An FDA-approved cancer drug improved quality of life for patients with Parkinson’s disease and Lewy body dementia.
The drug, nilotinib (Tasigna), was originally created as treatment for chronic myelogenous leukemia (CML). When given in high enough doses, it forces cancer cells into autophagy, a biological process leading to the death of tumor cells.
However, when given in smaller doses, nilotinib penetrates the blood-brain barrier and triggers neurons to clear toxic intracellular proteins from brain cells. This leads to statistically significant changes in toxic proteins linked to disease progression in Parkinson’s and Lewy body dementia, as well as dramatically improved cognition, motor skills, and non-motor function (such as constipation).
“To my knowledge, this study represents the first time a therapy appears to reverse — to a greater or lesser degree depending on stage of disease — cognitive and motor decline in patients with these neurodegenerative disorders,” Fernando Pagan, MD, director of the Movement Disorders Program at MedStar Georgetown University Hospital, Washington, D.C., said in a statement.
For this small phase I clinical trial, the researchers tested the safety of nilotinib by giving escalating doses (150 to 300 mg daily) to 12 study participants for 6 months. In all 11 participants who completed the trial, nilotinib produced benefit, with 10 participants reporting meaningful clinical improvements. The doses were much lower than the dose given to cancer patients (800 mg daily) and were well tolerated with no serious side effects.
These results should be viewed with caution, as there was no control group for comparison. “It is critical to conduct larger and more comprehensive studies before determining the drug's true impact,” Pagan said.
An FDA-approved drug for leukemia improved cognition, motor skills and non-motor function in patients with Parkinson’s disease and Lewy body dementia in a small phase I clinical trial, report researchers at Georgetown University Medical Center (GUMC) in Washington. In addition, the drug, nilotinib (Tasigna), led to statistically significant and encouraging changes in toxic proteins linked to disease progression (biomarkers).
Complete data will be presented at Neuroscience 2015, the annual meeting of the Society for Neuroscience, in Chicago on Oct. 17.
Charbel Moussa, MD, PhD, who directs Georgetown’s Laboratory of Dementia and Parkinsonism, conducted the preclinical research that led to the discovery of nilotinib for the treatment of neurodegenerative diseases.