LAUSR

Multiple system atrophy (MSA) is a rare, untreatable neurodegenerative disorder characterized by accumulation of α‐synuclein in oligodendroglial inclusions. As such, MSA is a synucleinopathy along with Parkinson's disease (PD) and dementia with Lewy bodies. Activation of the abelson tyrosine kinase c‐Abl leads to phosphorylation of α‐synuclein at tyrosine 39, thereby promoting its aggregation and subsequent neurodegeneration. The c‐Abl inhibitor nilotinib used for the treatment of chronic myeloid leukemia based on data collected in preclinical models of PD might interfere with pathogenic mechanisms that are relevant to PD and dementia with Lewy bodies, which motivated its assessment in an open‐label clinical trial in PD and dementia with Lewy bodies patients. The objective of this study was to assess the preclinical efficacy of nilotinib in the specific context of MSA.