NHS to offer £1.8million drug to cure babies of spinal disease that can kill infants before they turn two
- Around 56 babies born every year in England have spinal muscular atrophy
- The disease causes muscle weakness, paralysis and breathing issues
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Babies born with a genetic disease that can lead to a life expectancy of just two years without intervention will now be cured on the NHS with a new £1.8million drug.
Around 56 infants are born with severe spinal muscular atrophy (SMA) in England every year, which causes muscle weakness and can lead to paralysis.
The National Institute for Health and Care Excellence (Nice), the NHS watchdog, has approved the use of onasemnogene abeparvovec, branded as Zolgensma, for babies born with the condition.
The disease, which is the leading genetic cause of death in children, is triggered by a defect in a gene called SMN1, which makes a key protein that enables nerves in the spinal cord to control muscle movement.
SMA causes muscles to waste and gets worse over time, making it difficult for breathe, move and eat. Nine in 10 of those who have the most severe form of the disease, known as type 1, die by the age of two if they do not receive treatment.
Then five-month old Arthur Morgan with his father Reece Morgan in June 2021 after he became the first patient in England to undergo gene therapy using Zolgensma
The one-off infusion, given in just an hour, uses a harmless virus to deliver a healthy version of the SMN1 gene
But Zolgensma has been proven in studies to help sufferers sit, crawl and walk — something they would never normally be able to do — and also prevents them from being put on a ventilator.
The one-off infusion, given in just an hour, uses a harmless virus to deliver a healthy version of the SMN1 gene.
It passes into the nerve cells to replace the defective gene and restores normal function, allowing the baby to live a healthy life with minimal symptoms.
It comes after one-year-old Edward Willis-Hall, from Colchester in Essex, was given the drug and subsequently took his first assisted steps last January.
At the time, his mother Megan Willis, 30, spoke of her joy after her son achieved milestones she never thought possible.
READ MORE: Mother says £1.8million drug has given her son his life back
He can roll over, sit up, stand for five minutes in gaiters which support his legs, and take steps in a harness.
She told the BBC: ‘We are so proud of Edward. He’s doing incredibly well. He is far surpassing our expectations. We are so incredibly lucky.’
Ms Willis, who was told her son had spinal muscular atrophy when he was seven weeks old, set herself the goal of him sitting unaided. She said: ‘All I ever wanted was for him to be able to sit and I knew then he would have an amazing life.’
Nice has said purchasing the drug is a ‘cost-effective’ use of resources.
Currently only babies who have a family history of SMA will be tested.
But campaigners are pushing for all five-day-old infants to undergo checks diagnosing nine conditions.
Laurent Servais, a professor of paediatric neuromuscular disease at the University of Oxford, said: ‘I have seen too many families devastated by this disease, but we now have treatment options.
‘However, waiting until the onset of symptoms is still too late. Every day we delay in finding and treating these infants, we could be responsible for a child spending their life in a wheelchair.
‘There is a 100 per cent consensus among UK experts, among world experts, among patients: we need to screen to save life, we need to screen to save quality of life and we need to screen to save money.’
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