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Clinical Data

Epilepsy is the most well-known neurologic disorder in which cannabidiol has shown therapeutic promise.1 Despite advances in epilepsy treatment, approximately 1/3 of patients with epilepsy will have drug-resistance seizures, which can lead to cognitive impairment, and psychiatric and physical disabilities.2,3

Dravet Syndrome (DS) and Lennox-Gastaut syndrome (LGS) are severe epilepsy encephalopathy disorders with an early childhood onset. Tuberous sclerosis complex (TSC) is a genetic disease, which commonly manifests as focal seizures. Patients with DS, LGS, and TSC have seizures resistant to common antiepileptic medications and mutations in endocannabinoid and other receptors regulating brain hyperexcitability, which makes these diseases difficult to treat with most targeted therapies.2,4,5

Argueta D, et al. Front Pharmacol. 2020;11:561.

Randomized controlled trials (RCTs) have demonstrated that cannabidiol treatment, as an adjuvant therapy to antiseizure medications, reduces the frequency of seizures in patients with DS, LGS, TSC, or other forms of drug-resistant epilepsy, with a tolerable toxicity profile.2,6 The efficacy of cannabidiol in drug-resistant seizures is evidenced by reductions in seizures persisting with up to 48 weeks of treatment.3 Approval of cannabidiol by the FDA was based on evidence from several double-blind, placebo-controlled phase 3 RCTs:

  • Dravet syndrome: GWPCARE 1 and 2 (NCT02091375 and NCT02224703)7,8
  • Lennox-Gastaut syndrome: GWPCARE 3 and 4 (NCT02224560 and NCT02224690)9,10
  • Tuberous sclerosis complex: GWPCARE 6 (NCT02544763)11

Epilepsy continues to be the most common therapeutic area for ongoing cannabidiol clinical trials.12 Currently, there are a dozen or more registered planned or active trials of cannabidiol in epilepsy including GWP42003 in typical absence seizures (NCT04899050), an inhaled dried powder cannabidiol formulation (RLS103) in photosensitive epilepsy (NCT05678881), a cannabidiol-enriched cannabis herbal extract in refractory epileptic encephalopathy (NCT03024827), and a cannabidiol: THC oral solution (MGCND00EP1) in resistant epilepsy (NCT04406948).

More research is needed in the form of randomized clinical trials with regard to cannabidiol use for other specific conditions before implementation in everyday clinical practice. Other areas of study for cannabidiol include Parkinson’s disease, Alzheimer’s disease, multiple sclerosis, neuropathic pain, anxiety, depression, schizophrenia, and psychosis.

References

  1. Singh K, Bhushan B, Chanchal DP, et al. Emerging therapeutic potential of cannabidiol (CBD) in neurological disorders: a comprehensive review. Behav Neurol. 2023;2023:8825358.
  2. Silvinato A, Floriano I, Bernardo WM. Use of cannabidiol in the treatment of epilepsy: Lennox-Gastaut syndrome, Dravet syndrome, and tuberous sclerosis complex. Rev Assoc Med Bras (1992). 2022;68:1345-1357.
  3. Liu S, He Z, and Li J. Long-term efficacy and adverse effects of cannabidiol in adjuvant treatment of drug-resistant epilepsy: A systematic review and meta-analysis. Ther Adv Neurol Disord. 2023;16:1-21.
  4. Luz-Veiga M, Azevedo-Silva J, Fernandes JC. Beyond pain relief: A review on cannabidiol potential in medical therapies. Pharmaceuticals (Basel). 2023;16:155.
  5. Ghosh S, Sinha JK, Ghosh S, et al. A comprehensive review of emerging trends and innovative therapies in epilepsy management. Brain Sci. 2023;13:1305.
  6. Zavala-Tecuapetla C, Luna-Munguia H, López-Meraz ML, et al. Advances and challenges of cannabidiol as an anti-seizure strategy: Preclinical evidence. Int J Mol Sci. 2022;23:16181.
  7. Devinsky O, Cross JH, Laux L, et al. Trial of cannabidiol for drug-resistant seizures in the Dravet syndrome. N Engl J Med. 2017;376:2011-2020.
  8. Miller I, Scheffer IE, Gunning B, et al. Dose-ranging effect of adjunctive oral cannabidiol vs placebo on convulsive seizure frequency in Dravet syndrome–a randomized clinical trial. JAMA Neurol. 2020;77:613-621.
  9. Devinsky O, Patel AD, Cross H, et al. Effect of cannabidiol on drop seizures in the Lennox–Gastaut syndrome. N Engl J Med. 2018;378:1888-1897.
  10. Thiele EA, Marsh ED, French JA, et al. Cannabidiol in patients with seizures associated with Lennox-Gastaut syndrome (GWPCARE4): A randomised, double-blind, placebo-controlled phase 3 trial. 2018;391:1085-1096.
  11. Thiele EA, Bebin M, Bhathal H, et al. Add-on cannabidiol treatment for drug-resistant seizures in tuberous sclerosis complex–a placebo-controlled randomized clinical trial. JAMA Neurol. 2021;78:285-292.
  12. Jazz Pharmaceuticals Global Medical Affairs. Access our Medical Resources Library. Accessed 9/11/2024. https://www.jazzmedical.com/

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