New treatments for bladder cancer: when will we make progress?

OPINION STATEMENT: The prognosis and long-term survival for patients with metastatic urothelial carcinoma patients is poor. Traditionally, the mainstay of treatment has been combination chemotherapy with gemcitabine and cisplatin or with the classical M-VAC. Vinflunine has become an EMA-approved second-line option in Europe. Urothelial carcinomas contain genetic alteration in multiple genes that are potentially treated by targeted therapies. Recently, a number of these new therapies have been developed in this disease. But not one has been approved for clinical use in urothelial cancers. While clinical evaluation of these agents is still in its early days, some promising findings have begun to emerge. For example, everolimus demonstrated activity in those metastatic urothelial cancer patients who harbors TSC1 mutation. With the identification of the most relevant drug targets for tumors initiation and maintenance and the best way to assess drug candidates that may only account for a small fraction of patients, it is anticipated that the therapeutic arsenal for urothelial cancers will be expanded in the future.