Motor neuron disease: systematic reviews of treatment for ALS and SMA

Br Med Bull. 2010:93:145-59. doi: 10.1093/bmb/ldp049. Epub 2009 Dec 15.

Abstract

Introduction: There is no curative treatment for the common motor neuron diseases, amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy. Nevertheless, there is an increasing volume of published studies. This review assesses the current evidence for treatment of these conditions.

Sources of data: Primarily, the systematic reviews of the Cochrane Collaboration, with additional reference to other systematic reviews and online sites.

Areas of agreement: Riluzole remains the only medication with demonstrated efficacy and regulatory approval for the treatment of ALS. AREAS OF CONTROVERSY, GROWING POINTS, AND AREAS TIMELY FOR DEVELOPING RESEARCH: The design of clinical trials and the publication of unsatisfactory studies, in both human and animal models, continue to cause confusion in advising on patient management. Improvements in trial design, critical assessment of studies for publication and avoidance of bias towards publication of positive results are needed. A better understanding of pathogenesis should lead to more potent interventions.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Amyotrophic Lateral Sclerosis / therapy*
  • Databases as Topic
  • Humans
  • Motor Neuron Disease / therapy
  • Muscular Atrophy, Spinal / therapy*
  • Neuroprotective Agents / therapeutic use*
  • Randomized Controlled Trials as Topic
  • Riluzole / therapeutic use*

Substances

  • Neuroprotective Agents
  • Riluzole