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Small Molecule Inhibitors of ERG for Pediatric Leukemia and Sarcoma

Institution: 
University of Virginia, School of Medicine
Researcher(s): 
John Bushweller, PhD
Grant Type: 
Innovation Grants
Year Awarded: 
2015
Type of Childhood Cancer: 
Acute Myeloid Leukemia (AML), Ewing Sarcoma
Project Description: 

Background:

Current treatment for childhood acute myeloid leukemia (AML), T-ALL, and Ewing's sarcoma is limited in efficacy and has profound long-term side-effects due to the use of traditional cytotoxic agents rather than targeted drugs inhibiting specific drivers of the diseases. ERG is a protein which reads the DNA and regulates how much of many other proteins is made. ERG is altered or produced at an abnormally high level in Ewing's sarcoma, T-ALL, and AML. Studies in several labs show this alteration of ERG function is important for these diseases.

Project Goal:

Therefore, a targeted agent which inhibits ERG, clearly a driver of these diseases, has the potential to improve both survival and quality of life for children with AML, T-ALL, and Ewing's sarcoma. We are proposing to develop inhibitors of ERG as a novel approach to treatment for childhood AML, T-ALL, and Ewing's sarcoma.

Co-funded by: 
I Care I Cure
The Brian Morden Foundation