Neuroblastoma - Childhood: Latest Research

Approved by the Cancer.Net Editorial Board, 03/2015

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. To see other pages, use the menu on the side of your screen.

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 diagnostic and treatment options best for your child.

Many of the items below are explained earlier in the Treatment Options section. Studies are underway to improve their use and effectiveness.

  • New drug combinations. Clinical trials are underway to study the use of a drug called topotecan in combination with cyclophosphamide. Researchers hope that this drug combination will increase the effectiveness and decrease the side effects of initial treatment.

  • Stem cell transplantation.  A clinical trial comparing two cycles of high-dose chemotherapy to one cycle of high-dose chemotherapy and stem cell transplantation has recently been completed. The results of this trial should be available within the next two years. Previous Children’s Oncology Group studies used carboplatin, etoposide, and melphan as the conditioning regimen before a stem cell transplant.

    The Children’s Oncology Group is now testing a new consolidation regimen consisting of busulfan and melphalan before a stem cell transplant. A European study compared outcome for children who received conditioning prior to a stem cell transplant with carboplatin, etoposide, melphan vs. busulfan and melphalan. The study suggested that the outcome was better for children treated with busulfan and melphalan.

  • New radiation therapy techniques. A radioactive MIBG delivers radiation therapy to neuroblastoma cells for recurrent or refractory disease, with or without stem cell transplantation. By using a high-energy form of radioactive iodine with the MIBG, enough radiation therapy is given off to kill the neuroblastoma cells. This treatment is currently available at about a dozen centers in the United States. Clinical trials involving radioactive MIBG when there is a recurrence and for newly diagnosed children with high-risk neuroblastoma are ongoing.

  • Other treatment options. Research on the use of small molecules to target the cell functions that are abnormal in neuroblastoma cells are ongoing. Crizotinib and other drugs that inhibit ALK, a tyrosine kinase that is mutated in a small subset some types of neuroblastomas, as well as other tyrosine kinase inhibitors are being tested in early phase clinical trials. Studies testing crizotinib in newly diagnosed patients with neuroblastoma tumors with ALK mutations are planned in the Children’s Oncology Group.

  • Palliative care. Clinical trials are underway to find better ways of reducing symptoms and side effects of current neuroblastoma treatments in order 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 Side Effects and it offers some guidance in how to cope with the physical, emotional, and social changes that cancer and its treatment can bring. Or, use the menu on the side of your screen to choose another section to continue reading this guide.