神経治療学
Online ISSN : 2189-7824
Print ISSN : 0916-8443
ISSN-L : 2189-7824
シンポジウム10:ALSに対する治験の最新情報
筋萎縮性側索硬化症に対する肝細胞増殖因子を用いた治験
長野 清一割田 仁望月 秀樹青木 正志
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2019 年 36 巻 4 号 p. 462-464

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Amyotrophic lateral sclerosis (ALS) is a lethal degenerative disease of motor neurons that results in progressive muscle weakness. At present, riluzole and edaravone have been approved in Japan, but its effect on the progression of symptoms is limited, and development of more effective treatments is desired.

Hepatocyte growth factor (HGF) is an in–vivo substance discovered by Nakamura et al., and cell proliferation promoting/cell death suppressing activity of HGF has been reported in many cells including hepatocytes. Also, neurotrophic factor–like effects of HGF have been shown in neurons, and from that point HGF has been used to study the disease–suppressing effects on ALS in Tohoku University. Human recombinant HGF (hrHGF) showed about 63% prolongation of morbidity by administration into the cerebrospinal fluid from the onset of the disease in mutant superoxide dismutase 1 (SOD1) overexpressing rats. Next, a phase I study of 15 patients with mild ALS was conducted in Tohoku University Hospital from 2011 to 2014 to confirm the safety and pharmacokinetics of intrathecal administration of hrHGF in humans. Based on this result, a placebo–controlled double–blind phase II study in 48 patients with ALS was started in Tohoku University Hospital and Osaka University Hospital from May 2016.

Patients who show a mild to moderate symptom progression after 12 weeks of pre–registration are enrolled, and after catheter implantation surgery in the spinal cord cavity, the drug is administered once every two weeks for a total of 24 weeks. Patients who desire to continue are given the active drug for up to 24 weeks. As of the end of September 2018, 22 cases have been enrolled. No serious adverse events have been seen to date.

We will further promote recruitment of candidate patients and aim to complete the clinical trial sooner. If the clear efficacy of HGF to ALS patients is confirmed in this clinical trial, ALS treatment based on a novel mechanism of action using neurotrophic factors becomes possible, and the effects on other neurodegenerative diseases are also expected.

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