Grant to CHOP funds personalized treatments for children with neuroblastoma

December 10, 2015

A new $1.5 million grant to The Children's Hospital of Philadelphia (CHOP) from Alex's Lemonade Stand Foundation (ALSF) supports an innovative approach to treating relapsed or refractory neuroblastoma, a high-risk childhood cancer.

CHOP researchers are launching a pediatric clinical trial with a dynamic design allowing them to quickly incorporate new treatments based on ongoing lab studies of evolving genetic changes in an individual patient's tumor. The researchers will match those gene changes with available drugs.

"This is the first time this strategy is being applied to a prospective clinical trial in children with cancer," said principal investigator Yael P. Mosse, M.D., a pediatric oncologist at CHOP. "Our hope is that this trial will change the paradigm of treating children with neuroblastoma, offer a new model for improving treatment of other childhood cancers, and serve as a blueprint for similar trials in other diseases."

The three-year Bio-Therapeutics Impact Award from ALSF seeks to strategically advance research-based treatment of neuroblastoma, an-often lethal childhood cancer that remains difficult to cure. Usually appearing as a solid tumor in the chest or abdomen, neuroblastoma accounts for a disproportionate share of cancer deaths in children, despite many recent improvements in therapy.

"Through the dedication and tireless efforts of Alex's Lemonade Stand Foundation's volunteers, we are attempting to make it possible for childhood cancer patients to become childhood cancer survivors," said Jay Scott, executive director of Alex's Lemonade Stand Foundation. "With every grant we are able to award, we are one step closer to finding those cures our daughter dreamed of."

The CHOP physician-scientists expect to begin the next-generation personalized neuroblastoma therapy (NEPENTHE) trial in 2016. "Next-generation refers to the most current gene-sequencing technology that provides us with better knowledge of the genetic and molecular features of an individual patient's tumor," said co-investigator John M. Maris, M.D., who leads CHOP's internationally prominent research laboratory focused on neuroblastoma.

"Our improved understanding of neuroblastoma biology and treatment will guide this trial and help us to continually design combination therapies that are potentially more effective and less toxic for our young patients," Maris added. The NEPENTHE trial will be a collaborative effort, enlisting the expertise of numerous other specialists throughout CHOP and other institutions. It will enroll children who have experienced a relapse of neuroblastoma or whose neuroblastoma did not respond to the initial treatment.
About The Children's Hospital of Philadelphia: The Children's Hospital of Philadelphia was founded in 1855 as the nation's first pediatric hospital. Through its long-standing commitment to providing exceptional patient care, training new generations of pediatric healthcare professionals and pioneering major research initiatives, Children's Hospital has fostered many discoveries that have benefited children worldwide. Its pediatric research program is among the largest in the country. In addition, its unique family-centered care and public service programs have brought the 535-bed hospital recognition as a leading advocate for children and adolescents. For more information, visit

Children's Hospital of Philadelphia

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