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Updated: May 14, 2026
10:49
Intraspinal Cell Transplantation for Targeting Cervical Ventral Horn in Amyotrophic Lateral Sclerosis and Traumatic Spinal Cord Injury
Published on: September 18, 2011
[Restorative therapy in amyotrophic lateral sclerosis]
1(Department of Neurology, Tohoku University School of Medicine, 1-1, Seiryo-machi, Aoba-ku, Sendai 980-8574, Japan.
Nihon Shinkei Seishin Yakurigaku Zasshi = Japanese Journal of Psychopharmacology
|February 5, 2013
Summary
Hepatocyte growth factor (HGF) treatment slowed motor neuron degeneration in a rat model of Amyotrophic Lateral Sclerosis (ALS). This neuroprotective factor also promoted functional recovery in a primate spinal cord injury model, paving the way for human clinical trials.
Area of Science:
- Neuroscience
- Genetics
- Pharmacology
Context:
- Amyotrophic Lateral Sclerosis (ALS) is a fatal neurodegenerative disease.
- Mutations in the superoxide dismutase 1 (SOD1) gene cause familial ALS.
- A novel rat model expressing human SOD1 mutations was developed for ALS research.
Purpose:
- To investigate the therapeutic potential of hepatocyte growth factor (HGF) in ALS.
- To evaluate the efficacy of intrathecal administration of recombinant HGF (hrHGF) in a transgenic rat model of ALS.
- To assess the functional recovery effects of hrHGF in a primate model of spinal cord injury.
Summary:
- Transgenic rats with ALS-associated SOD1 mutations exhibited motor neuron degeneration and paralysis.
- Intrathecal delivery of hrHGF to these rats attenuated neurodegeneration and extended disease duration by 63%.
- Intrathecal hrHGF administration promoted functional recovery in common marmosets with induced spinal cord injury.
Impact:
- This study demonstrates the neuroprotective and functional recovery potential of HGF in ALS and spinal cord injury.
- The findings support the translation of continuous intrathecal HGF administration to human clinical trials for ALS.
- The developed rat model offers a valuable platform for future ALS research and therapeutic development.
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