"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."
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.
Read our blog post summarizing the most recent published paper.
Abstracts and Publications
Maintenance DFMO Increases Survival in High Risk Neuroblastoma
101 subjects enrolled on Stratum 1 and 100 were eligible for ITT analysis; two-year EFS was 84% (±4%) and OS 97% (±2%). Download PDF.
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.