A first-of-its-kind drug that targets toxic protein tangles in the brains of Alzheimer’s patients could bring new hope to millions of people living with the disease.
Developed by a team of international researchers led by British experts, the drug addresses two “hotspots” of a protein that leads to the memory-robbing condition worsening over time.
While drugs targeting these proteins have already been developed, they typically only work in one of these areas, not both.
Development is still in the early stages and the drug has only been tested on human cells in the laboratory and in animals.
But the experts behind the drug, called RI-AG03, say the initial findings are promising.
Developed by a team of international researchers led by British experts, the drug, called RI-AG03, addresses two “hotspots” in the protein that lead to the memory theft condition worsening over time. stock image
The drug targets tau proteins, which normally play a crucial role in maintaining the health of brain cells.
However, in people with Alzheimer’s disease, they clump together and form clumps that starve brain cells of nutrients and slow down signals in the organ.
These tangles eventually kill neurons, and as more die, memory and thinking ability become increasingly impaired.
This is where RI-AG03 comes into play. Professor Amritpal Mudher, a neuroscience expert at the University of Southampton, explained that it targets two parts of the tau protein where these clusters originate.
He said that “for the first time” we have a drug that is effective in curbing the formation of tau tangles.
It works on a different mechanism than the recent successful Alzheimer’s drugs lecanemab and donanemab, which recruit the immune system to clear the buildup of another harmful disease-related protein called amyloid.
Dr Anthony Aggidis, also from Southampton and author of a new paper on RI-AG03, added that the discovery could give hope to the millions of people suffering from Alzheimer’s.
“Our research represents an important step toward creating treatments that can prevent the progression of diseases such as Alzheimer’s disease,” he said.
“By targeting both key areas of the tau protein, this unique approach could help address the growing impact of dementia on society, providing a much-needed new option for the treatment of these devastating diseases.”
The team, which also included experts from the United States and Japan, published the results of experiments with the drug in the Alzheimer’s and Dementia: The Alzheimer’s Association Magazine.
Alzheimer’s disease is the most common cause of dementia. The disease can cause anxiety, confusion and short-term memory loss.
These involved administering RI-AG03 to fruit flies with tangles of toxic tau protein.
The researchers found that the drug not only suppressed the degeneration of the insects’ brain cells, but also extended their lifespan by two weeks compared to those who did not receive the drug.
Professor Mudher said: “The higher the dose administered, the greater the improvement we see in the lifespan of the fruit fly.”
The team also conducted experiments on human cells in the lab and found similar evidence that it reduced tau accumulation.
They said they now plan to conduct further studies in rodents and, if successful, begin clinical trials in humans.
The research on RI-AG03 was funded by the charity Alzheimer’s Society UK.
Its associate director of research and innovation, Dr Richard Oakley, welcomed the results.
“Dementia is the leading cause of death in the UK and puts enormous cost and pressure on our healthcare system, which is why we are committed to funding world-leading studies like this one,” he said.
‘This research is taking promising steps towards a unique new therapy.
‘This drug has the potential to be more specific than others currently being studied and we hope it will produce fewer toxic side effects.
“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.”
According to the Alzheimer’s Society, around 982,000 people in the UK are living with dementia.
It is currently believed that around 900,000 Britons suffer from this memory-robbing disorder. But scientists at University College London estimate this figure will rise to 1.7 million within two decades as people live longer. It marks a 40 percent increase from the previous forecast in 2017.
The number is expected to rise to 1.4 million by 2040.
After years of false starts with potential Alzheimer’s drugs, there has been an avalanche of drugs that show promise.
Last year, the medical world rejoiced when the first drug that clearly slows the progression of Alzheimer’s was approved in the United States.
The makers said trial results showed that lecanemab, also known as Leqembi, slowed cognitive and functional decline in early-stage Alzheimer’s patients by 27 percent over 18 months.
This is equivalent to a five-month slowdown in disease progression.
Results from donanemab followed, showing slower progression of Alzheimer’s by up to 35 percent.
But experts have warned that people who take these drugs actually lose volume in the brain.
There has also been concern about brain hemorrhages among patients.
Results from the lecanemab trials show that about 21 percent of participants who received the drug experienced brain inflammation or bleeding, compared with 9 percent of those who received a placebo.
Most patients had no side effects or very mild symptoms, while most of those who experienced swelling or bleeding in the brain saw these problems resolve by the end of the trial.
Experts have also previously highlighted how more than 80 per cent of Britons at risk of Alzheimer’s could miss out on new drugs due to a lack of testing for the disease.
The London scientists estimated that only 14 percent of patients referred to clinics were likely to benefit from the treatments.
Researchers said there was a “clear and urgent” need to improve NHS screening to determine who might benefit from new Alzheimer’s drugs.
Alzheimer’s is the leading cause of dementia and triggers memory theft disorder in about three out of five patients.
Dementia cases are expected to skyrocket in the coming years, in part due to a growing aging population.
Currently, the only medications available for Alzheimer’s are to treat its symptoms.