Danial Maarten, Stauffer Angela N, Wurm Frederik R, Root Michael J, Klok Harm-Anton
Institut des Matériaux and Institut des Sciences et Ingéniere Chimiques, École Polytechnique Fédérale de Lausanne, Laboratoire des Polymères , Bâtiment MXD, Station 12, 1015 Lausanne, Switzerland.
Department of Biochemistry and Molecular Biology, Thomas Jefferson University , 233 South 10th Street, Philadelphia, Pennsylvania 19107, U.S.A.
Bioconjug Chem. 2017 Mar 15;28(3):701-712. doi: 10.1021/acs.bioconjchem.6b00540. Epub 2016 Oct 27.
A popular strategy for overcoming the limited plasma half-life of peptide heptad repeat 2 (HR2) fusion inhibitors against HIV-1 is conjugation with biocompatible polymers such as poly(ethylene glycol) (PEG). However, despite improved resistance to proteolysis and reduced renal elimination, covalent attachment of polymers often causes a loss in therapeutic potency. In this study, we investigated the molecular origins of the loss in potency upon conjugation of linear, midfunctional, and hyperbranched PEG-like polymers to peptides that inhibit HIV-1-host cell membrane fusion. Fluorescence binding assays revealed that polymer conjugation imparted mass transport limitations that manifested as coexistent slower association and dissociation rates from the gp41 target on HIV-1. Furthermore, reduced association kinetics rather than affinity disruption was responsible for the loss in antiviral potency. Finally, the binding assays indicated that the unmodified HR2-derived peptide demonstrated diffusion-limited binding. The observed high potency of the unmodified peptide in HIV-1 inhibition assays was therefore attributed to rapid peptide conformational changes upon binding to the gp41 prehairpin structure. This study emphasizes that the view in which polymer ligation to therapeutic peptides inadvertently leads to loss in potency due to a loss in binding affinity requires scientific verification on a case-by-case basis and that high peptide potency may be due to rapid target-binding events.
克服针对HIV-1的肽七肽重复序列2(HR2)融合抑制剂血浆半衰期有限的一种常用策略是与生物相容性聚合物如聚乙二醇(PEG)缀合。然而,尽管对蛋白水解的抗性有所提高且肾脏清除率降低,但聚合物的共价连接通常会导致治疗效力的丧失。在本研究中,我们研究了将线性、中等官能度和超支化PEG样聚合物与抑制HIV-1-宿主细胞膜融合的肽缀合后效力丧失的分子起源。荧光结合试验表明,聚合物缀合导致了质量传递限制,表现为与HIV-1上的gp41靶点共存的较慢结合和解离速率。此外,结合动力学降低而非亲和力破坏是抗病毒效力丧失的原因。最后,结合试验表明未修饰的HR2衍生肽表现出扩散限制结合。因此,在HIV-1抑制试验中观察到的未修饰肽的高效力归因于与gp41前发夹结构结合后肽的快速构象变化。本研究强调,由于结合亲和力丧失,聚合物与治疗性肽连接会无意中导致效力丧失的观点需要逐案进行科学验证,并且高肽效力可能归因于快速的靶点结合事件。