Unbiased Proteomic Analysis of Clinical Trial CSF Samples to Identify Early Pharmacodynamic Response Signatures Associated with Disease-Modifying Therapies in ALS
Time: 1:30 pm
day: Conference Day One Track A PM
Details:
- Identification of tofersen PD-response biomarkers in SOD1-ALS VALOR clinical trial CSF via multiplexed quantitative proteomics
- Significant modulation from baseline abundance observed for 56 proteins in tofersen-treated participants relative to placebo, including proteins with significant changes from baseline as early as 4 weeks post-treatment
- Orthogonal validation of selected biomarker candidates across independent tofersen-treated cohorts
- Utility of integrating unbiased proteomic screening with targeted validation methods to identify new predictive, prognostic, and pharmacodynamic response biomarkers in clinical trial biospecimens
