According to the FDA (Food and Drug Administration), about 7,000 rare disorders affect more than 30 million individuals in the U.S.(1). If you want to try CBD to help with a specific condition, read on to know more about the non-psychoactive compound and its effect on rare disorders. 

How CBD May Help With Rare Disorders

The FDA has approved hundreds of substances for rare disorders ever since the passage of the Orphan Drug Act(2).

The Orphan Drug Act defines a rare condition as an illness that affects less than 200,000 individuals in the U.S.(3). The act also incentivizes drug development for rare diseases.

A study from the University of South Carolina School of Medicine has led the FDA to grant an orphan drug designation for CBD (cannabidiol) due to the compound’s potential therapeutic effects on autoimmune hepatitis(4).

Autoimmune hepatitis is a rare disorder in which the immune system attacks liver cells, leading to liver inflammation(5).

CBD is a plant-based cannabinoid derived from cannabis plants. The compound lacks the psychotropic effect of THC (tetrahydrocannabinol), the primary mind-altering component of cannabis.

CBD has purported therapeutic benefits, including analgesic and anti-inflammatory properties(6).

Aside from CBD’s therapeutic potential, its activity in the ECS (endocannabinoid system) may be why public and medical interest in the non-psychoactive compound have been rising.

The ECS comprises cannabinoid receptors, endocannabinoids, and enzymes.

The endocannabinoid system modulates various bodily functions, such as emotional processing, learning and memory, sleep, appetite, temperature and pain control, and inflammatory and immune responses(7).

CBD and THC may interact with the cannabinoid receptors, namely CB1 and CB2(8).

CBD is believed to have a low binding affinity for cannabinoid receptors. However, CBD may activate them indirectly through anandamide, a fatty acid neurotransmitter, and vanilloid receptors(9).

TRPV1, or transient receptor potential vanilloid subtype 1, is a potential target for some of the pharmacological effects of CBD(10).

Prakash Nagarkatti, Ph.D., said that CBD may suppress the processes that create inflammation by binding to the vanilloid receptors. This activity may result in an increased life expectancy and decreased risk of liver failure in patients with autoimmune hepatitis(11).

Meanwhile, a study published in the British Journal of Pharmacology using a rat model suggested that vanilloid receptors may mediate the antihyperalgesic effect of CBD(12).

Antihyperalgesics serve to counter hyperalgesia or increased pain sensitivity.

In pathological conditions like neuropathy, in which TRPV1 receptor sensitivity and expression are increased, CBD may represent a useful pharmacological option to help with disease-associated chronic pain(13).

Neuropathy refers to nerve damage that usually results in muscle weakness, pain, and numbness in the affected area(14).

A 2020 study suggested that topical CBD oil may provide pain relief to patients with peripheral neuropathy(15).

The putative usefulness of CBD for pain and inflammation is plausible considering the compound’s pharmacology. Still, more animal studies and clinical trials are warranted to elucidate how CBD may help with rare disorders.

Benefits of Using CBD for Rare Disorders

A study from the Journal of Korean Medical Science examining the efficacy and safety of CBD in patients with DS (Dravet syndrome) and LGS (Lennox-Gastaut syndrome) indicated that CBD may be safe and tolerable(16).

In addition, Korean researchers hypothesized that CBD may reduce the seizure frequency in pediatric patients with DS or LGS(17).

DS is a rare and drug-resistant form of epilepsy that starts during infancy and proceeds with accumulating morbidity, impacting people throughout their lifetime(18).

Meanwhile, LGS is a severe form of epilepsy characterized by recurring seizures early in life(19).

MS (multiple sclerosis) is another rare disorder characterized by chronic course with intermittent relapses. The condition usually ends with severe debilitation(20).

A study from Frontiers in Neurology suggested that CBD may improve mobility in individuals with multiple sclerosis(21).

On the other hand, corticobasal syndrome is a rare disorder that shares some features with Parkinson’s disease, like tremor at rest, postural instability, slowness of movement, and stiffness(22).

Corticobasal syndrome also causes problems with thinking and memory(23).

There is no scientific data directly examining the benefits of CBD on the condition.

However, a study from the Brazilian Journal of Psychiatry mentioned that oral CBD doses ranging from 150 to 400mg daily combined with antiparkinsonian agents may reduce psychotic symptoms among individuals with Parkinson’s disease(24).

