Fox Robert J, Thompson Alan, Baker David, Baneke Peer, Brown Doug, Browne Paul, Chandraratna Dhia, Ciccarelli Olga, Coetzee Timothy, Comi Giancarlo, Feinstein Anthony, Kapoor Raj, Lee Karen, Salvetti Marco, Sharrock Kersten, Toosy Ahmed, Zaratin Paola, Zuidwijk Kim
Mellen Center for Multiple Sclerosis, Neurological Institute, and Lerner College of Medicine, Cleveland Clinic, Cleveland, OH 44122, USA.
Mult Scler. 2012 Nov;18(11):1534-40. doi: 10.1177/1352458512458169. Epub 2012 Aug 23.
Despite significant progress in the development of therapies for relapsing MS, progressive MS remains comparatively disappointing. Our objective, in this paper, is to review the current challenges in developing therapies for progressive MS and identify key priority areas for research. A collaborative was convened by volunteer and staff leaders from several MS societies with the mission to expedite the development of effective disease-modifying and symptom management therapies for progressive forms of multiple sclerosis. Through a series of scientific and strategic planning meetings, the collaborative identified and developed new perspectives on five key priority areas for research: experimental models, identification and validation of targets and repurposing opportunities, proof-of-concept clinical trial strategies, clinical outcome measures, and symptom management and rehabilitation. Our conclusions, tackling the impediments in developing therapies for progressive MS will require an integrated, multi-disciplinary approach to enable effective translation of research into therapies for progressive MS. Engagement of the MS research community through an international effort is needed to address and fund these research priorities with the ultimate goal of expediting the development of disease-modifying and symptom-relief treatments for progressive MS.
尽管在复发型多发性硬化症(MS)治疗方法的开发方面取得了重大进展,但进展型MS仍然相对令人失望。在本文中,我们的目标是回顾进展型MS治疗方法开发中的当前挑战,并确定关键的优先研究领域。来自多个MS协会的志愿者和工作人员领导召集了一个协作组织,其任务是加速开发针对进展型多发性硬化症的有效疾病修饰和症状管理疗法。通过一系列科学和战略规划会议,该协作组织确定并就五个关键优先研究领域形成了新的观点:实验模型、靶点的识别与验证及重新利用机会、概念验证临床试验策略、临床结局指标,以及症状管理与康复。我们的结论是,应对进展型MS治疗方法开发中的障碍需要一种综合的多学科方法,以实现将研究有效地转化为进展型MS的治疗方法。需要通过国际努力让MS研究界参与进来,以解决这些研究优先事项并为其提供资金,最终目标是加速开发针对进展型MS的疾病修饰和症状缓解治疗方法。