NUC-1031 in biliary tract cancer: from bench to bedside and back?

Cancer Chemother Pharmacol. 2020 Jun;85(6):1011-1014. doi: 10.1007/s00280-020-04080-6. Epub 2020 May 31.


In preclinical models of biliary tract cancer, NUC-1031 showed less potency than gemcitabine, no correlation with potential biomarkers and only moderate additive interaction in combination with cisplatin. These findings should prompt further careful pharmacological and translational studies to better define the purported therapeutic advantage of NUC-1031 over gemcitabine. That would be a more cautious approach than the phase III clinical trial which is planning to enrol 828 patients with biliary tract tumours to compare gemcitabine/cisplatin "conventional" treatment with or without NUC-1031.

Publication types

  • Editorial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antineoplastic Agents / therapeutic use*
  • Biliary Tract Neoplasms / drug therapy*
  • Biliary Tract Neoplasms / pathology
  • Cytidine Monophosphate / analogs & derivatives*
  • Cytidine Monophosphate / therapeutic use
  • Humans
  • Prognosis
  • Translational Research, Biomedical*


  • Antineoplastic Agents
  • NUC-1031
  • Cytidine Monophosphate