{"id":3439,"date":"2024-10-28T09:50:42","date_gmt":"2024-10-28T08:50:42","guid":{"rendered":"https:\/\/www.centrodinoferrari.com\/senza-categoria\/identificata-una-nuova-potenziale-terapia-per-la-sla-legata-a-mutazioni-del-gene-c9orf72\/"},"modified":"2026-03-24T14:38:12","modified_gmt":"2026-03-24T13:38:12","slug":"identificata-una-nuova-potenziale-terapia-per-la-sla-legata-a-mutazioni-del-gene-c9orf72","status":"publish","type":"post","link":"https:\/\/www.centrodinoferrari.com\/en\/dal-centro\/identificata-una-nuova-potenziale-terapia-per-la-sla-legata-a-mutazioni-del-gene-c9orf72\/","title":{"rendered":"Identified a New Potential Therapy for ALS Linked to C9orf72 Gene Mutations"},"content":{"rendered":"\n<h4 class=\"wp-block-heading\">An important international study coordinated by the National Institute on Aging (NIH) and published in <em>Cell Genomics<\/em>, which involved several Italian research centers\u2014particularly in Milan\u2014has identified a promising therapeutic approach for patients with Amyotrophic Lateral Sclerosis (ALS) caused by mutations in the <strong>C9orf72<\/strong> gene, the most common genetic form of the disease.<\/h4>\n\n\n\n<p>The study identified <strong>acamprosate<\/strong>, a drug already approved for the treatment of alcohol dependence, as a potential therapy to slow disease progression in patients with <strong>C9orf72<\/strong> gene mutations. Experiments conducted on motor neuron cells derived from patients showed that <strong>acamprosate<\/strong> indeed has a neuroprotective effect comparable to or greater than <strong>riluzole<\/strong>, the current standard of care for ALS.<\/p>\n\n\n\n<p>Researchers from the <strong>\u201cCentro Dino Ferrari\u201d of the University of Milan<\/strong> and the <strong>Fondazione IRCCS Istituto Auxologico Italiano<\/strong> \u2013 including <strong>Dr. Silvia Peverelli, Dr. Cinzia Tiloca, Prof. Nicola Ticozzi, Dr. Federico Verde, Prof. Antonia Ratti, and Prof. Vincenzo Silani<\/strong> \u2013 as well as from the <strong>Fondazione IRCCS Ca&#8217; Granda Ospedale Maggiore Policlinico<\/strong> \u2013 including <strong>Prof. Daniela Galimberti, Dr. Maria Serpente, Dr. Chiara Fenoglio, Prof. Elio Scarpini, Prof. Giacomo P. Comi, Prof. Stefania Corti, and Dr. Roberto Del Bo<\/strong> \u2013 contributed to this important study, which analyzed genomic data from over 41,000 individuals affected by ALS. The Italian group is part of the <strong>SLAGEN Consortium<\/strong>, established years ago to promote genetic research on ALS. Co-senior author of the work is <strong>Dr. Isabella Fogh<\/strong>, who spent extended periods in the UK with support from <strong>Centro Dino Ferrari<\/strong>, which has always generously supported researchers.<\/p>\n\n\n\n<p>The researchers also discovered that genetic factors influencing the risk of developing sporadic ALS can modify the age of onset in patients with <strong>C9orf72<\/strong> mutations, providing new insights into the variable clinical expression of the disease.<\/p>\n\n\n\n<p>\u201cThe originality of the approach lies in initially focusing the research on gene variants influencing the age of onset in patients with <strong>C9orf72<\/strong> mutations,\u201d comments <strong>Prof. Nicola Ticozzi<\/strong>, \u201cand then using this strategy to identify therapeutically effective molecules.\u201d<\/p>\n\n\n\n<p>\u201cThis study represents an important advance in understanding the mechanisms underlying <strong>C9orf72-related ALS<\/strong> and in identifying new therapies,\u201d comments <strong>Prof.ssa Stefania Corti<\/strong>. \u201cThe innovative approach based on genomic data analysis also allowed the identification of an already available drug that could be rapidly tested in clinical trials.