A Brief History, Current Status Report and Options for Next Steps

A Brief History, Current Status Report And Options for Next steps Prepared for CIRM outside reviewers prior to October 13-15, 2010 on-site review 1 Index — Introduction, p.3 Getting Started, p. 4 Scientific Strategic Plan, p. 5 The Grant Review Process, p. 7 Business Systems, p. 9 Intellectual Property, p. 9 Medical and Ethical Standards, p. 10 The Science Program, p. 11 Intellectual Resources, p.11 Facilities Infrastructure, p. 13 Pipeline Strategy, p. 14 Foundational Biology, p. 14 Creating a Development Portfolio, p. 16 Assessing the Research Portfolio, p. 20 Outcomes, p. 22 Impact of Grants that Have Been Completed, p. 23 Collaborative Funding Leverages RFA Potential, p. 24 Managing the Portfolio, p. 26 The Business side of Grant Management, p. 26 Medical and Ethical Standards and Compliance, p. 27 Operations and Administration, p. 29 Outreach, p. 31 Partnering in the Stem Cell Community, p. 32 Industry Engagement, p. 33 Going Forward, p. 34 Our Loan Program, p. 35 Other Approaches to Attract Additional Funding, p. 36 Next Steps in Intellectual Property, p. 37 Our Science Program, p. 38 Core Programs and New Initiatives, p. 39 Refinancing CIRM, 41 Appendices, p. 42 2 Introduction The California Institute for Regenerative Medicine has matured into a deliberative, targeted funding agency in the nearly six years since 59 percent of California voters approved the initiative, Proposition 71, that created the agency in November 2004 http://www.cirm.ca.gov/AboutCIRM_Prop71 The agency has awarded 364 grants and loans for research and facilities to 54 institutions totaling $1.07 billion. About half of those commitments have been disbursed. This progress comes despite years of litigation and delays. A series of court cases were resolved in CIRM’s favor in May of 2007 and the state issued the first bonds under the initiative in October, nearly three years after the vote. The agency was able to fund its first round of grants a year earlier, in April 2006, through a loan from the state’s general fund arranged by Governor Arnold Schwarzenegger and through the generous support of individuals. To date, the agency has issued 22 rounds of funding. CIRM has established systems and processes for soliciting, evaluating, and monitoring high quality, targeted research projects and to do this in an ethically sound manner (Link to 2006 Scientific Strategic Plan at http://www.cirm.ca.gov/meetings/pdf/2006/12/120706_item_7.pdf ) It has seeded the stem cell research field in California with a greatly expanded workforce and dedicated facilities. It is now entering a second phase in which it can build on this robust foundation and deliver on its goal of accelerating this research toward the clinic (Link to 2009 Scientific Strategic Plan Update at http://www.cirm.ca.gov/files/PDFs/Publications/2009_Strategic_Plan-2.pdf ) The systems developed by CIRM have produced results. As of August 10, CIRM funding contributed toward the work published in 584 journal articles (Appendix 5 - List of Publications). Twenty-three percent of those have been in high profile journals, and the CIRM grantee was first or last author on 75 percent of all 584 papers. Seventeen shared lab facilities are up and running providing specialized equipment, facilities and training necessary to carry out stem cell protocols. Six of these labs were funded for specific Techniques Training Courses as well. Of the 12 major facilities grants that were awarded $271 million in May of 2008, two projects have been completed and opened this spring, three more are scheduled to open in October of this year and all but one of those remaining are scheduled for completion by December, 2011. These dedicated stem cell facilities and CIRM’s predictable and sustained funding stream have clearly impacted the ability of California institutions to recruit in this field. We have documented more than 100 faculty-level recruits since 2006 (Appendix 6 - Faculty Level Recruits). CIRM-funded research has contributed to moving two candidate therapeutics into phase I FDA- approved clinical trials. (Appendix 38, A new Political-Financial Paradigm for Medical Research: The California Model) These apparent successes notwithstanding, CIRM is now exploring whether or not its focus, systems and processes are optimal. Is a focus on pluripotent-derived therapies the optimal strategy to benefit patients? Are we attracting and selecting the research proposals that are most likely to accelerate the field toward therapies? Are we providing effective oversight to maximize the pace and success of individual projects and the synergy between projects? As we think about system gaps and process improvements, which should garner the greatest attention and assets? 