New horizons in drug discovery of lymphocyte-specific protein tyrosine kinase (Lck) inhibitors: a decade review (2011–2021) focussing on structure–activity relationship (SAR) and docking insights

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淋巴细胞特异性蛋白酪氨酸激酶(Lck)抑制剂药物研发新进展:十年回顾(2011–2021)——聚焦构效关系(SAR)与分子对接研究

作者 Ahmed Elkamhawy; Eslam M.H. Ali; Kyeong Lee 期刊 Journal of Enzyme Inhibition and Medicinal Chemistry 发表日期 2021 ISSN 1475-6366 DOI 10.1080/14756366.2021.1937143 类型 原创研究 (Original Research)

📄 中文摘要 Chinese Abstract

中文
淋巴细胞特异性蛋白酪氨酸激酶(Lck)是一种非受体型Src家族激酶,在T细胞受体(TCR)信号传导、T细胞发育和稳态中发挥关键作用。它调控包括增殖、分化、黏附和迁移在内的多种关键细胞过程。Lck表达或活性的失调与多种疾病相关,例如癌症(如白血病、实体瘤)、哮喘、糖尿病、类风湿性关节炎、动脉粥样硬化和神经元疾病。由于其在免疫信号传导和疾病发病机制中的核心地位,Lck已成为一个有前景的治疗靶点。过去十年间,大量研究工作致力于开发高效、选择性和安全的Lck抑制剂,构效关系(SAR)研究和分子对接为结合模式和抑制剂设计提供了重要见解。

📋 英文结构化总结 English Structured Summary

全文整理

EN

Background:

Lymphocyte-specific protein tyrosine kinase (Lck), a non-receptor Src family kinase, plays a critical role in T-cell receptor (TCR) signaling, T-cell development, and homeostasis. It regulates key cellular processes including proliferation, differentiation, adhesion, and motility. Dysregulation of Lck expression or activity is implicated in various diseases such as cancer (e.g., leukemia, solid tumors), asthma, diabetes, rheumatoid arthritis, atherosclerosis, and neuronal disorders. Due to its central role in immune signaling and disease pathogenesis, Lck has emerged as a promising therapeutic target. Over the past decade, significant research efforts have focused on developing potent, selective, and safe Lck inhibitors, with structure–activity relationship (SAR) studies and molecular docking providing crucial insights into binding modes and inhibitor design.

Methods:

This review compiles and analyzes literature and pharmaceutical patents from 2011 to 2020 reporting new Lck inhibitors. The methodology includes detailed SAR analysis of newly discovered chemical scaffolds and molecular docking simulations using the Molecular Operating Environment (MOE, 2014) software. X-ray crystal structures of Lck (PDB IDs: 2PL0, 3BYM, 3BYO, 6PDJ) were used for docking studies after energy minimization and protonation. Docking protocols were validated by re-docking native ligands and calculating RMSD values. Binding interactions—particularly hydrogen bonding with Met319 (hinge region), Thr316 (gatekeeper), and hydrophobic interactions with Asp382—were analyzed to evaluate inhibitor affinity and selectivity within the Src kinase family.

Results:

Multiple novel chemical classes of Lck inhibitors were identified, including halogenated alkaloids (e.g., purpuroines A and D), imidazo[1,5-a]pyrazines, pyrrolopyrimidines, substituted triazoles, dasatinib derivatives, prodan-based fluorescent inhibitors, phenoxypyrimidines, and pyrazolo[1,5-a]pyridines. Several compounds demonstrated nanomolar inhibitory activity against Lck. For example, purpuroine D showed IC₅₀ = 0.94 µg/mL; compound 4 exhibited pIC₅₀ ≥ 8; pyrrolopyrimidines 5, 14–19, 22, and 23 had Ki < 100 nM; triazoles 24 and 25 showed IC₅₀ < 0.1 µM; dasatinib derivative 31 had IC₅₀ = 1.5 nM; and pyrazolo[1,5-a]pyridine 38 achieved IC₅₀ = 26 nM. Docking revealed that high-affinity inhibitors consistently formed key interactions: H-bonding with Met319 in the hinge region, engagement with Thr316 at the gatekeeper site, and hydrophobic contacts with Asp382. Selectivity over other Src kinases was often linked to specific interactions with unique residues like Glu320 and Thr316.

Data Summary:

Quantitative data highlight the potency and selectivity trends across compound classes. Purpuroines A and D inhibited Lck with IC₅₀ values of 2.35 and 0.94 µg/mL, respectively. Among pyrrolopyrimidines, 9 out of 19 compounds showed Ki < 100 nM. Triazoles 24 and 25 were highly potent (IC₅₀ < 0.1 µM) and selective for Lck. Dasatinib derivative 31 maintained strong inhibition (IC₅₀ = 1.5 nM). Phenoxypyrimidines 34 and 35 exhibited IC₅₀ values of 6.5 ± 2 nM and 6 ± 0.5 nM, respectively. The most potent pyrazolo[1,5-a]pyridine (compound 38) showed a 10-fold improvement over its predecessor (IC₅₀ = 26 nM vs. 260 nM). Docking scores ranged from −5.57 to −11.39 kcal/mol, with lower (more negative) scores correlating with higher experimental potency in several series.

Conclusions:

Lck remains a compelling yet challenging drug target due to high homology among Src family kinases. While numerous potent inhibitors have been developed over the last decade, achieving selectivity, optimal pharmacokinetics, and safety profiles remains elusive. Key structural features for effective Lck inhibition include hydrogen bonding with Met319 and Thr316, and hydrophobic occupancy near Asp328. Molecular docking has proven instrumental in rationalizing SAR and guiding inhibitor optimization. Future progress may depend on targeting allosteric sites to enhance selectivity and reduce off-target effects, offering a promising path toward clinically viable Lck inhibitors for cancer and inflammatory diseases.

Practical Significance:

The development of selective Lck inhibitors holds significant therapeutic potential for treating T-cell malignancies (e.g., CLL, ALL), solid tumors (e.g., breast, lung, colon cancers), autoimmune and inflammatory diseases (e.g., asthma, rheumatoid arthritis), and transplant rejection. Inhibitors with improved selectivity and safety profiles could lead to more effective and targeted immunomodulatory therapies, minimizing broad immunosuppression and adverse effects associated with current kinase inhibitors.

📋 中文结构化总结 Chinese Structured Summary

中文

背景:

淋巴细胞特异性蛋白酪氨酸激酶(Lck)是一种非受体型Src家族激酶,在T细胞受体(TCR)信号传导、T细胞发育和稳态中发挥关键作用。它调控包括增殖、分化、黏附和迁移在内的多种关键细胞过程。Lck表达或活性的失调与多种疾病相关,例如癌症(如白血病、实体瘤)、哮喘、糖尿病、类风湿性关节炎、动脉粥样硬化和神经元疾病。由于其在免疫信号传导和疾病发病机制中的核心地位,Lck已成为一个有前景的治疗靶点。过去十年间,大量研究工作致力于开发高效、选择性和安全的Lck抑制剂,构效关系(SAR)研究和分子对接为结合模式和抑制剂设计提供了重要见解。

方法:

本综述整理并分析了2011年至2020年间报道新型Lck抑制剂的相关文献和药物专利。研究方法包括对新型化学骨架进行详细的构效关系(SAR)分析,以及使用分子操作环境(MOE, 2014)软件进行分子对接模拟。Lck的X射线晶体结构(PDB编号:2PL0、3BYM、3BYO、6PDJ)经能量最小化和质子化处理后用于对接研究。通过重新对接天然配体并计算均方根偏差(RMSD)值来验证对接方案。分析了结合相互作用,特别是与Met319(铰链区)、Thr316(看门残基)的氢键以及与Asp382的疏水相互作用,以评估抑制剂在Src激酶家族中的亲和力和选择性。

结果:

鉴定出多种新型Lck抑制剂化学类别,包括卤代生物碱(如紫红碱A和D)、咪唑并[1,5-a]吡嗪类、吡咯并嘧啶类、取代三唑类、达沙替尼衍生物、基于芘的荧光抑制剂、苯氧基嘧啶类和吡唑并[1,5-a]吡啶类。多个化合物对Lck表现出纳摩尔级抑制活性。例如,紫红碱D的IC₅₀ = 0.94 µg/mL;化合物4的pIC₅₀ ≥ 8;吡咯并嘧啶类化合物5、14–19、22和23的Ki < 100 nM;三唑类化合物24和25的IC₅₀ < 0.1 µM;达沙替尼衍生物31的IC₅₀ = 1.5 nM;吡唑并[1,5-a]吡啶类化合物38的IC₅₀ = 26 nM。对接研究表明,高亲和力抑制剂始终形成关键相互作用:与铰链区Met319形成氢键、与看门位点Thr316结合以及与Asp382产生疏水接触。对其他Src激酶的选择性通常与独特残基(如Glu320和Thr316)的特异性相互作用相关。

数据总结:

定量数据揭示了各类化合物的效力和选择性趋势。紫红碱A和D对Lck的IC₅₀值分别为2.35和0.94 µg/mL。在吡咯并嘧啶类中,19个化合物中有9个的Ki < 100 nM。三唑类化合物24和25对Lck具有高效力(IC₅₀ < 0.1 µM)和选择性。达沙替尼衍生物31保持了强效抑制(IC₅₀ = 1.5 nM)。苯氧基嘧啶类化合物34和35的IC₅₀值分别为6.5 ± 2 nM和6 ± 0.5 nM。最有效的吡唑并[1,5-a]吡啶类(化合物38)较其前体提高了10倍(IC₅₀ = 26 nM对比260 nM)。对接打分范围为−5.57至−11.39 kcal/mol,在多个系列中,较低(更负)的打分与较高的实验效力相关。

结论:

由于Src家族激酶之间的高度同源性,Lck仍然是一个引人注目但颇具挑战性的药物靶点。尽管过去十年间已开发出众多高效抑制剂,但在实现选择性、最佳药代动力学和安全性方面仍面临困难。有效抑制Lck的关键结构特征包括与Met319和Thr316形成氢键以及在Asp328附近占据疏水空间。分子对接在合理化构效关系和指导抑制剂优化方面已被证明具有重要价值。未来的进展可能依赖于靶向变构位点以增强选择性并减少脱靶效应,这为开发用于癌症和炎症性疾病的临床可行Lck抑制剂提供了一条有前景的途径。

实际意义:

选择性Lck抑制剂的开发在治疗T细胞恶性肿瘤(如慢性淋巴细胞白血病、急性淋巴细胞白血病)、实体瘤(如乳腺癌、肺癌、结肠癌)和自身免疫性及炎症性疾病(如哮喘、类风湿性关节炎)以及移植排斥反应方面具有重要的治疗潜力。具有改进选择性和安全性特征的抑制剂可带来更有效和更具针对性的免疫调节疗法,最大限度地减少广谱免疫抑制以及与当前激酶抑制剂相关的不良反应。

📖 英文全文 English Full Text

EN

3428 jenzimc Journal of Enzyme Inhibition and Medicinal Chemistry J Enzyme Inhib Med Chem Taylor & Francis PMC8274522 8274522 8274522 34233563 10.1080/14756366.2021.1937143 New horizons in drug discovery of lymphocyte-specific protein tyrosine kinase (Lck) inhibitors: a decade review (2011–2021) focussing on structure–activity relationship (SAR) and docking insights Elkamhawy Ahmed a b Ali Eslam M H c d e Lee Kyeong a ✉ a College of Pharmacy, Dongguk University-Seoul, Goyang, Republic of Korea b Department of Pharmaceutical Organic Chemistry, Faculty of Pharmacy, Mansoura University, Mansoura, Egypt c Center for Biomaterials, Korea Institute of Science & Technology (KIST School), Seoul, Republic of Korea; d University of Science & Technology (UST), Daejeon, Republic of Korea e Pharmaceutical Chemistry Department, Faculty of Pharmacy, Modern University for Technology and Information (MTI), Cairo, Egypt ✉ CONTACT Kyeong Lee kaylee@dongguk.edu

