An “encouraging” new medication, zorevunersen, appears safe and effective as a treatment for Dravet syndrome according to a new study in The New England Journal of Medicine.
A total of 81 children from the UK and the US aged 2-18 years entered the study and received one, two or three doses of zorevunersen over a three-month period.
Of these, 75 children went on into an extension study, continuing to have 45mg of the medication every four months for 20 months.
On average, the number of convulsive seizures in the children reduced by between 59% and 91%.
In this early trial, the researchers could see improvement in the children’s health, quality of life and adaptive behaviour.
Epilepsy Action’s health improvement and research manager, Tom Shillito, said: “This is an encouraging early result particularly given the urgent need for better treatments for children with Dravet syndrome.”
Dravet syndrome affects around one in 15,000 children. It tends to be difficult to treat, as seizures in this condition often don’t respond very well to current epilepsy medicines, the organisation says.
Zorevunersen was delivered through a lumbar puncture to combat the underlying cause of Dravet syndrome. The condition is often caused by a faulty SCN1A gene which means that there is too little of a particular protein produced which helps nerve cells to work properly. The medication helps to increase production of this protein.
The study authors Dr Linda Laux and colleagues found that in their research, the medication’s side effects were mild or moderate.
They conclude that the medication has the potential to be disease-modifying, meaning it doesn’t just treat the symptoms, but can reduce the progression of the condition, and warrants more research and development.
Healthier and happier lives
One family who took part in the trial said the effects of the medication have “completely changed our lives”.
Freddie Truelove from Huddersfield went from 12 seizures a night to one or two short seizures every few nights. His mum, Lauren, said: “We now have a life we didn’t ever think was possible and, most importantly, it’s a life that Freddie can enjoy.”
Consultant paediatric neurologist and study author Helen Cross 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.”
Tom Shillito added: “We regularly receive enquiries to our helpline from affected families and know just how challenging and heart-breaking it can be caring for a child with Dravet syndrome.
“The study suggests this targeted approach could make a meaningful difference and give families greater options in treatment and more positive outcomes for their child. We look forward to seeing further research on this drug and similar drugs.”
Find out more about Dravet syndrome on the Epilepsy Action website.