CBD and Rare disease

February 29 is the international rare disease day.

The main objective of rare disease day is to raise awareness amongst the general public and decision-makers about rare diseases and their impact on patients’ lives.

The campaign targets primarily the general public and also seeks to raise awareness amongst policymakers, public authorities, industry representatives, researchers, health professionals and anyone who has a genuine interest in rare diseases.

The term ‘rare diseases’ refers to diseases that affect a small number of people in relation to the general population. Rare diseases are often referred to as “orphan diseases”. Orphan means – a child whose parents are dead.

How was this name created?
Many rare diseases affect one in hundreds of thousands or even less. Such patients are vulnerable as “orphans” in terms of health care and social solidarity. They are the most vulnerable psychologically, socially and economically. This is because, unlike common diseases, rare diseases are mostly not on the list of priorities of politicians and health care providers, and therefore the care of patients is negligent and of poor quality. This is understandable as it is not possible to schedule when a patient with an extremely rare disease will appear in the doctor’s practice. Most family doctors do not encounter or even recognize most of the rare diseases throughout their working lives.

Similarly, the pharmaceutical industry has been reluctant to undertake in-depth research and development of new medicine in areas where there are only a few hundred patients worldwide, as no great profit is expected.

As a result, high-quality healthcare, including medicines, was not available to patients and often still is not available.

Therefore, in 1983, the US government adopted the Orphan Medicines Act, which promoted medicine research and development. For example, companies wishing to invest in pharmaceutical research and development have given the state of opportunity to set a price for this medicine in the future that guarantees a return on their investment. Due to the relatively small number of patients, the cost of the medicine is often relatively high and the usual methods for measuring the cost-effectiveness of the medicine do not work. Such medicines used to treat rare diseases are called ‘orphan medicines’. In the European Union and in several countries around the world, the Orphan designation (‘orphan designation’) is being granted to certain medicinal products during the registration process. Usually, this group of medicines is covered by state funds.

To date, there are dozens of medicines on the Orphanet list of “Orphan Medicinal Products”.

The disease is considered rare in the European Union if it affects no more than 5 per 10,000 (1 in 2,000) people.

The world is divided on what is a rare disease. That is why each country is developing its own strategy for rare diseases. Because what is a common disease in one country, may be listed as a rare disease in another.

To date, up to 8,000 rare diseases are known, affecting between 6% and 8% of the population in their lifetime. Thus, between 27 and 36 million people in the European Union suffer from them.

As you can see, this is already a large number of patients, but it should be borne in mind that many rare diseases are not life-threatening and people feel healthy.

Most rare diseases cannot be completely cured, but timely diagnosis, appropriate treatment and additional medical care can improve patients’ quality of life and extend their life expectancy.

Today, more and more new diseases are being recorded thanks to ever more precise laboratory testing methods. There are several classifications of rare diseases that are constantly being updated and developed.

In Europe, such a classification is available on Orphanet. Orphanet is an information portal providing information to the general public in the field of rare diseases and orphan medicine to help improve the diagnosis, care and treatment of rare disease patients. Orphanet is run by a European consortium of around 40 countries, coordinated by the project’s French lead partner, INSERM. The Orphanet database contains information on more than 6,000 diseases as well as specialized services across Europe.

Orphanet maintains a list of orphan drugs available in Europe and provides access to information on orphan medicine still under development. For medicines already on the market, Orphanet offers search by disease name, product type, active ingredient/medicine name, sponsor and country.

About 80% of rare diseases are of genetic origin. Many rare diseases have their origin in exposure to environmental factors such as infections and allergies. In many cases, the causes can be difficult to determine, such as autoimmune diseases, rare malignancies, degenerative and proliferative diseases. The causes of many rare diseases are still unknown.

Many rare diseases have a chronic and progressive course.

Signs of rare diseases are often seen as early as newborns or childhood. Examples of diseases include tyrosinemia, galactosemia, albinism, Williams syndrome, mucoviscidosis, osteogenesis imperfecta, chondrodysplasia, or Rett syndrome.

