神経治療学
Online ISSN : 2189-7824
Print ISSN : 0916-8443
ISSN-L : 2189-7824
シンポジウム6:神経難病におけるトランスレーショナル・リサーチ–2
筋萎縮性側索硬化症(ALS)に対する肝細胞増殖因子(HGF)治療
青木 正志割田 仁加藤 昌昭長野 清一望月 秀樹
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2020 年 37 巻 3 号 p. 401-405

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Amyotrophic lateral sclerosis (ALS) is an adult–onset, devastating neurodegenerative disease characterized by systemic loss of motor neurons in the central nervous system. Using a transgenic rat model overexpressing ALS–linked mutant Cu/Zn superoxide dismutase gene, which is the most frequent causative gene in Japanese ALS, we reported a dose–dependent neuroprotective effect of recombinant human hepatocyte growth factor (rhHGF) by intrathecal infusion, even with administration from onset of the motor neuron disease (Ishigaki A et al., J Neuropathol Exp Neurol 2007). Based on the pharmacokinetic and safety data in our phase I/first–in–human trial (Warita H et al., J Clin Pharmacol, 2018), we are going to accomplish the novel phase II proof–of–concept (POC) study of intrathecal rhHGF administration on ALS in the present study. In other CNS degenerative diseases, possible application of the intrathecal rhHGF delivery might be warranted.

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