FDA Approves the First Major New Alternative to Surgery for Bladder Cancer in 20 years

Dr. Arjun Balar, Director of Genitourinary Medical Oncology at NYU Langone Health's Perlmutter Cancer Center on immunotherapy for bladder cancer

The newly approved treatment uses the blockbuster immunotherapy drug pembrolizumab (which has the brand name KEYTRUDA), for people with what’s called “non-muscle invasive bladder cancer in situ,” or cancer in the lining of the bladder that does not involve the bladder muscle. Immunotherapy drugs are treatments that use the body’s own immune system to treat the disease.

About 80,000 people are diagnosed with bladder cancer each year in the United States, according to the National Cancer Institute, making up about 4.6 percent of cancer diagnoses. About 75 percent of those cases are non-muscle invasive.

The surgery that is usually used to treat non-muscle invasive bladder cancer, according to Dr. Balar, is very effective in treating the disease. But after the surgery associated with bladder cancer, patients usually live with a bag to divert urine and the risk of complications is very high. Moreover, 1 out of every 20 people who have the surgery die due to the treatment.

In the study that led to the approval, 40% of patients responded to the drug, and 20% were still responding to the drug after one year, meaning one out of five patients in the study wouldn't have to have their bladder removed.

This approval marks the first FDA approval for non-muscle invasive bladder cancer patients since 1998, when the FDA green-lighted Valrubicin, but doctors seldom use Valrubicin because it is not very effective. “[Valrubicin does] kill off a lot of the cancer cells, but they usually grow back, usually within six months,” said Dr. Balar.

“On January 8, 2020, the Food and Drug Administration approved pembrolizumab (KEYTRUDA®, Merck & Co. Inc.) for the treatment of patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy,” wrote the Director of the FDA Oncology Center of Excellence, Dr. Richard Pazdur in an official FDA statement.

The most common side effects of the drug, according to the FDA release, were fatigue, diarrhea, rash, itching, pain in the muscles and bones, blood in the urine, cough, joint pain, nausea, constipation, urinary tract infection, peripheral edema or swelling, hypothyroidism, and nasal inflammation, according to the statement.

 

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