Exp Neurobiol.  2014 Sep;23(3):207-214. 10.5607/en.2014.23.3.207.

Amyotrophic Lateral Sclerosis - Cell Based Therapy and Novel Therapeutic Development

Affiliations
  • 1Department of Bioscience and Biotechnology, Sejong University, Seoul 143-747, Korea. changkim@sejong.ac.kr
  • 2Department of Neurology, Seoul National University Hospital, Seoul 110-774, Korea. jjsaint@snu.ac.kr

Abstract

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease, characterized by the predominant loss of motor neurons (MNs) in primary motor cortex, the brainstem, and the spinal cord, causing premature death in most cases. Minimal delay of pathological development by available medicine has prompted the search for novel therapeutic treatments to cure ALS. Cell-based therapy has been proposed as an ultimate source for regeneration of MNs. Recent completion of non-autologous fetal spinal stem cell transplant to ALS patients brought renewed hope for further human trials to cure the disease. Autologous somatic stem cell-based human trials are now in track to reveal the outcome of the ongoing trials. Furthermore, induced pluripotent stem cell (iPSC)-based ALS disease drug screen and autologous cell transplant options will broaden therapeutic options. In this review paper, we discuss recent accomplishments in cell transplant treatment for ALS and future options with iPSC technology.

Keyword

ALS; cell transplant; stem cell; iPSC; drug screen; neural stem cell

MeSH Terms

Amyotrophic Lateral Sclerosis*
Brain Stem
Hope
Humans
Mortality, Premature
Motor Cortex
Motor Neurons
Neural Stem Cells
Neurodegenerative Diseases
Pluripotent Stem Cells
Regeneration
Spinal Cord
Stem Cells
Transplants
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