ALS Drug Development: Reflections from the Past and a Way Forward
Summary
Tremendous advances in our understanding of pathogenesis of amyotrophic lateral sclerosis (ALS) have provided a rich pipeline of drugs for clinical trialists. At least 32 unique compounds have been tested. Nevertheless, riluzole is currently the only treatment that prolongs survival. We present a critical overview of past clinical trials, how therapies are selected for testing in people, challenges with ALS clinical trial design and conduct, and ways to best move forward.
Department of Neurology, Massachusetts General Hospital, Neurology Clinical Trials Unit, Charlestown, Massachusetts 02129
Address correspondence and reprint requests to: Swati P. Aggarwal, M.D., Assistant in Neurology, Massachusetts General Hospital, Building 149, 13th Street, Charlestown, MA 02129
ABSTRACT