Jones Chris, Straathof Karin, Fouladi Maryam, Hargrave Darren, Prados Michael, Resnick Adam, Doz Francois, Jones David T W, Mueller Sabine
Division of Molecular Pathology, Institute of Cancer Research, London, United Kingdom.
Department of Oncology, University College London Cancer Institute, London, United Kingdom.
Front Oncol. 2023 Apr 5;13:1167082. doi: 10.3389/fonc.2023.1167082. eCollection 2023.
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.
尽管我们对儿童脑肿瘤潜在的疾病生物学有了越来越多的了解，但许多儿童脑肿瘤的临床预后仍然很差。分子诊断技术的进步提高了我们对肿瘤类型和亚型进行分类的能力，多个国际儿科神经肿瘤学联盟正在努力将对预后最差的实体瘤的新生物学见解应用于创新临床试验。虽然我们首次基于疾病特异性生物学数据设计此类研究，但启动这些试验所依据的适当模型系统中的临床前证据水平仍存在很大差异。我们在CONNECT、PNOC和ITCC-Brain之间讨论了这些问题，并制定了一个框架，在这个框架中我们可以评估为可能的临床转化而提出的新概念。这些标准并非旨在适用于每一种可能的情况，而是为实验室科学家提供了自我评估证据的基础，以及在临床推进之前进行讨论和合理决策的平台。
