Skip to main content

A role for cannabinoids in the treatment of myotonia? Report of compassionate use in a small cohort of patients



The symptomatic treatment of myotonia and myalgia in patients with dystrophic and non-dystrophic myotonias is often not satisfactory. Some patients anecdotally report symptoms’ relief through consumption of cannabis.


A combination of cannabidiol and tetrahydrocannabinol (CBD/THC) was prescribed as compassionate use to six patients (four patients with myotonic dystrophy types 1 and 2, and 2 patients with CLCN1-myotonia) with therapy-resistant myotonia and myalgia. CBD/THC oil was administered on a low dose in the first 2 weeks and adjusted to a higher dose in the following 2 weeks. Myotonia behaviour scale (MBS), hand-opening time, visual analogue scales (VAS) for myalgia and myotonia, and fatigue and daytime sleepiness severity scale (FSS, ESS) were performed weekly to monitor treatment response.


All patients reported an improvement of myotonia especially in weeks 3 and 4 of treatment: MBS improved of at least 2 points in all patients, the hand-opening time variously improved in 5 out of 6 patients. Chronic myalgia was reported by both DM2 patients at baseline, one of them experienced a significant improvement of myalgia under treatment. Some gastrointestinal complaints, as abdominal pain and diarrhoea, improved in 3 patients; however, 4 out of 6 patients reported new-onset constipation. No other relevant side effect was noticed.


These first empirical results suggest a potentially beneficial role of CBD/THC in alleviating myotonia and should encourage further research in this field including a randomized-controlled trial on larger cohorts.

This is a preview of subscription content, access via your institution.

Fig. 1
Fig. 2


  1. 1.

    Wenninger S, Montagnese F, Schoser B (2018) Core clinical phenotypes in myotonic dystrophies. Front Neurol 9:303.

    Article  PubMed  PubMed Central  Google Scholar 

  2. 2.

    Miró J, Gertz KJ, Carter GT, Jensen MP (2014) Pain location and intensity impacts function in persons with myotonic dystrophy type 1 and facioscapulohumeral dystrophy with chronic pain. Muscle Nerve 49(6):900–905.

    Article  PubMed  PubMed Central  Google Scholar 

  3. 3.

    Heatwole C, Bode R, Johnson N, Quinn C, Martens W, McDer MP, Rothrock N, Thornton C, Vickrey B, Victorson D, Mox-ley R (2012) Patient-reported impact of symptoms in myotonic dystrophy type 1 (PRISM-1). Neurology 79(4):348–357.

    Article  PubMed  PubMed Central  Google Scholar 

  4. 4.

    Heatwole C, Johnson N, Bode R, Dekdebrun J, Dilek N, Hilbert JE, Luebbe E, Martens W, McDermott MP, Quinn C, Rothrock N, Thornton C, Vickrey BG, Victorson D, Moxley RT (2015) Patient-reported impact of symptoms in myotonic dystrophy type 2 (PRISM-2). Neurology 85(24):2136–2146.

    CAS  Article  PubMed  PubMed Central  Google Scholar 

  5. 5.

    Johnson NE, Heatwole CR (2013) Teaching video neuroimages: trapezius myotonia percussion sign in myotonic dystrophy type 2. Neurology 80(24):e251.

    Article  PubMed  PubMed Central  Google Scholar 

  6. 6.

    Suokas KI, Haanpää M, Kautiainen H, Udd B, Hietaharju AJ (2012) Pain in patients with myotonic dystrophy type 2: a postal survey in Finland. Muscle Nerve 45(1):70–74.

    Article  PubMed  Google Scholar 

  7. 7.

    Sansone VA (2016) The dystrophic and nondystrophic myotonias. Continuum 22:1889–1915

    PubMed  Google Scholar 

  8. 8.

    Montagnese F, Schoser B (2018) Dystrophic and non-dystrophic myotonias. Fortschr Neurol Psychiatr 86(9):575–583.

    Article  PubMed  Google Scholar 

  9. 9.

