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


This case demonstrates the benefit of a lower than previously studied CBE dose for core social communicative and behavioral ASD symptoms, as well as improvements in co-occurring anxiety, sleep dysregulation, and weight, which led to substantial improvements in both our patient's and his family's quality of life and daily functioning. There was partial response at the initial dose of 0.1 mL twice a day (2 mg CBD and 0.1 mg THC) and a dramatic response at 0.2 mL twice a day (4 mg CBD and 0.2 mg THC). Seizures recurred when conventional anti-epileptic medication (lamotrigine) was weaned while on the CBE at the 0.1 mL twice a day dose (low doses), reiterating CBE at this dose did not have any significant anti-epileptic effect; lamotrigine has not been weaned while on the 0.2 mL twice a day (4 mg CBD and 0.2 mg THC) dose of CBE. Typically, significant higher CBD doses are needed for seizure control (> 20 mg/kg per day).[14,15]

The symptom improvement occurred within a 6-month period following the initiation of CBE treatment, during which time there were no new additions or significant alterations of/to any concurrent medications or therapies that could otherwise explain the improvements in symptomatology. It remains unclear whether the CBE directly modified the core ASD symptoms in some way, or whether the impact of CBE was secondary to its positive effects on comorbid conditions, namely anxiety and/or sleep dysregulation, which were producing or exacerbating underlying ASD behaviors. We must also consider there are limitations inherent in the method used to assess his clinical improvement, as the VAS and the AQ are not yet validated. These measures were chosen by default, as no scale is currently validated to assess clinical progress.[3] Seizures recurred at the initial 0.1 mL twice a day (2 mg CBD and 0.1 mg THC) dose of CBE. In addition to the fact that seizures were well controlled prior to starting CBE, the recurrence of seizures on the initial 0.1 mL twice a day dose of CBE, a dose at which symptoms were already starting to improve, suggests that improvements in ASD symptoms were not related to improvements in epilepsy control; the anti-seizure properties of CBD alone are unlikely to be the predominant mechanism responsible for the improvements in this patient's ASD symptoms.

The ECS is a unique biological system that is present in the majority of body tissues. It plays an important role in cellular processes at the early stages of development.[21] The ECS is an essential regulatory system of the central nervous system that modulates both neurotransmission and synaptic plasticity. It is also involved in emotional and social functioning, and cognition.[1,21] There is evidence that the ECS is underdeveloped in ASD.[1,22] CBD may be treating core symptoms in ASD by interacting with the ECS to boost function in one way. CBD may increase the availability of the endogenous cannabinoids, anandamide (AEA), by directly inhibiting one of its degrading enzymes, that is, fatty amide acid hydrolase (FAAH).[1,23,24] Wei et al. demonstrated that selective inhibition of FAAH in BTBR animals, increased AEA activity.[25] Further to this, a case–control study by Karhson et al. assessed AEA concentrations in ASD (n = 59) versus controls, and found lower AEA concentrations associated with ASD.[26]

High-dose CBD has been studied for seizures and has been approved by the FDA (Epidiolex) for the treatment of intractable epilepsy,[14,15,27,28] but there remains a lack of evidence for the use of phytocannabinoids in ASD.[11] Only a few low-powered studies address the clinical efficacy of cannabinoids for such symptoms, and there are no established recommendations for its use in ASD treatment.[5,6] The majority of published studies for ASD either involve synthetic cannabinoids[11,17,18] or synthetic enzyme-inhibitors.[25,29] Only a few studies offer evidence for the use of phytocannabinoids in ASD. An observational study by Bar-Lev Schleider et al. provided valuable information on safety and efficacy, but the study design was insufficient to draw strong conclusions on standard clinical care.[19] Clinicaltrials.gov lists an ongoing randomized trial comparing different phytocannabinoid extracts in the setting of behavioral symptoms, but results are not yet available.[30] Therefore, this case report is rare as it documents observed effects of CBE in ASD-related symptoms as opposed to other forms of cannabinoids (for example, nabilone, dronabinol, and nabiximols).

From a clinical perspective, the use of CBD-based products to treat neuropsychiatric symptoms must be done only after appropriate education and informed discussion with families, including consideration of risks and benefits of CBD compared to other available treatment options, and with vigilant monitoring.

Research into the role of cannabinoids in treating ASD symptoms and associated behaviors is in its infancy. Although there is an increasing amount of evidence providing biological plausibility for the use of CBD in treating ASD,[1,5,25,26,31] further research is essential to better understand the effects of phytocannabinoids on neurobiological pathways and their impact on behavior and brain function. Rigorous, controlled clinical trials are needed to further establish safety, especially long-term safety, optimal dosing, and efficacy, including further delineation of the effect of CBE on core versus associated ASD symptoms. Until sufficient, supportive evidence is found, CBE remains an unproven alternative treatment and should not replace conventional evidence-based treatments for children with autism. However, the unexpected and significant benefits of CBE in this case report highlight the urgent need and potential benefits of continuing to pursue research in this area.