In August 2015, The Association committed $3.5 million from money raised through the ALS Ice Bucket Challenge to the Genomic Translation for ALS Care (GTAC), a collaborative nationwide precision medicine initiative through Columbia University Medical Center (CUMC) and Biogen, which is being led by Dr. Matthew Harms. GTAC is actively enrolling now (scroll to the bottom for more details) with the ultimate goal of ensuring the genetic characterization of all people living with ALS.
“There are several exciting aspects of the GTAC study,” said Dr. Harms. “On a larger scale than ever before, this study will combine the genomes, gene expression profile and clinical symptoms of a patient to look for shared factors among patients with ALS. We hope this information will allow us to design more successful clinical trials and eventually design personalized therapies for patients. The GTAC study also plans to return genetic findings to its participants, allowing them to learn about how their genes might be contributing to their disease.”
How GTAC works
The study will follow 1,500 people living with ALS in the clinic over a three year period with three- month visit intervals. Clinical history, disease progression and family history data are collected during clinic visits. Blood samples will also be taken for multiple analyses, including whole genome sequencing (to read each person’s full DNA sequence), transcriptome analyses (study of RNA), and peripheral blood mononuclear cells (pBMCs) are stored to make induced pluripotent stem cells (iPSCs) in the future. The clinical data being collected from participants is harmonized with other genomics efforts in ALS and the epidemiologic studies being carried out by the Centers for Disease Control (CDC).
Collaboration is key to GTAC
This is a collaborative effort with multiple partners contributing. Whole genome sequencing and transcriptomics are performed in collaboration with the New York Genome Center. 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 Dr. Dhruv Sareen. 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).
There are 10 enrollment 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.
The GTAC Impact
This collaborative effort is already responsible for identifying ALS gene TBK1 in 2015 that was published in the journal Science. Research carried out through GTAC will not only contribute to the identification of new ALS genes, but will also seek to accomplish the following:
- Better understand how different genes contribute to various clinical forms of ALS. This will in turn assist researchers design better, more focused clinical trials for the development of more effective treatments;
- Shed light on the range and types of mutations in known inherited ALS genes;
- Pinpoint the understanding of which symptoms and disease patterns are associated with specific genes or mutations; and
- Create a library of cell lines for which complete genome and clinical data is already available, which will give the global ALS community an invaluable resource for studying the diverse forms of ALS.
How to get involved in GTAC
The project is actively enrolling participants now. For more information see:
Watch a recent webinar on
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.