New FDA approved medical food product to combat Alzheimer's Disease

Photo from Axona.com

When I first began reading about this new approach to fighting Alzheimer’s Disease, my first thought was that this was probably a vitamin supplement product, like the thousands of supplemental products that may or may not do what they promise. I started doing some research and discovered that there is actually an FDA category for medical food, which is what this new product is, named Axona. Axona is a product of Accera, Inc.

NOTE - (This is not an endorsement of a product, but a presentation of a new therapy in the fight against Alzheimer's Disease)

The first interview was with P. Murali Doraiswamy MD is professor of psychiatry at Duke University and co-author of The Alzheimer’s Action Plan.

Q1.) Can you tell me a little about Axona, what you’re role is in this discovery and what you’re general thoughts are about Alzheimer’s research going on right now?

Dr. Doraiswamy:) I had no role in it’s discovery and was not involved in any of it’s testing or studies. It was discovered by Dr Sam Henderson, a genetics researcher whose mother had Alzheimer’s. This is a great example of a discovery coming from a combination of personal motivation and scientific ingenuity. Axona’s main ingredient is a special mixture of medium chain triglycerides but it also contains whey protein, milk protein, palm oil, sunflower oil, soy lecithin, vitamins, minerals, and vanilla extract.

I first came across this product when the company presented their study results to me to get my feedback. Because of the huge unmet needs in this field and urgency for new therapies, we really need to welcome new ideas but remain objective. And people are more receptive to the idea of a food versus a drug.

In general, research in the Alzheimer field is at a very exciting stage now. There are several new diagnostic tests and drugs in the pipeline that promise to fundamentally change the way we may diagnose and manage this condition in the future. We are very close to being able to develop cocktails of therapies that can not only improve symptoms but also target the pathology.

Q2.) Can you speak to any further information about this new FDA medical food category and how Axona must deal with the possibility of being associated with supplemental products?

Dr. Doraiswamy:) Most doctors and health professionals have not heard of a medical food. I myself had not heard of it till a year ago. Everyone mixes it up with a supplement. Adding to the confusion is a new category called “functional foods” such as probiotics. So lot’s of educational efforts are needed to educate both professionals and the public. There are over a dozen medical foods already on the market for everything from memory to bowel problems. Again, it’s best you get the details of the FDA process and standards required for approval/making claims from the company.
Make sure you read the FDA link that provides important information.

Q3.) Do you know of any plans to bring this product into nursing homes and if not, would that be a possibility?

Dr. Doraiswamy:) The company would need to answer that. They would need to conduct a study in severe dementia patients before they brought it into the nursing home.

Q4.) In the PR Newswire video (http://www.prnewswire.com/broadcast/36441/press.shtml), speaking about Axona, it mentions how this therapy increases metabolism. Is that within the brain only or metabolism of the entire body? Would this have any other good or bad side effects, such as weight loss?

Dr. Doraiswamy:) I was not involved with that video and the company will need to answer this question.

But, in general, it is well established that there is a metabolic and energy deficit in Alzheimer nerve cells which occurs quite early and is quite well correlated with memory decline. For example, PET scans show that the Alzheimer brain takes up less glucose than normal brains, and even people at risk for Alzheimer’s show these changes. It is also well established that ketone bodies (a type of natural chemical produced by the body under conditions such as starvation or fasting) can serve as an alternate source of fuel. Diets rich in ketones have proved useful to treat other neurologic conditions such as refractory epilepsy in humans. Studies in mice and dogs have also shown benefits. The company has conducted a clinical trial in Alzheimer patients to test it’s effects on memory and cognition and those data can be found on their web site. I have not seen any study that shows that Axona increases metabolism in the human brain – I am hoping to conduct a study to test this.

Q5.) My mother has had AD for about 2 years now (showing symptoms and diagnosed by her family doctor) and is in a public nursing home now. She is showing signs of increased confusions and forgetfulness. Is cell and or associated memory damage already done completely irreversible? In other words, is it possible that a new drug approved now would be of help to someone who is already in mid to later stages of AD?

Dr. Doraiswamy:) Yes. Even moderate stage patients can demonstrate substantial improvements. However, there is no therapy being tested for late-stage patients currently. We don’t have a way to grow new brain cells to replace lost ones yet. There is a gene-therapy trial underway of nerve growth factor but it’s still in early stages.

Q6.) Have you heard of the anti-body clinical trial mentioned in the HBO documentary The Alzheimer’s Project, mentioned in the part titled, Momentum in Science, and if so, do you have any news or feedback about this trial?

Dr. Doraiswamy:) Yes we are one of the sites at Duke. The expectations are lower than they were a few years back. There are also other strategies, besides antibodies, that are targeting the plaques and tangles. The availability of newer brain scans will improve our ability to target the right patient.

Q7.) Is Axona, the only new approved drug for Alzheimer’s and is there any other new treatment available or soon to be available in the rest of 2009?

Dr. Doraiswamy:) Axona is NOT a drug and it would wrong to refer to it as such. It is a “medical food” which has different standards from a prescription drug (see the FDA link).

There are 4 prescription drugs for Alzheimer’s on the market today. Dimebon is a new drug product that is in late stage testing but will not be on the market in 2009.

The second interview is with Steve Orndorff, Ph.D., founder and CEO, Accera, Inc.

Q1.) Can you speak to any further information about this new FDA medical food category and how Axona must deal with the possibility of being associated with supplemental products?

Steve:) A medical food is an FDA-regulated product, in a relatively new category of medical protocols defined by Congress as part of the Orphan Drug Act. A medical food is formulated to be consumed or administered orally and is intended for the dietary management of a specific disease or condition for which distinctive nutritional requirements, based on recognized scientific principles, are established by medical evaluation.

Medical Foods are often prescription products, but are different than drugs or dietary supplements (also called nutraceuticals) in several aspects, such as their claims. Claims for both Medical Foods and drugs must be supported by solid laboratory and clinical data. Medical Food ingredients have GRAS designation, the highest FDA standard of safety given to foods. Medical Foods, sometimes prescribed in addition to pharmaceuticals, nonetheless represent an entirely different scientific and medical approach to managing diseases.

This is a very exciting and promising approach to Alzheimer's disease (AD). In randomized controlled clinical trials, Axona was demonstrated to be safe, effective and generally well-tolerated. It is available by prescription and is complementary to current AD therapies.

Q2) In the PR Newswire video, speaking about Axona, it mentions how this therapy increases metabolism. Is that within the brain only or metabolism of the entire body? Would this have any other good or bad side effects, such as weight loss?

Steve:) Axona targets the metabolic deficiencies and imbalances in the brain associated with AD by providing an alternative energy source for brain cells.

Alzheimer’s disease is characterized by a substantial decrease in the brain’s ability to metabolize glucose, the brain's primary source of energy. Axona is digested and metabolized by the liver to produce ketone bodies, naturally occurring compounds that the brain can use as an alternative energy source. Therefore, Axona does not increase metabolism. Instead, it provides an alternative energy source for the brain for patients whose brain's cannot metabolize glucose effectively.

Q3.) Is Axona, the only new approved drug for Alzheimer's and is there any other new treatment available or soon to be available in the rest of 2009?

Steve:) Just to clarify, Axona is not a drug, it is an FDA-regulated prescription Medical Food product. Axona offers a new approach to managing AD for patients in need of effective, well-tolerated therapies.