Idebenone is a synthetic drug that was initially developed to treat brain conditions. It has a very similar structure to an important antioxidant in the body, ubiquinone (coenzyme Q10). Idebenone may also be beneficial for genetic disorders with impaired mitochondria function (such as Friedreich’s ataxia and Leber’s disease). Read on to learn more about idebenone, what it does, its function, and its side effects.
Note: By writing this post, we are not recommending this drug. Some of our readers who were already taking the drug requested that we commission a post on it, and we are simply providing information that is available in the scientific literature. Please discuss your medications with your doctor.
What is Idebenone?
Idebenone is a synthetic drug developed in Japan in the 1980s to treat conditions involving brain degradation (neurodegenerative conditions). It has a similar structure to ubiquinone (coenzyme Q10), which is an antioxidant that also has an essential role in mitochondrial energy production [R, R].
Idebenone can be helpful in a number of diseases stemming from mitochondrial dysfunction. Because of its antioxidant ability, it is thought to boost skin health and play a beneficial role in the health of brain cells [R, R].
Since the core structure of idebenone is the same as ubiquinone, they function in a similar manner. They both carry electrons through the electron transport chain, the energy (ATP) producing process in the mitochondria. Idebenone mixes more easily in the blood than ubiquinone, and therefore, crosses the blood-brain barrier (BBB) more efficiently [R, R].
Mechanism of Action
Idebenone neutralizes free radicals called reactive oxidative species (ROS) in the mitochondria, preventing oxidative damage in cells. This improves the survival and recovery of cells. Additionally, by moving electrons down the electron transport chain, idebenone can restore energy (ATP) production in damaged cells [R, R].
Although glutamate is an important neurotransmitter, excess levels can cause issues in the brain (excitotoxicity). Idebenone can prevent brain cell death (neuronal death) due to oxidative stress that is sometimes caused by glutamate. Idebenone also increases nerve growth factor (NGF) levels, a neurotransmitter responsible for the growth, development, and survival of neurons. Increased NGF levels can improve overall cognitive function [R, R].
Uses of Idebenone
1) May Help Friedreich’s Ataxia
Friedreich’s ataxia is a genetically inherited disease that causes progressive damage to the nervous system. Most individuals with this condition are deficient in a mitochondrial protein (frataxin), resulting in an impaired electron transport chain. This impaired function reduces energy production and increases the number of ROS, causing cell damage [R, R].
Friedreich’s ataxia patients treated with idebenone in a 6-month study (double-blind randomized controlled trial) had improved brain function and daily living activities. The higher the dose, the more positive of an effect (dosages: 5 mg/kg, 15 mg/kg, and 45 mg/kg). This was further confirmed by a 12-month follow-up study (double-blind randomized controlled trial) [R, R, R].
38 more patients with Friedreich’s ataxia who received idebenone treatment (5 mg/kg a day) also experienced an improved condition (prospective trial) [R].
Two additional studies (open-label studies with 9 and 10 patients) gave idebenone treatment (5-20 mg/kg a day) to patients with Friedreich’s ataxia. In both studies, idebenone slowed progression of the disease [R, R].
2) May Help Alzheimer’s Disease
In a study (double-blind randomized controlled trial), 102 elderly patients with Alzheimer’s Disease were treated with idebenone (45 mg/2x a day). After 4 months, memory, attention, and behavior improved in the patients [R, R].
Multiple additional studies (3 double-blind randomized controlled trials with 83, 203, and 450 Alzheimer’s patients) also reported improvements in attention, memory, and behavior, with idebenone treatment (360 mg/day, 270 mg/day, respectively). Idebenone also slowed the progression of Alzheimer’s [R, R, R, R].
However, there have been some mixed results with idebenone. In a study (double-blind randomized controlled trial) with 500 Alzheimer’s patients, idebenone (120, 240, or 360 mg/3x a day) did not slow down the progression of the disease [R].
3) May Help Mitochondrial Disease
The antioxidative and cell health improvement effects of idebenone make it a strong candidate to treat various diseases caused by mitochondrial dysfunction. In a condition called MELAS syndrome (characterized by a specific set of symptoms: mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes), energy production in the mitochondria is impaired [R, R].
MELAS causes symptoms like dementia, stroke-like episodes, epilepsy, headaches, hearing impairment, and diabetes. A 36-year-old man with MELAS given high dosages (90-270 mg/day) of idebenone for 5 months had healthier brain cells (via the antioxidative function of idebenone) [R, R].
