Neuroblastoma in the Era of Precision Medicine: A Clinical Review

SIMPLE SUMMARY: Patients with high-risk neuroblastoma, especially those whose disease either does not respond or recurs, have a poor chance of survival. Additionally, the majority of survivors of modern day high-risk neuroblastoma therapy develop significant late-effects. In an effort to improve survival, new agents with less toxicity are being explored. As technologies advance, the ability to design therapies for specific molecular targets on neuroblastoma are crucial. The most notable existing targeted therapies for high-risk neuroblastoma thus far are ALK inhibitors and anti-GD2 therapies, both of which have become mainstays of treatment both in the upfront and relapsed settings. Development of additional molecular targeted therapy has proven challenging, with limited successes. Adding to this difficulty is the heterogeneity of high-risk neuroblastoma, especially in the relapsed setting. This heterogeneity makes the numbers of patients eligible for specific targeted therapy small, complicating optimal clinical trial design. Here, we review the known molecular targets of neuroblastoma and their clinical implications. ABSTRACT: The latest advances in treatment for patients with neuroblastoma are constantly being incorporated into clinical trials and clinical practice standards, resulting in incremental improvements in the survival of patients over time. Survivors of high-risk neuroblastoma (HRNBL), however, continue to develop treatment-related late effects. Additionally, for the majority of the nearly 50% of patients with HRNBL who experience relapse, no curative therapy currently exists. As technologies in diagnostic and molecular profiling techniques rapidly advance, so does the discovery of potential treatment targets. Here, we discuss the current clinical landscape of therapies for neuroblastoma in the era of precision medicine.