GW Pharmaceuticals, the British maker of marijuana distillate Sativex, is in clinical trials now for its new cannabis anti-seizure drug, Epidiolex. Cannabis has already been shown to be an immensely effective treatment for epilepsy and seizures. Additional research has also shown the CBD, specifically, is a potent treatment for epilepsy. GW’s formulation into an easy-to-use, safe and prescribable drug is a huge step forward for the cannabis industry.
According to the company website, the active ingredient in Epidiolex is cannabidiol (CBD), the most potent non-psychoactive component in marijuana.
The drug is in clinical trials to treat intractable epilepsy and other seizure disorders, including Dravet syndrome, Lennox-Gastaut syndrome, tuberous sclerosis complex, and infantile spasms.
These are all rare types of epilepsy disorders that cause seizures.
What is Dravet syndrome?
Dravet syndrome is a seizure disorder that begins in an infant’s first year and lasts the rest of their lives. The child’s development is normal until the onset of seizures, and because of this, it’s often misdiagnosed.
Charlotte Figi, the brave little girl with Dravet syndrome, helped guide the public perception towards recognizing cannabis as a safe and effective treatment method for Dravet syndrome. Charlotte’s Web, the now famous high-CBD strain, is named in her honor.
Kids with Dravet usually have developmental problems resulting from the syndrome.
Dravet is linked to a mutation on the SC1NA gene that affects the how the brain’s ion channels operate. Ion channels are pores in nerve cell membranes that control the flow of charged molecules in and out of the cell, thus regulating the electrochemical nerve signal.
This genetic abnormality isn’t typically inherited; it results from a mutation.
Dravet is resistant to standard anti-seizure medications, which is why GW is targeting it with the new CBD drug.
What is tuberous sclerosis complex and infantile spasms?
Tuberous sclerosis complex that causes infantile spasms is a genetic abnormality that causes noncancerous tumors to grow in different parts of the body.
Tumors in the brain are what causes seizures in babies (infantile spasms). Tuberous sclerosis complex causes other health problems, as well as developmental delays. Tumors can also arise in the skin, eyes, kidneys, heart and lungs.
What is Lennox-Gastaut syndrome?
Lennox-Gastaut syndrome is a particularly nasty form of childhood epilepsy. It’s marked by frequent violent seizures which can lead to injuries, and which can’t be controlled by existing medication.
The syndrome also comes with a host of developmental problems, including psychiatric disorders, cognitive deficits, and behavioral problems.
How does Epidiolex (Cannabidiol) work to suppress seizures?
Researchers haven’t figured out the exact mechanism by which CBD reduces seizures.
CBD activates the body’s cannabinoid receptors, named CB1 and CB2. Cannabidiol interacts with both receptors but mostly interacts with CB2. CBD mimics the natural endocannabinoids your body uses to control all sorts of nerve impulses related to mood, appetite, hormonal function and a host of other processes.
According to current understanding, endocannabinoids act as a sort of traffic cop for nerve impulses, making sure the stop and start at the right time and have the right intensity.
CBD also interacts with a number of other types of receptors — proteins that sit on the walls of nerve cells to receive neurotransmitters specifically meant for them, which are their “ligands.” The receptor and its ligand act like a lock and key. When the key is inserted, it kicks off a chemical process to achieve a specific physiological function.
Scientists have been investigating CBD’s interaction with about half a dozen other receptors and systems to try to pinpoint the exact mechanism of action to suppress seizures. They haven’t had any luck yet, largely because of a dearth of clinical trials with marijuana and its derivatives like CBD.
Epidiolex was in phase III clinical trials as of June, 2017, according to the company.
Ion Channel Definition
“Infantile Spasms and Tuberous Sclerosis Complex”
Directory of Disorders
Child Neurology Foundation
“Cannabidiol: pharmacology and potential therapeutic role in epilepsy and other neuropsychiatric disorders.”
O. Devinsky et al
Epidiolex: Epilepsy Patients and Caregivers