Keynote - New Directions in ALS Therapies

Presented by Robert H. Brown, Jr., D.Phil, MD; Leo P. and Theresa M. LaChance Chair in Medical Research, Director of the Program in Neurotherapeutics, University of Massachusetts Medical School (UMMS)
Dr. Brown has a longstanding research interest in identifying gene defects that underlie ALS and related neuromuscular disorders. With his colleague, Teepu Siddique, MD of the Les Turner ALS Center at Northwestern Medicine, he was a lead member of the team that identified the first ALS gene (SOD1) and, with colleagues, has subsequently participated in identifying several other defective genes in ALS including alsin, dynactin, FUS/TLS, ErbB4 and profilin1. He has identified causative gene defects s in other disorders including limb girdle dystrophy type 2B (dysferlin), hereditary sensory neuropathy (serine palmitoyl-transferase), and hyperkalemic paralysis (skeletal muscle sodium channel). His laboratory team has used insights from these investigations in genetics to generate cell and animal models of each of these disorders. These models have been useful in understanding pathological processes that trigger diseases like ALS and have assisted in therapy development. Most recently, he has initiated trials of gene suppression therapy (SOD1, C9orf72) in non-human primates and now in humans. He has published more than 300 peer-reviewed reports and more than 70 reviews and chapters on these topics. He is a member of the National Academy of Medicine (formerly the Institute of Medicine) and is a past president of the American Neurological Association.