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Genomic Translation for ALS Care (GTAC)

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Genomic Translation for ALS Care (GTAC) is a collaboration with Biogen and Columbia University Medical Center (CUMC) to better understand how different genes contribute to various clinical forms of ALS. This will in turn help researchers design better, more focused clinical trials for the development of more effective treatments. This kind of precision medicine, in which treatment is tailored to each person’s unique genetic makeup, will be effectively applied to ALS.

The ALS Association Commitment

The ALS Association committed $3.5 million that was announced in August 2015. This commitment comes directly from money raised through the Ice Bucket Challenge. This study is also being funded through Biogen’s strategic alliance with CUMC.

Goal

This initiative aims to set the stage for a nationwide effort to ensure the genomic characterization of all people with ALS. The study will follow 1,500 people living with ALS in the clinic over a three year period with three month visit intervals to collect clinical data, sequence their DNA and store blood samples to generate induced pluripotent stem cells (iPSCs). This information will allow the correlation of ALS clinical manifestations to the genetic causes and help stratify patients for future clinical trials. Ultimately, it will provide a basis for the development of precision medicine, or more individually tailored therapies for ALS.

How the GTAC advances the search for a treatment for ALS

  • Collects clinical progression data at the clinic
      • Historical ALS data (onset and disease progression) for deep clinical phenotyping (comprehensive study of clinical history and disease progression)
      • Family history
      • Exposures and epidemiologic data
      • Neurological examinations
  • Collects blood samples from people living with ALS
      • To sequence whole genomes to read the full sequence of DNA of each participant
      • Analyzes transcriptomics (study of messenger RNA (mRNA)
  • Collects pBMCs (peripheral blood mononuclear cells) archived to make iPSCs in the future
  • Shares information in a database that will allow for a large investigation of the clinical correlates of the genetic causes of ALS

Key Players:

  • Matthew Harms, M.D., Director GTAC and Assistant Professor of Neurology at CUMC
  • David Goldstein, Ph.D., Director of the Institute for Genomic Medicine at CUMC and Professor of Genetics and Development at CUMC
  • Dhruv Sareen, Ph.D., Director of Induced Pluripotent Stem Cell Core Facility, Cedars-Sinai, Los Angeles.
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(left to right) Dr. David Goldstein, Dr. Dhruv Sareen and Dr. Matthew Harms

Participant 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 in Los Angeles led by Dhruv Sareen, Ph.D. Clinical data will be collected and curated through The ALS Association supported NeuroBANK™ at Massachusetts General Hospital in Boston. Each sample will have a unique identifier called a global unique identifier {GUID). Whole genome sequencing and transcriptomics are performed in collaboration with the New York Genome Center.

There are 10 centers across the U.S. and in Scotland collecting samples. Participating clinical centers include: Cedars-Sinai Board of Governors Regenerative Medicine Institute, Columbia University Medical Center, University of Michigan, Houston Methodist, Scotland ALS Clinic Network, University of Minnesota and Hennepin County Medical Center, University of Utah, University of Washington, Penn State Hershey Medical Center and Washington University in St. Louis.

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GTAC enrollment centers

Progress:

Study started enrolling in June 2016 with a projected timeline of three years with follow-ups in three month intervals. Data will be shared immediately to the CGND consortium and publically at later stages. This collaborative effort is already responsible for identifying ALS gene TBK1 in 2015 that was published in the journal Science.

See the GTAC project page for more information and a recent progress report.

How to get involved in GTAC

The project is actively enrolling participants now. For more information see:


Past Webinar

Genomic Translation for ALS Care (GTAC study): Envisioning the Precision Medicine Future for ALS

Speaker: Matthew Harms, Ph.D., Director of GTAC and Assistant Professor of Neurology at Columbia University Medical Center

Genomic Translation for ALS Care (GTAC) is a collaboration with Biogen and Columbia University Medical Center (CUMC) to better understand how different genes contribute to various clinical forms of ALS. This will in turn help researchers design better, more focused clinical trials for the development of more effective treatments. This kind of precision medicine, in which treatment is tailored to each person’s unique genetic makeup, will be effectively applied to ALS. On October 11th, join us to learn more about this exciting strategic initiative supported by The ALS Association.

Watch this Webinar

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