Fate Therapeutics plans to announce this morning that it has begun its first clinical trial of a drug built on its knowledge of stem cell biology. Rather than injecting certain kinds of adult stem cells to regenerate tissues, this treatment involves a conventional small-molecule drug that’s designed to spur growth of blood-forming stem cells that patients need to recover from certain kinds of leukemia and lymphomas.
Xconomy reported in March that La Jolla, CA-based Fate—which was founded by top stem cell scientists at Harvard University, The Scripps Research Institute, the University of Washington, Stanford University, and the Whitehead Institute for Biomedical Research—was destined to test its first drug in humans. But the company is now disclosing that the study will be done at the Dana-Farber Cancer Institute in Boston, and provided more details on how its treatment could better the way hematopoietic stem cells from umbilical cord blood are transplanted into some cancer patients to restore their bone marrow function.
Fate’s drug, dubbed FT-1050, is a small molecule that the company plans to use to treat blood-forming stem cells from cord blood before the cells are transplanted into humans. By activating certain genes in the stem cells, the drug is intended to boost the ability of the cells to multiply and take up residence in bone marrow, Pratik Multani, vice president of clinical development at Fate, explains. The clinical trial is testing this use of the drug in patients with blood or immune system cancers whose bone marrow function—which is vital to blood production and the immune system—has been destroyed by chemotherapy and other cancer treatments.
Fate’s treatment was initially discovered in the lab of Leonard Zon, the director of stem cell research at Children’s Hospital Boston. Zon is one of several stem cell experts who was recruited by Polaris Venture Partners and Arch Venture Partners to form Fate, which is focused on the development of drugs intended to activate adult stem cells to treat diseases.
Dana-Farber, one of the world’s leading centers for adult stem cell transplants, is expected to be the clinical trial site for all 12 patients who will enroll in the Phase Ib study. The trial enrolled its first patient last week and is expected to end next year. Each patient will receive two different supplies of stem cells from different donors, and one of the two supplies will be treated with Fate’s drug for an hour before it is infused into patients. The primary purpose of the study is to test the safety of the drug. Yet by designing the trial to have a control group, Fate also hopes to get a preliminary sense of whether the drug is working, specifically, whether the stem cells treated with the drug are able to better multiply and find their way into bone marrow more quickly than the untreated stem cells. Fate hopes to show in future trials that the the drug can speed up the time needed for the stem cell transplants to restore immune function and blood production.
Cord blood—drawn from umbilical cords and placentas after babies are born—is a good source of stem cells for transplants because it requires less of a match from donor to patient than sources such as donated bone marrow or blood that circulates through the rest of the body, according to Fate. Because of this, patients often don’t need to wait as long for stem cell transplants from cord blood supplies as they do for transplants from the other two sources. Fate isn’t alone in developing ways to improve cord blood transplants; other companies—including Durham, NC-based Aldagen, Gamida Cell, of Jerusalem, Israel—are developing methods to increase stem cell numbers in cord blood supplies.
Despite its name, Fate wasn’t leaving anything to chance when selecting Dana-Farber to conduct the trial. “Transplantation itself is a very intense procedure, so you want to work with the best to make sure that’s not a variable in your study,” Multani said.
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