In the United States, Breakthrough Therapy designation is granted by the U.S. Food and Drug Administration (FDA) to expedite drug development and review. This designation is granted to therapies that are intended to treat serious conditions and which may show promise or benefits over existing therapies. As an incentive, Breakthrough Therapy designation also comes with benefits for the drug developers: namely Fast Track designation features, intensive FDA guidance on drug development, and additional organizational commitment. According to a December 2023 report on Drugs.com, the FDA granted Breakthrough Therapy designation to TAR-200, a potential treatment for individuals living with BCG-unresponsive high-risk non-muscle-invasive bladder cancer who either have not undergone surgical bladder removal or who cannot undergo surgical bladder removal.

So what is TAR-200, you might ask? TAR-200, developed by Johnson & Johnson, is a novel and investigational therapeutic releasing system. It offers localized gemcitabine release straight to the bladder over a 21-day dosing cycle, allowing for more targeted treatment. Additionally, this overcomes some of the more invasive nature of available therapies. So far, this investigational treatment has shown promise in clinical studies such as the Phase 2b SunRISe-1 trial, which is exploring how safe and effective this treatment is. Within the trial, researchers are comparing three potential treatment pathways:

• TAR-200 as a monotherapy
• Cetrelimab as a monotherapy
• TAR-200 in conjunction with cetrelimab

Additional studies are being performed on TAR-200. The treatment has also been shown to be effective in treating muscle-invasive bladder cancer, reports The Journal of Urology.

What is Bladder Cancer?

The Cleveland Clinic explains that bladder cancer is a relatively rare form of cancer that forms in the urothelium, or the bladder lining. While bladder cancer is treatable, approximately 75% of early-stage bladder cancers recur after treatment. Tobacco use, industrial product exposure, pelvic radiation therapy, prior chemotherapy use, Aristolochia fangchi use, a history of bladder infections, and older age all increase the risk of developing bladder cancer.

Bladder cancer may be characterized as noninvasive (meaning the tumors are only in a small section of tissue or near the surface), non-muscle-invasive (meaning that the cancer has spread but not to muscle yet), and muscle-invasive (meaning that the cancer has spread into the bladder wall muscle and possibly beyond the bladder). According to the Mayo Clinic, 75-80% of newly diagnosed bladder cancer is considered non-muscle-invasive. High-risk non-muscle-invasive bladder cancer has a heightened potential for recurrence and may become muscle-invasive.

While BCG induction therapy can treat high-risk non-muscle-invasive bladder cancer, the cancer recurs in about 50% of people – and some develop treatment-averse cancer. It is crucial to continue researching novel therapeutics to reach this patient population.

Symptoms of non-muscle-invasive bladder can may include:

• Hematuria (blood in your urine that might be visible)
• Dysuria (painful urination)
• Back and pelvic pain
• Frequent or urgent urination
• Difficulty urinating