The biggest barrier to treating NF1 tumors? Doctors don't know the drugs exist

Patients with neurofibromatosis type 1 (NF1) and its associated plexiform neurofibromas (PN) are missing out on effective systemic therapy — and the culprit isn't insurance or drug costs. At clinical forums in Houston (September 2026) and Phoenix (November 2026), pediatric neuro-oncologist Kevin Bielamowicz, MD, of Arkansas Children's Hospital, found that lack of awareness was the top barrier cited by attending community oncologists and neurologists — several of whom said they hadn't known MEK inhibitors were an option for NF1-PN until the forums themselves.

A major structural gap compounds the problem: robust pediatric NF1 multidisciplinary clinics exist at children's hospitals, but when patients age out, there's often nowhere to go. Adult neuro-oncology infrastructure is thin — Arkansas, for instance, has no dedicated adult neuro-oncologist — leaving patients to "fall through the cracks." Surgery, long the default for PN, frequently falls short because these tumors infiltrate nerves and resist clean removal. MEK inhibitors like selumetinib and mirdametinib have emerged as key systemic options, with one case showing a 24% tumor volume reduction at one year — modest, but clinically meaningful for pain, edema, and appearance.

Key Takeaways:

• NF1 affects ~1 in 3,000 births; plexiform neurofibromas are often inoperable due to nerve infiltration
• MEK inhibitors (selumetinib, mirdametinib) are central to systemic management, but cross-trial comparisons should be interpreted cautiously
• Combining MAP kinase pathway inhibitors in benign PN adds toxicity without improving response — unlike in malignant oncology
• Pediatric residents may receive as little as one lecture on NF1 in three years of training

Why it matters: For a rare disease where outcomes depend heavily on which specialist a patient happens to find first, closing the awareness gap among adult clinicians may be just as impactful as developing new therapies.