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Cholangiocarcinoma and Gallbladder Cancer Studies / Cancer Cells and Metastasis · Journal article
Cancer Research Communications · August 10, 2026
Early or partial results. Treat as a signal, not a conclusion.
This multicenter feasibility study demonstrates that patient-derived tumor organoids can be successfully generated from biliary tract cancer specimens and screened against multiple targeted agents. While 92.3% of organoids showed sensitivity to at least one drug, clinical validation is limited to five cases with one detailed responder, making this a proof-of-concept study requiring prospective controlled validation.
Multicenter organoid feasibility study with prospective drug sensitivity testing. Biliary tract cancer patients; 43 patients enrolled, 26 with successful organoid derivation; majority from late-stage disease. Intervention: Patient-derived tumor organoid (PDTO) drug sensitivity testing using PARIS® assay with average 50 cancer-directed therapies. n = 26. Multicenter (specific sites not stated).
26 tumor organoids successfully derived from 43 BTC patients (60.5% derivation rate) 24/26 organoids (92.3%) exhibited strong sensitivity to one or more targeted agents Average of 50 cancer-directed therapies tested per organoid using CLIA-certified PARIS® assay
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This work suggests organoid drug testing may expand therapeutic options beyond genomic biomarkers in BTC, but the evidence is preliminary. Prospective randomized controlled trials comparing organoid-guided therapy to standard care are needed before clinical implementation.
Single-centre feasibility study of organoid drug testing in a small sample with uncontrolled clinical outcomes; demonstrates proof-of-concept but lacks comparison group and prospective validation.
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This work suggests organoid drug testing may expand therapeutic options beyond genomic biomarkers in BTC, but the evidence is preliminary. Prospective randomized controlled trials comparing organoid-guided therapy to standard care are needed before clinical implementation.
Graded across the dimensions that decide whether you should act, each from what the source actually supports. There is no single score, and where a dimension was not assessed it says so.
Abstract Biliary tract cancers (BTCs) pose clinical challenges due to poor chemotherapy response and aggressive disease course. We evaluated patient derived tumor organoid (PDTO) based drug sensitivity testing as a tool to guide therapy. In this multicenter study, 26 tumor organoids were successfully derived from 43 BTC patients and tested with an average of 50 cancer-directed therapies using the CLIA certified PARIS® assay. Despite most organoids being from late-stage disease, 24/26 (92.3%) exhibited strong sensitivity to one or more targeted agents. Active drugs included inhibitors of EGFR/HER2, MEK, ERK, BCR-ABL and SRC family, mTOR, PI3K, MDM2, BCL2, and BET. Drug sensitivities aligned with known genetic biomarkers, but were also observed in cultures lacking them, indicating ex vivo testing can expand actionability beyond genomics. In five cases, results guided therapy; one patient with an FGFR-BICC1 fusion refractory to FGFR inhibitors responded to dasatinib, achieving symptomatic improvement, stable disease, and 8 month survival.
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