The California Institute for Regenerative Medicine: Science, Governance, and the Pursuit of Cures (2013)
Chapter: Appendix A: Data Sources and Methods
Appendix A
Data Sources and Methods
The Committee on a 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.
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
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, Davis (UC Davis), the University of California, 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
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
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
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 |
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.
| 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 |
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 | |
| 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 |
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, Ph.D. (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 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 | |
| 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 |
