# GD2-CART01 (Bambino Gesù phase 1/2)

Source: https://onco.cc/trials/gd2-cart01/  
OnCo record `gd2-cart01` (Trial). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

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.

## Fields

- Kind: Trial
- Status: positive
- Last checked: 2026-09-07
- Registry id: 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
- Enrolled: 27
- Result: ORR 63%, CR 33%; 3-year OS 60%.
- Outcomes: Objective response rate: GD2-CART01 63%; Overall survival at 3 years: GD2-CART01 60%
- Replication: Stanford GD2 CAR-T (with IL-15, in DIPG/DMG) and Baylor GD2 CAR-T show consistent GD2 targetability; no randomised trial yet.

## Sources

- ClinicalTrials.gov NCT03373097: https://clinicaltrials.gov/study/NCT03373097
- Nature Medicine 2025 long-term: https://www.nature.com/articles/s41591-025-03513-0

## Connected records

- cancers: [High-risk neuroblastoma](https://onco.cc/cancers/neuroblastoma-high-risk/), [Neuroblastoma (paediatric)](https://onco.cc/cancers/neuroblastoma/)
- technologies: [Armoured, logic-gated & next-gen CARs](https://onco.cc/technologies/armored-car/), [CAR-T cell therapy](https://onco.cc/technologies/car-t/)
- targets: [GD2 (disialoganglioside)](https://onco.cc/targets/gd2/)
- terms: [Cytokine release syndrome (CRS)](https://onco.cc/terms/crs/)
- people: [Crystal L. Mackall](https://onco.cc/people/crystal-mackall/)
- ideas: [GD2 CAR-T as consolidation in high-risk neuroblastoma](https://onco.cc/ideas/idea-gd2-car-t-frontline-consolidation/)
- bottlenecks: [Rare and paediatric cancers without markets](https://onco.cc/bottlenecks/b-rare-cancers/)
- roadmaps: [Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first](https://onco.cc/roadmaps/paediatric-oncology-roadmap/)

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