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13 th December 2006

Stem Cells Hope or Hype?. Paul Rodgers. 13 th December 2006. Ithaka Life Sciences Ltd. Business started in 2000 Commercialising life science technologies: Business creation Technical, commercial and market evaluation Intellectual property review

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13 th December 2006

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  1. Stem Cells Hope or Hype? Paul Rodgers 13th December 2006

  2. Ithaka Life Sciences Ltd • Business started in 2000 • Commercialising life science technologies: • Business creation • Technical, commercial and market evaluation • Intellectual property review • Business plans: due diligence and drafting • Business development, M&A, licensing, disposal • Sourcing investment finance • Interim management

  3. Ithaka Life Sciences Ltd • Stem Cells • Axordia Ltd • CereStem Ltd • British Stem Cell Registry Ltd • Centre of Excellence for Life Sciences Ltd • Plasticell Ltd • bioProcess UK • Cardiff University • Manchester University

  4. Overview • What’s the problem? • Hype? • Reality? • Hope? • Conclusions

  5. What’s the problem?

  6. Unmet Medical Need • Diabetes • 12.1 million sufferers in US (2002) • Costs $132 billion p.a. (2002) rising to $156 billion p.a. (2010) • Cardiovascular disease • 64.4 million cases in US (2004) • Costs $368.4 billion p.a. (2004) • Parkinson’s Disease • 100,000 sufferers in UK • Treatment costs £600 million p.a. (1998)

  7. Degenerative Diseases • Other examples • Alzheimer’s, spinal cord injuries, Amyotrophic Lateral Sclerosis, multiple sclerosis, liver diseaseetc • Restoration of cell or organ function • Current drugs don’t treat the cause • Organ transplants • Expensive, donor shortages, not applicable to many diseases

  8. Hype

  9. Embryonic Stem Cells • Immortal • A single cell line is all you need • Precursors of all cell types • Can produce any cell you need • Can divide without limit • Unlimited supply

  10. Adult Stem Cells • Can be isolated from most tissues • e.g. bone marrow, cord blood, fat, skin etc • Generate cell types of tissue of origin • Produce useful cells for therapies • Plasticity • Can be coaxed into forming cells of completely different tissues • e.g. neurons from blood cells

  11. Reality

  12. Embryonic Stem Cells: Issues (1) • Derivation • Ethics and logistics (is SNCT a solution?) • Manufacture • Feeder cells; TSE; genetic stability; GMP • Safety • Uncontrolled cell proliferation; reprogramming; immune rejection • Differentiation • Reproducible production of desired cell type with appropriate functionality (typical yield <10%)

  13. Embryonic Stem Cells: Issues (2) • Efficacy • Dose, delivery and functionality • Economics • Uncertain regulatory requirements • Freedom to operate

  14. Embryonic Stem Cells Survey • ES cell therapy is 10-15 years away • Will be used to treat certain diseases • Diabetes, cardiovascular, single gene defects, MS, Alzheimer’s, Parkinson’s, liver disease, spinal cord injury, retinal disease • Schering and Merck are the only pharma companies to say they are interested at this stage

  15. Adult Stem Cells: Issues • Derivation • Tissues other than bone marrow and cord blood? • Differentiation • Reproducible production of cells of completely different tissues • Therapeutic benefit • Implanted cells often die due to immune attack • Unexpected side effects (e.g. foetal neurons in Parkinsons) • Economics of autologous cell therapy

  16. Hope

  17. Islet Cell Therapy • Edmonton protocol (1999) • Cells injected into liver via hepatic portal vein under local anaesthetic • >300 patients: 84% success rate reported (insulin-free) • But • Immunosuppressive drugs required • Requires 2-4 pancreata per patient • Only 1,100 pancreata available for 1.1 million Type 1 diabetes sufferers in US • Side effects and decline in performance with time

  18. Islet Cell Therapy

  19. Clinical Use of Adult Stem Cells • Bone marrow transplants • Replace blood cells ablated by cancer therapies • ~ 300 clinical studies on haematopoietic stem cells • Cell expansion is a key issue • Haematopoietic stem cells may be able to restore function to damaged cardiac tissue (3 trials so far) • Neural stem cell trial started in 2006 • Batten’s disease (Stem Cells Inc.) • Developments in China and Korea

  20. Brain Repair: Neural Stem Cells

  21. Progress with ES Cells • Manufacture • Feeder cell-free culture of human ES cells • Efficacy • Cardiomyocytes derived from human ES cells can restore some myocardial function in a pig model of cardiac disease • Clinical studies may start in 2007 • Congenital heart failure (ES Cell), spinal cord injury (Geron) or diabetes (Geron)

  22. But……….. • All the key issues remain to be addressed • How will clinical grade cells be produced economically and used effectively? • Regulatory uncertainty • Registration requirements • Ethical issues (especially in USA)

  23. Conclusion • We still have mountains to climb • Alps for adult stem cells • Himalayas for embryonic stem cells

  24. Information Sources http://stemcells.nih.gov/info/basics/ www.stemcellresearchnews.com/ www.aastrom.com www.neuronova.com www.athersys.com www.novocell.com www.cellartis.com www.stemcellsinc.com www.escellinternational.com www.gamida-cell.com www.geron.com www.stemcellsciencesltd.com Nature Biotechnology July 2005 issue

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