UMR 788 INSERM and Université Paris-Sud, 94276 Kremlin-Bicêtre, France.
Brain. 2013 Jan;136(Pt 1):132-46. doi: 10.1093/brain/aws284.
Myelin regeneration is a major therapeutic goal in demyelinating diseases, and the failure to remyelinate rapidly has profound consequences for the health of axons and for brain function. However, there is no efficient treatment for stimulating myelin repair, and current therapies are limited to anti-inflammatory agents. Males are less likely to develop multiple sclerosis than females, but often have a more severe disease course and reach disability milestones at an earlier age than females, and these observations have spurred interest in the potential protective effects of androgens. Here, we demonstrate that testosterone treatment efficiently stimulates the formation of new myelin and reverses myelin damage in chronic demyelinated brain lesions, resulting from the long-term administration of cuprizone, which is toxic for oligodendrocytes. In addition to the strong effect of testosterone on myelin repair, the number of activated astrocytes and microglial cells returned to low control levels, indicating a reduction of neuroinflammatory responses. We also identify the neural androgen receptor as a novel therapeutic target for myelin recovery. After the acute demyelination of cerebellar slices in organotypic culture, the remyelinating actions of testosterone could be mimicked by 5α-dihydrotestosterone, a metabolite that is not converted to oestrogens, and blocked by the androgen receptor antagonist flutamide. Testosterone treatment also failed to promote remyelination after chronic cuprizone-induced demyelination in mice with a non-functional androgen receptor. Importantly, testosterone did not stimulate the formation of new myelin sheaths after specific knockout of the androgen receptor in neurons and macroglial cells. Thus, the neural brain androgen receptor is required for the remyelination effect of testosterone, whereas the presence of the receptor in microglia and in peripheral tissues is not sufficient to enhance remyelination. The potent synthetic testosterone analogue 7α-methyl-19-nortestosterone, which has been developed for long-term male contraception and androgen replacement therapy in hypogonadal males and does not stimulate prostate growth, also efficiently promoted myelin repair. These data establish the efficacy of androgens as remyelinating agents and qualify the brain androgen receptor as a promising drug target for remyelination therapy, thus providing the preclinical rationale for a novel therapeutic use of androgens in males with multiple sclerosis.
髓鞘再生是脱髓鞘疾病的主要治疗目标,而不能快速进行髓鞘修复对轴突的健康和大脑功能有深远的影响。然而,目前还没有有效的刺激髓鞘修复的治疗方法,并且当前的治疗方法仅限于抗炎药物。男性患多发性硬化症的可能性低于女性,但疾病的严重程度往往更高,而且比女性更早达到残疾的里程碑,这些观察结果激发了人们对雄激素潜在保护作用的兴趣。在这里,我们证明睾酮治疗能有效刺激新髓鞘的形成,并逆转由长期给予对少突胶质细胞有毒的 cuprizone 引起的慢性脱髓鞘脑损伤中的髓鞘损伤。除了睾酮对髓鞘修复的强烈作用外,激活的星形胶质细胞和小胶质细胞的数量也恢复到低对照水平,表明神经炎症反应减少。我们还确定神经雄激素受体是髓鞘恢复的新治疗靶点。在器官型培养的小脑切片急性脱髓鞘后,睾酮的促髓鞘形成作用可以被其代谢物 5α-二氢睾酮模拟,5α-二氢睾酮不会转化为雌激素,且可被雄激素受体拮抗剂氟他胺阻断。在缺乏功能性雄激素受体的小鼠中,慢性 cuprizone 诱导的脱髓鞘后,睾酮治疗也未能促进髓鞘形成。重要的是,在神经元和大胶质细胞中特异性敲除雄激素受体后,睾酮也不会刺激新髓鞘鞘的形成。因此,神经脑雄激素受体是睾酮促髓鞘形成作用所必需的,而受体在小胶质细胞和外周组织中的存在不足以增强髓鞘形成。已经开发出用于长期男性避孕和性腺功能减退男性雄激素替代治疗的合成睾酮类似物 7α-甲基-19-去甲睾酮,它不会刺激前列腺生长,也能有效促进髓鞘修复。这些数据确立了雄激素作为髓鞘修复剂的疗效,并将脑雄激素受体确定为髓鞘修复治疗的有希望的药物靶点,从而为雄激素在多发性硬化症男性中的新治疗用途提供了临床前依据。