Experimental brain gene therapy cuts seizures by more than 90% in three patients
In an early-stage trial reported in The New England Journal of Medicine, three adults with drug-resistant epilepsy had their monthly seizures fall by more than 90% after gene-carrying viruses were injected into the brain tissue where seizures began. The Nature report says the treatment uses clozapine to activate engineered receptors in targeted neurons; no serious side effects were reported, but a neurologist not involved in the study cautioned that the trial was very small and flagged concerns about long-term clozapine use.
Bottom line — Three patients saw seizures fall by more than 90%, but larger studies must test the approach and its reliance on clozapine.
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The participants had scarring on one side of the hippocampus and had been diagnosed with epilepsy 10–25 years earlier, Nature reports.
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All three had developed resistance to at least two anti-seizure medicines and averaged at least two seizures a month before the trial, according to Nature.
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The treatment used adeno-associated virus 9 to deliver a gene that makes targeted excitatory neurons produce a receptor activated by clozapine, Nature explains.
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University College London neurologist Matthew Walker, who was not involved, said the result was striking but stressed the small sample and concerns about long-term clozapine use.
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Chemical & Engineering News describes this as the first human use of designer receptors, known as DREADDs, to alter an adult brain’s activity.
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