Temporary amelioration of symptoms with intravenous cyclophosphamide in amyotrophic lateral sclerosis.

Based on the evidence that autoimmunity may play a role in the pathogenesis of amyotrophic lateral sclerosis (ALS), a variety of immunomodulating agents have been used in the treatment. In an uncontrolled trial we treated 44 patients of ALS with intravenous cyclophosphamide (IVCP) at a total dose of 1.5 g/m2 given over a period of 8 to 10 days. The patients were evaluated using neurological score which included bulbar, motor and daily activity scores before and following treatment. Twenty three patients showed a significant improvement (P=<0.001) in the composite and the individual scores. The improvement persisted only for 2 to 3 months. Amongst them the severely (7) and moderately (16) affected (score less than or more than 150) showed almost a similar response to treatment. A comparison of the improved group of 23 patients with the unimproved group of 21 patients did not reveal any significant factors which influenced the response to IVCP. However, there was a suggestion that patients below the age of 60 years and a duration of illness less than 12 months may respond to the drug. In conclusion, treatment with intravenous cyclophosphamide resulted in mild and temporary improvement in clinical status of the patients with amyotrophic lateral sclerosis. This may be considered as an alternative method of treatment in developing countries where newer drugs are not available and affordable.