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）题为神经变性药物发现的会议将于2008年2月4日至5日在华盛顿地区举行。会议的目的是通过培训下一代科学领导人将基础研究转化为新型治疗干预措施的过程来推进神经退行性疾病的药物发现。需要关于这一主题的正规教育，因为在这一研究领域存在有据可查的知识差距，而且在制药行业之外，科学家很少有机会了解这些过程。会议的目标与NIH的路线图是一致的：弥合药物发现和神经退行性疾病的知识差距;汇集跨学科科学家，以刺激新的方法结合技能和学科;为这些科学家提供网络机会，以交流思想和资源;并在同行评审的科学期刊上发表会议记录。这个为期两天的会议将为与会者提供关于创造治疗和预防神经退行性疾病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：目标识别与验证;铅识别与优化;铅发现;临床前概念验证与开发;推进药物发现的资源和服务;和神经退行性疾病药物发现中的疾病特定问题。阿尔茨海默氏病、帕金森氏病、亨廷顿氏病和肌萎缩侧索硬化症将被纳入讨论。会议将包括3到5个演讲，有足够的时间进行问答。关于路线图的公私伙伴关系倡议，ADDF将邀请来自学术界、生物技术和制药业以及国家卫生研究所的约25名国际公认的科学家，包括妇女和少数民族，担任主席或发言人。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，并促进他们共同努力开发商业上可行和有效的疾病修饰产品。 特色1： 目的：在药物发现方面培训学术科学家，交流思想，促进临床前研究，建立公私伙伴关系，出版会议记录，提供CME学分。6个主题：靶点、先导化合物识别、先导化合物优化、临床前POC、药物发现资源、特定疾病问题。已确认和拟议的演讲者包括来自行业和学术界的资深和公认的成功的药物开发科学家。 总的来说，拟议的会议可能对其学术研究人员的目标受众非常有用，并可能实现其教育这些研究人员进行药物开发的目标，并将促进公私伙伴关系。这些活动将提高提交给NIH的翻译提案的质量。AD、PD、HD和ALS这四个目标疾病领域非常适合神经退行性疾病领域。从目标验证到IND工作的全方位药物发现和开发培训学术科学家的重点仍然很少，尽管IBC赞助了包括其中一些领域的会议。最好能更具体地指出，拟议的会议与其他会议相比，有何优势。发言者的范围和专门知识可以通过减少两个或两个以上发言者来自同一机构的一些潜在冗余来扩大。应增加对成功开发获批药物实际作出直接和重大贡献的发言者的比例。 预算：总体预算是适当的，向NIA申请的金额（不到会议总费用的一半）是适当的。 将妇女和少数民族包括在内是适当的。

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.