A Little Over a Year Ago, a Man Was Told He Had Only Two Options: Die at Home or in the Hospital
Joseph Coates, then 37 and living in Renton, Wash., was barely conscious. 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 liters of fluid from his abdomen. He became too sick to receive a stem cell transplant, one of the only treatments that could have put him into remission.
"I gave up," he said. "I just thought the end was inevitable."
But Coates’s girlfriend, Tara Theobald, wasn’t ready to quit. So she sent an email begging for help to a doctor in Philadelphia named David Fajgenbaum, whom the couple had met a year earlier at a rare disease summit.
Finding Clues in Old Research
Repurposing is fairly common in pharmaceuticals: Minoxidil, developed as a blood pressure medication, has been repurposed to treat hair loss. Viagra, originally marketed to treat a cardiac condition, is now used as an erectile dysfunction drug. Semaglutide, a diabetes drug, has become best known for its ability to help people lose weight.
The first time Dr. Fajgenbaum repurposed a drug, it was in an attempt to save his own life. At 25, while in medical school, he was diagnosed with a rare subtype of a disorder called Castleman disease, which led to an immune system reaction that landed him in the intensive care unit.
There is no one way to treat Castleman disease, and some people don’t respond to any of the available treatments. Dr. Fajgenbaum was among them. Between hospitalizations and rounds of chemo that temporarily helped, Dr. Fajgenbaum spent weeks running tests on his own blood, poring over medical literature, and trying unconventional treatments.
The Power of Artificial Intelligence
Dr. Fajgenbaum’s lab had some early successes, including finding that a novel cancer drug helped another Castleman patient. But the process was laborious, requiring his team to examine "one drug and one disease at a time," he said. Dr. Fajgenbaum decided he needed to speed up the project. In 2022, he established a nonprofit called Every Cure, aimed at using machine learning to compare thousands of drugs and diseases all at once.
Conclusion
The use of artificial intelligence to repurpose existing drugs has the potential to revolutionize the way we approach rare diseases. By analyzing vast amounts of data and identifying patterns, AI models can quickly pinpoint potential treatments that might have otherwise gone unnoticed. This approach has already led to successful treatments for patients with a range of conditions, from Castleman disease to chronic vomiting.
As we move forward, it’s essential to continue developing and refining these AI models, ensuring that they are able to identify the most promising potential treatments for patients in need.
Frequently Asked Questions
Q: How does the AI model work?
A: The AI model uses machine learning algorithms to analyze vast amounts of data, including existing medical literature, to identify potential treatments for rare diseases.
Q: How accurate is the AI model?
A: The AI model is not infallible, and its predictions may not always be 100% accurate. However, it can still provide valuable insights and suggestions for further investigation.
Q: How does the AI model prioritize potential treatments?
A: The AI model prioritizes potential treatments based on their likelihood of success, as well as the strength of the evidence supporting their use.
Q: How does the AI model take into account the complexity of rare diseases?
A: The AI model is designed to account for the complexity of rare diseases, including the many variables that can influence their progression and treatment.
