Rare Central Nervous System Cancers Initiative

NIH是唯一适合创建一项罕见的中枢神经系统癌症倡议的机构。这项工作将利用美国国立卫生研究院的以下具体资源。这需要开发一个临床试验基础设施，包括数据管理系统，以监督疾病特定的治疗和自然病史研究。与成像技术专家合作，创建成像生物标记物和常规成像，以监测治疗反应。这一倡议将结合儿科和成人调查人员的专业知识，受益于这些调查人员的各种方法。为了促进对肿瘤生物学和发病机制的了解，建立了一个组织信息库，用于存储肿瘤和正常组织，用于包括基因组学、代谢组学和蛋白质组学在内的分子分析，从而获得更好的预后信息、分类和最终预测特定治疗反应的标志物。此外，还将根据项目需要建立具体的测试能力。例如，预计将进行一项免疫治疗试验，并将需要免疫监测能力作为这项研究的关键组成部分。将利用NIH的外展努力与患者倡导团体互动，以加强患者对这些努力的参与，并为教育计划提供场所，以提高患者和照顾者对他们罕见疾病的了解。

The NIH is uniquely suited to create a rare CNS cancer initiative. This effort will utilize the following specific resources of the NIH. This has required developing a clinical trial infrastructure, including data management system, to oversee the disease specific therapeutic and natural history studies. Collaborations with experts in imaging technologies to create imaging biomarkers and routine imaging for monitoring response to therapies. This initiative will combine the expertise from both pediatric and adult investigators, benefitting from the varied approaches from these investigators. A tissue repository has been developed to store both tumor and normal tissue for molecular analyses including genomics, metabolomics, and proteomics with the intent of advancing the understanding of tumor biology and pathogenesis leading to better prognostic information, sub-classification and ultimately markers predictive of response to specific treatments. Additionally, specific testing capabilities will be established based on project need. For example, an immunotherapy trial is anticipated and immunologic monitoring capabilities will be needed as a critical component of this study. NIH-based outreach efforts will be utilized to interact with patient advocacy groups to enhance patient participation in these efforts and provide a venue for educational programs to improve patient and caregiver understanding of their uncommon disease.