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Drug Evaluation

An evaluation of nadofaragene firadenovec-vncg for the treatment of high-risk BCG-unresponsive non-muscle-invasive bladder cancer

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Pages 415-423 | Received 21 Jan 2024, Accepted 05 Jun 2024, Published online: 13 Jun 2024

References

  • Bladder cancer: non-muscle invasive guideline - American urological association. n.d [cited 2023 Dec 26]. Available from: https://www.auanet.org/guidelines-and-quality/guidelines/bladder-cancer-non-muscle-invasive-guideline
  • Ritch CR, Velasquez MC, Kwon D, et al. Use and validation of the AUA/SUO risk grouping for nonmuscle invasive bladder cancer in a contemporary cohort. J Urol. 2020;203:505–511. doi: 10.1097/JU.0000000000000593
  • Grimm M-O, van der Heijden AG, Colombel M, et al. Treatment of high-grade non-muscle-invasive bladder carcinoma by standard number and dose of BCG instillations versus reduced number and standard dose of BCG instillations: results of the European association of urology research foundation randomised phase III clinical trial “NIMBUS”. Eur Urol. 2020;78(5):690–698. doi: 10.1016/j.eururo.2020.04.066
  • Yang LS, Shan BL, Shan LL, et al. A systematic review and meta-analysis of quality of life outcomes after radical cystectomy for bladder cancer. Surg Oncol. 2016;25:281–297. doi: 10.1016/j.suronc.2016.05.027
  • Collacott H, Krucien N, Heidenreich S, et al. Patient preferences for treatment of bacillus calmette-guérin–unresponsive non–muscle-invasive bladder cancer: a cross-country choice experiment. Eur UrolOpen Science. 2023;49:92–99. doi: 10.1016/j.euros.2022.12.016
  • Kamat AM, Colombel M, Sundi D, et al. BCG-unresponsive non-muscle-invasive bladder cancer: recommendations from the IBCG. Nat Rev Urol. 2017;14(4):244–255. doi: 10.1038/nrurol.2017.16
  • Steinberg G, Bahnson R, Brosman S, et al. Efficacy and safety of valrubicin for the treatment of Bacillus Calmette-Guerin refractory carcinoma in situ of the bladder. The valrubicin study group. J Urol. 2000;163(3):761–767. doi: 10.1016/S0022-5347(05)67799-3
  • Balar AV, Kamat AM, Kulkarni GS, et al. Pembrolizumab monotherapy for the treatment of high-risk non-muscle-invasive bladder cancer unresponsive to BCG (KEYNOTE-057): an open-label, single-arm, multicentre, phase 2 study. The Lancet Oncology. 2021;22:919–930. doi: 10.1016/S1470-2045(21)00147-9
  • Commissioner O of the. FDA approves first gene therapy for the treatment of high-risk, non-muscle-invasive bladder cancer. FDA; 2022 [cited 2023 Dec 26]. Available from: https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treatment-high-risk-non-muscle-invasive-bladder-cancer
  • Nieder AM, Brausi M, Lamm D, et al. Management of stage T1 tumors of the bladder: international consensus panel. Urology. 2005;66:108–125. doi: 10.1016/j.urology.2005.08.066
  • Lerner SP, Dinney C, Kamat A, et al. Clarification of bladder cancer disease states following treatment of patients with intravesical BCG. Bladder Cancer. 2015;1:29–30. doi: 10.3233/BLC-159002
  • Research C for DE and. Bacillus calmette-guérin-unresponsive nonmuscle invasive bladder cancer: developing drugs and biologics for treatment guidance for industry 2020. [cited 2023 Dec 26]. Available from: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/bacillus-calmette-guerin-unresponsive-nonmuscle-invasive-bladder-cancer-developing-drugs-and
  • Christophersen IS, Jordal R, Osther K, et al. Interferon therapy in neoplastic disease. A preliminary report. Acta Med Scand. 1978;204:471–476. doi: 10.1111/j.0954-6820.1978.tb08475.x
