In the late-2000s, the foundation supported many early-stage research programs to investigate therapeutics for NPC disease. One such class of compounds were called cyclodextrins, and they showed dramatic improvements in cell culture studies and animal models with the NPC mutation.
On March 15, 2017, Vtesse, Inc. (recently acquired by Sucampo Pharmaceuticals, Inc.) announced full enrollment in the Phase 2b/3 clinical trial of VTS-270, a well-characterized complex mixture of 2-hydroxypropyl-b-cyclodextrin (HPβCD) with a specific compositional fingerprint that distinguishes it from other HPβCD mixtures, in Niemann-Pick Type C1 (NPC) disease. The investigational drug candidate showed promise in pre-clinical and early clinical studies for NPC disease.
Twenty-three years after the launch of the foundation, the announcement that the VTS-270 clinical study has been fully enrolled with more than 51 NPC patients from around the globe is an exciting milestone. There is great enthusiasm from not only the Parseghian family, but for their many supporters, the families affected by NPC disease, researchers and clinicians that invested their time to find a cure, and the Notre Dame family.
You can see the entire press release at http://niemannpick.wpengine.com/research-supported-by-the-ara-parseghian-medical-research-fund-reaches-important-milestone/