Andras Heczey

2 months ago | nature.com | Che-Hsing Li |Sandhya Sharma |Andras Heczey |Mae Woods |Sachin G. Thakkar |Mengfen Wu | +7 more

In a phase 1 clinical trial open to accrual from 2004 to 2009, we treated children with neuroblastoma with Epstein–Barr virus (EBV)-specific T lymphocytes and CD3-activated T cells—each expressing chimeric antigen receptors (CARs) targeting GD2 but without an embedded co-stimulatory sequence (first-generation CARs). These CARs incorporated barcoded sequences to track each infused population. We previously reported outcomes up to 5 years and now report long-term outcomes up to 18 years. Of 11 patients with active disease at infusion, three achieved a complete response that was sustained in two patients, one for 8 years until lost to follow-up and one for more than 18 years. Of eight patients with no evidence of disease at the time of CAR-T administration, five are disease free at their last follow-up between 10 years and 15 years after infusion. Intermittent low levels of transgene were detected during the follow-up period with significantly greater persistence in those who were long-term survivors. Despite using first-generation vectors that are no longer employed because of the lack of co-stimulatory domains, patients with relapsed/refractory neuroblastoma achieved long-term disease control after receiving GD2 CAR-T cell therapy, including one patient now in remission of relapsed disease for more than 18 years. ClinicalTrials.gov identifier: NCT00085930 . In an extended follow-up of a phase 1 trial evaluating a first-generation GD2-directed CAR-T cell therapy in patients with neuroblastoma, one patient has had a sustained clinical response for over 18 years without requiring any additional therapy, with translational data from long-term survivors providing broader insights into potential determinants of CAR-T cell response.

Jan 9, 2024 | nature.com | Andras Heczey |Xin Xu |Amy N Courtney |Claudia Amador |Sachin G. Thakkar |TAO WANG | +2 more

Correction to: Nature Medicine https://doi.org/10.1038/s41591-023-02363-y, published online 15 May 2023. In the version of this article initially published, the Acknowledgements did not include thanks to the Cancer Prevention and Research Institute of Texas (CPRIT) for support under grant no. RP180785. The error has been corrected in the HTML and PDF versions of the article. About this articleHeczey, A., Xu, X., Courtney, A.N. et al.