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“Unique” new Alzheimer’s drug RI-AG03 shows promise

Researchers say they have made an “exciting” breakthrough in treating Alzheimer's disease by developing a drug that prevents toxic proteins from building up in the brain.

“This research takes promising steps towards a new, unique therapy that targets tau, a harmful protein in the brains of people with Alzheimer's disease, and prevents it from clumping together,” said Richard Oakley, deputy director of research innovation at Alzheimer's Society UK, who funded the research.

Researchers at the University of Southampton administered the drug to fruit flies that had toxic dew. Here is the brain of a 7 day old fruit fly with dew. The green outlines of the neurons begin to swell and degenerate due to the dew. The red shows where dew accumulates. University of Southampton

Tau is a naturally occurring protein that helps stabilize nerve cells in the brain. These proteins can spoil by clumping together into long, twisted fibrils that disrupt cell function.

Abnormal clumps of tau are called neurofibrillary tangles (NFTs) — they are a hallmark of Alzheimer's, a neurodegenerative disease that affects an estimated 7 million Americans.

Now an international team led by Lancaster University in the United Kingdom says it has developed a drug called RI-AG03 that targets and blocks the two “hotspots” where tau clumps occur.

Using a special imaging technique, scientists observed how tau protein clumped together 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 and allow aggregation,” explains Amritpal Mudher, professor of neuroscience at the University of Southampton. “For the first time we have a drug that effectively inhibits both regions.”

Researchers at the University of Southampton gave the drug to fruit flies containing toxic dew and found 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 in genetically modified cells and found that it reduced tau aggregation.

The findings were published Thursday in Alzheimer's & Dementia: The Journal of the Alzheimer's Association.

Anthony Aggidis, a former postdoctoral fellow 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 beginning clinical trials. However, previous promising tau-based therapies failed in clinical trials.

“It's important to note that the study is still in its early stages, so we don't yet know whether it will work or be safe for humans, but it's an exciting development and we look forward to seeing where it goes she leads,” Oakley said.

There is no cure for Alzheimer's, but certain medications aim to slow cognitive decline in people with the disease.

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