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7 September 2015, 07:17
A charity set up to support children with deadly brain disease has received an anonymous £300,000 donation to help fund clinical trials.
The money was pledged through an online crowd-funding page set up by Glasgow-based Funding Neuro to raise £900,000 to support treatment for 18 children with diffuse intrinsic pontine glioma (DIPG) brain tumours.
The charity, which was set up to support ground-breaking work by Bristol-based neurosurgeon Professor Steven Gill, said it was "blown away by the donation''.
Prof Gill works with a "robotic treatment'' where drugs are injected directly into the brain to overcome the need to penetrate the blood-brain barrier that can hamper traditional treatments of Alzheimer's, Parkinson's disease and brain tumours.
More than £600,000 has been raised for the trials so far.
Funding Neuro chief executive Sharon Kane said: "We were absolutely blown away when we received the donation. It's very unusual for anyone to make such a substantial one-off contribution.
"It's certainly by far the biggest single amount Funding Neuro has ever received. There was only one caveat to the donation, that it remain anonymous.
"The response to our crowd-funding drive has simply been phenomenal. Not only have members of the public been extremely generous in terms of financial support, they've also lent their valuable time and effort to help fund-raise on our behalf.
"We're a relatively small charity in terms of size and profile, but we believe the work Funding Neuro supports could have a hugely positive impact on the lives of millions of people.''
DIPG is said to be one of the most resistant of all cancers to chemotherapy treatment and symptoms include double vision, dropping of one side of the face and difficulty chewing and swallowing.
With few treatments available, symptoms usually worsen rapidly in children as the tumour grows.
Ms Kane said two young boys who are currently undergoing treatment from Prof Gill are responding well.
She said: "DIPG tumours are rare in the UK, with around 30 to 40 children diagnosed each year. The reality is they are normally a death sentence for those children.
"Professor Gill is currently treating a small number of children on compassionate grounds, giving these children a chance of survival when previously there would have been no hope, and providing much valued information to further develop the treatment.
"Two of the children currently being treated by the Professor were given just a few months to live when originally diagnosed.
"Following treatment, we're not only seeing their life expectancy increasing dramatically, their quality of life is also significantly improved.''
One of Dr Gill's current patients, 12-year-old Esten Pedersen Indset from Norway, has returned to school after five rounds of treatment.
His father Carl said: "It is now one year since Esten was diagnosed. The diagnosis came as a shock to us then, and the reality only got worse as we came to learn how horrible this disease really is.
"We do realise how fortunate Esten is to be accepted for the treatment in Bristol. The journey has been hard, but also filled with renewed hope.''
The appeal was launched after Prof Gill failed to secure NHS funding.
Speaking at the start of the appeal in June, he said he understood that the NHS was "not a bottomless pit''.
He said: "In this particular disorder, nothing that people have done has made any difference at all, including chemotherapy and radiotherapy.
"On the other hand, if we can crack this one - which is the hardest type of tumour to treat - that spins out into treating a whole range of other different things.''