At the Cedars-Sinai Regenerative Medicine Institute, future of medicine's here today

Under the leadership of Clive Svendsen, PhD, the Regenerative Medicine Institute is experiencing unprecedented growth as Cedars-Sinai scientists and physicians work together to translate stem cell science into potential leading-edge treatments for diseases and genetic disorders.

The Institute - home to prominent stem cell scientists who specialize in diseases, injuries and birth defects including spinal muscular atrophy, heart disease and diabetes to vertebral compression fractures and Alzheimer's - coordinates, assists and consults with stem cell researchers in most aspects of the medical center’s clinical activities.

The mission of the Institute is to bring together basic scientists with specialist clinicians, physician scientists and translational scientists across multiple medical specialties to move as quickly and safely as possible fundamental stem cell studies in the laboratory to therapies to benefit patients at the bedside.

The Institute has just opened a new Induced Pluripotent Stem Cell (iPSC) Core Facility capable of producing induced pluripotent stem cells (iPSCs) from adult human fibroblasts or other tissue samples. To produce a line of iPS cells, clinicians can take skin cells from patients with specific life-threatening medical conditions. Then, Regenerative Medicine Institute scientists can create "disease in a dish" models that enable them to more easily identify effective therapies.

"Now, for the first time, we can study human diseases by creating a laboratory specimen of afflicted cells," said Svendsen, director of the Cedars-Sinai Regenerative Medicine Institute. "The theory is that if we can put a disease in a Petri dish, we can treat it in the laboratory without needing human participants. We can also test novel drugs on the patients' own cells to check for potential side effects. One day, we predict that this will herald a new era of personalized medicine for patients at Cedars-Sinai and around the world."

The Stem Cell Core Facility already is supplying iPS cells to a five-member NIH consortium of researchers for development of potential therapies to treat Huntington's disease, an incurable neurodegenerative genetic disorder afflicting muscle coordination and some cognitive functions, such as memory. With funding from the state stem cell agency, the Cedars-Sinai core also has generated iPS cells from children with spinal muscular atrophy - a lethal disease that leaves children paralyzed. These are being used to develop novel drug compounds to treat this devastating disorder.

A record of accomplishment

The Institute resides in the Steven Spielberg Building, in state-of-the-art laboratories built specifically for stem cell and regenerative medicine research. Cells produced within the Institute are used in various 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.
Among the Institute's 62 employees are 22 scientists with PhDs – including 10 post-doctoral researchers – and five MDs – including three MDs who also have earned PhDs.

And the Institute’s success is a matter of record:

  • Since Svendsen joined Cedars-Sinai in 2009, when the Institute was founded, Regenerative Medicine Institute scientists have been awarded 19 grants totaling more than $8 million from the National Institutes of Health (NIH), the California Institute of Regenerative Medicine and the U.S. Department of Defense.
  • In the past two years, Institute scientists have had 43 studies and analyses published in peer-reviewed publications.
  • Fourteen percent of the embryonic stem cells approved, and funded, by the National Institutes of Health for research come from Cedars-Sinai. Cedars-Sinai has contributed 17 approved embryonic stem cells lines - second only to Harvard University.
  • Six Cedars-Sinai stem cell researchers, including Svendsen, Heart Institute Director Eduardo Marbán, MD, PhD and Dean of the Faculty Shlomo Melmed, MD, were featured at the 2011 World Stem Cell Summit.

Svendsen, formerly a professor of neurology and anatomy at the University of Wisconsin-Madison is conducting groundbreaking research using a combination of stem cells and powerful growth factors to both model and treat neurodegenerative disorders. These include amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease) and Parkinson's disease. As director of the National Institutes of Health-funded Stem Cell Training Program at Wisconsin and editor of the Encyclopedia of Stem Cell Research, he had a long interest in stem cell education, public policy and community outreach.