Boy, 11, denied life-extending drug as his incurable ‘octopus tumour’ is 1 MILLIMETRE too small
A SCHOOLBOY has been denied life-extending treatment because his incurable brain tumour is one millimetre too small, his heartbroken parents have said.
Ronnie Hood, 11, was given just months to live after being diagnosed with a diffuse midline glioma in March.
He underwent chemotherapy and radiotherapy, but this only helped to shrink his tumour very slightly.
It was, however, a big enough reduction that he no longer qualifies for ONC201 – an experimental drug being trialled for serious brain tumours in children and young adults.
Dad Nick told the BBC: “We got the news it had shrunk, which is obviously very good news, but the double-edged sword was that we are now not eligible for the drug.
“If there is a change, then we will be eligible, but in the meantime we are sourcing it ourselves.
“It costs about £700 per pill, or it would be about £4,000 a month.”
The family have set up a GoFundMe page to help pay for the private treatment.
So far, they have raised more than £30,000.
Mum Vicky said: “We can’t thank everyone enough.”
Ronnie, from Sudbury, Suffolk, had been experiencing unbearable neck pain and tingling in his fingers for about a year.
His parents took him to their GP but claim the doctor simply ordered physio – saying the issue was due to the gamer “playing on his phone too much”.
When his symptoms didn’t improve, and physio exercises left him “crying in pain”, Nick, 58, and Vicky, 50, pushed for an MRI scan.
Ronnie was finally examined in February – and his results revealed a tumour growing “like an octopus” on his brain stem near the top of his spine.
Nick said that finding out his little boy had brain cancer was like “being hit by a bus”.
“Ronnie wasn’t getting any better. He cried in pain and frustration and then had a numbness in his right hand,” he said.
“When we got the results of the MRI, we couldn’t believe the news that he had brain cancer. It was like we had been hit by a bus.
“He’s a handsome, confident, and well-loved little boy who was fit and healthy. How could this be happening?
“Ronnie has gone from bouncing off the walls, riding his bike, doing art and going down the park with his friends to not being able to use his hands.
“I have to dress him, feed him and shower him. He can do hardly anything now and so it is horrific to watch him.”
We don’t want to sit around and wait for this to take our boy
Nick
It is so difficult for his parents that they are doing anything they can to improve his chances.
Nick, who runs a courier company, added: “Ronnie has a rare mutation of this type of tumour which means there aren’t many options for treatment here in the UK.
“There is only so much radiation that he can have, and the oncologist said chemo is useless in his case.
“Currently, for Ronnie to be eligible for any other treatment, the tumour needs to grow before anyone will do anything.
“This seems counterproductive. We are trying to be proactive in prolonging Ronnie’s life.
“We don’t want to sit around and wait for this to take our boy.”
What is a diffuse midline glioma?
DIFFUSE midline gliomas are the second most common type of primary high grade brain tumour in children.
They grow in the midline between the two halves of the brain.
Symptoms can be vague, but can include:
- Headaches
- Nausea
- Fatigue
- Problems with walking, coordination, balance
- Weakness in the arms and legs
- Difficulty controlling facial expressions
- Speech problems
- Issues with swallowing and chewing
- Double vision
A patient’s prognosis will vary depending on the tumour and how they react to treatment, but the average life expectancy after diagnosis is less than a year – usually between eight and 11 months.
About 10 per cent of people survive at least two years, and only two per cent live beyond five years.
Diffuse midline gliomas affect around 25 children every year in the UK.
Source: The Brain Tumour Charity
Nick said the family is “touched” by the generosity people have shown to the fundraiser so far.
“We appreciate any other contributions as there is still a way to go,” the dad added.
Ronnie, who is unable to return to school, has been getting more uncomfortable each day.
He is on a high dosage of steroids in order to reduce the swelling around the tumour – but one of the major side effects is that his face and tummy have “blown up”.
