Researchers at the Florey Institute for Neuroscience and Mental Health in Melbourne, Australia, have developed an mRNA therapeutic approach that targets the toxic protein tau (τ) that accumulates in the brains of patients with Alzheimer’s disease and other forms of dementia.
Main “tool” for vaccine development
To date, the mRNA technology that became known worldwide as the basis of vaccines against COVID-19 is mainly used for vaccine development.
First time against Alzheimer’s
But now Florey researchers led by Dr Rebecca Nisbett are turning the ‘power’ of mRNA in a new direction. “This is the first time we have explored mRNA for use in Alzheimer’s disease,” noted Dr. Nisbett, adding that “our research in cell models demonstrates that this technology can serve purposes other than vaccine development.”
“Handbook” of cells
The researcher likened mRNA to a “manual” that provides instructions to cells. “When the mRNA reaches the cell, it ‘reads’ it and produces an antibody.”
Antibody production – “key”
Florey’s team used mRNA to instruct cells in the lab to produce RNJ1, an antibody that Dr. Nisbett developed to target the t protein that has been closely linked to Alzheimer’s. “This is the first time, to our knowledge, that a T antibody has been able to bind directly to the protein inside the cell,” said the study leader.
Application to any therapeutic antibody
The paper’s first author, PhD researcher Patricia Wongsontircho said, “Our technique can be applied to any therapeutic antibody, and we envision that this strategy, when combined with mRNA-carrying nanoparticles, will lead to better targeting of toxic molecules to the brain and will improve patient outcomes compared to conventional strategies.”
Limitations of existing antibody therapies
According to Dr. Nisbett, RNJ1 needs to be studied more thoroughly. Although new treatments for Alzheimer’s disease such as the antibody lecanemab (trade name Leqembi) show promise in slowing the progression of the disease, they are associated with high costs and are not an effective way to deliver an active antibody into brain cells. “With conventional antibodies, such as lecanemab, a very small amount of the antibody that eventually enters the brain removes only a small portion of the harmful plaque that is outside the brain cells. However, these antibodies do not have access to the toxic proteins such as ‘t’ which is ‘located’ inside the brain cells” concluded the researcher.
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