College of Pharmacy, Dongguk University-Seoul, Goyang, 10326, Republic of Korea. 7 7 2021 36 1 1574 1574–1602 20 7 2021 © 2021 The Author(s). Published by Informa UK Limited, trading as Taylor & Francis Group. This is an Open Access article distributed under the terms of the Creative Commons Attribution-NonCommercial License ( http://creativecommons.org/licenses/by-nc/4.0/ ), which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited. Abstract Lymphocyte-specific protein tyrosine kinase (Lck), a non-receptor Src family kinase, has a vital role in various cellular processes such as cell cycle control, cell adhesion, motility, proliferation, and differentiation. Lck is reported as a key factor regulating the functions of T-cell including the initiation of TCR signalling, T-cell development, in addition to T-cell homeostasis. Alteration in expression and activity of Lck results in numerous disorders such as cancer, asthma, diabetes, rheumatoid arthritis, atherosclerosis, and neuronal diseases. Accordingly, Lck has emerged as a novel target against different diseases. Herein, we amass the research efforts in literature and pharmaceutical patents during the last decade to develop new Lck inhibitors. Additionally, structure-activity relationship studies (SAR) and docking models of these new inhibitors within the active site of Lck were demonstrated offering deep insights into their different binding modes in a step towards the identification of more potent, selective, and safe Lck inhibitors. Keywords: Lck inhibitors, structure-activity relationship (SAR), Src family kinase, lymphocyte-specific protein tyrosine kinase (Lck), molecular modelling status released display-pdf yes is-olf no is-manuscript no is-preprint no is-journal-matter no is-scanned no is-retracted no Received 2021 Feb 25; Accepted 2021 May 25; Collection date 2021. 1. Introduction Lymphocyte-specific protein tyrosine kinase (Lck), a 56 KDa protein, is a member of the Src family of non-receptor protein kinases. Lck is involved in the phosphorylation process of a number of intracellular signalling molecules such as IL-2-inducible T-cell kinase (ITK), protein kinase C, Phosphoinositide 3-kinase (PI3K), and Zeta-chain-associated protein kinase 70 (ZAP-70). Accordingly, it regulates numerous cellular processes including cell cycle control, cell adhesion, motility, proliferation, and differentiation. The function of Lck has been extensively studied and various reports revealed different mechanistic insights into the regulation of its activity including its major role as a key activator of T cells via T cell antigen receptors (TCR) signalling 1–5 . In addition to T cells, Lck is expressed in natural killer (NK) cells, NK T cells, CD5 + B-1 B cells, germinal centre and to a lesser extent in mantle zone B cells, aryl hydrocarbon receptor-activated primary human B cells, and brain including the hippocampus, cerebellum and retina 6–10 . In addition to leukaemia, Lck expression was also detected in a number of solid cancers including colon cancer, lung carcinoma, and breast cancer 11–16 , which led to the hypothesis that Lck may also have cancer promoting functions and hence may act as a potential therapeutic target for solid cancers. Accordingly, Lck inhibitors were found to be promising not only for the treatment of leukaemia but also in various solid cancers. In this review, we focus on presenting the newly discovered Lck inhibitors during the last decade, discussing their structure-activity relationship (SAR), in addition to performing docking simulation models of the most promising candidates into the binding site(s) of Lck in an attempt to get insights for further investigations towards more selective, potent and safe Lck inhibitors. 2. Lck (structure, regulation, and physiological roles) The strcuture of Lck has the typical backbone found in all members of the Src kinase family ( Figure 1 ); an N-terminal site (SH4 domain), SH3 and SH2 domains, a catalytic domain at the carboxy terminal (SH1 domain), and a short C-terminal tail 17–19 . The C-terminal lobe contains the activation loop (alpha-helix) which forms the phosphorylation site. Both SH2 and SH3 domains are folded to be involved in protein-protein interactions responsible for the regulation of Lck activity and signal transmission; while the main function of SH2 domain is to regulate interactions with phosphotyrosine containing elements, the SH3 domain regulates interactions with proline rich elements. The SH4 domain contains a glycine and two cysteine residues, which are myristoylated and palmitoylated, respectively, to target Lck to the plasma membrane. Figure 1. (A) Structure of Lck kinase domains; (B) Schematic structure of Lck: SH4, unique region (UR), SH3, SH2, SH2-kinase domain linker region (LR), kinase domain, and the C-terminal negative regulatory tail (NR), Reprinted from Ref. 4 ; (C) Lck conformations and regulation of Lck activation, Reprinted from Ref. 4 . The regulation of Lck activity occurs via phosphorylation/dephosphorylation of crucial tyrosine residues, and by some conformational changes; Phosphorylation of Tyr505 residue by the C-terminal Src kinase (Csk) leads to Lck closure through an intramolecular interaction with the SH2 domain. The closed conformation of Lck is further stabilised by the interaction between the SH3 domain and a proline-reach region located in the SH2-kinase domain linker. On the other side, Lck opening depends on dephosphorylation of Tyr505 catalysed by the protein tyrosine phosphatase CD45. The open conformation of Lck auto- and transphosphorylates Tyr394 residue located in the activation loop within the catalytic domain resulting in Lck activation. In addition to Tyr505 and Tyr394, there are other amino acid residues regulate Lck activity; a recent study by Courtney et al. on a phosphomimetic Lck mutant found that phosphorylation of Tyr192 located in the SH2 domain may restrict the interaction between Lck and CD45, leading to hyperphosphorylation of Tyr505 and accordingly in Lck inactivation 20 . Another study proposed that the phosphorylation of this site is Zap-70-dependent, in addition, Tyr192 residue was found to be a part of an inhibitory feedback loop, which controls the regulation of the amount of active Lck and the strength/duration of TCR signalling 21 . Moreover, Lck activity was found to be also regulated by phosphorylation of Ser59 (another feedback circuit required for the regulation of TCR signalling) 22–24 . Accordingly, a number of biochemical modifications, conformational dynamics, and signalling circuits were found to regulate the activity of Lck. At physiological level, Lck is a key factor for development of T cells in the thymus and for the function of mature T cells. It also has a major role in the activation of TCR linked signal transduction pathways ( Figure 2 ) 25–28 . Plus, Lck is involved in regulation of neurite outgrowth since it plays an important role in maintaining long-term synaptic plasticity in neurons in addition to other roles related to spatial learning and memory 29 , 30 . As mentioned earlier, Lck is also expressed in NK T cells, NK cells, and B cells. Although the function of Lck in B-cell remains unclear, Lck was suggested to regulate B Cell Receptor Signalling (BCR) signalling 31 , 32 . Figure 2. The pathway of Lck signalling. Reprinted from Ref. 5 . 3. Lck-related diseases The human genome contains more than 500 protein kinases transfer a γ-phosphate group from ATP to serine, threonine, or tyrosine residues. Several kinases were found to be associated with different human disorders including cancer initiation and progression. Also, the recent medicinal chemistry research targeting development of small molecule kinase inhibitors for the treatment of various diseases including cancer has been proven to be a successful strategy 33–46 . Among these cancer-related kinases, Lck was reported to be the promotor of BCR signals in chronic lymphocytic leukaemia (CLL) via catalysis of the proximal phosphorylation of CD79a and the induction of distal signalling events involving phosphorylation of Syk, activation of MAPK, NF-kB, ERK, and PI3K/Akt signalling pathways that are responsible for CLL cell survival following BCR cross-linking. The treatment of CLL cells with Lck inhibitors suppressed BCR dependent cell survival leading to apoptosis suggesting the potential role of Lck inhibitors in the treatment of CLL 47–49 . Lck was also found to be overexpressed and hyperactivated in patients with B-cell precursor acute lymphoblastic leukaemia (BCP-ALL) 50 , 51 . Low levels of Lck were also detected in thymoma and suggested to be responsible of the abnormal proliferation of immature thymocytes causing thymic tumorigenesis. Co-expression of Lck-Fyn has been reported in the development of thymomas 52 . Other studies showed that Lck functions as a therapeutic target in acute myeloid leukaemia (AML) 53–55 . A recent study showed that Lck was expressed at a high level in primary central nervous system lymphoma (PCNSL) patients 56 . Lck expression was also detected in cholangiocarcinoma 57 , 58 , breast cancer 12 , 13 , 59 , 60 , colon cancer 14 , 15 , 61 , 62 , and lung carcinoma 16 , 62 , 63 . It was also found that Lck seems to play a role in cancer stem cells (CSC) in endometrioid cancer models and cisplatin resistance of glioma cancer stem cells 64 , 65 . An additional function of Lck in glioma cells has been recently described 66 . Moreover, Lck overexpression was reported in several small cell lung cancer (SCLC) and non-small cell lung cancer (NSCLC) cell lines and lung cancer specimens from patients 67 . The success of small molecule kinase inhibitors in the treatment of cancer, coupled with a greater understanding of inflammatory signalling cascades, has led to kinase inhibitors coming to the fore in the pursuit for new anti-inflammatory agents for the treatment of inflammatory and immune-mediated diseases 68 . Non-receptor tyrosine kinases of the Jak, Src, Syk, and Btk families play major roles in various inflammatory and immune-mediated disorders 69 . Lck was found to be a key player in the early allergic immune response. Antigen activation of TCR results in the activation of Lck and further downstream signalling, resulting in T cell differentiation as well as cytokine secretion. It was also reported that Lck mediates Th2 differentiation. The chronic inflammatory disease of the bronchial airways (asthma) was associated with activation of a Th2 type of T cell in the airway 70 , 71 . A study by Pernis et al. found that mice overexpressing or lacking Lck gene showed altered lung function suggesting their involvement in pathogenesis of asthma 72 . Histological assessment of mice lung tissues by Zhang et al. revealed that Lck specific siRNA attenuated the pulmonary inflammation in asthma mice proposing Lck as a potential therapeutic target for asthma 73 . Accordingly, since Lck was proved to be involved in the pathogenesis of asthma, a novel therapy for treatment of asthma can be developed based on Lck novel specific inhibitors. Different studies reported the relation between Lck and other diseases rather than cancer and inflammation; the expression of Lck with Type I diabetes suggesting Lck as one of the main targets for diabetes treatment 74 . A recent study reviewed the interplay of protein tyrosine phosphatases with Src kinases including Lck establishing their role in auto-immune mediated diabetes 75 . The concept of Lck inhibition for the management of Type 1 diabetes was supported by another report suggested that βig-h3 represses T cell activation in Type 1 diabetes via inhibition of Lck 76 . Moreover, blocking of Lck may provide a novel therapeutic target to manage atherosclerosis. A recent study showed T-cells in atherosclerosis patients to be cytotoxic towards vascular smooth muscle cells as well as endothelial cells, leading to vascular injury and plaque destabilisation. Lck might inhibit heat shock protein 65–mediated Reverse Cholesterol Transport in T cells which has been well established as one of the causes involved for atherosclerosis 77 . Lck was also reported as a potential therapeutic target for acute rejection after kidney transplantation 78 . Organ graft rejection occurs when the tissue transplanted in the recipient’s body is rejected by his immune system 79 . Thus, inhibition of Lck has been established as a potential target to prevent organ graft rejection 80 , 81 . 4. Early discovery of Lck kinase inhibitors (selected examples) By the year 2010, a large number of small molecules incorporating various chemical scaffolds were already reported to inhibit Lck 82 , 83 . In this section, we demonstrate some examples of the most promising candidates; the earliest members of this family are the ones possessing the pyrazolopyrimidine chemical scaffold; PP1 ( I ) and PP2 ( II ) ( Figure 3 ), reported by Pfizer in 1996 84 . Despite the low nanomolar Lck IC 50 range of these two compounds (0.005 and 0.004 µM for PP1 and PP2, respectively), they showed lack of selectivity within Src kinase family. Further extended studies offered a direct descendant of PP1 (A-770041, III , Figure 3 ) which demonstrated a specific inhibition over Lck with an IC 50 value of 0.147 µM. The final structure of this molecule is a result of both strategic modification and extensive SAR exploration aiming to improve the activity towards Lck and reduce activity against other members of the Src family while offering compounds with suitable pharmacokinetic properties 80 , 81 , 85–88 . Figure 3. Chemical structures of compounds I – IV . For different reasons such as the discovery of a chemical space available in the hydrophobic pocket and the solvent exposed binding region of Lck 80 , 81 , the difficulty of generating N7 variants of A-770041, and the hope to discover a highly selective Lck inhibitor via making productive contacts with the side chains of Tyr318 and the unique Glu320 in the extended hinge region of Lck, the pyrazolopyrimidine core was replaced with a thienopyridine scaffold offering compound IV with Lck IC 50 value of 0.21 µM ( Figure 3 ) 89 . The further SAR exploration confirmed that specificity could be generated through interactions with the hinge region. Analysis of compound IV against a larger kinase set showed improved selectivity within the Src family with significant decreases in activity against Src and Fyn relative to A-770041 ( III) . However, upon administration to mice, compound IV inhibited TCR stimulated IL-2 production with an ED 50 of 5 mg/kg; the pharmacokinetic analysis demonstrated poor performance regarding clearance and oral bioavailability. The benzothiazole compound BMS-243117 ( V , Figure 4 ) was then reported following SAR exploration of a thiazole compound initially obtained via high throughput screening. Although compound V demonstrated a highly potent nanomolar activity over Lck (IC 50 = 4 nM) and a promising inhibitory activity over T Cell proliferation with an IC 50 value of 1.1 µM, it showed high inhibitory activity against other isoforms of Src family (Src IC 50 = 632 nM, Fyn IC 50 = 128 nM, Hck IC 50 = 3.84 µM, Blk IC 50 = 336 nM, Lyn IC 50 = 1.32 µM, and Fgr IC 50 = 240 nM), in addition, no in vivo data is reported for this promising candidate to date 90 . Another aminoquinazoline-based highly potent Lck inhibitor ( VI , Figure 4 ), possessing IC 50 of 0.2 nM was identified via a high-throughput screening (HTS) 91 . Extended SAR studies of compound VI offered a series of novel aminoquinazolines possessing in vitro mechanism-based potency. Orally bioavailable optimised analogs of compound VI exhibited a promising anti-inflammatory activity over the anti-CD3-induced production of interleukin-2 (IL-2) in mice. Although the selectivity of compound VI within the Src family was not studied during these initial SAR studies, some analogs showed potent nanomolar activity against other Src family isoforms 91 . Screening of some pyrimidopyridazine-based small molecules against Lck led to the discovery of a novel 1,2-dihyrdropyrimido[4,5- c ]pyridazine derivative ( VII , Figure 4 ) with low micromolar activity towards Lck. Optimisation of this compound revealed the most promising analog of this series ( VIII , Figure 4 ) which demonstrated good solubility and activity towards Lck (IC 50 = 2 nM), although still with strong activity towards Src (IC 50 = 3 nM). Figure 4. Chemical structures of compounds V – VIII . A novel 4-amino-5,6-biaryl-furo[2,3- d ]pyrimidine lead ( IX , Figure 5 ) was discovered by DiMauro et al. as potent, non-selective inhibitor of Lck (IC 50 = 0.081 µM) via HTS 92 . The study further offered novel and expeditious synthetic route allowed for rapid diversification of the core scaffold and identification of compounds ( X and XI , Figure 5 ) possessing higher potency over Lck with IC 50 values of 0.009 and 0.036 µM, respectively. However, lack of selectivity was found; X and XI showed Src IC 50 values of 0.045 and 0.914 µM, and Ack1 IC 50 values of 0.098 and 0.078 µM, respectively. Further exploration of new 2,3-diarylfuro[2,3- b ]pyridin-4-amines by Martin et al. offered some derivatives with promising potency but the lack of selectivity and the non-optimal pharmacokinetic properties limited the research efforts in this area 93 . Martin et al. reported another series of 2-aminopyrimidine carbamates as a new class of compounds with potent and selective inhibition of Lck. The most promising compound of this series ( XII , Figure 5 ) exhibits good activity when evaluated in an in vivo model of T cell activation. It showed an IC 50 value of 0.0006 µM over Lck with an interesting selectivity profile (Src IC 50 = 0.001 µM, Kdr IC 50 = 0.14 µM, Syk IC 50 = 0.20 µM, Zap-70 IC 50 = 0.37 µM, and Btk IC 50 = 0.10 µM) 94 . Figure 5. Chemical structures of compounds IX – XII . 5. New horizons in drug discovery of Lck inhibitors (2011–2020) In the last decade, novel small molecules related to new chemical scaffolds were reported to inhibit Lck offering new horizons of drug discovery in this research area. By searching literature and pharmaceutical patents, we amass these efforts in this section. In addition, SAR studies and docking models of the most promising inhibitors within Lck active site were carried out to offer deep insights of their different binding modes in a step towards development of more potent, selective and safe Lck inhibitors as promising therapy for Lck-related human diseases. The molecular docking study of the following discussed Lck inhibitors was performed in an attempt to assist in defining and categorising the functional groups of each series (which are involved in the ligand binding and which are not detrimental in binding). Classifying these groups will determine which must be excised and which should be preserved or modified, which in turn will pave the way for the development of more potent and selective inhibitors. Guided by co-crystal structures of different ligands to their corresponding Lck domains, the key interactions in ATP pocket are determined as follow: (1) The native ligands anchored in the hinge binding adenine pocket by hydrogen bond interactions with either the NH or the carbonyl groups of the main chain of Met319 amino acid; however, some co-crystal structures showed additional hydrogen bond interaction in the adenine region with the carbonyl oxygen of Glu317 backbone, (2) The hydrophobic pocket of Lck is occupied by the ligand via Van der Waals interaction with Asp382 residue, (3) Amongst the employed crystal structures, staurosporine-Lck complex revealed deep embedding of the methylamino nitrogen of the glycoside ring in ATP ribose pocket via participation in hydrogen bond interaction with Ser323 residue, 4) Finally, the ligand is positioned in Lck gatekeeper residue via hydrogen acceptor bond with the γ–OH of Thr316 residue 95 , 96 . The molecular docking studies was performed using Molecular Operating Environment (MOE, 2014). The X-ray crystal structures of Lck domain were downloaded from the Protein Data Bank (PDB IDs: 1QPC, 1QPJ, 2OF2, 2OFU, 2PL0, 3BYM, 3BYO, 3LCK, and 6PDJ). Amino acid sequences of all protein were protonated and their energies were minimised. The employed crystal structures were docked with their native ligands, and their RMSD values were calculated. Only four PDB IDs: 2PL0, 3BYM, 3BYO, and 6PDJ of the lowest RMSD values were selected for operating docking protocol to the discussed inhibitors 1 – 38 ( Figures 6(A), 7(A), 8, 10, 12, 14(A), 15 ) aiming at evaluation of their binding scores, and determination of their crucial binding interactions within Lck active site, comparable to the native ligand of the corresponding PDB file ( Table 1 ). As depicted in Table 1 , most of the docked compounds preserved the key interaction in the hinge binding site by hydrogen bond formation with Met319; while, the hydrophobic pocket was occupied by some compounds via Van der Waals interaction with Asp382; however, the gatekeeper Thr316 H-bonded with majority of the compounds, that in turn hypothesised their selectivity to Lck kinase among Src-family kinases. The correlation between the docking findings and the variable inhibitory activities is discussed in more details for each class. Figure 6. (A) Chemical structures of halogenated alkaloids 1 – 3 ; (B) 3D molecular interaction docking model of compound 1 in Lck kinase domain active site (PDB ID: 3BYO) (C) 3D molecular interaction docking model of compound 3 in Lck kinase domain active site (PDB ID: 3BYO). Figure 7. (A) Chemical strucutre of compound 4 ; (B) 3D molecular interaction docking model of compound 4 in Lck kinase domain active site (PDB ID: 2PL0). Figure 8. Chemical structures of pyrrolopyrimidine-based Lck inhibitors 5 – 23 . Figure 10. Chemical structures of triazole-based compounds 24 – 29 . Figure 12. Chemical structure of Dasatinib ( 30 ) and its derivative 31 . Figure 14. (A) Chemical structure of compound 32 ; (B) 3D molecular interaction docking model of compound 32 in Lck kinase domain active site (PDB ID: 3BYM). Figure 15. Chemical structures of phenoxypyrimidine scaffold-based Lck inhibitors 33 – 35 . Table 1. Molecular docking study of compounds 1 – 38 in Lck kinase domain represented in 2D diagrams. Cpd. ID PDB ID Energy Score (Kcal/mol) 2D diagram Amino acids Binding group Molecular interactions (Native ligand 95 ) 6-(2,6-dimethylphenyl)-2-((4-(4-methyl-1-piperazinyl)phenyl)amino)pyrimido[5′,4′:5,6] pyrimido-[1,2- a ]benzimidazol-5(6 H )-one 3BYO −8.29 Met319 Pyrimidine (N)-NH H-bond Val259 Imidazole ring Arene-H 1 3BYO −5.57 Met319 Phenoxy (Br) H-bond Thr316 COOH (C = O) H-bond 2 3BYO −5.69 Met319 Phenoxy (I) H-bond Thr316 COOH (C = O) H-bond 3 3BYO −5.75 Met319 Phenoxy (Br) H-bond (Native ligand 97 ) Imatinib 2PL0 −10.66 Asp382 Amide-C = O H-bond Glu288 Amide-NH H-bond Ile361 Piperazine-NH H-bond Met292 Amide-NH H-bond Phe383 Pyrimidine ring Ar-Ar 4 2PL0 −8.36 Ala381 Amide-C = O H-bond Asp382 Amide-C = O H-bond Met292 Amide-NH H-bond Phe383 Pyrimidine ring Ar-Ar (Native ligand 95 ) N -phenyl-1-(4-((3,4,5-trimethoxyphenyl)amino)-1,3,5-triazin-2-yl)-1 H -benzo[ d ]imidazol-2-amine 3BYM −8.55 Asp382 Phenyl ring Arene-H Glu317 Triazine-CH H-bond Met319 Triazine (N)-NH H-bond Val259 benzo[ d ]imidazole Arene-H 5 3BYM −6.63 Asp382 Phenoxy ring Arene-H Gly322 Pyrimidine ring Arene-H Leu251 Pyrimidine ring Arene-H Met319 NH 2 H-bond 6 