Great Ormond Street Hospital in London – credit, Fast Track Images, CC BY-SA 2.0.
A phase 2 clinical trial in England has demonstrated smashing success in improving the conditions of children with a rare form of treatment-resistant epilepsy called Dravet syndrome.
Believed to affect 3,000 children in England, it’s just one out of hundreds of forms of genetic epilepsy that have no pharmacological options.
Scientists not involved with the study said the success of the drug, called Zorevunersen, shows that pharmaceuticals can work to improve the lives of patients with these epilepsies, giving it a chance to spur innovation toward other treatments and cures.
The trial involved 81 children between the ages of 2 and 18 that suffered up to 18 seizures a month resulting from Dravet syndrome. Zorevunersen was given in 3 doses at Great Ormond Street Hospital, where the trial was co-organized by University College London.
After 1 dose, monthly seizures were reduced by 50%. After 3 doses, that improved to 80%. The drug was well-tolerated by all 81 patients, with no significant side effects reported.
Additionally, this reduction in seizures led to real improvements in life quality for the patients. Being children, it led to improved development outcomes, particularly with motor skills and communication. It also improved their markers for “coping” with Dravet syndrome.
The trail was led by Helen Cross, director and professor of childhood epilepsy at the UCL Institute of Child Health, and consultant pediatric neurologist at Great Ormond.
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“I regularly see patients with hard-to-treat genetic epilepsies, who can have multiple seizures a week,” said Professor Cross. “Many are unable to do anything independently for themselves; they require around the clock care and are at high risk of sudden expected death in epilepsy.”
She’s hopeful that Zorevunersen “could help children with Dravet syndrome lead much healthier and happier lives,” and in pursuit of that end, is organizing a phase 3 trail that will study the drug over a much longer period of time to control for potentially serious side effects.
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“There are now over 800 genetic epilepsies that need therapeutics similar to Zorevunersen,” the uninvolved Dr. Alfredo Gonzalez-Sulser from the Institute for Neuroscience and Cardiovascular Research, University of Edinburgh, told the Guardian.
“This sets a clear path to achieve effective interventions for these severe life-altering diseases for both patients and carers.”
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