TY  - JOUR
AU  - Muqaku, Besnik
AU  - Dorst, Johannes
AU  - Wiesenfarth, Maximilian
AU  - Otto, Markus
AU  - Ludolph, Albert C
AU  - Oeckl, Patrick
TI  - Peptidomic analysis of CSF reveals new biomarker candidates for amyotrophic lateral sclerosis.
JO  - EMBO molecular medicine
VL  - 17
IS  - 8
SN  - 1757-4676
CY  - [London]
PB  - Nature Publishing Group UK
M1  - DZNE-2025-00948
SP  - 1926 - 1949
PY  - 2025
AB  - Amyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disease, and novel biomarkers are needed. We applied mass-spectrometry-based peptidomic analysis in cerebrospinal fluid (CSF) samples of ALS and non-neurodegenerative control patients (Con) from a discovery (n = 48) and validation (n = 109) cohort for biomarker discovery. Systematic selection revealed a panel of eight novel peptide biomarker candidates for ALS (out of 33,605) derived from seven proteins. In the validation cohort, NFL, MAP1B, MYL1, and APOC1 peptides were upregulated, and peptides from CADM3, SCG1, and PENK were downregulated in ALS compared to Con. The peptides (except NFL) were not changed in other neurodegenerative diseases, including Alzheimer´s disease, frontotemporal dementia and Parkinson´s disease. Combination of all peptides in a logistic regression model led to an area under the curve value of 98
KW  - Humans
KW  - Amyotrophic Lateral Sclerosis: diagnosis
KW  - Amyotrophic Lateral Sclerosis: cerebrospinal fluid
KW  - Amyotrophic Lateral Sclerosis: pathology
KW  - Biomarkers: cerebrospinal fluid
KW  - Female
KW  - Male
KW  - Middle Aged
KW  - Aged
KW  - Peptides: cerebrospinal fluid
KW  - Cohort Studies
KW  - Mass Spectrometry
KW  - Proteomics: methods
KW  - Adult
KW  - ALS (Other)
KW  - Biomarker (Other)
KW  - Muscle (Other)
KW  - Neurodegeneration (Other)
KW  - Peptidomics (Other)
KW  - Biomarkers (NLM Chemicals)
KW  - Peptides (NLM Chemicals)
LB  - PUB:(DE-HGF)16
C6  - pmid:40681694
DO  - DOI:10.1038/s44321-025-00272-w
UR  - https://pub.dzne.de/record/280293
ER  -