Molecular targeted therapy for biliary tract malignancy: defining the target

Malignancies of the biliary tract are rare, diagnosed in approximately 9,000 patients each year in the United States (1). Unfortunately, most patients present with advanced disease. Of the small subset who present with resectable (and thus potentially curable) disease, many experience recurrence (2), reinforcing the fact that a multidisciplinary approach to these cancers is critical. Traditionally, our success with medical therapy for patients with locally advanced or metastatic disease has been poor.