Could a Common Cancer Drug Help Treat Parkinson’s Disease?

Scientists are saying, this week, they believe they may have found a way to stabilize the debilitating effects of Parkinson’s disease with a  drug that is usually prescribed to cancer patients.  

In an experimental trial at Georgetown University Medical Centre, researchers found that the cancer drug nilotinib appears to increase dopamine levels in the brain, which helps to lower the concentration of toxic proteins. Normally, this drug is used in the treatment for chronic myeloid leukemia but, in this case, also appeared to slow motor control decline in Parkison’s disease. 

Georgetown University associate professor of neurology Dr. Charbel Moussa explains, “We found that nilotinib is reasonably safe using doses 25 to 50 percent lower than the cancer dose. We also discovered that nilotinib increases the level of a brain chemical called dopamine that is lost or reduced in Parkinson’s disease.”  

The drug trial involved 75 patients at an average age of 68.4 years who have been diagnosed with moderately advanced Parkinson’s disease and have stable symptoms.  This group was randomly split into three equal cohorts of 25 patients:  a 150mg dosage group, a 300mg dosage group, and a placebo group. 

The lead study author goes on to say, “After one year of treatment, the nilotinib group was stable and did not decline on both motor and non-motor clinical scales.”

Specifically, the study found fewer than one-third of those taking the drug, at all, showed a reduction in at least two types of toxic proteins that are associated with the development of Parkinson’s disease. Also, 50 percent of these patients showed significant in increases in dopamine levels. 

Indeed, all study groups appeared to show improvement in motor testing at six months into the test.  However, those on the placebo and those at the 300mg dose remained stable at later stages of 12 and 15 months. The 150mg group simply showed improvement between the start and end of the trial (so, over several years). 

In all, then, this data suggests the drug should definitely be considered for further evaluation.  Accordingly, Dr. Moussa concludes: “These data are a green light to study nilotinib in a larger phase 3 trial to finally validate its clinical effects.”

The results of this study have been published online in the journal JAMA Neurology.