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Dr. Laughlin completes phase 1 clinical trial using umbilical cord blood to treat leukaemia

Case Western Reserve University : 18 January, 2007  (Technical Article)
A relatively new source of stem cells has been used to treat leukemia. Mary Laughlin, who is a researcher at the National Center for Regenerative Medicine and an associate professor of hematology and oncology, and her colleagues at the Cleveland-based center successfully concluded a phase I clinical trial using umbilical cord blood stem cells to treat leukemia.
Patients with leukemia were transplanted with umbilical cord blood stem cells from mismatched, unrelated donors after receiving full body irradiation. The study proved that umbilical cord blood stem cells, even from unrelated donors, is a feasible alternative source of stem cells for transplantation in adults because these stem cells can repair the blood-producing bone marrow with a low risk for rejection.

Bone marrow transplantation is an effective medical therapy for life-threatening blood-related disorders. However, BMT is limited by the lack of available matched donors. A mis-match between the donor and the recipient can result in a rejection response which can cause from skin rashes, diarrhea, intestinal bleeding, liver damage and even death.

Umbilical cord blood stem cells have been used as an alternative stem cell source to bone marrow stem cells and offer the advantages of easy procurement, no risk to donors, reduced risk of transmitting infections, immediate availability of frozen units, and acceptable partial HLA mismatches.

The importance of finding new sources of stem cells to treat various blood-related diseases is evident in the more than 40,000 BMT performed each year worldwide. Of these, 9,000 people in North America undergo stem cell transplantation for leukemia, lymphoma or myeloma. The disease prevalence continues to increase with an estimated 35,000 new cases of leukemia diagnosed and 22,600 deaths in the United States in 2005.

Dr. Laughlin's trial enrolled patients suffering from severe leukemia. Each was transplanted with mismatched, unrelated umbilical cord blood stem cells after receiving full-body irradiation to kill the bone marrow. Only one third of these patients were diagnosed with graft vs host disease after transplantation compared to 70-90% of patients who have received bone marrow stem cells from unrelated donors. Dr. Laughlin reports that 56% of her patients are alive and event free at median follow up over 3 years. These findings herald the use of UCB from unrelated, unmatched donors as a feasible alternative source of stem cells for treatment of adults.
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