Idebenone
Encyclopedia
Idebenone is an experimental drug, initially developed by Takeda Pharmaceutical Company
for the treatment of Alzheimer's disease
and other cognitive defects. This has been met with limited success. The Swiss company Santhera Pharmaceuticals has started to investigate it for the treatment of neuromuscular diseases. , early clinical trial
s for the treatment of Friedreich's ataxia
and Duchenne muscular dystrophy
have been completed. Chemically, idebenone is an organic compound
of the quinone
family. It is also promoted commercially as a synthetic analog of coenzyme Q10
(CoQ10).
s like electroretinography
, auditory evoked potentials and visual analogue scale
s also suggested positive nootropic
effects, but larger studies with hard endpoints are missing.
Research on idebenone as a potential therapy of Alzheimer's disease
have been inconsistent, but there may be a trend for a slight benefit. In May 1998, the approval for this indication was cancelled in Japan due to the lack of proven effects. In some European countries, the drug is available for the treatment of individual patients in special cases.
.
(mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes) and primary progressive multiple sclerosis are recruiting patients , a study investigating Leber's hereditary optic neuropathy
is under way.
properties, and has therefore been used in anti-aging on the basis of free-radical theory
. Clinical evidence for this use is missing. It has been used in topical
applications to treat wrinkle
s.
issued a Notice of Compliance under the Notice of Compliance with Conditions (NOC/c) Policy to Santhera Pharmaceuticals (Switzerland) for the drug product Catena. The product was authorized under the NOC/c Policy on the basis of the promising nature of the clinical evidence, and the need for a confirmatory study to verify its clinical benefit. Patients should be advised of the fact that the market authorization was issued with conditions.
Takeda Pharmaceutical Company
is the largest pharmaceutical company in Japan and Asia and a top 15 pharmaceutical company. The company has over 19,000 employees worldwide and achieved $15.7 billion USD in revenue during the 2008 fiscal year...
for the treatment of Alzheimer's disease
Alzheimer's disease
Alzheimer's disease also known in medical literature as Alzheimer disease is the most common form of dementia. There is no cure for the disease, which worsens as it progresses, and eventually leads to death...
and other cognitive defects. This has been met with limited success. The Swiss company Santhera Pharmaceuticals has started to investigate it for the treatment of neuromuscular diseases. , early clinical trial
Clinical trial
Clinical trials are a set of procedures in medical research and drug development that are conducted to allow safety and efficacy data to be collected for health interventions...
s for the treatment of Friedreich's ataxia
Friedreich's ataxia
Friedreich's ataxia is an inherited disease that causes progressive damage to the nervous system, resulting in symptoms ranging from gait disturbance to speech problems; it can also lead to heart disease and diabetes....
and Duchenne muscular dystrophy
Duchenne muscular dystrophy
Duchenne muscular dystrophy is a recessive X-linked form of muscular dystrophy, which results in muscle degeneration, difficulty walking, breathing, and death. The incidence is 1 in 3,000 boys. Females and males are affected, though females are rarely affected and are more often carriers...
have been completed. Chemically, idebenone is an organic compound
Organic compound
An organic compound is any member of a large class of gaseous, liquid, or solid chemical compounds whose molecules contain carbon. For historical reasons discussed below, a few types of carbon-containing compounds such as carbides, carbonates, simple oxides of carbon, and cyanides, as well as the...
of the quinone
Quinone
A quinone is a class of organic compounds that are formally "derived from aromatic compounds [such as benzene or naphthalene] by conversion of an even number of –CH= groups into –C– groups with any necessary rearrangement of double bonds," resulting in "a fully conjugated cyclic dione structure."...
family. It is also promoted commercially as a synthetic analog of coenzyme Q10
Coenzyme Q
Coenzyme Q10, also known as ubiquinone, ubidecarenone, coenzyme Q, and abbreviated at times to CoQ10 , CoQ, Q10, or Q, is a 1,4-benzoquinone, where Q refers to the quinone chemical group, and 10 refers to the number of isoprenyl chemical subunits in its tail.This oil-soluble, vitamin-like substance...
(CoQ10).
Nootropic effects and Alzheimer's disease
Idebenone improved learning and memory in experiments with mice. In humans, evaluation of Surrogate endpointSurrogate endpoint
In clinical trials, a surrogate endpoint is a measure of effect of a certain treatment that may correlate with a real clinical endpoint but doesn't necessarily have a guaranteed relationship...
s like electroretinography
Electroretinography
Electroretinography measures the electrical responses of various cell types in the retina, including the photoreceptors , inner retinal cells , and the ganglion cells. Electrodes are usually placed on the cornea and the skin near the eye, although it is possible to record the ERG from skin electrodes...
