As per the findings of a recent study published in the Journal 'Seizure', the long term use of Everolimus was found effective, safe and tolerable in patients with tuberous sclerosis complex (TSC) associated therapy-resistant epilepsy. The present compassionate use trial aimed to assess the therapeutic efficacy and tolerability of Everolimus for treating TSC and therapy-resistant epilepsy. Patients were administered with Everolimus, after a baseline period of 3 months.

The overall treatment was categorized into 3 phases, each of which lasts for 9 months. One of the three target serum levels was started in patients and subsequent treatment levels with another target serum level were started in case of insufficient seizure control. During the baseline and initial 3 treatment phases, the accompanying anti-seizure medication remained stable. The changes in the seizure frequency and seizure-free days was compared to baseline values in each patient.

A total of 15 patients were included and followed up for 10 years. A significant decrease in the seizure frequency and an increase in the seizure-free days was reported in 80% of patients. Around 40% of patients were found to be seizure-free and 26.7% (n=4) patients remained seizure-free for more than > 7 years and 3 patients required no additional anti-seizure medication. At least one adverse effect was observed in each participant, however, the majority of the side effects were mild to moderate.

Everolimus can be a good therapeutic option for treating therapy-resistant epilepsy in TSC, however, it may take a longer time for maintaining seizure relief.