{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,11]],"date-time":"2026-09-11T03:50:38Z","timestamp":1789098638159,"version":"build-2803163510"},"reference-count":38,"publisher":"Oxford University Press (OUP)","issue":"5","license":[{"start":{"date-parts":[[2024,9,5]],"date-time":"2024-09-05T00:00:00Z","timestamp":1725494400000},"content-version":"vor","delay-in-days":248,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100003197","name":"AIFA","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100003197","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100003197","name":"Agenzia Italiana del Farmaco","doi-asserted-by":"publisher","award":["2016-02364678"],"award-info":[{"award-number":["2016-02364678"]}],"id":[{"id":"10.13039\/501100003197","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2024,9,2]]},"abstract":"<jats:title>Abstract<\/jats:title>\n               <jats:p>In preclinical studies, the anti-inflammatory drug colchicine, which has never been tested in amyotrophic lateral sclerosis, enhanced the expression of autophagy factors and inhibited accumulation of transactive response DNA-binding protein 43\u2005kDa, a known histopathological marker of amyotrophic lateral sclerosis. This multicentre, randomized, double-blind trial enrolled patients with probable or definite amyotrophic lateral sclerosis who experienced symptom onset within the past 18 months. Patients were randomly assigned in a 1:1:1 ratio to receive colchicine at a dose of 0.005\u2005mg\/kg\/day, 0.01\u2005mg\/kg\/day or placebo for a treatment period of 30 weeks. The number of positive responders, defined as patients with a decrease lesser than 4 points in the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised total score during the 30-week treatment period, was the primary outcome. Disease progression, survival, safety and quality of life at the end of treatment were the secondary clinical outcomes. Secondary biological outcomes included changes from baseline to treatment end of stress granule and autophagy responses, transactive response DNA-binding protein 43\u2005kDa, neurofilament accumulation and extracellular vesicle secretion, between the colchicine and placebo groups. Fifty-four patients were randomized to receive colchicine (n = 18 for each colchicine arm) or placebo (n = 18). The number of positive responders did not differ between the placebo and colchicine groups: 2 out of 18 patients (11.1%) in the placebo group, 5 out of 18 patients (27.8%) in the colchicine 0.005\u2005mg\/kg\/day group (odds ratio = 3.1, 97.5% confidence interval 0.4\u201337.2, P = 0.22) and 1 out of 18 patients (5.6%) in the colchicine 0.01\u2005mg\/kg\/day group (odds ratio = 0.5, 97.5% confidence interval 0.01\u201310.2, P = 0.55). During treatment, a slower Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised decline was detected in patients receiving colchicine 0.005\u2005mg\/kg\/day (mean difference = 0.53, 97.5% confidence interval 0.07\u20130.99, P = 0.011). Eight patients experienced adverse events in placebo arm (44.4%), three in colchicine 0.005\u2005mg\/kg\/day (16.7%) and seven in colchicine 0.01\u2005mg\/kg\/day arm (35.9%). The differences in adverse events were not statistically significant. In conclusion, colchicine treatment was safe for amyotrophic lateral sclerosis patients. Further studies are required to better understand mechanisms of action and clinical effects of colchicine in this condition.<\/jats:p>","DOI":"10.1093\/braincomms\/fcae304","type":"journal-article","created":{"date-parts":[[2024,9,5]],"date-time":"2024-09-05T06:10:57Z","timestamp":1725516657000},"source":"Crossref","is-referenced-by-count":8,"title":["Colchicine treatment in amyotrophic lateral sclerosis: safety, biological and clinical effects in a randomized clinical trial"],"prefix":"10.1093","volume":"6","author":[{"given":"Giulia","family":"Gianferrari","sequence":"first","affiliation":[{"name":"Department of Biomedical, Metabolic and Neural Sciences, University of Modena and Reggio Emilia , Modena 41121 ,","place":["Italy"]},{"name":"Department of Neurosciences, Azienda Ospedaliero Universitaria di Modena , Modena 41126 ,","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Riccardo","family":"Cuoghi Costantini","sequence":"additional","affiliation":[{"name":"Unit of Statistical and Methodological Support to Clinical Research, Azienda Ospedaliero-Universitaria , Modena 41121 ,","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3058-5711","authenticated-orcid":false,"given":"Valeria","family":"Crippa","sequence":"additional","affiliation":[{"name":"Dipartimento di Scienze Farmacologiche e Biomolecolari \u2018Rodolfo Paoletti\u2019, Universit\u00e0 degli Studi di Milano , Milan 20122 ,","place":["Italy"]},{"name":"Dipartimento di Eccellenza 2018-2027, Universit\u00e0 degli Studi di Milano , Milan 20122 ,","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Serena","family":"Carra","sequence":"additional","affiliation":[{"name":"Department of Biomedical, Metabolic and Neural Sciences, University of Modena and Reggio Emilia , Modena 41121 ,","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0456-2054","authenticated-orcid":false,"given":"Valentina","family":"Bonetto","sequence":"additional","affiliation":[{"name":"Research Center for ALS, Istituto di Ricerche Farmacologiche Mario Negri IRCCS , Milan 20156 ,","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Orietta","family":"Pansarasa","sequence":"additional","affiliation":[{"name":"Cellular Model and Neuroepigenetics Unit, IRCCS Mondino Foundation , Pavia 27100 ,","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cristina","family":"Cereda","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Center of Functional Genomics and Rare diseases, \u2018V. 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