Identification of tofersen PD-response biomarkers in VALOR clinical trial CSF via multiplexed quantitative proteomics - PubMed
3 hours ago
- #biomarkers
- #proteomics
- #ALS
- Tofersen is the first approved genetically targeted therapy for ALS, specifically for adults with SOD1 gene mutations.
- The study analyzed CSF samples from the VALOR trial to identify biomarkers of tofersen treatment response in SOD1-ALS patients.
- 56 proteins showed significant modulation in tofersen-treated participants compared to placebo, with CSF GPNMB being continuously elevated post-treatment.
- GPNMB elevation was confirmed in independent cohorts using immunoassay, validating it as a pharmacodynamic-response biomarker.
- The findings highlight the potential of proteomic screening combined with targeted validation to identify treatment-response biomarkers in clinical trials.