Cannabis for medical use in rare diseases

Abstract

Rare diseases (RDs) are severe and usually chronically debilitating and RD patients often lack effective and accessible treatment options. Medicinal Cannabis (MC) is used for symptoms such as pain, spasticity, nausea and vomiting, seizures and anxiety which may be experienced by RD patients. The aim of the study was to identify RDs for which MC can be used and issues related to its use in patients with RDs. The methodology was in two parts: (1) systematic literature review using search engines: PubMed and MEDLINE. Open access peer review journal articles, published between January 2010 – October 2021 were included; (2) development, validation and dissemination of two questionnaires: for RD patients and for healthcare professionals (HCPs) in Malta. The literature revealed 36 papers that described the use of MC as a possible therapeutic option in 22 different RDs, mainly epileptic conditions (n=7) and neurodegenerative diseases (n=6). Respondents of the questionnaire for HCPs (n=101) were pharmacists (n=40), general practitioners (n=17) and occupational therapists (n=13), with more than 11 years of practice (n=46). HCPs encountered two to four RD patients a year on average. Symptoms experienced by RD patients were pain (n=51), mainly chronic neuropathic pain (n=31), anxiety (n=34) and muscle spasticity (n=33). Fifty-nine HCPs agreed to reply to MC related questions. Twenty-six of 59 HCPs have used MC in their practice. Fifty two out of 59 HCPs consider it to be effective for pain relief, 38 for anxiety and 38 for muscle spasticity. Thirty six out of 59 HCPs agreed on the use of MC in their practice. Regarding the side-effects of MC, confusion (n=30) and addiction (n=29) were reported to be of the most concern. Study included thirty-eight patients with RDs, mostly 41-50 years old (n=11) and reported pain (n=24), anxiety (n=22) and muscle spasticity (n=10) as commonly experienced symptoms associated with RD. Seven reported experiencing side-effects associated with the currently used medications. Two respondents had been prescribed MC by a HCP, though 20 would consider MC use to relieve symptoms of their disease. Confusion, possibly associated with MC usage, was reported as the side-effect causing most concern (n=8). Eighteen patients were not concerned with MC side-effects. Literature supports the use of MC for management of RDs. MC can be effective to relief pain, anxiety and muscle spasticity possibly experienced by RD patients. HCPs and RD patients consider that MC can be used in management of RD symptoms. In lack of efficacious treatment options for RD patients, MC can be an alternative therapy for symptom relief