Five of six patients with drug-resistant epilepsy caused by rare GPI-anchored protein deficiencies responded to purified CBD, with an 83% responder rate sustained over a median of 27 months.
Epileptologists, rare disease specialists, geneticists
83% responder rate at 12 months
What the researchers found
Six patients with genetically confirmed GPI-anchored protein deficiency and drug-resistant epilepsy received add-on CBD (Epidyolex). At 12 months, 5 of 6 (83%) were responders. No severe adverse events. Mean CBD dose was 17.85 mg/kg/day.
Why it matters
GPI-anchored protein deficiencies cause severe, drug-resistant epilepsy with few treatment options. An 83% response rate suggests a potential targeted therapy.
The numbers in context
6 patients; 83% responders at 12 months; mean dose 17.85 mg/kg/day; median treatment duration 27 months; no severe adverse events.
How the study worked
Case series of 6 patients with genetically proven GPI-anchored protein deficiency epilepsy treated with pharmaceutical-grade CBD as add-on therapy.
What this study cannot tell us
Only 6 patients. No placebo control. Ultra-rare condition limits replication possibilities.
How to read the evidence
Very small case series in an ultra-rare condition. Encouraging but cannot establish efficacy.
When this study was published
2024 publication
The bigger picture
CBD is approved for specific epilepsy syndromes, but many rare genetic epilepsies remain without evidence-based treatments. This small series suggests GPI-related epilepsy may be another responsive condition.
Questions still open
- Is there a mechanistic explanation for why GPI-deficiency epilepsy responds to CBD?
- Would larger registries confirm these response rates?
Common questions
Can CBD help with rare genetic epilepsy?
How long does CBD take to work for epilepsy?
Read the original research
Cannabidiol Add-On in Glycosylphosphatidylinositol-Related Drug-Resistant Epilepsy.
Cannabis and cannabinoid research, 9(4), 990-995
Citation
Riva, Antonella; D'Onofrio, Gianluca; Pisati, Angelica; Roberti, Roberta; Amadori, Elisabetta; Bosch, Friedrich; de Souza, Carolina Fischinger Moura; Thomas, Ashley; Russo, Emilio; Striano, Pasquale; Bayat, Allan. (2024). Cannabidiol Add-On in Glycosylphosphatidylinositol-Related Drug-Resistant Epilepsy.. Cannabis and cannabinoid research, 9(4), 990-995. https://doi.org/10.1089/can.2022.0255
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