Targeting ALK Mutations In Children With Neuroblastoma

A collaboration between CHOP oncologists and researchers at UPenn aims to provide targeted therapy to children with neuroblastoma, based on their ALK mutation status.

Oncology researchers studying gene mutations in the childhood cancer neuroblastoma are refining their diagnostic tools to predict which patients are more likely to respond to drugs called ALK inhibitors that target such mutations. Removing some of the guesswork in diagnosis and treatment, the researchers say, may lead to more successful outcomes for children with this often-deadly cancer.

“Some mutations are more important than others,” said Yaël P. Mossé, MD, a pediatric oncologist at The Children’s Hospital of Philadelphia, and a co-leader of the new study published online today in the journal Cancer Cell. “By integrating biochemistry into our clinical strategies, we can better match a patient’s specific ALK-mutation profile with an optimum treatment.” Mossé is also an assistant professor of Pediatrics in the Perelman School of Medicine, University of Pennsylvania.

“Understanding the specific mutations that trigger signals in cell receptors to stimulate cell growth will help us identify biomarkers for specific subtypes of neuroblastoma,” said study co-leader Mark A. Lemmon, PhD, professor and chair of Biochemistry and Biophysics at Penn. Lemmon’s research focuses on cell receptors in cancer.

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