Research suggests that 40% of individuals with Parkinson’s disease have anxiety disorders(25). CBD has purported anti-anxiety properties(26).

Although these studies seem promising, they are inconclusive. Therefore, more longitudinal research is warranted to confirm the health benefits of CBD for rare disorders.

How to Use CBD Oil for Rare Disorders

Ensure to work with a healthcare professional with a background in recommending CBD so that delivery methods and dosage can be fine-tuned.

Suppose you do not know a doctor experienced in cannabis use. The details below may help you initiate a conversation with your healthcare provider regarding the use of CBD for rare disorders.

Delivery Methods

The sublingual delivery method is a fast-acting solution with a 15- to 30-minute onset(27). This administration route relies on the mucous membrane under the tongue to absorb cannabidiol(28).

Meanwhile, research from the University of Minnesota suggested that high-fat foods may improve CBD absorption into the body(29). In contrast to fasting, taking CBD with food may increase the amount of the compound in the body by four times.

If you choose to take CBD via the ingestion route, the compound’s onset time is between 30 and 90 minutes(30).

CBD Dosage 

The U.S. FDA (U.S. Food and Drug Administration) has not approved the marketing of products with cannabis extracts as a treatment for most medical conditions(31).

However, the agency approved Epidiolex, a drug that contains CBD, to treat rare and severe forms of epilepsy and seizures linked to tuberous sclerosis complex in patients one year and older(32).

Tuberous sclerosis complex refers to a genetic disorder characterized by the growth of benign tumors.

Korean researchers who examined the efficacy and safety of CBD in DS and LGS administered CBD orally to patients at a starting dose of 5mg per day. They maintained the dosage at 10mg daily(33).

The results showed that the use of CBD may be safe and tolerable and may help reduce seizure frequency in pediatric patients with DS or LS(34).

Since the study only focused on specific forms of epilepsy, it is best to consult a doctor versed in cannabis use to know how much CBD you need to take for your rare medical condition.

How Long Does CBD Take to Help With Rare Disorders?

How long CBD takes to help with rare disorders depends on several factors, including the administration route and body weight.

For instance, you will feel the effects of CBD via the ingestion route between 30 and 90 minutes. Usually, the compound’s effects will last for 6 to 8 hours(35).

Meanwhile, taking CBD sublingually has an onset time of 15 to 30 minutes, and the effect of CBD typically lasts for 2 to 4 hours(36).

How Long Will CBD Help With Rare Disorders?

How long CBD will last after it takes effect to help with rare disorders depends on its half-life.

The half-life of a particular drug is the time taken for its plasma concentration to be reduced by 50% of its original value.

A systematic review from Frontiers in Pharmacology mentioned that CBD’s half-life was between 1.4 and 10.9 hours following oromucosal spray use and two to five days after chronic oral administration(37).

Choosing the Best CBD Products for Rare Disorders

Various CBD companies are dedicated to offering the best CBD oil for rare disorders and other medical conditions. Therefore, it is difficult to determine the most trusted CBD brand in the market.

Below are a few pertinent factors that may help you sort through various brands’ offerings’ so you can find the best CBD product for you:

Hemp Extracts

Full-spectrum CBD oil contains all compounds of the cannabis plant, including CBD and other cannabinoids, essential oils, flavonoids, and terpenes.

Terpenes are the components of essential oils responsible for the aroma of cannabis(38).

Meanwhile, flavonoids are active compounds in cannabis that may have antioxidant, anti-inflammatory, and antiviral actions(39).

The full-spectrum CBD variant also has less than 0.3% THC.

Although broad-spectrum CBD oil also contains all of the natural compounds of cannabis, this CBD variant is THC-free, whereas CBD isolates are made from pure CBD.

Source of Hemp

It is necessary to identify the source of hemp to determine whether a CBD product is high-quality. Some CBD products may contain pesticides and harmful residues that may put your health at risk.

Therefore, choose a CBD brand that provides sufficient information on the hemp used. If possible, you should go for organic hemp or non-GMO CBD products manufactured by reputable companies.

Home-grown hemp is subject to the quality-control procedures of the USDA (United States Department of Agriculture).

Third-Party Lab Testing

The company must provide its clients with lab reports of all their products as tested by a third-party laboratory. These documents should be readily accessible on the brand’s official website.

