PNET5 trial: transforming treatments for childhood brain tumours
- Official title: SIOP-PNET5-MB: An international prospective study on clinically standard-risk medulloblastoma in children older than 3 to 5 years with low-risk biological profile or average-risk biological profile - Molecular Testing
- Lead researcher: Professor Steve Clifford
- Where: Newcastle University
- When: November 2017 - March 2020
- Cost: £280,000
- Research type: Paediatric, Medulloblastoma (High Grade), Clinical Trial
Medulloblastomas are the most common high grade brain tumours in children. The standard treatment for this tumour type is removing the tumour with surgery, followed with chemotherapy and radiotherapy.
However, these treatments are quite aggressive and cause long-term, life-altering disabilities. The PNET5 clinical trial aims to improve the quality of survival of these children by providing kinder and tailored treatments.
Each child on the trial will have their tumour tested for specific biomarkers. A biomarker (or indicator) is any change in a cancer cell that can be analysed. These changes can range from DNA modifications to unique molecules produced by the cancer cell.
The biomarker will then determine if the tumour is standard-risk or low-risk. Children with standard-risk will receive the standard dose of chemotherapy and radiotherapy; whereas, children with low-risk will receive a reduced dose of chemotherapy and radiotherapy.
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, in the UK, that biomarker testing will be routine for children diagnosed with a brain tumour and we hope it will prove how valuable it is and become a standard across all brain tumour types.
What impact will this project have?
Up till now, all children with a medulloblastoma are treated in exactly the same way, even though there are different subtypes of this tumour.
This trial should demonstrate that kinder treatments can be given to those children with lower-risk tumours without risking their survival, but still improving their quality of life.
Alex was the first patient in the UK to join the PNET5 trial for children with medulloblastoma. He had fewer doses of maintenance chemotherapy to try to reduce long-term damage to his health without risking his survival.
A vital part of the PNET5 trial 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.
What's been achieved so far?
There are seven centres in the UK now open and actively recruiting children. These centres are: Birmingham Children's Hospital, Bristol Children's Hospital, Alder Hey Hospital (Liverpool), Great Ormond Street Hospital (London), University College Hospital (UCLH, London), Royal Victoria Infirmary (Newcastle), The Royal Marsden Hospital (London).
So far five children have been successfully screened.
How will my money help?
In the first year of the trial we need to raise £250,000
- £10 will pay for an antibody test to identify how the immune system is responding
- £60 will pay for a genetic test to assess a particular gene that plays a role in cell growth, division and change, called the MYC gene
- £350 will pay for a test called a DNA methylation array to assess the molecular subgroup of a tumour
- £2,000 will pay for a full molecular diagnostic analysis on a single medulloblastoma tumour for one child