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.

项目摘要 转化治疗核心（TTC）将提供必要的基础架构和专业知识 加速对阿尔茨海默氏病（AD）和广告相关的新型治疗的临床前发展 痴呆症（ADRD）。 TTC将在广告/ADRD中开发新的治疗剂时填补关键的空白 通过促进2个迫切支持的转化科学的关键组成部分，来领域 学术界：（1）人类目标验证基础科学发现，以及（2）网络医学方法 确定已经存在的药物重新使用的代理。一个关键的方面是针对目标的人类数据 通过临床经验，人遗传学和组织表达的领域进行验证。尤其是 由于质量组织和疾病复杂性的稀缺性，在逻辑上挑战了AD/ADRD领域。 通过建立克利夫兰阿尔茨海默氏病研究中心（CADRC）核心TTC 将与生物标志物核（BC）紧密合作，不仅可以提供深入的表型生物学访问 样本，但专家指导ADRD的异质性。 TTC将为CadRC带来丰富的财富 共享资源，包括大学医院的哈灵顿发现研究所（HDI），这是 哈灵顿发现与发展项目，一项旨在使创造力的国际倡议 科学家将他们的发现推向新颖的药物，这些药物可以通过提供来改善人类健康 资金和药物开发支持以弥合基本发现与临床领域之间的差距。 TTC由阿尔茨海默氏症毒品发现基金会（ADDF）主任安德鲁·皮珀（Andrew Pieper）博士领导 - 哈灵顿学者计划，该计划支持科学家开发可以预防，治疗和治愈的药物 广告/adrd。 TTC将识别和支持正在开发治疗的研究人员，目前针对的 通过Pieper博士与Addf的关系 和HDI（特定目标1）。为与之相关的新假设提供了临床前支持的研究人员 AD/ADRD将通过Cheng博士的方法来支持基于网络的方法，以识别这些药物 可以重新使用用于治疗AD/ADRD。 TTC还将应用与特定目标1中相同的原则 支持研究人员在医学研究的其他领域开发新疗法或假设 也可以应用于AD/ADRD，以支持创新的关键多样性以开发新的治疗 对于AD/ADRD（特定目标2）。此外，与CADRC生物标志物核心（BC）的协调将使 TTC促进与新的治疗方法相关的标志物的识别，可以应用于 未来的临床试验或患者护理。通过提供访问和资金，用于使用人体组织中的目标验证 CADRC生物标志物和神经病理学核心中不断增长的生物晶体收集，TTC将启用 CADRC有意义地提高了针对患者的新疗法的开发。