Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development

Current therapeutic development in amyotrophic lateral sclerosis (ALS) relies on individual randomized clinical trials to test a specific investigational product in a single patient population. This approach has intrinsic limitations, including cost, time, and lack of flexibility. Adaptive platform trials represent a novel approach to investigate several interventions for a single disease in a continuous manner. Already in use in oncology, this approach is now being employed more often in neurology. Here, we describe a newly launched platform trial for ALS. The Healey ALS Platform Trial is testing multiple investigational products concurrently in people with ALS, with the goal of rapidly identifying novel treatments, biomarkers, and trial endpoints.

Automated summary

The current therapeutic development in ALS faces limitations in terms of cost, time, and flexibility due to individual randomized clinical trials. Adaptive platform trials provide a novel approach to investigate multiple interventions for a single disease continuously. The Healey ALS Platform Trial is a recently launched trial that aims to identify novel treatments, biomarkers, and trial endpoints rapidly by testing multiple investigational products concurrently in ALS patients.