Give them back their childhood: Funding plea for pioneering treatment of rare brain tumour that no child has survived
- Most die within months of diagnosis of intrinsic pontine glioma (DIPG)
- Innovative treatment targets tumour directly rather than intravenously
- New technique also means there is no risk of side effects like hair loss
- Charities are urging the public to help fund trial which costs £900,000
A ground-breaking treatment is giving hope to the families of children suffering from a rare and deadly brain tumour.
No child has ever survived diffuse intrinsic pontine glioma (DIPG), with most dying within a few months of diagnosis after suffering rapidly worsening symptoms of sight and speech loss, then paralysis.
But there is new hope in an innovative technique that delivers drugs directly to the tumour, rather than intravenously, using micro-catheters – tiny tubes – placed surgically into the brain.
Ground-breaking: The new technique delivers drugs directly to the tumour. Pictured, how the treatment works
This method bypasses the membrane ‘wall’ known as the blood-brain barrier, which stops intravenous drugs passing from the bloodstream.
And targeting the tumour directly without damaging healthy cells means there is a reduced risk of side effects such as hair loss and nausea.
But at £35,000 a time, the operation does not come cheap. Neurosurgeon Professor Steven Gill, of the Bristol Royal Hospital for Children, who is using the technique, is relying on the generosity of the public to fund a clinical trial.
A charity, Funding Neuro, has raised £680,000 of a £900,000 online target for the trial – with £300,000 being given by a single mystery donor in September.
The push has been backed by pop star Emeli Sandé, who performed at a gala event to further boost the coffers.
One of Prof Gill’s first trialists was five-year-old Gughi Grasselini, who has been given a second chance at his childhood thanks to the procedure.
Once unable to walk and given just weeks to live, Italian-born Gughi’s tumour has shrunk dramatically since treatment began, and he is walking again. Though weak, he is even well enough to go swimming.
Brain cancer is notoriously under-funded compared with other cancers. Figures from charity Brain Tumour Research reveal that of the £4.5 billion spent on UK cancer research from 2002 to 2012, just £35 million – less than one per cent – was spent on brain tumour research.
Sue Farrington Smith, chief executive of Brain Tumour Research, lost her seven-year-old niece Alison to DIPG in 2001.
‘Brain-tumour research is currently about 20 years behind other cancers,’ she says. ‘The fact that we simply don’t know what causes brain tumours is shocking.
‘Research relies heavily on the generosity of the general public. So it’s not surprising charities such as Funding Neuro are resorting to crowd-funding methods to raise funds for such an important trial.’
About 40 UK children are diagnosed with DIPG brain tumours every year. ‘It is the most horrendous death,’ says Funding Neuro chief executive Sharon Kane.
Keira Wrenn, of Lincolnshire, pictured, was the first British child to use the ground-breaking tumour treatment
‘They are trapped inside their bodies, but completely aware of everything that is happening to them.’
Until now, radiotherapy, which shrinks the tumour temporarily, has been the only treatment available. Chemotherapy is ineffective due to the blood-brain barrier.
Prof Gill and his team will be using the pioneering system called convection enhanced delivery (CED), and the trial will involve 18 children.
First, the child has a detailed MRI scan of the brain to make a model of the tumour and plan where to position the tubes to deliver the chemotherapy.
Then, under general anaesthetic, an incision is made in the scalp and tiny 1mm-wide plastic ‘guide tubes’ are implanted by a neurosurgical robot.
The treatment tubes are fed through these and connected to a titanium ‘port’ the size of a small button inserted into the skull behind an ear. The port acts as an entry for drugs to be administered.
The technique uses the blood-brain barrier to the doctors’ advantage, as it also serves to keep the drug in the brain.
Procedure: Keira, seven, pictured in hospital, went through a 13-hour operation during the treatment in October
‘The drugs do not get into the blood system so you don’t get any of the side-effects you might associate with chemotherapy such as hair loss and nausea, and it carries on working for several weeks,’ says Prof Gill.
Operations carried out so far have been funded through charitable support and royalties from Prof Gill’s surgeries.
Keira Wrenn, a seven-year-old from Lincolnshire, is the first UK child to have the treatment. She was diagnosed with a DIPG tumour in August 2014 after she collapsed while swimming on holiday.
Radiotherapy shrank her tumour to half the size, but Keira remained terminally ill.
Her parents read about Prof Gill’s work online and asked that she be considered for the innovative treatment.
Keira underwent a 13-hour operation to fit the port and administer the chemotherapy drugs in October, and now has chemotherapy every six weeks.
‘It’s still very early days, but we have hope now while before we had none,’ says her mother Emma, 31.
- Donate at fundingneuro.com
Read more: http://www.dailymail.co.uk/health/article-3391695/Give-childhood-Funding-plea-pioneering-treatment-rare-brain-tumour-no-child-survived.html#ixzz3yBZK4Tal
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