Scientists have hailed a drug that could change the lives of children with a difficult-to-treat form of epilepsy, after promising results from early clinical trials.
Dravet syndrome is a genetic disorder that causes treatment-resistant epilepsy that is often accompanied by speech and developmental delays. Around 3,000 people are thought to suffer from the condition in the UK. Current treatments aim to control the number and severity of seizures, but they often do not work.
These preliminary trials, led by UCL and Great Ormond Street Hospital (GOSH), found that the drug appeared to be safe and well tolerated by the 81 children who took part.
Before the study, participants, ages two to 18, experienced an average of 17 seizures per month. But after taking 70 mg doses of Zorevunersen, they had on average 50% fewer seizures and about 80% fewer seizures after three doses.
The study, published in The New England Journal of Medicine, also showed improved quality of life, including motor skills, communication and coping skills.
A Phase 3 clinical trial will be conducted to study Zorevunersen over time, identify potential long-term risks and any rare but potentially serious side effects, and determine which patients are most likely to benefit.
Lead author Helen Cross, director and professor of childhood epilepsy at the UCL Institute of Child Health and honorary consultant in pediatric neurology at GOSH, said: “I regularly see patients with difficult-to-treat genetic epilepsies, who can have multiple seizures a week. Many cannot do anything independently for themselves; they require 24-hour care and are at high risk of sudden expected death in the case of epilepsy.”
If phase 3 trials are successful, he added, this new treatment “could help children with Dravet syndrome lead much healthier and happier lives.”
Epilepsy experts welcomed the findings. Jowinn Chew, a researcher at London South Bank University, said the preliminary results were a “clinically significant step forward” towards future treatment that targets the underlying cause of Dravet syndrome rather than just managing symptoms.
Dr Alfredo González-Sulser, from the Institute of Neuroscience and Cardiovascular Research at the University of Edinburgh, said the findings were “incredibly exciting” and could suggest new treatment avenues for other difficult-to-treat forms of epilepsy. “There are currently more than 800 genetic epilepsies requiring therapies similar to Zorevunersen. This establishes a clear path to effective interventions for these serious, life-altering diseases for both patients and caregivers.”
Deb Pal, professor of epilepsy at King’s College London, said the landmark study offers “enormous hope for the families of thousands of children and young people affected by monogenic epilepsies (caused by a single gene mutation) around the world.”
