A promising experimental gene therapy for the treatment of drug-resistant epilepsy has yielded remarkable results in a small-scale clinical trial. According to the research published in The New England Journal of Medicine, the method, applied directly to the brain, reduced the monthly seizure frequency by over 90 percent in three adult patients.
The study was conducted on patients whose seizures could not be controlled with standard antiepileptic drugs and who had been living with epilepsy for 10 to 25 years due to hippocampal sclerosis. In the study, surgeons injected a genetically modified virus into the damaged area of the brain.
In the approach called chemogenetics, the virus was programmed to carry a specific gene to excitatory neurons. This enabled these neurons to respond to clozapine, a drug already in use. According to the researchers, this mechanism aims to reduce the electrical activity of overactive cells that lead to seizures.
At the end of a two-month follow-up, the monthly seizure count in two participants dropped from 4.5 to 0.5. In the third participant, it was reported that the number of seizures, which was 4 per month, fell to zero. These results have raised the possibility of a new treatment avenue, especially for patients who do not benefit from existing medications.
CAUTIOUS OPTIMISM FROM EXPERTS
Experts state that the method could offer a more conservative approach compared to options based on removing or laser-ablating brain tissue. The ability to adjust the treatment effect via the drug dose in the chemogenetic method is also considered a significant advantage.
However, it is emphasized that the results are still in the early stages because the research was conducted on only three patients. Furthermore, risks such as potential cardiovascular side effects that may arise from long-term and high-dose use of clozapine need to be monitored. The researchers plan to follow the patients in the long term and test the method with larger clinical trials.
News Source: 12punto
