Baby given months to live may now outlive parents - after receiving world’s most expensive drug

A baby given just months to live after being born with a rare genetic disorder is now expected to outlive his parents - after he received the world’s most expensive drug.

One-year-old Theo Greenhall has spinal muscular atrophy (SMA), which restricted his breathing, caused his limbs to become floppy and prevented him from swallowing.

His parents, Natalie and Sean, both 33, found out he had the most severe form of the illness soon after he was born, at which point he was given less than two years to live.

Last March, the drug Zolgensma became available to SMA sufferers aged under one in the UK, giving the family hope.

Zolgensma, which has a list price of £1.795 million per single dose, was made available after the NHS struck a deal with US-based manufacturer Novartis Gene Therapies.

The one-off infusion, given in just an hour, uses a harmless virus to deliver a healthy version of the SMN1 gene, which is defective in those with spinal muscular atrophy.

While Theo qualified for the drug in July, doctors later discussed pulling his treatment when he was admitted to hospital for six months with breathing problems.

His nervous parents were relieved when his symptoms eased just in time and he got the critical infusion in November.

Since then, Natalie said that Theo’s mobility has increased dramatically - and the couple are now hopeful that he will enjoy a long, happy life with them.

‘We always said if we could give our lives for Theo, we would’

Natalie, who is Theo’s full-time carer, said: “We were told it was terminal, he’d never sit, he’d never stand or walk, and now we’re seeing a few of those boundaries being broken.”

“We always said if we could give our lives for Theo, we would, and effectively, if we had the money, straight away we’d pay for it. In this day and age, we’re just so grateful for it.

“We’re just hopeful that he will outlive us as parents, and that we’ll have a better quality than families that have had SMA children in the past.”

Natalie, from Warrington, first found out the tot may be unwell when a midwife noticed he was “quite floppy” in the days after he was born, on November 3, 2021.

Just over three weeks later, she and her husband were called to Alder Hey Children’s Hospital, in Liverpool, where medical experts told them that their child has SMA.

Natalie said: “They basically had to give us the news that Leo had got one of the most fatal, most horrible diseases, and his life expectancy is less than 18 months.

“In that moment, I was quite emotional. I couldn’t even speak. My husband didn’t really process it there and then. He asked questions about it.”

The moment both parents were told that the NHS would fund £1.79m drug

Theo was first put on a drug called Spinraza, requiring him to have “traumatic” monthly spinal injections, which doctors hoped would stabilise and improve his condition.

But early one morning in March 2021, Natalie awoke to find that the NHS would be funding a new £1.79m drug, which could have life-changing effects for kids like Theo.

She said: “We were back in hospital for a sleep study, and at 6 am, I was awake, and I looked at the news, and I was like “Oh my life! The gene therapy has been approved in England.”

“I had nobody to talk to – no nurses were around, and my husband wasn’t there – but I was like, ‘This is so exciting!’”

Theo was among the first babies to become eligible for the drug, called Zolgensma, after getting his doctor’s approval in July last year.

But when he entered intensive care on July 4, medics held back and set a deadline for his health to improve by his first birthday, on November 3, in order to qualify for it.

Natalie said: “In intensive care, he couldn’t breath for himself, and he had to be off the ventilator for at least 18 hours [to get the drug].”

“So from July to October, each day we took him off it for 20 mins, an hour, and by October, he was just on it overnight again.”

As the deadline approached, Natalie said doctors were “surprised” to see his progress and gave him the all-clear to get the medicine in the same week as he would turn one.

Natalie said she and her husband were incredibly grateful that he was selected for the new treatment given its incredibly hefty price tag.

Natalie said: “The treatment is a one-off infusion, which blows my mind because it’s nearly £2 million and it was one hour. And that was it!”

“For that amount of money to be spent on our child, and other children in the same position, it’s just incredible.”

“We’re grateful for that money and for the NHS, but for us as a family, we don’t see the money – we see the benefit it has on our child.”

‘We’re just hopeful that he will outlive us as parents’

Now looking to the future, the parents are hopeful that the gene therapy treatment will allow Theo to walk and see “many birthdays”– which was unimaginable when he was diagnosed.

Natalie said: “We hope to see many birthdays, and we hope to see a lot of holidays and time together.

“With the gene therapy, they would hope that the child would live to be an adult, but they can’t really tell you what to expect, so we’re just hopeful that he will outlive us as parents.”

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