The US Food and Drug Administration (FDA) Oncology Drugs Advisory Committee (ODAC) voted unanimously to recommend approval for CTL019, an experimental chimeric antigen receptor T cell (CAR-T) cancer therapy, for the treatment of young adults and children with advanced leukemia.1
CTL019 is a novel customized therapy, created by collecting white blood cells from individual patients and genetically reprogramming them to target tumor cells.
The FDA advisory panel based its recommendation on evidence from an interim analysis of the ELIANA study (ClinicalTrials.gov Identifier: NCT02435849), which showed that 83% of patients with acute lymphoblastic leukemia (ALL) refractory to prior therapy achieved complete remission after 3 months of CAR-T therapy.
A previous dose optimization study (ClinicalTrials.gov Identifier: NCT01747486) enrolled 24 patients with relapsed and refractory chronic lymphocytic leukemia (CLL) and assigned them to high dose or low dose CTL019 arms. The overall response rate (ORR) was 42%, the complete remission (CR) rate was 25%, and the partial response rate was 17%.2
To address the potential long term toxic effects of CAR-T therapy, which include viral infections, tumorigenicity, and brain toxicity, the manufacturers of CTL019 have pledged a 15 year follow-up period to monitor safety in treatment patients.
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CAR-T is a novel therapy and could lead to changing the treatment standards for cancer. The FDA will make a final decision for approval by October 3rd.
The ODAC meeting briefing document can be accessed here.
- Feurerstein A, Garde D. Novartis CAR-T cancer therapy wins expert support for FDA approval. STAT website. https://www.statnews.com/2017/07/12/novartis-car-t-fda-approval/. Published July 12, 2017. Accessed July 13, 2017.
- Porter DL, Frey NV, Melenhorst JJ, et al. Randomized, phase II dose optimization study of chimeric antigen receptor (CAR) modified T cells directed against CD19 in patients (pts) with relapsed, refractory (R/R) CLL. J Clin Oncol. 34(suppl 15). doi: 10.1200/JCO.2016.34.15_suppl.3009