Oncology Frontier: What is the role of targeted therapy in providing durable responses against drug resistance from mutations in myeloma?

　　《肿瘤瞭望》：对于骨髓瘤治疗，针对突变的靶向治疗效果如何？

　　Dr Anderson: Targeted therapies in myeloma that are specifically targeting mutations are not very common. In some diseases， melanoma for example， the BRAF mutation is very common and specific inhibitors such as vemurafenib can be used in malignant melanoma very commonly and can be very effective. In multiple myeloma， we haven’t really a dominant mutation. The BRAF mutation I mentioned occurs in 4-8% of patients with myeloma. There is some early data now that inhibitors such as vemurafenib can work in that subset of patients. Ongoing work will define further and further new targets and subsets of patients. Perhaps the most exciting development in myeloma though is in the immune treatments. Monoclonal antibodies， immunomodulatory drugs and even some of the newer modalities， vaccines and checkpoint inhibitor therapies， PD-1 or PD-L1， in very early experiences are very active. I mention these because the immune treatments seem to be active even across many subsets of patients with myeloma. So on the one hand， we have efforts being made to divide the disease and treat it with targeted therapies; and on the other， we have this very exciting immune treatment development that I think will be able to treat broad populations of patients with this disease.

　　Anderson博士：不常采取针对突变的靶向药物治疗骨髓瘤。某些肿瘤（比如黑素瘤）的BRAF突变很常见，BRAF抑制剂（如威罗菲尼，vemurafenib）常用于治疗恶性黑素瘤并且非常有效。还没有发现真正的多发性骨髓瘤显性突变，BRAF突变的骨髓瘤患者占4~8%，一些早期的数据显示BRAF抑制剂（如威罗菲尼）对这类患者亚群有效。目前一些研究正在确认新靶点和和患者亚群。也许最令人兴奋的骨髓瘤治疗进展是免疫疗法：目前免疫疗法（单克隆抗体和免疫调节药物，甚至一些新治疗方式如疫苗和PD-1/PD-L1检查点抑制剂）治疗MM的经验不多但是疗效令人期待，免疫治疗能对多个患者亚群起效。所以，目前研究者正在研究MM的各个亚型，对不同亚型的患者亚群采取不同的靶向疗法，免疫治疗的疗效令人振奋，我认为这种疗法可以治疗广泛的患者人群。