Breakthrough Could Lead to Treatments for Many Neurological Disorders…

Hope Center researchers have identified a molecule that could prevent or even reverse the buildup of a protein that causes neurological disorders, including Alzheimer’s.

The tau protein ordinarily contributes to normal, healthy brain function, but in some cases, tau accumulates in tangles that damage brain cells.

Dr. Timothy Miller and his colleagues studied a synthetic molecule that targets the genetic instructions for building tau. Their findings show great promise for development of treatment and cures for Alzheimer’s and similar neurological disorders.

“We’ve shown that this molecule lowers levels of the tau protein, preventing and, in some cases, reversing the neurological damage,” said Miller, MD, PhD, a Hope Center scientist who led the study. “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.”

Researchers first studied effects of the molecule, known as an antisense oligonucleotide, on mice. They then studied the molecule’s effect on monkeys. Miller and his team hope to study ways to reduce tau in people. By successfully showing that this molecule can safely and effectively reduce tau in a primate, the researchers moved closer to human trails and treatments.

“Tau tangles correlate with cognitive decline in several diseases,” Miller said. “This is a promising new approach to lowering tau, but we have to test whether it is safe in people, and whether it actually lowers tau, as it is designed to do, before we get to the question of whether it has any effect on the disease. But everything we’ve seen so far says that this is worth investigating as a potential treatment for people.”

About Dr. Miller and His Collaborative Research

Dr. Timothy Miller is the David Clayson Professor of Neurology at Washington University School of Medicine. He is the director of the Christopher Wells Hobler Lab for ALS Research at the Hope Center for Neurological Disorders. Like so many other Hope Center scientists, he works and collaborates across multiple disciplines and conducts research that could lead to breakthroughs in many disorders. While this study is associated with Alzheimer’s, Dr. Miller notes that it has implications for other disorders.

Dr. Miller’s approach and research reflect a key principle and purpose for Hope Happens: Breakthroughs related to one disorder will lead to breakthroughs in others.

For more information about this study, click here.


Dr. Timothy Miller

In some people, the brain protein tau collects into toxic tangles that damage brain cells and contribute to diseases such as Alzheimer’s. Researchers at Washington University School of Medicine in St. Louis have found a drug that can lower tau levels and prevent some neurological damage. In neurons that contain the drug (above, in red) there are no tau tangles (in green). (Image: Sarah Devos)