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

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。

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).