  • Lamm D, Brausi M, Ma O, et al. Interferon alfa in the treatment paradigm for non–muscle-invasive bladder cancer. Urol Oncol. 2014;32(1):35.e21–30. doi: 10.1016/j.urolonc.2013.02.010
  • Joudi FN, Smith BJ, O’Donnell MA, et al. Final results from a national multicenter phase II trial of combination bacillus Calmette-Guérin plus interferon α-2B for reducing recurrence of superficial bladder cancer. Urol Oncol. 2006;24(4):344–348. doi: 10.1016/j.urolonc.2005.11.026
  • Shepherd AR, Shepherd E, Brook NR. Intravesical Bacillus Calmette-Guérin with interferon-alpha versus intravesical Bacillus Calmette-Guérin for treating non-muscle-invasive bladder cancer. Cochrane Database Of Systematic Reviews. 2017;3(3):CD012112. doi: 10.1002/14651858.CD012112.pub2
  • Belldegrun AS, Franklin JR, O’Donnell MA, et al. Superficial bladder cancer: the role of interferon-alpha. J Urol. 1998;159:1793–1801. doi: 10.1016/S0022-5347(01)63160-4
  • Benedict WF, Tao Z, Kim C-S, et al. Intravesical Ad-IFNα causes marked regression of human bladder cancer growing orthotopically in nude mice and overcomes resistance to IFN-α protein. Mol Ther. 2004;10(3):525–532. doi: 10.1016/j.ymthe.2004.05.027
  • Mulligan RC. The basic science of gene therapy. Sci. 1993;260:926–932. doi: 10.1126/science.8493530
  • Wirth T, Parker N, Ylä-Herttuala S. History of gene therapy. Gene. 2013;525:162–169. doi: 10.1016/j.gene.2013.03.137
  • Narayan VM, Dinney CPN. Intravesical gene therapy. Urol Clin North Am. 2020;47(1):93–101. doi: 10.1016/j.ucl.2019.09.011
  • Iqbal Ahmed CM, Johnson DE, Demers GW, et al. Interferon α2b gene delivery using adenoviral vector causes inhibition of tumor growth in xenograft models from a variety of cancers. Cancer Gene Ther. 2001;8(10):788–795. doi: 10.1038/sj.cgt.7700364
  • Klingler CH. Glycosaminoglycans: how much do we know about their role in the bladder? Urologia. 2016;83(Suppl 1):11–14. doi: 10.5301/uro.5000184
  • Lilly JD, Parsons CL. Bladder surface glycosaminoglycans is a human epithelial permeability barrier. Surg Gynecol Obstet. 1990;171:493–496.
  • Nickel JC, Downey J, Morales A, et al. Relative efficacy of various exogenous glycosaminoglycans in providing a bladder surface permeability barrier. J Urol. 1998;160(2):612–614. doi: 10.1016/S0022-5347(01)62968-9
  • Engler H, Anderson SC, Machemer TR, et al. Ethanol improves adenovirus-mediated gene transfer and expression to the bladder epithelium of rodents. Urology. 1999;53:1049–1053. doi: 10.1016/s0090-4295(98)00641-4
  • Connor RJ, Engler H, Machemer T, et al. Identification of polyamides that enhance adenovirus-mediated gene expression in the urothelium. Gene Ther. 2001;8:41–48. doi: 10.1038/sj.gt.3301348
  • Yamashita M, Rosser CJ, Zhou J-H, et al. Syn3 provides high levels of intravesical adenoviral-mediated gene transfer for gene therapy of genetically altered urothelium and superficial bladder cancer. Cancer Gene Ther. 2002;9:687–691. doi: 10.1038/sj.cgt.7700488
  • Benedict WF, Tao Z, Kim C-S, et al. Intravesical Ad-IFNα causes marked regression of human bladder cancer growing orthotopically in nude mice and overcomes resistance to IFN-α protein. Mol Ther. 2004;10:525–532. doi: 10.1016/j.ymthe.2004.05.027
  • Papageorgiou A, Dinney CPN, McConkey DJ. Interferon-α Induces TRAIL expression and cell death via an IRF-1-dependent mechanism in human bladder cancer cells. Cancer Biol Ther. 2007;6:872–879. doi: 10.4161/cbt.6.6.4088
  • Dinney CP, Bielenberg DR, Perrotte P, et al. Inhibition of basic fibroblast growth factor expression, angiogenesis, and growth of human bladder carcinoma in mice by systemic interferon-alpha administration. Cancer Res. 1998;58:808–814.
