Awareness, Not Access, Remains the Barrier to NF1 Therapy

On October 7, 2026, NeurologyLive convened a Clinical Forum in Phoenix, bringing together community oncologists and neurologists to discuss the recognition and treatment of neurofibromatosis type 1 (NF1) and its associated plexiform neurofibromas (PN). The program was led by Kevin J. Bielamowicz, MD, a pediatric neuro-oncologist who co-directs the multidisciplinary NF1 clinic at Arkansas Children’s Hospital in Little Rock. He opened by noting the nonstop flight that gets him from Little Rock to Phoenix in a few hours, a detail that doubled as an explanation for why he was in the room at all: Arkansas has no dedicated adult neuro-oncologist, so Bielamowicz has increasingly become the de facto adult specialist too.

That gap shaped much of the evening’s conversation. Pediatric NF1 clinics are well built out at children’s hospitals, Bielamowicz said, but patients often lose that infrastructure the moment they age out of it. “We develop these amazing pediatric multidisciplinary clinics at children’s hospitals, and they all get funneled into that clinic, and then it’s really challenging as we’re transitioning,” he said.

One attendee, an adult neurologist who sees NF1 patients only for seizure management, described the same problem from the other side: without one clinician coordinating their care, patients “fall through the cracks.” Bielamowicz said he has spent 11 years building his own answer to that gap, assembling an informal network of neurosurgeons, orthopedic surgeons, endocrinologists, and ophthalmologists across Arkansas so adult patients have somewhere to land.

Bielamowicz walked attendees through a case pulled from the medical literature, a 44-year-old man with a large, inoperable facial PN, to illustrate why surgery so often falls short for these tumors. Resection can reduce recurrence risk, he said, but only if most of the tumor comes out, and PN frequently grows in clusters that resist clean removal. He described a teenager who underwent an 80 percent resection of a head and neck PN only to find it unchanged five years later. On a MEK inhibitor, the case patient saw a 24 percent reduction in tumor volume over a year, a result Bielamowicz frames deliberately for families before treatment begins. “If you have a 24 percent response in this giant tumor, is that going to be a response that you’re going to be happy with?” he asked. “Maybe it’s all you have to try.”

Attendees also pressed Bielamowicz on drug selection and combination strategies. He explained that unlike in malignant conditions, stacking multiple MAP kinase pathway inhibitors in benign PN tends to add toxicity without improving response, a distinction he said often surprises oncologists used to combination regimens in cancer care. Polling during the forum found that lack of awareness and familiarity, not access or cost, was attendees’ top-cited barrier to referring or prescribing systemic therapy, a finding consistent with earlier NeurologyLive forums on the same topic.

Not every finding was discouraging. Asked whether dermatologists reliably catch NF1 in adults, Bielamowicz pushed back on the assumption that primary recognition is the weak link. “I actually feel like outside dermatologists that we get referrals from actually pick up on it pretty nicely,” he said, adding that the average age of diagnosis at his clinic has dropped steadily over the past decade as community physicians have gotten more comfortable with the condition.

Bielamowicz closed by acknowledging how little runway he had to cover a dense topic over dinner. “I’m breezing through these because I tend to talk and not necessarily go with the slides,” he said, thanking the group for its attention. For a rare disease that depends heavily on which specialist a patient happens to find first, that kind of candid, in-the-room exchange, between a pediatric specialist who flew in and the adult clinicians who will see these patients next, may matter as much as any single therapy discussed.