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Prize4Life: Inducement Prizes for ALS (Lou Gehrig’s Disease) Nicole Szlezak Member, Board of Directors Prize4Life

Prize4Life: Inducement Prizes for ALS (Lou Gehrig’s Disease) Nicole Szlezak Member, Board of Directors Prize4Life. What is ALS (Lou Gehrig’s Disease)?. Progressive paralysis of unknown cause, 100% lethal Death within 3-5 years from diagnosis (respiratory failure)

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Prize4Life: Inducement Prizes for ALS (Lou Gehrig’s Disease) Nicole Szlezak Member, Board of Directors Prize4Life

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  1. Prize4Life: Inducement Prizes for ALS (Lou Gehrig’s Disease) Nicole Szlezak Member, Board of Directors Prize4Life

  2. What is ALS (Lou Gehrig’s Disease)? • Progressive paralysis of unknown cause, 100% lethal • Death within 3-5 years from diagnosis (respiratory failure) • ~30.000 individuals are affected in the U.S. at any given time • No cure exists, only existing treatment prolongs life by 3 months (Rilutek) • http://www.dollar4life.org/?p4l-hbs-004 Source: www.nwabr.org Source: web

  3. ALS is an orphan disease. Patients die so fast that total ALS “patient pool” is relatively small (30.000/120.000 in US/world) Annual investment in ALS research is comparatively low (~$60 million USD worldwide) Tools that industry needs for systematic, large-scale drug development are not in place Why is There no Effective ALS treatment?Some Reasons… We know little about the causes of ALS ALS CASES 100% 90% “sporadic” ? 10% inherited

  4. ALS Therapy Development is Risky Human Trials Certainty ? Mouse model ? target ALS clinical trials are expensive due to lack of suitable biomarker. Large intra- and interpersonal variablity of ALS symptoms makes disease progression hard to measure. Most frequently used biomarker = survival time Requires long (=expensive) trials Questionable predictiveness of existing ALS mouse model Causes of ALS unknown, targets uncertain Cost

  5. Universities/ Research Institutions Biotech – Pharma Health Systems Hospitals Health Services Innovation in Basic Research often doesn’t get translated into direct benefit for patients Research -–- Development --- Access: “Valley of Death” = gap between academic research and industry involvement Without industry commitment, basic research does not get translated into tangible results for patients

  6. Prizes as “Lighthouse”: Prize4Life prizes highlight scientific breakthroughs that will accelerate ALS therapy development Triple mission: Accelerate existing efforts in ALS research Bring in new ideas and new minds Complement existing funding models Draw attention to ALS research Raise funding from heretofore untapped sources Prize4Life’s Approach : Can we make ALS Breakthroughs more likely?

  7. Founded in 2006; US 501c3 status in 2007 Mission: To accelerate the discovery of a treatment and a cure for ALS by using powerful incentives to attract new people and new ideas and to leverage existing efforts and expertise in the ALS field Prize4Life: Background/Structure

  8. The Prizes

  9. The Prize Design and Selection Process • Step 1: Prize4Life develops prize criteria in cooperation with our Scientific Advisory Board and input from the scientific community • Step 2: Prize4Life launches Prize • Step 3: Scientific Advisory Board selects winner/s according to pre-set criteria

  10. Robert H. Brown, M.D., D. Phil, Professor of Neurology, Harvard Medical School Lucie Bruijn, PhD., Science Director and Vice Presiedent, the ALS Association (ALSA) Valerie Estess, Director of Research and Cofounder, Project A.L.S. Adrian Ivinson, PhD., Director, Harvard NeuroDiscovery Center at Harvard Medical School Tom Maniatis, PhD., The Thomas H. Lee Professor of Molecular and Cellular Biology, Harvard University David P. Meeker, M.D., President, Lysosomal Storage Disorders, Genzyme Therapeutics Alfred Sandrock, M.D., Ph.D., Senior Vice President of Neurology R&D, Biogen Inc. Prize4Life’s Scientific Advisory Board represents academia, industry and patient associations

  11. Raised > 4.5 Million USD since 2006 First prize (Prize4Life ALS Biomarker Prize) launched in November 2006 on InnoCentive $1.000.000 for an ALS Biomarker that will reduce the cost of ALS clinical trials Teams from different disciplines are currently working to submit their solutions Deadline: November 2008 Five small “idea prizes” awarded in May 2007 to researchers who submitted theoretical solutions to finding a biomarker Several of these proposals have generated new, interesting projects for the development of an ALS biomarker (new minds) Preparing to launch second prize (2008) Progress

  12. Outreach to the scientific community Reach researchers from a wide variety of fields and disciplines Overcome scepticism Resources Upfront funding: how can we support researchers interested in winning our prizes in their search to find upfront funding? Other resources Challenges

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