Drug Re-Purposing & Re-Positioning Studies Funded by Cure Sanfilippo Presented At International Conference

Last month, the research institute TIGEM presented preliminary results of two studies funded by Cure Sanfilippo Foundation at the Conference of Telethon Fundamental Associations in Italy.

Cure Sanfilippo partnered with TIGEM (Telethon Institute of Genetics and Medicine) on research projects to explore re-positioning and re-purposing FDA-approved compounds to determine if identify they can activate the clearance of pathologic lysosomal accumulation in MSD cellular models independently of the missing enzyme in each particular disease.

Posters were presented by TIGEM at the conference for both projects. 

Top of poster presentation at Telethon scientific meeting Oct 2019 about re-positioning FDA-approved compounds for MPS III


Bottom of poster presentation at Telethon scientific meeting Oct 2019 about re-positioning FDA-approved compounds for MPS III

The team developed a new high-content screening assay to determine lysosomal activity. They have identified similar primary “hit compound” in MSD and MPS IIIA. The same compound in the screening reduces cellular heparan sulfate GAGs in cell models for MSD and MPS IIIA.

Poster presentation at Telethon scientific meeting Oct 2019 about disease mechanisms and pharmacological targeting of MPS III

Read more about each specific research project:

Further work to determine efficacy and proper concentration doses and delivery is ongoing in animal models and efficacy.

Fellow Sanfilippo Advocate Katia Moletta (mother to Francesco, MPS IIIA) was able to be attend the scientific meeting and connect with the presenters from TIGEM.

Katia Moletta at Telethon scientific meeting Oct 2019   Katia Moletta and Sandro Montefusco of TIGEMat Telethon scientific meeting Oct 2019

Cure Sanfilippo Foundation

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