Helen DeVos Childrens Hospital Archives

2017

Young Investigator

Dr. Jenna Gedminas

Grand Rapids, MI

Development of a Novel Therapeutic Approach for Desmoplastic Small Round Cell Tumor

Desmoplastic small round cell tumor (DSRCT). is a rare, aggressive malignancy that develops in the abdominal peritoneum and is typically highly metastatic at the time of diagnosis. It is most commonly seen in adolescent and young adult males and prognosis is extremely poor, with a 5-year survival of less than 15%. There is currently no accepted treatment for this disease.
In nearly all cases of DSRCT, the tumor contains a specific translocation, which fuses the EWSR1 gene to the WT1 gene creating transcription factor that drives tumor growth. Previous therapies targeting specific receptors in DSRCT including IGF1 R, VEG FR, and PDGFR have been tried with little success.

The goal of this study is to develop a new therapeutic approach for Desmoplastic Small Round Cell Tumor (DSRCT). The approach is to target the oncogenic driver of the disease, the EWS-WT1 transcription factor, using a compound that we have shown inhibits a related EWS fusion protein in another tumor.