IUSM Alzheimer's Disease Drug Discovery Center

IUSM 阿尔茨海默病药物研发中心

• 批准号: 10684136
• 负责人: Bruce T Lamb
• 金额: $ 729.79万
• 依托单位: INDIANA UNIV-PURDUE UNIV AT INDIANAPOLIS
• 依托单位国家: 美国
• 财政年份: 2019
• 资助国家: 美国
• 起止时间: 2019-09-30 至 2024-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10684136
• 关键词: Alzheimer disease prevention; Alzheimer' s Disease; Amyloid beta-Protein; Animal Disease Models; Basic Science; Biological Markers; Biology; Brain; Cause of Death; Clinical Research; Clinical Sciences; Clinical Trials Design; Communities; Data; Dimensions; Disease; Drug Targeting; Early Onset Alzheimer Disease; Elements; Ensure; Feasibility Studies; Generations; Genetic; Goals; Human Pathology; Immunization; Indiana; Intervention; Investments; Lead; Methods; Mission; Modality; Modeling; Molecular; Nerve Degeneration; Neuroimmune; Pathway interactions; Patients; Performance; Pharmaceutical Preparations; Pharmacologic Substance; Pharmacology; Probability; Process; Qualifying; Research; Research Personnel; Resources; Risk; Science; Scientist; Technology; Testing; Therapeutic; Toxicology; Translating; Translational Research; United States; United States National Institutes of Health; Universities; Validation; Work; abeta accumulation; beta secretase; data access; data sharing; design; disability; drug candidate; drug development; drug discovery; experimental study; flexibility; gamma secretase; human disease; inhibitor; innovation; insight; medical schools; novel; open data; physical property; pre-clinical; sharing platform; success; translational study; validation studies

The strategic goal of the Indiana University School of Medicine Alzheimer’s Disease Drug Discovery (ADDD) Center is to integrate sophisticated capability for early drug discovery and contribute to a broader study of emerging Alzheimer’s disease (AD) target hypotheses (beyond Ab) with the goal of generating new classes of potential therapeutics. Specifically, the ADDD CENTER will establish itself as a strategic and operational partner for the NIA AMP-AD and MODEL-AD initiatives. By design, this will provide drug discovery capability to bridge the foundational work in target discovery (AMP-AD) with newly discovered lead molecules characterized in AD animal models based on human pathology, genetics and translational biomarkers (MODEL-AD). To accomplish the mission of the ADDD CENTER, we have assembled a world-class team of scientists and capabilities from Indiana University, Purdue University, and the Indiana Clinical and Translational Sciences Institute (CTSI). This team has considerable expertise in Alzheimer’s disease biology, CNS pharmaceutical drug discovery and development, and scientific excellence in the core discovery technologies that will drive the ADDD CENTER’s contributions and deliverables. A key advantage and differentiated strength of the proposed ADDD CENTER is the primary scientific coordination and administration through the Indiana University School of Medicine. Specifically, this concentrates a strong and long-standing commitment to neurodegenerative research through co-presence with the NIA-supported Indiana Alzheimer’s Disease Center under the direction of Dr. Andy Saykin, the MODEL-AD consortium under the direction of Dr. Bruce Lamb, and the Longitudinal Early Onset Alzheimer’s Disease (LEAD) study under the direction of Dr. Liana Apostolova. The Specific Aims of the ADDD CENTER are: 1. Create a dynamic portfolio of well-characterized AD drug discovery targets representing novel intervention hypotheses that capitalize on significant investments in basic research and emerging disease understanding. 2. Perform advanced pre-clinical target validation and enablement studies to prioritize the best opportunities for therapeutic discovery. 3. Create high-quality and well-characterized lead molecules for prioritized targets that meet rigorous milestone criteria and are valued opportunities for further translational investment. 4. Create a robust and flexible data sharing platform to enable global researchers with data and target enablement packages to expand on the work of the ADDD CENTER.

印第安纳大学医学院阿尔茨海默病药物发现(ADDD)中心的战略目标是整合早期药物发现的尖端能力，促进对新出现的阿尔茨海默病(AD)靶点假说(超越抗体)的更广泛研究，目标是产生新的潜在疗法类别。具体地说，ADDD中心将成为NIA AMP-AD和MODEL-AD倡议的战略和业务合作伙伴。通过设计，这将提供药物发现能力，将靶标发现的基础工作(AMP-AD)与新发现的以人类病理、遗传学和翻译生物标记物(模型-AD)为基础的AD动物模型中特征的前导分子联系起来。为了完成ADDD中心的使命，我们组建了一支由来自印第安纳大学、普渡大学和印第安纳临床与转化科学研究所(CTSI)的世界级科学家和能力组成的团队。该团队在阿尔茨海默病生物学、中枢神经系统药物发现和开发方面拥有相当多的专业知识，并在推动ADDD中心贡献和交付成果的核心发现技术方面拥有卓越的科学能力。拟议的ADDD中心的一个关键优势和差异化优势是通过印第安纳大学医学院进行主要的科学协调和管理。具体地说，这集中了对神经退行性研究的坚定和长期的承诺，通过与NIA支持的印第安纳州阿尔茨海默病中心(由Andy Saykin博士指导)、Model-AD联盟(由Bruce Lamb博士指导)以及纵向早发性阿尔茨海默病(LEAD)研究(由Liana Apostolova博士指导)共同开展研究。ADDD中心的具体目标是：1.创建具有良好特征的AD药物发现目标的动态组合，代表新的干预假设，利用在基础研究和对新出现的疾病的了解方面的重大投资。2.执行先进的临床前目标验证和启用研究，以确定治疗发现的最佳机会的优先顺序。3.为符合严格里程碑标准的优先目标创造高质量和特征良好的先导分子，是进一步翻译投资的宝贵机会。4.建立一个强大和灵活的数据共享平台，使全球研究人员能够利用数据和目标赋能一揽子计划扩大发展数据中心的工作。