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NEJM:初步研究表明idelalisib可有效治疗转移性Merkel细胞瘤

2015-10-17 sunshine 译 MedSci原创

1

识别患者对现有药物的基因突变可清除患者已转移的Merkel细胞瘤。在没有有效的治疗下,转移性Merkel细胞瘤(Merkelcellcarcinoma,MCC)对患者来说无疑是致命的。

本研究作者Ling Gao博士,来自于阿肯色大学医学院,其86岁的女性患者于2013年诊断为IIIB Merkel细胞瘤,部位为右侧的太阳穴处。2013年5月患者接受了手术及放射治疗,并与2014年11月再次接受了手术治疗,医生确诊患者体内癌细胞已发生了转移。

Gao博士是一名皮肤科医生,治疗了大量来自阿肯色州及其周边国家的Merkel细胞瘤患者。她在Merkel癌细胞上进行了遗传学试验,并发现癌细胞存在多个基因突变,包括PI3Kδ。

Idelalisib是一种新型的PI3K通路抑制剂,已经FDA批准用于治疗B细胞淋巴瘤。Gao博士发现,近期的研究表明PI3Kδ突变使得机体可启动一个有效对抗肿瘤的免疫反应。

在其实验室研究以及对idelalisib认识的基础上,2015年6月Gao开始使用idelalisib治疗患者。经过3个月的治疗,Gao发现,患者的肝脏中肿瘤的迹象消失。

基于自己所掌握的知识,Gao博士认为,此病例可为PI3Kδ做进一步的研究,正如idelalisib用于实性肿瘤的治疗,而非仅仅限于血液肿瘤。

Gao表示,她们的研究中,Idelalisib对患者的治疗效果为靶向PI3Kδ,为治疗Merkel细胞瘤提供了初步的临床证据。

原始出处:

Researchers' cure of metastatic skin cancer revealed. Science Daily, October 16, 2015.

Mallory B. Shiver, Fade Mahmoud, Ling Gao. Response to Idelalisib in a Patient with Stage IV Merkel-Cell Carcinoma. New England Journal of Medicine, 2015; 373 (16): 1580 DOI: 10.1056/NEJMc1507446


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    2016-05-14 chenlianhui
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