Updated: Jun 17, 2019
Can marijuana treat autism? A new clinical trial is looking to see if cannabidiol (CBD) gel is a safe and effective method of treating Fragile X syndrome, a type of autism linked to deficiencies in the endocannabinoid system, which controls emotional response, behavioral reactivity to context, and social interaction. Autism, or more technically referred to as Autism Spectrum Disorder (ASD), is a wide range of conditions grouped together because they all affect development and behavior. People with ASD typically experience difficulty communicating with other people, have restricted interests and repetitive behaviors.
Fragile X syndrome is a heritable condition caused by changes in the Fragile X mental retardation 1 gene (FMR1). FMR1 prevents the production of the Fragile X mental retardation protein, which is needed for normal brain development. There is no cure currently for Fragile X syndrome. CBD may result in health benefits such as pain and anxiety relief as well as mood management without the psychoactive effects of THC (Tetrahydrocannabinol), another prominent cannabis compound.
“CBD is a known modulator of the endocannabinoid system and therefore may have therapeutic potential in ameliorating some of those core symptoms,” Dr. Raphe Bernier, the principal investigator of the study explained. Bernier is also a UW professor of psychiatry and behavioral sciences and the executive director of Seattle Children’s Autism Center.
“For individuals with ASD caused by other gene variants or other causes, we wouldn’t anticipate that CBD would have an impact because the underlying cause doesn’t have anything to do with the endocannabinoid system,” Bernier said.
The trial is sponsored by the company that produces the CBD gel, Zynerba Pharmaceuticals, and will study children from 3 to 17-years-old with Fragile X syndrome. Participants will be given ZYN002, the first and only pharmaceutically produced cannabinoid (CBD). Parents will rub the gel into their child’s arm twice a day and the researchers will look for behavioral changes in follow-up visits. The study is set up in a double-blind procedure format which means neither the researchers nor the participants will not know if they are receiving the CBD gel or a control gel.
“Topical gel can help make things easier for children with developmental disabilities,” Bernier said. “There is no need to swallow a pill, which can be a challenge, or apply a patch that can be removed because it’s uncomfortable. It also bypasses the GI system and many children with Fragile X syndrome have GI disturbances, and [there is] no need to introduce anything else into the system.” Orally-administered CBD may lead to side effects in the gastrointestinal tract such as nausea, vomiting, and diarrhea, according to Will Roberts, vice president of corporate communication at Zynerba Pharmaceuticals.
Twenty-one sites around the United States, New Zealand, and Australia are participating in the study, one of which will be located at the UW. The study will last around 14 weeks. Micah Pepper, the trial coordinator and manager of program operations of the UW clinical site, said. “The basic idea for Zygel is that we believe in the medicinal potential of CBD and that it can potentially address a number of behavioral and emotional symptoms of a number of neuropsychiatric disorders,” Roberts said. “The potential for CBD is enormous, but it must be developed like any other pharmaceutical drug.”
In order to run the study Bernier went through registrations with the Washington State Department of Health and the Federal Drug Enforcement Administration in order to handle the CBD (only required due to the unfortunate fact, and regardless of known benefits, is still listed as a Schedule 2 drug). The experts are unsure if the drug will effectively treat Fragile X symptoms. “I don’t know if it’s going to work. That’s why we do research and are using a double-blind procedure. Only then can we know if this is a potential treatment option,” Pepper said. Bernier concurred. “I’m a scientist. I’m withholding judgment until after the trial. There is a theoretical rationale for why it could work, but we’ve got to try it out.”
Zynerba Pharmaceuticals is sponsoring other ongoing clinical trials using their CBD gel as a treatment for other autism disorders, anxiety, and epilepsy. “We continuously assess the spectrum of rare and near-rare neuropsychiatric disorders for potential future indications of interest,” Roberts said.