Drug Discovery in ALS: Opportunities and Challenges

描述（由申请人提供）：ALS是一种毁灭性疾病，患者在诊断后2-5年内死亡。尽管为阻止疾病的进展做出了巨大的努力，利鲁唑是市场上唯一一种仅能延长患者生存2-3个月的药物。找到一种能够阻止这种疾病的药物，不仅要了解这种疾病的潜在机制，而且还要努力将基础研究的发现转化为临床应用。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“肌萎缩侧索硬化症药物发现：机遇与挑战”。研讨会的重点是邀请ALS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ALS转化研究面临的挑战。本次研讨会还为处于职业生涯早期阶段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，并学习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、工业界、ALS协会和加拿大ALS协会的指导委员会成员与NINDS项目主任Amelie Gubitz博士共同选择，旨在概述ALS研究和药物开发的ALS靶点。ALS专家将介绍ALS研究的现状和正在用于ALS药物开发的策略。来自产业界的专家将分享关于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。工业和学术伙伴关系；2. 加强生物标志物工作的机会；3. 临床试验网络。第一次翻译

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.