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One Drug, Other Uses

UVA researchers think outside the label for treatments

What if, like a multitool, a drug could be used to treat multiple conditions?
Timothy Cook

Developing new drugs can be lengthy and costly; according to the National Institutes of Health, the process from “promising molecule” to “approved drug” can take well over a decade and considerable expense—with many failing along the way. But increasingly sophisticated research tools, including large-scale data analysis, are making it easier to discover how existing drugs might be repurposed for other conditions, and UVA researchers across a range of disciplines are finding promise in the potential to apply approved drugs to new therapeutic purposes.

In cancer treatment and care, researchers in the School of Medicine published a paper last summer in the journal Cancer Research that demonstrated the potential of palbociclib, a drug used for some forms of advanced breast cancer, to treat bone marrow scarring that is characteristic of a rare form of leukemia known as myelofibrosis. And in the journal Cancer Medicine, a team from the Department of Public Health Sciences, along with colleagues from Brazil, outlined an analysis of medical records that found evidence that common blood pressure drugs appeared to improve survival in colorectal cancer patients over age 65.

Researchers have also found evidence that HIV drugs may be effective with other conditions. A large team of researchers at UVA and other institutions analyzed medical records and found evidence that the risk of developing diabetes—a health condition on the rise globally—appeared to be lower in patients taking certain drugs to treat HIV and hepatitis B. And HIV drugs known as nucleoside reverse transcriptase inhibitors are also seen as a potential treatment for one form of age-related macular degeneration, a progressive condition that can lead to severe vision loss. Previous research had shown an association between the drug and reduced risk of the condition. Experts including UVA professor of ophthalmology Jayakrishna Ambati have discovered how inflammation builds up in the eye and damages retinal cells in the disease, and their work in animal models suggests the HIV drugs could protect against that inflammation.

“Runaway” inflammation also characterizes the potentially deadly condition of sepsis—and the most serious cases of COVID-19. But researchers in the lab of associate professor Alban Gaultier in the Department of Neuroscience found evidence that the antidepressant drug fluvoxamine could be effective in stopping sepsis. Building on that work, a clinical trial led by a team at Washington University School of Medicine in St. Louis has found that fluvoxamine significantly reduced the risk of hospitalization or death in COVID-19 patients.