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

•Twelve patients with chronic cerebellar ataxia received daily nilotinib therapy.•Patients were nonresponsive to other treatment options (baseline mRS scores of > 2).•At follow-up, favorable mRS scores (≤ 2) were found in 6/12 (50.0%) patients.•No severe adverse event was reported.•Atrophy in cerebellar vermis was negatively associated with favorable outcomes.

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: 150-300 mg) 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.

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