School of Pharmacy and Pharmaceutical Sciences, Binghamton University, P.O. Box 6000, Binghamton, NY 13902, United States.
School of Pharmacy and Pharmaceutical Sciences, Binghamton University, P.O. Box 6000, Binghamton, NY 13902, United States.
Bioorg Med Chem Lett. 2022 Nov 1;75:128953. doi: 10.1016/j.bmcl.2022.128953. Epub 2022 Sep 1.
In spite of their value in prodrug applications, the use of esters in antibody-drug-conjugate (ADC) payloads and linkers has generally been avoided due to the ubiquitous and promiscuous nature of human esterases. ADCs generally have a long circulating half life (3-7 days) that makes them susceptible to esterase-mediated metabolism. Moreover, it is largely unclear whether lysosomal and cytosolic esterases cleave ester-containing linkers upon ADC internalization. Due to our interest in the targeted delivery of immune-modulators, our team has recently prepared a series of ester-linked dexamethasone ADCs. Herein, we report our studies of the functional activity of these ADCs, with a particular focus on their catabolism in various biological milieu. We found that esters are selectively but inefficiently cleaved upon cellular uptake, likely by cytosolic esterases. Lysosomal catabolism studies indicate that, in spite of the strong proteolytic activity, very little cleavage of ester-containing linkers occurs in the lysosome. However, ADCs bearing the ester-linked payloads are active in various immune-suppressive assays, suggesting that cytosolic cleavage is taking place. This was confirmed through LCMS quantitation of the payload following cell lysis. Finally, the stability of the ester linkage was evaluated in mouse and human plasma. We found, similar to other reports, there is a significant site-dependence on the cleavage. Esters attached at highly exposed sites, such as 443C, were rapidly cleaved in plasma while esters at more hindered sites, such at 334C, were not. Together, these results help to unravel the complexities of ester-incorporation into ADC linkers and pave a path forward for their utility in ADC applications.
尽管酯类在前药应用中具有价值,但由于人类酯酶无处不在且具有混杂性,抗体药物偶联物 (ADC) 有效载荷和连接子中通常避免使用酯类。ADC 通常具有较长的循环半衰期(3-7 天),这使其容易受到酯酶介导的代谢影响。此外,尚不清楚溶酶体和细胞质酯酶在 ADC 内化时是否会切割含酯的连接子。由于我们对免疫调节剂的靶向递送感兴趣,我们的团队最近制备了一系列酯连接的地塞米松 ADC。在此,我们报告了我们对这些 ADC 的功能活性的研究,特别关注它们在各种生物环境中的代谢情况。我们发现,酯类在细胞摄取时会被选择性但低效地切割,可能是由细胞质酯酶介导的。溶酶体代谢研究表明,尽管溶酶体具有很强的蛋白水解活性,但含酯连接子的很少发生切割。然而,带有酯连接有效载荷的 ADC 在各种免疫抑制测定中具有活性,这表明细胞质切割正在发生。通过细胞裂解后对有效载荷进行 LCMS 定量证实了这一点。最后,在小鼠和人血浆中评估了酯键的稳定性。我们发现,与其他报告类似,酯键的切割存在显著的位点依赖性。附着在高度暴露位点(如 443C)的酯类在血浆中迅速切割,而附着在更受阻碍的位点(如 334C)的酯类则不会。这些结果有助于揭示将酯类纳入 ADC 连接子的复杂性,并为其在 ADC 应用中的实用性铺平道路。