Multiple Sclerosis: Prospects and Promise
Multiple Sclerosis: Prospects and Promise
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DOI：
10.1002/ana.24009
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发表时间：
2013-09-01
影响因子：
11.2
通讯作者：
Oksenberg, Jorge R.
中科院分区：
文献类型：
--
作者：
Hauser, Stephen L.;Chan, Jonah R.;Oksenberg, Jorge R.
我们已经进入了多发性硬化症（MS）研究的黄金时代。20年前，我们对这种疾病的理解主要是描述性的，没有批准的治疗方法来改变MS的自然史。今天，与MS相关的免疫途径的描述已经澄清;影响风险的基因的综合图谱汇编;确定环境触发因素的线索; MS疾病过程的非侵入性体内监测已经被高场MRI彻底改变;并且现在存在许多针对MS的早期、复发性组分的有效疗法。然而，重大挑战依然存在。我们仍然没有有效的治疗进展性MS（MS研究的圣杯），没有修复受损轴突或保护神经元的方法，并且指导治疗决策的证据非常有限。最近的进展为开发针对患者的越来越有选择性的免疫疗法奠定了基础;应用遗传和基因组发现来改善治疗选择;开发用于进行性MS的髓鞘再生或神经保护疗法;以及整合临床，成像和基因组数据用于个性化医疗。MS现在已经从自身免疫性疾病研究的死水发展到了前线，包括治愈在内的明确答案现在是未来十年的现实目标。MS中的许多突破性发现也来自跨学科的有意义的互动，特别是来自与临床医生密切合作的转化和基础科学家，强调发现的临床价值通常是在实验室开发的想法在床边进行测试时揭示的。《神经病学年鉴》2013;74：317-327
We have entered a golden era in multiple sclerosis (MS) research. Two decades ago, our understanding of the disease was largely descriptive and there were no approved therapies to modify the natural history of MS. Today, delineation of immune pathways relevant to MS have been clarified; a comprehensive map of genes that influence risk compiled; clues to environmental triggers identified; noninvasive in vivo monitoring of the MS disease process has been revolutionized by high-field MRI; and many effective therapies for the early, relapsing, component of MS now exist. However, major challenges remain. We still have no useful treatment for progressive MS (the holy grail of MS research), no means to repair injured axons or protect neurons, and extremely limited evidence to guide treatment decisions. Recent advances have set in place a foundation for development of increasingly selective immunotherapy for patients; application of genetic and genomic discoveries to improve therapeutic options; development of remyelination or neuroprotection therapies for progressive MS; and integrating clinical, imaging and genomic data for personalized medicine. MS has now advanced from the backwaters of autoimmune disease research to the front-line, and definitive answers, including cures, are now realistic goals for the next decade. Many of the breakthrough discoveries in MS have also resulted from meaningful interactions across disciplines, and especially from translational and basic scientists working closely with clinicians, highlighting that the clinical value of discoveries are most often revealed when ideas developed in the laboratory are tested at the bedside. Ann Neurol 2013;74:317-327