Branched-chain amino acids and amyotrophic lateral sclerosis: a treatment failure? The Italian ALS Study Group

Neurology. 1993 Dec;43(12):2466-70. doi: 10.1212/wnl.43.12.2466.


We initiated a double-blind, placebo-controlled trial to test the efficacy and safety of branched-chain amino acids (BCAA) (L-leucine 12 g, L-isoleucine 6 g, and L-valine 6 g daily) in amyotrophic lateral sclerosis (ALS) patients. There was an excess mortality in subjects randomized to active treatment (24 BCAA, 13 placebo) when a total of 126 ALS patients had been recruited. This finding, associated with the lack of efficacy of BCAA (measured by comparing the disability scales in the two treatment groups), led the Data Monitoring Committee to require cessation of the trial.

Publication types

  • Clinical Trial
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Amino Acids, Branched-Chain / adverse effects
  • Amino Acids, Branched-Chain / therapeutic use*
  • Amyotrophic Lateral Sclerosis / drug therapy*
  • Amyotrophic Lateral Sclerosis / mortality
  • Amyotrophic Lateral Sclerosis / physiopathology
  • Double-Blind Method
  • Female
  • Humans
  • Male
  • Meta-Analysis as Topic
  • Middle Aged
  • Multivariate Analysis
  • Patient Advocacy
  • Safety Management
  • Severity of Illness Index
  • Survival Analysis
  • Treatment Failure
  • Vital Capacity


  • Amino Acids, Branched-Chain