Regarding the familiar form, the genes mainly involved are SOD1, TARDBP, FUS, OPTN, VCP, UBQLN2, C9ORF72, and PFN1. The familial forms of ALS are reported only in 5–10% of cases on the contrary, the majority of ALS cases are sporadic. Noninvasive ventilation, considered a standard treatment for respiratory support for ALS patients, was demonstrated to improve the quality of life but also to increase the survival of patients. Unfortunately, respiratory symptoms are not easy to recognize and alterations of blood gas analysis become evident only in a late stage of the disease or when an acute episode of respiratory failure happens. The loss of function of the phrenic nerve induces diaphragm weakness, leading as a consequence to dyspnea, orthopnea, and hypoventilation. The appearance of respiratory failure is caused by the impairment of the respiratory musculature, and it is influenced by the concomitant presence of other respiratory pathologies. Respiratory failure is recognized as one of the main complications of ALS and one of the main causes leading to death. Usually, ALS leads to death within 3–5 years. Even if ALS main symptoms are correlated with motor dysfunction, about 50% of patients show also cognitive and behavioural impairment. However, other than by a progressive and asymmetric weakness and atrophy in limb, thoracic, abdominal, and bulbar muscles, ALS is associated also with extrapyramidal features, postural abnormalities, small fiber neuropathy, and mild oculomotor disturbance. In Europe, the ALS incidence was estimated to be 2 to 3 cases per 100,000 individuals. The patients can show an initial presentation with spinal-onset disease, which is the most common form characterized by limb muscle weakness, or with bulbar-onset disease, whose characteristics are dysarthria and dysphagia. Specifically, UMN symptoms include weakness, speech difficulties, spasticity, and inappropriate emotionality, while LMN symptoms are represented by cramps, twitching, muscle wasting and weakness. The main symptoms of ALS are muscle weakness, wasting, in particular in the limbs, cramps, twitching, and problems in speaking. 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. IntroductionĪmyotrophic 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. For this reason, more studies and trials are needed to clarify the real effectiveness of MSC-based therapy in ALS. However, only partial improvements were shown. In addition, clinical trials evidenced the feasibility and safety of MSC transplantation in ALS patients, given that no major adverse events were recorded. 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 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 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. Stem cell therapy is arising as a new promising approach for the treatment of neurodegenerative disorders.

A cure is not available at this time, and the disease leads to death within 3–5 years, mainly due to respiratory failure. Patients show both motor and extra-motor symptoms. Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease characterized by the degeneration of both upper and lower motor neurons.

Stem cell treatment for als