The US Food and Drug Administration claims that a drug used to treat leukemia has the potential of curing acute diseases like dementia and Parkinson's. Nearly one million Americans and up to 10 million people worldwide have Parkinson's, according to the Parkinson's Disease Foundation.
Parkinson's disease is the second most common neurodegenerative disorder that causes a range of motor and non-motor symptoms. It is a chronic, progressive movement disorder that causes tremors, stiffness in the limbs, slowed movement and problems with balance and coordination. Most people also have symptoms that are unrelated to movement, according to the foundation. For some, those include memory issues, fuzzy thinking or even full-blown dementia. No one is sure exactly what causes Parkinson's, but it involves the death of certain brain cells, including ones that produce dopamine, which help regulate movement. During the course of the disease, dopamine producing neurons are lost and bundles of proteins known as Lewy Bodies form in the brain. For the past 50 years, Parkinson's disease has been treated using levodopa, a drug that boosts brain dopamine levels.
‘Leukemia drug nilotinib may restore brain dopamine and reduce toxic proteins associated with Lewy Bodies formation associated with neurodegenerative conditions like Parkinson's disease and dementia patients.’
The researchers conducted a small phase one study that included only 12 patients who were asked to take low doses of nilotinib everyday for six months, primarily intended to evaluate whether patients could tolerate the drug. The results showed unanticipated improvements in clinical outcomes and motor function. The findings were reported in the Journal of Parkinson's Disease
The researchers found that the leukemia drug nilotinib that belongs to a group of cancer agents called tyrosine kinase inhibitors, which block certain proteins that fuel cancer growth may also help restore brain dopamine and reduce toxic proteins associated with Lewy Bodies formation in patients with Parkinson's disease and dementia. The patients typically showed some improvement in both physical symptoms and problems with memory and thinking, the findings showed.
Lead investigator Charbel Moussa an assistant professor of neurology at Georgetown University Medical Center (GUMC) in Washington, D.C. explained, "This is the first study to treat subjects with advanced Parkinson's disease 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 Parkinson's disease or dementia with Lewy Bodies (DLB), and although the data are supportive of additional trials, caution must be used in any future studies." Moussa added "We have to be very cautious about the safety profile of this drug in Parkinson's patients."
James Beck, vice president of scientific affairs for the Parkinson's Disease Foundation shared the same views about the trial still being in its early stage. One of the limitations was that the study included only people who were in advanced stages of Parkinson's and had some degree of dementia, including memory loss and thinking problems. Another one was that the study was designed only as an early 'proof of concept' and lacked the rigors that are used in later-stage trials. There was no comparison group given a placebo (inactive treatment) and all of the study patients (and researchers) knew they were on the drug. Another drawback associated with nilotinib is that it carries a boxed warning about potentially risky changes in heart rhythm.
Moussa acknowledged those limitations and said his team is planning two trials that will put nilotinib to a tougher test where patients will be randomly assigned to take either the drug or a placebo. One study will include patients with moderate-stage Parkinson's. The other will focus on people with mild to moderate Alzheimer's disease, since there is evidence the drug could affect dementia symptoms.
The study was supported by government and philanthropic funds.