People with Down syndrome are likely to develop Alzheimer's disease at a young age, with autopsy studies showing that by age 40 years, the brains of individuals with Down syndrome have amyloid plaques. Yet people with Down syndrome have been excluded from or underrepresented in clinical trials of new therapies for treating AD. Lecanemab, which has been shown to target and remove beta-amyloid plaques, has been approved by the U.
S. Food and Drug Administration to treat AD early in the disease's progression. A new study led by investigators at Brigham and Women's Hospital (a founding member of the Mass General Brigham healthcare system) and University of California, Irvine tested lecanemab to see if it could bind to amyloid plaques in tissue samples from people with Down syndrome, finding that it effectively targeted amyloid in all 15 samples.
However, the drug also bound to brain blood vessels, which raises safety concerns. Results are published in JAMA Neurology . Our study is highly clinically relevant, as we focus on the usage of a recently approved disease modifying therapy for Alzheimer's disease, lecanemab, in people with Down syndrome.
" Lei Liu, MD, PhD, co-corresponding author of the Department of Neurology, Brigham and Women's Hospital "Our findings underscore the exciting promise of anti-amyloid drugs for helping people with Down syndrome, but also the need for careful consideration of safety, especially the risk of hemorrhagic complications," said co-corresponding au.