Cannabinoid Treatments for Rare and Less-Common Diseases: What We Know So Far | Research

The takeaway

Cannabinoids show promise for rare conditions like refractory epilepsies, dystonia, Tourette syndrome, and epidermolysis bullosa, but advanced drug delivery systems are needed to overcome bioavailability challenges.

Patients with rare diseases exploring cannabinoid options, rare disease researchers, and pharmaceutical developers working on cannabinoid formulations

What the researchers found

Recent evidence supports cannabinoid use in rare epilepsies beyond Dravet/Lennox-Gastaut, movement disorders, and rare skin diseases, while Fragile X syndrome trials revealed methodological challenges instructive for future research.

Why it matters

Patients with rare diseases often have few treatment options — cannabinoids could fill therapeutic gaps, but only if formulation challenges like poor bioavailability are solved.

The numbers in context

Review covered evidence from 2020-2025 across multiple rare conditions including refractory epilepsies, dystonia, Tourette syndrome, epidermolysis bullosa, and Crohn's disease.

How the study worked

Critical review synthesizing 2020-2025 evidence from PubMed and Scopus on cannabinoid therapies in less-common disorders, including formulation and delivery optimization strategies.

What this study cannot tell us

Most evidence is preclinical or from small clinical studies; the review covers heterogeneous conditions making direct comparisons difficult.

How to read the evidence

Comprehensive review of recent literature, but underlying evidence base is largely preclinical with small clinical studies for most conditions reviewed.

When this study was published

Published in 2026, covering the most recent 5 years of evidence (2020-2025) in this rapidly evolving field.

The bigger picture

The intersection of cannabinoid medicine and orphan diseases represents a frontier where unmet medical need is high and novel delivery systems could make the difference between theoretical promise and clinical reality.

Questions still open

Which rare disease applications will advance to pivotal trials first? Can nanocarrier systems and transdermal gels solve cannabinoid bioavailability problems?

Common questions

Can cannabinoids help with rare diseases?

There's emerging evidence for conditions like rare epilepsies, dystonia, Tourette syndrome, and epidermolysis bullosa, but most evidence is preliminary and more clinical trials are needed.

Why is drug delivery a challenge for cannabinoid medicines?

Cannabinoids have poor bioavailability when taken orally, meaning much of the dose is lost before reaching its target. New delivery systems like nanocarriers and transdermal gels aim to solve this.

Read the original research

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

Diseases (Basel, Switzerland), 14(2)

View on PubMed ↗Publisher’s paper ↗

Citation

Afonso, Silvia; Gonçalves, Joana; Brinca, Ana T; Rosendo, Luana M; Rosado, Tiago; Duarte, Ana Paula; Gallardo, Eugenia. (2026). Cannabinoid Therapies in Less-Common Disorders: Clinical Evidence and Formulation Strategies.. Diseases (Basel, Switzerland), 14(2). https://doi.org/10.3390/diseases14020083