Personalized gene therapy eases severe epilepsy in two boys
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A personalized gene therapy reduced seizures by over 90% in two boys with severe epilepsy, researchers reported in Nature Medicine. The treatment, tailored to each patient's genetic mutation, allowed both to resume normal activities. The results mark the first successful application of individualized gene therapy for epilepsy.
The Treatment
Researchers at the University of Texas Southwestern Medical Center designed a custom antisense oligonucleotide for each boy, targeting a specific sodium channel gene mutation. The therapy, delivered via intrathecal injection every three months, reduced seizure frequency by 93% in one patient and 97% in the other over 18 months. Both patients had previously failed multiple anti-epileptic drugs.
Clinical Outcomes
The first patient, a 7-year-old with SCN2A mutation, experienced developmental regression before treatment; after therapy, he began speaking and walking independently. The second, a 5-year-old with SCN8A mutation, achieved seizure-free periods lasting up to six months. No serious adverse events were reported, according to the study published July 24 in Nature Medicine.
What's Next
The researchers plan to expand the trial to 10 additional patients with similar mutations. It remains unclear whether the therapy will be effective for other genetic forms of epilepsy or require further customization.
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Personalized gene therapy eases severe epilepsy in two boys
