我国嵌合抗原受体T细胞产品治疗儿童急性淋巴细胞白血病的申报现状和临床审评考虑

Current Status and Clinical Review Considerations of Chimeric Antigen Receptor T-cell Therapy Products Applying for Pediatric Acute Lymphoblastic Leukemia in China

近年来,随着肿瘤免疫治疗和基因修饰技术的进展,嵌合抗原受体T细胞(CAR-T)成为药品研发热点。以CD19为靶点的CAR-T药物对儿童急性淋巴细胞白血病(acute lymphoblastic leukemia,ALL),尤其是化疗和造血干细胞移植等常规方法疗效较差的复发难治性ALL的治疗效果显著。目前已有多款CAR-T产品获得药品监管机构的批准上市,但我国尚未有针对儿童ALL的CAR-T产品上市。该研究主要介绍了目前获我国许可开展药物临床试验的CAR-T产品的情况,探讨了针对儿童急性B细胞淋巴细胞白血病突出的临床需求开展药物临床研发的关键要素,为同类产品的注册和临床试验设计提供参考。

In recent years,with the progress of tumor immunotherapy and gene modification technology,chimeric antigen receptor T cells(CAR-T)has become a hot topic in drug research and development.CAR-T drugs targeting CD19 have significant therapeutic effects on acute lymphoblastic leukemia(ALL)in children,especially relapsed/refractory ALL with poor efficacy of conventional methods such as chemotherapy and hematopoietic stem cell transplantation.Several CAR-T products have been approved by drug regulatory administration for marketing,but no CAR-T products for children with ALL have been approved in China.This study mainly introduces the current situation of CAR-T products licensed to carry out drug clinical trials in China.Then the clinical needs and the critical elements of drug clinical research and development for acute B-cell lymphocytic leukemia in children is discussed,to provide reference for the design of registered clinical trials of similar products.