英国《新科学家》杂志、BBC等媒体6月7日（北京时间）报道，在日前举行的美国临床肿瘤学会2011年年会上公布的两种新药能减缓肿瘤恶化，显著提高晚期黑色素瘤患者存活率，被誉为30年来皮肤癌治疗领域的最大突破。相关论文发表在《新英格兰医学杂志》（NEJM）上。
黑色素瘤是出现在黑色素细胞中的一种恶性肿瘤，如果发现早还可以治愈，但如果已发展到晚期，患者预期存活时间平均只有6个月。此前，唯一有效的治疗方法是在肿瘤厚度小于1毫米时进行手术切除。
其中的一种新药名为“vemurafenib”，由美国斯隆凯特琳癌症中心的保罗·查普曼带领的团队研发。恶性皮肤癌病例中一半与BRAF基因变异反应相关，该药可抑制BRAF基因变异反应，药效优于目前在治疗转移性黑色素瘤中最常使用的化疗药物达卡巴嗪。查普曼团队在临床实验中，将675名无法通过手术治愈的晚期黑色素瘤患者分为两组，一组自愿服用新药，另一组自愿接受传统疗法——在化疗的同时服用达卡巴嗪。6个月后，服用新药小组存活率高达84%，而另一组的存活率仅为64%。由于效果显著，研究人员果断提前停止了实验，为所有参与实验的患者都换上了这种新药。
为该项研究提供资助的罗氏制药公司全球发展部主管哈尔·巴龙在声明中说，这是黑色素瘤治疗领域的一项重要成果，“vemurafenib”不仅延长了患者的寿命，更减少了黑色素瘤恶化的风险，同时也使肿瘤出现了缩小。
在该次会议上公布的另一项成果也让人欢欣鼓舞。由施贵宝制药公司研制的一种名为“Ipilumumab”的抗体药物在与达卡巴嗪配合使用时可提高后者的疗效，该抗体药物能通过刺激免疫系统的方式对肿瘤产生作用。研究人员发现，使用复合疗法的250名黑色素瘤患者中有28.5%的存活时间超过了两年，而只采用达卡巴嗪疗法的患者中两年存活率只有17.9%。
在看到该药物的早期研究结果后，美国食品和药物管理局已于3月批准该药在晚期黑色素瘤患者中进行使用。欧洲药品管理局也将该药提上了审批日程，预计8月前获批。
英国癌症研究所首席医师彼得·约翰逊说，这是晚期黑色素瘤治疗上的一项重大突破，是首次获得一项有实质疗效的疗法。巴龙说：“我们已经在治疗转移性黑色素瘤上获得了一个显著成果，下一步我们还将对其做进一步的完善，并期待与施贵宝公司合作，为患者提供更多新选择。”（生物谷Bioon.com）
专题：MicroRNA 和 癌症
生物谷推荐原文出处：
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.
