Parents in desperate battle to secure drugs to save their children’s lives

Ollie, 14, and Amelia Carroll, 11, are among 36 children in the UK receiving treatment for the heartbreaking neurodegenerative Batten disease, also known as CLN2.

NHS England must decide before May whether to agree to the funding of an enzyme therapy drug called Brinuera – which slows the cruel progress of the aggressive disease.

It causes seizures and dementia, robbing children of their sight, mobility, ability to talk and swallow, and the average lifespan is between six and 12 years.

Brave Ollie first hit the headlines in 2016 when he mustered all his strength to stand and hug Prince Harry at the Wellchild Awards ceremony.

The pair met again a few months later when Prince Harry paid a surprise visit to him and Amelia as they received treatment in Great Ormond Street Children’s hospital.

Their fortnightly brain fusions of enzyme therapy have been preventing the deadly disease from ravaging their young bodies as quickly as it would without treatment.

But now the Carrolls, whose plight has been repeatedly publicised by the Express, fear they will be forced to watch their children succumb to the disease and, ultimately, a painful death. Desperately hoping further funding will be granted, parents Lucy, 39, and builder Mike, 40, have been left fighting to prove that their children’s lives are each worth the £520,000 a year the drug costs per child.

Lucy said: “It’s hard because you’re being asked questions about your child’s quality of life, and you’re having to prove that your child has a right to live.

“For example, with Ollie, they are asking a lot of questions about children in wheelchairs who can’t walk and can’t talk and want to know what they are still able to do.

“I feel judged that I’m trying to explain that just because he can’t walk and can’t talk, it doesn’t mean that he can’t experience things and doesn’t have a good quality of life.

“He can’t talk but he communicates in his own way. A lot of it is facial expressions; he laughs, he smiles. If he doesn’t want to do something he’ll roll his eyes.”

Lucy and Mike, from Poynton, Cheshire, are also parents to Danny, 17, Micky, 16, and five-year-old Renesmee.

The decision on whether to continue funding the drug rests in the lap of a panel at NHS England which is currently in commercial discussions with US pharmaceutical giant BioMarin, which makes the drugs.

NICE, the National Institute for Healthcare and Excellence, has twice refused funding for the drug but Lucy and Mike were instrumental in challenging the decision and won a Supreme Court battle.

In 2019 the drug was granted under a five-year Managed Access Agreement, which ran out in November 2024, with a six-month extension being given.

“The worry is that if they can’t negotiate the cost down, they’ll try to cut costs by taking some children off the treatment,” says Lucy.

“I feel like they’re on the cusp of discriminating against the children who can’t walk and talk. How can you say that one child’s life is worth more than another?”

She insists, even though non-verbal, her son does have the ability to communicate his needs to his family and carers.

“He was working with his teacher earlier and he really doesn’t like messy play, so he pulled his hands away,” adds Lucy.

“Ollie tells us whether he’s happy or sad, he still has an understanding of what he likes and dislikes. He’s still having an education, teachers come out to the house and interact with him, he still goes swimming every week and has hydrotherapy, and he can still go on holiday.”

Ollie and Amelia were previously granted treatment by the pharmaceutical company on compassionate grounds.

A ruling could come any day now.

And the parents of the 40 children in the UK and Northern Ireland are praying their families are not about to be ripped apart.

Lucy added: “The treatment provided under the current five-year Managed Access Agreement is life changing.

“It has proven to slow the progression of the disease, and it sustains a good quality of life for the child and the family.

“If you stopped the treatment the disease would take over the child. Before treatment began when he was five, Ollie had up to 50 seizures a day. They would return.

“For Ollie this would mean his health would quickly decline and his organs would start to lose function, resulting eventually in a painful death.

“Amelia started the treatment at a younger age than Ollie, so for her it would mean she would lose the ability to walk independently and play.

“Her mobility would be lost, her muscles, her speech would go and so would her ability to eat and drink orally.”

Both youngsters would end up bed-ridden, needing 24-hour care and moving towards an uncomfortable and frightening premature death.

“Seeing the child’s life taken away like this, knowing that treatment is available but has been deemed unaffordable to the NHS, would be very distressing for the whole family and impact significantly on mental health and wellbeing.

“We can just hope they agree to fund the treatment, and if they don’t, we will fight it and appeal.”

Amelia, who has lost her sight two years ago, is in full time education at a special school which she adores.

Lucy said: “She has wonderful friendships both inside and outside of school. She has always had a love of gymnastics and is a current international gymnastics champion in her class.

“She can attend gymnastics sessions weekly thanks to the dedication and support of her coaches at CC Gymnastics in Poynton.

“She is still able to speak in short sentences as well as eat orally, something that would never have been possible without this treatment.”

The brain infusions do not target the eyes, so in 2021 doctors started a pioneering trial to inject Brinuera directly into patient’s eyes.

And while it was too late for Amelia, the treatment has prevented sight loss in other

children. Lucy has written to health secretary Wes Streeting to ask that funding for both uses of the drug is considered at the same time.

A drop-in event for MPs has been set up by Lord Ian Botham and his daughter Sarah Botham, long-time supporters of the Batten Disease Family Association, in the Houses of Parliament on March 12 from 3.15pm until 5.30pm.

Families will be gathering outside Parliament before the event at 1pm to raise awareness.

They plan to wear special T-shirts and wave banners to raise awareness of their campaign to access treatment.