A groundbreaking AI tool has saved the life of a patient suffering from idiopathic multicentric Castleman’s disease (iMCD), a rare and deadly condition with limited treatment options. The AI, after analyzing over 4,000 existing medications, predicted that adalimumab, a monoclonal antibody already approved for conditions like arthritis and Crohn’s disease, could be an effective treatment for iMCD. This discovery, published in NEJM, is a potential game-changer for patients with rare diseases.
The research team, led by Dr. David Fajgenbaum from the Perelman School of Medicine, also uncovered that elevated tumor necrosis factor (TNF) levels were a key factor in the severity of iMCD. By targeting TNF with adalimumab, the patient—previously entering hospice care—has now been in remission for almost two years.
This success not only offers hope to iMCD patients but also demonstrates the vast potential of machine learning in drug repurposing. Drug repurposing is a process where existing medications are used to treat new conditions, which is particularly important for rare diseases with limited therapeutic options. Fajgenbaum, who also has iMCD, has long been a champion of this approach, having previously found his own life-saving treatment through repurposing.
The study emphasizes the potential of AI to repurpose existing drugs for rare diseases. This marks a new era where AI could analyze vast datasets to find effective treatments for conditions previously lacking options, potentially saving many lives in the future.
Article written by Frank Otto
05/02/2025
Source:
Penn Medicine