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) 药理学与实验治疗科 (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。特别关注需求未得到满足的罕见肿瘤。有前景的新型药物在早期临床试验中得到研究，评估毒性、活性、药代动力学和药效学。通过利用独特的 NIH 资源，我将难治性癌症的药物开发原理应用于 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的综合 NF1 临床试验项目（MPNST）。