Local Man Fights for Access to Groundbreaking MND Treatment
A man from County Down is hopeful that a new treatment for a rare type of Motor Neurone Disease (MND) can provide him with more quality time with his family.
Conaire Quinn, aged 38, has a nine-year-old daughter named Autumn. He feels that time is not on his side.
Mr. Quinn, residing in Ballynahinch, is advocating for the groundbreaking drug, Tofersen, to be made widely available. The Department of Health has indicated that Tofersen isn’t currently commissioned in the UK, but it’s under appraisal by NICE (the National Institute for Health and Care Excellence).
He received his first treatment via a lumbar puncture at Altnagelvin Hospital in mid-August.
Mr. Quinn first sensed that something was off “around Covid time when I felt my foot dropping.” He described how he started stumbling while walking and experienced increasing difficulties with stairs, abdominal pains, and cramps. The tipping point came at a Manchester United match when he had to be assisted from the terrace.
“They said right away that this was a neurological issue,” he recalled. “I initially thought it was just a sports injury, but I ended up needing a crutch and I’ve been using it ever since.”
He mentioned having a family history of MND on his mother’s side. “It might sound silly, but I wanted a diagnosis since it was being downplayed. I needed it to be acknowledged,” he noted.
What is Tofersen?
Tofersen has the potential to slow or stop symptoms for individuals with a rare form of MND caused by a SOD1 gene mutation.
Consultant Gavin McCloskey from Altnagelvin Hospital reported that there are around 50 new MND cases in Northern Ireland annually. However, only one person every one to two years will have access to this drug.
“This is the first new treatment for MND in 30 years,” he explained. “It represents a new class of treatment that aims to mitigate the effects of the abnormal gene, which could, in turn, reduce nerve damage and slow progression.”
Dr. McCloskey thinks this breakthrough could “change the narrative” surrounding MND, offering new opportunities to manage the disease.
‘Breakthrough’ Treatment
Mr. Quinn is optimistic that this treatment will extend his independence. He has noticed significant improvements in his fingers, hands, and overall strength.
“This (new treatment) is essential for me to spend time with my family and my daughter. I want to take vacations and go to the park. If things keep progressing fast, that might not be possible,” he expressed. He views the new drug as a “breakthrough” not just for him, but potentially for his family and others facing MND.
“If I’m paving the way for this treatment, my loved ones will see what’s ahead and how they can access it,” he added.
‘You Don’t Get Time Back’
The MND Association is leading a nationwide campaign to make Tofersen more available and ensure there are enough specialists to administer it. This summer, they showcased a melting ice sculpture in Parliament Square, symbolizing the plight of those with MND.
A petition with 21,000 signatures has been presented to MP Karin Smyth, urging the government to expedite access to the drug.
According to the MND Association, over 30 patients are currently on Tofersen through an early access program provided free of charge by its developer, Biogen. However, about 20 patients are unable to receive it due to a lack of local services.
For Mr. Quinn, the focus remains on accessing this new treatment. “Time is incredibly valuable. Over the past few years, I’ve realized that you don’t get time back,” he stated.
The Department of Health has indicated that while the timeline for NICE’s decision is unclear, treatments approved for regular use in England are typically accessible in Northern Ireland as well. The department pledges to “make the treatment available” if recommended.
They added that if treatment requests fall outside of this framework, considerations for individual patient funding will be evaluated.
