用于阿尔茨海默病及其他神经退行性疾病的药物重新定位。

Drug repurposing for Alzheimer's disease and other neurodegenerative disorders.

作者信息

Cummings Jeffrey L, Zhou Yadi, Van Stone Alexandra, Cammann Davis, Tonegawa-Kuji Reina, Fonseca Jorge, Cheng Feixiong

机构信息

Chambers-Grundy Center for Transformative Neuroscience, Department of Brain Health, Kirk Kerkorian School of Medicine, University of Nevada, Las Vegas (UNLV), Las Vegas, NV, 89106, USA.

Genomic Medicine Institute, Lerner Research Institute, Cleveland Clinic, Cleveland, Ohio, 44195, USA.

出版信息

Nat Commun. 2025 Feb 19;16(1):1755. doi: 10.1038/s41467-025-56690-4.

DOI:10.1038/s41467-025-56690-4

PMID:39971900

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11840136/

Abstract

Repurposed drugs provide a rich source of potential therapies for Alzheimer's disease (AD) and other neurodegenerative disorders (NDD). Repurposed drugs have information from non-clinical studies, phase 1 dosing, and safety and tolerability data collected with the original indication. Computational approaches, "omic" studies, drug databases, and electronic medical records help identify candidate therapies. Generic repurposed agents lack intellectual property protection and are rarely advanced to late-stage trials for AD/NDD. In this review we define repurposing, describe the advantages and challenges of repurposing, offer strategies for overcoming the obstacles, and describe the key contributions of repurposing to the drug development ecosystem.

摘要

老药新用为阿尔茨海默病（AD）和其他神经退行性疾病（NDD）提供了丰富的潜在治疗来源。老药新用拥有来自非临床研究、1期剂量测定以及针对原始适应症收集的安全性和耐受性数据。计算方法、“组学”研究、药物数据库和电子病历有助于识别候选疗法。通用的老药新用药物缺乏知识产权保护，很少能进入AD/NDD的后期试验。在本综述中，我们定义了老药新用，描述了老药新用的优势和挑战，提供了克服障碍的策略，并阐述了老药新用对药物开发生态系统的关键贡献。