Several years ago, when Arjun V. Balar, MD, would deliver a diagnosis of advanced bladder cancer to a typical patient—a man over the age of 70—the conversation was a grim one for doctor and patient alike. Dr. Balar, a medical oncologist at NYU Langone’s Perlmutter Cancer Center, had to explain that if the cancer had traveled beyond the lining of the bladder, that organ and others nearby would need to be removed; that for cancer that had spread beyond the bladder, most older patients found the standard chemotherapy regimen intolerable; and that the less potent alternative would extend life by only 10 months or so. “The patient would be devastated,” recalls Dr. Balar.

But earlier this year, despair gave way to hope. Two ongoing international clinical trials led by Dr. Balar, director of the Genitourinary Medical Oncology Program at Perlmutter Cancer Center, and funded by pharmaceutical manufacturers, have paved the way for the approval of two novel treatments for patients with advanced bladder cancer who are ineligible for the standard chemotherapy. The medications—the first ever approved for this typically frail population— give older patients a nearly 25 percent chance of survival.

Trial results showed that the immunotherapy treatments shrank tumors by at least 30 percent in one-quarter of the nearly 500 study participants combined in both studies. Of those, 5 percent got the news that that their tumors had disappeared.

Atezolizumab (Tecentriq^®), approved in April 2017, and pembrolizumab (Keytruda^®), green-lighted one month later, belong to a class of cancer drugs known as immune checkpoint inhibitors, which are designed to release a break on immune cells, allowing them to attack tumor cells. Trial results showed that the immunotherapy treatments shrank tumors by at least 30 percent in one-quarter of the nearly 500 study participants combined in both studies. Of those, 5 percent got the best news imaginable: their tumors had totally disappeared.

“While this percentage may seem small,” Dr. Balar notes, “remember that on average, these patients would otherwise have died within 10 months.”

Each year, about 79,000 Americans are diagnosed with bladder cancer. The disease claims some 17,000 lives annually, and less than 15 percent of those with a late-stage tumor survive more than 5 years. Bladder cancer strikes four times as many men as women, making it the fourth most common cancer among men. About 80 percent of those diagnosed are current or former smokers.

The malignancy is usually confined to the inner lining of the bladder, requiring most patients to undergo surgery to remove the tumor. If the cancer has invaded deeper, from the inner lining into the muscle, however, the bladder must be removed, along with the prostate in men or the uterus and ovaries in women.

“Removal of the entire bladder and reconstruction of a new urinary tract is a complex surgical procedure associated with a high rate of complications,” explains urologic oncology surgeon William C. Huang, MD, who was not involved in the trials. Dr. Balar, in collaboration with Dr. Huang, is now leading a trial to see if surgery can be avoided by using immunotherapy in combination with chemoradiation. “If immunotherapy can reduce the number of people who may need to have their entire bladder removed,” says Dr. Huang, “that would be a huge step forward.”

Dr. Balar first recognized the potential of immunotherapy during the clinical trial with atezolizumab. He recalls one participant, an 80-year-old woman with a very aggressive cancer that had widely metastasized, who looked and felt better within weeks of her first dose. Dr. Balar reports that three years later, the woman remains cancer free.

Nearly a dozen clinical trials are currently under way at NYU Langone to test novel therapies for bladder cancer, including new immunotherapies, targeted therapies, and combinations. “Patients used to choose between chemotherapy and hospice care,” says Dr. Balar. “It’s a privilege to provide them with hope instead.”