For many rare diseases, the first clinical symptoms can be seen predominantly in adults, such as Huntington’s disease, Crohn’s disease, amyotrophic lateral sclerosis, Kaposi’s sarcoma, thyroid malignancy.

Some rare diseases can be successfully treated by following a specific diet without the need for specific medication, but many diseases require specific medication.

When it comes to cannabidiol (CBD) and rare diseases, the only official mediums with CBD on Orphans right now are the EPIDIOLEX mentioned on our blogs before for Dravet and Lennox-Gastaut syndrome patients. EPIDIOLEX is the first and only FDA-approved cannabidiol (CBD) for the treatment of Dravet and LGS.
It is used as adjunctive therapy to seizures associated with Lennox-Gastaut Syndrome (LGS) or Dravet Syndrome (DS) in combination with clobazam in patients aged 2 years and over.

Dravet’s Syndrome (DS) is a genetic childhood epilepsy characterized by a variety of drug-resistant seizures, often caused by fever, occurring in previously healthy children and often leading to cognitive and motor disorders.
Lennox-Gastaut Syndrome (LGS) is a rare syndrome that belongs to the group of severe childhood epilepsy encephalopathies.

Benefits of using CBD oil to treat the symptoms of Dravet’s syndrome.
It is no secret that CBD oil has a number of potential health benefits, from reducing inflammation and anxiety to helping people with ADHD.

Many traditional treatments for Dravet’s Syndrome have a number of harmful side effects and some conventional medications can worsen this condition. These side effects make the treatment of Dravet’s syndrome particularly difficult.

First-line anticonvulsants commonly prescribed to Dravet’s syndrome sufferers include: Clobazam, which may cause side effects such as drowsiness, fatigue, weakness, constipation including insomnia, urinary tract infections, vomiting, pneumonia, severe suicidal thoughts and behavior in rare cases; valproic acid, which has side effects including dizziness, drowsiness, problems with walking or body coordination, vomiting and nausea, mood changes that may include anxiety, depression and irritability, ramping up, in rare cases.

Among children, these side effects can be prolonged or more severe, and can make an already difficult condition even more painful.

Use of CBD oil to treat the symptoms of Dravet’s syndrome.
In contrast, CBD oil has little or no side effects and, according to the World Health Organization (WHO) report, is “generally well tolerated, with a good safety profile”.
Unlike the whole Cannabis plant, CBD oil contains only tetrahydrocannabinol (THC) trace elements, so it has no intoxicating effects that are typically associated with Cannabis use.

There are several options available for the types of CBD products you can use to manage your child’s Dravet-induced epilepsy. CBD oil capsules and drops are the most common choice, but your choice depends on what is the easiest and most comfortable for your child. For small children, capsules may be difficult to swallow, and water-soluble droplets of CBD oil are easier to take.

Talk to your child’s doctor and try some admission techniques until you find the best one that best suits your needs. You may also want to consider talking to a doctor who specializes in CBD as an adjunct therapy.

CBD can help to reduce inflammationpainanxiety and depressionstabilize moodimprove sleep. CBD has antioxidant and immune-boosting properties.
Most health problems also result from imbalances in the whole body, including rare diseases.

In a healthy body, everything is as it should be, and in the body, everything is in balance. In an unhealthy body, something is too much or too little and it disrupts the body’s natural balance.

When it comes to homeostasis in the body, we cannot fail to mention the endocannabinoid system. The endocannabinoid system is a system of the human body that plays an important role in our internal biological system throughout the rest of our lives: affecting or regulating appetite and digestion; metabolism; chronic pain; inflammation and other immune system reactions; mood; learning and memory; motor skills and movement; sleep; cardiovascular function; muscle building; bone regeneration; health and growth; liver function; function of the reproductive system; stress; skin and nerve function; communication between the cells of the human body and much more.

