Watchful Waiting

In some children with low-risk neuroblastoma, the disease may disappear on its own without any treatment at all. For these children, the doctor may suggest "watchful waiting" or "active surveillance," more so in patients with Stage 4S. The doctor will closely monitor the patient and watch for any signs of cancer progression. If the doctor detects any cancer progression or the disease does not go away on its own, active treatment will be started.

Surgery

Doctors commonly perform surgery to remove neuroblastoma tumors. The doctor will remove as much of the tumor as possible. The doctor may be able to remove the entire tumor, or may be able to remove only a part of it if it has attached itself to vital organs. If the doctor can't remove the entire tumor, he or she will most likely recommend adjuvant chemotherapy after surgery. The doctor may then attempt to surgically remove the rest of the tumor. If the tumor is very large, the doctor may also give neoadjuvant chemotherapy before surgery to shrink the tumor.

Chemotherapy

Chemotherapy is the use of drugs to target rapidly growing cells in an effort to destroy cancer cells. Chemotherapy medications may be given through the vein (intravenously) or by mouth (orally). Doctors may give neoadjuvant chemotherapy to reduce the tumor prior to surgery, or adjuvant chemotherapy to destroy any remaining cancer cells after surgery. Chemotherapy may also be the primary treatment for more widespread cases of neuroblastoma that have spread throughout the body.

Radiation Therapy

Doctors most commonly use external beam radiation therapy or MIBG radiotherapy to treat neuroblastoma. In external beam radiation therapy, waves of high-energy rays are used to target and kill cancer cells. In MIBG radiotherapy, the doctor injects a more radioactive form of MIBG than is used in an MIBG scan. The MIBG attaches itself to the neuroblastoma cells and delivers radiation directly to the tumor. This modality is used when neuroblastoma progresses or comes back after initial therapy.

High-dose Chemotherapy & Stem-cell Transplantation

The doctor may recommend this procedure for children with high-risk neuroblastoma. This procedure allows patients to receive extra-large doses of chemotherapy. These high doses destroy normal cells in the blood and bone marrow, as well as cancer cells. After chemotherapy, the patient receives his own blood-forming stem cells that help the body form new, healthy blood cells.

Retinoid Therapy

Retinoids are substances that are related to vitamin A. Retinoids may help neuroblastoma cells mature into normal, benign (non-cancerous) cells in some patients. The doctor may recommend retinoid therapy after a stem-cell transplantation to reduce the risk of tumor recurrence.

Immunotherapy/Monoclonal Antibody Therapy

The doctor may recommend a combination of immunotherapy and targeted therapy to treat neuroblastoma. Certain proteins found on the surface of neuroblastoma cells can cause the cancer to grow more aggressively. Monoclonal antibodies target these proteins on the surface of neuroblastoma cells, stopping or slowing the growth of cancer. Doctors commonly give cytokines (immune system hormones) with monoclonal antibodies to stimulate the immune system and help it destroy neuroblastoma cells.

CAR-T Immunotherapy

One approach to immunotherapy currently being studied in clinical trials involves engineering a patient’s own immune cells to recognize and attack tumor cells.  T-cells are a type of immune cell collected from the patient’s own blood.  After collection, the T-cells are genetically engineered to produce special receptors on their surface called chimeric antigen receptors (CARs).  These genetically engineered cells are then re-infused into the patient’s bloodstream.  After re-infusion, the T-cells multiply in the patient’s body and with guidance from their engineered receptor, are able to recognize and kill cancer cells. Click here to see introduction of CAR T-cells.

Clinical Trials

Clinical trials allow patients to try a new treatment before it is available to the general public. In some cases, this may be a new drug that has not been used in humans before, or it may be a drug or drug combination that is not currently used for that specific type of cancer. Early phase clinical trials are often used to test side effects of a drug or drug combination, while later phase clinical trials are used to see how effective a new treatment might be for a certain type of cancer. Clinical trials allow doctors and researchers to improve the treatment of cancers with possibly more effective therapies. A clinical trial may be a new, groundbreaking drug or it may have no effect. It is important to talk with your doctor about the pros and cons of clinical trials for your particular situation.

Different new treatments have been studied for patients with neuroblastoma:

• Targeted therapy is the use of a drug that attacks specifically tumor cells and spares normal cells. Crizotinib is a tyrosine kinase inhibitor studied in neuroblastoma, it blocks signals needed for tumor growth.
• Angiogenesis inhibitors are molecules that target blood vessels and prevent the tumor from growing.
• Vaccine therapy is the use of a substance that boosts the immune system to fight tumor cells.