Neuroblastoma - Childhood: Latest Research

Approved by the Cancer.Net Editorial Board, 04/2018

ON THIS PAGE: You will read about the scientific research being done now to learn more about this type of tumor 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 the Types of Treatment section. Studies are underway to improve their use and effectiveness.

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

  • Bone marrow/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 with MIBG has been shown to have the tumor respond in about 30% of patients with recurrent or refractory neuroblastoma. This treatment is currently available at about a dozen cancer centers in the United States. Clinical trials involving radioactive MIBG alone or combined with radiation-sensitizers are ongoing through the NANT Consortium. The Children’s Oncology Group has conducted a feasibility pilot study evaluating radioactive MIBG combined with busulfan and melphalan during consolidation therapy for newly diagnosed patients with a high-risk tumor. These results are currently being analyzed. A new Children’s Oncology Group Phase III study will test the activity and side effects of MIBG therapy in induction in newly diagnosed patients with a high-risk tumor. Other radiopharmaceutical therapies that are being evaluated include 277 (277Lu)‐DOTATE and astatine‐211 (211At)‐mIBG.

  • Molecularly targeted therapies. Research on the use of small molecules to target the cell functions that are abnormal in neuroblastoma cells are ongoing. Crizotinib, 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 early-phase clinical trials. A new Children’s Oncology Group clinical trial will test the activity and side effects of crizotinib in newly diagnosed patients with neuroblastoma with ALK mutations. Other treatments currently in phase I or phase II studies include bromodomain and extra‐terminal motif (BET) bromodomain inhibitors (for their inhibition of MYCN), although this clinical trial is not yet open for children. 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 characterization of tumors, Studies are in progress to analyze circulating tumor DNA using comprehensive gene panels.

  • New imaging techniques: New imaging approaches, such as FDG‐ PET‐MRI, 124I‐mIBG PET‐CT or 68Ga‐DOTATATE are being evaluated in patients with neuroblastoma. These newer modalities may lead to more more‐precise tumor localization.

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

Looking for More About the Latest Research?

If you would like additional information about the latest areas of research regarding 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 in how to cope with the physical, emotional, and social changes that cancer and its treatment can bring. Use the menu to choose a different section to read in this guide.