Drug Repurposing
A main focus of MEPAN Foundation has working with Perlara PBC and others to support drug repurposing research using various models of MEPAN Syndrome. These projects can efficiently test thousands of FDA-approved drugs to identify existing medicines that may address MEPAN symptoms.
Because these drugs have already been reviewed by the FDA and tested in patients for safety, repurposing offers a potentially faster and more cost-effective path to finding interventions that may help individuals with MEPAN Syndrome.
Repurposing Overview
Our support of drug repurposing followed a linear path since 2019 and has generated promising leads that may lead to therapeutic discoveries for MEPAN.​​​​
​High-throughput screen in yeast models at UCSF
This project tested five yeast models carrying patient-specific mutations to identify drugs that could rescue cell growth, offering potential therapeutic leads.
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A follow-up screen at the University of Oulu
Researchers tested a subset of the promising yeast drug hits in four sets of patient-derived fibroblasts to further assess their therapeutic potential.
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Additional fibroblast screening done at UCLA
This effort further tested the yeast leads in patient fibroblasts along with some candidates identified in a 2023 paper from the Bellen Lab at Baylor University.
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We also funded a pilot project at Kenyon College to explore drug combinations in roundworms with mecr-1 deficiency, providing insights into potential multi-drug treatment strategies for MEPAN.