Therapy for NF1-Related Tumors and other Genetic Tumor Predisposition Syndromes

NF1相关肿瘤和其他遗传性肿瘤易感综合征的治疗

• 批准号: 9556368
• 负责人: Brigitte Widemann
• 金额: $ 100.17万
• 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/9556368
• 关键词: Biology; Child; Clinical Trials; Drug Kinetics; Inherited; Investigational Therapies; Knowledge; Malignant Neoplasms; Mission; Neurofibromatosis 1; Neurofibromatosis 2; Neurofibrosarcoma; Other Genetics; Pathogenesis; Pediatric Oncology; Pharmacodynamics; Pharmacology; Plexiform Neurofibroma; Predisposition; Refractory; Resources; Syndrome; Toxic effect; Tumor-Associated Process; United States National Institutes of Health; cancer genetics; clinical development; drug development; effective therapy; medullary thyroid carcinoma; novel; programs; targeted agent; tumor; young adult

The mission of the Pharmacology & Experimental Therapeutics Section (PETS) of NCI's Pediatric Oncology Branch (POB) is to develop more effective treatments for children and young adults with refractory cancers and genetic tumor predisposition syndromes (GTPS). A particular focus is on rare tumors with unmet need. Promising novel agents are studied in early clinical trials that evaluate toxicities, activity, pharmacokinetics, and pharmacodynamics. By leveraging unique NIH resources, I have applied the principles of drug development for refractory cancers to neurofibromatosis type 1 (NF1)-related tumors and built the nation's largest comprehensive NF1 clinical trials program directed at plexiform neurofibromas (PN) and malignant peripheral nerve sheath tumors (MPNST).

NCI儿科肿瘤科(POB)的药理学和实验治疗科(PETS)的使命是为患有难治性癌症和遗传性肿瘤易感综合征(GTPS)的儿童和年轻人开发更有效的治疗方法。特别关注的是需求未得到满足的罕见肿瘤。有希望的新药在早期临床试验中被研究，评估毒性、活性、药代动力学和药效学。通过利用NIH独特的资源，我将难治性癌症的药物开发原则应用于1型神经纤维瘤病(NF1)相关肿瘤，并建立了全国最大的针对丛状神经纤维瘤(PN)和恶性周围神经鞘瘤(MPNST)的综合NF1临床试验计划。