Gemfibrozil is a member of the fibrate group of medications, which typically are prescribed to control cholesterol levels. But now gemfibrozil is being investigated as a possible treatment for Alzheimer’s disease. Researchers at the University of Kentucky are interested in gemfibrozil because it affects the production of two molecules that may play a role in Alzheimer’s: miRNA 107 and β-secretase (BACE).

How gemfibrozil works

The exact cause of Alzheimer’s disease is unknown, but the disease leads to loss of connections between brain cells, called neurons, and death of these cells. One characteristic of the disease is the abnormal accumulation of two proteins: tau and beta-amyloid. These proteins can form clumps called “plaques” and “tangles” that are thought to damage neurons.

Genes essentially are the coded instructions for making a protein. When a gene is “expressed” it means the protein it codes for is made by the cell. There are a number of steps in the process from gene to protein and along the way, many factors can affect if and how much of a protein is made. MicroRNAs are one of these factors. They are small fragments of genetic material that are involved in regulating gene expression.

One particular type of microRNA called miRNA 107, appears to reduce the production of the enzyme BACE. BACE is involved in the production of beta-amyloid. Researchers have found that the amount of miRNA 107 in the brain decreases as the disease progresses. This may lead to an increase in the amount BACE and ultimately cell-damaging beta-amyloid plaques.

Some studies have shown that treatment with fibrate medications like gemfibrozil reduces the risk of Alzheimer’s by 50 percent. Gemfibrozil has been shown to increase levels of miRNA 107. Researchers hope that treatment with gemfibrozil will increase the production of miRNA 107, reducing the production of BACE and ultimately beta-amyloid plaques.

Gemfibrozil in clinical trials

Gemfibrozil has been tested in one Phase 1 clinical trial (NCT00966966) in healthy volunteers to determine how well it was absorbed and how it interacted with another Alzheimer’s medication, cerlapirdine (SAM-531). The results of this study have not been published.

There is a second, early Phase 1 trial (NCT02045056) in progress to evaluate the safety and effectiveness of gemfibrozil in increasing miRNA 107 levels to prevent Alzheimer’s. This trial includes participants with either no cognitive impairment or only mild impairment. The trial is no longer recruiting and is expected to be completed in December 2018.


Alzheimer’s News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

Meredith Walker AuthorBNS Writer
Meredith Walker AuthorBNS Writer