Life saved: AI discovers existing drug works for rare disease

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artificial intelligence AI

A patient’s life was saved after researchers at the University of Pennsylvania, Castleman Disease Collaborative Network, Medidata, and Every Cure, used artificial intelligence to use an existing drug to treat idiopathic multicentric Castleman disease (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.

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. 

Albert, a 50-year-old iMCD patient who exhausted all known treatments, was prescribed adalimumab by Luke Chen from the University of British Columbia after consultation with David Fajgenbaum and his team from Every Cure, who had identified the potential benefit of this medication using an AI-guided proteomics approach.

“After a two-year battle with iMCD, I was unfortunately out of options, causing my family and me to prepare for hospice care,” Albert said. 

The Every Cure team identified the treatment just as Albert began to exhibit the worst symptoms in his two-year battle with iMCD. Within a few days of taking adalimumab, his organs regained function, his symptoms subsided, and he went into remission. The use of adalimumab to treat iMCD would be a novel application.

Monoclonal antibody

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 (FDA) in 2002. Treating iMCD with adalimumab is not a currently approved use. With the groundbreaking discovery, Every Cure and its research partners are beginning to lay the groundwork for conducting clinical trials to further validate its use for iMCD.

Scoring existing 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, including David Koslicki, Alex Tropsha, and Chris Bizon 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.

Every Cure is a non-profit seeking immediate funding to further optimize its algorithms and evaluate its highest-ranked results to bring directly to patients. It is also seeking partnerships for data contributions. 

Every Cure excited about potential future impact

“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 Every Cure co-founder and CEO, Grant Mitchell. 

“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. Our mission is to offer options to patients who have otherwise run out of hope. We do that by taking on the responsibility of ensuring that every drug is used to treat every disease it possibly can. It’s a huge responsibility but desperately needed and we are positioned to do it.”

Every Cure co-founder and director of the Center for Cytokine Storm Treatment & Laboratory (CSTL), Fajgenbaum, announced this significant breakthrough at the STAT Summit in San Francisco.

“I’m alive thanks to a repurposed drug that I discovered when I was dying from a rare disease during medical school. Now, I’m on a mission to unlock every cure so that no patient is told we’ve tried everything when a life-saving cure is sitting on the pharmacy shelf,” Fajgenbaum stated.

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