打开APP

美国癌症研究中心筛选失败药物用于治疗特殊基因型患者群体

  1. NCI
  2. TSC1
  3. 癌症
  4. 药物研发

来源:生物谷 2014-04-14 08:56

美国国家癌症研究中心筛选失败药物用于治疗特殊基因型患者群体

2014年4月13日讯 /生物谷BIOON/ --去年美国国家癌症研究中心(National Cancer Institute, NCI)宣布开始一项研究旨在重新分析过去十年中在临床试验中未能对大多数患者产生疗效的抗癌药物以用于治疗一些特殊基因型的癌症患者。纽约的Memorial Sloan-Kettering Cancer Center、波士顿的Dana-Farber Cancer Institute和Massachusetts General Hospital以及麻省坎布里奇市的Broad Institute都参与到这项研究中。

根据NCI的定义,特殊反应患者是在临床上对药物能够做出完全的反应或是在至少6个月的治疗中做出部分反应的癌症患者,而这种药物对普通患者的响应率则在10%以下。

基于以上设想,NCI的研究人员进行了一项利用everolimus (Afinitor)和pazopanib (Votrient)联合治疗晚期膀胱癌特殊反应患者的临床一期研究。结果显示其中一名患者对这种疗法做出了完全响应,而另外几名患者则出现了部分响应的表现。通过对这些患者的基因进行分析,研究人员还发现一个名为TSC1的基因型突变可能是这一现象的原因。

尽管这些发现令人振奋,但是研究人员首先还是要搞清楚特定人群响应某种药物的基因机制才能进一步有针对性的开发药物。(生物谷Bioon.com)

详细英文报道:

Last year the National Cancer Institute announced the launch of an initiative meant to examine failed clinical trials in search of "exceptional responders": patients who responded well to drugs that the bulk of the trial population didn't.

Now, after sifting through 10 years' worth of clinical trials, NCI's Cancer Diagnosis Program has identified about 100 of these unique patients so far, according to Bloomberg.

Memorial Sloan-Kettering Cancer Center in New York, Dana-Farber Cancer Institute and Massachusetts General Hospital in Boston and the Broad Institute in Cambridge, MA, have teamed up with NCI to look for these outlier patients and eventually establish a national database of patient data for researchers.

The NCI defines exceptional responders as cancer patients who had a complete response or partial response to treatment for at least 6 months in a clinical trial in which less than 10% of patients responded.

One such patient was the subject of a study published in March in Cancer Discovery. The patient, who had advanced bladder cancer, experienced a complete response for 14 months to the drug combination everolimus (Afinitor) and pazopanib (Votrient) in a Phase I trial. In the trial, investigators recruited 9 patients with advanced solid tumors, including 5 with bladder cancer, whose diseases had progressed despite treatment with standard therapies. Patients received one to 13 cycles of everolimus and pazopanib.

One of 5 of the bladder cancer patients had a complete response to the treatment, and genomic profiling of his tumor revealed two unique sequences that may have caused this exceptional response. Investigators identified a mutation in a gene called TSC1 that may have led to the result. The same gene mutation was found in several other patients in the clinical trial, including two who had minor responses to everolimus. The findings indicate that everolimus may be used to treat bladder cancer in patients with these specific gene mutations.

Exceptional responders such as these may provide the impetus to resurrect other so-called failed drugs for use in certain patients. But first, scientists need to understand the genetic mechanisms at work in outlying cases. Then, investigators can work toward finding hypertargeted anticancer therapies that could improve patient selection for certain treatments as well as boost patient response to therapy and advance new treatment regimens.

 

版权声明 本网站所有注明“来源:生物谷”或“来源:bioon”的文字、图片和音视频资料,版权均属于生物谷网站所有。非经授权,任何媒体、网站或个人不得转载,否则将追究法律责任。取得书面授权转载时,须注明“来源:生物谷”。其它来源的文章系转载文章,本网所有转载文章系出于传递更多信息之目的,转载内容不代表本站立场。不希望被转载的媒体或个人可与我们联系,我们将立即进行删除处理。

87%用户都在用生物谷APP 随时阅读、评论、分享交流 请扫描二维码下载->