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The first drug whose potential was discovered in zebrafish has now made it to clinical trial. In studies led by Children’s Hospital Boston’s Stem Cell Program Director, Leonard Zon, MD, and former research fellows Trista North, PhD, and Wolfram Goessling, MD, PhD, large-scale screening in zebrafish revealed that prostaglandin E2 (PGE2) boosts numbers of blood stem cells in zebrafish. It’s now hoped that the same drug will help people with blood diseases rebuild their blood systems more quickly.
As reported in Nature in 2007, Zon and colleagues homed in on PGE2 after bathing thousands of zebrafish embryos in more than 2,500 different chemicals. The embryos took up the compounds through their skin, and because they’re transparent, their blood stem cell numbers could be easily assessed under the microscope. A derivative of PGE2 was a potent "hit," increasing blood stem cell formation and helping zebrafish and mice recover their blood cell populations after injury.
In the Phase I clinical trial, which began in early May at the Dana-Farber Cancer Institute and Massachusetts General Hospital, 24 adults undergoing treatment for leukemia or lymphoma will each receive blood stem cells from two donor umbilical cords, one or both of which will be pre-treated with PGE2. If the drug works as hoped, a single cord may provide enough stem cells to successfully engraft in patients’ bone marrow and produce the range of healthy blood cells needed. This could make cord blood a more viable option for patients who cannot receive a bone marrow transplant due to lack of a matched donor.
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