By Caroline Purslow, Head of Health, Challenge Works
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.
As such, investment in this area is relatively risky, further hindering the chance of successful drug treatment.
However, Tofersen proves that ALS is not insurmountable, and that it can be solved despite its complexity.
Knocking down this first brick in the wall is not only a huge triumph for those living with SOD1-ALS, but it also shows that drug treatments are no longer an impossibility for the other 97 per cent of people living with ALS.
The next brick to knock down in this wall is the identification of viable biological drug targets to treat sporadic disease.
This breakthrough is key to interrupting the current bleak dynamic of continued trial failure rendering ALS drug discovery high risk and therefore a low-investment area of drug research.
To de-risk pursuing the development of treatments and incentivise pharmaceutical companies to focus on this area, we need to create a trustworthy map of drug targets that have been identified as having high potential for successful drug discovery.
This is where the £7.5 million Longitude Prize on ALS comes in.
The Prize is run by Challenge Works, part of Nesta, and is seeking entries until December 3rd 2025.
Principally funded by the Motor Neurone Disease Association, alongside numerous additional international funders, the Longitude Prize is incentivising AI-led discovery of new drug targets to accelerate treatment development for ALS.
The key to AI-enabled drug discovery is data – and access to the right data is one of the leading components of the prize.
Historically, ALS research data has been fragmented across various institutions, each with their own unique access restrictions and formations.
This has posed a number of challenges to researchers – another hurdle to finding a cure for ALS.
The prize has convened one of the largest and most comprehensive collections of ALS patient data, and participants will be offered access.
These datasets have been curated to be interoperable, so that AI algorithms can be trained to scour through the biology of the disease and identify the most promising drug targets for future treatments.
This database, on which AI models can be built and trained, will accelerate the pathway towards drug discovery – at incredible speed.
The teams entering will require a varied set of expertise, spanning complex neurodegenerative disease and an understanding of and ability to use AI to analyse data.
However, we recognise that some teams may not have all of this expertise; so to support applicants, the prize is facilitating connections between participants with a ‘match making’ process that will ensure each team has a balance of biological and technical expertise.
Following the initial entry period, the prize will support teams through various stages.
Initially, it will reward 20 of the most promising entrants with ‘Discovery Awards’ of £100,000 each – as well as full access to the incredibly valuable datasets.
After the initial £100,000 ‘Discovery Awards’ have been awarded in 2026, 10 teams will progress to a second stage, receiving a further £200,000 in 2027 to build the evidence base for their proposed drug targets in silico and in the lab.
In 2028, five teams will then receive £500,000 to undertake validation of the highest potential identified targets in the wet lab.
The winning team will be announced in early 2031 and will be awarded £1 million for identifying the target with the strongest evidence of therapeutic potential.
Rapid advancements in AI, a deeper understanding of ALS, and the availability of vast amounts of biological data have converged to make now the ideal moment to intensify efforts toward finding a treatment for ALS.
Tofersen is proving life-changing for those with the SOD-1 gene mutation; now it’s time to transform the lives of all ALS patients.
Identifying drug targets is the essential first step toward pharmaceutical companies developing new treatments. The Longitude Prize on ALS is helping to make this dream become a reality.
