COBRE Faculty Recruit, Matthew E. R. Butchbach, Ph.D. Receives Grant from AurimMed Pharma, Inc.
Matthew E. R. Butchbach, Ph.D., a COBRE faculty recruit, has been awarded a grant from AurimMed Pharma, Inc. Dr. Butchbach heads the Motor Neuron Diseases Research Laboratory (MNDRL) which focuses on understanding the molecular mechanisms underlying early-onset motor neuron diseases like spinal muscular atrophy (SMA) and developing therapeutic strategies designed to treat these disorders. SMA results from reduced expression of SMN (survival motor neuron) protein caused by the loss or mutation of the SMN1 gene. In this pilot grant, the MNDRL will identify those compounds within AurimMed’s library of orally bioavailable, CNS penetrant compounds which increase the expression of SMN2, an endogenous modifier of SMA severity. Dr. Butchbach will use the data obtained for these screens to identify those compounds which will be brought forward in preclinical drug trials using mouse models for SMA. This pilot grant, along with support from the CPR and Nemours Biomedical Research, will help Dr. Butchbach obtain further extramural support need to facilitate his establishment as an independent researcher.