Libmeldy est un m\u00e9dicament de th\u00e9rapie g\u00e9nique avanc\u00e9e destin\u00e9 au traitement d\u2019enfants atteints de leucodystrophie m\u00e9tachromatique (MLD), une maladie g\u00e9n\u00e9tique rare li\u00e9e \u00e0 une mutation du g\u00e8ne ARSA, n\u00e9cessaire \u00e0 la production de l\u2019enzyme arylsulfatase A. Cette d\u00e9ficience entra\u00eene une accumulation de sulfatides, responsable de l\u00e9sions du syst\u00e8me nerveux et d\u2019autres organes, se traduisant notamment par des troubles de la marche, une d\u00e9t\u00e9rioration mentale progressive et, \u00e0 terme, le d\u00e9c\u00e8s. En raison de la raret\u00e9 de la pathologie, Libmeldy a obtenu le statut de \u00ab m\u00e9dicament orphelin \u00bb le 13 avril 2007.<\/p>\n
Libmeldy est indiqu\u00e9 chez des enfants pr\u00e9sentant une MLD avec mutation du g\u00e8ne ARSA, dans deux situations cliniques pr\u00e9cises : d\u2019une part, les formes infantiles tardives ou juv\u00e9niles pr\u00e9coces chez des patients encore asymptomatiques ; d\u2019autre part, les formes juv\u00e9niles pr\u00e9coces d\u00e9j\u00e0 symptomatiques, \u00e0 condition que l\u2019enfant marche encore de fa\u00e7on autonome et n\u2019ait pas d\u00e9velopp\u00e9 de d\u00e9t\u00e9rioration mentale. Le m\u00e9dicament ne peut \u00eatre administr\u00e9 qu\u2019\u00e0 l\u2019enfant dont les cellules ont servi \u00e0 sa fabrication et son utilisation est limit\u00e9e \u00e0 des centres de greffe sp\u00e9cialis\u00e9s, sur prescription m\u00e9dicale.<\/p>\n
Le traitement repose sur l\u2019utilisation de cellules souches CD34+ autologues issues de la moelle osseuse ou du sang du patient. Ces cellules, capables de produire des globules blancs, sont pr\u00e9lev\u00e9es, puis modifi\u00e9es ex vivo par insertion d\u2019une copie fonctionnelle du g\u00e8ne ARSA au moyen d\u2019un lentivirus g\u00e9n\u00e9tiquement modifi\u00e9, inoffensif pour l\u2019\u00eatre humain mais capable d\u2019int\u00e9grer le g\u00e8ne dans les cellules. Les cellules ainsi modifi\u00e9es constituent le produit Libmeldy, administr\u00e9 en une seule perfusion intraveineuse, avec une posologie adapt\u00e9e au poids de l\u2019enfant.<\/p>\n
Avant la perfusion, les patients re\u00e7oivent un traitement de conditionnement par busulfan afin d\u2019\u00e9liminer les cellules m\u00e9dullaires existantes et de permettre l\u2019implantation des cellules modifi\u00e9es. D\u2019autres m\u00e9dicaments sont administr\u00e9s en amont pour limiter les r\u00e9actions li\u00e9es au traitement. Apr\u00e8s perfusion, les cellules CD34+ migrent vers la moelle osseuse, se multiplient et g\u00e9n\u00e8rent des globules blancs capables de produire une ARSA fonctionnelle. Ces cellules se distribuent ensuite dans l\u2019organisme et contribuent \u00e0 la d\u00e9gradation des sulfatides, avec un effet attendu durable sur le contr\u00f4le des sympt\u00f4mes de la maladie.<\/p>\n
Les informations disponibles insistent sur le caract\u00e8re hautement sp\u00e9cialis\u00e9 de ce traitement (th\u00e9rapie g\u00e9nique, utilisation de cellules autologues, recours \u00e0 un vecteur lentiviral, hospitalisation en centre de greffe) et sur le fait qu\u2019il s\u2019agit d\u2019une prise en charge unique, potentiellement de longue dur\u00e9e. Aucune donn\u00e9e chiffr\u00e9e de r\u00e9sultats cliniques, d\u2019efficacit\u00e9 ou de s\u00e9curit\u00e9 n\u2019est fournie dans l\u2019extrait disponible, mais le cadre r\u00e9glementaire de m\u00e9dicament orphelin et de th\u00e9rapie g\u00e9nique, ainsi que les conditions strictes d\u2019indication et d\u2019administration, pr\u00e9sentent