Researchers at the University of Southampton have celebrated a promising breakthrough in drugs to treat Alzheimer’s disease.
For the first time, scientists have developed a drug that targets both key areas of a protein linked to the disease.
The study found that the drug, called RI-AG03, was effective in preventing the formation of tau proteins in both laboratory and fruit fly studies.
The main author Dr. Anthony Aggidis, visiting researcher at the University of Southampton, said: “Our research represents an important step towards developing treatments that can prevent the progression of diseases such as Alzheimer’s disease.”
“By targeting both key areas of the tau protein, this unique approach could help address the growing impact of dementia on society and provide a much-needed new option to treat these devastating diseases.”
Tau proteins help stabilize the internal skeleton of nerve cells (neurons) in the brain, but Alzheimer’s disease causes these proteins to malfunction and clump together into tangles that clog the neurons.
As more neurons die, memory, thinking, and behavior become more impaired, leading to the cognitive decline seen in Alzheimer’s disease.
Researchers say there are two hotspots of the tau protein where this clumping occurs. While current treatments target one of these hotspots, the new drug exclusively targets and blocks both hotspots.
Amritpal Mudher, professor of neuroscience at the University of Southampton, said: “For the first time we have a drug that effectively inhibits both regions.
“This dual-targeting mechanism is significant because it targets both domains that stimulate tau aggregation, potentially paving the way for more effective treatments for neurodegenerative diseases such as Alzheimer’s.”
When tested on fruit flies, the drug suppressed damage to neurons and extended the flies’ lives by about two weeks – a significant increase given the insects’ lifespan.
Researchers now plan to test the drug on rodents before moving on to clinical trials.
They believe their work will have a significant impact on drug development efforts in the area of neurodegenerative diseases.