Epilepsy Drug Discovered in Fish Model Shows Promise in Pediatric Clinical Trial

A zebrafish model of Dravet syndrome, a severe form of pediatric epilepsy, may help scientists quickly find drugs for the disorder.

Photo: Scott Baraban, UCSF

“Bench-to-bedside” describes research that has progressed from basic science in animal models that has led to therapies used in patients. Now, a study in the journal Brain describes what could be considered a direct “aquarium-to-bedside” approach, taking a drug discovered in a genetic zebrafish model of epilepsy and testing it, with promising results, in a small number of children with the disease. The study was supported by NINDS.

“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 Dr. Vicky Whittemore, NINDS program director. “These findings suggest that it may be possible to treat neurological disorders caused by genetic mutations through an efficient and precision medicine-style approach.”

Dr. Scott C. Baraban, the William K. Bowes Jr. endowed chair in neuroscience research and professor of neurological surgery at the University of California, San Francisco, postdoctoral fellow Dr. Aliesha Griffin and colleagues used a zebrafish model of Dravet syndrome to test the drug lorcaserin and found that it suppressed seizure activity in the fish. Dravet syndrome is a severe form of pediatric epilepsy characterized by frequent daily drug-resistant seizures and developmental delays. It is caused by a genetic mutation, which Baraban’s group was able to introduce into the zebrafish to cause epilepsy.