Patients with high-risk non-muscle-invasive bladder cancer (NMIBC) not responding to intravesical Bacillus Calmette-Guérin (BCG) therapy now have a new treatment alternative to cystectomy, which has almost always been their only treatment option. The US Food and Drug Administration (FDA) has approved the first gene therapy “nadofaragene firadenovec-vncg” for the treatment of adult patients with high-risk BCG-unresponsive NMIBC with carcinoma in situ (CIS) with or without papillary tumors.^1,2

Nadofaragene firadenovec-vncg, available as Adstiladrin, is a non-replicating adenoviral vector based gene therapy and is instilled into the bladder through a urinary catheter once every three months for one year. “The adenovirus vector enters the cells of the bladder wall, releasing a gene that directs the cells to secrete high quantities of interferon alfa-2b, a naturally occurring cancer-fighting protein”.²

Contraindication: Immunosuppressed patients or those with immunodeficiency

Side effects: Fatigue, chills, fever, bladder discharge, bladder spasm, urgency, hematuria, dysuria.

Nadofaragene firadenovec-vncg is expected to be available in 2023, in the later part of the year. The cost of the therapy is yet to be revealed.

References

1. FDA news release. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treatment-high-risk-non-muscle-invasive-bladder-cancer, Dec. 16, 2022. Accessed on Dec. 19, 2022.
2. FDA approves first gene therapy for bladder cancer - Medscape - Dec 16, 2022.