Types of CBD Products

Most CBD-derived products available in the market are:

The type of cannabis products or CBD items you choose will depend on your lifestyle preferences, specific conditions, and expected effects.

Carrier Oils

Usually, coconut oil and hempseed oil are used as carrier oils. Hempseed oil does not have CBD. However, it is rich in nutrients and fatty acids(40).

Risks and Side Effects of Using CBD for Rare Disorders

The FDA warns the public about the use of CBD because cannabidiol may cause side effects(41).

Generally, the risks and side effects of using CBD include(42):

  • Changes in alertness
  • Changes in mood
  • Gastrointestinal distress

Legality of CBD

Rules and regulations regarding medical cannabis may be confusing. Hence, consumers should familiarize themselves with various state laws.

For instance, Colorado permits recreational and medical marijuana use. The local government decriminalized the use of marijuana by passing Amendment 64 to the Colorado Constitution in November 2012(43).

Meanwhile, in October 2021, California Governor Gavin Newsom signed Assembly Bill No. 45 into law(44).

The bill allowed the inclusion of hemp and cannabinoids, extracts, or hemp derivatives in dietary supplements, food and beverages, cosmetics, and processed pet food provided that they contain less than 0.3% THC(45).

The 2018 Farm Bill states that Cannabis sativa L. and other derivatives of cannabis with less than 0.3% THC content on a dry weight basis are deemed legal under federal law(46).


  1. How can CBD help with rare disorders? 

Studies regarding the effect of CBD on rare disorders are still in their infancy. However, CBD may be a novel therapeutic agent for autoimmune hepatitis(47).

Autoimmune hepatitis is a rare disorder in which the immune system attacks liver cells.

  1. What evidence or research exists to say that CBD helps with rare disorders?

Corticobasal syndrome is a rare disorder that shares some features with Parkinson’s disease(48).

Parkinson’s disease is a brain disorder that affects movement.

There is no available scientific data that says CBD can help with corticobasal syndrome.

However, CBD may be a potential therapeutic agent for Parkinson’s disease due to the identification of numerous pharmacological targets in the central nervous system(49).

  1. Is there any evidence that CBD can make rare disorders worse?

No evidence suggests that CBD can make rare disorders worse. However, Epidiolex, a drug that contains CBD, may cause adverse reactions, such as reduced appetite, fatigue, diarrhea, poor quality of sleep, and possible infections(50).

Epidiolex is an FDA-approved drug used to treat rare and severe forms of epilepsy and seizures associated with tuberous sclerosis complex(51).

  1. Will CBD interact with any current medication I may be taking for rare disorders?

CBD is not a biologically inert compound. Thus, it may trigger potential interactions with other medications(52).

  1. Are there other treatments I should consider alongside CBD to help with rare disorders?

Always seek medical advice, especially if you consider using CBD along with other treatments to help with rare disorders like autoimmune hepatitis.

Doctors often prescribe prednisone as an initial medication for autoimmune hepatitis(53). The substance is widely used as an immunosuppressive and anti-inflammatory drug.

CYP3A4 is the enzyme that metabolizes prednisone(54). It is worth noting that CBD is a potent inhibitor of CYP3A4(55). Hence, taking CBD alongside prednisone may alter the drug’s pharmacological effects.

  1. Can I fail a drug test if I use CBD for rare disorders?

CBD does not induce a euphoric high(56). Still, some CBD products have small amounts of THC that may build up in the body when taken regularly, in a span of four to six days(57).

An increased THC level in the bloodstream may result in a positive drug test.

  1. What is the CBD dosage for rare disorders?

There is no standard CBD dosage for rare disorders.

However, Korean researchers administered CBD orally to patients with rare and severe forms of epilepsy at a starting dose of 5mg daily. They maintained the dosage at 10mg per day(58).

The experts hypothesized that CBD may be safe to use for such medical conditions(59).

Still, the study only focused on specific types of epilepsy. Hence, you should consult a doctor versed in cannabis use to determine the accurate CBD dosage for a rare disorder.

  1. Is CBD legal?

The legality of CBD varies from state to state. Thus, you should be aware of your current state laws regarding the use of CBD to help with rare disorders.

  1. Does the FDA approve CBD?

Despite the 2018 Farm Bill passage, Epidiolex is the only CBD-derived product recognized by the FDA. Other CBD products marketed for medical use are still subject to FDA approval(60).

