Mum of Leeds boy Toby Nye speaks out over children's cancer drug decision

CANCER battle Leeds youngster Toby Nye's mother today said more children fighting the disease  will be put at risk of relapsing after health chiefs ruled a drug Toby is being treated with will not be made available on the NHS.

Toby Nye with Leeds United captain Liam Cooper

Five-year-old Leeds United fan Toby, of Osmondthorpe, is battling rare childhood cancer neuroblastoma and is currently undergoing specialist £200,000 antibody therapy treatment not previously routinely available on the NHS after Leeds United and YEP readers backed a massive fundraising drive to pay for it.

His mother Stacey Worsley said the antibody therapy treatment with drug dinutuximab beta, which stared in January, has cleared cancer from his bone marrow for the first time since he was diagnosed with neuroblastoma on his fourth birthday in January 2017.

Sign up to our daily newsletter

The i newsletter cut through the noise

The National Institute for Health and Care Excellence (Nice) has said it would like to recommend dinutuximab beta for inclusion in the Cancer Drugs Fund but it is currently too expensive to do so and there remains "substantial uncertainty" about its long-term benefits.

Toby Nye with mum Stacey Worsley

Miss Worsley said all children who need the treatment should get it free on the NHS and families should not have to go through the stress of fundraising for vital treatment

Charities that support children with high-risk neuroblastoma - a cancer of the nerve tissue - said the decision was "immoral" and families will now have to try to raise thousands of pounds to send their children for the treatment overseas.

Miss Worsley said Toby was originally on a trial for free dinutuximab beta treatment on the NHS, but he needed additional treatment and had to come off the trial.

It meant his family had no choice but to launch a fundraising drive to raise the £200,000 needed to pay for the treatment he is currently undergoing at Leeds General Infirmary.;

Miss Worsley said: "I think this treatment should be available on the NHS. They are going to be putting more children at risk of their cancer getting worse and relapsing.

"I believe every child who needs it should have free access to this drug. Families should not have to go through the struggles that we have had to go through to raise the money to pay for it.

"It has been hard work trying to look after a poorly child and the rest of your family and keep your house and work in between. Having to fundraise takes your focus away from your child.

"The treatment obviously works with Toby. The cancer was still in his bone marrow before he started the antibody therapy and now his bone marrow is clear.

"We were lucky we were able to raise the money because other families haven't been able to."

Miss Worsley said previous chemotherapy, radiotherapy and stem cell treatment had failed to clear the cancer from Toby's bone marrow.

Miss Worsley said the antibody therapy treatment is due to finish at the end of June and the family will not know of Toby is cancer free or not until July.

She said Toby has more energy now after undergoing the antibody therapy treatment.

Toby underwent an operation to remove a cancerous tumour on one of his kidneys last September.

There was a small amount of tumour that could not be removed safely but latest tests show there is no sign of the tumour re-growing.

Stephen Richards, chief executive of Solving Kids' Cancer, said: "Anti-GD2 antibody therapy given at the end of standard therapy represents the best chance of long-term survival for children diagnosed with high-risk neuroblastoma.

"It is recognised as standard of care by clinicians internationally.

"This is devastating for families - it takes away hope in the fight against high-risk neuroblastoma, and it's immoral to deny children the chance of an essential treatment that could enable them to live longer or cancer-free.

"When we know that there is an effective drug available, it's immensely frustrating for parents to be entangled within a system that leaves them no choice but to raise hundreds of thousands of pounds for their children to receive this treatment overseas.

"Having the extreme pressure of fundraising plus the anxiety and logistics of travelling abroad for treatment puts an enormous emotional and practical strain on families."

Neuroblastoma develops from specialised nerve cells (neuroblasts) left behind from a baby's development in the womb and affects around 100 children each year in the UK, most commonly under the age of five.

Mr Richards was also speaking on behalf of the charities Neuroblastoma UK, the Children's Cancer and Leukaemia group, JACK, The Bradley Lowery Foundation, Hugs from Henry, Christopher's Smile, Niamh's Next Steps and Smile with Siddy.

Football mascot Bradley Lowery, six, from Blackhall, County Durham, died in July last year after being diagnosed with neuroblastoma when he was 18 months old.

The Sunderland fan was a mascot for his beloved club along with Everton and England as he battled the condition.

Nice said it was estimated dinutuximab beta would cost the NHS between £62,300 and £79,900 per quality-adjusted life year gained - higher than what is normally considered to be a cost-effective use of resources, which is between £20,000 and £30,000 per quality-adjusted life year gained.

Mr Richards added: "Nice has been directed to work within an appraisal route that is based entirely on complex health economics.

"This doesn't take into account the nature of the disease and any of the unique contexts, challenges and considerations that exist within rare children's cancers.

"Instead, a drug that saves the lives of some children and extends the lives of others has been appraised against metrics designed to evaluate treatments for chronic conditions affecting adults. The system's one-size fits all approach is simply not fit for purpose.

"We owe it to these vulnerable children to give them the best chance of survival, and we will do everything in our power to keep fighting for these families."

He said the decision leaves the UK lagging behind the United States and other European countries.

Meindert Boysen, director of the Nice Centre for Health Technology Evaluation, said: "Dinutuximab beta shows a lot of promise, but the evidence is uncertain and we must acknowledge this.

"There is opportunity for the company to collect longer-term data from the ongoing trials. This could make dinutuximab beta a candidate for inclusion in the Cancer Drugs Fund.

"However, the company needs to demonstrate that the drug has at least the potential to be cost-effective before we can consider recommending dinutuximab beta be included in the CDF.

"As such, we are keen to work with the company and NHS England to help them explore options."