Researchers in Australia have made a significant breakthrough in the fight against neuroblastoma, a severe childhood cancer and the most common solid tumor in children outside the brain. This new discovery could potentially improve treatment outcomes for relapsed cases, which historically have low survival rates. The drug, romidepsin, traditionally used to treat lymphoma, has shown promise in overcoming the resistance that these tumors typically develop. Generally, neuroblastoma is known for its lethality; nine out of ten pediatric patients who face a recurrence unfortunately do not survive.
The Garvan Institute of Medical Research led the study that revealed how romidepsin can trigger neuroblastoma cell death through alternative pathways that bypass the damaged JNK pathway, which is often dysfunctional in relapsed tumors. Conventional chemotherapy treatments target the JNK pathway to induce cell death; however, in resistant neuroblastoma cases, this pathway is frequently non-functional, rendering standard treatments ineffective.
In experiments conducted using animal models, the research team found that combining romidepsin with standard chemotherapy drugs not only halted tumor growth but also extended survival and allowed for lower doses of chemotherapy. This reduction in dosage could minimize the side effects typically experienced by young patients undergoing treatment, making the regimen safer overall. The results of this research are published in the journal Science Advances, underscoring the potential impact of this combination therapy on pediatric oncology.
The lead researcher, David Croucher, emphasized the importance of overcoming the resistant nature of high-risk neuroblastomas, highlighting the devastating statistics for families once children reach a relapsed stage. Romidepsin's existing approval for other cancer types and its prior testing for safety in children is expected to speed up its development as a potential treatment for neuroblastoma. Despite these promising findings, Croucher notes the necessity for additional clinical trials to fully evaluate the safety and effectiveness of this new treatment regime.
In conclusion, the discovery of romidepsin as a viable option in combination with standard chemotherapy marks a significant step forward in neuroblastoma treatment. If clinical trials succeed, this could change the trajectory of care for pediatric patients battling this aggressive form of cancer.