A treatment against bouts of tuberous sclerosis in Bourneville
A highly purified oral cannabidiol (CBD) solution reduces epilepsy associated with Tuberous sclerosis of Bourneville (STB), which is characterized by benign tumors developing in many parts of the body. Seizures in young children, which manifest as repetitive spasms of the head and legs, are often a sign of the disease.
“CBD appears to be an effective, safe and well-tolerated drug in TBS, so it offers us another treatment option for epilepsy, where there is a significant and unmet need,” said Dr. Elizabeth Thiele, Director of the Herscot Center for Tuberous Sclerosis Complex and director of the Pediatric Epilepsy Program, Massachusetts General Hospital in Boston.
The results were presented here at the 73rd Annual Meeting of the American Epilepsy Society (AES) in 2019.
Symptoms resistant to other treatments
As reported by Medscape Medical News, the purified CBD solution used in the study (Epidiolex, GW Pharmaceuticals) was approved in 2018 by the US Food and Drug Administration for the treatment of Dravet syndrome and Lennox-Gastaut syndrome (LGS), two resistant forms other treatments.
It is estimated that the STB touches an individual on 5500. It is caused by defects in the TSC1 and TSC2 genes that negatively control the rapamycin (mTOR) pathway in mammals and cause abnormal cell growth in organ systems, particularly in the skin and brain.
About 85% of people with TBS have epilepsy, usually of pediatric origin. In about two-thirds of cases, the epilepsy is refractory, says Dr Thiele.
To determine the effectiveness of Epidiolex in this population, the researchers recruited 224 patients (median age, 11 years) at 46 sites in six countries.
Patients had already tried a median of four antiepileptic drugs and took a median of three. Valproate (45%), vigabatrin (33%), levetiracetam (28%) and clobazam (27%) were the most common.
Participants were randomly assigned to receive 25 mg / kg / day or 50 mg / kg / day of CBD, or a placebo. After a 4 week dose adjustment period, patients continued to receive these products for 12 weeks. The primary endpoint of the study was the percent change in the frequency of focal and generalized seizures associated with TBS.
Analysis of the 201 patients who participated in the study showed that the two doses of CBD resulted in a significantly greater reduction in seizure frequency - 49% for the 25 mg dose (P = 0,0009) and 48% for the 50 mg dose (P = 0,0018) - compared to placebo, i.e. 27%.