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Eighteen-year survival after GD2-directed Chimeric Antigen Receptor-Modified Immune Effector Cell Treatment for Neuroblastoma

GSE284343 Homo sapiens Other; Expression profiling by high throughput sequencing 12 samples Submitted 2025/01/08 Platform GPL24676
Summary
We report long-term outcomes up to 18 years of a clinical trial treating children with neuroblastoma with EBV-specific T lymphocytes and CD3-activated T cells – each expressing chimeric antigen receptors (CAR) targeting GD2 but without an embedded costimulatory sequence (1st generation CARs). These CARs incorporated barcoded sequences to track each infused population. Of 11 patients with active disease at infusion, three patients achieved a complete response that was sustained in 2, one for 8 years until lost to follow up and one for 18+ years. Of eight patients with no evidence of disease at time of CAR-T administration, five are disease-free at their last follow-up between 10-15 years post-infusion. Intermittent low levels of transgene were detected during the follow up period with significantly greater persistence in those who were long-term survivors. In conclusion, despite using first-generation vectors that are nowadays no longer employed because of the lack of costimulatory 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 >18 years.
Published in
Long-term outcomes of GD2-directed CAR-T cell therapy in patients with neuroblastoma
Li CH, Sharma S, Heczey AA et al. · Nature medicine 2025 · PMID 39962287 · doi:10.1038/s41591-025-03513-0
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Direct links to NCBI, no account and no request form: the whole study as GSE284343_RAW.tar, processed values as the series matrix, the supplementary file directory, and per-sample supplementary files for any of the 12 samples. Raw sequencing reads are also available from ENA.

Also filed as BioProject PRJNA1198629 and SRA study SRP551617. Searching any of these in the dataset finder brings you back here.

Study design
12 conditions, each sampled once — no replicated groups

Read from 12 sample titles: 12 distinct titles with little repetition. Check it against the sample list below before relying on it.

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