un int\u00e9r\u00eat pour le suivi des politiques de sant\u00e9 concernant les maladies rares, l\u2019organisation de l\u2019offre de soins sp\u00e9cialis\u00e9e et l\u2019acc\u00e8s \u00e0 l\u2019innovation th\u00e9rapeutique en p\u00e9diatrie. (contenu r\u00e9sum\u00e9 en fran\u00e7ais par l’IA)\u00a0\u200bLire la Suite<\/a><\/p>","protected":false},"excerpt":{"rendered":" Libmeldy est un m\u00e9dicament de th\u00e9rapie g\u00e9nique avanc\u00e9e destin\u00e9 au traitement d\u2019enfants atteints de leucodystrophie m\u00e9tachromatique (MLD), une maladie g\u00e9n\u00e9tique rare li\u00e9e \u00e0 une mutation du g\u00e8ne ARSA, n\u00e9cessaire \u00e0 […]<\/p>\n","protected":false},"author":1,"featured_media":17698,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"site-sidebar-layout":"default","site-content-layout":"","ast-site-content-layout":"default","site-content-style":"default","site-sidebar-style":"default","ast-global-header-display":"","ast-banner-title-visibility":"","ast-main-header-display":"","ast-hfb-above-header-display":"","ast-hfb-below-header-display":"","ast-hfb-mobile-header-display":"","site-post-title":"","ast-breadcrumbs-content":"","ast-featured-img":"","footer-sml-layout":"","ast-disable-related-posts":"","theme-transparent-header-meta":"","adv-header-id-meta":"","stick-header-meta":"","header-above-stick-meta":"","header-main-stick-meta":"","header-below-stick-meta":"","astra-migrate-meta-layouts":"default","ast-page-background-enabled":"default","ast-page-background-meta":{"desktop":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"tablet":{"background-color":"","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"mobile":{"background-color":"","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""}},"ast-content-background-meta":{"desktop":{"background-color":"var(--ast-global-color-4)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"tablet":{"background-color":"var(--ast-global-color-4)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"mobile":{"background-color":"var(--ast-global-color-4)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""}},"footnotes":""},"categories":[7],"tags":[257,227,226],"class_list":["post-21707","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-medicaments-et-produits-de-sante","tag-ema","tag-ia","tag-sources-etrangeres"],"_links":{"self":[{"href":"https:\/\/veilles-affaires-sociales.fr\/index.php?rest_route=\/wp\/v2\/posts\/21707","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/veilles-affaires-sociales.fr\/index.php?rest_route=\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/veilles-affaires-sociales.fr\/index.php?rest_route=\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/veilles-affaires-sociales.fr\/index.php?rest_route=\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/veilles-affaires-sociales.fr\/index.php?rest_route=%2Fwp%2Fv2%2Fcomments&post=21707"}],"version-history":[{"count":0,"href":"https:\/\/veilles-affaires-sociales.fr\/index.php?rest_route=\/wp\/v2\/posts\/21707\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/veilles-affaires-sociales.fr\/index.php?rest_route=\/wp\/v2\/media\/17698"}],"wp:attachment":[{"href":"https:\/\/veilles-affaires-sociales.fr\/index.php?rest_route=%2Fwp%2Fv2%2Fmedia&parent=21707"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/veilles-affaires-sociales.fr\/index.php?rest_route=%2Fwp%2Fv2%2Fcategories&post=21707"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/veilles-affaires-sociales.fr\/index.php?rest_route=%2Fwp%2Fv2%2Ftags&post=21707"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}