尽管我们对潜在疾病的生物学有了越来越多的了解，许多儿童脑肿瘤的临床结果仍然不佳。分子诊断的进步使我们能够更精确地分类肿瘤类型和亚型，多个国际小儿神经肿瘤组织正在努力将最坏预后实体的新生物学见解引入创新的临床试验中。虽然我们首次根据疾病特异性生物学数据设计这些研究，但这些试验在适当的模型系统中启动的前期证据水平仍然存在极大的差异。我们考虑了CONNECT、PNOC和ITCC-Brain之间的这些问题，并制定了一个框架，以评估引入可能进行临床转化的新概念。虽然这些标准并非面面俱到，但它们为实验室科学家自我评估证据提供了基础，并为在临床前进行讨论和理性决策提供了平台。版权所有©2023 Jones, Straathof, Fouladi, Hargrave, Prados, Resnick, Doz, Jones和Mueller。

Clinical outcomes for many childhood brain tumours remain poor, despite our increasing understanding of the underlying disease biology. Advances in molecular diagnostics have refined our ability to classify tumour types and subtypes, and efforts are underway across multiple international paediatric neuro-oncology consortia to take novel biological insights in the worst prognosis entities into innovative clinical trials. Whilst for the first time we are designing such studies on the basis of disease-specific biological data, the levels of preclincial evidence in appropriate model systems on which these trials are initiated is still widely variable. We have considered these issues between CONNECT, PNOC and ITCC-Brain, and developed a framework in which we can assess novel concepts being brought forward for possible clinical translation. Whilst not intended to be proscriptive for every possible circumstance, these criteria provide a basis for self-assessment of evidence by laboratory scientists, and a platform for discussion and rational decision-making prior to moving forward clinically.Copyright © 2023 Jones, Straathof, Fouladi, Hargrave, Prados, Resnick, Doz, Jones and Mueller.