Boy getting ‘most expensive drug’ for genetic condition is ‘best thing ever’

A couple who fought for their young son to have “the most expensive drug in the world” to treat his genetic condition say they are “overwhelmed” after he finally got the treatment.

Rosie-Mae Walton and Wes Powell had been desperately trying to raise cash for their son Marley to have the £1.8 million genetic drug Zolgensma in the United States when it was licensed in the UK and NHS England struck a landmark deal with the American manufacturer.

Marley, who is now 16 months old and has Type 1 spinal muscular atrophy (SMA), was given the one-off treatment of the “miracle drug” at Sheffield Children’s Hospital on Tuesday afternoon and is now recovering.

Ms Walton said it had been a “rollercoaster” since the drug was licensed in March, as they waited to find out whether Marley was eligible.

She said they just cried when he was finally approved.

“You can imagine that was just so overwhelming,” she told the PA news agency.

“It was like the best news ever that had come to us.”

Ms Walton added: “As soon as we found out that it came to the UK, we just cried our eyes out. Then, when we got the antibody test, and that came back negative, that he was eligible for Zolgensma, I read the email and we just cried again. We just cried all day.

“It was just the happiest moment ever… just to know that he’s actually getting it, after all the time waiting and how much we’ve been through and how much Marley’s been through.”

She said Marley appears to be doing really well since Tuesday’s treatment.

“It feels so surreal, like it hasn’t hit home yet that he’s actually had it – that a drip is just so expensive and has worked wonders.

“It’s just crazy considering all we’ve been through and the amount of scares you get put through thinking that your baby won’t reach the age of two or a second birthday.

“Then you think that he’s had this most expensive drug in the world. It’s just crazy. It’s the best thing ever.”

Ms Walton and Mr Powell, who live in Driffield, East Yorkshire, said money raised for Marley will be used for continuing treatment, including private physiotherapy and equipment.

Ms Walton said: “As much as he’s got his treatment, it’s not just going to be an easy ride. So, we just thank every single person that’s helped out for that.”

Tony Hart, consultant paediatric neurologist at Sheffield Children’s Hospital, said SMA is a genetic condition which causes a range of life-limiting problems, meaning that most children with the condition die within a couple of years.

“Zolgensma is a breakthrough because it really is a miracle drug,” he said.

“Without Zolgensma, most children would have died.”

He said Zolgensma stops further deterioration but more research is needed to know whether it can repair existing damage.

But he said some children who have had the treatment do appear to have got stronger.

Mr Hart said more than 30 children have now received Zolgensma in the four centres in England and Wales where is it being given – Sheffield, London Manchester and Bristol.

Asked about handling the “most expensive drug in the world”, the consultant said he leaves that to the nurses, adding: “One of the great things about the NHS is that decisions about costs are taken out of our hands and our priority is giving as many children who need it the drug when we can, and as fast as we can.”

NHS England announced in March that it had struck a “landmark confidential deal” with US-based manufacturer Novartis Gene Therapies which then-chief executive Sir Simon Stevens described as a “life-changer”.

In May, five-month-old Arthur Morgan became the first patient in England to be treated with Zolgensma, at Evelina London Children’s Hospital in London.

In December, PA photographer Joe Giddens battled the worst weather of the winter to scale England’s highest peak, Scafell Pike, six times in 24 hours to raise funds for Marley’s trip to the United States.