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Review Article

Exp Neurobiol 2014; 23(3): 207-214

Published online September 30, 2014

https://doi.org/10.5607/en.2014.23.3.207

© The Korean Society for Brain and Neural Sciences

Amyotrophic Lateral Sclerosis - Cell Based Therapy and Novel Therapeutic Development

Changsung Kim1*, Hee Chul Lee1 and Jung-Joon Sung2*

1Department of Bioscience and Biotechnology, Sejong University, Seoul 143-747, 2Department of Neurology, Seoul National University Hospital, Seoul 110-774, Korea

Correspondence to: *To whom correspondence should be addressed.
Changsung Kim
TEL: 82-2-3408-4485, FAX: 82-2-3408-4334
e-mail: changkim@sejong.ac.kr
Jung-Joon Sung
TEL: 82-2-3672-7553, FAX: 82-2-2072-1015
e-mail: jjsaint@snu.ac.kr

Received: August 6, 2014; Revised: August 27, 2014; Accepted: August 27, 2014

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.

Keywords: ALS, cell transplant, stem cell, iPSC, drug screen, neural stem cell