Cats and Febreze may hold key to treating devastating Niemann-Pick disease

Several years ago, scientists at the Albert Einstein College of Medicine in New York made a serendipitous discovery that the active ingredient in the odor-fighting household spray Febreze could greatly alleviate symptoms of a rare neurological disease called Niemann-Pick type C (NPC). But the finding was made in mouse models of the disease, and they needed a more realistic model to further develop their unusual but highly promising therapy.

Enter cats. A new study by the University of Pennsylvania School of Veterinary Medicine shows that cats with naturally occurring NPC--which is nearly identical to the human form of the disease--are greatly helped by the experimental Febreze-related compound, called cyclodextrin. The study was recently published in the medical journal Science Translational Medicine.

NPC affects just one in 150,000 people, but many of its victims are children, and the progression of the disease can be devastating. The illness, caused by a mutation in one of two genes, impedes the processing of cholesterol in cellular structures known as lysosomes. That leads to a stark neurological decline, and in children the disease is often fatal by age 20.

Cats suffer a similar fate and therefore present great opportunities for research that might translate to human therapies, says Penn's associate professor of veterinary neurology Charles Vite in a press release from the university. "Large animal models of human disease, like cats, are really helpful for determining what's going to happen in a child when you're treating them," Vite says. "By using these natural models, the ultimate goal is to find something that helps your cat and your dog population and your human population, too."

The Einstein team discovered cyclodextrin's power when they were testing a drug dissolved in one form of the compound called 2-hydroxypropyl-beta-cyclodextrin. Turns out, that drug wasn't having any effect on NPC but the cyclodextrin was. Coincidentally, that same cyclodextrin compound is what gives Febreze its odor-fighting ability.

A Phase I clinical trial of cyclodextrin is now underway in children, with a Phase II/III likely to begin later this year. The human trials will be sponsored by Vtesse, a biotech company founded around the cyclodextrin discovery. Vtesse was launched in January and funded by New Enterprise Associates, Pfizer ($PFE), Lundbeckfond Ventures, Bay City Capital and Alexandria Venture Investments.

As for the cats, the Penn team administered cyclodextrin to the brains of NPC-affected animals starting when they were just three weeks old and continuing every two weeks. Some of the treated cats have lived past age 3 and have gone on to have kittens, while untreated cats died by 6 weeks of age, according to the university. Vite says he and his colleagues will continue researching NPC, to try to determine why cholesterol buildup causes damage to neurons and to better understand how the experimental compound clears cholesterol from cells.

- here's the press release
- access an abstract of the study here