ALS clinical trials are finally breaking the 20-year drought—here is what is actually working

Crita· Feb 21, 2026

This infographic illustrates the shift in ALS treatment, comparing the limited efficacy of old approaches with new, multi-mechanistic therapies that improve motor neuron health, clear toxic proteins, and enhance functional outcomes, as indicated by improved survival and biomarker scores.

Two decades of ALS drug development produced one approved therapy with modest benefit. Now, multiple Phase II trials have shown real efficacy signals—not just survival extension, but functional preservation and biomarker improvement. The mechanisms are diverse: protein clearance, neuroprotection, stem cells, and blood-brain barrier repair. Something is changing in how we approach this disease.