Centre d'Investigation Cliniques Plurithématique 1433, INSERM U1116, CHRU Nancy, F-CRIN INI-CRCT, Nancy, France.
Department of Medicine, University of Michigan School of Medicine, Ann Arbor, MI, USA.
Lancet Healthy Longev. 2021 Jun;2(6):e371-e379. doi: 10.1016/S2666-7568(21)00061-1. Epub 2021 Jun 3.
Histone deacetylase inhibitors (HDACi) regulate gene expression via epigenetic mechanisms. Accumulating evidence suggests that HDACi exert antiproliferative, antioxidant, antineoplastic, and proapoptotic effects through epigenetic mechanisms. Furthermore, HDACi also exert antithrombotic and antifibrotic effects through regulation of thrombotic and fibrotic transduction mechanisms. One of the oldest HDACi is valproic acid, which was first synthesised in 1882. After the discovery of its anticonvulsant properties for the treatment of epilepsy, the use of valproic acid was extended to other conditions, such as bipolar disorder and migraine. Given the accumulating evidence supporting the role of HDACi in the treatment of multiple medical conditions beyond epilepsy, the interest in novel potential indications for HDACi has been renewed. Considering the pleotropic epigenetic effects of HDACi, future studies could assess their efficacy and safety for cardiovascular disease prevention and treatment; treatment of venous thrombosis, Alzheimer's disease, autoimmune and proinflammatory conditions, chronic thromboembolic pulmonary hypertension, and pulmonary arterial hypertension; and as a coadjuvant therapy for cancer. Adequately designed and powered clinical trials are required to assess the efficacy and safety of HDACi before their clinical repurposing.
组蛋白去乙酰化酶抑制剂(HDACi)通过表观遗传机制调节基因表达。越来越多的证据表明,HDACi 通过表观遗传机制发挥抗增殖、抗氧化、抗肿瘤和促凋亡作用。此外,HDACi 还通过调节血栓形成和纤维化转导机制发挥抗血栓形成和抗纤维化作用。最早的 HDACi 之一是丙戊酸,它于 1882 年首次合成。在发现其具有抗惊厥作用可用于治疗癫痫后,丙戊酸的用途扩展到其他疾病,如双相情感障碍和偏头痛。鉴于越来越多的证据支持 HDACi 在治疗癫痫以外的多种疾病中的作用,人们对新型潜在适应症的兴趣又重新燃起。考虑到 HDACi 的多效性表观遗传效应,未来的研究可以评估它们在心血管疾病预防和治疗中的疗效和安全性;治疗静脉血栓形成、阿尔茨海默病、自身免疫和炎症性疾病、慢性血栓栓塞性肺动脉高压和肺动脉高压;以及作为癌症的辅助治疗。在重新应用之前,需要进行充分设计和有力的临床试验来评估 HDACi 的疗效和安全性。