Science Questions

Is gene therapy for epilepsy in the clinic yet?

Tue, 13th Aug 2013

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Richard Salmon asked:

Has the UCL 'gene cure' for seizure disorders been elevated to human trials, yet?


Thank you.



Kat - Now it’s time to look at your burning genetics questions, with the help of Naked Scientist Martha Henriques.

Martha -   Listener Richard Salmon wanted to know whether the experimental epilepsy group at UCL, working on a gene cure for seizure disorders have progressed to human trials.  Their study, published last November in Science Translational Medicine, showed that introducing a genetically modified virus to a brain region which generates the epileptic seizures suppressed excitability of that region, significantly reducing the effect of seizures. 

Stephanie Schorge, an author of that study at UCL told me that the group’s application for human trials has been submitted.  She explained that the first human trials for this viral treatment will be in patients who could have the current standard treatment for this type of epilepsy in which the brain region generating the seizures is surgically removed.  Stephanie Schorge...

Stephanie -   We give maybe five patients our treatment and then if the seizures get better, great!  We’re happy.  If the seizures don’t get better, we could have that secondary treatment of having the tissue that was injected with the viruses causing seizures and having that cut out.  So in a way that's a safety thing in humans that we’re injecting tissue that we know otherwise would have been removed anyway. 

And then if it proves safe and effective in these five patients, we go to our next population which is our ultimate population for patients who have seizures coming from parts of the brain which we know cannot be cut out.  And these patients who have seizures rising say, from the motor cortex which controls an arm, if we can show that in animals injecting this virus has no effect on movement, then we think that if we inject the same virus into the human cortex it’s also unlikely to affect movement.

Martha -   Thanks to Stephanie Schorge for that answer and to Richard Salmon for his question.

Kat - If you’ve got any questions about genes, DNA and genetics you’d like us to answer, just email them to, tweet us @nakedgenetics or post on our Facebook page.


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I think you're referring to this study:

As Stephanie Schorge, the author and interviewee, highlights, clinical trials are a goal but not a reality yet. I am also unaware of any other groups around the world doing this yet, but if anyone hears different, please do say.

Chris chris, Fri, 19th Jul 2013

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