Korean J Clin Neurophysiol.  2015 Jun;17(1):1-16. 10.14253/kjcn.2015.17.1.1.

Update of Therapeutic Clinical Trials for Amyotrophic Lateral Sclerosis

• Kim NH
• Lee MO

• KMID: 2225214
• DOI: http://doi.org/10.14253/kjcn.2015.17.1.1

Abstract

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease that is characterized by progressive death of motor neurons in the cortex, brainstem, and spinal cord. Until now, many treatment strategies have been tested in ALS, but so far only Riluzole has shown efficacy of slightly slowing disease progression. The pathophysiological mechanisms underlying ALS are multifactorial, with a complex interaction between genetic factors and molecular pathways. Other motor neuron disease such as spinal muscular atrophy (SMA) and spinobulbar muscular atrophy (SBMA) are also progressive neurodegenerative disease with loss of motor neuron as ALS. This common thread of motor neuron loss has provided a target for the development of therapies for these motor neuron diseases. A better understanding of these pathogenic mechanisms and the potential pathological relationship between the various cellular processes have suggested novel therapeutic approaches, including stem cell and genetics-based strategies, providing hope for feasible treatment of ALS.

Keyword

Amyotrophic lateral sclerosis; Clinical trial; Treatment

MeSH Terms

Amyotrophic Lateral Sclerosis*
Brain Stem
Disease Progression
Hope
Motor Neuron Disease
Motor Neurons
Muscular Atrophy, Spinal
Muscular Disorders, Atrophic
Neurodegenerative Diseases
Riluzole
Spinal Cord
Stem Cells
Riluzole

Figure

• 

  Fig. 1. Update of therapeutic clinical trials for ALS. CoQ10; conenzyme Q10, TUDCA; tauroursodeoxycholic acid, G-CSF; granulocyte colony stimulating factor, BDNF; brain-derived neurotrophic factor, IGF-1; insulin-like growth factor-1, CNTF; ciliary neurotrophic factor, PBA; 4-phenylbutyrate, SC; stem cell.

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