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GD2-CART01 was the first CAR-T to produce durable complete remissions in a childhood solid tumour: two-thirds responded and a third achieved complete remission.
NEJM 2023: ORR 63%, CR 33%; 3-year OS 60%, EFS 36%; grade 1-2 CRS common, one grade 3; safety switch not needed. Nature Medicine 2025 long-term update: ORR 66%, complete responses sustained 8-18+ years in earlier-generation recipients, and 5 of 8 patients treated in remission disease-free at 10-15 years. Phase 2 expansion and commercialisation (Italy) under way.
Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.
Shares GD2 (disialoganglioside), High-risk neuroblastoma, Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first, Neuroblastoma (paediatric).
Shares High-risk neuroblastoma, Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first, Neuroblastoma (paediatric), Rare and paediatric cancers without markets.
Shares GD2 (disialoganglioside), High-risk neuroblastoma, Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first, Neuroblastoma (paediatric).
Shares GD2 (disialoganglioside), High-risk neuroblastoma, Neuroblastoma (paediatric).
Shares Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first, Neuroblastoma (paediatric), Rare and paediatric cancers without markets.
Shares Armoured, logic-gated & next-gen CARs, Neuroblastoma (paediatric), CAR-T cell therapy.
Shares Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first, Neuroblastoma (paediatric), Rare and paediatric cancers without markets.
Shares GD2 (disialoganglioside), High-risk neuroblastoma, Neuroblastoma (paediatric), Rare and paediatric cancers without markets.