Known drug may help treat childhood epilepsy

RUTGERS (US)—A widely available therapeutic drug may prove effective in treating childhood epilepsy, according to Rutgers researchers.

In a quest for new therapeutic approaches, Rutgers neuroscientist Gabriella D’Arcangelo is investigating the molecular basis of the disease. Her article in the journal Disease Models and Mechanisms describes the first use of a mouse model of cortical dysplasia, a malformation of the brain that is most often the cause of childhood epilepsy. Introduction of the drug rapamycin, originally used to prevent rejection in organ transplants, suppressed epileptic seizures in the mice.

Epilepsy is the third most common neurological disorder in the United States after Alzheimer’s disease and stroke. It currently affects more than 326,000 children under age 15. More than 90,000 of them have severe seizures that cannot be adequately treated. The children often go on to develop cognitive problems due to recurrent and uncontrolled seizures and the combined effects of heavy medication. They may also suffer consequences from having parts of their brains removed during surgery. Approximately 45 percent of pediatric epilepsy surgery cases are due to cortical dysplasia.

“The surgery is not without risks, and while it may help control the seizures, it does not work in all cases,” says D’Arcangelo, an associate professor in the Department of Cell Biology and Neuroscience at Rutgers. “Clearly there is a pressing need to come up with new strategies for treatment.”

“We demonstrated that rapamycin is a novel and effective anti-epileptic agent that suppresses seizures in our mice . . . and this has raised some hope for the future,” says D’Arcangelo. “This drug is being tested on human patients of treating tuberous sclerosis complex (TSC), in a multicenter study involving six TSC clinics throughout the United States. I hope it will soon be tested for all cortical dysplasia patients.”

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