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儿童脑瘤网络(CBTN)——通过协作和开放科学加速小儿中枢神经系统肿瘤的研究。

The children's brain tumor network (CBTN) - Accelerating research in pediatric central nervous system tumors through collaboration and open science.

机构信息

Children's Hospital of Philadelphia, Philadelphia, PA, USA.

Sydney Children's Hospital, Sydney, Australia.

出版信息

Neoplasia. 2023 Jan;35:100846. doi: 10.1016/j.neo.2022.100846. Epub 2022 Nov 3.

DOI:10.1016/j.neo.2022.100846
PMID:36335802
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC9641002/
Abstract

Pediatric brain tumors are the leading cause of cancer-related death in children in the United States and contribute a disproportionate number of potential years of life lost compared to adult cancers. Moreover, survivors frequently suffer long-term side effects, including secondary cancers. The Children's Brain Tumor Network (CBTN) is a multi-institutional international clinical research consortium created to advance therapeutic development through the collection and rapid distribution of biospecimens and data via open-science research platforms for real-time access and use by the global research community. The CBTN's 32 member institutions utilize a shared regulatory governance architecture at the Children's Hospital of Philadelphia to accelerate and maximize the use of biospecimens and data. As of August 2022, CBTN has enrolled over 4700 subjects, over 1500 parents, and collected over 65,000 biospecimen aliquots for research. Additionally, over 80 preclinical models have been developed from collected tumors. Multi-omic data for over 1000 tumors and germline material are currently available with data generation for > 5000 samples underway. To our knowledge, CBTN provides the largest open-access pediatric brain tumor multi-omic dataset annotated with longitudinal clinical and outcome data, imaging, associated biospecimens, child-parent genomic pedigrees, and in vivo and in vitro preclinical models. Empowered by NIH-supported platforms such as the Kids First Data Resource and the Childhood Cancer Data Initiative, the CBTN continues to expand the resources needed for scientists to accelerate translational impact for improved outcomes and quality of life for children with brain and spinal cord tumors.

摘要

小儿脑肿瘤是美国儿童癌症相关死亡的主要原因,与成人癌症相比,其潜在寿命损失的比例过高。此外,幸存者经常遭受长期的副作用,包括继发性癌症。儿童脑瘤网络(CBTN)是一个多机构的国际临床研究联盟,旨在通过收集和快速分发生物样本和数据,通过开放科学研究平台,实时提供给全球研究界使用,从而推进治疗方法的发展。CBTN 的 32 个成员机构利用费城儿童医院的共享监管治理架构,加速和最大化生物样本和数据的使用。截至 2022 年 8 月,CBTN 已经招募了超过 4700 名受试者、1500 多名家长,并收集了超过 65000 份生物样本用于研究。此外,还从收集的肿瘤中开发了 80 多个临床前模型。目前已有超过 1000 个肿瘤和种系材料的多组学数据,正在生成 >5000 个样本的数据。据我们所知,CBTN 提供了最大的开放获取小儿脑肿瘤多组学数据集,其中包含纵向临床和结果数据、成像、相关生物样本、儿童-家长基因组系谱以及体内和体外临床前模型。在 NIH 支持的平台(如 Kids First Data Resource 和 Childhood Cancer Data Initiative)的支持下,CBTN 继续扩大资源,为科学家提供所需的资源,以加速转化影响,改善患有脑和脊髓肿瘤的儿童的结果和生活质量。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1dfa/9641002/ee489fd56df7/gr1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1dfa/9641002/ee489fd56df7/gr1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1dfa/9641002/ee489fd56df7/gr1.jpg

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Upfront Biology-Guided Therapy in Diffuse Intrinsic Pontine Glioma: Therapeutic, Molecular, and Biomarker Outcomes from PNOC003. upfront 生物学指导弥漫性内在脑桥胶质瘤的治疗:来自 PNOC003 的治疗、分子和生物标志物结果。
Clin Cancer Res. 2022 Sep 15;28(18):3965-3978. doi: 10.1158/1078-0432.CCR-22-0803.
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Novel mouse model reveals neurodevelopmental origin of PMM2-CDG brain pathology.新型小鼠模型揭示了PMM2-CDG脑病理的神经发育起源。
bioRxiv. 2025 Jun 3:2025.06.01.657261. doi: 10.1101/2025.06.01.657261.
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Global DNA methylation differences involving germline structural variation impact gene expression in pediatric brain tumors.涉及种系结构变异的全基因组DNA甲基化差异影响小儿脑肿瘤中的基因表达。
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