Mom's Insight May Lead to Heart Disease Treatment

Compound used to treat kids' rare disorder unclogs arteries, too
By Elizabeth Armstrong Moore,  Newser Staff
Posted Apr 8, 2016 1:11 PM CDT
Mom's Insight May Lead to Heart Disease Treatment

The unlikely use of a compound in powdered booze could ultimately help save millions of people from heart disease—and it was the mother of twin girls with a rare genetic disorder who first pitched the idea to scientists. The compound is called beta-cyclodextrin, which is already approved by the FDA and is widely used in medications and foods (including powdered alcohol), reports Ars Technica. It's long been seen as more of a "carrier"—it delivers other drugs and makes them more effective—but now it seems cyclodextrin itself is plenty potent, reports the Wall Street Journal. Writing in the journal Science Translational Medicine, researchers say it dissolved cholesterol crystals and plaque building up around the hearts of mice. “This is a potentially promising therapeutic approach,” says a Michigan State University cardiologist not involved with the study.

An unusual twist is that one of the co-authors listed is Chris Hempel, an American mom without formal medical training who suggested the heart disease study. She is the mother of twin daughters with a rare genetic disease called Niemann-Pick type C, or NPC, often referred to as childhood Alzheimer's. Cyclodextrin has shown promise in treating NPC—it is currently being tested in clinical trials, thanks in large part to the advocacy of Hempel and her husband, Hugh—and Chris Hempel suggested to heart researchers that it might help on that front, too. The initial findings suggest her hunch was correct. More research is needed, but if further studies back up the results, cyclodextrin would be an improvement over the statins that don't always work on patients with clogged arteries, reports Popular Science. (Check out a profile of one of the NPC kids taking cyclodextrin.)

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