{"entity":{"id":"paper-nadofaragene-firadenovec-lancet-oncol-2021","kind":"paper","name":"Nadofaragene firadenovec gene therapy for BCG-unresponsive non-muscle-invasive bladder cancer","aka":[],"tldr":"A single instillation of a virus carrying the interferon gene into the bladder every three months cleared carcinoma in situ in about half of patients who had failed BCG, and became the first gene therapy approved for bladder cancer.","summary":"Single-arm phase 3 study of 157 patients with BCG-unresponsive high-grade non-muscle-invasive bladder cancer treated with intravesical nadofaragene firadenovec, a non-replicating adenovirus vector expressing interferon alfa-2b, every three months.\n\nAmong patients with carcinoma in situ, 53.4 percent had a complete response within 12 months and about a quarter remained free of high-grade recurrence at one year. Treatment was well tolerated with mostly transient bladder symptoms.","asOf":"2026-09-17","links":[{"label":"Lancet Oncol 2021","url":"https://doi.org/10.1016/S1470-2045(20)30540-4"},{"label":"PubMed","url":"https://pubmed.ncbi.nlm.nih.gov/33253641/"}],"tags":[],"related":[],"cancers":["non-muscle-invasive-bladder-cancer"],"sections":[],"technologies":[],"targets":[],"drugs":["nadofaragene-firadenovec"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":["lancet-oncology"],"dependsOn":[],"notes":[],"journal":"The Lancet Oncology","year":2021,"doi":"10.1016/S1470-2045(20)30540-4","pmid":"33253641","authors":"Boorjian SA, Alemozaffar M, Konety BR, et al.","paperType":"observational","findings":["Complete response in 53.4 percent of patients with carcinoma in situ within 12 months.","45.5 percent of responders remained in complete response at 12 months."],"whatItMeans":"Bladder-sparing gene therapy is a realistic option for BCG-unresponsive disease, given four times a year in clinic. Durability is limited and cystoscopic follow-up continues.","caveats":["Single-arm design with no cystectomy or chemotherapy comparator.","Approval in the United States came in 2022; access outside the United States remains limited."],"changedPractice":true,"participants":157},"route":"/key-papers/paper-nadofaragene-firadenovec-lancet-oncol-2021/","neighbours":{"cancer":[{"id":"non-muscle-invasive-bladder-cancer","kind":"cancer","name":"Non-muscle-invasive bladder cancer","route":"/cancers/non-muscle-invasive-bladder-cancer/"}],"drug":[{"id":"nadofaragene-firadenovec","kind":"drug","name":"Nadofaragene firadenovec","route":"/drugs/nadofaragene-firadenovec/"}],"journal":[{"id":"lancet-oncology","kind":"journal","name":"The Lancet Oncology","route":"/journals/lancet-oncology/"}]}}