Puget Sound Blood Center Release: Blood Center Gene Therapy Researcher Awarded Grant

SEATTLE, March 28 /PRNewswire/ -- The Hemophilia Association of New York recently awarded Blood Center Principal Investigator Neil Josephson, M.D., a two-year grant to study methods for the prevention and treatment of immune responses in severe hemophiliacs to certain blood clotting proteins. The study is scheduled to begin in April 2005.

Dr. Josephson, who is also an Assistant Professor in the Hematology Department at the University of Washington Department of Medicine, was awarded the $227,513 grant on the strength of his proposal, "Induction of Immune Tolerance to Factor VIII by Tolerogenic Dendritic Cells." The proposal suggests that a special class of immune cells, known as tolerogenic dendritic cells, may prevent or diminish immune reactions to the Factor VIII concentrates that control bleeding in patients with severe hemophilia.

Patients with hemophilia A produce reduced levels of normal functioning Factor VIII, a protein that is critical for clot formation. Individuals with the most severe form of the disease need frequent infusions of Factor VIII concentrates to control bleeding that occurs spontaneously or as the result of minimal injury. Unfortunately, a significant number of these patients develop immune responses to the infused factor concentrates, making them ineffective for the control of bleeding episodes.

While alternative clotting factors are available to control bleeding in these patients, they are all less effective and significantly more expensive than Factor VIII concentrates. Other treatments can eliminate the immune response so that the body no longer recognizes Factor VIII as foreign. However, this type of therapy, called immune tolerance induction, can take up to one year to be effective and costs about $1 million for the average patient. It also fails about one-third of the time.

By isolating and treating the cells responsible for directing the immune system to down regulate its response to specific foreign proteins, Dr. Josephson hopes to develop a faster, more effective, and less expensive method for inducing immune tolerance in hemophilic patients to Factor VIII.

"The grant from the Hemophilia Association of New York is a tremendous validation for the work we are doing at the Blood Center," said Dr. Josephson. "As one of only four research blood centers in the country, the Blood Center is in a unique position to collaborate among its various laboratories, leveraging our nationally recognized research in hemophilia, immunology and stem cell gene therapy to discover new therapies and advance transfusion medicine."

Joining the Blood Center in 2002, Dr. Josephson's other research projects focus on developing strategies for efficiently transferring therapeutic genes into blood stem cells. Stem cell gene therapy has the potential to improve the quality of life for thousands of people, offering great promise in the fight against disorders such as sickle cell disease, thalassemia, and inherited immune deficiencies.

Other Blood Center researchers collaborating with Dr. Josephson on the hemophilia study will be Arthur R. Thompson, M.D., Ph.D., director of Puget Sound Blood Center's Hemophilia Program, Yvette Latchman, Ph.D., a senior investigator at the Blood Center with expertise in immunology, and Ruijun Su, M.D., Ph.D., a post-doctoral fellow with the Blood Center.

The Hemophilia Association of New York's seeks to improve the quality of life for all people affected by hemophilia and other coagulation disorders. The organization supports research that either leads to greater understanding of the disease or brings advances in the treatment of hemophiliacs and others with congenital bleeding disorders.

About Puget Sound Blood Center

Celebrating 60 years in the community, the Blood Center is internationally recognized for groundbreaking research in transfusion and transplantation medicine. The nonprofit Blood Center is the resource for patients in Western Washington who need blood, tissue and specialized laboratory services. Founded in 1944, the Blood Center has a long and unique tradition of blending community volunteerism, medical science and research to improve patients' lives. The organization directly affects the lives of over half million patients annually in more than 70 hospitals and clinics in 14 counties of Washington State and provides tissue and transplantation support to 185 hospitals across the Northwest.

Puget Sound Blood Center

CONTACT: Steve McLean, +1-206-292-6511, or stevemc@psbc.org, or pager,+1-206-680-2610

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