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Penn Vet Researchers Identify Effective Treatment

Penn Vet Researchers Identify Effective Treatment for Niemann Pick Type C
UPenn - 2/25/15

Niemann Pick Disease type C, or NPC, is a disease most people have never heard of, affecting just one person in 150,000. Yet the disease is a devastating one. Frequently diagnosed in children in their elementary school years, sufferers usually die by the time they’re 20. The disease is sometimes referred to as “childhood Alzheimer’s” because of the progressive mental and physical decline seen in the children it afflicts.

There is no specific, FDA-approved treatment for NPC, only drugs to treat the symptoms. That is now changing.

A study coming out in Science Translational Medicine and led by University of Pennsylvania School of Veterinary Medicine researchers has shown that cats with NPC — which mirrors the human version of the disease — show vast improvements when treated with a compound called cyclodextrin. While NPC typically results in inexorable neurological decline, administering cyclodextrin into the fluid around the cats’ brains largely halted the progression of disease.

This study showed that the treatment was safe enough to begin a clinical trial in children, which launched last year.

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