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现有或失败的候选药物重新利用的开源方法:跨疾病借鉴并应用经验教训。

Open-source approaches for the repurposing of existing or failed candidate drugs: learning from and applying the lessons across diseases.

作者信息

Allarakhia Minna

机构信息

Department of Management Sciences, University of Waterloo, Waterloo, ON, Canada.

出版信息

Drug Des Devel Ther. 2013 Aug 8;7:753-66. doi: 10.2147/DDDT.S46289. eCollection 2013.

Abstract

Repurposing has the objective of targeting existing drugs and failed, abandoned, or yet-to-be-pursued clinical candidates to new disease areas. The open-source model permits for the sharing of data, resources, compounds, clinical molecules, small libraries, and screening platforms to cost-effectively advance old drugs and/or candidates into clinical re-development. Clearly, at the core of drug-repurposing activities is collaboration, in many cases progressing beyond the open sharing of resources, technology, and intellectual property, to the sharing of facilities and joint program development to foster drug-repurposing human-capacity development. A variety of initiatives under way for drug repurposing, including those targeting rare and neglected diseases, are discussed in this review and provide insight into the stakeholders engaged in drug-repurposing discovery, the models of collaboration used, the intellectual property-management policies crafted, and human capacity developed. In the case of neglected tropical diseases, it is suggested that the development of human capital be a central aspect of drug-repurposing programs. Open-source models can support human-capital development through collaborative data generation, open compound access, open and collaborative screening, preclinical and possibly clinical studies. Given the urgency of drug development for neglected tropical diseases, the review suggests elements from current repurposing programs be extended to the neglected tropical diseases arena.

摘要

药物重新利用的目标是将现有药物以及失败、被放弃或尚未开发的临床候选药物用于新的疾病领域。开源模式允许共享数据、资源、化合物、临床分子、小型文库和筛选平台,以经济高效地推动旧药和/或候选药物进入临床再开发阶段。显然,药物重新利用活动的核心是合作,在许多情况下,合作不仅限于资源、技术和知识产权的开放共享,还包括设施共享和联合项目开发,以促进药物重新利用方面的人力能力发展。本综述讨论了正在进行的各种药物重新利用举措,包括针对罕见病和被忽视疾病的举措,并深入探讨了参与药物重新利用发现的利益相关者、所采用的合作模式、制定的知识产权管理政策以及发展的人力能力。对于被忽视的热带病,建议将人力资本开发作为药物重新利用计划的核心内容。开源模式可以通过协作数据生成、开放化合物获取、开放和协作筛选、临床前以及可能的临床研究来支持人力资本开发。鉴于被忽视热带病药物开发的紧迫性,本综述建议将当前重新利用计划中的要素扩展到被忽视热带病领域。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/868d/3743608/6bc2b89bf6a0/dddt-7-753Fig1.jpg

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