Loss-of-function variants in the ELP1 gene are the most prevalent predisposing genetic factors in childhood medulloblastoma, accounting for about 30% of sonic hedgehog medulloblastoma (SHH-3 subtype).
Previous research revealed that nuclear factor of activated T cells 1 (NFAT1) is a novel regulator of the mouse double minute 2 homolog (MDM2) oncogene, which acts by directly binding to the MDM2 P2 promoter and as such, enhancing MDM2 transcription independent of p53. Researchers from the University of Houston and Baylor College of Medicine presented preclinical data for MA-242, a dual inhibitor of MDM2 and NFAT1, being developed for the treatment of cancer.
Roivant Discovery Inc. has disclosed PROTACs (proteolysis targeting chimeras) comprising an E3 ubiquitin ligase binding moiety covalently linked to an E3 ubiquitin-protein ligase Mdm2 (Hdm2)-targeting moiety through a linker.
Lamassu Biotech Inc. has announced that its IND for SA53-OS has been cleared by the FDA in the U.S. The planned phase I/IIa trials will investigate the genetically targeted therapy that blocks the MDM2 protein, a key regulator of the tumor suppressor p53 gene.
Unnatural Products Inc. has synthesized cell-permeable cyclic peptides acting as E3 ubiquitin-protein ligase Mdm2 (Hdm2) and/or Mdm4 (Mdmx) inhibitors reported to be useful for the treatment of cancer.
