Epilepsy is a spectrum disorder characterized by recurring, unprovoked seizures. Although people often equate seizures with convulsions due to their obvious and dramatic nature; seizures are actually an abnormal, uncontrollable firing of neural networks in the brain. As a result, seizures can impact any function controlled by the brain.
While there are many types and causes of epilepsy, they all ultimately result from an imbalance of excitory and inhibitory brain-cell function. Restoring this balance can reduce or eliminate seizures, bringing epilepsy under control. While current medications manage some types of epilepsy effectively, other types are resistant to therapy (i.e. they are refractory). In addition, many patients feel lethargic and sleepy while taking their medication due to the sedative nature of these drugs. These sedative effects are largely due to activation of GABAA receptors containing α1 subunit.
Neurocycle Therapeutics is working on developing an α1-sparing neurosteroid, that is capable of treating refractory types of epilepsy without sedation. Unlike benzodiazepines, neurosteroids potentiate both synaptic and extrasynaptic GABAA receptors. Potentiation of extrasynaptic receptors induces long lasting tonic inhibition which is thought to be particularly important for control of epilepsy. Currently, we are evaluating our compounds in models of epilepsy associated with traumatic brain injury and tuberous sclerosis complex.