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干细胞替代治疗可能是一种能彻底治愈肌萎缩侧索硬化症的方法。体外培养的胚胎干细胞和神经干细胞能分化为具有电生理活性并能支配肌管的运动神经元。在运动神经元缺失的成年大鼠脊髓内,移植的神经干细胞能分化为运动神经元,并能支配骨骼肌及部分恢复实验动物的运动功能。另一些研究表明,干细胞可通过释放神经营养因子及重建运动神经元微环境等机制,保护濒死的运动神经元,从而延缓疾病进展。在目前看来,这种干细胞保护治疗更具实际意义。
Stem cell replacement therapy may be a way to completely cure ALS. In vitro cultured embryonic stem cells and neural stem cells can differentiate into motoneurons that have electrophysiological activity and can dominate the myotubes. Transplantation of neural stem cells differentiates into motor neurons in the spinal cord of adult rats with motor neuron loss and can dominate skeletal muscle and partially restore motor function in experimental animals. Other studies show that stem cells can protect the dying motor neurons by releasing neurotrophic factors and rebuilding motor neuron microenvironment to delay the progression of the disease. So far, this kind of stem cell protection therapy has more practical significance.