In an unprecedented leap from lab to patients, a potential treatment for childhood epilepsy identified in experiments with zebrafish by UC San Francisco scientists was administered in a clinical trial to children with Dravet syndrome, a rare and devastating genetic form of epilepsy that can cause hundreds of seizures per day. The therapy tested in the small, compassionate–use trial, a drug known as lorcaserin (Belviq), significantly reduced the frequency of seizures in all five patients enrolled in the trial, and two of those children continue to take the drug with no increase in seizure activity.

The decision to evaluate lorcaserin as a Dravet therapy grew out of research in the UCSF laboratory of Scott C. Baraban, PhD, professor of neurological surgery, who has developed platforms with which hundreds of drug compounds can be rapidly assessed for effectiveness in zebrafish larvae that carry gene mutations paralleling those known to cause childhood epilepsy.

“This is the first time that scientists have taken a potential therapy discovered in a fish model directly into people in a clinical trial,” said Vicky Whittemore, PhD, program director at the National Institute of Neurological Disorders and Stroke (NINDS), a division of the National Institutes of Health (NIH) that provided funding for the new study. “These findings suggest that it may be possible to treat neurological disorders caused by genetic mutations through an efficient and precision medicine–style approach.”

The clinical trial results, as well as the basic research leading up to the trial, were reported in the March 2017 issue of the journal Brain.