In this process, the team selectively removed the alpha/beta-positive T cells and CD19-positive B cells from the donor graft, as those are more likely to trigger donor cells to attack recipient cells, resulting in a dangerous complication known as graft-versus-host disease (GVHD). At the same time, the process preserved healthy, mature, immune-active cells known as natural killer and gamma/delta-positive T cells that help prevent disease relapse and protect against infection. A total of 45 patients with acute leukemia were treated with genetically engineered stem cells from one of their parents. Transplants engrafted in 44 of the 45 patients, with a 29 percent cumulative incidence of mild GVHD. One month after transplant, follow-up analyses showed that transplanted cells had persisted in the patients and demonstrated potential anti-leukemic activity, which continued to increase over time."Our results, which demonstrate that transplantation of selectively modified, half-matched donor stem cells boasts success rates equivalent to those of a fully matched transplant, preventing GVHD and reducing transplant-related death, help continue to establish this approach as a viable option for patients without a matched donor," said study author Alice Bertaina, MD, of the Bambino Gesu Children's Hospital in Rome, Italy. "This has the potential to make this lifesaving treatment more accessible to a much larger population of patients who may not have a perfect donor match."
High-Tech Advances In Gene Therapy Overcome Challenges, Offer Hope For Patients With Hard-to-Treat Blood Disorders
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