Novel Pyrrolopyridone Bromodomain and Extra-Terminal Motif (BET) Inhibitors Effective in Endocrine-Resistant ER+ Breast Cancer with Acquired Resistance to Fulvestrant and Palbociclib

J Med Chem. 2020 Jul 9;63(13):7186-7210. doi: 10.1021/acs.jmedchem.0c00456. Epub 2020 Jun 15.

Abstract

Acquired resistance to fulvestrant and palbociclib is a new challenge to treatment of estrogen receptor positive (ER+) breast cancer. ER is expressed in most resistance settings; thus, bromodomain and extra-terminal protein inhibitors (BETi) that target BET-amplified ER-mediated transcription have therapeutic potential. Novel pyrrolopyridone BETi leveraged novel interactions with L92/L94 confirmed by a cocrystal structure of 27 with BRD4. Optimization of BETi using growth inhibition in fulvestrant-resistant (MCF-7:CFR) cells was confirmed in endocrine-resistant, palbociclib-resistant, and ESR1 mutant cell lines. 27 was more potent in MCF-7:CFR cells than six BET inhibitors in clinical trials. Transcriptomic analysis differentiated 27 from the benchmark BETi, JQ-1, showing downregulation of oncogenes and upregulation of tumor suppressors and apoptosis. The therapeutic approach was validated by oral administration of 27 in orthotopic xenografts of endocrine-resistant breast cancer in monotherapy and in combination with fulvestrant. Importantly, at an equivalent dose in rats, thrombocytopenia was mitigated.

Publication types

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

MeSH terms

  • Animals
  • Breast Neoplasms / metabolism
  • Breast Neoplasms / pathology*
  • Drug Resistance, Neoplasm / drug effects*
  • Fulvestrant / pharmacology*
  • Humans
  • MCF-7 Cells
  • Mice
  • Models, Molecular
  • Piperazines / pharmacology*
  • Protein Domains
  • Pyridines / pharmacology*
  • Pyridones / chemistry*
  • Pyridones / pharmacokinetics
  • Pyridones / pharmacology*
  • Receptors, Estrogen / metabolism
  • Tissue Distribution
  • Transcription Factors / antagonists & inhibitors*
  • Transcription Factors / chemistry
  • Xenograft Model Antitumor Assays

Substances

  • Piperazines
  • Pyridines
  • Pyridones
  • Receptors, Estrogen
  • Transcription Factors
  • Fulvestrant
  • palbociclib