Structural Basis for Achieving GSK-3β Inhibition with High Potency, Selectivity, and Brain Exposure for Positron Emission Tomography Imaging and Drug Discovery

J Med Chem. 2019 Nov 14;62(21):9600-9617. doi: 10.1021/acs.jmedchem.9b01030. Epub 2019 Oct 21.

Abstract

Using structure-guided design, several cell based assays, and microdosed positron emission tomography (PET) imaging, we identified a series of highly potent, selective, and brain-penetrant oxazole-4-carboxamide-based inhibitors of glycogen synthase kinase-3 (GSK-3). An isotopologue of our first-generation lead, [3H]PF-367, demonstrates selective and specific target engagement in vitro, irrespective of the activation state. We discovered substantial ubiquitous GSK-3-specific radioligand binding in Tg2576 Alzheimer's disease (AD), suggesting application for these compounds in AD diagnosis and identified [11C]OCM-44 as our lead GSK-3 radiotracer, with optimized brain uptake by PET imaging in nonhuman primates. GSK-3β-isozyme selectivity was assessed to reveal OCM-51, the most potent (IC50 = 0.030 nM) and selective (>10-fold GSK-3β/GSK-3α) GSK-3β inhibitor known to date. Inhibition of CRMP2T514 and tau phosphorylation, as well as favorable therapeutic window against WNT/β-catenin signaling activation, was observed in cells.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Blood-Brain Barrier / metabolism
  • Brain / diagnostic imaging
  • Brain / metabolism*
  • Catalytic Domain
  • Drug Discovery*
  • Glycogen Synthase Kinase 3 beta / antagonists & inhibitors*
  • Glycogen Synthase Kinase 3 beta / chemistry
  • HEK293 Cells
  • Humans
  • Mice
  • Models, Molecular
  • Neuroimaging
  • Oxazoles / chemistry
  • Oxazoles / metabolism
  • Oxazoles / pharmacology
  • Positron-Emission Tomography / methods*
  • Protein Kinase Inhibitors / chemistry*
  • Protein Kinase Inhibitors / metabolism
  • Protein Kinase Inhibitors / pharmacology*
  • Triazoles / chemistry
  • Triazoles / metabolism
  • Triazoles / pharmacology

Substances

  • Oxazoles
  • PF-04802367
  • Protein Kinase Inhibitors
  • Triazoles
  • Glycogen Synthase Kinase 3 beta