Not least, Cannabis plays an important role in the human endocannabinoid system. The human biological system responds to the active ingredients of Cannabis, called cannabinoids, and through these receptors, the beneficial effects of the plant work. ECS exists and is active in our body even when we are not using Cannabis.

The endocannabinoid system is important for many basic human functions such as appetite, immune system, metabolism, digestive system and disorders, pain, sleep, memory, mood, inflammatory response, etc.
In addition to regulating these basic functions of the body, it also has an effect on the physiological response of the disease.

For inflammation. Scientists have discovered that cannabidiol (CBD) is effective against inflammation, and research has pointed out several ways that this natural molecule has anti-inflammatory effects.
These and other reasons why so many people incorporate CBD products into their anti-inflammatory routine.

With CBD for pain, there are several ways to improve well-being, pain relief or complete pain relief, sleep quality improvement, anxiety reduction, and, not least, the emergence of hope. CBD helps with sleep problems. Sleep is an extremely important aspect of life. This is the time when our bodies and brains renew themselves and prepare us for the next day.

For sleep. CBD does not cause sleep through sedation or make the user feel tired or sleepy. In fact, CBD can even make users feel more alert during the day while promoting a balanced sleep-wake cycle. In addition, the CBD helps to fall asleep by easing conditions that might keep people awake at night, such as anxiety, stress and pain.
By promoting a balanced sleep cycle and reducing symptoms that make it difficult to fall asleep, CBD can soothe and relax the user, making it easier to fall asleep.

As antioxidant. New studies show that cannabinoids are a great, if not the richest, source of antioxidants known to man (a study published in 1998 was one of the first to document the antioxidant properties of CBD and found that CBD is a stronger antioxidant than vitamins C and E).
The CBD structure also contains a free oxygen atom2. It is an atom that is donated to free radicals, thus neutralizing them. It is a free oxygen atom that provides the antioxidant property of CBD, and it does a great job of getting rid of free radicals.
There are many ways in which antioxidants can help clean up or even prevent cell contamination.
More importantly, CBD helps the body regulate its own antioxidant systems, which help to boost overall homeostasis and slow down aging.

For the immune system. Cannabinoids such as THC and CBD are immunosuppressive (those that block the response of our body’s defense system), which explains the relief Cannabis medical users experience with autoimmune diseases and chronic inflammation. Other studies suggest that regular use of Cannabis can increase the amount of white blood cells in immunodeficiency disorders, such as HIV, which is indicative of an immune-boosting effect.
Scientists also believe that the effects of Cannabis not only affect the body’s endocannabinoid system, but also interact with all the biological activities in the body, including our immune system.

For mental health. In most studies, CBD has a positive interaction with serotonin receptors in the brain. Serotonin affects a variety of bodily functions, including a person’s emotional state and well-being or happiness. Maintaining serotonin levels is often the main therapy for people who are depressed or depressed due to a difficult diagnosis.

The function of the endocannabinoid system is homeostasis – the regulation of the body system under all conditions while maintaining internal balance.
Bad health is largely the result of the body’s inability to achieve homeostasis. Therefore, the endocannabinoid system and balance in the body are crucial and in this case, CBD can be a good support tool for many rare diseases.

The information on the internet about CBD products and their use will be ambiguous, so we always recommend reading safe and reliable sources. We also use websites related to the Cannabis industry, research and education to prepare information.

The content of this site is for informational purposes only. We are not medical experts and this should not be interpreted as medical advice. Be sure to consult your doctor before undergoing CBD or other treatment.

The chemistry of every human body and brain is unique, so the use of CBD to improve mental as well as physical health does not guarantee results. However, many GWEEPRODUCTS CBD oil users have included it into their daily routine as one of the therapies for improving mental, physical health and quality of life.

References: cbdoilreview.org; orpha.net; epidolex.com; vesels.lv

Photo from Unsplash.

Scroll to Top