In July, following a lengthy regulatory review process and heavy patient campaigning across the UK – which gained extensive media coverage – the Medicines and Healthcare Products Regulatory Agency (MHRA) approved the use of Tofersen to treat those who are living with ALS (the most common form of MND), caused by mutations in their SOD-1 gene.
While only approximately 3 per cent of ALS patients have a mutant SOD-1 gene, the approval marks meaningful progress in MND research, and offers a powerful beacon of hope for further discoveries in this space.
1 in 300 of us will develop MND in our lifetimes – whether or not we have a family history of the disease.
Signals from the brain cease to reach the body’s muscles, causing a loss of mobility, speech, and eventually the ability to eat, drink and ultimately breathe.
A staggering 90 per cent of patients with ALS have so called sporadic-onset ALS, or ALS with no known genetic link.
For sporadic ALS, there are only very limited treatments that have been shown to slow progression for a short time, and alleviate symptoms; those diagnosed have an average life expectancy of just three to five years.
ALS’ biological complexity and the lack of reliable ways to track its progression have made it incredibly difficult for scientists to gain a comprehensive understanding of the whole disease.
Although there have been many clinical trials for potential new treatments, high rates of failure persist.