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

 Progressive paralysis of unknown cause, 100% lethal  Death within 3-5 years from diagnosis (respiratory failure)  ~ individuals are affected in the U.S. at any given time  No cure exists, only existing treatment prolongs life by 3 months (Rilutek)  Source: webSource: What is ALS (Lou Gehrig’s Disease)?

Why is There no Effective ALS treatment? Some Reasons… 90% “sporadic” ? 10% inherited  ALS is an orphan disease. Patients die so fast that total ALS “patient pool” is relatively small (30.000/ 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 ALS CASES 100% We know little about the causes of ALS

Human Trials ? Mouse model ? target ALS Therapy Development is Risky Cost Certainty Causes of ALS unknown, targets uncertain Questionable predictiveness of existing ALS mouse model 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

Biotech – Pharma Universities/ Research Institutions Health Systems Hospitals Health Services 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 Innovation in Basic Research often doesn’t get translated into direct benefit for patients

Prize4Life’s Approach : Can we make ALS Breakthroughs more likely? 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: Background/Structure  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

The Prizes PrizeDescriptionDurationAmount Biomarker/ Clinical Trials Outcome Measure The discovery of one or more biological parameters that allow clinicians to measure how fast ALS progresses in a patient. 2 years after launch $1 Million Launched Nov 6, 2006 New ALS Treatment Candidates New treatments that have been proven effective in the ALS mouse model. 2 years after launch $2.5 Million ALS Biological Target The discovery of one or more of the biological mechanisms that cause ALS. 2 years after launch $5 Million

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

Prize4Life’s Scientific Advisory Board represents academia, industry and patient associations  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.

Progress  Raised > 4.5 Million USD since 2006  First prize (Prize4Life ALS Biomarker Prize) launched in November 2006 on InnoCentive  $ 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)

Challenges  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