ZIA BC 010801 (ZIA) | |||
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Title | Therapy for NF1-Related Tumors and other Genetic Tumor Predisposition Syndromes | ||
Institution | NCI, Bethesda, MD | ||
Principal Investigator | Widemann, Brigitte | NCI Program Director | N/A |
Cancer Activity | N/A | Division | CCR |
Funded Amount | $719,531 | Project Dates | 00/00/0000 - 00/00/0000 |
Fiscal Year | 2017 | Project Type | Intramural |
Research Topics w/ Percent Relevance | Cancer Types w/ Percent Relevance | ||
Cancer (100.0%) Childhood Cancers (80.0%) Neurofibromatosis (100.0%) |
Central Nervous System - Not Including Brain (25.0%) Nervous System (75.0%) Sarcoma (25.0%) |
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Research Type | |||
Systemic Therapies - Discovery and Development Systemic Therapies - Clinical Applications |
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Abstract | |||
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). |