Therapeutic Potential of NTRK3 Inhibition in Desmoplastic Small Round Cell Tumor

Abstract

Our results indicate that EWSR1-WT1 directly activates NTRK3 expression in DSRCT cells, which are dependent on its expression and activity for growth. Pharmacologic inhibition of NTRK3 by entrectinib significantly reduces growth of DSRCT cells both <i>in vitro</i> and <i>in vivo</i>, providing a rationale for clinical evaluation of NTRK3 as a therapeutic target in DSRCT.