Relative Katy-Jade Church, who set up the fundraising page, said: “At an age where he is starting to become particularly conscious of his looks, Ronnie is finding these side effects particularly distressing and is increasingly not wanting to see friends or leave the house.
“The steroids have also affected his ability to sleep and made him understandably irritable.
“And all of this has happened at a time where Ronnie’s life is just getting started.”
Brain tumours kill more children and adults under the age of 40 than any other cancer – but just one per cent of the national spend on cancer research has been allocated to them since records began in 2002.
Vicky, Nick, and Ronnie’s four older sisters have all been shocked with the lack of investment and research into the devastating disease.
Nick said: “In the months since Ronnie was diagnosed with DIPG, his appearance has changed drastically.
“Although he can walk and talk, it’s becoming more and more difficult, and in March, the overwhelming side effects of the tumour and medication meant he stopped going to school.
“Nobody should have to face this devastation because of brain tumours, let alone a child.
“We feel like we are clinging on to any hope which has been met with a lack of investment into research into the disease.”
‘BRAIN TUMOURS CAN AFFECT ANYONE’
Charlie Allsebrook, community development manager at Brain Tumour Research, said: “It’s heartbreaking to learn of Ronnie’s diagnosis.
“It’s a stark reminder that brain tumours are indiscriminate; they can affect anyone at any age.
“It’s horrendous that patients and their families feel forced to take treatment into their own hands.
“If we are to find kinder treatments and eventually a cure for all types of brain tumours, we must invest in research in the UK to improve outcomes so parents such as Nick and Vicky don’t face the reality of losing a child to a brain tumour.”
Brain tumours are the most deadly form of cancer for children and young people.
Around 16,000 people are diagnosed every year in the UK. Just 12 per cent survive beyond five years.
There have been no new treatments in almost 50 years.
Brain Tumour Research funds sustainable research at dedicated centres in the UK and campaigns for the government and larger cancer charities to invest more in research, treatments, and finding a cure.
A spokesperson for Cambridge University Hospitals NHS Foundation Trust, which is handling Ronnie’s treatment, said: “While we have every sympathy for Ronnie and his family, and have approached the pharmaceutical company on their behalf, it is important to understand this drug is only available through a managed access programme directly from the pharmaceutical company by meeting their eligibility criteria.
“We would like to reassure patients that this Trust is dedicated to providing the very best levels of care.”
What is ONC201?
ONC201, also called dordaviprone, is an experimental drug to treat serious brain tumours in children and young adults.
This includes diffuse midline glioma, including DIPG, which has no approved treatments.
People with this type of tumour usually don’t live more than nine months.
In small trials, ONC201 has at least doubled expected survival time and been shown to have few safety-related side effects.
The drug is still at clinical trial stage, so it is not available on the NHS.
Only once there is enough clinical trial data will there be an assessment to see if the drug should be approved for use on the NHS.
The trial is recruiting in 12 centres in the UK, but as with all clinical trials, it has eligibility criteria for people to enrol.
Worldwide, 141 sites are trialling ONC201 to determine how effective the treatment is for newly-diagnosed children with diffuse glioma.
There are very few clinical trials for those diagnosed with a brain tumour.
ONC201 is generally well tolerated in children, and it has been shown to reduce the size of some brain tumours but more evidence is needed.
Dr Michele Afif, chief executive of The Brain Tumour Charity, said: “It is hugely encouraging to see so many UK sites participating in the ONC201 ACTION study so that as many eligible patients as possible can access this potential new treatment, despite the barriers associated with opening brain tumour specific clinical trials in the UK.
“On a personal level, it is heartbreaking to know that people affected by a brain tumour diagnosis all too often still find themselves in the terrible situation of finding there is no alternative but to privately fund potential treatments for this devastating disease.
“It’s vital that we work together with clinicians, drug manufacturers and regulatory bodies to ensure kinder, more effective treatments reach patients so that those affected by a brain tumour diagnosis live longer, better lives.”
Source: The Brain Tumour Charity, Chimerix and Musella Foundation for Brain Tumour Research & Information