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.

Classifying neuroblastomas

Researchers now have better tools to look for changes in the genes of neuroblastoma cells. They have made a great deal of progress in recent years in to help doctors find out which neuroblastomas are likely to be cured with standard treatment, and which will need more aggressive treatment.

For example, using newer lab tests, researchers recently found that certain DNA changes on the short arm of chromosome 6 (6p22) were more likely to be seen in neuroblastomas that grew more aggressively.

In the near future, doctors may be able to use these types of test results to aid in choosing treatments.

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.

Chemotherapy: Doctors continue to search for the best combinations of chemotherapy drugs to treat neuroblastoma. Several drugs that are already used to treat other cancers, such as topotecan, irinotecan, and temozolomide, are now being studied for use against neuroblastoma as well.

Stem cell transplants: Doctors are also trying to improve the success rate with high-dose chemotherapy and stem cell transplants, using different combinations of chemotherapy, radiation therapy, retinoids, and other treatments. Some clinical trials are studying the use of more than one stem cell transplant in the same patient.

Retinoids: Retinoids such as 13-cis-retinoic acid (isotretinoin) have shown promise in reducing the risk of recurrence after treatment. Newer, potentially more effective retinoids, such as fenretinide, are now being developed and studied in clinical trials.

Newer forms of treatment: Knowledge about what makes neuroblastoma cells different from normal cells may lead to new approaches to treating this disease. Several newer forms of therapy, which target neuroblastoma cells more specifically than standard treatments, are now being studied in clinical trials.

For example, monoclonal antibodies are manmade versions of immune system proteins that can be made to attack a very specific target. These molecules can be injected into the body to seek out and attach to cancer cells. Monoclonal antibodies have been developed to attach to the ganglioside GD2, a substance found on the surface of many neuroblastoma cells.

Some of these antibodies are used by themselves to try to stimulate the body's immune system to attack the cancer, while others are first attached 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 these treatments have shown promise, but they are available only in clinical trials at this time.

Forms of gene therapy and cancer vaccines are also being studied for use against neuroblastoma, although these treatments are still in the earliest stages of clinical trials.

Last Medical Review: 10/22/2008
Last Revised: 10/22/2008