Individuals with Down syndrome have a higher risk of developing Alzheimer’s disease at an earlier age. Autopsy findings reveal that by the time they reach 40, their brains commonly contain amyloid plaques. However, those with Down syndrome have been either excluded from or not adequately represented in clinical trials evaluating new treatments for Alzheimer’s disease. Recently, lecanemab, a drug that targets and eliminates beta-amyloid plaques, received approval from the U.S. Food and Drug Administration for early intervention in Alzheimer’s. A study conducted by researchers from Brigham and Women’s Hospital and the University of California, Irvine, examined lecanemab’s effectiveness in binding to amyloid plaques in brain tissue from Down syndrome patients. The results demonstrated that the drug successfully targeted amyloid in all 15 tissue samples. Nonetheless, it was also found to bind to blood vessels in the brain, which raises some safety issues. These findings are detailed in JAMA Neurology.
“This study is very important for clinical practice as it evaluates a newly approved treatment for Alzheimer’s, lecanemab, specifically for those with Down syndrome,” explained co-corresponding author Lei Liu, MD, PhD, from the Department of Neurology at Brigham and Women’s Hospital.
“Our results highlight the potential of anti-amyloid medications for benefiting individuals with Down syndrome, while also stressing the importance of addressing safety concerns, particularly the risk of bleeding complications,” said co-corresponding author Elizabeth Head, PhD, of the Department of Pathology and Laboratory Medicine at the University of California, Irvine.
The research team analyzed brain tissue samples from 15 individuals with Down syndrome, aged between 43 and 68 years. The study’s limited sample size and age range prompted plans to broaden future research by including samples from younger donors to assess the impact of age on drug binding to blood vessels. Additionally, they aim to investigate the drug’s binding characteristics in patients with late-onset Alzheimer’s to see if similar trends are present. The researchers expressed their appreciation to the individuals with Down syndrome who contributed their brain tissue for study.