Cholangiocarcinoma

After knocking off HIF-1α, both the number of migrating and invading cells decreased (both P<0.05). miR-199a-5p is downregulated in ICC and suppresses tumor migration and invasion by directly targeting HIF-1α and regulating downstream molecules involved in proliferation, invasion and apoptosis.

P1/2, N=52, Recruiting, Bold Therapeutics Inc.

The incidence of FGFRi-associated nail toxicities varies by agent and can affect quality of life and treatment adherence. The pathogenesis remains unclear, and no predictive biomarkers exist. Further research into optimized management and preventative strategies is needed. Early recognition and proactive multidisciplinary management are essential to minimizing complications and maintaining oncologic treatment continuity. &nbsp.

In this LT cohort, CCA molecular profiling informed prognosis and treatment response. Heterogeneity may depend on early tumorigenesis disruptions, potentially explaining outcome differences.

Parallel translational work using cfDNA-based liquid biopsy has mapped a spectrum of secondary kinase-domain mutations that underlie acquired resistance, informing the development of next-generation FGFR2-selective inhibitors (eg, lirafugratinib) and combination strategies with EGFR/ERBB blockade. Collectively, these data underscore the need for comprehensive molecular profiling and innovative umbrella trial designs to optimise targeted therapy in this rare, biologically heterogeneous malignancy.

P1, N=66, Terminated, Imugene Limited | N=100 --> 66 | Trial completion date: Nov 2026 --> Jan 2026 | Active, not recruiting --> Terminated | Trial primary completion date: Jun 2026 --> Jan 2026; Portfolio prioritization

We characterize distinct molecular signatures of AKT and YAP in iCCA progression and identify YAP as a key regulator of the tumor immune microenvironment (TIME). Our findings support combining YAP inhibition with immune checkpoint inhibitors (ICIs) for iCCA treatment.

P=N/A, N=300, Not yet recruiting, Aarhus University Hospital

Furthermore, PSMD14 and ENO1 were highly expressed in tumor tissues and closely associated with a poor ICC prognosis. Overall, our study reveals the importance of the L-lactate/PSMD14/ENO1 axis in regulating ferroptosis resistance in ICC, suggesting a novel therapeutic target and strategy for treating this disease.

P2, N=54, Recruiting, Yale University | Not yet recruiting --> Recruiting

P3, N=757, Active, not recruiting, AstraZeneca | Recruiting --> Active, not recruiting

P1/2, N=52, Recruiting, Bold Therapeutics Inc. | N=38 --> 52