In a new study, researchers found that a new drug that could prevent Alzheimer’s disease by modulating, rather than inhibiting, a key enzyme involved in forming amyloid plaques in the brain.
They demonstrated that the drug is safe and effective in animals, paving the way for future clinical trials in humans.
The research was conducted by a team at the University of California San Diego and elsewhere.
A key pathological hallmark of Alzheimer’s disease involves the formation of amyloid plaques composed of small protein fragments known as amyloid-β (Aβ) peptides.
Some of these fragments are particularly prone to forming amyloid plaques, and their production is elevated in patients with mutations predisposing them to early-onset Alzheimer’s disease.
In the study, the team developed a novel drug and tested it on mice, rats, and monkeys.
Repeated low doses of the drug completely eliminated amyloid-β production in mice and rats without causing any toxic side effects.
The drug was also safe and effective in monkeys, reducing amyloid-β levels by up to 70%.
The researchers then tested the drug in a mouse model of early-onset Alzheimer’s disease, treating the animals either before or shortly after they began to form amyloid plaques.
In both cases, the novel drug decreased plaque formation and reduced plaque-associated inflammation, which is thought to contribute to the development of the disease.
These findings suggest that the drug could be used to prevent Alzheimer’s disease, either in patients with genetic mutations that increase susceptibility to the disease or in cases where amyloid plaques have been detected by brain scans.
The team says future clinical trials will determine whether this promising drug is safe in humans and could be used to effectively treat or prevent Alzheimer’s disease.
One author of the study is Steven L. Wagner, Ph.D.
The study is published in the Journal of Experimental Medicine.
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