Neuroblastoma is a relatively common cancer affecting infants and young babies. Research into this disease is important so that accurate diagnostic and screening methods can be developed to diagnose the condition early. Research also focuses on treating the condition as early as possible. Neuroblastoma research is ongoing in several institutions, medical centers and hospitals.

Some of the areas of research where progress is being made are outlined below.

Several cytogenetic patterns identified in babies with neuroblastoma have been shown to predict a worse outcome, even after treatment. Research involves testing for certain DNA changes, especially on the short arm of chromosome 6 (6p22), as these changes increase the risk of neuroblastomas growing more rapidly.

Another study has shown that neuroblastomas in older children are associated with changes in the ATRX tumor suppressor gene. Normally this gene would suppress the tumor but when altered or mutated, the tumor grows unchecked. Children with this gene tend to have a slow growing but hard to cure neuroblastoma.

Retinoids are effective in reducing the recurrence of neuroblastoma after treatment and research is being carried out into what are hoped to be improved versions, such as fenretinide.Research also focuses on the development of the most effective combination of chemotherapy agents for treating neuroblastoma. Some agents already used to treat other cancers are now being tested in the treatment of neuroblastoma and include irinotecan, topotecan and temozolomide. Also, some more recent agents with different mechanisms to standard chemotherapy agents are being studied and include vorinostat, nifurtimox, bortezomib and lestaurtinib. Research also focuses on minimizing the use of less effective chemotherapy agents.

Researchers are looking at how neuroblastoma cells differ from normal cells with a view to developing drugs that target these abnormal cells more specifically than current treatment. Crizotinib (Xalkori), for example, is a drug that targets cells with changes in the ALK gene. Alterations in this gene are seen in around 15% of children with neuroblastoma. Researchers are testing whether the addition of an mTOR inhibitor can enhance the effects of crizotinib.

Research is also being carried out into the use of several cancer vaccines. This involves the injection of modified neuroblastoma cells into the body to try and trigger an immune response that targets and attacks the cancer cells.