Neuroblastoma is a cancer that often begins in childhood. It impacts the nerve tissue, especially in the adrenal glands.
Research + Clinical Trials
This study is being done to test the effect of a drug, nifurtimox, against neuroblastoma and medulloblastoma in children. Nifurtimox is a drug that has been used in South America for many years to treat a parasitic disease known as Chagas Disease. It is not approved by the Food and Drug Administration for routine use in neuroblastoma or medulloblastoma in the United States, but the FDA is allowing it to be used in research studies like this one. Limited early observations, suggest that nifurtimox may have anti-tumor activity for neuroblastoma and medulloblastoma. We do not know whether nifurtimox will shrink/kill tumor cells effectively in children. Therefore, the major goal of the study is to learn if nifurtimox in combination with other common chemotherapy drugs is effective in shrinking/killing neuroblastoma and medulloblastoma cells. We will also be collecting information about any side effects that the drug may have.
In this study we are using new biologic features, in addition to the standard risk factors, to help decide which subjects need treatment and what the best treatment is. People with non-high-risk NBL generally respond well to treatment and the majority of patients can be treated successfully with standard therapy. But standard therapy can include risks from surgery and/or side effects from chemotherapy.Based on the evaluation of non-high-risk NBL patients treated in the past, we think that we can change the amount of therapy for subjects and still treat the cancer successfully. Small studies have shown that some subjects can be watched without having surgery or getting chemotherapy and still have an excellent outcome. By not having surgery or lowering the amount of chemotherapy we hope to prevent complications and harmful side effects. For other subjects we think a change in therapy will treat the cancer more successfully.
The purpose of this research study is to evaluate an investigational drug (DFMO) for Neuroblastoma that is in remission. Remission means that there are no current signs of active cancer. An investigational drug is one that has not been approved by the U.S. Food and Drug Administration (FDA). This study will look at the ability of this study drug to keep neuroblastoma in remission and will also look at the safety and tolerability of DFMO.