Stem Cells in Science and Medicine

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Enabling Stem Cell Research in California

• Gil Sambrano, Ph.D.
• California Institute for Regenerative Medicine

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What is a stem cell?
1. Mature/specialize 2. Self-renew
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Stem Cell Capacity
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Stem Cell Capacity
Human embryonic stem cell lines were first
derived in 1998 by Dr. James Thompson.
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Embryonic Stem Cellsvia In Vitro Fertilization
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Potential of Stem Cell Research
An enabling technology for

• Tissue/cell replacement
• Gene therapy/drug delivery
• Models of disease in vitro
• Drug screening and drug development
• Basic knowledge of human development

A path to new therapies and cures for many
diseases
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Somatic Cell Nuclear Transfer
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Scale of a Human Egg
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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

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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

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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

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• 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.

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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
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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)

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

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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.
• Governors Loan
• 150M loan provided to CIRM by Governor
  Schwarzenegger in July, following Presidential
  veto on Castle-DeGette bill

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Making a plan
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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
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Strategic Planning Framework
Resources
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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

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Commitment Goals Context

• Scientifically young field
• Therapeutic drug development takes time and
  fails more often than it succeeds
• New treatment modality cellular therapy

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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

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Laying the Foundation
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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

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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

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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

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Whats Next

• New President
• New Initiatives
• Develop for-profit funding
• Community outreach
• Growing the Institute

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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

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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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