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Elucidation of the biochemical mechanisms responsible for the efficacy of the Ketogenic Diet

The ketogenic diet has only recently been shown to definitely be effective where drugs have failed, with the first randomised controlled trial in children age 2 to 16 years published by our group in 2008.

In addition, the same study demonstrated reduction in antiepileptic drugs where the diet was successful. However, the exact mechanism of action of the diet is still not known. Consequently, patients are required to consume a particularly difficult diet in order to retain seizure control.

Based on our previous considerable experience of mitochondrial metabolism, we propose that the ketogenic diet exerts its beneficial effects via medium chain fatty acids.


To determine changes seen in medium chain fatty acids in children treated with the ketogenic diet, and determine their possible role in the underlying mechanism of effect.

Outline plan

Blood samples of patients who have received, or are currently receiving the ketogenic diet will be analysed to see whether there are any similarities in those who have responded well to treatment and those who have not. The research team will assess the plasma medium chain fatty acid profile in the patients.


Blood from participants recruited from Great Ormond Street Hospital Ketogenic Diet Clinic is now being assessed for medium chain fatty acids. The possibility of a clinical trial is under discussion.


Sean Hughes, Helen Cross, Simon Heales.

Cyber EssentialsFundraising RegulatorYoung Epilepsy is the operating name of The National Centre for Young People with Epilepsy (NCYPE).
Registered Charity number 311877 (England and Wales)

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