Bladder Cancer Coverage from Every Angle

AUA 2021: Trial Update on Novel Gene-Mediated Therapy for High-Grade Bladder Cancer

By: Kayci Reyer
Posted: Wednesday, September 29, 2021

Findings presented during the 2021 American Urological Association (AUA) Annual Meeting (Abstract MP16-02) suggest that treatment with nadofaragene firadenovec, a novel gene-mediated therapy that causes prolonged expression of IFNα2b, appears to be well tolerated and effective in some patients with bacillus Calmette-Guérin (BCG)-unresponsive, high-grade non–muscle-invasive bladder cancer. Yair Lotan, MD, of The University of Texas Southwestern Medical Center, Dallas, and colleagues evaluated the efficacy, safety, and durability of response at a long-term follow-up of 24 months.

The multicenter, phase III trial included two cohorts of patients with BCG-unresponsive non–muscle-invasive bladder cancer: carcinoma in situ (± Ta/T1) and high-grade Ta/T1 alone. Among the high-grade Ta/T1 cohort, 48 patients were included in the efficacy analysis, and 50 patients were in the safety analysis. All patients received 75 mL of nadofaragene firadenovec once every 3 months for a maximum of four doses before undergoing a five-site biopsy at 12 months. Patients without disease recurrence at that time could continue treatment in 3-month intervals.

At data cutoff in September 2020, 9 of the 48 patients in the efficacy analysis (18.8%) had received 24 months of treatment; 33 continued to be followed and underwent restricted-mean follow-up at 23.5 months. At 3, 12, and 24 months, 35 (72.9%), 21 (43.8%), and 16 (33.3%) patients were high-grade disease recurrence-free, respectively. A total of 16 patients (45.7%) who were disease free at 3 months remained so at 24 months.

Instillation-site discharge was the most common treatment-related adverse event (26%), followed by dysuria (16%), bladder spasm (14%), and micturition urgency (14%). Most adverse events were a maximum of grade 2. One patient discontinued treatment due to an adverse event. One grade 4 adverse event occurred, though it was thought not to be related to treatment, and no adverse events resulting in death were reported.

Disclosure: For full disclosures of the study authors, visit

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