Nilotinib hydrochloride, an Abelson tyrosine kinase inhibitor used in the treatment of chronic myeloid leukemia, has been in the neurology news since the results of the open-label, phase 1 study in a small group of patients with advanced Parkinson disease (PD) and dementia with Lewy bodies was reported at the Society for Neuroscience in Chicago in October 2015. The extensive media coverage that followed, supported by videos of nilotinib-treated participants getting up and walking from their wheelchairs, suggested that disease modification in PD was within reach with this drug. The corresponding publication documented that, at 150 and 300 mg/d, doses lower than required for treating leukemia, nilotinib was detectable in the cerebrospinal fluid (CSF), increased CSF homovanillic acid levels, seemed to engage the target Abelson, and was relatively safe and well tolerated at 24 weeks.1 Although the authors emphasized that “motor and cognitive outcomes suggest a possible beneficial effect on clinical outcomes,”1(p503) the accompanying editorial noted that, owing to the small sample size and lack of a control group, it was impossible to rule out a placebo effect.2
Identify all potential conflicts of interest that might be relevant to your comment.
Conflicts of interest comprise financial interests, activities, and relationships within the past 3 years including but not limited to employment, affiliation, grants or funding, consultancies, honoraria or payment, speaker's bureaus, stock ownership or options, expert testimony, royalties, donation of medical equipment, or patents planned, pending, or issued.
Err on the side of full disclosure.
If you have no conflicts of interest, check "No potential conflicts of interest" in the box below. The information will be posted with your response.
Not all submitted comments are published. Please see our commenting policy for details.
Espay AJ, Hauser RA, Armstrong MJ. The Narrowing Path for Nilotinib and Other Potential Disease-Modifying Therapies for Parkinson Disease. JAMA Neurol. 2020;77(3):295–297. doi:10.1001/jamaneurol.2019.3983
Customize your JAMA Network experience by selecting one or more topics from the list below.
Create a personal account or sign in to: