Rare Central Nervous System Cancers Initiative

罕见中枢神经系统癌症倡议

• 批准号: 9344012
• 负责人: Mark Gilbert
• 金额: $ 39.33万
• 依托单位: DIVISION OF BASIC SCIENCES - NCI
• 依托单位国家: 美国
• 资助国家: 美国
• 起止时间: 至
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/9344012
• 关键词: Adult; Brain Stem Glioma; Brain Stem Neoplasms; Caregivers; Central Nervous System Neoplasms; Childhood; Chordoma; Choroid Plexus Carcinoma; Classification; Clinical Trials; Collaborations; Consensus; Diffuse intrinsic pontine glioma; Disease; Ependymoma; Evaluation; Funding; Genomic DNA; Genomics; Glioblastoma; Glioma; Gliomatosis cerebri; Image; Imaging technology; Immunologic Monitoring; Immunotherapy; Incidence; International; Investigation; Knowledge; Laboratories; Malignant - descriptor; Malignant Neoplasms; Malignant neoplasm of central nervous system; Molecular; Monitor; Monograph; Mutation; National Cancer Institute; Natural History; Neoplasms; Neuraxis; Normal tissue morphology; Oligodendroglioma-Astrocytoma; Pathogenesis; Patient Participation; Patients; Primary Brain Neoplasms; Primary Neoplasm; Proteomics; Publishing; Qualifying; Rare Diseases; Regimen; Research; Research Infrastructure; Research Personnel; Resources; Rhabdoid Tumor; System; Testing; Therapeutic; Tissues; Tumor Biology; Tumor Tissue; United States; United States National Institutes of Health; base; clinical infrastructure; data management; imaging biomarker; improved; medulloblastoma; metabolomics; outreach; patient advocacy group; pineoblastoma; predicting response; prognostic; programs; repository; response; tumor

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.

美国国立卫生研究院非常适合开展罕见的中枢神经系统癌症计划。这项工作将利用 NIH 的以下特定资源。这需要开发临床试验基础设施，包括数据管理系统，以监督疾病特定的治疗和自然历史研究。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治疗反应。该计划将结合儿科和成人研究人员的专业知识，并受益于这些研究人员的不同方法。已经开发出一个组织存储库来存储肿瘤和正常组织，以进行包括基因组学、代谢组学和蛋白质组学在内的分子分析，目的是增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、子分类和最终预测特定治疗反应的标记物。此外，将根据项目需要建立特定的测试能力。例如，预计将进行免疫治疗试验，并且需要免疫监测能力作为本研究的关键组成部分。 NIH 的外展工作将用于与患者倡导团体互动，以加强患者对这些工作的参与，并为教育项目提供场所，以提高患者和护理人员对其罕见疾病的了解。