急性髓性白血病是血液病的一種，一直是很多醫(yī)學(xué)研究進(jìn)行討論的一種疾病，較近有好消息爆出，Agios公司研發(fā)的IDH1突變蛋白抑制劑AG-120，被FDA火速批準(zhǔn)用于急性髓性白血病，實(shí)在令人驚喜，這個(gè)藥具體是怎么回事呢？馬上來一起看看吧：

話說Agios公司開發(fā)出IDH1突變蛋白抑制劑AG-120好幾年了，沒想到因?yàn)橐黄谂R床試驗(yàn)結(jié)果良好，F(xiàn)DA居然就直接批準(zhǔn)用于臨床了，實(shí)在是讓我吃驚。IDH1是個(gè)三羧酸循環(huán)代謝酶，第120位的氨基酸突變成組氨酸后產(chǎn)生新功能，產(chǎn)生致癌代謝物誘發(fā)白血病和腦癌。所以對(duì)于非靶向藥物，只要藥物殺不死癌細(xì)胞，就無法病愈，這是很淺顯的道理。
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于是Agios公司一直致力于開發(fā)針對(duì)IDH1/IDH2這兩個(gè)突變酶的抑制劑，分別是AG-120和AG-221. AG-221前不久已經(jīng)被FDA批準(zhǔn)，我有文章介紹， 今天說的是IDH1 突變體抑制劑 AG-120， 商品名Ivosidenib.

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這1期臨床試驗(yàn)發(fā)表在今年6月的新英格蘭醫(yī)學(xué)雜志上：

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先看看英文的結(jié)果：

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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.

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中文簡述： 125名病人中， 總效果對(duì)比41.6%, 35%的病人不在需要輸血，34名病愈的病人中有7位體內(nèi)檢查不到攜帶IDH1突變的癌細(xì)胞。

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如此驚艷的1期臨床試驗(yàn)結(jié)果才是FDA火速批準(zhǔn)的原因，救人如救火，什么時(shí)候CFDA的老爺們有如此快的速度就好了。