Axona was previously marketed as a

medical food Medical food is food that is 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, it was defined in the Food and Drug Admin ...

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 neurodegenerative disease and the cause of 60–70% of cases of dementia. The most common early symptom is difficulty in remembering recent events. As the disease advances, symptoms can include problems wit ...

. It is a proprietary formulation of

fractionated Fractionation is a separation process in which a certain quantity of a mixture (of gasses, solids, liquids, enzymes, or isotopes, or a suspension) is divided during a phase transition, into a number of smaller quantities (fractions) in which t ...

palm kernel oil Palm kernel oil is an edible plant oil derived from the kernel of the oil palm tree ''Elaeis guineensis''. It is related to two other edible oils: ''palm oil'', extracted from the fruit pulp of the oil palm, and ''coconut oil'', extracted from ...

( caprylic triglyceride), a

medium-chain triglyceride A medium-chain triglyceride (MCT) is a triglyceride with two or three fatty acids having an aliphatic tail of 6–12 carbon atoms, i.e. a medium-chain fatty acid (MCFA). Rich food sources for commercial extraction of MCTs include palm kernel oil ...

. Cericin, the company that makes Axona, states that during digestion, caprylic triglyceride is broken down into

ketones In organic chemistry, a ketone is an organic compound with the structure , where R and R' can be a variety of carbon-containing substituents. Ketones contain a carbonyl group (a carbon-oxygen double bond C=O). The simplest ketone is acetone ( ...

, 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 sugar with the Chemical formula#Molecular formula, molecular formula , which is often abbreviated as Glc. It is overall the most abundant monosaccharide, a subcategory of carbohydrates. It is mainly made by plants and most algae d ...

, is impaired in Alzheimer's disease. Axona was first sold in March 2009. In 2013, US

Food and Drug Administration The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is respo ...

(FDA) determined Axona was misbranded because the product was 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 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 The Alzheimer's Association is a nonprofit voluntary health organization that focuses on Alzheimer's disease care, support and research. History Jerome H. Stone founded the Alzheimer's Association with the help of several family support groups ...

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

. caprylic triglyceride is

generally recognized as safe Generally recognized as safe (GRAS) is a United States Food and Drug Administration (FDA) designation that a chemical or substance added to food is considered safe by experts under the conditions of its intended use. An ingredient with a GRAS d ...

(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 An orphan drug is a medication, pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce without government assistance, due to the small population of patients affected by th ...

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 Dementia is a syndrome associated with many neurodegenerative diseases, characterized by a general decline in cognitive abilities that affects a person's ability to perform everyday activities. This typically involves problems with memory, ...

s and

neurofibrillary tangle Neurofibrillary tangles (NFTs) are intracellular aggregates of hyperphosphorylated tau protein that are most commonly known as a primary Biomarker (medicine), biomarker of Alzheimer's disease. Their presence is also found in numerous other disea ...

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 An enzyme () is a protein that acts as a biological catalyst by accelerating chemical reactions. The molecules upon which enzymes may act are called substrate (chemistry), substrates, and the enzyme converts the substrates into different mol ...

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 metabolic organ (anatomy), organ exclusively found in vertebrates, which performs many essential biological Function (biology), functions such as detoxification of the organism, and the Protein biosynthesis, synthesis of var ...

, where they undergo oxidation to form

ketone In organic chemistry, a ketone is an organic compound with the structure , where R and R' can be a variety of carbon-containing substituents. Ketones contain a carbonyl group (a carbon-oxygen double bond C=O). The simplest ketone is acetone ( ...

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 trial The phases of clinical research are the stages in which scientists conduct experiments with a health intervention to obtain sufficient evidence for a process considered effective as a medical treatment. For drug development, the clinical phases ...

s, paid for and conducted by Cerecin, only one of which was published in an

open-access journal Open access (OA) is a set of principles and a range of practices through which nominally copyrightable publications are delivered to readers free of access charges or other barriers. With open access strictly defined (according to the 2001 de ...

in 2009.John Reville for Wall Street Journal. July 19, 2012
Nestlé Buys U.S. Maker of 'Brain Health' Shake
/ref>

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 False advertising, deceptive marketing, health care fraud, or a scam. Similarly, snake oil salesman is a common label used to describe someone who sells, promotes, or is a general proponent of some valueless ...

." 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

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 Mayo Clinic () is a Nonprofit organization, private American Academic health science centre, academic Medical centers in the United States, medical center focused on integrated health care, healthcare, Mayo Clinic College of Medicine and Science ...

, 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 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
/ref>

References