Common Oral Diuretic Pill as Alzheimer’s Treatment, Study Shows


A commonly available, FDA-approved oral diuretic pill may be a potential candidate for Alzheimer's disease treatment, according to new research.

A commonly available oral diuretic pill, bumetanide, has been approved by the U.S. Food and Drug Administration and shows potential for Alzheimer's disease treatment.

In an analysis of clinical trials conducted, those who took bumetanide reported having a lower prevalence of Alzheimer’s disease compared to those who did not take the drug. Researchers analyzed data 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 may potentially be repurposed to treat Alzheimer's. The findings were published in Nature Aging.1

"Though further tests and clinical trials are needed, this research underscores the value of big data-driven tactics combined with more traditional scientific approaches to identify existing FDA-approved drugs as candidates for drug repurposing to treat Alzheimer's disease," said NIA Director Richard J. Hodes, M.D.

Researchers retrieved data from 213 brain tissue samples and researchers identified Alzheimer's gene expression signatures and studied the levels to which the genes were turned on or off, specific to APOE4 carriers. One of the main genetic risk factors for Alzheimer's is a form of the apolipoprotein E gene, APOE4.

Further, scientists compared the APOE4-specific Alzheimer's signatures against over 1,300 established FDA-approved drugs. Five drugs became known with a gene expression signature thought to counteract the disease. The strongest candidate of those studied was bumetanide. Bumetanide is used to treat fluid retention caused by heart, kidney, and liver disease.

Bumetanide was tested in both mouse models of Alzheimer's and induced pluripotent stem cell-derived human neurons. It was found that treating mice which expressed the human APOE4 gene lessened learning and memory deficits.

The neutralizing effects were established in the human cell-based models. Therefore, people already taking bumetanide were expected to have lower rates of Alzheimer's. Health record data sets from over 5 million people were studied in two groups: those over age 65 who took bumetanide and a control group who did not take the drug.

The results showed that those who had the genetic risk and took bumetanide had a 35 percent to 75 percent lower prevalence of Alzheimer's disease compared to those not taking the drug.

"We know that Alzheimer's disease will likely require specific types of treatments, perhaps multiple therapies, including some that may target an individual's unique genetic and disease characteristics -- much like cancer treatments that are available today," said Jean Yuan, M.D., Ph.D., Translational Bioinformatics and Drug Development program director in the NIA Division of Neuroscience.1


1. News Release. Precision Medicine Data Dive shows diuretic pill may be viable to test as Alzheimer’s treatment. ScienceDaily. Accessed October 12, 2021.

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