A Novel Cell-Based Assay to Identify Small Molecules for Galactocerebrosidase (GALC)

National Center For Advancing Translational Sciences

Investigators

Abstract

The NCATS Early Translation Branch (ETB) hosts a broad and comprehensive program for the discovery of drug candidates directed towards rare diseases and pharmacological tools to probe the function of the human genome. ETB conducts research to understand the underlying principles driving the translation of basic research discoveries into tangible improvements in human health. During this reporting period, the collaborative team has worked on validation and characterization of hit compounds previously identified in the primary GALC screen. Additionally, a medium-throughput assay to detect psychosine from iPSC derived oligodendrocytes using mass spectrometry has been advanced. This assay will enable the quantification of lipid reduction upon treatment with analogs of hit molecules. Structure activity relationship studies are in progress with a selected chemotype.