Small Molecule Inhibitors of ERG for Pediatric Leukemia and Sarcoma

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.