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Carrie Munk
The ALS Association
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The ALS Association and CReATe Consortium Announce Biomarker Discovery and Validation Projects

Washington, D.C. (September 29, 2015) — The ALS Association and the CReATe Consortium are pleased to announce the selection for funding of two new projects that will advance the discovery and validation of biomarkers relevant to ALS therapy development.

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 goal of the first project is to develop a single molecule detection assay for the dipeptide repeat proteins (DPRs) generated by the ALS- and frontotemporal degeneration (FTD)-causing repeat expansion in C9orf72. Researchers will employ a methodology that is capable of single molecule detection and has sensitivity up to 1000-fold higher than current enzyme-linked immunosorbent assay (ELISA) approaches. Using this approach, they hope to detect these DPRs in the blood of patients with C9orf72 ALS/FTD. Successful completion of this project would represent a major advance, as a simple blood test could then be used to inform how well a drug is working and greatly simplify the assessment of new therapies for ALS or FTD caused by mutations in the C9orf72 gene.

The second project will build on researchers’ prior observation that populations of innate lymphoid cells are increased in the blood of ALS patients. The researchers hope to better define the role of these cells in ALS by tracking specific innate lymphoid cell populations in individual patients over the course of a year and determining whether these cells express higher levels of pro-inflammatory signals, which may exacerbate disease. Demonstrating that innate lymphoid cells are associated with disease progression will allow investigators to use these cells to both track and predict disease progression. This research may permit alteration of innate lymphoid cell function using novel or existing therapeutics to extend the lifespan of patients with ALS.

“The ALS Association is extremely excited to partner with the CReATe Consortium in supporting these efforts to develop biomarkers that will be so essential to our collective efforts to identify effective therapies for this group of disorders,” said ALS Association Chief Scientist Lucie Bruijn, Ph.D., M.B.A.

The first study will be led by Dr. Adrian Isaacs, a Reader (Associate Professor) in the Department of Neurodegenerative Disease at University College London. The second will be led by Dr. Benjamin Murdock, a Research Investigator in the Department of Neurology at the University of Michigan.

About CReATe Consortium
The CReATe (Clinical Research in ALS and related disorders for Therapeutic development) is a Rare Diseases Clinical Research Consortium (RDCRC) that forms part of the National Institutes of Health (NIH) Rare Diseases Clinical Research Network (RDCRN). A major goal of the CReATe Consortium is to foster the discovery and validation of biomarkers relevant to therapy development for patients with ALS and related disorders. In an exciting partnership with the ALS Association, CReATe is able to advance this goal through the release an annual RFA to fund biomarker pilot projects. CReATe also fosters biomarker development by making its substantial repository of biological samples available to the broader scientific community.

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

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