A 16-year-old boy with mitochondrial encephalopathy (MELAS) had seizures, muscle weakness, hearing loss, mental retardation, and muscle spasms (myoclonus). After treating him with combined therapy (120 mg/day or 4 mg/kg/day for 16 months) of ubiquinone and idebenone, symptoms did not progress. Idebenone (90-180 mg/day) and ubiquinone (210 mg/day) therapy for 11 months produced similar positive results in two additional cases of MELAS [R, R].
4) May Help Leber’s disease
Leber’s disease (Leber’s hereditary optic neuropathy) is another rare, genetically inherited condition, where dysfunctional mitochondria cause rapid vision loss. It is the most common mitochondrial disorder [R].
Idebenone treats Leber’s by restoring ATP production in cells through antioxidation (as an electron carrier). This prevents more vision damage and promotes recovery by restoring eye cell function [R].
In a retrospective study with 28 patients with Leber’s, half were treated with idebenone (180 mg/day), vitamin B2, and vitamin C for a year. Patients who received this treatment had faster vision recovery [R].
However, two Leber’s patients treated with idebenone (270 mg/day), vitamin C, and vitamin B2 for one year, and had no improvements in their vision [R].
5) May Help Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy is a genetic disease (X-linked disorder) that affects 1 out of 5000 boys. It is one of the most severe muscular disorders to affect children and if left untreated, usually results in death by 20 years of age [R, R].
In two studies (randomized controlled trials), patients with Duchenne muscular dystrophy (64 and 66 patients) were treated with idebenone (900 mg/day for both studies). Individuals treated with idebenone had improved lung function with fewer complications. Idebenone may be used to improve lung function in such cases [R, R].
6) May Help Symptoms of Leigh Syndrome
Leigh syndrome (also known as Leigh’s disease) is a devastating genetic condition that causes damage to certain parts of the brain, resulting in loss of mental and motor function. Lung failure is a common outcome of Leigh syndrome. Usually, symptoms appear early on, in infants and children, and results in death from breathing issues a few years later. Occasionally, symptoms develop in adulthood or very slowly [R, R].
One of Leigh’s patient treated with idebenone (90 mg/day for 6 months) had improved lung function [R].
7) Can Be Used as an Anti-Aging Product
Because idebenone is an antioxidant, it could prove useful as an anti-aging product. A cream containing either 0.5% and 1.0% idebenone was developed. Women with moderately sun damaged skin used the cream 2x/day for 6 weeks. Idebenone cream improved roughness, hydration, and wrinkles of the skin (DB-RCT with 41 female subjects) [R].
8) May Help Sun Burns
A study treated sunburnt skin cells with idebenone. Idebenone significantly prevented further oxidative damage in these cells and improved their overall health [R].
9) May Help Symptoms of Liver Disease
An experiment damaged rat liver cells to mimic liver disease. When treated with idebenone, the health of the cells significantly improved due to protection from oxidative damage. A follow-up experiment confirmed the same results. Idebenone may be applied in the future to treat liver disease [R].
10) Multiple Sclerosis (MS)
Most of the experiments reported no side effects in their patients using idebenone. However, in one study (on Alzheimer’s patients), subjects experienced some side effects. In several studies, idebenone was given to healthy male subjects to test its safety and how it is metabolized in the body. Idebenone was given at various dosages (total daily dosage of 2,250 mg and a large, single dose of 1050 mg). The side effects reported by these studies are listed below [R, R, R]:
Idebenone is primarily available in the form of capsules, most commonly in 150 mg capsules. It is not found in pharmacies but can be ordered online.
Idebenone is available as an anti-wrinkle cream, usually containing 0.5% and 1.0% idebenone.
Since this drug is yet to be approved by the FDA, no dosage guidelines have been provided. One study showed that idebenone is well-tolerated in single oral doses of 1050 mg and daily dosages up to 2250 mg [R].
Websites selling idebenone recommend using dosages of 90-120 mg/day. Doses in the studies mentioned in this article were usually 150 mg or higher [R].
Limitations and Caveats
Studies on idebenone are generally limited, and this drug is not approved by the FDA. Most of the clinical trials testing the efficacy of idebenone have small sample sizes with small dosages, while other research was done on rats or cells. There is not enough research to confirm idebenone as a treatment for any of the above conditions [R].
One user was disappointed in the strength of the product and therefore, didn’t think it was worth the price.
Another user said the idebenone face cream produced noticeable results (fuller cheeks and a youthful appearance) within 6-8 weeks and even as quickly as 2 days.
However, another user said the cream is too heavy and didn’t noticeably improve wrinkles.