Did you know that there are over 80 chemicals, called cannabinoids, that can be sourced from the Cannabis sativa plant (marijuana or hemp)? Now, Europe is harnessing this for good within the medical community. According to Medscape, a cannabidiol solution called Epidyolex has been approved in Europe for the adjunctive treatment of seizures in patients with tuberous sclerosis complex (TSC).
Epidyolex
According to MedLine Plus:
Cannabidiol has effects on the brain [and] seems to prevent the breakdown of a chemical in the brain that affects pain, mood, and mental function. Preventing the breakdown of this chemical and increasing its levels in the blood seems to reduce psychotic symptoms associated with conditions such as schizophrenia [and] seems to reduce pain and anxiety.
In the United States, cannabidiol is approved for patients over 1 year old whose epilepsy is associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome, or TSC. Alternately, in Europe, Epidyolex is currently approved for patients aged 2+ with these same conditions. Now, it is newly indicated for patients with TSC in conjunction with clobazam. This fills an unmet need for patients with TSC, whose condition often does not respond to the current standard-of-care for epilepsy.
This approval comes following data from a Phase 3 clinical trial evaluating the safety, efficacy, and tolerability of Epidyolex. Patients received 25mg/kg/d Epidyolex. Altogether, researchers found the therapy to be relatively safe and well-tolerated. Additionally, Epidyolex significantly reduced the frequency and rate of seizures.
Tuberous Sclerosis Complex (TSC)
TSC1 and TSC2 gene mutations cause tuberous sclerosis complex (TSC), a rare genetic disease. Typically, both of these genes play a role in controlling cell proliferation. By managing the way cells are growing, and preventing them from growing too quickly, TSC1 and TSC2 keep the body healthy and in stasis. But mutations in these genes cause cells to grow at an uncontrolled rate. As a result, patients with TSC experience the widespread growth of noncancerous tumors throughout their body. In many cases, TSC diagnoses are made in infancy and childhood. However, in patients with mild TSC, the condition may not be diagnosed until adulthood. Symptoms include:
- Seizures
- Facial lesions resembling acne
- Non-cancerous tumors which often grow in the brain, skin, heart, lungs, and kidneys
- Skin abnormalities (light colored skin, thickened skin, growths on/around the nails)
- Developmental delays
- Retinal damage and/or other eye abnormalities
- Aggression or irritability
- Repetitive behaviors
- Social anxiety
- issues with heart, lung, or kidney function
- Hyperactivity
