Treatment of Fragile X Syndrome with Cannabidiol: A Case Series Study and Brief Review of the Literature
- PMID: 30944868
- PMCID: PMC6446166
- DOI: 10.1089/can.2018.0053
Treatment of Fragile X Syndrome with Cannabidiol: A Case Series Study and Brief Review of the Literature
Abstract
Fragile X syndrome (FXS) is an X-linked dominant disorder caused by a mutation in the fragile X mental retardation 1 gene. Cannabidiol (CBD) is an exogenous phytocannabinoid with therapeutic potential for individuals with anxiety, poor sleep, and cognitive deficits, as well as populations with endocannabinoid deficiencies, such as those who suffer from FXS. The objective of this study was to provide a brief narrative review of recent literature on endocannabinoids and FXS and to present a case series describing three patients with FXS who were treated with oral CBD-enriched (CBD+) solutions. We review recent animal and human studies of endocannabinoids in FXS and present the cases of one child and two adults with FXS who were treated with various oral botanical CBD+ solutions delivering doses of 32.0 to 63.9 mg daily. Multiple experimental and clinical models of FXS combine to highlight the therapeutic potential of CBD for management of FXS. All three patients described in the case series exhibited functional benefit following the use of oral CBD+ solutions, including noticeable reductions in social avoidance and anxiety, as well as improvements in sleep, feeding, motor coordination, language skills, anxiety, and sensory processing. Two of the described patients exhibited a reemergence of a number of FXS symptoms following cessation of CBD+ treatment (e.g., anxiety), which then improved again after reintroduction of CBD+ treatment. Findings highlight the importance of exploring the therapeutic potential of CBD within the context of rigorous clinical trials.
Keywords: CBD; cannabidiol; endocannabinoid system; fragile X syndrome; oral solution.
Conflict of interest statement
N.T. has received funding from Neuren, Alcobra, Roche, and Seaside to carry out treatment trials in FXS. She has also consulted with Novartis, Zynerba, and Ovid regarding treatment in FXS. M.B.M. is an employee of Zynerba Pharmaceuticals. R.H. has received funding from Novartis, Marinus, Neuren, Alcobra, Seaside, and Roche to carry out treatment trials in FXS. She has also consulted with Fulcrum, Zynerba, and Ovid regarding treatment in FXS.
Similar articles
-
Parent and Caregiver Perspectives towards Cannabidiol as a Treatment for Fragile X Syndrome.Genes (Basel). 2022 Sep 6;13(9):1594. doi: 10.3390/genes13091594. Genes (Basel). 2022. PMID: 36140762 Free PMC article.
-
A phase 1/2, open-label assessment of the safety, tolerability, and efficacy of transdermal cannabidiol (ZYN002) for the treatment of pediatric fragile X syndrome.J Neurodev Disord. 2019 Aug 2;11(1):16. doi: 10.1186/s11689-019-9277-x. J Neurodev Disord. 2019. PMID: 31370779 Free PMC article. Clinical Trial.
-
Role of the endocannabinoid system in fragile X syndrome: potential mechanisms for benefit from cannabidiol treatment.J Neurodev Disord. 2023 Jan 9;15(1):1. doi: 10.1186/s11689-023-09475-z. J Neurodev Disord. 2023. PMID: 36624400 Free PMC article. Review.
-
Cannabidiol (CBD) reduces anxiety-related behavior in mice via an FMRP-independent mechanism.Pharmacol Biochem Behav. 2019 Jun;181:93-100. doi: 10.1016/j.pbb.2019.05.002. Epub 2019 May 4. Pharmacol Biochem Behav. 2019. PMID: 31063743
-
Sensory Processing Phenotypes in Fragile X Syndrome.ASN Neuro. 2018 Jan-Dec;10:1759091418801092. doi: 10.1177/1759091418801092. ASN Neuro. 2018. PMID: 30231625 Free PMC article. Review.
Cited by
-
Unraveling the Endocannabinoid System: Exploring Its Therapeutic Potential in Autism Spectrum Disorder.Neuromolecular Med. 2024 May 14;26(1):20. doi: 10.1007/s12017-024-08781-6. Neuromolecular Med. 2024. PMID: 38744725 Free PMC article. Review.
-
Les données probantes et les recommandations sur le cannabis à des fins médicales chez les enfants.Paediatr Child Health. 2024 Apr 5;29(2):104-121. doi: 10.1093/pch/pxad077. eCollection 2024 May. Paediatr Child Health. 2024. PMID: 38586491 Review.
-
Medical cannabis for children: Evidence and recommendations.Paediatr Child Health. 2024 Apr 5;29(2):104-121. doi: 10.1093/pch/pxad078. eCollection 2024 May. Paediatr Child Health. 2024. PMID: 38586483 Review.
-
State-of-the-art therapies for fragile X syndrome.Dev Med Child Neurol. 2024 Jul;66(7):863-871. doi: 10.1111/dmcn.15885. Epub 2024 Feb 22. Dev Med Child Neurol. 2024. PMID: 38385885 Review.
-
Cannabidiol (Epidyolex®) for severe behavioral manifestations in patients with tuberous sclerosis complex, mucopolysaccharidosis type III and fragile X syndrome: protocol for a series of randomized, placebo-controlled N-of-1 trials.BMC Psychiatry. 2024 Jan 4;24(1):23. doi: 10.1186/s12888-023-05422-3. BMC Psychiatry. 2024. PMID: 38177999 Free PMC article.
References
-
- Hagerman RJ, Berry-Kravis E, Hazlett HC, et al. . Fragile X syndrome. Nat Rev Dis Primers. 2017;3:1706–5. - PubMed
-
- Turner G, Webb T, Wake S, et al. . Prevalence of fragile X syndrome. Am J Med Genet. 1996;64:196–197 - PubMed
-
- Hunter J, Rivero-Arias O, Angelov A, et al. . Epidemiology of fragile X syndrome: a systematic review and meta-analysis. Am J Med Genet A. 2014;164a:1648–1658 - PubMed
Publication types
LinkOut - more resources
Full Text Sources
Other Literature Sources