3BYM −5.98 Asp382 Pyrimidine ring Arene-H Thr316 Pyrrole ring Arene-H 7 3BYM −6.57 Lys273 4-Br Phenoxy ring Arene-H Met292 4-Br Phenoxy (Br) H-bond Met319 NH 2 H-bond 8 3BYM −6.46 Asp382 4-Cl Phenoxy (Cl) H-bond Leu251 Phenyl ring Arene-H 9 3BYM −6.66 Leu371 Phenyl ring Arene-H Thr316 Phenoxy ring Arene-H 10 3BYM −7.54 Asp382 NH 2 H-bond Met319 SO 2 H-bond Tyr318 SO 2 H-bond 11 3BYM −6.89 Leu371 Phenyl ring Arene-H Thr316 Phenoxy ring Arene-H 12 3BYM −6.66 Glu320 Piperidine (NH) H-bond Gly322 Pyrrole ring Arene-H Thr316 Phenoxy ring Arene-H 13 3BYM −7.71 Asp382 Phenyl ring Arene-H Gly322 Pyrimidine ring Arene-H Leu251 Pyrimidine ring Arene-H 14 3BYM −5.69 Asp382 Pyrimidine ring Arene-H Thr316 Pyrrole ring Arene-H Val301 Pyrimidine (N) H-bond 15 3BYM −6.38 Leu251 Benzyl group Arene-H Thr316 Pyrimidine ring Arene-H Val259 Pyrrole Arene-H 16 3BYM −6.64 Asp382 Phenyl ring Arene-H Gly322 Pyrimidine ring Arene-H Leu251 Pyrimidine ring Arene-H Met319 NH 2 H-bond 17 3BYM −6.44 Asp382 Phenoxy ring Arene-H Gly322 Pyrimidine ring Arene-H Leu251 Pyrimidine ring Arene-H Met319 NH 2 H-bond 18 3BYM −6.97 Gly322 Pyrrole ring Arene-H Ser323 Pyrimidine ring Arene-H Thr316 Phenoxy ring Arene-H 19 3BYM −6.61 Asp382 Phenoxy ring Arene-H Gly322 Pyrimidine ring Arene-H Leu251 Pyrimidine ring Arene-H Met319 NH 2 H-bond 20 3BYM −6.62 Asp382 3-CN phenoxy (CN) H-bond Leu251 Pyrrolo pyrimidine Arene-H Thr316 Phenyl ring Arene-H 21 3BYM −6.50 Asp382 Amide-(C = O) H-bond Met319 NH 2 H-bond Val301 Amide-(NH 2 ) H-bond 22 3BYM −6.70 Asp382 Phenyl ring Arene-H Leu251 Pyrrolo pyrimidine Arene-H Met319 NH 2 H-bond 23 3BYM −6.81 Leu251 Phenoxy ring Arene-H Lys269 4-CF 3 phenyl group Arene-H Thr316 Furan (O) H-bond Val259 Pyrimidine ring Arene-H 24 3BYM −5.86 Leu251 Thiophene ring Arene-H Met319 Triazole (N) H-bond Thr316 2-Cl,6-F-phenyl group Arene-H 25 3BYM −6.17 Met319 Triazole (N) H-bond Thr316 2-Cl,6-F-phenyl group Arene-H       26 3BYM −6.57 Asp382 2-Cl,6-F-phenyl group Arene-H 27 3BYM −6.87 Glu317 Triazole (NH) H-bond Leu251 3-NH 2 , 4-OMe phenyl group Arene-H Met319 Triazole (N) H-bond 28 3BYM −6.34 Met319 Triazole (N) H-bond 29 3BYM −6.16 Met319 Triazole (N) H-bond Thr316 2-Cl,6-F-phenyl group Arene-H 30 3BYM −7.00 Leu251 Thiazole ring Arene-H Met319 Thiazole (S) H-bond   NH H-bond 31 3BYM −7.63 Asp382 Pyridine ring Arene-H Glu249 Piperazine (NH) Metal/Ione Gly322 Pyrimidine ring Arene-H Leu251 Pyrimidine ring Arene-H Leu371 Thiazole ring Arene-H Met319 Thiazole (N) H-bond   NH H-bond 32 3BYM −6.98 Met319 NH 2 H-bond Val259 Naphthyl group Arene-H (Native ligand 98 ) N -(4-(6-methoxypyrazolo[1,5- a ]pyridine-3-carboxamido)-3-methylphenyl)-1-methyl-1 H -indazole-3-carboxamide ( 37 ) 6PDJ −11.39 Asp382 Amide-C = O H-bond Met319 Pyrazole (N) H-bond Phe283 Pyrazole ring Arene-H 33 6PDJ −7.59 Met319 Pyrimidine (N) H-bond Tyr318 CH Arene-H 34 6PDJ −7.95 Met319 Pyrimidine (N) H-bond 35 6PDJ −7.17 Met319 Pyrimidine (N) H-bond 36 6PDJ −6.52 Ala284 Amide (NH) H-bond Asp382 Azetidine (CH) Arene-H Met292 Urea (NH) H-bond Phe285 Indazole (Pyrazole ring) Arene-H Phe256 3-Cl phenyl group Arene-H 38 6PDJ −9.50 Ala289 CN group H-bond Asp382 Amide (NH) H-bond Met292 Indazole (Pyrazole ring) H-bond Phe383 Amide (NH) Arene-H 5.1. Halogenated alkaloids HPLC-ESIMS (High-performance liquid chromatography combined with electrospray mass spectrometry) guiding fractionation of the sponge I. purpurea resulted in the isolation of ten polyhalogenated alkaloids (Purpuroine A–J) 99 . The newly isolated purpuroines were assayed for their antibiotic and kinase inhibition activities. Although the initial assays were limited to a small panel of three different kinases including Lck, cyclin-dependent kinase 2 (CDK2), and polo-like kinase 1 (PLK1), purpuroines A ( 1 ) and D ( 2 ) ( Figure 5(A) ) showed potent inhibitory activity against Lck kinase with IC 50 values of 2.35 and 0.94 µg/mL, respectively. Purpuroine D was also found to inhibit PLK1 with an IC 50 value of 1.45 µg/mL. As a reference, staurosporine (a broad-spectrum protein kinase inhibitor) exhibited IC 50 values of 3.73 and 0.92 µg/mL over Lck and PLK1, respectively. All purpuroines displayed weak inhibition to CDK2 (IC 50 > 50 µg/mL). The primary SAR analysis of the trihalogen substituted analogs including the most potent compound (purpuroine D) presented their ability to show more inhibitory activity against Lck than the dihalogentaed analogs as in purpuroine B ( 3 , Figure 6(A) ). A molecular docking simulation was performed to get more insights about the different binding modes of this series within the Lck active site and to understand the possible reason(s) behind the difference in their biological activities. The docking study indicated that compounds 1 ( Figure 6(B) ) and 2 possessing tri-halogenated phenoxy group were deeply embedded in the hinge binding region via formation of H-bond with Met319 residue, leading to orientation of molecule’s lateral carboxylic acid group towards H-bonding with Thr316. On the other hand, the di-halogenated phenoxy in compound 3 ( Figure 6(C) ) is H-bonded through the bromo group with Met319, even though, the molecule didn’t show any additional H-bonds with amino acid residues in the adenine binding area. 5.2. 8-Methyl-1-phenyl-imidazo[l,5-a]pyrazines Using Lck IMAP assay, design of a new series of 8-methyl-1-phenyl-imidazo[l,5- a ]pyrazines as Lck inhibitors resulted in the discovery of novel Lck inhibitory derivatives with a wide range of pIC 50 values against Lck (≥6 – ≥8) 100 . Compound 4 ( Figure 7(A) ) is reported as one among many derivatives exhibited a potent inhibitory activity over Lck with pIC 50 value ≥8. Docking of compound 4 ( Figure 7(B) ) in the active site of Lck (PDB ID: 2PL0) illustrated a similar binding behaviour of imatinib (selective inhibitor of Lck among Src-family kinases); the amide linker of compound 4 conserved the essential hinge binding interactions with the back bone of Met292 and Glu288 amino acids via H-bond formation with NH, while the carbonyl part H-bonds with the NH of both Ala381and Asp381 residues. Moreover, the imidazopyrimidine moiety occupied the hydrophobic pocket and involved in Van der Waals interactions with Phe383. 5.3. Pyrrolopyrimidines Novel pyrrolopyrimidine-based Lck inhibitors were patented by Laurent et al. from the Canadian pharmaceutical company Pharmascience Inc. 101 At the molecular level, the kinase inhibitory activity (expressed as K i values) of the newly synthesised compounds was assessed against Lck and Bruton’s tyrosine kinase (Btk). Using splenic cell proliferation assay, EC 50 values (50% proliferation in the presence of compound as compared to vehicle treated controls) were also determined at the cellular level. As illustrated in Figure 8 , nineteen compounds belonging to five different general structures were selected to elucidate the SAR of this new series ( Table 2 ). Table 2. Biological activity of compounds 5 – 23 over Lck. Cpd K i Lck (nM) 5 , 14 , 15 , 16 , 17 , 18 , 19 , 22 , and 23 <100 7 , 8 , 9 , 10 , 11 , 12 , and 13 >100 – <1000 6 , 20 , and 21 >1000 It was noted that compounds 5 – 8 possessing cyclopentene ring exhibited a wide range of Lck inhibition; while compound 5 possessing unsubstituted phenoxy moiety exhibited a potent inhibition constant ( K i value < 100 nM), compounds 7 and 8 with bromo and chloro substituted phenoxy, respectively, exhibited higher K i values (> 100 – < 1000 nM). A total loss of the nanomolar activity was found in case of compound 6 possessing p- fluorophenoxy moiety ( K i value > 1000 nM). Alteration of the cyclopentene ring into the 5-membered (un)substituted 2,5-dihydro-1 H -pyrrole ( 9 and 10 ) and ring expansion into the 6-membered cyclohexene ( 11 ) and 1,2,3,6-tetrahydropyridine ( 12 ) retrieved the modest activity ( K i value > 100 – < 1000 nM). While substitution of the free NH in 1,2,3,6-tetrahydropyridine ring with benzenesulphonyl moiety ( 13 ) did not improve this modest activity, substitution with a small size ethyl group ( 14 ) greatly increased the inhibitory activity against Lck (K i value < 100 nM). Retrieving the 5-membered cyclopentene moiety along with replacement of the phenoxy moiety with benzyl resulted in compound 15 which also demonstrated a potent Lck inhibition ( K i value < 100 nM). Although an introduction of the isostere 5-membered 2,5-dihydrofuran ( 16 ) or 2-methyl-2,5-dihydrofuran ( 17 – 19 ) instead of the cyclopentene ring, along with keeping the phenoxy moiety, maintained the high potency, substitution of the phenoxy moiety in the meta position with cyano ( 20 ) or carboxamide group ( 21 ) resulted in loss of the nanomolar activity. Interestingly, the high potency was retrieved when the phenoxy moiety was substituted in the meta position with pyridin-3-ylmethoxy group ( 22 ) and 4-(trifluoromethyl)benzyloxy group ( 23 ). Molecular modelling studies in the active site of Lck (PDB ID: 3BYM) were carried out to understand the superiority in activity of the 5-membered rings (cyclopentene, 2,5-dihydrofuran, and 2-methyl-2,5-dihydrofuran ring) over the 6-membered ring 1,2,3,6-tetrahydropyridine, in addition, to figure out the role of the meta position substitution of the phenoxy moiety in the biological activity over Lck. As demonstrated in Table 1 , the docked derivatives exhibited variable interaction modes; however, the most potent inhibitors exhibited the highest affinity to the enzyme active site. For instance, compound 5 ( Figure 9(A) ) embedded deeply via multiple interactions within the pocket residues. Compound 5 anchored to the adenine area by H-bond with Met319 residue, also, the un-substitution on the phenoxy moiety allowed its deep interaction into the hydrophobic pocket via Arene-H bond with Asp382 back chain, while, the pyrrolopyrimidine scaffold contributed in holding the compound in this position by hydrophobic interaction with Gly322 and Leu251 amino acid residues. In contrary, p -fluoro substitution on the phenoxy group in compound 6 ( Figure 9(B) ) flipped the compound in the active site and resulted in moving the amino group away from the hinge binder which is supposed to badly affect the compound stability in the enzyme active site and reduce its activity. However, the observed moderate activity upon replacement of the cyclopentene ring into substituted 2,5-dihydro-1 H -pyrrole in compound 10 ( Figure 9(C) ) could be explained due to the contribution of the substituted sulphonyl (SO 2 ) group in two H-bonds with Met319 in the hinge binder and Thr316 in the hydrophobic pocket. Figure 9. 3D molecular interaction docking models of compound 5 (A), compound 6 (B), and compound 10 (C) in Lck kinase domain active site (PDB ID: 3BYM). 5.4. Substituted triazoles A series of substituted triazole-based compounds were designed and synthesised as new kinase inhibitors from the national institute of biological sciences in Beijing 102 , 103 . Upon screening of forty-two compounds over a panel of seven autoimmune disease-related kinases including Lck, Btk, P38a, Fyn, Lyn, BMX, and Blk, only two compounds ( 24 and 25 , Figure 10 ) exhibited highly potent and selective activities over Lck with IC 50 values less than 0.1 µM. While most of other compounds exhibited moderate activities against Lck with an IC 50 range of 0.1–10 µM, it was noted that only compound 26 ( Figure 10 ) was totally inactive over Lck (IC 50 > 10 µM) 102 . It was also found that compounds 27 , 28 , and 29 ( Figure 10 ) belonging to the same series were able to inhibit Lck in a high nanomolar IC 50 range (0.077 ± 0.022, 0.018 ± 0.007, and 0.044 ± 0.02, respectively) despite their high activity against other kinases. A molecular docking study of this groupoffered insights into their different binding modes in the active site of Lck and proposed an explanation for their variable activities. The highly potent derivatives 24 ( Figure 11(A) ) and 25 were able to fit into the active site, where the triazole nitrogen atom is conserving H-bonding interaction with Met319 amino acid backbone in the hinge binding region, while, the lateral substituted benzylamine moiety was oriented towards the gatekeeper pocket through Arene-H interaction with Thr316 residue. The conformation of the moderately active non selective inhibitors 27 – 29 preserved the central triazole ring held in the adenine binding region, but hindered the benzyl moiety interaction in the hydrophobic pocket ( Figure 11(C) ). Compound 26 did not exhibit the fundamental binding interactions in the hinge region ( Figure 11(B) ). Figure 11. 3D molecular interaction docking models of compound 24 (A), compound 26 (B), and compound 27 (C) in Lck kinase domain active site (PDB ID: 3BYM). 5.5. Dasatinib-derived Lck inhibitor Dasatinib ( 30 , Figure 12 ) is one of the tyrosine kinase inhibitors (TKIs) which has transformed the treatment of Chronic Myeloid Leukaemia (CML), with chronic-phase CML now considered a manageable chronic disease. It is an orally administered small molecule inhibitor of many tyrosine kinases at nanomolar concentrations, including BCR-ABL1, c-Kit, EphA2, platelet-derived growth factor receptor-b and the Src family of kinases (e.g. Src, Lck, Yes, Fyn) 104–107 . However, dasatinib which is metabolised in humans primarily by the cytochrome P450 enzyme 3A4 (CYP3A4) is also a time-dependent inhibitor of CYP3A4, accordingly, the dosage of dasatinib must be significantly decreased if the patient is concomitantly medicated with a strong CYP3A4 inhibitor such as ketoconazole, clarithromycin, and indinavir, since these drugs may increase the plasma concentration of dasatinib to unsafe levels. The administration of dasatinib should be stopped upon occurrence of myelosuppression. In addition, dasatinib causes inhibition of hERG (the human “Ether-a-go-go-Related Gene”) which is an ion channel involved in the electrical activity of the heart and the coordination of heart beating.Dasatinib also suffers from an extremely short half-life, with an overall mean terminal half-life of only 3–5 h. Accordingly, in a recent trial to develop a dasatinib-derived new inhibitor with better pharmacological and safety profile, compound 31 ( Figure 12 ) was reported by Sennthenn et al. and found to inhibit multiple kinases including Lck with an IC 50 value of 1.5 nM 108 . Docking the structurally modified derivative 31 ( Figure 13(B) ) revealed significant changes in the compound conformation in the active site of Lck (PDB ID: 3BYM), compared to the lead compound (dasatinib, Figure 13(A) ) . While the main hinge binder interaction with Met319 via the 2-aminothiazole central scaffold was conserved in the modified compound, such a small change in the terminal aromatic amide in dasatinib by the pyridinyl amide in compound 31 oriented the compound to bind deeply in the hydrophobic pocket via Arene-H interaction with Asp382. Additional molecular interactions were observed as a result of these conformational changes, the pyrimidine ring contributed by a pair of Arene-H interactions with Gly322 and Leu 251 amino acids. Also, the nitrogen atom of the lateral piperazine participated in Metal/Ione interaction with Glu249. Figure 13. 3D molecular interaction docking models of dasatinib ( 30 ) (A) and compound 31 (B) in Lck kinase domain active site (PDB ID: 3BYM). 5.6. Prodan-derived Lck inhibitor In an attempt to find a prodan-derived Lck inhibitor which could serve as a molecular tool for real-time intracellular studies of Lck signalling, a small ATP-competitive Lck inhibitor ( 32 , IC 50 = 124 nM, Figure 14(A) ) with innate fluorescent properties has been discovered by Fleming et al. through the integration of a prodan-derived fluorophore into the pharmacophore of the kinase inhibitor 109 . Docking of compound 32 in the Lck active site (PDB ID: 3BYM, Figure 14(B) ) revealed the pyrazolopyrimidine main scaffold to be buried in the adenine binding site by H-bonding between the amino group in the compound and Met319 backbone. While, the hydrophobic pocket was occupied by the naphthyl moiety which participated in Arene-H interaction with Val259. 5.7. Phenoxypyrimidines A novel series of phenoxypyrimidine scaffold-based inhibitors was recently reported from Korea Institute of Science and Technology (KIST) targeting Lck and FMS kinases for inflammatory disorders 110 . While this study concluded the discovery of a new Lck/FMS dual inhibitor ( 33 , Figure 15 ) with highly potent nanomolar IC 50 values of 22.0 ± 10.0 and 4.6 ± 0.05 nM against Lck and FMS kinases, respectively, in addition to its ability to demonstrate a promising anti-inflammatory effect, compounds 34 and 35 ( Figure 15 ) with Lck IC 50 value of 0.0065 ± 0.002 and 0.006 ± 0.0005 µM, respectively, were found to be the most potent Lck inhibitors in this series. Molecular docking of the synthesised phenoxypyrimidine derivatives 33 – 35 in the Lck active site (PDB ID: 6PDJ, Figure 16 ), revealed the fundamental role of 2-aminopyrimidine core in stabilising the inhibitors in the active site of the enzyme. The nitrogen atom of the pyrimidine acted as a H-bond acceptor and kept the molecules in the hinge binding region by forming a H-bond interaction with Met319. Furthermore, the substituted phenoxy moiety was oriented towards the hydrophobic pocket, even though it didn’t show remarkable interactions with the amino acid residues in this area. Figure 16. 3D molecular interaction diagrams of compound 33 (A), compound 34 (B), and compound 35 (C) in Lck kinase domain active site (PDB ID: 6PDJ). 5.8. Pyrazolo[1,5- a ]pyridines Bristol-Myers Squibb screened an internal kinase inhibitor collection which led to identify a pyridazinone lead compound ( 36 , Figure 17 ) as a starting point for development of novel inhibitors of C-terminal Src Kinase to evaluate the potential of this target for an immuno-oncology therapy 98 . Upon a series of modifications included switching from a pyridazinone to pyrazolopyridine hinge binder, the optimised analog 37 ( Figure 17 ) showed a promising ability to increase T cell proliferation induced by T cell receptor signalling and an excellent potential to reduce Lck phosphorylation in vivo upon oral dosing with Lck IC 50 = 260 nM. The most potent compound in this series over Lck was compound 38 ( Figure 17 , IC 50 = 26 nM) which showed 10-folds of potency compared to compound 37 . Figure 17. Chemical structures of pyrazolo[1,5- a ]pyridine-based Lck inhibitors 36 – 38 . The molecular interactions of the developed inhibitors were elaborated by their docking in the Lck active site (PDB ID: 6PDJ). As illustrated in Figure 18 , the native ligand ( 37 ) showed fit binding in the enzyme pocket; where the pyrazolo[1,5- a ]pyridine’s N1 formed a H-bond with Met319 in the hinge binding area, while, the hydrophobic pocket was occupied by the lateral indazole-3-carboxamide moiety via H-bonding with Asp382 and Arene-H interaction with Phe383. On the other hand, the pyridazinone moiety of the initial identified lead 36 was positioned away from the hinge region with no observed interactions. In addition, the indazole-3-carboxamide moiety was anchored in the hydrophobic pocket through H-bonding with Met292, and a couple of Arene-H interactions between the azetidine ring and the indazole moiety with Asp382 and Phe285, respectively. The modified potent inhibitor 38 showed the highest affinity to the binding site; the pyrazolo[1,5- a ]pyridine carboxamide moiety was positioned to the hinge region, while, the indazole-3-carboxamide moiety was bound in the hydrophobic pocket where the carboxamide-NH group H-bound with both Met292 and Asp382. Also, the indazole moiety formed an Arene-H interaction with Phe285. Moreover, the long chain substitution on the indazole N1 of 38 allowed the compound to extend deeply in the pocket via formation of a H-bond between the terminal CN group and Ala289 residue. Figure 18. 3D molecular interaction diagrams of compound 36 (A), compound 37 (B), and compound 38 (C) in Lck kinase domain active site (PDB ID: 6PDJ). 6. Conclusion Fuelled by the recent development of kinase inhibitor small molecules as an area of intense research, Lck is well established as a promising target for the next generation of kinase inhibitors. However, due to the high homology of Lck with other members of the Src family isoforms, complications in the development of Lck inhibitors are still found. There is no doubt that off target inhibition of other Src family members has the potential to inhibit numerous essential cellular functions. Accordingly, the successful Lck inhibitory chemical scaffold must show high activity towards Lck, relatively little activity towards other Src kinases as well as a promising in vitro cell-based and in vivo data to support its further consideration as promising clinical candidate. Despite the extensive research efforts to optimise a promising Lck inhibitor possessing the above-mentioned criteria, the development of Lck specific inhibitors with good bioavailability and pharmacokinetics is still elusive. The efforts thrown in developing potent selective inhibitors are focussing on deep analysis of the targeted enzyme active site and defining its specific key interactions. Among Src kinases family, Lck has an advantage of sequence differences where Lck gatekeeper is characterised by hydrogen bonding between the γ OH of Thr316 and a H-bond acceptor group in the corresponding inhibitors. Considering this specific hydrogen bonding might help in designing Lck selective inhibitors by introducing well-positioned groups to accept H-bond from Thr316. Molecular docking of the presented inhibitors showed variable interaction modes in Lck active site; however, following their reported SAR revealed that the key point for improving the activity is conserving the essential interactions in the Lck active site including H-bond interaction with Met319 in the hinge binder, Van der Waals interaction with Asp382 in the hydrophobic pocket, and binding to Lck gatekeeper with Thr316. Moreover, additional molecular interactions were detected by some inhibitors, which in turn boosted the inhibitors affinity and stability in Lck active site and explained their improved activity. In summary, deep understanding of the different structural interactions of inhibitor molecules with multiple closely related enzymes has the potential to provide data useful in the rational design of kinase inhibitors and the development of novel Lck inhibitors. Moreover, small‐molecule allosteric kinase inhibitors possessing the significant advantages over ATP‐competitive kinase inhibitors such as greater selectivity and lower off‐target toxicity could be the next generation of specific Lck inhibitors that can be optimised for clinical use. Thus, the efficient rational approaches for rapid discovery of new allosteric hits for Lck, as well as systematic biological assay technologies, are urgently needed. CRediT authorship contribution statement Ahmed Elkamhawy: Conceptualisation, Methodology, Writing-original draft, and Data curation. Eslam M.H. Ali: Visualisation, Software, Writing-modelling section. Kyeong Lee: Supervision, Funding acquisition, Review and editing. Funding Statement This work was supported by the National Research Foundation of Korea (NRF) grant funded by the Korea government (MSIT) [No. NRF-2018R1A5A2023127]. Disclosure statement The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper. References 1. Voronova AF, Sefton BM..

