Adamant seizure control in young children is necessary to avoid the development of the epileptic encephalopathy. Compared to children without disease, children with refractory epilepsy are at risk of higher rates of morbidity and death.
Early intervention to control seizure frequency is also critical to reducing seizure-related morbidity and mortality. Resective and palliative surgeries continue to be the mainstay of treatment in a majority of these children. However, 20-40% patients will continue to have seizures after surgery.
mTOR is dysregulated in a number of human diseases, including tuberous sclerosis and epilepsy. A phase 1 study is currently evaluating ABI-009 in conjunction with pre-existing antiepileptic drugs (AEDs) regimen in pediatric patients with surgically-refractory epilepsy (NCT03646240).
For recent update on the trial see New Drug Trial Gives Hope for Treatment-Resistant Epilepsy