THURSDAY, April 6, 2023 (HealthDay News) — Chimeric antigen receptor (CAR)-expressing T cells that target the disialoganglioside GD2 expressed on tumor cells (GD2-CAR T cells) are feasible and safe for children with heavily pretreated neuroblastoma, according to a study published in the April 6 issue of the New England Journal of Medicine.

Francesca Del Bufalo, M.D., from IRCCS Ospedale Pediatrico Bambino Gesù in Rome, and colleagues conducted a phase 1/2 clinical trial involving 27 children with heavily pretreated neuroblastoma (12 with refractory disease, 14 with relapsed disease, and one with a complete response at the end of first-line therapy) who received GD2-CAR T cells expressing the inducible caspase 9 suicide gene (GD2-CART01).

The researchers observed no failure to generate GD2-CART01. In the phase 1 part of the trial, three dose levels were tested (3-, 6-, and 10×10⁶ CAR-positive T cells/kg body weight), with no dose-limiting toxic effects reported. For the phase 2 part of the trial, the recommended dose was 10×10⁶ CAR-positive T cells/kg. In 74 percent of patients, cytokine release syndrome occurred and was mild in all but one patient. The suicide gene was activated in one patient, with rapid elimination of GD2-CART01. GD2-targeted CAR T cells expanded in vivo; up to 30 months after infusion, they were detectable in peripheral blood in 26 of 27 patients. Overall response was 63 percent, with nine and eight patients having a complete and partial response, respectively. Three-year overall survival and event-free survival were 60 and 36 percent, respectively, among children receiving the recommended dose.

“GD2-CART01 may induce sustained eradication of disease in a proportion of patients with relapsed or refractory neuroblastoma,” the authors write.

Bellicum Pharmaceuticals donated rimiducid for the trial.

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