Overall and event-free survival of 100 and 95 percent, respectively, seen with autologous CD34+ hematopoietic stem-cell lentiviral gene therapy<\/p>\n<\/h3>\n
By Elana Gotkine HealthDay Reporter<\/b>
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FRIDAY, Oct. 17, 2025 (HealthDay News) — Autologous CD34+ hematopoietic stem-cell lentiviral gene therapy shows sustained clinical efficacy for severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency (ADA-SCID), according to a study published online Oct. 15 in the New England Journal of Medicine<\/em>.<\/p>\n Claire Booth, M.B.B.S., Ph.D., from the University College London Great Ormond Street Institute of Child Health, and colleagues reported the long-term results from a cohort of patients with ADA-SCID who were treated with busulfan nonmyeloablative conditioning followed by transplantation with autologous CD34+ hematopoietic stem cells transduced ex vivo with a lentiviral vector encoding human ADA<\/em>. Overall survival and event-free survival were examined as the primary efficacy end points.<\/p>\n The cohort included 62 patients with ADA-SCID in the United States and United Kingdom (33 and 29 patients, respectively); patients were followed for a median of 7.5 years (474 patient-years of follow-up). The researchers found that overall and event-free survival were 100 and 95 percent, respectively. Fifty-nine patients with successful gene-marked engraftment at six months continued not to receive enzyme-replacement therapy and through last follow-up had stable gene marking, ADA enzyme activity, metabolic detoxification, and immune reconstitution; 58 discontinued immunoglobulin G replacement therapy and had a robust response to vaccinations. There were no leukoproliferative events or clonal expansion.<\/p>\n \u201cThese results are what we hoped for when we first began developing this approach,\u201d said coauthor Donald B. Kohn, M.D., from the University of California, Los Angeles, said in a statement. \u201cThe durability of immune function, the consistency over time, and the continued safety profile are all incredibly encouraging. … What’s most remarkable is that everything has been completely stable beyond the initial three- to six-month recovery period.”<\/p>\n Several authors disclosed ties to industry.<\/p>\n Abstract\/Full Text (subscription or payment may be required)<\/a><\/p>\n