Discovery of small-molecule inhibitors selectively targeting the DNA-binding domain of the human androgen receptor

J Med Chem. 2014 Aug 14;57(15):6458-67. doi: 10.1021/jm500802j. Epub 2014 Aug 4.

Abstract

The human androgen receptor (AR) is considered as a master regulator in the development and progression of prostate cancer (PCa). As resistance to clinically used anti-AR drugs remains a major challenge for the treatment of advanced PCa, there is a pressing need for new anti-AR therapeutic avenues. In this study, we identified a binding site on the DNA binding domain (DBD) of the receptor and utilized virtual screening to discover a set of micromolar hits for the target. Through further exploration of the most potent hit (1), a structural analogue (6) was identified demonstrating 10-fold improved anti-AR potency. Further optimization resulted in a more potent synthetic analogue (25) with anti-AR potency comparable to a newly FDA-approved drug Enzalutamide. Site-directed mutagenesis demonstrated that the developed inhibitors do interact with the intended target site. Importantly, the AR DBD inhibitors could effectively inhibit the growth of Enzalutamide-resistant cells as well as block the transcriptional activity of constitutively active AR splice variants, such as V7.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, Non-P.H.S.

MeSH terms

  • Androgen Receptor Antagonists / chemical synthesis
  • Androgen Receptor Antagonists / chemistry*
  • Androgen Receptor Antagonists / pharmacology
  • Animals
  • Antineoplastic Agents / chemical synthesis
  • Antineoplastic Agents / chemistry*
  • Antineoplastic Agents / pharmacology
  • Benzamides
  • Binding Sites
  • Cell Line, Tumor
  • Cell Survival / drug effects
  • Computer Simulation
  • DNA / metabolism*
  • Databases, Chemical
  • Drug Resistance, Neoplasm
  • Humans
  • Imidazoles / chemical synthesis
  • Imidazoles / chemistry*
  • Imidazoles / pharmacology
  • Male
  • Molecular Conformation
  • Molecular Docking Simulation
  • Morpholines / chemical synthesis
  • Morpholines / chemistry*
  • Morpholines / pharmacology
  • Mutation
  • Nitriles
  • Phenylthiohydantoin / analogs & derivatives
  • Phenylthiohydantoin / pharmacology
  • Prostate-Specific Antigen / metabolism
  • Prostatic Neoplasms / drug therapy
  • Pyrvinium Compounds / chemistry
  • Pyrvinium Compounds / pharmacology
  • Rats
  • Receptors, Androgen / genetics
  • Receptors, Androgen / metabolism*
  • Structure-Activity Relationship
  • Thiazoles / chemical synthesis
  • Thiazoles / chemistry*
  • Thiazoles / pharmacology
  • Thiophenes / chemical synthesis
  • Thiophenes / chemistry
  • Thiophenes / pharmacology
  • Transcription, Genetic

Substances

  • 4-(4-(4,5-dibromo-1H-imidazol-1-yl)thiazol-2-yl)morpholine
  • Androgen Receptor Antagonists
  • Antineoplastic Agents
  • Benzamides
  • Imidazoles
  • Morpholines
  • Nitriles
  • Pyrvinium Compounds
  • Receptors, Androgen
  • Thiazoles
  • Thiophenes
  • Phenylthiohydantoin
  • pyrvinium
  • DNA
  • enzalutamide
  • Prostate-Specific Antigen