{"id":130339,"date":"2024-12-11T16:11:06","date_gmt":"2024-12-11T21:11:06","guid":{"rendered":"https:\/\/wfh.org\/article\/le-new-england-journal-of-medicine-evoque-une-nouvelle-therapie-genique-revolutionnaire\/"},"modified":"2024-12-17T09:16:47","modified_gmt":"2024-12-17T14:16:47","slug":"le-new-england-journal-of-medicine-evoque-une-nouvelle-therapie-genique-revolutionnaire","status":"publish","type":"post","link":"https:\/\/wfh.org\/fr\/article\/le-new-england-journal-of-medicine-evoque-une-nouvelle-therapie-genique-revolutionnaire\/","title":{"rendered":"Le New England Journal of Medicine \u00e9voque une nouvelle th\u00e9rapie g\u00e9nique r\u00e9volutionnaire"},"content":{"rendered":"\t\t
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L\u2019\u00e9tude a \u00e9t\u00e9 effectu\u00e9e sur cinq hommes de 22 \u00e0 41 ans, atteints d\u2019h\u00e9mophilie A s\u00e9v\u00e8re sans inhibiteurs du facteur VIII. Ils ont \u00e9t\u00e9 suivis pendant une m\u00e9diane de 14 mois (pour des p\u00e9riodes allant de 9 \u00e0 27 mois). Les r\u00e9sultats montrent que la th\u00e9rapie a r\u00e9ussi \u00e0 maintenir le facteur VIII \u00e0 des niveaux permettant de pr\u00e9venir les saignements spontan\u00e9s (niveau d\u2019activit\u00e9 m\u00e9dian du facteur\u00a0: 20-40%), sans \u00e9pisodes de saignements pendant ou apr\u00e8s le traitement. Avant le traitement, les participants \u00e0 l\u2019\u00e9tude pr\u00e9sentaient un taux de saignement annuel de 20 \u00e0 120 \u00e9pisodes.<\/p>\n

Certains effets secondaires \u00e9taient attendus et ont \u00e9t\u00e9 rapport\u00e9s, tels qu\u2019une neutrop\u00e9nie s\u00e9v\u00e8re (faible taux de globules blancs), ainsi qu\u2019une thrombocytop\u00e9nie s\u00e9v\u00e8re (faible taux de plaquettes), mais ont \u00e9t\u00e9 r\u00e9solus au bout de 11 jours. Hormis cela, aucun effet secondaire grave n\u2019a \u00e9t\u00e9 observ\u00e9. Fait notable, aucun participant n\u2019a d\u00e9velopp\u00e9 d\u2019inhibiteurs du facteur VIII apr\u00e8s la th\u00e9rapie, et aucun d\u00e9c\u00e8s n\u2019a \u00e9t\u00e9 rapport\u00e9 pendant la p\u00e9riode de l\u2019\u00e9tude.<\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t

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Cette \u00e9tude men\u00e9e sur cinq personnes atteintes d\u2019h\u00e9mophilie A \u00e9tablit qu\u2019un nouveau syst\u00e8me de vecteur viral peut apporter le g\u00e8ne du facteur VIII jusqu\u2019aux cellules souches de la moelle \u00e9pini\u00e8re et produit une prot\u00e9ine FVIII stable qui peut circuler. Puisque le facteur VIII s\u2019int\u00e8gre dans les chromosomes, on s\u2019attend \u00e0 ce que cela dure de nombreuses ann\u00e9es, voire tout au long de la vie. S\u2019il faudra encore beaucoup de travail avant que cette technologie devienne une modalit\u00e9 de traitement viable, cette \u00e9tude pr\u00e9sente des donn\u00e9es qui sugg\u00e8rent fortement que cela pourrait constituer une approche s\u00fbre et efficace pour mettre au point un potentiel rem\u00e8de.<\/h2>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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\u2014 Dr Glenn Pierce (MD, PhD), vice-pr\u00e9sident m\u00e9dical de la FMH<\/p>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t

