Groundbreaking development in epilepsy treatment: Experimental gene therapy reduced seizures by more than 90 percent

An experimental gene therapy applied directly to the human brain has yielded results that could radically change the quality of life of patients with drug-resistant epilepsy, according to a new study that has caused excitement in the medical world. In the study published in The New England Journal of Medicine, it was observed that the number of seizures in severe cases that could not be controlled with standard antiepileptic drugs decreased by more than 90 percent.

Around 600,000 people in the UK live with epilepsy, with around a third of patients being drug-resistant and unresponsive to standard medications, according to data from the research-focused charity Epilepsy Society. Uncontrolled electrical storms seen in these patients can lead to serious injuries, cognitive decline and an increased risk of premature death.

Within the scope of the study, surgeons injected a genetically modified virus directly into the damaged brain tissue in the method tested on three adult patients who had been living with epilepsy for 10 to 25 years due to hippocampal sclerosis. The virus is programmed to insert a specific gene into excitatory neurons, allowing surrounding neurons to bind to clozapine, a currently used drug. In this way, clozapine safely reduces the electrical firing rate of overactive cells that cause seizures.

At the end of two months, the number of monthly seizures in two of the participants decreased from 4.5 to 0.5, while the number of seizures in the third participant decreased from 4 to zero. Experts point out that, unlike standard gene therapies, thanks to this dual approach called "chemogenetics", physicians can adjust the intensity of the treatment by changing the drug dose.

Neurologist Dr. from the University of California San Diego campus. Olivia Kim-McManus stated that current treatment options require surgical cutting or laser destruction of the damaged area in the brain, and that the new technique protects the patient's brain tissue by combining surgical precision and gene therapy in a single intervention.