PARP Theranostic Auger Emitters Are Cytotoxic in BRCA Mutant Ovarian Cancer and Viable Tumors from Ovarian Cancer Patients Enable Ex-Vivo Screening of Tumor Response

Molecules. 2020 Dec 19;25(24):6029. doi: 10.3390/molecules25246029.

Abstract

Theranostics are emerging as a pillar of cancer therapy that enable the use of single molecule constructs for diagnostic and therapeutic application. As poly adenosine diphosphate (ADP)-ribose polymerase 1 (PARP-1) is overexpressed in various cancer types, and is localized to the nucleus, PARP-1 can be safely targeted with Auger emitters to induce DNA damage in tumors. Here, we investigated a radioiodinated PARP inhibitor, [125I]KX1, and show drug target specific DNA damage and subsequent killing of BRCA1 and non-BRCA mutant ovarian cancer cells at sub-pharmacological concentrations several orders of magnitude lower than traditional PARP inhibitors. Furthermore, we demonstrated that viable tumor tissue from ovarian cancer patients can be used to screen tumor radiosensitivity ex-vivo, enabling the direct assessment of therapeutic efficacy. Finally, we showed tumors can be imaged by single-photon computed tomography (SPECT) with PARP theranostic, [123I]KX1, in a human ovarian cancer xenograft mouse model. These data support the utility of PARP-1 targeted radiopharmaceutical therapy as a theranostic option for PARP-1 overexpressing ovarian cancers.

Keywords: Auger therapy; BRCA1; HRD; PARP-1; ovarian cancer; radiopharmaceutical therapy; theranostic.

MeSH terms

  • Animals
  • Antineoplastic Agents / pharmacology*
  • BRCA1 Protein / genetics*
  • Cell Line, Tumor
  • DNA Damage / drug effects
  • DNA Damage / genetics
  • Disease Models, Animal
  • Female
  • Heterografts
  • Humans
  • Iodine Radioisotopes / pharmacology
  • Mice, SCID
  • Ovarian Neoplasms / drug therapy*
  • Ovarian Neoplasms / genetics*
  • Poly (ADP-Ribose) Polymerase-1 / genetics*
  • Poly(ADP-ribose) Polymerase Inhibitors / pharmacology*

Substances

  • Antineoplastic Agents
  • BRCA1 Protein
  • BRCA1 protein, human
  • Iodine Radioisotopes
  • Poly(ADP-ribose) Polymerase Inhibitors
  • PARP1 protein, human
  • Poly (ADP-Ribose) Polymerase-1
  • Iodine-125