Brigitte C. Widemann
布里吉特·魏德曼
MD
Chief, Pediatric Oncology Branch; Senior Investigator儿科肿瘤分部主任；高级研究员
👥Biography 个人简介
Dr. Widemann is the leading authority on NF1 clinical trials at NCI, pioneering selumetinib (MEK inhibitor) therapy for pediatric plexiform neurofibromas. Her SPRINT trial established the first FDA-approved treatment for this condition, transforming care for NF1 patients worldwide.
🧪Research Fields 研究领域
🎓Key Contributions 主要贡献
Selumetinib for Pediatric NF1
Led the landmark SPRINT Phase II trial demonstrating selumetinib's efficacy in children with NF1 and inoperable plexiform neurofibromas, leading to FDA approval in 2020 and establishing MEK inhibition as standard of care.
NF1 MPNST Treatment
Developed clinical trial frameworks for malignant peripheral nerve sheath tumors in NF1, advancing combination chemotherapy and targeted therapy approaches for this aggressive and historically treatment-resistant malignancy.
Representative Works 代表性著作
Selumetinib in Children with Inoperable Plexiform Neurofibromas
New England Journal of Medicine (2020)
Pivotal SPRINT trial results demonstrating objective response in 70% of NF1 patients, leading to FDA approval of selumetinib for pediatric plexiform neurofibromas.
Phase I Trial of Selumetinib in Adults with NF1 and Plexiform Neurofibromas
Clinical Cancer Research (2019)
Established selumetinib dosing and safety profile in adult NF1 patients with progressive plexiform neurofibromas, expanding treatment access beyond the pediatric population.
🏆Awards & Recognition 奖项与荣誉
📄Data Sources 数据来源
Last updated: 2026-01-15 | All information from publicly available academic sources
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