David Fajgenbaum was a third-year medical student when he was diagnosed with the rare Castleman disorder, which causes the immune system to attack vital organs. He nearly died five times before he discovered that sirolimus, a drug used to prevent transplant patients from rejecting a new kidney, might keep his disease in check. 

He hadn’t responded to the only drug in development for Castleman Disease. “I knew I didn’t have a billion dollars and ten years to create a new drug from scratch,” he said. “If I wanted to survive, I would need to find an existing drug that I could repurpose for my disease.”

Sirolimus worked. He has been in remission for 10 years, and takes the drug every day. And he has spent the decade helping to match patients with rare diseases with drugs that are already in pharmacies for other conditions, leveraging existing knowledge. Over the past decade, he and his colleagues have found 17 drugs that could be repurposed, mainly for treating cancers and inflammatory diseases.

In 2022, he helped found a nonprofit called Every Cure, which aims to help the 300 million people globally who are battling diseases with no approved treatments by unlocking the full potential of every existing drug to treat every disease it possibly can.

With funding from the federal Advanced Research Projects Agency that could surpass $48 million, Every Cure will build a drug-repurposing database and algorithms, ML/AI-enabled Therapeutic Repurposing In eXtended uses (MATRIX), that patients, doctors and researchers can use to find drugs for untreated diseases. 

It will be a comprehensive, open-source data engine to generate predictive efficacy scores for all 3,000 FDA-approved drugs against all 12,000 diseases and rapidly advance treatments to patients. While many diseases share common mechanisms and can benefit from the same drugs, says Every Cure, approximately 9,000 diseases affecting millions of people do not have any approved treatments. Given that it takes between $1 and 2 billion and 10 to 15 years to develop a single new FDA-approved drug, it is faster and cheaper to repurpose safe, widely available drugs.

“I’m alive thanks to a repurposed drug that I discovered when I was dying from a rare disease during medical school,” Dr. Fajgenbaum said at the Philadelphia Citizen of the Year ceremony earlier this year. “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.”

The MATRIX project builds on previous work led by the National Center for Advancing Translational Sciences (NCATS) Biomedical Data Translator Program to structure biomedical data and perform targeted queries. In 2023, Every Cure partnered with David Koslicki, an NCATS funded investigator at Penn State University, and other members of the Translator Program, to generate pilot rankings for all 3,000 FDA-approved drugs to treat all human diseases. One of the top hits was for a drug that was recently used to save a patient’s life.

Every Cure is working to identify 100 potential treatment opportunities in the next five years and advance 25 into further research, including clinical trials.

“Rather than the current, one-step-at-a-time drug discovery process, we have an opportunity to use artificial intelligence to rapidly understand how already approved drugs could be effective against other diseases,” said ARPA-H Director Renee Wegrzyn. “Through this project, we hope to unlock the full potential of existing medicines to quickly and safely bring therapies to people with rare or currently untreatable diseases.” 

Within the last year, Every Cure has received funding from leading organizations such as the Chan Zuckerberg Initiative, Flagship Pioneering, Elevate Prize Foundation, and Lyda Hill Philanthropies. With government and philanthropic support, it is seeking partnerships with healthcare organizations that may be open to sharing data and well-positioned to bring cures to patients who need them most.

“Society’s greatest life-saving resource is the trove of medicines we already have, says Dr. Grant Mitchell, co-founder and CEO of Every Cure. “Leveraging a systematic, disease agnostic approach for drug repurposing enables us to rapidly unlock new diseases they can treat and confirm the validity of AI discoveries in patients. We’re excited to make our research tools available to the public, advancing data-driven discovery and offering hope to patients worldwide.” 

Earlier this year, Every Cure was one of five winning startup companies of the first Cure Xchange Challenge: Health AI for Good, designed to foster interdisciplinary collaborations that will help AI accelerate the transition of health systems from ‘sick care’ to ‘well care’.

Cure designed the challenge to incubate innovations that leverage AI across sectors and disciplines to accelerate basic research, diagnose conditions, and develop novel treatments. Cure partnered with MIT Solve, and an independent advisory board of healthcare experts selected both the winners and finalists.

The winners receive one-year residencies at Cure, unrestricted seed money, mentoring, in-kind resources and support services from Cure’s partners, such as Amazon Web Services, ThermoFisher and NVIDIA.

“AI-driven tools have the potential to be a game changer in improving human health and solving health inequities that persist in underserved communities,” said Seema Kumar, Chief Executive Officer of Cure. “This innovative group of Challenge winners is pioneering the future of healthcare, where AI responsibly empowers us to create and deliver equitable, accessible and transformative care for all. We welcome these trailblazing companies into the Cure community and look forward to helping their solutions thrive and grow to successful ventures.”

I was really struck by something Dr. Fajgenbaum said to PBS.

“You know, I’m not supposed to be here. I was supposed to die many years ago from a disease that didn’t have treatments available for it. And I’m only alive today because we figured out that a drug that was made for something else could save my life. And so all I can think about all day long is how many more of these drugs are just sitting out there at your neighborhood CVS, at your neighborhood Walgreens that are just waiting to be matched to patients who are suffering.”

Sources:

This Doctor Found His Own Miracle Drug. Now He Wants to Do It for Others Wall Street Journal, Feb. 28, 2024

Every Cure to Receive $48.3M from ARPA-H to Develop AI-Driven Platform to Revolutionize Future of Drug Development and Repurposing Every Cure, Feb. 28, 2024

Every Cure Wins Cure. Xchange Challenge: Health AI for Good Every Cure, Feb. 22, 2024

Every Cure Co-Founder Dr. David Fajgenbaum Honored as Philadelphia Citizen of the Year to Commemorate 10 Years in Remission After Discovering a Cure for His Deadly Disease Every Cure, Jan. 31, 2024

UPenn professor receives $48.3 million to find new treatments in old drugs. WHYSS PBS, Mar. 10, 2024