Neuroblastoma Program

The Texas Children's Cancer Center Neuroblastoma Program is dedicated to the integration of laboratory and clinical research to increase our understanding of neuroblastoma and to develop new, more effective therapies for the disease. The Program is comprehensive, researching new means of diagnosing, monitoring, predicting behavior of, as well as treating and preventing disease. It is multidisciplinary, utilizing experts from pediatric oncology, surgery, pharmacology, marrow and stem cell transplant, molecular oncology, gene therapy and cell therapy. The principle areas of research focus on understanding the genetic determinants of tumor biology, exploiting the relationship of neuroblastoma with the immune system to develop immunotherapy, and in new drug development.

Neuroblastoma is the most common solid tumor that occurs in children outside of the brain. Approximately 500 children are diagnosed with this type of cancer each year in the United States. Depending on the age of the child, the extent of disease at diagnosis, and the inherent characteristics of the tumor cells, which we call the tumor biology, neuroblastoma may require minimal therapy or may behave very aggressively, ultimately resisting treatment efforts.

The disease is unique in many ways: It has the highest rate of spontaneous regression of any human cancer. Less than complete resection of localized disease may be curative. Tumors may be highly infiltrated with cells from the immune system. Disease that on the surface appears to be identical in infants and in older children may be easily cured in the infants, with minimal therapy, yet is nearly always fatal in the older children. We believe that the clues for understanding neuroblastoma biology lie in the genes of the tumor cells.

The Neuroblastom Program conducts numerous clinical trials for children with newly diagnosed neuroblastoma or ganglioneuroblastoma, and for children with recurrent or resistant disease. The program is unique for its concentration of nationally and internationally recognized experts in the fields of pediatric oncology, neuroblastoma, gene and cell therapy, new drug development, marrow transplant, and in the design and conduct of clinical trials. The country's leading gene therapy and new drug development programs participate in the Program. For children with recurrent or resistant neuroblastoma, clinical trials of tumor vaccine and of a variety of new agents are currently being conducted. For children with newly diagnosed high-risk disease, trials incorporating a promising new drug called irinotecan, innovative scheduling of induction chemotherapy, modification of the patient's response to stem cell therapy, of prevention of recurrence of progression through modification of tumor biology are open.

Page modified on August 1, 2021