Translational and Therapeutics Core

转化和治疗核心

• 批准号: 10675670
• 负责人: ANDREW A PIEPER
• 金额: $ 20.17万
• 依托单位: CLEVELAND CLINIC LERNER COM-CWRU
• 依托单位国家: 美国
• 财政年份: 2021
• 资助国家: 美国
• 起止时间: 2021-09-01 至 2026-06-30
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10675670
• 关键词: Academia; Acceleration; Advanced Development; Alzheimer' s Disease; Alzheimer' s disease related dementia; Alzheimer' s disease therapeutic; Area; Basic Science; Biological; Biological Markers; Center for Translational Science Activities; Clinic; Clinical; Clinical Trials; Collection; Development; Disease; Doctor of Philosophy; FDA approved; Fostering; Foundations; Funding; Future; Goals; Health; Heterogeneity; Human; Human Genetics; Incidence; Infrastructure; International; Laboratories; Laboratory Research; Medical Research; Medicine; Methods; Nerve Degeneration; Network-based; Pathway interactions; Patient Care; Patients; Pennsylvania; Pharmaceutical Preparations; Phase I Clinical Trials; Phenotype; Preparation; Prevalence; Process; Psychiatry; Research; Research Institute; Research Personnel; Resource Sharing; Safety; Sampling; Scholars Program; Scientist; Source; Texas; Therapeutic; Tissue Banks; Tissues; Translational Research; Universities; University Hospitals; Validation; Work; biobank; biomarker identification; brain health; clinical center; design; drug development; drug discovery; drug repurposing; experience; human data; human tissue; improved; induced pluripotent stem cell; innovation; medical schools; neuropathology; novel; novel strategies; novel therapeutic intervention; novel therapeutics; pre-clinical; preclinical development; prevent; professor; programs; therapeutic target; translational therapeutics

PROJECT SUMMARY The Translational Therapeutics Core (TTC) will provide the infrastructure and expertise necessary to accelerate preclinical development of novel therapeutics for Alzheimer’s disease (AD) and AD-related dementias (ADRD). The TTC will fill a critical void in the development of new therapeutics in the AD/ADRD field by facilitating 2 critical components of translational science that are desperately under supported in academia: (1) human target validation of basic science findings, and (2) network medicine approaches to identify already existing agents for drug repurposing. A critical aspect of this is human data towards target validation, through the realms of clinical experience, human genetics, and tissue expression. This is particularly logistically challenging in the field of AD/ADRD due to scarcity of quality tissue and disease complexity. Through the establishment of the Cleveland Alzheimer’s Disease Research Center (CADRC) Cores, the TTC will work closely with the Biomarker Core (BC) to provide not only access to deeply phenotyped biological samples, but expert guidance in the heterogeneity of ADRD. The TTC will bring to the CADRC a wealth of shared resources including the Harrington Discovery Institute (HDI) at University Hospitals, which is part of the Harrington Project for Discovery & Development, an international initiative designed to enable inventive scientists to advance their discoveries into novel medicines that will improve human health by providing funding and drug development support to bridge the gap between basic discovery and the clinical realm. The TTC is led by Dr. Andrew Pieper, Director of the Alzheimer’s Drug Discovery Foundation (ADDF) – Harrington Scholar Program, which supports scientists to develop drugs that will prevent, treat, and cure AD/ADRD. The TTC will identify and support researchers developing therapeutics currently targeted for AD/ADRD locally in the CADRC, and nationally and internationally through Dr. Pieper’s relationship with ADDF and HDI (Specific Aim 1). Researchers who have developed preclinical support for novel hypotheses related to AD/ADRD will be supported through Dr. Cheng’s methods for network-based approaches to identify drugs that might be repurposed for treating AD/ADRD. The TTC will also apply the same principles as in Specific Aim 1 to support researchers developing novel therapeutics or hypotheses in other areas of medical research that might also be applied to AD/ADRD, in order to support critical diversity of innovation for developing new treatments for AD/ADRD (Specific Aim 2). In addition, coordination with the CADRC Biomarker Core (BC) will enable the TTC to facilitate identification of markers related to new therapeutic approaches, which might be applied in future clinical trials or patient care. By providing access and funds for target validation in human tissue using the growing biospecimen collections in the CADRC Biomarker and Neuropathology Cores, the TTC will enable the CADRC to meaningfully advance development of new treatments for patients.

项目概要 转化治疗核心（TTC）将提供必要的基础设施和专业知识 加速阿尔茨海默病 (AD) 及 AD 相关新疗法的临床前开发 痴呆症（ADRD）。 TTC 将填补 AD/ADRD 新疗法开发的关键空白 领域，通过促进转化科学的两个关键组成部分，而这些组成部分在 学术界：（1）基础科学发现的人体目标验证，以及（2）网络医学方法 确定现有的药物再利用药物。其中一个关键方面是针对目标的人类数据 通过临床经验、人类遗传学和组织表达领域进行验证。这一点特别 由于优质组织的稀缺性和疾病的复杂性，AD/ADRD 领域的后勤工作面临着挑战。 通过建立克利夫兰阿尔茨海默病研究中心 (CADRC) 核心，TTC 将与生物标记核心（BC）密切合作，不仅提供深入表型生物 样本，但 ADRD 异质性方面的专家指导。 TTC 将为 CADRC 带来丰富的经验 共享资源，包括大学医院的哈林顿探索研究所 (HDI)，该研究所是 哈林顿发现与开发项目，一项旨在实现创造性的国际倡议 科学家们将他们的发现推进到新药中，通过提供改善人类健康的药物 资金和药物开发支持，以弥合基础发现和临床领域之间的差距。 TTC 由阿尔茨海默病药物发现基金会 (ADDF) 主任 Andrew Pieper 博士领导 – 哈林顿学者计划，支持科学家开发预防、治疗和治愈的药物 AD/ADRD。 TTC 将确定并支持研究人员开发目前针对的治疗方法 AD/ADRD 在 CADRC 本地开展，并通过 Pieper 博士与 ADDF 的关系在国内和国际范围内开展 和 HDI（具体目标 1）。为相关新假设提供临床前支持的研究人员 AD/ADRD 将通过 Cheng 博士的基于网络的方法来支持，以识别药物 可能会被重新用于治疗 AD/ADRD。 TTC 还将应用与具体目标 1 中相同的原则来 支持研究人员在其他医学研究领域开发新的疗法或假设 也适用于 AD/ADRD，以支持开发新疗法的关键创新多样性 AD/ADRD（具体目标 2）。此外，与 CADRC 生物标记核心 (BC) 的协调将使 TTC 有助于识别与新治疗方法相关的标记物，这些方法可能应用于 未来的临床试验或患者护理。通过使用人体组织中的目标验证提供途径和资金 随着 CADRC 生物标志物和神经病理学核心中生物样本收藏的不断增加，TTC 将使 CADRC 有意义地推进针对患者的新疗法的开发。