Biogen collaborates with ALSA and Columbia University on genomic-based ALS treatments
pharmafile | August 18, 2015 | News story | Research and Development |
Biogen, the ALS Association and Columbia University Medical Center (CUMC) have announced a new collaboration aimed at better understanding the differences and commonalities in the 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 next generation genetic sequencing and detailed clinical phenotyping in 1500 people with ALS: a neurodegenerative disease that affects neurons in the brain and the spinal cord.
The disease currently has no cure or life-prolonging treatment, and can often lead to total paralysis and death within two to five years of diagnosis.
GTAC aims to provide a basis for the development of precision medicine, or more bespoke therapies for ALS, and also seeks to set the stage for a nationwide effort to ensure the genomic characterisation of all ALS patients.
The project is being funded through US-based 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, the publicity from which helped the Association gain a record $115 million in donations last year.
ALS Association chief scientist, Lucie Bruijn, says: “We want to bring genomics right to the point of care in ALS where instead of focusing on retrospective DNA samples with limited clinical information, we focus on patients who are under active clinical management. By focusing on patients seen by participating ALS clinics, this project will allow investigators to ask how different genetic causes of ALS translate into different clinical consequences.”
“We know that ALS is not just one disease,” adds Tim Harris, senior vice president, Precision Medicine at Biogen. “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.”
“This kind of ‘precision medicine,’ in which a treatment is tailored to a person’s unique genetic make-up, is already being used in the cancer field. It is an approach we feel is ready for ALS too.”
Patient 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.
“The ability to create patient iPS cells from such a genetically well-annotated ALS blood repository will allow us to model causes of motor neuron degeneration in ALS at a scale that has never been possible,” concludes Dhruv Sareen, leader of the Cedars-Sinai iPSC Core.
Joel Levy
