Drexel is among 65 sites testing AC-1204, a vanilla-flavored white powder that supplies a type of triglycerides.
The double-blind, placebo-controlled study is funded by the AC-1204 maker Accera Inc., of Broomfield, Colo. Abington Neurological Associates and Comprehensive Clinical Research in Berlin, N.J., also are participating. Results are expected in early 2016.
Accera hopes to recruit nearly 500 subjects for its NOURISH trial, the first test of AC-1204. Either the drug or a placebo will be mixed in participants' food or liquid for six months, after which they will have the option of getting AC-1204 for sure for another six months.
Lippa said she was attracted to two kinds of clinical trials. One uses the hardest of science to try to alter the course of the disease at a cellular level. The other tests more "natural" products that might help, while unlikely to do much harm. The AC-1204 trial is among the latter.
She found the product more appealing than many supplements. "This is different," she said, "because it's not a gobbledygook of antioxidants and vitamins."
AC-1204 is based on the idea that the body, when it has been fasting, produces an alternative energy source called ketone bodies. "The brain really seems to like these ketone bodies," she said. "It uses them really efficiently and kind of preferentially."
The powder, containing medium-chain triglycerides, is a way to get ketone bodies without the fasting. It's hard to get much of this form of triglycerides from foods, Lippa said.
The trial is evidence that Accera is hedging its bets.
The company markets an earlier iteration of the powder, Axona, as a medical food. The product - not covered by insurance - is available from pharmacies by prescription for $70 to $100 a month, said Sam Henderson, a molecular geneticist who is Accera's vice president for research and development.
The Food and Drug Administration in December sent Accera a warning letter after concluding that Axona was a drug, not a medical food. It called the product's labeling "false and misleading" and said "there are no distinctive nutritional requirements or unique nutrient needs for individuals with mild to moderate Alzheimer's disease."
Henderson said medical foods do not require FDA approval, while drugs do. Accera continues to assert that Axona is a medical food. Henderson refused to release the company's FDA response. The FDA declined comment.
The NOURISH trial positions AC-1204 as an investigational new drug with the goal of achieving FDA approval.
Henderson described AC-1204 as a "next-generation product" similar to Axona, but tweaked to reduce gastrointestinal side effects. He characterized it as a "semisynthetic" fatty acid derived from coconut oil. (You couldn't eat enough coconut oil to get the benefits, he said.)
Steven Arnold, director of the Penn Memory Center, the University of Pennsylvania's program for older people with memory problems, said he was skeptical of the "alternative energy" argument for using products such as Axona or coconut oil. "Why dying cells can use this energy source better than glucose has never been clear to me," he said in an e-mail.
While he has heard anecdotal reports that Axona has helped some patients, he has not seen any benefits in his own patients.
A 2009 double-blind, placebo-controlled trial found significant improvements in cognitive functioning among patients who took Axona. The results were best for those who were negative for the APOE4 mutation, which increases risk for developing Alzheimer's. On a 70-point cognitive test, patients without this mutation scored 4.77 to 6.26 points better than the placebo group at 45 days, and 3.36 to 5.33 points better at 90 days.
Henderson said a change of four points was considered "clinically meaningful," or large enough that family members might notice it.
Lippa said she did not expect a product like AC-1204 to cure or prevent Alzheimer's, but that it might help people use their remaining brain cells more effectively.
Loretta Tucker encouraged her 80-year-old mother to try the trial because the older woman had always been interested in alternative medicines. "This was just kind of right for her," she said.
Her mother, who lives in Burlington County, has been putting the powder - it could be drug or placebo - in Ovaltine each morning for six months.
Tucker, who shares her mother's interest in nutrition and supplements, thinks her mother's memory and mood have been more stable since she joined the trial.
Lippa said enrollment in the trial had been slow. She has only three patients so far. Henderson said that has been a problem across the board in Alzheimer's clinical trials, and is one reason that a new Alzheimer's drug has not been approved since 2003. He urged people with the disease to participate in clinical trials of all sorts, not just his.
"It's so important," he said. "I can't overstate."