Study summary · research use only
Retrospective observational study on the use of acetyl-L-carnitine in ALS
Plain-language summary
Paraphrased from the published abstract below — not a verdict on whether anything works.
This retrospective, observational, multicentre case-control human study from Italy compared subjects with ALS treated with acetyl-L-carnitine (ALCAR) at 1.5 g/day or 3 g/day to matched untreated subjects (45 per group). With ALCAR 3 g/day, 51.1% of treated subjects were alive at 24 months compared to 48.9% of untreated subjects, with no statistically significant difference in ALSFRS, FVC, or self-sufficiency. With ALCAR 1.5 g/day, 71.1% of treated subjects were alive at 24 months compared to 48.9% of untreated subjects; the ALSFRS-R slope was less negative in treated subjects, though this difference did not reach statistical significance. The authors reference an earlier phase II trial reporting positive effects on self-sufficiency, ALSFRS-R score, and FVC, and state that additional evidence is needed to confirm the drug's effects and dosage.
Abstract
ALCAR (Acetyl-L-carnitine) is a donor of acetyl groups and increases the intracellular levels of carnitine, the primary transporter of fatty acids across the mitochondrial membranes. In vivo studies showed that ALCAR decrease oxidative stress markers and pro-inflammatory cytokines. In a previous double-blind placebo-controlled phase II trial showed positive effects on self-sufficiency (defined as a score of 3+ on the ALSFRS-R items for swallowing, cutting food and handling utensils, and walking) ALSFRS-R total score and FVC. We conducted an observational, retrospective, multicentre, case-control study to provide additional data on the effects of ALCAR in subjects with ALS in Italy. Subjects treated with ALCAR 1.5 g/day or 3 g/day were included and matched with not treated subjects by sex, age at diagnosis, site of onset, and time from diagnosis to baseline, (45 subjects per group). ALCAR 3 g/day vs not treated: 22 not treated subjects (48.9%) were still alive at 24 months after baseline, compared to 23 (51.1%) treated subjects (adj. OR 1.18, 95% CI 0.46-3.02). No statistically significant differences were detected in ALSFRS nor FVC nor self-sufficiency. ALCAR 1.5 g/day vs not treated: 22 not treated subjects (48.9%) were still alive at 24 months after baseline, compared to 32 (71.1%) treated subjects (adj. OR 0.27, 95% CI 0.10-0.71). For ALSFRS-R, a mean slope of - 1.0 was observed in treated subjects compared to - 1.4 in those not treated (p = 0.0575). No statistically significant difference was detected in the FVC nor self-sufficiency. Additional evidence should be provided to confirm the efficacy of the drug and provide a rationale for the dosage.
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