A clinical trial, published in JAMA Neurology, has found that Nilotinib, a drug used for the treatment of leukemia, has been found safe and effective in people with Parkinson’s disease.
The main purpose of the clinical trial was to assess the safety, tolerability and efficacy of the drug. Also, the study wanted to determine how Nilotinib behaves in the body of the people with moderate to severe Parkinson’s disease.
Researchers from Georgetown University Medical Center (GUMC) in Washington DC investigated the impact of the drug on certain substances that could be useful biomarkers for tracking the progression of Parkinson’s.
The biomarkers include two proteins – alpha-synuclein and tau – and products of dopamine metabolism, which can be measured in the cerebrospinal fluid (CSF).
Senior study author Dr. Charbel Moussa said, “Determining the safety of nilotinib in people with Parkinson’s was our primary objective.”
In Parkinson’s disease, the brain cells that produce a neurotransmitter called dopamine, which helps in controlling the movements, stop working and die, giving rise to motor and non-motor symptoms.
Motor symptoms of Parkinson’s include tremors, sluggishness, stiffness, and balance difficulties, while non-motor symptoms include poor memory, depression, mood changes, and constipation.
In the United States, more than 60,000 people are diagnosed with the neurological condition every year, according to the Parkinson’s Foundation. Over 1 million Americans are living with the condition.
The U.S. Food and Drug Administration (FDA) has approved nilotinib for the treatment of myeloid leukemia in children.
In animal studies, low doses of nilotinib entered the brain and lowered alpha-synuclein and tau proteins, showing promising results that it could be used for treating Parkinson’s.
Another study found “that nilotinib may increase dopamine metabolism and potentially treat motor and non-motor symptoms of Parkinson’s disease.”
In the current trial, the researchers found that the doses of 150 mg and 300 mg of nilotinib “were reasonably safe.” However, some participants experienced severe side effects. Dr. Moussa noted, “Our study shows that at these lower doses, nilotinib does not seem to cause Abl inhibition, suggesting it shouldn’t have the same safety concerns that are potentially associated with Abl inhibition as might be the case at higher doses.”
