Sheffield hospital to trial potentially life-saving £1.79m drug for children

A Sheffield hospital will be one of four in the UK to administer a revolutionary new drug which could treat babies born with a rare genetic condition which is a leading cause of child mortality.

Tuesday, 1st June 2021, 3:40 pm

Sheffield Children’s NHS Foundation Trust will be one of the first places in the country to make use of the potentially life-saving drug on the NHS that can prolong the lives of children with spinal muscular atrophy.

Until two years ago, there were no treatment options available for children with spinal muscular atrophy (SMA), which is the leading genetic cause of death for children.

But babies could potentially have the ability to sit, crawl and walk after being treated with US gene therapy Zolgensma, which has been called the most expensive drug in the world.

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Sheffield Children's Hospital

Zolgensma, which has a list price of £1.79 million per dose, was made available on the NHS after the health service struck a deal with manufacturers Novartis Gene Therapies in March.

This comes after a five-month-old baby has become the first patient in England treated with the drug.

Arthur Morgan, who was diagnosed with the condition earlier this month, received the one-off gene therapy at Evelina London Children’s Hospital on May 25.

Baby Arthur, who was born six weeks premature in December, underwent the gene therapy infusion last week after being diagnosed with SMA less than three weeks earlier.

His father Reece Morgan, 31, who works as a self-employed plasterer, said: “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.

“We still don’t know what the future will hold, but this gives Arthur the best possible chance to give him the best possible future.”

Babies born with Type 1 SMA, which is the most common form of the condition, experience progressive muscle weakness, loss of movement, difficulty breathing, and have a life expectancy of just two years.

Studies found that a single treatment with Zolgensma has helped babies with SMA to sit, crawl and walk, and also prevented them from having to be put on a ventilator.

Four specialist NHS centres have now been commissioned across the country to administer the treatment, including Evelina London Children’s Hospital, where Arthur was treated.

The other sites are Manchester University NHS Foundation Trust, Sheffield Children’s NHS Foundation Trust and University Hospitals Bristol and Weston NHS Foundation Trust.

Dr Elizabeth Wraige, consultant paediatric neurologist at Evelina London Children’s Hospital, said: “This treatment will bring hope to families affected by SMA who have fought so courageously against it.”

NHS chief executive Sir Simon Stevens added: “It is fantastic news that this revolutionary treatment is now available for babies and children like Arthur on the NHS.

“The NHS Long Term Plan committed to securing cutting edge treatments for patients at a price that is fair to taxpayers.

“Zolgensma is the latest example of the life-changing therapies that the NHS is now routinely using to transform the lives of patients and their families.”