Janero David R, Yaddanapudi Suma, Zvonok Nikolai, Subramanian Kumar V, Shukla Vidyanand G, Stahl Edward, Zhou Lei, Hurst Dow, Wager-Miller James, Bohn Laura M, Reggio Patricia H, Mackie Ken, Makriyannis Alexandros
†Center for Drug Discovery and Departments of Chemistry and Chemical Biology and Pharmaceutical Sciences, Northeastern University, Boston, Massachusetts 02115, United States.
‡Departments of Molecular Therapeutics and Neuroscience, Scripps Research Institute, Jupiter, Florida 33458, United States.
ACS Chem Neurosci. 2015 Aug 19;6(8):1400-10. doi: 10.1021/acschemneuro.5b00090. Epub 2015 May 29.
The cannabinoid 1 receptor (CB1R) is one of the most abundant G protein-coupled receptors (GPCRs) in the central nervous system. CB1R involvement in multiple physiological processes, especially neurotransmitter release and synaptic function, has made this GPCR a prime drug discovery target, and pharmacological CB1R activation has been demonstrated to be a tenable therapeutic modality. Accordingly, the design and profiling of novel, drug-like CB1R modulators to inform the receptor's ligand-interaction landscape and molecular pharmacology constitute a prime contemporary research focus. For this purpose, we report utilization of AM3677, a designer endocannabinoid (anandamide) analogue derivatized with a reactive electrophilic isothiocyanate functionality, as a covalent, CB1R-selective chemical probe. The data demonstrate that reaction of AM3677 with a cysteine residue in transmembrane helix 6 of human CB1R (hCB1R), C6.47(355), is a key feature of AM3677's ligand-binding motif. Pharmacologically, AM3677 acts as a high-affinity, low-efficacy CB1R agonist that inhibits forskolin-stimulated cellular cAMP formation and stimulates CB1R coupling to G protein. AM3677 also induces CB1R endocytosis and irreversible receptor internalization. Computational docking suggests the importance of discrete hydrogen bonding and aromatic interactions as determinants of AM3677's topology within the ligand-binding pocket of active-state hCB1R. These results constitute the initial identification and characterization of a potent, high-affinity, hCB1R-selective covalent agonist with utility as a pharmacologically active, orthosteric-site probe for providing insight into structure-function correlates of ligand-induced CB1R activation and the molecular features of that activation by the native ligand, anandamide.
大麻素1受体(CB1R)是中枢神经系统中最丰富的G蛋白偶联受体(GPCR)之一。CB1R参与多种生理过程,尤其是神经递质释放和突触功能,这使得该GPCR成为主要的药物发现靶点,并且药理学上CB1R激活已被证明是一种可行的治疗方式。因此,设计和分析新型的、类药物的CB1R调节剂以了解受体的配体相互作用格局和分子药理学,构成了当代的主要研究重点。为此,我们报告了利用AM3677,一种经反应性亲电异硫氰酸酯官能团衍生化的设计型内源性大麻素(花生四烯乙醇胺)类似物,作为一种共价的、CB1R选择性化学探针。数据表明,AM3677与人CB1R(hCB1R)跨膜螺旋6中的半胱氨酸残基C6.47(355)反应,是AM3677配体结合基序的关键特征。在药理学上,AM3677作为一种高亲和力、低效的CB1R激动剂,抑制福斯可林刺激的细胞cAMP形成,并刺激CB1R与G蛋白偶联。AM3677还诱导CB1R内吞和不可逆的受体内化。计算对接表明,离散的氢键和芳香相互作用作为AM3677在活性态hCB1R配体结合口袋内拓扑结构的决定因素的重要性。这些结果构成了对一种强效、高亲和力、hCB1R选择性共价激动剂的初步鉴定和表征,该激动剂可用作药理活性的正构位点探针,以深入了解配体诱导的CB1R激活的结构-功能相关性以及天然配体花生四烯乙醇胺激活的分子特征。