Phase 2 gemcitabine + Jelmyto mitomycin (DNA crosslinker) chemoablation in high-grade UTUC

Rutgers (Rutgers Cancer Institute of New Jersey) is running this Phase 2, kidney-sparing, single-arm study testing sequential endoluminal gemcitabine and Jelmyto (mitomycin gel) delivered into the affected collecting system via nephrostomy. Gemcitabine is a nucleoside analog antimetabolite that inhibits DNA synthesis (via incorporation into DNA and inhibition of ribonucleotide reductase), and mitomycin is an alkylating/crosslinking agent that damages DNA; Jelmyto is UroGen Pharma’s reverse-thermal gel formulation designed to increase local dwell time in the upper tract. Eligible patients have “favorable high-risk” non–muscle-invasive UTUC (papillary high-grade Ta ± carcinoma in situ) that is not endoscopically resectable, where kidney preservation is a key goal. The main endpoint is complete response, with follow-on dosing based on response and longer-term tracking of recurrence-free outcomes and renal preservation. Today’s care for high-grade, non–muscle-invasive but non-resectable UTUC often escalates to radical nephroureterectomy (kidney/ureter removal) due to progression risk; this trial explores whether intensified local chemoablation can safely avoid that morbidity in a carefully selected subset.