A Novel Epigenetic Strategy to Treat Ewing Sarcoma

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Mary Beckerle, PhD

2013-2016

Project: A Novel Epigenetic Strategy to Treat Ewing Sarcoma

Mary Beckerle
  • Mary Beckerle, PhD
  • Ralph E. and Willia T. Main Presidential Professor
    CEO and Director, Huntsman Cancer Institute
    The University of Utah

Ewing sarcoma is the most common bone cancer in children. The disease is typically aggressive and difficult to treat. Treatment of localized tumors involves surgery and chemotherapy. Despite these interventions, the vast majority of tumors move to other locations in the body (metastasize). Patients with metastatic or recurrent disease face poor prognosis with less than a 20% five-year survival rate. Therapeutic advanced for Ewing sarcoma are desperately needed.

Ewing sarcoma occurs because of a chromosomal abnormality that causes an atypical protein, known as EWS/FLI, to be expressed. Mary Beckerle, PhD and a collaborative team at the Huntsman Cancer Institute have demonstrated that the EWS/FLI mutation disrupts the internal cellular skeleton, which allows cells to move out of their normal environment. A cell that does not remain in its normal environment is more likely to travel to another area of the body, facilitating the spread of the tumor. Therefore, being able to stop EWS/FLI from changing a cell’s “stickiness” might help stop the spread of cancer. Dr. Beckerle’s team identified a novel targeted treatment for Ewing sarcoma that disrupts the effects of EWS/FLI and disrupts the spread of the cancer. Dr. Beckerle’s Acceleration Award contributed to the clinical studies required to move this novel therapy into clinical trials.

As of the conclusion of her CureSearch-funded project, Dr. Beckerle has supported the movement of a new therapeutic for Ewing sarcoma into the clinic:

  • 1 new metastatic Ewing sarcoma mouse model created
  • 1 novel drug carried through preclinical testing and licensed to Salarius Pharmaceuticals
  • 1 IND application filed for drug assessed during CureSearch funding
  • 1 Phase I clinical trial initiated for Ewing sarcoma based on data obtained during the award period
“Discovering new targeted treatments for metastatic Ewing sarcoma.”

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