UM171

UM171 is a small molecule drug that acts as a potent agonist of human hematopoietic stem cell (HSC) renewal. It functions primarily by promoting the degradation of lysine-specific demethylase 1 (LSD1) and histone deacetylase 1/2, thereby modulating epigenetic marks and enhancing HSC self-renewal capacity[5][3][8]. The mechanism involves modulation of the CoREST complex via activation of the CULLIN3-E3 ubiquitin ligase complex, with specificity conferred by KBTBD4, leading to proteasomal degradation of LSD1/RCOR1 corepressor components[8]. Additionally, UM171 reduces MYC activity in HSCs through KBTBD4-mediated degradation, helping preserve stem cell function under culture-induced stress[6]. Originally discovered for its ability to expand cord blood-derived HSCs ex vivo for transplantation in blood cancers such as leukemias and myelodysplasias[1], it is also being investigated for anti-cancer applications due to its effects on cancer stem cells and potential utility in solid tumors like breast cancer[7].

Other names

UM-171UM171UM 171