DFMO

“We believe that by using DFMO to target an important cancer stem cell pathway to ‘turn cells off,’ we may prevent children from relapsing. Cancer cells have pathways that drive the cancer to grow and DFMO targets a specific pathway to turn these cells off.”Dr Giselle Sholler

DFMO, or difluoromethylornithine, is an ODC inhibitor which we’ve been funding research into since 2010. Ornithine decarboxylase (ODC) expression is an indicator of poor prognosis in NB patients, and the hope was DFMO would stop kids from relapsing with neuroblastoma.

Results have been motivating, and we are encouraged and continue to fund work with DFMO.

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.

DFMO Videos

DFMO Trials

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.

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.

Safety Study for Refractory or Relapsed Neuroblastoma With DFMO Alone and in Combination With Etoposide

The purpose of this research study is to evaluate a new investigational drug to treat neuroblastoma. This study drug is called DFMO. The objectives of this study will be to monitor for safety and to find a maximum tolerated dose in this population. A secondary objective will be to 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 and/or etoposide may continue on treatment with the expectation that there will be an overall clinical benefit.

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.

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.