    Cannon SC (2015) Channelopathies of skeletal muscle excitability. Compr Physiol 5(2):761–790.

    Article  PubMed  PubMed Central  Google Scholar 

  10. 10.

    Trivedi JR, Cannon SC, Griggs RC (2014) Nondystrophic myotonia: challenges and future directions. Exp Neurol 253:28–30.

    Article  PubMed  Google Scholar 

  11. 11.

    Matthews E, Fialho D, Tan SV, Venance SL, Cannon SC, Sternberg D, Fontaine B, Amato AA, Barohn RJ, Griggs RC, Hanna MG, CINCH Investigators (2010) The non-dystrophic myotonias: molecular pathogenesis, diagnosis and treatment. Brain 133(Pt 1):9–22.

    CAS  Article  PubMed  Google Scholar 

  12. 12.

    Statland JM, Bundy BN, Wang Y, Rayan DR, Trivedi JR, Sansone VA, Salajegheh MK, Venance SL, Ciafaloni E, Matthews E, Meola G, Herbelin L, Griggs RC, Barohn RJ, Hanna MG, Consortium for Clinical Investigation of Neurologic Channelopathies (2012) Mexiletine for symptoms and signs of myotonia in nondystrophic myotonia: a randomized controlled trial. JAMA 308(13):1357–1365.

    CAS  Article  PubMed  PubMed Central  Google Scholar 

  13. 13.

    Stunnenberg BC, Raaphorst J, Groenewoud HM, Statland JM, Griggs RC, Woertman W, Stegeman DF, Timmermans J, Trivedi J, Matthews E, Saris CGJ, Schouwenberg BJ, Drost G, van Engelen BGM, van der Wilt GJ (2018) Effect of mexiletine on muscle stiffness in patients with nondystrophic myotonia evaluated using aggregated N-of-1 trials. JAMA 320(22):2344–2353.

    CAS  Article  PubMed  PubMed Central  Google Scholar 

  14. 14.

    Lorusso S, Kline D, Bartlett A, Freimer M, Agriesti J, Hawash AA, Rich MM, Kissel JT, David AW (2019) Open-label trial of ranolazine for the treatment of paramyotonia congenita. Muscle Nerve 59(2):240–243.

    CAS  Article  PubMed  Google Scholar 

  15. 15.

    Andersen G, Hedermann G, Witting N, Duno M, Andersen H, Vissing J (2017) The antimyotonic effect of lamotrigine in non-dystrophic myotonias: a double-blind randomized study. Brain 140(9):2295–2305.

    Article  PubMed  Google Scholar 

  16. 16.

    Schneider-Gold C, Weber F, Schoser B, Ellrichmann G, Quasthoff S, Lehmann-Horn F, Sinnreich M (2018) Myotone Dystrophien, nicht dystrophe Myotonien und periodische Paralysen. Aktuelle Neurologie 45(03):167–177.

    Article  Google Scholar 

  17. 17.

    Trivedi JR, Bundy B, Statland J, Salajegheh M, Rayan DR, Venance SL, Wang Y, Fialho D, Matthews E, Cleland J, Gorham N, Herbelin L, Cannon S, Amato A, Griggs RC, Hanna MG, Barohn RJ, CINCH Consortium (2013) Non-dystrophic myotonia: prospective study of objective and patient reported outcomes. Brain 136(Pt 7):2189–2200

    Article  Google Scholar 

  18. 18.

    Montagnese F, White M, Klein A, Stahl K, Wenninger S, Schoser B (2019) Cannabis use in myotonic dystrophy patients in Germany and USA: a pilot survey. J Neurol 266(2):530–532.

    Article  PubMed  Google Scholar 

  19. 19.

    Stockings E, Campbell G, Hall WD et al (2018) Cannabis and cannabinoids for the treatment of people with chronic noncancer pain conditions: a systematic review and meta-analysis of controlled and observational studies. Pain 159:1932–1954.

