Protein Aggregation and the Development of NU-9 for Upper Motor Neuron Diseases, Including ALS

Presented by Richard B. Silverman, PhD; Patrick G. Ryan/Aon Professor in the Department of Chemistry, Department of Molecular Biosciences (Weinberg College of Arts and Sciences) and Department of Pharmacology (Feinberg School of Medicine), Northwestern University.
Dr. Silverman is the Patrick G. Ryan/Aon Professor in the Department of Chemistry with courtesy appointments in the Department of Molecular Biosciences (Weinberg College of Arts and Sciences) and Department of Pharmacology (Feinberg School of Medicine). His research is focused on the design, synthesis, and evaluation of small molecules that inhibit enzymes, cancer, and protein aggregation. Target enzymes include GABA aminotransferase for the treatment of epilepsy and addiction, ornithine aminotransferase for the treatment of hepatocellular carcinoma, and neuronal nitric oxide synthase for the treatment of neurodegenerative diseases and melanoma. Protein aggregation inhibitors are targeted for the treatment of neurodegenerative diseases, including ALS. Inactivation mechanisms for molecules designed as irreversible inhibitors are investigated with the use of several mass spectral techniques, X-ray crystallography, and computer modeling. The ultimate goal is to bring small molecules into clinical trials. His research is supported primarily by NIH.