A randomized controlled trial of recombinant interferon beta-1a in ALS

Objective: To evaluate the efficacy of recombinant interferon beta (IFN bet a)-1a in the treatment of ALS. Background: It has been proposed that IFNs a ffect the progression of ALS by interfering with putative immune mechanisms involved in the pathogenesis of the disease. Methods: Patients (n = 61) 40 to 70 years of age with a 6- to 24-month history of confirmed ALS with mil d to moderate disability received IFN beta-1a, 12 mIU (n = 31), or placebo (n = 30) subcutaneously three times a week for 6 months and were followed u p for an additional 6 months. Patients were assessed after 4, 12, 24, 36, a nd 48 weeks. Medical Research Council scale, Norris scale, and bulbar score s as well as forced vital capacity were used to assess disability. Selected electrophysiologic measures (latency, amplitude, and duration of the compo und muscle action potential) were also used. Results: Twenty patients rando mized to IFN beta-1a and 17 patients given placebo completed the study. A t otal of 16 patients receiving IFN beta-1a became non-self-supporting compar ed with 16 on placebo (52% versus 53%). There were no significant differenc es between the two treatment groups for any of the measures of disease prog ression and disability. Deaths were reported in six patients treated with I FN beta-1a and four patients on placebo. Adverse events were reported more frequently with IFN beta-1a (77%: of patients) compared with placebo (57%), with flu-like symptoms and local erythema being the commonest complaints. Conclusions: This pilot study suggests that IFN beta-1a is not effective in the treatment of ALS.