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J Neuroimmunol. 2017 Aug 15;309:82-87. doi: 10.1016/j.jneuroim.2017.05.015. Epub 2017 May 24.

The c-Abl inhibitor, nilotinib, as a potential therapeutic agent for chronic cerebellar ataxia.

Author information

1
Department of Neurology, Comprehensive Epilepsy Center, Laboratory for Neurotherapeutics, Biomedical Research Institute, Seoul National University Hospital, Seoul, South Korea.
2
Department of Neurology, Comprehensive Epilepsy Center, Laboratory for Neurotherapeutics, Biomedical Research Institute, Seoul National University Hospital, Seoul, South Korea; Program in Neuroscience, Neuroscience Research Institute of SNUMRC, College of Medicine, Seoul National University, Seoul, South Korea.
3
Department of Radiology, Seoul National University Hospital, Seoul, South Korea.
4
Program in Neuroscience, Neuroscience Research Institute of SNUMRC, College of Medicine, Seoul National University, Seoul, South Korea; Department of Neurology, Seoul National University Healthcare System Gangnam Center, Seoul, South Korea.
5
Department of Neurology, Comprehensive Epilepsy Center, Laboratory for Neurotherapeutics, Biomedical Research Institute, Seoul National University Hospital, Seoul, South Korea; Program in Neuroscience, Neuroscience Research Institute of SNUMRC, College of Medicine, Seoul National University, Seoul, South Korea; Protein Metabolism Research Center, Seoul National University College of Medicine, Seoul, South Korea.
6
Department of Neurology, Comprehensive Epilepsy Center, Laboratory for Neurotherapeutics, Biomedical Research Institute, Seoul National University Hospital, Seoul, South Korea; Program in Neuroscience, Neuroscience Research Institute of SNUMRC, College of Medicine, Seoul National University, Seoul, South Korea. Electronic address: stemcell.snu@gmail.com.

Abstract

Nilotinib is a potent inhibitor of tyrosine kinase BCR-ABL that penetrates the blood-brain barrier. To evaluate the effect of nilotinib in chronic cerebellar ataxia, twelve patients with chronic cerebellar ataxia nonresponsive to other treatment options (modified Rankin scale [mRS] scores: >2) and received nilotinib therapy (daily doses: 150-300mg) for >4 (range 5-16) weeks were reviewed. At follow-up, improved mRS scores were found in 7/12 (58.3%) patients and favorable mRS scores (≤2) were found in 6/12 (50.0%) patients. No severe adverse event was observed. Atrophy in the cerebellar vermis appeared to be negatively associated with favorable outcomes.

KEYWORDS:

Cerebellar ataxia; Cerebellar atrophy; Nilotinib; Outcome; Treatment

PMID:
28601294
DOI:
10.1016/j.jneuroim.2017.05.015
[Indexed for MEDLINE]

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