Development of a Novel Therapy for Amyotrophic Lateral Sclerosis (ALS) Using Hepatocyte Growth Factor (HGF)
update：2020-06-16
Features
Amyotrophic lateral sclerosis (ALS) is an adult-onset neurodegenerative disease characterized by systematic motor neuron degeneration. Approximately 20% cases of familial ALS are caused by mutations in the superoxide dismutase 1 (SOD1) gene. We developed transgenic rats that express a human SOD1 gene with two different ALS-linked mutations (G93A or H46R) showing progressive motor neuron degeneration and paralysis. The larger size of the rat ALS models as compared with existing mouse models will facilitate studies on neuroprotective and neuro-regenerative strategy involving manipulations of cerebrospinal fluid and spinal cord.Targeted Application(s)/Industry
Hepatocyte growth factor (HGF) is one of the most potent survival-promoting factors for motor neurons. Continuous intrathecal infusion of human recombinant HGF (hrHGF) attenuated loss of spinal motor neurons, astrocytosis, and microglial activation, leading to prolonged survival in the ALS rats. Safety and toxicology testing of the hrHGF protein in non-human primates should prompt further clinical trials in human ALS patients.Researchers
Department of Neurology, Graduate School of Medicine
AOKI Masashi
, Professor
Doctor of Medicine
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