Trastuzumab plus nivolumab with gemcitabine and cisplatin as first-line therapy for HER2-positive unresectable biliary tract cancer: a phase 1b/2 trial

A phase 1b/2 trial showed encouraging response rates when first-line trastuzumab, the HER2-targeted antibody, was added to the checkpoint inhibitor nivolumab plus standard gemcitabine and cisplatin chemotherapy in patients with unresectable biliary tract cancer. The level of HER2 expression in tumours correlated with clinical benefit, supporting its use as a biomarker to select patients for this quadruple regimen. Biliary tract cancers are rare and carry a poor prognosis, so an early-phase signal in the first-line setting is notable, but randomised phase 3 confirmation is still required before practice changes.