Promising Drug Compound Blocks Alzheimer's-Related Brain Damage in Mice


Researchers have found a way to potentially combat the damaging effects of neurodegenerative diseases such as Alzheimer’s.

Researchers have found a way to potentially combat the damaging effects of neurodegenerative diseases such as Alzheimer’s. A study spearheaded by the Washington University School of Medicine in St. Louis is focusing on a drug that lowers tau levels.

Tau proteins are responsible for the buildup of toxic tangles that lead to brain cell damage and possibly Alzheimer’s disease. Tau levels in the brain have been linked to cognitive decline in many neurodegenerative diseases. The study’s findings support a molecule known as an antisense oligonucleotide as a potential treatment for neurodegenerative diseases attributed to abnormal tau proteins. The molecule, which interferes with the genetic instruction for building tau proteins, can be used as a means of impeding tau levels.

The researchers noted that a drug compound — an anti-tau oligonucleotide – halted hippocampus shrinkage and cell death in treated mice. Mice received a dose of the compound every day for a month, and then their tau RNA levels, total tau protein, and tangles of tau protein in the brain were measured. Levels of tau and tau tangles in the brains of treated mice were significantly lower than in the untreated mice, indicating that treatment also reversed tau levels. The treated mice lived an average of 36 days longer than their untreated counterparts, and exhibited indications of social behavior, cognitive performance, and motor capabilities.

“We’ve shown that this molecule lowers levels of the tau protein, preventing and, in some cases, reversing the neurological damage,” Timothy Miller, MD, PhD, said in a news release. “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.”

If effective in humans, the drug could be a treatment option for preventing and reversing tau-related damage caused by previously incurable diseases such as Alzheimer’s. The FDA recently approved oligonucleotide treatments for Duchenne’s muscular dystrophy and spinal muscular atrophy, and human trials for several other neurological diseases such as Huntington’s disease and amyotrophic lateral sclerosis are underway.


Drug compound halts Alzheimer’s-related damage in mice [news release]. St. Louis. WUSTL’s website. Accessed Jan. 26, 2017.

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