Dr. David Fajgenbaum’s nonprofit research organization, Every Cure, has received a $48 million contract from the U.S. Advanced Research Projects Agency for Health (ARPA-H) to develop an AI-powered platform, MATRIX, for rare disease research. The platform aims to match existing, approved medicines with rare diseases that lack treatments. The funding will allow Every Cure to scale their approach across diseases and pioneer new treatments. The organization’s work will build upon the Biomedical Data Translator created by the National Center for Advancing Translational Sciences (NCATS). The project could potentially cut years off the FDA approval process.
AI-Powered Research for Rare Diseases Receives Significant Funding
The U.S. Advanced Research Projects Agency for Health (ARPA-H) has recently awarded a three-year contract worth $48 million to Every Cure, a nonprofit research organization. The funding will be used to develop an AI-powered platform, MATRIX (ML/AI-enabled Therapeutic Repurposing In eXtended uses), which aims to match existing, approved medicines with rare diseases that currently lack treatments. The platform will utilize machine learning (ML) to expedite the research process.
Dr. David Fajgenbaum, the physician-researcher who founded Every Cure, believes that the key to treating many rare diseases may lie in the repurposing of existing medicines. Fajgenbaum himself found a cure for his own life-threatening rare disease, Castleman disease, in an already-approved medicine. The funding from ARPA-H will allow Every Cure to expand their approach across a wider range of diseases, potentially pioneering new treatments for millions of people worldwide who currently have no other options.
The Role of AI in Drug Repurposing
Every Cure’s AI work will build upon the Biomedical Data Translator created by the National Center for Advancing Translational Sciences (NCATS). This tool looks beyond symptom-based diagnoses to the molecular and cellular characterizations of disease, such as cell pathology. It collates biomedical data from various sources and creates knowledge models, enabling different biomedical and healthcare datasets to be searched and compared.
The use of AI in this context offers the opportunity to rapidly understand how already-approved drugs could be effective against other diseases. This approach could significantly expedite the drug discovery process, which traditionally proceeds one step at a time.
The Impact of Every Cure’s Approach
As part of the U.S.-funded research, Every Cure will undertake several key initiatives. These include the development of an open-source medicine repurposing database, the establishment of a portal for physicians, researchers, and patients to contribute repurposing ideas, and the public release of predictive efficacy scores for all medicines against all diseases. The organization will also select numerous medicines for advancement to address neglected diseases in neglected populations.
The approach taken by Every Cure could deliver quick progress and cut years off the Food and Drug Administration (FDA) approval process. Existing treatments have already been researched for safety as part of the clinical trial process, and launching a new medicine, from development to approval, can take 10 years or more.
ARPA-H’s Role in Funding Healthcare Projects
ARPA-H was established to fund healthcare projects, including rare disease research, that traditionally struggle to secure funding. By working with already approved products, ARPA-H aims to expedite the process of finding effective treatments for rare diseases. The agency’s recent funding of Every Cure’s AI-powered research is a testament to this commitment.
The Personal Journey of Dr. David Fajgenbaum
Dr. David Fajgenbaum, one of the co-founders of Every Cure, has a personal connection to the mission of the organization. His own health was nearly destroyed by Castleman disease, a rare condition. However, after overcoming life-threatening complications, Fajgenbaum discovered a potential treatment in an existing, approved medicine. This experience inspired him to establish Every Cure and work towards finding more treatments for rare diseases.
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