In 3 infants with KCNT1-related epilepsy of infancy with migrating focal seizures, pharmaceutical CBD produced no overall seizure frequency reduction, though one patient showed notably reduced seizure intensity and possible developmental gains.
Pediatric epileptologists, families of children with KCNT1 mutations, and rare disease researchers.
1 of 3 patients showed reduced seizure intensity with CBD
What the researchers found
Three patients with EIMFS secondary to KCNT1 mutations received pharmaceutical-grade CBD. Two patients showed no benefit and discontinued treatment. One patient showed no overall seizure frequency reduction but had notable reduction in seizure intensity and possible developmental progression. The results contrast with CBD efficacy in Dravet and Lennox-Gastaut syndromes.
Why it matters
EIMFS is a devastating rare epilepsy with almost no treatment options. While results were mostly negative, the one partial responder suggests that seizure intensity (not just frequency) may be a meaningful outcome measure for CBD.
The numbers in context
3 patients with KCNT1 mutations; 2 showed no benefit; 1 showed reduced seizure intensity but not frequency; the responder also had possible developmental progression.
How the study worked
Open-label, prospective interventional study of 3 patients with EIMFS caused by KCNT1 mutations treated with pharmaceutical-grade CBD as adjunctive therapy.
What this study cannot tell us
Only 3 patients (no statistical power); open-label; no control group; mixed results; EIMFS is extremely rare, making large studies difficult; seizure intensity assessment is subjective.
How to read the evidence
Preliminary: only 3 patients in an uncontrolled open-label study, though the rare disease context limits feasibility of larger trials.
When this study was published
Published 2020.
The bigger picture
CBD mechanism likely differs by epilepsy subtype. The lack of response in KCNT1-related epilepsy (a potassium channelopathy) contrasts with Dravet syndrome (sodium channel), suggesting CBD anti-seizure effects may be channel-specific.
Questions still open
- Does the seizure mechanism (sodium vs potassium channel) determine CBD responsiveness? Should seizure intensity be a primary outcome measure for refractory epilepsy trials?
Common questions
Does CBD work for all types of epilepsy?
What is EIMFS?
Read the original research
Response to cannabidiol in epilepsy of infancy with migrating focal seizures associated with KCNT1 mutations: An open-label, prospective, interventional study.
European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society, 25, 77-81
Citation
Poisson, Kelsey; Wong, Matthew; Lee, Chon; Cilio, Maria Roberta. (2020). Response to cannabidiol in epilepsy of infancy with migrating focal seizures associated with KCNT1 mutations: An open-label, prospective, interventional study.. European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society, 25, 77-81. https://doi.org/10.1016/j.ejpn.2019.12.024
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