What Role Do Zebrafish Models Play in Epilepsy Treatment?

Scientists have discovered a potential new drug therapy by using a zebrafish model of epilepsy. The study, which was supported by the National Institute of Neurological Disorders (NINDS), is the first time a drug has been taken from a fish model and applied directly to a human trial.

Researchers discovered the potential therapy using a zebrafish model of Dravet syndrome, a severe form of pediatric epilepsy, to test the drug lorcaserin. The findings revealed that the drug was able to suppress seizure activity in the fish, which prompted the researchers to test the drug’s efficacy in children with Dravet syndrome in a clinical trial.

“These findings suggest that it may be possible to treat neurological disorders caused by genetic mutations through an efficient and precision medicine-style approach,” Vicky Whittemore, PhD, program director at the NINDS, said in a news release.

The clinical trial tested lorcaserin in 5 children with Dravet syndrome who were resistant to other anti-epileptic drugs and participated in the study through a compassionate use, off-label program. Initial results demonstrated decreased seizure frequency in all the children who were taking the drug. However, after a few months, seizure activity increased, but it was not at the level of frequency reported at the beginning of the trial.

The study team’s previous research identified the compound clemizole as another potential anti-epileptic therapy that decreases seizure activity by acting on the serotonin system. They are currently developing the drug for further research in clinical trials.

Reference

Epilepsy drug discovered in fish model shows promise in small pediatric clinical trial [news release]. NIH’s website. https://www.nih.gov/news-events/news-releases/epilepsy-drug-discovered-fish-model-shows-promise-small-pediatric-clinical-trial?utm_source=dlvr.it&utm_medium=twitter. Accessed Feb. 10, 2017.