![]() It's an uphill battle for genetic therapy, but if they can prove the technology works, I think you will see the pendulum swing that way." That can be lifesaving itself and is well-established. The competing technology now is allogeneic bone marrow transplantation where you go to a sibling donor or an unrelated donor. He added that "at the moment, I would go to transplantation and wait for these data to mature. They found that the CD3+ T cell count at 6 months for their patients was a median 548/mcl versus a median 1,755/mcl for those in the earlier study ( P=0.14 in two-sided Wilcoxon rank sum test). Pai's group also did an early immune recovery and peripheral blood insertion site analysis between six patients from their trial and the 20 subjects enrolled in the earlier study. They are free of SCID-related infections, and humoral immune evaluation is ongoing," the authors wrote. "At last follow-up, the remaining seven patients have evidence of transgene expression in T cells, naive T-cell generation, and normal diversity. The two patients who did not achieve T cell count >300/mcl received CD34+ cells with the lowest VCN, the authors explained. Seventy-one percent of the patients achieved CD3+ T cell count >300/mcl. Another patient experienced treatment failure and underwent hematopoietic stem cell transplantation. One patient with a low initial transduction underwent a repeat procedure 1 month after infusion of the first product. One patient died while the others recovered with treatment these patients are alive and now have normal T-cell numbers and diversity, Pai's group reported. In the French trial that Pai mentioned, 18 of 20 boys treated with a MLV-based gammaretroviral vector achieved reconstituted immune systems, but five developed T-cell acute lymphoblastic leukemia. The work by Pai and colleagues is similar to previous attempts with genetic therapy in this fatal disease. For these patients, gene therapy is an attractive option." "Outcomes for boys who do not have well-matched donors are suboptimal. "Without curative therapy such as bone marrow transplantation, these boys die of opportunistic or community-acquired viral infection," Pai said. The current standard treatment for X-SCID is hematopoietic stem cell transplantation, but finding a suitable donor is problematic. "But the emergence of leukemia occurred in the 3- to 5-year time frame in the previous trial. ![]() "We are 3 years' post-therapy for some of these children, and have not seen any sign of leukemia," Pai said at the American Society of Hematology annual meeting.
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