FDA's Jelmyto Approval Offers Patients 'Very Sorely Needed' Treatment Option

The Food and Drug Administration’s recent approval of the first drug to treat patients with low-grade upper tract urothelial cancer offers patients a “very sorely needed” option, according to Dr. John L. Gore.

“It’s a space where we have had a hard time helping patients for a long time,” Gore, a professor in urology and adjunct professor in surgery at the University of Washington, said in an interview with CURE®. “(Jelmyto [mitomycin gel]) gives patients an option. As someone who participated in the trial, it was pretty easy to recruit patients for the study because their alternatives were not very satisfying.”

Upper tract urothelial cancer (UTUC), a cancer of the lining of the urinary system, may block a patient’s ureter or kidney, which can cause swelling, infections and impairment of kidney function. Low-grade UTUC is rare and affects approximately 6,000 to 8,000 patients in the United States every year.

The approval was based off a study involving 71 patients, of which 58% achieved a complete response following six treatments of Jelmyto administered weekly. Nineteen patients who originally achieved a complete response maintained the response after 12 months of follow-up.

When a patient presents with high-grade UTUC, meaning the cancer cells look more aggressive under the microscope, common practice is to remove the kidney, Gore, who is also an affiliate investigator at the Fred Hutchinson Cancer Research Center in Seattle, said.

However, when a patient presents with low-grade UTUC, Gore noted that it can be excessive to remove the patient’s kidney. But he acknowledged that it can be difficult to deliver effective treatments into the kidney.

What physicians typically do is laser the tumor down and then sometimes insert a liquid chemotherapy directly into the organ. However, given that kidneys are constantly sending urine down to the bladder, chemotherapy can only stay in the kidney for a couple minutes.

“What this gel does is it has these reverse thermal properties, meaning that when you think about water, water is liquid when warm and solid when cooled,” Gore said. “This gel is liquid when cooled and sort of a semi-solid gel when warmed. When you squirt it into the kidney, it immediately forms a cast inside the kidney, but it’s impregnated with the chemotherapy so it allows this chemotherapy a much longer dwell time that's on the order of a couple of hours instead of a couple of minutes. It allows us to expose our patient’s cancer to chemotherapy for a much longer time and because of that, it's much more effective, we think, than just lasering the tumor.”

If laser treatments and spraying chemotherapy into the kidney didn’t work previously, the remaining option was to remove the kidney.

“Patients either had a successful course and the laser treatment worked, or they lost their kidney,” he said. “If one of our goals is to help people keep their kidney, it’s kind of the only thing out there that’s going to help them out.”

Like with anything, Gore stressed, the success rate was not 100%. There could be some side effects which Gore noted are important for patients to know about.

For instance, mitomycin, the chemotherapy used in this treatment, could cause some significant irritative cystitis — a chronic condition causing pain and pressure in the bladder — and some cases of stricturing both inside the kidney and in the ureter, or the tube from the kidney down to the bladder.

“That can be temporary, or patients could require long-term stents to manage it, so you may be able to keep your kidney but you may have an area of scarring in the drainage of the kidney that requires some long-term management,” he said. “For most of the patients, that’s a positive trade-off, but it is a trade-off.”

Although there are some side effects associated with the therapy, Gore noted that the drug was still successful in more than half of the patients. Additionally, in patients who had a response, the response seemed to be long-lasting.

“Some patients may still lose their kidney despite the treatment, but most patients would at least like to try the treatment,” he concluded.

Read CURE®’s original coverage on Jelmyto’s approval.