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  • Title: Temsirolimus for metastatic desmoplastic small round cell tumor.
    Author: Thijs AM, van der Graaf WT, van Herpen CM.
    Journal: Pediatr Blood Cancer; 2010 Dec 15; 55(7):1431-2. PubMed ID: 20730888.
    Abstract:
    Desmoplastic small round cell tumor (DSRCT) is a rare and aggressive neoplasm that mostly occurs in young males. No curative treatment options currently exist for this type of tumor and long-term survival remains poor. In vitro rapamycin induces apoptotic death of JN-DSRCT-1 cells, a possible model for desmoplastic small round cell tumors in which the EWS gene is fused to the WT1 gene. We therefore demonstrate the prolonged activity of temsirolimus, an mTOR-inhibitor, in a patient with DSRCT.
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