By AMERICAN HEART ASSOCIATION NEWS
The latest research news in heart health didn’t start in a lab, a hospital or a doctor’s office.
It all began with one smart mom, sitting at home reading scientific journals.
Chris Hempel of Reno, Nevada, was the catalyst behind a study published in April in Science Translational Medicine that shows cyclodextrin could one day be used to treat heart patients who don’t respond well to statins.
“This is important news because it potentially gives us another tool to fight a problem that continues to plague our society … it’s also pretty remarkable when an astute mother can put two and two together and say ‘This might work for people who have cholesterol problems,’” said Dr. Clyde Yancy, a cardiologist at Northwestern Memorial Hospital and the chief of cardiology at Northwestern University Feinberg School of Medicine. “It speaks to the beauty and serendipity of the scientific discovery process.”
Hempel is the mother of twin girls, Addison and Cassidy, with Niemann-Pick Type C, a rare and fatal genetic cholesterol-metabolism disorder.
After reading a story in Nature about cholesterol crystals and heart disease, she wondered if the crystals might be dissolved by cyclodextrin, a compound which her girls receive as part of an experimental treatment approved by the Food and Drug Administration.
“Anybody can have an idea,” Hempel said. “I was just trying to put together the pieces of the puzzle.”
Her first move was to email the article’s author, Dr. Eicke Latz, director of The Institute of Innate Immunity at the University of Bonn in Germany.
“He was intrigued enough that he started doing experiments with mice,” Hempel said. “After six years of loading them up with Western diets, the cyclodextrin is having enormous effects.”
According to the study, cyclodextrin “reduced atherosclerotic plaque size and cholesterol crystal load, and promoted plaque regression even with a continued cholesterol-rich diet.”
The study also said “because cyclodextrin is already known to be safe in humans, this drug is now a potential candidate for testing in human patients for the treatment of atherosclerosis.”
“We’re still very far away from clinical utility, but it’s worth pursuing,” said Dr. Yancy, who wasn’t involved in the study. “It potentially exposes another strategy to deal with the problem of abnormal lipids and high cholesterol, especially for individuals who have difficulties with statins.”
Hempel – who is listed as a co-author of the study in Science Translational Medicine — said her daughters, now 12, have been taking cyclodextrin for seven years with positive results and no side effects.
“It’s one of the first drugs in history that has stopped a progressive neurological disease. They shouldn’t even be alive today,” she said.
She said she believes cyclodextrin can have similar positive effects for people with heart disease, and she hopes further studies will be conducted.
“It’s such a promising drug that can benefit so many people who are suffering. We really need public support and government support to move it forward,” she said.
Hempel also wants to see the emergence of more “patient advocates” such as herself.
“The average person without a medical degree can make a difference and spark some kind of change. Sometimes, you just have to use logic,” she said
Dr. Yancy agreed. “Statins are still ideal for most patients with cholesterol problems, but this approach using cyclodextrin may be uniquely different — and it would be very eye-opening to recognize this got started by an astute mother’s observations,” Dr. Yancy said.
“We live in a very different world now where ideas can percolate up almost instantly, whether through social media or through word of mouth,” he said. “The discovery process has really become a broad net that we cast.
Sometimes in the net, we find a pearl that can really impact health and disease.”