The aim of this study was to establish new biliary tract
carcinoma (BTC) cell lines and identify predictive
biomarkers for the potential effectiveness of
gemcitabine therapy. Surgical specimens of BTC were transplanted directly into immunodeficient mice to establish xenografts, then subjected to in vitro cell culture. The
gemcitabine sensitivity of each cell line was determined and compared with the genome-wide gene expression profile. A new predictive
biomarker candidate was validated using an additional cohort of
gemcitabine-treated BTC cases. From 55 BTC cases, we established 19 xenografts and six new cell lines. Based on their
gemcitabine sensitivity, 10 BTC cell lines (including six new and four publicly available ones) were clearly categorized into two groups, and MAGEH1
mRNA expression in the
tumor cells showed a significant negative correlation with their sensitivity to
gemcitabine. Immunohistochemically, MAGEH1
protein was detected in three (50%) out of six sensitive cell lines, and four (100%) out of four resistant cell lines. In the validation cohort of
gemcitabine-treated recurrence cases, patients were categorized into "effective" and "non-effective" groups according to the RECIST guidelines for assessment of chemotherapeutic effects. MAGEH1
protein expression was detected in two (40%) out of five "effective" cases and all four (100%) "non-effective" cases. We have established a new BTC bioresource that covers a wide range of
biological features, including
drug sensitivity, and is linked with clinical information. Negative expression of MAGEH1
protein serves as a potential predictive marker for the effectiveness of
gemcitabine therapy in BTC.