Epilepsy drug stops nervous system tumor growth in mice

Author: Tamara Bhandari

Published On: 07/01/22

People with neurofibromatosis type 1 (NF1) develop tumors on nerves throughout their bodies. These tumors are usually benign — meaning they don’t spread to other parts of the body and are not considered life-threatening — but they can still cause serious medical problems such as blindness, especially when they form in the brain and nerves.

Researchers at Washington University School of Medicine in St. Louis have discovered that neurons carrying a mutation in the Nf1 gene are hyperexcitable and that suppressing this hyperactivity with lamotrigine, a drug approved by the Food and Drug Administration to treat epilepsy, stops tumor growth in mice.

“Tumors are very common in people with NF1,” said senior author David H. Gutmann, MD, PhD, the Donald O. Schnuck Family Professor and director of the Washington University Neurofibromatosis (NF) Center. “We’ve shown that we can block the growth of NF1 tumors by shutting off neuronal hyperexcitability. We’ve done it now a couple of different ways, and there’s no question that repurposing antiepileptics is an effective way to inhibit tumor growth, at least in mice. This underscores the critical role that neurons play in tumor biology.”

The study is published May 19 in Nature Communications.

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