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Lentiviral Gene Therapy Beneficial for Early Cerebral Adrenoleukodystrophy

Medically reviewed by Carmen Pope, BPharm. Last updated on Oct 11, 2024.

By Elana Gotkine HealthDay Reporter

FRIDAY, Oct. 11, 2024 -- For boys with early-stage cerebral adrenoleukodystrophy and evidence of active inflammation, lentiviral elivaldogene autotemcel (eli-cel) gene therapy offers lasting benefits, according to a study published in the Oct. 10 issue of the New England Journal of Medicine.

Florian Eichler, M.D., from Massachusetts General Hospital and Harvard Medical School in Boston, and colleagues conducted a phase 2 to 3 study to examine the efficacy and safety of eli-cel therapy in boys with early-stage cerebral adrenoleukodystrophy and evidence of active inflammation. Survival without any of six major functional disabilities at month 24 was examined as the primary efficacy end point. Overall, 32 patients received eli-cel; 29 completed the 24-month study and are being monitored for long-term follow-up.

The researchers found that none of the 29 patients had major functional disabilities at month 24, with 94 percent overall survival. At the most recent assessment (median follow-up of six years), 30 of 32 patients had a stable neurologic score compared with the baseline score (94 percent); 81 percent of the patients had no major functional disabilities. Adverse events that were directly related to eli-cel occurred in four patients. One patient developed myelodysplastic syndrome (MDS) with excess blasts at 92 months; the patient underwent allogeneic hematopoietic stem cell transplantation and, at most recent follow-up, did not have MDS.

"Most patients with cerebral adrenoleukodystrophy in this study appear to have benefited from eli-cel, with benefit shown over a median duration of follow-up of 60.2 months, and up to 8.9 years in two patients, and with a low incidence of serious adverse events," the authors write.

The study was funded by Bluebird Bio, the manufacturer of eli-cel.

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Posted October 2024

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