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Repurposed Cancer Drug Restores Brain Function in Mouse Models of Alzheimer’s Disease

By Meg Bouvier

I stay current on NIH happenings and I’d be delighted to keep you informed.

Recently, a team of researchers at the Yale University School of Medicine discovered that a drug originally developed for the use of cancer therapy could be used to treat Alzheimer’s disease (March 21, 2015 Annals of Neurology; click here for NIH story). The drug is called saracatinib, originally developed by the biopharmaceutical company AstraZeneca. However, through the NIH-funded initiative to repurpose experimental drugs, the research team discovered that the drug restores memory loss and reverses brain problems in mouse models of Alzheimer’s disease.

Nearly 5 million Americans have Alzheimer’s disease, the most common form of dementia. When a person develops Alzheimer’s disease, clumps of amyloid beta protein build up in the brain, damaging brain cells and synapses. Current Alzheimer’s treatments only ease the symptoms of the disease. When saracatinib was given to mice with symptoms similar to Alzheimer’s patients, mice showed a complete reversal of spatial learning and memory loss after four weeks. The researchers found that the drug actually restored the synapses.

Normally it takes around ten years for an experimental drug to be approved for a Phase 2a human clinical trial. This process took the Yale research team only 18 months. “The investigational drug already had been developed, optimized and studied in animals as well as tested for safety in humans, so our ability to obtain this asset through NCATS and AstraZeneca gave us an incredible shortcut in the drug development process,” explained Strittmatter, the senior author on the study. This discovery serves as a testament to the importance of collaboration and knowledge sharing across groups. The human trial will involve 152 subjects, who will receive either saracatinib or a placebo for one year. The researchers expect to have results in the next two years.

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