By Kate Morgan March 21, 2025 — 9.16am
A little over a year ago, Joseph Coates was told there was only one thing left to decide: Did he want to die at home, or in the hospital?
Coates – then 37 and living in Renton, Washington state, in the north-west of the US – was barely conscious. For months, he had been battling a rare blood disorder called POEMS syndrome, which had left him with numb hands and feet, an enlarged heart and failing kidneys.
Every few days, doctors needed to drain litres of fluid from his abdomen. He became too sick to receive a stem cell transplant, one of the few treatments that could have put him into remission.
Dr David Fajgenbaum (left) found a lifesaving drug regimen for Joseph Coates (right) that AI suggested. Credit: New York Times
“I gave up,” he said. “I just thought the end was inevitable.”
But Coates’ girlfriend, Tara Theobald, wasn’t ready to quit. So she sent an email begging for help to a doctor on the other side of the country named David Fajgenbaum, whom the couple met a year earlier at a rare disease summit.
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By the next morning, Fajgenbaum had replied, suggesting an unconventional combination of chemotherapy, immunotherapy and steroids previously untested as a treatment for Coates’ disorder.
Within a week, Coates was responding to treatment. In four months, he was healthy enough for a stem cell transplant. Now, he’s in remission.
The lifesaving drug regimen wasn’t thought up by the doctor – or any person. It had been spat out by an artificial intelligence model.
In labs around the world, scientists are using AI to search among existing medicines for treatments that work for rare diseases. Drug repurposing, as it’s called, is not new, but the use of machine learning is speeding up the process – and could expand the treatment possibilities for people with rare diseases and few options.
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Thanks to versions of the technology developed by Fajgenbaum’s team at Philadelphia’s University of Pennsylvania and elsewhere, drugs are being quickly repurposed for conditions including rare and aggressive cancers, fatal inflammatory disorders and complex neurological conditions. And often, they’re working.
The handful of success stories so far have led researchers to ask: How many other cures are hiding in plain sight?
There is a “treasure trove of medicine that could be used for so many other diseases. We just didn’t have a sy
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