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BACKGROUNDER: NIH funds clinical trials testing new approaches for Alzheimer’s

May 15, 2012

Kathleen Sebelius, secretary of the U.S. Department of Health and Human Services, today announced funding of two major clinical trials to test potential therapies for Alzheimer’s disease. The awards were announced at the HHS- and NIH-sponsored Research Summit on Alzheimer’s Disease 2012: Path to Treatment and Prevention. (View press release)

A nasal spray for Alzheimer’s?

Growing evidence points to insulin playing a vital role in brain function, with decreased levels possibly contributing to memory problems, atrophy and other Alzheimer’s-related brain changes. Recently reported findings suggest that normalizing brain insulin levels might be beneficial in treating the disease. Researchers led by Suzanne Craft, Ph.D., of the University of Washington and Veterans Affairs Puget Sound, reported from a four-month pilot trial of insulin nasal spray therapy that memory, cognition and daily functioning improved among 104 participants with mild cognitive impairment (MCI) and mild to moderate Alzheimer’s. Treatments were administered through an innovative device that quickly delivered the insulin to the brain, and no side effects were reported.

The new NIH grant will allow Craft and her co-investigators to conduct a larger trial. The new study, expected to total $7.9 million over five years, will recruit 240 volunteers for a year-long treatment trial at multiple sites across the U.S. People with MCI or mild Alzheimer’s dementia will receive insulin treatment or a placebo, and at the end of the year, researchers will compare cognition, memory and functional performance between the two groups. Additionally, biomarkers associated with Alzheimer’s disease (levels of tau and amyloid proteins) will be measured in cerebrospinal fluid. Alzheimer’s-associated brain atrophy will be measured through brain imaging. At the end of the year, all participants will receive the insulin spray for six months.

The trial will be conducted in collaboration with the Alzheimer’s Disease Cooperative Study Consortium, an NIA-supported network of academic medical centers and clinics experienced in designing and conducting Alzheimer’s clinical trials.

Innovative international prevention trial

The second study to receive funding is a five-year prevention trial, the first to focus on people who are cognitively normal but at extremely high risk of developing Alzheimer’s disease. The effort, toward which NIH expects to contribute $16 million, is an international collaboration led by grantees at the Banner Alzheimer’s Institute in Phoenix, working with a unique and large family population in Colombia sharing a genetic mutation known to cause observable signs of Alzheimer’s disease at around age 45.

The study is generating excitement because its approach reflects new thinking—that testing therapies before signs of memory loss appear might be most effective in fighting Alzheimer’s disease. In this study, Drs. Eric Reiman and Pierre Tariot of Banner and colleagues will examine whether up to five years of an antibody treatment, crenezumab, designed to bind to and possibly clear away abnormal amounts of amyloid protein in the brains of people with Alzheimer’s, can prevent decline in cognitive function.

The study participants—some 300 adult members of the Colombian clan with a family history of early-onset Alzheimer’s, as well as a smaller number of U.S. participants age 30 and older—will include both carriers and non-carriers of the gene. The Banner researchers will work in collaboration with a Colombian team led by Dr. Francisco Lopera of the University of Antioquia. The Banner team will also collaborate with researchers from the NIA-sponsored Dominantly Inherited Alzheimer’s Network to identify and recruit the U.S. participants. The study will use brain scans, fluid biomarkers and cognitive testing to track amyloid levels, changes in brain structure and function and cognitive performance.

Banner is contributing $15 million to the collaboration and Genentech, a Roche biotech company based in San Francisco, is providing the study drug, clinical support and a large share of funding for the trial.

The study aims to do more than look at the potential effectiveness of one therapy. It will be a test of how and to what extent biomarkers may predict disease progress or treatment success and will contribute vitally important information and samples to the study of Alzheimer’s disease. This study will demonstrate how international collaboration in key areas of research can make a major contribution to the understanding and testing therapies for Alzheimer’s disease.

For more information, please contact the NIA Office of Communications at 301-496-1752; nianews3@mail.nih.gov.