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3428 jenzimc 酶抑制与药物化学杂志 J Enzyme Inhib Med Chem Taylor & Francis PMC8274522 8274522 8274522 34233563 10.1080/14756366.2021.1937143 淋巴细胞特异性蛋白酪氨酸激酶(Lck)抑制剂药物研发新视野:十年回顾(2011–2021),聚焦构效关系(SAR)与对接洞察 Elkamhawy Ahmed a b Ali Eslam M H c d e Lee Kyeong a ✉ a 韩国庆熙大学药学学院,高阳,韩国 b 曼苏拉大学药学院药物有机化学系,曼苏拉,埃及 c 韩国科学技术研究院(KIST)生物材料中心,首尔,韩国 d 韩国科学技术院(UST),大田,埃及 e 现代技术与信息大学(MTI)药学系药物化学系,开罗,埃及 ✉ 通讯作者:Kyeong Lee,邮箱:kaylee@dongguk.edu 韩国庆熙大学药学学院,高阳,10326,韩国 2021年7月7日 36卷 1期 1574–1602页 2021年7月20日 © 2021 作者。由Informa UK Limited以Taylor & Francis Group名义出版。本文采用知识共享署名-非商业性使用许可协议(http://creativecommons.org/licenses/by-nc/4.0/)进行开放获取分发,允许在任何媒介中无限制地非商业性使用、分发和复制,前提是正确引用原始作品。

