TREATMENT OF ALS WITH HIGH-DOSE PULSE CYCLOPHOSPHAMIDE

Autoimmune abnormalities have been described in sporadically acquired amyotrophic lateral sclerosis (ALS), but ALS patients do not benefit f rom conventional immunosuppressive therapy. Because Multifocal Motor N europathy causes a syndrome mimicking the lower motor neuron involveme nt in ALS and responds to high dose cyclophosphamide, we tested whethe r ALS patients would respond to the same treatment. Eighteen patients with classical ALS initially received a high loading dose of intraveno us cyclophosphamide (3 g/m(2)) followed by 6-monthly injections of 750 -1000 mg/m(2). We monitored isometric strength, fine motor coordinatio n and pulmonary function monthly for 3 months prior to the study, duri ng the treatment phase, and for 6 months after treatment. Treatment su bjects were matched to control subjects from the WALS natural history data base and compared on the basis of decline rate (megaslopes). Trea tment did not alter the course of ALS.