Fleur Ferguson, PhD
Unlocking the Potential of Targeted Protein Degradation in Neurological Disorders
Summary
Targeted protein degradation (TPD) is an exciting new therapeutic modality that has expanded the druggable target space. Targeted protein degraders hijack cells’ natural “trash disposal” mechanisms for protein turnover and redirect cells to dispose of disease causing proteins. Recently, numerous new anticancer drugs that act via a targeted protein degradation mechanism have entered clinical trials. However, TPD has not been applied broadly to targets in neuro-oncology because it is challenging to develop TPD drugs that can cross the blood-brain barrier (a network of cells meant to protect the brain from harmful substances). My research will investigate generalizable approaches to improving TPD pharmacokinetics (how the drugs move through the body) to enable application of this transformative technology to neurological disorders.
I’m grateful to the PhRMA Foundation for their support, which has enabled me to pursue an exciting new direction in the lab. I hope to enable and accelerate the application of TPD to neuro-oncology, with the goal of developing improved therapies for various neurological disorders.
