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2019 Alzheimer's Innovation Award (WWDAIA)

Developing HDAC6 inhibitors as novel therapeutics for Alzheimer's Disease

Can a novel chemical molecule restore three major biological mechanisms severely disrupted by Alzheimer’s?
 

Janice Kranz, Ph.D.
Eikonizo Therapeutics, Inc.
Cambridge, MA - United States



Background

Many therapies being developed to treat Alzheimer’s target a specific biology, like amyloid or tau. However, past studies have revealed that multiple biological pathways play a role in Alzheimer’s dementia. As a result, an emerging concept in the field is to target these different pathways to more broadly delay, or prevent Alzheimer’s progression.
 
Emerging research has found the chemical substance HDAC6 (histone deacetylase 6) plays a key role in at least three mechanisms directly related to Alzheimer’s progression 1) including tau protein accumulation into tangles, which is a hallmark of Alzheimer’s, 2) biology related to the overall health of the cell, including the way tau and other proteins fold properly and remain stable, and 3) interference in biological pathways that assist in nerve cell communication in the brain. Preliminary studies have shown blocking HDAC6 in genetically engineered Alzheimer’s-like mouse model improves cognition.
 

Research Plan

Dr. Janice Kranz and colleagues have developed a molecule that could block HDAC6. The researchers will test their potential therapy in genetically engineered Alzheimer’s-like mice and measure its impact on downstream biology.  Dr. Kranz will also assess whether their experimental drug can safely enter the brain in these mice.
 

Impact

If successful, the study may advance this novel potential therapy to clinical trials for Alzheimer’s.
 

The Alzheimer’s Innovation Award is jointly funded by the Alzheimer's Association and Johnson & Johnson.
 

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