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Biogen, ALS Association, Columbia to Study ALS Genomics in 1,500 Patients

NEW YORK (GenomeWeb) – Biogen, the ALS Association, and Columbia University Medical Center (CUMC) are collaborating to study the genetics of amyotrophic lateral sclerosis (ALS).

Under a new project called Genomic Translation for ALS Clinical Care (GTAC), the partners will perform next-generation sequencing and detailed clinical phenotyping on 1,500 ALS patients. Patient blood cells will be stored at the Induced Pluripotent Stem Cell Core at the Cedars-Sinai Board of Governors Regenerative Medicine Institute, allowing researchers to create cell lines for further study.

The project is funded through a $30 million strategic alliance between Biogen and CUMC, announced earlier this year, and $3.5 million from the ALS Association that was raised through the Ice Bucket Challenge. Biogen Idec changed its name to Biogen in March.

"This study will help in developing a detailed understanding of how different genes contribute to different clinical forms of ALS. This will in turn help us design better, more focused clinical trials for the development of more effective treatments," said Tim Harris, senior vice president of precision medicine at Biogen, in a statement.

While other ALS projects have used archived DNA samples, "this project reflects our commitment to using ALS genomic studies to benefit patients directly through diagnoses and to set the stage for genetically stratified clinical trials," David Goldstein, professor of genetics and development and director of the Institute for Genomic Medicine at CUMC, said in a statement.

Clinical sites participating in the project include Cedars-Sinai, CUMC, Duke Medical Center, Houston Methodist, the Scotland ALS clinic network, the University of Minnesota and Hennepin County Medical Center, the University of Utah, the University of Washington, and Washington University in St. Louis. Clinical data will be collected and curated by the NeuroBank system at Massachusetts General Hospital.

One aim of the project is to prepare for a nationwide effort to characterize the genomes of all patients with ALS.

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