  1. What are the possible side effects of CBD for rare disorders?

Generally, commonly reported side effects of CBD include drowsiness, low blood pressure, and dry mouth(61).

  1. Rare Diseases at FDA
  2. Ibid.
  3. Ibid.
  4. Research Leads to Potential Treatment for Rare Disease
  5. Autoimmune Hepatitis
  6. Cannabidiol
  7. The Endocannabinoid System: Essential and Mysterious
  8. Cannabidiol
  9. Vanilloid TRPV1 Receptor Mediates the Antihyperalgesic Effect of the Nonpsychoactive Cannabinoid, Cannabidiol, in a Rat Model of Acute Inflammation
  10. CBD Effects on TRPV1 Signaling Pathways in Cultured DRG Neurons
  11. Research Leads to Potential Treatment for Rare Disease
  12. Vanilloid TRPV1 Receptor Mediates the Antihyperalgesic Effect of the Nonpsychoactive Cannabinoid, Cannabidiol, in a Rat Model of Acute Inflammation
  13. Ibid.
  14. Neuropathy (Peripheral Neuropathy)
  15. The Effectiveness of Topical Cannabidiol Oil in Symptomatic Relief of Peripheral Neuropathy of the Lower Extremities
  16. Cannabidiol for Treating Lennox-Gastaut Syndrome and Dravet Syndrome in Korea
  17. Ibid.
  18. What Is Dravet Syndrome?
  19. Lennox-Gastaut Syndrome
  20. Estimating Epidemiological Data of Multiple Sclerosis Using Hospitalized Data in Shandong Province, China
  21. Cannabidiol to Improve Mobility in People with Multiple Sclerosis
  22. Corticobasal Syndrome
  23. Ibid.
  24. Cannabidiol in Parkinson’s Disease
  25. Could an Anxiety Disorder Be a Sign of Parkinson’s Disease?
  26. Cannabidiol as a Potential Treatment for Anxiety Disorders
  27. Healing With CBD, page 207.
  28. Ibid.
  29. High-Fat Foods Can Increase Oral Cannabidiol Absorption Into the Body
  30. Healing With CBD, page 191.
  31. FDA Regulation of Cannabis and Cannabis-Derived Products, Including Cannabidiol (CBD)
  32. FDA Approves New Indication for Drug Containing an Active Ingredient Derived from Cannabis to Treat Seizures in Rare Genetic Disease
  33. Cannabidiol for Treating Lennox-Gastaut Syndrome and Dravet Syndrome in Korea
  34. Ibid.
  35. Healing With CBD, page 191.
  36. Healing With CBD, page 192.
  37. A Systematic Review on the Pharmacokinetics of Cannabidiol in Humans
  38. The Cannabis Terpenes
  39. Flavonoids As Antioxidants
  40. Hemp Seed Oil Properties
  41. What You Need to Know (And What We’re Working to Find Out) About Products Containing Cannabis or Cannabis-derived Compounds, Including CBD
  42. Ibid.
  43. Legal Marijuana Use in Colorado
  44. Assembly Bill No. 45
  45. Ibid.
  46. FDA Regulation of Cannabis and Cannabis-Derived Products, Including Cannabidiol (CBD)
  47. Research Leads to Potential Treatment for Rare Disease
  48. Corticobasal Syndrome
  49. Cannabidiol in Parkinson’s disease
  50. Epidiolex (Cannabidiol) Oral Solution
  51. FDA Approves First Drug Comprised of an Active Ingredient Derived from Marijuana to Treat Rare, Severe Forms of Epilepsy
  52. Potential Adverse Drug Events and Drug-Drug Interactions with Medical and Consumer Cannabidiol (CBD) Use
  53. Autoimmune Hepatitis
  54. The Effect of Cytochrome P450 Metabolism on Drug Response, Interactions, and Adverse Effects
  55. Potent Inhibition of Human Cytochrome P450 3A Isoforms by Cannabidiol: Role of Phenolic Hydroxyl Groups in the Resorcinol Moiety
  56. Cannabidiol (CBD)-What We Know and What We Don’t
  57. Can You Take CBD and Pass a Drug Test?
  58. Cannabidiol for Treating Lennox-Gastaut Syndrome and Dravet Syndrome in Korea
  59. Ibid.
  60. FDA Regulation of Cannabis and Cannabis-Derived Products, Including Cannabidiol (CBD)
  61. CBD: What You Need to Know Before You Try

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