After seeing some success around a treatment for epilepsy, the cannabis industry is increasingly eyeing for products for people with the autism spectrum disorder.
Autism is a lifelong developmental disorder that affects communication, behavior and the ability to socialize. As many as one in 54 children are currently diagnosed with autism, according to Centers for Disease Control, and so are millions of adults.
In addition to companies looking expand their customer base and come up with new treatments, the Autism Science Foundation is also urging further study on the safety and efficacy of medical marijuana in autism.
"While the FDA has eased some regulatory requirements to study CBD, the legality of CBD is still in question making human-based research highly challenging," said the foundation recently. "We urge the DEA to move medical marijuana from a Schedule I to a Schedule II compound to expand future research possibilities."
Despite limitations on research, three cannabis companies have begun working towards finding cannabis treatments for autism.
While GW Pharmaceuticals (GWPH) is known more for its work on epilepsy, another neurological condition, the company is tackling autism, as well. GW Pharmaceuticals has said that many of the childhood-onset intractable epilepsy conditions share considerable overlap with autism spectrum disorder - which is often shortened as ASD - and these conditions often fall within the "orphan" disease space.
"Initial clinical observations from treating physicians suggest a potential role for cannabinoids in addressing problems associated with ASD such as deficits in cognition, behavior, and communication," said the company.
GW has evaluated the cannabinoids CBD and CBDV in autism, yielding promising signals on cognitive and social functioning as well as repetitive behaviors. In the company's annual report it wrote that it is working on various clinical initiatives for CBDV in the field of autism spectrum disorder and last year initiated a company-sponsored open label trial in ASD and an investigator-led 100 patient placebo-controlled trial.
GW Pharmaceuticals has made great strides in getting its epilepsy drug Epidiolex removed from controlled-drug status. The company has a strong track record of running respected and credible drug studies. The results coming out of GW Pharmaceuticals will no doubt be closely watched.
Last month, Zynerba Pharmaceuticals, Inc. (ZYNE) announced positive top-line results from the exploratory, open-label Phase 2 BRIGHT study, which looked at the efficacy, safety and tolerability of Zygel, a transdermal gel potentially to be used for children and adolescents with autism.
The trial was designed to assess the safety, tolerability, and efficacy of the Zynerba drug called Zygel in pediatric and adolescent patients with autism spectrum disorder (ASD). Zygel was administered to patients with moderate-to-severe symptoms of autism as add-on therapy to their standard of care utilizing a variety of efficacy assessments.
"We are very encouraged by the compelling top-line results of the BRIGHT trial and we expect to meet with the FDA to discuss the clinical pathway for developing Zygel for the treatment of behavioral symptoms of ASD in the second half of this year," said Armando Anido, Zynerba's chairman and CEO.
He said the goal is to develop Zygel for patients with debilitating neuropsychiatric disorders, such as autism and fragile-X syndrome.
The study consisted of 36 patients for 14 weeks with a mean age of 9.2 years old. Nearly all, 92%, were boys and 94% of enrolled patients had moderate-to-severe symptoms of autism. The company reported that patients on Zygel experienced improvement by week 14 and that 57% of patients were assessed as "very much improved" or "much improved" at week 14. The drug was also well-tolerated by the patients.
"I am very impressed with the improvements my patients made over the 14-week treatment period while receiving Zygel; the reduction in irritability, communication deficits, and repetitive movements were especially noteworthy since some of these are core autistic behaviors," said Helen Heussler, an associate professor at Children's Health Queensland, Medical Director Child Development and principal investigator in the BRIGHT trial. "The magnitude of effect on autistic behaviors in this trial is significant, including hyperactivity and stereotypy, which are among the most difficult behaviors to improve with therapeutic intervention. The results of this study strongly suggest the potential of this drug as an important treatment for ASD and I look forward to participating in future clinical studies with Zygel."
Zelira Therapeutics Ltd (OTCMKTS: ZLDAF) is working closely with leading researchers and organizations including Curtin University in Perth, Western Australia, the Telethon Kids Institute, the University of Western Australia, in Perth, St. Vincent's Hospital in Melbourne, Australia, and the Children's Hospital of Philadelphia in the United States. Zelira was formed as a merger of Ilera Therapeutics and Zelda Therapeutics.
The company recently released a white paper detailing the survey results of 50 patients within the Hope Grows for Autism network in Pennsylvania. All the patients in the survey used Ilera Healthcare's HOPE product line. They were all residents of Pennsylvania and able to legally purchase medicinal cannabis.
This wasn't a clinical trial, but instead an informal approach to treatment. The survey responses were based on caregiver responses with no control group, nor did the survey collect information on the patient's ASD history or associated symptoms. Nevertheless, "there is a strong and widely accepted belief that the main caregivers are the most reliable source of information to evaluate the status of a patient with ASD."
The white paper wrote, "Respondents who used HOPE 1 tincture with a THC:CBD ratio of 1:1 with Eucalyptol, Valencene and Alpha-Pinene terpenes, HOPE 2 tincture with a THC:CBD ratio of 5:1 with beta-Caryophyllene, Myrcene and Alpha-Bisabolol terpenes or both products for a range of 5-133 days (average of 68 days of use) reported the following: Significant improvements in patient irritability (68%), meltdowns (60%), aggression (55%), anxiety (49%) as well as capacity to focus (38%); while 69% of participants reported an improved quality of life for the patient. In addition, 58% reported improved quality of life for the entire family."
According to the data, the majority of patients, however, noted significant improvement, with a small percentage noticing some improvement. None of the patients said that their symptoms worsened. Under 30% of participants reported no change in symptoms of anxiety, aggression, meltdowns, and irritability, with roughly 42% of participants reporting no change in the patient's ability to focus.
Parents of children with autism as well as adults with the disorder can have some comfort in knowing these companies are trying to find some relief for autism's symptoms.