Cannabinoid Gel Treats Fragile X Syndrome

July 31, 2018

"Fragile X syndrome is a genetic disorder characterized by developmental problems such as intellectual disabilities, cognitive impairment, and behavioral and learning challenges. Zynerba Pharmaceuticals announced in 2017 that a cannabinoid gel they have produced improved symptoms of fragile x syndrome in children and adolescents when applied daily to the upper arm.

Multiple cannabinoids are derived from cannabis plants, and include cannabidiol, which likely conveys some of the plant’s positive effects, and tetrahydrocannabinol (THC), which lends marijuana its psychoactive or psychomimetic effects, such as delusion or delirium. Cannabidiol is the active ingredient in the gel, and no THC was found in participants’ blood tests after using the gel.

The open study of 20 patients aged 6 to 17 years found that the participants showed improvement on a scale measuring anxiety, depression, and mood after 12 weeks of using the gel. The gel also appeared to improve aberrant behaviors including social avoidance, temper tantrums, repetitive movements, and hyperactivity. Treatment began at a dose of 50mg per day, and could be increased up to 250 mg per day within the first six weeks of the study. The dose then remained stable for the next six weeks.

Zynerba Pharmaceuticals hope to begin controlled clinical trials in 2018, with the goal of attaining approval for the drug from the US Food and Drug Administration. Other companies are also competing to garner the first FDA approval of a cannabis-based drug. Many of the drugs currently in development are intended to target neurological or behavioral conditions."

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