肌萎缩侧索硬化症临床试验者的困境：寻找治愈方法的障碍

The Dilemma of the Clinical Trialist in Amyotrophic Lateral Sclerosis: The Hurdles to Finding a Cure.

作者信息

Katz Jonathan S, Barohn Richard J, Dimachkie Mazen M, Mitsumoto Hiroshi

机构信息

Department of Neurology, Forbes Norris MDA/ALS Center, California Pacific Medical Center, 2324 Sacramento Street, Suite 111, San Francisco, CA 94115, USA.

Department of Neurology, The University of Kansas Medical Center, 3901 Rainbow Boulevard, Mail Stop 2012, Kansas City, KS 66160, USA.

出版信息

Neurol Clin. 2015 Nov;33(4):937-47. doi: 10.1016/j.ncl.2015.07.014.

DOI:10.1016/j.ncl.2015.07.014

PMID:26515630

Abstract

Amyotrophic lateral sclerosis can be described as a disease with a poorly understood pathophysiologic mechanism and no treatment that dramatically impacts the course of the disease. Clinical trialists are faced with finding small treatment effects against a background of multiple potential treatments, a past history of failed trials, and heterogenous clinical outcomes. This article summarizes this environment and provides a rationale for drug development going forward.

摘要

肌萎缩侧索硬化症可被描述为一种病理生理机制尚不清楚且没有能显著影响疾病进程的治疗方法的疾病。临床试验人员面临着在多种潜在治疗方法、过去试验失败的历史以及异质性临床结果的背景下寻找微小治疗效果的挑战。本文总结了这种情况，并为未来的药物开发提供了理论依据。