21 January 2026

Editor’s Choice
Journal of the Neurological Sciences | Clinical safety of ultra-high-dose methylcobalamin in patients with amyotrophic lateral sclerosis: Open-label extension of a phase 2/3 randomized controlled study

A new phase 3 extension study evaluates the long-term safety of ultra-high-dose methylcobalamin in patients with advanced amyotrophic lateral sclerosis (ALS). The open-label study followed patients receiving 50 mg intramuscular methylcobalamin twice weekly for up to one year after completion of a prior randomized phase 2/3 trial.

The results show that this high-dose regimen was generally well tolerated, with few treatment-related adverse reactions and no discontinuations or deaths attributed to the therapy. Survival rates at 52 weeks remained high across ALS severity stages, and functional decline, measured by the ALS Functional Rating Scale, was limited over the study period.

Read the full article for detailed safety outcomes and discussion of how ultra-high-dose methylcobalamin may serve as a foundation for future combination therapies in ALS.

Article link:https://doi.org/10.1016/j.jns.2025.125701

Source: World Federation Neurology