Development of Novel Inhibitors Targeting the D-Box of the DNA Binding Domain of Androgen Receptor

Int J Mol Sci. 2021 Mar 2;22(5):2493. doi: 10.3390/ijms22052493.

Abstract

The inhibition of the androgen receptor (AR) is an established strategy in prostate cancer (PCa) treatment until drug resistance develops either through mutations in the ligand-binding domain (LBD) portion of the receptor or its deletion. We previously identified a druggable pocket on the DNA binding domain (DBD) dimerization surface of the AR and reported several potent inhibitors that effectively disrupted DBD-DBD interactions and consequently demonstrated certain antineoplastic activity. Here we describe further development of small molecule inhibitors of AR DBD dimerization and provide their broad biological characterization. The developed compounds demonstrate improved activity in the mammalian two-hybrid assay, enhanced inhibition of AR-V7 transcriptional activity, and improved microsomal stability. These findings position us for the development of AR inhibitors with entirely novel mechanisms of action that would bypass most forms of PCa treatment resistance, including the truncation of the LBD of the AR.

Keywords: androgen receptor; computer-aided drug discovery; dimerization; prostate cancer; small-molecule inhibitors.

MeSH terms

  • Androgen Receptor Antagonists / chemistry
  • Androgen Receptor Antagonists / pharmacology*
  • Computer Simulation
  • DNA, Neoplasm / antagonists & inhibitors
  • DNA, Neoplasm / metabolism*
  • Gene Expression Regulation, Neoplastic / drug effects*
  • High-Throughput Screening Assays
  • Humans
  • Male
  • Prostatic Neoplasms / drug therapy*
  • Prostatic Neoplasms / metabolism
  • Prostatic Neoplasms / pathology
  • Protein Conformation
  • Protein Domains
  • Receptors, Androgen / chemistry*
  • Receptors, Androgen / genetics
  • Receptors, Androgen / metabolism
  • Small Molecule Libraries / chemistry
  • Small Molecule Libraries / pharmacology*
  • Transcription, Genetic*
  • Tumor Cells, Cultured

Substances

  • AR protein, human
  • Androgen Receptor Antagonists
  • DNA, Neoplasm
  • Receptors, Androgen
  • Small Molecule Libraries