Neurorestorative Treatment Strategies Hold Promise for Most Severe Forms of Epilepsy
Posted on 08 Apr 2025
Epilepsy affects approximately 50 million people worldwide, with around 30% of these individuals, or roughly 15 million people, suffering from drug-resistant epilepsy (DRE). While some patients experience only a few seizures per month, others may have hundreds of seizures each day, ranging from mild episodes to life-threatening events. Current treatment options for DRE include surgical interventions like brain resection, where a portion of the brain tissue responsible for seizures is removed. Another approach is laser ablation therapy, which targets and destroys abnormal brain tissue using focused energy. Although these surgical methods can be effective, they carry the risk of side effects such as memory problems, motor deficits, and speech difficulties. Another surgical option, neuromodulation, uses electrical or magnetic stimulation to interrupt abnormal brain activity without removing brain tissue. However, a new and innovative trend in research is exploring the use of regenerative medicine as a "reparative" approach to help heal the brain in patients with DRE.
Researchers at Mayo Clinic (Rochester, MN, USA) are conducting the first-in-human clinical trial to evaluate the use of specialized inhibitory brain cells implanted as a potential reparative treatment for DRE. This minimally invasive technique involves injecting these inhibitory cells through a small, pencil eraser-sized incision at the back of the head. The goal is for these cells to integrate into the brain over time, helping to repair the neural circuitry and reducing or preventing seizures without causing the side effects associated with traditional surgical methods. Mayo Clinic's Arizona location is one of 29 sites nationwide taking part in this trial, which focuses on patients with focal epilepsy, where seizures are localized to a specific brain region.
At Mayo Clinic in Florida, another clinical trial is exploring the potential of regenerative medicine as a reparative treatment for DRE. This trial is investigating the combination of implanted stem cells and neuromodulation therapy. One of the most recent FDA-approved neuromodulation therapies for epilepsy is deep brain stimulation (DBS). While DBS has been shown to reduce seizures by up to 70% after five years, it is rare for patients to become completely seizure-free. To address this, researchers are combining DBS with stem cell therapy in hopes of enhancing the effectiveness of neuromodulation. This clinical trial involves implanting adipose-derived mesenchymal stem cells (MSCs), a type of adult stem cell with anti-inflammatory properties that may aid in healing, as an adjunct to DBS for patients with DRE.
"We've thought about this for generations, we just didn't have these technologies to enable it. Now we do," said Sanjeet Grewal, M.D., director of stereotactic and functional neurosurgery at Mayo Clinic. "So, whether it's wound healing, neurodegeneration, epilepsy or stroke, there are so many different studies going on investigating the potential of regenerative or reparative therapies."
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