'We're being left to die': West Lothian mum with motor neurone disease denied life-saving treatment
A West Lothian mum with a rare form of motor neurone disease is fighting for the chance to receive treatment which she says could save her life.
Angie Brown, from Bathgate, was diagnosed with the SOD1 gene variation, a form of MND that is thought to affect around two per cent of people with the condition, in February last year.
But although there is an available treatment known to slow the condition, the treatment called Tofersen is not being made available to patients in Scotland.
Angie said: “This treatment will save my life; it slows the type of MND I've got and stops it progressing. If there's a treatment that will mean I can live longer and live better, I really want that to be available for anybody who needs it.
“The drug is actually free and it’s being provided by a company called Biogen, so the NHS aren't having to pay for the drug at all. The problem is the red tape around it and the funding to have it administered in a clinic.
“But it seems nobody is listening. I’ve written to the Heath Secretary but got no response, I contacted the Scottish Government several times and got one reply saying to basically speaks to my doctor about it.
“This issue really needs to be highlighted because people with SOD1 in Scotland are being left to die. Every day matters – with MND something stops working a little bit more every day.”
My Name’5 Doddie Foundation described Tofersen as ‘the first truly effective life-extending treatment for people living with SOD1-MND’ adding it is ‘unacceptable’ that eligible patients are being denied the treatment.
It is understood the Scottish Government is exploring ways to safely administer the drug and the Scottish Medicines Consortium (SMC) ‘has not yet received a submission for Tofersen’ from the pharmaceutical company.
Angie said the procedure involves a bilateral injection in the spine ‘similar to a lumbar puncture’. She said: “I’ve been told it’s a very easy process. There are even medical professionals saying they are willing to come up and help Scotland set up and train people on how to administer the drug it. But at the moment I’m trying to find a place in England because Scotland is still catching up.”
The 47-year-old florist who owns Bloom Room Florist in Bathgate, said she first experienced back problems and weakness in her right leg around six years ago, which doctors now believe was the onset her condition. Angie, whose brother Tam Agnew and cousin passed away from MND said there is also a 50 per cent chance she will pass the gene onto her two teenage children.
She said: “I'm still working and trying to do bits and bobs, but I'm actually training my niece because I don't think I'll be able to do it much longer. My whole right leg is quite weak now, I can still walk slowly and can do stairs and stuff like that but it’s now affecting my right arm and dexterity in my hand.”
The Bathgate resident added: “Doddie Weir’s foundation has provided the most support to me and they really want to support people and help. But there’s all this red tape that's stopping people like myself getting lifesaving treatment.
“There are some patients in England who receive the medication through the National Institute for Clinical Excellence (NICE) early access programme but it’s a postcode lottery as to who gets it and people in Scotland are getting left behind.”
A spokesman from the My Name’5 Doddie Foundation said: “The drug is licensed in the UK but still needs to be approved for NHS use by the National Institute of Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC).
“While this process goes on, the manufacturer, Biogen, is making the drug free of charge to eligible patients on a ‘named patient basis’. This means NHS Trusts and Health Boards can choose to make the drug available and currently, only some have done so.
“This postcode lottery is preventing some eligible patients accessing the treatment. This is a particular issue in Scotland, where no Health Boards are currently offering it at all.”
A Scottish Government spokesperson said: “The Scottish Government is committed to ensuring that people living with conditions like Motor Neurone Disease (MND) have access to high quality care and support, and benefit from healthcare services that are safe, effective and person-centred.
“We have a clear and consistent route in Scotland for licensed medicines to be appraised through the Scottish Medicines Consortium (SMC). The SMC has not yet received a submission for Tofersen and would welcome a submission from the pharmaceutical company at any time.
“A pharmaceutical company may choose to offer a medicine free of charge prior to appraisal, sometimes known as an Early Access Programme (EAP). While such schemes provide the medicine at no cost, they do not normally cover the additional resource needed to support service implementation.”
Angie’s great niece and nephew will take part in a fun run to raise money for research into the disease this summer. Three-year-old twins Campbell and Harper Flemming will run 1k to raise vital funds for MND research. You can support their fundraiser by visiting the JustGiving website.