April 1988

A Double-blind Study of the Effectiveness of Cyclosporine in Amyotrophic Lateral Sclerosis

Author Affiliations

From the Department of Neurology, Baylor College of Medicine (Drs Appel, Stewart, and Harati, and Ms Appel) and the Veterans Administration Medical Center (Drs Stewart and Harati), Houston; and the Sandoz Research Institute (Drs Mietlowski and Belendiuk), Hanover, NJ. Mr Weiss is in statistical consulting in Vienna, Va.

Arch Neurol. 1988;45(4):381-386. doi:10.1001/archneur.1988.00520280027011

• In a double-blind placebo-controlled trial of cyclosporine in amyotrophic lateral sclerosis, no differences were observed in the monthly rate of progression or the relative risk of progression in comparing 38 patients randomized to the placebo group and 36 patients randomized to the cyclosporine group. In comparing three subgroups of patients, cyclosporine appeared to benefit men who entered the study within 18 months of the onset of first symptoms, whereas it was of no value to women or to men who entered later than 18 months. For the men with recent onset of disease, the relative risk of progression was 0.403; the monthly rate of progression was 5.2 ± 1.1 points with placebo and 3.5 ± 0.7 points with cyclosporine. These provocative results support the need for a full study of cyclosporine in men with recent onset of disease.