Drug Discovery for Neurodegeneration

描述（由申请人提供）：阿尔茨海默病药物发现基金会（ADDF）会议题为神经退行性疾病药物发现将于2008年2月4-5日在华盛顿特区举行。会议的目的是通过培训下一代科学领袖，将基础研究转化为新的治疗干预措施，促进神经退行性疾病的药物发现。关于这一主题的正规教育是必要的，因为在这一研究领域存在记录在案的知识缺口，而且在制药业之外，科学家了解这些过程的机会很少。会议的目标与NIH的路线图一致：弥合药物发现和神经退行性疾病方面的知识差距；将跨学科的科学家聚集在一起，激发结合技能和学科的新方法；为这些科学家提供交流思想和资源的网络机会；并在同行评议的科学杂志上发表论文。这个为期两天的会议将为与会者提供有关创造治疗和预防神经退行性疾病的新药的基本知识和资源。它还将解决与以下子主题相关的障碍和挑战：目标识别和验证；引线识别及优化；先导化合物;临床前概念验证和开发；促进药物发现的资源和服务；神经退行性疾病药物开发中的疾病特异性问题。阿尔茨海默病、帕金森病、亨廷顿病和肌萎缩性侧索硬化症将包括在讨论中。会议将包括3到5个演讲，并有充足的问答时间。关于路线图的公私伙伴关系倡议，亚洲发展基金会将邀请来自学术界、生物技术和制药行业以及美国国立卫生研究院的大约25名国际公认的科学家担任主席或发言人，其中包括妇女和少数民族。预计本次会议将为来自不同背景的150名科学家提供服务，其中27%将是女性，少数民族，年轻/初级研究人员和研究生。ADDF寻求解决的公共卫生问题是减少神经退行性疾病的发病率，如阿尔茨海默病、帕金森病、亨廷顿病和肌萎缩性侧索硬化症。通过举办这次会议，ADDF将增加科学家对药物发现过程的了解，并促使他们共同努力开发商业上可行和有效的疾病修饰产品。

DESCRIPTION (provided by applicant): The Alzheimer's Drug Discovery Foundation's (ADDF) conference entitled Drug Discovery for Neurodegeneration will be held on February 4-5, 2008 in the Washington, DC area. The purpose of the conference is to advance drug discovery for neurodegenerative disease by training the next generation of scientific leaders on the process of translating basic research into novel therapeutic interventions. Formal education on this topic is needed because there is a documented knowledge gap in this area of research and there are very few opportunities outside the pharmaceutical industry for scientists to learn about these processes. The goals of the conference are consistent with the NIH's Roadmap: to bridge the knowledge gap in drug discovery and neurodegenerative disease; to bring together interdisciplinary scientists to stimulate new ways of combing skills and disciplines; to provide networking opportunities for these scientists to exchange ideas and resources; and to publish the proceedings in a peer-reviewed scientific journal. This two-day conference will give participants fundamental knowledge and resources on creating new drugs to treat and prevent neurodegenerative disease. It will also address the barriers and challenges associated with the following sub-topics: Target Identification & Validation; Lead Identification & Optimization; Lead Discovery; Pre-Clinical Proof-of-Concept & Development; Resources and Services for Advancing Drug Discovery; and Disease Specific Issues in Drug Discovery for Neurodegenerative Disease. Alzheimer's disease, Parkinson's disease, Huntington's disease, and Amyotrophic Lateral Sclerosis will be included in the discussions. Sessions will consist of 3 to 5 presentations with ample time for Q&A. With respect to the public-private partnership initiative of the Roadmap, ADDF will invite approximately 25 internationally recognized scientists, including women and minorities, from academia, the biotechnology and pharmaceutical industries, as well as the NIH, to serve as chairs or speakers. It is anticipated that this conference will serve an audience of 150 scientists from diverse backgrounds, of which 27% will be women, minorities, young/junior investigators, and graduate students. The public health problem ADDF seeks to solve is to reduce the incidence of neurodegenerative diseases, such as Alzheimer's disease, Parkinson's disease, Huntington's disease and Amyotrophic Lateral Sclerosis. By offering this conference, ADDF will increase scientists' knowledge of the drug discovery processes and catalyze them to work together to develop commercially viable and effective disease modifying products. CTITIQUE 1: Objectives: Training of academic scientists in drug discovery, exchange of ideas, stimulate pre-clinical research, build public-private partnerships, publish proceedings, provide CME credits. 6 topics: targets, leads identification, lead optimization, pre-clinical POC, drug discovery resources, disease-specific issues. The confirmed and proposed speakers include senior and well recognized successful scientists in drug development from both industry and academics. Overall, the proposed conference would likely be quite useful to its targeted audience of academic investigators and would likely accomplish its goal of educating these investigators in drug development and would facilitate public-private partnerships. These activities would strengthen the quality of translational proposals submitted to the NIH. The four targeted disease areas of AD, PD, HD and ALS are quite appropriate for the neurodegenerative field. The focus of training academic scientists in the full range of drug discovery and development from target validation through IND work remains rare although IBC sponsors conferences that include some of these areas. It would be helpful to point out in more specific terms how the proposed conference compares to and is advantageous to these other conferences. The range and expertise of speakers could be broadened by decreasing some of the potential redundancy in which two or more speakers are from the same institution. The proportion of speakers who have actually made a direct and major contribution to the successful development of an approved drug should be increased. Budget: The overall budget is appropriate and the amount requested from NIA (less than half the total cost of the conference) is appropriate. Inclusion of women and minorities is appropriate.