Enhancing therapeutic efficacy in homologous recombination-proficient pancreatic cancer via the combination of PARP1-PROTAC and a BRD4 inhibitor

Bioorg Med Chem. 2024 Dec 1:115:117970. doi: 10.1016/j.bmc.2024.117970. Epub 2024 Oct 26.

Abstract

Currently, poly (ADP-ribose) polymerase inhibitors (PARPi) have been approved by U.S. Food and Drug Administration for BRCA-mutated pancreatic cancer therapy. However, limited indications hinder their further application. Repression of bromodomain-containing protein 4 (BRD4) can block the homologous recombination (HR) repair pathway and has the potential to enhance the response to PARPi in HR-proficient pancreatic cancer therapy. In addition, proteolysis targeting chimeras (PROTACs) can hijack E3 ligase within the cell to ubiquitinate degradation-targeted proteins effectively and quickly, thus enhancing the therapeutic effect on tumors. In the present study, the LB23 compound, which induces PARP1 degradation, was employed in combination with the BRD4 inhibitor JQ1, confirming their synergistic effect in HR-proficient pancreatic cancer through various methods. Moreover, compared to the JQ1 and PARPi olaparib combination, PARP1-PROTAC and JQ1 had more notable synergistic effects. Further research into the synergistic mechanism demonstrated that combination therapy enhanced DNA damage and suppressed DNA repair by inducing cell cycle arrest and cell apoptosis. The present study therefore provides the experimental data for this type of combination therapy, which is expected to be an innovative approach for the treatment of HR-proficient pancreatic cancer.

Keywords: BRD4 inhibiton; DNA repair and damage; PARP1-PROTAC; Pancreatic cancer; Synthetic lethality.

MeSH terms

  • Antineoplastic Agents* / chemical synthesis
  • Antineoplastic Agents* / chemistry
  • Antineoplastic Agents* / pharmacology
  • Apoptosis* / drug effects
  • Azepines / chemistry
  • Azepines / pharmacology
  • Bromodomain Containing Proteins
  • Cell Cycle Proteins* / antagonists & inhibitors
  • Cell Cycle Proteins* / metabolism
  • Cell Line, Tumor
  • Cell Proliferation* / drug effects
  • Dose-Response Relationship, Drug
  • Drug Screening Assays, Antitumor
  • Homologous Recombination / drug effects
  • Humans
  • Molecular Structure
  • Pancreatic Neoplasms* / drug therapy
  • Pancreatic Neoplasms* / pathology
  • Poly (ADP-Ribose) Polymerase-1* / antagonists & inhibitors
  • Poly (ADP-Ribose) Polymerase-1* / metabolism
  • Poly(ADP-ribose) Polymerase Inhibitors* / chemical synthesis
  • Poly(ADP-ribose) Polymerase Inhibitors* / chemistry
  • Poly(ADP-ribose) Polymerase Inhibitors* / pharmacology
  • Proteolysis / drug effects
  • Structure-Activity Relationship
  • Transcription Factors* / antagonists & inhibitors
  • Transcription Factors* / metabolism
  • Triazoles / chemistry
  • Triazoles / pharmacology

Substances

  • BRD4 protein, human
  • Transcription Factors
  • Poly (ADP-Ribose) Polymerase-1
  • Cell Cycle Proteins
  • PARP1 protein, human
  • Poly(ADP-ribose) Polymerase Inhibitors
  • Antineoplastic Agents
  • Triazoles
  • (+)-JQ1 compound
  • Azepines
  • Bromodomain Containing Proteins