Discovery of Small Molecules Targeting the Synergy of Cardiac Transcription Factors GATA4 and NKX2-5

J Med Chem. 2017 Sep 28;60(18):7781-7798. doi: 10.1021/acs.jmedchem.7b00816. Epub 2017 Sep 15.

Abstract

Transcription factors are pivotal regulators of gene transcription, and many diseases are associated with the deregulation of transcriptional networks. In the heart, the transcription factors GATA4 and NKX2-5 are required for cardiogenesis. GATA4 and NKX2-5 interact physically, and the activation of GATA4, in cooperation with NKX2-5, is essential for stretch-induced cardiomyocyte hypertrophy. Here, we report the identification of four small molecule families that either inhibit or enhance the GATA4-NKX2-5 transcriptional synergy. A fragment-based screening, reporter gene assay, and pharmacophore search were utilized for the small molecule screening, identification, and optimization. The compounds modulated the hypertrophic agonist-induced cardiac gene expression. The most potent hit compound, N-[4-(diethylamino)phenyl]-5-methyl-3-phenylisoxazole-4-carboxamide (3, IC50 = 3 μM), exhibited no activity on the protein kinases involved in the regulation of GATA4 phosphorylation. The identified and chemically and biologically characterized active compound, and its derivatives may provide a novel class of small molecules for modulating heart regeneration.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Cell Line
  • GATA4 Transcription Factor / agonists
  • GATA4 Transcription Factor / antagonists & inhibitors
  • GATA4 Transcription Factor / metabolism*
  • Homeobox Protein Nkx-2.5 / agonists
  • Homeobox Protein Nkx-2.5 / antagonists & inhibitors
  • Homeobox Protein Nkx-2.5 / metabolism*
  • Humans
  • Isoxazoles / chemistry*
  • Isoxazoles / pharmacology*
  • Mice
  • Models, Molecular
  • Protein Interaction Maps / drug effects
  • Small Molecule Libraries / chemistry*
  • Small Molecule Libraries / pharmacology*
  • Transcriptional Activation / drug effects*

Substances

  • GATA4 Transcription Factor
  • Homeobox Protein Nkx-2.5
  • Isoxazoles
  • Small Molecule Libraries