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Anti-Amyloid Drug Shows Promise In Preventing Alzheimer's
- March 24, 2025
- Dennis Thompson HealthDay Reporter
The best evidence yet that cutting-edge Alzheimer’s disease drugs might indeed ward off the degenerative brain disease has emerged from a small-scale study.
An experimental drug that clears amyloid beta from the brain cut the risk of developing Alzheimer’s by 50% among a group of 22 people with genetic mutations that all but guarantee they would eventually develop the brain disease, researchers reported March 19 in The Lancet Neurology.
The drug, gantenerumab, was abandoned by its developers, but subsequent anti-amyloid drugs lecanemab (Leqembi) and donanemab (Kisunla) have since received federal government approval and are being used to treat early Alzheimer’s.
“Everyone in this study was destined to develop Alzheimer’s disease and some of them haven’t yet,” senior author Dr. Randall Bateman, a professor of neurology at Washington University School of Medicine in St. Louis, said of the gantenerumab study.
“We don’t yet know how long they will remain symptom-free – maybe a few years or maybe decades,” Bateman continued. “In order to give them the best opportunity to stay cognitively normal, we have continued treatment with another anti-amyloid antibody in hopes they will never develop symptoms at all.”
“What we do know is that it’s possible at least to delay the onset of the symptoms of Alzheimer’s disease and give people more years of healthy life,” he concluded.
All these drugs were developed based on what’s called the “amyloid hypothesis” of Alzheimer’s, which holds that the disease is caused at least in part by sticky, toxic proteins called amyloid beta that build up in the brain.
For this study, Bateman and his colleagues tested the anti-amyloid drug gantenerumab among 73 people who carry genetic mutations that make them destined to fall prey to Alzheimer’s in middle-age.
The 22 patients highlighted in the new paper had no cognitive problems at the start of the study, and received gantenerumab the longest, researchers said.
When the trial concluded in 2020, researchers reported that gantenerumab had indeed lowered amyloid levels in the patient’s brains, but the drug’s effectiveness in warding off Alzheimer’s was inconclusive.
Despite ongoing research, gantenerumab makers Roche and Genentech discontinued the drug’s development in November 2022, given that clinical trials had failed to prove any benefit in staving off cognitive decline.
But researchers kept following the initially healthy patients treated with gantenerumab, who by this time had received the drug for eight years on average.
Eventually, they found a strong effect in this longest-treated group, with the drug cutting the risk of developing Alzheimer’s symptoms in half.
“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,” Bateman said. “One day soon, we may be delaying the onset of Alzheimer’s disease for millions.”
Anti-amyloid drugs are expensive and not without risk. They can cause brain swelling, and in this small gantenerumab group rates of this side effect were one-third higher than in the original clinical trial, 30% versus 19%.
But these results show that anti-amyloid drugs might one day be proven effective as preventive medications for Alzheimer’s disease, rather than treatments given once someone has the disorder, researchers said.
“These exciting preliminary findings hint very clearly at the potential role of lowering beta amyloid in prevention of Alzheimer’s disease,” Maria Carrillo, chief science officer for the Alzheimer’s Association, concluded in a news release.
More information
The Alzheimer’s Association has more about treatments for Alzheimer’s.
SOURCE: Washington University in St. Louis, news release, March 19, 2025
