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$16.8 million grant awarded to develop platelet recovery treatments

SEATTLE The National Heart, Lung and Blood Institute of the National Institutes of Health has awarded a $16.8 million, seven-year grant to launch a bicoastal research partnership between Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium and Children's Hospital of Philadelphia.

The collaboration, led by scientists at the Hutchinson Center, will develop molecular- and cell-based therapies for a range of blood diseases, with an initial focus on a life-threatening complication of stem-cell transplantation called thrombocytopenia. The condition, which is sometimes associated with abnormal bleeding, is caused by a delayed recovery of blood-clotting cells called platelets.

The problem is that some stem cell transplant patients, particularly those receiving stem cells from cord blood, often do not produce enough platelets in a timely fashion which puts them at risk for life-threatening bleeding. Platelet production can be delayed by several months in persons who receive a stem cell transplant to treat certain cancers. Transfusions can help, but the number of platelet donors is limited, transfusions carry a risk of blood-borne diseases, and patients who receive multiple transfusions can develop antibodies that destroy the platelets.

According to principal investigator Beverly Torok-Storb, Ph.D., a member of the Hutchinson Center's Clinical Research Division, the goal is to control the process of blood cell production so that all types of blood cells - red cells to carry oxygen, white cells to fight infection and platelets to prevent bleeding - recover together within a few days.

The partner institutions will focus on two complementary strategies to address delayed platelet production. Scientists at the Hutchinson Center and the UW will develop reagents that can be administered to patients to stimulate the differentiation and proliferation of precursor cells into platelets. The Philadelphia group will work to generate "ex vivo," or outside the body, platelets and their precursors from embryonic stem cells for use as cell therapy. The principal investigator in Philadelphia is Mortimer Poncz, M.D., chief of the Division of Hematology at Children's Hospital of Philadelphia.

The Hutchinson Center/UW group will receive a total of $8.2 million during the seven-year grant; the Philadelphia research group will receive $8.6 million.

"This coast-to-coast collaborative effort will take advantage of complementary skills and interests in these two institutions to develop new therapies for all patients needing platelet transfusions," Poncz said.


Contact: Dean Forbes
Fred Hutchinson Cancer Research Center

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