{"entity":{"id":"idea-gd2-car-t-frontline-consolidation","kind":"idea","name":"GD2 CAR-T as consolidation in high-risk neuroblastoma","aka":[],"tldr":"Give engineered GD2 T cells to children in remission after standard therapy, where the long-term data show the deepest and longest cures.","summary":"This idea would give GD2-directed CAR-T cells to children with high-risk neuroblastoma who are in remission after induction, surgery and transplant, replacing or following tandem transplant and anti-GD2 antibody therapy. Long-term follow-up of the GD2-CART01 phase 1/2 trial at Bambino Gesù showed that most children infused with no evidence of disease have remained disease-free for many years, far better than expected with standard therapy. The rationale is that minimal residual disease is the ideal setting for CAR-T, that GD2 is retained after chemotherapy, and that a safety switch mitigates risk. The proposed test is a randomised phase 2/3 within COG or SIOPEN; at an early clinical stage, it addresses the bottleneck of rare and paediatric cancers and sits in the paediatric roadmap.","asOf":"2026-09-07","links":[{"label":"ClinicalTrials.gov NCT03373097: GD2-CART01 (Bambino Gesù phase 1/2)","url":"https://clinicaltrials.gov/study/NCT03373097"}],"tags":[],"related":[],"cancers":["neuroblastoma"],"sections":[],"technologies":["car-t","armored-car"],"targets":["gd2"],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["gd2-cart01"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"hypothesis":"GD2 CAR-T consolidation after induction, surgery and transplant improves 3-year EFS from ~60% to ≥75% in high-risk neuroblastoma with acceptable CRS and no chronic neuropathy.","rationale":"Minimal residual disease is the ideal CAR-T setting; GD2 is retained after chemotherapy; safety switch mitigates risk.","test":"Randomised phase 2/3 within COG or SIOPEN: standard anti-GD2 immunotherapy vs anti-GD2 followed by GD2 CAR-T; EFS primary.","maturity":"early-clinical"},"route":"/ideas/idea-gd2-car-t-frontline-consolidation/","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/"}],"trial":[{"id":"gd2-cart01","kind":"trial","name":"GD2-CART01 (Bambino Gesù phase 1/2)","route":"/trials/gd2-cart01/"}],"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/"}]}}