World's most expensive drug gives terminally ill baby future back
A baby with a rare genetic disorder who was given just 18 months to live is now expected to experience adulthood, after being treated with the world's most expensive drug, Zolgensma.
Baby Theo Greenhall, one, 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, said they are now "hopeful that he will outlive us as parents" thanks to the new life-changing drug, after originally being faced with the worst.
Zolgensma, costing nearly £2 million, became available through the NHS last year to patients with SMA under one in the UK. This came after the organisation made a deal with the US-based manufacturer Novartis Gene Therapies.
Natalie and Sean discovered Theo had the most severe form of SMA just a few weeks after he was born (November 3, 2020), when he was given less than two years to live.
Natalie, also 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."
On being told Theo's prognosis, Natalie said, “In that moment as a woman, I was quite emotional. I couldn’t even speak. My husband didn’t really process it there and then. He asked questions about it.”
Theo was originally put on a drug called Spinraza, leading him to have "traumatic" monthly spinal injections, which doctors hoped would stabilise and improve his condition. But then by fate, baby Theo's parents got some better news.
Early one morning in March 2021, Natalie woke to find out that the NHS would be funding a new drug costing £1.795 million per single dose, which has the potential to have drastic benefits for children like Theo.
"We were back in hospital for a sleep study, and at 6am, I was awake, and I looked at the news, and I was like "Oh my life! The gene therapy has been approved in England," she said.
"I had nobody to talk to – no nurses were around, and my husband wasn't there – but I was like, 'This is so exciting!'"
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The one-off treatment is given in just an hour and uses a harmless virus to deliver a healthy version of the SMN1 gene, which is defective in those with SMA.
Theo was one of the first babies to become eligible for Zolgensma, after his doctor gave him approval in July last year. However, things were still a little touch and go for a while.
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After going to intensive care on July 4, medics stalled and set a deadline for his health to improve by his first birthday in order for Theo to qualify.
Natalie explained that her son couldn't breath for himself, and he had to be off the ventilator for at least 18 hours (in order 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," she said.
As the deadline given by the medics grew closer, Natalie said doctors were "surprised" to see Theo's progress and gave him the all-clear just in time to get get the much-needed treatment – in the same week he turned one.
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Theo's parents are incredibly grateful that he was selected for the new treatment that "blows" Natalie's mind, considering how expensive it is.
"For that amount of money to be spent on our child, and other children in the same position, it's just incredible," she said.
"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."
She added, "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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