Alex’s Lemonade Stand presented UVA’s John Bushweller with a check for $250,000. He plans to use that money to explore and develop new precision medicine treatments for children with cancer. 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. 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.
“It targets one specific protein that we know is a causative agent for a number of types of pediatric cancer, so we think if we can turn that protein off that that will kill these cancers,” said Bushweller.
Bushweller is hopeful his more targeted approach will also lead to less long-term side effects of treatment.