Yale Cancer Center Study Shows Targeted Therapy Improves Survival for Advanced Bladder Cancer

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Enfortumab vedotin significantly prolonged survival as compared with standard chemotherapy in patients with locally advanced or metastatic urothelial carcinoma or bladder cancer, who had previously received platinum-based treatment and a PD-1–PD-L1 inhibitor.

In a new study led by researchers at Yale Cancer Center (YCC), the drug enfortumab vedotin (EV) significantly prolonged survival as compared with standard chemotherapy in patients with locally advanced or metastatic urothelial carcinoma or bladder cancer, who had previously received platinum-based treatment and a PD-1–PD-L1 inhibitor.


The results were published today in The New England Journal of Medicine (NEJM).

“These findings are very encouraging as they continue to show EV is an effective tool in the fight to improve survival rates for patients with advanced bladder cancer,” said Daniel P. Petrylak, MD, Professor of Medicine (Medical Oncology) and Urology and Co-Director of the Cancer Signaling Research Program at YCC and senior author of the published study. “We continually strive to identify new treatment options for this deadly disease and bring hope and encouraging options to our patients.”

EV is described by Petrylak as a “smart bomb,” or an antibody-drug conjugate that targets a specific protein on the surface of tumor cells with a load of powerful chemotherapy. In 2019, the U.S. Food and Drug Administration (FDA) granted accelerated approval to EV to treat adult patients with advanced urothelial or bladder cancer. The approval is the direct result of a multi-institutional clinical trial led by YCC and Smilow Cancer Hospital researchers.

For this study, physicians conducted a global, open-label, phase 3 trial of EV for the treatment of patients with locally advanced or metastatic urothelial carcinoma who had previously received platinum-containing chemotherapy and had had disease progression during or after treatment with a PD-1–PD-L1 inhibitor. Patients were randomly assigned in a 1:1 ratio to receive EV or investigator-chosen chemotherapy administered on day 1 of a 21-day cycle. A total of 608 patients underwent randomization; 301 were assigned to receive EV and 307 to receive chemotherapy. Overall survival was longer in the EV group than in the chemotherapy group. (median overall survival, 12.88 vs. 8.97 months; hazard ratio for death, 0.70; 95% confidence interval) Progression-free survival was also longer in the EV group than in the chemotherapy group.

The study was funded by Astellas Pharma US, Inc. and the biotechnology company Seagen.

About Yale Cancer Center and Smilow Cancer Hospital

Yale Cancer Center (YCC) is one of only 51 National Cancer Institute-designated comprehensive cancer centers in the nation and the only such center in Connecticut. Cancer treatment for patients is available at Smilow Cancer Hospital through 13 multidisciplinary teams and at 15 Smilow Cancer Hospital Care Centers in Connecticut and Rhode Island. Comprehensive cancer centers play a vital role in the advancement of the NCI’s goal of reducing morbidity and mortality from cancer through scientific research, cancer prevention, and innovative cancer treatment.