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Neuroblastoma Treatments

Neuroblastoma experts Kim Kramer (left) and Nai-Kong Cheung

At MSK, we’re committed to providing your child with the right treatment at the right time. Over the past 20 years, we have successfully spared many young children with neuroblastoma from unnecessary toxic treatments.

No matter which treatment we recommend, rest assured that your child is getting the very best in care.

We treat neuroblastoma depending 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, radiation therapy, and surgery. Our surgical skill and expertise are world-renowned. We have unparalleled success in removing the most difficult tumors.

After initial treatment, some patients may benefit from the antibody medicines Hu3F8 and 8H9, both of which were developed in our labs. Hu3F8 detects and destroys neuroblastoma cells that have survived chemotherapy or radiation therapy. 8H9 is liquid radiation that targets tumor cells in the brain while sparing nearby brain and spinal cord tissue. We have been giving Hu3F8 since the 1980s and 8H9 since 2004, all on an outpatient basis, so you and your family can get back to what matters. Thanks to these medications, many neuroblastoma patients who were once told they were out of options are now long-term survivors of the disease. In fact, 8H9 was recently given Breakthrough Status by the US Food and Drug Administration, meaning it has proven to be so effective that it could be approved sooner than originally thought.

After your child’s immune system recovers from the effects of chemotherapy, we may use an anti-neuroblastoma vaccine to train the immune system to destroy any remaining cancer cells. So far more than 100 children have received this vaccine, which is currently being tested in a clinical trial at MSK.

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.
  • The FDA recently granted Breakthrough Therapy Designation to a drug called burtomab for the treatment of children and adults whose neuroblastoma has spread to the brain. Burtomab — also known as 8H9 — is a radioactive antibody that delivers precise liquid radiation to cancer cells when it is injected into the spinal fluid. Early clinical data has shown that burtomab may demonstrate substantial improvement over current therapies, and the FDA’s designation is meant to speed up the drug’s review process.
  • 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.