急性髓性白血病是血液病的一种，一直是很多医学研究进行讨论的一种疾病，较近有好消息爆出，Agios公司研发的IDH1突变蛋白抑制剂AG-120，被FDA火速批准用于急性髓性白血病，实在令人惊喜，这个药具体是怎么回事呢？马上来一起看看吧：

话说Agios公司开发出IDH1突变蛋白抑制剂AG-120好几年了，没想到因为一期临床试验结果良好，FDA居然就直接批准用于临床了，实在是让我吃惊。IDH1是个三羧酸循环代谢酶，第120位的氨基酸突变成组氨酸后产生新功能，产生致癌代谢物诱发白血病和脑癌。所以对于非靶向药物，只要药物杀不死癌细胞，就无法病愈，这是很浅显的道理。
 

 

于是Agios公司一直致力于开发针对IDH1/IDH2这两个突变酶的抑制剂，分别是AG-120和AG-221. AG-221前不久已经被FDA批准，我有文章介绍， 今天说的是IDH1 突变体抑制剂 AG-120， 商品名Ivosidenib.

 

这1期临床试验发表在今年6月的新英格兰医学杂志上：

 

先看看英文的结果：

 

In the primary efficacy population (125 patients), the rate of complete remission or complete remission with partial hematologic recovery was 30.4% (95% confidence interval [CI], 22.5 to 39.3), the rate of complete remission was 21.6% (95% CI, 14.7 to 29.8), and the overall response rate was 41.6% (95% CI, 32.9 to 50.8). The median durations of these responses were 8.2 months (95% CI, 5.5 to 12.0), 9.3 months (95% CI, 5.6 to 18.3), and 6.5 months (95% CI, 4.6 to 9.3), respectively. Transfusion independence was attained in 29 of 84 patients (35%), and patients who had a response had fewer infections and febrile neutropenia episodes than those who did not have a response. Among 34 patients who had a complete remission or complete remission with partial hematologic recovery, 7 (21%) had no residual detectableIDH1mutations on digital polymerase-chain-reaction assay.

 

中文简述： 125名病人中， 总效果对比41.6%, 35%的病人不在需要输血，34名病愈的病人中有7位体内检查不到携带IDH1突变的癌细胞。

 

如此惊艳的1期临床试验结果才是FDA火速批准的原因，救人如救火，什么时候CFDA的老爷们有如此快的速度就好了。