Child Neurology Society's Brainstorm CNS Brainstorm Episode 48 "Pediatric NF1 Therapeutics" with guest Bonnie Kaur, MD
Dec 17, 2025
Join Dr. Bonnie Kaur, Assistant Professor of Neurology and Pediatrics, as she dives into the world of neurofibromatosis type 1 (NF1). She discusses the evolution of treatments, including the groundbreaking MEK inhibitors, selumetinib and mirdametinib, which show promise in halting tumor growth. Dr. Kaur shares insights on eligibility, potential side effects, and the impressive results from recent clinical trials. She also highlights emerging therapies and resources for patients, making it a must-listen for those interested in NF1 advancements.
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NF1 Is A Lifelong, Multi-System Condition
- Neurofibromatosis type 1 (NF1) causes tumors on nerves and affects about 1 in 2,500 births worldwide.
- NF1 is lifelong and requires specialized multidisciplinary care rather than a one-time cure.
MEK Inhibitors Shift NF1 Care From Observation
- Two FDA-approved MEK inhibitors now target plexiform neurofibromas, marking a therapeutic shift from pure surveillance.
- These drugs block RAS–RAF–MEK–ERK signaling to inhibit tumor cell growth or cause cell death.
Treat Eligible Patients With MEK Inhibitors
- Offer MEK inhibitors to patients older than one year with inoperable, symptomatic plexiform neurofibromas per FDA approvals.
- Apply the same eligibility to adults and coordinate with surgical teams for resectability assessments.