“Lupus research is very complicated, like a four-dimensional puzzle,” says UNC School of Medicine Professor of Microbiology and Immunology Barbara Vilen. Mysteries about the mechanistic underpinnings of the disorder have made designing effective therapeutics challenging. But that’s changing thanks to a discovery made by Vilen and her team that links non-functioning lysosomes with lupus symptoms.
A cell’s lysosomes act as its waste-disposal system. They break down and digest old cellular components, such as proteins, nucleic acids and sugars, with the help of enzymes and acids. Vilen found that lupus patients who were in inactive disease had functional lysosomes, while patients in active disease did not have functional lysosomes.
With funding from Eshelman Innovation, Vilen then developed a therapeutic that could restore lysosome function as a strategy to maintain inactive disease. The next step is getting the therapeutic into human clinical trials.
“It’s not going to be a cure because there’s a genetic underpinning in there somewhere, but it’s going to be a better treatment [than steroids],” says Vilen, the latest guest in the video series “North Carolina Innovators You Should Know,” hosted by Eshelman Innovation Executive Director John Bamforth. “And that’s going to be a win for lupus patients.”
