Rare Central Nervous System Cancers Initiative

罕见中枢神经系统癌症倡议

• 批准号: 10262379
• 负责人: Mark Gilbert
• 金额: $ 43.65万
• 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/10262379
• 关键词: Astrocytoma; Caregivers; Caring; Central Nervous System Neoplasms; Central Nervous System Sarcoma; Choroid Plexus Neoplasms; Classification; Clinical; Clinical Trials; Collaborations; Consensus; Data Management Resources; Development; Disease; Enrollment; Ensure; Ependymoma; Evaluation; Fostering; Funding; Gene Amplification; Gene Fusion; Genomic DNA; Genomics; Glioblastoma; Glioma; Gliomatosis cerebri; Goals; Histones; Imaging technology; Immune checkpoint inhibitor; Immunologic Monitoring; Incidence; Infrastructure; International; Investigation; Knowledge; Laboratories; Malignant - descriptor; Malignant Childhood Neoplasm; Malignant Neoplasms; Malignant neoplasm of central nervous system; Methylation; Molecular; Molecular Analysis; Monitor; Monograph; Mutate; Mutation; National Cancer Institute; Natural History; Neoplasms; Nivolumab; Normal tissue morphology; Oncology; Pathogenesis; Pathology; Patient Participation; Patient advocacy; Patients; Pediatric Oncology; Phase II Clinical Trials; Phenotype; Pineal Region Tumor; Primary Brain Neoplasms; Primary Neoplasm; Proteomics; Provider; Publishing; Rare Diseases; Recurrence; Regimen; Research; Research Personnel; Resources; Rhabdoid Tumor; Sampling; Spinal Cord; Supratentorial; Testing; Therapeutic; Tissue Banks; Tumor Biology; Tumor Tissue; United States; United States National Institutes of Health; base; clinical infrastructure; exome sequencing; imaging biomarker; immunotherapy trials; improved; medulloblastoma; member; meningioma; metabolomics; molecular pathology; mutant; oligodendroglioma; outreach; patient advocacy group; pediatric patients; phase II trial; predicting response; predictive marker; prognostic; programs; rare cancer; response; routine imaging; transcriptome sequencing; tumor

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.

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用于特定罕见的中枢神经系统癌症患者；19-C-0011 Marizomib第二阶段临床试验用于治疗复发的低度恶性和间变性幕上、幕下和脊髓室管膜瘤；18-C-0137 Sunitinib治疗中枢神经系统肉瘤的第二阶段试验；19-C-0006第二阶段试验评估具有和不具有超突变表型的IDH突变胶质瘤患者的Nivolumab)。最近，我们建立了NCI-CONNECT：综合肿瘤学网络，评估罕见的中枢神经系统肿瘤，以促进患者-倡导-提供者伙伴关系和网络，以改进护理和治疗方法。