Axona was previously marketed as a

medical food Medical foods are foods that are specially formulated and intended for the dietary management of a disease that has distinctive nutritional needs that cannot be met by normal diet alone. In the United States they were defined in the Food and Drug A ...

for the clinical dietary management of the impairment of metabolic processes associated with mild to moderate

Alzheimer's disease Alzheimer's disease (AD) is a neurodegeneration, neurodegenerative disease that usually starts slowly and progressively worsens. It is the cause of 60–70% of cases of dementia. The most common early symptom is difficulty in short-term me ...

. It is a proprietary formulation of fractionated palm kernel oil ( caprylic triglyceride), a medium-chain triglyceride. Cericin, the company that makes Axona, states that during digestion, caprylic triglyceride is broken down into

ketones In organic chemistry, a ketone is a functional group with the structure R–C(=O)–R', where R and R' can be a variety of carbon-containing substituents. Ketones contain a carbonyl group –C(=O)– (which contains a carbon-oxygen double bon ...

, which provide an alternative energy source for the brain. Its use is based on the idea that the brain's ability to use its normal energy source,

glucose Glucose is a simple sugar with the molecular formula . Glucose is overall the most abundant monosaccharide, a subcategory of carbohydrates. Glucose is mainly made by plants and most algae during photosynthesis from water and carbon dioxide, using ...

, is impaired in Alzheimer's disease. Axona was first sold in March 2009. In 2013, FDA determined that Axona is misbranded under section 403(a)(1) of the Federal Food, Drug, and Cosmetic Act (the Act) 1 U.S.C. § 343(a)(1)because the labeling is false and misleading in that the product is labeled and marketed as a medical food but does not meet the statutory definition of a medical food. Axona has not been approved by the US Food and Drug Administration (FDA) as a drug to treat Alzheimer's and the efficacy of managing the health of Alzheimer's patients by use of this medical food has been questioned by experts in the field, including the Alzheimer's Association.

Description

Axona is a

marketed to assist with dietary management of mild to moderate Alzheimer's disease. Axona is formulated for oral administration and is sold by prescription. The largest ingredient in Axona is caprylic triglyceride, also known as fractionated coconut oil, a medium-chain triglyceride. caprylic triglyceride is Generally Recognized As Safe (GRAS) by the FDA. A

in the US is an official FDA product classification, and was originally defined by Congress as part of the Orphan Drug Amendments of 1988 as "a food which is formulated to be consumed or administered through a feeding tube under the supervision of a physician and which is intended for the specific dietary management of a disease or condition for which distinctive nutritional requirements, based on recognized scientific principles, are established by medical evaluation."Staff, FDA. May 1997; Revised May 200
Guidance for Industry: Frequently Asked Questions About Medical Foods
/ref> Medical foods are not drugs and cannot be marketed as treating or preventing a disease or condition; the FDA does not evaluate the efficacy and safety of medical foods.

Proposed mechanism of action of Axona

Alzheimer's disease is clinically characterized by a progressive decline in memory and language, and pathologically by accumulation of

senile plaque Amyloid plaques (also known as neuritic plaques, amyloid beta plaques or senile plaques) are extracellular deposits of the amyloid beta (Aβ) protein mainly in the grey matter of the brain. Degenerative neuronal elements and an abundance of mi ...

s and

neurofibrillary tangle Neurofibrillary tangles (NFTs) are aggregates of hyperphosphorylated tau protein that are most commonly known as a primary biomarker of Alzheimer's disease. Their presence is also found in numerous other diseases known as tauopathies. Little is kn ...

s. Because Alzheimer's disease is also characterized by a reduced ability of some areas of the brain to use glucose, some scientists have proposed that treatments targeting metabolic deficits in the brain of Alzheimer's patients may have efficacy. The makers of Axona claim that after oral administration of Axona, the caprylic triglyceride in Axona are processed by

enzyme Enzymes () are proteins that act as biological catalysts by accelerating chemical reactions. The molecules upon which enzymes may act are called substrates, and the enzyme converts the substrates into different molecules known as products. A ...

s in the gut, and the resulting medium-chain fatty acids (MCFAs) are absorbed into the blood supply leading to the liver. The MCFAs rapidly pass directly to the

liver The liver is a major Organ (anatomy), organ only found in vertebrates which performs many essential biological functions such as detoxification of the organism, and the Protein biosynthesis, synthesis of proteins and biochemicals necessary for ...

, where they undergo oxidation to form

ketone In organic chemistry, a ketone is a functional group with the structure R–C(=O)–R', where R and R' can be a variety of carbon-containing substituents. Ketones contain a carbonyl group –C(=O)– (which contains a carbon-oxygen double bo ...

s. Since the liver does not use ketones, they are released into the circulation to be used by nonliver tissues. These ketones can cross the blood-brain barrier and are then taken up by brain cells. While glucose is the brain's chief energy source, ketones normally serve as the "backup" energy source. Ketones act as an alternative energy source for brain cells that have an impaired ability to use

(sugar) as a result of Alzheimer's disease, and the makers of Axona claim that this may have medical benefits.

Clinical trials

Axona has been evaluated in Phase II clinical trials, paid for and conducted by Cerecin, only one of which was published in an open-access journal in 2009.John Reville for Wall Street Journal. July 19, 2012
Nestlé Buys U.S. Maker of 'Brain Health' Shake
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Sales and marketing

The product launched in 2009. By 2012 it was being administered to about 30,000 patients in the US.

Criticism

A 2011 story by ABC News noted widespread concern about Axona in the medical community, with some calling it "

snake oil Snake oil is a term used to describe deceptive marketing, health care fraud, or a scam. Similarly, "snake oil salesman" is a common expression used to describe someone who sells, promotes, or is a general proponent of some valueless or fraudu ...

." The theory that the brain in Alzheimer's disease patients is better able to use ketones than glucose is not widely accepted among AD clinicians and researchers. The Alzheimer's Association has classified Axona an "alternative treatment", has "expressed concern that there is not enough evidence to assess the potential benefit of medical foods for Alzheimer’s disease", and notes that the safety of Axona is not regulated in the way that drugs are. Glenn Smith, Ph.D, a clinical neuropsychiatrist at the Mayo Clinic, also noted that Axona's safety and efficacy are not known, and noted that "the Alzheimer's Association doesn't recommend the use of medical foods, including Axona, for the treatment of Alzheimer's disease."Glenn Smith for the Mayo Clinic. Aug. 4, 201
Alzheimer's disease, Expert Answers - Axona: Medical food to treat Alzheimer's
Accessed May 10, 2012 In 2013, FDA determined that Axona is misbranded under section 403(a)(1) of the Federal Food, Drug, and Cosmetic Act (the Act) 1 U.S.C. § 343(a)(1)because the labeling is false and misleading in that the product is labeled and marketed as a medical food but does not meet the statutory definition of a medical food.FDA Warning Letter
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References