Treatments Needed for Rare Epilepsy Disorders

Jacqueline A. French, MD, NYU Epilepsy Center, New York, NY discussed the latest research in the field of epilepsy and seizure disorders during a presentation at Neurology Week.

Dr French emphasized a need for anti-epilepsy drugs. Currently, anti-seizure drugs, which block excitation, increase inhibition, and prevent repetitive firing but do not stop the cause of seizures, are used to treat epilepsy, and are considered as anti-epilepsy drugs.

“The International League Against Epilepsy, which is our naming organization, is very much considering an official name change from anti-epileptic drugs to two different combinations: anti-seizure medicine and disease-modifying epilepsy medication,” Dr French said. “Again, this is to highlight to everybody, including our patients, that most medications don't alter the course of epilepsy.”

Current treatment options exist for focal epilepsy and idiopathic generalized epilepsy, often referred to as common epilepsies, which account for about 3/4 of patients. Other patients have rare forms of epilepsy, which Dr French referred to as orphan syndromes, that do not currently have efficient treatment options.

Dr French emphasized a need for treatment options for these rare epilepsies, which could be obtainable due to the premium price that a medication for a rare disorder could command, the idea that rare "orphan" diseases can get special benefits from regulators (the FDA) to encourage drug development, and that the marketplace is not "crowded". Additionally, motivated patient groups may help speed development. 

Dr French broke down opportunities for targets of precision medicine or precision therapies. While genes and genetic markers are one target for precision medicine, other precision targets of therapy to improve treatment of epilepsies include drugs that target neuroinflammation, blood-brain barrier disruption, and disruption of the M-tor pathway.

The presentation at Neurology Week highlighted some new treatment options in the realm of epilepsy that have been approved as well as research currently in the works, including wearable devices to treat, detect, and forecast seizures. Additionally, subscalp or subcutaneous implants for long-term EEG monitoring are in development.  

“Epilepsy is a unique disease, in that our individualized choices for patients can be the difference between seizure control or continued epilepsy disability,” Dr French concluded. “We hope in the future to move to therapies that are disease-modifying or curative.”

—Erin McGuinness

French, J. State of the Science: Research and New Therapies in Epilepsy. Presented at Neurology Week 2021; July 14-18. Virtual.