Researchers to use $6.3M grant to develop novel therapeutic approaches for pediatric sarcomas

Researchers at Nationwide Children's Hospital will use a $6.3 million grant to further their study of pediatric sarcomas, a rare form of the disease that affects bone or soft tissue and accounts for 11 percent of all childhood cancers. The project, funded by the National Institutes of Health and the National Cancer Institute, will be led by Peter Houghton, PhD, director of the Center for Childhood Cancer and Blood Diseases in The Research Institute at Nationwide Children's.

"The ultimate goal of this project is to develop novel therapeutic approaches for advanced childhood sarcoma," said Dr. Houghton, who has spent more than three decades studying pediatric cancer through work designed to bring knowledge from the laboratory to the bedside.

While more than 70 percent of children with sarcoma are cured, the outcome is still poor for those with advanced or metastatic disease. Specifically, the five-year, event-free survival rates are 30 percent or less in children with advanced or metastatic Ewing sarcoma, osteosarcoma or rhabdomyosarcoma. Intensive chemo-radiotherapy has not significantly altered this outcome, making the search for effective new therapies a critical pursuit.

Each of the three sarcomas targeted by this grant has distinct characteristics requiring in-depth analysis of disease pathways and treatment opportunities.

"The projects will characterize the interrelationship of these pathways and identify combinatorial inhibitory approaches most likely to yield biologic activity in the clinical setting," said Dr. Houghton, also a faculty member at The Ohio State University College of Medicine.

Experts in sarcoma biology, cellular signaling pathways and drug development will collaborate on the grant, bringing together researchers from Nationwide Children's and The Ohio State University. Dr. Houghton is the principal investigator on the grant, and Nationwide Children's is responsible for its management. The researchers will collaborate on each of six smaller sections of the grant, sharing personnel and facilities. Each division of the grant will be overseen by a special director and team to create the best opportunity for synergy and innovation.