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The first drug discovered using zebrafish has now made it to clinical trial in cancer patients. It’s now hoped that the same drug will help patients rebuild their blood systems more quickly.
The original studies, led by Stem Cell Program Director Leonard Zon, MD, of Children’s Division of Hematology/Oncology, bathed thousands of zebrafish embryos in more than 2,500 different chemicals, hoping to find one that would enhance multiplication of blood stem cells. As reported in Nature in 2007, a derivative of prostaglandin E2 (PGE2) was a potent "hit," increasing hematopoietic stem cell formation (assessed by viewing the transparent embryos under a microscope) and helping zebrafish and mice recover their blood cell populations after injury.
In the Phase I clinical trial, launched in May at the Dana-Farber Cancer Institute and Massachusetts General Hospital, 24 adults undergoing treatment for leukemia or lymphoma each receive hematopoietic 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 engraft in patients’ marrow and produce the range of blood cells needed. This could make cord blood a more viable option for patients who lack a matched marrow donor, Dr. Zon says.
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