In the first clinical trial of difluoromethylornithine (DFMO), an investigational agent for children suffering from neuroblastoma, researchers found minimal side effects with long-term survival of three patients. This is the first clinical study of an oral dosing form of DFMO in any pediatric population.

“This DFMO trial is an important advancement in neuroblastoma research,” explained study leader and pediatric oncologist Giselle Sholler, MD, of Spectrum Health Helen DeVos Children’s Hospital in Grand Rapids, MI, and the Neuroblastoma and Medulloblastoma Translational Research Consortium (NMTRC). “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. Sholler recently published her laboratory studies describing how this drug works in neuroblastoma in preventing tumor formation in lab models and also published the full results of the phase 1 trial in PLOS ONE (2015; doi:10.1371/journal.pone.0127246).

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The trial found that DFMO was well tolerated with minimal side effects in children with relapsed neuroblastoma. Children with specific genetic changes are predicted to have better response to DFMO. There were three patients enrolled in the trial who are now long-term survivors.

Dr. Sholler’s laboratory investigated the effectiveness of combining DFMO with the drug etoposide. She incorporated early work performed by Andre Bachmann, PhD, professor of pediatrics and human development at Michigan State University College of Human Medicine, in East Lansing, MI. Bachmann’s work identified the relationship of this drug targeting the ODC gene in neuroblastoma.

Sholler then designed and led a clinical trial to test the combination of drugs in children being treated for the disease at sites participating in the NMTRC. Dr. Sholler and NMTRC are now testing this concept in a phase 2 clinical trial to prevent relapse.