A Phase II Study of High-Dose 3F8/GM-CSF Immunotherapy Plus 13-Cis-Retinoic Acid for Primary Refractory Neuroblastoma in the Bone Marrow

Full Title
3F8/GM-CSF Immunotherapy Plus 13-Cis-Retinoic Acid for Primary Refractory Neuroblastoma in Bone Marrow: A Phase II Study

Neuroblastoma is a solid tumor that mainly affects young persons and can be very difficult to treat successfully. Although a combination of chemotherapy, radiation therapy, and surgery can help patients with advanced disease achieve remission, many patients experience a relapse of their disease. Doctors are continually pursuing new, more effective therapies.

The purpose of this study is to evaluate the safety and effectiveness of high-dose 3F8 combined with GM-CSF compared with standard-dose 3F8 for treating neuroblastoma that has a high risk of returning in patients with evidence of the disease remaining only in the bone marrow. Researchers also want to see if the 3F8 antibody works against a very small amount of neuroblastoma (minimal residual disease) remaining in the bone marrow.

3F8 has been shown to kill cancer cells in some patients. This antibody attaches to neuroblastoma cells and helps focus a patient’s own immune system — especially white blood cells — to attack the neuroblastoma cells. Granulocytes make up one class of white blood cells. GM-CSF increases the number of granulocytes, and makes them better killers of cancer cells.


To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • Patients must have a confirmed diagnosis of neuroblastoma that is determined to have a high risk of recurrence.
  • Patients must have evidence of disease only in the bone marrow that remains despite prior therapy.
  • Patients must not have evidence of progressive disease.

For more information about this study and to inquire about eligibility, please contact Dr. Brian Kushner at 212-639-6793.

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