ALS的干细胞疗法再现曙光

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ALS的干细胞疗法再现曙光

2012-01-18 14:15 来源：丁香园 作者：xxsl_2007

本周二，BrainStorm 公司宣布，在一项治疗肌萎缩性侧索硬化症（ALS）的临床试验中，其研发的细胞治疗（成人干细胞疗法）被证明是安全的，未发生明显副作用。/ \  n; `2 l6 a# t8 X8 J* Y/ U

2 q; s& N% ~1 I% D以色列BrainStorm公司研发的新药NurOwn主要用于肌萎缩性侧索硬化症（ALS）的治疗。它又被称为卢伽雷氏病（Lou Gehrig病），是一种的渐进和致命的神经退行性疾病，主要累及大脑和脊髓。去年，美国食品药品管理局批准了NurOwn用于罕见病的治疗。此外，FDA的罕见病治疗药物计划还为罕见病治疗的发展给予了财政扶持。
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以色列哈达萨医疗中心的多发性硬化症分部负责人，也是这项试验的主持者Dimitrios Karussis说到：“在试验中，患者接受了自体骨髓干细胞移植和NurOwn干细胞技术的治疗。BrainStorm的新药NurOwn未出现明显副作用。并且，早期的临床随访还发现干细胞治疗能使患者获益：其呼吸、咀嚼功能和肌力都得到了一定的改善。”. ~3 q' n0 V( H* z8 O# T2 \4 U# p- D
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第一阶段试验的主要目的是评估NurOwn的安全性，接下来将致力于验证其是否真的有效。BrainStorm 公司总裁Chaim Lebovits 说到“由于第一阶段的试验已经证实了NurOwn的安全性，医院伦理和安全委员会已经同意其用于下一批病人的治疗。”
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; }" g  c3 e6 h4 R9 ?1 L" F根据肌萎缩性侧索硬化症联合会的资料，每年美国新发ALS患者5,600例，截止目前全美预计已有30,000例ALS患者。英国物理学家霍金就是由于ALS而导致的瘫痪。4 P' D. r/ V6 u, T$ U' x* M- W
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http://www.reuters.com/article/2012/01/17/us-brainstorm-trial-embargoed-idUSTRE80G07Q20120117?feedType=RSS&feedName=healthNews&utm_source=feedburner&utm_medium=feed&utm_campaign=Feed%3A+reuters%2FhealthNews+%28News+%2F+US+%2F+Health+News%291 M* \" d% N) i( [7 V6 t7 v) ~

( V7 d4 t2 p' l5 I  I5 A7 \BrainStorm sees positive data in ALS stem cell trial
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(Reuters) - Data from the first ALS patients in a clinical trial treated with BrainStorm Cell Therapeutics adult stem cell therapy did not show significant side effects and the treatment has so far proven to be safe, the company said on Tuesday.
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+ o  Z: ~( t& W/ nIsrael-based BrainStorm is developing NurOwn for the treatment of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's Disease, a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord.
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The U.S. Food and Drug Administration last year granted orphan drug designation to NurOwn. The FDA's orphan drug program encourages the development of treatments for rare diseases by providing financial incentives.
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  C  h5 _) |3 `. O% U8 w8 A/ D"There have been no significant side effects in the initial patients we have treated with BrainStorm's NurOwn technology," said Dimitrios Karussis, the head of Israel's Hadassah Medical Center's Multiple Sclerosis unit, who is leading the clinical trial.% q: M/ s$ b/ N  F, l

  h' S: h- I. O7 d" h"In addition, even though we are conducting a safety trial, the early clinical follow-up of the patients treated with the stem cells shows indications of beneficial clinical effects, such as an improvement in breathing and swallowing ability as well as in muscular power."7 i9 e  r1 @( f, @9 p

8 J1 A3 D! k1 K* R0 @; A8 TPatients in the trial are transplanted with stem cells derived from their own bone marrow and treated with the NurOwn stem cell technology.+ }* {, Q. F  ^! k9 r2 m
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The initial phase of the study is designed to establish the safety of NurOwn and will later be expanded to assess efficacy.+ o; ]- e/ J: `/ P

2 C8 E+ b, X4 }0 M/ }+ l7 Y+ ], r"Based on the interim safety report, the hospital ethical and safety committee granted the company approval to proceed with treating the next patients," said Chaim Lebovits, president of BrainStorm.
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# L. T8 J# r* V2 V- yAccording to the ALS Association, 5,600 people in the United States are diagnosed with the disease each year. It is estimated that as many as 30,000 Americans may have the disease -- which has severely disabled British physicist Stephen Hawking -- at any given time.
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5 T5 |! U. {; D  K9 `% }" u(Reporting by Tova Cohen; Editing by Helen Massy-Beresford)
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http://news.dxy.cn/bbs/topic/22055237 9 Q1 ?2 z5 z. T