AMPK synthetic activators

National Center For Advancing Translational Sciences

Investigators

Abstract

During this period the collaborative team has worked to characterize a number of small molecule hits previously found to activate 5' adenosine monophosphate-activated protein kinase (AMPK). The team has optimized select chemotypes via medicinal chemistry efforts and implemented a novel high-throughput mass spectrometry assay to measure cholesterol to test activity of the compounds. Top actives were tested in the context of Niemann Pick C, a lysosomal storage disease characterized by cholesterol accumulation. The team is utilizing mass spectrometry data to nominate a small number of compounds for additional cell based characterization, followed by in vivo efficacy studies.