Re-Purposing of FDA-Approved Drugs for the Treatment of LYSOSOMAL STORAGE DISEASES (LSD)
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 MSD 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 1,280 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: March 2017 – March 2018
UPDATE: October 2019
In October 2019, TIGEM presented preliminary results of this study at the Conference of Telethon Fundamental Associations in Italy. Read more.