Transformative advances in bladder cancer treatment

March 06, 2025

Bladder cancer treatment has undergone remarkable advancements over the past decade, driven by innovative clinical trials and new therapeutic options. These developments are not only enhancing patient outcomes but also offering bladder-sparing alternatives.

"Just 10 years ago, people with localized bladder cancer that didn't respond to treatment often required bladder removal," says Mark D. Tyson II, M.D., M.P.H., a urologic surgeon at Mayo Clinic Comprehensive Cancer Center in Phoenix, Arizona. "Now, we have a whole host of new drugs and clinical trials that allow us to offer more options.

Revolutionizing treatment with drug combinations

One of the most significant advancements in bladder cancer treatment is the Food and Drug Administration's 2023 approval of a combination therapy involving enfortumab vedotin and pembrolizumab (EV/pembro) for locally advanced or metastatic urothelial bladder cancer. This combination, previously reserved for patients ineligible for cisplatin-based chemotherapy, has now become the first line treatment.

"Clinical trial results showed that EV/pembro was better than cisplatin-based combination chemotherapy in the first line setting," says Dr. Tyson. The combination therapy achieved a 55% reduction in disease progression or death compared with cisplatin-based chemotherapy, establishing it as the new standard of care for metastatic urothelial bladder cancer. The study results were published in the New England Journal of Medicine in 2021.

Advancements in surgical techniques with single-port cystectomy

For patients with muscle-invasive bladder cancer or nonmuscle-invasive bladder cancer unresponsive to other treatments, cystectomy remains a critical option. Mayo Clinic is pioneering the refinement of single-incision robotic surgery for bladder removal and reconstruction. This technique provides reduced postoperative pain, fewer complications and shorter hospital stays. While further research is necessary to fully validate these benefits, single-port cystectomy is a promising advancement for patients with bladder cancer.

Clinical trials pioneering new options in bladder cancer treatment

Clinical trials are at the forefront of advancing bladder cancer treatment, offering new hope and improved outcomes for patients. These trials are essential for developing innovative therapies and less invasive methods that enhance patient care and quality of life.

Immunotherapy for treatment-resistant nonmuscle-invasive bladder cancer

Clinical trials have been instrumental in advancing immunotherapy for nonmuscle-invasive bladder cancer, particularly for patients whose priority is bladder preservation. Bacillus Calmette-Guerin (BCG) has long been a standard treatment, but new immunotherapeutic options are emerging through ongoing research.

Dr. Tyson highlights a phase 3 clinical trial testing cretostimogene grenadenorepvec for BCG-unresponsive, high-grade nonmuscle-invasive bladder cancer. "We presented data at the annual meeting of the American Urological Association in 2024 showing promising results," says Dr. Tyson. The trial involves an initial six-week treatment course followed by maintenance doses. Preliminary results indicate a 75.2% complete response rate, with 83% of patients maintaining their response beyond one year. Dr. Tyson presented these results at the 25th Annual Meeting of the Society of Urologic Oncology in December 2024.

Urine test for nonmuscle-invasive bladder cancer recurrence monitoring

Surveillance is crucial for managing bladder cancer due to its high recurrence rate. Traditional cystoscopy, while effective, can be uncomfortable for patients. Clinical trials are exploring less invasive alternatives to improve patient experience and compliance.

Mayo Clinic is conducting a study on a urinary biomarker test as a noninvasive method for monitoring nonmuscle-invasive bladder cancer recurrence. "One of the exciting studies we're doing at Mayo Clinic uses a urinary biomarker from a urine sample to identify recurrence without the need for cystoscopy," explains Dr. Tyson. This trial aims to evaluate patient preferences, comfort and confidence in the urine test compared with traditional cystoscopy.

The landscape of bladder cancer treatment has evolved significantly, offering patients more options and better outcomes. Clinical trials continue to play a pivotal role in these advancements, providing new therapies and improving existing ones. As research progresses, the goal remains to enhance patient care and quality of life through innovative and effective treatment strategies.

For more information

Powles T, et al. Enfortumab vedotin in previously treated advanced urothelial carcinoma. New England Journal of Medicine. 2021;384:1125.

Tyson M, et al. BOND-003- Cohort P: A multi-national, single-arm study of intravesical cretostimogene grenadenorepvec for the treatment of high-risk, papillary only, BCG-unresponsive non-muscle invasive bladder cancer. Presentation at: Society of Urologic Oncology 25th Annual Meeting; 2024; Dallas, Texas.

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