Six-month-old given just two years to live starts pioneering NHS treatment that could save his life
By Toby Porter
A battling six-month-old tot given just two years to live because of a crumbling spine has started a pioneering NHS treatment which may save his life.
Arthur Morgan, from Lewisham, become the first patient in England to treated with a new, potentially life-saving drug for spinal muscular atrophy (SMA) – which costs £1.79million per dose.
The new gene replacement therapy, Zolgensma, can help babies to reach motor milestones such as sitting, crawling and walking. It can also prevent them from becoming dependent on a ventilator after this single infusion treatment.
Babies born with the most common form of the condition, type 1 SMA, experience progressive muscle weakness with loss of movement and have a life expectancy of just two years.
Zolgensma – dubbed the world’s most expensive drug because of its cost – is now available to patients in four centres across England, including Evelina London Children’s Hospital.
Arthur, who was born six weeks premature in December 2020, was the first patient to receive the new treatment at Evelina London last week.
The five-month-old had been diagnosed with SMA in May.
His Lewisham-based parents Reece and Rosie noticed Arthur wasn’t reaching his expected motor milestones and was becoming immobile and floppy, so they took him to their local A&E department.
Doctors suspected that he had SMA and carried out genetic tests to confirm the diagnosis, before they transferred him to Evelina London.
Reece Morgan, who works as a plasterer, said: “This is the best possible treatment – it 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 cannula.
“We still don’t know what the future will hold, but this gives Arthur the best possible chance for a future.”
Dr Elizabeth Wraige, consultant paediatric neurologist at Evelina London said: “We are very excited to be able to provide this potentially life-changing treatment at Evelina London to infants with the severe form of SMA.
“Babies like Arthur may now be able to have this one-off gene therapy to treat the root cause of SMA, with the aim of preventing the further progression or even onset of this debilitating condition.
“This treatment will bring hope to families affected by SMA who have fought so courageously against it.”
