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儿童神经肿瘤学免疫治疗临床试验的转化考虑

英文原题:Translational considerations for immunotherapy clinical trials in pediatric neuro-oncology.

PubMed 2023/05/25(内容时间) Neoplasia Q2 · IF 4.8(JCR 2025)

研究概要

尽管近几十年来儿童肿瘤免疫治疗取得了巨大进展,包括FDA批准了dinutuximab和tisgenlecleucel等药物,但这些成功很少惠及儿童中枢神经系统(CNS)肿瘤患者。

中文摘要

近几十年来,儿童癌症免疫治疗取得了长足进展,包括dinutuximab和tisgenlecleucel等药物获美国FDA批准,但这些成功很少惠及儿童中枢神经系统(CNS)肿瘤患者。随着对这些肿瘤生物学基础的认识不断发展,专为儿童CNS肿瘤设计的新型免疫疗法正迅速进入临床转化阶段。最近,溶瘤病毒、疫苗、过继性细胞治疗和免疫检查点抑制取得了一些显著临床成功。本文中,太平洋儿童神经肿瘤联盟(PNOC)免疫治疗工作组回顾了CNS免疫治疗临床试验的现状和未来,重点关注临床试验开发。基于近期治疗试验,作者讨论了免疫治疗临床试验的独特挑战,包括毒性考量、疾病评估和相关性研究,并探讨联合策略和未来方向。通过国际合作和联盟协作,作者希望推动这一前景广阔的免疫肿瘤学领域迈向成功应用于儿童CNS肿瘤治疗的新阶段。

展开英文摘要原文

While immunotherapy for pediatric cancer has made great strides in recent decades, including the FDA approval of agents such as dinutuximab and tisgenlecleucel, these successes have rarely impacted children with pediatric central nervous system (CNS) tumors. As our understanding of the biological underpinnings of these tumors evolves, new immunotherapeutics are undergoing rapid clinical translation specifically designed for children with CNS tumors. Most recently, there have been notable clinical successes with oncolytic viruses, vaccines, adoptive cellular therapy, and immune checkpoint inhibition. In this article, the immunotherapy working group of the Pacific Pediatric Neuro-Oncology Consortium (PNOC) reviews the current and future state of immunotherapeutic CNS clinical trials with a focus on clinical trial development. Based on recent therapeutic trials, we discuss unique immunotherapy clinical trial challenges, including toxicity considerations, disease assessment, and correlative studies. Combinatorial strategies and future directions will be addressed. Through internationally collaborative efforts and consortia, we aim to direct this promising field of immuno-oncology to the next frontier of successful application against pediatric CNS tumors.

论文信息

作者
Foster JB、Alonso MM、Sayour E、Davidson TB、Persson ML、Dun MD、Kline C、Mueller S
单位
Division of Oncology, Children's Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA USA. Electronic address: fosterjb@chop.edu.United States
期刊
Neoplasia (New York, N.Y.)2023 Aug
原文标识
PubMed 37244226 · DOI 10.1016/j.neo.2023.100909