In some people, the normal brain protein tau collects into toxic tangles that damage brain cells and contributes to diseases such as Alzheimer’s. Now, researchers report that they have found a drug can lower tau levels and prevent some of the neurological damage. The findings suggest a potential treatment for tau-related neurodegenerative diseases.
Under ordinary circumstances, the protein tau contributes to the normal, healthy functioning of brain neurons. In some people, though, it collects into toxic tangles that damage brain cells. Such tangles are a hallmark of Alzheimer’s and other neurodegenerative diseases.
The study, in mice and monkeys, is published Jan. 25 in Science Translational Medicine. The findings suggest that the molecule — known as an antisense oligonucleotide — potentially could treat neurodegenerative diseases characterized by abnormal tau, including Alzheimer’s.
“We’ve shown that this molecule lowers levels of the tau protein, preventing and, in some cases, reversing the neurological damage,” said Timothy Miller, MD, PhD, the David Clayson Professor of Neurology and the study’s senior author. “This compound is the first that has been shown to reverse tau-related damage to the brain that also has the potential to be used as a therapeutic in people.”