Vanderbilt NeuroCline, a consortium of researchers from Vanderbilt Health and Vanderbilt University, has received a prestigious award to explore using artificial intelligence to find new drug targets for amyotrophic lateral sclerosis (ALS), the most common form of motor neuron disease (MND).
The Longitude Prize on ALS is a £7.5 million global challenge prize that rewards cutting edge AI-based approaches to transform drug discovery to treat ALS. Following a worldwide call to action in June 2025, almost 100 teams representing the world’s leading universities, technology giants, advanced medical research organizations and AI pioneers entered the prize.
Vanderbilt NeuroCline was among the 20 entrants recently selected to receive “Discovery Awards” of £100,000 each, based on their potential to use AI to identify and validate drug targets that aim to drive understanding of the disease and support future drug discovery. The awardees include King’s College London (UK) in partnership with GlaxoSmithKline and Bielefeld University (Germany), Aperture Therapeutics (USA) in partnership with Harvard Medical School and Tufts University (USA), and the National University of Singapore and Stockholm University (Sweden) and The University of Pennsylvania (USA).
Vanderbilt NeuroCline is a collaboration led by Veronique Belzil, director of the Vanderbilt ALS Research Center at Vanderbilt Health, in partnership with Bennett Landman, director of the Vanderbilt Lab for Immersive AI Translation (VALIANT) at Vanderbilt University. Belzil is a leader in ALS genetics and translational biology, and Landman is an expert in large-scale AI and biomedical data systems. The team brings together complementary strengths in disease biology and advanced computational modeling.
The team’s project is structured around milestones that progressively transform raw whole-genome, epigenomic, and transcriptomic data into biologically interpretable, therapeutically actionable ALS targets. These milestones create a systematic pipeline from genome to target, enabling the identification of biologically informed therapeutic opportunities in ALS, a progressive neurodegenerative disease that damages the motor neurons in the brain and spinal cord.
ALS is a fatal neurodegenerative disease that can affect adults at any age, although it is most commonly diagnosed in mid- to late adulthood. In the United States, an estimated 1 in 400 people will develop ALS during their lifetime.
“In NeuroCline, we combine genomic and epigenomic signals with AI to pinpoint higher-confidence ALS targets, reducing false leads before they even reach the lab,” Belzil said. “Through this prize, we’re uniting biological and AI expertise across Vanderbilt to bridge data silos and decode the genomic and epigenomic complexity of ALS to drive more effective therapeutics.”
Said Landman, “I am excited to partner with Dr. Belzil and her newly launched Vanderbilt ALS Research Center on this high risk, high reward exploration to find a cure for ALS.”
Vanderbilt NeuroCline now has access to the largest and most comprehensive ALS patient dataset of its kind, combining multiple types of biological information which have not been available in one place previously – including the genomic sequences of 9,000 ALS patients and epigenomics, transcriptomics, and proteomics data for over 2,000 cases.
Next year, 10 teams will progress to a second stage, receiving an additional £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 and validating the target with the strongest evidence of therapeutic potential.