Stem Cells   A Technology Assessment June2009
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Stem Cells A Technology Assessment June2009

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Stem Cells   A Technology Assessment June2009 Stem Cells A Technology Assessment June2009 Presentation Transcript

  • June 1 st , 2009 STEM CELLS Jie He Tony Jia Ji Young Lee Andy Yen
  • HISTORICAL PERSPECTIVE AND SCIENTIFIC BACKGROUND
    • Timeline
      • 1963 – Discovered by McCulloch and Till
      • 1981 – Mouse Embryonic Stem Cells Isolated
      • 1998 – First Human Embryonic Cell Line Developed
    • Scientific Background
      • Self-Renewing
      • Pluripotent
      • Two Main Types
        • Adult
        • Embryonic
  • SCIENTIFIC BACKGROUND
    • Advantages/Disadvantages
      • Embryonic Stem Cells will divide indefinitely
      • Adult Stem Cells have a finite lifetime
      • Not all tissues contain Adult Stem Cells
      • Adult Stem Cells are not controversial
      • Embryonic cell lines
      • Media
      • Reagents
      • Safety
  • RESEARCH METHODOLOGY
    • Lots of Primary Research
      • Industry and Academia
    • Some supporting Secondary Research
    • Issues:
      • How Big?
      • Future Potential
      • Obstacles
      • Driving Forces
      • Most Promising Opportunities
      • Intellectual Property
      • Commercialization
      • Business Model
  • LIST OF INTERVIEWS CONDUCTED
    • Dr. David Baltimore
      • Robert Andrews Millikan Professor of Biology at Caltech, Nobel La ureate
    • Dr. Martin F. Pera
      • Director of Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research at USC
    • Dr. Agnes Lukaszewicz
      • Postdoctoral Scholar for Dr. David Anderson (Founder of StemCells, In c.) at Caltech
    • Dr. Owen Witte
      • Founding Director of Eli and Edythe Broad Center of Regener ative Medicine and Stem Cell Research at UCLA
    • Dr. Marie Csete
      • Chief Scientific Officer of California Institute of Regenerative M edicine
    • Kenneth Aldrich
      • Chairman and CEO of International Stem Cell Corporation
  • S CURVE ANALYSIS
  • DEVELOPMENT CYCLE
    • Three Phases
    • Biotech – long, complicated, expensive development cycle
    • Stem Cells
      • Complexity of cellular biology
      • Risks of new, unproven therapy
    Research Testing/Approval Commercialization
  • DEVELOPMENT - RESEARCH
    • Where is this research taking place and why?
  • DEVELOPMENT - RESEARCH
    • Most research is in academia
    • CIRM grant data
    • Most Stem Cell companies NOT involved in research.
    • Research is still in the early stages.
    • Current trend, private sector unwilling to invest until Stage 2 or Stage 3
    • Too expensive and complicated
    • Low success rate
  • DEVELOPMENT – TESTING AND APPROVAL
    • Human use – requires rigorous testing and approval
    • No precedent, very unique procedure
    • A form of personalized medicine
    How can FDA regulate biotech they have no experience in? GMP Labs Researchers must educate the FDA Physician Initiated Trials
  • COMMERCIALIZATION
    • Bone Marrow Transplantation
      • Not a Commercial Product
        • Fixed Cost
        • Similar to existing treatments
          • No potential for business applications
  • COMMERCIALIZATION
    • Month
    • Kidney Transplantation
      • Immune response due to incompatibilities
        • Expensive Immunodepressant have to be taken throughout lifetime
      • Stem cell technologies avoids it
        • Cost reduced dramatically
  • COMMERCIALIZATION
    • Windpipe Transplant
  • COMMERCIALIZATION
    • Patents
      • WARF – Preparation of primate and human embryonic stem cells
      • Geron – Cell therapy and Drug screening applications using cells derived from hESC
      • Kyoto University– Derivation of iPS cells from a somatic cell
    • Time
      • Estimates vary, Hard to predict
      • Depends on Therapy/Application
        • Macular degeneration: 3 - 5 years
  • POTENTIAL MARKETS
    • Stem Cell Therapy
      • Most Obvious
      • Most Future Potential
    • Peripherals
      • Media and Reagents
      • Supplies
      • Cultures
    • Disease Study and Drug Development
      • Unlimited Supply of Human Cells
      • Use Stem Cells to test drugs
      • Safer, more Efficient
    • Most VC+Startup is in the latter 2
  • DRIVERS
    • Mainly Intellectual Curiosity
    • Later:
      • When more money is involved
      • Demand for Cures
      • “ Money, Fame, Sex.” –David Baltimore
  • HOW BIG AND THE NEXT BIG THING
    • Lots of Funding at USC/UCLA
    • CIRM Funding
    • 180+ Startups
    • Ken Aldrich – $100 billion market after required FDA approvals.
    • Next Big Thing
      • Induced Pluripotent Stem Cells (iPS)
      • Macular Degeneration
      • Advanced Melanoma
  • IPS CELLS
    • Essentially Pluripotent Stem Cells Derived from non-Pluripotent Adult Cells
      • Uses Skin Cells
    • Ethical Considerations
    Advantages Disadvantages Not Limited by Existing Cell Lines Low Efficiency Can be Derived Universally High Cost Genetic Compatability Very New
  • MACULAR DEGENERATION
    • Possible immediate applicability
    • Leading cause of blindness and vision loss for elderly people
    • Advantages
      • Eye is a small organ
      • Small number of cells required
      • Wide Applicability
      • Optical Tools Exist to Observe Results
      • Eye is a noncritical organ
    Age Percentage Afflicted 66-74 10% 75-85 30%
  • Advanced Melanoma
    • Already in Clinical trials
    • Promising Results
    • Stem Cells used as messengers to modify patient’s immune system
    • Uses Adult Stem cells
    • Does not require detailed understanding of how stem cells interact with human tissue.
    • Easier to get approval for Stage 4 patients
  • ROADBLOCKS/RISKS
    • Cancer
      • Teratoma
    • Dangers with iPS cells
      • Oncogene
      • retrovirus
    • These are not “killer” problems
    • Biggest issue – sheer complexity of human body
    • High cost of production
      • Stanford GMP facility – $200M
  • BUSINESS MODEL
  • BUSINESS MODEL
  • CONCLUSIONS
    • Lots of therapies are possible
      • Some are close: Macular Degeneration (3 years)
      • Some are far off: Neural Diseases (5+ years)
      • Impossible to Tell
      • Peripherals Market
    • Limited by Regulation, Cost, and Science
    • Too Early for Private Investors
      • Government Funding is Crucial for Basic Research and Commercialization
    • When it reaches the Market, initial cost will be high but decrease over time