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Ces premiers r\u00e9sultats r\u00e9pondent \u00e0 des difficult\u00e9s observ\u00e9es dans d\u2019autres types de th\u00e9rapies g\u00e9niques et semblent prometteurs pour un traitement \u00e0 long terme (et possiblement \u00e0 vie) de l\u2019h\u00e9mophilie A. Cette approche pourrait aussi rendre la th\u00e9rapie g\u00e9nique accessible \u00e0 un plus grand nombre de patient\u00b7es, y compris des enfants.<\/p>\n

Pour lire l\u2019article du professeur Srivastava (en anglais), cliquez ici<\/a>. Un \u00e9ditorial du professeur Johnny Mahlangu de l\u2019universit\u00e9 de Witswatersrand compare la th\u00e9rapie g\u00e9nique avec lentivirus et avec virus ad\u00e9no-associ\u00e9 (VAA), qui \u00e9tait jusqu\u2019\u00e0 aujourd\u2019hui utilis\u00e9 dans le d\u00e9veloppement des th\u00e9rapies g\u00e9niques pour l\u2019h\u00e9mophilie \u2013 il est intitul\u00e9 \u00ab\u00a0Could Lentivirus Overcome the AAV Gene-Therapy Challenges in Hemophilia A?\u00a0\u00bb, et \u00e9galement publi\u00e9 dans le New England Journal of Medicine.<\/em> Cliquez ici<\/a> pour lire cet \u00e9ditorial (en anglais).<\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<\/div>\n\t\t","protected":false},"excerpt":{"rendered":"

Une nouvelle \u00e9tude publi\u00e9e dans le New England Journal of Medicine, \u00ab Lentiviral Gene Therapy with CD34+ Hematopoietic Cells for Hemophilia A \u00bb, rapporte l\u2019essai d\u2019une nouvelle th\u00e9rapie g\u00e9nique r\u00e9volutionnaire utilisant pour porter le g\u00e8ne du Facteur VIII un virus diff\u00e9rent de celui utilis\u00e9 dans les th\u00e9rapies pr\u00e9c\u00e9dentes. Cette \u00e9tude pr\u00e9sente des donn\u00e9es prometteuses sur la s\u00e9curit\u00e9 et l\u2019efficacit\u00e9 de cette nouvelle approche de th\u00e9rapie g\u00e9nique.<\/p>\n","protected":false},"author":13,"featured_media":130304,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"inline_featured_image":false,"footnotes":""},"categories":[177],"tags":[],"search-category":[85],"class_list":["post-130339","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-actualite-medicale","search-category-article"],"yoast_head":"\nLe New England Journal of Medicine \u00e9voque une nouvelle th\u00e9rapie g\u00e9nique r\u00e9volutionnaire | FMH - F\u00e9d\u00e9ration mondiale de l\u2019h\u00e9mophilie<\/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:\/\/wfh.org\/fr\/article\/le-new-england-journal-of-medicine-evoque-une-nouvelle-therapie-genique-revolutionnaire\/\" \/>\n<meta property=\"og:locale\" content=\"fr_FR\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Le New England Journal of Medicine \u00e9voque une nouvelle th\u00e9rapie g\u00e9nique r\u00e9volutionnaire\" \/>\n<meta property=\"og:description\" content=\"Une nouvelle \u00e9tude publi\u00e9e dans le New England Journal of Medicine, \u00ab Lentiviral Gene Therapy with CD34+ Hematopoietic Cells for Hemophilia A \u00bb, rapporte l\u2019essai d\u2019une nouvelle th\u00e9rapie g\u00e9nique r\u00e9volutionnaire utilisant pour porter le g\u00e8ne du Facteur VIII un virus diff\u00e9rent de celui utilis\u00e9 dans les th\u00e9rapies pr\u00e9c\u00e9dentes. 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