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2016年6月13日 讯 /生物谷BIOON/ --最新的一项研究指出，一种新的疗法有望治愈多发性硬化。 最近一项在加拿大进行的、囊括24名病患的临床试验中，一名患者从治疗前需要坐轮椅的状态，在治疗后能够获得正常人的生活。 不过需要注意的是，该疗法程度十分猛烈，一名患者在治疗过程中死亡。就此，研究者们希望能够进一步优化治疗用药物，以达到对多发性硬化患者安全的标准。 这一疗法是加拿大渥太华医院研究所的研究者们偶然发现的，当时他们正在为同时患有白血病与多发性硬化的患者进行治疗。 治疗白血病的主流方法之一就是将骨髓细胞抽出，杀死体内残留的免疫细胞，之后再将净化后的骨髓细胞注入用于骨髓重建。 结果显示，这一重建后的骨髓不仅有助于白血病的治疗，而且能够有效抵抗多发性硬化。多发性硬化的病因是免疫细胞攻击大脑、脊髓以及神经系统中的神经细胞外周的保护组织。基于上述结果，研究者们决定开展专门的临床试验：将免疫系统摧毁后再重建。 这一疗法是用于阻止多发性硬化的恶化，但在大多数情况下它甚至能够完全治愈疾病，这说明神经系统具有一定的自愈能力。 该临床试验开展于2000年，在24名多发性硬化病人中，有17名产生了明显的效果。 但这并不是一个简单的治疗方法，它具有严重的副作用，因此仅仅适用于多发性硬化病情严重的患者。如果医生们能够找到安全的优化方法，将会是一个巨大的进步。 目前，多发性硬化的疗法主要包括限制疾病的效应以及降低急性发作数量，虽然不能根本上解决问题，但不失为一个折中的办法。科学家们目前仍不知道为什么在多发性硬化中免疫系统会主动攻击神经细胞，但折中摧毁-重建的方式似乎能够消除原本免疫系统的记忆。 相关结果发表在《Lancet》杂志上。（生物谷Bioon.com） 本文系生物谷原创编译整理，欢迎转载！点击 获取授权 。更多资讯请下载生物谷APP. 生物谷推荐英文原文报道：This new drug is super effective at clearing up severe psoriasis, trials find  DOI: [url]http://dx.doi.org/10.1016/S0140-6736(16)30169-6[/url] PMC: PMID: Immunoablation and autologous haemopoietic stem-cell transplantation for aggressive multiple sclerosis: a multicentre single-group phase 2 trial Dr Harold L Atkins, MDcorrespondenceemail, Marjorie Bowman, MScN, David Allan, MD, Grizel Anstee, MD, Prof Douglas L Arnold, MD, Prof Amit Bar-Or, MD, Isabelle Bence-Bruckler, MD, Paul Birch, MLT, Prof Christopher Bredeson, MD, Jacqueline Chen, PhD, Prof Dean Fergusson, PhD, Mike Halpenny, MLT, Linda Hamelin, RN, Prof Lothar Huebsch, MD, Brian Hutton, PhD, Pierre Laneuville, MD, Yves Lapierre, MD, Hyunwoo Lee, BSc, Lisa Martin, MLT, Sheryl McDiarmid, RN, Prof Paul O'Connor, MD, Timothy Ramsay, PhD, Mitchell Sabloff, MD, Lisa Walker, PhD, Prof Mark S Freedman, MD Background Strong immunosuppression, including chemotherapy and immune-depleting antibodies followed by autologous haemopoietic stem-cell transplantation (aHSCT), has been used to treat patients with multiple sclerosis, improving control of relapsing disease. We addressed whether near-complete immunoablation followed by immune cell depleted aHSCT would result in long-term control of multiple sclerosis. Methods We did this phase 2 single-arm trial at three hospitals in Canada. We enrolled patients with multiple sclerosis, aged 18–50 years with poor prognosis, ongoing disease activity, and an Expanded Disability Status Scale of 3·0–6·0. Autologous CD34 selected haemopoietic stem-cell grafts were collected after mobilisation with cyclophosphamide and filgrastim. Immunoablation with busulfan, cyclophosphamide, and rabbit anti-thymocyte globulin was followed by aHSCT. The primary outcome was multiple sclerosis activity-free survival (events were clinical relapse, appearance of a new or Gd-enhancing lesion on MRI, and sustained progression of Expanded Disability Status Scale score). This study was registered at ClinicalTrials.gov, NCT01099930. Findings Between diagnosis and aHSCT, 24 patients had 167 clinical relapses over 140 patient-years with 188 Gd-enhancing lesions on 48 pre-aHSCT MRI scans. Median follow-up was 6·7 years (range 3·9–12·7). The primary outcome, multiple sclerosis activity-free survival at 3 years after transplantation was 69·6% (95% CI 46·6–84·2). With up to 13 years of follow-up after aHSCT, no relapses occurred and no Gd enhancing lesions or new T2 lesions were seen on 314 MRI sequential scans. The rate of brain atrophy decreased to that expected for healthy controls. One of 24 patients died of transplantation-related complications. 35% of patients had a sustained improvement in their Expanded Disability Status Scale score. Interpretation We describe the first treatment to fully halt all detectable CNS inflammatory activity in patients with multiple sclerosis for a prolonged period in the absence of any ongoing disease-modifying drugs. Furthermore, many of the patients had substantial recovery of neurological function despite their disease's aggressive nature.
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