Leukemia drug may treat Parkinson's disease, dementia

Washington : A drug approved by the US Food and Drug Administration to treat leukemia has the potential to improve motor and cognitive functions of patients suffering from Parkinson's disease and dementia, a small study has found.

Parkinson's disease (PD) is the second most common neurodegenerative disorder that causes a range of motor and non-motor symptoms.
During the course of the disease, dopamine (DA)-producing neurons are lost and bundles of proteins known as Lewy Bodies (LBs) form in the brain.

The researchers found that the leukemia drug nilotinib may restore brain dopamine and reduce toxic proteins associated with Lewy Bodies formation in Parkinson's disease and dementia patients.

The findings were reported in the Journal of Parkinson's Disease.

The researchers conducted a small phase one study that included only 12 patients, primarily intended to evaluate whether patients could tolerate the drug.

The results showed unanticipated improvements in clinical outcomes and motor function.

"This is the first study to treat subjects with advanced PD with a tyrosine kinase inhibitor,"

"This study suggests that low doses of nilotinib appear to be relatively safe in a small cohort of participants with advanced PD or dementia with Lewy Bodies (DLB), and although the data are supportive of additional trials, caution must be used in any future studies," explained lead investigator Charbel Moussa from Georgetown University Medical Center (GUMC) in Washington, D.C.

"The data give a clear signal to move forward with more definitive trials to determine an appropriate therapeutic dose and evaluate nilotinib effects in larger, randomized, double-blinded, placebo-controlled trials," Moussa noted.