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Could a novel chemical compound be effective in preventing tau tangle formation in brain diseases?
Janice Kranz, Ph.D.
Eikonizo Therapeutics, Inc.
Cambridge, MA - United States
Background
Several neurodegenerative disorders are associated with the abnormal build-up and spreading of the tau protein, which collapses into twisted strands called tangles, a hallmark brain change seen in Alzheimer’s, frontotemporal dementia, Parkinson’s disease and over 20 other brain diseases. These tangles form inside cells that die and tend to move throughout the brain as Alzheimer’s and related disorders progress.
Emerging research has found the enzyme (a protein) HDAC6 (called histone deacetylase 6), modifies the structure of tau that may promote the formation of tangles. Dr. Janice Kranz and her team at Eikonizo believe that reducing HDAC6 activity may have a therapeutic effect of preventing disease-related tau modifications and blocking the downstream formation of tau tangles in the brain.
Research Plan
Dr. Kranz and colleagues believe that blocking HDAC6 with chemical compounds can reduce accumulation of tau in brain diseases. To conduct their study, the researchers will use a genetically engineered mouse model with abnormal human tau in their brains. Dr. Kranz’s team has developed molecules that block HDAC6 and they will first test them on nerve cells (taken from these mice) in a laboratory dish. Using this model, the researchers will study the biological mechanisms that can decrease accumulation of tau protein. They will select 2-3 promising molecules from their compound pool for further evaluation.
Impact
If successful, the study results could lead to a potentially novel therapy that could be tested in human clinical trials.
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