Abstract

A change in our current approach toward drug development is required to improve the likelihood of finding effective treatment for patients with amyotrophic lateral sclerosis (ALS). The aim of the Treatment Research Initiative to Cure ALS (TRICALS) is to extend the collective effort with industry and consolidate drug development paths. TRICALS has begun a series of meetings on how to best move the field forward collaboratively, thereby addressing five major topics in ALS clinical trials: (1) preclinical research, (2) biomarker development, (3) eligibility criteria, (4) efficacy endpoints and (5) innovative trial design. There is an appetite for ongoing discussions of these major topics in clinical trials between representatives from academia, patient advocacy groups, industry partners and funding bodies. Industry is open to fundamentally change drug development for ALS and shorten the time to effective therapy for patients by implementing promising innovations in biomarker development, trial design, and patient selection. There is however, a pressing need from all stakeholders for regulatory discussions and amendments of current guidelines to successfully adopt innovation in future clinical development lines.

摘要

我们目前的药物开发方法需要改变，以提高为肌萎缩侧索硬化 (ALS) 患者找到有效治疗方法的可能性。治愈 ALS 的治疗研究计划 (TRICALS) 的目的是扩大与行业的集体努力并巩固药物开发路径。TRICALS 已开始就如何最好地合作推动该领域向前发展举行一系列会议，从而解决 ALS 临床试验中的五个主要主题：(1) 临床前研究，(2) 生物标志物开发，(3) 资格标准，(4) 疗效终点(5) 创新的试验设计。来自学术界、患者权益团体、行业合作伙伴和资助机构的代表希望就临床试验中的这些主要主题进行持续讨论。业界愿意通过在生物标志物开发、试验设计和患者选择方面实施有前景的创新，从根本上改变 ALS 的药物开发并缩短患者获得有效治疗的时间。然而，所有利益相关者迫切需要对当前指南进行监管讨论和修订，以在未来的临床开发线中成功采用创新。