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Alzheimer’s disease remains one of the most challenging neurodegenerative disorders, affecting millions worldwide. However, a groundbreaking study led by the Knight Family Dominantly Inherited Alzheimer Network-Trials Unit (DIAN-TU) at Washington University School of Medicine in St. Louis has provided new hope. An experimental drug has shown significant promise in reducing the risk of Alzheimer’s-related dementia, particularly in individuals genetically predisposed to develop the disease in their 30s, 40s, or 50s.
Published on March 19 in The Lancet Neurology, the study involved 73 individuals carrying rare inherited genetic mutations that result in the overproduction of amyloid plaques, an established hallmark of Alzheimer’s disease. Without intervention, these individuals were almost certain to develop Alzheimer’s in middle age. However, the trial’s results suggest that early treatment aimed at removing amyloid plaques from the brain may delay the onset of dementia symptoms.
Among the participants, a crucial subgroup of 22 individuals who had no cognitive impairments at the start and received the drug for an average of eight years experienced a dramatic decrease in their risk of developing symptoms—from nearly 100% to about 50%. These findings underscore the potential of early intervention in altering the course of the disease.
The study provides further validation for the amyloid hypothesis of Alzheimer’s disease, which suggests that the accumulation of amyloid plaques is the initial step leading to dementia. The experimental drug was administered to determine whether it could prevent the onset of Alzheimer’s symptoms.
Originally part of the DIAN-TU-001 trial—the world’s first Alzheimer’s prevention trial—the participants continued into an extension study receiving anti-amyloid therapy. The trial, funded by the Alzheimer’s Association, GHR Foundation, and the National Institutes of Health (NIH), has been instrumental in evaluating amyloid-targeting drugs for Alzheimer’s prevention.
Initially, researchers were uncertain whether reducing amyloid levels would translate into cognitive benefits. However, the extended study revealed that prolonged treatment could significantly delay symptom onset. The longest-treated group, who received gantenerumab for an average of eight years, exhibited the strongest effect, cutting the risk of developing symptoms by 50%.
Despite these promising results, the trial faced a setback when Roche and its U.S. affiliate, Genentech, discontinued the development of gantenerumab in late 2022 following the failure of Phase 3 trials to meet primary endpoints for slowing clinical decline. However, participants transitioned to lecanemab, another FDA-approved anti-amyloid drug, to continue evaluating long-term benefits.
Like other anti-amyloid drugs, gantenerumab has been associated with amyloid-related imaging abnormalities (ARIA), which include brain swelling and micro-hemorrhages. While most cases are asymptomatic and resolve on their own, severe cases required discontinuation of the drug for a small number of participants. Nonetheless, no life-threatening adverse events were reported.
To build on these findings, the Knight Family DIAN-TU has initiated the Amyloid Removal Trial, funded by the Alzheimer’s Association, to assess how long dementia onset can be delayed by early amyloid removal. With the discontinuation of gantenerumab, most participants in the extension study have now been switched to lecanemab. Data from this phase of the study is currently pending analysis.
While the study focused on individuals with genetic forms of early-onset Alzheimer’s, researchers anticipate that the findings will inform prevention and treatment strategies for all forms of the disease. Alzheimer’s—both early-onset and late-onset—follows a similar pattern, with amyloid accumulating decades before cognitive symptoms appear. If future trials in late-onset Alzheimer’s mirror these results, Alzheimer’s prevention could become a reality for the general population.
Dr. Randall J. Bateman, senior author and the Charles F. and Joanne Knight Distinguished Professor of Neurology at WashU Medicine, expressed optimism: “I am highly optimistic now, as this could be the first clinical evidence of what will become preventions for people at risk for Alzheimer’s disease. One day soon, we may be delaying the onset of Alzheimer’s for millions.”
With continued advancements in anti-amyloid therapies, the fight against Alzheimer’s is making significant strides. While gantenerumab’s development has been discontinued, alternative drugs are being explored, offering hope for future prevention and treatment breakthroughs. The Alzheimer’s Association remains committed to expanding research efforts to ensure that Alzheimer’s and other dementias are met with effective preventive strategies in the near future.
The upcoming 42nd International Conference on Advances in Psychiatry and Mental Health will take place on November 10-11, 2025, at the Hilton Hotel, Dubai, UAE. The event will feature discussions on the latest advancements in psychiatric care, including neurodegenerative diseases such as Alzheimer’s. Attendees can earn 21 CME credits and engage with experts on groundbreaking research and therapeutic strategies.
Website: www.neurologyconf.com/psychiatry
For more details, contact team@neurologyconf.com.
As research continues, experts hope that this drug, along with other emerging therapies, will pave the way for more effective interventions in the fight against Alzheimer’s disease.