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New Therapies For Neuroblastoma

Approximately 40% of children with neuroblastoma have "high-risk" tumors with poor 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. The use of immunotherapy for children with neuroblastoma has been an attractive option for treatment, as the immune system provides a way to target tumor cells directly while avoiding the typical side effects of chemotherapy such as hair loss and low blood counts. However, the use of immunotherapy for cancer treatment has been limited in the past by the ability of tumor cells to evade the immune system. Current clinical trials that treat neuroblastoma with antibodies or with immune cells are currently underway at many centers nationwide. Researchers at Texas Children's Hospital and the Center for Cell and Gene Therapy at Baylor College of Medicine have developed novel treatments that are currently being tested in clinical trials for children with neuroblastoma here in Houston. 

A new clinical trial ("Autologous Actviated T-cells Transduced with A 3rd Generation GD-2 Chimeric Antigen Receptor And iCaspase9 Safety Switch Administered to Patients With Relapsed or Refractory Neuroblastoma (GRAIN)") recently opened at Texas Children's and is now enrolling patients, providing a new and exciting treatment option for children with neuroblastoma. In the previous clinical trial at Texas Children's Hospital, 1st generation GD2 CAR T cells were given to 19 patients with high-risk neuroblastoma. Five of 11 patients with active disease showed tumor responses, and in 3 of these individuals, the responses were complete, and persistence of the GD2 T-cells was associated with prolonged survival (1,2). In order to promote the long-term persistence of these GD2 CAR T cells for this new clinical trial, additional components of the natural T-cell activation pathway have been included in these new GD2 CAR T cells, along with a "safety switch" that allows the signal to be rapidly turned off in the event of severe side effects. The successes of these and other immunotherapy clinical trials for children with neuroblastoma provide hope for the routine use of these treatments as a part of future standard neuroblastoma treatment. Additional new treatments are currently undergoing testing in the neuroblastoma laboratories at Texas Children's Hospital, and new clinical trials are currently being developed to make these exciting new treatment options available for the children who need them most in order to provide the most appropriate and most effective therapy for each and every child with neuroblastoma. For more information on the neuroblastoma research program, visit here.

References: 1. Louis CU, Savoldo B, Dotti G et al. Antitumor activity and long-term fate of chimeric antigen receptor-positive T cells in patients with neuroblastoma. Blood 2011; 118:  6050-6056. 2. Pule MA, Savoldo B, Myers GD et al. Virus-specific T cells engineered to coexpress tumor-specific receptors: persistence and antitumor activity in individuals with neuroblastoma. Nat Med 2008; 14:  1264-1270.

Dr. Peter Zage, Pediatric Oncologist