What are Rare Disorders?
According to the National Center for Advancing Translational Sciences (NCATS), a rare disorder or disease is a condition that affects fewer than 200,000 people in the United States. Rare diseases are also known as orphan diseases because drug companies were uninterested in developing treatments for these (1).
Hence, the U.S. Congress created the Orphan Drug Act of 1983 to provide financial incentives for pharmaceutical companies to develop a treatment for these rare diseases (2).
NCATS says that there may be around 7,000 rare diseases, with 25 to 30 million Americans afflicted with these. When someone is diagnosed, only a few types of rare diseases are tracked, making it difficult to determine the exact number of rare diseases and sufferers in the U.S.
One of these rare diseases is corticobasal degeneration.
What is Corticobasal Degeneration?
Corticobasal degeneration is a rare, progressive disorder that causes nerve cell
loss and degeneration of several parts of the brain.
Causes of corticobasal degeneration have yet to be determined, but researchers have observed that people suffering from the disease have accumulated abnormal levels of tau, a protein that is found in the brain(3).
Too much tau in the brain cells could lead to the deterioration of these cells and cause symptoms of corticobasal degeneration. Tau protein is also associated with neurodegenerative diseases. These include Alzheimer’s, progressive supranuclear palsy (PSP), and frontotemporal dementia. (4)
The symptoms usually appear in people around 60 years old. Initially, only one side of the body is affected, but eventually, the symptoms will affect both sides.
Symptoms include (5):
- Poor coordination and rigidity (similar to Parkinson’s)
- Memory loss
- Visual-spatial problems
- Apraxia (inability to make familiar and purposeful movements)
- Hesitant and halting speech
- Myoclonus (involuntary muscular jerks)
- Dysphagia (difficulty swallowing)
People who are suffering from other degenerative diseases like Alzheimer’s, Lewy body disease, and PSP experience the same corticobasal degeneration symptoms. Because of this, these symptoms have been dubbed as “corticobasal syndrome.” (6)
In the advanced stages, patients experience dementia, loss of inhibition, and behavioral changes like lack of empathy. They also experience an inability to communicate and ambulate as well as difficulty walking and balancing. (7)
Over six to eight years, the disease progresses gradually and could lead to death. The usual causes are pneumonia, a severe infection of the blood (sepsis), or a blood clot in the lungs (pulmonary embolism). (8)
Some medications like clonazepam can help with the involuntary muscle jerks. Likewise, Botox and therapy (occupational, physical, and speech therapy) can help manage corticobasal degeneration symptoms; however, there is no specific treatment for the disease (9).
CBD and Corticobasal Degeneration
A study in 2005 (10) reveals that cannabidiol (CBD), the non-psychoactive component of cannabis plants, inhibits Tau.
Tau is the protein that accumulates in the brain cells of people suffering from corticobasal degeneration. It is also associated with several neurodegenerative diseases.
The researchers conclude that CBD is a possible pharmacological tool in treating these diseases, especially since it has “extremely low toxicity in humans.” (11)
Another study in 2017 reports that CBD has beneficial effects on patients who have Alzheimer’s disease, Parkinson’s disease, and multiple sclerosis. The study discusses CBD’s therapeutic properties, like its neuroprotective effects on several pathological conditions (12).
According to the researchers of the 2017 study, CBD’s antioxidant and anti-inflammatory properties benefit parts of the brain responsible for the development and maintenance of neurodegenerative diseases (13).
Some of the diseases in the 2017 study have symptoms shared by patients suffering from corticobasal degeneration. Hence, CBD use may be an alternative that can be explored.
CBD comes in various forms like gummies, tinctures (drops), patches, balms, and even gelcaps.
The use of CBD has not been approved by the U.S. Food and Drug Administration, so there is no standard dosage. It is recommended that patients begin with small doses and, if there are no adverse effects, gradually increase the dosage.
Before adding CBD products to a patient’s medication regimen, it is recommended to consult with a doctor first.
Several rare diseases have no treatment yet. One of these is corticobasal degeneration.
Upon the symptoms’ appearance in patients, it only takes six to eight years before the disease becomes life-threatening.
There are no known treatments for corticobasal degeneration. A study (14), however, has found that CBD may inhibit Tau, the protein that may be one of the causes of corticobasal degeneration.
Another study on neurodegenerative diseases also reports CBD’s neuroprotective benefits on pathological conditions (15).
CBD may be an alternative that can help treat corticobasal degeneration. However, there is no proven benefit of CBD yet for rare diseases at this early stage of research. It is essential to consult with a doctor first before taking CBD for any reason.
- “FAQs About Rare Diseases.” Genetic and Rare Diseases Information Center, U.S. Department of Health and Human Services, rarediseases.info.nih.gov/diseases/pages/31/faqs-about-rare-diseases.
- “Corticobasal Degeneration.” Stanford Health Care (SHC) – Stanford Medical Center, stanfordhealthcare.org/medical-conditions/brain-and-nerves/corticobasal-degeneration.html.
- U.S. Department of Health and Human Services. op. cit.
- Stanford Health Care. op. cit.
- “Corticobasal Syndrome (CBS).” Baylor College of Medicine, www.bcm.edu/healthcare/care-centers/parkinsons/conditions/corticobasal-syndrome.
- Stanford Health Care. op. cit.
- Esposito, Giuseppe, et al. “The Marijuana Component Cannabidiol Inhibits Beta-Amyloid-Induced Tau Protein Hyperphosphorylation through Wnt/Beta-Catenin Pathway Rescue in PC12 Cells.” Journal of Molecular Medicine (Berlin, Germany), U.S. National Library of Medicine, Mar. 2006, www.ncbi.nlm.nih.gov/pubmed/16389547.
- Mannucci, Carmen, et al. “Neurological Aspects of Medical Use of Cannabidiol.” CNS & Neurological Disorders Drug Targets, U.S. National Library of Medicine, 2017, www.ncbi.nlm.nih.gov/pubmed/28412918.
- Esposito, G. (2006 Mar). op. cit.
- Mannucci, C. (2017) op. cit.