Scientists laud life-changing drug for children with resistant form of epilepsy | Epilepsy
Scientists have welcomed a potentially life-changing drug for children with a difficult-to-treat form of epilepsy after promising early clinical trial results.
Dravet syndrome It is a genetic disease that causes treatment-resistant epilepsy and is often accompanied by speech and developmental delays. Around 3,000 people in the UK are thought to have the condition. Current treatments aim to control the number and severity of seizures but often do not work.
These preliminary trials, conducted by UCL and Great Ormond Street Hospital (GOSH), found that the drug appeared safe and was well tolerated by the 81 children who took part.
Before the study, participants aged between 2 and 18 had an average of 17 seizures per month. But after taking 70 mg of Zorevunersen, they had, on average, 50% fewer seizures, and after three doses, they had about 80% fewer seizures.
Study, Published in the New England Journal of MedicineIt also showed improved quality of life, including motor skills, communication, and coping ability.
A phase 3 clinical trial will be conducted to study Zorevunersen over time, identify possible long-term risks and 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 epilepsy who may have more than one seizure per week. Many are unable to do anything independently; they need 24-hour care and the expected risk of sudden death from epilepsy is high.”
He added that if Phase 3 trials are successful, this new treatment “could help children with Dravet syndrome live much healthier and happier lives.”
Epilepsy experts celebrated the findings. Researcher Jowinn Chew, of London South Bank University, said the preliminary results were a “clinically important step” towards a future treatment that targets the underlying cause of Dravet syndrome rather than just managing symptoms.
Dr. from the Institute of Neuroscience and Cardiovascular Research at the University of Edinburgh. Alfredo Gonzalez-Sulser said the findings were “incredibly exciting” and could suggest new treatments for other difficult-to-treat forms of epilepsy. “There are currently more than 800 cases of genetic epilepsy needing treatments similar to Zorevunersen. This opens a clear path for both patients and caregivers to achieve effective interventions for these serious life-changing diseases.”
Deb Pal, professor of epilepsy at King’s College London, said the groundbreaking study gave “tremendous hope for the families of the thousands of children and young people affected by monogenic epilepsy”. [caused by a single gene mutation] epilepsies worldwide”.
