Cholangiocarcinoma

P1/2, N=180, Recruiting, Alentis Therapeutics AG

Notably, Hel potentiated gemcitabine efficacy, resulting in pronounced tumor inhibition and improved survival. Collectively, these findings establish the USP5-HSPB1 axis as a ferroptosis checkpoint in ICC and suggest pharmacologic USP5 inhibition as a strategy to improve chemotherapy response.

Multi-omic profiling of BTC within the MASTER program uncovered novel therapeutic opportunities. By the identification of ultra-rare and composite biomarkers, MASTER-guided therapies resulted in clinical benefit also in heavily pretreated patients.

In this issue of Cancer Cell, Entrialgo-Cadierno et al. raise the bar for the ∼20%-25% of patients with oncogenic KRAS mutations by unlocking the therapeutic potential of direct RAS-GTP inhibition.

Polymorphism co-occurrence patterns indicate a relatively low mutational burden, with epigenetic dysregulation and oncogenic signaling representing central mechanisms in iCCA development. Further large-scale studies integrating tissue and circulating DNA analyses are warranted to validate these findings and identify clinically actionable biomarkers in iCCA.

This platform allows quantitative, side-by-side comparison of genetic, pharmacological, and microenvironmental perturbations on bile duct morphogenesis. By combining spatial control, a tunable microenvironment, and high-resolution morphometric analysis, it provides a versatile tool for studying developmental defects, dissecting pathway-specific contributions, and exploring regenerative strategies for biliary disorders.

CCNJL has the potential to act as a valuable prognostic indicator and immunotherapy target in CHOL. Its expression pattern and associations with clinical outcomes, immune characteristics, and drug sensitivity highlight its potential for improving diagnostic and therapeutic approaches. Future research should focus on elucidating the underlying mechanisms and validating these findings in larger cohorts.

We established a novel and clinically applicable pyroptosis-based classifier for iCCA prognosis. Our findings hint at a possible connection of the TGF-β-GSDMB-CDH3 axis with enhanced tumor aggressiveness, providing new insights for patient risk stratification and revealing potential therapeutic targets for iCCA.

P=N/A, N=2000, Active, not recruiting, Fondazione Policlinico Universitario Agostino Gemelli IRCCS

P2, N=50, Recruiting, Cancer Institute and Hospital, Chinese Academy of Medical Sciences | Initiation date: Jul 2025 --> Dec 2025 | Not yet recruiting --> Recruiting

P2/3, N=168, Active, not recruiting, Compass Therapeutics | Completed --> Active, not recruiting | Trial completion date: Jan 2026 --> Dec 2026 | Trial primary completion date: Jan 2026 --> Dec 2026

In this study, we establish a quantitative extravasation imaging platform for evaluating CAR-NK cell trafficking in pancreatic and cholangiocarcinoma tumor models. This approach provides a structured framework for assessing immune cell delivery and therapeutic distribution within the tumor microenvironment.