A new clinical study published in the scientific journal The New England Journal of Medicine suggests that an experimental treatment can significantly reduce epileptic seizures in children with Dravet syndrome.
According to researchers from University College London, the number of seizures in some patients fell by up to 91 percent after treatment with the drug zorevunersen.
Based on the initial results, a larger phase 3 study has now been launched in which the treatment will be tested on more patients.
The researchers report that most side effects observed during the trial were mild and that the treatment was generally well tolerated by the participants.
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Trial with 81 children
The study included 81 children and adolescents between the ages of 2 and 18 diagnosed with Dravet syndrome.
Before treatment began, the participants experienced an average of about 17 epileptic seizures per month.
Patients received up to 70 milligrams of the medication through a lumbar puncture in the lower back over a six-month period.
Afterwards, 75 of the participants continued in a follow-up study in which the treatment was administered approximately every four months.
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During the follow-up period, researchers recorded a reduction in seizures of between 59 and 91 percent among patients who received the highest dose.
The genetic cause of the disease
Dravet syndrome is a rare genetic disease that causes frequent and severe epileptic seizures from early childhood.
The disease is linked to errors in the SCN1A gene, which plays a role in how nerve cells send signals in the brain.
According to the researchers, zorevunersen has been developed to increase the production of an important protein from the healthy copy of the gene.
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The aim is to improve signaling between nerve cells and thereby reduce symptoms.
The researchers emphasize that the results are still preliminary and that larger studies are needed to confirm the effect.
Sources: Science Daily, and New England Journal of Medicine.
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