Researchers tested a novel drug on people with a genetically inherited form of Alzheimer’s in a recent trial. It is almost impossible for someone who has dementia to prevent developing it. Gantenerumab helps remove amyloid plaques, which are one of the neurological characteristics of Alzheimer’s. The drug lowered some participants’ likelihood of developing the condition by more than 50%
A new study offers much-needed hope for the treatment of Alzheimer’s disease. According to a recent clinical experiment conducted by the Washington University School of Medicine, early anti-amyloid medication intervention may postpone or perhaps prevent Alzheimer’s.
The research published in The Lancet Neurology focused on people who had rare genetic abnormalities that result in early-onset Alzheimer’s disease [1]. It offered substantial evidence that clearing the brain of amyloid plaques long before symptoms appear could considerably slow cognitive deterioration.
The researchers have identified an anti-amyloid drug that reduced some participants’ risk of developing the condition by up to 50%.
Alzheimer’s Disease: The Amyloid Hypothesis
Although there are many unanswered concerns about Alzheimer’s and a substantial dearth of viable treatments, scientists believe a protein known as amyloid-beta plays an integral part in the disease.
Amyloid-beta is present in healthy brains, but in Alzheimer’s patients, it aggregates to create plaques that impair the brain’s ability to function normally. Scientists believe that this is the primary cause of severe cognitive deterioration.
The amyloid concept prompted researchers to create monoclonal antibody treatments, such as aducanumab, lecanemab, and donanemab, that target and eliminate these protein accumulations.
According to research, these medications may reduce cognitive deterioration in the early stages of Alzheimer’s disease by preventing the buildup of amyloid-beta [2].
Nevertheless, whether monoclonal antibody treatments could stop or postpone the beginning of Alzheimer’s in at-risk patients has not yet been studied by researchers. The most recent addresses this question while providing evidence in favor of the amyloid hypothesis.
DIAD: Focus of the Recent Study
The most recent study focused on persons with dominantly inherited Alzheimer’s disease (DIAD). These people have mutations in particular genes that lead to amyloid-beta buildup.
Due to this genetic propensity, their chances of having Alzheimer’s disease in their early to mid-adult years are about 100%. Since the disorder is almost unavoidable, these people offer a unique paradigm for testing innovative strategies to prevent Alzheimer’s.
The Study Findings: Gantenerumab Lowers Risk in Some Patients
The study tracked 73 individuals who had inherited genetic variants that almost always result in Alzheimer’s disease in their 30s, 40s, or 50s.
Participants in this study first signed up for the first-ever Alzheimer’s prevention trial, DIAN-TU-001. Individuals in this anti-amyloid drug trial had either no symptoms at all or only minor cognitive issues when the trial started in 2012. At this point, they were between 15 and 10 years younger than the average age at which Alzheimer’s disease would emerge.
The trial concluded in 2020 and demonstrated that gantenerumab, an anti-amyloid drug developed by Roche/Genentech, significantly lowered amyloid-beta levels while improving several other Alzheimer’s disease markers.
Although Roche/Genentech discontinued gantenerumab research in 2022 due to unsatisfactory results in a larger trial, the expanded study found that long-term early treatment significantly impacted cognitive deterioration.
Despite the favorable signs, there were no discernible cognitive improvements compared to the placebo group because neither group had acquired symptoms yet. Therefore, the researchers continued the study and increased the dosage to observe how gantenerumab would perform over time.
Although gantenerumab did not make a substantial difference in all of the study’s groups, it did benefit some.
The effect was evident for those who had taken the medicine the longest. After receiving the experimental drug for an average of eight years, the probability of acquiring symptoms was reduced from nearly 100% to roughly 50% among a subgroup of 22 people who did not initially exhibit any cognitive symptoms.
Some participants are only nearing or have recently passed their expected age of disease onset. Therefore, as the study progresses, this percentage may grow if participants remain symptom-free or decline if they develop the condition.
Senior author Randall J. Bateman, MD, of WashU Medicine in St. Louis, MI, claimed in a press release that everyone in this study was predestined to get Alzheimer’s, while some have yet to do so [3].
He added that they were unsure about how long they would be symptom-free, estimating it may be several years or even decades. To give them the best chance of remaining cognitively normal, they continued treatment with another anti-amyloid antibody in hopes of never developing symptoms.
Bateman concluded that they only know that it is possible to delay the onset of Alzheimer’s symptoms and give people more years of healthy life.
