Baby receives new potentially life-changing NHS gene therapy at our hospital
Posted on Tuesday 1st June 2021
Baby Arthur with his father
Evelina London has become one of four children’s hospitals in England to provide an exciting, potentially life-saving gene therapy to babies.
The new drug, Zolgensma, can allow babies and young children with a rare genetic disorder Spinal Muscular Atrophy (SMA) to reach motor milestones such as sitting and crawling, to breathe without a ventilator, and to live for longer. Evelina London is one of four centres in the country who can now provide this new therapy on the NHS and five month old Arthur Morgan was the first baby to receive the treatment at Evelina London.
Baby Arthur, who was born in December six weeks premature, underwent the gene therapy infusion last week after being diagnosed with SMA less than three weeks previously.
His parents Reece and Rosie had taken him to their local A&E department after they noticed that he wasn’t reaching his motor milestones and was becoming immobile and floppy. After a genetic test confirmed a diagnosis of SMA he was transferred to our specialist children’s hospital.
Arthur’s dad, Reece, said: “This is the best possible treatment and will give him the best possible life he can have, and we are so grateful that the NHS is here for him.
“When we found out that Arthur would get the treatment, and be the first patient, I just broke down. It had been such a whirlwind few weeks, filled with lots of anxiety and adjustment as we learnt about his condition and what it might mean for him and our family.
“The way the treatment works is incredible, where the gene is hidden in a virus, and is given to him in a simple canula. We still don’t know what the future will hold, but this gives Arthur the best possible chance for a future.”
Babies like Arthur may now be able to have a one-off gene therapy to treat the root cause of SMA with the aim of preventing the further progression or even start of this debilitating condition. The treatment, which has been dubbed the ‘world’s most expensive drug’ at £1.79 million list price per dose, is now available to eligible patients on the NHS. As with all treatments our clinical teams will have full discussions with families of babies with SMA to see if this treatment option is appropriate for their child.
Marian Ridley, Director of Evelina London said: “At our specialist children’s hospital we look after the sickest children and young people. Spinal Muscular Atrophy is a devastating condition, so to be able to provide this life-changing treatment is hugely exciting.
“Research and innovation are a big part of what we do at Evelina London, including big leaps forward in treatment for rare conditions such as SMA. Healthcare is always evolving and through new treatments we are able to offer more life-changing care and treatment to the growing number of children and families that need us.”