Thompson Alan J
Faculty of Brain Sciences, UCL Institute of Neurology, University College London, London, UK.
Curr Opin Neurol. 2017 Jun;30(3):237-240. doi: 10.1097/WCO.0000000000000453.
Understanding the mechanisms underlying progression in multiple sclerosis (MS) and identifying appropriate therapeutic targets is a key challenge facing the MS community. This challenge has been championed internationally by organizations such as the Progressive MS Alliance, which has raised the profile of progressive MS and identified the key obstacles to treatment. This review will outline the considerable progress against these challenges.
New insights into mechanisms underlying progression have opened up potential therapeutic opportunities. This has been complemented by ongoing validation of clinical and imaging outcomes for Phase II trials of progression, coupled with the development of innovative trial designs. The field has been greatly encouraged by recent positive Phase III trials in both primary and secondary progressive MS, albeit with modest benefit. Early trials of neuroprotection and repair have provided important new data with which to drive the field. Improving symptom management and advancing rehabilitation approaches, critical for this patient population which, taken together with identifying and managing comorbidities and risk factors, has an appreciable impact on health-related quality of life.
Raising the profile of progressive MS has resulted in the first effective treatments with the promise of more to come.
了解多发性硬化症(MS)进展的潜在机制并确定合适的治疗靶点是MS领域面临的一项关键挑战。这一挑战得到了诸如进行性MS联盟等国际组织的支持,该联盟提高了进行性MS的关注度并确定了治疗的关键障碍。本综述将概述在应对这些挑战方面取得的重大进展。
对进展潜在机制的新见解开辟了潜在的治疗机会。这得到了对进展性II期试验的临床和影像学结果持续验证的补充,同时还有创新试验设计的发展。尽管获益不大,但近期在原发性和继发性进行性MS中的III期试验取得的积极成果极大地鼓舞了该领域。神经保护和修复的早期试验提供了推动该领域发展的重要新数据。改善症状管理和推进康复方法对这一患者群体至关重要,这与识别和管理合并症及风险因素一起,对健康相关生活质量有显著影响。
提高进行性MS的关注度已带来了首批有效治疗方法,且有望有更多进展。