Neuroblastoma: Basic Treatment Options

January 26, 2012


Neuroblastoma is the most common solid tumor in childhood and accounts for approximately 8% of childhood cancers. Neuroblastoma tumors arise from tissues in the autonomic nervous system in the body, which controls body functions like movement of food within the intestines, sweating and blood pressure, among others. Neuroblastoma tumors can be found next to the spinal cord and in the adrenal glands above the kidneys, which are parts of the autonomic nervous system. It can also spread to lymph nodes, the liver or other organs, the bones and bone marrow, and, in the most severe cases, to the lungs and brain.

Neuroblastoma occurs most often in infants and young children, but can be found in older children and adolescents in rare instances. Cases of neuroblastoma are divided into low-, intermediate-, and high-risk, based on the age of the child, the spread of the tumor, and biologic features of the tumor itself. While children with low- and intermediate-risk neuroblastoma have cure rates of over 90%, approximately 40% of children with neuroblastoma have "high-risk" tumors with much lower survival rates, despite aggressive treatment with combinations of chemotherapy, stem cell transplantation, surgery and radiation therapy. Cases of "high-risk" neuroblastoma are also associated with frequent recurrences and tumors that are resistant to treatment. New therapies are needed for these children to improve the cure rates and reduce the occurrence and severity of the side effects of treatment.

Diagnosis of and treatment for children with neuroblastoma requires

the combined efforts of a variety of specialists, including pediatric oncologists, surgeons, radiologists, radiation oncologists, pathologists, pharmacists, nurses, child life specialists and social workers, among others, to ensure the best outcomes. Developing the best treatment plan for each patient is an ongoing priority for the Neuroblastoma Program at Texas Children's Cancer and Hematology Centers, and our doctors and scientists are continuously striving to translate laboratory discoveries into new treatments for children with neuroblastoma.

Our dedicated team of clinicians coordinates the treatment of children with new and recurrent neuroblastoma, and we are continuing to evaluate new therapies and new strategies for delivering therapies. We offer a wide range of clinical trials for all children with neuroblastoma, including trials for patients with newly diagnosed neuroblastoma through the Children's Oncology Group and through the Center for Cell and Gene Therapy at Baylor College of Medicine.

Children with low- and intermediate-risk neuroblastoma will have surgery to resect as much tumor as possible by one of our surgeons who specialize in pediatric cancer surgery. Additional treatment will be directed by one of the pediatric oncologists from the Pediatric Solid Tumor Team specializing in neuroblastoma, following the guidelines of the Children's Oncology Group and focused on the specific needs of the individual child.

Children with high-risk neuroblastoma will be treated following a protocol developed for use at the Texas Children's Cancer and Hematology Centers, incorporating:

  • chemotherapy using combinations of drugs found to be most effective against neuroblastoma,
  • surgery to remove the tumors by one of the specialized pediatric cancer surgeons at Texas Children's Hospital,
  • radiation therapy, including the option of proton beam radiation therapy, a more focused type of radiation,
  • autologous stem cell transplantation,
  • maintenance therapy with 13-cis-retinoic acid (Accutane), with or without added antibody therapy directed against neuroblastoma, and
  • the option of enrolling on a vaccine protocol only available at Texas Children's Hospital through the Center for Cell and Gene Therapy at Baylor College of Medicine.

New studies exploring the role of kidney function in the responses of children to chemotherapy and the importance of the expression of a small subset of genes (identified in the neuroblastoma laboratories at Texas Children's Cancer and Hematology Centers) in the outcomes of children with neuroblastoma are currently being developed.

Children with relapsed or refractory neuroblastoma represent a unique challenge for treatment. We offer a wide range of phase I and II clinical trials using exciting new therapies for these children. We offer clinical trials through the Children's Oncology Group Phase I consortium and the New Approaches to Neuroblastoma Therapy (NANT) consortium. We also offer unique cell therapy trials for children with relapsed neuroblastoma through the Center for Cell and Gene Therapy at Baylor College of Medicine to attempt to harness the natural immune system to fight the neuroblastoma tumor cells resistant to chemotherapy. Additional new treatments are currently undergoing testing in the neuroblastoma laboratories of the Texas Children's Cancer and Hematology Centers, and new clinical trials are currently being developed to make these exciting new treatment options available for the children who need them most.

Future research by the scientists in the neuroblastoma program at Texas Children's Cancer and Hematology Centers will continue to explore new pathways, new targets, and new treatments in order to provide the most appropriate and most effective therapy for each and every child with neuroblastoma.

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