BRD4-specific PROTAC inhibits basal-like breast cancer partially through downregulating KLF5 expression

Oncogene. 2024 Sep;43(39):2914-2926. doi: 10.1038/s41388-024-03121-1. Epub 2024 Aug 20.

Abstract

Interest in the use of proteolysis-targeting chimeras (PROTACs) in cancer therapy has increased in recent years. Targeting bromodomain and extra terminal domain (BET) proteins, especially bromodomain-containing protein 4 (BRD4), has shown inhibitory effects on basal-like breast cancer (BLBC). However, the bioavailability of BRD4 PROTACs is restricted by their non-selective biodegradability and low tumor-targeting ability. We demonstrated that 6b (BRD4 PROTAC) suppresses BLBC cell growth by targeting BRD4, but not BRD2 and BRD3, for cereblon (CRBN)-mediated ubiquitination and proteasomal degradation. Compound 6b also inhibited expression of Krüppel-like factor 5 (KLF5) transcription factor, a key oncoprotein in BLBC, controlled by BRD4-mediated super-enhancers. Moreover, 6b inhibited HCC1806 tumor growth in a xenograft mouse model. The combination of 6b and KLF5 inhibitors showed additive effects on BLBC. These results suggest that BRD4-specific PROTAC can effectively inhibit BLBC by downregulating KLF5, and that 6b has potential as a novel therapeutic drug for BLBC.

MeSH terms

  • Animals
  • Breast Neoplasms* / drug therapy
  • Breast Neoplasms* / genetics
  • Breast Neoplasms* / metabolism
  • Breast Neoplasms* / pathology
  • Bromodomain Containing Proteins
  • Cell Cycle Proteins* / antagonists & inhibitors
  • Cell Cycle Proteins* / metabolism
  • Cell Line, Tumor
  • Cell Proliferation / drug effects
  • Down-Regulation / drug effects
  • Female
  • Gene Expression Regulation, Neoplastic / drug effects
  • Humans
  • Kruppel-Like Transcription Factors* / genetics
  • Kruppel-Like Transcription Factors* / metabolism
  • Mice
  • Mice, Nude
  • Proteolysis* / drug effects
  • Transcription Factors* / antagonists & inhibitors
  • Transcription Factors* / metabolism
  • Ubiquitination / drug effects
  • Xenograft Model Antitumor Assays*

Substances

  • KLF5 protein, human
  • Kruppel-Like Transcription Factors
  • BRD4 protein, human
  • Transcription Factors
  • Cell Cycle Proteins
  • Bromodomain Containing Proteins