Here’s how parents of kids with rare disease found what may be blockbuster drug.
Two parents’ quest to save their twin daughters’ lives from a rare, degenerative genetic disorder may end up saving and improving the lives of millions.
After digging through medical literature and fitting pieces of data together, the non-medically trained couple contacted German researchers and suggested that a chemical called cyclodextrin may be able to treat atherosclerosis—the hardening of arteries with cholesterol-rich plaques, which is a precursor to heart attack, stroke, and other cardiovascular diseases.
The researchers, Eicke Latz at the University of Bonn and colleagues, followed up on the parents’ hypothesis and found that in mice, cyclodextrin indeed blocked plaque formation, melted away plaques that had already formed in arteries, reduced atherosclerosis-associated inflammation, and revved up cholesterol metabolism—even in rodents fed cholesterol-rich diets. In petri dish-based tests, the researchers found that the drug seemed to have the same effects on human cells and plaques.
The findings, published Wednesday in Science Translational Medicine, suggest that cyclodextrin—a drug already approved for use in humans by the US Food and Drug Administration—may be highly effective at treating and preventing heart disease.