Contemplative senior man at home
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A drug made to treat cancer could be a viable treatment for neurodegenerative diseases such as Alzheimer’s disease, a new study finds.

The report was published Thursday in Science

When researchers blocked an enzyme called indoleamine-2,3-dioxygenase 1 (IDO1), it was able to rescue memory and brain function in models that mimic Alzheimer’s disease. 

IDO1 inhibitors that are currently being created to treat cancers like melanoma, leukemia and breast cancer could be repurposed to treat the neurodegenerative diseases in their early stages. 

“We’re showing that there is high potential for IDO1 inhibitors, which are already within the repertoire of drugs being developed for cancer treatments, to target and treat Alzheimer’s,” Melanie McReynolds, PhD, an author and researcher at Penn State, said in a statement. “In the broader context of aging, neurological decline is one of the biggest co-factors of being unable to age healthier. The benefits of understanding and treating metabolic decline in neurological disorders will impact not just those who are diagnosed, but our families, our society, our entire economy.”

Existing treatments for Alzheimer’s disease center on managing symptoms and slowing progression by targeting amyloid and tau plaque build-up in the brain. Still, there aren’t any approved treatments for combating the onset of the disease, McReynolds said.

The team used in vitro cellular models with amyloid and tau proteins, in vivo mouse models and in vitro human cells from Alzheimer’s patients to conduct their research. In the trial, they found that stopping IDO1 can restore healthy glucose metabolism in brain cells that provide metabolic support to neurons.

“Scientists have been targeting the downstream effects of what we identify as an issue with the way the brain powers itself,” Praveena Prasad, a doctoral student at Penn State and co-author on the paper, said in the statement. “The therapies that are currently available are working to remove peptides that are likely the result of a bigger issue we can target before those peptides can start forming plaques. We’re demonstrating that by targeting the brain’s metabolism, we can not only slow, but reverse the progression of this disease.”