Department of Clinical Neurosciences and National Institute for Health Research (NIHR) Biomedical Research Centre, University of Cambridge, Cambridge, UK.
Cambridge Innovation Technologies Consulting (CITC) Ltd, Cambridge, UK.
Trends Mol Med. 2020 Oct;26(10):898-912. doi: 10.1016/j.molmed.2020.04.005. Epub 2020 May 21.
Multiple disease-modifying medications with regulatory approval to treat multiple sclerosis (MS) are unable to prevent inflammatory tissue damage in the central nervous system (CNS), and none directly promote repair. Thus, there is an unmet clinical need for therapies that can arrest and reverse the persistent accumulation of disabilities associated with progressive forms of MS (P-MS). Preclinical research has revealed an unexpected ability of neural stem cell (NSC) therapies to provide neurotrophic support and inhibit detrimental host immune responses in vivo following transplantation into the chronically inflamed CNS. We discuss NSC transplantation as a promising therapy for P-MS, elaborate on the necessities of clinical trial validation and formalized usage guidelines, and caution about unscrupulous 'clinics' marketing unproven therapies to patients.
多种已被监管机构批准用于治疗多发性硬化症(MS)的疾病修正治疗药物,无法预防中枢神经系统(CNS)的炎症性组织损伤,也没有一种药物可以直接促进修复。因此,临床上迫切需要能够阻止和逆转与进展型多发性硬化症(P-MS)相关的持续性残疾累积的治疗方法。临床前研究揭示了神经干细胞(NSC)疗法的一个意外能力,即在移植到慢性炎症的中枢神经系统后,能够提供神经营养支持并抑制有害的宿主免疫反应。我们讨论了 NSC 移植作为 P-MS 的一种有前途的治疗方法,详细阐述了临床试验验证和规范化使用指南的必要性,并对不道德的“诊所”向患者推销未经证实的治疗方法提出了警告。