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Download AD biomarker introduction- Annemieke Aartsma
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Biomarker introduction Annemieke Aartsma-Rus November 7 2015 What is a biomarker? A biomarker is a characteristic that is objectively measured and evaluated as an indicator of normal biological processes, pathogenic processes or pharmacological responses to a therapeutic intervention Annemieke Aartsma-Rus Different types of biomarkers • Diagnostic biomarkers • Something is wrong • Prognostic biomarkers • Predict disease course mild/severe • Therapeutic monitoring biomarkers • Predict whether therapy will work/is working • Sometimes one biomarker is both diagnostic and prognostic and monitoring but generally NOT Annemieke Aartsma-Rus Biomarkers used in DMD already • Diagnostic biomarkers • Serum creatine kinase (CK) • Elevated in DMD at young age • Not disease specific • Indication of muscle damage • Helps clinicians to make differential diagnosis • Gene analysis needed to confirm diagnosis (DMD mutation) Annemieke Aartsma-Rus Biomarker research for DMD • Prognostic biomarkers • May allow stratification in trials • Research ongoing • DNA differences (SNPs) in genes involved in inflammation can influence age at loss of ambulation • Osteopontin • LTBP4 • Challenges: • Which genes to focus on? • Large cohorts needed to confirm findings Annemieke Aartsma-Rus Biomarker research for DMD • Therapeutic biomarkers • Used for therapeutic monitoring of treatment • Identify responders • Before trial (more homogeneous population) • During trial (to continue with responders) • Pharmacodynamic biomarker • Surrogate endpoint Annemieke Aartsma-Rus Biomarker research for DMD • Pharmacodynamic biomarker • Did the treatment work as proposed? • Confirmation drug mechanism • E.g. dystrophin restoration for exon skipping/ataluren or reduced fibrosis for antioxidant Annemieke Aartsma-Rus Biomarker research for DMD • Surrogate endpoints • Correlates with clinical benefit (now or in future) • A tool that can be used to accelerate the process of making the drug available on the market • Advantage: may be quicker and less prone to variation/motivation than functional tests • No surrogate endpoint available yet for DMD Annemieke Aartsma-Rus
