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 clinical trial, say researchers at Georgetown University Medical Center (GUMC).
The drug, nilotinib (known as Tasigna® by Novartis) also led to statistically significant and encouraging changes in toxic proteins linked to disease progression. The study’s findings were presented at the annual meeting of the Society for Neuroscience, in Chicago.
The investigators report that one individual confined to a wheelchair was able to walk again and that three others who could not speak were able to hold conversations.
The researchers report that during use of the medication by the participants, production of dopamine increased in many patients, requiring doses of L-dopa and other dopamine-sparing drugs used to treat Parkinson’s to be lowered or stopped.
Stopping nilotinib treatment appears to lead to cognitive and motor decline despite reinstating L-dopa therapies.
“The use of nilotinib in doses much smaller than are used to treat cancer, which is up to 800 mg daily, was well tolerated with no serious side effects,” author explains. “The dose used in this study was 150 and 300 mg daily.”
The researchers also found that the drug penetrates the blood-brain barrier in amounts greater than dopamine drugs.
The observed efficacy in cognition, motor skills and non-motor function improvement (such as constipation) for many patients was the most dramatic result, author notes.
Alan Hoffman, a professor emeritus of social science education at Georgia State University, was diagnosed with Parkinson’s disease in 1997. He says he participated in several clinical trials with no benefit until he enrolled in Pagan’s study.
“Before the nilotinib, I did almost nothing around the house,” he says. “Now, I empty the garbage, unload the dishwasher, load the washer and the dryer, set the table, even take responsibility for grilling.”
In the three weeks prior to enrolling in the study, Hoffman says he fell eight times, but he only fell once during six months on the study. His speech has improved, as has his thinking.
“My wife says it’s life-changing for her and for my children and grandchildren,” Hoffman says. “To say that nilotinib has made a change in our lives is a huge understatement.”