Leukemia drug shows promise in fight against dementia, Parkinson's

Georgetown University Medical Center found that the drug nilotinib, approved to treat a form of leukemia, appeared to help a small group of people with Parkinson’s disease and a related form of dementia.

This preliminary study back in 2015 boosted excitement in the scientific community but because it was a small trial with no placebo control, J.Paul Taylor, chair of the cell and molecular biology department at St. Jude Children’s Research Hospital in Memphis said that it really wasn’t designed to assess efficacy.

Now backed with input from the Food and Drug Administration, Georgetown is launching two larger trials of nilotinib. The Parkinson’s disease trial will enroll 75 patients and the Alzheimer’s trial, 42 patients.

“Nilotinib seems to work by eliminating toxic proteins that build up in the brains of people with Parkinson's and Alzheimer's. The drug activates a mechanism in brain cells that acts like a sort of garbage disposal,” said Fernando Pagan, medical director of the translational neurotherapeutics program at Georgetown.

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