p97 inhibitors for treatment of IBMPFD/ALS

National Center For Advancing Translational Sciences

Investigators

Abstract

This collaborative team aims to identify compounds that are capable of inhibiting p97, with a view to developing novel therapies for Inclusion Body Myopathy with Early-Onset Paget Disease of Bone and/or Frontotemporal Dementia (IBMPFD) and Amyotrophic Lateral Sclerosis (ALS). During this period, the collaborative team worked to further characterize and validate hit compounds previously identified through screening for inhibitors of p97 ATPase activity. Screening of an additional 40k compounds was performed. Candidate p97 activators and inhibitors were selected for testing in orthogonal assays and cell-based models in the Chou lab. The team is also assessing virtual screening approaches using structural models of p97.