New drug brings new hope for advanced bladder cancer
Forty percent of bladder cancer patients saw their tumors shrink with a new targeted therapy drug.
When Gary Price joined a clinical trial testing a new drug for patients with advanced bladder cancer, he knew his odds weren’t good. His doctors had told him he had just a few months to live, but still, he decided to give it a shot.
That was more than three years ago.
Price was first diagnosed with bladder cancer in 2013 after noticing blood in his urine. A series of tests with his primary care doctor confirmed his fears.
“Nobody wants to hear the words, ‘you have cancer.’ I immediately worried this would be my death sentence,” says Price, an oil well supervisor. “But back then, my doctor assured me the cancer was treatable and my outlook was good.”
A few days later, a surgeon removed Price’s tumor. He then began receiving Bacillus Calmette-Guerin, or BCG, an immunotherapy drug for early bladder cancer, delivered directly into the bladder. Immunotherapy is used to prompt the immune system to attack cancer cells. But small tumors kept reappearing in Price’s bladder that each required a new surgery.
“Over that next year or so, I had five or six additional surgeries to remove the tumors that kept growing,” he says. “My doctor told me the new tumors were not as aggressive as the original cancer, but it was also time I visit MD Anderson.”
Surgery and chemotherapy were among his limited bladder cancer treatment options
Surgeons at MD Anderson recommended removing Price’s bladder, right kidney, right ureter and prostate as his best chance for keeping cancer away. Despite knowing the surgery would leave him with an ostomy bag that, in the absence of a bladder, reroutes urine into a collection pouch outside the body, Price had the surgery in June 2015.
Unfortunately, a routine follow-up scan the following April found a suspicious spot in an old scar on his side. A biopsy confirmed his cancer had spread.
It was then that Price met Arlene Siefker-Radtke, M.D., professor of Genitourinary Medical Oncology and an expert in treating bladder cancer. She prescribed an aggressive course of chemotherapy, which required Price to be hospitalized for several days.
However, his tumor continued growing, and Siefker-Radtke explained that few treatment options remained.
“Chemotherapy has long been the standard of care for patients with bladder and other urothelial cancers, but it’s an aggressive treatment with many side effects, and only a small percentage of patients see real benefit,” explains Siefker-Radtke. “The recent emergence of immunotherapy has improved outcomes for some, but new treatments remain a major unmet need for these patients.”
Targeted therapy clinical trial offers hope for effective bladder cancer treatment
Siefker-Radtke learned that Price’s tumor carried a mutation in a gene named FGFR3. The mutation causes cancer cells to proliferate and grow rapidly. About 15 to 20% of patients with advanced bladder cancer and up to 35% of patients with other urothelial cancers have mutations in FGFR genes.
It just so happened that Siefker-Radtke was launching a clinical trial that would test a new drug called erdafitinib (trade name Balversa). The drug was designed to turn off the mutation that causes the tumor cells to proliferate.
Price was the first MD Anderson patient to enroll.
The drug, an oral medication taken at home, proved largely effective, and Price’s tumor has been stable for the past three years.
This year, Siefker-Radtke and colleagues published the results of the clinical trial in the New England Journal of Medicine. 40% of patients enrolled in the trial saw their tumors shrink, remain stable, or disappear.
In April 2019, the Food and Drug Administration approved erdafitinib, making it the first targeted therapy ever approved for patients with advanced urothelial cancers. Targeted therapy drugs block specific proteins or genes that help cancers grow and spread.
“With this approval, we have an additional drug to add to our toolbox,” says Siefker-Radtke. “My hope is we’ll be able to continue our research and bring its benefits to more and more patients.”
“Proof that new treatments can change everything”
Today, Price’s tumor remains roughly the size of a pea. He knows he’s not cured, but he also never expected to be here today.
Though it keeps his tumor at bay, erdafitinib does come with its share of side effects. Price has severe pain in his feet and hands, which keep him from one of his favorite pastimes – playing guitar. He’s also lost most of his sense of taste.
But he’s managed to keep working in the oil field and participating in his favorite hobby – competitive clay pigeon shooting.
“I make the best I can of every day, and I’m grateful that I’ve had as many days as I have,” says Price. “I hope my story will inspire others, and make more people aware of the need for researching new bladder cancer therapies. I’m proof that new treatments can change everything.”