Project information

Genetic abnormalities in paediatric brain tumours

We want to expedite new treatments for children with cancer. We have funded a new genetic sequencing test specifically for children and we now need a researcher to analyse data generated from the test to enable clinical decisions to be made in terms of new targeted treatments for children.

4 Years with start date in 2017

Charity information: Christopher's Smile

Christopher's Smile logo
  • Need

    Need

    Approximately 40% of children diagnosed with Medulloblastoma, an aggressive childhood brain tumour, do not survive. Those that do survive suffer lifelong side effects from the harsh treatments that were administered in order to rid the children of their disease. There is a need to identify those children who face the worst prognosis from the outset in order to offer them an innovative treatment which may be more effective and result in less side effects that current treatments.

    Solution

    This project will identify any trends between genetic abnormality of the tumour identified using Next Generation Sequencing at diagnosis, treatment given and short term outcome. The objective is to identify those children who are least likely to benefit from current standard treatments in order to offer them a clinical trial from the outset.

  • Aims

    Aim 1

    Collate tumour sequencing data from children in the UK with Medulloblastoma over a given period.

    Activities

    » Obtain sequencing data from all UK centres where children were diagnosed with Medulloblastoma

    Access and retrieve all required data from every centre in the UK


    Aim 2

    Collate the treatments these children were given and the short term outcome for these children.

    Activities

    » Obtain treatment and short term survival data from all UK centres where children were diagnosed with Medulloblastoma.

    Access and retrieve all required data from every centre in the UK


    Aim 3

    Identify any trends between tumour genetic abnormality, treatment given and outcome.

    Activities

    » Analyse data to identify any trends that may be present

    Report on the results of the data analysis highlighting any trends


    Aim 4

    Produce hypothesis as to why treatments may not be effective for a given tumour genetic abnormali

    Activities

    » If any trends exist, produce a theory as to why there could be a link between tumour genetic abnormality at diagnosis and short term outcome.

    Produce scientific paper for peer review


    Aim 5

    Use the information as evidence to offer some children enrolment onto a clinical trial in the fir

    Activities

    » Present findings in peer reviewed journal
    » Use evidence to offer children the opportunity to enrol on a clinical study from the outset and therefore not be given current standard treatments

    Children are enrolled on clinical studies at diagnosis as a result of this work


  • Impact

    Impact

    Sub populations of children with Medulloblastoma will be identified at diagnosis as having a significantly lower chance of survival with current treatments and will be offered the opportunity to enrol on the most appropriate clinical study from the outset.

    Risk

    Timescales - the project is dependent on the timely rollout of Panel Sequencing at all UK centres
    Mitigation – delay start of project
    Data access – If access to patient data is blocked the project cannot proceed
    Mitigation – Ensure access to all relevant patient data is approved before project start
    Costs - Additional costs may be needed if additional sequencing is required.
    Mitigation - Create contingency for additional sequencing if advised by Newcastle University

    Reporting

    Updates will be available through the Christopher's Smile website in the research page
    www.christopherssmile.org.uk/research

  • Budget

    Budget - Project Cost: £120,000

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      Amount Heading Description
      £120,000 4 year PhD 4 year PhD student at Newcastle University

    Current Funding / Pledges

    Source Amount
    Christopher's Smile £100,000 Guaranteed
  • Background

    Location

    The project will be undertaken at Newcastle University

    Beneficiaries

    Children with Medulloblastoma who currently receive the worst prognosis.

  • Why Us?

    Why Us?

    Our charity has championed the development and roll out of Next Generation Sequencing (NGS) for children with cancer. Christopher's Smile has invested over £400,000 in NGS for children with cancer to date. As the major stakeholder in this area we want to see the investment benefiting as many children as soon as practicably possible.

    Read more about the Charity running this project.

    People

    Professor Steven Clifford

    PhD student supervisor

    PhD Student

    Once approved a PhD student will be selected using standard Newcastle University selection procedures