Eflornithine (DFMO), an antiparasitic drug repurposed for cancer treatment, has shown promising results in combating neuroblastoma, an aggressive cancer primarily affecting young children. The drug received FDA approval in December 2023 and is emerging as a significant advancement in pediatric oncology.
Clinical Efficacy of DFMO
Neuroblastoma patients often face high relapse rates, with the cancer returning in approximately 50% of cases after initial treatment. Dr. Giselle Sholler, chief of Penn State Health Children’s Hospital’s Division of Pediatric Hematology and Oncology, has been instrumental in researching DFMO as a maintenance therapy to prevent relapse. Clinical trials have demonstrated that DFMO reduces relapse rates to 15% when administered as a twice-daily pill for two years following initial treatment.
"With DFMO added at the end (of treatment) for two years, they take a pill twice a day, they go back to school and their immune systems are fine," said Dr. Sholler, emphasizing the improved quality of life for children undergoing treatment.
Mechanism of Action
DFMO's efficacy in treating neuroblastoma is linked to its ability to suppress the MYCN gene, a known driver of the cancer. Originally developed as an antiparasitic drug for African sleeping sickness, DFMO was found to inhibit neuroblastoma tumor formation in preclinical studies. Mice injected with neuroblastoma that were given DFMO in their drinking water did not develop tumors, which shifted the focus of research toward preventing relapse.
Funding and Support from Families
Funding for pediatric cancer research is often limited, with only about 4% of federal cancer research funds allocated to pediatric cancers. The development of DFMO has been significantly supported by families of children affected by neuroblastoma. Tyler's parents, whose son was the first patient Dr. Sholler ever lost, provided initial funding for clinical trials. The Beat Childhood Cancer Foundation, founded by Pat Lacey, whose son Will was saved by DFMO, has also been a crucial source of funding.
Meryl Witmer, a director at Berkshire Hathaway, played a key role in ensuring the drug's production when the initial pharmaceutical company ceased supply. Kristen Gullo, a senior vice president at US WorldMeds, facilitated her company's backing of DFMO's FDA approval bid. The drug's patent name, Iwilfin, honors Will Lacey and Finn Johnson, Gullo's nephew, both of whom benefited from DFMO treatment.
Future Directions
Dr. Sholler's ongoing research at Penn State College of Medicine focuses on translating research into clinical trials. With the FDA approval of DFMO, the focus is now on expanding access to the drug and continuing to explore its potential in treating neuroblastoma and other cancers. The commitment to research and the support from organizations like Four Diamonds are expected to further advance treatment options for children with cancer.