  • Izawa JI, Sweeney P, Perrotte P, et al. Inhibition of tumorigenicity and metastasis of human bladder cancer growing in athymic mice by interferon-beta gene therapy results partially from various antiangiogenic effects including endothelial cell apoptosis. Clin Cancer Res. 2002;8:1258–1270.
  • Plote D, Choi W, Mokkapati S, et al. Inhibition of urothelial carcinoma through targeted type I interferon-mediated immune activation. Oncoimmunology. 2019;8:e1577125. doi: 10.1080/2162402X.2019.1577125
  • Diamond MS, Kinder M, Matsushita H, et al. Type I interferon is selectively required by dendritic cells for immune rejection of tumors. J Exp Med. 2011;208:1989–2003. doi: 10.1084/jem.20101158
  • Dinney CPN, Fisher MB, Navai N, et al. Phase I trial of intravesical recombinant adenovirus mediated interferon-α2b formulated in Syn3 for Bacillus Calmette-Guérin failures in nonmuscle invasive bladder cancer. J Urol. 2013;190:850–856. doi: 10.1016/j.juro.2013.03.030
  • Mitra AP, Narayan VM, Mokkapati S, et al. Antiadenovirus antibodies predict response durability to nadofaragene firadenovec therapy in BCG-unresponsive non–muscle-invasive bladder cancer: secondary analysis of a phase 3 clinical trial. Eur Urol. 2022;81:223–228. doi: 10.1016/j.eururo.2021.12.009
  • Shore ND, Boorjian SA, Canter DJ, et al. Intravesical rAd–IFNα/Syn3 for patients with high-grade, bacillus calmette-guerin–refractory or relapsed non–muscle-invasive bladder cancer: a phase II randomized study. JCO. 2017;35:3410–3416. doi: 10.1200/JCO.2017.72.3064
  • Boorjian SA, Alemozaffar M, Konety BR, et al. Intravesical nadofaragene firadenovec gene therapy for BCG-unresponsive non-muscle-invasive bladder cancer: a single-arm, open-label, repeat-dose clinical trial. The Lancet Oncology. 2021;22(1):107–117. doi: 10.1016/S1470-2045(20)30540-4
  • Society of urologic oncology - efficacy of intravesical nadofaragene firadenovec for patients with BCG-unresponsive carcinoma in situ of the bladder: 36-month follow-up from a phase 3 trial. n.d [cited 2023 Dec 31]. Available from: https://suo-abstracts.secureplatform.com/a/gallery/rounds/18/details/2938
  • Ferring Pharmaceuticals. A phase 3, open label trial to evaluate the safety and efficacy of FE 999326 administered intravesically to Japanese subjects with high-grade, BCG unresponsive, non-muscle invasive bladder cancer (NMIBC). clinicaltrials. gov; 2023.
