Pharmaceutical and academic leaders come together to discuss biomarkers in ALS

May 19 2014

Leaders from academic institutions, non-profit organizations, and pharmaceutical companies will meet in Cambridge, Mass., on Monday, May 19, to discuss biomarkers in amyotrophic lateral sclerosis (ALS).

ALS, which is also known as Lou Gehrig's Disease, is a progressive neurodegenerative disease that affects neurons (nerve cells) in the brain and the spinal cord. Eventually, people with ALS lose the ability to initiate and control muscle movement, which often leads to total paralysis and death within two to five years of diagnosis. There is no cure and no life-prolonging treatments for the disease. 

A biomarker is any measurement, such as a blood test or imaging result, that tracks the disease process and the effects of treatment. Cholesterol is a biomarker for heart disease, for instance, because its level correlates with the risk for the disease. Biomarkers are especially important for therapy development because they allow researchers to determine if a new treatment is having the intended biological effect. The existence of a reliable progression biomarker can influence a pharmaceutical company's decision whether to invest the millions of dollars needed to support a large clinical trial. Most importantly, biomarkers would expedite clinical trials and determine whether a treatment effect is beneficial or not and whether the drug has reached its target. Although several biomarkers are emerging as promising for ALS, significant effort is required to validate and standardize biomarkers. A consortium effort to make this happen is essential and is the focus of the upcoming meeting.

This meeting will be held in collaboration with ALS Therapy Development Institute (ALS TDI), and leaders from 24 companies and academic institutions will attend this event. Other participants include Genentech, Pfizer, Astra Zeneca, Biogen Idec, Bristol-Myers Squibb, NextGen Sciences, University of Massachusetts Medical School, Massachusetts General Hospital, SUNY Upstate Medical University, Barrow Neurological Institute, Voyager Therapeutics, Michael J. Fox Foundation and Brigham and Women's Hospital, Harvard Medical School.

"This is an extremely important initiative and collaboration between academia and industry is critical to the development of biomarkers, said Lucie Bruijn, Ph.D., M.B.A., The ALS Association's Chief Scientist. "I look forward to productive discussions and fruitful partnerships in the months ahead."

SOURCE The ALS Association