Emerging treatments

131-iodine-metaiodobenzylguanidine (MIBG) as induction therapy

Clinical trials and pilot studies continue to assess the feasibility and tolerability of incorporating MIBG therapy in induction regimens for patients with high-risk neuroblastoma.[115][116]

Lorlatinib

One phase 3 study to determine the effect of the addition of lorlatinib, an ALK/ROS1 inhibitor, to intensive multimodality therapy in patients with high-risk ALK-driven neuroblastoma is active.​[117] Interim results from an ongoing phase 1 study suggest that lorlatinib, given alone or in combination with chemotherapy, is safe and tolerable in pediatric, adolescent, and adult patients with relapsed or refractory ALK-mutated or ALK-amplified neuroblastoma.[118]​​

Adavosertib

An investigational inhibitor of WEE1 tyrosine kinase, adavosertib (plus irinotecan) met the protocol-defined efficacy end point (3 objective responses from a cohort of 20 patients) in a phase 2 trial of pediatric patients with recurrent neuroblastoma.[119]

RIST protocol

In a multicenter, open-label, controlled, phase 2 trial, 129 patients ages 1-25 years with high-risk relapsed or refractory neuroblastoma were randomly assigned to the RIST protocol (comprising metronomic irinotecan plus temozolomide and dasatinib plus rapamycin), or to irinotecan plus temozolomide (control group).[120]​ At median follow-up of 72 months, median progression-free survival was 11 months in the RIST group and 5 months in the control group.

Chimeric antigen receptor (CAR) T-cell therapy

Clinical trials and studies suggest that disialoganglioside 2 (GD2)-specific CAR T-cell therapy is feasible and safe in patients with high-risk or relapsed/refractory neuroblastoma.[23][121][122]​​​[123]​ Grade 3/4 hematological toxicity has been reported.[23][123]​ Phase 2 trials are ongoing.[124]

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