Despite advances in the treatment of other genitourinary malignancies, no novel therapies have been approved by the US Food and Drug Administration for urothelial carcinoma (UC) in the last 20 years. To date, no clinical trials of targeted agents in UC have led to improvements in survival compared with cytotoxic therapy. This article outlines representative trials of targeted therapies in UC and discusses the significance of genetic preselection in trial design as a method to optimize responses to these agents, thus, hopefully expanding the armamentarium of treatment options against this lethal disease.
Keywords: Angiogenesis; MAPK pathway; Next-generation sequencing; PI3K/Akt/mTOR pathway; Small molecule inhibitors; Targeted therapy; Urothelial carcinoma.
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