摘要 淋巴细胞特异性蛋白酪氨酸激酶(Lck)是一种非受体型Src家族激酶,在细胞周期调控、细胞黏附、运动、增殖和分化等多种细胞过程中发挥关键作用。研究表明,Lck是调控T细胞功能的关键因子,包括T细胞抗原受体(TCR)信号启动、T细胞发育以及T细胞稳态。Lck表达或活性异常与多种疾病相关,如癌症、哮喘、糖尿病、类风湿性关节炎、动脉粥样硬化和神经系统疾病。因此,Lck已成为治疗多种疾病的新型靶点。本文系统总结了2011年至2021年间文献和制药专利中关于新型Lck抑制剂的研究进展,并展示了这些抑制剂的构效关系(SAR)研究及其与Lck活性位点的分子对接模型,深入揭示其不同结合模式,旨在为开发更具效力、选择性和安全性的Lck抑制剂提供理论依据。

关键词:Lck抑制剂;构效关系(SAR);Src家族激酶;淋巴细胞特异性蛋白酪氨酸激酶(Lck);分子建模 状态:已发布;显示PDF:是;是否为OLF:否;是否为手稿:否;是否为预印本:否;是否为期刊内容:否;是否为扫描件:否;是否为撤回文章:否 收稿日期:2021年2月25日;接受日期:2021年5月25日;出版年份:2021年

