Leveraging an NQO1 Bioactivatable Drug for Tumor-Selective Use of Poly(ADP-ribose) Polymerase Inhibitors

Cancer Cell. 2016 Dec 12;30(6):940-952. doi: 10.1016/j.ccell.2016.11.006.

Abstract

Therapeutic drugs that block DNA repair, including poly(ADP-ribose) polymerase (PARP) inhibitors, fail due to lack of tumor-selectivity. When PARP inhibitors and β-lapachone are combined, synergistic antitumor activity results from sustained NAD(P)H levels that refuel NQO1-dependent futile redox drug recycling. Significant oxygen-consumption-rate/reactive oxygen species cause dramatic DNA lesion increases that are not repaired due to PARP inhibition. In NQO1+ cancers, such as non-small-cell lung, pancreatic, and breast cancers, cell death mechanism switches from PARP1 hyperactivation-mediated programmed necrosis with β-lapachone monotherapy to synergistic tumor-selective, caspase-dependent apoptosis with PARP inhibitors and β-lapachone. Synergistic antitumor efficacy and prolonged survival were noted in human orthotopic pancreatic and non-small-cell lung xenograft models, expanding use and efficacy of PARP inhibitors for human cancer therapy.

Keywords: ARQ761; NQO1; NQO1 bioctivatable drugs; NSCLC; Olaparib; PARP1; PDA; ROS; Rucaparib; combination chemotherapy; synthetic lethality; β-lapachone.

MeSH terms

  • Animals
  • Carcinoma, Non-Small-Cell Lung / drug therapy*
  • Carcinoma, Non-Small-Cell Lung / genetics
  • Cell Line, Tumor
  • Cell Proliferation / drug effects
  • Cell Survival / drug effects
  • DNA Damage
  • Drug Synergism
  • Gene Expression Regulation, Neoplastic
  • Humans
  • Lung Neoplasms / drug therapy*
  • Lung Neoplasms / genetics
  • Mice
  • NAD(P)H Dehydrogenase (Quinone) / genetics*
  • Naphthoquinones / administration & dosage*
  • Naphthoquinones / pharmacology
  • Pancreatic Neoplasms / drug therapy*
  • Pancreatic Neoplasms / genetics
  • Poly(ADP-ribose) Polymerase Inhibitors / administration & dosage*
  • Reactive Oxygen Species / metabolism
  • Up-Regulation
  • Xenograft Model Antitumor Assays

Substances

  • Naphthoquinones
  • Poly(ADP-ribose) Polymerase Inhibitors
  • Reactive Oxygen Species
  • beta-lapachone
  • NAD(P)H Dehydrogenase (Quinone)
  • NQO1 protein, human