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

Tristan Dale1, Jenny Downs2, Heather Olson3

  • 1UWA 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).

Epilepsy Research
|February 17, 2019
PubMed

Insights

Cannabis-based treatments, particularly cannabidiol, show promise for managing seizures in CDKL5 Deficiency Disorder (CDD), a severe pediatric epilepsy. While evidence is emerging, it suggests a potential role for cannabinoids where other therapies fail.

Area of Science:

  • Neuroscience
  • Pharmacology
  • Genetics

Background:

  • CDKL5 Deficiency Disorder (CDD) is a severe, early-onset pediatric epilepsy with refractory seizures.
  • Current treatments for CDD are often ineffective and carry significant side effects.
  • Cannabis-based products have gained attention for potential therapeutic benefits in epilepsy.

Purpose of the Study:

  • To provide a comprehensive overview of cannabis and cannabinoids for treating CDD.
  • To examine the history, mechanism of action, efficacy, and safety of cannabis-based preparations in CDD.
  • To review the burden of CDD and existing treatment limitations.

Main Methods:

  • Literature review and synthesis of existing evidence on cannabis and cannabinoids in epilepsy.
  • Analysis of anecdotal reports and clinical trial data for CDD.
  • Discussion of the pharmacological properties of cannabinoids.

Main Results:

  • Cannabinoids, especially cannabidiol (CBD), are effective in other refractory epilepsies like Dravet and Lennox-Gastaut syndromes.
  • Anecdotal evidence and an open-label trial suggest CBD reduces seizure activity in CDD.
  • Limited but growing evidence supports the use of cannabinoids for CDD.

Conclusions:

  • Cannabis-based preparations, particularly CBD, represent a potential therapeutic option for refractory seizures in CDD.
  • Further clinical and observational research is warranted to establish the efficacy and safety of cannabinoids in CDD.
  • The review highlights the need for regulatory consideration of cannabis-based treatments for severe pediatric epilepsies.

Keywords:
CDKL5 Deficiency DisorderCannabidiolCannabisEncephalopathyEpilepsyPaediatric

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