Research progress of stem cell transplantation on neuromuscular diseases
2020-06-23
Neuromuscular diseases are a variety of acquired or inherited diseases that involve nerves, muscles, and neuromuscular junctions. The loss of muscle contraction and muscle atrophy due to nerve damage or signaling pathway failure affects the quality of life of patients.
Classification of neuromuscular diseases
There are hundreds of neuromuscular diseases, mainly divided into two types: rapid progression and chronic progression. The former is mainly motor neuron diseases, such as progressive muscle atrophy, amyotrophic lateral sclerosis (commonly known as "gradual freezing"), spinal muscular atrophy and primary lateral sclerosis, etc.; the latter are mostly genetic diseases, common There are spinal muscular atrophy, post-polio syndrome, and facial and shoulder brachial muscular dystrophy [1].
Current status of neuromuscular
According to the "Diagnosis and Treatment Guide for Rare Diseases" released in 2019, neuromuscular diseases such as amyotrophic lateral sclerosis are mostly rare diseases [2], but rare diseases are actually "not rare". There are about 4.5 million NMD patients in China. Life is threatened for this.
At present, clinical damage cannot effectively treat neuromuscular diseases by repairing damaged nerve cells. Studies have found that stem cells have the potential for directional differentiation and secretion of nerve factors. Under certain environmental factors, they can repair damaged tissues and play a certain role in improving neuromuscular diseases!
1. Stem cells differentiate into neurons
Stem cells are a type of cells with the potential for self-replication and multi-directional differentiation. Under certain conditions, they can differentiate into various cells required by the human body, such as neurons or glial cells. These differentiated nerve cells also have functions and can be replaced. Damaged cells.
2. Neural factors secreted by stem cells
Stem cells co-cultured with motor neurons and glial cells can induce increased secretion of glial cell-derived neurotrophic factor (GDNF) and ciliary nerve factor (CNTF); increased expression of vascular endothelial cytokine (VGEF) reduces cytokines Expression of IL-6, TNF-α, iNOS [3].
Research on stem cell transplantation
For many years, scientists have devoted themselves to the research of stem cells on neuromuscular diseases, from basic research to animal models to clinical research, revealing a positive message: stem cell transplantation to repair neuromuscular atrophy is scientifically feasible.
Animal model research: In March 2020, Wang et al. released a report of bone marrow mesenchymal stem cell transplantation to repair denervated muscle atrophy. The researchers performed stem cell transplantation on 10 model rats and found that the neurological function score of the hind limbs had improved; the stem cells entered the muscle fibers and repaired the muscle fiber intervals. The arrangement of muscle fibers was more regular and returned to normal; the measured values of muscle fiber diameter and muscle fiber cross-sectional area increased Big [4].
Clinical research: Stem cell transplantation to repair neuromuscular diseases has been verified by phase I and phase II clinical trials. In 2014, the stem cell product NurOwn has been designated by the FDA fast-track product, and completed the safety certification of phase II (48 subjects) in 2016; in August 2017, the researchers opened a phase III clinical trial, and In August 2018, it passed the review of the Drug Safety Monitoring Committee and was deemed to have no obvious safety issues [5].
South Korea launched Neuronata-R, a stem cell product targeting motor neuron diseases, in 2015. This is the world’s first stem cell product for neuromuscular diseases. To this end, it can be seen that stem cell transplantation has huge clinical applications for neuromuscular diseases. Value is highly valued by scientists!
Scientists will shoulder the responsibility of "thawing" and gradually freezing people, bringing a glimmer of light to neuromuscular patients!
