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Advances in Bladder Cancer Research

Microscopic view of a papillary urothelial (transitional cell) carcinoma.

Credit: iStock

NCI-funded researchers are working to improve our understanding of how to treat bladder cancer. With recent advances in immunotherapy and targeted therapy, treatment has the potential to become more effective and less toxic.

This page highlights some of the latest research in bladder cancer, including clinical advances that may soon translate into improved care and research findings from recent studies.

Bladder Cancer Treatment

Bladder cancer treatments are based on the type of bladder cancer and the stage of the disease. The most common type of bladder cancer is transitional cell carcinoma, also called urothelial carcinoma, which begins in cells in the innermost tissue layer of the bladder. There are other types of bladder cancers such as squamous cell carcinoma, small cell carcinoma, and adenocarcinoma among others.

The mainstays of bladder cancer treatment are surgery, radiation therapy, chemotherapy, and immunotherapy, depending on the stage. Scientists continue to study novel treatments and drugs, along with new combinations of existing treatments.

Nonmuscle-invasive bladder cancer—bladder cancer that has grown through the lining of the bladder but hasn’t yet invaded the muscle layer of the bladder—is usually removed by scraping the tumor from the bladder wall. After surgery, some patients may receive additional treatment with an immune-based therapy called bacillus Calmette-Guérin (BCG), or with chemotherapy drugs such as mitomycin C (Jelmyto) or gemcitabine put directly into their bladder to reduce the risk that the cancer will recur. 


Immunotherapy is treatment that helps the body’s immune system fight cancer more effectively. In recent years, several different immunotherapy drugs, all of a type called immune checkpoint inhibitors, have been approved for treating certain patients with locally advanced or metastatic bladder cancer. These drugs work by “releasing the brakes” on the immune system and allowing immune cells to detect and kill tumor cells.

Patients whose bladder cancers respond to immune checkpoint inhibitors tend to maintain those responses for long periods. Ongoing clinical trials will help researchers learn whether these extended responses help patients live longer. 

However, only a small number of patients respond to immune checkpoint inhibitors. Scientists are trying to develop biomarkers that could help doctors identify which patients with bladder cancer are likely to respond to these drugs. The PD-L1 protein, for example, has been studied as a biomarker for response to treatment with immune checkpoint inhibitors.

Scientists have now begun to test immune checkpoint inhibitors in earlier stages of bladder cancer and in combination with other treatments, such as chemotherapy. 

The NCI-sponsored AMBASSADOR trial is comparing the immune checkpoint inhibitor pembrolizumab (Keytruda) with observation (the standard of care) in patients with bladder cancer that invades the muscle layer of the bladder wall (localized muscle-invasive disease) or has spread to nearby lymph nodes (locally advanced disease) and has been surgically removed. The trial will see if pembrolizumab improves overall survival or disease-free survival.

In July 2020, the FDA approved the immune checkpoint inhibitor avelumab (Bavencio) for people with advanced bladder cancer that has shrunk or stopped growing after chemotherapy using a platinum-based drug. The approval is for the use of avelumab as maintenance therapy for advanced disease that has not spread (locally advanced) or disease that has spread beyond the bladder (metastatic).

Targeted Therapy 

Targeted therapies use drugs to identify and attack specific types of cancer cells with less harm to normal cells. In 2019, erdafitinib (Balversa) became the first targeted therapy to be approved by FDA to treat patients with locally advanced or metastatic urothelial carcinoma. This drug can be used to treat some patients whose cancers have certain alterations in the FGFR2 gene or FGFR3 gene. Only about 20% of bladder cancers harbor an FGFR gene alteration.

An ongoing phase 3 study is comparing erdafitinib with standard chemotherapy and with pembrolizumab in patients with advanced bladder cancer whose tumors have an FGFR gene alteration. This study could help researchers learn whether patients with FGFR-altered bladder cancer benefit more from erdafitinib or an immune checkpoint inhibitor versus chemotherapy. 

The drug enfortumab vedotin-ejfv (Padcev) has also been approved to treat advanced/metastatic bladder cancer. It showed positive results in patients who had previously been treated with chemotherapy and a subsequent immune checkpoint inhibitor. Researchers are continuing to study this drug to see whether it can be used to treat bladder cancer earlier in the disease process and to evaluate it in combination with immunotherapy and/or chemotherapy.

Combination Therapy

Researchers are testing many combinations of therapies for bladder cancer, either by combining several immunotherapy drugs or by combining an immunotherapy drug with another type of treatment.

Clinical Trials for Bladder Cancer

NCI funds and oversees both early- and late-phase clinical trials to develop new treatments and improve patient care. Trials are available for bladder cancer treatment.

NCI-Supported Research Programs

See the full list of bladder cancer research projects that NCI funded in FY 2018.

Many NCI-funded researchers working at the NIH campus and across the United States and the world are seeking ways to address bladder cancer more effectively. Some research is basic, exploring questions as diverse as the biological underpinnings of cancer. And some is more clinical, seeking to translate this basic information into improving patient outcomes.

The Bladder Specialized Program of Research Excellence, or SPORE, is a cornerstone of the NCI’s efforts to promote collaborative, interdisciplinary translational research on bladder cancer. It is currently located at Memorial Sloan Kettering Cancer Center

NCI’s Division of Cancer Epidemiology and Genetics (DCEG) conducts studies on bladder cancer to learn about risk factors for the disease. Some of their areas of study are described in these bladder cancer research studies

In addition, NCI has funding opportunities for researchers aimed at encouraging investigations of the biology and underlying mechanisms of bladder cancer. 

Bladder Cancer Research Results

The following are some of our latest news articles on bladder cancer research.

View the full list of Bladder Cancer Research Results and Study Updates.

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