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Cancer Drug May Potentially Treat Parkinson's, Alzheimer's and Other Dementias

Update Date: May 10, 2013 12:58 PM EDT
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New findings suggest that small doses of a leukemia drug may help prevent neurodegenerative conditions like Parkinson's disease, Alzheimer's disease and others forms of dementia.

Scientists found that tiny doses of the drug nilotinib, which is used to treat chronic myelogenous leukemia (CML), can stop the abnormal protein build-up in the brains of mice, according to a new study published Friday in Human Molecular Genetics.

The study targeted the alpha-Synuclein and tau proteins, which have been linked to the development of neurodegenerative diseases like Parkinson's disease, Alzheimer's disease, amyotrophic lateral sclerosis (ALS), frontotemporal dementia, Huntington disease and Lewy body dementia.

Lead researcher Dr. Charbel E-H Moussa, head of the neuroscience department at Georgetown University, told Fox News that in neurodegenerative diseases, these proteins accumulate and kill the cell.

"The best strategy to make the cell survive is to clean the debris," Moussa said, according to Fox News.

"This drug, in very low doses, turns on the garbage disposal machinery inside neurons to clear toxic proteins from the cell," he said in a news release.

"By clearing intracellular proteins, the drug prevents their accumulation in pathological inclusions called Lewy bodies and/or tangles, and also prevents amyloid secretion into the extracellular space between neurons, so proteins do not form toxic clumps or plaques in the brain," Moussa explained.

Researchers say that when nilotinib is used to treat CML, the drug forces cancer cells into autophagy - a biological process that leads to death of tumor cells in cancer. For the study, Moussa and his team wanted to see if nilotinib could also be used to treat patients with neurodegenerative diseases. Initial experiments revealed that the leukemia drug could cross the blood-brain barrier, the separation that keeps may materials in the blood from entering the brain.

Later researchers found that mice treated with nilotinib experienced an improvement in cognitive and motor functions compared with mice treated with a placebo.  Researchers added that mice treated with the leukemia drug also lived longer.

"We know this drug enters the brain, and this is the first drug that we know reduces alpha-Synuclein and tau at the same time," Moussa told Fox News. "And when we decreased the protein build-up, we saw behavioral improvement in these mice."

Moussa and his team now hope to test the drug in patients suffering conditions linked to a build-up of alpha-Synuclein like Lewy body dementia, Parkinson's disease and Alzheimer's disease.

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