Innovative Alzheimer’s Trials Focus on Prevention

By Susan McClure

Jonathan Bailey, NHGRI

The second issue of Genome is getting ready to go to press, and this time our editorial “deep dive” will explore how personalized medicine is being used to find innovative treatment options for the millions of Americans suffering from Alzheimer’s disease.

Those who have been touched by Alzheimer’s know that this is one of the most devastating illnesses we face as a society. The slow and painful process of watching a loved one slip away is heart-wrenching, and the behavioral changes associated with the illness can cause tremendous stress and fatigue for caregivers. There are only a few treatment options available, and while they may help slow the disease’s progression, there is no known cure. Given that more than 5.2 million people are living with the disease and that the number is expected to triple by 2050, it’s imperative that a cure or at least an effective treatment is found soon. Some forward-thinking researchers have decided that the best way to beat this disease is to prevent it from ever taking hold in the first place.

This week, researchers from the Banner Alzheimer’s Institute (BAI) announced a partnership with Novartis to determine whether two investigative drugs can prevent or delay Alzheimer’s in healthy, high-risk individuals. The APOE4 trial will kick off in 2015 and will study 1,300 cognitively healthy people between the ages of 60 and 75 who are at high risk of developing Alzheimer’s because they inherited two copies of the APOE4 gene — one from each parent. Only 2 percent of the world’s population carries two copies of this gene. The drugs used in this study are designed to either attack or prevent the production of amyloid protein, which many researchers believe plays a starring role in the development of Alzheimer’s.

Another project, the A4 Study, is funded by the National Institute on Aging, Eli Lilly and Company, and several philanthropic organizations. Like the APOE4 study, this trial also targets amyloid protein. Investigators are seeking 1,000 healthy individuals between the ages of 65 and 80 who have normal brain function but may be at risk of developing Alzheimer’s. Researchers will use an imaging test called a PET scan to determine whether potential participants have evidence of amyloid plaque buildup in their brain. The investigational treatment in this case is an anti-amyloid antibody intended to decrease amyloid buildup.

What excites me about both of these trials is that they are working to prevent disease rather than reverse it. That’s a novel concept and one that I hope we see more of across a variety of diseases.