这项研究也让辉瑞公司鼓足勇气再次向FDA递交了批准申请。FDA将在几周内进行讨论。
正如Meshinchi教授所说:「我们想要一种能治愈一切的药物,这种想法是及其错误的。过去,该药被证明是失败的,或许失败的不是药物本身,而是我们没有选对合适的患者。」[7]
看到徒弟的最新研究成果,Bernstein教授甚是欣慰:「此结果将会让这个药重获新生!在有生之年还能看到这样好的结果,我真是高兴啊!」[7]
回顾了几十年的坎坷历程,Appelbaum 教授也感慨道:「我想,我们从这些经历中收获了许多,开发这个药物是令人着迷的,但有时又真的令人沮丧!我们希望好的结果,能够看到GO可以治愈某些患者,真是欣慰!」[7]
Meshinchi教授认为,现在已有足够的证据证明GO可以作为标准疗法,用于治疗那些能够表达完整CD33蛋白的AML患者。并且,还可以利用他们发现的这个特殊基因位点,去预测哪些患者接受GO治疗效果会更好,他们已经开发出了针对这个特殊基因位点的快速检测方法。
接下来,Meshinchi教授及其团队将进行一个大型的AML临床试验,旨在利用GO治疗能够产生完整CD33蛋白的AML患者。
Meshinchi教授也希望其他癌症研究人员能从GO的故事中得到启示:药物的研发过程中,选对受益的患者人群是很重要的,这样才能给患者带来革命性的一大步!
写到最后,奇点糕不禁有两点感想。一是想跟还坚持在制药行业里的朋友们说:你们做的是为人类疾控事业谋福利的大事,要坚持住!说不定多年以后你们也可以成为大牛,到时候奇点糕也可以写你们的故事。二是想跟还在实验室里奋斗的硕硕博博们说:报答师恩最好的方式,或许就是超越你导,完成你导尚未完成的事业!加油哦!
参考资料:
[1] Lamba JK, Chauhan L, Shin M, Loken MR, Pollard JA, et al. 2017. CD33 Splicing Polymorphism Determines Gemtuzumab Ozogamicin Response in De Novo Acute Myeloid Leukemia: Report From Randomized Phase III Children's Oncology Group Trial AAML0531. Journal of clinical oncology : official journal of the American Society of Clinical Oncology:Jco2016712513
[2] Bross PF, Beitz J, Chen G, Chen XH, Duffy E, et al. 2001. Approval summary: gemtuzumab ozogamicin in relapsed acute myeloid leukemia. Clinical cancer research : an official journal of the American Association for Cancer Research 7:1490-6
[3] Petersdorf SH, Kopecky KJ, Slovak M, Willman C, Nevill T, et al. 2013. A phase 3 study of gemtuzumab ozogamicin during induction and postconsolidation therapy in younger patients with acute myeloid leukemia. Blood 121:4854-60
[4] Almeida A M, Ramos F. Acute myeloid leukemia in the older adults[J]. Leukemia research reports, 2016, 6: 1-7.
[5]http://www.chinablood.com.cn/system/2008/09/26/010014166.shtml
[6] Estey E, Dohner H. 2006. Acute myeloid leukaemia. Lancet (London, England) 368:1894-907
[7]https://www.fredhutch.org/en/news/center-news/2017/06/mylotarg-precision-medicine.html
[8] Andrews RG, Torok-Storb B, Bernstein ID. 1983. Myeloid-associated differentiation antigens on stem cells and their progeny identified by monoclonal antibodies. Blood 62:124-32
[9] Strebhardt K, Ullrich A. 2008. Paul Ehrlich’s magic bullet concept: 100 years of progress. Nat Rev Cancer 8: 473-80
[10] Dinndorf PA, Andrews RG, Benjamin D, Ridgway D, Wolff L, Bernstein ID. 1986. Expression of normal myeloid-associated antigens by acute leukemia cells. Blood 67:1048-53
[11] Appelbaum FR, Matthews DC, Eary JF, Badger CC, Kellogg M, et al. 1992. The use of radiolabeled anti-CD33 antibody to augment marrow irradiation prior to marrow transplantation for acute myelogenous leukemia. Transplantation 54:829-33
[12] Maiese WM, Lechevalier MP, Lechevalier HA, Korshalla J, Kuck N, et al. 1989. Calicheamicins, a novel family of antitumor antibiotics: taxonomy, fermentation and biological properties. The Journal of antibiotics 42:558-63
[13] Sievers EL, Appelbaum FR, Spielberger RT, Forman SJ, Flowers D, et al. 1999. Selective ablation of acute myeloid leukemia using antibody-targeted chemotherapy: a phase I study of an anti-CD33 calicheamicin immunoconjugate. Blood 93:3678-84
[14] Rowe JM, Lowenberg B. 2013. Gemtuzumab ozogamicin in acute myeloid leukemia: a remarkable saga about an active drug. Blood 121:4838-41
[15] Pollard JA, Loken M, Gerbing RB, Raimondi SC, Hirsch BA, et al. 2016. CD33 Expression and Its Association With Gemtuzumab Ozogamicin Response: Results From the Randomized Phase III Children's Oncology Group Trial AAML0531. Journal of clinical oncology : official journal of the American Society of Clinical Oncology 34:747-55