Temsirolimus for metastatic desmoplastic small round cell tumor

Pediatr Blood Cancer. 2010 Dec 15;55(7):1431-2. doi: 10.1002/pbc.22755.

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.

Publication types

  • Case Reports

MeSH terms

  • Abdominal Neoplasms / pathology*
  • Antineoplastic Agents / therapeutic use*
  • Humans
  • Liver Neoplasms / drug therapy
  • Liver Neoplasms / secondary
  • Lung Neoplasms / drug therapy
  • Lung Neoplasms / secondary
  • Lymphatic Metastasis
  • Male
  • Protein Kinase Inhibitors / therapeutic use*
  • Sirolimus / analogs & derivatives*
  • Sirolimus / therapeutic use
  • TOR Serine-Threonine Kinases / antagonists & inhibitors*
  • Young Adult

Substances

  • Antineoplastic Agents
  • Protein Kinase Inhibitors
  • temsirolimus
  • TOR Serine-Threonine Kinases
  • Sirolimus