Objective: The aim of this study was to identify additional new biomarkers using mass spectrometry-based proteomics in well-defined clinical samples from a phase II clinical trial and biobank.
Background: There is an urgent need to identify biomarkers for multiple system atrophy (MSA) to enable earlier, more accurate diagnosis, monitor disease progression, and assess response to drug therapies.
Method: CSF samples from patients with MSA (n = 37) and controls (n = 30) were analyzed using advanced mass spectrometry–based proteomics to discover and validate novel biomarkers for disease detection and treatment monitoring. Proteomics sample preparation used heat-denatured reduction/alkylation, tryptic digestion for peptide generation, and solid phase extraction for peptide purity. Data acquisition employed a Vanquish Neo LC online coupled to an Orbitrap Astral mass spectrometer operating in nDIA for comprehensive proteomics.
Results: A total of 6,505 protein groups were quantified in the entire study, with a mean of 5,088 proteins per sample. Comparison of MSA and control samples identified 195 significantly altered proteins, including several previously reported MSA biomarkers. Neurofilament proteins, including neurofilament light (NEFL), neurofilament medium (NEFM), and neurofilament heavy (NEFH) chains, showed among the strongest upregulation in MSA, whereas synaptic proteins such as chromogranin B (CHGB) were among the most significantly decreased. The identification of established biomarkers alongside novel candidates highlights the potential of this approach to uncover proteins relevant to MSA detection and disease progression. Analysis of all significantly altered proteins is ongoing.
Conclusion: CSF proteomic profiling in MSA identified a set of established and novel protein candidates with potential utility for disease detection and monitoring. These findings support the development of targeted biomarker assays and their validation in future clinical studies.
To cite this abstract in AMA style:
P. Kallunki, M. Lubas, J. Fussing Tengberg, V. Carmelo, S. Virreira Winter, P. Geyer. Identification of Candidate Cerebrospinal Fluid Biomarkers in Multiple System Atrophy Using Mass Spectrometry‑Based Proteomics in CSF Biobank Samples [abstract]. Mov Disord. 2026; 41 (suppl 1). https://www.mdsabstracts.org/abstract/identification-of-candidate-cerebrospinal-fluid-biomarkers-in-multiple-system-atrophy-using-mass-spectrometry-based-proteomics-in-csf-biobank-samples/. Accessed October 1, 2026.« Back to 2026 International Congress
MDS Abstracts - https://www.mdsabstracts.org/abstract/identification-of-candidate-cerebrospinal-fluid-biomarkers-in-multiple-system-atrophy-using-mass-spectrometry-based-proteomics-in-csf-biobank-samples/