1. 引言 淋巴细胞特异性蛋白酪氨酸激酶(Lck)是一种56 kDa的蛋白,属于非受体型Src蛋白激酶家族成员。Lck参与多种细胞内信号分子的磷酸化过程,如IL-2诱导型T细胞激酶(ITK)、蛋白激酶C、磷脂酰肌醇3-激酶(PI3K)和Zeta链相关蛋白激酶70(ZAP-70),从而调控细胞周期、黏附、运动、增殖和分化等过程。大量研究揭示了Lck活性调控的机制,尤其是其作为T细胞关键激活因子通过T细胞抗原受体(TCR)信号通路发挥作用¹⁻⁵。除T细胞外,Lck还在自然杀伤(NK)细胞、NKT细胞、CD5⁺ B-1 B细胞、生发中心B细胞及套区B细胞、芳烃受体激活的人原代B细胞以及大脑(包括海马体、小脑和视网膜)中表达⁶⁻¹⁰。此外,Lck不仅在白血病中表达,也在多种实体瘤中被检测到,如结肠癌、肺癌和乳腺癌¹¹⁻¹⁶,提示Lck可能具有促癌功能,并可作为实体瘤的潜在治疗靶点。因此,Lck抑制剂不仅有望用于白血病治疗,也可能在多种实体瘤中发挥作用。本综述重点介绍近十年来发现的新型Lck抑制剂,讨论其构效关系(SAR),并对最具前景的化合物进行Lck结合位点的分子对接模拟,以期为开发更具选择性、强效且安全的Lck抑制剂提供深入见解。

2. Lck的结构、调控与生理功能 Lck的结构具有Src激酶家族典型的骨架特征(图1):N端区域(SH4结构域)、SH3和SH2结构域、C端催化结构域(SH1结构域)以及短的C端尾部¹⁷⁻¹⁹。C端叶包含激活环(α-螺旋),形成磷酸化位点。SH2和SH3结构域折叠参与蛋白质-蛋白质相互作用,调控Lck活性和信号传导;其中SH2结构域主要调控与含磷酸酪氨酸元件的相互作用,而SH3结构域则调控与富含脯氨酸元件的相互作用。SH4结构域含有一个甘氨酸和两个半胱氨酸残基,分别发生豆蔻酰化和棕榈酰化,引导Lck定位于质膜。

图1. (A) Lck激酶结构域结构;(B) Lck示意图:SH4、独特区(UR)、SH3、SH2、SH2-激酶结构域连接区(LR)、激酶结构域及C端负调控尾部(NR),引自文献⁴;(C) Lck构象及其激活调控,引自文献⁴。

Lck活性通过关键酪氨酸残基的磷酸化/去磷酸化及构象变化进行调控。C端Src激酶(Csk)对Tyr505的磷酸化导致Lck通过SH2结构域发生分子内相互作用而闭合。Lck的闭合构象进一步由SH3结构域与SH2-激酶连接区中富含脯氨酸区域的相互作用稳定。另一方面,Lck的开放依赖于CD45蛋白酪氨酸磷酸酶催化的Tyr505去磷酸化。开放构象的Lck可自磷酸化及反式磷酸化催化结构域激活环中的Tyr394,从而激活Lck。除Tyr505和Tyr394外,其他氨基酸残基也参与调控Lck活性。Courtney等人对磷酸模拟Lck突变体的研究发现,SH2结构域中Tyr192的磷酸化可能限制Lck与CD45的相互作用,导致Tyr505过度磷酸化,进而使Lck失活²⁰。另一项研究表明,该位点的磷酸化依赖于Zap-70,且Tyr192是调控活性Lck数量及TCR信号强度/持续时间的负反馈环路的一部分²¹。此外,Ser59的磷酸化也被发现参与调控TCR信号的反馈回路²²⁻²⁴。综上,多种生化修饰、构象动态和信号回路共同调控Lck活性。

在生理水平上,Lck是胸腺中T细胞发育和成熟T细胞功能的关键因子,并在TCR相关信号转导通路激活中发挥主要作用(图2)²⁵⁻²⁸。此外,Lck还参与神经突生长的调控,因其在维持神经元长期突触可塑性中起重要作用,并与空间学习记忆等功能相关²⁹,³⁰。如前所述,Lck也在NKT细胞、NK细胞和B细胞中表达。尽管Lck在B细胞中的功能尚不明确,但有研究提出其可能调控B细胞受体(BCR)信号³¹,³²。

图2. Lck信号通路,引自文献⁵。

3. Lck相关疾病 人类基因组包含500多种蛋白激酶,可将ATP的γ-磷酸基转移至丝氨酸、苏氨酸或酪氨酸残基。多种激酶与人类疾病(包括癌症的发生发展)密切相关。近年来,针对小分子激酶抑制剂治疗多种疾病(包括癌症)的药物化学研究已被证明是一种成功策略³³⁻⁴⁶。在这些癌症相关激酶中,Lck被报道为慢性淋巴细胞白血病(CLL)中BCR信号的促进因子,通过催化CD79a的近端磷酸化并诱导涉及Syk磷酸化、MAPK、NF-κB、ERK和PI3K/Akt信号通路激活的远端信号事件,促进CLL细胞在BCR交联后的存活。用Lck抑制剂处理CLL细胞可抑制BCR依赖性细胞存活并诱导凋亡,提示Lck抑制剂在CLL治疗中的潜在作用中的潜在作用⁴⁷⁻⁴⁹。Lck也在B细胞前体急性淋巴细胞白血病(BCP-ALL)患者中高表达和过度激活⁵⁰,⁵¹。胸腺瘤中也检测到低水平Lck,可能与未成熟胸腺细胞异常增殖导致胸腺肿瘤发生有关。Lck与Fyn的共表达被报道与胸腺瘤的发展相关⁵²。其他研究显示Lck在急性髓系白血病(AML)中作为治疗靶点发挥作用⁵³⁻⁵⁵。近期研究发现,原发性中枢神经系统淋巴瘤(PCNSL)患者中Lck高表达⁵⁶。Lck表达亦见于胆管癌⁵⁷,⁵⁸、乳腺癌¹²,¹³,⁵⁹,⁶⁰、结肠癌¹⁴,¹⁵,⁶¹,⁶²和肺癌¹⁶,⁶²,⁶³。研究还发现Lck可能在子宫内膜样癌模型中的癌症干细胞(CSC)及胶质瘤癌细胞的顺铂耐药中发挥作用⁶⁴,⁶⁵。最近有研究描述了Lck在胶质瘤细胞中的新功能⁶⁶。此外,Lck过表达见于多种小细胞肺癌(SCLC)和非小细胞肺癌(NSCLC)细胞系及患者肺癌组织⁶⁷。

小分子激酶抑制剂在癌症治疗中的成功,结合对炎症信号级联反应的深入理解,使激酶抑制剂成为开发治疗炎症和免疫介导疾病新型抗炎剂的研究热点⁶⁸。Jak、Src、Syk和Btk家族的非受体型酪氨酸激酶在多种炎症和免疫介导疾病中发挥重要作用⁶⁹。Lck被发现在早期过敏免疫反应中起关键作用。抗原激活TCR导致Lck激活及下游信号传导,进而促进T细胞分化和细胞因子分泌。研究还报道Lck介导Th2分化。支气管气道的慢性炎症性疾病(哮喘)与气道中Th2型T细胞的激活相关⁷⁰,⁷¹。Pernis等人研究发现,过表达或缺失Lck基因的小鼠肺功能发生改变,提示其参与哮喘发病机制⁷²。Zhang等人对小鼠肺组织的组织学评估显示,Lck特异性siRNA可减轻哮喘小鼠的肺部炎症,提出Lck作为哮喘治疗的潜在靶点⁷³。因此,鉴于Lck已被证实参与哮喘发病机制,基于新型特异性Lck抑制剂的哮喘治疗策略值得开发。

多项研究报道了Lck与癌症和炎症以外的其他疾病的关系:Lck与I型糖尿病的关联提示其可作为糖尿病治疗的主要靶点⁷⁴。近期研究综述了蛋白酪氨酸磷酸酶与Src激酶(包括Lck)的相互作用,确立其在自身免疫介导糖尿病中的作用⁷⁵。Lck抑制用于管理I型糖尿病的概念得到另一项报告支持,即βig-h3通过抑制Lck在I型糖尿病中抑制T细胞激活⁷⁶。此外,阻断Lck可能为动脉粥样硬化管理提供新靶点。最新研究表明,动脉粥样硬化患者的T细胞对血管平滑肌细胞和内皮细胞具有细胞毒性,导致血管损伤和斑块不稳定。Lck可能抑制T细胞中热休克蛋白65介导的逆向胆固醇转运,这是动脉粥样硬化的已知病因之一⁷⁷。Lck也被报道为肾移植后急性排斥反应的潜在治疗靶点⁷⁸。器官移植排斥是指受体免疫系统对移植组织的排斥反应⁷⁹。因此,抑制Lck已被确立为预防器官移植排斥的潜在靶点⁸⁰,⁸¹。

4. Lck激酶抑制剂的早期发现(精选案例) 截至2010年,已有大量含有不同化学骨架的小分子被报道具有Lck抑制活性ck抑制活性⁸²,⁸³。本节展示其中一些最具前景的候选药物:该家族最早成员为含有吡唑并嘧啶骨架的化合物PP1(I)和PP2(II)(图3),由辉瑞公司于1996年报道⁸⁴。尽管这两种化合物对Lck的IC₅₀值在纳摩尔级别(PP1为0.005 µM,PP2为0.004 µM),但它们在Src激酶家族中缺乏选择性。进一步研究获得了PP1的直接衍生物A-770041(III,图3),其对Lck具有特异性抑制作用,IC₅₀值为0.147 µM。该分子的最终结构源于策略性修饰和广泛的构效关系探索,旨在提高对Lck的活性并降低对Src家族其他成员的活性,同时赋予化合物良好的药代动力学性质⁸⁰,⁸¹,⁸⁵⁻⁸⁸。

图3. 化合物I–IV的化学结构。

由于发现Lck的疏水口袋和溶剂暴露结合区域存在可用化学空间⁸⁰,⁸¹,A-770041的N7变体难以合成,以及希望通过与Lck延伸铰链区中Tyr318和独特Glu320侧链形成有效接触来发现高选择性Lck抑制剂,研究者将吡唑并嘧啶核心替换为噻吩并吡啶骨架,得到化合物IV,其Lck IC₅₀值为0.21 µM(图3)⁸⁹。进一步的构效关系研究表明,特异性可通过与铰链区的相互作用实现。对化合物IV在更大激酶谱中的分析显示,其在Src家族内的选择性有所提高,对Src和Fyn的活性相对于A-770041(III)显著降低。然而,在小鼠给药后,化合物IV抑制TCR刺激的IL-2产生的ED₅₀为5 mg/kg;药代动力学分析显示其清除率和口服生物利用度表现不佳。

随后,通过对高通量筛选获得的噻唑类化合物进行构效关系研究,报道了苯并噻唑类化合物BMS-243117(V,图4)。尽管化合物V对Lck表现出强效纳摩尔活性(IC₅₀ = 4 nM)和对T细胞增殖的抑制活性(IC₅₀ = 1.1 µM),但它对Src家族其他亚型也具有高抑制活性(Src IC₅₀ = 632 nM,Fyn IC₅₀ = 128 nM,Hck IC₅₀ = 3.84 µM,Blk IC₅₀ = 336 nM,Lyn IC₅₀ = 1.32 µM,Fgr IC₅₀ = 240 nM),且至今未见该候选药物的体内数据报道⁹⁰。

另一种基于氨基喹唑啉的高效Lck抑制剂(VI,图4),IC₅₀为0.2 nM,通过高通量筛选(HTS)发现⁹¹。对化合物VI的扩展构效关系研究获得了一系列具有体外机制基础效价的新型氨基喹唑啉类化合物。化合物VI的可口服优化类似物在小鼠中表现出良好的抗炎活性,可抑制抗CD3诱导的白细胞介素-2(IL-2)产生。尽管在这些初始构效关系研究中未评估化合物VI在Src家族内的选择性,但部分类似物对其他Src亚型也表现出强效纳摩尔活性⁹¹。

对一类嘧啶并哒嗪类小分子进行Lck筛选,发现了一种新型1,2-二氢嘧啶并[4,5-c]哒嗪衍生物(VII,图4),对Lck具有微摩尔级活性。该化合物的优化得到该系列中最有前景的类似物(VIII,图4),其具有良好的溶解性和对Lck的活性(IC₅₀ = 2 nM),尽管对Src仍有较强活性(IC₅₀ = 3 nM)。

