Bladder cancer treatment survival improves with drug combination

A bladder cancer treatment survival study found that enfortumab vedotin plus pembrolizumab helped patients with muscle-invasive disease live longer without progression, recurrence or death than standard chemotherapy, according to results published in The New England Journal of Medicine. The finding matters because cisplatin-based chemotherapy has been the main presurgery treatment before bladder removal for more than two decades, according to Mount Sinai Hospital.

The international phase 3 trial compared the drug combination with standard cisplatin-based chemotherapy in patients who were candidates for surgery. Mount Sinai said the study was led by Matthew D. Galsky, a bladder cancer specialist at the Icahn School of Medicine at Mount Sinai and deputy director of the Mount Sinai Tisch Cancer Center.

What is the new bladder cancer treatment?

The treatment pairs enfortumab vedotin, an antibody-drug conjugate that targets Nectin-4 on bladder cancer cells, with pembrolizumab, a PD-1 inhibitor immunotherapy. Mount Sinai said pembrolizumab is designed to help the immune system identify and attack cancer cells.

Patients in the newer-treatment group received the two drugs before and after surgery. Patients in the comparison group received cisplatin-based chemotherapy before surgery, according to the trial report described by Mount Sinai.

What did the bladder cancer trial find?

The randomized trial enrolled 808 patients at 158 sites worldwide, Mount Sinai said. Patients assigned to enfortumab vedotin plus pembrolizumab had a 47% lower risk of cancer progression, recurrence or death than those assigned to standard chemotherapy.

Two years after treatment started, nearly 80% of patients in the combination-treatment group were alive without disease progression, recurrence or death, compared with 66% in the chemotherapy group, according to the published findings. The study also found that more than half of patients who received the newer regimen had no detectable cancer in tissue removed during surgery, compared with about one-third of patients who received standard chemotherapy.

Galsky said patients with muscle-invasive bladder cancer have had few changes in presurgery treatment for many years. He said the trial showed better results with the combination than with cisplatin-based chemotherapy and could change care for patients globally, according to Mount Sinai.

Why does muscle-invasive bladder cancer carry higher risk?

Muscle-invasive bladder cancer is an aggressive form of the disease in which cancer has grown into the muscle layer of the bladder. Mount Sinai said it carries a high risk of coming back and spreading to other parts of the body.

Bladder cancer is the sixth most common cancer in the United States, according to Mount Sinai, citing the American Cancer Society. The American Cancer Society estimates that nearly 85,000 Americans will be diagnosed with bladder cancer this year, and Mount Sinai said about one in four bladder cancers are muscle-invasive.

FDA approval followed the trial results

Mount Sinai said the Food and Drug Administration approved pembrolizumab in combination with enfortumab vedotin on July 10, 2026, as a perioperative treatment for muscle-invasive bladder cancer. Perioperative treatment means therapy given around the time of surgery, which can include treatment before surgery, after surgery or both.

The FDA action expanded the label from cisplatin-ineligible patients to all patients with muscle-invasive bladder cancer who are candidates for cystectomy, according to Mount Sinai. A cystectomy is surgery to remove the bladder.

The researchers also reported that the combination had higher rates of side effects than standard chemotherapy. Mount Sinai said the finding points to the need for close monitoring and side-effect management by experienced cancer care teams.

This story draws on original reporting from Medical Xpress.