Alzheimer’s Study in People at Risk, a Response to FDA Call for Early Research, Now Recruiting

The U.S. Food and Drug Administration (FDA) issued a draft guidance in 2013 to encourage research  in people at the earliest stages of Alzheimer’s disease, with no symptoms or no obvious symptoms. The update has had effect, with new clinical trials looking at Alzheimer’s patients without noticeable dementia, when they might possibly benefit most from treatment, the agency reported in a news release.

Previous studies have shown that there is a delay of several years between the first changes in a patient’s brain and the onset of Alzheimer’s symptoms, and some scientists suggest that the greatest benefits of treatment are most likely only very early in the disease course.

The FDA intended that the draft prod research into early stage Alzheimer’s therapies, with the document serving as a platform for continued discussions among the agency, industry sponsors of drug therapies, the scientific community and the general public.

Among the clinical trials underway in response — and now recruiting participants — is the ‘A4 Study’ (Anti-Amyloid Treatment in Asymptomatic Alzheimer’s), a landmark public-private effort funded by the National Institute on Aging, Eli Lilly and Company, and several philanthropic organizations.

People being recruited are ages 65 to 85 with unimpaired thinking and memory function, but thought to be at risk for developing Alzheimer’s as determined by an advanced brain scan.

The three-year study, taking place in the U.S., Canada and Australia, is testing whether a new investigational treatment, known as an anti-amyloid antibody, can slow memory loss caused by Alzheimer’s.

More information on the trial and its locations is available through this link. Researchers hope to enroll 1,000 people with “elevated” levels of amyloid plaque in the brain as determined through PET scans. An online “prescreening” test is available to all interested in taking part, but not sure if they might quality.

“Earlier and more precise identification of patients with early changes in the brain who will go on to develop Alzheimer’s is important for the success of these clinical studies,” Billy Dunn, MD, a neurologist and the director of FDA’s Division of Neurology Products, said in the release. “We hope that earlier interventions, before further extensive damage to the brain sets in, will be more successful. We’re very excited about the potential for this research to result in safe and effective treatments for early Alzheimer’s disease.”

One of the biggest challenges in Alzheimer’s research is to accurately spotting people at risk for developing the disease, even though researchers have recently identified biomarkers that may indicate a higher risk.

“There may be a window of opportunity to affect the disease before people experience symptoms,” said Eric Bastings, MD, a neurologist and the FDA division’s deputy director. “If we can use biomarkers to better choose who should be in which clinical trials, these biomarkers hopefully will help increase the likelihood that we’ll be able to show the drug effect in clinical trials.”

Some of these biomarkers are gene mutations.“In a small proportion of patients, having someone in the family with Alzheimer’s dramatically raises the risk that they, too, will have the disease,” Dunn said.

The A4 trial is being coordinated by the University of Southern California’s Alzheimer’s Therapeutic Research Institute.

Over 5 million Americans live with Alzheimer’s disease, making it the most common cause of dementia among people age 60 or older. Over the last two decades, only five treatments have been approved by the FDA, with the most recent one coming 10 years before the agency issued the draft guidance.