Drug Discovery in ALS: Opportunities and Challenges

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DESCRIPTION (provided by applicant): ALS is a devastating disease leading to the death of patients in 2-5 years from the time of diagnosis. Despite tremendous efforts to halt the progression of the disease, Riluzole is the only drug on the market which prolongs the survival of patients by only 2-3 months. Finding a drug that would halt the disease depends on understanding the mechanisms underlying the disease but also on efforts to translate findings in basic research into the clinic. The ALS Association is organizing a three day workshop entitled "Drug Discovery for ALS: Opportunities and Challenges". The focus of the workshop is to bring clinical and scientific experts in ALS research and related fields to share their experience and knowledge in order to identify and overcome challenges to ALS translational research. This workshop also provides an opportunity for scientists at a very early stage in their career to have a global vision on drug development and to learn how basic research can be translated into a marketable drug. The workshop agenda selected by the steering committee members from academia, industry, The ALS association and The ALS Society of Canada, together with Dr. Amelie Gubitz, NINDS program director, is designed to provide an overview of ALS research and ALS targets for drug development. ALS experts will present the current status of ALS research and strategies that are being used for drug development in ALS. Experts from industry will share information on the various strategies used for drug development in other disease areas. The program includes three discussion panels: 1. Industry and Academic Partnerships; 2. Opportunities to Enhance Biomarker Efforts; 3. Clinical Trial Networks. The first translational workshop for ALS was hosted by The ALS Association and co- funded by NINDS and The Office of Rare Disease in January, 2008. Since this time several new clinical trials for ALS have been initiated and many more biotechs are focusing on ALS. This workshop provides an excellent opportunity to expand on the prior successful meeting and introduces new areas for discussion. The relatively small size of the workshop (around 85 participants) creates a stimulating and interactive environment for scientists from academia and industry to share new ideas and concepts and to bridge the gap between basic research and drug development. A post-meeting report will be published in a peer-reviewed scientific journal to share workshop outcomes with the entire ALS community. PUBLIC HEALTH RELEVANCE: The goal of the workshop is to identify key barriers and enablers for translating findings in basic research into the clinic for ALS. It is an opportunity fr clinician and scientific leaders in industry and academia in neurodegeneration to share their findings and discuss the future directions and opportunities for ALS drug discovery.