Gallbladder Cancer: Current and Emerging Therapies

Despite its rare prevalence, gallbladder cancer (GBC) is associated with a poor prognosis. Patients typically experience vague symptoms and present with advanced stages of disease. Gallstones are the most prevalent risk factors. Tumors that are associated with gallstones have a different pathogenesis compared to those originating from anomalous pancreaticobiliary duct junction or on a background of adenomatous polyps. Approximately 10–30% of patients will qualify for surgical resection, which is the only potentially curable treatment. Patients with resected, node- or margin-positive disease are candidates for adjuvant therapy. Options include fluoropyrimidine-based chemoradiation with or without chemotherapy, as well as fluoropyrimidine- or gemcitabine-based chemotherapy alone. For patients with advanced disease, prognosis is poor despite treatment, which includes fluoropyrimidine- or gemcitabine-based chemotherapy, with or without fluoropyrimidine chemoradiation. Targeted biologic therapy, such as inhibitors of VEGF, EGFR, and HER2, as well as pathways with aberrant expression, have been evaluated in the setting of advanced disease. Unfortunately, the trials are underpowered. Further studies are in dire need.