UVA Cancer Center receives $250,000 to develop innovative new approach to treating leukemia

Children's Cancer Research Fund has awarded $250,000 to an innovative new approach to treating leukemia – blood cancer – being developed at UVA Cancer Center.

The grant to John H. Bushweller, PhD, of the University of Virginia School of Medicine, is part of the national nonprofit's efforts to accelerate the development of new and better treatments for difficult-to-treat cancers.

This funding makes it possible to continue developing a novel approach to treatment for a form of pediatric leukemia with a very poor prognosis. For pediatric cancers in particular, the development of highly targeted approaches that directly inhibit the protein that drives the cancer has the potential to be more effective and to limit the substantial toxicity of current approaches, vastly improving outcomes for these patients. This is exactly the approach we are pursuing with the support of CCRF."

John H. Bushweller, PhD, UVA's Department of Molecular Physiology and Biological Physics

Better leukemia treatment

Bushweller and his team are developing drugs to block the effect of an abnormal protein that is formed when the protein MLL becomes fused to other proteins and alters the cell, resulting in either acute myeloid leukemia or acute lymphocytic leukemia. Bushweller and his team found in their lab models that blocking the ability of these abnormal proteins to bind to DNA completely prevented it from causing leukemia. Based on those results, they are working to develop drugs that disrupt this DNA binding as a novel approach for treatment.

Based on the promising results so far, Bushweller believes his new approach could be both more effective and less toxic than existing options for treating leukemia. Further, he hopes that the new approach could be combined with existing drugs to provide unique benefits for patients.

"The potential benefits to patients include improved efficacy leading to better outcomes and much reduced toxicity leading to far better quality of life and reduced long-term health effects from the treatment," he said.