Researchers at the Beat Childhood Cancer Research Consortium, led by Dr. Giselle Saulnier Sholler, have achieved a major breakthrough in the treatment of pediatric high-risk neuroblastoma. The U.S. Food and Drug Administration (FDA) has granted approval for the first drug, called IWILFIN (formerly known as eflornithine or DFMO), based on the consortium’s research and clinical trials. This drug has the potential to reduce the risk of relapse in pediatric neuroblastoma patients by more than 50% after undergoing multiagent, multimodality therapy.
Neuroblastoma is a rare form of cancer that primarily affects children under the age of six. It develops in immature nerve cells and commonly occurs in the adrenal gland or near the spine. Unfortunately, by the time of diagnosis, approximately 50% of patients already have advanced metastatic disease, which significantly decreases their chances of survival. Furthermore, about 40% of high-risk patients who enter remission will experience relapse within four years, and less than 10% of those relapsed patients survive for another five years.
The approval of IWILFIN is the result of extensive research conducted by Dr. Sholler and her team over the past 20 years. In their studies, they discovered that neuroblastoma tumors have highly activated expression of the ODC1 gene, which promotes the synthesis of polyamines. However, excessive polyamines can lead to uncontrolled cell division and cancer development. Dr. Sholler found that IWILFIN, in addition to inhibiting polyamine synthesis, also blocks the LIN28 signaling pathway, which helps cancer stem cells survive and contribute to relapse.
The initial clinical trials of IWILFIN showed promising results. In a Phase I trial, 17 children with incurable neuroblastoma participated, and three of them survived. This led Dr. Sholler to investigate further and found that DWILFIN was particularly effective in preventing relapse in some patients. Subsequent trials focused on high-risk neuroblastoma children in remission, using DWILFIN as a maintenance therapy. Results showed that 97% of children who took DWILFIN twice a day for two years survived, with 84% remaining in remission. Even after four years, 96% were still alive, and 83% were still in remission.
Although the clinical trials were promising, an issue arose regarding the absence of a randomized controlled trial, which the FDA generally requires for drug approval. To address this, Dr. Sholler collaborated with the FDA on an externally controlled study, where they compared the effects of DWILFIN in her trial with a separate group of neuroblastoma patients who had received the same treatment.
The breakthrough therapy designation received from the FDA in 2020 facilitated the testing and review process for DWILFIN, ultimately leading to the partnership between Kids Cure Pharmaceuticals and US WorldMeds for streamlined production. Over 850 pediatric cancer patients participated in more than eight trials under the Beat Childhood Cancer Research Consortium, which greatly contributed to the evidence supporting the approval of DWILFIN.
After presenting the compelling results to the FDA’s Oncologic Drugs Advisory Committee, the committee voted 14 to 6 in favor of DWILFIN, acknowledging its potential in reducing the risk of neuroblastoma relapse. This decision was met with immense support from the community, with over 160 families writing letters advocating for DWILFIN.
The FDA’s approval of DWILFIN marks a significant milestone in the treatment of pediatric high-risk neuroblastoma. This breakthrough drug provides hope for children and their families affected by this devastating disease. IWILFIN, expected to be commercially available in early 2024, symbolizes the collaboration and dedication of researchers, pharmaceutical companies, and patient advocates in improving outcomes for pediatric cancer patients. Dr. Sholler expressed her gratitude to all those involved and emphasized the transformative power of research in bringing about positive change in the field of pediatric oncology.
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