Another Small Step Forward in ALS Treatment

Source: Lai EC, et al. Effect of recombinant human insulin-like growth factor-I on progression of ALS: A placebo-controlled study. Neurology 1997;49:1621-1630.

Als is slowly yielding its fatal path to the tenacious onslaught of systemic drugs. Among 266 patients with ALS randomized in a nine-month, double-blind, placebo-controlled trial using low- (0.05 mg/kg/d s/c qd) or high-dose (0.05 mg/kg/d s/c bid) recombinant human insulin-like growth factor-I (rhIGF-I), functional decline was 26% slower in the high-dose group than in those receiving placebo (P = 0.01). Quality of life, as assessed by the Sickness Impact Profile (SIP), declined more slowly in the high-dose group, and patients in the low-dose group tended toward a similar benefit without reaching significance, suggesting the presence of a dose- dependent effect. Significant adverse experiences were similar in the three groups, most frequently including injection site complaints of pain, inflammation, and bleeding. rhIGF-I has yet to be shown to prolong survival, but, perhaps more importantly, it is the first agent to slow symptom progression and, thus, will likely play a role in the treatment of ALS. -mr