Question: What are the treatments for Neuroblastoma ?
Answer: Key Points - There are different types of treatment for patients with neuroblastoma. - Children with neuroblastoma should have their treatment planned by a team of doctors who are experts in treating childhood cancer, especially neuroblastoma. - Children who are treated for neuroblastoma may have late effects, including an increased risk of second cancers. - Seven types of standard treatment are used: - Observation - Surgery - Radiation therapy - Iodine 131-mIBG therapy - Chemotherapy - High-dose chemotherapy and radiation therapy with stem cell rescue - Targeted therapy - New types of treatment are being tested in clinical trials. - Immunotherapy - Other drug therapy - Patients may want to think about taking part in a clinical trial. - Patients can enter clinical trials before, during, or after starting their cancer treatment. - Follow-up tests may be needed. There are different types of treatment for patients with neuroblastoma. Different types of treatment are available for patients with neuroblastoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment. Children with neuroblastoma should have their treatment planned by a team of doctors who are experts in treating childhood cancer, especially neuroblastoma. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating children with neuroblastoma and who specialize in certain areas of medicine. These may include the following specialists: - Pediatric surgeon. - Pediatric radiation oncologist.. - Endocrinologist. - Neurologist. - Pediatric neuropathologist.. - Neuroradiologist. - Pediatrician. - Pediatric nurse specialist. - Social worker. - Child life professional. - Psychologist. Children who are treated for neuroblastoma may have late effects, including an increased risk of second cancers. Some cancer treatments cause side effects that continue or appear years after cancer treatment has ended. These are called late effects. Late effects of cancer treatment may include: - Physical problems. - Changes in mood, feelings, thinking, learning, or memory. - Second cancers (new types of cancer). Some late effects may be treated or controlled. It is important that parents of children who are treated for neuroblastoma talk with their doctors about the possible late effects caused by some treatments. See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information. Seven types of standard treatment are used: Observation Observation is closely monitoring a patient's condition without giving any treatment until signs or symptoms appear or change. Surgery Surgery is used to treat neuroblastoma unless it has spread to other parts of the body. Depending on where the tumor is, as much of the tumor as is safely possible will be removed. If the tumor cannot be removed, a biopsy may be done instead. Radiation therapy Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: - External radiation therapy uses a machine outside the body to send radiation toward the cancer. - Internal radiation therapy uses a radioactive substance sealed in needles, seeds, wires, or catheters that are placed directly into or near the cancer. The way the radiation therapy is given depends on the type of cancer being treated and the child's risk group. External radiation therapy is used to treat neuroblastoma. Iodine 131-mIBG therapy Iodine 131-mIBG therapy is a treatment with radioactive iodine. The radioactive iodine is given through an intravenous (IV) line and enters the bloodstream which carries radiation directly to tumor cells. Radioactive iodine collects in neuroblastoma cells and kills them with the radiation that is given off. Iodine 131-mIBG therapy is sometimes used to treat high-risk neuroblastoma that comes back after initial treatment. Chemotherapy Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on the type of cancer being treated and the child's risk group. The use of two or more anticancer drugs is called combination chemotherapy. See Drugs Approved for Neuroblastoma for more information. High-dose chemotherapy and radiation therapy with stem cell rescue High-dose chemotherapy and radiation therapy with stem cell rescue is a way of giving high doses of chemotherapy and radiation therapy and replacing blood -forming cells destroyed by cancer treatment for high-risk neuroblastoma. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient and are frozen and stored. After chemotherapy and radiation therapy are completed, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. Maintenance therapy is given after high-dose chemotherapy and radiation therapy with stem cell rescue to kill any cancer cells that may regrow and cause the disease to come back. Maintenance therapy is given for 6 months and includes the following treatments: - Isotretinoin: A vitamin -like drug that slows the cancer's ability to make more cancer cells and changes how these cells look and act. This drug is taken by mouth. - Dinutuximab: A type of monoclonal antibody therapy that uses an antibody made in the laboratory from a single type of immune system cell. Dinutuximab identifies and attaches to a substance, called GD2, on the surface of neuroblastoma cells. Once dinutuximab attaches to the GD2, a signal is sent to the immune system that a foreign substance has been found and needs to be killed. Then the body's immune system kills the neuroblastoma cell. Dinutuximab is given by infusion. It is a type of targeted therapy. - Granulocyte-macrophage colony-stimulating factor (GM-CSF): A cytokine that helps make more immune system cells, especially granulocytes and macrophages (white blood cells), which can attack and kill cancer cells. - Interleukin-2 (IL-2): A type of immunotherapy that boosts the growth and activity of many immune cells, especially lymphocytes (a type of white blood cell). Lymphocytes can attack and kill cancer cells. See Drugs Approved for Neuroblastoma for more information. Targeted therapy Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack cancer cells with less harm to normal cells. There are different types of targeted therapy: - Tyrosine kinase inhibitors are small-molecule drugs that go through the cell membrane and work inside cancer cells to block signals that cancer cells need to grow and divide. Crizotinib is used to treat neuroblastoma that has come back after treatment. - Antibody-drug conjugates are made up of a monoclonal antibody attached to a drug. The monoclonal antibody binds to specific proteins or receptors found on certain cells, including cancer cells. The drug enters these cells and kills them without harming other cells. Lorvotuzumab mertansine is an antibody-drug conjugate being studied to treat neuroblastoma that has come back after treatment. New types of treatment are being tested in clinical trials. