
12:45 p.m. PERSPECTIVES ON CIRM FROM DISEASE TEAM INVESTIGATORS
Donald Kohn, M.D.
Professor, Department of Microbiology, Immunology, and Molecular Genetics
and Department of Pediatrics
University of California, Los Angeles
Larry Goldstein, Ph.D.
Professor, Department of Cellular and Molecular Medicine and Department of
Neurosciences
University of California, San Diego School of Medicine
Dennis Clegg, Ph.D.
Professor, Molecular, Cellular, and Developmental Biology
University of California, Santa Barbara
Catriona Jamieson, M.D., Ph.D.
Associate Professor, Division of Hematology-Oncology
University of California, San Diego Moores Cancer Center
2:00 p.m. PERSPECTIVES ON CIRM FROM INDUSTRY
Gregory A. Bonfiglio, J.D., Panel Moderator
Managing Partner, Proteus Venture Partners
Michael D. West, Ph.D.
Chief Executive Officer, BioTime, Inc.
Allan Robins, Ph.D.
Acting Chief Executive Officer, Vice President and Chief Technical Officer,
ViaCyte, Inc.
Gail K. Naughton, Ph.D.
CEO, Chairman of the Board, Histogen, Inc.
3:30 p.m. BREAK
3:45 p.m. PERSPECTIVES ON CIRM FROM TECHNOLOGY TRANSFER OFFICERS
Casie Kelly, Ph.D.
Technology Transfer Officer, Life Sciences, Office of Intellectual Property &
Industry Sponsored Research
University of California, Los Angeles
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DATA SOURCES AND METHODS A-11
4:30 p.m. PUBLIC COMMENT
Individuals register for a slot and have 5 minutes for comments on any topic
related to the study charge.
5:30 p.m. ADJOURN OPEN SESSION
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