Flynn Aidan, O'Brien Timothy
Regenerative Medicine Institute, National Centre for Biomedical Engineering Science, National University of Ireland, Galway, Ireland.
IDrugs. 2008 Apr;11(4):283-93.
Alferminogene tadenovec is a replication-deficient human adenovirus serotype 5 that encodes human FGF4, an angiogenic protein that enhances the formation of new blood vessels. Following early clinical development by Collateral Therapeutics Inc (now Bayer Schering Pharma AG), Cardium Therapeutics Inc is currently developing alferminogene tadenovec as a potential gene therapy to improve the reperfusion of ischemic myocardium. In phase I and II clinical trials, the administration of alferminogene tadenovec was well tolerated and resulted in significant improvements in treadmill exercise capacity. However, two phase IIb/III clinical trials for the gene therapy were discontinued before completion because a high placebo response had occurred, and the trial was considered unlikely to demonstrate a benefit under the design employed. A post-hoc subgroup analysis revealed a substantial benefit from the therapy in female patients only. A phase III clinical trial is currently evaluating alferminogene tadenovec as a therapy for myocardial ischemia in women who are not candidates for revascularization. If further investigations confirm the safety and efficacy of the gene therapy, then alferminogene tadenovec may be considered a realistic therapeutic option for myocardial ischemia in selected patient populations.
阿夫米诺基因他地诺韦克是一种复制缺陷型人5型腺病毒,其编码人成纤维细胞生长因子4(FGF4),这是一种可促进新血管形成的血管生成蛋白。在抵押治疗公司(现为拜耳先灵医药公司)进行早期临床开发之后,心脏治疗公司目前正在将阿夫米诺基因他地诺韦克开发为一种潜在的基因疗法,用于改善缺血心肌的再灌注。在I期和II期临床试验中,阿夫米诺基因他地诺韦克的给药耐受性良好,并使跑步机运动能力得到显著改善。然而,该基因疗法的两项IIb/III期临床试验在完成前被中止,原因是出现了较高的安慰剂反应,并且该试验被认为在所采用的设计下不太可能显示出益处。一项事后亚组分析显示,该疗法仅对女性患者有显著益处。一项III期临床试验目前正在评估阿夫米诺基因他地诺韦克作为无法进行血运重建的女性心肌缺血治疗方法的效果。如果进一步研究证实该基因疗法的安全性和有效性,那么阿夫米诺基因他地诺韦克可能被视为特定患者群体中心肌缺血的一种切实可行的治疗选择。