Gemfibrozil is a medication typically prescribed to control cholesterol levels. It is also 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 might play a role in Alzheimer’s disease: 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 nerve cells and their death. One characteristic of the disease is the abnormal accumulation of two proteins, tau and beta-amyloid, in the brain These proteins can form clumps called “plaques” and “tangles” that are thought to damage nerve cells.

MicroRNAs are small fragments of genetic material that are involved in regulating gene function. One particular type of microRNA, called miRNA 107, appears to reduce the production of an enzyme called BACE. This enzyme 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 of BACE and, ultimately, the amount of beta-amyloid plaques.

Some studies have shown that treatment with fibrate medications such as gemfibrozil reduces the risk of Alzheimer’s disease 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

A study in an Alzheimer’s mouse model showed a reduction in amyloid plaque buildup and improved memory in the animals. A more recent Chinese study also found a reduction in amyloid plaque in vitro with the administration of gemfibrozil. Both studies found that one of the ways gemfibrozil worked was through activation of PPARA (peroxisome proliferator-activated receptor alpha), which activated autophagy in neuronal cells. In autophagy, the cells break down unnecessary or broken cellular components such as amyloid-beta.

Gemfibrozil has been tested in a 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 disease. This trial includes participants with either no cognitive impairment or only mild impairment. The trial is still active but no longer recruiting participants.


Last updated: July 21, 2019


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