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Discovery of Translational Pharmacodynamic Biomarkers and Biomarkers of Endo-lysosomal Pathway Pathology by Biased & Unbiased Lipidomics and Metabolomics

Study Rationale: The endolysosomal system within living cells is comprised of small, specialized structures that contain enzymes that degrade proteins and remove waste materials from the cell. Their impaired function leads to a build-up of toxic proteins and neurodegenerative diseases such as Parkinson’s disease (PD). Our mission is to discover therapeutics that improve the impaired function of the endolysosomal system and benefit PD patients. Biomarkers (or indicators) of target and pathway manipulation are key in drug discovery. We are proposing a large-scale study of small molecules, commonly known as metabolites to identify biomarkers that accelerate our drug discovery efforts.

Hypothesis: We hope to discover biomarkers of our target manipulation and the endolysosomal pathology. This will be key to transforming our ongoing drug discovery efforts into therapeutics.  

Study Design: We will conduct a study that combines informative models of endolysosomal pathology in Parkinson’s disease, human samples from patients and healthy controls with powerful technologies to identify key metabolites that are signatures of our drug discovery target and endolysosomal pathology. We will then test our biomarkers with small chemical compounds that we are optimizing in our drug discovery efforts.

Impact on Diagnosis/Treatment of Parkinson’s disease: If successful, the proposed studies will accelerate our ground-breaking drug discovery efforts to find new therapeutics that improve endolysosomal function and slow down progression of Parkinson’s disease and potentially other neurodegenerative diseases.

Next Steps for Development: Currently there are disease modifying treatment for Parkinson’s disease. Novel therapeutics that improve endolysosomal function would likely benefit ParkinSon’s disease.  


  • Joanna Wolak, MSc, PhD

    London United Kingdom

  • Richard Wade-Martins, MA, DPhil

    Oxford United Kingdom

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