2020 Volume 37 Issue 3 Pages 401-405
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.
