Because of the pioneering work of the Department of Pediatrics at Memorial Sloan Kettering, more than 50 percent of patients treated here today survive the disease. In the 1980s, that number was less than 5 percent.

Treatment options depend on whether the disease is low- or high-risk or has returned (relapsed) after initial treatment. Most children with neuroblastoma will receive one or more forms of treatment. These generally include chemotherapy, surgery, and radiation therapy. At MSK, we follow these initial therapies with the immunotherapy-humanized monoclonal antibodies Hu3F8 and 8H9, which were developed in our labs.

Cancer immunotherapy uses drugs and vaccines to harness the body’s own immune system and direct it toward cancer cells, fighting a tumor in the same way that it would a virus. Hu3F8 detects and destroys neuroblastoma cells that have survived chemotherapy or radiation therapy.  After the patient’s immune system recovers from the effects of chemotherapy, we use anti-neuroblastoma vaccines to train the patient’s immune system to destroy any remaining cancer cells.

Pioneering Therapies

Through the dedicated research efforts of MSK’s neuroblastoma program, our physician-scientists have made great strides in developing safer and more-effective therapies for patients with this disease. Some of these therapies have the potential to be used for other solid tumors and brain tumors in children. The following are some studies currently under way:

  • Exciting work continues to further refine the Hu3F8 antibody using new drug combinations and approaches.,
  • While effective in killing cancer cells, radiation therapy also harms healthy surrounding organs and tissue. This is a particular concern when it is used in children. By attaching the antibody Hu3F8 to radioactive iodine, investigators are using PET scan imaging to track how Hu3F8 travels through the body to the tumor sites. Researchers want to determine if this radiolabeled antibody (known as 124I-Hu3F8) can be safely used as a means to deliver higher doses of radiation to tumor cells in patients with neuroblastoma. If so, the application of this treatment could have wider implications — benefiting other cancers associated with the Hu3F8 antibody such as osteosarcoma, a pediatric tumor that develops in the bone.
  • Another radioactive antibody under evaluation is 131I-8H9, used to treat children and adults with cancer of the brain or brain covering that has returned, or for which no standard therapy exists. Included in the trial are patients with neuroblastoma. This is the first-ever study assessing 131I-8H9 administered directly into the spinal fluid to kill cancer cells. It has already made a major impact on cure rates for children who experience a relapse.
  • An ongoing study of a “bivalent” vaccine targets two antigens found in patients with neuroblastoma: GD2L and GD3L. Invented at MSK, the vaccine can help train the body to do the work of the Hu3F8 antibody. For a patient who is disease free, the goal is to render ongoing treatment with Hu3F8 unnecessary.
  • Previous MSK studies have demonstrated that natural killer (NK) cells — white blood cells that recognize and kill abnormal cells in the body — work well in tandem with Hu3F8 to target neuroblastoma. Other work has shown that introducing NK cells obtained from a donor (also known as a “mismatch”) can boost the killer cells’ antitumor activity against neuroblastoma. To take this even further, an MSK trial has opened to evaluate a regimen that includes Hu3F8, the anticancer drug cyclophosphamide, and NK cells from a donor. Patients will also receive a treatment called interleukin-2, or IL-2, which stimulates NK cells and helps them grow and survive.
  • Exome sequencing is performed on every neuroblastoma tumor both at diagnosis and at relapse to identify possible genetic mutations that can be targeted by novel anticancer agents. The development of the sophisticated sequencing platform MSK-IMPACT™ has been invaluable to perform these studies with a view to personalizing therapy for each individual patient.

Within MSK’s neuroblastoma program, there is reason for hope that this work will lead to a cure — even for children with high-risk or relapsed cancer. Our breakthrough research in neuroblastoma has been instrumental in saving the lives of hundreds of children. Additional studies are critical to maintaining the pace and progress of research so that we can help children fighting these cancers, not just at MSK but everywhere.