Enhancing clinical trials in neurodegenerative disorders: lessons from amyotrophic lateral sclerosis

This review article is focused on strategies that may enhance clinical trial efficiency in neurodegenerative disorders, as demonstrated within the research field of amyotrophic lateral sclerosis (ALS).

Unravelling ALS pathophysiology will result in an increased number of candidate therapeutics. Recent ALS clinical trials have employed novel study designs that expedite the drug development process and limit sample size, including futility, lead-in, selection, adaptive and sequential designs. The search for sensitive and specific biomarkers in ALS continues to develop, and they are essential in accelerating the drug discovery process. Several candidate cerebrospinal fluid (CSF), neuroimaging and electrophysiological biomarkers have been recently described in ALS, and some have been successfully employed as secondary outcome measures in clinical trials. The advent of web-based technologies has provided a complementary platform to expedite clinical trials, through electronic data capture, teleconferencing and online registries. In addition, the formation of ALS consortia has enhanced collaborative multicentre studies.

ALS research studies have employed novel strategies to accelerate the efficiency and pace of drug discovery. The importance of adapting to novel measures that enhance study efficiency is not unique to ALS and can be applied to other neurodegenerative diseases in search of effective treatments.