A groundbreaking new treatment for motor neurone disease (MND) has been approved for use in the UK, offering fresh hope after decades of despair.
But campaigners warn that patients may still have to wait for access on the NHS.
The drug, called QALSODY (tofersen), is the first in Britain to directly target a genetic cause of the disease.
It is being hailed as a “genuine breakthrough” - the first new treatment for MND in almost 30 years.
The new drug will only help those with a rare inherited type of MND caused by a fault in the SOD1 gene - thought to affect fewer than 60 people in the UK.
However experts say the breakthrough demonstrates the potential of a new era of gene-targeted therapies, which are currently being trialled in other forms of MND offering hope to thousands of patients diagnosed each year.
Professor Chris McDermott, a leading consultant neurologist and Sheffield MND Care Centre Co-Director said: “This drug is a gamechanger for many of the patients who have taken it who have noticed a step change in the way their disease progresses with some also noticing improvement in their function. Some of these patients would not be alive now were it not for this drug.”
He added: “This technology is a major milestone for all MND sufferers and is now being used in trials to target other key genes and proteins for other forms of MND.
"This is a very exciting time for MND and looking to the future our understanding of MND means we should have a meaningful treatment for everyone in years rather than decades and we are not giving up on a cure.”
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The treatment is being hailed as a 'genuine breakthrough' - the first new treatment for MND in almost 30 years (stock pic)
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MND attacks the nerves controlling movement, slowly paralysing sufferers until they can no longer walk, speak, swallow, or even breathe.
In the UK, around 2,000 people are diagnosed every year. Some 5,000 people live with the disease at any given time, and every day it claims the lives of six more people.
Life expectancy is short: most die within two to five years of symptoms starting.
Campaigners point out that the UK has lagged behind in offering new therapies, despite huge public pressure.
The three main UK MND charities — the MND Association, MND Scotland and the My Name’5 Doddie Foundation - welcomed the announcement, calling it “a significant milestone for the MND community.”
Stephen Hawking lived with MND for 55 years
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QALSODY, developed by biotechnology company Biogen, works by blocking production of a harmful protein that causes nerve damage.
In trials, it reduced levels of a key marker of nerve injury by 55 per cent compared to placebo.
Dr Kylie Bromley, Biogen’s UK & Ireland General Manager, said: “This approval is a moment of real hope for the MND community. We now need to work with the NHS to make sure patients can access it as quickly as possible.”
Despite approval from the UK medicines regulator, the Medicines and Healthcare products Regulatory Agency (MHRA), the drug is not yet available through the NHS.
Before that can happen, it must be assessed by the National Institute for Health and Care Excellence (NICE), which decides whether treatments offer value for money.
Because the SOD1 type of MND is so rare, trial data is limited. That makes it harder for NICE to approve, despite the desperate need.
Campaigners are urging NICE and NHS England to show flexibility. Last week, the MND community held a powerful protest in London, unveiling a life‑sized ice sculpture of a patient in a wheelchair outside Parliament.
The melting figure symbolised how quickly time runs out for those living with the disease, with campaigners pleading: “We cannot wait.”
MND has taken some of the UK’s most beloved figures. Rugby legend Doddie Weir, who lent his name and passion to the fight, died in 2022. Former footballer Stephen Darby has spoken movingly of his own struggle.
Families often describe the illness as being forced to watch a loved one slowly fade away, while they remain fully aware of what’s happening.
Motor neurone disease (MND): a fatal condition that attacks the nerves controlling muscles.
Life expectancy: Usually two– five years after symptoms begin.
UK impact: 2,000 new cases each year, 5,000 people living with the disease. Daily toll: Six deaths per day. Inherited cases: Around 15 per cent are genetic.
The SOD1 mutation affects about two per cent worldwide, with fewer than 60 in the UK. QALSODY is already available in the US and Europe.
The MHRA’s decision puts Britain on the map, but campaigners stress that this is only the first step.