An experimental drug appears to reduce the risk of Alzheimer’s-related dementia in people destined to develop the disease in their 30s, 40s or 50s, according to the results of a study led by the Knight Family Dominantly Inherited Alzheimer Network-Trials Unit (DIAN-TU), based at Washington University School of Medicine in St. Louis.
Published in The Lancet Neurology, the findings suggest early treatment to remove amyloid plaques from the brain years before symptoms arise can delay the onset of Alzheimer’s dementia.
The international study involved 73 individuals with rare genetic mutations that cause amyloid overproduction.
For a subgroup of 22 participants who began the treatment while cognitively normal and continued for an average of eight years, the drug reduced the risk of developing dementia symptoms from nearly 100% to about 50%.
“This is the first evidence in a clinical trial that Alzheimer’s symptoms can be delayed by treating amyloid plaques early,” said senior author Dr. Randall J. Bateman.
The study supports the amyloid hypothesis, suggesting amyloid plaque buildup initiates the path to dementia. Participants originally joined the DIAN-TU-001 trial in 2012 and then an open-label extension. All participants had no or mild cognitive symptoms and were within 15 years before or 10 years after their family’s expected age of onset.
Initially, the anti-amyloid drug gantenerumab showed plaque reduction but no cognitive benefit. During the extension, all participants received gantenerumab. Results showed that early, long-term treatment was key: the subgroup treated for about eight years showed significantly delayed symptom onset.
Due to Roche/Genentech’s discontinuation of gantenerumab development in 2022, the extension ended early. Participants had an average treatment duration of 2.6 years. Longer treatment was needed for cognitive benefit; shorter durations showed no observable impact yet.
Gantenerumab was associated with ARIA (amyloid-related imaging abnormalities), mostly asymptomatic but sometimes severe. Two participants were withdrawn due to serious ARIA but recovered. No life-threatening events or deaths occurred.
Following gantenerumab’s discontinuation, participants began treatment with lecanemab, another FDA-approved anti-amyloid drug. Data on this phase are pending. A new NIH-funded trial is proposed to continue the research.
The results are promising for prevention in both early- and late-onset Alzheimer’s. The Knight Family DIAN-TU has launched a new Primary Prevention Trial with Eli Lilly’s remternetug, enrolling genetically at-risk individuals as young as 18, up to 25 years before expected symptoms.
“This could be the beginning of Alzheimer’s prevention for millions,” Bateman said.
Maria C. Carrillo, Chief Science Officer of the Alzheimer’s Association, emphasized the importance of continuing and expanding dementia research in light of these groundbreaking findings.
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