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儿童侵袭性脑肿瘤的新疗法

2017-04-12 MedSci MedSci原创

儿童侵袭性脑肿瘤新疗法

科学家利用最先进的基因编辑技术发现了一个非典型畸胎样/横纹肌样瘤（AT/RT）的潜在治疗靶点。AT/RT是一种高度侵袭性和耐药的脑肿瘤，常在婴幼儿期发病。

科学家利用最先进的基因编辑技术发现了一个非典型畸胎样/横纹肌样瘤（AT/RT）的潜在治疗靶点。AT/RT是一种高度侵袭性和耐药的脑肿瘤，常在婴幼儿期发病。


研究人员发现这些肿瘤的生长和转移趋势受Polo样激酶4（PLK4）蛋白激酶的调节，该酶的水平在AT / RT中升高。他们还证明了一种实验药物，PLK4抑制剂，可以阻止肿瘤生长。研究结果发表在Pediatric Blood & Cancer上。

“这是首次将PLK4作为脑肿瘤或儿科癌症的治疗靶点。”西北大学费因伯格医学院和斯坦利Manne儿研所Lurie儿童癌症研究院小儿神经外科副教授Simone T. Sredni博士说道。

Sredni及其团队利用CRISPR / Cas9新型基因编辑技术鉴定PLK4作为治疗的潜在目标。CRISPR / Cas9基因编辑技术能够突变单个激酶基因——细胞功能的关键调节因子——从而揭示对肿瘤细胞生长影响最显着的激酶。然后，利用相应的激酶抑制剂靶向该激酶，目前正在进行乳腺癌的测试。

他们还发现PLK4抑制剂 (CFI-400945)只攻击肿瘤细胞，对正常组织是安全的。“我们使用的Plk4抑制剂能明显抑制肿瘤的增殖、存活、侵袭和迁移，而对正常细胞无影响。”Sredni说。“这可能是治疗AT / RT和可能的其他儿科脑肿瘤的新方法”。

科学家将斑马鱼幼鱼长时间暴露于极高剂量的药物下测试药物的安全性。他们观察到这种药物对鱼的发育无影响，这意味着它可能能安全地运用到儿科患者身上。

Sredni说：“这也可能是精准医疗的一次机会，因为我们可以根据PLK4在肿瘤中的表达水平来施予不同的治疗方法。

该组正在进行该药物在AT / RT以及其他类型的脑肿瘤的动物实验。 Sredni希望能快速进入一期临床试验。

原文出处：https://www.luriechildrens.org/en-us/news-events/Pages/new_potential_treatment_for_agressive_brain_cancer_in_children_450.aspx?utm_medium=lc.org&utm_source=homepage&utm_campaign=sredni-braintumor&utm_term=#REF!&utm_content=research

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    2017-04-14 sunyl07

    #侵袭性#

    0

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    2017-04-14 839640779

    #脑肿瘤#

    0

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    2017-04-14 kcb074

    #新疗法#

    0