ARPA-H Takes on Rare Diseases

MATRIX, Short for ML/AI-Aided Therapeutic Repurposing in eXtended uses

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. 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.

• Every Cure seeks expert insights through an online form. These insights are pivotal in recognizing untapped potentials, hidden connections, and unexplored avenues within the realm of drug development.
• Experts’ specialized understanding of drug mechanisms, disease pathways, and medical research methodologies guides our decision-making process, reducing uncertainties and increasing the likelihood of success are encouraged to contribute at https://everycure.org/we-need-your-expert-insights/

Please note that any drug repurposing ideas or suggestions submitted through our website are considered non-confidential and non-proprietary. By submitting your ideas, you agree that these submissions do not contain intellectual property that you or a third party own or control. Every Cure does not claim ownership of your submissions; however, by providing your ideas, you grant us an unrestricted, worldwide, royalty-free license to use, reproduce, and distribute the ideas for the purpose of research, analysis, and furtherance of our mission. We appreciate your contributions to our efforts in advancing healthcare innovation.

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.

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.”