A groundbreaking clinical trial led by the Knight Family Dominantly Inherited Alzheimer Network-Trials Unit (DIAN-TU) has shown promising results in reducing the risk of early-onset Alzheimer’s dementia using an experimental anti-amyloid drug called gantenerumab. The study involved 73 participants with rare genetic mutations that virtually guarantee Alzheimer’s development in middle age. For a subgroup of 22 individuals with no initial cognitive problems who received the drug for an average of eight years, the treatment lowered the risk of developing symptoms from nearly 100% to about 50%.
These findings provide new evidence supporting the amyloid hypothesis, which suggests that removing amyloid plaques from the brain before symptoms arise can delay or potentially prevent the onset of Alzheimer’s disease. Although the trial was cut short when Roche/Genentech discontinued gantenerumab’s development, the researchers remain optimistic about the potential for preventing Alzheimer’s, with most participants now transitioning to another anti-amyloid drug called lecanemab. Overall, this study, which focused on early-onset Alzheimer’s, could have significant implications for prevention efforts across all forms of the disease, and researchers believe similar results might be achievable for the general population.