LAUSR

OBJECTIVE To examine the safety and efficacy of iltamiocel, an investigational cellular therapy of autologous muscle-derived cells, as a treatment for fecal incontinence (FI) in adults. BACKGROUND Limited therapeutic options are available for patients with FI refractory to conservative treatments. Cell therapy using autologous muscle-derived cells represents a promising, minimally invasive approach for restoring anal sphincter function. METHODS In this multicenter, prospective, non-randomized study, 48 participants were treated with a single iltamiocel dose of 250×106 cells. The primary outcome was the incidence of product or procedure-related adverse events (AEs) and serious adverse events (SAEs). Secondary outcomes were changes in number of FI episodes, Cleveland Clinic Incontinence Score (CCIS), Fecal Incontinence Quality of Life (FIQL), and anorectal manometry at 3, 6, and 12 months compared to baseline. RESULTS No SAEs and only one product-related AE of inflammation at the injection site were reported. At 12 months, there was a reduction in median FI episodes (-6.0; 95% CI: -10.0, -1.0) and days with episodes (-4.0; 95% CI: -8.0, -1.0). A ≥50% reduction in FI episodes was observed in 53.7% of participants, and 24.4% had complete restoration of continence. Symptom severity and quality of life improved with mean CCIS reduction (-2.9; 95% CI: - 3.7, -2.1), and FIQL increased (2.2; 95% CI:1.4, 2.9). No significant changes were detected in anorectal manometry measurements. A history of episiotomy was significantly associated with treatment response in multivariate analysis. CONCLUSION The administration of iltamiocel cellular therapy is safe. Iltamiocel shows promise for significantly improving fecal incontinence symptoms and quality of life.