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The Advanced Research Projects Agency for Health (ARPA-H) is allocating up to $48 million to the non-profit Every Cure in Philadelphia for repurposing approved drugs for rare diseases. The award includes the potential for additional funds to validate the most promising drug-disease matches in clinical trials.

“This project will harness AI to find answers and treatments for the 30 million Americans who are currently facing rare diseases,”  said Arati Prabhakar, PhD, director of the White House Office of Science of Technology Policy.

The ML/AI-Aided Therapeutic Repurposing In eXtended uses (MATRIX) project intends to build a machine learning platform to rapidly pinpoint and validate existing medications to treat diseases that currently have no therapies. For the more than 10,000 known rare diseases, today only a few hundred have safe, effective treatments. 

Every Cure works with,  “…partners in medicine, pharma, tech, and philanthropy, we are building out a comprehensive, open-source database of drug-repurposing opportunities.”

The non-profit  points out, on its website, that, “300M people suffer from 9,000 diseases without any approved treatments, and underserved communities suffer the most. In fact, one in 10 of us will develop a disease with no treatments! While many of these diseases could be treated with drugs sitting on the local pharmacy’s shelf.” 

But drug repurposing, they add, typically happens by chance. This project aims to change that.

“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, PhD. “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.”  

While a rare disease is defined as impacting less than 200,000 people in the United States, in total, these conditions impact 25 to 30 million, or one in ten Americans. If successful, MATRIX will develop an open-source platform and interactive heatmap displaying predicted efficacy scores for approved drugs against other diseases, thereby greatly accelerating traditional drug discovery timelines and providing an avenue to scale discovery across a vast set of diagnoses. 

Every Cure will leverage ML/AI standards, knowledge graphs, and data models developed through the Biomedical Data Translator Program funded by the National Center for Advancing Translational Sciences, part of the National Institutes of Health. 

Wegrzyn added, “It’s exciting to consider the potential of a systematic approach to identifying and validating the top repurposing candidates across all drug-disease combinations. The team will work towards developing new treatment options and improved health outcomes for more people, all within significantly reduced timelines.” 

This project is one of many solicited through ARPA-H’s Open Broad Agency Announcement (Open BAA), which “seeks transformative ideas for health research or technology.”

“America’s health outcomes demand new innovation and action, which is exactly why President Biden started ARPA-H,” said Prabhakar. 

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