New “one-of-a-kind” Alzheimer’s drug RI-AG03 holds promise

Researchers say they have made an “exciting” breakthrough in the treatment of Alzheimer’s disease by developing a drug to prevent toxic proteins from building up in the brain.

“This research is taking promising steps toward a new one-of-a-kind therapy that targets tau, a harmful 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 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 become damaged by clumping together to form long, twisted fibrils that disrupt cell function.

Abnormal clumps of tau are called neurofibrillary tangles (NFTs)—they’re a hallmark of Alzheimer’s, a neurodegenerative condition that affects about 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 aggregation tends to occur.

“There are two regions of the tau protein that act as a chain to enable it to aggregate,” explained Amritpal Mudher, professor of neuroscience at the University of Southampton. “For the first time, we have a drug that is effective at inhibiting both of 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 lifespan of fruit flies is 40 to 50 days.

Researchers at the University of Texas Southwestern Medical Center also tested the drug on genetically engineered cells, finding that it reduced the accumulation of tau.

The results were published Thursday in Alzheimer’s & Dementia: Journal of the Alzheimer’s Association.

The team plans to test RI-AG03 in rodents before starting clinical trials. However, previous 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’re looking forward to seeing where it goes.” he leads,” Oakley said.

There is no cure for Alzheimer’s, but some drugs aim to slow cognitive decline in people with the disease.