干细胞疗法!治疗1型糖尿病取得积极数据

2021-10-18 Allan MedSci原创

近日,Vertex Pharmaceuticals报告了第一个参加VX-880 I/II期试验的1型糖尿病患者的三个月阳性数据,VX-880是该公司的实验性干细胞衍生的胰岛细胞替代疗法。

近日,Vertex Pharmaceuticals报告了第一个参加VX-880 I/II期试验的1型糖尿病患者的三个月阳性数据,VX-880是该公司的实验性干细胞衍生的胰岛细胞替代疗法。

VX-880是一种研究性同种异体干细胞衍生的、完全分化的、产生胰岛素的胰岛细胞疗法,使用专有技术制造。VX-880有可能通过恢复胰岛细胞功能来恢复身体调节葡萄糖水平的能力,包括葡萄糖响应胰岛素的产生。VX-880通过注入肝门静脉输送,需要长期免疫抑制治疗以保护胰岛细胞免受免疫排斥。

根据Vertex的说法,结果显示VX-880恢复了患者的胰岛素产生,该患者接受了目标剂量一半的VX-880单次输注并结合免疫抑制治疗。患者还在多项措施上取得了“快速而稳健”的益处,包括血糖控制的改善(包括 HbA1c)以及每日胰岛素需求量的减少。

该患者大约在40年前被诊断出患有T1D,并且一直依赖于外源性胰岛素。在治疗前一年,患者经历了5次严重的、可能危及生命的低血糖发作。在使用VX-880治疗之前,患者的胰岛素剂量为每天34 个单位,并且无法检测到空腹和受刺激的C肽水平,这表明患者不是自己制造胰岛素。根据研究方案,患者通过肝门静脉输注目标剂量一半的VX-880,并结合标准免疫抑制剂方案。

空腹C肽、HbA1c和7天平均每日胰岛素剂量在VX-880治疗后至第90天的不同时间间隔测量。VX-880治疗后第90天,该患者的空腹C肽为280pmol/L,反映基础胰岛素产量恢复,MMTT刺激后增加至峰值560pmol/L,表明VX-880恢复葡萄糖反应性胰岛素产量。同样在第90天,HbA1c从基线时的8.6% 提高到7.2%,每日胰岛素剂量从VX-880治疗前的每天34单位减少到7天期间的平均每天2.9单位(表1)。第90 天就诊,患者每日外源性胰岛素使用量减少了91%。

表1.患者还在多项措施上取得了“快速而稳健”的益处

Vertex细胞和基因疗法负责人Bastiano Sanna称这些结果“前所未有”,真正让它们与众不同的是,它们是在目标剂量一半的情况下实现的。虽然还为时尚早,但这些结果支持VX-880临床研究的持续进展。

 

原始出处:

https://www.firstwordpharma.com/node/1871904?tsid=28&region_id=6

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    2021-11-06 ms9000000266694635

    学习了

    0

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  4. [GetPortalCommentsPageByObjectIdResponse(id=1803244, encodeId=65ed180324499, content=<a href='/topic/show?id=7ef0e51191e' target=_blank style='color:#2F92EE;'>#积极数据#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=33, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=75119, encryptionId=7ef0e51191e, topicName=积极数据)], attachment=null, authenticateStatus=null, createdAvatar=http://thirdwx.qlogo.cn/mmopen/vi_32/Q0j4TwGTfTK17mLkWpia3T92NlEWTkEhrhZa7XPNic2JqQumEicZXGYFG6Ww5SCIl0Uq62bhhPnibAqIZDHtRva92A/132, createdBy=e0752500025, createdName=江川靖瑶, createdTime=Fri Jan 21 15:58:03 CST 2022, time=2022-01-21, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1067575, encodeId=dae0106e5753f, content=学习了, beContent=null, objectType=article, channel=null, level=null, likeNumber=66, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=c3045695343, createdName=ms9000000266694635, createdTime=Sat Nov 06 14:18:25 CST 2021, time=2021-11-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1314497, encodeId=ce64131449e53, content=<a href='/topic/show?id=4c6348e80a9' target=_blank style='color:#2F92EE;'>#干细胞疗法#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=50, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=48780, encryptionId=4c6348e80a9, topicName=干细胞疗法)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=5319428, createdName=jjjiang0199, createdTime=Wed Oct 20 13:58:03 CST 2021, time=2021-10-20, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1061731, encodeId=26791061e3190, content=学习学习, beContent=null, objectType=article, channel=null, level=null, likeNumber=88, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20210901/969603a2377544169c0528539edd5ab7/50bbe992202d43569f6cc40d89829e36.jpg, createdBy=b54c5537441, createdName=智慧药, createdTime=Tue Oct 19 09:25:49 CST 2021, time=2021-10-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1061728, encodeId=7c481061e28c1, content=学习学习<a href='/topic/show?id=23e3454e353' target=_blank style='color:#2F92EE;'>#学习#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=87, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=45473, encryptionId=23e3454e353, topicName=学习)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=97905634470, createdName=ms8000000850807940, createdTime=Tue Oct 19 09:20:27 CST 2021, time=2021-10-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1061692, encodeId=b7ce10616922c, content=学习了, beContent=null, objectType=article, channel=null, level=null, likeNumber=77, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=d1e05489924, createdName=ms1000002136354097, createdTime=Tue Oct 19 07:45:13 CST 2021, time=2021-10-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1061687, encodeId=acf9106168e74, content=重症和危重症病人达到78,678例;全球新, beContent=null, objectType=article, channel=null, level=null, likeNumber=74, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=http://thirdwx.qlogo.cn/mmopen/vi_32/Q0j4TwGTfTIrrxxDxnLic8t6EAs38Qfwo6H2h6JMleIgb9OSYf6LMnVDkQEqUXWoSDWboibjhOBI9SJDTXweqQIA/132, createdBy=50aa5321359, createdName=查查佳佳, createdTime=Tue Oct 19 07:11:06 CST 2021, time=2021-10-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1061683, encodeId=117e1061683ec, content=学习, beContent=null, objectType=article, channel=null, level=null, likeNumber=50, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f6d64838705, createdName=zhangyali10, createdTime=Tue Oct 19 07:04:01 CST 2021, time=2021-10-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1061663, encodeId=1d12106166317, content=糖尿病, beContent=null, objectType=article, channel=null, level=null, likeNumber=29, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=ece61961273, createdName=雲深無迹, createdTime=Tue Oct 19 01:19:42 CST 2021, time=2021-10-19, status=1, ipAttribution=)]
    2021-10-19 智慧药

    学习学习

    0

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    2021-10-19 ms8000000850807940

    学习学习#学习#

    0

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    2021-10-19 ms1000002136354097

    学习了

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    2021-10-19 查查佳佳

    重症和危重症病人达到78,678例;全球新

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    2021-10-19 zhangyali10

    学习

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    2021-10-19 雲深無迹

    糖尿病

    0

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