A sensitive and specific LC-MS/MS method for rapid diagnosis of Niemann-Pick C1 disease from human plasma

J Lipid Res. 2011 Jul;52(7):1435-45. doi: 10.1194/jlr.D015735. Epub 2011 Apr 24.

Abstract

Niemann-Pick type C1 (NPC1) disease is a rare, progressively fatal neurodegenerative disease for which there are no FDA-approved therapies. A major barrier to developing new therapies for this disorder has been the lack of a sensitive and noninvasive diagnostic test. Recently, we demonstrated that two cholesterol oxidation products, specifically cholestane-3β,5α,6β-triol (3β,5α,6β-triol) and 7-ketocholesterol (7-KC), were markedly increased in the plasma of human NPC1 subjects, suggesting a role for these oxysterols in diagnosis of NPC1 disease and evaluation of therapeutics in clinical trials. In the present study, we describe the development of a sensitive and specific LC-MS/MS method for quantifying 3β,5α,6β-triol and 7-KC human plasma after derivatization with N,N-dimethylglycine. We show that dimethylglycine derivatization successfully enhanced the ionization and fragmentation of 3β,5α,6β-triol and 7-KC for mass spectrometric detection of the oxysterol species in human plasma. The oxysterol dimethylglycinates were resolved with high sensitivity and selectivity, and enabled accurate quantification of 3β,5α,6β-triol and 7-KC concentrations in human plasma. The LC-MS/MS assay was able to discriminate with high sensitivity and specificity between control and NPC1 subjects, and offers for the first time a noninvasive, rapid, and highly sensitive method for diagnosis of NPC1 disease.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Calibration
  • Case-Control Studies
  • Child
  • Child, Preschool
  • Cholestanols / blood
  • Cholestanols / chemistry
  • Cholestanols / isolation & purification
  • Chromatography, High Pressure Liquid / methods*
  • Female
  • Humans
  • Infant
  • Infant, Newborn
  • Ketocholesterols / blood
  • Ketocholesterols / chemistry
  • Ketocholesterols / isolation & purification
  • Male
  • Middle Aged
  • Niemann-Pick Disease, Type C / blood*
  • Niemann-Pick Disease, Type C / diagnosis*
  • Sarcosine / analogs & derivatives
  • Sarcosine / chemistry
  • Sensitivity and Specificity
  • Tandem Mass Spectrometry / methods*
  • Time Factors
  • Young Adult

Substances

  • Cholestanols
  • Ketocholesterols
  • cholestane-3,5,6-triol
  • dimethylglycine
  • 7-ketocholesterol
  • Sarcosine