Advancing ALS Research With DHTs: Where Are We Now?

By Sylvain Zorman, Ph.D.

Amyotrophic Lateral Sclerosis (ALS) is a devastating, progressive neuromuscular disease with minimal therapeutic progress in the past 30 years. Despite a robust drug pipeline, with over 110 Phase 2 and Phase 3 trials launched from 2013 to 2023, recent clinical trials, including Phase 3 for Relyvrio and Phase 2 for SAR443820/DNL788, have failed to show improvements on the ALS Functional Rating Scale-Revised (ALSFRS-R), the primary endpoint used.

Are these trials failing due to ineffective drugs, or are the endpoints not sensitive enough to detect changes in patient function?

The ALSFRS-R, a staff-administered questionnaire, provides valuable insights into ALS patient functioning but requires in-clinic visits and is prone to recall bias and inter-rater variability. These limitations suggest it may not capture the daily variabilities in ALS progression effectively. Wearable, sensor-based digital health technologies (DHTs) offer a solution by continuously collecting objective data on gross and fine motor functions.

In our Digital Health Monthly webinar series, we explored new data showing that DHTs can provide continuous, sensitive measures to better track ALS patient function.