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A Potential New Clinical Biomarker for Multiple Sclerosis – Quantification of Galactosylceramide

A Potential New Clinical Biomarker for Multiple Sclerosis – Quantification of Galactosylceramide
Advancements in the discovery and development of new therapeutics are often limited by the lack of robust biomarkers to assess drug activity. In a recent paper in Clinical and Translational Science (CTS), Kanhai and colleagues from the Centre for Human Drug Research in the Netherlands, report a new method that may prove useful to evaluate the effects of remyelinating agents. Enhancing remyelination is a compelling therapeutic strategy to develop new treatments for multiple sclerosis, a disease characterized by a loss of myelin coupled with incomplete remyelination, which leads to progressive demyelination of nerves in the CNS. The method reported in this paper may be applied to investigate the effects of remyelinating agents for the treatment of not only multiple sclerosis, but other demyelinating diseases such as Krabbe disease, neuromyelitis optica, and metachromatic leucodystrophy. 
 
In their study, Kanhai and colleagues measured the turnover of galactosylceramide, a component of myelin, in six healthy subjects using a labeling approach with orally administered deuterated water (D2O) followed by mass spectrometry detection and compartmental modeling. The results from this study suggests a slow turnover rate of 0.00168 day-1, corresponding to a half-life of 413 days for galactosylceramide in CSF. The authors suggest that that a proof-of-concept study employing this method could be conducted in as little as 15 weeks by measuring peak galactosylceramide enrichment. This approach to assess myelin kinetics may prove to be useful to investigate the potential of new therapies to enhance remyelination and may be a more robust method than the imagining techniques that are currently available. 
 
The work reported in this paper represents important progress in identifying a potential new biomarker for remyelinating agents. A critical next step includes conducting additional studies in patients with multiple sclerosis to investigate if galactosylceramide turnover could be used as a measure of activity for remyelination therapies in patients.

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