Christopher’s Smile believes that personalised medicine for children diagnosed with solid tumour malignancies is the best strategy to improve outcomes. To introduce personalised medicine there must be robust tumour genomic analysis methods as standard that are cost effective and can be performed in clinically relevant timescales. NGS sequencing meets this need and the introduction of NGS Panel Sequencing for paediatric tissue samples is now in progress.
Christopher’s Smile would like to now advance to the genomic analysis of circulating tumour cells in blood and cerebral spinal fluid. We are confident that ‘liquid biopsies’ can offer huge advantages for children, especially those with CNS malignancies.
Christopher’s Smile would like to oversee the introduction of liquid biopsies into the clinic in a 3-stage process:
Stage 1 – Clinical Validation of blood and CSF samples
Stage 2 – Clinical Trial
Stage 3 – Implementation into standard NHS care.
Christopher’s Smile would like at this stage to fund a project to fulfil stage 1 and is prepared to make a significant grant for Stage 2. Christopher’s Smile views this programme of work as technology application and implementation, not scientific discovery. Liquid biopsies as a technology are already being used in some spheres of cancer diagnosis and the charity would like them to be introduced into the paediatric arena as soon as practicably possible.
Christopher’s Smile would welcome interest from UK institutions that could manage the first two stages and work with the charity to obtain Stage 3. The successful institution should show a proactive approach to completing Stage 1 as soon as possible and while Stage 1 is in progress make all the preparations for Stage 2. This approach will ensure that Stage 2 happens as early as possible.
As part of the selection process, Christopher’s Smile would like the institution to present a plan showing the timescales and costing for Stage 1 in addition to timescales and costings for Stage 2. The charity expects the institution to present background information that defines the scope and content of Stage 1 and not simply base the stage duration on a length of time. Likewise the scope of Stage 2 should be sufficient to progress to Stage 3 without unnecessary duration and expenditure.
Christopher’s Smile would like this programme of work to benefit as many children with solid tumours as possible and especially those with the following malignancies:
|Medulloblastoma||Glioma incl. DIPG||Osteosarcoma|
|Rhabdomyosarcoma||Ewings Sarcoma||Wilm’s Tumour|
The applicant should provide details of how these malignancies will be pre clinically validated so children with these malignancies can be enrolled in the trial as early as possible.
Stage 1 should also test the feasibility of being able to undertake Whole Genome Sequencing (WGS) from a liquid biopsy. This small feasibility study should be included within the scope of Stage 1. The rationale defining the size of this feasibility study being proposed should be shown.
If an institution would like to be considered to partner Christopher’s Smile in this project please email info(Replace this parenthesis with the @ sign)christopherssmile.org.uk by 24th November 2017 with your draft response showing your best estimate costings. The selection process will involve a number of stages which will be outlined to each applicant once their response has been received.