Theeler Brett J, Dalal Yamini, Monje Michelle, Shilatifard Ali, Suvà Mario L, Aboud Orwa, Camphausen Kevin, Cordova Christine, Finch Elizabeth, Heiss John D, Packer Roger J, Romo Carlos G, Aldape Kenneth, Penas-Prado Marta, Armstrong Terri, Gilbert Mark R
Department of Neurology and John P. Murtha Cancer Center, Walter Reed National Military Medical Center, Bethesda, Maryland, USA.
Neuro-Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, Maryland, USA.
Neurooncol Adv. 2020 Jan 16;2(1):vdaa007. doi: 10.1093/noajnl/vdaa007. eCollection 2020 Jan-Dec.
Histone mutations occur in approximately 4% of different cancer types. In 2012, mutations were found in the gene encoding histone variant H3.3 ( gene) in pediatric diffuse intrinsic pontine gliomas and pediatric hemispheric gliomas. Tumors with mutations in the gene are generally characterized as histone mutated gliomas (HMGs) or diffuse midline gliomas. HMGs are a rare subtype of glial tumor that is malignant and fast growing, carrying a poor prognosis. In 2017, the Beau Biden Cancer Moonshot Program appropriated $1.7 billion toward cancer care in 10 select areas. The National Cancer Institute (NCI) was granted support to focus specifically on rare central nervous system (CNS) tumors through NCI-CONNECT. Its mission is to address the challenges and unmet needs in CNS cancer research and treatment by connecting patients, providers, researchers, and advocacy organizations to work in partnership. On September 27, 2018, NCI-CONNECT convened a workshop on histone mutated midline glioma, one of the 12 CNS cancers included in its initial portfolio. Three leaders in the field provided an overview of advances in histone mutated midline glioma research. These experts shared observations and experiences related to common scientific and clinical challenges in studying these tumors. Although the clinical focus of this workshop was on adult patients, one important objective was to start a collaborative dialogue between pediatric and adult clinicians and researchers. Meeting participants identified needs for diagnostic and treatment standards, disease biology and biological targets for this cancer, disease-specific trial designs, and developed a list of action items and future direction.
组蛋白突变发生在约4%的不同癌症类型中。2012年，在小儿弥漫性脑桥内在胶质瘤和小儿半球胶质瘤中发现了编码组蛋白变体H3.3（基因）的基因发生突变。该基因发生突变的肿瘤通常被归类为组蛋白突变型胶质瘤（HMGs）或弥漫性中线胶质瘤。HMGs是一种罕见的胶质肿瘤亚型，具有恶性、生长迅速、预后不良的特点。2017年，博·拜登癌症“登月计划”拨款17亿美元用于10个特定领域的癌症治疗。美国国立癌症研究所（NCI）获得支持，通过NCI-CONNECT专门关注罕见的中枢神经系统（CNS）肿瘤。其使命是通过将患者、医疗服务提供者、研究人员和倡导组织联系起来合作，应对CNS癌症研究和治疗中的挑战及未满足的需求。2018年9月27日，NCI-CONNECT召开了一次关于组蛋白突变型中线胶质瘤的研讨会，这是其初始项目组合中包含的12种CNS癌症之一。该领域的三位领军人物概述了组蛋白突变型中线胶质瘤研究的进展。这些专家分享了与研究这些肿瘤时常见的科学和临床挑战相关的观察结果和经验。尽管本次研讨会的临床重点是成年患者，但一个重要目标是启动儿科和成年临床医生及研究人员之间的合作对话。与会者确定了对诊断和治疗标准、该癌症的疾病生物学和生物学靶点、针对该疾病的试验设计的需求，并制定了一份行动项目清单和未来方向。
