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Comment
. 2016 Jul 12;6(3):519-22.
doi: 10.3233/JPD-160904.

Nilotinib - Differentiating the Hope from the Hype

Richard K Wyse  1 , Patrik Brundin  2 , Todd B Sherer  3
Affiliations
Free PMC article
Comment

Nilotinib - Differentiating the Hope from the Hype

Richard K Wyse et al. J Parkinsons Dis. .
Free PMC article

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.

Keywords: Drug repositioning; Nilotinib; neuroprotection.

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Comment on

  • Nilotinib Effects in Parkinson's disease and Dementia with Lewy bodies.
    Pagan F, Hebron M, Valadez EH, Torres-Yaghi Y, Huang X, Mills RR, Wilmarth BM, Howard H, Dunn C, Carlson A, Lawler A, Rogers SL, Falconer RA, Ahn J, Li Z, Moussa C. Pagan F, et al. J Parkinsons Dis. 2016 Jul 11;6(3):503-17. doi: 10.3233/JPD-160867. J Parkinsons Dis. 2016. PMID: 27434297 Free PMC article. Clinical Trial.

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