There are more than 10,000 known rare diseases and only a few hundred have safe, effective treatments. ARPA-H’s latest project aims to change that and deliver hope and improved outcomes to millions of Americans.
Introducing MATRIX. Short for ML/AI-Aided Therapeutic Repurposing in eXtended uses, MATRIX intends to build a machine learning platform to rapidly pinpoint and validate existing medications to treat diseases that have no therapies.
- The platform will be open-sourced and include an interactive heatmap displaying predicted efficacy scores for approved drugs against other diseases.
ARPA-H is allocating up to $48 million to the non-profit Every Cure in support of this research, with a potential for additional funds to validate the most promising drug-disease matches in clinical trials.
“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 Wegrzyn at the event. “It’s exciting to consider the potential of a systematic approach to identifying and validating the top repurposing candidates across all drug-disease combinations.”
Why ARPA-H? Rare diseases impact 25 to 30 million people, or one in ten Americans, yet each rare disease can impact anywhere from 1 to 200,000 people. As result, the limited market for rare disease treatments can repel investment in drug development. By funding Every Cure’s work in building this platform, ARPA-H aims to develop new treatment options where others have not.
“America’s health outcomes demand new innovation and action, which is exactly why President Biden started ARPA-H,” said Director of the White House Office of Science and Technology Policy Arati Prabhakar, Ph.D.
To learn more, visit the MATRIX press release and check out the Wall Street Journal’s piece on Every Cure, “This Doctor Found His Own Miracle Drug. Now He Wants to Do It for Others.”
