Clinical Study of Stem Cells Transplantation for the Treatment of Amyotrophic Lateral Sclerosis/Motor Neuron Disease
By Like Wu , Xiaojuan Wang , Bo Cheng, Susan Chu, Shuangshuang Liu , Fang Peng , Xiang Wang and Shengjie Liu
Wu Medical Center (3-10-2016)
ALS (Amyotrophic Lateral Sclerosis) is also known as MND (Motor Neuron Disease), is a kind of motor neuron degeneration (upper and lower motor neurons) caused by a gene mutation which leads to the gradual diffused muscle weakness and atrophy including the bulbar area (mainly indicates the muscles controlling the respiratory function, swallowing and speech pronunciation), four limbs, trunk, chest and abdomen, and finally leads to respiratory and circulatory failure. ALS is a kind of deadly disease which progresses very quickly, the patient’s body condition will decline at a fast rate, and because the motor neurons affected by the disease and those neurons can not be regenerated again, the current medical method cannot provide an effective treatment for ALS, though we had targeted a therapeutic drug, Riluzole, which was approved by the FDA for ALS clinical treatment. Riluzole cannot prevent the quick progress of ALS, it only can help patients prolong their lives for roughly 3-6 months, according to research. The stem cell therapy is a kind of new technology that can supply ALS patients new motor neurons which carry normal healthy genes after implantation, improve the healthy motor neurons, and slow down/block the further progress of ALS.
From Jan, 2013 to June, 2016, there were 68 ALS diagnosed patients at Wu Medical Center(WMC). The patients were administered three neural stem cell transplants and three mesenchymal stem cell transplants within 2-3 weeks besides the general internal medicine treatment. Doctors had evaluated the patients’ functional changes with ALSFRS before and after the treatment, and monitored the patients’ side effects during the treatment period and their treatment compliance. From the results we could see the patients’ ALSFRS scale increase 5.5 after the treatment, and differences were statistically significant. The side effects during the stem cell treatment period were minimal, there were only 2 cases with a slight fever, 1 case had a headache, while the symptoms were totally controlled after proper medications were given. There was no damage to the organs. The adherence to the medication was better and there were no bad responses reported. The clinical treatment data had fully demonstrated the safety and effectiveness of the combination of the two kinds of stem cells used for the ALS treatment.
Wu Medical Center offered a proposal based on recent years of treatment experience and combined with the specific basic scientific research, the stem cells can be controlled in vivo artificially more effectively, and doctors can guide the stem cells to differentiate into the targeted cells according to the actual clinical treatment requirements. In terms of ALS therapy, ALS is a kind of motor neuron system degenerative change caused by gene mutation, the number of neurons decrease gradually, and the patient’s weakness and muscle atrophy worsens day by day, eventually leading to complete paraplegia, even affecting the respiratory system and respiratory failure will eventually occur. Once the CNS(central nervous system) motor neurons are damaged, they cannot regenerative, and while neurological doctors cannot find an effective method for treatment of this condition, doctors at Wu Medical Center have applied stem cells for ALS treatment, increasing the number of motor neurons, enhancing the motor neuron system’s resistance to the pathogenetic factors, repairing the nerve damage, helping patients to recover and improve the muscles’ function, improve the patient’s quality of life and prolong their survival.
To make stem cells work efficiently after implantation, the doctors must be familiar with the cells’ characteristics, use complicated medical technology to regulate and monitor the cells’ growth and work in vivo, use the proper mediation for assistance and use physical rehabilitation training designed according to the instructions of the clinical index. The strict clinical cells regulations will guarantee the real effectiveness of the stem cell treatment, while from past experience, the pure stem cell implantation without cell control treatment could not achieve ideal treatment outcomes, since the existence of the pathology environment, neither the patient’s own motor neurons nor the implanted stem cells (or the neural precursor cells) did not allow the stem cells to survive.
Currently Wu Medical Center has achieved some good progress for ALS treatment, such as doctors discovering that the ALS/MND patient’s abnormal protein accumulation is related to prion protein degeneration, and based on this knowledge, there is an option to treat and improve the motor neurons’ anti-disease fighting ability. For example: motor neurons have prion proteins which are a kind of functional protein. When prion protein gene mutation occurs, there will be a faulty transformation from α-helix to β-pleated sheets, leading to heterodimer formation and a gradual accumulation of abnormal proteins. This can affect neighboring motor neurons via the axon nerves’ conduction and the motor neurons will start the programmed cell necrosis. Wu Medical Center doctors use implanted neural precursor cells which have healthy genes to build internuncial neurons among the genetically mutated nerve cells. They can provide molecular chaperones, and change the methionine into valine, correct the faulty β-pleated sheet transformations, allowing normal nucleoprotein to be produced to heal the nerve cells. The therapy can help patients restore their motor neuron functioning, improve the capability of disease resistance, and provide a new effective method to deal with ALS. However, this therapy requires a series of clinical monitoring and control processes toward the cells in-vivo in order to achieve effective treatment outcome.
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