First Life Saved with AI-Discovered Repurposed Medicine for Castleman Disease with Significant Potential Applications to Treat Other Illnesses

A 50-year-old patient battling life-threatening Castleman disease was provided adalimumab as a treatment of last resort following its identification by AI-guided discovery.

Every Cure and its partners are expanding upon this discovery to harness a novel AI algorithm that calculates the potential use of every drug to treat every disease. Initial promising candidates for further study include bosutinib for ALS, metreleptin for anorexia, and folinic acid for autism spectrum disorder.

 

 

PHILADELPHIA, PENNSYLVANIA – May 4, 2023 – A patient’s life was saved after researchers at the University of Pennsylvania, Castleman Disease Collaborative Network, Medidata, a Dassault Systèmes company, and Every Cure, led by Dr. David Fajgenbaum, utilized AI to uncover a previously unknown use for an existing drug, adalimumab, 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. Al, a 50-year-old iMCD patient who exhausted all known treatments, was prescribed adalimumab by Dr. Luke Chen from the University of British Columbia after consultation with Dr. 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,” Al stated. The Every Cure team identified this treatment just as Al began to exhibit the worst symptoms in his 2-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.

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.

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. “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), Dr. David Fajgenbaum, announced this significant breakthrough at the STAT Summit in San Francisco today.

 

“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,” Dr. Fajgenbaum shared.

 

For further information or to express interest in partnering with Every Cure, please contact Brent Shaw ([email protected], 818-876-2691).

 

You can watch the full feature story on Al’s journey here.

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About Every Cure

Every Cure is a nonprofit organization dedicated to unlocking the full potential of every existing medicine to treat every disease possible. Repurposing existing drugs is the fastest and most efficient way to treat diseases with the greatest return on investment for saving lives. However, systemic barriers impede repurposing, so patients suffer while potential treatments are not fully utilized. Every Cure overcomes these barriers to systematically identify and advance promising repurposing opportunities and save lives. Every Cure is taking a revolutionary and disruptive approach to overcome these systemic barriers by developing a comprehensive, open-source data engine to generate predictive efficacy scores for all 3,000 drugs against all 12,000 diseases and rapidly advance treatments to patients. In partnership with academia, industry, and government, Every Cure is integrating proprietary and public data into a comprehensive dataset, optimizing AI ranking algorithms, validating the most promising drug-disease matches, performing trials, and ensuring patient access to effective therapies. Every Cure announced its launch in September 2022 at the Clinton Global Initiative.

 

For more information, please visit EveryCure.org or connect with us on Twitter, LinkedIn, Facebook, and Instagram.

About Every Cure’s Founders

Dr. David Fajgenbaum, Co-Founder, Every Cure

David Fajgenbaum, MD, MBA, MSc, is a physician-scientist at the University of Pennsylvania, co-Founder of Every Cure, co-Founder & President of the Castleman Disease Collaborative Network, and national bestselling author of ‘Chasing My Cure: A Doctor’s Race to Turn Hope Into Action.’ He is also a patient battling a deadly disease called idiopathic multicentric Castleman disease (iMCD), which he discovered a treatment for that is saving his life and others. Through his work at the University of Pennsylvania’s Center for Cytokine Storm Treatment & Laboratory, he has also identified and advanced 14 other treatment approaches for iMCD and cancer. He also serves on the Board of Directors for the Reagan-Udall Foundation for the FDA. Fajgenbaum has published scientific papers in high-impact journals such as the New England Journal of Medicine, Journal of Clinical Investigation, and The Lancet, been recognized with awards such as the 2016 Atlas Award which he received along with then Vice President Joe Biden, and has been profiled in a New York Times cover story as well as by Good Morning America, CNN, Forbes 30 Under 30, and the Today Show. Dr. Fajgenbaum earned a BS from Georgetown University, MSc from the University of Oxford, MD from the University of Pennsylvania, and MBA from The Wharton School.

Dr. Grant Mitchell, Co-Founder and Chief Executive Officer, Every Cure

Grant Mitchell, MD, MBA, is Co-Founder and CEO of Every Cure. He is a seasoned entrepreneur, operator and investor in the areas of health, technology, and machine learning. He has launched multiple health tech companies and operated in the CEO role in life science and tech organizations. Previously, Dr. Mitchell was a Principal in Analytics at McKinsey & Company’s artificial intelligence division, QuantumBlack. There he led teams that pioneered the use of machine learning algorithms and real world evidence databases to predict health outcomes in subpopulations by drug type. Dr. Mitchell was an integral part of the effort to support Dr. Fajgenbaum in the search for the cure of his own rare disease, and further collaborated with Dr. Fajgenbaum on the CORONA project. Every Cure is the culmination of Dr. Mitchell and Dr. Fajgenbaum’s experience in rare disease research, drug repurposing, and advanced analytics. Dr. Mitchell earned a BA in economics from New York University, MD from the University of Pennsylvania, and MBA from the Wharton School.

Tracey Sikora, Co-Founder, Every Cure

Tracey Sikora studied repurposed drugs in rare diseases, including Niemann Pick C, MPS I, Familial Hypercholesterolemia, and others. She led clinical trials by working alongside partners at biotech companies and rare disease organizations, developing study protocols that may adapt to the challenging realities of conducting clinical research in the real-world. Inspired by the innovative clinical trials launched for COVID-19 research, Tracey is bringing bold plans for efficient, well-executed clinical trials to our team. Tracey has a BS from Cornell University.

About the Castleman Disease Collaborative Network

The Castleman Disease Collaborative Network (CDCN) is a global nonprofit organization dedicated to accelerating research, treatment, and patient care for Castleman disease (CD), a rare and often deadly disease that presents with a broad range of symptoms similar to autoimmune disorders and cancers. The CDCN’s groundbreaking approach, which includes a highly collaborative research model and a focus on repurposing existing medications approved for other diseases, has saved thousands of lives and become the exemplar for rare disease research. The organization was established in 2012 by David Fajgenbaum, MD, MBA, MSc, FCPP, and Frits van Rhee, MD, PhD, and is based at the University of Pennsylvania, where Dr. Fajgenbaum―a physician-scientist and CD patient―has made it his life’s mission to cure this disease, and revolutionize biomedical research to cure countless other diseases. More information is available at cdcn.org.

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