CX-5461 is a DNA G-quadruplex stabilizer with selective lethality in BRCA1/2 deficient tumours

Nat Commun. 2017 Feb 17:8:14432. doi: 10.1038/ncomms14432.

Abstract

G-quadruplex DNAs form four-stranded helical structures and are proposed to play key roles in different cellular processes. Targeting G-quadruplex DNAs for cancer treatment is a very promising prospect. Here, we show that CX-5461 is a G-quadruplex stabilizer, with specific toxicity against BRCA deficiencies in cancer cells and polyclonal patient-derived xenograft models, including tumours resistant to PARP inhibition. Exposure to CX-5461, and its related drug CX-3543, blocks replication forks and induces ssDNA gaps or breaks. The BRCA and NHEJ pathways are required for the repair of CX-5461 and CX-3543-induced DNA damage and failure to do so leads to lethality. These data strengthen the concept of G4 targeting as a therapeutic approach, specifically for targeting HR and NHEJ deficient cancers and other tumours deficient for DNA damage repair. CX-5461 is now in advanced phase I clinical trial for patients with BRCA1/2 deficient tumours (Canadian trial, NCT02719977, opened May 2016).

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • BRCA1 Protein / deficiency*
  • BRCA2 Protein / deficiency*
  • Base Sequence
  • Benzothiazoles / pharmacology*
  • Benzothiazoles / therapeutic use*
  • Benzoxazines / pharmacology
  • Caenorhabditis elegans / drug effects
  • Cell Line, Tumor
  • Chromosomal Instability / genetics
  • DNA Damage
  • DNA Repair / drug effects
  • DNA Replication / drug effects
  • DNA, Ribosomal / genetics
  • Female
  • G-Quadruplexes* / drug effects
  • Genome, Human
  • Genotype
  • Homologous Recombination / drug effects
  • Humans
  • Mice
  • Naphthyridines / pharmacology*
  • Naphthyridines / therapeutic use*
  • Neoplasms / drug therapy*
  • Quinolones / pharmacology
  • Saccharomyces cerevisiae / metabolism
  • Transcription, Genetic / drug effects
  • Xenograft Model Antitumor Assays

Substances

  • BRCA1 Protein
  • BRCA2 Protein
  • Benzothiazoles
  • Benzoxazines
  • CX 3543
  • CX 5461
  • DNA, Ribosomal
  • Naphthyridines
  • Quinolones

Associated data

  • ClinicalTrials.gov/NCT02719977