A Fluorescence Polarization Activity-Based Protein Profiling Assay in the Discovery of Potent, Selective Inhibitors for Human Nonlysosomal Glucosylceramidase

J Am Chem Soc. 2017 Oct 11;139(40):14192-14197. doi: 10.1021/jacs.7b07352. Epub 2017 Sep 29.

Abstract

Human nonlysosomal glucosylceramidase (GBA2) is one of several enzymes that controls levels of glycolipids and whose activity is linked to several human disease states. There is a major need to design or discover selective GBA2 inhibitors both as chemical tools and as potential therapeutic agents. Here, we describe the development of a fluorescence polarization activity-based protein profiling (FluoPol-ABPP) assay for the rapid identification, from a 350+ library of iminosugars, of GBA2 inhibitors. A focused library is generated based on leads from the FluoPol-ABPP screen and assessed on GBA2 selectivity offset against the other glucosylceramide metabolizing enzymes, glucosylceramide synthase (GCS), lysosomal glucosylceramidase (GBA), and the cytosolic retaining β-glucosidase, GBA3. Our work, yielding potent and selective GBA2 inhibitors, also provides a roadmap for the development of high-throughput assays for identifying retaining glycosidase inhibitors by FluoPol-ABPP on cell extracts containing recombinant, overexpressed glycosidase as the easily accessible enzyme source.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Drug Evaluation, Preclinical / methods
  • Enzyme Assays / methods*
  • Enzyme Inhibitors / chemistry
  • Enzyme Inhibitors / pharmacology*
  • Fluorescence Polarization / methods*
  • Glucosylceramidase
  • Humans
  • Imino Sugars / chemistry
  • Imino Sugars / pharmacology*
  • beta-Glucosidase / antagonists & inhibitors*
  • beta-Glucosidase / metabolism

Substances

  • Enzyme Inhibitors
  • Imino Sugars
  • beta-Glucosidase
  • GBA2 protein, human
  • Glucosylceramidase