What are the newest ALS treatments in 2026?

Short Answer: Key 2026 ALS developments include tofersen (FDA-approved for SOD1-ALS), masitinib (Phase III), and emerging gene therapy approaches targeting C9orf72 and TDP-43.

Evidence Level: strong

Detailed Answer

The ALS treatment landscape has evolved significantly: Tofersen (Qalsody) received accelerated FDA approval for SOD1-mutation ALS (~2% of cases), demonstrating neurofilament light chain reduction and functional stabilization. Masitinib, a tyrosine kinase inhibitor targeting neuroinflammation, showed positive Phase III results with 25% slowing of functional decline. AMX0035 (relyvrio/sodium phenylbutyrate-taurursodiol) was initially approved then withdrawn due to confirmatory trial failure. Gene therapy approaches targeting C9orf72 repeat expansions (the most common genetic cause, ~10% of all ALS) are in Phase I/II trials. TUDCA remains under investigation as a neuroprotective adjunct. Stem cell therapies (NurOwn/MSC-NTF) showed signals in post-hoc subgroup analyses but failed primary endpoints.

Sources

• Miller TM et al. (2022) NEJM — tofersen
• Mora JS et al. (2020) Amyotroph Lateral Scler — masitinib