CIRM Governing Board Receives Report from External Review Panel— It Praises CIRM’s Success to Date and Makes Broad Recommendations for Further Accelerating the Development of Therapies

Irvine, Calif., December 8, 2010 — Members of the eight-person External Advisory Panel (EAP), who in October investigated CIRM’s progress to date and trajectory moving forward, presented their joint findings and unanimous report to the state stem cell agency’s Governing Board today.

“In our judgment CIRM is at an interesting flash point in its history, moving from stage 1 to stage 2," said the Chair of the EAP, Alan Bernstein, Executive Director of the Global HIV Vaccine Enterprise, and former President of the Canadian Institutes of Health Research. "We were very impressed with the speed with which stage 1 got going and the number of programs that are underway.,”

“The extraordinary endorsement of the phase 1 work of our agency and the innovative ideas on how we can advance translational medicine in the second phase of CIRM’s mission provide an invaluable contribution to advancing our therapies to reach patients with cancer, diabetes, Alzheimer’s, heart disease, and the whole range of chronic disease and injury,” said Robert Klein, Chair of the CIRM Governing Board. “The review panel emphasized the outstanding science that has been funded and the remarkable progress of building the research and facilities platform on which we can launch phase 2 of the CIRM mission. We thank the 7 million visionary California voters who gave us the privilege to drive therapies for California and the world and to achieve the clear global leadership for stem cell research attributed to us by the review panel. “

A list of panel members and their biographies is available here, along with documents provided by CIRM to the panel, and the final report.

The EAP report summarized CIRM’s first years this way: “progress during this first stage of CIRM’s development has been remarkable; CIRM has built significant additional research capacity in the state, has attracted scores of talented young people to stem cell research, and has catalyzed large and important stem cell projects across the state. The EAP was most impressed with this rapid start up, the overall quality of the scientists and projects that have been funded, the development of major buildings and other facilities for stem cell research, the forging of several important international partnerships and the innovative training programs that are in place.”

Senator Art Torres, Co Vice-Chair of the governing board, thanked the panel for their detailed report. "For the members of the panel, I felt your work was done very, very well; your work was very detailed. I don’t think there has ever been such a thorough evaluation of a California agency."

The bulk of the report addresses recommendations for CIRM to do even more to accelerative the field by developing ways to make its funding more flexible, opportunistic and able to quickly respond to major discoveries, particularly those that are close to the clinic.

“The EAP feels confident that CIRM is poised to build on the success of the first stage to drive further growth towards its long-term mission of providing significant health and economic benefits to the people of California,” stated the EAP report.

“The panel has made some very thoughtful, and helpful recommendations for delivery of the CIRM mandate so as to become an even more effective organization going forward,” said CIRM President Alan Trounson. “We have already begun the internal process of considering the opportunities and revision of processes and timelines for responding to the challenging recommendations with our Board and adapting them as appropriate to deliver the revolution in regenerative medicine envisaged in Proposition 71, the voter initiative that created CIRM."

George Daley, who was a member of the external advisory board and is director of Stem Cell Transplantation at the Children's Hospital Boston, said during the board discussion, "The work of the grants review committee is spectacular. We were impressed with the caliber of science that is being funded. The Grants working group at CIRM is a higher standard than at NIH study groups."

A summary of the recommendations:

1. Maintain Focus on Meaningful, Targeted Scientific Excellence
CIRM has quickly developed an international reputation for excellence in science coupled with the strong underlying principle that the research funding needs to be aligned with the mission of CIRM.

2. Sustain Fundamental Discovery
The basic biology programs are the fuel that will continue to drive discovery and innovation in stem cell research and to create new opportunities for applying the unique properties of these cells to the clinic.

3. Paving a Path from Fundamental to Translational Research, Translational Medicine, Product Development and Healthcare Delivery
CIRM should increasingly move away from a traditional funding model and adopt a more aggressively proactive approach to indentifying innovative projects across the stem cell therapeutic landscape that show promise for moving into translational research, clinical trials and product development.

4. Portfolio Prioritization Process
CIRM should conduct a critical assessment of its current portfolio and it must begin the difficult process of focusing the number of disease areas to those that it believes have the greatest chance of development progress and clinical success, given reasonable timelines and budget.

5. Develop an Open Innovation, Porous Pipeline Strategy
Develop a model that allows the most likely source of clinical projects to come from either inside or outside of CIRM funded research, perhaps out of industry and even from outside of California. CIRM should capture these projects and bring them to California under CIRM funding of California collaborators. These projects could enter the CIRM pipeline at any stage of preclinical or clinical development.
(The report does not expect CIRM to fund projects outside of California.)

6. Social, Ethical, Healthcare delivery and Regulatory Issues
There are critical ethical, economic, manufacturing, health delivery, and social issues that require research, discussion and translation into policy and practice. These activities should be viewed as an integral part of CIRM’s portfolio.

7. Industry Engagement
Granting processes and funding criteria could be clarified and streamlined from an industry perspective and timelines for decision-making could be aligned with industry norms.

8. International Partnerships
CIRM should broaden these partnerships to other regions and countries and diversify those partnerships.

9. Outreach and Education
CIRM should significantly increase the breadth of it's community outreach and education programs.

10. Governing Board Composition and Corporate Governance
It is imperative that the roles and responsibilities of the Governing Board Chair and the CEO positions remain distinct but complementary to ensure the continued positive, collaborative partnership of these two key individuals.

Governing Board member Leeza Gibbons commented on the panel's recommendation to increase outreach to the California public. "Everyone wants their heroes. If we can tell our stories and make the connection between the science and the people we will show that there are heroes in this field," she said.

Contact:
Don Gibbons
dgibbons@cirm.ca.gov
(415) 396-9117

© 2013 California Institute for Regenerative Medicine