ÌìÃÀ´«Ã½

Newswise — DALLAS – March 10, 2025 – While randomized comparative trials are needed, a relatively new treatment option for upper urinary tract cancers shows promise for lowering long-term recurrence in many patients with low-grade disease, according to a multicenter study led by researchers at UT Southwestern Medical Center.

UGN-101 – a reverse thermal mitomycin gel sold under the brand name Jelmyto – was the first FDA-approved treatment for upper tract urothelial carcinoma (UTUC). Since the drug entered the market in 2020, researchers at 15 academic and community centers have collaborated to measure its effectiveness and better understand possible adverse effects. This latest study, published in , found that UGN-101 resulted in three-year, recurrence-free survival in 65% of patients with low-grade UTUC who had an initial complete response.

“This is a relatively rare type of cancer and one that is difficult to treat,” said the study’s corresponding author, , Associate Professor of  and a member of the  at UT Southwestern. “The durability of response we observed is promising because it suggests that UGN-101 may provide long-term benefit as a maintenance therapy, extending patients’ lives beyond what was possible before without the need for kidney or ureter removal and lifelong dialysis.”

The retrospective data review included 132 patients treated with UGN-101 at the 15 institutions. Of those, 55 had no evidence of disease on the first endoscopic evaluation following UGN-101 induction. Those patients were included in the recurrence analysis to determine the longer-term efficacy of the treatment at three years.

The study builds on  by the multi-institutional cohort, published in 2023, showing UGN-101’s effectiveness at three months after treatment.

UGN-101 is administered via a catheter directed into the lining of the kidney. Once the catheter is in place, the body’s natural temperature activates the gel, releasing mitomycin to target any remaining cancer cells. Because the mitomycin is gel-based, it remains in contact with the upper urinary tract longer, increasing its effectiveness. After diagnosis of a low-grade UTUC, treatment plans include an induction course of six weekly doses administered either through a nephrostomy in the kidney in the outpatient clinic or via a ureteral catheter in the operating room. In some patients, additional maintenance doses are administered monthly. 

“While UGN-101 may offer benefits to a cohort of patients with low-grade cancers, we still have work to do to better understand even longer-term outcomes, the risks and benefits of maintenance therapy, and the clinical guidelines for effectively administering therapy over many years,” said study leader , Chief of Urologic Oncology, Professor of Urology, and a member of the Experimental Therapeutics Research Program at Simmons Cancer Center.

Brett Johnson, M.D., and Isamu Tachibana, M.D., Assistant Professors of Urology at UT Southwestern, also contributed to this study. 

Drs. Woldu and Lotan serve as consultants for UroGen Pharma Ltd., which manufactures UGN-101.

Dr. Lotan holds the Jane and John Justin Distinguished Chair in Urology, in Honor of Claus G. Roehrborn, M.D.

About UT Southwestern Medical Center   

UT Southwestern, one of the nation’s premier academic medical centers, integrates pioneering biomedical research with exceptional clinical care and education. The institution’s faculty members have received six Nobel Prizes and include 25 members of the National Academy of Sciences, 23 members of the National Academy of Medicine, and 14 Howard Hughes Medical Institute Investigators. The full-time faculty of more than 3,200 is responsible for groundbreaking medical advances and is committed to translating science-driven research quickly to new clinical treatments. UT Southwestern physicians provide care in more than 80 specialties to more than 120,000 hospitalized patients, more than 360,000 emergency room cases, and oversee nearly 5 million outpatient visits a year.