世界首个干细胞治疗药物Prochymal获加拿大卫生部批准上市（附原文）

细胞海洋

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作者：jewell 来源：生物谷 2012-5-21 13:30:54 0 13 X- e: k: ?- `/ U* w
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一家小型生物技术公司日前宣称由其研发的干细胞治疗药物获加拿大监管部门批准上市，成为世界首个获批上市的人造干细胞药物，这对再生医学的发展又推进了一步。
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4 z7 w. {- N2 E! ^% B位于哥伦比亚的奥西里斯诊疗公司（Osiris）17日宣布获得加拿大卫生部对其干细胞疗法药物Prochymal的上市批准，用于治疗儿童移植物抗宿主病（GVHD）。" L$ c3 B1 }- ]6 j0 [7 `

! T6 k. ?' O) _* E* J: W( T“今天是对干细胞疗法理念及其实现颇具意义的一天，”Osiris公司行政总裁C-兰德尔-米尔斯（C. Randal Mills）在采访中表示。" x5 x) j( V4 l& d
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Prochymal由来自于健康的青年捐献者骨髓中的间质干细胞制备而成，从一个供源的骨髓中分离出干细胞并进行扩大培养，即可获得多达1万个剂量的Prochymal。( `0 X7 k; ?! t8 S* Y

2 i$ ?  R+ i  bProchymal取材于成体干细胞，因而并不涉及胚胎干细胞相关的伦理问题。
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GVHD致死率高，多发于移植时配型不完全匹配的受者，由骨髓移植后的免疫排异反应引起，常对皮肤、肝脏和消化道等造成严重损伤。现多用激素类药物治疗，但疗效甚微。, R4 t  r: e( ~

/ F7 X1 L1 X$ tProchymal在加拿大获批用于治疗那些对激素类药物无反应的GVHD儿童患者。在一个小规模临床试验中，约60%的GVHD儿童患者使用此药后症状得到改善，Osiris称。
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& h7 |9 L* W0 }" e0 g7 B7 R4 a2009年，Prochymal在其II期临床试验中惨遭失败——其疗效与安慰剂相差无几。公司在探索Prochymal用于治疗克隆氏症、糖尿病和心脏病等其他病症时也屡屡受挫。
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* A- L5 a- Q+ U6 j曾拥有Prochymal在北美以外地区销售权的赛诺菲（Sanofi），已在今年二月宣布放弃此药。6 \& D5 x8 @/ M+ x: s6 i

9 Y( M. g  R; P- x3 Y6 eMills表示，研究人员最终在一次小型药物试验中发现，Prochymal用于治疗对激素类药物无反应的严重GVHD儿童患者是最有效的。
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FDA表示Prochymal要获得审批必须提供更多有力的临床数据支持，因此Osiris首先申请了加拿大监管部门的批准，Mills说，今年底会向FDA提起申请。
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如今，骨髓或干细胞移植用于治疗各种癌症和遗传病已不少见，但都属于医疗程序，而不是药业公司生产出售的药物。
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5 w7 ]5 H) u+ i. `) w市面上已有几种获监管部门批准的细胞疗法，如健赞公司（Genzyme）推出的组织工程软骨移植疗法（Carticel），即使用患者自己的细胞来修复受损伤的软骨，以及去年FDA批准的移植中可使用脐血，与它们不同的是，Prochymal是可生产的实物现货，Mills如是说。（生物谷Bioon.com）
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# I& z7 h& v" t4 z9 U编译自A Stem-Cell-Based Drug Gets Approval in Canada
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; b! E  o! B; h8 _' v3楼原文 感谢sunsong7 提供

sunsong7

it  is a big milestone in stem cell therapeautic products！

sunsong7

Canada Approves Prochymal, World’s First Stem-Cell Drug
4 P' D; u  \: G# v3 ^) [, MPosted: May 19, 2012
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Canadian health regulators have approved the world’s first stem-cell drug for children, according to Osiris Therapeutics Inc.
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5 j5 W- R( m1 @1 AAccording to Reuters, the company released a statement on Thursday, which stated that the regulators approved the treatment for “acute graft-versus host disease in children.”
5 Q" ]/ a4 F) G! C7 VThe Digital Journal reports that Andrew Daly, M.D., Clinical Associate Professor, Department of Medicine and Oncology at the University of Calgary, Canada stated in a press release:, s" {* N6 j& s! P7 I6 z6 A/ n

