A new experimental drug has been identified that dramatically reduces seizures in children with rare Dravet syndrome, offering hope for improved quality of life and advancing future treatment options
A new experimental drug has been shown to dramatically reduce seizures in children with rare Dravet syndrome, offering hope for improved quality of life. Early trials led by Great Ormond Street Hospital and UCL found the treatment cut seizures by up to 91% and showed potential benefits for behaviour and development.
The findings, published in The New England Journal of Medicine, pave the way for larger phase three studies aimed at confirming long‑term benefits.
What is Dravet syndrome?
Dravet syndrome is a genetic condition which causes frequent, hard-to-control seizures and long-term neurodevelopmental impairment. The condition causes feeding difficulties, movement problems, and has a high risk of premature death. Current treatments do not control seizures in most patients, and there are no approved medicines that tackle the cognitive and behavioural impacts.
How does zorevunersen work?
Following early trials, the researchers found that zorevunersen, produced by Stoke Therapeutics in collaboration with Biogen, targeted the underlying cause of the disease: a faulty gene.
Humans typically have two copies of the SCN1A gene, and in most people with Dravet syndrome, one copy produces insufficient protein for their nerve cells to function properly.
Zorevunersen works by increasing the levels of the protein produced by the healthy SCN1A gene, aiming to restore proper nerve-cell function.
Lead author Professor Helen Cross, Director and Professor of Childhood Epilepsy at the UCL Great Ormond Street Institute of Child Health and an Honorary Consultant in Paediatric Neurology at GOSH, said: “I regularly see patients with hard-to-treat genetic epilepsies with impacts that go beyond seizures, and it’s heartbreaking 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.”
Trial results and patient stories
The trial involved 81 children aged between two and 18 years, and patients had an average of 17 seizures per month before the trial started. 19 of the trial participants were patients at a UK hospital, and the trial was expanded to include patients worldwide.
The 81 children were given up to 70mg of zorevunersen by lumbar puncture, either as a single dose or with additional doses two or three months later over a six-month period. Of those patients, 75 went on to take part in extension studies. Those patients continued to receive the drug every four months.
Dravet Syndrome UK Chair of Trustees, Galia Wilson, said: “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.”
6-year-old Jorge, from Las Palmas de Gran Canaria, was diagnosed with Dravet syndrome when he was just five months old after having seizures. Dad Juan said taking part in the trial at GOSH had “drastically” changed Jorge’s life for the better.
Dad Juan said, “Jorge was really susceptible to the smallest thing triggering his seizures, such as a cold wind, whereas since taking part in the trial, he can now jump into the cold sea!”
Jorge began participating in the trial and received his first dose in January 2023.
Dad Juan continued: “Taking part in the trial has been life-changing. We now have a normal family life. It’s given Jorge his childhood back, and he can take part in activities like his friends. We’re still careful and monitor his temperature, as previously, even a small change could trigger a seizure, but we’re no longer on edge. He can go to school by himself and stay with family members. We also decided to have a second baby, who’s now two years old, as Jorge is doing so well.”
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