LA JOLLA, CA--The Salk Institute for Biological Studies and Columbia University Medical Center have been awarded a $15 million grant by The Leona M. and Harry B. Helmsley Charitable Trust, establishing a collaborative program to fast-track the use of induced pluripotent stem (iPS) cells to gain new insight into disease mechanisms and screen for novel therapeutic drugs.
"Stem cell research is of immense importance to the future of biomedical research and will have a major impact in treating and preventing devastating diseases," said Fred H. Gage, Ph.D., a professor in the Laboratory for Genetics at the Salk Institute and the Vi and John Adler Chair for Research on Age-Related Neurodegenerative Diseases.
"The funding from The Helmsley Trust will accelerate and deepen our research efforts in stem cell biology, already an area of strength at the Salk Institute. In addition, this funding allows researchers at the Salk to join forces with outstanding researchers at Columbia University in a synergistic enterprise that will bring stem cell research closer to fulfilling its promise," he said.
The ability to reprogram adult human cells into iPS cells, which by all appearances look and act like embryonic stem cells, creates a unique opportunity to study human disease in revolutionary ways. After taking a few skin cells from patients, researchers can generate iPS cells and differentiate them into the type of tissue where a disease is manifest.
Over the course of the three-year grant, the Salk will develop a stem cell bank of well-characterized iPS cells derived from patients suffering from debilitating neurological, cardiac and hematological conditions. Both institutions will use these cell lines and other stem cell-based tools to generate the cell types (nerve cells, muscle, blood cells and other tissues) affected in a wide range of diseases, and to determine the molecular causes of disease phenotypes.
These cell-based models of disease will allow investigators from both institutions to screen at Columbia tens of thousands of chemical compounds to uncover novel drug therapies for hitherto untreatable diseases. The grant will therefore create a pipeline of new models and molecules that will start from individual patients and create new avenues back to the clinic.
Christopher E. Henderson, Ph.D., Director of the Columbia Stem Cell Initiative, and Co-Director of the Columbia Motor Neuron Center, said, "Although much public interest has been focused on the use of stem cells for tissue replacement, we and our collaborators at the Salk believe that in many clinical areas their major benefit will be to allow us to model diseases in the culture dish. Such cell-based models can generate new knowledge about the disease process itself, while also opening the way to high-throughput screening for test drug candidates."
Henderson added, "The Helmsley Trust support at Columbia will be focused on freeing up two key bottlenecks in this process. It will fund new protocols to allow scientists to transform stem cells into specific classes of differentiated cells, and will help support a facility that will allow researchers with a disease model to perform drug screening on a scale inaccessible in their own laboratories."
"The Helmsley Trust is showing great vision by investing in two scientific groups operating at the leading edge of stem cell research," said Salk Institute President William R. Brody. "We are deeply grateful to the trustees and look forward to a very fruitful collaboration with our Columbia University colleagues."
"The generosity of The Helmsley Trust enables research that ultimately may lead to prevention or treatment of diseases that currently have no cure," said Lee Goldman, M.D., Dean of the Faculties of Health Sciences and Medicine and Executive Vice President for Health and Biomedical Sciences at Columbia University Medical Center. "We look forward to working with our collaborators at the Salk Institute and delivering on the promise of stem cell research."
John Codey, Trustee of The Helmsley Trust stated, "We are excited to bring together two world-class institutions to work collaboratively from the bench to the bedside in diseases including Alzheimer's, ALS and Parkinson's Disease. We hope this work will help advance this important work in these and other diseases."
|Contact: Kat Kearney|