Neuroblastoma research gets a boost

July 17, 2013 | by

Pediatric neuroblastomas are rare, but many children diagnosed never recover from the disease – and treatment options are few. Better options for these patients may now be a bit closer.

Neuroblastomas are cancers in the peripheral nervous system that affect mainly infants and children.

Neuroblastomas are cancers in the peripheral nervous system that affect mainly infants and children.

Linda Malkas, Ph.D., deputy director of basic research at City of Hope, has been awarded $100,000 by the St. Baldrick’s Foundation for research on pediatric neuroblastomas. Already, her work has suggested that a certain peptide could hold the key to an effective treatment.

Neuroblastomas are rare tumors of the peripheral nervous system that affect about 600 U.S. children a year. Fewer than half of patients with the disease are cured, even with the use of high-dose chemotherapy followed by bone marrow transplant.

“These funds are allowing us to explore a new research avenue,” Malkas said. “Our laboratory is so grateful for this support, and we will do our best to ensure the people who donated to St. Baldrick’s see that we are committed to the completion of this research. It is our hope that this work points us toward a new path to treat children with neuroblastoma.”

Recent data discovered by researchers in Malkas’ lab suggests that a specific peptide they’ve discovered and have been studying could be useful in treating neuroblastomas. When used in a laboratory model, the peptide has been effective in fighting the tumors, particularly those with a certain gene amplification that is associated with more severe disease.

This peptide is uniquely expressed by neuroblastoma cells, and a small portion of the peptide can be used as a decoy to bind up other peptides to selectively kill cancer cells. The grant money will be put toward understanding the mechanism causing the peptide to work so that treatments can be developed around it, Malkas said.

St. Baldrick’s is the largest private funder of childhood cancer research grants, awarding more than $125 million to support lifesaving research since 2005.

Malkas is among more than 50 researchers and institutions awarded a total of $22 million in funding today by St. Baldrick’s Foundation, a volunteer-driven and donor-centered charity dedicated to funding childhood cancer research. Worldwide, a child is diagnosed with cancer every three minutes, and one in five children diagnosed in the U.S. will not survive.