According to a new study published today in the journal Nature Genetics, researchers from various sites around the world have found that the majority of pediatric patients with relapsed neuroblastoma studied harbored mutations that promoted RAS-MAPK pathway signaling. Increased signaling in this pathway results in the progression of neuroblastoma.
"Our greater understanding of mutations on a crucial biological pathway offers prospects of acting on these pathways for direct benefit to children with this aggressive form of neuroblastoma," said senior author John M. Maris, M.D., a pediatric oncologist at The Children's Hospital of Philadelphia.
A class of drugs known as MEK inhibitors have demonstrated activity against this signaling pathway, suggesting that MEK inhibitors may benefit relapsed patients who possess mutations on the RAS-MAPK pathway.
Neuroblastoma is a solid tumor of the peripheral system that often grows in the chest or abdomen. It is the most common type of cancer in infants and is known for a high mortality rate in children. Most patients initially respond to chemotherapy, but many relapse and die from the disease.
Researchers studying the pediatric cancer neuroblastoma have detailed how cancer-driving mutations evolve during chemotherapy, and they hope to exploit this knowledge to design better treatments for children.