Fragile X syndrome is a genetic disorder that causes different developmental problems. There are no standard treatment options available at this time; however, researchers are starting to look at the possibility of using cannabis to ease the symptoms.
People with Fragile X syndrome suffer from limited synapse communication, which causes their behavioral and cognitive afflictions. Cannabis is believed to be effective because it stimulates the production of an endocannabinoid transmitter in the brain called 2-AG. This transmitter has neuroregenerative potential and repaired brain function in mouse models with Fragile X syndrome.
What is Fragile X Syndrome?
The symptoms of Fragile X Syndrome include cognitive impairment, learning and intellectual disabilities, and various physical conditions include seizures and muscle spasms; and symptoms can vary from mild to severe. Fragile X syndrome can occur in both genders, although it’s more prevalent and typically worse in males. The symptoms are usually similar to those presented in patients with autism, but they often differ slightly based on the patient’s gender.
It’s caused by the fragile x mental retardation gene, and it affects 1 in every 4,000 males and 1 in every 8,000 females. Fragile X Syndrome is the most common inherited intellectual disability in males. Although there are a few treatments that can help reduce the severity of symptoms, there is no go-to, standard medication option for people with this affliction. Early intervention is pertinent for children who are born with fragile x, as it is possible to outgrow the condition in their adult years.
Twenty patients, 15 males and 5 females all 6 to 17 years of age were enrolled in the study. ZYN002, a CBD gel medication that’s still in the testing phase, was added on to other medications being administered. The first six weeks of research were designed to titrate dosing in patients. The initial dose was set at 50 mg per day and could be increased to 250 mg. Whatever dose was established during week 6 was then administered daily until the end of week 12. After the study was completed, participants could enter an “open label extension study” for up to 1 year.
“The data from the FAB-C trial are very exciting and demonstrate that ZYN002 may have a profound effect on improving many of the most disabling symptoms of Fragile X, such as anxiety and difficult behaviors,” Steven Siegel, MD, PhD, Professor and Chair of Psychiatry and Behavior sciences at Keck School of Medicine, USC, said in the release. “Fragile X is a challenging genetic autism spectrum disorder, with complex symptomatology that significantly impacts patients and their families. Many children with Fragile X and their families struggle with the lack of approved drugs to safely treat their symptoms. This study suggests that ZYN002 is ready for the next phase of development, and I believe that this drug holds great promise as a potential treatment for these very difficult-to-treat symptoms.”
Zynerba Pharmaceuticals, the company that manufactures ZYN002, expects to meet with the Food and Drug Administration before June 2018 to get the ball rolling on this new medication. They plan to quickly get into a “pivotal phase 2/3 program in pediatric and adolescent patients with Factor X syndrome” by the end of next year.[Image credit- Pixabay]