Understanding Catastrophic Epilepsy in Children
Children with severe forms of epilepsy often experience seizures multiple times each week, or even daily, despite being on several anti‑seizure medications. For families, the relentless cycle of seizures creates anxiety and a urgent need for additional treatment options.
Why Conventional Treatments Often Fall Short
Standard antiepileptic drugs work for many patients, but in certain epilepsy syndromes the brain’s electrical activity remains resistant to these therapies. When seizures persist, they can impair cognitive development, reduce quality of life, and increase the risk of injury or sudden unexpected death in epilepsy (SUDEP).
Cannabidiol as a Potential Option
Interest in cannabidiol (CBD) grew after anecdotal reports suggested that a non‑psychoactive compound from the cannabis plant could reduce seizure frequency. Parents began seeking CBD oil from dispensaries or online retailers, but the potency and purity of these products are inconsistent. The U.S. Food and Drug Administration (FDA) has warned that many over‑the‑counter CBD preparations contain little or no CBD, and some may pose risks such as liver enzyme elevations or drug interactions.
The FDA‑Approved CBD Medication: Epidiolex
In June 2018 the FDA granted approval to Epidiolex, a purified cannabidiol oral solution developed by GW Pharmaceuticals (marketed in the United States by Greenwich Biosciences). This marked the first time a cannabis‑derived medication received FDA approval for a medical indication in the United States. Epidiolex is specifically indicated for patients aged two years and older who have seizures associated with Dravet syndrome or Lennox‑Gastaut syndrome—two rare, severe forms of childhood‑onset epilepsy.
How Epidiolex Works: Mechanism of Action
Although the exact mechanisms are still under investigation, research suggests that CBD interacts with neuronal receptors that are not targeted by traditional anti‑seizure drugs. Dr. Orrin Devinsky, director of NYU Langone’s Comprehensive Epilepsy Center, notes that CBD appears to modulate the GPR55 receptor, a pathway implicated in seizure generation. By blocking this receptor, CBD may help break the positive feedback loop that amplifies seizure activity.
Clinical Evidence: Dravet and Lennox‑Gastaut Syndromes
Two pivotal randomized, double‑blind, placebo‑controlled trials provided the data that supported FDA approval.
- Dravet syndrome study (NEJM, 2017) – 120 children and young adults received either 20 mg/kg of CBD per day or placebo, in addition to their usual anti‑seizure regimen. Over 14 weeks, median convulsive seizures dropped from about 12.6 to 5.9 per month in the CBD group, compared with a change from 14.9 to 14.1 per month in the placebo group. Five percent of participants in the CBD arm became seizure‑free, while none in the placebo group did. Common side effects included diarrhea, vomiting, fatigue, decreased appetite, and transient elevations in liver enzymes.
- Lennox‑Gastaut syndrome study (NEJM, 2018) – 250 participants aged 2–55 years were assigned to low‑dose CBD, high‑dose CBD, or placebo. The high‑dose group experienced a 42 % reduction in drop seizures, the low‑dose group a 37 % reduction, and the placebo group a 17 % reduction. Discontinuation due to adverse events occurred in six patients receiving the high dose and one receiving the low dose.
Expanding Research: Other Epilepsy Types
Beyond the syndromes for which Epidiolex is approved, investigators have explored CBD’s effect in broader, treatment‑resistant epilepsies. Dr. Martina Bebin of the University of Alabama at Birmingham led an expanded‑access study of 132 adults and children with various epilepsy genotypes. Over 48 weeks, average seizure frequency fell from 144 to 52 seizures per two‑week period, and seizures tended to be less severe. These findings, published in Epilepsy & Behavior (October 2018), suggest that CBD may benefit a wider population, although further controlled trials are needed.
Cost, Access, and Safety Considerations
The list price for Epidiolex is approximately $32,500 per year. Many insurance plans and Medicare Part D provide coverage for patients with the approved indications, and manufacturer‑sponsored assistance programs can help reduce out‑of‑pocket expenses. Because CBD is metabolized by the liver, clinicians typically monitor liver function tests before and during treatment, especially when patients are taking other medications that share hepatic pathways.
What Lies Ahead for CBD in Epilepsy Care
Early data indicate that Epidiolex may also reduce seizures in patients with tuberous sclerosis complex, prompting ongoing studies to evaluate its utility in additional genetic epilepsies. Experts caution that CBD is not a cure‑all; a substantial proportion of patients continue to experience seizures despite treatment. Nevertheless, the availability of a pharmaceutical‑grade, FDA‑approved CBD product offers families a more reliable alternative to unverified CBD oils.
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