New ‘gel’ drug treats rare cancer

A rare cancer is one that affects fewer than 40,000 people per year. Finding efficient drugs and impacting a cure is also rare because of their limited use, which is why a new drug is so exciting for researchers.

While urothelial cancer is the tenth most commonly diagnosed cancer in the world, a form of it called LG-UTUC is extremely rare. Low-grade upper tract urothelial cancer has only 8,000 confirmed cases in the U.S. each year. It involves tumors in the tube that connects the kidney and the bladder.

With a traditionally high mortality rate, there was little funding for research or new treatments.

But now, the FDA has approved the first non-surgical option for these patients. It’s a new drug that can hopefully delay progression and avoid surgery to remove the kidney. The drug is called Jelmyto. It utilizes a unique hydrogel technology to deliver a chemo medication directly to the tumor site in the urinary tract.

Urologic oncologist Dr. Amirali Salmasi with UC San Diego Health describes how this new material helps get the chemo into the affected areas. “When it is in the cold, it’s like liquid. When we put in the body temperature, it forms like a gel,” Salmasi explained.

With this combo, the focused treatment is making a significant impact. A phase three clinical trial found 50% of patients with LG-UTUC experienced complete resolution of their disease for at least one year.

These results are remarkable, according to Salmasi. “I think it changed the field significantly,” he said.

A rare success story, it also paves the way for more promising treatments for rare and challenging cancers.