神经母细胞瘤（NB）仍然是一种具有挑战性的儿科恶性肿瘤，治疗选择有限，特别是对于高危病例。药物再利用为治疗 NB 等罕见疾病提供了一种方便且经济有效的策略。使用具有已知安全性的现有药物可以加速新疗法的推出，降低开发成本并降低风险，为在具有挑战性的条件下改善患者的治疗结果带来希望。这篇综述概述了将药物重新用于 NB 治疗的方法的进展。作者讨论了药物再利用所采用的策略，包括计算和实验方法以及合理的药物设计，重点介绍了具有良好临床效果的药物再利用的关键例子。此外，作者还研究了 NB 中药物再利用相关的挑战和机遇，并讨论了未来的方向和进一步研究的潜在领域。事实是，过去 30 年里只有一种新药被批准用于治疗神经母细胞瘤，而且其中很大一部分药物被批准用于治疗神经母细胞瘤。仍然无法治愈的高风险 NB 患者的出现证明需要新的快速且具有成本效益的替代方案。药物再利用可以加速治疗开发过程，同时降低费用和风险。这种方法可以迅速将有效的 NB 疗法推向市场，提高生存率和患者的生活质量。

Neuroblastoma (NB) remains a challenging pediatric malignancy with limited treatment options, particularly for high-risk cases. Drug repurposing offers a convenient and cost-effective strategy for treating rare diseases like NB. Using existing drugs with known safety profiles accelerates the availability of new treatments, reduces development costs, and mitigates risks, offering hope for improved patient outcomes in challenging conditions.This review provides an overview of the advances in approaches used to repurpose drugs for NB therapy. The authors discuss strategies employed in drug repurposing, including computational and experimental methods, and rational drug design, highlighting key examples of repurposed drugs with promising clinical results. Additionally, the authors examine the challenges and opportunities associated with drug repurposing in NB and discuss future directions and potential areas for further research.The fact that only one new drug has been approved in the last 30 years for the treatment of neuroblastoma plus a significant proportion of high-risk NB patients that remain uncurable, evidences the need for new fast and cost-effective alternatives. Drug repurposing may accelerate the treatment development process while reducing expenses and risks. This approach can swiftly bring effective NB therapies to market, enhancing survival rates and patient quality of life.