Amyotrophic lateral sclerosis, or Lou Gehrig’s disease, is the most common form of motor neurone disease (MND), with more than 5000 people in the United States being diagnosed with MND annually.
Motor neuron damaged by Amyotrophic Lateral Sclerosis (ALS) - 3d illustration closeup view | Image Credit: © LASZLO - stock.adobe.com
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).
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.
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.”
As Dyson mentioned, a treatment from Biogen Netherlands, Qalsody (tofersen), was recommended by the European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use for marketing authorization in February 2024 (3). The treatment, an antisense oligonucleotide that binds to the messenger RNA of the superoxide dismutase 1 (SOD1) gene, reduces the production of SOD1 protein, which leads to an improvement of ALS symptoms, according to EMA.
The Longitude Prize will initially bestow 20 applicants “Discovery Awards” of approximately $135,000 each (1). Teams will be judged on the potential of their approach and its translation into drug discovery, with progressive monetary tiers for teams whose research advances, and one winning team eligible to earn up to $1.3 million by 2031.
The entry window is open through Dec. 3, 2025, with the initial group of 20 successful applicants announced in the first half of 2026. Entries can be submitted at this link.
1. Challenge Works. Longitude Prize on ALS Launches to Award $10 million to AI Drug Target Discoveries for Most Common Form of MND. Press Release. June 25, 2025.
2. Lutz, J. MLB Commemorates Fifth Annual Lou Gehrig Day. MLB.com, May 31, 2025 (accessed June 24, 2025).
3. EMA. New Treatment for Rare Motor Neurone Disease Recommended for Approval. Press Release. Feb. 23, 2024.
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