Currently, there is no FDA-approved treatment for Niemann Pick Disease type C, only drugs that treat the symptoms. That might be changing though, thanks to a radical new compound called cyclodextrin that has been shown to improve cats afflicted with the disease.
Niemann Pick Disease type C, is commonly called “childhood Alzheimer’s” because of the mental and physical deterioration it has on kids is a terrible disease and scientists don’t know much about it. Only about one out of 150,000 people get the disease and some patients who have it don’t show symptoms until adulthood, but when it flares up in children it normally becomes fatal relatively quickly.
Researchers are excited about a new breakthrough in the treatment of the disease that shows when cats carrying the disease are treated with the compound cyclodextrin, their brains stave off the progression of the disease. John Dietschy of the University of Texas Southwestern Medical Center and Steven Walkley of Albert Einstein College of Medicine, found that previous studies in combating the disease with HPβCD — a main ingredient in the odor-fighting household product Febreze — showed positive effects on cell culture, but resulted in lung disease for the cats.
To try and avoid this negative outcome, the scientists administered the compound when the cats were just three weeks of age and repeated the dosage every two weeks. Amazingly, the cats were neurologically normal at six months and showed only mild symptoms at 18 months. Some of the cats have even gone on to have kittens.
The next step for the study is to continue looking for alternative compounds that elicit a similar benefit to HPβCD without the negative side effects. “It doesn’t get any better than finding something that treats animals and then will go on to treat kids,” said Charles Vite, associate professor of veterinary neurology at University of Pennsylvania.