Selectivity, ligand deconstruction, and cellular activity analysis of a BPTF bromodomain inhibitor

Org Biomol Chem. 2019 Feb 13;17(7):2020-2027. doi: 10.1039/c8ob02599a.

Abstract

Bromodomain and PHD finger containing protein transcription factor (BPTF) is an epigenetic protein involved in chromatin remodelling and is a potential anticancer target. The BPTF bromodomain has one reported small molecule inhibitor (AU1, rac-1). Here, advances made on the structure-activity relationship of a BPTF bromodomain ligand are reported using a combination of experimental and molecular dynamics simulations leading to the active enatiomer (S)-1. Additionally, a ligand deconstruction analysis was conducted to characterize important pharmacophores for engaging the BPTF bromodomain. These studies have been enabled by a protein-based fluorine NMR approach, highlighting the versatility of the method for selectivity, ligand deconstruction, and ligand binding. To enable future analysis of biological activity, cell growth analyses in a panel of cancer cell lines were carried out using CRISPR-Cas9 and (S)-1 to identify cell-based model systems that are sensitive to BPTF inhibition.

Publication types

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

MeSH terms

  • Antigens, Nuclear
  • Cell Proliferation
  • Crystallography, X-Ray
  • Humans
  • Ligands
  • Magnetic Resonance Spectroscopy
  • Molecular Dynamics Simulation
  • Molecular Structure
  • Nerve Tissue Proteins / antagonists & inhibitors*
  • Pyrazoles / chemical synthesis
  • Pyrazoles / chemistry
  • Pyrazoles / pharmacology*
  • Pyridines / chemical synthesis
  • Pyridines / chemistry
  • Pyridines / pharmacology*
  • Small Molecule Libraries / chemical synthesis
  • Small Molecule Libraries / chemistry
  • Small Molecule Libraries / pharmacology*
  • Structure-Activity Relationship
  • Transcription Factors / antagonists & inhibitors*

Substances

  • Antigens, Nuclear
  • Ligands
  • Nerve Tissue Proteins
  • Pyrazoles
  • Pyridines
  • Small Molecule Libraries
  • Transcription Factors
  • fetal Alzheimer antigen
  • rac-1 compound