Accelerating ALS Research: Translating Basic discoveries into Therapies for ALS

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。ALS协会将于2008年1月13日至16日在佛罗里达州坦帕市组织为期三天的研讨会，主题为“加速ALS研究：将基础发现转化为ALS治疗方法”。主要目标是确定将基础研究成果转化为ALS临床的关键障碍和推动因素。ALS研究专家和其他疾病药物发现的领导者将分享他们的经验和知识，以确定和克服ALS转化研究的挑战。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、NINDS和ALS协会的指导委员会成员选定，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。认识到成功开发针对ALS等疾病的药物的最大障碍是将学术界的重要发现转化为临床的能力，会议将以一天的重点讨论这些障碍，并研究ALS和其他疾病的成功和失败，以提供未来推动药物发现的方向。这是第一次组织以ALS药物开发为重点的转化研究研讨会。研讨会旨在促进小组讨论和互动，以确定ALS药物发现的机会和未来方向。研讨会规模相对较小（约85名参与者），ALS专家和业界领袖之间的平衡旨在为科学家创造一个激励和互动的环境，以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ALS社区分享研讨会成果。公共卫生相关性：研讨会的目标是确定将基础研究成果转化为ALS临床的关键障碍和推动因素。这是ALS专家和药物开发领导者分享他们的发现并讨论ALS药物发现的未来方向和机会的机会。

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 "Accelerating ALS Research: Translating Basic Discoveries into Therapies for ALS" in Tampa, Florida, January 13- 16, 2008. The main goal is to identify key barriers and enablers for translating findings in basic research into the clinic for ALS. Experts in ALS research and leaders in drug discovery for other diseases will 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, NINDS and The ALS association, 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. Recognizing that the most significant hurdle to successful development of drugs for diseases such as ALS is the ability to translate important findings in academia to the clinic, the meeting will conclude with a focused day to discuss these barriers and look at successes and failures in ALS and other diseases to provide future directions to move drug discovery forward. This is the first time a workshop on translational ALS research focused on drug development for ALS is being organized. The workshop aims to stimulate group discussions and interactions, to identify opportunities and future directions for ALS drug discovery. The relatively small size of the workshop (around 85 participants) and the balance between ALS experts and leaders from industry are designed to create a stimulating and interactive environment for scientists 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 for ALS experts and leaders in drug development to share their findings and discuss the future directions and opportunities for ALS drug discovery.