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Oregon Stem Cell Center Hunt for Human Disease Therapies

Published on March 23, 2004 at 6:28 PM · No Comments

Oregon Health & Science University's fast-growing stem cell research program, which already has made significant strides in the hunt for human disease therapies, now has a place to call home.

The multidisciplinary Oregon Stem Cell Center, the first of its kind in the Northwest, is studying cell and gene therapy as an alternative to organ transplantation for pancreatic and liver disorders, diabetes, cancer and a host of other human diseases. Its focus will be on adult stem cells.

Launched in January, the center is housed among several laboratories on the Marquam Hill Campus, but it will occupy a single floor in the 270,000-square-foot Biomedical Research Building now under construction and to be completed in fall 2005.

Its director, Markus Grompe, M.D., professor of molecular and medical genetics, and pediatrics in the OHSU School of Medicine, said the center is a hub for all areas of OHSU's rapidly expanding stem cell biology program. It aims to maximize the potential of various stem cells as therapies for human diseases through a rapid, "bench-to-bedside" approach involving basic research in stem cell biology and preclinical trials of therapies in animal models, followed by human trials.

"This is something we needed but didn't have," said Grompe, who has long studied gene and cell therapy for metabolic genetic diseases, as well as the molecular genetics of a rare, cancer-susceptibility syndrome called Fanconi anemia. "The consensus here at OHSU is that this is an area that is going to be important."

While the center's offerings will widen over time to cover other diseases -- Parkinson's disease and diabetes are considered "hot" areas of stem cell research -- it will concentrate on two areas for now and "build on that."

"My idea is to focus on the liver and the pancreas, and focus on adult stem cells," he said. "Our research already is advanced in liver reconstitution by stem cells and the repair of liver disease. We're clearly identified as one of the leaders in that area."

OHSU researchers like Grompe are wasting no time demonstrating the importance of stem cells. His laboratory is renowned as an international leader in cell therapy for liver and pancreas diseases as an alternative to organ transplantation.

Last year, Grompe published a study showing that bone marrow-derived stem cells from mice can combine with liver cells through cell fusion, which occurs when two or more cells combine to form one cell containing more genetic material than normal. The method reversed liver damage.

Other studies indicate bone marrow stem cells can meld with cells of other tissues, such as brain, spinal cord, lung, intestine and heart muscle.

The center's formation reflects a swell of research at OHSU involving stem cells. Scientists are conducting basic research, including molecular-level studies, in cardiology, endocrinology, genetics, hematology and oncology, neurology, neurological and general surgery, and reproduction.

"Basically all diseases which are currently being treated by organ transplantation are, at least on paper, amenable to cell therapy," Grompe said. "Our hope is that procedures as effective as whole organ transplantation will come out of (the center)."

Of particular interest to School of Medicine scientists are recently discovered clonally self-renewing stem cells. These unique cells have energized stem cell research – and are broadening the discipline's appeal to a larger group of scientists – because of their ability to generate copies of themselves and further divide into special-purpose offspring.

Clonally self-renewing stem cells come in several forms, such as mesenchymal stem cells, neural stem cells and multipotent progenitor cells, and can be used to create multiple cell types, including nerve cells, liver cells and muscle cells. They can be isolated from mice and primates, including humans, manipulated outside the living organism, and transplanted for reconstituting tissue.

"Their ability to be expanded in culture and then differentiated make them attractive for use in cell therapy," Grompe said.

Dan Dorsa, Ph.D., OHSU vice president for research and professor of physiology and pharmacology in the School of Medicine, said stem cells hold promise for treating many disorders. As a result, OHSU has the potential to make "a very broad impact."

"The use of stem cells for treating diseases will be at the forefront," Dorsa said. "The question we want to answer is: What is it about stem cells that allow them to be viable and take on the roles in the body you hope they will?"

The Oregon facility is one of only a handful of stem cell research centers around the country. Other sites include the University of Minnesota, Stanford University, the University of California, San Francisco, and the University of California, San Diego.

The heart of the center is three core facilities that provide cell development and management services for all campus research. They include:

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