Biliary tract cancer research focuses on chemoimmunotherapy combinations, FGFR2 and IDH1-directed targeted therapy, and molecular profiling of cholangiocarcinoma subtypes.
PATHFINDER 2, a prospective, interventional cohort study of 35,878 adults aged 50 and older with no clinical suspicion of cancer, evaluated the performance and safety of a blood-based multi-cancer early detection (MCED) test that analyzes cell-free DNA methylation patterns to detect a shared cancer signal and predict its tissue of origin.
A single-arm, phase 1b/2 trial (HERBOT) tested adding the HER2-targeted antibody trastuzumab to first-line nivolumab plus gemcitabine-cisplatin chemoimmunotherapy in 40 patients with HER2-positive, previously untreated, unresectable or metastatic biliary tract cancer — the first prospective study to introduce HER2-directed therapy immediately rather than reserving it for a later treatment line.
A randomized phase II trial testing whether the CD27 costimulatory agonist varlilumab, combined with atezolizumab and the MEK inhibitor cobimetinib, could rescue checkpoint-inhibitor activity in previously treated, unresectable biliary tract cancer closed early for futility after a preplanned interim analysis.
A registry-based analysis of 11,681 US interventional cancer trials registered on ClinicalTrials.gov between 2008 and 2024 found that industry sponsors now outnumber federal sponsors nearly 5 to 1 (4.53-to-1 overall), with the ratio widening from 2.72-to-1 in 2008–2012 to 6.44-to-1 in 2019–2024.
Final post hoc analysis of the global, double-blind, placebo-controlled phase 3 TOPAZ-1 trial (685 patients randomized 1:1) reporting the longest published follow-up — approximately 4 years — of an immunotherapy-plus-chemotherapy regimen in first-line advanced biliary tract cancer (aBTC)