Researchers Uncover Possible Cause of Pain in Neurofibromatosis Type 1

Author: News Medical Life Sciences, Cincinnati Children's Hospital Medical Center

Published On: 7/1/26

Researchers at Cincinnati Children's have identified a potential new way to relieve chronic pain linked to neurofibromatosis type 1 (NF1), a genetic condition best known for causing tumors to grow along nerves.

The new findings suggest that pain in NF1 may begin before tumors appear and may be driven by abnormal signaling from Schwann cells, which normally support and protect nerves. The abnormal signaling produces excess glial cell line–derived neurotrophic factor, or GDNF, a protein that can heighten pain signaling.

Details were published May 26, 2026, in Science Signaling. Namrata G. R. Raut, PhD, was first author. Michael P. Jankowski, PhD, was corresponding author.

The work helps explain why many people with NF1 report significant pain even in areas where no tumors are present. Importantly, we also found that blocking MAPK signaling with a MEK inhibitor lowered GDNF levels in Schwann cells and reduced pain-like responses in the mice."

Michael P. Jankowski, PhD, corresponding author

Although tumor-related pain in NF1 is well recognized, non-tumor pain has remained poorly understood and difficult to treat. The new study focused on that gap.

Using a mouse model in which the NF1 gene was deleted in Schwann cells, the investigators found that those cells were the main source of excess GDNF. The protein acted through a receptor called GFRα1 on pain-sensing nerve fibers, helping drive mechanical hypersensitivity.

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