PNET5 clinical trial: transforming treatments for childhood brain tumours
The Brain Tumour Charity has committed nearly £1 million to PNET5 – a vital clinical trial studying improved treatments for children diagnosed with medulloblastoma. Now we need your help to ensure that every child in the UK diagnosed with a medulloblastoma has the chance to be part of it.
Medulloblastoma is the most common paediatric brain tumour but treatments are aggressive and can leave long-term, life-altering disabilities for children. It's a terrible burden for children to bear for the rest of their lives.
But, with PNET5, we're on the verge of a major breakthrough for medulloblastoma treatment.
PNET5 aims to prove that children who are diagnosed with a lower risk medulloblastoma would benefit from less aggressive treatment regimens, and that those with standard risk medulloblastoma could benefit from a different treatment regimen resulting in fewer side effects, without any negative impact on length of survival.
Kinder, less aggressive treatments will drastically improve quality of life for these children; reducing the time spent unwell from treatment, reducing time in hospital and, critically, reducing the risk of long-term disabilities.
However, research and clinical trials are expensive – we need to raise £250,000 to fund the first year of the UK trial centre, ensure biomarker testing for every child and fund the quality of life study within the trial. And in total, over the seven year trial, we need to raise over £870,000. That's why, whether you can spare £2 a month or are able to make a one off donation of £200, every pound is important to us – it all adds up.
In order to establish whether a child has a lower or standard risk medulloblastoma, The Brain Tumour Charity has committed to pay for all children diagnosed with a medulloblastoma in the UK to receive biomarker testing. This is the first time biomarker testing will be routine for those diagnosed with a brain tumour and we hope it will prove how valuable it is, with a view that biomarker testing becomes standard across all brain tumour types.
You can help transform treatments for children
With your support, we'll make sure that every child in the UK who's been diagnosed with a medulloblastoma gets a biomarker test, so they can get the best treatment for them.
You can make a one-off gift or set up a regular donation to provide ongoing support.
The biomarker tests, developed by Professor Steve Clifford, will identify which subtype of medulloblastoma each child has. Those classified as standard or lower risk will be eligible to join the PNET5 trial and benefit from a more tailored, appropriate treatment level.
How has The Brain Tumour Charity helped us get to PNET5 and this important trial?
We’ve funded Professor Clifford since 2001. His long-term research into medulloblastoma has identified seven sub-types of medulloblastoma, which have different diagnostic markers, behaviours and prognosis. PNET5 is using this information to test kinder, better and more tailored treatments.
A vital part of PNET5 is the quality of life and quality of survival assessment which was developed by Professor Kennedy – a researcher we’ve long supported and whose achievements in the quality of life field have been widely acknowledged as important progress.
“We're pioneering the development and introduction of advanced molecular diagnostics and, with the support of The Brain Tumour Charity, we're the first country to offer routine biomarker testing for all children diagnosed with medulloblastoma, leading the way for more tailored, effective treatments." Professor Clifford
In the first year of the trial we need to raise £250,000
Research and clinical trials are expensive – the biomarker testing in PNET5 alone costs £2,000 per child. That's why, whether you can spare £2, £20 or £200, every pound is important to us – it all adds up.
We're raising money to fund three elements of the PNET5 clinical trial: the UK trial centre, the biomarker testing, and the quality of life assessment which is integrated into the trial. To fund this, in year one of the trial, we need to raise £250,000.
To give you an idea of how your donation to the PNET5 clinical trial could be spent, here's a breakdown of some of the costs for biomarker testing – a series of tests that allow scientists to accurately diagnosis the type of medulloblastoma each child has:
£10 will pay for an antibody test (to identify how the immune system is responding)
£60 will pay for a genetic test to assess the MYC gene (a gene that plays a role in cell growth, division and change)
£350 will pay for a DNA methylation array (a test) to assess the molecular subgroup of a tumour
£500 will pay for the mutation status of critical medulloblastoma genes to be assessed
£2,000 will pay for a full molecular diagnostic analysis on a single medulloblastoma tumour
I was delighted The Charity was able to fund the UK part of this European trial. It is a major step forward in our strategy to halve the harm. Having successfully identified the low risk medullablastoma group it is vital we treat the patients accordingly and save them from long term side effects caused by the traditional intense treatment. This trial means a lot, as we originally funded the basic science in the mid 2000s.To witness it come full circle, from "bench to bedside", is a very proud moment for us and illustrates what a difference The Charity is making in translating basic research into new treatments.
Neil Dickson MBE
Please donate whatever you can to the PNET5 trial today or set up a regular donation to provide ongoing support. Together, we'll make sure that every child diagnosed with a medulloblastoma has a brighter future.