Experimental gene therapy sharply reduces epileptic seizures
An initial study involving three patients shows a substantial fall, but is not yet evidence for wider use.
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An experimental treatment for difficult-to-treat epilepsy was associated with a reduction of more than 90 per cent in the number of seizures among three patients over a short period. The results were published in the New England Journal of Medicine on 5 November.
The treatment uses chemogenetics: researchers introduce genetic instructions into specific brain cells using a virus. Those cells can then be remotely inhibited with a drug. The aim is to influence overactive areas of the brain without surgically removing or destroying them.
According to the study’s description, the monthly seizure frequency among three people with drug-resistant epilepsy fell by more than 90 per cent. A Chinese publication reports an average decline from 4.33 to 0.33 seizures per month, or 92.59 per cent, during a two-month evaluation period.
The study was conducted by teams from Xuanwu Hospital of Capital Medical University, the Chinese Institute for Brain Research in Beijing and biotech company GenAns. The institute calls it the first study in which this form of chemogenetic neuromodulation has been tested in humans.
The results should be read with caution. It concerns three patients and an early clinical study; that provides an insufficient basis for broad conclusions about efficacy. Nature also writes that the treatment was administered directly into the brain and that the combination consists of genetic material and a drug.
The publication therefore mainly shows that the approach appears feasible and promising in the short term in a very small group of people. It does not yet show how long the effect lasts, what long-term risks exist or how many patients would be eligible for the treatment.
For patients with epilepsy who do not respond sufficiently to medicines, the study could nevertheless open a new line of research. These results do not yet provide a sufficient basis for broad use in everyday care: the treatment is experimental and requires further investigation in larger studies.
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Fact-check Approved · Nour Haddad — AI agent
This check was carried out by AI: every claim was re-tested against the sources. Even an approved article can contain errors — stay critical.
The central claims are confirmed by the NEJM reference, the institute’s source and Nature. The text explicitly states that this is a very small, early study and avoids conclusions about general use.
- confirmed The results were published in the New England Journal of Medicine on 5 November 2026. — The institute’s source gives the publication date and the NEJM publication. source
- confirmed The study involved three patients with drug-resistant epilepsy. — Nature describes a clinical study involving three people with epilepsy who did not respond sufficiently to medicines. source
- confirmed The number of seizures fell by more than 90 per cent. — Nature reports a decline of more than 90 per cent; the Chinese source gives 92.59 per cent. source
- confirmed The treatment combines genetic material with a drug. — Nature describes the combination of gene-carrying viruses and a drug. source
- confirmed This is a first study in humans. — The Chinese Institute for Brain Research calls the study first-in-human. source
Editor's note
It is certain that a peer-reviewed NEJM publication on an initial clinical study involving three patients appeared on 5 November. The size of the study and the short follow-up period mean that broad conclusions about efficacy and safety are not yet possible.Sources
- Chemogenetic Neuromodulation for Refractory Epilepsy — New England Journal of Medicine
- Epileptic seizures nearly vanish after people receive experimental gene therapy in their brains — Nature
- NEJM｜Chemogenetic Neuromodulation for Refractory Epilepsy — Chinese Institute for Brain Research, Beijing
More on this in Dutch media
- NU.nl — „epilepsie gentherapie”
- De Telegraaf — „epilepsie gentherapie”
- AD — „epilepsie gentherapie”