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September 15, 2006

Validity of Proposed Markers of ALS to be Established by New Funding

Roberta Friedman, Ph.D., Research Department Information Coordinator

[Quick Summary: New funding by The ALS Association will verify that a proposed set of marker molecules identified in ALS patients can reliably reflect disease stage and progression to provide faster diagnosis and accelerate clinical trials.]

The need to speed diagnosis of ALS and to reliably measure progression of the disease may be met by a set of metabolic markers identified by research funded by The ALS Association. Now The Association is funding a study, as part of a consortium effort to provide ALS biomarkers, that will follow the appearance of metabolic signposts over time in patient samples to validate the ability to accurately reflect the degree of disease.

The ability of a set of marker molecules to show that a patient has ALS and to reflect the degree of damage to the nervous system would provide more accurate and rapid diagnosis and would also accelerate the testing of candidate therapies. A particular set of biochemical markers, serving as signatures of the disease process, were determined to appear in ALS patients, but a further validation will be required to establish these as true diagnostic markers.

The research on biomarkers must establish how early in the disease these biomarkers appear and whether the markers truly correlate with disease progression.  To fully validate the proposed disease signatures, funded researchers at Metabolon, Inc., will examine blood plasma samples from 66 subjects who participated in a 12 month clinical trial of topiramate in ALS. These patients had been assigned to the placebo treatment in that trial, and their collected samples at several time points were stored and are still preserved. 

Three or four samples were obtained from each person over the 12 months of the trial.  Moreover, each patient had clinical measurements of their disease progression.  These include muscle strength testing, respiration (forced vital capacity) and the questionnaire of daily living challenges called the ALS functional rating scale. These established measures of the disease should correlate over time with changing chemistry that can be measured in the blood plasma collected and stored if these proposed biomarkers are valid.

The investigators will be looking at changes in metabolic molecules that reflect a patient’s biochemical activities, ones that appear to be specific for the disease process. The researchers will track those metabolic molecules that increase or decrease as the disease progresses and pick from those the ones that truly correlate with each other and with disease progression. 

The researchers will establish which of these molecules are the most informative and reliably able to measure progression of the disease. 

For further information about the promise for biomarkers in ALS, click here.

 

 



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