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Observational Study
. 2023 Nov;270(11):5344-5357.
doi: 10.1007/s00415-023-11844-6. Epub 2023 Jun 28.

Retrospective observational study on the use of acetyl-L-carnitine in ALS

Serena Sassi #  1 , Elisa Bianchi #  1 , Luca Diamanti  2 , Danilo Tornabene  2   3 , Elisabetta Sette  4 , Doriana Medici  5 , Sabrina Matà  6 , Deborah Leccese  6 , Martina Sperti  6 , Ilaria Martinelli  7   8 , Andrea Ghezzi  9 , Jessica Mandrioli  7   9   10 , Valentina Virginia Iuzzolino  11 , Raffaele Dubbioso  11 , Francesca Trojsi  12 , Carla Passaniti  12 , Giulia D'Alvano  12 , Massimiliano Filosto  13 , Alessandro Padovani  14 , Letizia Mazzini  15 , Fabiola De Marchi  15 , Lucia Zinno  16 , Andi Nuredini  16 , Paolo Bongioanni  17 , Cristina Dolciotti  17 , Elena Canali  18 , Giulia Toschi  18 , Antonio Petrucci  19 , Alessia Perna  19 , Vittorio Riso  19 , Maurizio Inghilleri  20 , Laura Libonati  21 , Chiara Cambieri  20 , Elisabetta Pupillo  1
Affiliations
Observational Study

Retrospective observational study on the use of acetyl-L-carnitine in ALS

Serena Sassi et al. J Neurol. 2023 Nov.

Erratum in

  • Correction to: Retrospective observational study on the use of acetyl-L-carnitine in ALS.
    Sassi S, Bianchi E, Diamanti L, Tornabene D, Sette E, Medici D, Matà S, Leccese D, Sperti M, Martinelli I, Ghezzi A, Mandrioli J, Iuzzolino VV, Dubbioso R, Trojsi F, Passaniti C, D'Alvano G, Filosto M, Padovani A, Mazzini L, De Marchi F, Zinno L, Nuredini A, Bongioanni P, Dolciotti C, Canali E, Toschi G, Petrucci A, Perna A, Riso V, Inghilleri M, Libonati L, Cambieri C, Pupillo E. Sassi S, et al. J Neurol. 2023 Nov;270(11):5358-5359. doi: 10.1007/s00415-023-11960-3. J Neurol. 2023. PMID: 37682317 Free PMC article. No abstract available.

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.

Keywords: Amyotrophic lateral sclerosis; Case–control; Efficacy; Neurological disease; Observational study; Treatment.

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Conflict of interest statement

The authors declare that they have no conflict of interest.

Figures

Fig. 1
Fig. 1
Study flow chart
Fig. 2
Fig. 2
Cumulative survival probability and cumulative probability of remaining self-sufficient in treated with ALCAR and not treated
Fig. 3
Fig. 3
Progression of ALSFRS-R and FVC% in treated with ALCAR and not treated
See this image and copyright information in PMC

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