New epilepsy treatment offers hope to children with Dravet syndrome

A new epilepsy treatment for children with Dravet Syndrome, a genetic condition that causes frequent, hard-to-control seizures and long-term neurodevelopmental impairment, has been shown to reduce seizures by 91%.

The new medication called zorevunersen works by addressing the root cause of the disease – the SCN1A gene. In most people with Dravet syndrome, one copy of this gene doesn’t produce enough of a protein for their nerve cells to function properly.

In a new study published in The New England Journal of Medicine, the researchers discovered that zorevunersen could potentially lessen the impact of the condition on a child’s mental processes and behaviour. The children’s quality of life also improved over a three-year period, and most of the treatment’s side effects were mild.

What is Dravet syndrome?

Dravet syndrome affects approximately one in 15,000 children. It also leads to feeding difficulties, movement issues, and carries a high risk of premature death. Current treatments generally fail to control seizures in most patients, and there are no approved medicines that tackle the condition’s cognitive and behavioural effects.

The researchers from UCL and Great Ormond Street Hospital concluded that zorevunersen has the potential to be disease-modifying, meaning it doesn’t just treat the symptoms but can also reduce the progression of the condition, and it warrants further research and development.

Zorevunersen was administered via a lumbar puncture to 81 children with Dravet syndrome in the UK and the US. Patients in these studies experienced an average of 17 seizures per month before the trials commenced.

The 81 children received up to 70mg of zorevunersen either as a single dose or with additional doses two or three months later over a six-month period. Of those patients, 75 participated in extension studies. Those patients continued to receive the drug every four months.

Patients given a 70mg dose in the initial phase of the trial saw their seizures reduce by between 59% and 91% over the first 20 months of the extension studies compared with the number of seizures they were having before the trial started.

Heartbreaking when epilepsy treatments are limited

Lead author Professor Helen Cross, Director and Professor of Childhood Epilepsy at the UCL Great Ormond Street Institute of Child Health, said: “I regularly see patients with hard-to-treat genetic epilepsies with impacts that go beyond seizures and it’s heart-breaking when treatment options are limited. This new treatment could help children with Dravet syndrome lead much healthier and happier lives.

“Overall, our findings showed that zorevunersen is safe to use and well tolerated by most patients and supports further evaluation in the ongoing Phase Three study.”

Dravet Syndrome UK Chair of Trustees, Galia Wilson, added: “We regularly see the devastating impact that this condition has on the lives of families. That’s why we’re so thrilled about these latest results from the initial zorevunersen clinical trials.

“We’re now looking forward to the Phase Three clinical trials taking place to see if the early promise we see here will translate into real hope for all those families currently affected by Dravet syndrome.”

Alison Bloomer

Alison Bloomer is Editor of Learning Disability Today.

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