Bladder Cancer Coverage from Every Angle

Targeting Bladder Carcinoma in Situ With Novel Gene Therapy

By: Lauren Harrison, MS
Posted: Wednesday, November 18, 2020

The use of a novel intravescial gene-mediated therapy, nadofaragene firadenovec, seemed to demonstrate activity in patients with Bacillus Calmette-Guérin (BCG)-unresponsive carcinoma in situ of the bladder. Nadofaragene firadenovec works through delivering the human interferon alpha-2b gene, leading to an increase in expression of interferon alpha-2b. This work was published in the Journal of Clinical Oncology by Stephen A. Boorjian, MD, of Mayo Clinic in Rochester, Minnesota, and colleagues.

“These data represent a potentially significant management advancement for a historically difficult-to-treat disease state,” concluded the authors.

This phase III, open-label, multicenter trial enrolled 157 patients with high-grade non–muscle-invasive bladder cancer (carcinoma in situ and/or Ta/T1 disease) that was unresponsive to BCG. Patients received 75 mL of 3 x 1011 vp/mL of nadofaragene firadenovec once every 3 months for up to four doses in the first year of therapy. Additional dosing was administered at the investigator’s discretion.

A total of 103 patients had carcinoma in situ of the bladder, and 53.4% of these patients achieved a complete response to therapy. These responses were all seen by month 3 after treatment. Within this group of complete responders, 45.5% remained free of high-grade recurrence at month 12, as confirmed by biopsy. Among the patients with high-grade Ta/T1 disease alone, 72.9% did not have recurrence at 3 months, and 43.8% were recurrence-free at 12 months.

Treatment-emergent adverse events were generally transient among this cohort. They included instillation-site discharge (33.1%), fatigue (23.6%), bladder spasm (19.7%), micturition urgency (17.8%), and hematuria (16.6%). Two grade 4 adverse events were reported, including sepsis and an anaphylactic reaction, which were not related to the study drug. There were no grade 5 adverse events observed.

Disclosure: For a full list of authors’ disclosures, visit

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