‘One-of-a-kind’ new Alzheimer’s drug shows promise: ‘An exciting development’

· New York Post

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 toward 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.

University of Southampton researchers gave the drug to fruit flies that had toxic tau. Here is the brain of a 7-day-old fruit fly with tau. The green outlines the neurons, which are starting to swell and degenerate due to the tau. The red shows where tau is building up. University of Southampton

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 — 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 “hot spots” where tau clumping tends to occur.

Scientists used a special imaging technique to observe how tau protein clumps in the brain tissue of genetically modified flies in response to treatment with RI-AG03. University of Southampton

“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.

Anthony Aggidis, former postdoctoral research associate at Lancaster University and visiting researcher at the University of Southampton, led the research. Lancaster University

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.

Dr. Liron Sinvani — North Shore University Hospital director of geriatric hospital services, who was not involved in the new research — told The Post that RI-AG03 is “more targeted than current treatments, potentially making it safer, with fewer side effects.”

“While these findings are very exciting and could provide a much-needed treatment for this devastating disease, it is important to note that there is significant work that needs to be done,” Sinvani added, “including testing to see whether RI-AG03 is effective in safely preventing tau protein build-up in people themselves, and more importantly whether this translates into a clinical benefit, meaning does it prevent or slow the progression of Alzheimer’s disease.”