Cardiothoracic Pharmacology, NHLI, Imperial College London, United Kingdom.
PLoS One. 2010 Mar 4;5(3):e9526. doi: 10.1371/journal.pone.0009526.
Pulmonary vascular diseases are increasingly recognised as important clinical conditions. Pulmonary hypertension associated with a range of aetiologies is difficult to treat and associated with progressive morbidity and mortality. Current therapies for pulmonary hypertension include phosphodiesterase type 5 inhibitors, endothelin receptor antagonists, or prostacyclin mimetics. However, none of these provide a cure and the clinical benefits of these drugs individually decline over time. There is, therefore, an urgent need to identify new treatment strategies for pulmonary hypertension.
METHODOLOGY/PRINCIPAL FINDINGS: Here we show that the PPARbeta/delta agonist GW0742 induces vasorelaxation in systemic and pulmonary vessels. Using tissue from genetically modified mice, we show that the dilator effects of GW0742 are independent of the target receptor PPARbeta/delta or cell surface prostacyclin (IP) receptors. In aortic tissue, vascular relaxant effects of GW0742 were not associated with increases in cGMP, cAMP or hyperpolarisation, but were attributed to inhibition of RhoA activity. In a rat model of hypoxia-induced pulmonary hypertension, daily oral dosing of animals with GW0742 (30 mg/kg) for 3 weeks significantly reduced the associated right heart hypertrophy and right ventricular systolic pressure. GW0742 had no effect on vascular remodelling induced by hypoxia in this model.
CONCLUSIONS/SIGNIFICANCE: These observations are the first to show a therapeutic benefit of 'PPARbeta/delta' agonists in experimental pulmonary arterial hypertension and provide pre-clinical evidence to favour clinical trials in man.
肺血管疾病日益被认为是重要的临床病症。由多种病因引起的肺动脉高压难以治疗,且与进行性发病率和死亡率相关。目前肺动脉高压的治疗方法包括磷酸二酯酶 5 抑制剂、内皮素受体拮抗剂或前列环素类似物。然而,这些方法都无法治愈疾病,而且这些药物的临床疗效会随时间推移而逐渐下降。因此,迫切需要寻找肺动脉高压的新治疗策略。
方法/主要发现:我们发现过氧化物酶体增殖物激活受体β/δ(PPARβ/δ)激动剂 GW0742 可引起体循环和肺血管舒张。通过使用基因修饰小鼠的组织,我们发现 GW0742 的舒张作用不依赖于其靶受体 PPARβ/δ或细胞表面前列环素(IP)受体。在主动脉组织中,GW0742 的血管舒张作用与 cGMP、cAMP 或超极化的增加无关,而是归因于 RhoA 活性的抑制。在缺氧诱导的肺动脉高压大鼠模型中,GW0742(30mg/kg)每日口服治疗 3 周,可显著减轻相关的右心肥厚和右心室收缩压。在该模型中,GW0742 对缺氧引起的血管重塑没有影响。
结论/意义:这些观察结果首次表明“PPARβ/δ”激动剂在实验性肺动脉高压中有治疗益处,并为临床试验提供了临床前证据。