Biomarkers are important tools for diagnosing and monitoring diseases and are useful in assessing patient responses to new therapies. Neurodegenerative diseases such as Parkinson's disease (PD) and Alzheimer's disease (AD) are particularly difficult to monitor because the site of disease (the brain) isn't readily accessible.
In this issue of the Journal of Clinical Investigation, researchers led by Patrizia Fanara of KineMed, Inc. in Emeryville, CA and Marc Hellerstein of the University of California, San Francisco report the development of a novel class of cerebrospinal fluid (CSF)-based kinetic biomarkers.
The biomarkers measure axonal transport, a cellular process that is altered in PD, AD, Huntington's disease (HD), and amyotrophic lateral sclerosis (ALS). The researchers first analyzed the biomarkers in a mouse model of Parkinsons' disease. Prior to the test, the mice drank "labeled" water, which was metabolized and incorporated into molecules that could then be measured in CSF.
The study was then repeated in humans. Fanara and colleagues observed marked alterations in CSF from patients with PD compared to healthy subjects. In a companion piece, William Potter, a member of the National Institutes of Health Neuroscience Steering Committee, discusses the use of CSF as a biomarker source and the implications of this technology for the study of neurological diseases.
TITLE:Cerebrospinal fluid–based kinetic biomarkers of axonal transport in monitoring neurodegeneration