4SCAR-GD2-modified T-cell therapy in neuroblastoma with MYCN amplification: A case report with over 4-year follow-up data

Xiao Xu; Wen Zhao; Zhixia Yue; Maoquan Qin; Mei Jin; Lung-Ji Chang; Xiaoli Ma

doi:10.1002/ped4.12181

4SCAR-GD2-modified T-cell therapy in neuroblastoma with MYCN amplification: A case report with over 4-year follow-up data

Pediatr Investig. 2020 Mar 17;4(1):55-58. doi: 10.1002/ped4.12181. eCollection 2020 Mar.

Authors

Xiao Xu¹, Wen Zhao¹, Zhixia Yue¹, Maoquan Qin¹, Mei Jin¹, Lung-Ji Chang², Xiaoli Ma¹

Affiliations

¹ Beijing Key Laboratory of Pediatric Hematology Oncology; National Discipline of Pediatrics, Ministry of Education; MOE Key Laboratory of Major Diseases in Children; Hematology Oncology Center, Beijing Children's Hospital Capital Medical University, National Center for Children's Health Beijing 100045 China.
² Shenzhen Geno-immune Medical Institute and America Yuva Biomed Inc Shenzhen China.

Abstract

Introduction: Neuroblastoma (NB) is the most common extracranial solid tumor among children. The 5-year event-free survival rate for high-risk (HR) NB is still poor, especially for patients with advanced NB with MYCN gene amplification. Chimeric antigen receptor T (CAR-T) cell therapy is a new treatment for HR-NB.

Case presentation: A 55-month-old boy with stage IV HR-NB received 4th-generation CAR-T cells that target disialoganglioside GD2, as consolidation maintenance treatment after intensive chemotherapy, surgery, and autologous stem-cell transplantation. As of February 2019, his CAR-T follow-up time was 37.5 months, indicating prolonged survival. Cranial MRI and ultrasound showed no mass; ¹²³I-metaiodobenzylguanidine (¹²³I-MIBG) scan was negative.

Conclusion: GD2-CAR-T cells may be an effective treatment option for NB patients with MYCN amplification.

Keywords: Bone marrow; CAR‐T; Encephalic metastasis; GD2; Long term survival; MYCN amplification; Metastasis; Neuroblastoma.

Publication types

Case Reports