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。这项工作利用了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。计划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。已经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，目的是促进对肿瘤生物学和发病机制的了解，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记。此外，已经开发了特定的测试能力，如上文描述的CNS分子面板，其他测试正在开发中。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。最近开放了四项临床试验(17C0102免疫检查点抑制剂Nivolumab用于罕见的中枢神经系统癌症患者；Sunitinib用于肉瘤和胶质肉瘤；Marizomib用于复发融合的室管膜瘤；Nivolumab用于高突变的低级别胶质瘤，包括少突胶质瘤。

The NIH is uniquely suited to create a rare CNS cancer initiative. This effort utilizes 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 are planned. A tissue repository has been established 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 have been developed such as the CNS Molecular Panel described above and additional tests are under development. 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. Four clinical trials are recently open (17C0102 Immune Checkpoint Inhibitor Nivolumab in People With Select Rare CNS Cancers; Sunitinib for Sarcoma and Gliosarcoma; Marizomib for RELA-fused ependymoma; and Nivolumab for Hypermutated Lower Grade Glioma including oligodendroglioma.