, auditory evoked potentials and visual analogue scale
Visual Analogue Scale
A visual analogue scale is a psychometric response scale which can be used in questionnaires. It is a measurement instrument for subjective characteristics or attitudes that cannot be directly measured. When responding to a VAS item, respondents specify their level of agreement to a statement by...
s also suggested positive nootropic
Nootropic
Nootropics , also referred to as smart drugs, brain steroids, memory enhancers, cognitive enhancers, and intelligence enhancers, are drugs, supplements, nutraceuticals, and functional foods that improve mental functions such as cognition, memory, intelligence, motivation, attention, and concentration...
effects, but larger studies with hard endpoints are missing.
Research on idebenone as a potential therapy of Alzheimer's disease
Alzheimer's disease
Alzheimer's disease also known in medical literature as Alzheimer disease is the most common form of dementia. There is no cure for the disease, which worsens as it progresses, and eventually leads to death...
have been inconsistent, but there may be a trend for a slight benefit. In May 1998, the approval for this indication was cancelled in Japan due to the lack of proven effects. In some European countries, the drug is available for the treatment of individual patients in special cases.
Friedreich's ataxia
Preliminary testing has been done in humans and found idebenone to be a safe treatment for Friedreich's ataxia, exhibiting a positive effect on cardiac hypertrophy and neurological function. The latter was only significantly improved in young patients. In a different experiment, a one-year test on eight patients, idebenone reduced the rate of deterioration of cardiac function, but without halting the progression of ataxiaAtaxia
Ataxia is a neurological sign and symptom that consists of gross lack of coordination of muscle movements. Ataxia is a non-specific clinical manifestation implying dysfunction of the parts of the nervous system that coordinate movement, such as the cerebellum...
.
Duchenne muscular dystrophy
After experiments in mice and preliminary studies in humans, idebenone has entered Phase II clinical trials in 2005 and Phase III trials in 2009.Other neuromuscular diseases
Phase I and II clinical trials for the treatment of MELASMELAS
Mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes – abbreviated to MELAS – is one of the family of mitochondrial cytopathies, which also include MERRF, and Leber's hereditary optic neuropathy. It was first characterized under this name in 1984...
(mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes) and primary progressive multiple sclerosis are recruiting patients , a study investigating Leber's hereditary optic neuropathy
Leber's hereditary optic neuropathy
Leber’s hereditary optic neuropathy or Leber optic atrophy is a mitochondrially inherited degeneration of retinal ganglion cells and their axons that leads to an acute or subacute loss of central vision; this affects predominantly young adult males...
is under way.
Life style
Idebenone is claimed to have properties similar to CoQ10 in its antioxidantAntioxidant
An antioxidant is a molecule capable of inhibiting the oxidation of other molecules. Oxidation is a chemical reaction that transfers electrons or hydrogen from a substance to an oxidizing agent. Oxidation reactions can produce free radicals. In turn, these radicals can start chain reactions. When...
properties, and has therefore been used in anti-aging on the basis of free-radical theory
Free-radical theory
The free-radical theory of aging states that organisms age because cells accumulate free radical damage over time. A free radical is any atom or molecule that has a single unpaired electron in an outer shell. While a few free radicals such as melanin are not chemically reactive, most...
. Clinical evidence for this use is missing. It has been used in topical
Topical
In medicine, a topical medication is applied to body surfaces such as the skin or mucous membranes such as the vagina, anus, throat, eyes and ears.Many topical medications are epicutaneous, meaning that they are applied directly to the skin...
applications to treat wrinkle
Wrinkle
A wrinkle is a fold, ridge or crease in the skin. Skin wrinkles typically appear as a result of aging processes such as glycation or, temporarily, as the result of prolonged immersion in water. Wrinkling in the skin is caused by habitual facial expressions, aging, sun damage, smoking, poor...
s.
History
On July 23, 2008, Health CanadaHealth Canada
Health Canada is the department of the government of Canada with responsibility for national public health.The current Minister of Health is Leona Aglukkaq, a Conservative Member of Parliament appointed to the position by Prime Minister Stephen Harper.-Branches, regions and agencies:Health Canada...
issued a Notice of Compliance under the Notice of Compliance with Conditions (NOC/c) Policy to Santhera Pharmaceuticals (Switzerland) for the drug product Catena. The product was authorized under the NOC/c Policy on the basis of the promising nature of the clinical evidence, and the need for a confirmatory study to verify its clinical benefit. Patients should be advised of the fact that the market authorization was issued with conditions.