GD2 CAR-T as consolidation in high-risk neuroblastoma

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.