Comprehensive Transcriptomic Analysis of EWSR1:WT1 Targets Identifies CDK4/6 Inhibitors as an Effective Therapy for Desmoplastic Small Round Cell Tumors
This study of note identified a new potential therapeutic avenue for desmoplastic small round cell tumors (DSRCT). DSRCT are a type of aggressive, pediatric sarcoma characterized by the EWSR1:WT1 fusion oncogene and with a 5-year survival rate of 15-25%. In this study, “Comprehensive Transcriptomic Analysis of EWSR1:WT1 Targets Identifies CDK4/6 Inhibitors as an Effective Therapy for Desmoplastic Small Round Cell Tumors,” investigators determined that the fusion stimulated tumor cell growth in a specific cell pathway called the cyclin D–CDK4/6–RB axis. Additional studies indicated that treatment with the CDK4/6 inhibitor Palbociclib reduced cell growth in two DSRCT models. Additional studies need to be conducted, but this study supports clinical investigation of Palbociclib for treating DSRCT.