RARE Daily

ARPA-H Awards $48.3 Million Contract to Every Cure to Develop AI-Driven Platform for Drug Repurposing

February 29, 2024

Rare Daily Staff

The Advanced Research Projects Agency for Health has awarded a three-year, $48.3 million contract to Every Cure to accelerate the nonprofit’s mission to unlock the full potential of existing drugs to treat more diseases through the development of an AI-powered platform drug repurposing.

Established in 2022 by the Biden-Harris Administration, ARPA-H is a new federal agency that aims to advance high-potential, high-impact biomedical and health research that cannot be readily accomplished through traditional research or commercial activity. The announcement was made during a rare disease day event hosted by the White House.

“While Ozempic is a recent example of drug repurposing, the concept is not new,” said David Fajgenbaum, co-founder and president of Every Cure and associate professor of medicine at the University of Pennsylvania. “This funding comes at a pivotal time, allowing us to scale our approach across diseases to pioneer new treatments for millions of people around the world without options.”

Every Cure’s platform called MATRIX, an acronym for ML/AI-enabled Therapeutic Repurposing In eXtended uses, enables Every Cure to look across all drugs and all diseases simultaneously and then quantifies the strength of those connections to find the most promising new treatments to help patients.

The MATRIX project builds upon previous work led by the National Center for Advancing Translational Sciences Biomedical Data Translator Program to structure biomedical data and perform targeted queries. In 2023, Every Cure partnered with an NCATS funded investigator at Penn State University, David Koslicki and other members of the Translator Program, to generate pilot rankings for all 3,000 FDA-approved drugs to treat all human diseases. One of the top hits was for a drug that was recently used to save a patient’s life.

By leveraging novel AI techniques to analyze the world’s biomedical knowledge, Every Cure is working to identify 100 potential treatment opportunities in the next five years and advance 25 into further research, including clinical trials. Promising early leads include arginine for sickle cell disease, folinic acid for autism spectrum disorder, and bosutinib for ALS.

The funding from ARPA-H will be used to develop an open-source drug repurposing database; establish a portal for physicians, researchers and patients to contribute repurposing ideas; publicly release predictive efficacy scores for all drugs against all diseases; and select numerous drugs for advancement to address neglected diseases in neglected populations.

“Rather than the current, one-step-at-a-time drug discovery process, we have an opportunity to use artificial intelligence to rapidly understand how already approved drugs could be effective against other diseases,” said ARPA-H Director Renee Wegrzyn. “Through this project, we hope to unlock the full potential of existing medicines to quickly and safely bring therapies to people with rare or currently untreatable diseases.”

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