GPR52 Antagonist Reduces Huntingtin Levels and Ameliorates Huntington's Disease-Related Phenotypes

J Med Chem. 2021 Jan 28;64(2):941-957. doi: 10.1021/acs.jmedchem.0c01133. Epub 2020 Nov 13.

Abstract

GPR52 is an orphan G protein-coupled receptor (GPCR) that has been recently implicated as a potential drug target of Huntington's disease (HD), an incurable monogenic neurodegenerative disorder. In this research, we found that striatal knockdown of GPR52 reduces mHTT levels in adult HdhQ140 mice, validating GPR52 as an HD target. In addition, we discovered a highly potent and specific GPR52 antagonist Comp-43 with an IC50 value of 0.63 μM by a structure-activity relationship (SAR) study. Further studies showed that Comp-43 reduces mHTT levels by targeting GPR52 and promotes survival of mouse primary striatal neurons. Moreover, in vivo study showed that Comp-43 not only reduces mHTT levels but also rescues HD-related phenotypes in HdhQ140 mice. Taken together, our study confirms that inhibition of GPR52 is a promising strategy for HD therapy, and the GPR52 antagonist Comp-43 might serve as a lead compound for further investigation.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Behavior, Animal / drug effects
  • Cell Survival / drug effects
  • Cells, Cultured
  • Corpus Striatum / cytology
  • Corpus Striatum / drug effects
  • Drug Design
  • Humans
  • Huntingtin Protein / biosynthesis*
  • Huntington Disease / drug therapy*
  • Huntington Disease / physiopathology
  • Huntington Disease / psychology
  • Mice
  • Models, Molecular
  • Neurons / drug effects
  • Receptors, G-Protein-Coupled / antagonists & inhibitors*
  • Structure-Activity Relationship

Substances

  • GPR52 protein, human
  • HTT protein, human
  • Huntingtin Protein
  • Receptors, G-Protein-Coupled