英國《新科學家》雜志,、BBC等媒體6月7日（北京時間）報道，在日前舉行的美國臨床腫瘤學會2011年年會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，被譽為30年來皮膚癌治療領域的最大突破,。相關論文發(fā)表在《新英格蘭醫(yī)學雜志》（NEJM）上,。
黑色素瘤是出現(xiàn)在黑色素細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,，患者預期存活時間平均只有6個月,。此前,，唯一有效的治療方法是在腫瘤厚度小于1毫米時進行手術切除。
其中的一種新藥名為“vemurafenib”,，由美國斯隆凱特琳癌癥中心的保羅·查普曼帶領的團隊研發(fā),。惡性皮膚癌病例中一半與BRAF基因變異反應相關，該藥可抑制BRAF基因變異反應,，藥效優(yōu)于目前在治療轉移性黑色素瘤中最常使用的化療藥物達卡巴嗪,。查普曼團隊在臨床實驗中,，將675名無法通過手術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——在化療的同時服用達卡巴嗪,。6個月后，服用新藥小組存活率高達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驗,，為所有參與實驗的患者都換上了這種新藥,。
為該項研究提供資助的羅氏制藥公司全球發(fā)展部主管哈爾·巴龍在聲明中說，這是黑色素瘤治療領域的一項重要成果,，“vemurafenib”不僅延長了患者的壽命,，更減少了黑色素瘤惡化的風險，同時也使腫瘤出現(xiàn)了縮小,。
在該次會議上公布的另一項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”的抗體藥物在與達卡巴嗪配合使用時可提高后者的療效，該抗體藥物能通過刺激免疫系統(tǒng)的方式對腫瘤產生作用,。研究人員發(fā)現(xiàn),，使用復合療法的250名黑色素瘤患者中有28.5%的存活時間超過了兩年，而只采用達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果后,，美國食品和藥物管理局已于3月批準該藥在晚期黑色素瘤患者中進行使用。歐洲藥品管理局也將該藥提上了審批日程,，預計8月前獲批,。
英國癌癥研究所首席醫(yī)師彼得·約翰遜說，這是晚期黑色素瘤治療上的一項重大突破,，是首次獲得一項有實質療效的療法,。巴龍說：“我們已經在治療轉移性黑色素瘤上獲得了一個顯著成果，下一步我們還將對其做進一步的完善,，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.
