Science子刊：癌症药物或可用来治疗血管变形疾病

2016-04-07 佚名生物谷

日前发表在国际著名杂志Science Translational Medicine上的一项研究论文中，来自斯隆凯德琳癌症纪念研究中心(Memorial Sloan-Kettering)等机构的研究人员通过研究发现，一种当前在癌症患者中进行试验的药物或可被用于治疗血管畸形病。文章中，研究者对基因PIK3CA突变的小鼠进行工程化操作，PIK3CA基因和癌症发病直接相关，随后这些小鼠机体中出现了静

日前发表在国际著名杂志Science Translational Medicine上的一项研究论文中，来自斯隆凯德琳癌症纪念研究中心(Memorial Sloan-Kettering)等机构的研究人员通过研究发现，一种当前在癌症患者中进行试验的药物或可被用于治疗血管畸形病。

文章中，研究者对基因PIK3CA突变的小鼠进行工程化操作，PIK3CA基因和癌症发病直接相关，随后这些小鼠机体中出现了静脉畸形（VMs），静脉畸形的主要特点为血管变形肿胀，这些血管位于皮下，就会引发疼痛和缺陷，如果位于机体深处的话就会引发多种疾病，据估计每5000个新出生儿体中就有1人患有该病，而且目前并无有效的疗法进行治疗。

研究者对来自伦敦大学儿童健康研究所中参与皮肤遗传性研究中的13名儿童患者以及32名来自斯隆凯德琳癌症纪念研究中心的患者的样本进行遗传性检测，结果显示，大约有四分之一的患者机体中会出现和小鼠机体中相同的基因PIK3CA的突变。Sandra Castillo博士说道，雷帕霉素可以阻断基因PIK3CA下游发生的信号过程，因此其就可以阻断基因PIK3CA的效应，但却不能从根源上阻断该基因的表达；当给予小鼠雷帕霉素后，其就会表现出一定的临床效益，但在患者机体中其就会引发严重的副作用同时还会中和机体免疫反应。

随后研究人员在PIK3CA功能被直接抑制的小鼠机体中进行检测，结果显示这些药物可以很好地发挥作用并且明显减少畸形血管的尺寸。尽管PIK3CA抑制剂相比很多癌症药物而言非常特殊，但其在机体中仍然存在不可避免的副作用，而进行更为深入的研究获将为开发治疗静脉畸形疾病的新型疗法提供新的思路。

研究者Bart Vanhaesebroeck教授说道，起初我们想通过诱导小鼠机体中PIK3CA的突变来调查癌症的发生，但没有想到的是该基因却和其它疾病直接相关；本文研究支持了此前的研究结论，即相比其它细胞而言，血管对于基因PIK3CA非常敏感，此前研究者非常清楚当PIK3CA失活后会发生什么情况，但本文研究中研究者却发现，PIK3CA基因的活性突变还会引发血管的过度增长；在癌症中肿瘤需要形成新型的血管才能够生存和扩散，而阻断该过程的药物目前正在临床开发之中，而研究者也希望这样的药物可以用来治疗静脉畸形疾病。

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2016-04-14 李继凯

文章不错

143 0
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2016-04-14 李继凯

值得关注

168 0
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2016-04-09 jichang

#SCIE#

51 0
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2016-04-09 surilei

#癌症药物#

29 0
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2016-04-08 1de3b290m83(暂无匿称)

是药

79 0

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