A significant development has advanced in the fight against neuroblastoma, a type of cancer most often occurring in young children that affects immature nerve cells outside of the brain. On December 13, The Food and Drug Administration (FDA) approved the drug DFMO (eflornithine, now known as IWILFIN) to reduce the risk of relapse in pediatric patients with high-risk neuroblastoma.
Behind the initial research leading to this approval is André S. Bachmann and his research team. Bachmann was formerly an associate professor at the University of Hawaiʻi Cancer Center until 2010. He then moved to UH Hilo as department chair and professor of pharmaceutical sciences before joining Michigan State University in 2015.
While at the UH Cancer Center, Bachmann and his team were the first to suggest that DFMO could potentially change how medical professionals treat pediatric neuroblastoma. Bachmann credits his time at the UH Cancer Center for laying the groundwork for this breakthrough.
"The FDA announcement is a testament that basic science and pre-clinical research are important," said Bachmann.
Based on keen insights into MYCN, a gene that plays a vital role in regulating cell division and tumor growth and is often amplified in this type of tumor, Bachmann's team proposed that DFMO could target a MYCN-activated protein called ornithine decarboxylase (ODC), an indicator of poor prognosis in neuroblastoma patients.
This FDA approval serves as a compelling reminder of the essential role that basic research plays in advancing medicine
—Naoto Ueno
"This FDA approval serves as a compelling reminder of the essential role that basic research plays in advancing medicine," said Naoto T. Ueno, director of the UH Cancer Center. "Although the spotlight often shifts to clinical trials and FDA approvals, the meticulous, foundational work sets the stage for these breakthroughs. This underscores the importance of the work initiated here at the UH Cancer Center and later continued at Michigan State University. Thank you to Dr. André S. Bachmann and his team for laying the groundwork for this life-saving therapy."
