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A phase 1-2 clinical trial was conducted to test the safety and efficacy of GD2-CAR T cells expressing a suicide gene in treating high-risk neuroblastoma. The trial enrolled 27 children with relapsed or refractory disease who received GD2-CART01, with no failure to generate the CAR T cells. Cytokine release syndrome occurred in 74% of patients but was mostly mild, and the suicide gene was activated in one patient, leading to the elimination of GD2-CART01. GD2-targeted CAR T cells were detectable in peripheral blood in most patients up to 30 months after infusion. The overall response rate was 63%, and the recommended dose for phase 2 was 10×106 CAR-positive T cells per kilogram. The use of GD2-CART01 was feasible and safe, and it may have a sustained antitumor effect.
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