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GD2-CART01 effective for R/R high-risk neuroblastoma 

GD2-CART01 has been found in a phase 1-2 clinical trial to be both safe and effective for children with heavily pretreated neuroblastoma.

GD2-CART01 is a safe and feasible therapy for children with relapsed or refractory (R/R) high-risk neuroblastoma according to a phase 1 – 2 trial by Italian researchers.

Neuroblastoma is responsible for 11% of paediatric cancer deaths and these cancer cells express high levels of disialoganglioside GD2.

Moreover, targeting this protein, while a valid and safe strategy, requires further modification to promote CAR-T cell longevity.

In the current trial, researchers used a third-generation CAR-T cell therapy in patients with R/R high-risk neuroblastoma. The therapy included the inducible caspase 9 suicide gene, designed to kill cells in the presence of dangerous toxic effects.

GD2-CART01 outcomes

The trial enrolled 27 children with heavily pretreated neuroblastoma.

The overall response rate was 63%; 9 children had a complete response and 8 a partial response. The three-year overall survival and event-free survival were 60% and 36%, respectively.

Overall, after infusion 33% of patients (eight patients) had a complete response or maintained a complete response (one patient).

Cytokine release syndrome occurred in 74% of patients but was of mild severity in the majority (95%). The suicide gene activation occurred in only one patient. GD2-targeted CAR T cells were detectable in peripheral blood in 26 of 27 patients up to 30 months after infusion.

The authors concluded that GD2-CART01 may induce sustained eradication of disease in a proportion of patients with R/R neuroblastoma.

Del Bufalo F et al. GD2-CART01 for Relapsed or Refractory High-Risk Neuroblastoma. N Eng J Med 2023.