Treatment Options

for NPC

While there is no definitive therapy for NPC, research is continuing to identify potential treatments that could either slow or stop the progression of the disease.

A clinical trial of Zavesca® (or Miglustat) for NPC is underway in the U.S. and Europe. Zavesca has slowed, but not stopped, neurological decline when tested on NPC mice.

A drug assay is being conducted by Dr. Laura Liscum. Nearly 50,000 compounds have been tested by Bristol Meyers Squibb for potential effectiveness with NPC. Fifty compounds were identified as candidates for further testing but none has proven suitable for human use. Work is continuing on related compounds.

Laboratory studies of neurosteroids have had encouraging results when tested on mice but more work needs to be conducted before a clinical trial can be considered.

Many of the symptoms of Niemann-Pick Type C can be controlled or tempered by drugs and supportive treatment.

Cate Walsh-Vockley, National NP Coordinator, can provide guidance to families in this area. Each patient must be considered individually, depending on symptoms. Supportive treatments should be re-evaluated on a regular basis as the disease progresses.

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