New Meds Target Seizures from Happening in the First Place
Epilepsy affects approximately 3 million adults and almost a half a million children in the United States. Roughly 30% to 50% of children with autism also suffer with epilepsy. There are a number of anti-seizure drugs on the market, however, 30% of people with the disorder have tried at least three different medications and still experience seizures. According to Paul Matteis, the head of the biotech tech research group at the investment firm Stifel, “Once you fail three drugs, your probability of becoming seizure-free is only 6%.” This sobering statistic has created an opening for drug companies to develop new medications using novel approaches to address the limitations of existing drug choices. Currently, most epilepsy drugs suppress seizures. However, in the last ten years researchers started focusing on molecular targets which could stop seizures from occurring in the first place. They hope to do this by balancing the communication networks in the brain that misfire in individuals with epilepsy. Right now, there are four companies working to change the treatment landscape for the disease. Marinus Pharmaceuticals has already filed for Food and Drug Administration approval for its drug, Ganaxolone. Their medication successfully reduced seizure activity in children with a rare form of pediatric epilepsy by regulating the way neurons in the brain communicate with one another. The Canadian biopharmaceutical company, Xenon, is working on a treatment for adults with focal epilepsy, a condition where seizures only affect half the brain. This drug also aims to balance excitatory and inhibitory neurons but does it by regulating the potassium in a person’s brain that normally dampens excitation. Cerevel Therapeutics is in a Phase 2 clinical trial with a drug that showed anti-epileptic activity in patients with photosensitive epilepsy, triggered by flashing lights or contrasting light and dark patterns. Finally, Stoke Therapeutics is developing a drug that aims to reduce the frequency of convulsive seizures in children with a rare form of epilepsy called Dravet syndrome. Their treatment is RNA-based and targets the underlying genetic cause of the disorder. One drawback to therapy, which has already shown positive early results, the medication must be injected into the patient’s spine. With these four new medications in development, the treatment horizon for the epilepsy community is looking more promising.
