Doctors Debate Effectiveness of Alzheimer's Milkshake

VIDEO: Dr. Paul Aisen and Dr. Paul Schulz discuss Axona for treating
WATCH Does Alzheimer's Milkshake Work?

Some call it a novel approach to treating Alzheimer's disease. Others call it "snake oil."

Either way, Axona is different from other treatments. It's a milkshake -- not a drug, but a "medical food," and it's a controversial alternative for Alzheimer's patients and their caregivers, who face a frustrating lack of options when it comes to treating the disease successfully. Like drugs, "medical foods" are regulated by the U.S. Food and Drug Administration (FDA), but the agency's approval standards for this category are far less rigorous than for drugs.

According to the Alzheimer's Association, there are just five FDA-approved drugs to treat the symptoms of Alzheimer's, compared with the dozens available for conditions like cancer or heart disease. And scientists say none of the Alzheimer's drugs provide patients any lasting protection from the gradual advance of the disease's symptoms.

"Current treatments are modestly useful, but certainly inadequate. There is an enormous need for more effective therapy for Alzheimer's disease," said Dr. Paul Aisen, a neuroscience professor at the University of California, San Diego. "Patients and families want to find anything that may help."

Without any "magic bullet" cures, alternative treatments like Axona can offer a glimmer of hope to patients who have tried everything without success. But doctors are divided over whether these treatments are worth exploring or if they merely give false hope to patients with few options.

According to Accera, the milkshake's manufacturer, Axona was created as a solution to the problem of brain cells in Alzheimer's patients not being able to use the fuel they need to function: glucose. Instead, Axona provides the brain with an alternative fuel source: fats called ketones. On its website, Accera says that one Axona milkshake each day will "improve cognitive function in some AD patients."

When Catherine Yanda heard about Axona, she was ready to try anything. She has cared for her 87-year-old mother, Mary, for nine years, and watched her become steadily less alert, conversant and active. Her mother has tried three of the FDA-approved Alzheimer's drugs -- Namenda, Reminyl, and Aricept -- without much success.

"We were definitely looking for other avenues," Yanda, who is an active member of the online caregiver, said. She paid for her mother's daily Axona milkshakes for six months until she couldn't afford them anymore. The milkshakes costs $70 to $90 for a 30-day supply, and aren't covered by most insurance plans

"I did see what I thought was some benefit," Yanda said. "But the doctors weren't sure if it was the Axona or not."

"It's fairly outrageous that little can be done to impede the exploitative marketing of unproven and unlikely substances to vulnerable Alzheimer's patients and their desperate caregivers," said Dr. Sam Gandy, chair of Alzheimer's disease research at Mount Sinai School of Medicine.

Gandy is one of a group of doctors who study and treat Alzheimer's patients who say Axona is worthless in treating the disease. These scientists point to a lack of data showing that the treatment has any real effect on the disease.

In 2009, Accera studied a small number of patients who drank one Axona milkshake per day, and found that they improved 1.9 points on a 70-point cognitive testing scale than patients who drank a placebo milkshake. However, the benefits didn't last as long as the company hoped -- only 45 days as opposed to the 90-day goal.

That short fall is enough to persuade scientists like Dr. Rudolph Tanzi, a neurology professor at Harvard Medical School, that the treatment is worthless.

"Getting extra fuel to the brain would provide an energy boost that could potentially lead to modest improvements," he said. "But it is very short-lived and does nothing to stop or treat the disease. I would think a low-fat frozen yogurt would achieve the same thing."

"This is just expensive coconut oil," said Dr. Roger Brumback, professor of neurology at the Creighton University School of Medicine. "It's another example of false hopes and an entrepreneur's financial gain in a disease that is clearly devastating to patients and families."

But other physicians aren't so quick to dismiss Axona. Steven Ferris, director of the Aging and Dementia Research Center at New York University, serves on the scientific advisory board at Accera, Axona's manufacturer. He said Axona's method of using different fuel to power Alzheimer's brain cells is a legitimate idea that scientists have explored since the 1980s. Although Axona's benefits are not proven, he said he doesn't believe the idea behind it is entirely worthless.

"I wouldn't characterize it as snake oil, simply because it does have a scientific basis and there is some data that suggests a potential benefit," he said. "If this were out there available in the supermarkets, I'd really be concerned. But we do have gatekeepers, the physicians. It really comes down to individual judgments by prescribers as to whether this is appropriate for their patients or not."

Richard Isaacson, a neurologist at the University of Miami School of Medicine who is also a paid consultant for Accera, said he likes having new options to present to patients who have so few.

"I don't want to give patients false hope, but I want to give them options," Isaacson said. "I want to do anything and everything I can for them. As long as it's safe, I'm still going to try it."