Migliaccio Anna Rita
Altius Institute for Biomedical Sciences, Seattle, WA 98121, USA.
Biomolecules. 2024 Mar 27;14(4):408. doi: 10.3390/biom14040408.
The identification of the hormone erythropoietin (EPO), which regulates red blood cell production, and its development into a pharmaceutical-grade product to treat anemia has been not only a herculean task but it has also been the first of its kind. As with all the successes, it had "winners" and "losers", but its history is mostly told by the winners who, over the years, have published excellent scientific and divulgate summaries on the subject, some of which are cited in this review. In addition, "success" is also due to the superb and dedicated work of numerous "crew" members, who often are under-represented and under-recognized when the story is told and often have several "dark sides" that are not told in the polished context of most reviews, but which raised the need for the development of the current legislation on biotherapeutics. Although I was marginally involved in the clinical development of erythropoietin, I have known on a personal basis most, if not all, the protagonists of the saga and had multiple opportunities to talk with them on the drive that supported their activities. Here, I will summarize the major steps in the development of erythropoietin as the first bioproduct to enter the clinic. Some of the "dark sides" will also be mentioned to emphasize what a beautiful achievement of humankind this process has been and how the various unforeseen challenges that emerged were progressively addressed in the interest of science and of the patient's wellbeing.
调节红细胞生成的激素促红细胞生成素(EPO)的鉴定及其发展成为治疗贫血的医药级产品,不仅是一项艰巨的任务,而且也是同类中的首例。与所有成功一样,它有“赢家”和“输家”,但其历史大多由赢家讲述,多年来,他们发表了关于该主题的优秀科学和普及性综述,本文引用了其中一些。此外,“成功”还归功于众多“团队成员”的卓越和专注工作,在讲述这个故事时,他们往往代表性不足且未得到认可,而且在大多数综述的完美背景中,他们往往有几个“阴暗面”未被提及,但正是这些“阴暗面”引发了当前生物治疗立法的发展需求。尽管我只是略微参与了促红细胞生成素的临床开发,但我个人认识了这个传奇故事的大多数(如果不是全部)主角,并且有多次机会在推动他们活动的动力方面与他们交谈。在这里,我将总结促红细胞生成素作为首个进入临床的生物制品的主要开发步骤。还将提及一些“阴暗面”,以强调这一过程是人类多么了不起的成就,以及为了科学和患者福祉,如何逐步应对出现的各种意外挑战。