图4. 化合物V–VIII的化学结构。

DiMauro等人通过HTS发现了一种新型4-氨基-5,6-二芳基-呋喃并[2,3-d]嘧啶先导化合物(IX,图5),作为Lck的强效非选择性抑制剂(IC₅₀ = 0.081 µM)⁹²。该研究进一步提供了快速合成路线,便于核心骨架的快速多样化,并鉴定出对Lck具有更高活性的化合物(X和XI,图5),IC₅₀值分别为0.009 µM和0.036 µM。然而,这些化合物缺乏选择性:X和XI对Src的IC₅₀分别为0.045 µM和0.914 µM,对Ack1的IC₅₀分别为0.098 µM和0.078 µM。Martin等人对新型2,3-二芳基呋喃并[2,3-b]吡啶-4-胺的进一步探索获得了一些具有前景活性的衍生物,但缺乏选择性和非最优的药代动力学性质限制了此领域的研究⁹³。

Martin等人还报道了另一类2-氨基嘧啶氨基甲酸酯作为新型强效选择性Lck抑制剂。该系列中最有前景的化合物(XII,图5)在T细胞激活的体内模型中表现出良好活性。其对Lck的IC₅₀为0.0006 µM,具有优异的选择性特征(Src IC₅₀ = 0.001 µM,Kdr IC₅₀ = 0.14 µM,Syk IC₅₀ = 0.20 µM,Zap-70 IC₅₀ = 0.37 µM,Btk IC₅₀ = 0.10 µM)⁹⁴。

图5. 化合物IX–XII的化学结构。

5. Lck抑制剂药物研发新视野(2011–2020) 近十年来,多种新型小分子被报道具有Lck抑制活性,为该领域药物研发开辟了新视野。通过文献和制药专利检索,本文系统总结了这些研究。此外,对最具前景的抑制剂进行了构效关系研究和Lck活性位点的分子对接模型分析,深入揭示其不同结合模式,旨在为开发更强效、更具选择性和安全性的Lck抑制剂提供理论支持,以用于治疗Lck相关人类疾病。

以下讨论的Lck抑制剂的分子对接研究旨在帮助定义和分类各系列化合物的功能基团(哪些参与配体结合,哪些对结合无益)。通过分类这些基团,可确定哪些应被去除,哪些应保留或修饰,从而为开发更强效、更具选择性的抑制剂铺平道路。

基于不同配体与Lck结构域的共晶结构,ATP口袋中的关键相互作用确定如下:(1) 天然配体通过氢键与Met319主链的NH或羰基结合,锚定于铰链结合腺嘌呤口袋;部分共晶结构还显示与Glu317主链羰基氧的额外氢键;(2) 配体通过范德华力与Asp382残基结合,占据Lck的疏水口袋;(3) 在所用晶体结构中,星形孢菌素-Lck复合物显示糖苷环的甲氨基氮深入嵌入ATP核糖口袋,并与Ser323残基形成氢键;(4) 最后,配体通过氢键受体与Thr316残基的γ-OH结合,定位于Lck的守门员残基⁹⁵,⁹⁶。

分子对接研究使用Molecular Operating Environment(MOE, 2014)软件完成。Lck结构域的X射线晶体结构从蛋白质数据库(PDB ID: 1QPC, 1QPJ, 2OF2, 2OFU, 2PL0, 3BYM, 3BYO, 3LCK, 6PDJ)下载。所有蛋白的氨基酸序列经质子化并能量最小化。将所用晶体结构与其天然配体对接,并计算RMSD值。仅选择RMSD值最低的四种PDB ID(2PL0, 3BYM, 3BYO, 6PDJ)用于对接协议,对讨论的抑制剂1–38(图6(A)、7(A)、8、10、12、14(A)、15)进行结合评分评估及Lck活性位点内关键结合相互作用的测定,并与相应PDB文件的天然配体进行比较(表1)。

如表1所示,大多数对接化合物通过与Met319形成氢键保留了铰链结合位点的关键相互作用;部分化合物通过范德华力与Asp382结合占据疏水口袋;多数化合物与守门员Thr316形成氢键,提示其对Lck激酶在Src家族中的选择性。对接结果与可变抑制活性之间的相关性将在每类化合物中详细讨论。

图6. (A) 卤代生物碱1–3的化学结构;(B) 化合物1在Lck激酶结构域活性位点的3D分子相互作用对接模型(PDB ID: 3BYO);(C) 化合物3在Lck激酶结构域活性点的3D分子相互作用对接模型(PDB ID: 3BYO)。

图7. (A) 化合物4的化学结构;(B) 化合物4在Lck激酶结构域活性位点的3D分子相互作用对接模型(PDB ID: 2PL0)。

图8. 基于吡咯并嘧啶的Lck抑制剂5–23的化学结构。

图10. 基于三唑的化合物24–29的化学结构。

图12. 达沙替尼(30)及其衍生物31的化学结构。

图14. (A) 化合物32的化学结构;(B) 化合物32在Lck激酶结构域活性位点的3D分子相互作用对接模型(PDB ID: 3BYM)。

图15. 基于苯氧基嘧啶骨架的Lck抑制剂33–35的化学结构。

表1. 化合物1–38在Lck激酶结构域中的分子对接研究(2D示意图)。

| Cpd. ID | PDB ID | 能量评分 (Kcal/mol) | 2D示意图 | 氨基酸 | 结合基团 | 分子相互作用 | |----------|---------|---------------------|----------|--------|----------|--------------| | (Native ligand ⁹⁵) | 3BYO | −8.29 | Met319 | 嘧啶(N)-NH | H键 | | | | | Val259 | 咪唑环 | Arene-H | | 1 | 3BYO | −5.57 | Met319 | 苯氧基(Br) | H键 | | | | | Thr316 | COOH(C=O) | H键 | | 2 | 3BYO | −5.69 | Met319 | 苯氧基(I) | H键 | | | | | Thr316 | COOH(C=O) | H键 | | 3 | 3BYO | −5.75 | Met319 | 苯氧基(Br) | H键 | | (Native ligand ⁹⁷) | 2PL0 | −10.66 | Asp382 | 酰胺-C=O | H键 | | | | | Glu288 | 酰胺-NH | H键 | | | | | Ile361 | 哌嗪-NH | H键 | | | | | Met292 | 酰胺-NH | H键 | | | | | Phe383 | 嘧啶环 | Ar-Ar | | 4 | 2PL0 | −8.36 | Ala381 | 酰胺-C=O | H键 | | | | | Asp382 | 酰胺-C=O | H键 | | | | | Met292 | 酰胺-NH | H键 | | | | | Phe383 | 嘧啶环 | Ar-Ar | | (Native ligand ⁹⁵) | 3BYM | −8.55 | Asp382 | 苯基 | Arene-H | | | | | Glu317 | 三嗪-CH | H键 | | | | | Met319 | 三嗪(N)-NH | H键 | | | | | Val259 | 苯并[d]咪唑 | Arene-H | | 5 | 3BYM | −6.63 | Asp382 | 苯氧基 | Arene-H | | | | | Gly322 | 嘧啶环 | Arene-H | | | | | Leu251 | 嘧啶环 | Arene-H | | | | | Met319 | NH₂ | H键 | | 6 | 3BYM | −5.98 | Asp382 | 嘧啶环 | Arene-H | | | | | Thr316 | 吡咯环 | Arene-H | | 7 | 3BYM | −6.57 | Lys273 | 4-Br苯氧基 | Arene-H | | | | | Met292 | 4-Br苯氧基(Br) | H键 | | | | | Met319 | NH₂ | H键 | | 8 | 3BYM | −6.46 | Asp382 | 4-Cl苯氧基(Cl) | H键 | | | | | Leu251 | 苯基 | Arene-H | | 9 | 3BYM | −6.66 | Leu371 | 苯基 | Arene-H | | | | | Thr316 | 苯氧基 | Arene-H | | 10 | 3BYM | −7.54 | Asp382 | NH₂ | H键 | | | | | Met319 | SO₂ | H键 | | | | | Tyr318 | SO₂ | H键 | | 11 | 3BYM | −6.89 | Leu371 | 苯基 | Arene-H | | | | | Thr316 | 苯氧基 | Arene-H | | 12 | 3BYM | −6.66 | Glu320 | 哌啶(NH) | H键 | | | | | Gly322 | 吡咯环 | Arene-H | | | | | Thr316 | 苯氧基 | Arene-H | | 13 | 3BYM | −7.71 | Asp382 | 苯基 | Arene-H | | | | | Gly322 | 嘧啶环 | Arene-H | | | | | Leu251 | 嘧啶环 | Arene-H | | 14 | 3BYM | −5.69 | Asp382 | 嘧啶环 | Arene-H | | | | | Thr316 | 吡咯环 | Arene-H | | | | | Val301 | 嘧啶(N) | H键 | | 15 | 3BYM | −6.38 | Leu251 | 苄基 | Arene-H | | | | | Thr316 | 嘧啶环 | Arene-H | | | | | Val259 | 吡咯 | Arene-H | | 16 | 3BYM | −6.64 | Asp382 | 苯基 | Arene-H | | | | | Gly322 | 嘧啶环 | Arene-H | | | | | Leu251 | 嘧啶环 | Arene-H | | | | | Met319 | NH₂ | H键 | | 17 | 3BYM | −6.44 | Asp382 | 苯氧基 | Arene-H | | | | | Gly322 | 嘧啶环 | Arene-H | | | | | Leu251 | 嘧啶环 | Arene-H | | | | | Met319 | NH₂ | H键 | | 18 | 3BYM | −6.97 | Gly322 | 吡咯环 | Arene-H | | | | | Ser323 | 嘧啶环 | Arene-H | | | | | Thr316 | 苯氧基 | Arene-H | | 19 | 3BYM | −6.61 | Asp382 | 苯氧基 | Arene-H | | | | | Gly322 | 嘧啶环 | Arene-H | | | | | Leu251 | 嘧啶环 | Arene-H | | | | | Met319 | NH₂ | H键 | | 20 | 3BYM | −6.62 | Asp382 | 3-CN苯氧基(CN) | H键 | | | | | Leu251 | 吡咯并嘧啶 | Arene-H | | | | | Thr316 | 苯基 | Arene-H | | 21 | 3BYM | −6.50 | Asp382 | 酰胺-(C=O) | H键 | | | | | Met319 | NH₂ | H键 | | | | | Val301 | 酰胺-(NH₂) | H键 | | 22 | 3BYM | −6.70 | Asp382 | 苯基 | Arene-H | | | | | Leu251 | 吡咯并嘧啶 | Arene-H | | | | | Met319 | NH₂ | H键 | | 23 | 3BYM | −6.81 | Leu251 | 苯氧基 | Arene-H | | | | | Lys269 | 4-CF₃苯基 | Arene-H | | | | | Thr316 | 呋喃(O) | H键 | | | | | Val259 | 嘧啶环 | Arene-H | | 24 | 3BYM | −5.86 | Leu251 | 噻吩环 | Arene-H | | | | | Met319 | 三唑(N) | H键 | | | | | Thr316 | 2-Cl,6-F-苯基 | Arene-H | | 25 | 3BYM | −6.17 | Met319 | 三唑(N) | H键 | | | | | Thr316 | 2-Cl,6-F-苯基 | Arene-H | | 26 | 3BYM | −6.57 | Asp382 | 2-Cl,6-F-苯基 | Arene-H | | 27 | 3BYM | −6.87 | Glu317 | 三唑(NH) | H键 | | | | | Leu251 | 3-NH₂,4-OMe苯基 | Arene-H | | | | | Met319 | 三唑(N) | H键 | | 28 | 3BYM | −6.34 | Met319 | 三唑(N) | H键 | | 29 | 3BYM | −6.16 | Met319 | 三唑(N) | H键 | | | | | Thr316 | 2-Cl,6-F-苯基 | Arene-H | | 30 | 3BYM | −7.00 | Leu251 | 噻唑环 | Arene-H | | | | | Met319 | 噻唑(S) | H键 | | | | | | NH | H键 | | 31 | 3BYM | −7.63 | Asp382 | 吡啶环 | Arene-H | | | | | Glu249 | 哌嗪(NH) | 金属/离子 | | | | | Gly322 | 嘧啶环 | Arene-H | | | | | Leu251 | 嘧啶环 | Arene-H | | | | | Leu371 | 噻唑环 | Arene-H | | | | | Met319 | 噻唑(N) | H键 | | | | | | NH | H键 | | 32 | 3BYM | −6.98 | Met319 | NH₂ | H键 | | | | | Val259 | 萘基 | Arene-H | | (Native ligand ⁹⁸) | 6PDJ | −11.39 | Asp382 | 酰胺-C=O | H键 | | | | | Met319 | 吡唑(N) | H键 | | | | | Phe283 | 吡唑环 | Arene-H | | 33 | 6PDJ | −7.59 | Met319 | 嘧啶(N) | H键 | | | | | Tyr318 | CH | Arene-H | | 34 | 6PDJ | −7.95 | Met319 | 嘧啶(N) | H键 | | 35 | 6PDJ | −7.17 | Met319 | 嘧啶(N) | H键 | | 36 | 6PDJ | −6.52 | Ala284 | 酰胺(NH) | H键 | | | | | Asp382 | 氮杂环丁烷(CH) | Arene-H | | | | | Met292 | 脲(NH) | H键 | | | | | Phe285 | 吲唑(吡唑环) | Arene-H | | | | | Phe256 | 3-Cl苯基 | Arene-H | | 38 | 6PDJ | −9.50 | Ala289 | CN基团 | H键 | | | | | Asp382 | 酰胺(NH) | H键 | | | | | Met292 | 吲唑(吡唑环) | H键 | | | | | Phe383 | 酰胺(NH) | Arene-H |

