Rare Central Nervous System Cancers Initiative

罕见中枢神经系统癌症倡议

基本信息

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
项目类别：
财政年份：
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

项目摘要

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.

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。这项工作利用了 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。计划与成像技术专家合作，创建成像生物标志物和用于监测治疗反应的常规成像。已经建立了一个组织存储库来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、子分类和最终预测反应的标记物到具体的治疗。此外，还开发了特定的测试功能，例如上述 CNS 分子面板，并且正在开发其他测试。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。最近开放了四项临床试验（17C0102 免疫检查点抑制剂纳武单抗治疗特定罕见中枢神经系统癌症患者；19-C-0011 Marizomib 治疗复发性低度和间变性幕上、幕下和脊髓室管膜瘤的 II 期临床试验；18-c- 0137 舒尼替尼治疗中枢神经系统肉瘤的 II 期试验， 19-C-0006 II 期试验评估 Nivolumab 在具有或不具有高突变表型的 IDH 突变胶质瘤患者中的作用。最近，我们建立了 NCI-CONNECT：评估罕见中枢神经系统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，以改进护理和治疗方法。