Designer compound may lead to new dementia treatment

Feb. 8 (UPI) -- Research from the National Institutes of Health, or NIH, suggests the potential to prevent or reverse brain damage caused by tau protein in dementia patients.

The study of mice and monkeys showed that tau antisense oligonucleotides, which are genetically engineered to block a cell's production of tau, have the potential to treat neurodegenerative disorders such as dementia.

Toxic forms of tau are clumped together in several neurodegenerative disorders like Alzheimer's disease, tau-associated frontotemporal dementia, chronic traumatic encephalopathy and progressive supranuclear palsy, and there is currently no treatment for toxic tau.

"This compound may literally help untangle the brain damage caused by tau," Dr. Timothy Miller, Ph.D., the David Clayson Professor of Neurology at Washington University and the study's senior author, said in a press release.

Researchers tested the antisense oligonucleotide compound designed to turn tau genes off in mice that are genetically engineered to produce abnormally high levels of a mutant form of the human protein. Tau clusters began to appear in the brains of mice starting at 6 months of age and increased with age. Those mice then developed neurologic issues and died earlier than mice in the control group.

The antisense oligonucleotide compound was injected into the fluid-filled spaces of the mice brains and prevented tau from clustering in 6- to 9-month-old mice. The compound reversed the clustering of tau in older mice, causing them to live longer and have healthier brains than mice that received a placebo.

"These results open a promising new door," Margaret Sutherland, Ph.D., program director at NIH's National Institute of Neurological Disorders and Stroke. "They suggest that antisense oligonucleotides may be effective tools for tackling tau-associated disorders."

The study was published in Science Translational Medicine.