Abstract
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease characterized by the degeneration of both upper and lower motor neurons. Patients show both motor and extra-motor symptoms. A cure is not available at this time, and the disease leads to death within 3–5 years, mainly due to respiratory failure. Stem cell therapy is arising as a new promising approach for the treatment of neurodegenerative disorders. In particular, mesenchymal stem cells (MSCs) seem the most suitable type of stem cells, thanks to their demonstrated beneficial effects in different experimental models, to the easy availability, and to the lack of ethical problems. In this review, we focused on the studies involving ALS rodent models and clinical trials in order to understand the potential beneficial effects of MSC transplantation. In different ALS rodent models, the administration of MSCs induced a delay in disease progression and at least a partial recovery of the motor function. In addition, clinical trials evidenced the feasibility and safety of MSC transplantation in ALS patients, given that no major adverse events were recorded. However, only partial improvements were shown. For this reason, more studies and trials are needed to clarify the real effectiveness of MSC-based therapy in ALS.
Highlights
Amyotrophic lateral sclerosis (ALS) is a lethal neurodegenerative disorder characterized by the selective degeneration of both upper (UMN) and lower motor neurons (LMN), causing both motor and extra-motor symptoms [1, 2].LMNs are in the brainstem and spinal cord and transmit impulses from the UMNs to the muscles at the level of the neuromuscular synapses to innervate the skeletal muscles that control the arms and the legs
Even if there were no significant differences in the symptom onset time, they evidenced that the highest dose (1 × 106 cells) was able to prolong the survival and to delay the decline of motor function, associated with a higher average number of motor neurons compared to untreated animals and to those receiving the lowest mesenchymal stem cells (MSCs) dose
It was necessary to evaluate if MSCs derived from ALS patients may be useful for stem cell therapy and able to exert the same effects of MSCs obtained from healthy donors
Summary
Amyotrophic lateral sclerosis (ALS) is a lethal neurodegenerative disorder characterized by the selective degeneration of both upper (UMN) and lower motor neurons (LMN), causing both motor and extra-motor symptoms [1, 2]. The main symptoms of ALS are muscle weakness, wasting, in particular in the limbs, cramps, twitching, and problems in speaking [3]. It is known that about two-thirds of familial cases are caused by the genes C9ORF72, SOD1, TARDBP, and FUS [7]. In the context of developing new therapeutic strategies, stem cell therapy is reaching promising results in the treatment of different neurodegenerative disorders. The assumption of this approach is that stem cells, which are able to differentiate into neuronal cells, may replace the degenerated neurons. Stem cells are able to release different growth factors that may create a microenvironment that promotes neuroprotection. Being adult stem cells, MSCs do not face the ethical concerns linked to the use of embryonic stem cells
Sign-in/Register to view highlights & summary Sign-in/Register to access full text options 
Free) 
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a call
