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Biomarkers Pave the Way for ALS Therapeutics

Clinical trials could more quickly determine that a treatment will help patients with amyotrophic lateral sclerosis (ALS) if the onset and progress of the disease were easier to identify. Diagnosis of ALS is notably difficult, and is often delayed by the insidious beginnings of symptoms that mimic other conditions. Merit Cudkowicz, M.D., of Massachusetts General Hospital in Boston, is part of a collaborative effort to find biomarkers of ALS, to aid trials and to improve diagnosis.

Biomarkers are the biological signature of a process or disease that scientists can detect in the bloodstream or even in other body fluids, such as that bathing the brain and spinal cord. A sample of fluid can show the presence of molecules that reflect altered metabolism or toxic byproducts in a disease process. Modern techniques can find just a few molecules in very small amounts of fluid. These techniques can be harnessed to serve the clinicians who are seeking new treatments and designing new trials for ALS.

Cudkowicz, funded by ALSA, is part of the effort to find unique markers of ALS. She is working with groups in several academic centers and with a North Carolina biotech company called Metabolon, to bring biomarkers to the ALS field. So far, the path towards obtaining biomarkers for ALS has a hopeful beginning. Signals appear in assays that measure components of fluid samples taken from people with ALS. These signals represent small molecules that are more abundant in blood samples from ALS patients as compared to other people sampled.

By looking for patterns in the small molecules present in the blood of ALS patients, clinicians in the future will likely be able to follow the disease course, and get ideas about the pathways that are involved in ALS. Trials of potential therapies would benefit immensely if a simple biomarker could tell if the disease process is being changed.




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