IDDF2024-ABS-0384 Efficacy and safety of fecal microbiota transplantation in amyotrophic lateral sclerosis

<h3>Background</h3> Amyotrophic lateral sclerosis (ALS) is a fatal, progressive neurodegenerative disease with unclear pathogenesis. Studies have found that gut microbiota are essential among them. The feasibility of fecal microbiota transplantation (FMT) has been reported in two ALS patients with respiratory failure. More clinical validation is needed. <h3>Methods</h3> We performed a single-center, single-arm clinical trial to explore the safety and efficacy of FMT for ALS. After strict enrollment, patients receive three FMT treatments by colonoscopy at a 6-week interval. Assessments were performed one day before each FMT. The primary measure was the changes of the Revised ALS Functioning Rating Scale (ALSFRS-R). The response was defined as the scale decreased by ≤ 0 points. Secondary measures were assessed by the ALS Assessment Questionnaire (ALSAQ-40), Short Form Health Survey (SF-36), Self-rating Depression Scale (SDS), and Self-rating Anxiety Scale (SAS). Safety was assessed by the number and severity of adverse events. The trial had been approved and with Clinical Trial Registry (ChiCTR2100046015). IDDF2024-ABS-0384 Figure 1. Flowchart of study design <h3>Results</h3> We recruited 59 patients with ALS and 34 of them completed the final assessment (IDDF2024-ABS-0384 Figure 1. Flowchart of study design). There were 22 males and 12 females, with an average onset age of 52.88 ± 10.20 years, disease duration of 23.12 ± 15.84 months, and ALSFRS-R of 36.97 ± 7.36 at baseline. For primary measure, responses occurred in 55.88% (19/34) of the patients after the first FMT, and 61.76% (21/34) after the second FMT. The slope of the ALSFRS-R score was -0.60 ± 0.50 points/month at baseline, -0.53 ± 0.35 after the first FMT, and -0.50 ± 0.34 after the second FMT. Detailedly, FMT ameliorated the symptoms of bulbar and respiratory function rather than motor function when measured by ALSFRS-R subscales. Concerning secondary measures, the scores of ALSAQ40, SF36, SDS, and SAS showed an improvement tendency after FMT. No adverse events were reported during the trial. <h3>Conclusions</h3> FMT showed efficacy and safety for ALS with slower functional decline. FMT might improve bulbar and respiratory function and ameliorate life quality. Large randomized controlled trials are needed to further elucidate the efficacy and safety of FMT in ALS.