This Doctor Found His Own Miracle Drug. Now He Wants to Do It for Others.
Kaila Mabus, an athletic teenager in the Chicago area, went to the emergency room in 2019 in renal failure. It took another month before she was diagnosed with Castleman disease, a rare disorder that causes the immune system to attack vital organs. The Food and Drug Administration has approved just one drug to treat Castleman. It didn’t help Kaila. Her parents feared she might die.
Her doctors sought help from Dr. David Fajgenbaum, an immunologist at the University of Pennsylvania and fellow Castleman patient who studies the disease. He has matched rare-disease patients with drugs that are already in pharmacies for other conditions for over 10 years, starting with himself.
Fajgenbaum said that just two weeks earlier, his lab experiments had suggested the drug ruxolitinib, approved to treat a blood cancer, might work in some Castleman patients. Kaila started taking it twice a day, and it put her in remission. Kaila, now 18, plans to study nursing at Marquette University in Milwaukee in the fall.
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Every Cure’s database will use artificial intelligence to rank FDA-approved drugs that are most likely to work against every known disease. The rankings will be based on what Fajgenbaum calls “the world’s biomedical knowledge”: studies, case reports, clinical-trial data, health records and adverse-event reports that contain tantalizing clues that haven’t been systematically mined to help many patients. Every Cure will also use the money to identify and advance at least 25 potential treatments into clinical trials or to gather enough data to persuade doctors to prescribe them to patients.
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It will take about two years to build the database and publish the scores, Fajgenbaum says. Meanwhile, Every Cure will keep making matches. The organization is soliciting more ideas from doctors and patients like Kaila.
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