Targeted Therapies in Neuroblastoma: A History and a View to the Future

Neuroblastomas remain a disproportionately morbid and mortal pediatric cancer. Representing 8% of all childhood cancer diagnoses, it still is the cause of 14% of cancer-related deaths in children despite the use of aggressive multimodal treatments with significant long-term sequelae. The development of targeted therapies for neuroblastoma has been and will continue to be the optimal approach to improve durable long-term survival in these patients while reducing the consequences of treatment. Here, I review the history of the development of targeted therapies for the treatment of neuroblastoma. I discuss the history of therapies that have been integrated into current treatment regimens, including isotretinoin, anti-GD2 antibodies and eflornithine. I review the history of candidate biologic targets in this malignancy as well as ongoing and future efforts to improve outcomes.