thalidomide

Thalidomide is an immunomodulatory and anti-inflammatory small molecule drug originally developed as a non-barbiturate hypnotic. It was withdrawn from the market in the 1960s due to severe teratogenic effects but has since been reintroduced for specific indications under strict regulation. Thalidomide is used primarily in combination with dexamethasone to treat multiple myeloma and alone or with other medications to treat and prevent skin symptoms of erythema nodosum leprosum (ENL) associated with leprosy[1][2][4]. Its mechanism of action involves modulation of the immune system, including inhibition of tumor necrosis factor-alpha (TNF-α), interleukin-6 (IL-6), and angiogenesis through suppression of basic fibroblast growth factor (bFGF) and vascular endothelial growth factor (VEGF)[4][8]. The direct molecular target identified for thalidomide is cereblon (CRBN), a substrate receptor for the E3 ubiquitin ligase complex CRL4, which mediates some of its therapeutic as well as teratogenic effects[8]. Due to its teratogenicity, use is restricted via special risk management programs.

Brand names

Thalomid

Other names

N-PhthaloylglutamimideN-Phthalylglutamic acid imide3-Phthalimidoglutarimidealpha-Phthalimidoglutarimide2,6-Dioxo-3-phthalimidopiperidineN-(2,6-Dioxo-3-piperidyl)phthalimide2-(2,6-dioxopiperidin-3-yl)isoindoline-1,3-dione