Allosteric modulation of the farnesoid X receptor by a small molecule

Sci Rep. 2018 May 1;8(1):6846. doi: 10.1038/s41598-018-25158-5.

Abstract

The bile acid activated transcription factor farnesoid X receptor (FXR) regulates numerous metabolic processes and is a rising target for the treatment of hepatic and metabolic disorders. FXR agonists have revealed efficacy in treating non-alcoholic steatohepatitis (NASH), diabetes and dyslipidemia. Here we characterize imatinib as first-in-class allosteric FXR modulator and report the development of an optimized descendant that markedly promotes agonist induced FXR activation in a reporter gene assay and FXR target gene expression in HepG2 cells. Differential effects of imatinib on agonist-induced bile salt export protein and small heterodimer partner expression suggest that allosteric FXR modulation could open a new avenue to gene-selective FXR modulators.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • ATP Binding Cassette Transporter, Subfamily B, Member 11 / metabolism
  • Allosteric Regulation
  • Bile Acids and Salts / metabolism
  • Gene Expression Regulation
  • HEK293 Cells
  • HeLa Cells
  • Hep G2 Cells
  • Humans
  • Imatinib Mesylate / analogs & derivatives
  • Imatinib Mesylate / chemistry
  • Imatinib Mesylate / pharmacology*
  • Receptors, Cytoplasmic and Nuclear / antagonists & inhibitors
  • Receptors, Cytoplasmic and Nuclear / chemistry
  • Receptors, Cytoplasmic and Nuclear / metabolism*
  • Transcription Factors* / analysis
  • Transcription Factors* / metabolism

Substances

  • ABCB11 protein, human
  • ATP Binding Cassette Transporter, Subfamily B, Member 11
  • Bile Acids and Salts
  • Receptors, Cytoplasmic and Nuclear
  • Transcription Factors
  • nuclear receptor subfamily 0, group B, member 2
  • farnesoid X-activated receptor
  • Imatinib Mesylate