SANTA BARBARA, Calif. — For nearly one-third of the 3 million adults in the United States living with epilepsy, medications fail to control seizures. But new research is offering hope through minimally invasive therapies designed to heal the brain rather than remove parts of it.
Clinical trials underway at Mayo Clinic and other centers are exploring the use of cell-based therapies, which involve injecting special cells into the brain. These cells integrate into brain tissue over time and aim to restore the balance between excitatory and inhibitory signals — potentially preventing seizures before they start.
“What cell-based therapies are trying to do is restore that normal balance between things that are exciting the brain and things that are inhibiting the brain,” said Dr. Jonathan Parker, a neurosurgeon at Mayo Clinic.
Traditional treatment options for drug-resistant epilepsy include invasive surgeries such as removing portions of the brain or using lasers to destroy affected tissue. While these procedures can be effective, they also carry risks such as speech impairment and memory loss.
Parker described these surgeries as “destructive” and emphasized that outcomes are not guaranteed.
“We want all of our patients to be seizure-free, and that’s the goal that we’re working towards,” he said.
In addition to cell-based therapies, other emerging treatments include neuromodulation techniques. Devices like the NeuroPace RNS system, a type of brain pacemaker, and laser ablation are becoming more common as less invasive alternatives to surgery.
Dr. Phillip McCarthy, an epileptologist at Corewell Health West Epilepsy Center in Michigan, said he is encouraged by the new developments.
“There’s a lot of promise in either stem cell or viral vector gene therapies,” McCarthy said.
McCarthy’s center is involved in gene therapy trials using viral vectors to deliver RNA that targets specific neural pathways linked to seizures. These treatments may offer relief for patients with medically refractory epilepsy, a condition where seizures persist despite medication.
“Regardless of the new medications we come up with for epilepsy, we still hit that same statistic,” McCarthy said, referencing the long-standing finding that about 30% of patients do not respond to drug therapies.
Epilepsy can have various causes, including tumors, strokes, and genetic abnormalities. However, some origins remain unclear. One such condition, medial temporal sclerosis, may be linked to early childhood viral infections, though the exact cause is still under investigation.
“There’s a lot of hypotheses,” McCarthy said. “In most cases — or some 30% of those cases — patients can experience medically refractory epilepsy.”
While there is still no cure, researchers hope that cell-based and gene therapies will reshape the future of epilepsy treatment and offer a path to seizure freedom for more patients.
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