Stem Cell Therapies in the Fight of Amyotrophic Lateral Sclerosis

Dear Colleagues,

Amyotrophic lateral sclerosis (ALS) is a progressive, incurable, fatal neurodegenerative disease, the pathogenesis of which still remains elusive. ALS is characterized by selective loss of upper and lower motor neurons (MNs) in the motor cortex and spinal cord, respectively, leading to progressive weakness of voluntary muscles and finally to death within 2 ± 5 years due to respiratory decline. ALS has an incidence of 1 up to 4 per 100,000 people worldwide, displaying notable differences between ethnic groups. There are two types of ALS: familial ALS (fALS) and sporadic ALS (sALS). The vast majority, 90%, of total ALS cases are sporadic, while the remaining 10% of cases are related to specific gene mutations in the C9ORF72, SOD1, NEK1, TDP43 (also known as TARDBP), FUS, and KIF5A genes. Until today, there is no cure for ALS, and the existing drugs that have been approved by the Food and Drug Administration (FDA), riluzole and edaravone, have been shown to be not effective enough against ALS, as they delay the disease and prolong the survival of ALS patients by several months only.

Stem cell therapy for fighting ALS is a hot topic in basic and clinical research. During the past years, there has been considerable excitement about stem-cell-based therapies for ALS and other neurodegenerative diseases, as well as for spinal cord injury. Stem cell approaches are applied in ALS basic research using animal models of the disease, as well as in clinical trials of ALS patients. Stem cell therapies mainly aim to provide neuroprotection and survival of damaged motor neurons. Stem cells also provide immunomodulation, secrete growth factors, and serve to produce supporting cells, such as astrocytes and oligodendrocytes, or interneurons, which may provide a supportive environment to the vulnerable MNs. Neuronal replacement is another promising therapeutic strategy for ALS. Nowadays, stem cell therapies are in phase 1, 2, and 3 clinical trials and they use a variety of stem cell types such as embryonic stem cells (ESCs), neural stem cells (NSCs), glial progenitor cells (GPCs), mesenchymal stem cells (MSCs), mononuclear cells (MCs), and induced-pluripotent stem cells (iPSCs).

In this Special Issue, our scope is to present a comprehensive collection of research articles, data reports, and reviews contributed by leading experts in the ALS research field. This Special Issue highlights the use of stem cells therapies in the fight against ALS. Basic and preclinical research and advanced clinical trials of phases 1, 2, and 3 that use many stem cell types, virally transduced to deliver therapeutic genes or not, in combination with novel differentiation protocols of stem cells and transplantation approaches, combined or not with additional medication, are expected to achieve effective treatments for this incurable and fatal disease in the future.

Dr. Maria Gaitanou
Guest Editor

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