A mother has been left devastated by a government ruling set to take away a “miracle drug” that has stopped her motor neurone disease from progressing. Eleanor Dalley, who has a 14-year-old daughter, was given just 18 months to two years to live when she was diagnosed with a rare form of the brutal terminal condition in 2019.
Just two per cent of MND sufferers have this condition caused by a mutation in a gene called SOD1.
She started taking tofersen, a promising new drug that slows the progression of the neuro-degenerative disease, in November 2022 through an early access programme – the phase following a clinical trial.
By that point she could not turn over in bed without using her arms to lift her legs, but since the first treatment there has been “no further decline”.
However, the National Institute for Health and Care Excellence (NICE) has decided not to classify the new drug as treating a clinically distinct disease, a Highly Specialised Technologies.
Instead the public body attached to the Department of Health looked at as a standard drug, which experts say makes it “highly unlikely” to be available on the NHS.
Ms Dalley, 48, from London, said the “miracle she and her family have prayed for’ has been taken away.
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.
“My daughter said to me ‘with this treatment it gives you hope, and it gives us a 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.”
Ms Dalley is also worried that tofersen will not be available if her 14-year-old daughter needs it, as two of her aunts and her father were diagnosed with MND.
She added: “My daughter has already worked out the genetic connection. I told her there is a 50 per cent chance but said, ‘let’s hope you get Daddy’s genes for this’.
“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.”
A NICE spokesperson said: “This therapy does not meet the criteria for the Highly Specialised Technologies programme.
“NICE will not therefore be reviewing this decision.”
*The Sunday Express helped secure £50million for research to find effective treatments through our Fund The Fight To Cure MND crusade in 2021, but tofersen was found separately to this ongoing mission.
As a former specialist nurse focusing on end of life and palliative care, I became well aware of the brutality of motor neurone disease many years before taking my post in January last year.
Since then I have had the great privilege of working with people affected by MND across the country.
I know that across the MND community today there is deep concern over the impact of NICE’s approach to appraising tofersen, a new precision medicinefor treating MND.
Tofersen is targeted at people living with MND who carry a specific gene mutation, known as SOD-1 MND.
This constitutes around two per cent of the MND patient population, estimated to be 60 to 100 people at any one time.
Tofersen has been shown to slow disease progression and increase life expectancy for this group.
MND is a progressive, incurable, and terminal condition, for which there is only one licensed treatment available in the UK.
Naturally, the prospect of a new, effective treatment on the horizon is a source of great hope and excitement across the MND community. The NICE decision has extremely concerning implications for people with rare sub-types of diseases in the UK.
At a time of growing global interest in the potential of precision medicine to transform outcomes for patients, NICE’s approach to tofersen risks consigning the UK to the back of the queue.
It is directly contrary to the Government’s stated vision for the UK to move away from a “one size fits all” approach to be a world centre for precision medicine.
If NICE does not change its routing decision on tofersen, people with MND in the UK will be denied access to an innovative and effective new treatment, even as their counterparts in the EU, the United States and elsewhere are able to access it.
I believe this is unacceptable – and so do all those currently living with this devastating disease.
Tanya Curry, chief executive of the MND Association
AS A former specialist nurse focusing on end of life and palliative care, I
became well aware of the brutality of motor neurone disease many years before taking my post in January last year.
Since then I have had the great privilege of working with people affected by MND across the country.
I know that across the MND community today there is deep concern over the impact of NICE’s approach to appraising tofersen, a new precision medicinefor treating MND.
Tofersen is targeted at people living with MND who carry a specific gene mutation, known as SOD-1 MND.
This constitutes around two per cent of the MND patient population, estimated to be 60 to 100 people at any one time.
Tofersen has been shown to slow disease progression and increase life expectancy for this group.
MND is a progressive, incurable, and terminal condition, for which there is only one licensed treatment available in the UK.
Naturally, the prospect of a new, effective treatment on the horizon is a source of great hope and excitement across the MND community. The NICE decision has extremely concerning implications for people with rare sub-types of diseases in the UK.
At a time of growing global interest in the potential of precision medicine to transform outcomes for patients, NICE’s approach to tofersen risks consigning the UK to the back of the queue.
It is directly contrary to the Government’s stated vision for the UK to move away from a “one size fits all” approach to be a world centre for precision medicine.
If NICE does not change its routing decision on tofersen, people with MND in the UK will be denied access to an innovative and effective new treatment, even as their counterparts in the EU, the United States and elsewhere are able to access it.
I believe this is unacceptable – and so do all those currently living with this devastating disease.
