Banner Alzheimer's Institute (BAI) has announced a major prevention trial to evaluate a treatment in cognitively healthy older adults at the highest known genetic risk for developing Alzheimer's disease at older ages. An NIH grant, expected to total $33.2 million, will support this research.
The study is part of the Alzheimer's Prevention Initiative (API), an international collaboration led by BAI to accelerate the evaluation of promising but unproven prevention therapies. It will test an anti-amyloid treatment in about 650 adults, ages 60 to 75, who have two copies of the apolipoprotein E (APOE4) gene, the major genetic risk factor for late-onset Alzheimer's. None of the participants will have impairments in memory or thinking at the time they enter the study.
The randomized, placebo-controlled trial, which will take place at BAI and other U.S. sites, will test the treatment's ability to stave off the memory and thinking declines associated with Alzheimer's. It will also assess the treatment's effects on different brain imaging and cerebrospinal fluid measurements of the disease. The specific compound to be evaluated has not been decided.
The trial will test what is often called the amyloid hypothesis, which suggests that accumulation of the protein amyloid in the brain plays a key role in the disease's progression. Major funding from philanthropy and industry will also support the trial, and its leaders expect to provide data and biological samples to the research community after the trial's conclusion to help in the scientific fight against Alzheimer's.
Individuals in the study will learn their APOE4 status. To help them prepare for this information, BAI has convened an expert committee to develop a comprehensive genetic testing and disclosure plan and to assess the impact of this disclosure during the trial.
"Under the National Plan to Address Alzheimer's Disease, our goal is to prevent and effectively treat the disorder by 2025," said Dr. Neil Buckholtz, of the National Institute on Aging, which leads Alzheimer's research at the NIH.
The research is intended to complement API's initial trial, which is primarily focused in Colombia and involves about 300 people from an extended family with a rare genetic mutation that typically triggers Alzheimer's symptoms around age 45. That work also is focusing on an anti-amyloid therapy and its potential in slowing or blocking the disease while preserving cognitive abilities. The investigation, including a smaller U.S. companion study, dovetails with prevention trials that have been planned or started by other research groups during the past 16 months.
"We are now looking at potential treatments to prevent both the early and late onset forms of the disease," said Dr. Pierre N. Tariot, BAI director.
The new trial will draw participants mainly from the Alzheimer's Prevention Registry, an online community of people who are committed to helping in the fight against Alzheimer's. The Registry provides regular updates on the latest scientific advances, as well as information on overall brain health. To overcome one of the biggest obstacles to clinical research, the Registry supports enrollment in a variety of Alzheimer's prevention studies within members' communities.
Alzheimer's is a debilitating and incurable disease that affects more than 5.2 million Americans, with a new diagnosis every 68 seconds. Without the discovery of successful prevention therapies, the number of U.S. cases is projected to nearly triple by 2050.