Somanath Payaningal R, Chernoff Jonathan, Cummings Brian S, Prasad Sandip M, Homan Harvey D
Department of Clinical & Administrative Pharmacy, College of Pharmacy, University of Georgia, Augusta, GA 30912, USA.
MetasTx LLC, Basking Ridge, NJ 07920, USA.
Cancers (Basel). 2023 Apr 11;15(8):2236. doi: 10.3390/cancers15082236.
Metastatic prostate cancer (mPCa) has limited therapeutic options and a high mortality rate. The p21-activated kinase (PAK) family of proteins is important in cell survival, proliferation, and motility in physiology, and pathologies such as infectious, inflammatory, vascular, and neurological diseases as well as cancers. Group-I PAKs (PAK1, PAK2, and PAK3) are involved in the regulation of actin dynamics and thus are integral for cell morphology, adhesion to the extracellular matrix, and cell motility. They also play prominent roles in cell survival and proliferation. These properties make group-I PAKs a potentially important target for cancer therapy. In contrast to normal prostate and prostatic epithelial cells, group-I PAKs are highly expressed in mPCA and PCa tissue. Importantly, the expression of group-I PAKs is proportional to the Gleason score of the patients. While several compounds have been identified that target group-I PAKs and these are active in cells and mice, and while some inhibitors have entered human trials, as of yet, none have been FDA-approved. Probable reasons for this lack of translation include issues related to selectivity, specificity, stability, and efficacy resulting in side effects and/or lack of efficacy. In the current review, we describe the pathophysiology and current treatment guidelines of PCa, present group-I PAKs as a potential druggable target to treat mPCa patients, and discuss the various ATP-competitive and allosteric inhibitors of PAKs. We also discuss the development and testing of a nanotechnology-based therapeutic formulation of group-I PAK inhibitors and its significant potential advantages as a novel, selective, stable, and efficacious mPCa therapeutic over other PCa therapeutics in the pipeline.
转移性前列腺癌(mPCa)的治疗选择有限且死亡率高。p21激活激酶(PAK)蛋白家族在生理过程中的细胞存活、增殖和运动中起着重要作用,在诸如感染性、炎症性、血管性和神经性疾病以及癌症等病理状况中也很重要。I组PAK(PAK1、PAK2和PAK3)参与肌动蛋白动力学的调节,因此对于细胞形态、与细胞外基质的粘附以及细胞运动不可或缺。它们在细胞存活和增殖中也发挥着重要作用。这些特性使I组PAK成为癌症治疗的一个潜在重要靶点。与正常前列腺和前列腺上皮细胞相比,I组PAK在mPCA和PCa组织中高表达。重要的是,I组PAK的表达与患者的 Gleason评分成正比。虽然已经鉴定出几种靶向I组PAK的化合物,它们在细胞和小鼠中具有活性,并且一些抑制剂已经进入人体试验,但截至目前,尚无一种获得美国食品药品监督管理局(FDA)批准。这种缺乏转化的可能原因包括与选择性、特异性、稳定性和疗效相关的问题,这些问题导致了副作用和/或疗效不佳。在当前的综述中,我们描述了PCa的病理生理学和当前治疗指南,将I组PAK作为治疗mPCa患者的潜在可药物化靶点,并讨论了PAK的各种ATP竞争性和变构抑制剂。我们还讨论了基于纳米技术的I组PAK抑制剂治疗制剂的开发和测试,以及其作为一种新型、选择性、稳定且有效的mPCa治疗方法相对于其他正在研发中的PCa治疗方法的显著潜在优势。