Tiny doses of a drug used for leukemia has halted toxic brain proteins linked to Parkinson’s disease in a new Georgetown University Medical Center study using animal models.
The senior investigator of the study is planning a clinical trial in humans to study the effects of the drug, called nilotinib.
The study, recently published online in Human Molecular Genetics, provides a novel strategy in treating neurodegenerative diseases that feature abnormal buildup of such proteins involved in Parkinson’s, Alzheimer’s disease, amyotrophic lateral sclerosis (ALS), frontotemporal dementia, Huntington disease and Lewy body dementia, among others.
“This drug, in very low doses, turns on the garbage disposal machinery inside neurons to clear toxic proteins from the cell,” says the study’s senior investigator, neuroscientist Charbel E-H Moussa. “By clearing intracellular proteins, the drug prevents their accumulation in pathological inclusions called Lewy bodies and/or tangles, and also prevents protein secretion into the extracellular space between neurons, so proteins do not form toxic clumps or plaques in the brain.”