Researchers say they have sparked an “exciting” breakthrough in treating Alzheimer’s disease by developing a drug to prevent the build-up of toxic proteins in the brain.
“This research is taking promising steps towards a new one-of-a-kind therapy which targets tau, a damaging protein in the brains of people living with Alzheimer’s, preventing it from clumping together,” said Richard Oakley, associate director of research and innovation at the Alzheimer’s Society UK, which funded the research.
Tau is a naturally occurring protein that helps stabilize nerve cells in the brain. These proteins can go bad by clumping together to form long, twisting fibrils that disrupt cell function.
Abnormal clumps of tau are called neurofibrillary tangles (NFTs) — they are a hallmark of Alzheimer’s, a neurodegenerative condition that affects an estimated 7 million Americans.
Now, an international team led by Lancaster University in the UK says it has developed a drug called RI-AG03 that targets and blocks the two “hotspots” where tau clumping tends to occur.
“There are two regions of the tau protein that act like a zipper to enable it to aggregate,” explained Amritpal Mudher, professor of neuroscience at the University of Southampton. “For the first time, we have a drug which is effective in inhibiting both these regions.”
University of Southampton researchers gave the drug to fruit flies that had toxic tau, noting that it suppressed neurodegeneration and extended the flies’ lives by about two weeks.
The average fruit fly lifespan is 40 to 50 days.
Researchers at the University of Texas Southwestern Medical Center also tested the drug in genetically engineered cells, finding that it reduced tau aggregation.
The results were published Thursday in Alzheimer’s & Dementia: The Journal of the Alzheimer’s Association.
The team plans to test RI-AG03 in rodents before launching clinical trials. However, prior promising tau-based therapies have failed in clinical trials.
“It’s important to note that the study is in its early stages, so we don’t yet know if it will work or be safe for humans, but it’s an exciting development and we look forward to seeing where it leads,” Oakley said.
There is no cure for Alzheimer’s, but certain medications aim to slow cognitive decline in people with the disease.
