A recently published study from researchers at the Cleveland Clinic has found that sildenafil, an approved drug for erectile dysfunction, popularly known as Viagra, can help in treating Alzheimer’s disease.
The neurodegenerative disease that currently does not have a cure is estimated to impact 13.8 million Americans by 2050, the press release said. With new drug development being a time and cost-intensive process, researchers in the pharma industry have turned to repurpose already approved drugs. The team led by Feixiong Cheng, at Genomic Medicine Institute at Cleveland, was studying subtypes of neurodegenerative diseases to find common mechanisms that could be targeted with FDA-approved drugs.
In the case of Alzheimer’s disease, two proteins, namely beta-amyloid and tau, begin accumulating inside the brain, leading to clots and tangles. Clinical trials targeting these proteins have failed in the past decade, the press release said. Recent research has also shown that the two proteins that work together cause the disease. The researchers began investigating which of the approved drugs could act on both the proteins at the same time instead of targeting just one.
To their surprise, sildenafil turned up to have the best scores. The database that the researchers used consisted of data of more than seven million people in the U.S. and even included drugs such as losartan or metformin, that are currently under active clinical trials to treat Alzheimer’s. After six years of follow-up, sildenafil users were 69 percent less likely to develop the neurodegenerative disease, the press release said.
“We found that sildenafil use reduced the likelihood of Alzheimer’s in individuals with coronary artery disease, hypertension, and type 2 diabetes, all of which are comorbidities significantly associated with risk of the disease,” added Dr. Cheng.
To further verify their findings, the team created model cells of Alzheimer’s disease in the lab using stem cells and treated them with sildenafil. The drug not only increased brain cell growth but also decreased hyperphosphorylation of tau proteins, a process that usually leads to tangles.
The research team will now carry out a phase II randomized clinical trial to confirm their findings, following which the approach will be applied to other neurodegenerative diseases like Parkinson’s or Amyotrophic Lateral Sclerosis (ALS).
“This is an example of a growing area of research in precision medicine where big data is key to connecting the dots between existing drugs and complex diseases,” said Jean Yuan, M.D., Ph.D., at the National Institute on Aging (NIA), which funded the work. The study was published in Nature Aging.
A trial for a nasal vaccine for Alzheimer’s is also scheduled to begin soon.
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