\u201d<\/p>\n\n\n\n<p>\u201cThe completeness of the study lies in confirming efficacy using in vitro models,\u201d explains <strong>Prof.ssa Antonia Ratti<\/strong>, \u201cobtained from induced pluripotent stem cells (iPSCs) differentiated into human motor neurons, providing a biologically valuable model to test clinically relevant molecules.\u201d<\/p>\n\n\n\n<p>\u201cThese results open new prospects for developing personalized therapies for ALS patients,\u201d adds **Prof. Giacomo P. Comi. \u201cThe possibility of using an already approved drug could significantly accelerate the development of new treatments.\u201d<\/p>\n\n\n\n<p>\u201cThe availability of a close-knit consortium like <strong>SLAGEN<\/strong> and the decade-long collaboration between the two <strong>Centro Dino Ferrari<\/strong> sites,\u201d concludes <strong>Prof. Vincenzo Silani<\/strong>, \u201crepresents a guarantee for scientific progress in a poorly treatable disease like ALS and a reference point for the growth of future generations of researchers.\u201d<\/p>\n\n\n\n<p><strong>For more information:<\/strong><br>Pierangelo Garzia<br>Head of Press Office<br>Auxologico IRCCS<br><a>garzia@auxologico.it<\/a><br>Tel. 0261911.2896<\/p>\n\n\n\n<p><strong>Researcher contacts:<\/strong><br><strong>Prof. Nicola Ticozzi:<\/strong> <a>nicola.ticozzi@unimi.it<\/a><br><strong>Prof.ssa Stefania Corti:<\/strong> <a>stefania.corti@unimi.it<\/a><br><strong>Prof.ssa Antonia Ratti:<\/strong> <a>antonia.ratti@unimi.it<\/a><br><strong>Prof.ssa Daniela Galimberti:<\/strong> <a>daniela.galimberti@unimi.it<\/a><br><strong>Prof. Giacomo P. Comi:<\/strong> <a>giacomo.comi@unimi.it<\/a><br><strong>Prof. Vincenzo Silani:<\/strong> <a>vincenzo.silani@unimi.it<\/a><\/p>\n","protected":false},"excerpt":{"rendered":"<p>An important international study coordinated by the National Institute on Aging (NIH) and published in Cell Genomics, which involved several Italian research centers\u2014particularly in Milan\u2014has identified a promising therapeutic approach for patients with Amyotrophic Lateral Sclerosis (ALS) caused by mutations in the C9orf72 gene, the most common genetic form of the disease. The study identified [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":1560,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"give_campaign_id":0,"footnotes":""},"categories":[262],"tags":[272,336,315,337,300,338,339],"class_list":["post-3439","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-dal-centro","tag-sla","tag-cura-sclerosi-laterale-amiotrofica","tag-cura-sla","tag-ricerca-scientifica-sclerosi-laterale-amiotrofica","tag-ricerca-sla","tag-terapia-per-sclerosi-laterale-amiotrofica","tag-terapia-sla"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.1.1 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Identified a New Potential Therapy for ALS Linked to C9orf72 Gene Mutations - Centro Dino Ferrari<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/www.centrodinoferrari.com\/en\/dal-centro\/identificata-una-nuova-potenziale-terapia-per-la-sla-legata-a-mutazioni-del-gene-c9orf72\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Identified a New Potential Therapy for ALS Linked to C9orf72 Gene Mutations - Centro Dino Ferrari\" \/>\n<meta property=\"og:description\" content=\"An important international study coordinated by the National Institute on Aging (NIH) and published in Cell Genomics, which involved several Italian research centers\u2014particularly in Milan\u2014has identified a promising therapeutic approach for patients with Amyotrophic Lateral Sclerosis (ALS) caused by mutations in the C9orf72 gene, the most common genetic form of the disease. 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