Cholangiocarcinoma

This integrative framework combining transcriptomics, CRISPR dependency mapping, machine learning, and pharmacogenomic annotation identifies clinically relevant HB-essential genes and predictive molecular signatures for tumor identity. The derived expression-based scores provide tools for patient stratification, while drug-gene mapping highlights actionable vulnerabilities on HDAC11 with pediatric approved drugs that support rational drug repurposing strategies in hepatoblastoma.

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

Given the refractory nature of his disease course, he was subsequently treated with a repeat dose of IVIG and prednisone, then transferred to an outside facility for plasma exchange. This case highlights the rare but potentially fatal concurrent occurrence of immune-related myasthenia gravis and myocarditis as irAEs in a patient receiving durvalumab for cholangiocarcinoma. While checkpoint inhibitors have revolutionized outcomes across many solid tumor malignancies, this case underscores the diagnostic and management challenges posed by severe, refractory irAEs, and the importance of early recognition and aggressive treatment in this patient population.

Zenocutuzumab demonstrated clinically meaningful and durable antitumor activity with a favorable safety profile in patients with advanced NRG1+ cholangiocarcinoma.

As Ex1C exhibited homotypic self-association, these effects are consistent with interference with ECL1-dependent claudin-2 interactions. Collectively, our findings identify claudin-2 as a characteristic molecular feature of the examined cHCC-CCA cell lines and support the utility of extracellular loop-mimetic peptides as probes to dissect claudin-dependent regulation of proliferation and cell adhesion.

P=N/A, N=100, Recruiting, Minia University | Trial completion date: Jul 2026 --> Oct 2026 | Trial primary completion date: Jun 2026 --> Sep 2026

This case highlights that spontaneous tumor regression does not exclude malignancy. Careful long-term surveillance and timely surgical intervention are crucial when imaging findings and tumor marker trends raise suspicion of malignancy despite temporary regression.

The combinations of atezolizumab and varlilumab with/without cobimetinib were safe but neither meaningfully improved outcomes in BTC treated in the later lines. Correlative tissue studies validated preclinical work that MEK inhibition increases CD8+TILs.

Our data indicates that arsenite and DCM are relevant environmental chemicals which induced cellular changes that are consistent with cholangiocarcinogenesis. Our in vitro approach, like others, may also be useful as a valuable compliment to in vivo carcinogenesis investigations.

Mechanistically, RNF216 promoted p53 degradation through ubiquitination, leading to upregulated SLC7A11 expression and ultimately inhibiting ferroptosis.125I seed inhibited SLC7A11 expression by preventing RNF216-mediated ubiquitination of p53, ultimately inducing ferroptosis in CCA. This provides crucial molecular evidence for the anticancer effects of 125I seed.

P2, N=22, Not yet recruiting, University of Washington

Chemoimmunotherapy is the reference first-line regimen for biomarker-unselected advanced cholangiocarcinoma. Early comprehensive genomic profiling is essential to enable timely, matched targeted therapy and maximize population-level benefit.