Alzheimer's Breakthrough

Cancer drug shows promise in reversing symptoms in mice.

Neuroscientists at Case Western Reserve University School of Medicine have made a dramatic breakthrough in their efforts to find better treatments for Alzheimer's disease. The researchers' findings, published in the journal Science, show that use of a drug currently prescribed to treat cancer appears to reverse Alzheimer's symptoms in mice. The results point to the significant potential that the medication, bexarotene, has to help the roughly 5.4 million Americans with the progressive brain disease.

"When used in mice, the drug was successful in removing the buildup of amyloid plaque in the brain-a pathological hallmark of Alzheimer's disease-as well as reversing cognitive symptoms and memory deficits.

Bexarotene even helped restore lost nesting behaviors in mice with Alzheimer's disease-within 72 hours of treatment. The drug also improved the ability of the mice to sense and respond to odors.

"This is an unprecedented finding," says Paige Cramer, PhD candidate and first author of the study. "Previously, the best existing treatment for Alzheimer's disease in mice required several months to reduce plaque in the brain."

Bexarotene has been approved for the treatment of cancer by the U.S. Food and Drug Administration for more than a decade. It has a good safety and side-effect profile, which researchers hope will help speed the transition to clinical trials of the drug.

"This is a particularly exciting and rewarding study because of the new science we have discovered and the potential promise of a therapy for Alzheimer's disease," says Case Western Reserve neuroscientist and senior author of the study Gary Landreth, PhD. "We need to be clear; the drug works quite well in mouse models of the disease. Our next objective is to ascertain if it acts similarly in humans. We are at an early state in translating this basic science discovery into a treatment."