UC San Diego Health offers new bladder-saving gene therapy to treat localized bladder cancer

University of California - San DiegoJul 8 2024

UC San Diego Health is the first health system in San Diego County to offer a new bladder-saving gene therapy to treat localized bladder cancer. 

The novel treatment is the first and only FDA-approved gene therapy delivered directly into the bladder for non-muscle-invasive bladder cancer (NMIBC). Called nadofaragene firadenovec (Adstiladrin), the gene therapy addresses an unmet need for patients who are no longer responding to the longstanding first line of defense -; bacillus calmette-guerin (BCG), a bacteria-based immunotherapy for cancer management. While BCG is a common first therapy, it can eventually stop working, ultimately leading to complete bladder removal.

The American Cancer Society estimates that 83,000 new cases of bladder cancer will be diagnosed this year, and approximately 17,000 will die of the disease. NMIBC is the most common type of bladder cancer, accounting for nearly 75% of cases. It is a type of localized bladder cancer that has not metastasized or invaded into the muscle wall of the bladder.

"The primary treatment for NMIBC in the past typically involved surgical resection, followed by therapies into the bladder, like immunotherapy or chemotherapy. Unfortunately, if these interventions proved ineffective, the standard course of action involved bladder removal, which poses a substantial morbidity risk," said Amirali Salmasi, MD, associate professor of urology at University of California San Diego School of Medicine, urologist at UC San Diego Health and UC San Diego Moores Cancer Center member. "This innovative gene therapy approach holds promise in delaying or even eliminating the necessity for bladder removal."

UC San Diego Health urologists have initiated the bladder gene treatments on five patients to date. The gene therapy dosing is delivered once every three months into the bladder through a urinary catheter and is a treatment option for any patient who is no longer responding to BCG therapy.

Here's how it works: The bladder therapy is a recombinant adenovirus gene therapy, which uses a form of a virus to deliver genetic material into a cell. When instilled into the bladder, it infiltrates the bladder cells and delivers a gene encoding interferon -; a natural substance that helps the body's immune system fight disease -; which is incorporated into the cells' DNA. The bladder cells then express the interferon, which blocks bladder cancer growth. 

This exciting gene therapy represents a significant milestone for bladder cancer treatment. There have been limited advancements for patients who are unresponsive to BCG treatment -; until now."

Aditya Bagrodia, MD, associate professor of urology at UC San Diego School of Medicine, urologic oncologist at UC San Diego Health and UC San Diego Moores Cancer Center member

UC San Diego Health offers comprehensive bladder cancer care and is repeatedly ranked among the nation's best in both cancer care and urology by U.S. News & World Report.

"As the region's only NCI-designated Comprehensive Cancer Center, UC San Diego Health is a champion of implementing innovative new therapies system-wide," said Diane Simeone, MD, director of Moores Cancer Center at UC San Diego Health.

Moores Cancer Center at UC San Diego Health is the only National Cancer Institute-designated Comprehensive Cancer Center in San Diego County, the highest possible rating for a U.S. cancer center. NCI recognizes centers around the country that meet rigorous standards for transdisciplinary, state-of-the-art research focused on developing new and better approaches to preventing, diagnosing, and treating cancer.