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.

NIH非常适合创建一项罕见的CNS癌症计划。这项工作将利用NIH的以下特定资源。这需要开发包括数据管理系统在内的临床试验基础设施，以监督疾病特定的治疗和自然史研究。与成像技术专家的合作，以创建成像生物标志物和常规成像，以监视对疗法的反应。该倡议将结合儿科和成人研究人员的专业知识，从这些研究者的各种方法中受益。已经开发了组织储存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，目的是推进对肿瘤生物学和发病机理的理解，从而获得更好的预后信息，亚分类，并最终标志着对特定处理反应的预测。此外，将根据项目需求建立特定的测试功能。例如，预计将进行免疫疗法试验，并且将需要作为本研究的关键组成部分的免疫监测能力。基于NIH的外展工作将用于与患者倡导小组互动，以增强患者参与这些努力，并为教育计划提供一个教育计划的场所，以改善患者和看护者对他们不常见疾病的理解。