Speeding Up Success With Precision Medicine Cancer Trial

Researchers are increasingly finding that neuroblastoma, like many childhood cancers, is much more complicated than it first appeared. An innovative new clinical trial launching in 2016 at The Children’s Hospital of Philadelphia aims to address the numerous molecular and genetic causes of neuroblastoma with a dynamic design. 

This method allows researchers to quickly translate findings from the lab based on the evolving individual characteristics of each patient’s tumor. It is the first time such a strategy is being applied to a prospective clinical trial in children with cancer. Known as the NExt-generation Personalized NEuroblastoma THErapy (NEPENTHE) trial, it may be a model for how precision medicine clinical trials can spur better and faster cancer therapy discoveries in the future.

Neuroblastoma, a cancer of the nervous system that remains difficult to cure, accounts for a disproportionate share of cancer deaths in children, despite many recent improvements in therapy. The many different underlying genetic and molecular causes of neuroblastoma can interact to affect treatment responsiveness and change over time in cases where the cancer relapses after treatment. These biological patterns have not routinely been addressed in the design of traditional clinical trials for childhood cancers.

“The novelty of this trial could be viewed on numerous levels,” said principal investigator Yael Mossé, MD, a CHOP pediatric oncologist and assistant professor at the Perelman School of Medicine at the University of Pennsylvania. “It’s based on rigorous preclinical data, understanding the molecular drivers that are important in this disease. It’s combining multiple novel drugs, not just one at a time. And it’s bringing that to the clinic and assigning patients to therapy based on what their tumor genetics are teaching us at the time that they meet us with relapsed or refractory cancer.”

NEPENTHE has received funding from the Band of Parents and Arms Wide Open, dedicated to supporting new treatments for neuroblastoma, Solving Kids’ Cancer Foundation, the Open Hands Overflowing Hearts Foundation, and a $1.5 million grant from Alex’s Lemonade Stand Foundation.

See the full version of this article in the January 2016 issue of Bench to Bedside.

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