School of Medicine

Investigational drugs didn’t slow memory loss, cognitive decline in rare, inherited Alzheimer’s, initial analysis indicates

Randall J. Bateman, MD, of Washington University School of Medicine in St. Louis, led an international trial evaluating whether investigational drugs could slow memory loss and cognitive decline in a rare, inherited form of Alzheimer's disease. The trial was conducted at 24 sites in Australia, Canada, France, Spain, the United Kingdom and the United States. (Photo: Huy Mach)

An international clinical trial evaluating whether two investigational drugs can slow memory loss and cognitive decline in people in the early stages of a rare, inherited form of Alzheimer’s disease has yielded disappointing results, an initial analysis of the data has shown. However, the researchers continue to explore data from the trial’s cognitive and clinical outcomes, and await analyses of biomarkers and other information so they can further understand the study’s results.

The study ( Identifier: NCT01760005) is a phase 2/3 trial led by Washington University School of Medicine in St. Louis through its Dominantly Inherited Alzheimer Network-Trials Unit (DIAN-TU). The trial separately evaluated the effects of two drugs – solanezumab, made by Eli Lilly and Co., and gantenerumab, made by Roche and its U.S. affiliate, Genentech – in people with a rare, inherited, early-onset form of Alzheimer’s called dominantly inherited Alzheimer’s disease or autosomal dominant Alzheimer’s disease. Such people experience declines in memory and thinking skills starting in their 50s, 40s or even 30s.

The initial analysis indicated that neither drug met the primary outcome of the study, which was a slowing of cognitive decline as measured by multiple tests of thinking and memory.

“Although the drugs we evaluated were not successful, the trial will move us forward in understanding Alzheimer’s,” said principal investigator Randall J. Bateman, MD, director of DIAN-TU and the Charles F. and Joanne Knight Distinguished Professor of Neurology at Washington University. “The trial’s innovative design – developed in collaboration with a consortium of pharmaceutical companies, the National Institutes of Health (NIH), regulatory agencies and academic leaders – will make advances for future Alzheimer’s trials. Ongoing and continued research and trials will bring us closer to our goal to stop Alzheimer’s. We will continue until we are successful.”

Despite the trial’s results, the study yielded new insight into the development and progression of Alzheimer’s, which can inform future research into the disease, including the more common form that typically strikes after age 65, Bateman said. The brain changes that occur as Alzheimer’s progresses are much the same in people with the inherited, early-onset and the late-onset forms of the disease.

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