Approximately six weeks ago, David Fajgenbaum, a medical professor at the University of Pennsylvania, was contacted by the relatives of a man in Washington state battling a rare blood disorder known as POEMS syndrome.
Upon learning that the patient was on the brink of being moved to hospice care and that the medical team had exhausted their treatment options, Fajgenbaum, despite not specializing in POEMS, recommended three drugs commonly used for multiple myeloma, a condition akin to POEMS based on his organization Every Cure’s research.
In a bold move, the patient’s physicians opted to try Fajgenbaum’s suggested treatment regimen, which ultimately proved to be life-saving. The patient’s condition improved significantly, leading to his discharge from the hospital, marking a pivotal success in the experimental treatment.
This groundbreaking initiative is part of a larger endeavor spearheaded by Fajgenbaum, who recently secured $48.3 million in federal funding for MATRIX, an innovative AI-driven platform designed to match diseases with existing FDA-approved medications.
Fajgenbaum’s personal battle with Castleman Disease in 2010 served as the catalyst for Every Cure’s inception two years ago. Confronted with a lack of effective treatments, Fajgenbaum embarked on a quest to repurpose existing drugs for his condition after conventional therapies failed.
Through meticulous research and self-experimentation, Fajgenbaum identified the mTOR pathway as a crucial target in his disease and repurposed the drug sirolimus, traditionally used for organ rejection, with remarkable success, achieving a decade of remission in January.
Motivated by this triumph, Fajgenbaum and his team have since identified 17 repurposable drugs, primarily for cancer and inflammatory disorders, at a significantly accelerated pace compared to traditional drug development processes.
The development of an AI-driven platform by Every Cure aims to revolutionize the drug repurposing landscape by efficiently matching thousands of diseases with existing medications, a task beyond human capacity due to the vast number of potential combinations.
Supported by ARPA-H, a federal agency dedicated to advancing biomedical research, Every Cure’s platform is set to streamline the identification of potential drug-disease matches, offering a promising avenue for swift and cost-effective treatments.
While the program’s initial focus is on integrating diverse data sources and generating drug-disease scores, the ultimate goal is to identify optimal treatment candidates within a three-year timeframe, with plans to engage physicians, researchers, and patients in the process.
Fajgenbaum’s personal journey underscores the transformative potential of repurposing existing drugs, shedding light on the untapped therapeutic possibilities awaiting discovery in everyday pharmacies, poised to alleviate the suffering of countless individuals.
