As noted in this interesting article, I think the approach of supplying alternate forms of bioenergetic substrates to patients with Alzheimer disease is worth further exploration, and future studies must be designed and powered to test a differential APOE response, which we have observed in our own studies of insulin/energy-modulating agents. In the interest of full disclosure, as the article described, I received a small grant from Accera to conduct an acute dosing study of an MCT formulation in 2004; additionally, I also serve as a consultant for Accera, a fact that was not mentioned in the article.

View all comments by Suzanne Craft

The premise of this article is the notion that companies are using the medical food route as a “fallback” or backup strategy if their drug compound fails in the clinic. As I will discuss below, this premise is flawed. I wish to point out that this was never the intent for Axona (AC-1202). As I stated in the Tangled Neuron interview, Axona was originally intended to be a surrogate for testing our new therapeutic approach (ketone treatment for neuronal hypometabolism) in AD patients so the company could secure venture funding for its drug development platform. Based on our research, we found evidence that the dietary addition of ketones can delay and reduce the magnitude of cognitive dysfunction in patients with mild to moderate AD and can be an effective part of the dietary management of the disease. As a result, we concluded that the product could be appropriately marketed as a medical food. The company never filed an IND for Axona or intended to develop it as a drug. However, we did perform our clinical studies to pharmaceutical standards with industry and FDA-accepted...  Read more

The premise of this article is the notion that companies are using the medical food route as a “fallback” or backup strategy if their drug compound fails in the clinic. As I will discuss below, this premise is flawed. I wish to point out that this was never the intent for Axona (AC-1202). As I stated in the Tangled Neuron interview, Axona was originally intended to be a surrogate for testing our new therapeutic approach (ketone treatment for neuronal hypometabolism) in AD patients so the company could secure venture funding for its drug development platform. Based on our research, we found evidence that the dietary addition of ketones can delay and reduce the magnitude of cognitive dysfunction in patients with mild to moderate AD and can be an effective part of the dietary management of the disease. As a result, we concluded that the product could be appropriately marketed as a medical food. The company never filed an IND for Axona or intended to develop it as a drug. However, we did perform our clinical studies to pharmaceutical standards with industry and FDA-accepted outcomes for safety and efficacy. These data have been presented at major scientific conferences such as AAN and ICAD, and have been reviewed by internationally acclaimed experts on AD. The clinical data are also incorporated into our 10,000+ page product dossier that supports its positioning as a medical food. As a result of our laboratory and clinical studies with Axona during the past seven years we now have two preclinical New Chemical Entity compounds in development as drugs that work through the ketosis therapeutic mechanism. In terms of the article’s overall premise, there were some significant inaccuracies. First, NCEs and drug products cannot be marketed as medical food since medical food must meet the general food requirement of having ingredients that are either GRAS or approved food additives. Additionally, as noted in the Q&A with Sue Anderson from the FDA, medical food “is not a product that is merely provided to sick people … It is a formulated product that contains ingredients shown to be directly linked biochemically or metabolically to the disease or condition in humans.” I would also note that Vivimind (aka Alzhemed) is a dietary supplement, not a medical food. Supplements and medical food have distinct definitions and regulatory requirements. We are not aware of any examples of a product in the marketplace that was switched from drug development to medical food. While it is true that medical food is not required to report adverse events to FDA, this statement holds true for all food products. Nevertheless, Accera has in place a full medical information and safety system that compiles safety-related information and relays all serious adverse events via MedWatch to the FDA. We believe this is a responsible way to identify and address potential side effects and drug interactions in an elderly patient population that often has many comorbidities. Lastly, I wish to point out that the discussion of “what’s in a label” shows a fundamental misunderstanding of the key features that distinguish a medical food from a drug. As Ms. Anderson notes in the Q&A discussion, the product’s intended use and the provision of nutritional support distinguish medical food from a drug. In other words, to be considered a medical food, a product must be intended for the specific dietary management of a disease or condition and must be an integral part of the clinical treatment of patients. It may not, however, state or imply that the product treats, cures, or prevents a disease. Those are drug claims. To imply that the Axona label is insufficient or indicates that the product failed drug trials is simply inaccurate. The Axona label appropriately reflects the product’s intended use as a medical food.

View all comments by Steve Orndorff

I think it would have been a major advantage to get Ketasyn/AC1202 FDA-approved as a drug. Ketasyn/AC1202 could have then been used by health professionals and prescribed to the right persons. There is a strong rationale in using medium chain triglycerides (MTCs) as a source of ketone bodies to boost brain metabolism. It is likely that certain specific patients in “energy crisis”, such as very old persons for example, could benefit from MCTs. Unfortunately, the use of Ketasyn/AC1202 as a medical food will dilute its true therapeutic benefit.

In summary, I might be wrong but I think Ketasyn would have had more chance to achieve its full therapeutic potential as a drug than as a medical food.

View all comments by Frederic Calon