Case study: Repurposing of PI3K pathway modulators for PHTS

Key factions

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 2025

Project goals

To conduct pre-clinical drug repurposing studies to identify new treatment options for PHTS.

Project overview

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.

Anticipated outcomes of this work

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.

More information about the CROs can be found on their corporate websites.