In the last three years, the U.S. Food and Drug Administration (FDA) approved ﬁve new drugs for treating advanced bladder cancer; before that, no new therapy for the disease had been approved in 30 years.
This is signiﬁcant because more advanced stages of the disease are particularly challenging to treat. “It requires chronic treatment—when one treatment stops working, we switch to another,” says Elizabeth Plimack, M.D., chief of the division of genitourinary medical oncology at Fox Chase Cancer Center in Philadelphia.
The new drugs are immunotherapy drugs. Instead of attacking cancer cells directly, they help your immune system do so. Atezolizumab (Tecentriq), durvalumab (Imﬁnzi), and avelumab Bavencio) target the protein PD-L1, while nivolumab (Opdivo) and pembrolizumab (Keytruda) target PD-1, another protein. These proteins are used by the cancer cells to hide from the immune system. But by blocking these proteins, the drugs enable the immune system to identify the cancer cells as alien, and take aggressive action against them. These drugs are also known as checkpoint inhibitors.
“Fifteen to 25 percent of people with advanced bladder cancer respond to immunotherapy, depending on whether it’s their ﬁrst or second treatment,” says Arjun V. Balar, M.D., an assistant professor of medicine and director of the genitourinary medical oncology program at NYU Langone’s Perlmutter Cancer Center in New York City. “If a patient’s cancer responds, we may be able to keep it under control for many years.”
“These are really exciting treatments that are changing the way we treat bladder cancer,” says Arlene Siefker-Radtke, M.D., a professor of genitourinary medical oncology at the MD Anderson Cancer Center in Houston.
Other promising therapies include combining immunotherapy with other treatments; using another class of immune checkpoint inhibitors along with a PD-1 drug; and using new targeted therapies, such as drugs that target mutations in the protein FGFR3. One such inhibitor, erdaﬁtinib, was recently submitted for FDA review for the treatment of advanced bladder cancer.
The potential beneﬁts of targeted therapy—analyzing the tumor’s DNA and matching a treatment accordingly—are being explored with other mutations, too. The idea is that depending on the DNA mutation in the tumor, a drug used for another type of cancer might be used to ﬁght bladder cancer.
“There’s a lot in the pipeline,” Dr. Balar says. “Our hope is that some people might not need anything besides immunotherapy, and that perhaps we may be able to cure more patients with more modern treatments.”