英國(guó)《新科學(xué)家》雜志,、BBC等媒體6月7日（北京時(shí)間）報(bào)道,，在日前舉行的美國(guó)臨床腫瘤學(xué)會(huì)2011年年會(huì)上公布的兩種新藥能減緩腫瘤惡化,，顯著提高晚期黑色素瘤患者存活率,，被譽(yù)為30年來(lái)皮膚癌治療領(lǐng)域的最大突破,。相關(guān)論文發(fā)表在《新英格蘭醫(yī)學(xué)雜志》（NEJM）上,。
黑色素瘤是出現(xiàn)在黑色素細(xì)胞中的一種惡性腫瘤，如果發(fā)現(xiàn)早還可以治愈,，但如果已發(fā)展到晚期,，患者預(yù)期存活時(shí)間平均只有6個(gè)月。此前,，唯一有效的治療方法是在腫瘤厚度小于1毫米時(shí)進(jìn)行手術(shù)切除,。
其中的一種新藥名為“vemurafenib”，由美國(guó)斯隆凱特琳癌癥中心的保羅·查普曼帶領(lǐng)的團(tuán)隊(duì)研發(fā),。惡性皮膚癌病例中一半與BRAF基因變異反應(yīng)相關(guān),，該藥可抑制BRAF基因變異反應(yīng),，藥效優(yōu)于目前在治療轉(zhuǎn)移性黑色素瘤中最常使用的化療藥物達(dá)卡巴嗪。查普曼團(tuán)隊(duì)在臨床實(shí)驗(yàn)中,，將675名無(wú)法通過手術(shù)治愈的晚期黑色素瘤患者分為兩組,，一組自愿服用新藥，另一組自愿接受傳統(tǒng)療法——在化療的同時(shí)服用達(dá)卡巴嗪,。6個(gè)月后,，服用新藥小組存活率高達(dá)84%，而另一組的存活率僅為64%,。由于效果顯著,，研究人員果斷提前停止了實(shí)驗(yàn)，為所有參與實(shí)驗(yàn)的患者都換上了這種新藥,。
為該項(xiàng)研究提供資助的羅氏制藥公司全球發(fā)展部主管哈爾·巴龍?jiān)诼暶髦姓f,，這是黑色素瘤治療領(lǐng)域的一項(xiàng)重要成果，“vemurafenib”不僅延長(zhǎng)了患者的壽命,，更減少了黑色素瘤惡化的風(fēng)險(xiǎn),，同時(shí)也使腫瘤出現(xiàn)了縮小。
在該次會(huì)議上公布的另一項(xiàng)成果也讓人歡欣鼓舞,。由施貴寶制藥公司研制的一種名為“Ipilumumab”的抗體藥物在與達(dá)卡巴嗪配合使用時(shí)可提高后者的療效,，該抗體藥物能通過刺激免疫系統(tǒng)的方式對(duì)腫瘤產(chǎn)生作用。研究人員發(fā)現(xiàn),，使用復(fù)合療法的250名黑色素瘤患者中有28.5%的存活時(shí)間超過了兩年,，而只采用達(dá)卡巴嗪療法的患者中兩年存活率只有17.9%。
在看到該藥物的早期研究結(jié)果后,，美國(guó)食品和藥物管理局已于3月批準(zhǔn)該藥在晚期黑色素瘤患者中進(jìn)行使用,。歐洲藥品管理局也將該藥提上了審批日程，預(yù)計(jì)8月前獲批,。
英國(guó)癌癥研究所首席醫(yī)師彼得·約翰遜說，這是晚期黑色素瘤治療上的一項(xiàng)重大突破,，是首次獲得一項(xiàng)有實(shí)質(zhì)療效的療法,。巴龍說：“我們已經(jīng)在治療轉(zhuǎn)移性黑色素瘤上獲得了一個(gè)顯著成果，下一步我們還將對(duì)其做進(jìn)一步的完善,，并期待與施貴寶公司合作,，為患者提供更多新選擇。”（生物谷Bioon.com）
專題：MicroRNA 和 癌癥
生物谷推薦原文出處：
The New England Jourmal of Medicine DOI:10.1056/NEJMoa1103782
Improved Survival with Vemurafenib in Melanoma with BRAF V600E Mutation
Paul B. Chapman, M.D., Axel Hauschild, M.D., Caroline Robert, M.D., Ph.D., John B. Haanen, M.D., Paolo Ascierto, M.D., James Larkin, M.D., Reinhard Dummer, M.D., Claus Garbe, M.D., Alessandro Testori, M.D., Michele Maio, M.D., David Hogg, M.D., Paul Lorigan, M.D., Celeste Lebbe, M.D., Thomas Jouary, M.D., Dirk Schadendorf, M.D., Antoni Ribas, M.D., Steven J. O'Day, M.D., Jeffrey A. Sosman, M.D., John M. Kirkwood, M.D., Alexander M.M. Eggermont, M.D., Ph.D., Brigitte Dreno, M.D., Ph.D., Keith Nol
Background Phase 1 and 2 clinical trials of the BRAF kinase inhibitor vemurafenib (PLX4032) have shown response rates of more than 50% in patients with metastatic melanoma with the BRAF V600E mutation. Full Text of Background... Methods We conducted a phase 3 randomized clinical trial comparing vemurafenib with dacarbazine in 675 patients with previously untreated, metastatic melanoma with the BRAF V600E mutation. Patients were randomly assigned to receive either vemurafenib (960 mg orally twice daily) or dacarbazine (1000 mg per square meter of body-surface area intravenously every 3 weeks). Coprimary end points were rates of overall and progression-free survival. Secondary end points included the response rate, response duration, and safety. A final analysis was planned after 196 deaths and an interim analysis after 98 deaths. Full Text of Methods... Results At 6 months, overall survival was 84% (95% confidence interval [CI], 78 to 89) in the vemurafenib group and 64% (95% CI, 56 to 73) in the dacarbazine group. In the interim analysis for overall survival and final analysis for progression-free survival, vemurafenib was associated with a relative reduction of 63% in the risk of death and of 74% in the risk of either death or disease progression, as compared with dacarbazine (P<0.001 for both comparisons). After review of the interim analysis by an independent data and safety monitoring board, crossover from dacarbazine to vemurafenib was recommended. Response rates were 48% for vemurafenib and 5% for dacarbazine. Common adverse events associated with vemurafenib were arthralgia, rash, fatigue, alopecia, keratoacanthoma or squamous-cell carcinoma, photosensitivity, nausea, and diarrhea; 38% of patients required dose modification because of toxic effects. Full Text of Results... Conclusions Vemurafenib produced improved rates of overall and progression-free survival in patients with previously untreated melanoma with the BRAF V600E mutation.
