Iwilfin (eflornithine) approved by the FDA as the first and only oral maintenance therapy for high-risk neuroblastoma in adult and pediatric patients: Narrative review

Neural crest progenitor cells give rise to neuroblasts, the growing nerve cells of the sympathetic nervous system. These cells can undergo changes leading to neuroblastoma, a malignancy responsible for 15% of all pediatric cancer-related deaths. The molecular pathogenesis of this pediatric cancer involves complex genetic alterations, such as MYCN amplification, chromosomal abnormalities, and gene expression changes. Despite aggressive therapies, survival rates for children with high-risk neuroblastoma (HRNB) have not improved significantly compared to those with less severe forms of the disease. This highlights the challenge of managing HRNB and underscores the need for new, effective treatments. A comprehensive treatment regimen, including immunotherapy, radiation therapy, myeloablative chemotherapy, and surgical removal, has been employed to achieve remission in HRNB patients. While dinutuximab beta immunotherapy is an effective and widely used treatment, it has several potential side effects that must be carefully monitored. New drugs are being developed to reduce these side effects without compromising efficacy. One such drug is DL-alpha-difluoromethylornithine (DFMO), approved by the FDA under the brand name Iwilfin. Numerous clinical trials have shown that DFMO, when used as maintenance therapy, significantly improves event-free survival and overall survival in neuroblastoma patients. However, DFMO has adverse effects that require continuous monitoring. Further research is needed to minimize these side effects and improve its efficacy, particularly in addressing resistance caused by long-term use.