Edward Willis-Hall as a rare genetic condition called type one spinal muscular atrophy which often ends in death before the age of two but £1.79million pioneering gene therapy Zolgensma has seen him ‘reborn’
Image: Sheffield Children’s NHS Foundat SWNS)
Little Edward Willis-Hall is celebrating after being given the best birthday present ever a month early – the world’s most expensive drug.
Edward had his landmark first birthday today (September 7) and will see many more after being given a new lease of life thanks to pioneering gene therapy Zolgensma, which costs the NHS a massive £1.79million.
Mum Megan Willis says it has been worth every penny for her son, who has a rare genetic condition called type one spinal muscular atrophy (SMA) and initially was not expected to see his second birthday.
“Getting the drug has affected Edward in every way,” says former events manager Megan, 30, who lives in Colchester, Essex.
“Children with SMA lose the ability to swallow, stand, breathe – everything that your muscles do, they will lose that.
“But Zolgensma is an incredible drug – it’s as if Edward has been reborn, there’s already been such a difference in him since he was given it last month.
“The past year has been a rollercoaster. We didn’t even know if Edward would survive and the fact that he’s not only surviving but completely thriving and getting stronger every day means Zolgensma has transformed all of our lives.”
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Sheffield Children’s NHS Foundat SWNS)
Sheffield Children’s NHS Foundat SWNS)
Edward had the incredible treatment on August 2 at Sheffield Children’s Hospital, and celebrated his first birthday with his parents at home with a cake.
As the Mirror reported in June, Edward’s parents Megan and her partner John Hall, a 37-year-old mortgage broker, were initially crowdfunding to raise the huge amount needed for the gene therapy, as it was only made available on the NHS in England for newly-diagnosed babies.
The pair managed to raise £170,000, and they now plan to put this towards treatments such as private physiotherapy three times a week for Edward.
Finding out that Zolgensma was going to be approved on the NHS for children over six months old with SMA, which affects around 65 babies born per year, was “amazing” for Megan and her family.
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“We managed to raise a lot of money, but trying to get over £1m was very difficult,” she says.
“Time was very much of the essence with trying to fund Edward’s treatment. Once the damage is done to a person with SMA’s muscles, even a wonder drug such as Zolgensma can’t reverse this.
“It was amazing to find out that he was approved to get it on the NHS and that we no longer had to worry about getting the best drug for our child.”
Edward was one of the first 20 babies to get the drug on the NHS. It involved him going into hospital for a transfusion into his arm, which took just one hour, and then he was kept in for a week for observation afterwards.
Children with SMA have a genetic defect, which means they are unable to produce enough of the protein SMN in their body – this protein is vital for muscle development and movement.
SMA is a progressive condition, meaning it gets worse over time and around 95% of babies with it will not make past the age of two, usually due to problems with their chest muscles affecting their breathing.
Zolgensma contains a copy of the gene SMN1, which produces the SMN protein in the body. It is inserted into a harmless virus, which is adminstered through an intravenous drip, and once it is inside the body the treatment ‘infects’ cells with the gene, which then makes the once-missing SMN protein be produced in the body.
This improves children with SMA’s movement and motor function, can prevent paralysis and extend their lives.
Sheffield Children’s Hospital say that the expectation for Edward after receiving Zolgensma is that he will have a better chance of sitting, crawling and walking.
“It’s an expensive drug but it is a miracle so effective, so that’s why,” says Megan.
“We don’t know what the future holds for Edward and if he will need to use a wheelchair or assistance aids in the future, but he will have a fulfilling life either way.”
At first, Megan says her son was a healthy baby, but six weeks after he was born he was rushed to Colchester Hospital after he choked and started turning blue.
After being resuscitated, Edward spent a week in a coma. He was formally diagnosed last November following a series of tests.
Initially, Edward was put on Spinraza, a drug that needs to be injected straight into the spinal vertebrae with a large needle every four months.
Again, it is costly – £450,000 for a child’s first year and £225,000 for every year following that, for the rest of their life.
But Megan and John were also told Zolgensma, was in the clinical trial stages.
After researching it themselves, they knew straight away it was what they wanted for their son, as it had the best outcome and was a lot less invasive than spinal injections.
Initially, they faced red tape but the drug was approved for babies over six months earlier in the summer on the NHS.
“Having to worry about getting the best medication for our child was really hard, especially knowing there was this drug out there but we couldn’t get hold of it,” says Megan.
“Now he’s had it, the hard part is over and our focus is on getting him as strong and able as possible.”