Neuroblastoma Research & Clinical Trials

Leaders in Neuroblastoma Research

Our physicians are leading clinical trials testing new treatments to improve outcomes in children with newly diagnosed high-risk and non-high-risk neuroblastoma. We are also leading clinical trials for children with relapsed neuroblastoma or tumors that have not responded to conventional treatments and clinical studies investigating late effects in high-risk neuroblastoma survivors.

In addition, physicians and scientists are collaborating to:

• Develop new biomarkers of treatment response and survival that have the potential to transform treatment approaches
• Identify new therapeutic targets and investigate the biological underpinnings that drive aggressive neuroblastoma growth

Ongoing Neuroblastoma Research Studies

• Determining the role of immune cells in high-risk neuroblastoma tumors as predictors of a favorable prognosis
• Developing novel, liquid biopsy epigenetic biomarker that correlates with disease burden and response to treatment in children with neuroblastoma
  
• Refining the neuroblastoma risk classification system to improve treatment stratification and patient outcome; read more here and here
  
• Identifying germline genetic factors associated with increased risk of developing high-risk neuroblastoma with MYCN amplification
  
• Evaluating the late effects and second-cancers in neuroblastoma survivors

In addition, Susan Cohn, MD, serves as co-chair of the International Neuroblastoma Risk Group (INRG) task force, a group of pediatric oncologists dedicated to transforming neuroblastoma research and clinical care. In collaboration with Samuel Volchenboum, MD, PhD, MS, the INRG task force developed the INRG Data Commons, an ecosystem that currently includes clinical data on more than 22,000 patients from around the world that can be linked to genomic data in GEO, TARGET, and other external data bases. The data are available to the research community, and the INRG Data Commons has facilitated international, interdisciplinary research in childhood neuroblastoma never before possible. In 2022, the INRG data will be available on the Pediatric Cancer Data Commons (PCDC), a platform that harnesses clinical data from patients with other types of pediatric cancers from around the globe, enabling new, cross-disease research studies.