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CAR-T 细胞疗法的演进:克服儿童中枢神经系统肿瘤治疗障碍

英文原题:Evolving CAR T-Cell Therapy to Overcome the Barriers in Treating Pediatric Central Nervous System Tumors.

PubMed 2025/05/02(内容时间) Cancer Discov Q1 · IF 29.5(JCR 2025)

研究概要

CNS 肿瘤是儿童癌症相关死亡的首要原因,凸显了对新治疗策略的迫切需求。

中文摘要

中枢神经系统(CNS)肿瘤是儿童癌症相关死亡的首要原因,凸显了开发新治疗策略的迫切需求。CAR-T细胞提供了一种独特治疗方式,有别于数十年来主导临床试验的细胞毒性化疗和小分子抑制剂。1期CAR-T细胞试验已显示治疗儿童CNS肿瘤的可行性和潜在疗效;但若要使多数患病儿童获益,仍需克服诸多挑战。快速的转化研究和早期临床试验迅速加深了我们的认识,而目前儿童CNS CAR-T领域亟需深入评估,并开放讨论如何解决仍存障碍。

展开英文摘要原文

CNS tumors are the leading cause of cancer-related death in children, highlighting the dire need for new treatment strategies. CAR T cells represent a unique approach, distinct from the cytotoxic chemotherapies and small-molecule inhibitors that have dominated the clinical trial space for decades. Phase I CAR T-cell trials have shown feasibility and possible efficacy against pediatric CNS tumors; however, many challenges must be overcome if these therapeutics are going to be beneficial to most affected children. Although rapid translational development and early-phase trials have quickly evolved our understanding, the pediatric CNS CAR T-cell community now yearns for critical assessments and open dialogue about overcoming the remaining obstacles ahead.

论文信息

作者
Timpanaro A、Song EZ、Amwas N、Chiu CH、Ronsley R、Taylor MR、Foster JB、Wang LD
单位
Ben Towne Center for Childhood Cancer and Blood Disorders Research, Seattle Children's Research Institute, Seattle, Washington.United States
文献类型
综述
期刊
Cancer discovery2025 May 2
原文标识
PubMed 40300089 · DOI 10.1158/2159-8290.CD-24-1465