EXCLUSIVE: ALS is at a turning point – and investing in treatments makes strategic sense
Research into ALS is at a pivotal moment, thanks to an uptick in funding, a growing understanding of the disease, increased availability of large-scale datasets, and an expanding number of AI and in-silico technologies that can accelerate drug discovery.
Recognising this, the Longitude Prize on ALS was launched on 25 June 2025: a £7.5 million prize to incentivise the creation of AI-based approaches to transform drug discovery for the treatment of ALS. It is principally funded by the Motor Neurone Disease Association, and designed and delivered by Challenge Works.
Tris Dyson, Managing Director, Challenge Works, explains why a successful new treatment for ALS could be worth billions.
Globally, we’ve made real strides within neurodegenerative disease research. Thanks to advances in AI, increased availability of datasets, an uptick in funding and the tireless work of scientists around the world, the promise of an altered course of reality for some of these devastating conditions is becoming possible.
Recently, researchers at the University of Sheffield have discovered and developed M102 – a new drug that has been found to protect nerve cells damaged by motor neurone disease (MND). Although still at the preclinical stage, results so far suggest M102 could significantly extend life expectancy.
For the first time, Huntingdon’s has been successfully treated in trials by a research team in the UK. A gene therapy, administered during highly precise brain surgery, has been shown to slow disease progression by 75 per cent.
However, amyotrophic lateral disease (ALS), the most common form of MND, remains incurable, and the drugs that are available have limited impact.
I’d be remiss not to note the progress that has been made – notably the development of tofersen, which successfully slows the progression of ALS in the small proportion of people living with the inherited form of the disease who have the SOD-1 gene mutation.
While this development is cause for hope, 95% of us living with ALS do not have this gene mutation. Where do we turn to next? Bolstered investment to fuel continued efforts is undeniably key to advancing where we are today.
The dilemma before us
That said, we have found ourselves at an unhelpful crossroads. Given the high rate of failure in ALS drug trials over the years, the impression is that investing in this space is high risk and low return.
However, AI is transforming this dynamic. The application of AI to the drug discovery process promises to radically improve accuracy and timeframes for drug development – and has the most transformational potential in diseases that are highly complex, such as ALS.
I firmly believe that AI has the power to materially alter the economics of innovation for ALS, by finding and validating high-potential drug targets at speed that will de-risk and attract investment from the pharmaceutical industry. It’s for this reason that Challenge Works launched the Longitude Prize on ALS last year: an international challenge prize to incentivise the use of AI-based approaches to transform drug discovery for the treatment of ALS.
To put this belief to the test and truly understand the economic value of developing a successful treatment for ALS – beyond the incalculable value of improving the lives of those living with the disease – the Longitude Prize on ALS recently partnered with the Office of Health Economics (OHE).
The story the research reveals is powerful: investment in drug treatments for ALS should be embraced. The value that could be unlocked for both economies and drug developers around the world is huge.
If advances in detection and treatment could effectively eradicate ALS, the total value generated over 10 years would exceed $143 billion across seven key markets: the UK, US, Canada, Australia, Italy, Germany and France.
Depending on assumptions around treatment efficacy, the commercial value of a successful treatment over a decade could reach $26-40 billion in the US alone, around $1.7 billion in the UK, and between $1.1 and $4 billion in each of the other major markets studied.
It’s time to evolve the rhetoric of ALS investment being high-risk and instead view this space as one that presents glaring opportunities – both commercially and morally. This is also a chance to put recent technologies to the test – namely AI and in-silico analysis.
What’s more, success here will lay firm foundations for the future of neurodegenerative disease treatments more broadly, given that there is significant genetic and mechanistic overlap with ALS and other diseases, such as frontotemporal dementia. Any future treatments for ALS are therefore likely to find a wider market.
The opportunity to make a difference
This Spring, the Longitude Prize on ALS will announce its 20 Discovery Award winners. The teams of biomedical and AI scientists, researchers and innovators will be awarded £100,000 each to identify new, high-potential therapeutic targets and progress their research.
In 2027, ten teams will progress to a second stage, receiving a further £200,000 to build the evidence base for their proposed therapeutic 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 greatest therapeutic potential.
I want to send a clear message to those looking to invest in this space: ALS is one of the most dynamic areas for translational research and presents a compelling case for strategic investment.
This will take collaboration among key players across the entire ecosystem – from scientists, to AI innovators, investors, biomedical researchers, pharmaceutical companies and beyond.
The understanding of the SOD-1 gene came from one paper. Thanks to the collaboration between leading neuroscientists and neurologists across the world, we now have tofersen. Identifying a gene link with inherited forms of ALS in 1993 has led us to a moment in time where the five per cent of ALS patients that have a mutant SOD-1 gene can be successfully treated.
That’s the power of global collaboration – and an example of what’s possible if we all work together towards a shared goal.
OHE’s pivotal research is a compelling economic case for reaching this goal. For too long, ALS has sat at the fringes of investors’ priorities – but it presents a $143 billion opportunity to transform the lives of those living with the disease, as well as the many unnamed heroes who provide hours of unpaid care.
To read OHE’S The commercial case for investing in ALS in full, follow this link.