“I am very proud of the leadership role Canada has taken in advancing stem cell therapy and particularly gratified that this historic decision benefits children who would otherwise have little hope.”
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Graft-versus host disease (GvD) occurs after a patient goes through a bone marrow transplant. The Digital Journal reports that the disease will kill about 80 percent of the children who are infected.
, R2 ^4 [0 h4 w5 i; HAccording to Newser, the drug is called Prochymal, and in trials, it helped a subgroup of patients, who saw an improvement in 61% to 64% of cases.: \% t% R6 _. @
Daly, the Principal Investigator of Prochymal, went on to say that:
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“As a result of Health Canada’s comprehensive review, physicians now have an off-the-shelf stem cell therapy in their arsenal to fight GvHD. Much like the introduction of antibiotics in the late 1920′s, with stem cells we have now officially taken the first step into this new paradigm of medicine.”) ]3 T% a! C' F, R" |" S# r. ~

C. Randal Mills, Ph.D., President and Chief Executive Officer of Osiris also praised Canada’s decision, stating:
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“Today is not only a great day for Osiris, but for everyone involved in the responsible development of stem cell therapies. Most importantly, today is a great day for children and their families who bravely face this horrific disease. While today marks the first approval of a stem cell drug, now that the door has been opened, it will surely not be the last.”
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Osiris Therapeutics Inc plans to seek F.D.A. approval for Prochymal later this year
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/ V! D- ^! |* i! \4 F9 CRead more at http://www.inquisitr.com/239383/canada-approves-prochymal-worlds-first-stem-cell-drug/#TasphTdxxlf5FF55.99

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一一

这是drug？真的是吗？摆货架了吗？

zhumd

虽然之前已有若干干细胞产品获批上市，相比于之前获批的干细胞产品，osiris公司的Prochymal更接近真正意义上的Drug，这也是为什么其在新闻通报上冠以 World's First Approved Stem Cell Drug标题的原因。

sunsong7

Prochymal治疗移植物抗宿主病（Graft versus Host Disease）
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7 l5 r: H) L/ ]8 y& c: G8 cProchymal is currently in clinical trials for the treatment of acute graft versus host disease (GvHD). GvHD is a life-threatening immune reaction that can occur in patients following bone marrow transplantation. Steroids are typically used to control the disease, however they are often ineffective. In patients that fail to respond to steroids, mortality can reach 85%.
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Below is a pediatric patient with severe acute GvHD of the skin that failed to respond to multiple lines of therapy including high dose steroids and other immunosuppressants. At the time of treatment with Prochymal (left), severe skin GvHD is characterized by peeling of the skin and inflammation. At five days post treatment, the inflammation is no longer present and skin regeneration is beginning to take place. At day 18, no signs of GvHD are present.# t5 G# \. y; b( Z2 \

JOEL

回复 sunsong7 的帖子/ Y0 D0 @  e2 o, P- Z$ [
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" C2 u6 a# c! q7 Y---“Prochymal 由来自于健康的青年捐献者骨髓中的间质干细胞制备而成，从一个供源的骨髓中分离出干细胞并进行扩大培养，即可获得多达1万个剂量的Prochymal。
( m! u8 }. b* S2 f1 U---Prochymal 取材于成体干细胞，因而并不涉及胚胎干细胞相关的伦理问题。”
% G8 l7 |! @! }; }---“the drug is called Prochymal, and in trials, it helped a subgroup of patients, who saw an improvement in 61% to 64% of cases.”
3 A! v4 t4 S7 E0 m9 N---"approved the treatment for ‘acute graft-versus host disease in children.’ ”
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It is a milestone of the SCR on the way to SC-Drugs, cytotherapeutic drugs, though the clinical data is not very contented.  Am autiously optimistic.

JOEL

回复 细胞海洋 的帖子
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---“Prochymal 由来自于健康的青年捐献者骨髓中的间质干细胞制备而成，从一个供源的骨髓中分离出干细胞并进行扩大培养，即可获得多达1万个剂量的Prochymal。
, X' k' P) m4 d6 D+ U0 ~---Prochymal 取材于成体干细胞，因而并不涉及胚胎干细胞相关的伦理问题。”+ Y. @" J7 N+ ^$ N& g
---“the drug is called Prochymal, and in trials, it helped a subgroup of patients, who saw an improvement in 61% to 64% of cases.”" Q/ _% ^: O8 W* D) C* m
---"approved the treatment for ‘acute graft-versus host disease in children.’ ”) d; @; `1 l) M* }% z& V
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It is a milestone of the SCR on the way to SC-Drugs, cytotherapeutic drugs, though the clinical data is not very contented.  Am autiously optimistic.

雁南飞

不错，好消息。这会给中国SFDA一定的压力,让他们不能再打酱油.

drxia

是好消息。不过SFDA还要看FDA的结论才行；再说国内医疗体制更倾向于使干细胞技术化，而不是干细胞制剂工业化生产，任重道远啊！