Data dive finds cheap diuretic could be Alzheimer's drug
A data-mining study conducted by researchers in the US has found that an already-approved diuretic drug could have potential as a treatment for some patients with Alzheimer's disease.
The research by a team of National Institutes of Health (NIH) scientists found that people who took bumetanide, a generic drug usually used to treat heart failure and oedema, had a significantly lower prevalence of Alzheimer's compared to those not taking the drug.
Bumetanide's potential was discovered by analysing data on more than 1,300 FDA-approved drugs derived from brain tissue samples, laboratory experiments involving animal and human cells, and human population studies.
The research has focused on Alzheimer's associated with an apolipoprotein E4 (APOE4) gene variant known to be a major risk factor for late-onset forms of the disease.
The scientists looked at brain samples to identify gene expression signatures associated with APOE4-specific Alzheimer's, and then compared those to the signatures of the drugs. All told, five drugs emerged with a gene expression signature that the researchers believed might help neutralise the disease, but the strongest candidate was bumetanide.
According to the study, which is published in Nature Aging, treatment with bumetanide "rescued electrophysiological, pathological or cognitive deficits" in animal and human cell models of APOE4 Alzheimer's.
Meanwhile, bumetanide exposure was associated with a 35% to 75% lower Alzheimer's prevalence in individuals over the age of 65 years in two electronic health record databases, suggesting bumetanide could play a role in preventing the disease.
"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," according to Richard Hodes, director of the National Institute on Aging (NIA).
The results are strong enough to back a proof-of-concept study in people with genetic risk of Alzheimer's according to the researchers.
It is the second time in a matter of weeks that data-mining studies have suggested that an already-approved drug could be repurposed for Alzheimer's disease.
A Canadian study recently suggested that Pfizer's cancer drug Inlyta (axitinib) could be repurposed for Alzheimer's, based on the hypothesis that it could interrupt the formation of abnormal blood vessels in the brains of people with the disease.