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£3m for childhood brain tumour research

Brain tumours account for a quarter of childhood cancer diagnoses and more than a third of childhood cancer deaths. Childhood brain tumours are treated aggressively to give each child the very best chance of survival. But survival comes at a cost, and many young patients are left with devastating long-term and late effects that severely impact on their future health and wellbeing.

Brain Tumour Initiative

Despite the devastating effects of brain tumours, they have not attracted the same level of research investment as research into some other childhood cancers. To counter this, in 2014, we launched our three-year Brain Tumour Initiative, with a pledge to invest at least £3m in new brain tumour research over this period.

In March 2015, we awarded the first major project grants under this call, at a total cost of £1.81m. In December 2016 the second phase of funding was agreed and an additional four projects were awarded at a total cost of£1.24m. This has brought our total expenditure of the Brain Tumour Initiative to over £3m as planned in 2014.

December 2016 – the second phase of funding

Four new projects at a total cost of £1.24m. For this second round we invited research applications which sought to enhance the effectiveness of therapy through improved drug delivery systems or the use of novel technologies. The four projects agreed for funding use distinctly different approaches to deliver treatments of brain tumour patients.

  • Convection-enhanced delivery of N3-propargyl, a novel analogue of temozolomide – Dr Beth Coyle, University of Nottingham:
    The chemotherapy drug (temozolomide) used to treat adult high-grade gliomas are less effective in children as sufficient concentrations of the drug are unable to infiltrate the brain to target the cancerous cells. The aim of this project is to test the effectiveness of a more potent modification of temozolomide delivered directly into the tumour using a technique called convection-enhanced delivery (CED).
  • Precision delivery of chemotherapeutics across the blood-brain barrier for treatment of brain tumour – Professor Giuseppe Battaglia, UCL:
    Professor Battaglia will lead a multidisciplinary team and use nanotechnology to develop an extremely small molecular vesicle loaded with therapeutic drugs. These vesicles are able to access the brain and deliver targeted drugs to eradicate brain tumours. The team will initially test the effectiveness of their nano-carrier with clinically available drugs which will allow the potential impact of their work to be realised in a much shorter timeframe.
  • TCR-transduced T cells for immunotherapy of paediatric high-grade glioma – Professor John Anderson, UCL Great Ormond Street Institute of Child Health:
    In collaboration with the University of California, San Francisco the researchers will use a novel immunotherapeutic approach to target diffuse midline gliomas. The aim of the project is to develop gene-modified T cells which target a protein found specifically on cancerous cells like DIPG. This approach is highly targeted and, if successful, will allow them to investigate the treatment in a clinical trial.
  • Investigation of a harmless prokaryotic virus for intravenous targeting delivery of therapeutic nucleic acids to DIPG – Dr Amin Hajitou, Imperial College London:
    Diffuse intrinsic pontine glioma is a type of brain tumour which is currently incurable. This project aims to use bacteriophages to deliver therapeutic nucleic acids to DIPG cells in a preclinical setting. If successful, this phage guided anticancer therapy can rapidly enter clinical trials and could also be applicable to other brainstem gliomas.

March 2015 – the first phase of funding

Four new projects at a total cost of £1.81m. An important feature of the four funded projects is that they are highly collaborative, with researchers working together to share samples, data and expertise for maximum progress.

  • BIOmarkers of Ependymomas in Children and Adolescents- Professor Richard Grundy, Children’s Brain Tumour Research Centre, University of Nottingham:
    Ependymoma is a devastating brain tumour that kills up to 40 per cent of young patients, with little improvement over recent years. BIOmarkers of Ependymomas in Children and Adolescents (BIOMECA) is a unique partnership between leading European ependymoma specialists who are now collecting tumour samples from young patients being treated under the international ependymoma clinical trial. They are aiming to develop new risk categories and work towards the future personalisation of therapy.
  • Diagnosing childhood medulloblastomas by improved MRI scans – Professor Andrew Peet, University of Birmingham:
    Medulloblastomas are the most common childhood malignant brain tumours and only around 60 per cent of young patients survive. Different medulloblastoma subtypes respond differently to treatment and rapid diagnosis is essential to aid treatment planning. Professor Peet is working with colleagues from other UK centres to develop a new tool to allow rapid, non-invasive diagnosis that can inform critical initial treatment decisions.
  • Understanding the genetics of paediatric glioblastoma – Dr Steven Pollard, University of Edinburgh:
    Paediatric glioblastoma is a devastating brain tumour that less than 20 per cent of young patients survive. This collaborative project brings together research teams from the UK, Canada and Sweden to define the role of a recently discovered genetic mutation in paediatric glioblastoma and determine whether it represents a possible target for a new therapeutic approach. The team will also create new cellular models of the disease for on-going drug-discovery efforts.
  • New tools to improve treatment and care in paediatric craniopharyngioma – Dr Juan Pedro Martinez-Barbera, UCL Institute of Child Health:
    Paediatric craniopharyngioma is a brain tumour that has high survival but can leave survivors with very poor quality of life. New, targeted treatments are urgently needed but progress is hampered by our poor understanding of the biology of these tumours. This collaborative project brings together teams from the UK and Germany to help profile these tumours and work towards the development of improved treatments.