    CAS  Article  PubMed  Google Scholar 

  20. 20.

    Whiting PF, Wolff RF, Deshpande S, Di Nisio M, Duffy S, Hernandez AV et al (2015) Cannabinoids for medical use. JAMA 313:2456

    CAS  Article  Google Scholar 

  21. 21.

    Bagüés A, Martín MI, Sánchez-Robles EM (2014) Involvement of central and peripheral cannabinoid receptors on antinociceptive effect of tetrahydrocannabinol in muscle pain. Eur J Pharmacol 745:69–75.

    CAS  Article  PubMed  Google Scholar 

  22. 22.

    Cavuoto P, McAinch AJ, Hatzinikolas G, Janovská A, Game P, Wittert GA (2007) The expression of receptors for endocannabinoids in human and rodent skeletal muscle. Biochem Biophys Res Commun 364(1):105–110

    CAS  Article  Google Scholar 

  23. 23.

    Hammarén E, Kjellby-Wendt G, Lindberg C (2005) Quantification of mobility impairment and self-assessment of stiffness in patients with myotonia congenita by the physiotherapist. Neuromuscul Disord 15(9–10):610–617

    Article  Google Scholar 

  24. 24.

    Freeman TP, Hindocha C, Green SF, Bloomfield MAP (2019) Medicinal use of cannabis based products and cannabinoids. BMJ 4(365):l1141.

    Article  Google Scholar 

  25. 25.

    Mackie K (2008) Cannabinoid receptors: where they are and what they do. J Neuroendocrinol 20(Suppl 1):10–14.

    CAS  Article  PubMed  Google Scholar 

  26. 26.

    Boggs DL, Nguyen JD, Morgenson D, Taffe MA, Ranganathan M (2018) Clinical and preclinical evidence for functional interactions of cannabidiol and Δ9-tetrahydrocannabinol. Neuropsychopharmacology 43(1):142–154.

    CAS  Article  PubMed  Google Scholar 

  27. 27.

    Maccarrone M, Bab I, Bíró T, Cabral GA, Dey SK, Di Marzo V, Konje JC, Kunos G, Mechoulam R, Pacher P, Sharkey KA, Zimmer A (2015) Endocannabinoid signaling at the periphery: 50 years after THC. Trends Pharmacol Sci 36(5):277–296.

    CAS  Article  PubMed  PubMed Central  Google Scholar 

  28. 28.

    Pertwee RG (2008) The diverse CB1 and CB2 receptor pharmacology of three plant cannabinoids: delta9-tetrahydrocannabinol, cannabidiol and delta9-tetrahydrocannabivarin. Br J Pharmacol 153(2):199–215

    CAS  Article  Google Scholar 

  29. 29.

    Friedman D, French JA, Maccarrone M (2019) Safety, efficacy, and mechanisms of action of cannabinoids in neurological disorders. Lancet Neurol 18(5):504–512.

    CAS  Article  PubMed  Google Scholar 

  30. 30.

    Maccarrone M, Maldonado R, Casas M, Henze T, Centonze D (2017) Cannabinoids therapeutic use: what is our current understanding following the introduction of THC, THC:CBD oromucosal spray and others? Expert Rev Clin Pharmacol 10(4):443–455.

    CAS  Article  PubMed  Google Scholar 

  31. 31.

    Pisanti S, Malfitano AM, Ciaglia E, Lamberti A, Ranieri R, Cuomo G, Abate M, Faggiana G, Proto MC, Fiore D, Laezza C, Bifulco M (2017) Cannabidiol: State of the art and new challenges for therapeutic applications. Pharmacol Ther 175:133–150.

    CAS  Article  PubMed  Google Scholar 

  32. 32.

    Fijał K, Filip M (2016) Clinical/therapeutic approaches for cannabinoid ligands in central and peripheral nervous system diseases: mini review. Clin Neuropharmacol 39(2):94–101

    Article  Google Scholar 

  33. 33.

