Wu Wenze, Kong Fanhao, Chen Zhen, Huang Lixin, Nie Xiaowei, Bian Jin-Song
Department of Pharmacology Joint Laboratory of Guangdong-Hong Kong Universities for Vascular Homeostasis and Diseases, School of Medicine, Southern University of Science and Technology Shenzhen Guangdong China.
Department of Human Cell Biology and Genetics, School of Medicine Southern University of Science and Technology Shenzhen Guangdong China.
J Am Heart Assoc. 2025 Aug 5;14(15):e042713. doi: 10.1161/JAHA.125.042713. Epub 2025 Jul 29.
Pulmonary arterial hypertension (PAH) is a severe cardiovascular disease with high morbidity, significant mortality, and poor prognosis. To date, the pathogenesis of PAH is still not clarified, and the effectiveness of current treatment strategies is limited. Therefore, it is essential to understand the pathophysiology of PAH and to explore optimal treatment approaches. RTKs (receptor tyrosine kinases) are a family of enzyme-linked receptors that can be bound with specific ligands and then phosphorylate tyrosine residues of substrate targets, playing a crucial role in various biological processes. Accumulating evidence indicates that RTKs are strongly associated with the pathogenesis of PAH. Interestingly, even the same RTK may exhibit opposite effects in PAH under different circumstances. We hypothesize that this may stem from factors such as genetic background, cell line specificity, hypoxia gradient variations, and other underlying mechanisms. Although several RTK-targeting small molecule drugs have entered clinical trials, concerns regarding their toxicity and limited efficacy have been reported. To facilitate the clinical translation of these therapeutic agents, we highlight the pressing need for developing highly selective drugs (eg, monoclonal antibodies) and more sophisticated drug delivery systems. In this review, we therefore summarized and constructed a multilevel regulatory framework of RTKs with PAH pathogenesis, aiming to guide future clinical practice.
肺动脉高压(PAH)是一种严重的心血管疾病,发病率高、死亡率显著且预后较差。迄今为止,PAH的发病机制仍未阐明,当前治疗策略的有效性也有限。因此,了解PAH的病理生理学并探索最佳治疗方法至关重要。受体酪氨酸激酶(RTKs)是一类酶联受体,可与特定配体结合,然后使底物靶点的酪氨酸残基磷酸化,在各种生物过程中发挥关键作用。越来越多的证据表明,RTKs与PAH的发病机制密切相关。有趣的是,即使是相同的RTK在不同情况下在PAH中也可能表现出相反的作用。我们推测这可能源于遗传背景、细胞系特异性、缺氧梯度变化等因素以及其他潜在机制。尽管几种靶向RTK的小分子药物已进入临床试验,但有关其毒性和疗效有限的问题已有报道。为促进这些治疗药物的临床转化,我们强调迫切需要开发高选择性药物(如单克隆抗体)和更先进的药物递送系统。因此,在本综述中,我们总结并构建了一个与PAH发病机制相关的RTKs多层次调控框架,旨在指导未来的临床实践。