Pediatric Cancer Researchers Receive $1.3 Million to Study Precise Treatment for Neuroblastoma

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Today Children’s Hospital of Philadelphia (CHOP) and Gustave Roussy, a cancer-research institute based in France, were awarded a grant from a group of neuroblastoma clinical research charities led by Solving Kids’ Cancer UK and Solving Kids’ Cancer (US) that will support parallel phase 3 clinical trials developed by the Children’s Oncology Group (COG) and The International Society of Paediatric Oncology Europe Neuroblastoma Group (SIOPEN). This is the first-ever collaboration between the North American and European pediatric cancer consortia focused on neuroblastoma. The grant, distributed over a four-year period, will be split between CHOP and Gustave Roussy.

Additional charity partners providing the research funds include Band of Parents, Joining Against Cancer in Kids (J-A-C-K), Ronan Thompson Foundation, Wade’s Army, and Zoé4life.

Yael Mossé, MD, a physician-scientist who focuses on neuroblastoma in CHOP’s Cancer Center, will co-lead this collaboration with COG and Dominique Valteau-Couanet, MD, PhD, the division chief of Pediatric Oncology at Gustave Roussy.

“This exciting collaboration is an important step in developing better treatments for children with neuroblastoma,” said Dr. Mossé. “Only one new drug has been approved by the FDA for neuroblastoma in the past 30 years. We hope this collaboration between North American and European researchers will bring more targeted therapies to children newly diagnosed with this disease.”

“This collaboration will allow us to evaluate a targeted therapy upfront in combination with the standard treatment developed by COG and SIOPEN for children with high-risk neuroblastoma. It is a unique opportunity to demonstrate the impact of such targeted therapies in this disease,” said Dr. Valteau-Couanet. “The collaboration is the result of a confident relationship developed between our two groups during the last decade. This trial is the first step of a collaboration that will help to answer questions that could not be solved otherwise”.

Neuroblastoma is the most common pediatric cancer diagnosed in infancy, with approximately 90 percent of children with the disease diagnosed before the age of five. In North America and Europe, some 1,500 children are diagnosed with severely malignant, high-risk neuroblastoma each year, which requires intense and grueling treatment. Fewer than half of those with high-risk disease live more than five years after diagnosis, and those who do survive often suffer lifelong side effects, including hearing loss, learning disabilities, and secondary malignant cancers.

In order to develop a more effective and less toxic treatment for neuroblastoma, clinical trials developed within COG and SIOPEN will target mutations in the ALK oncogene, which are found in 14 percent or more of patients with newly diagnosed neuroblastoma and have recently been implicated in relapses of the disease. Through a partnership with Pfizer Pharmaceuticals and the New Approaches to Neuroblastoma Therapy (NANT), researchers at CHOP led by Mossé’s laboratory have discovered a highly specific and potent ALK inhibitor, lorlatinib, a drug currently used to treat lung cancer that would impede the pro-cancer activities of these ALK mutations and potentially have a significant impact in neuroblastoma patients.

In parallel Phase 3 clinical trials within COG in North America and SIOPEN in Europe, the researchers hope to use lorlatinib to substantially improve the survival for patients with newly diagnosed high-risk neuroblastoma whose tumors harbor an activated ALK gene mutation. The collaboration, known as TITAN: Transatlantic Integration Targeting ALK in Neuroblastoma, will involve simultaneous evaluation of the addition of lorlatinib to both COG’s and SIOPEN’s high-risk neuroblastoma treatment regimens, merging the data with the aim of accelerating lorlatinib approval if the drug is effective.

“This is a landmark step in clinical research for children with neuroblastoma,” said Andy Pearson, MD, Chair of the Solving Kids’ Cancer UK Scientific Advisory Board, which managed the grant review and award process. “With pediatric cancer hugely underfunded in comparison to adult cancer, there is an urgent need for breakthrough treatments for the most difficult-to-cure childhood cancers like neuroblastoma. This trial has the potential to accelerate the discovery of a new treatment and lay the foundation for future collaborations of this nature.”

About Children’s Hospital of Philadelphia: 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 595-bed hospital recognition as a leading advocate for children and adolescents. For more information, visit

About Gustave Roussy: Gustave Roussy, a leading cancer center in Europe, covers the full range of expertise in the world of oncology and is totally committed to looking after patients. Its 3,100 professional staff are dedicated to patient care, research and teaching. The Child and Adolescent Cancer Department of Gustave Roussy was the first department dedicated to pediatric oncology in France. Its pediatric team is dedicated to patient- and family-centered care and to clinical and translational research. The physicians are actively participating in national and international protocols and research programs that significantly improve pediatric cancer patients’ outcomes. Visit

Contact: Jennifer Lee, The Children’s Hospital of Philadelphia, 267-426-6084 or