Biomarker introduction Annemieke Aartsma-Rus November 7 2015.

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Presentation transcript:

Biomarker introduction Annemieke Aartsma-Rus November

What is a biomarker? Annemieke Aartsma-Rus 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

Different types of biomarkers Annemieke Aartsma-Rus 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

Biomarkers used in DMD already Annemieke Aartsma-Rus 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)

Biomarker research for DMD Annemieke Aartsma-Rus 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

Biomarker research for DMD Annemieke Aartsma-Rus 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

Biomarker research for DMD Annemieke Aartsma-Rus Pharmacodynamic biomarker Did the treatment work as proposed? Confirmation drug mechanism E.g. dystrophin restoration for exon skipping/ataluren or reduced fibrosis for antioxidant

Biomarker research for DMD Annemieke Aartsma-Rus 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