Timpanaro Andrea, Song Edward Z, Amwas Nour, Chiu Chu-Hsuan, Ronsley Rebecca, Taylor Mallory R, Foster Jessica B, Wang Leo D, Vitanza Nicholas A
Ben Towne Center for Childhood Cancer and Blood Disorders Research, Seattle Children's Research Institute, Seattle, Washington.
Department of Immuno-oncology, Beckman Research Institute, City of Hope National Medical Center, Duarte, California.
Cancer Discov. 2025 May 2;15(5):890-902. doi: 10.1158/2159-8290.CD-24-1465.
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.
中枢神经系统肿瘤是儿童癌症相关死亡的主要原因，这凸显了对新治疗策略的迫切需求。嵌合抗原受体（CAR）T细胞代表了一种独特的方法，不同于数十年来主导临床试验领域的细胞毒性化疗和小分子抑制剂。I期CAR T细胞试验已显示出针对小儿中枢神经系统肿瘤的可行性和可能的疗效；然而，如果这些疗法要使大多数受影响的儿童受益，还必须克服许多挑战。尽管快速的转化研究和早期试验迅速增进了我们的理解，但小儿中枢神经系统CAR T细胞领域现在渴望进行批判性评估并就克服未来的剩余障碍展开开放对话。
