Research: Lennox Gastaut Syndrome
Lennox Gastaut syndrome (LGS) is a devastating epileptic encephalopathy of childhood. It presents with frequent seizures of multiple types (tonic, atonic, tonic-clonic, absence, myoclonic), cognitive delays and generalized slow spike wave on EEG studies. The seizures are notoriously difficult to control and patients often fail multiple medications, dietary therapy and palliative surgeries. Recognizing the need to find better treatments for this devastating condition, Cook Children's continues to participate in new research using drugs such as low-dose fenfluramine for treatment of seizures associated with LGS.
Cook Children's was the only site in Texas to participate in an earlier study of fenfluramine for Dravet syndrome. Initial data from these earlier studies have demonstrated remarkable reduction in seizure frequency with patients on 0.8 mg/kg/d of fenfluramine experiencing a median 72.4% reduction in convulsive seizures, versus only 17.4% in the placebo group (p<0.001). Nearly half (45%) of treated patients had >75% reduction of seizures, versus only 2.5% in the placebo group (p<0.001). Similarly, mean number of seizure free-days was longer in the treatment group (20.5 days), versus the placebo.
Fenfluramine was formerly a component of the diet drug Fen-Phen and was associated with cardiac valvulopathy prompting withdrawal from the market. Thus far, no significant cardiac valvulopathy or pulmonary hypertension has been noted in trials for epilepsy, possibly due to the lower dosing used in these trials.
Cook Children's Neurosciences team strives to offer new and innovative medication treatments to our patients through clinical trials and research. A number of studies are currently open and enrolling for diagnoses such as epilepsy, dystonia and muscular dystrophy at Cook Children's.