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Huntington's Stem Cell Project is First for Regenerative Medicine Institute

The Cedars-Sinai Regenerative Medicine Institute is providing stem cells to a five-member National Institutes of Health consortium of researchers for development of potential therapies to treat Huntington's disease.

As part of a $3.7 million grant from the NIH, the Cedars-Sinai Regenerative Medicine Institute will supply scientists at five leading laboratories, including Cedars-Sinai, with all the adult stem cells used in the study. The consortium comprises a partnership of five leading Huntington's disease laboratories at Cedars-Sinai, the Gladstone Institutes, Johns Hopkins, Massachusetts General Hospital and the University of California at Irvine.

The goal of the project is to compare stem cells from patients with Huntington's to stem cells from healthy patients in an effort to understand why brain cells die in Huntington's patients, causing uncontrollable body movements and psychological changes. The project will use induced pluripotent stem cell technology, which enables specialized stem cells to be generated from adults' skin samples.

"Regenerative medicine could enable us to untangle the mystery of this inexorably fatal disease," said Clive Svendsen, Ph.D., director of the Cedars-Sinai Regenerative Medicine Institute. "One of the problems we have faced is that treatments that work in animals are ineffective in people. Now we have an opportunity to study this disease at a cellular level and collaborate with others dedicated to finding effective treatments."

The Huntington's project is the first endeavor announced since Dr. Svendsen was selected as director of the Cedars-Sinai Regenerative Medicine Institute, which brings together basic scientists with specialist clinicians, physician scientists and translational scientists across multiple medical specialties to translate fundamental stem cell studies to therapeutic regenerative medicine.

The Institute will be housed in new state-of-the-art laboratories being constructed for stem cell and regenerative medicine research. At the heart of the Institute will be a specialized core facility for the production of pluripotent stem cells (capable of making all tissues in the human body) from adult human skin biopsies. Cells produced within the Institute would be used in a variety of Cedars-Sinai Medical research programs (initially focusing on understanding the causes of and finding treatments for diseases of the brain, heart, eye, liver, kidney, pancreas and skeletal structures, as well as cancer and metabolic disorders).

The Institute's new space in the Steven Spielberg Building is nearly completed.