Chinese researchers made breakthrough in treating Pakinson's disease with "genetically engineered mesenchymal stem cells".
Paper in English:
Abstract
Although striatal delivery of three critical genes for dopamine synthesis by viruses is a potential clinical approach for treating Parkinson’s disease (PD), the approach makes it difficult to finely control dopamine secretion amounts and brings safety concerns. Here, we generate genetically engineered mesenchymal stem cells encoding three critical genes for dopamine synthesis (DOPA-MSCs). DOPA-MSCs retain their MSC identity and stable ability to secrete dopamine during passaging. Following transplantation, DOPA-MSCs reinstate striatal dopamine levels and correct motor function in PD rats. Importantly, after grafting into the caudate and putamen, DOPA-MSCs provide homotopic reconstruction of midbrain dopamine pathways by restoring striatal dopamine levels, and safely and long-term (up to 51 months) correct motor disorders and nonmotor deficits in acute and chronic PD rhesus monkey models of PD even with advanced PD symptoms. The long-term benefits and safety results support the idea that the development of dopamine-synthesized engineered cell transplantation is an important strategy for treating PD.
News in Chinese:
基因工程化间充质干细胞 为帕金森病治疗带来曙光
科技日报讯 (记者赵汉斌)帕金森病是继肿瘤、心脑血管疾病之后中老年健康的“第三杀手”。近日,科技日报记者从昆明理工大学灵长类转化医学研究院了解到,该院季维智院士、李天晴教授团队在应用基因工程化间充质干细胞治疗帕金森病方面取得重要进展。相关研究成果在线发表于国际期刊《自然伙伴期刊—帕金森病》。
目前,我国帕金森病患者已超300万人,年医疗费用超过2000亿元。这种疾病发病率和死亡率都非常高,6年内的死亡率高达66%。常规的临床治疗方法,长期使用会带来明显的副作用,后期还有失效之虞。因此开发安全、有效、经济并适合广大帕金森病患者的治疗方法,一直是整个医疗领域努力的方向。
李天晴、季维智团队耗时8年,潜心开发出用于帕金森病治疗的可持久稳定分泌多巴胺神经递质的基因工程化间充质干细胞,将其移植到大鼠和猴帕金森病模型后,可通过恢复纹状体多巴胺水平,实现中脑多巴胺环路的原位重建,并可快速恢复急性和慢性帕金森病猴模型的运动和非运动障碍缺陷。长达5年的结果评价,证明了该方法的安全性和有效性,显示出基因工程化间充质干细胞在未来帕金森病临床治疗应用中的巨大潜力。
此项研究有力地支持了基因工程化间充质干细胞移植在不同帕金森病模型中的显著治疗效果,其安全性可在移植前得以充分评估;同时可实现细胞的规模化和标准化扩增以及建立稳定的质控体系,用于细胞药物开发。此方法可以治疗包括重症在内的大部分帕金森病,且移植结果在2周内可发挥功效,有利于移植效果评价,减少临床费用。