A Pediatric Patient With Autism Spectrum Disorder and Epilepsy Using Cannabinoid Extracts as Complementary Therapy

A Case Report

Juliana Andrea Ponton; Kim Smyth; Elias Soumbasis; Sergio Andres Llanos; Mark Lewis; Wilhelm August Meerholz; Robert Lawrence Tanguay


J Med Case Reports. 2020;14(162) 

In This Article


Autism spectrum disorder (ASD) is a neurodevelopmental disorder that is characterized by deficits in two major domains: restrictive, repetitive patterns of behavior, interests, or activities; and deficits in social communication and interaction.[1,2] ASD is associated with a higher incidence of comorbid conditions including attention deficit hyperactivity disorder, anxiety, gastrointestinal disturbances, motor impairments, and epilepsy. Symptoms appear in early childhood and vary in severity leading to a broad range of clinical manifestations.[2]

The pathogenesis of ASD is not completely understood.[3] Given its complexity and diverse clinical manifestations, it is believed that the etiopathogenesis of ASD is a combination of genetic, epigenetic, neurobiological, diet, and other environmental factors.[4] Hundreds of genes (NLGN, SHANK3, ZNF8034A, and UNC13A)[5,6] have been linked to ASD, most of which are closely related to the development of the nervous system.[1]

There is a myriad of theories that attempt to explain the occurrence of ASD,[1,3,7] although the two most accepted are impaired synaptic transmission and disruption of neural connectivity. The endocannabinoid system (ECS) has attracted considerable attention as a potential contributor to ASD, as the development of the ECS is essential for regulating synaptic function by inhibiting the release of neurotransmitters from presynaptic neurons.[1]

The management of ASD requires individualized, comprehensive treatment. Non-psychopharmacologic interventions (for example, cognitive behavioral therapy) modify disruptive behaviors and improve social communication skills with varying degrees of success. Traditional psychopharmacologic medications target specific ASD core behaviors (for example, repetitive behaviors) and associated behaviors (for example, hyperactivity, aggression, anxiety, and sleep disturbances), but do not treat core social communication deficits.[8,9] These medications are well known for their substantial side effects. For example, aripiprazole and risperidone, the only two medications approved by the US Food and Drug Administration (FDA) to treat irritability and agitation in ASD, frequently cause somnolence, increased appetite, and weight gain.[10] No other medication has been approved for management of behavioral and/or core ASD symptoms. Challenges with these traditional treatment approaches include barriers to access (economical, geographic), lack of efficacy, and undesirable side effects, which have led many families to seek complementary and alternative medicine (CAM) to augment or replace standard therapy.[8] One of the newest CAM options now being explored in ASD (and, in fact, the wider medical community) is cannabinoids: for example, cannabidiol-based extract (CBE), which is an extract from the cannabis plant, rich in cannabidiol (CBD).[11]

Follow-up of these patients must also be individualized as presentation of the disorder is highly variable. There are no validated questionnaires to accurately assess clinical progress, therefore, conducting an objective clinical assessment of related behavioral and core symptoms is challenging. Despite this, there are tools available for characterizing the overall functionality of patients with ASD, for example, Autism Spectrum Quotient (AQ) adults version.[3]

The World Health Organization stated that CBD should not be scheduled with the International Drug Control Conventions because of growing evidence of its medicinal applications.[12,13] It is imperative for health care providers to understand the minutiae of how cannabinoids interact with the human body and the different forms of cannabinoids that are available for medical use (for example, synthetocannabinoids, phytocannabinoids).[1] Delta-9-tetrahydrocannabinol (THC) and CBD are the most well-known and studied phytocannabinoids. THC is associated with the impairing psychoactive effects of cannabis, resulting in potentially undesirable side effects (dizziness, anxiety, paranoia, dependency, cognitive impairment, and so on). In contrast, CBD is only minimally psychoactive and not impairing or intoxicating at typically used doses (for example, ≥ 20 mg/kg of CBD referred in the majority of intractable seizures studies).[1,8,10,11]

A multitude of studies have analyzed the use of high-dose CBD extract (~ 20 mg/kg of weight per dose) in the context of intractable seizure treatment.[14,15] It has been reported that CBD effects are dose-dependent (for example, > 160 mg/day elicits a sedating effect and lower doses have been associated with increased wakefulness).[16] A few case reports and observational studies have suggested the safety and efficacy of lower dose CBD, for treating behavioral symptoms in ASD.[11,17,18] In a prospective study, 188 patients with ASD were treated with lower to medium doses of phytocannabinoids (from 15 mg of CBD three times a day to 300 mg of CBD three times a day), the majority taking 1:20 CBE: 30% CBD to 1.5% THC.[19] This study found that cannabis was well tolerated, safe, and effective in relieving certain ASD symptoms. More research is needed to assess the long-term effects of CBD, as well as optimal dosing, formulation, delivery method, and so on to maximize both safety and efficacy.

This case report describes the clinical presentation of a pediatric, overweight patient with ASD, epilepsy, anxiety, insomnia, and social deficits who benefited clinically with even lower doses of CBE (4 mg of CBD and 0.2 mg of THC twice a day) compared to the ones already studied.[19]