What is Epidiolex?
Epidiolex is a strawberry flavored, oral liquid medication taken twice daily that has been proven to help reduce the frequency of seizures in people suffering from two rare, severe forms of epilepsy: Lennox-Gastaut syndrome and Dravet syndrome. The drug comes directly from the cannabis sativa, or marijuana, plant itself. It is a highly purified liquid formulation of a component of the plant called cannabidiol, or CBD. It does not have any of the psychoactive properties of marijuana.
How does it work?
Preclinical data suggests that there’s an interaction between cannabidiol and brain receptors that produces an anti-seizure effect. Seizures can come from the temporal lobe, and these brain receptors might be in higher concentration in the area called the hippocampus, a very small area deep inside the temporal lobe. Some studies have shown that there is a higher density of brain receptors that specifically respond to cannabidiol in people with injured hippocampi, suggesting a possible reason for the effectiveness of CBD therapies in people with epilepsy.
There may also be what we call an additive, or synergistic, effect when combined with other medications. Cannabidiol inhibits a process in the liver that breaks down other, traditional anti-seizure medications. So, when you include cannabidiol and other anti-convulsant regimens, it may increase the levels of other seizure medications, perhaps making them more effective.
How will this new drug help children with these severe forms of epilepsy?
Dravet and Lennox-Gastaut are childhood epilepsy syndromes that are tough to treat. Many of these patients have weekly or daily clusters of seizures, and some have “drop seizures,” when they just suddenly drop without warning, creating a high chance of injury. Many of these kids have to wear helmets for this reason.
These conditions impact their ability to do basic daily activities like bathing, feeding, and dressing. There’s also a social stigma. They have to have additional equipment that other children don’t have. So, if there is a reduction in seizures, this may help impact other outcomes, such as better motor and neurologic development.
Does the new medication have side effects?
It’s a little early to know the long-term implications of cannabidiol. Side effects from the studies have shown an elevation in liver enzymes that stopped once the medication was removed, and other mild reactions such as fatigue or decrease of appetite. In the short term, it seems both effective for reducing seizures and relatively safe.
Are there any current medications that are effective?
There are several treatments that work to reduce the frequency of seizures, but there’s no one drug that has proven to be highly effective. Most children with this condition are what we call medication resistant and continue to have seizures.