Neurological and Eye Diseases

Multiple Sclerosis (MS): The current rating instruments for assessing the clinical course of MS are a major challenge to the field. Although there are other useful biomarkers to monitor the disease, there is no biomarker for the secondary, progressive stage of MS. Like those working in addiction, workers in the MS field have turned outside their field to cancer studies to glean valuable models that have proved successful. Most important, the cancer field was especially successful in setting up networks in the United States and Europe to foster growth in the design and conduct of, and report development for, cancer biomarker studies. The incorporation of potentially new biomarkers into clinical trials has the potential to be used in future analysis, including the possibility of identifying a surrogate marker.

Stroke: A promising biomarker for acute ischemic stroke, using MRI technology, may hold the key to applications for stroke clinical trials. The field of stroke research needs a formal way to share clinical trial and observational studies data, specifically during Phase II trials, which would help to standardize and optimize MRI data and patient selection and outcomes. Currently, a promising proposal for a multistage approach to standardize, optimize, and establish the use of MRI biomarkers in stroke drug development is being examined.

Spinal Muscular Atrophy (SMA): SMA is ripe for biomarker development, given the identification of a single defective gene over 10 years ago. It has been hypothesized that therapeutics that could increase the expression of this deleted gene may improve motor performance and muscular strength. However, this requires further delineation given that the detection of the SMA biomarker is only correlated with a certain subset of patients.

Retinal Degeneration: Major advances have been made, and are under way, for identifying biomarkers for retinal degeneration and even several neurodegenerative diseases such as MS. Advanced technologies such as optical coherence tomography and adaptive optics, in addition to metabolic biomarker candidates, are lending to further advancement in this area. However, despite the plethora of therapeutic targets, there is a need for increased understanding of the pathophysiology of the disorders.