Rare Central Nervous System Cancers Initiative

NIH是唯一适合创建一个罕见的中枢神经系统癌症倡议。这项工作利用了NIH的以下具体资源。这需要开发临床试验基础设施，包括数据管理系统，以监督疾病特异性治疗和自然史研究。计划与成像技术专家合作，创建成像生物标志物和常规成像，以监测对治疗的反应。已经建立了一个组织库，用于存储肿瘤和正常组织，以进行分子分析，包括基因组学、代谢组学和蛋白质组学，目的是促进对肿瘤生物学和发病机制的理解，从而获得更好的预后信息、子分类和最终预测对特定治疗反应的标志物。此外，已经开发了特定的检测能力，如上述CNS分子组，其他检测正在开发中。例如，预计将进行免疫治疗试验，免疫监测能力将作为本研究的关键组成部分。基于NIH的外展工作将用于与患者倡导团体互动，以提高患者对这些工作的参与，并为教育计划提供场所，以提高患者和护理人员对其罕见疾病的了解。最近有四项临床试验（17 C 0102免疫检查点抑制剂Nivolumab在患有特定罕见CNS癌症的人中的应用; 19-C-0011 Marizlavine用于复发性低级别和间变性幕上、幕下和脊髓室管膜瘤的II期临床试验; 18-c-0137舒尼替尼在中枢神经系统肉瘤中的II期试验，19-C-0006纳武单抗在具有和不具有超变子表型的IDH突变型胶质瘤患者中评价的II期试验）。最近，我们建立了NCI-RECT：评估罕见CNS肿瘤的综合肿瘤学网络，以促进患者-倡导者-提供者的伙伴关系和网络，以改善护理和治疗方法。

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; 19-C-0011 Phase II Clinical Trial of Marizomib for Recurrent Low-Grade and Anaplastic Supratentorial, Infratentorial and Spinal Cord Ependymoma; 18-c-0137 Phase II Trial of Sunitinib in Sarcomas of the Central Nervous System, 19-C-0006 Phase II Trial Evaluating Nivolumab in Patients with IDH-Mutant Gliomas with and without Hypermutator Phenotype). Most recently, we have established the NCI-CONNECT: Comprehensive Oncology Network Evaluating Rare CNS Tumors for fostering patient-advocacy-provider partnerships and networks to improve approaches to care and treatment.