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

Abstract

Background

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.

Methods

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.

Results

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.

Conclusions

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.

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Acknowledgements

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.

Funding

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

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Correspondence to Federica Montagnese.

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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.

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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). https://doi.org/10.1007/s00415-019-09593-6

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Keywords

  • Myotonia
  • Cannabis
  • Cannabidiol
  • Tetrahydrocannabinol
  • Myotonic dystrophy