ALS research pivots to biology-driven biomarkers to capture disease heterogeneity

ALS drug development is moving beyond traditional measures such as survival time and the ALS Functional Rating Scale. Researchers are now adding biology‑focused approaches that aim to reflect the full range of disease variation.

The condition remains deadly, with most patients surviving only two to five years after diagnosis, and no therapy has yet altered that outlook. A recent regulatory decision to approve tofersen based on reductions in the neurofilament light biomarker gave sponsors a clear tool for trial design and patient stratification.

The field is expanding to include biomarker panels that monitor neuronal injury, inflammation and muscle health across distinct ALS subtypes. Experts say this shift represents an exciting new era for ALS research, offering more precise ways to evaluate potential treatments.