How AI Is Saving Lives By Repurposing Old Drugs

A recent report by The New York Times tells the story of Joseph Coates, 37, whose future seemed grim after doctors gave him only months to live.

Diagnosed with POEMS syndrome, a rare blood disorder, he was too ill to undergo a stem cell transplant—the only known treatment. “I gave up,” Coates said, as reported by The Times. “I just thought the end was inevitable,” he added.

But his girlfriend, Tara Theobald, refused to accept defeat. She reached out to Dr. David Fajgenbaum, a physician specializing in rare diseases, who suggested an unconventional treatment plan, as reported by The Times.

The lifesaving regimen wasn’t devised by Dr. Fajgenbaum—it was generated by an artificial intelligence model. Within weeks, Coates responded to the treatment, regained his health, and underwent a successful stem cell transplant. Today, he’s in remission, says The Times.

This breakthrough is part of a growing trend: scientists are using AI to repurpose existing drugs for rare diseases. Known as drug repurposing, this approach leverages machine learning to identify overlooked treatments among thousands of approved medications.

“There is a treasure trove of medicine that could be used for so many other diseases. We just didn’t have a systematic way of looking at it,” said Donald C. Lo, a scientific lead at Remedi4All, a group focused on drug repurposing, as reported by The Times.

Rare diseases, defined as those affecting fewer than 200,000 people in the U.S., collectively impact tens of millions worldwide. Yet, over 90% of these conditions lack approved treatments, says NORD.

Pharmaceutical companies often avoid investing in rare diseases due to limited profitability. “There isn’t typically much money to be made developing a new drug for a small number of patients,” said Christine Colvis of the National Center for Advancing Translational Sciences, reports The Times.

AI is changing that. By analyzing vast datasets, machine learning models can quickly identify potential drug-disease matches. The Times reports that Dr. Fajgenbaum’s team at the University of Pennsylvania, for example, compares 4,000 drugs against 18,500 diseases, scoring each for potential efficacy. Similar efforts are underway globally, from Stanford to Japan.

One success story involves a 19-year-old patient in Alabama suffering from chronic vomiting. An AI model suggested inhaling isopropyl alcohol, which provided instant relief, reported The Times.

“Essentially, we ran a query that said, ‘Show us every proposed treatment there has ever been in the history of medicine for nausea,’” said Matt Might, a professor at the University of Alabama at Birmingham. “It popped to the top of our list, and it worked instantly,” he added, as reporter by The Times

According to The Times, Dr. Fajgenbaum’s journey into drug repurposing began when he was diagnosed with Castleman disease, a rare immune disorder, during medical school. After failing to respond to standard treatments, he discovered that sirolimus, a generic drug used to prevent organ rejection, saved his life.

This experience inspired him to establish Every Cure, a nonprofit using AI to accelerate drug repurposing. Despite its potential, drug repurposing faces challenges. Many repurposed drugs are generics, offering little financial incentive for pharmaceutical companies, says The Times.

“If you use AI to find a new use for an old, inexpensive drug, no one makes any money off of it,” Dr. Fajgenbaum said, as reported by The Times. Still, the impact is undeniable. For patients like Coates, AI offers hope where traditional medicine falls short. “

This is one example of AI that we don’t have to fear, that we can be really excited about,” said Dr. Grant Mitchell, a co-founder of Every Cure, reported The Times. “This one’s going to help a lot of people,” he added.

As Coates stood healthy and smiling beside Dr. Fajgenbaum last month, his story served as a testament to the transformative power of AI in medicine. “Someone had to be the first to try,” said Dr. Wayne Gao, Coates’s oncologist, reported The Times.