2015年2月3日讯 /生物谷BIOON/ --自从上世纪末人类开始基因组计划之后，科学家们一直希望有一天可以通过编辑修饰患者的致病基因从根本上治愈疾病。随着一代又一代科研工作者的努力这一梦想正在一步一步向现实转变。最近美国基因疗法的先驱Bluebird公司表示公司开发的用于治疗β-地中海贫血的基因治疗方案LentiGlobin BB305已经获得了FDA的突破性药物地位认证。这也是继罗氏公司的PD-L1药物MPDL3280A之后FDA今年授予的第二个突破性药物认证。
Bluebird公司方面为FDA做出的这一决定表示欢迎。一方面这再次奠定了公司在基因疗法方面领跑者的地位，另一方面也向市场表明了向患者体内输送修正基因以治疗疾病的可行性和安全性。负责这一研究的Dr. David Davidson表示目前获得的早期临床研究中，这种药物对于多种基因型的地中海贫血患者都起到了良好的治疗效果。
位于麻省坎布里奇市的Bluebird公司一直是基因疗法开发的领头羊，LentiGlobin BB305是利用慢病毒载体将修正后的β-球蛋白基因导入到由患者体内分离而来的造血干细胞中，然后将这些基因重组的造血干细胞经过扩增再回输至患者体内，以恢复患者正常合成血红蛋白的能力。这一疗法能够使地中海贫血患者彻底摆脱输血维持的局面。而就在去年12月份，Bluebird公司在美国血液学会上公布了接受这种疗法的四名患者已经在未输血治疗的条件下正常生活了三个月以上。
而在稍早时候，制药巨头罗氏公司也获得了FDA授予其开发的PD-L1药物MPDL3280A用于治疗非细小细胞肺癌的突破性药物认证。此前这种药物已经被批准用于治疗膀胱癌。近年来，FDA一直在努力采取各种措施加速新型药物的批准过程，而突破性药物认证的设计就是基于这一考虑。在过去几年中，FDA已经向十数种药物颁发了这一认证，大大加速了这些特效药物的批准速度。
详细英文报道：
Bluebird bio nabbed the FDA's breakthrough drug title for LentiGlobin BB305 as a new treatment for beta-thalassemia major, putting one of the industry's top experimental gene therapies back squarely in a promising spotlight. The biotech put out the news alongside Roche's announcement that its PD-L1 checkpoint inhibitor MPDL3280A picked up its second breakthrough drug designation, this time for non-small cell lung cancer.
Bluebird's ($BLUE) BTD win helps solidify its rep as one of the leading gene therapy developers in biotech. The biotech believes that the scientific roller coaster ride in gene therapy work is over and that its lead therapy for beta-thalassemia and sickle cell diseases will prove that you can safely insert a corrective gene into patients. And their approach has drawn some positive attention at the agency.
"Our early clinical data investigating the use of LentiGlobin in patients with multiple genotypes of beta-thalassemia major, including beta-0/beta-0, the most severe genotype, are very encouraging, and we remain on track to complete enrollment in the Northstar and HGB-205 studies in 2015," says Dr. David Davidson, chief medical officer of bluebird bio, in a statement. "In light of the breakthrough designation, we look forward to working even more closely with the FDA to expedite the development of LentiGlobin for the treatment of beta-thalassemia major."
Cambridge, MA-based Bluebird has been testing an improved lentiviral vector needed to get a corrective beta-globin gene in place to repair patients' ability to produce hemoglobin and wean them off the blood transfusions needed to stay alive.
Just last December bluebird noted in preliminary results presented at the annual the American Society of Hematology meeting that the first four patients treated with bluebird's therapy remain transfusion-free after at least three months follow up, affirming the treatment's promise as a functional cure for the disease. That update added two more transfusion-free patients to the two bluebird disclosed over the summer, news that sent its shares up more than 50% in June.
Roche, meanwhile, added boasting rights for a second BTD on its PD-L1 drug, which had earlier won a nod for bladder cancer. The pharma giant has been pushing along its checkpoint inhibitor, designed to unleash an immune system attack, behind the lead drugs from Bristol-Myers Squibb and Merck, which are both pushing hard on lung cancer programs of their own. Lung cancer is considered a major market opportunity for the leading players in this field, and Roche has been mixing and matching cancer therapies for an ambitious in-house program.
Over the last two years the FDA has handed out dozens of these breakthrough designations to drug developers, promising to work closely with researchers to help speed trial development and advance cutting-edge therapies to a final marketing decision. Over that time a number of developers have noted that the FDA's new program has helped accelerate their work, shaving time out of the process. And several of these drugs in cancer have gone on to accelerated approval.