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：
10486897
负责人：
Mark Gilbert
金额：
$ 42.53万
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/10486897
关键词：
Astrocytoma Caregivers Caring Central Nervous System Neoplasms Chordoma Choroid Plexus Carcinoma Classification Clinical Clinical Trials Collaborations Consensus Data Management Resources Development Diagnosis Disease Enrollment 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 Joints Knowledge Laboratories Malignant - descriptor Malignant Neoplasms Malignant neoplasm of central nervous system 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 Phenotype Primary Brain Neoplasms Primary Neoplasm Proteomics Provider Publishing Rare Diseases Recruitment Activity Regimen Research Research Personnel Resources Rhabdoid Tumor Sampling Testing Therapeutic Tissue Banks Tumor Biology Tumor Tissue United States United States National Institutes of Health base clinical infrastructure imaging biomarker immunotherapy trials improved medulloblastoma member metabolomics methylome molecular pathology mutant oligodendroglioma outreach patient advocacy group patient engagement phase II trial pineoblastoma predicting response predictive marker prognostic programs rare cancer response routine imaging 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 underway and actively recruiting. This study includes immunologic monitoring that are being performed in Dr. Gilbert's laboratory. 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. Two clinical trials are recently open (17C0102 Immune Checkpoint Inhibitor Nivolumab in People With Select Rare CNS Cancers;19-C-0006 Phase II Trial Evaluating Nivolumab in Patients with IDH-Mutant Gliomas with and without Hypermutator Phenotype). 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 is very active with clinical trial participation among the members of the network.

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，一项免疫疗法试验正在进行中并正在积极招募患者。这项研究包括吉尔伯特博士实验室正在进行的免疫监测。 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。最近开放了两项临床试验（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-0006 评估纳武单抗治疗具有或不具有高突变表型的 IDH 突变胶质瘤患者的 II 期试验）。 NCI-CONNECT：评估罕见中枢神经系统肿瘤的综合肿瘤学网络非常活跃，网络成员积极参与临床试验，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。