The Next Step: Extending Alzheimer’s Prevention Trials
To investigate the long-term impacts of amyloid removal, the Knight Family DIAN-TU research team has started the DIAN-TU Amyloid Removal Trial [4]. The FDA-approved anti-amyloid medication lecanemab, which slows cognitive loss in symptomatic Alzheimer’s patients, has replaced gantenerumab, which is no longer available, for the majority of participants.
Maria C. Carrillo, PhD, CSO of the Alzheimer’s Association, stressed the necessity of future research, adding that these findings suggest a potential role of decreasing beta-amyloid in Alzheimer’s prevention. They hope future research will expand on and validate these ground-breaking findings [3].
At the same time, scientists are recruiting younger people as young as 18 to participate in a Primary Prevention Trial to see if halting amyloid buildup decades before symptoms manifest may prevent the disease entirely [5, 6].
What’s Next?: Future Prospects for Alzheimer’s Prevention
Although the study’s focus was on hereditary early-onset Alzheimer’s, its conclusions might have wider ramifications. Future research could ascertain whether the same preventive strategy could help millions of people at risk for late-onset Alzheimer’s as early-onset and late-onset Alzheimer’s share comparable amyloid-driven pathways.
Bateman expressed his optimism that we would be able to delay or perhaps prevent Alzheimer’s disease for millions of people soon.
Alzheimer’s Research Association is a non-profit organization dedicated to helping caregivers of Alzheimer’s disease and dementia. We provide the latest information and news about the illness and helpful tips to help caregivers cope with their daily caregiving challenges. We realize the most important thing that a caregiver needs is financial assistance. Therefore, we provide grants to caregivers to ease their financial burden. Caregivers can apply for grants here: Alzheimer’s Grant Application.
You can also help caregivers in their endeavor by donating as much as possible: Donation To Alzheimer’s Research Associations.
References
- Bateman, R.J., Li, Y., McDade, E.M., Llibre-Guerra, J.J., Clifford, D.B., Atri, A., Mills, S.L., Santacruz, A.M., Wang, G., Supnet, C. and Benzinger, T.L., 2025. Safety and efficacy of long-term gantenerumab treatment in dominantly inherited Alzheimer’s disease: an open-label extension of the phase 2/3 multicentre, randomised, double-blind, placebo-controlled platform DIAN-TU trial. The Lancet Neurology, 24(4), pp.316-330.
- Budd Haeberlein, S., Aisen, P.S., Barkhof, F., Chalkias, S., Chen, T., Cohen, S., Dent, G., Hansson, O., Harrison, K., Von Hehn, C. and Iwatsubo, T., 2022. Two randomized phase 3 studies of aducanumab in early Alzheimer’s disease. The journal of prevention of Alzheimer’s disease, 9(2), pp.197-210.
- Schneider, T. Anti-amyloid drug shows signs of preventing Alzheimer’s dementia. News Release. WashU Medicine. https://medicine.washu.edu/news/anti-amyloid-drug-shows-signs-of-preventing-alzheimers-dementia/. Published Online: 19th March, 2025. Accessed: 9th April, 2025.
- DIAN-TU Amyloid Removal Trial (ART) in Dominantly Inherited Alzheimer’s Disease (DIAN-TU). ClinicalTrials.gov. NIH National Library of Medicine. https://clinicaltrials.gov/study/NCT06384573. Last Updated: 3rd April, 2025. Accessed: 10th April, 2025.
- International Alzheimer’s prevention trial in young adults begins. News Release. WashU Medicine. https://medicine.washu.edu/news/international-alzheimers-prevention-trial-in-young-adults-begins/. Published Online: 4th February, 2025. Accessed: 10th April, 2025.
- Primary Prevention Trial. Dominantly Inherited Alzheimer’s Network (DIAN). WashU Medicine. https://dian.wustl.edu/clinical-trials/primary-prevention-trial/. Accessed: 10th April, 2025.
- Newman, T. Anti-amyloid drug may help reduce the risk of early-onset Alzheimer’s by 50%. Medical News Today. https://www.medicalnewstoday.com/articles/anti-amyloid-drug-may-help-reduce-risk-early-onset-alzheimers-symptoms. Published Online: 20th March, 2025. Accessed: 9th April, 2025.
- Anti-Amyloid Drug Shows Potential to Delay Alzheimer’s Symptoms. Inside Precision Medicine. https://www.insideprecisionmedicine.com/topics/precision-medicine/anti-amyloid-drug-shows-potential-to-delay-alzheimers-symptoms/. Published Online: 21st March, 2025. Accessed: 9th April, 2025.
