Reduction of Tau Protein Levels Help Minimize Symptoms of Dravet Syndrome

Dravet syndrome is classified as a genetic and developmental epilepsy syndrome that is diagnosed during infancy. Though this neurological health issue is rare, researchers set out to determine how tau proteins could affect children with severe childhood epilepsy.

Findings published in the journal Annals of Neurology found that reducing the protein helped to suppress seizure activity and reduce the risk of premature death. Tau reduction was even linked to reduced risk of cognitive and behavioral abnormalities commonly associated with the health issue.

"It would really be wonderful if tau reduction turned out to be useful not only in Alzheimer's disease, but also in other disabling neurological conditions for which there currently are no effective treatments," said senior author Lennart Mucke, MD, the director of the Gladstone Institute of Neurological Disease and a professor of Neurology and Neuroscience at the University of California, San Francisco, in a news release. "We suspected that this approach might be beneficial in Dravet, but we couldn't be sure because of the severity of this syndrome and the corresponding model. We are thrilled that our strategy was so effective, but a lot more work is needed to advance it into the clinic."

For the study, researchers zeroed in on Dravet syndrome and how tau protein levels can affect the brain. Together, they created mouse models with the health issue. During the study, they reduced their levels of tau protein by suppressing the activity of the gene that makes up the protein.

By deleting just one copy of the gene, the models dramatically improved. Eliminating more copies showed more symptoms disappear. 

"I am especially excited about the improvements we observed in cognitive and behavioral dysfunctions because these abnormalities are particularly hard on the kids-and their parents," said first author Ania Gheyara, MD, PhD, a staff scientist at Gladstone who is also affiliated with the UCSF Department of Pathology, reported in the press release. "Our hope is that this approach will be broadly applicable to many different types of epilepsy."