Fragile X Syndrome, or "Martin Bell Syndrome," is a hereditary disorder that causes symptoms similar to the autistic spectrum, and in many cases also leads to developmental mental disabilities.
According to the Center for Genetic Diagnosis at Assuta Hospital, Fragile X Syndrome is the most common hereditary cause of autism and mental retardation, with Down Syndrome being a little more common but in most cases not hereditary.
The syndrome appears in one of each 4,000 boys and one of the 7,000 girls. The average IQ of boys with the syndrome is below 55, and in many of them it is also manifested in hyperactivity.
In the past decade, pharmaceutical companies have invested a lot of money and resources in developing drugs for fragile X syndrome, but to date there is no FDA-approved treatment for the disease.
Instead, drug therapy focuses on alleviating the secondary symptoms of the disease such as self-harm, aggression, depression, and anxiety - and includes mainly antidepressants and antipsychotic drugs.
Many hoped that a breakthrough in the field would come from the Novartis drug giant, but that Has announced In 2014 because it is discontinuing trials with its drug for fragile X syndrome following disappointing results.
At the end of February it was approved Patent Application Filed by Zynerba Pharmaceuticals on a CBD (cannabidiol) gel developed for the treatment of fragile X syndrome.
The patent was approved based on studies conducted by the company in several countries on the efficacy of its CBD treatment in 17-infected boys with the syndrome.
Zinrava's full research results will be published later this year, when the company will also seek FDA approval for marketing the drug in the US. In the meantime, another, smaller, study of the effectiveness of CBD in treating the syndrome has been published with particularly optimistic results.
Research(March 2019) in Cannabis and Cannabinoid Research, examined the efficacy of cannabidiol (CBD) treatment on three patients with fragile X syndrome.
In all three patients, improvement was observed, including reduction in social abstinence and anxiety, improvement in sleep, diet, lip skills, motor coordination, and sensory processing.
Patients in the study received oral CBD solutions at a daily dose of 32-63.9 mg. In two of the patients, the symptoms worsened back after discontinuation of CBD and improved again after treatment resumed.
"Previous studies have shown that many of the abnormal symptoms of fragile X syndrome are caused by a lack of regulation of the endocannabinoid system, and reduced stimulation of their receptors due to a lack of natural endocannabinoids to bind to them. Studies have shown that CBD intake increases the levels of endocannabinoid anandamide and 2-AG, thus reducing and even neutralizing one of the biological mechanisms of abnormal cell function in fragile X syndrome. "