Bou Malhab Lara J, Alsafar Habiba, Ibrahim Saleh, Rahmani Mohamed
Research Institute of Medical and Health Sciences, University of Sharjah, Sharjah, United Arab Emirates.
Center for Biotechnology, Khalifa University, Abu Dhabi, United Arab Emirates.
Front Pharmacol. 2023 Feb 20;14:1086946. doi: 10.3389/fphar.2023.1086946. eCollection 2023.
Proteolysis targeting chimeras (PROTACs) are heterobifunctional small molecules that uses the proteasome ubiquitin system to target proteins of interest and promote their degradation with remarkable selectivity. Importantly, unlike conventional small molecule inhibitors, PROTACs have proven highly effective in targeting undruggable proteins and those bearing mutations. Because of these considerations, PROTACs have increasingly become an emerging technology for the development of novel targeted anticancer therapeutics. Interestingly, many PROTACs have demonstrated a great potency and specificity in degrading several oncogenic drivers. Many of these, following extensive preclinical evaluation, have reached advanced stages of clinical testing in various cancers including hematologic malignancies. In this review, we provide a comprehensive summary of the recent advances in the development of PROTACs as therapeutic strategies in diverse hematological malignancies. A particular attention has been given to clinically relevant PROTACs and those targeting oncogenic mutants that drive resistance to therapies. We also discus limitations, and various considerations to optimize the design for effective PROTACs.
蛋白酶靶向嵌合体(PROTACs)是异双功能小分子,其利用蛋白酶体泛素系统靶向感兴趣的蛋白质,并以显著的选择性促进其降解。重要的是,与传统小分子抑制剂不同,PROTACs已被证明在靶向不可成药蛋白和携带突变的蛋白方面非常有效。基于这些考虑,PROTACs越来越成为开发新型靶向抗癌疗法的新兴技术。有趣的是,许多PROTACs在降解多种致癌驱动因子方面已显示出强大的效力和特异性。其中许多在经过广泛的临床前评估后,已进入包括血液系统恶性肿瘤在内的各种癌症的临床试验后期阶段。在本综述中,我们全面总结了PROTACs作为多种血液系统恶性肿瘤治疗策略开发的最新进展。特别关注了临床相关的PROTACs以及那些靶向导致治疗耐药的致癌突变体的PROTACs。我们还讨论了局限性以及优化有效PROTACs设计的各种考虑因素。