Biogen, the ALS Association and Columbia University Medical Center (CUMC) have formed a new collaboration to better understand the differences and commonalities in the amyotrophic lateral sclerosis (ALS), disease process and how genes influence the clinical features of the disease. The project, “Genomic Translation for ALS Clinical care” (GTAC), will involve a combination of genetic sequencing and clinical phenotyping in 1,500 people with ALS. The goal of the project is to provide a basis for the development of precision medicine, or more individually tailored therapies for ALS. ALS 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.

The project is being funded through Biogen's $30 million strategic alliance with CUMC and $3.5 million from the ALS Association. The ALS Association's commitment comes from funds raised directly through the Ice Bucket Challenge.

Under the collaboration, patients’ blood cells will be stored at the Induced Pluripotent Stem Cell (iPSC) Core, a facility supported by the ALS Association, at the Cedars-Sinai Board of Governors Regenerative Medicine Institute. This cell bank will allow researchers to create cell lines for further study, based on leads provided by genome sequencing. Clinical data will be collected and curated through the NeuroBank system at the Massachusetts General Hospital, and cell lines will be developed at Cedars-Sinai in Los Angeles. Currently, participating clinical sites in this effort include the Cedars-Sinai Board of Governors Regenerative Medicine Institute, Columbia University Medical Center, Duke Medical Center, Houston Methodist, the Scotland ALS clinic network, University of Minnesota and Hennepin County Medical Center, University of Utah, University of Washington, and Washington University in St. Louis.

Source: Biogen