Drug now available on NHS for mother who spent thousands on son's brain tumour medication

Anila Dhami went to meet the family and hear about what this breakthrough means.

A mother who was spending thousands of pounds a month on medication to treat her son’s brain tumour has spoken of her relief after the drug became available on the NHS.

The combination of dabrafenib and trametinib was approved for NHS use for youngsters with brain tumours in April.

However, Alexander has been accessing the medication privately at a cost of about £3,700 a month – or more than £11,000 for a three-month supply – for the past two-and-a-half years.

The family had turned to crowdfunding to help raise the cash, setting up a gofundme page.

"I know we had to fundraise for it at first, but the fact that it was even there for us to fundraise for, we are very grateful for," said Alexander's Mum, Rhonda Josephs.

Alexander has glioma, the most common type of brain cancer in children and young people, which develops from the glial cells that support the nerve cells of the brain and spinal cord.

The combination of dabrafenib and trametinib was given the green light for NHS use by the National Institute for Health and Care Excellence (NICE) for patients aged one and over with either low-grade or high-grade glioma with the genetic mutation BRAF V600E.

The two drugs work together by blocking the growth signal coming from the mutant BRAF protein and can slow or even stop the tumour from growing.

Mrs Josephs described her son as “perfectly fit and healthy, really strong and really into sports” before his first seizure at age six in September 2020, during the Covid-19 pandemic.

"Mum's sleep with one eye open," explained Ms Josephs.

"It was in the middle of the night and I just hard these noises so I went into this room and he was having a full seizure."

His family called an ambulance, and doctors decided to do an MRI as a precaution.

Doctors initially thought the seizure was a one-off and Alexander was booked in for a routine MRI on his brain, which took place in the November.

"I didn't hear anything and I thought no news must be good news," she continued.

"The week before Christmas he had another seizure, so I took him to A and E, whilst the MRI was flagged as having a brain tumour present, they had kind of lost it in the system."

It was when Alexander returned to the emergency department following another seizure in December 2020, the scan images were recovered and the family were informed he had a brain tumour - two months after the scan.

"The way we found out, in A and E, and Xander was with me - I imagine usually they have all these ways they tell children you've got a brain tumour, but unfortunately we were told there and then," she said.

"Xander is a really bright boy, he was really interested in the human body and knows how important the brain is so it was really hard for him to find out that way."

Medics believed the tumour was slow growing, but Ms Josephs urged them to perform surgery in January 2021.

"I think we met with them three or four days before Christmas. so everything shuts down, I asked for elective surgery so the day after boxing day I phoned up and said I feel strongly if it can come out it should come out," she said.

Weeks later Alexander’s cancer was confirmed as an aggressive glioma which had grown.

Alexander eventually had a 10-hour operation to remove the tumour in March 2021, when it was discovered the cancer had spread to his spine.

"They knew it wasn't behaving like a lower-grade tumour," she continued.

"After they removed it completely, they saw the lesions on the spine, they thought they'd wait to see and aim to remove the lesions on the spine, but within the weeks, he started seizing again and the brain tumour returned within weeks."

His second and third surgeries taking place in June of that year.

"We just tried to stay positive, and keep everyone positive. Just tried to explain things to them in child-friendly ways as we went along," said Ms Josephs.

"We were going through surgeries and then six weeks of daily radiotherapy to his whole head and spine, he was so unwell. He lost weight and lost his hair, he was hospitalised many times for uncontrollable illness because of the harsh treatment."

The medication came as a relief after the family spoke to doctors all around the world.

Mrs Josephs explained: "We researched and researched, I reached out to family friends, looking for trials all around the world and we spoke to doctors all around the world.

"It was actually a doctor in America, a world leader on paediatric brain tumours, and the doctors here in the UK, they all work together.

"They put forward this medication. Whilst it was in a trial the data was really good and they recommended that he take it straight after radio therapy.

"He was the first child in the UK to have it straight after radiotherapy - because Xander's was considered so aggressive, they thought it was life or death and he needed to access is straight away."

The medication wasn't available on the NHS so the family began fundraising.

"We had a really good response, but we didn't get to our target just through that alone and that point, I broke down," said Mrs Josephs.

"It just felt so enormous, the amounts of money and Xander was so unwell, just coping with everything and putting Xander and our other son first. The fundraising felt like an impossible task."

It was thanks to a group of mothers who got behind the family and helped organise events to raise money for the medication. These events included marathons, climbing mountains and jumping out planes.

Alexander's school also did sponsored fun-runs, and sponsored zip lines - something both him and his brother got involved in.

"It was a lot of work, but thankfully I had four amazing women behind me to do it, and when I needed to look after Xander, they just stepped forward. They have children of their own and they help save him," she added.

Medics told the family that Alexander was in remission in January 2024, leaving Mrs Josephs “cautiously optimistic, as he’s still undergoing treatment”.

“They were words we’d waited to hear,” she added.

“Alexander is getting back to his sports and he’s just becoming a normal little boy again. He’s doing remarkably well.

“He’s just getting his life back. I don’t want to say he’s unscathed because he’s not.

“But it could have been so much worse. We’re really grateful for how he’s doing.”

The family now plan to enjoy summer, Mrs Josephs said: “The weather is definitely not going to stop us.”

Cameron Miller, director of strategy and external affairs at the Brain Tumour Charity, said: “Twenty years ago, our research funding helped identify BRAF mutations in brain tumours.

“When a breakthrough came following clinical trials, we worked with the National Institute for Health and Care Excellence to ensure the patient voice was heard.

“Our representatives, including a patient expert, participated in the scoping meeting and provided essential evidence to the committee meeting. So, we are delighted at this outcome.

“Yet Alexander’s struggle underscores the immense challenge of finding cures for this complex and unpredictable group of diseases.

“It’s vital that we continue to pursue these relentlessly and prioritise funding for research into brain tumours to accelerate the development and NHS availability of effective new treatments.”

Mrs Josephs said “cancer doesn’t wait for red tape”, adding: “I do feel children with brain tumours are written off and this shows they shouldn’t be.”

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