The Long Road to a Medical Milestone: How Basic Research Paves the Way for Breakthroughs

December 22, 2023

A journey from UH Cancer Center to the FDA: The FDA announced a breakthrough and approved the use of DFMO/eflornithine (IWILFIN) in adult and pediatric patients with neuroblastoma

A significant development recently occurred in the fight against a type of cancer called neuroblastoma, which affects immature nerve cells outside of the brain, most often occurring in young children. On December 13, The Food and Drug Administration (FDA) approved a drug called DFMO (eflornithine, now known as IWILFIN) to reduce the risk of relapse in patients with high-risk neuroblastoma.

The person behind the initial research leading to this approval is André S. Bachmann, PhD, 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 until he was recruited to Michigan State University in 2015.

The drug was initially approved for treating African sleeping sickness and hirsutism, the latter being a condition in women with excessive facial hair growth..

Bachmann and his team were the first to suggest that DFMO could potentially change how we treat pediatric neuroblastoma in the future. Although Bachmann is now a professor and associate chair for research at Michigan State University, he credits his time at the UH Cancer Center for laying the early groundwork for this breakthrough.

“This FDA announcement is a testament that basic science research is 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 concept was cemented in two seminal first papers published in the Hawaiʻi Medical Journal in 2004 and in Oncogene in 2005, followed by several other key publications in the American Association for Cancer Research, Journal of Biological Chemistry, International Journal of Cancer, and PLoS that confirmed a key role of ODC/DFMO in neuroblastoma. Bachmann also secured a prestigious National Cancer Institute Research Project Grant (R01) in 2006 to further this line of inquiry and a U.S. patent with support from the UH Office of the Vice President for Research and Innovation.

“This FDA approval serves as a compelling reminder of the essential role that basic research plays in advancing medicine,” said Naoto T. Ueno, MD, PhD, 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 Bachmann and his team for laying the groundwork for this life-saving therapy.”