An HIV Drug to Treat Alzheimer’s? Researchers See Promising Results in Mice

As research into a cure — or at least treatment — for Alzheimer’s continues, scientists are looking beyond the pathways of targeting tau or beta-amyloid protein. Recent research has turned to an HIV/AIDs drug that shows potential in treating Alzheimer’s.

The study, published in the Journal of Medicinal Chemistry, was conducted by researchers at Case Western Reserve University School of Medicine in Cleveland, Ohio.

The researchers focused on an anti-AIDS drug called efavirenz (EFV), an antiretroviral medication. When EFV was modified, the researchers were able to boost its ability to spur a cholesterol-removing enzyme in the brain.

Cholesterol, a waxy, fatty substance that’s most often associated with clogged arteries and heart disease, can also cause problems in the brain. Past research has found a link between cholesterol and beta-amyloid buildup in the brain, suggesting that cholesterol helps beta-amyloid stick together.

Irina Pikuleva is professor of Pharmacology and Visual Sciences at Case Western Reserve University. As part of the research looking into the HIV/AIDs drug for Alzheimer’s, she honed in on an enzyme called CYP46A1, which works to remove cholesterol from the brain.

Pikuleva discovered a way to activate CYP46Aq in mice if they were given low doses of the HIV/AIDs drug, efavirenz. During her work at Case, Pikuleva began a study that tested small doses of efavirenz on genetically-engineered mice that were developing amyloid plaques on the brain.

When the researchers gave the mice drinking water with efavirenz, the mice developed fewer amyloid plaques in the brain than mice who didn’t take the drug.

It’s well-established that amyloid plaques, or tangles of a toxic protein that accumulate on the brain, are associated with neurodegeneration and Alzheimer’s disease. But there are countless ways amyloid may develop, and researchers are busy investigating different channels.

Recently, researchers have honed in on inflammation as a main driver of amyloid. They’ve found that inflammation actually triggers the buildup of amyloid protein.

Other researchers have been examining how HIV/AIDs drugs, or even diabetes and organ transplant drugs, may aid in battling Alzheimer’s. One current Phase 1 clinical trial out of Brown University will investigate whether an HIV/AIDs medication can lower inflammation in the brain as a therapy for Alzheimer’s.