Department of Oncology, R&D, Sanofi, Vitry-sur-Seine, France.
Department of Alliance Management and Public Private Partnerships, Sanofi, Gentilly, France.
Handb Exp Pharmacol. 2024;286:33-50. doi: 10.1007/164_2024_725.
The outcome for children with cancer has improved significantly over the past 60 years, with more than 80% of patients today becoming 5-year survivors. Despite this progress, cancer remains the leading cause of death from disease in children in the United States and Europe, with significant short- and long-term toxicity of treatment continuing to impact most children. While the past 15 years have witnessed dramatic scientific innovation for certain cancers in adult patients, pediatric cancer treatment innovation lags increasingly behind. To help bridge the adult-pediatric therapeutic development gap, collaborative efforts are essential among stakeholders within and outside the pediatric oncology community. Prioritizing collaboration in areas such as cancer characterization, target identification and validation, drug discovery, and approaches to currently "undruggable" targets is imperative to improving the outcomes for children with cancer.
过去 60 年来，儿童癌症患者的治疗效果显著提高，目前超过 80%的患者成为了 5 年幸存者。尽管取得了这些进展，但癌症仍然是美国和欧洲儿童因病死亡的主要原因，治疗带来的短期和长期毒性仍然对大多数儿童产生影响。尽管过去 15 年来，某些成人癌症的治疗取得了显著的科学创新，但儿科癌症治疗的创新却越来越滞后。为了帮助弥合成人与儿科治疗开发之间的差距，儿科肿瘤学领域内外的利益相关者之间必须进行协作。在癌症特征描述、靶点识别和验证、药物发现以及针对目前“不可成药”靶点的方法等领域，优先开展合作，对于改善儿童癌症患者的治疗效果至关重要。
