Cholangiocarcinoma with isocitrate dehydrogenase-1 (IDH1) mutation

Cholangiocarcinoma with an isocitrate dehydrogenase-1 (IDH1) mutation is a distinct molecular subtype of bile duct cancer, most frequently arising within the intrahepatic biliary tree

Cholangiocarcinoma with an isocitrate dehydrogenase-1 (IDH1) mutation is a distinct molecular subtype of bile duct cancer, most frequently arising within the intrahepatic biliary tree Pathophysiologically, somatic missense mutations in the *IDH1* gene confer neomorphic enzymatic activity that catalyzes the aberrant reduction of alpha-ketoglutarate into the oncometabolite D-2-hydroxyglutarate (2-HG) Intracellular accumulation of 2-HG inhibits alpha-ketoglutarate-dependent dioxygenases, resulting ...

This molecular biomarker defines a targeted subset of patients—predominantly adults with unresectable, locally advanced, or metastatic disease—whose tumors are susceptible to targeted small-molecule IDH1 inhibitors