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NIH Record - National Institutes of Health

Could Water Pill Be Treatment for Alzheimer’s?

An older woman holds a pill in one hand a glass of water in the other.

People taking bumetanide had significantly lower prevalence of Alzheimer’s.

Photo: Fizkes/Shutterstock

A commonly available FDA-approved oral diuretic pill may be a potential candidate for an Alzheimer’s disease treatment for those who are at genetic risk, according to findings published in Nature Aging

The research included analysis showing that those who took bumetanide—a commonly used, potent diuretic—had a significantly lower prevalence of Alzheimer’s disease compared to those not taking the drug. The NIA-funded study advances a precision medicine approach for individuals at greater risk of the disease due to their genetic makeup.

The research team analyzed information in databases of brain tissue samples and FDA-approved drugs, performed mouse and human cell experiments and explored human population studies to identify bumetanide as a leading drug candidate that potentially may be repurposed to treat Alzheimer’s.

Knowing that one of the most significant genetic risk factors for late-onset Alzheimer’s is a form of the apolipoprotein E gene called APOE4, researchers analyzed data derived from 213 brain tissue samples. Next, they compared the APOE4-specific Alzheimer’s signatures against those of more than 1,300 known FDA-approved drugs. Five drugs emerged with a gene expression signature that potentially might help neutralize the disease, the strongest of which was bumetanide.

The researchers then validated the data-driven discoveries by testing bumetanide in mouse models and induced pluripotent stem cell-derived human neurons. Researchers found that treating mice that expressed the human APOE4 gene reduced learning and memory deficits. The neutralizing effects were also confirmed in the human cell-based models, which led to the hypothesis that people already taking bumetanide should have lower rates of Alzheimer’s. 

To test this, the team pared down electronic health record data sets from more than 5 million people to 2 groups—adults over age 65 who took bumetanide and a matching group who did not take the drug. The analysis showed that those who had the genetic risk and took bumetanide had a 35 to 75 percent lower prevalence of Alzheimer’s disease compared to those not taking the drug.

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