Cannabis for refractory epilepsy in children: A review focusing on CDKL5 Deficiency Disorder

Tristan Dale; Jenny Downs; Heather Olson; Ann Marie Bergin; Stephanie Smith; Helen Leonard

doi:10.1016/j.eplepsyres.2019.02.001

Cannabis for refractory epilepsy in children: A review focusing on CDKL5 Deficiency Disorder

Epilepsy Res. 2019 Mar:151:31-39. doi: 10.1016/j.eplepsyres.2019.02.001. Epub 2019 Feb 5.

Authors

Tristan Dale¹, Jenny Downs², Heather Olson³, Ann Marie Bergin⁴, Stephanie Smith⁵, Helen Leonard⁶

Affiliations

¹ UWA Medical School, Faculty of Health and Medical Sciences, University of Western Australia, Perth, WA, Australia(1); Telethon Kids Institute, University of Western Australia, Perth, WA, Australia(2). Electronic address: Tristan.Dale@telethonkids.org.au.
² Telethon Kids Institute, University of Western Australia, Perth, WA, Australia(2); School of Physiotherapy and Exercise Science, Curtin University, Perth, WA, Australia(3). Electronic address: Jenny.Downs@telethonkids.org.au.
³ Department of Neurology, Division of Epilepsy and Clinical Neurophysiology, Boston Children's Hospital, Boston, MA, USA(4). Electronic address: Heather.Olson@childrens.harvard.edu.
⁴ Department of Neurology, Division of Epilepsy and Clinical Neurophysiology, Boston Children's Hospital, Boston, MA, USA(4). Electronic address: AnnMarie.Bergin@childrens.harvard.edu.
⁵ Telethon Kids Institute, University of Western Australia, Perth, WA, Australia(2). Electronic address: Stephanie.Smith@telethonkids.org.au.
⁶ Telethon Kids Institute, University of Western Australia, Perth, WA, Australia(2). Electronic address: Helen.Leonard@telethonkids.org.au.

PMID: 30771550
DOI: 10.1016/j.eplepsyres.2019.02.001

Abstract

Severe paediatric epilepsies such as CDKL5 Deficiency Disorder (CDD) are extremely debilitating, largely due to the early-onset and refractory nature of the seizures. Existing treatment options are often ineffective and associated with a host of adverse effects, causing those that are affected to seek alternative treatments. Cannabis based products have attracted significant attention over recent years, primarily driven by reports of miraculous cures and a renewed public preference for 'natural' therapies, thus placing intense pressure on health professionals and the government for regulatory change. This study provides a comprehensive overview of the potential role for cannabis in the treatment of CDD. Key areas discussed include the history, mechanism of action, efficacy and safety of cannabis based preparations as well as the burden related to CDD. The evidence supports the use of cannabinoids, especially cannabidiol, in similar forms of refractory epilepsy including Dravet and Lennox-Gastaut syndromes. Evidence for cannabinoids specifically in CDD is limited but growing, with multiple anecdotal reports and an open-label trial showing cannabidiol to be associated with a significant reduction in seizure activity. This review provides the first comprehensive overview of the potential role for cannabis based preparations in the treatment of CDD and provides justification for further clinical and observational research.

Keywords: CDKL5 Deficiency Disorder; Cannabidiol; Cannabis; Encephalopathy; Epilepsy; Paediatric.

Publication types

Research Support, Non-U.S. Gov't
Review

MeSH terms

Anticonvulsants / therapeutic use*
Cannabinoids / therapeutic use*
Cannabis / chemistry
Drug Resistant Epilepsy / drug therapy
Epileptic Syndromes / drug therapy*
Humans
Spasms, Infantile / drug therapy*

Substances

Anticonvulsants
Cannabinoids

Supplementary concepts

CDKL5 deficiency disorder

Grants and funding

K23 NS107646/NS/NINDS NIH HHS/United States