Investigating FDA-approved drugs in an animal model of KCNT1-childhood epilepsy to identify potential new therapies for treating children

Investigating FDA-approved drugs in an animal model of KCNT1-childhood epilepsy to identify potential new therapies for treating children

Chief Investigator: Professor Leanne Dibbens

Funding Amount: $ 100,000

Recipient: University of South Australia

Overview:

KCNT1 mutations cause one of the most severe forms of epilepsy in children, resulting in frequent drug resistant seizures and cognitive decline. Currently, there are no drugs available which specifically target the underlying cause of the seizures, the mutated KCNT1 ion channel. We have recently identified several FDA-approved drugs which block the action of the hyperactive mutated KCNT1 channel. We will investigate the ability of these drugs (up to 10) to reduce the seizure phenotype in our Drosophila model of KCNT1-childhood epilepsy. We predict this study will identify new therapies to treat children with KCNT1-drug resistant epilepsy.