Cochrane review: gabapentin is not effective in ALS

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  • Gabapentin did not improve survival or slow disease progression when compared with placebo in amyotrophic lateral sclerosis (ALS).

Why this matters

  • Gamma aminobutyric acid (GABA) imbalance may play a role in the pathogenesis of ALS.

Key results

  • Only 2 trials met inclusion criteria; both were double-blind randomized controlled trials (RCTs) of oral gabapentin (Neurontin and others) vs placebo, and they had a total of 355 patients.
  • The trials did not last long enough to fully assess 1-y survival, but the estimated rate was 78% with gabapentin and 77% with placebo, a nonsignificant difference.
  • Gabapentin also did not slow the rate of decline of muscle strength or respiratory function.
  • There also was little evidence that it improved QoL or slowed monthly decline in the ALS Functional Rating Scale.
  • Adverse effects included light-headedness, drowsiness, and edema, whereas fatigue was similar.

Study design

  • A systematic review and meta-analysis of double-blind RCTs or quasi-RCTs of GABA modulators among adults with probable or definite ALS.
  • The main outcome was 1-y survival.
  • Funding: Study funding not disclosed.


  • Available evidence for gabapentin was limited.
  • The search failed to identify any RCTs of baclofen or other GABA modulators.