A genetically engineered compound that blocks the production of a protein known as tau could lead the way toward preventing a variety of disorders, including Alzheimer's disease, the National Institutes of Health (NIH) reported recently.
Researchers working with mice and monkeys said the compound, called tau antisense oligonucleotide, effectively prevented brain cells from forming toxic tau tangles, which are present in Alzheimer’s, chronic traumatic encephalopathy, progressive supranuclear palsy and tau-associated frontotemporal dementia.
If successful, the compound would be the first effective treatment for toxic tau.
"This compound may literally help untangle the brain damage caused by tau,” Dr. Timothy Miller, senior author of the study and a professor of neurology at Washington University in St. Louis, said.
In the study, some mice began accumulating tau clusters beginning at 6 months of age. These mice died earlier than control mice after developing neurological problems. When the researchers injected the designer compound into the mice, tau clustering was blocked and even appeared to be reversed in older mice.
“These results open a promising new door,” Margaret Sutherland, program director at the NIH’s National Institute of Neurological Disorders and Stroke, said. “They suggest that antisense oligonucleotides may be effective tools for tackling tau-associated disorders.”
The study’s results were published in the journal Science Translational Medicine.
