New, worldwide approaches based on artificial intelligence (AI) technology, that are on the cutting edge of drug discovery for the treatment of amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease), could be eligible for more than $10 million with the June 25, 2025 announcement of the Longitude Prize on ALS, which is principally funded by the Motor Neurone Disease Association and designed and delivered by Challenge Works, with support from Nesta and other stakeholders (1).
Why AI?
Key Takeaways
- The Longitude Prize on ALS will award over $10 million to AI-driven drug discovery projects targeting ALS and other forms of motor neurone disease.
- AI advancements are helping researchers analyze vast patient data sets, aiming to accelerate treatment development for ALS, a complex and progressive disease.
- Biogen’s Qalsody (tofersen), targeting SOD1-related ALS, shows promise; the prize seeks to foster similarly innovative, scalable therapeutic approaches.
According to a press release, advancements in AI are making it easier for innovators to unlock massive quantities of patient data generated in the past decade, giving them a chance to “outpace” disease progression (1).
ALS is the most common form of motor neurone disease (MND) (1). It is a progressive disease that damages nerves in the brain and spinal cord, also known as the motor neurons. Eventually, signals from the brain cease to reach the muscles, causing those muscles—including the ones used to swallow and breathe—to degenerate.
Challenge Works says there is a 1 in 300 chance that a person will develop MND in their lifetime, with approximately 90% of those cases being ALS (1). Approximately 30,000 people in the United States are living with MND at a given time, with more than 5000 cases diagnosed annually.
‘The luckiest man...’
A public face was given to ALS in 1939, when star New York Yankees first baseman Lou Gehrig was diagnosed with the disease, forcing him to end his then-record streak of 2130 consecutive games played (2). Gehrig delivered his now-famous “luckiest man on the face of the Earth” speech to the Yankee Stadium crowd on July 4 of that year, succumbing to the disease less than two years later.
“ALS is astonishingly complex, which is why it has been so difficult to develop treatments that truly fight this hideous disease. Tireless fundraising in the last decade has created a wealth of data on ALS that just didn’t exist before, and we are at a turning point. In the last year, tofersen, the first drug treatment to show real promise for people with the very rare inherited form of MND (affecting around 2% of patients), shows that the disease is no longer a black box that we cannot penetrate,” Tris Dyson, managing director at Challenge Works who was diagnosed with ALS in 2023, said in the press release (1). “We are now on the right path for treatments for all MND patients—including those of us living with ALS. The real game-changer, though, is the rapid advancement of AI. It means we can turn the path into a superhighway. Never before have we had the power to unlock the complexity of MND, and in particular ALS, and accelerate along the road to long-term treatments, and, I hope one day, a cure.”