Childhood Cancer Research Growing Up

Neuroblastoma treatment research will require different perspective of genomics

January 25, 2013 / Author:  / Reviewed by: Joseph V. Madia, MD

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(dailyRx News) The goal of personalized therapy is to identify and target a patient’s unique genetic makeup. When it comes to cancer, though, the disease can keep causing new genetic changes. In childhood cancer, this can be particularly challenging.

A new study of the childhood cancer neuroblastoma has uncovered very few genetic mutations that can be targeted with personalized therapy. As a result, researchers are now looking at how tumors change in response to treatment.

Neuroblastoma is a rare cancer of the nervous system that occurs almost always in children under the age of 10. It’s diagnosed in about 750 children in the US every year. Most cases appear around the age of 2, and it’s the most common cancer in infants.

"Take your child to the doctor if you notice a bulging in the abdomen."

John M. Maris, MD, director of the Center for Childhood Cancer Research at The Children’s Hospital of Philadelphia (CHOP), led the multi-center study called TARGET (Therapeutically Applicable Research to Generate Effective Treatments).

Researchers examined the DNA from 240 children who had high-risk neuroblastomas. The DNA found in the children's tumors was compared to the DNA in healthy cells. The study goal was to use this information to outline various treatment options for this aggressive cancer.

Of this group, about 10 percent of the children had a mutation in the ALK gene, which clinicians are able to treat with some success. However, there were very few other genetic mutations that were consistently seen in the neuroblastoma patients.

The results only confirmed the complexity of treating this disease.

Dr. Maris said in a statement, “Personalized medicine is more complex than we had hoped. While there are successes such as those in treating patients whose tumors harbor ALK mutations, this study implies that we must think very differently about how we’ll use genomics to define treatment.”

The authors concluded that scientists will have to change their thinking about this disease. Instead of looking at static genetic mutations, researchers will have to look at the dynamic nature of the disease, which is constantly changing as the child’s nervous system develops.

Findings from the TARGET study were published January 20 in Nature Genetics.

Support for this study came from the National Institutes of Health, the Canada Research Chair in Genome Science, the Giulio D’Angio Endowed Chair, the Alex’s Lemonade Stand Foundation, the Arms Wide Open Foundation and the Cookies for Kids Foundation. No conflicts of interest were reported.

Reviewed by: 
Review Date: 
January 24, 2013
Last Updated:
January 25, 2013