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 figuring 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 have found that certain DNA changes on the short arm of chromosome 6 (6p22) are more likely to be seen in neuroblastomas that grow more aggressively.

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

Treatment

Survival rates from childhood cancers have gotten better as doctors have found ways to improve on current treatments. Researchers continue to look for better ways to treat neuroblastoma.

Chemotherapy

Doctors continue to search for the best combinations of chemotherapy drugs to treat neuroblastoma.

Several chemotherapy drugs that are already used to treat other cancers, such as topotecan, irinotecan, and temozolomide, are now being studied for use against neuroblastoma. Some newer drugs that work in different ways from standard chemotherapy drugs are being studied against neuroblastoma as well. Examples include bortezomib, nifurtimox, and lestaurtinib.

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 (known as a tandem transplant).

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 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, doctors are now studying medicines that specifically target the machinery of neuroblastoma cells, such as inhibitors of the ALK pathway or aurora A pathway.

Some monoclonal antibodies that target substances on neuroblastoma cells are used routinely for children with high-risk neuroblastoma, to help immune system cells find and destroy the cancer cells. Clinical trials are now in under way to test the effectiveness of attaching radioactive particles or chemotherapy drugs these antibodies. By zeroing in on their targets, these antibodies may be able to deliver chemotherapy or radiation to the neuroblastoma cells with minimal damage to normal cells.

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: 11/23/2009
Last Revised: 11/23/2009