Cannabis for medicinal use in patients with rare diseases

• Published in: Translational science of rare diseases Vol. 6; no. 4; pp. 123 - 135
• Main Authors: Parovincaka, Jekaterina, Szijj, Janis Vella, Serracino-Inglott, Anthony, Azzopardi, Lilian M.
• Format: Journal Article
• Language: English
• Published: London, England SAGE Publications 30.09.2023
• Subjects: Rare diseases; CBD; medicinal cannabis; THC
• ISSN: 2214-6490; 2214-6512
• DOI: 10.3233/TRD-230060

BACKGROUND: Patients with Rare Diseases (RDs) present with chronic and debilitating symptoms such as pain, anxiety and epileptic seizures. Symptoms can be unresponsive to conventional treatment and may lead to a decreased Quality of Life for patients. Cannabinoids have been reported to be efficacious against chronic pain refractory to conventional analgesics, anxiety and seizures. OBJECTIVE: Identification of RDs for which Medicinal Cannabis (MC) can be used and identification of issues related to RDs and perceptions on the use of MC in patients with RDs. METHODS: Study was divided into 2 phases. Phase 1: Literature Review to identify RDs in which cannabis or cannabinoids are used Phase 2: Development, validation and dissemination of 2 questionnaires for: (i) Health Care Professionals (HCPs) and (ii) RD patients. RESULTS: Cannabinoids were described as possible therapeutic agents in 20 RDs. The questionnaires were completed by 101 HCPs and 38 RD patients. Thirty-three HCPs had no experience on use of MC but would consider using it in their practice for management of RDs. Most patients (n = 29) did not have experience with use of MC and 20 patients would consider using MC to treat their condition or relieve symptoms of their disease. CONCLUSION: The study helps identify the potential of MC use in RDs associated with chronic symptoms such as pain, muscle spasticity, seizures and anxiety.