The application of biomarkers in clinical trials for motor neuron disease

The interest and research into disease-related biomarkers has greatly accelerated over the last 10 years. The potential clinical benefits for disease-specific biomarkers include a more rapid and accurate disease diagnosis, and potential reduction in size and duration of clinical drug trials, which would speed up drug development. The application of biomarkers into the clinical arena of motor neuron disease should both determine if a drug hits its proposed target and whether the drug alters the course of disease. This article will highlight the progress made in discovering suitable biomarker candidates from a variety of sources, including imaging, neurophysiology and proteomics. For biomarkers to have clinical utility, specific criteria must be satisfied. While there has been tremendous effort to discover biomarkers, very few have been translated to the clinic. The bottlenecks in the biomarker pipeline will be highlighted as well as lessons that can be learned from other disciplines, such as oncology.