As Alzheimer's disease now affects 7 million Americans (the largest number of Americans ever) there is a growing demand for new treatments.
Scientists at the University of California, Irvine have discovered a "groundbreaking" new treatment to fight the disease.
According to a UCI press release, the treatment involves using stem cells to "program" human immune cells, called microglia, to counteract signs of brain dementia.
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According to the senator, microglia are immune cells found in the central nervous system that act as the "main line of defense against infection and damage" for the brain.
Using CRISPR gene editing, scientists designed the cells to produce an enzyme called Neprilysin, which has been shown to destroy toxic beta-amyloid plaques that accumulate in the brains of patients with Alzheimer's disease.
As Alzheimer's disease now affects 7 million Americans (the largest number of Americans ever) there is a growing demand for new treatments. (iStock)
The researchers found that in the brains of mice, engineered cells were found to retain neurons, reduce inflammation, reduce the accumulation of beta-amyloid and reverse neurodegeneration.
The study was funded by the National Institutes of Health and was published in the journal Cell Cells.
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"Driven biologics to the brain has always been a major challenge because of the disorder of the blood-brain barrier," said Mathew Blurton-Jones, professor of neurobiology and behavior.
“We have developed a programmable living transport system that solves this problem by living in the brain itself and reacting only when and where it is needed.”
The researchers noted that programmed cells target only amyloid plaques, making the method “highly targeted but widely effective.”
“We have developed a programmable living transport system that solves this problem by living in the brain itself and reacting only when and where it is needed.”
The study also found that microglia can effectively combat other central nervous system diseases such as brain cancer and multiple sclerosis.
“This work opens the door to a completely new brain therapy,” said Robert C. Spitale, a professor of pharmaceutical sciences, in a press release.
“We don’t use synthetic drugs or viral vectors, but instead use immune cells from the brain as precise delivery vehicles.”
Using CRISPR gene editing, scientists designed the cells to produce an enzyme called Neprilysin, which has been shown to destroy toxic beta-amyloid plaques that accumulate in the brains of patients with Alzheimer's disease. (iStock)
Dr. Joel Salinas, a behavioral neurologist and associate professor at New York University’s Grossman School of Medicine, said the study was an “impressive proof of concept” of a highly targeted and responsive brain therapy.
"One of the most exciting aspects is precision - rather than releasing treatments throughout the brain, it activates these cells when the disease-related damage occurs," Salinas, who was not involved in the study, told Fox News Digital.
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“This targeted action can help limit damage to healthy brain tissue, reduce side effects, and focus on therapeutic effects when most needed.”
Although the results were predated by mice, Salinas noted that this strategy opened up “promising new avenues.”
A neurologist noted that while the results were early and limited to mice, the strategy opened up “promising new avenues”. (iStock)
According to release, the researchers aimed at conducting human trials that may use stem cells from each patient to reduce the risk of immune rejection.
"If future research shows that this approach is safe, durable and effective in humans, it may not only adapt to Alzheimer's, but also to other diseases with spotted or localized processes, such as brain tumors, multiple sclerosis, or other neurodegenerative diseases that cause dementia," Salinas said.
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Courtney Kloske, director of scientific participation in the Chicago Alzheimer's Association, also reviewed the UCI findings.
"As the population continues to age, strategic research funding has expanded the pipeline of treatment for Alzheimer's and other diseases that cause dementia," she told Fox News Digital.
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“It is equally important to explore different drug delivery methods, such as using genetically modified cells demonstrated by this newly published study.”
Klosk pointed out that these findings are promising and promising, but preliminary.
"Other studies are needed to determine how this type of drug delivery mechanism affects people with or at risk for Alzheimer's disease," said a representative from the Alzheimer's Association. (iStock)
“Other studies are needed to determine how this type of drug delivery mechanism affects people at or at risk of Alzheimer’s,” she said.
“This work was done in animal models; the authors highlight the importance of advancing this study into clinical trials in order to better understand the therapeutic potential of this drug delivery mechanism.”
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In addition to NIH, the study was supported by the California Institute of Regenerative Medicine and the Cure Alzheimer's Fund.