Next-generation sequencing panel for solid tumour cancers in children

Patient organisation comments

Two representatives from the Christopher's Smile charity, 1 individual patient expert who is also a consumer member from the National Cancer Research Institute Children's Cancer & Leukaemia Clinical Studies Group (NCRI CCL CSG) and 1 representative from the Abbie's Army charity, provided comments on the NGS panel test as summarised below.

Christopher's Smile recognises the importance of personalised medicine and supports the chief medical officer's 2016 annual report, which promotes the need for accurate, quick, cost-effective testing to determine the genetic abnormalities that cause cancer. The charity funded the preclinical development and validation of the NGS panel technology and, although large clinical trials are still underway, the charity believes that it should now become part of standard care as a diagnostic tool. The opportunity to collect sequencing data is a huge potential benefit: adopting the NGS panel would build evidence to show that specific drug targets in adults also occur in children, which may persuade some companies to make drugs available for research in children earlier in the pipeline. Data from the NGS panel test will be critical when planning and assessing the treatment plans for cancer in children.

The individual patient expert agrees that genomic sequencing of childhood cancers should become standard practice in the UK, stating that the UK is already behind in terms of sequencing these tumours. Sequencing of tumours at both diagnosis and relapse will also help unlock additional areas of research, exploring and understanding the changes that occur with the tumour, which may in turn be used to develop new ways of treating these diseases.

Abbie's Army focuses on diffuse intrinsic pontine glioma (DIPG), a childhood cancer with a survival rate of less than 1%. A meta-analysis of 1,000 children with high-grade gliomas and DIPG revealed at least 10 disease and biological subgroups (McKay et al. 2017). The charity agrees that clinical treatments must keep pace with the science and that the NGS panel is the starting point. The benefits of a more personalised therapeutic approach have been seen in the BIOMEDE trial, the results of which are due to publish in 2018.