Important research into neuroblastoma is under way right now in many university hospitals, medical centers, and other institutions around the world. Each year, scientists find out more about what causes the disease and how to improve treatment.

In recent years, survival rates from childhood cancers have improved. Most of these results have come when doctors find ways to improve on the use of already available and accepted therapies. For example, studies are helping doctors learn which combinations of drugs are more likely to improve survival.

Clinical trials are under way to study the use of a drug called topotecan, alone and in combination with cyclophosphamide. Researchers hope that this agent will allow less intense doses of chemotherapy drugs to be used for treatment of neuroblastoma.

Differentiating agents such as retinoids have begun to show some promise in reducing the risk of recurrence after treatment. New, more effective retinoids are now being developed and studied.

Another new treatment under evaluation involves the use of monoclonal antibodies. These are special molecules that can be injected into the body to seek out and attach to cancerous cells. An example of the monoclonal antibodies now in clinical trials are those that have been developed to attach to the ganglioside GD2, which is present on the surface of many neuroblastoma cells.

Sometimes, these antibodies can stimulate the child’s immune system to fight the cancer more effectively. Scientists are also learning how to attach these antibodies to radioactive particles or chemotherapy drugs. By zeroing in on their targets, these antibodies than can deliver chemotherapy or radiation to the neuroblastoma cells with minimal damage to normal cells. Early results of studies show that perhaps 40% of children with neuroblastoma that has returned after initial treatment show some response to some monoclonal antibodies.

Revised: 08/04/2006