Bench to bedside Parkinson’s Research

Dr. Esther Sammler

Abstract

The greatest unmet need in Parkinson’s are treatments that slow the relentless progression of its symptoms. The discovery of genetic changes in different genes that cause Parkinson’s have prompted new therapeutic approaches. LRRK2 is the most frequently mutated gene in Parkinson’s and LRRK2 inhibitor drugs have recently entered early clinical trial stages. However, many questions remain including markers of disease progression, biochemical markers for LRRK2 activation status and biomarkers for drug target engagement. These biomarkers will be necessary to confirm patient subgroups with high LRRK2 activity that might receive greater benefits from these therapies and ensure that the drug has the desired effect on the pathway targeted.

My talk will focus on a test that we have developed to measure LRRK2 activity in human peripheral blood and I will show very exciting data demonstrating the usefulness of this test.

For people interested in participating in research, I will also briefly mention a translational research project of mine (involving donating a blood sample) that will soon launch in the East and South East of Scotland.

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