Crowd-funding to pay for trials
A British neurosurgeon who developed a "revolutionary" robotic treatment for brain disease has turned to crowd-funding to pay £900,000,000 required for clinical trials.
Professor Steven Gill aims to treat 18 children with diffuse intrinsic pontine glioma (DIPG) brain tumours at the Bristol Royal Hospital for Children.
His micro-catheter system injects drugs directly to the brain to deliver medication where it is needed - bypassing the blood-brain barrier.
The system offers hope to sufferers of brain tumours such as DIPG, as well as those with neurological conditions including Alzheimer's and Parkinson's disease.
Five-year-old Italian boy Guglielmo Grasselini is able to walk for the first time since March 2014 after four cycles of chemotherapy administered through the system.
"We've known for years that the protective membrane which surrounds the brain - the blood-brain barrier - stops anything getting through which the brain doesn't recognise, including many man-made drugs," Prof Gill said.
"It's been hugely frustrating that effective drugs currently being used to treat the most devastating neurological diseases, such as Parkinson's, Alzheimers, cancer and Motor Neurone Disease cannot effectively cross the barrier, until now.
"We have actually turned the traditional obstacle of the blood-brain barrier to our advantage - the fact that the drugs cannot re-cross the barrier, with regards to treating brain tumours means we can administer far greater doses of effective drugs, without causing the toxic side-effects of traditional chemotherapy, such as hair and weight loss and nausea."
Prof Gill said detailed clinical research had been carried out using the micro-catheters to target specific parts of the brain "with incredible accuracy".
The initial trial for the system, called Convection Enhanced Delivery (CED), took place in 2001 and involved five patients with Parkinson's disease.
Results from the trial have been "exceptional", Prof Gill said.
"It's a tremendously exciting development and one which offers hope of real progress against some of the most widespread and devastating illnesses," he added.
The method has now been trialled in children with brain stem tumours, one of the most common causes of cancer death in children.
Prof Gill said children diagnosed with the condition currently have a life expectancy of between seven and nine months, with many dying before they reach 18 months old.
"Crowd-funding seems to be a method to raise this amount of money quickly," he said.
"We have applied for funding but we have not be successful because we are competing with diseases such as breast and prostate cancer.
"It can take up to 18 months to secure funding and we don't have this long to wait."
Around 40 children in the UK and 400 in Europe are diagnosed with DIPG each year.
Currently, children receiving CED are treated on "compassionate" grounds by Prof Gill and his team at University of Bristol and North Bristol NHS Trust, with funds raised privately by their parents and charities.
"To date, because of the experimental nature of the procedures, we have only been able to treat children who have failed to respond to all other currently available treatments and as such, are extremely poorly when we first meet them," Prof Gill added.
The crowd-sourcing drive was launched by charity Funding Neuro, a registered charity based in Scotland to fund research into conditions which affect the brain or spinal column.
Sharon Kane, chief executive of the charity, said: "The micro-catheter technology offers real hope to people suffering some of the worst diseases but we've turned to crowd-funding in an attempt to allow this technology to be developed far more quickly.
"Desperate parents across the world are seeking a cure for children suffering from brain stem tumours and the work being undertaken by Professor Gill and his team could literally be the difference between life and death."
Guglielmo, known as Gughi, was diagnosed with a brain tumour in March 2014 and is the youngest child to receive chemotherapy via Prof Gill's system.
His parents Matteo and Valentina took him around the world after being given a "death sentence" by his diagnosis.
He arrived in Bristol unable to walk unaided and had double vision. He is currently undergoing his fifth round of chemotherapy at the hospital.
Business consultant Mr Grasselini, 40, from Florence, Italy, said his son could now swim, walk, sprint and his tumour was shrinking.
"At the moment, these tumours are a death sentence for the children affected," he added.
"As a parent, I am very happy to think that other children will be able to have the same treatment in the future."
To donate visit https://www.indiegogo.com/projects/revolutionary-treatment-for-deadliest-child-cancer#/story