Idebenone is a synthetic drug with a very similar structure to the body antioxidant coenzyme Q10 that was initially developed to treat brain conditions. 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, its potential benefits, and side effects.
Disclaimer: 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 clinical and 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 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 [1, 2].
Idebenone is claimed to 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 [1, 2].
Since the core structure of idebenone is the same as ubiquinone, they may 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 [1, 3].
Mechanism of Action
Idebenone neutralizes free radicals called reactive oxidative species (ROS) in the mitochondria, helping prevent oxidative damage in cells. Additionally, by moving electrons down the electron transport chain, idebenone can restore energy (ATP) production in damaged cells [1, 2].
Although glutamate is an important neurotransmitter, excess levels can damage brain cells due to their overexcitation (excitotoxicity). Idebenone can prevent brain cell damage caused by glutamate. It also increases the levels of nerve growth factor (NGF), a neurotransmitter responsible for the growth, development, and survival of brain cells. Increased NGF levels can improve overall cognitive function [1, 4].
Snapshot
Proponents
- May improve cognitive decline in people with Alzheimer’s and other types of dementia
- May help with some rare hereditary disorders such as Duchenne muscular dystrophy and Leber’s disease
Skeptics
- Insufficient evidence for some purported benefits
- Not approved
- Unknown safety profile
Health Benefits
The effects of idebenone are due to its ability to preserve or restore mitochondrial function and reduce the levels of ROS [2].
Possibly Effective
1) Alzheimer’s Disease and Dementia
In a clinical trial on 102 elderly people with Alzheimer’s disease, idebenone (45 mg, 2x/day). Improved memory, attention, and behavior after 4 months [5].
Four further clinical trials on over 1,000 people with this condition also reported improvements in attention, memory, and behavior, and slow progression of the disease with idebenone (240-360 mg/day) [6, 7, 8, 9].
However, there have been some mixed results. In a clinical trial on 500 Alzheimer’s patients, idebenone (120-360 mg, 3x/day) failed to slow down the progression of the disease [10].
Dementia is the loss of cognitive function occurring with age, most commonly due to the damage of brain blood vessels. In 3 clinical trials from the 90s on almost 300 elderly people with vascular dementia, idebenone slightly improved cognitive function, as measured with several tests [11, 12, 13].
All in all, the evidence suggests that idebenone may help with cognitive decline caused by Alzheimer’s disease and other types of dementia. However, other drugs approved by the FDA and possibly safer and more effective are available. Discuss the most suitable treatment with your doctor.
2) Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMM) is a genetic disease (X-linked disorder) that affects 1 out of 5,000 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 [14, 15].
In 3 clinical trials on 151 people with DMM, idebenone (450-900 mg/day) improved respiratory function and reduced the incidence of lung complications. The results of one of the studies were maintained during a 1-year follow-up period [16, 17, 18, 19].
Although limited, the evidence suggests that idebenone may help with Duchenne muscular dystrophy. However, other medications (such as eteplirsen, corticosteroids, and ACE inhibitors), exercise, assistive devices, and surgery may be safer and more effective measures to improve the symptoms.
3) 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 [20].
Idebenone restores ATP production in cells through antioxidation (as an electron carrier). This may prevent vision damage from worsening and promote recovery by restoring eye cell function [20].
Idebenone (300 mg, 3x/day for 6 months) slightly improved vision in a clinical trial on 85 people with Leber’s disease, especially in those whose eyes had different visual acuity. A 30-month follow-up of 60 patients found that the benefits continued despite discontinuing the treatment [21, 22].
In another trial on 39 people with this condition, idebenone (300 mg, 3x/day for 24 weeks) protected from color vision loss [23].
In a clinical trial on 28 people with Leber’s, those treated with idebenone (180 mg/day), vitamin B2, and vitamin C for a year showed faster vision recovery. However, 2 people treated with this combination but a higher dose of idebenone (270 mg/day) showed no improvement [24, 25].
Because Leber’s disease is a very rare genetic condition, the evidence is based on only a few clinical trials. It suggests that idebenone may help with this condition, but further research on its effectiveness and safety is needed.
Insufficient Evidence
1) Friedreich’s Ataxia
Friedreich’s ataxia is a genetically inherited disease that causes progressive damage to the nervous system. Most people with this condition are deficient in a mitochondrial protein (frataxin), resulting in an impaired electron transport chain. This reduces energy production and increases the number of ROS, causing cell damage [26, 1].
In a 6-month clinical trial on 48 children and adolescents with Friedreich’s ataxia, idebenone (5-45 mg/kg) dose-dependently improved brain function and daily living activities. A 12-month trial on 68 children had similar results [27, 28].
However, a 24-week trial on 70 children and adolescents with this condition found that idebenone (450-2250 mg/day) only caused a slight, non-significant improvement in cognitive function [29].
In a long-term (5 years) follow-up study on 104 children, the cognitive status worsened over time regardless of idebenone treatment [30].
Similarly, idebenone didn’t improve exercise capacity in another trial on 48 children with Friedreich’s ataxia [31].
In 3 trials on 75 people with this condition, idebenone (5 mg/kg per day) protected the heart muscle from thickening (cardiomyopathy). However, it failed to do so in another trial on 70 people [32, 33, 34, 35].
