Leukemia drug improved symptoms among patients with Parkinson’s

Individuals with Parkinson’s disease and Lewy body dementia had improved cognition, motor skills and nonmotor functions, as well as significant changes in toxic proteins linked to disease progression, after receiving Tasigna, an FDA-approved drug for leukemia, according to a press release.

“The changes in Tau, p-Tau, α-synuclein and Abeta-40 and -42 in spinal fluid suggest the clearance of toxic proteins in the brain…Study participants with earlier stage disease responded best, as did those diagnosed with Lewy body dementia, often described as a combination of Parkinson’s and Alzheimer’s diseases,” study co-author Fernando Pagan, MD, associate professor of neurology at MedStar Georgetown University Hospital, said in a press release.

To assess the efficacy of Tasigna (nilotinib, Novartis) in patients with advanced Parkinson’s disease and Lewy body dementia, Pagan and colleagues randomly assigned 12 patients to 150 mg or 300 mg nilotinib once daily for 6 months.

Results demonstrated that nilotinib reversed loss of CSF alpha-Synuclein and significantly reversed Abeta-40 and -42 levels.

Significant reductions in cerebrospinal fluid total Tau and p-Tau (P < .05), as well as increases in homovanillic acid were seen in patients between baseline and 6 months.

Many patients had increased levels of dopamine, which required patients L-dopa medications and other dopamine sparing drugs to be lowered or stopped, according to a press release.

The researchers noted that the study did not include a control group, so the data should be interpreted with caution. Larger, placebo-controlled trials are needed to determine the overall safety and efficacy of nilotinib for patients with various stages of Parkinson’s disease and Lewy body dementia, according to the researchers.

“To my knowledge, this study represent 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. But it is critical to conduct larger and more comprehensive studies before determining the drug’s true impact,” Pagan said in the release.

Disclosures: The researchers report no relevant financial disclosures.