title: Nilotinib - Differentiating the Hope from the Hype.
abstract: We discuss a report in the current issue on clinical and biochemical findings from a safety trial using the cAbl tyrosine kinase inhibitor Nilotinib (150 mg or 300 mg given daily for 24 weeks) in a small group of patients with either advanced Parkinson's disease or Dementia with Lewy Bodies. Despite some side effects (one serious), the authors claim that Nilotinib, which is normally used at much higher doses for treating leukemia, is safe and tolerated. Furthermore, they report a possible benefit on motor and cognitive outcomes. We debate the safety of Nilotinib and the reported efficacy signals. We emphasize that due to the small sample size, and lack of a control group, it is impossible to rule out a placebo effect. We briefly discuss a range of aspects surrounding the current and possible future use of this cAbl inhibitor in patients with alpha-synucleinopathy, and what must now be done to obtain definitive information about its safety and efficacy in this population of patients.
authors.affiliation: The Cure Parkinson's Trust, London, UK.
authors.forename: Richard K
authors.initials: RK
authors.lastname: Wyse
authors.affiliation: Translational Parkinson's Disease Research, Center for Neurodegenerative Science, Van Andel Research Institute, Grand Rapids, MI, USA.
authors.forename: Patrik
authors.initials: P
authors.lastname: Brundin
authors.affiliation: Michael J Fox Foundation for Parkinson's Research, New York, USA.
authors.forename: Todd B
authors.initials: TB
authors.lastname: Sherer
mesh_terms: D003704:Dementia; D006801:Humans; D020961:Lewy Body Disease; D010300:Parkinson Disease; D011743:Pyrimidines
chemical_list: D011743:Pyrimidines; C498826:nilotinib
keywords: Drug repositioning; Nilotinib; neuroprotection