In 2 small studies on 19 people with Friedreich’s ataxia, idebenone (5-20 mg/kg per day) slowed the progression of the disease [36, 37].
Triple therapy with idebenone, riboflavin, and the chelator deferiprone or the synthetic EPO analog darbepoetin alpha caused a slight improvement in brain and heart function in 2 small trials on 18 people with Friedreich’s disease [38, 39].
Due to the small size of many studies and the mixed results, it’s difficult to draw conclusions on the potential effects of idebenone on Friedreich’s ataxia. Larger, more robust clinical trials are needed to shed some light on this potential use.
2) Skin Health
A cream containing 0.5%-1.0% applied 2x/day for 6 weeks improved skin roughness, hydration, and wrinkles in a clinical trial on 41 women with moderately sun-damaged skin [40].
In a small trial on 5 people, topical idebenone protected the skin from UV damage. Idebenone was similarly effective in cultured skin cells [41].
However, idebenone was ineffective in pigs exposed to UV radiation [42].
Two small clinical trials and a study in pigs (with negative results) are clearly insufficient to support the use of idebenone in skin care. More clinical trials on larger populations are needed to evaluate its effectiveness.
Anecdotal Evidence (Case Studies)
Mitochondrial Disease
In a rare 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. This causes symptoms like dementia, stroke-like episodes, epilepsy, headaches, hearing impairment, and diabetes [43, 1].
A 36-year-old man with MELAS given high dosages (90-270 mg/day) of idebenone for 5 months had healthier brain cells, possibly due to the antioxidant effects of idebenone [44].
In a 16-year-old boy with MELAS, a combined therapy of ubiquinone and idebenone for 6 months stopped symptom progression [45].
Idebenone (90-180 mg/day) and ubiquinone (210 mg/day) therapy produced similar results after 11 months in two additional cases [46].
Leigh Syndrome
Leigh syndrome (also known as Leigh’s disease) is a genetic condition that damages certain parts of the brain, resulting in loss of mental, motor, and lung function. Usually, symptoms appear during early childhood and result in death from breathing issues a few years later. Occasionally, symptoms develop in adulthood or very slowly [47, 48].
A patient with Leigh syndrome treated with idebenone (90 mg/day for 6 months) improved lung function [49].
Animal and Cell Research
No clinical evidence supports the use of idebenone for any of the conditions listed in this section. Below is a summary of the existing animal and cell-based research, which should guide further investigational efforts. However, the studies should not be interpreted as supportive of any health benefit.
Liver Disease
In rats with liver damage, idebenone protected liver cells from oxidative damage. A follow-up experiment confirmed the initial results [50].
Possibly Ineffective
Huntington’s Disease
In a 1-year clinical trial on 91 people with Huntington’s disease, idebenone wasn’t better than the placebo at improving the symptoms [51].
Side Effects
Keep in mind that the safety profile of idebenone is relatively unknown, given the lack of well-designed clinical studies. The list of side effects below is not a definite one and you should consult your doctor about other potential side effects based on your health condition and possible drug or supplement interactions.
Most of the clinical trials using idebenone for different health conditions reported no side effects.
However, in one study on 83 Alzheimer’s patients, some of them experienced [6]:
The most common adverse effects observed in 2 studies on healthy people given idebenone (up to 2,250 mg/day in single doses of up to 1,050 mg) were [52, 53]:
- Loose stools
- Fatigue
- Headaches
- Attention disturbances
- High blood pressure
- Upset digestion
- Discoloration of the urine
Forms
Capsules
Idebenone is primarily available in the form of capsules, most commonly 150-mg capsules. It is not found in pharmacies but can be ordered online
Cream
Idebenone is available as an anti-wrinkle cream, usually containing 0.5% or 1.0% idebenone.
Remember that idebenone is not approved for any conditions and its safety profile is insufficiently investigated.
Dosage
There is no safe dosage for idebenone, since it is an unapproved new drug that poses a significant safety risk.
Below is a summary of the dosages used in scientific research, outlined for informational purposes. We highly advise against the use of idebenone.
One study showed that idebenone is well-tolerated in single oral doses of 1,050 mg and daily dosages up to 2,250 mg [53].
Websites selling idebenone recommend using dosages of 90-120 mg/day. The doses used in the studies discussed in this article were usually 150 mg or higher [53].
Limitations and Caveats
Studies on idebenone are generally limited since this drug is not approved by the FDA. Most clinical trials testing the efficacy of idebenone had small sample sizes with small dosages, while other research was done in rats or cells. There is not enough research to confirm idebenone as a treatment for any of the above conditions [1].
Reviews
The opinions expressed in this section are solely those of idebenone users, who may or may not have medical or scientific training. Their reviews do not represent the opinions of SelfHacked. SelfHacked does not endorse any specific product, service, or treatment.
Do not consider user experiences as medical advice. Never delay or disregard seeking professional medical advice from your doctor or other qualified healthcare providers because of something you have read on SelfHacked. We understand that reading individual, real-life experiences can be a helpful resource, but it is never a substitute for professional medical advice, diagnosis, or treatment from a qualified healthcare provider.
One user was disappointed with the strength of the product and therefore, didn’t think it was worth the price.
Another user reported that 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 found the cream too heavy and didn’t notice any improvement in her wrinkles.