    Oláh T, Bodnár D, Tóth A, Vincze J, Fodor J, Reischl B, Kovács A, Ruzsnavszky O, Dienes B, Szentesi P, Friedrich O, Csernoch L (2016) Cannabinoid signalling inhibits sarcoplasmic Ca2+ release and regulates excitation-contraction coupling in mammalian skeletal muscle. J Physiol 594(24):7381–7398

    Article  Google Scholar 

  34. 34.

    Iannotti FA, Pagano E, Moriello AS, Alvino FG, Sorrentino NC, D'Orsi L, Gazzerro E, Capasso R, De Leonibus E, De Petrocellis L, Di Marzo V (2019) Effects of non-euphoric plant cannabinoids on muscle quality and performance of dystrophic mdx mice. Br J Pharmacol 176(10):1568–1584.

    CAS  Article  PubMed  Google Scholar 

  35. 35.

    Nielsen S, Germanos R, Weier M, Pollard J, Degenhardt L, Hall W, Buckley N, Farrell M (2018) The Use of Cannabis and Cannabinoids in Treating Symptoms of Multiple Sclerosis: a Systematic Review of Reviews. Curr Neurol Neurosci Rep 18(2):8.

    CAS  Article  PubMed  Google Scholar 

  36. 36.

    Moshourab R, Palada V, Grunwald S, Grieben U, Lewin GR, Spuler S (2016) A molecular signature of myalgia in myotonic dystrophy 2. EBioMedicine 7:205–211. 2016 Mar 14

    Article  PubMed  PubMed Central  Google Scholar 

  37. 37.

    Hilbert JE, Barohn RJ, Clemens PR, Luebbe EA, Martens WB, McDermott MP, Parkhill AL, Tawil R, Thornton CA, Moxley RT, National Registry Scientific Advisory Committee/Investigators (2017) High frequency of gastrointestinal manifestations in myotonic dystrophy type 1 and type 2. Neurology 89(13):1348–1354.

    Article  PubMed  PubMed Central  Google Scholar 

  38. 38.

    Duncan M et al (2008) Cannabinoid CB2 receptors in the enteric nervous system modulate gastrointestinal contractility in lipopolysaccharide-treated rats. Am J Physiol Gastrointest Liver Physiol 295:G78–G87

    CAS  Article  Google Scholar 

  39. 39.

    Sexton M, Cuttler C, Finnell JS, Mischley LK (2016) A crosssectional survey of medical cannabis users: patterns of use and perceived efficacy. Cannabis Cannabinoid Res 1(1):131–138.

    Article  PubMed  PubMed Central  Google Scholar 

Download references


We like to thank all our six patients, the Diagnosegruppe Myotone Dystrophien, Deutsche Gesellschaft für Muskelkranke, Freiburg Germany for all the enthusiasm and ongoing support for all our projects, and the Myotonic dystrophy foundation MDF USA, here especially Molly White. We thank the team at the Hubertus Apotheke in Berlin, Germany for the cooperation in preparing the CBD/THC oil used.


Hubertus Apotheke Berlin was supported by an unrestricted grant of Nexien Biopharma, Inc., for the preparation of the CBD/THC oil.

Author information



Corresponding author

Correspondence to Federica Montagnese.

Ethics declarations

Conflicts of interest

FM, KS, and SW have no conflicts of interest. BS is a scientific advisor of Nexien BioPharma, Inc.

Ethical standard

This study followed the principles of the Declaration of Helsinki. As open labelled compassionate use treatment, no formal IRB approval was submitted.

Rights and permissions

Reprints and Permissions

About this article

Verify currency and authenticity via CrossMark

Cite this article

Montagnese, F., Stahl, K., Wenninger, S. et al. A role for cannabinoids in the treatment of myotonia? Report of compassionate use in a small cohort of patients. J Neurol 267, 415–421 (2020).

Download citation


  • Myotonia
  • Cannabis
  • Cannabidiol
  • Tetrahydrocannabinol
  • Myotonic dystrophy