Chu Cong-Qiu
Division of Arthritis and Rheumatic Diseases, Oregon Health & Science University, Portland, OR, USA.
Rheumatology Section, Veterans Affairs Portland Health Care System, Portland, OR, USA.
Med Rev (2021). 2024 Jan 4;3(6):521-525. doi: 10.1515/mr-2023-0051. eCollection 2023 Dec.
The success and safety seen in treating complement-mediated hemolysis conditions has sparked the development of targeted therapies for rare autoimmune diseases, with expansion to more common autoimmune conditions. Various classes of drugs, including small molecules, peptides, monoclonal antibodies, and small interfering RNA (siRNA), are undergoing development to specifically address complement activity. A dual approach targeting both complement and other immune components may be required for autoimmune diseases characterized by inflammation and complex pathogenic mechanisms. siRNA, which suppresses complement production, is emerging as a potent therapeutic tool. Combining a complement-blocking siRNA drug with a treatment that reduces autoantibodies could prove clinically feasible and impactful in managing these conditions.
在治疗补体介导的溶血疾病中所见到的成功与安全性激发了针对罕见自身免疫性疾病的靶向治疗的发展，并扩展到更常见的自身免疫性疾病。包括小分子、肽、单克隆抗体和小干扰RNA（siRNA）在内的各类药物正在研发中，以专门针对补体活性。对于以炎症和复杂致病机制为特征的自身免疫性疾病，可能需要采用同时针对补体和其他免疫成分的双重方法。抑制补体产生的siRNA正在成为一种有效的治疗工具。将一种补体阻断性siRNA药物与一种减少自身抗体的治疗方法相结合，在管理这些疾病方面可能证明在临床上是可行且有效的。
