The epidemiology and treatment of ALS: focus on the heterogeneity of the disease and critical appraisal of therapeutic trials

Amyotroph Lateral Scler. 2011 Jan;12(1):1-10. doi: 10.3109/17482968.2010.502940. Epub 2010 Aug 11.


Abstract Effective treatments for amyotrophic lateral sclerosis (ALS) have remained elusive. Only riluzole, a drug thought to affect glutamate metabolism, improves survival albeit to modest extent. Explanations for the negative results of therapeutic trials include a likely heterogeneity, both in disease susceptibility and pathogenic mechanisms, and faulty methodology of clinical trials. Further understanding of these factors will lead to improvements in patient stratification, and in the design of future clinical trials.

Publication types

  • Research Support, N.I.H., Intramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Aged
  • Amyotrophic Lateral Sclerosis / drug therapy
  • Amyotrophic Lateral Sclerosis / epidemiology*
  • Amyotrophic Lateral Sclerosis / metabolism
  • Amyotrophic Lateral Sclerosis / therapy*
  • Antioxidants / therapeutic use
  • Clinical Trials as Topic
  • Enteral Nutrition
  • Europe / epidemiology
  • Glutamic Acid / metabolism
  • Humans
  • Incidence
  • Intercellular Signaling Peptides and Proteins / therapeutic use
  • Middle Aged
  • Prognosis
  • Randomized Controlled Trials as Topic
  • Registries
  • Research Design
  • Respiration, Artificial
  • Riluzole / therapeutic use
  • Treatment Outcome
  • United States / epidemiology
  • White People / statistics & numerical data


  • Antioxidants
  • Intercellular Signaling Peptides and Proteins
  • Glutamic Acid
  • Riluzole