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December 11, 2000

UW receives $12.83 million for gene therapy research

A federal agency is providing $12.83 million over five years to the University of Washington School of Medicine to investigate both present and future uses of gene therapy.

The University of Washington will have one of four multidisciplinary Programs of Excellence in Gene Therapy that the National Heart, Lung and Blood Institute (NHLBI), one of the National Institutes of Health, is funding over five years.

Also collaborating are researchers from the Fred Hutchinson Cancer Research Center, who are also on the UW faculty. The UW has the only center where all the scientists are on the faculty of a single institution; other recipients include consortiums of universities.

The UW became a center of excellence because of the great depth of talent already gathered here for gene therapy research, says Dr. George Stamatoyannopoulos, the principal investigator for the grant and a UW professor of medicine. He is the head of the Division of Medical Genetics and director of the Markey Molecular Medicine Center at the UW. He is also the founder of the American Society for Gene Therapy.

“This is an extremely exciting program because it utilizes the great depth of talent here at the university,” Stamatoyannopoulos said.

This is the second major grant for investigators at the Markey Molecular Medicine Center that has been announced recently, for a total of more than $18.1 million. In October, the National Institute for Diabetes, Digestive and Kidney Illnesses announced it would provide $5.3 million for work with the stem cells that produce blood. The two grants combined will help support the research of 15 investigators.

In September 1999, investigators at the Markey Molecular Center received another $9.36 million for research into stem cell gene therapy.

“The latest grant and the two previously awarded grants on stem cell molecular biology and gene therapy make the UW the premier institution worldwide for research in the stem cell field,” Stamatoyannopoulos said.

There are three segments to the latest NHLBI award: basic research, clinical trials, and education.

“The emphasis of our research at the University of Washington is on basic research in gene therapy. We will do clinical investigations, but gene therapy is a field in the early stages of its development. We do not lose sight of this reality. We have a lot of hard work to do — a lot of important, basic science — in order to advance gene therapy into its adolescent and then adult stages,” Stamatoyannopoulos said.

Basic science: Investigators will explore the mysterious world of stem cell gene therapy. Stem cells are cells that can transform themselves into vital elements of life, such as blood, brain or muscle cells. Research has shown that stem cells have what scientists call “plasticity.” Stem cells from the muscle can be teased into producing blood as if they were bone marrow cells. Stem cells from the blood can be used to repopulate the liver. Scientists think this plasticity can be a key to new treatments: for example, using stem cells to grow new liver cells for someone with an ailing liver.

The investigators include Dr. Andre M. Lieber, assistant professor of medicine in the Division of Medical Genetics, and Dr. David Russell, associate professor of medicine in the Division of Hematology, who was honored by President Clinton in April with one of the Presidential Early Career Awards for Scientists and Engineers.

“They are investigating in the frontiers of medicine. There is no question that in the next 10 to 15 years, the way we think about gene therapy will change radically,” Stamatoyannopoulos said. “The plasticity of stem cells is an important part of that future.”

Clinical research: A very important element of the Program of Excellence is establishment of a Clinical Gene Therapy Core Facility, under the direction of Dr. Tony Blau, associate professor of medicine, Division of Hematology. Clinical gene therapy studies will be facilitated by the Gene and Cell Therapy Laboratory of the UW Clinical Research Center.

The clinical core facility will include a clinical coordinator and a registered nurse to help with studies on patients. This increased staffing for clinical trials represents a big change for UW gene therapy research. The clinical research component includes:

1. A gene therapy project to develop technologies and then conduct human trials for treatment of cystic fibrosis. The co-directors are Dr. Dusty Miller of the Fred Hutchinson Cancer Research Center’s Human Biology and Basic Sciences divisions and an affiliate professor of pathology at the UW; and Dr. Moira Aitken, associate professor in the Division of Pulmonary and Critical Care Medicine.

2. A gene therapy trial to monitor graft-vs.-host disease. Graft-vs.-host disease occurs after a bone marrow transplant when the bone marrow of a donor starts destroying the cells of the recipient. Researchers hope to use gene therapy to make that disease less likely. The director is Dr. Stanley Riddell, a member of the Hutchinson Cancer Research Center and a UW professor of medicine.

3. Development and testing of a gene therapy to treat sickle cell anemia and thalassemia. Stamatoyannopoulos is the director. The co-investigators are Blau and Dr. David Emery, an assistant professor in the Division of Medical Genetics. The research program will begin not with gene therapy, but with cell therapy studies that involve manipulation of cells. It is expected it will be about three or four years before therapeutic genes could be inserted into the patient’s stem cells.

No clinical gene therapy studies are expected to begin right away. They are either going through the intensive federal approval for such studies, or through pre-clinical research.

Education/training: The university will offer an intensive training program for postdoctoral students in gene therapy. Russell will be the director.

“The goal is to create future investigators who are not only skilled in the laboratory, but are also good scholars. They will understand the breadth of the field in addition to the depth of the field,” Stamatoyannopoulos said.

A total of 14 consortiums and single institutions applied to NHLBI for the four slots. Other Programs of Excellence in Gene Therapy will be led by investigators at Stanford University, Weill Medical College of Cornell University and the University of Pittsburgh.