Background & Aims Intrahepatic cholangiocarcinoma (ICC) is a lethal malignancy without
effective treatment options. MLN0128, a second generation pan-mTOR inhibitor, shows
efficacy for multiple tumor types. We evaluated the therapeutic potential of MLN0128 vs.
gemcitabine/oxaliplatin in a novel ICC mouse model. Methods We established a novel ICC
mouse model via hydrodynamic transfection of activated forms of AKT (myr-AKT) and Yap
(YapS127A) protooncogenes (that will be referred to as AKT/YapS127A). Genetic …

Methods: In the present study, we evaluated the therapeutic potential of MLN0128 and
compared it with gemcitabine/oxaliplatin (the standard therapeutic option for this tumor
entity) in a novel ICC mouse model ad hoc generated. Specifically, we established a novel
ICC model via hydrodynamic transfection of activated forms of AKT (myr-AKT) and Yap
(YapS127A) protooncogenes (that will be referred to as AKT/YapS127A) in the mouse liver.
Results: We found that co-expression of myr-AKT and YapS127A promoted rapid ICC …