CBD Reduced Seizures in 5 of 6 Patients With a Rare Genetic Epilepsy
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.
Quick Facts
What This Study 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.
Key Numbers
6 patients; 83% responders at 12 months; mean dose 17.85 mg/kg/day; median treatment duration 27 months; no severe adverse events.
How They Did This
Case series of 6 patients with genetically proven GPI-anchored protein deficiency epilepsy treated with pharmaceutical-grade CBD as add-on therapy.
Why This Research Matters
GPI-anchored protein deficiencies cause severe, drug-resistant epilepsy with few treatment options. An 83% response rate suggests a potential targeted therapy.
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.
What This Study Doesn't Tell Us
Only 6 patients. No placebo control. Ultra-rare condition limits replication possibilities.
Questions This Raises
- ?Is there a mechanistic explanation for why GPI-deficiency epilepsy responds to CBD?
- ?Would larger registries confirm these response rates?
Trust & Context
- Key Stat:
- 83% responder rate at 12 months
- Evidence Grade:
- Very small case series in an ultra-rare condition. Encouraging but cannot establish efficacy.
- Study Age:
- 2024 publication
- Original Title:
- Cannabidiol Add-On in Glycosylphosphatidylinositol-Related Drug-Resistant Epilepsy.
- Published In:
- Cannabis and cannabinoid research, 9(4), 990-995 (2024)
- Authors:
- Riva, Antonella(4), D'Onofrio, Gianluca(2), Pisati, Angelica, Roberti, Roberta, Amadori, Elisabetta, Bosch, Friedrich, de Souza, Carolina Fischinger Moura, Thomas, Ashley, Russo, Emilio, Striano, Pasquale, Bayat, Allan
- Database ID:
- RTHC-05655
Evidence Hierarchy
Frequently Asked Questions
Can CBD help with rare genetic epilepsy?
In this small study, 5 of 6 patients with epilepsy caused by GPI-anchored protein deficiency responded to purified CBD, with sustained seizure reduction over a median of 27 months.
How long does CBD take to work for epilepsy?
Response was assessed at 12 months, with all 5 responders maintaining benefit through the median 27-month follow-up. CBD was titrated from 2 mg/kg/day up to a median of 25 mg/kg/day.
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Cite This Study
https://rethinkthc.com/research/RTHC-05655APA
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
MLA
Riva, Antonella, et al. "Cannabidiol Add-On in Glycosylphosphatidylinositol-Related Drug-Resistant Epilepsy.." Cannabis and cannabinoid research, 2024. https://doi.org/10.1089/can.2022.0255
RethinkTHC
RethinkTHC Research Database. "Cannabidiol Add-On in Glycosylphosphatidylinositol-Related D..." RTHC-05655. Retrieved from https://rethinkthc.com/research/riva-2024-cannabidiol-addon-in-glycosylphosphatidylinositolrelated
Access the Original Study
Study data sourced from PubMed, a service of the U.S. National Library of Medicine, National Institutes of Health.
This study breakdown was produced by the RethinkTHC research team. We analyze and report published research findings without making health recommendations. All interpretations are based solely on the published abstract and study data.