Contemporary approach to diagnosis and treatment of neuroblastoma

The diagnostic value of Metaiodobenzylguanidine (MIBG) scintigraphy in the management of neuroblastoma is well established. The specificity of MIBG is virtually 100% and remains the most specific imaging modality. Numerous semi-quantitative scores and guidelines have emerged in the last decade that illustrate standardization of the procedure. Other pharmaceuticals such as norcholesterol derivatives, [111In]pentetreotide and [68Ga]somatostatin analogs, [18F]fluorodeoxyglucose, [18F]fluorodopa, [18F]fluorodopamine, [11C]meta hydroxyephedrine, and [11C] /[18F] /[123I]Metomidate (MTO) have been or are being evaluated currently (including development of new analogues labeled with positron emitting radionuclides such as [124I], [18F], and [76Br]. Radiopharmaceutical therapy of neuroblastoma, initiated over 30 years ago, demonstrates that a significant fraction of patients enter partial remission but complete remission is rare and relapse is frequent. With the combination of chemotherapy, radiosensitizers, and autologous stem cell support, some centers have seen overall response rates of up to 30% in refractory or recurrent diseases. Topoisomerase I inhibitor topotecan may improve upon existing [131I]MIBG therapy. Areas of future development may be in vitro cultures and animal models, proper instrumentation to acquire sub-centimeter resolution and human clinical trials to evaluate treatment at earlier times or stages of disease, evaluation with concomitant immunotherapy with monoclonal antibodies targeting the GD2 ganglioside or inhibitors of anaplastic lymphoma kinase. Because of the complexity of those trials, progress remains extremely slow as well designed multicenter studies are required. Nonetheless, the future has never been so hopeful.