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Immunotherapy Immunotherapy is a treatment that uses the patients immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the bodys natural defenses against cancer. This type of cancer treatment is also called biotherapy or biological therapy. Vaccine therapy uses a substance to stimulate the immune system to destroy a tumor. Vaccine therapy is being studied to treat neuroblastoma that has come back after treatment. Other drug therapy Lenalidomide is a type of angiogenesis inhibitor. It prevents the growth of new blood vessels that are needed by a tumor to grow. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI's listing of clinical trials. Follow-up tests may be needed. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. Treatment Options for Neuroblastoma Low-Risk Neuroblastoma Treatment of low-risk neuroblastoma may include the following: - Surgery followed by observation. - Chemotherapy with or without surgery, for children with symptoms, children whose tumor has continued to grow and cannot be removed by surgery, or children with unfavorable tumor biology. - Observation alone for infants younger than 6 months who have small adrenal tumors. - Observation alone for infants who do not have signs or symptoms of neuroblastoma. - Radiation therapy to treat tumors that are causing serious problems and do not respond quickly to chemotherapy or surgery. - A clinical trial of treatment based on the tumor's response to treatment and tumor biology. Intermediate-Risk Neuroblastoma Treatment of intermediate-risk neuroblastoma may include the following: - Chemotherapy for children with symptoms or to shrink a tumor that cannot be removed by surgery. Surgery may be done after chemotherapy. - Surgery alone for infants. - Observation alone for certain infants. - Radiation therapy to treat tumors that are causing serious problems and do not respond quickly to chemotherapy or surgery. - Radiation therapy for tumors that do not respond to other treatment. - A clinical trial of treatment based on the tumor's response to treatment and tumor biology. High-Risk Neuroblastoma Treatment of high-risk neuroblastoma may include the following: - A regimen of combination chemotherapy, surgery, stem cell rescue, radiation therapy, and monoclonal antibody therapy (dinutuximab) with interleukin-2 (IL-2), granulocyte-macrophage colony-stimulating factor (GM-CSF), and isotretinoin. Stage 4S Neuroblastoma There is no standard treatment for stage 4S neuroblastoma but treatment options include the following: - Observation with supportive care for certain children who have favorable tumor biology and do not have signs or symptoms. - Chemotherapy, for children who have signs or symptoms of neuroblastoma or unfavorable tumor biology, or for very young infants. - A clinical trial of treatment based on the tumor's response to treatment and tumor biology. Recurrent Neuroblastoma Patients First Treated for Low-Risk Neuroblastoma Treatment for recurrent neuroblastoma that is found only in the area where the cancer first formed may include the following: - Surgery followed by observation or chemotherapy. - Chemotherapy that may be followed by surgery. Treatment for recurrent neuroblastoma that has spread to other parts of the body may include the following: - Observation. - Chemotherapy. - Surgery followed by chemotherapy. - Treatment as for newly diagnosed high-risk neuroblastoma for children older than 1 year. Patients First Treated for Intermediate-Risk Neuroblastoma Treatment for recurrent neuroblastoma that is found only in the area where the cancer first formed may include the following: - Surgery that may be followed by chemotherapy. - Treatment as for newly diagnosed high-risk neuroblastoma for neuroblastoma that has spread to other parts of the body. Recurrent neuroblastoma that has spread to other parts of the body is treated the same way as newly diagnosed high-risk neuroblastoma. Patients First Treated for High-Risk Neuroblastoma Treatment for recurrent neuroblastoma may include the following: - Combination chemotherapy. - Iodine 131-mIBG therapy to relieve symptoms and improve quality of life. It may be given alone or in combination with other therapy, or before stem cell rescue. - A second course of high-dose chemotherapy and stem cell rescue. - Tyrosine kinase inhibitor therapy (crizotinib) for patients with changes in the ALK gene. Because there is no standard treatment for recurrent neuroblastoma in patients first treated for high-risk neuroblastoma, patients may want to consider a clinical trial. For information about clinical trials, please see the NCI website. Patients with Recurrent CNS Neuroblastoma Treatment for neuroblastoma that recurs (comes back) in the central nervous system (CNS; brain and spinal cord) may include the following: - Surgery to remove the tumor in the CNS followed by radiation therapy. - A clinical trial of a new therapy. Treatments Being Studied for Progressive/Recurrent Neuroblastoma Some of the treatments being studied in clinical trials for neuroblastoma that recurs (comes back) or progresses (grows, spreads, or does not respond to treatment) include the following: - Combination chemotherapy and monoclonal antibody therapy (dinutuximab). - Lenalidomide and monoclonal antibody therapy (dinutuximab) with or without isotretinoin. - Iodine 131-mIBG given alone or with other anticancer drugs. - Immunotherapy (vaccine therapy). - Tyrosine kinase inhibitor (crizotinib) and combination chemotherapy. - Targeted therapy with an antibody-drug conjugate (lorvotuzumab mertansine).