California Stem Cell and Biofocus DPI to Collaborate in ALS Association-Funded Research Using Human Motor Neurons

Through its TREAT ALS drug discovery and clinical trials program, The ALS Association announces that California Stem Cell (CSC) has signed a contract to supply international drug discovery organization BioFocus DPI, the service division of Galapagos, with its MOTORPLATE 96 assay-ready well plates, containing motor neuron progenitors derived from human embryonic stem cells. BioFocus DPI will use the high purity, clinical grade human motor neurons to perform assay development and screening for amyotrophic lateral sclerosis (ALS or Lou Gehrigs Disease).

The Associations TREAT ALS (Translational Research Advancing Therapy for ALS) program is funding this collaborative research project as part of its global initiative to speed the discovery of new drugs and therapies for ALS.

The ALS Association is focused on accelerating new and promising developments from the laboratory bench into treatments for ALS, said Lucie Bruijn, Ph.D., science director and vice president of The Association. The unique ability of CSC to generate human motor neurons on a large scale, and the target discovery engine provided by BioFocus DPI, will contribute to the development of medicines that may significantly slow the progression of this disease and ultimately lead to a cure.
 
BioFocus DPI chose to source human motor neurons from CSC because its multi-well plate format enables us to develop specialized high-throughput assays not previously possible, said Katherine Hilyard, PhD, vice president of biological sciences, BioFocus DPI. This the first source of high purity human motor neurons available for use in high-content screening assays. This significant advancement allows us to develop innovative human cell-based assays for the discovery of drugs for ALS and other neurological disorders.

CSC is delighted to help our lead customer BioFocus DPI to accelerate its drug discovery programs, and to begin to supply the marketplace with stem cell derived human motor neurons, produced using state of the art, proprietary and commercially scalable manufacturing processes. The potential market for this product in high-throughput and high-content screening analyses for predictive toxicology and drug discovery is very significant, said Dr. Chris Airriess, CSC chief operating officer. More importantly, CSC is proud to work toward the common mission of developing a treatment for ALS.

BioFocus DPI aims to expand its partners drug pipelines by accelerating the gene-to-candidate discovery process. This is achieved through a comprehensive discovery platform, which includes target discovery in human primary cells, focused as well as diverse compound libraries, in vitro and cell-based screening, medicinal chemistry and ADME/PK services, supported by unique chemogenomic and informatics tools. As the service division of Galapagos, BioFocus DPI has over 300 employees in five countries worldwide.

California Stem Cell, Inc. is a privately held company focused on the manufacturing of high-purity human cells for therapeutic development and clinical application. Since its founding in 2005, CSC has developed and has intellectual property surrounding methods for scalable production of human motor neurons, neuronal progenitors, cardiac muscle and sino-atrial node cells at its Irvine, Calif. facility. CSC is currently in the pre-clinical development stage of stem cell based therapies for amyotrophic lateral sclerosis (ALS, or Lou Gehrigs Disease), spinal muscular atrophy (SMA), spinal cord injury (SCI) and coronary heart disease.

The ALS Association is a leader in ALS research and the only national not-for-profit voluntary health organization dedicated solely to the fight against ALS. The mission of The ALS Association is to lead the fight to cure and treat ALS through global cutting-edge research, and to empower people with Lou Gehrigs Disease and their families to live fuller lives by providing them with compassionate care and support.