Wonder If: Precision Ketogenic Therapy Is The Needed Proof Of Principle Required To Document Long Term Efficacy Of Using Food As Medicine For Seizures

December 3, 2023




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Precision Ketogenic Therapy Is The Needed Proof Of Principle Required To Document Long Term Efficacy Of Using Food as Medicine For Seizures

This paper published in January 2024, in the European Journal of Pediatric Neurology on behalf of Network for Therapy in Rare Epilepsies (NETRE) is encouraging a precision medicine approach to the use of ketogenic therapy.

Is ketogenic diet a ‘precision medicine’? Recent developments and future challenges  

R. Falsaperla, V. Sortino, P. Striano, G. Kluger, G. Ramantani, M. Ruggieri and E.

Network for Therapy in Rare Epilepsies (NETRE)

Eur J Paediatr Neurol 2023 Vol. 48 Pages 13-16  

Accession Number: 37984007 DOI: 10.1016/j.ejpn.2023.11.002 


Recently, precision medicine has attracted much attention in the management of epilepsies, but it remains unclear if the increasingly utilized ketogenic diet approaches can truly be considered precision medicine in all epilepsy treatments.
Ketogenic diet is presumed to be a precision medicine tool when applied to the treatment of seizures secondary to GLUT1 transporter deficiency Precision medicine refers to a multidisciplinary approach utilizing genetic profiling, environmental and lifestyle information (one’s dietary pattern is an integral component of one’s environmental and lifestyle information) to determine the optimal path either to prevention or to treatment of a specific disease.

The paper emphasizes that most studies use ketogenic therapy for a matter of months, but patients will need a longer-term therapy. 

Notably, most RCTs have evaluated treatment efficacy at 3–4 months after KD initiation, whereas only one RCT focused on long-term (16-month) outcome in 48 children. (We have patients with a variety of diagnoses accompanied by seizures who have been on PKT for more than 2 decades.)
Robust data on the efficacy, side effects, and adherence to long-term ketogenic diet therapy (are needed).
Future directions should involve the use of precision medicine, applied to each patient with the help of “omics”, whose use should be expanded and inclusive.

This paper focuses on the genetic component of precision medicineWhen studying a medication, the chemical identity and quantity of the active compound being studied is well defined. It would be unacceptable to not define both the identity and the dose of the medication.  However, the chemical composition of the ketogenic diets, the nutritional state of the patient and the design of the ketogenic diet to meet nutritional needs of the patient are frequently not well defined.  


  • As of December 2023, we have 16 patients who have been on PKT for 5 years to more than 20 years with improvement in seizure control and/or reduced need for antiseizure medications with controllable side effects.  Wonder if this is enough proof of principle to move to scaling up the use of PKT? 
  • Wonder if our small group of adult patients on PKT is adequate to mobilize resources to scale up the Adult PKT program? 
  • The genetics component of precision medicine is important, but it is also critical that we precisely know the identity and quantity of chemicals in the food used for PKT. Wonder if the small sample size that we have treated using manual calculation methods are sufficient to move to more automated methods? 
Post by Paige Applegate