(HealthDay News) — Chimeric antigen receptor (CAR) T-cell therapy targeting GD2 appears safe and active in children with heavily pretreated neuroblastoma, according to a study published in The New England Journal of Medicine.
Researchers conducted a phase 1/2 trial to test GD2-CAR T cells expressing the inducible caspase 9 suicide gene (GD2-CART01) in children with heavily pretreated neuroblastoma. The trial included 27 patients — 12 who had refractory disease, 14 who had relapsed disease, and 1 who had a complete response at the end of first-line therapy.
In the phase 1 part of the trial, patients received GD2-CART01 at 1 of 3 dose levels — 3×106, 6×106, or 10×106 CAR T cells/kg body weight. No dose-limiting toxicities were reported. For the phase 2 part of the trial, the recommended dose was 10×106 CAR T cells/kg.
The CAR T cells expanded in vivo. They were detectable in the peripheral blood in 26 of 27 patients up to 30 months after infusion.
The overall response rate was 63%. Nine patients had a complete response, and 8 patients had a partial response. Among patients who received the recommended dose, the 3-year overall survival rate was 60% and the 3-year event-free survival rate was 36%.
Cytokine release syndrome occurred in 74% of patients. It was mild in all but 1 patient. The suicide gene was activated in 1 patient, and this resulted in rapid elimination of GD2-CART01.
“GD2-CART01 may induce sustained eradication of disease in a proportion of patients with relapsed or refractory neuroblastoma,” the study authors wrote.
Bellicum Pharmaceuticals donated rimiducid for the trial.