Cannabis and cannabinoid compounds may provide many complex solutions to these rare diseases. Medical cannabis programs are now helping to discover these solutions and deliver them to the patients in dire need of them. 

There has been an increasing awareness that cannabis can help manage pain, inflammation, or neurogenerative diseases such as Alzheimer’s and Parkinson’s. But the true potential of using medical cannabis for rare diseases and disorders has yet to be unlocked. 

Let’s see what research says so far.

Myasthenia Gravis

An autoimmune condition causes progressive weakening of the skeletal muscles. It affects around 200 million people worldwide. The main symptoms are drooping eyelids, blurred vision, difficulty chewing, swallowing, and speaking. The autoimmune condition is caused by antibodies attacking nicotinic acetylcholine receptors that block the standard transmission of acetylcholine, the neurotransmitter responsible for managing nerve impulses with muscle movement. 

There is no cure for it – however, many studies have shown THC’s ability to inhibit acetylcholinesterase and lessen the degradation rate of acetylcholine.

Tuberous Sclerosis Complex (TSC)

A rare genetic disorder causes benign tumors to grow within the brain, eyes, and other vital organs. Diagnosed usually in childhood, it can cause epilepsy, autism, and developmental delay – these symptoms may vary between individuals. In addition, it has been suggested that the endocannabinoid system might play a role in cell division and death processes. 

In addition, CBD is a crucial anti-epilepsy drug that can manage seizures in TSC. 

Mitochondrial Disease

It is a group of genetic disorders affecting the mitochondria, the energy-generating engines of cells. It causes neurological impairment, chronic pain, muscle weakness, seizures, poor hearing and vision, multi-organ disease, learning disabilities, and respiratory disorders. The children born with this usually display symptoms by the age of 10. 

It is thought that the antioxidant abilities of some cannabinoids may work directly to improve mitochondrial function. 

Behcet’s Disease (BD)

It is an autoimmune disorder in which the immune system attacks multiple parts of the body. It can cause skin and genital lesions, joint swelling, chronic pain, swelling in the brain, blood clots, etc. there’s no cure; however, anti-inflammatory drugs might provide relief.

Several cannabinoids, the most famous ones including THC and CBD, are recognized for their anti-inflammatory effect and properties, and some US BD patients have stated subjective relief of symptoms when using cannabis. 

Neuromyelitis Optica (Devic’s Syndrome)

It is a rare autoimmune disease that causes recurrent inflammation and demyelination of the optic nerve and spine. As a result, it causes ongoing loss of vision and spinal cord function; along with that, it can lead to muscle weakness, bowel/bladder control, lack of coordination, and loss of sensation. 

In a study conducted in 2013 into neuropathic pain and hyperintensity resulting from NMO, the endocannabinoids 2-AG and anandamide were raised in patients compared to healthy controls. It was then concluded that these endocannabinoids are released at higher levels in NMO sufferers to reduce pain and sensitivity and prevent hyperalgesia. 

Ehlers-Danlos Syndrome (EDS)

Ehlers-Danlos syndrome (EDS) can trigger hypermobile, unstable joints, hyperplastic skin, chronic pain, deformities of joint and spine, muscle spasms, and cardiovascular complications. It is a group of genetic disorders that affects the skin and the connective tissues of the joints. EDS is caused by mutations in specific vital genes, which help control the expression of proteins crucial to collagen production. 

EDS sufferers have anecdotally reported subjective relief from neuropathic pain and spasm when using cannabis and cannabinoid-based treatments. In addition, the compound palmitoylethanolamide (PEA) has also been used to keep neuropathic pain in control in EDS. It is available under the brand name PeaPure in some countries. 

Although it is not a standard cannabinoid, PEA is known to have an affinity for GPR55 and GPR119 receptors, which are part of the endocannabinoid system. It is also known to augment the effects of anandamide by an entourage effect.


It is a rare group of autoimmune disorders affecting the skin and the mucous membranes, particularly the mouth. Pemphigus causes itchiness, often painful blisters and sores that can spread to cover many bodies. If left uncured, it can cause runaway infections, which can be deadly. The most common treatments include several high-strength steroids, including prednisone, which have a range of severe side effects when used long-term. 

It is caused by pathogenic antibodies that attack a protein known as desmoglein. Without this protein, the epidermal cells cannot stick together as expected, and the skin and mucous membranes layer slough gradually off. 

Cannabis-based treatments are effective against several autoimmune disorders affecting the skin, including psoriasis and epidermolysis bullosa. Furthermore, cannabinoid treatments (especially topical creams and ointments) may also confer significant antibacterial, antiviral, and antifungal effects, helping to prevent secondary infection. 

Numerous patent applications for cannabis-based topicals for pemphigus have been submitted, and there are several anecdotal reports of patients experiencing relief from their symptoms. 

The Bottom Line

Cannabis has become a regular part of medicinal therapeutic in patients because of its healing properties and purposes. However, these aren’t the only rare diseases that may benefit from cannabis. Plenty of disorders may help shortly, but that requires further research and robust results. 

Medical Cannabis In The Fight Against Rare Diseases

Medical Cannabis In The Fight Against Rare DiseasesMedical Cannabis In The Fight Against Rare Diseases Medical Cannabis In The Fight Against Rare Diseases Medical Cannabis In The Fight Against Rare DiseasesMedical Cannabis In The Fight Against Rare Diseases
Medical Cannabis In The Fight Against Rare Diseases
Medical Cannabis In The Fight Against Rare Diseases
Medical Cannabis In The Fight Against Rare Diseases
Medical Cannabis In The Fight Against Rare Diseases