{"entity":{"id":"gd2-cart01","kind":"trial","name":"GD2-CART01 (Bambino Gesù phase 1/2)","aka":[],"tldr":"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.","summary":"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.","status":"positive","asOf":"2026-09-07","links":[{"label":"ClinicalTrials.gov NCT03373097","url":"https://clinicaltrials.gov/study/NCT03373097"},{"label":"Nature Medicine 2025 long-term","url":"https://www.nature.com/articles/s41591-025-03513-0"}],"tags":[],"related":[],"cancers":["neuroblastoma","neuroblastoma-high-risk"],"sections":[],"technologies":["car-t","armored-car"],"targets":["gd2"],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":["crs"],"trials":[],"people":["crystal-mackall"],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"nct":"NCT03373097","phase":"1/2","setting":"Relapsed/refractory high-risk neuroblastoma: third-generation GD2 CAR-T with inducible caspase-9 safety switch","sponsor":"Ospedale Pediatrico Bambino Gesù","result":"ORR 63%, CR 33%; 3-year OS 60%.","started":"2018-01-05","startedType":"actual","yearReported":2023,"enrolled":27,"enrolledBasis":"treated","enrolledNote":"ClinicalTrials.gov lists an estimated 42 participants for the phase 1/2 study, which is still active; the NEJM 2023 paper reports the 27 patients treated.","outcomes":[{"endpoint":"Objective response rate","primary":true,"unit":"%","arms":[{"name":"GD2-CART01","n":27,"value":63}],"source":"https://www.nejm.org/doi/full/10.1056/NEJMoa2210859"},{"endpoint":"Overall survival at 3 years","unit":"%","arms":[{"name":"GD2-CART01","value":60}],"source":"https://doi.org/10.1056/NEJMoa2210859"}],"replication":"Stanford GD2 CAR-T (with IL-15, in DIPG/DMG) and Baylor GD2 CAR-T show consistent GD2 targetability; no randomised trial yet."},"route":"/trials/gd2-cart01/","neighbours":{"cancer":[{"id":"neuroblastoma-high-risk","kind":"cancer","name":"High-risk neuroblastoma","route":"/cancers/neuroblastoma-high-risk/"},{"id":"neuroblastoma","kind":"cancer","name":"Neuroblastoma (paediatric)","route":"/cancers/neuroblastoma/"}],"technology":[{"id":"armored-car","kind":"technology","name":"Armoured, logic-gated & next-gen CARs","route":"/technologies/armored-car/"},{"id":"car-t","kind":"technology","name":"CAR-T cell therapy","route":"/technologies/car-t/"}],"target":[{"id":"gd2","kind":"target","name":"GD2 (disialoganglioside)","route":"/targets/gd2/"}],"term":[{"id":"crs","kind":"term","name":"Cytokine release syndrome (CRS)","route":"/terms/crs/"}],"person":[{"id":"crystal-mackall","kind":"person","name":"Crystal L. Mackall","route":"/people/crystal-mackall/"}],"idea":[{"id":"idea-gd2-car-t-frontline-consolidation","kind":"idea","name":"GD2 CAR-T as consolidation in high-risk neuroblastoma","route":"/ideas/idea-gd2-car-t-frontline-consolidation/"}],"bottleneck":[{"id":"b-rare-cancers","kind":"bottleneck","name":"Rare and paediatric cancers without markets","route":"/bottlenecks/b-rare-cancers/"}],"roadmap":[{"id":"paediatric-oncology-roadmap","kind":"roadmap","name":"Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first","route":"/roadmaps/paediatric-oncology-roadmap/"}]}}