Subcutaneous IGF-1 is not beneficial in 2-year ALS trial

Neurology. 2008 Nov 25;71(22):1770-5. doi: 10.1212/01.wnl.0000335970.78664.36.


Background: Previous human clinical trials of insulin-like growth factor type I (IGF-1) in amyotrophic lateral sclerosis (ALS) have been inconsistent. This phase III, randomized, double-blind, placebo-controlled study was undertaken to address whether IGF-1 benefited patients with ALS.

Methods: A total of 330 patients from 20 medical centers were randomized to receive 0.05 mg/kg body weight of human recombinant IGF-1 given subcutaneously twice daily or placebo for 2 years. The primary outcome measure was change in their manual muscle testing score. Secondary outcome measures included tracheostomy-free survival and rate of change in the revised ALS functional rating scale. Intention to treat analysis was used.

Results: There was no difference between treatment groups in the primary or secondary outcome measures after the 2-year treatment period.

Conclusions: Insulin-like growth factor type I does not provide benefit for patients with amyotrophic lateral sclerosis.

Publication types

  • Clinical Trial, Phase III
  • Randomized Controlled Trial
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Amyotrophic Lateral Sclerosis / drug therapy*
  • Amyotrophic Lateral Sclerosis / physiopathology*
  • Central Nervous System Agents / administration & dosage*
  • Central Nervous System Agents / adverse effects
  • Deglutition
  • Double-Blind Method
  • Female
  • Hand Strength
  • Humans
  • Injections, Subcutaneous
  • Insulin-Like Growth Factor I / administration & dosage*
  • Insulin-Like Growth Factor I / adverse effects
  • Kaplan-Meier Estimate
  • Male
  • Middle Aged
  • Research Design
  • Thromboembolism / chemically induced
  • Time Factors
  • Tracheostomy
  • Treatment Failure


  • Central Nervous System Agents
  • Insulin-Like Growth Factor I