CX-5461 activates the DNA damage response and demonstrates therapeutic efficacy in high-grade serous ovarian cancer

Nat Commun. 2020 May 26;11(1):2641. doi: 10.1038/s41467-020-16393-4.

Abstract

Acquired resistance to PARP inhibitors (PARPi) is a major challenge for the clinical management of high grade serous ovarian cancer (HGSOC). Here, we demonstrate CX-5461, the first-in-class inhibitor of RNA polymerase I transcription of ribosomal RNA genes (rDNA), induces replication stress and activates the DNA damage response. CX-5461 co-operates with PARPi in exacerbating replication stress and enhances therapeutic efficacy against homologous recombination (HR) DNA repair-deficient HGSOC-patient-derived xenograft (PDX) in vivo. We demonstrate CX-5461 has a different sensitivity spectrum to PARPi involving MRE11-dependent degradation of replication forks. Importantly, CX-5461 exhibits in vivo single agent efficacy in a HGSOC-PDX with reduced sensitivity to PARPi by overcoming replication fork protection. Further, we identify CX-5461-sensitivity gene expression signatures in primary and relapsed HGSOC. We propose CX-5461 is a promising therapy in combination with PARPi in HR-deficient HGSOC and also as a single agent for the treatment of relapsed disease.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Benzothiazoles / pharmacology*
  • Cell Line, Tumor
  • Cystadenocarcinoma, Serous / drug therapy*
  • Cystadenocarcinoma, Serous / genetics
  • Cystadenocarcinoma, Serous / metabolism
  • DNA Damage*
  • DNA Replication / drug effects
  • Drug Resistance, Neoplasm
  • Enzyme Inhibitors / pharmacology
  • Female
  • Heterografts
  • Homologous Recombination
  • Humans
  • Mice
  • Mice, Inbred NOD
  • Mice, Knockout
  • Mice, SCID
  • Models, Biological
  • Naphthyridines / pharmacology*
  • Ovarian Neoplasms / drug therapy*
  • Ovarian Neoplasms / genetics
  • Ovarian Neoplasms / metabolism
  • Poly(ADP-ribose) Polymerase Inhibitors / pharmacology
  • RNA Polymerase I / antagonists & inhibitors
  • Transcriptome

Substances

  • Benzothiazoles
  • CX 5461
  • Enzyme Inhibitors
  • Naphthyridines
  • Poly(ADP-ribose) Polymerase Inhibitors
  • RNA Polymerase I