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Scientists have developed an "innovative" method to attack toxic particles that cause brain disorders such as Alzheimer's disease.
The team hopes that drugs developed as a result of their research could be tested in two or three years, said one researcher. Newsweek. The team reported its findings in the journal Proceedings of the National Academy of Sciences of the United States of America.
Alzheimer's disease is a form of dementia that sees the death of nerve cells and the loss of tissue in the brain. As it shrinks, patients experience symptoms, including personality changes, memory failure, and difficulty performing daily tasks.
The disease affects approximately 5.7 million Americans, according to reports from Centers for Disease Control and Prevention (CDC). Associated with aging, it is the fifth leading cause of death among people aged 65 and over. In 2016, the United States had 49 million people aged 65 and over, and the CDC expects this number to double by 2060.
At the molecular level, Alzheimer's disease is linked to the accumulation of dangerous clusters of proteins in the brain, said Michele Vendruscolo, a researcher at Cambridge University and author of the study. Newsweek. Small and soluble "toxic particles" are formed during this process and kill the neurons, he explained.
Vendruscolo and his team used an "innovative approach to drug discovery" to develop small molecules that can slow the formation of these toxic particles, he said. His team's method was "the first" to directly target these particles and "dramatically" reduce their number.
Until now, researchers have tested the molecules during laboratory tests and roundworms. "We are testing them on a mouse model," said Vendruscolo, adding that his team could see the first results of clinical trials in two or three years.
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Although hundreds of clinical trials have focused on Alzheimer's disease, none has yet targeted the toxic particles that can cause this disease. "Although this is currently the most widely accepted hypothesis, it has not been proven yet," added Vendruscolo. "Our small molecules provide a powerful way to prove or disprove this hypothesis."
"Innovative" research could provide a "simplified path" for drug discovery that prevents early protein accumulation, said Jagan Pillai, an Alzheimer neurologist at Cleveland Clinic. Newsweek. "It remains to be seen whether promising compounds could also be evaluated for other [related] disorders, including Parkinson's disease and Huntington's disease.
Nikhil Palekar, Medical Director of the Center of Excellence for Alzheimer's Disease and Associate Professor of Psychiatry at Stony Brook Medicine, called the results important. Current treatments "are not very effective," he said. Newsweekand "recent attempts at drug development have failed to improve symptoms or slow the progression of the disease."
Read more: Zombie brain cells could play a key role in the fight against Alzheimer's disease
While Alzheimer's therapies have been gaining enthusiasm and interest, Pillai added that targeted studies are urgently needed to develop a sustainable cure.
The new research is promising in theory, said Palekar, but it "must translate into successful drug development." The complex nature of diseases such as Alzheimer's disease could complicate things, he added. "Alzheimer's disease is an extremely important public health problem, with millions of people around the world. The need for more research into new therapeutic targets can not be overstated.
"We hope that our research will help the public no longer consider Alzheimer's as a death sentence to a curable or preventable disease," Vendruscolo said.
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