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Stem Cells in Science and Medicine

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Connecticut: Connecticut Stem Cell Research Grant Project ... 'Turning stem cells into cures.' Roman Reed. Working Groups of the CIRM ... – PowerPoint PPT presentation

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Title: Stem Cells in Science and Medicine


1
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2
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
5
Stem Cell Capacity
6
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
10
Somatic Cell Nuclear Transfer
11
Scale of a Human Egg
12
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

13
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

14
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

15
  • 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.

16
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
17
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)

18
Building a State Agency
Grants management Compliance Tracking
Procedures Regulations
and a Funding Agency
19
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

20
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

21
Making a plan
22
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
23
Strategic Planning Framework
Resources
24
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

25
Commitment Goals Context
  • Scientifically young field
  • Therapeutic drug development takes time and
    fails more often than it succeeds
  • New treatment modality cellular therapy

26
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

27
Laying the Foundation
28
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

29
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

30
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

31
Whats Next
  • New President
  • New Initiatives
  • Develop for-profit funding
  • Community outreach
  • Growing the Institute

32
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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