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Cannabinoid Therapies in Less-Common Disorders: Clinical Evidence and Formulation Strategies.

Silvia Afonso1, Joana Gonçalves2, Ana T Brinca1,3,4

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Cannabinoids show therapeutic promise for rare diseases beyond epilepsy and pain. Further research and innovative drug delivery systems are needed to overcome challenges and explore their full potential.

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Area of Science:

  • Pharmacology
  • Medical Research
  • Drug Development

Background:

  • Cannabinoids possess therapeutic potential beyond established uses like chronic pain and epilepsy.
  • Emerging research suggests cannabinoids may be effective for orphan and rare diseases.
  • This opens new avenues for pharmaceutical research and clinical applications.

Purpose of the Study:

  • To critically synthesize recent evidence (2020-2025) on cannabinoid use in rare and complex disorders.
  • To review challenges and opportunities in cannabinoid-based therapies.
  • To highlight advancements in pharmaceutical formulations and delivery systems.

Main Methods:

  • Literature review of studies published between 2020-2025.
  • Searches conducted in PubMed and Scopus databases.
  • Focus on refractory epilepsies, movement disorders, rare dermatological conditions, and Crohn's disease.

Main Results:

  • Cannabinoids show potential in conditions like refractory epilepsies, dystonia, Tourette syndrome, epidermolysis bullosa, and Crohn's disease.
  • Negative trial outcomes (e.g., Fragile X syndrome) provide insights into challenges.
  • Optimized pharmaceutical formulations (oromucosal sprays, transdermal gels, nanocarriers) aim to improve bioavailability and patient response.

Conclusions:

  • Cannabinoids are a promising, yet underexplored, therapeutic option for rare and complex disorders.
  • Further investigation is warranted, supported by innovative pharmaceutical approaches.
  • Addressing safety, regulatory aspects, and conducting robust clinical trials are crucial.