Development of an in vitro Parkinson’s disease model of L-dopa-induced dyskinesia

Parkinson’s disease (PD) is a serious unmet medical need. Drug failures in neurological
conditions are driving the Pharmaceutical Industry to exit the field and there is an urgent need
for new therapeutic approaches that will change the underlying course of disease. Although Ldopa
remains the gold standard in treating PD, the treatment regime results in toxic side
effects that include disabling dyskinesia. Dyskinesia is not painful but is a distressing side
effect that often results uncontrolled flailing of the limbs and involuntary repetitive
movements of the face, tongue, mouth and neck. It results in a significant degradation in the
patient's quality of life and an increased burden of care to society at large.
This project aims to develop and validate an innovative in vitro PD model of L-dopa-induced
dyskinesia (LID) using differentiated induced pluripotent stem cells (iPS). This proprietary
disease modelling assay catalyses and is central to a network pharmacology-based drug
discovery platform to identify disease-modifying agents that will prevent the onset of
dyskinesia. Such a tool represents a valuable scientific and commercial inflection point that
will enable the identification of lead drug candidates to be taken into a full clinical
development plan.