First Clinical Trial of DFMO for Neuroblastoma Completed; Drug Targets a Cancer Stem Cell Pathway and Shows Early Promise

Researchers at Spectrum Health Helen DeVos Children's Hospital in Grand Rapids, Michigan have completed the first clinical trial of a new treatment for children suffering from neuroblastoma. In a clinical trial led by Giselle Sholler, M.D., Pediatric Oncologist at Helen DeVos Children's Hospital and the Neuroblastoma and Medulloblastoma Translational Research Consortium (NMTRC), DFMO (difluoromethylornithine), an investigational agent, showed 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. DFMO has previously been shown to inhibit ornithine decarboxylase (ODC), an independent indicator of poor prognosis in neuroblastoma. "This DFMO trial is an important advancement in neuroblastoma research," explained Dr. Sholler. "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 one trial. The work was published online on May 27, 2015 in the open-access journal PLOS ONE. The article is titled “A Phase I Trial of DFMO Targeting Polyamine Addiction in Patients with Relapsed/Refractory Neuroblastoma.” Will Lacey, a patient in the phase 1 clinical trial, was free from side effects and needed no additional treatment following the trial. This is a new way of life for a 10-year old whose neuroblastoma had kept him in and out of hospitals and on various treatments for most of his life. Patrick Lacey, Will's father explained, "His quality-of-life has been amazing!
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