mira-1

MIRA-1 (1-[(1-Oxopropoxy)methyl]-1-pyrrole-2,5-dione), also known as NSC 19630, is a small molecule maleimide derivative originally identified as a mutant p53-reactivating agent. It induces apoptosis and inhibits cell viability, colony formation, and migration in a variety of tumor cells, including multiple myeloma, regardless of p53 status[1]. MIRA-1 was selected from a pharmacological screen for compounds that can restore wild-type conformation and function to mutant p53, but it also exerts anti-tumor effects in cells with wild-type p53. Its mechanism includes upregulation of pro-apoptotic proteins Puma and Bax, downregulation of anti-apoptotic Mcl-1, and reduction of c-Myc[1]. MIRA-1 triggers endoplasmic reticulum stress, indicated by activation of PERK and IRE-α and splicing of XBP1, and shows synergistic effects when combined with conventional drugs such as dexamethasone, doxorubicin, or bortezomib (velcade)[1]. There is no evidence from the literature that MIRA-1 is a combination drug, formulation, biosimilar, or generic of another agent. The drug is still in preclinical development and has not progressed to clinical trials in humans[1].