Malignant rhabdoid tumors (MRT) are rare but deadly pediatric tumors characterized by mutations in the SMARCB1/SNF5/INI1/BAF47 gene. Currently, there are no targeted therapies for MRTs. In a previous issue of Cancer Research, Howard and colleagues utilize the power of genome-wide RNAi and CRISPR screening to identify MDM2 and MDM4 as potential drug targets for MRTs. Most MRTs retain an intact p53 pathway and the authors show that these cells are particularly sensitive to MDM2 and MDM4 inhibition due to SMARCB1's role in regulating p53-depedent apoptotic genes. This discovery suggests potential clinical trials of MDM2 inhibitors in patients with MRT.See related article by Howard and colleagues; Cancer Res 79(9):2404-14.
©2019 American Association for Cancer Research.