Is There a Role for Checkpoint Inhibitors as Neoadjuvant Therapy in Bladder Cancer?

There has long been a disconnect between the data supporting the role of cisplatin-based neoadjuvant chemotherapy prior to radical cystectomy and its actual broad implementation. Among the challenges include a very modest impact on disease outcome, its well-known toxicity, and the inability of a substantial number of patients who are medically ineligible to receive cisplatin. Meanwhile, five different checkpoint inhibitors have been FDA-approved for use in metastatic urothelial cancer.

Now, investigators have conducted an industry-supported, multicenter, single-arm, open-label, window-of-opportunity, phase II trial (PURE-01) of the anti-programmed cell death (PD)-1 inhibitor pembrolizumab in 50 patients with muscle-invasive bladder cancer (M…