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First results of the multicohort phase 2 LEAP-005 study suggest that lenvatinib plus pembrolizumab combination immunotherapy has promising antitumor activity in patients with biliary tract cancer.

Preclinical evidence using an genetically engineered mouse model of IDH1-mutated intrahepatic CCA indicates that the selective IDH1 inhibitor ivosidenib mediates its antitumor activity by promoting hepatic progenitor-cell differentiation, and inducing T-cell–mediated antitumor immune response.

Data reported at the ASCO 2020 GI Cancers Symposium indicate that treatment with neoadjuvant definitive chemoradiation followed by orthotopic liver transplantation was associated with better outcomes compared with definitive chemoradiation alone in patients with unresectable extrahepatic/hilar CCA.

Molecular analyses of biliary tract cancers indicate that neurotrophic tyrosine receptor kinase (NTRK) gene fusions are rare, suggesting that it is not a major driver of BTC pathogenesis.

Preclinical data implicate the ELR+ chemokine/CXCR2 axes in the pathogenesis of CCA, warranting further studies to define its role in CCA.

Preliminary results of a phase 2 study suggest that the addition of the PI3K inhibitor copanlisib to chemotherapy in the first-line setting was not associated with survival benefit in patients with advanced CCA.

AKT and NOTCH intracellular domain (NICD) targets YAP and SOX9 might be potential targets for therapeutic intervention in intrahepatic CCA subsets, while cholestatic injury or NASH might induce hepatocyte-to-cholangiocyte reprogramming that increases risk of intrahepatic CCA.

Interim analysis of the part 2 dose-expansion portion of an ongoing 2-part phase 1 Japanese study shows that the FGFR1 tyrosine kinase inhibitor E7090 was active with a manageable safety profile in CCA patients with FGFR2 gene fusion.

Preliminary recommendations made based on cases from the SOLAR-1 trial suggest that alpelisib-induced hyperglycemia can be managed with early detection, close monitoring, and prompt intervention.

For patients with early HR-positive, HER2-positive breast cancer who initiated neratinib within 1 year of completing trastuzumab and in patients who did not achieve a pathologic complete response after neoadjuvant treatment, neratinib may be a cost-effective treatment option.