3 Figure 1. CIRM was tangled in court cases for more than two years but used the time to set up its processes and to issue two rounds of grants through borrowed and donated funds. Getting Started CIRM’s governing board conducted an organizing meeting in December 2004 and it held its first business meeting in January, at which time the agency hired its first staff. The board met monthly the first year setting up procedures for creating its working groups for peer review, facilities and ethical standards; selecting the members of those groups; determining which city would host the agency; and numerous other start-up activities. (For a more extensive discussion regarding Proposition 71 and its requirements refer to Report of the Office of the Chair to External Reviewers.) During that first year, the agency was quick to create the processes necessary to fund promising science. CIRM issued its first Request for Applications (RFA) – for training grants – in May, organized the 15-member Grant Review Group to judge the applications in August and the Board approved the first awards at its September meeting. But by that time litigation had been filed preventing the agency from raising money through bonds, and the grants were put on hold. By December, staff had written an Interim Grants Administration Policy (GAP) able to handle the limited scope of training grants, pending available funds. Also, the board had adopted the National Academy of Science’s standard for human embryonic stem cell research as interim standards. In 2006, a slowly growing staff started to formalize and clarify the policies and procedures created in the first year. In April the agency used funds from Bond Anticipation Notes (BANs) to issue the first round of awards, the training grants approved the prior September. The BANs, from wealthy supporters of CIRM, would not have been taken out as bonds if the plaintiffs had succeeded in their challenge to Proposition 71 and would have been converted to gifts to the state. During the second half of the year the agency issued three RFAs that were known as the Jump Start Program: SEED grants to bring new investigators and innovative ideas into human embryonic cell research, Comprehensive grants to support mature projects of researchers with a track record in stem cell research, and Shared Labs to provide critical infrastructure and training in human embryonic stem cell use. Also, mid-year 2006 the board approved the agency’s policy on medical and ethical standards after a year of 4 work by the committee charged with developing the standards. Most important, the board and staff completed the Scientific Strategic Plan, which was approved by the board at its December 2006 meeting. THE SCIENTIFIC STRATEGIC PLAN — The agency developed the plan over a period of 14 months having sought the advice of more than 170 scientists, ethicists, patient advocates and public and private representatives. Work on the plan began with a scientific meeting that brought leading stem cell scientists together to discuss the challenges and opportunities of this emerging field on October 1-2, 2005, “Stem Cell Research: Charting New Directions for California.” A team from PricewaterhouseCooper facilitated later stages of the effort. Three smaller scientific conferences on specific topics followed along with two focus group meetings. The Board devoted two full meetings to developing the mission statement, values and strategic principals. The centerpiece of the plan is two sets of goals for CIRM, 10 five-year goals (Appendix 2 – CIRM 5 Year Goals annotated with success-to-date) and 10 ten-year goals (Appendix 3 – CIRM 10 Year Goals annotated with success-to-date). Collectively, those goals set aim on providing evidence that cell replacement therapy using derivatives of human embryonic stem cells is effective for at least one disease, producing a rich pipeline of therapeutic candidates for other diseases, and laying a broad foundation of knowledge about stem cells and disease mechanisms on which future research can build new therapies. The 2006 Strategic Plan was intended to be a living document. In recognition of the field’s rapid development, the agency updated the plan in 2009 (Appendix 4 – 2009 Strategic Plan Key Revisions). That update maintains the aims of the 2006 plan but increases focus on a “pipeline to cures” (Fig. 2) with significant increases in funding for translational and clinical programs and through five specific strategies: • Acceleration of Therapeutic Discoveries, by fostering teams, retaining flexibility to respond to advances in the field, actively managing the development portfolio, capturing data on our progress and sharing that data, fostering the sharing of expertise, promoting partnership with industry, initiating the linkage of stem cell and immunology researchers to

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