Eleanor Dalley’s motor neurone disease has been improved by a treatment which is unlikely to receive approval for NHS patients

Photo: RADAR

A Barnet mother whose terminal illness has been stopped in its tracks by a new drug is ’devastated’ after a decision by the National Institute for Health and Care Excellence (NICE) means it is unlikely the treatment will be accessible to patients through the NHS.

Eleanor Dalley, 48, from Barnet was diagnosed with motor neurone disease (MND) in July 2019, first noticing when exercising and stretching she couldn’t stand on tip toes with her left foot. MND attacks the nerves that control movement so muscles no longer work. MND does not usually affect the senses such as sight, sound or feeling.

Both of Eleanor’s aunts were diagnosed with MND, along with her father in November 2020. He died just nine months later in August 2021.

Eleanor said: “We discovered we have the gene mutation in the family. We have a family group chat and I share updates when I go for my treatments.”

The SOD1 form of MND is caused by a rare genetic mutation that is found in around 2% of all cases of the disease, but as much as in one in five cases where MND runs in families, estimated at up to 100 people in the UK. The faulty gene triggers the cellular production of a protein which is toxic to motor neurons. Tests showed she had the SOD1 MND gene and spent six months in what she calls ’the crying phase’ and thinking of ’what I was going to miss’.

Eleanor said: “I didn’t want my daughter to see me crying so I would put on a sad film so I could say that’s why I was in tears.”

By 2021, she relied on a walker and a wheelchair. When she started to feel further changes in her legs, Eleanor had to stop driving and working.

In November 2022S through an early access programme – the phase following a clinical trial Eleanor started taking tofersen, a drug targeted to treat people living with motor neurone disease who have alterations in the SOD1 gene, which involves a lumbar puncture every four weeks.

By this point, she couldn’t turn over in bed without using her arms to lift her legs but since that first treatment, Eleanor said there has been ’no further decline’.

She said: “There have been small, but for me, significant improvements. I can move my legs in bed unaided and can get back on the massage table on my own. I still can’t walk unaided, but these small things are mini miracles to me. I wanted to give myself a year taking the drug before I started to really hope.

She said: “Prior to tofersen, my MND was slowly progressing. When I was given my diagnosis, I was told the stats – 18 months to two years prognosis. In July it will be five years since I was diagnosed. I am outliving the odds and tofersen has given me the courage to think longer term.”

“My daughter said to me ‘with this treatment it gives you hope, and it gives us chance of a cure’. I haven’t told her the latest news.

“For me, it feels like tofersen is giving the research community time to find more answers and find a cure. I feel in a privileged and lucky position to have the drug.”

“We must show our community that we believe in them and will fight for them. It’s literally life and death for us.”

NICE has confirmed its decision to appraise the drug tofersen through the Single Technology Appraisal (STA) route rather than the Highly Specialised Technologies (HST) route is final. This is despite an appeal lodged by drug developers Biogen, with input from the MND Association and leading neurologists.

Ordinarily, products targeted at rare diseases that affect less than 1 in 50,000 people in England are assessed through the HST route. However, NICE confirmed it will conduct a standard appraisal because it does not view SOD1 MND as a clinically distinct disease.

The MND Association has been advised it is highly unlikely that tofersen will receive a positive decision if evaluated through the standard route – meaning that it will not become accessible to patients through the NHS.

Eleanor says she is ’devastated’ by the decision and the ’miracle she and her family have prayed for’ has been ’snatched away’. “My daughter has already worked out the genetic connection. I have told her there is a 50% chance but said ‘let’s hope you get Daddy’s genes for this’”.

Eleanor, who turns 50 in October 2025, is now planning a girls’ trip with her friends and a holiday with her daughter to celebrate the milestone.

She added: “I never thought I would be able to do that and plan that far ahead. Previously I’ve worked in six-month blocks – which I know is still better than for many living with MND.

“I have already seen milestones I didn’t expect – I didn’t think I would see my daughter go to secondary school but she’s now 14.

“I never thought after my diagnosis I was going to get to 50 but now the date is in my diary, and I am planning for it. MND has taken my loved ones but it’s not going to take my hope for the future.”

Dr Brian Dickie, Director of Research Development at the MND Association, said: “I struggle to understand the logic behind this decision by NICE.

“Neurologists know that this is not one single disease but is actually made up of different subtypes which will, as in the case of SOD1 MND, require highly specific and specialised approaches to treatment.

“NICE appears to believe that if the disease looks the same ‘clinically’ on the outside, it therefore must be the same process killing the nerve cells on the inside, but research has shown this is not the case.

“This is a level of understanding of MND that, frankly, belongs to the last century.”For more information about MND and the MND Association please visit www.mndassociation.org

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