Experts suggest that expanding eligibility for amyotrophic lateral sclerosis (ALS) trials could benefit patients and drug development. Currently, 60% of ALS patients are excluded from clinical trials by the time they are diagnosed, which is an understatement according to investigators. Expanding inclusion criteria would allow more patients to access experimental treatments and help drug developers get a truer sense of how well those treatments work. In addition, including fast progressors in trials may have affected previous studies' results as certain molecules take longer to work and skew results in the wrong direction if fast progressors do not receive the full benefit. Stratification based on biomarkers could be an effective way to expand trial populations in the future.
ALS investigators are working towards a robust stratification model which is predictive of speed of progression. The Personalized European Network for the Cure of ALS survival prediction model has been widely tested and neurofilament levels have been shown to be a strong predictor of speed of progression in SOD1-ALS patients. Expanding eligibility criteria while defining priority analysis populations could offer benefits such as furthering ALS biomarker data.
Expanding eligibility for ALS