Future of Clinical Trials and Application of Precision Medicine

Neuroblastoma is one of the most complex and challenging malignancies for the pediatric oncologist. It is characterized by an extreme heterogeneity both at the clinical and molecular level. To date, neuroblastoma serves as a paradigm for the prognostic benefit of combining molecular data with clinically defined risk factors as well as for the potential to tailor therapy for patient subgroups according to their individual risk for recurrence. However, despite tremendous progress in understanding the pathogenesis and molecular mechanisms of neuroblastoma, prognosis of patients with advanced disease remains dismal, with long-term event-free survival (EFS) < 50%. This article summarizes key aspects of future clinical trial design and personalized medicine approaches, and points out their impact on opening new avenues for risk stratification and targeted therapies.