CHARLOTTESVILLE, United States – The Children's Cancer Research Fund (CCRF) has awarded US$250,000 to an innovative new approach to treating leukaemia (blood cancer) being developed at the UVA Cancer Center.

The grant to John H Bushweller, PhD, of the University of Virginia School of Medicine, is part of the national non-profit'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 paediatric leukaemia with a very poor prognosis," said Bushweller of UVA's Department of Molecular Physiology and Biological Physics. "For paediatric 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."

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 leukaemia or acute lymphocytic leukaemia. Bushweller and his team found in their lab models that blocking the ability of these abnormal proteins to bind to DNA completely prevented them from causing leukaemia. 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 leukaemia. Further, he hopes that the new approach can 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 [a] far better quality of life and reduced long-term health effects from the treatment," he said.

Children's Cancer Research Fund is a national non-profit dedicated to ending childhood cancer. It backs promising research from top scientists across the country, funding projects that could make the greatest impact for children battling cancer. Since 1981 the group has contributed more than US$200 million to research, support programmes for children and their families, and towards cancer awareness and education outreach efforts.

"At Children's Cancer Research Fund we select and fund the best and most innovative research to advance breakthrough treatments for children's cancer, and we are excited about Dr Bushweller's promising results," said Jean Machart, chief operating officer and interim chief executive officer at CCRF.