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免疫细胞治疗有望更有效治愈髓母细胞瘤

英文原题:Immune Cell Therapy Promises More Effective Cure for Medulloblastoma.

PubMed 2026/06/18(内容时间) J Pers Med

研究概要

髓母细胞瘤是最常见的儿童脑肿瘤之一。

中文摘要

髓母细胞瘤是最常见的儿童脑肿瘤之一。目前针对这一严重癌症的治疗只能延长生存,但副作用严重且疾病易复发。近三分之一患者无法通过现有治疗治愈,多数生存者生活质量较差。将免疫检查点抑制剂(ICI)加入免疫治疗,为患者带来希望。尽管ICI为免疫治疗提供重要助力,但如何将免疫检查点抑制纳入现有治疗策略以治愈髓母细胞瘤,仍研究不足。然而,识别髓母细胞瘤主要分子亚组被认为是肿瘤学的一项成功。分子分型进展为按亚组设计临床试验铺平道路,可能带来有效的免疫靶向疗法。但这一相对较新的进展仍受疾病显著生物学异质性,以及对其耐受现有治疗机制缺乏全面认识所阻碍。将嵌合抗原受体(CAR)T细胞及CAR-NK细胞疗法纳入多种治疗策略和在研临床试验,为这一致命疾病患者带来新的希望。然而,在研试验提示,这一极具前景的疗法仍可能受到多项严重局限影响,包括细胞因子释放综合征、移植物抗宿主病、靶抗原稀少及严重不良事件。部分临床试验还提示,CAR-NK可能较少受到其中一些局限的影响。本综述也强调质谱蛋白质组学的贡献,以及液体活检相较组织活检日益增加的作用。

展开英文摘要原文

Medulloblastoma is one of the most prevalent pediatric brain tumors. Currently, existing therapies for this devastating type of cancer can only prolong survival time with severe side-effects and relapse. These therapies are not curative for almost a third of treated patients, while most survivors are condemned to a poor quality of life. The addition of immune checkpoint inhibitors (ICIs) to immune therapy has given some hope to those suffering from this type of cancer. Although ICIs provide a valuable contribution to immunotherapy, the exploitation of immune checkpoint inhibition within existing therapeutic strategies to cure Medulloblastoma remains understudied. However, the identification of the main molecular subgroups of medulloblastoma is considered one of the success stories of oncology. This advancement in molecular profiling of MB paved the way to subgroup-directed clinical trials, which may lead to efficacious immune-targeted therapy. However, this relatively new development is still hampered by a substantial biological heterogeneity of the disease and the absence of a full understanding of the various mechanisms behind its resistance to existing therapeutic modalities. The inclusion of chimeric antigen receptor (CAR) T and CAR NK cell therapy within various therapeutic strategies and ongoing clinical trials has given fresh hope those suffering from this fatal disease. However, ongoing clinical trials suggest that this highly promising therapy can be impaired by a number of serious limitations, including cytokine release syndrome, Graft-versus-host disease, the scarcity of target antigens, and severe adverse events. Some of the ongoing clinical trials also suggest that CAR NK is less prone to some of these limitations. This review also highlights the contribution of mass spectrometry-based proteomics, and the increasing role of liquid biopsy rather than tissue biopsy.

论文信息

作者
Agostini M、Traldi P、Hamdan M
单位
Corso Stati Uniti 4, Istituto di Ricerca Pediatrica Città della Speranza, 35100 Padova, Italy.Italy
文献类型
综述
期刊
Journal of personalized medicine2026 Jun 18
原文标识
PubMed 42346637 · DOI 10.3390/jpm16060326