Cholangiocarcinoma

ABC is an independent prognostic factor for OS and DFS in patients undergoing radical surgery for ICC. The preoperative clinical staging of patients with ICC should fully consider the anatomical, biological, and PS factors.

RARγ also acts as a co-factor to Smad3 and reduced or enhanced TGFβ-driven and Smad3-mediated events when liganded and non-liganded, respectively. Collectively the findings support the view that RARγ plays a crucial role in controlling stem and progenitor cell behavior.

Selective FGFR tyrosine kinase inhibitors, including the reversible inhibitor pemigatinib and the irreversible inhibitor futibatinib, have demonstrated clinically meaningful response rates and durable disease control in patients with previously treated FGFR2-altered iCCA, leading to regulatory approvals and the incorporation of FGFR inhibition into contemporary treatment paradigms. In addition, we highlight the growing role of circulating tumor DNA as a noninvasive tool for longitudinal molecular monitoring and treatment guidance. Together, these insights underscore the central role of FGFR2-directed therapy in precision oncology for biliary tract cancer and provide a framework for optimizing and extending targeted treatment in this molecularly defined disease subset.

P1/2, N=7, Terminated, Servier Bio-Innovation LLC | Completed --> Terminated; The sponsor decided to terminate the study during the safety lead-in phase.

P2, N=224, Recruiting, AstraZeneca | Trial completion date: Dec 2026 --> Sep 2027 | Trial primary completion date: Dec 2025 --> Aug 2026

Adding ivosidenib to gemcitabine and cisplatin in this study demonstrated a challenging safety profile in advanced CCA. Further research and dose optimization are warranted to confirm these findings and optimize the integration of targeted therapies into first-line regimens.

Co-targeting SOX9 and YAP1 offers a promising and safe broad-spectrum preventive/therapeutic approach for iCCA, potentially overcoming resistance to YAP1 inhibition. The adaptive resistance mechanism identified may extend to other malignancies, providing insights for addressing the advanced resistant to YAP1-TEAD-directed therapies.

Our pan-cancer analysis highlights OGN as a context-dependent regulator linking extracellular matrix (ECM) remodeling with immune and angiogenic signaling. Its pan-cancer dysregulation, diagnostic/prognostic value, and crosstalk with immune evasion mechanisms nominate OGN as a promising multi-functional biomarker and therapeutic target.

Literature suggests that mucinous components in solid tumors are associated with a worse prognosis. In intrahepatic mucinous cholangiocarcinoma, surgery and chemotherapy appear to improve survival. Our case supports the potential role of LT in selected patients with early-stage tumors (e.g., pT1-pT2), even in rare or aggressive histologies. Although LT indications remain strict, selected cases of mucinous bile duct tumors with favorable profiles may benefit from transplantation. Multidisciplinary evaluation and individualized treatment strategies are essential in the era of transplant oncology.

It further confirmed its pivotal role in CCA progression. Targeting the USP10-PRMT1-DDX1 axis may represent a significant therapeutic approach for CCA.

P2, N=88, Active, not recruiting, National Cancer Institute (NCI) | Trial completion date: Mar 2026 --> Jun 2026 | Trial primary completion date: Mar 2026 --> Jun 2026

P2, N=62, Recruiting, Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University | Trial completion date: Oct 2025 --> Dec 2027 | Trial primary completion date: Jan 2024 --> Jan 2027