Research category: Translational medicine
Contract Research Organisations (CROs):
Evotec – headquarters in Hamburg, Germany
Charles River Laboratories – headquarters in Wilmington, MA, USA
Project start: October 2020 Expected completion: End of 2023
To conduct pre-clinical drug repurposing studies to identify new treatment options for PHTS.
PTEN Research has established collaborations with two leading drug discovery and development CROs to pursue basic and translational contract research activities that will support exploratory drug repurposing studies. A number of bespoke in vitro models have been established to identify candidates for in vivo evaluation in models representing PHTS neurodevelopment delay and autism (Evotec), and vascular anomalies and tissue overgrowth/oncology (Charles River Laboratories). Our current focus is on small molecules targeting the PI3K pathway and other pathways of interest.
These research activities are managed by joint PTEN Research and CRO steering committees, with input from independent experts and oversight from the Trustees.
In addition to identifying potential drug repurposing candidates for clinical testing, it is hoped that studying these models will also allow us to develop valuable insights into the dysregulated signalling that results from PTEN mutations, and the relative importance of PI3K and other pathways to the different phenotypes associated with PHTS.