GD2-CART01 (Bambino Gesù phase 1/2)
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.
Setting
Relapsed/refractory high-risk neuroblastoma: third-generation GD2 CAR-T with inducible caspase-9 safety switch
Phase
Phase 1/2
Sponsor
Ospedale Pediatrico Bambino Gesù
Registry
Headline result
ORR 63%, CR 33%; 3-year OS 60%.
Reported
2023
Enrolled
27
Replication
Stanford GD2 CAR-T (with IL-15, in DIPG/DMG) and Baylor GD2 CAR-T show consistent GD2 targetability; no randomised trial yet.
In plain words
What these results mean for people, not percentages
Objective response rateprimaryresponse endpoint
- 63 out of 100 people had their tumour shrink with GD2-CART01.
- A response rate counts how many people had their tumours shrink by a set amount; shrinking is encouraging but does not by itself show that people live longer or feel better.
Overall survival at 3 yearssurvival endpoint
- 60 out of 100 people alive at 3 years with GD2-CART01.
- Overall survival counts deaths from any cause, so it is the most direct measure of whether a treatment helps people live longer.
Be careful
- There was no comparison group, so this number cannot tell you how much of the effect is due to the treatment.
- These results apply to the people the trial enrolled: Relapsed/refractory high-risk neuroblastoma: third-generation GD2 CAR-T with inducible caspase-9 safety switch. People in a different situation may not see the same effect.
- The trial selected people by a biomarker (GD2); the result should not be assumed for people whose cancer does not have it.
- Only 27 people took part, so the numbers are less certain than in a large trial.
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.
27 participants enrolled.
Overall survival at 3 years
GD2-CART0160 of 100
| Endpoint | Arm | n | Value | HR (95% CI) | p | Source |
|---|---|---|---|---|---|---|
| Objective response rateprimary | GD2-CART01 | 27 | 63% | — | — | link |
| 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.