Huge advances have been made in our understanding of neurodegenerative diseases – but ALS still has no cure and new investment is urgently needed. By Dr Amanda Cole, Director, Office of Health Economics (OHE).
LS, the most common form of motor neurone disease (MND), is devastating. Signals from the brain stop reaching muscles – and eventually those living with the disease are unable to swallow food, drink or breathe. There is a 1 in 300 chance that a person will develop MND in their lifetime, but because of its complexity, it is currently incurable.
The burden of ALS is particularly stark when measured in quality-adjusted life years (QALYs). In the UK, an individual diagnosed with ALS is expected to lose an average of 12.6 QALYs over their lifetime. Few diseases carry such a heavy toll in such a short period.
Beyond the immense personal devastation experienced by those diagnosed, ALS also places huge pressure on healthcare systems, families and informal carers. In the UK alone, medical management of ALS costs an estimated £133 million each year; in the US, this figure is around $2.5 billion. When wider societal costs such as lost productivity and unpaid caregiving are included, these total costs roughly triple.
Despite this, ALS has remained at the margins of biomedical investment – perceived until now to be too complex, rare and risky to justify large-scale investment. But things are changing.
Advances in AI, a much better understanding of the disease biology, and the availability of unprecedented volumes of patient data mean that we find ourselves at a turning point for uncovering potential new treatment pathways. Now, the question facing investors within the biopharmaceutical industry is not whether progress is possible, but to what extent we are ready to seize the opportunity to move closer to a cure.
On behalf of The Longitude Prize on ALS, we at the Office of Health Economics (OHE) recently undertook research to quantify the value of a successful treatment for ALS in order to truly understand the size of this opportunity.
What we found is striking. If advances in detection and treatment could effectively eradicate ALS, the total value generated over 10 years would exceed $143 billion across the seven key markets that we studied: 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.
These figures send a strong message: we must reshape how ALS is viewed by investors and the wider biomedical innovation ecosystem.
Rather than being a disease characterised by high risk and low investment, ALS may offer biopharmaceutical companies a strategic investment opportunity. The characteristic rapid progression of ALS enables shorter and more cost-effective clinical trials; approved treatments – even those offering modest benefit – have historically seen fast uptake; novel platform trials allow multiple therapies to be tested efficiently; and orphan drug status accelerates regulatory approval.
Crucially, the genetic, pathological and clinical overlap between ALS and other neurodegenerative diseases means that advances in ALS treatment may serve as proof-of-concept for technologies relevant to diseases like Parkinson’s, Huntingdon’s and some forms of dementia, thereby unlocking broader opportunities.
Success in identifying effective treatments for ALS would not stand alone, but would represent the first skittle to fall in the fight to accelerate progress across the wider neurodegenerative disease landscape. Viewed through this lens, the commercial opportunities are boundless.
The timing of our economic research could not be more opportune. ALS research is benefiting from an uptick in funding, a growing understanding of disease mechanisms, and expanding access to large-scale datasets. Additionally, AI and in-silico technologies – well-suited to ALS drug target discovery – are transforming how potential therapies are identified and tested, dramatically reducing time and cost.
The Longitude Prize on ALS has recognised this momentum. Launched in June 2025, the prize, run by Challenge Works, is incentivising and rewarding cutting-edge AI-based approaches to transform drug discovery for the treatment of ALS.
Principally funded by the MND Association, and backed by an impressive fleet of additional funders across the globe, the prize is an ambitious signal of intent: one we hope will catalyse investment and collaboration.
It’s clear that the human cost of inaction against ALS is immense, and as we at OHE have shown, the economic case for progress is compelling. With the right investment, tools and partnerships, ALS is well-placed to become a story of success – at long last.
