Drug Discovery in ALS: Opportunities and Challenges

描述(申请人提供)：肌萎缩侧索硬化症是一种毁灭性的疾病，从确诊之日起2-5年内导致患者死亡。尽管做出了巨大的努力来阻止疾病的发展，但利鲁唑是市场上唯一只将患者的生存时间延长2-3个月的药物。找到一种可以阻止这种疾病的药物，不仅取决于对疾病潜在机制的理解，还取决于将基础研究的结果转化为临床的努力。肌萎缩侧索硬化症协会正在组织一个为期三天的研讨会，题为“肌萎缩侧索硬化症的药物发现：机遇和挑战”。研讨会的重点是让肌萎缩侧索硬化症研究和相关领域的临床和科学专家分享他们的经验和知识，以确定和克服肌萎缩侧索硬化症翻译研究的挑战。该研讨会还为职业生涯非常早期的科学家提供了一个机会，让他们对药物开发有一个全球视野，并了解如何将基础研究转化为可销售的药物。研讨会议程由来自学术界、产业界、肌萎缩侧索硬化症协会和加拿大肌萎缩侧索硬化症协会的指导委员会成员以及NINDS项目主任阿梅莉·古比茨博士共同选择，旨在提供肌萎缩侧索硬化症研究和肌萎缩侧索硬化症药物开发目标的概述。肌萎缩侧索硬化症专家将介绍肌萎缩侧索硬化症研究的现状和用于肌萎缩侧索硬化症药物开发的策略。来自业界的专家将分享在其他疾病领域用于药物开发的各种战略的信息。该计划包括三个讨论小组：1.行业和学术伙伴关系；2.加强Biomarker努力的机会；3.临床试验网络。第一个翻译版本 ALS研讨会于2008年1月由ALS协会主办，NINDS和罕见疾病办公室共同资助。自那时以来，已经开始了几个新的ALS临床试验，更多的生物技术公司正在关注ALS。这次研讨会提供了一个极好的机会来扩大上一次成功的会议，并介绍了新的讨论领域。研讨会的规模相对较小(约85人参加)，为来自学术界和工业界的科学家创造了一个激励和互动的环境，让他们分享新的想法和概念，并弥合基础研究和药物开发之间的差距。会后报告将发表在同行评议的科学期刊上，与整个肌萎缩侧索硬化症社区分享研讨会成果。

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.