Nilotinib hydrochloride, an Abelson tyrosine kinase inhibitor used in the treatment of chronic myeloid leukemia, has been in the neurology news since the results of the open-label, phase 1 study in a small group of patients with advanced Parkinson disease (PD) and dementia with Lewy bodies was reported at the Society for Neuroscience in Chicago in October 2015. The extensive media coverage that followed, supported by videos of nilotinib-treated participants getting up and walking from their wheelchairs, suggested that disease modification in PD was within reach with this drug. The corresponding publication documented that, at 150 and 300 mg/d, doses lower than required for treating leukemia, nilotinib was detectable in the cerebrospinal fluid (CSF), increased CSF homovanillic acid levels, seemed to engage the target Abelson, and was relatively safe and well tolerated at 24 weeks.¹ Although the authors emphasized that “motor and cognitive outcomes suggest a possible beneficial effect on clinical outcomes,”^1(p503) the accompanying editorial noted that, owing to the small sample size and lack of a control group, it was impossible to rule out a placebo effect.²

尼罗替尼盐酸盐是一种用于治疗慢性粒细胞白血病的 Abelson 酪氨酸激酶抑制剂，自从在一小群晚期帕金森病 (PD) 和痴呆症患者中进行的开放标签 1 期研究的结果以来，它一直出现在神经病学新闻中2015 年 10 月，芝加哥神经科学学会报道了路易体。随后的广泛媒体报道，以及尼罗替尼治疗的参与者从轮椅上起身和行走的视频的支持，表明 PD 的疾病改变是触手可及的药品。相应的出版物记载，在 150 和 300 mg/d 的剂量下，低于治疗白血病所需的剂量，可在脑脊液 (CSF) 中检测到尼罗替尼，增加 CSF 高香草酸水平，似乎与目标 Abelson 接触，¹尽管作者强调“运动和认知结果表明可能对临床结果产生有益影响” ^{1 (p503)}，但随附的社论指出，由于样本量小且缺乏对照组，因此无法排除安慰剂效应。²