Identify metabolites associated with clinical diagnosis and pathology of Alzheimer's Disease (AD)
Exogenous and endogenous metabolites in plasma were measured in 300 clinical AD and 430 healthy non-demented individuals of Caribbean Hispanic ancestry using untargeted liquid-chromatography, performed on HILIC (+ ionization) and C18 (- ionization) columns, coupled to a Thermo Orbitrap HF-X mass spectrometer. Genome-wide SNP data and plasma biomarkers including Ab40, Ab42, P-tau181 and Neurofilament light chain (NfL) were obtained on all participants. Metabolite association with AD and P-tau181 levels were tested after adjustment for age and sex. Metabolites associated with P-tau181 levels (but not with NfL, a non-specific marker of neurodegeneration) were categorized as AD-specific.
Over 4000 metabolomic features were measured with high accuracy in the sample. 96 metabolites were associated with P-tau181 levels, but no metabolites survived multiple testing correction for association with clinical AD alone. Features putatively annotated as phosphatidylcholine (4.11E-07) and Carotamine (p=1.2e-05) were strongly associated P-tau181 levels. We observed similar results when using a diagnostic cutoff of P-tau181 levels for AD as the outcome. Metabolites associated with P-tau181 levels were enriched in amino acid metabolism, glutathione metabolism, tryrosine metabolism and hexose phosphorylation pathways. Ramipril (p=2.9e-04) and glycerophosphocholine (p=9.9e-04) were among the top associated with AD diagnosis. PLS-DA analysis identified biopterin metabolism, glycosphingolipid biosynthesis and tyrosine metabolism as the most discriminating pathways between AD and healthy controls when augmented by biomarkers.
Metabolites specifically lipids and those involved in cardiovascular function were associated with AD and P-tau181 levels. Metabolite profiling can identify perturbed pathways in clinical and pre-clinical AD and integration with genome-wide SNP data will identify mechanisms underlying genetic association with disease.