Neuroblastoma - Childhood: Latest Research

Approved by the Cancer.Net Editorial Board, 06/2022

ON THIS PAGE: You will read about the scientific research being done to learn more about neuroblastoma and how to treat it. Use the menu to see other pages.

Doctors are working to learn more about neuroblastoma, ways to prevent it, how to best treat it, and how to provide the best care to people diagnosed with this disease. The following areas of research may include new options for patients through clinical trials. Always talk with your child’s doctor about the best diagnostic and treatment options for your child.

Many of the items below are explained earlier in this guide's Types of Treatment section. Additional studies are underway to improve the use and effectiveness of current treatments, as well as to find new approaches to treating neuroblastoma.

  • New drug combinations. Clinical trials are underway to study the use of chemotherapy combined with immunotherapy, tyrosine kinase inhibitors, or other drugs. Researchers hope that these drug combinations will increase the effectiveness and decrease the side effects of induction treatment.

  • Stem cell transplantation. A Children’s Oncology Group clinical trial comparing 2 cycles of high-dose chemotherapy to 1 cycle of high-dose chemotherapy and stem cell transplantation has recently been completed. Patients who received tandem cycles of high-dose therapy had improved event-free survival. Based on these results, the Children’s Oncology Group now considers 2 cycles of high-dose therapy with stem cell transplant the new standard of care for high-risk neuroblastoma patients. A European study compared the results for children who received conditioning prior to a stem cell transplant with carboplatin, etoposide, and melphan as compared to busulfan and melphalan. The study suggested that the outcome was better for children treated with busulfan and melphalan.

  • Targeted radiation therapy. Treatment with a high-energy form of radioactive iodine (I-131) with MIBG has been shown to have the tumor respond in about 30% of patients with recurrent or refractory neuroblastoma. This treatment is currently only available at certain cancer centers in the United States. Recently completed New Approaches to Neuroblastoma Therapy (NANT) clinical trials testing radioactive MIBG alone or combined with radiation sensitizers have shown higher response rates when MIBG is combined with vorinostat. Other NANT studies are testing MIBG combined with dinutuximab, and there are plans to amend this study to test vorinostat combined with MIBG and dinutuximab. The Children’s Oncology Group is conducting a randomized phase 3 study evaluating the efficacy of adding radioactive MIBG to the induction regimen in newly diagnosed patients with a high-risk neuroblastoma. Other radiopharmaceutical therapies that are being evaluated include 277 (277Lu)‐DOTATE and astatine‐211 (211At)‐mIBG.

  • Molecular targeted therapies. Research on the use of small molecules to target the cell functions that are abnormal in neuroblastoma cells is ongoing. Lorlatinib, and other drugs that inhibit ALK, a tyrosine kinase that is mutated in a subset of neuroblastomas, as well as other tyrosine kinase inhibitors are being tested in clinical trials. Other treatments currently in phase 1 or phase 2 studies include bromodomain and extra‐terminal motif (BET) bromodomain inhibitors (for their inhibition of MYCN). Aurora kinase inhibitors have also been combined with chemotherapy for the treatment of neuroblastoma.

  • Biomarkers. New tools are being developed to help find out the clinical aggressiveness of the tumor and response to treatment that may be used in the future to tailor therapies to individual patients. These include the use of circulating tumor DNA for genomic and epigenetic characterization of tumors and for determining how the tumor will respond to treatment. Learn more about tumor marker tests.

  • Imaging techniques. New imaging approaches, such as FDG‐PET‐MRI, 124I‐mIBG PET‐CT, or 68Ga‐DOTATATE, are being evaluated to see how they may help patients with neuroblastoma in the hope that these newer scanning methods may lead to more precise tumor localization.

  • Palliative care/supportive care. Clinical trials are underway to find better ways of reducing symptoms and side effects of current neuroblastoma treatments to improve comfort and quality of life for patients.

Looking for More About the Latest Research?

If you would like more information about the latest areas of research in neuroblastoma, explore these related items that take you outside of this guide:

The next section in this guide is Coping with Treatment. It offers some guidance on how to cope with the physical, emotional, social, and financial changes that cancer and its treatment can bring. Use the menu to choose a different section to read in this guide.