RARE Daily

Every Cure Says First Life Saved with AI-Discovered Repurposed Medicine for Castleman Disease

July 13, 2023

Rare Daily Staff

Every Cure, the nonprofit using artificial intelligence to identify drugs to repurpose to treat patients with rare diseases, said for the first time it was able to use AI to identify an approved drug to save the life of a dying patient.

Researchers at the University of Pennsylvania, Castleman Disease Collaborative Network, Medidata and Every Cure, led by David Fajgenbaum, utilized AI to uncover a previously unknown use for an existing drug, adalimumab, to treat a 50-year-old man with idiopathic multicentric Castleman disease (iMCD). Adalimumab is marketed under the brand name Humira.

iMCD is a rare and life-threatening disorder that involves hyperactivation of the body’s immune system which causes uncontrolled organ dysfunction. The only FDA-approved treatment works in a portion of those diagnosed with iMCD, leaving the majority of patients with limited options. The patient, who had exhausted all known treatments, was prescribed adalimumab by his physician at the University of British Columbia after consultation with Fajgenbaum and his team from Every Cure, who had identified the potential benefit of this medication using an AI-guided proteomics approach.

Within a few days of taking adalimumab, the man’s organs regained function, his symptoms subsided, and he went into remission. The use of adalimumab to treat iMCD would be a novel application.

Adalimumab is a monoclonal antibody typically used to treat severe and often life-debilitating inflammatory conditions such as rheumatoid arthritis, juvenile idiopathic arthritis, psoriatic arthritis, and Crohn’s disease, among others. It is administered via an injection under the skin. Adalimumab was initially approved by the U.S. Food and Drug Administration in 2002.

Treating iMCD with adalimumab is not a currently approved use. With the discovery, Every Cure and its research partners are beginning to lay the groundwork for conducting clinical trials to further validate its use for iMCD.

Every Cure and its partners are developing medical databases and AI algorithms to unlock new uses for existing medicines across all diseases, and the latest scoring identified adalimumab as the most promising treatment for iMCD out of all 3,000 approved drugs.

To help scale this case study for other potential uses, Every Cure, a non-profit organization dedicated to unlocking the full potential of approved medicines to treat additional diseases, has partnered with researchers at the University of Pennsylvania, Penn State University, and the University of North Carolina to develop an AI-powered Every Cure LinkMap. The algorithm scores every existing drug’s potential to treat every known disease based on worldwide knowledge from publications and databases.

The Every Cure LinkMap has initially ranked all 3,000 FDA-approved drugs to treat all 12,000 human diseases, generating 36 million evaluations. Every Cure is currently analyzing the top results from its initial findings in its Every Cure LinkMap, in addition to consulting industry experts, to identify the most promising candidates for repurposing, including opportunities such as the use of metreleptin for anorexia, folinic acid for autism spectrum disorder, anakinra for sepsis, and bosutinib for ALS. These candidates and others will be further evaluated and prioritized for future clinical trials.

“We’re incredibly encouraged by the progress we’ve achieved with limited resources that has already saved the lives of patients diagnosed with Castleman Disease, angiosarcoma, and COVID-19 by repurposing existing medicines,” said Grant Mitchell, co-founder and CEO of Every Cure. “However, we’re even more excited about the potential future impact as we integrate more data, optimize the algorithms, and investigate our most promising leads.”

Photo: Grant Mitchell, co-founder and CEO of Every Cure

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