Stem Cells A Technology Assessment June2009


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Stem Cells A Technology Assessment June2009

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