The ALS Association
FOR IMMEDIATE RELEASE
Collaborative Review Published in Leading Scientific Journal
Washington, D.C., (January 12, 2016) — A review supported by The ALS Association titled “ALS Biomarkers for Therapy Development: State of the Field and Future Directions,” authored by leading experts in the field of amyotrophic lateral sclerosis (ALS), was recently published in in the journal Muscle & Nerve. The review provides an overview of the state of the development of biomarkers, which are used to track disease progression, and calls for the formation of an ALS Biomarker Consortium to coordinate efforts among all stakeholders in an effort to speed progress toward finding effective treatments for people living with ALS.
ALS is a progressive neurodegenerative disease that affects 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. For unknown reasons, veterans are twice as likely to develop ALS as the general population. There is no cure, and only one drug approved by the U.S. Food and Drug Administration (FDA) modestly extends survival.
The review was penned by Michael Benatar M.D., Ph.D., of the University of Miami, Kevin Boylan M.D., of the Mayo Clinic in Jacksonville, Florida; Andreas Jeromin Ph.D., of Iron Horse Diagnostics in Phoenix; Seward B. Rutkove M.D., of Beth Israel Deaconess Medical Center in Boston; James Berry M.D. and Nazem Atassi M.D., both of Massachusetts General Hospital in Boston; and Lucie Bruijn, Ph.D., M.B.A., Chief Scientist for the ALS Association. The collaboration of this publication’s authors is also vital in advising the formation of the FDA Guidance Document for ALS clinical trials.
“The ALS Association has invested significant funds towards the development of biomarkers for ALS and will continue its commitment by investing further funds to establish the ALS Biomarker Consortium and seeks dedicated involvement from all stakeholders,” said Lucie Bruijn, Ph.D., M.B.A., and Chief Scientist for The ALS Association.
The need for validated biomarkers for ALS has taken on increased significance with the ongoing development of a Guidance Document, spearheaded by The ALS Association using funds from the ALS Ice Bucket Challenge. This guidance will be submitted to the U.S. Food and Drug Administration and will serve as a roadmap to help industry navigate the development process and provide the agency with an ALS community-centered view of how it should approach therapies for ALS. The named authors are currently working together and are key in advising the biomarker topic area that will be incorporated into the Guidance Document.
The review and the recognition of a pressing need for an ALS Biomarker Consortium grew out of a series of meetings, including the following: a biomarker workshop co-sponsored by The ALS Association and ALS Therapy Development Institute (TDI) in May 2014; a biomarker symposium at the first annual ALS Research Group meeting in September 2014; and, the emergence of the Clinical Research in ALS and related disorders for Therapeutic Development (CReATe) Consortium, a National Institutes of Health (NIH)-supported effort for which the discovery and validation of biomarkers relevant to therapy development is a specific focus.
According to the authors of the review, “This is an opportune time for the establishment an ALS Biomarker Consortium that includes all relevant stakeholders. If it is to succeed, the structure and governance of this consortium must recognize and accommodate the complexity of the ALS research landscape, including the number and diversity of stakeholders who may often have competing interests,” like academic researchers, small biotech companies, and large pharmaceutical companies.
About The ALS Association
The ALS Association is the only national non-profit organization fighting Lou Gehrig’s Disease on every front. By leading the way in global research, providing assistance for people with ALS through a nationwide network of chapters, coordinating multidisciplinary care through certified clinical care centers, and fostering government partnerships, The Association builds hope and enhances quality of life while aggressively searching for new treatments and a cure. For more information about The ALS Association, visit our website at www.alsa.org.