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

Objective: To evaluate the efficacy of recombinant interferon beta (IFNβ)-1a in the treatment of ALS. Background: It has been proposed that IFNs affect 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 mild to moderate disability received IFNβ-1a, 12 mIU (n = 31), or placebo (n = 30) subcutaneously three times a week for 6 months and were followed up for an additional 6 months. Patients were assessed after 4, 12, 24, 36, and 48 weeks. Medical Research Council scale, Norris scale, and bulbar scores as well as forced vital capacity were used to assess disability. Selected electrophysiologic measures (latency, amplitude, and duration of the compound muscle action potential) were also used. Results: Twenty patients randomized to IFNβ-1a and 17 patients given placebo completed the study. A total of 16 patients receiving IFNβ-1a became non- self-supporting compared with 16 on placebo (52% versus 53%). There were no significant differences between the two treatment groups for any of the measures of disease progression and disability. Deaths were reported in six patients treated with IFNβ-1a and four patients on placebo. Adverse events were reported more frequently with IFNβ-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 IFNβ-1a is not effective in the treatment of ALS.