Re-Purposing of FDA-Approved Drugs for MPSs

Re-Purposing of FDA-Approved Drugs for the Treatment of Mucoploysaccharidoses (MPSs)

Project is funded by Cure Sanfilippo Foundation

Project Summary: The study aims to identify FDA-approved compounds that activate the clearance of pathologic lysosomal accumulation in MPS cellular models independently of the missing enzyme in each particular disease.

Relevant cell-based high content (HC) screening assays have been developed, targeting common features of most MPS diseases, such as expansion and aggregation of lysosomal compartment and lysosomal degradation impairment.

These assays will be used to screen a FDA collection of 1500 compounds that are marketed drugs. Thus, the hits derived from the HCS, once validated in secondary assays, could in principle be immediately tested in patients to allow repositioning of known drugs as correctors of MPS.

Repositioning of FDA-approved drugs may reduce the clinical translation time of the findings.

Dates: March2017 – March 2018

Cure Sanfilippo Foundation

501c3 non-profit
(Tax ID: 46-4322131)

P.O. Box 6901
Columbia, SC 29260