肿瘤细胞治疗研究
英文原题：The Road to CAR T-Cell Therapies for Pediatric CNS Tumors: Obstacles and New Avenues.
儿童中枢神经系统（CNS）肿瘤是儿童中最常见的实体瘤，也是儿童癌症相关死亡的主要原因。
儿童中枢神经系统（CNS）肿瘤是儿童中最常见的实体肿瘤，也是儿童癌症相关死亡的主要原因。那些幸存下来的患儿面临着当前标准治疗方案——化疗、放疗和手术——所带来的长期不良反应。迫切需要新的治疗策略，以更有效地治疗儿童CNS肿瘤，同时降低毒性——其中一种新方法就是嵌合抗原受体（CAR）T细胞疗法。目前已获批用于多种血液系统恶性肿瘤，且有前景的临床前和早期临床数据表明，CAR-T细胞可能改变儿童CNS肿瘤的治疗格局。然而，要开发出安全有效的针对CNS肿瘤的CAR T细胞疗法，必须克服若干挑战。在此，我们详细阐述这些挑战，重点关注儿童患者特有的问题，包括抗原选择、肿瘤免疫原性和毒性。我们还讨论了我们对CAR T细胞疗法未来方向及潜在联合治疗方法的看法。
Pediatric central nervous system (CNS) tumors are the most common solid tumors diagnosed in children and are the leading cause of pediatric cancer-related death. Those who do survive are faced with the long-term adverse effects of the current standard of care treatments of chemotherapy, radiation, and surgery. There is a pressing need for novel therapeutic strategies to treat pediatric CNS tumors more effectively while reducing toxicity - one of these novel modalities is chimeric antigen receptor (CAR) T-cell therapy. Currently approved for use in several hematological malignancies, there are promising pre-clinical and early clinical data that suggest CAR-T cells could transform the treatment of pediatric CNS tumors. There are, however, several challenges that must be overcome to develop safe and effective CAR T-cell therapies for CNS tumors. Herein, we detail these challenges, focusing on those unique to pediatric patients including antigen selection, tumor immunogenicity and toxicity. We also discuss our perspective on future avenues for CAR T-cell therapies and potential combinatorial treatment approaches.
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