MRC Centre for Drug Safety Science, Department of Molecular and Clinical Pharmacology, University of Liverpool, Liverpool, UK.
Wellcome Sanger Institute, Wellcome Genome Campus, Cambridge, UK.
Nat Rev Drug Discov. 2019 Jan;18(1):41-58. doi: 10.1038/nrd.2018.168. Epub 2018 Oct 12.
Given the high attrition rates, substantial costs and slow pace of new drug discovery and development, repurposing of 'old' drugs to treat both common and rare diseases is increasingly becoming an attractive proposition because it involves the use of de-risked compounds, with potentially lower overall development costs and shorter development timelines. Various data-driven and experimental approaches have been suggested for the identification of repurposable drug candidates; however, there are also major technological and regulatory challenges that need to be addressed. In this Review, we present approaches used for drug repurposing (also known as drug repositioning), discuss the challenges faced by the repurposing community and recommend innovative ways by which these challenges could be addressed to help realize the full potential of drug repurposing.
鉴于高淘汰率、高成本以及新药研发速度缓慢,将“老”药重新用于治疗常见和罕见疾病的做法越来越具有吸引力,因为它涉及使用风险降低的化合物,潜在的总成本更低,开发时间更短。已经提出了各种基于数据和实验的方法来识别可重新定位的药物候选物;然而,也存在需要解决的重大技术和监管挑战。在这篇综述中,我们介绍了用于药物重新定位(也称为药物重定位)的方法,讨论了重新定位社区面临的挑战,并提出了创新的方法来解决这些挑战,以帮助充分发挥药物重新定位的潜力。