Cannabidiol Based Medical Cannabis in Children with Autism- a Retrospective Feasibility Study

Background: Anecdotal evidence of successful cannabis treatment in children with autism spectrum disorder (ASD) are accumulating but formal studies are lacking.

Objectives: This retrospective study assessed safety, tolerability and efficacy of cannabidiol (CBD) based medical cannabis, as an adjuvant therapy, for refractory behavioral problems in children with ASD.

Methods: 60 children with ASD (age = 11.8± 3.5, range 5.0-17.5; 77% low cognitive functioning; 83% boys) were treated with oral CBD and tetrahydrocannabinol (THC) at a ratio of 20:1. The dose was up-titrated to effect (maximal CBD dose -10mg/kg/d). Tolerability and efficacy were assessed using a modified Liverpool Adverse Events Profile, the Caregiver Global Impression of Change (CGIC) scale, the Home Situations Questionnaire–Autism Spectrum Disorder (HSQ-ASD) and the Autism Parenting Stress Index (APSI).

Results: Following the cannabis treatment, behavioral outbreaks were much improved or very much improved (on the CGIC scale) in 61% of patients. The anxiety and communication problems were much or very much improved in 39% and 47% respectively. Disruptive behaviors, were improved by 29% from 4.74±1.82 as recorded at baseline on the HSQ-ASD to 3.36±1.56 following the treatment. Parents reported less stress as reflected in the APSI scores, changing by 33% from 2.04±0.77 to 1.37±0.59. The effect on all outcome measures was more apparent in boys with non-syndromic ASD. Adverse events included sleep disturbances (14%) irritability (9%) and loss of appetite (9%).

Conclusions: This preliminary study support the feasibility of CBD based medical cannabis as a promising treatment option for refractory behavioral problems in children with ASD. Following this preliminary study, we launched a randomized double blind placebo controlled trial.