Garcinoic Acid Is a Natural and Selective Agonist of Pregnane X Receptor

J Med Chem. 2020 Apr 9;63(7):3701-3712. doi: 10.1021/acs.jmedchem.0c00012. Epub 2020 Mar 20.

Abstract

Pregnane X receptor (PXR) is a master xenobiotic-sensing transcription factor and a validated target for immune and inflammatory diseases. The identification of chemical probes to investigate the therapeutic relevance of the receptor is still highly desired. In fact, currently available PXR ligands are not highly selective and can exhibit toxicity and/or potential off-target effects. In this study, we have identified garcinoic acid as a selective and efficient PXR agonist. The properties of this natural molecule as a specific PXR agonist were demonstrated by the screening on a panel of nuclear receptors, the assessment of the physical and thermodynamic binding affinity, and the determination of the PXR-garcinoic acid complex crystal structure. Cytotoxicity, transcriptional, and functional properties were investigated in human liver cells, and compound activity and target engagement were confirmed in vivo in mouse liver and gut tissue. In conclusion, garcinoic acid is a selective natural agonist of PXR and a promising lead compound toward the development of new PXR-regulating modulators.

Publication types

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

MeSH terms

  • ATP Binding Cassette Transporter, Subfamily B, Member 1 / metabolism
  • Animals
  • Benzopyrans / metabolism
  • Benzopyrans / pharmacology*
  • Benzopyrans / toxicity
  • Cell Line, Tumor
  • Crystallography, X-Ray
  • Cytochrome P-450 CYP3A / metabolism
  • Gene Expression / drug effects
  • Humans
  • Liver / metabolism
  • Male
  • Mice, Inbred C57BL
  • Pregnane X Receptor / agonists*
  • Pregnane X Receptor / metabolism

Substances

  • ATP Binding Cassette Transporter, Subfamily B, Member 1
  • Benzopyrans
  • Pregnane X Receptor
  • garcinoic acid
  • Cytochrome P-450 CYP3A
  • cytochrome P450 3A4, mouse