5.1. 卤代生物碱 通过HPLC-ESIMS(高效液相色谱-电喷雾质谱联用)引导分离海绵I. purpurea,获得十种多卤代生物碱(Purpuroine A–J)⁹⁹。对新分离的purpuroines进行了抗菌和激酶抑制活性测试。尽管初步测试仅针对三种激酶(Lck、细胞周期蛋白依赖性激酶2(CDK2)和 polo样激酶1(PLK1)),但purpuroines A(1)和D(2)(图5(A))对Lck激酶表现出强效抑制活性,IC₅₀值分别为2.35和0.94 µg/mL。Purpuroine D还能抑制PLK1,IC₅₀为1.45 µg/mL。作为对照,广谱蛋白激酶抑制剂星形孢菌素对Lck和PLK1的IC₅₀分别为3.73和0.92 µg/mL。所有purpuroines对CDK2的抑制活性均较弱(IC₅₀ > 50 µg/mL)。对三卤代类似物(包括最强效化合物purpuroine D)的初步构效关系分析表明,其抑制Lck的能力优于二卤代类似物如purpuroine B(3,图6(A))。

为深入理解该系列在Lck活性位点的不同结合模式并解释其生物活性差异,进行了分子对接模拟。对接研究表明,含三卤代苯氧基的化合物1(图6(B))和2通过Met319残基形成氢键深入嵌入铰链结合区,使其侧链羧基朝向Thr316形成氢键。而化合物3(图6(C))的二卤代苯氧基虽通过溴原子与Met319形成氢键,但未在腺嘌呤结合区与氨基酸残基形成额外氢键。

5.2. 8-甲基-1-苯基-咪唑并[l,5-a]吡嗪 利用Lck IMAP实验设计了一系列新型8-甲基-1-苯基-咪唑并[l,5-a]吡嗪类Lck抑制剂,获得了对Lck具有广泛pIC₅₀值(≥6 – ≥8)的衍生物¹⁰⁰。化合物4(图7(A))是其中对Lck具有强效抑制活性的代表之一,pIC₅₀ ≥ 8。化合物4在Lck活性位点的对接(图7(B),PDB ID: 2PL0)显示其结合行为与伊马替尼(Src家族中选择性抑制Lck的抑制剂)相似:化合物4的酰胺连接基团通过NH与Met292和Glu288主链形成氢键,保留关键的铰链结合相互作用;羰基部分与Ala381和Asp381的NH形成氢键。此外,并咪唑并嘧啶部分占据疏水口袋,与Phe383发生范德华相互作用。

5.3. 吡咯并嘧啶类 加拿大制药公司Pharmascience Inc.的Laurent等人获得了新型吡咯并嘧啶类Lck抑制剂的专利¹⁰¹。在分子水平上,评估了新合成化合物对Lck和布鲁顿酪氨酸激酶(Btk)的激酶抑制活性(以Kᵢ值表示)。通过脾细胞增殖实验,在细胞水平测定了EC₅₀值(与溶剂对照组相比,50%增殖时的化合物浓度)。如图8所示,选取了属于五种不同通用结构的十九个化合物,以阐明该新系列的构效关系(表2)。

表2. 化合物5–23对Lck的生物活性

| Cpd | Kᵢ Lck (nM) | |-----|-------------| | 5, 14, 15, 16, 17, 18, 19, 22, 23 | <100 | | 7, 8, 9, 10, 11, 12, 13 | >100 – <1000 | | 6, 20, 21 | >1000 |

研究发现,含环戊烯环的化合物5–8对Lck抑制活性范围较广:含未取代苯氧基的化合物5表现出强效抑制常数(Kᵢ < 100 nM);而含溴代和氯代苯氧基的化合物7和8的Kᵢ值较高(>100 – <1000 nM)。含对氟苯氧基的化合物6则完全丧失纳摩尔级活性(Kᵢ > 1000 nM)。将环戊烯环改为五元(未)取代的2,5-二氢-1H-吡咯(9和10)或扩环为六元环己烯(11)和1,2,3,6-四氢吡啶(12)可恢复中等活性(Kᵢ > 100 – <1000 nM)。在1,2,3,6-四氢吡啶环的自由NH上引入苯磺酰基(13)未能改善此中等活性,而引入小尺寸乙基(14)则显著提高对Lck的抑制活性(Kᵢ < 100 nM)。恢复五元环戊烯并替换苯氧基为苄基得到化合物15,同样表现出强效Lck抑制(Kᵢ < 100 nM)。尽管用五元2,5-二氢呋喃(16)或2-甲基-2,5-二氢呋喃(17–19)替代环戊烯并保持苯氧基可维持高活性,但在间位引入氰基(20)或甲酰胺基(21)则导致纳摩尔活性丧失。有趣的是,当苯氧基间位被吡啶-3-基甲氧基(22)或4-(三氟甲基)苄氧基(23)取代时,高活性得以恢复。

为理解五元环(环戊烯、2,5-二氢呋喃、2-甲基-2,5-二氢呋喃)相对于六元环1,2,3,6-四氢吡啶的活性优势,并阐明苯氧基间位取代对Lck生物活性的影响,进行了Lck活性位点的分子建模研究(PDB ID: 3BYM)。如表1所示,对接衍生物表现出不同的相互作用模式,但最强效抑制剂对酶活性位点具有最高亲和力。例如,化合物5(图9(A))通过多个相互作用深入嵌入口袋残基:通过Met319残基的氢键锚定于腺嘌呤区域;苯氧基未取代使其通过Arene-H键与Asp382主链深入相互作用进入疏水口袋;吡咯并嘧啶骨架通过Gly322和Leu251的疏水相互作用帮助固定化合物位置。相反,化合物6(图9(B))苯氧基的对氟取代导致化合物在活性位点翻转,使氨基远离铰链结合区,严重影响化合物在酶活性位点的稳定性并降低其活性。然而,如化合物10(图9(C)所示,将环戊烯替换为取代的2,5-二氢-1H-吡咯后活性中等,这可能归因于取代磺酰基(SO₂)在铰链结合区与Met319及在疏水口袋与Thr316形成两个氢键的贡献。

图9. 化合物5(A)、化合物6(B)和化合物10(C)在Lck激酶结构域活性位点的3D分子相互作用对接模型(PDB ID: 3BYM)。

5.4. 取代三唑类 北京国家生物科学研究所设计并合成了一系列取代三唑类化合物作为新型激酶抑制剂¹⁰²,¹⁰³。在针对七种自身免疫相关激酶(包括Lck、Btk、P38a、Fyn、Lyn、BMX和Blk)筛选的42个化合物中,仅两个化合物(24和25,图10)对Lck表现出强效且选择性的抑制活性,IC₅₀值低于0.1 µM。大多数其他化合物对Lck具有中等活性,IC₅₀范围为0.1–10 µM。值得注意的是,仅化合物26(图10)对Lck完全无活性(IC₅₀ > 10 µM)¹⁰²。研究还发现,同系列的化合物27、28和29(图10)虽对其他激酶具有高活性,但仍能在高纳摩尔IC₅₀范围内抑制Lck(分别为0.077 ± 0.022、0.018 ± 0.007和0.044 ± 0.02 µM)。该组的分子对接研究深入揭示了其在Lck活性位点的不同结合模式,并为其可变活性提供了合理解释。

强效衍生物24(图11(A))和25能够适配活性位点,其中三唑氮原子与铰链结合区的Met319氨基酸主链保持氢键相互作用;侧链取代的苄胺部分通过Arene-H相互作用朝向守门员口袋的Thr316残基。中等活性非选择性抑制剂27–29的构象保持中心三唑环在腺嘌呤结合区,但阻碍了苄基在疏水口袋的相互作用(图11(C))。化合物26未表现出铰链区的关键结合相互作用(图11(B))。

图11. 化合物24(A)、化合物26(B)和化合物27(C)在Lck激酶结构域活性位点的3D分子相互作用对接模型(PDB ID: 3BYM)。

5.5. 达沙替尼衍生的Lck抑制剂 达沙替尼(30,图12)是一种酪氨酸激酶抑制剂(TKI),已彻底改变慢性髓系白血病(CML)的治疗,慢性期CML现被视为可管理的慢性疾病。它是一种口服小分子抑制剂,在纳摩尔浓度下可抑制多种酪氨酸激酶,包括BCR-ABL1、c-Kit、EphA2、血小板衍生生长因子受体-b及Src激酶家族(如Src、Lck、Yes、Fyn)¹⁰⁴⁻¹⁰⁷。然而,达沙替尼在人体中主要经细胞色素P450酶3A4(CYP3A4)代谢,同时也是CYP3A4的时间依赖性抑制剂。因此,若患者同时使用强效CYP3A4抑制剂(如酮康唑、克拉霉素、吲哚那韦),达沙替尼剂量需显著降低,因这些药物可能使其血浆浓度升至不安全水平。出现骨髓抑制时应停用达沙替尼。此外,达沙替尼还抑制hERG(人类“Ether-a-go-go相关基因”),该离子通道参与心脏电活动和心跳协调。达沙替尼的半衰期极短,总体平均终末半衰期仅为3–5小时。

因此,近期为开发具有更好药理学和安全性的达沙替尼衍生新型抑制剂,Sennthenn等人报道了化合物31(图12),发现其对包括Lck在内的多种激酶具有抑制活性,IC₅₀值为1.5 nM¹⁰⁸。对接结构修饰的衍生物31(图13(B))显示,与先导化合物达沙替尼(图13(A))相比,其在Lck活性位点(PDB ID: 3BYM)的构象发生显著变化。虽然通过2-氨基噻唑中心骨架与Met319的主要铰链结合相互作用在修饰化合物中得以保留,但达沙替尼末端芳香酰胺被吡啶基酰胺取代的微小变化,使化合物通过Arene-H相互作用与Asp382深入结合至疏水口袋。这些构象变化还带来了额外的分子相互作用:嘧啶环与Gly322和Leu251氨基酸形成一对Arene-H相互作用;侧链哌嗪的氮原子与Glu249发生金属/离子相互作用。

图13. 达沙替尼(30)(A)和化合物31(B)在Lck激酶结构域活性位点的3D分子相互作用对接模型(PDB ID: 3BYM)。

5.6. 普罗丹衍生的Lck抑制剂 为寻找一种可用于Lck信号实时胞内研究的普罗丹衍生Lck抑制剂作为分子工具,Fling等人通过将普罗丹衍生的荧光团整合至激酶抑制剂的药效团中,发现了一种具有固有荧光特性的小分子ATP竞争性Lck抑制剂(32,IC₅₀ = 124 nM,图14(A))¹⁰⁹。化合物32在Lck活性位点的对接(PDB ID: 3BYM,图14(B))显示,吡唑并嘧啶主骨架通过化合物中氨基与Met319主链的氢键埋入腺嘌呤结合位点;萘基部分通过Arene-H相互作用与Val259结合,占据疏水口袋。

5.7. 苯氧基嘧啶类 韩国科学技术研究院(KIST)近期报道了一类新型苯氧基嘧啶骨架抑制剂,靶向Lck和FMS激酶用于炎症性疾病治疗¹¹⁰。该研究发现了一种新型Lck/FMS双重抑制剂(33,图15),对Lck和FMS激酶具有强效纳摩尔IC₅₀值(分别为22.0 ± 10.0和4.6 ± 0.05 nM),并表现出良好的抗炎效果。此外,化合物34和35(图15)的Lck IC₅₀值分别为0.0065 ± 0.002和0.006 ± 0.0005 µM,是该系列中