Neuroblastoma is a solid cancerous tumor that begins in the nerve cells outside the brain of infants and young children. It can start in the nerve tissue near the spine in the neck, chest, abdomen, or pelvis, but it most often begins in the adrenal glands. The adrenal glands are located on top of both kidneys. These glands make hormones that help control body functions, such as heart rate and blood pressure.

Neuroblasts are immature nerve cells found in unborn babies. Normal neuroblasts mature into nerve cells or adrenal medulla cells, which are cells found in the center of the adrenal gland. Neuroblastoma forms when neuroblasts don’t mature properly. Sometimes, babies are born with small clusters of neuroblasts that eventually mature into nerve cells and do not become cancer. A neuroblast that does not mature can continue to grow, forming a mass called a tumor.

Neuroblastoma develops most often in infants and children younger than 5. It can form before the baby is born and can sometimes be found during a prenatal (before birth) ultrasound. Most often, however, neuroblastoma is found after the cancer has spread to other parts of the body, such as the lymph nodes, which are tiny, bean-shaped organs that help fight infection, liver, lungs, bones, and bone marrow, the spongy, red tissue in the inner part of large bones.

Neuroblastoma Trials


Pediatric Precision Laboratory Advanced Neuroblastoma Therapy (PEDS-PLAN)
A prospective open label, multicenter study to evaluate the feasibility and acute toxicity of using molecularly guided therapy in combination with standard therapy followed by maintenance therapy with DFMO in subjects with newly diagnosed high risk neuroblastoma.


Neuroblastoma Maintenance Therapy Trial (NMTT)
Difluoromethylornithine (DFMO) will be used in an open label, single agent, multicenter, study for patients with neuroblastoma in remission. In this study subjects will receive 730 Days of oral difluoromethylornithine (DFMO) at a dose of 500 to 1000 mg/m2 BID on each day of study. This study will focus on the use of DFMO in high risk neuroblastoma patients that are in remission as a strategy to prevent recurrence.


Study of DFMO in Combination With Bortezomib for Relapsed or Refractory Neuroblastoma
The purpose of this research study is to evaluate an investigational drug (DFMO) in combination with bortezomib, for relapsed and refractory neuroblastoma. DFMO is an investigational drug because it has not been approved by the U.S. Food and Drug Administration (FDA). This study will look at the safety and tolerability of DFMO in combination with bortezomib as well as the tumors response to this study drug.


Preventative Trial of Difluoromethylornithine (DFMO) in High Risk Patients With Neuroblastoma That is in Remission
The purpose of this research study is to evaluate a new investigational drug to prevent reoccurrence of neuroblastoma that is in remission. This study drug is called DFMO. The objectives of this study will be to monitor for safety and look at efficacy of DFMO.

The safety of the proposed dosing regimen in this trial will be tested by an on-going risk/benefit assessment during the study. A patient benefiting from treatment, not progressing on therapy, and in the absence of any safety issues associated with DFMO may continue on treatment up to 27 cycles with the expectation that there will be an overall clinical benefit.


A Phase I Trial of Tolcapone and Oxaliplatin in Patients with Relapsed or Refractory Neuroblastoma


Molecular-guided therapy for the treatment of patients with relapsed and refractory childhood cancers


A Two-Part Study of TB-403 in Pediatric Subjects with Relapsed or Refractory Medulloblastoma, Neuroblastoma, Ewing Sarcoma, or Alveolar Rhabdomyosarcoma.


A Phase II Trial of Nifurtimox for Refractory or Relapsed Neuroblastoma or Medulloblastoma


A Phase I Trial of DFMO Targeting Polyamine Addiction in Patients with Relapsed/Refractory Neuroblastoma
Neuroblastoma (NB) is the most common cancer in infancy and most frequent cause of death from extracranial solid tumors in children. Ornithine decarboxylase (ODC) expression is an independent indicator of poor prognosis in NB patients. This study investigated safety, response, pharmacokinetics, genetic and metabolic factors associated with ODC in a clinical trial of the ODC inhibitor difluoromethylornithine (DFMO) ± etoposide for patients with relapsed or refractory NB.

Abstracts and Publications

PLOS Phase 1 Published Paper

A Phase I Trial of DFMO Targeting Polyamine Addiction in Patients with Relapsed/ Refractory Neuroblastoma. Download PDF.

Oncotarget LIN28/Let 7 Published Paper

Targeting ornithine decarboxylase reverses the LIN28/Let-7 axis and inhibits glycolytic metabolism in neuroblastoma. Download PDF.

ANR 2016 Abstract Book

DFMO maintains remission and increases overall survival in high risk neuroblastoma: results of a phase II prevention trial. PDF.