Eleanor Dalley didn’t think she’d live to see 50 after being diagnosed with motor neurone disease (MND) in 2019.
There is no cure, and most people die within a few years as they lose their ability to move, eat, talk and breathe.
But for Eleanor, there was a lifeline – a breakthrough drug that has stopped the condition in its tracks.
“It’s a miracle to be honest, it’s like I’ve frozen in time,” the mum-of-one told Sky News.
Eleanor Dalley celebrated her 50th birthday in the Maldives
MND affects the brain and nerves, causing muscles to get progressively weaker. While Eleanor has lost some movement in her legs, her symptoms have not got any worse – allowing her to reach milestones she did not think she would live to see.
“My worst fear was that I’d be dead within a year,” Eleanor said.
“I would put things on TV that would make me cry, so that I could say it was the TV that was making me cry, but it wasn’t really. It was me worrying about, I’m not going to see my daughter grow up or I’m not going to get to 50.
“Having this new drug has meant I’ve achieved all those things.
“But I’m really lucky – and not everybody is getting that same opportunity.”
Eleanor, pictured on holiday, said Tofersen had halted her symptoms
The drug, Tofersen, is the first effective treatment for MND symptoms. Eleanor was able to get it through an early access programme ran by its manufacturer Biogen, which is currently offering it to the NHS for free.
However, it requires regular spinal injections, and while the drug itself is free, some NHS trusts say they don’t have the staff or resources to administer it.
These issues have resulted in a postcode lottery for access, with people like 19-year-old Lillia Jakeman losing out.
‘Mental torture’
“It’s like a carrot that’s dangled, and then it’s taken away,” said Lillia’s stepmum Rachel, a GP of 30 years. “It’s a very cruel, unjust situation.”
Lillia was diagnosed with MND in August, when she was given a leaflet about Tofersen. However, she has not been able to access the treatment upon referral.
She now uses a wheelchair, while her upper arms are getting weaker, affecting her ability to do what she loves.
Lillia Jakeman, 19
Lillia with her grandad, who died of MND
“One of the biggest things for her is that she used to love doing Lego and artwork, and 1769773718 she can’t,” said Rachel.
“She’s still got a degree of thumb function, so she can use an electric wheelchair, she can do certain things. But the point is that Tofersen needs to be given to these patients as early as possible to preserve function, and you know every day matters.
“Lillia describes it really as mental torture, not knowing when you wake up the next day, whether you might have lost something, some other function that’s gone.”
Lillia has not been able to get the drug through her local NHS trust
The All Party Parliamentary Group on MND, chaired by Labour MP Ian Byrne, said it was aware of 20 people unable to access Tofersen and four people who had died while awaiting treatment.
The drug is for those with an inherited form of MND called SOD1 – which makes up 2% of cases.
Caroline Nokes, Lillia’s local MP and a senior Conservative backbencher, said it should not be difficult to extend access to a small handful of people.
She joined Lillia’s family and Mr Byrne in handing in a petition to Downing Street on Wednesday, calling for the government to intervene.
NHS ‘red tape’
The issue has sparked wider concern about accessing new treatments on the NHS, given the millions of pounds being invested in the life sciences sector on research projects and clinical trials.
Tofersen received marketing authorisation in July from the UK’s Medicines and Healthcare Regulatory Agency.
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However the National Institute for Health and Care Excellence (NICE) is the body that advises whether drugs should be made available for routine funding on the NHS.
It will begin its evaluation process for Tofersen in March, but it is not clear how long it will take.
Ministers are said to be reluctant to interfere as they feel this would undermine the official process.
Lillia’s family walked from Southampton to Downing Street as part of their campaign to widen access to Tofersen
Professor Ammar Al-Chalabi, a consultant neurologist at King’s College Hospital, said there needed to be a simpler system.
He said: “The government would like us to be a science superpower, and for motor neurone disease, we really are.
“And this drug is available but it’s not available to patients despite the scientific breakthrough, what does that say?
“What’s the purpose of finding these new treatments if they’re not available on the NHS? The pathway has to be complete.”
Professor Ammar Al-Chalabi
A Department of Health and Social Care spokesperson said: “We are committed to improving care for people with motor neurone disease like Lillia and ensuring they receive the support and treatment they need.
“Tofersen is approved for use in the UK and NICE is working with the manufacturer on whether to recommend it for routine funding by the NHS.
“If recommended, NHS England will work to make it available to NHS patients as rapidly as possible.”
