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How AI Is Repurposing Existing Drugs as New Remedies

With the help of artificial intelligence, researchers are finding new uses for old medications to fight back against a surprising array of ‘incurable’ diseases


pill capsule digital illustration
Glenn Harvey

Allen Jones had bought a cemetery plot and was ready to start hospice care, his body and mind ravaged by a rare immune condition that had resisted chemotherapy, a stem cell transplant and a slew of powerful drugs. 

A physician from Canada, Jones was in his late 40s, with a wife and young child. “We had all the end-of-life protocols ready,” he says.

Jones, now 51, has the most debilitating form of Castleman disease, which causes the immune system to churn out a torrent of inflammatory proteins that attack vital organs such as the liver and kidneys. Up to 25 percent of people with this condition die within five years. 

There is no cure for the form of the disease that struck Jones. But then his doctor heard about an unlikely treatment: An AI-enabled computer program being built in the U.K. had identified the arthritis drug adalimumab (Humira) as a potential treatment for Jones’ condition. 

“In a situation where you’re dying, you would do anything,” Jones says. He started weekly injections of the drug. “After a few weeks, it worked. The vast majority of my symptoms got better. I’m alive today because of it.”

Dr. David Fajgenbaum meets with patient Kaila Mabus, 18, and her mother Sara Barrett at the Every Cure office. Every Cure co-founder Fajgenbaum and Mabus were both diagnosed with Castleman Disease, which previously had no known treatment. Every Cure is a nonprofit initiative that searches for existing medicines that can treat other diseases.
Alyssa Schukar/Redux

The drug’s off-label use was discovered by Every Cure, a nonprofit organization that has developed an AI-driven mathematical formula to compare 18,500 diseases with more than 4,000 FDA-approved drugs. 

“We are on a mission to save lives with the drugs we already have,” says David Fajgenbaum, M.D., associate professor of translational medicine and human genetics at the University of Pennsylvania, who cofounded Every Cure in 2022. “We could potentially double the impact of our medicines in very short order by using them in new ways.”

As drug companies and researchers turn to AI to develop and even design brand-new drugs faster (some are even in human trials), Every Cure and a handful of similar projects in the U.S., Europe and Japan are using artificial intelligence to find existing drugs that can be repurposed to treat the world’s approximately 10,000 rare diseases — and more common conditions, too. 

“We literally look at every drug and every disease,” says Fajgenbaum. Every Cure’s platform has already flagged little-known possible repurposed treatments for autism spectrum disorder and amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease).

Two years after Jones started adalimumab, his story was published as a case study in the New England Journal of Medicine in February 2025. 

At press time, Jones was doing well. “I’m a testament to the potential of this approach. It’s a drug we never, ever would have considered. It was a last-ditch option. I think it’s amazing.”

Advances in drug discovery

Identify new antibiotics. Researchers at MIT reviewed 12 million compounds for a 2024 study on drug-resistant bacteria. Phare Bio, a nonprofit company, is now creating what it calls “the world’s first generative AI antibiotics discovery engine” to bring lifesaving drugs to market.

Link Alzheimer’s genes with treatments. At the Cleveland Clinic’s Alzheimer’s Network Medicine Laboratory, researchers used AI-enabled software to identify 156 different Alzheimer’s risk genes, each of which is now a potential new drug target. They’ve already identified several drugs as possible weapons against Alzheimer’s.

Develop a new gut drug. An experimental drug for inflammatory bowel disease, engineered using generative AI, has entered human trials. ABS-101, developed by Absci, will first be tested for safety; drugs for other disorders are in phase 2 clinical trials.

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