With the increasing epidemiology of
autoimmune disease worldwide, there is an urgent need for effective drugs with low cost in clinical treatment.
Triptolide, the most potent bioactive compound from traditional Chinese herb Tripterygium Wilfordii Hook F, possesses immunosuppression and anti-inflammatory activity. It is a potential
drug for the treatment of various
autoimmune diseases, but its clinical application is still restricted due to severe toxicity. In this review, the pharmacodynamic effects and pharmacological mechanisms of
triptolide in
autoimmune diseases are summarized.
Triptolide exerts
therapeutic effect by regulating the function of immune cells and the expression of
cytokines through inflammatory signaling pathways, as well as maintaining redox balance and gut microbiota homeostasis. Meanwhile, the research progress on toxicity of
triptolide to liver, kidney, reproductive system, heart, spleen, lung and gastrointestinal tract has been systematically reviewed. In vivo experiments on different animals and clinical trials demonstrate the dose- and time- dependent toxicity of
triptolide through different administration routes. Furthermore, we focus on the strategies to reduce toxicity of
triptolide, including chemical structural modification, novel drug delivery systems, and combination
pharmacotherapy. This review aims to reveal the potential therapeutic prospect and limitations of
triptolide in treating
autoimmune diseases, thus providing guiding suggestions for further study and promoting its clinical translation.