The ALS Association
FOR IMMEDIATE RELEASE
Washington, D.C. (July 12, 2016)— The ALS Association is pleased to work in partnership with the NIH’s National Institute of Neurological Disorders and Stroke (NINDS) to support NeuroLINCS, a collaborative effort among multiple research groups with expertise in induced pluripotent stem cell (iPSC) technology, disease modeling, OMICs and computational biology that is focused on gaining a deeper understanding of neurons and the causes of neurological diseases. The ALS Association contribution to NeuroLINCS is a $2.5 million commitment in partnership with the Greater Philadelphia Chapter over a five-year period to extend the current activities NeuroLINCS, which is one of six National Institute of Health (NIH) Library of Integrated Network-based Cellular Signature (LINCS) centers.
The research laboratories involved include: Steve Finkbeiner, Ph.D., at Gladstone Institutes in San Francisco, Ernest Fraenkel, Ph.D. at Massachusetts Institute of Technology in Cambridge, Mass., Jeffrey Rothstein, M.D., Ph.D. at Johns Hopkins University in Baltimore, Leslie Thompson, Ph.D. at University of California Irvine in Irvine, Calif. and Dhruv Sareen, Ph.D., Clive Svendsen, Ph.D. and Jennifer Van Eyk, Ph.D. all at Cedars-Sinai in Los Angeles.
“The ALS Association is pleased to partner with NINDS on this important initiative. Data generated will contribute to many of the ongoing big data initiatives including Answer ALS and will bring us closer to understanding key pathways and unique signatures involved in ALS, which will better inform clinical trial design and development of treatments,” commented Lucie Bruijn, Ph.D., M.B.A., Chief Scientist, The ALS Association.
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 leads to total paralysis and death, usually 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 NeuroLINCS program is a component of the Library of Integrated Network Signatures (LINCS) program, a trans-NIH initiative supported by the NIH Common Fund, to elucidate how cells respond to diverse chemical perturbations, from toxins to therapeutics. The NINDS and The ALS Association are partnering to support the ALS study as a part of this LINCS large initiative. The ALS arm of this study plans to look at both ALS and Spinal Muscular Atrophy (SMA) that both involve loss of motor neurons that control muscle movement.
“NeuroLINCS represents a highly innovative approach to research that can provide valuable insight into ways in which brain cells are affected by devastating disorders, such as ALS. We are eager to work with the ALS Association to advance our knowledge of ALS and many other neurological diseases,” said Walter Koroshetz, M.D., NINDS director.
The goal of the NeuroLINCS program is to develop cell signatures of an array of human brain cell types under different conditions that should be broadly applicable to the entire LINCS community. To generate a cell signature, data will be collected from the following tests: whole genomic sequencing and transcriptomics and proteomics studies, both in an unperturbed state and importantly, in response to chemical perturbations. From this information, researchers will be able to achieve a larger goal, which is to understand the commonalities and differences among types of ALS, in order to better tailor treatments to specific ALS subtypes.
Specifically, this study will explore signatures in human induced pluripotent stem cells (iPSCs) generated from people living with both ALS and SMA. Currently, the study is analyzing 20 total iPSC lines including four healthy participants, four people living with sporadic ALS, four people with SOD1-ALS, four people with C9orf72 ALS and four people living with SMA. All data generated will be publically available.
For interviews with Dr. Margaret Sutherland, NINDS Program Director, please contact NINDSpressteam@ninds.nih.gov or 301-496-5751
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.