  • Flaig TW, Spiess PE, Abern M, et al. NCCN guidelines® insights: bladder cancer, version 2.2022: featured updates to the NCCN guidelines. Journal Of The National Comprehensive Cancer Network. 2022;20:866–878. doi: 10.6004/jnccn.2022.0041
  • Steinberg RL, Thomas LJ, Brooks N, et al. Multi-institution evaluation of sequential gemcitabine and docetaxel as rescue therapy for nonmuscle invasive bladder cancer. J Urol. 2020;203:902–909. doi: 10.1097/JU.0000000000000688
  • Tan WS, Grajales V, Bree K, et al. Bacillus Calmette–Guérin (BCG) unresponsive non‐muscle‐invasive bladder cancer: are the subgroups equal? BJU Int. 2023;132:384–386. doi: 10.1111/bju.16087
  • Chamie K, Chang SS, Kramolowsky E, et al. IL-15 superagonist NAI in BCG-unresponsive non–muscle-invasive bladder cancer. NEJM Evidence. 2022;2(1):EVIDoa2200167. doi: 10.1056/EVIDoa2200167
  • Uchio EM, Lamm DL, Shore ND, et al. A phase 3, single-arm study of CG0070 in subjects with nonmuscle invasive bladder cancer (NMIBC) unresponsive to Bacillus Calmette-Guerin (BCG). JCO. 2022;40:TPS598–TPS598. doi: 10.1200/JCO.2022.40.6_suppl.TPS598
  • Packiam VT, Lamm DL, Barocas DA, et al. An open label, single-arm, phase II multicenter study of the safety and efficacy of CG0070 oncolytic vector regimen in patients with BCG-unresponsive non–muscle-invasive bladder cancer: Interim results. Urol Oncol. 2018;36(10):440–447. doi: 10.1016/j.urolonc.2017.07.005
  • Necchi A, Jacob JM, Daneshmand S, et al. LBA105 Results from SunRISe-1 in patients (Pts) with bacillus Calmette–Guérin (BCG)-unresponsive high-risk non–muscle-invasive bladder cancer (HR NMIBC) receiving TAR-200 monotherapy. Annals Oncology. 2023;34:S1343–4. doi: 10.1016/j.annonc.2023.10.111
  • Chen W, Liu N, Yuan Y, et al. ALT-803 in the treatment of non-muscle-invasive bladder cancer: preclinical and clinical evidence and translational potential. Front Immunol. 2022;13:1040669. doi: 10.3389/fimmu.2022.1040669
  • Daneshmand S, Brummelhuis ISG, Pohar KS, et al. The safety, tolerability, and efficacy of a neoadjuvant gemcitabine intravesical drug delivery system (TAR-200) in muscle-invasive bladder cancer patients: a phase I trial. Urol Oncol. 2022;40(7):.e344.1–.e344.9. doi: 10.1016/j.urolonc.2022.02.009
  • Burke JM, Lamm DL, Meng MV, et al. A first in human phase 1 study of CG0070, a GM-CSF expressing oncolytic adenovirus, for the treatment of nonmuscle invasive bladder cancer. J Urol. 2012;188:2391–2397. doi: 10.1016/j.juro.2012.07.097
  • Li R, Steinberg GD, Uchio EM, et al. CORE1: Phase 2, single-arm study of CG0070 combined with pembrolizumab in patients with nonmuscle-invasive bladder cancer (NMIBC) unresponsive to bacillus Calmette-Guerin (BCG). JCO. 2022;40:4597–4597. doi: 10.1200/JCO.2022.40.16_suppl.4597
  • Mokkapati S, Narayan VM, Manyam GC, et al. Lentiviral interferon: a novel method for gene therapy in bladder cancer. Mol Ther Oncolytics. 2022;26:141–157. doi: 10.1016/j.omto.2022.06.005
  • Apolo AB, Ballman KV, Sonpavde GP, et al. AMBASSADOR alliance A031501: phase III randomized adjuvant study of pembrolizumab in muscle-invasive and locally advanced urothelial carcinoma (MIUC) vs observation. JCO. 2024;42:LBA531–LBA531. doi: 10.1200/JCO.2024.42.4_suppl.LBA531
  • Basile G, Bandini M, Gibb EA, et al. Neoadjuvant pembrolizumab and radical cystectomy in patients with muscle-invasive urothelial bladder cancer: 3-year median follow-up update of PURE-01 trial. Clin Cancer Res. 2022;28:5107–5114. doi: 10.1158/1078-0432.CCR-22-2158
  • Ferro M, Barone B, Crocetto F, et al. Predictive clinico-pathological factors to identify BCG, unresponsive patients, after re-resection for T1 high grade non-muscle invasive bladder cancer. J Urol Oncol. 2022;40(11):.e490.13–.e490.20. doi: 10.1016/j.urolonc.2022.05.016
  • Ferro M, Tătaru OS, Musi G, et al. Modified glasgow prognostic score as a predictor of recurrence in patients with high grade non-muscle invasive bladder cancer undergoing intravesical bacillus calmette–guerin immunotherapy. Diagnostics. 2022;12:586. doi: 10.3390/diagnostics12030586

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