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Enabling Stem Cell Research in California

Enabling Stem Cell Research in California. Gil Sambrano, Ph.D. California Institute for Regenerative Medicine. What is a stem cell?. 1. Mature/specialize 2. Self-renew. Stem Cell Capacity. Stem Cell Capacity. Human embryonic stem cell lines were first derived in 1998 by Dr. James Thompson.

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Enabling Stem Cell Research in California

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  1. Enabling Stem Cell Research in California Gil Sambrano, Ph.D. California Institute for Regenerative Medicine

  2. What is a stem cell? 1. Mature/specialize 2. Self-renew

  3. Stem Cell Capacity

  4. Stem Cell Capacity Human embryonic stem cell lines were first derived in 1998 by Dr. James Thompson.

  5. Embryonic Stem Cellsvia In Vitro Fertilization

  6. Potential of Stem Cell Research • Tissue/cell replacement • Gene therapy/drug delivery • Models of disease in vitro • Drug screening and drug development • Basic knowledge of human development An enabling technology for: A path to new therapies and cures for many diseases

  7. Somatic Cell Nuclear Transfer

  8. Scale of a Human Egg

  9. 2001 Presidential Executive Order for Embryonic Human Stem Cell Research • Prohibits use of Federal funds on embryonic stem cell lines derived prior to August 9, 2001 • Lines must have been derived from unused embryos that were made for IVF

  10. States Respond • California: California Institute of Regenerative Medicine • Connecticut: Connecticut Stem Cell Research Grant Project • Illinois: Illinois Regenerative Medicine Institute • Maryland: Maryland Stem Cell Commission • Massachusetts: Governor's Life Science Initiative • Minnesota: Stem Cell Institute • New Jersey: Stem Cell Institute of New Jersey • New York: Empire State Stem Cell Trust Fund • Ohio: Center for Stem Cell and Regenerative Medicine

  11. Proposition 71 • Approved by 59% of CA voters • Authorized $3 billion to fund stem cell research in CA • Affirmed the right to conduct research not supported by federal funding • Banned reproductive cloning • Required development of medical and ethical standards

  12. Independent Citizens Oversight Committee (ICOC): 29 members • Chair: Robert Klein • Vice-Chair: Ed Penhoet, Ph.D • California Institute for Regenerative Medicine (CIRM): 50 member staff (currently 26) • President: Alan Trounson, Ph.D.

  13. Mission Statement To support and advance stem cell research and regenerative medicine under the highest ethical and medical standards for the discovery and development of cures, therapies, diagnostics and research technologies to relieve human suffering from chronic disease and injury. “Turning stem cells into cures.” Roman Reed

  14. Working Groups of the CIRM • Standards and Ethics Working Group (19) • (prominent ethicists, scientists, patient advocates) • Grants Review Working Group (23) • (distinguished scientists from outside California, patient advocates) • Facilities Working Group (11) • (real estate experts; patient advocates)

  15. Building a State Agency Grants management Compliance Tracking Procedures Regulations and a Funding Agency

  16. Legal Challenges For over two years legal challenges prevented the institute from issuing bonds • Two consolidated lawsuits challenged our constitutional authority to spend state money • Very strong decision in Superior Court in May 2006 upholding CIRM position; appealed • In May 2007, the California Supreme Court declined to hear appeal and ended the legal challenge • A third lawsuit (dismissed) asserted that we are depriving frozen embryos of their constitutional rights

  17. Funds for a Funding Agency Bond Anticipation Notes (BANs) -Authorized $14 M in BANs in April, 2006 for first year of training grants; -Additional $36 M in BANs for research grants. Governor’s Loan $150M loan provided to CIRM by Governor Schwarzenegger in July, following Presidential veto on Castle-DeGette bill

  18. Making a plan

  19. Scientific Strategic Plan • Define long-term objectives that CIRM will pursue over ten years • Involved interviews with scientists, clinicians, ethicists, patient advocates, public interest groups • Focus group discussions and public meetings • Heard from ~200 individuals • A “living plan” with mechanisms for review and modification Published December 2006

  20. Laying the Foundation Preparing for the Clinic Clinical Research Scientific Training & Development Innovation Science Mission-Oriented Science Tools, Technologies & Infrastructure Facilities Communities of Science Responsibility to the Public Funding Initiatives Strategic Planning Framework Resources

  21. Strategic Plan Goals • Aspirational Goals: • What we hope to achieve • Use stem cells to cure disease • California as world-wide leader in stem cell research • Commitment Goals: • Our covenant with the people of California for what we will achieve over the next ten years

  22. Commitment Goals: Context • Scientifically young field • Therapeutic drug development: takes time and fails more often than it succeeds • New treatment modality: cellular therapy

  23. Commitment Goals • Focused on human embryonic stem cells, with emphasis on cell replacement therapy • Ten year goals • Goal 1: Clinical proof of principle that transplanted cells derived from pluripotent cells can be used to restore function for at least one disease. • Goal 2: Therapies based on stem cell research in Phase I or Phase II clinical trials for 2-4 additional diseases

  24. Laying the Foundation

  25. CIRM Training Program • 16 non-profit institutions in California • 169 Trainees (pre-doc, post-doc, clinical) • Course in stem cell biology • Course in ethical, legal, and social issues • Annual meeting of trainees • Grants awarded in April, 2006 • Total ~$38M for 3 years

  26. “Jumpstart” Initiative to Enable Stem Cell Research • CIRM SEED Grants • $200K/yr, 2 yrs • Innovative projects • Comprehensive Research Grants • $400K/yr, 4 yrs • Established investigators in SCB/related field • Shared Laboratory Space • Fund renovation of lab space for hESC work • Fund instructional course

  27. Latest Initiatives • New Faculty Awards • Enable young faculty scientists and physicians to • New Cell Lines Awards • Enable the development of new pluripotent stem cell lines for research and therapies • Disease Teams • Create teams of researchers

  28. What’s Next New President New Initiatives Develop for-profit funding Community outreach Growing the Institute

  29. Acknowledgements • Bob Klein • Zach Hall • Arlene Chiu • Alan Trounson • Rich Murphy • CIRM Team • All those who have generously contributed their time and expertise to making CIRM and the vision embodied in Proposition 71 a reality

  30. Scientific Challenges • Capabilities of different types of stem cells • Control division in vitro and in vivo • Control paths of differentiation • Safe production of large numbers of cells • Immunological tolerance • Production of SC lines with disease phenotypes

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