[et_pb_section fb_built=\u201d1\u2033 fullwidth=\u201don\u201d disabled_on=\u201doff|off|off\u201d _builder_version=\u201d4.16\u2033 border_width_bottom=\u201d55px\u201d border_color_bottom=\u201d#29327a\u201d global_colors_info=\u201d{}\u201d][et_pb_fullwidth_header _builder_version=\u201d4.16\u2033 title_font_size=\u201d55\u2033 background_color=\u201d#29327a\u201d background_image=\u201dhttps:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2019\/04\/About-Header.jpg\u201d background_position=\u201dcenter_left\u201d custom_padding=\u201d9vw||9vw||true\u201d custom_padding_tablet=\u201d\u201d custom_padding_phone=\u201d|56px||\u201d custom_padding_last_edited=\u201dsur|bureau\u201d title_font_size_tablet=\u201d45px\u201d title_font_size_phone=\u201d40px\u201d title_font_size_last_edited=\u201dsur|t\u00e9l\u00e9phone\u201d z_index_tablet=\u201d500\u2033 custom_css_main_element=\u201dbackground-position: center 18% !important;\u201d global_colors_info=\u201d{}\u201d]<\/p>\n
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L'inhibiteur de la farn\u00e9syltransf\u00e9rase (FTI) lonafarnib (commercialis\u00e9 sous le nom de Zokinvy) est le premier et le seul traitement m\u00e9dicamenteux connu pour les enfants atteints de prog\u00e9ria.<\/p>\n
Historique de cette d\u00e9couverte historique : En ao\u00fbt 2005 et f\u00e9vrier 2006, des chercheurs ont publi\u00e9 des \u00e9tudes qui soutenaient un traitement m\u00e9dicamenteux potentiel pour les enfants atteints de prog\u00e9ria. D\u00e9velopp\u00e9 \u00e0 l'origine pour traiter potentiellement le cancer, le lonafarnib a invers\u00e9 les anomalies dramatiques de la structure nucl\u00e9aire qui caract\u00e9risent les cellules des enfants atteints de prog\u00e9ria. De plus, ce m\u00e9dicament FTI a am\u00e9lior\u00e9 certains signes de la maladie dans un mod\u00e8le murin de type prog\u00e9ria.<\/p>\n
Pourquoi les chercheurs ont-ils pens\u00e9 que ce m\u00e9dicament fonctionnerait dans le cas de la prog\u00e9ria ? La prot\u00e9ine que nous pensons responsable de la prog\u00e9ria s\u2019appelle la prog\u00e9rine. Pour bloquer la fonction cellulaire normale et provoquer la prog\u00e9ria, une mol\u00e9cule appel\u00e9e \u00ab groupe farn\u00e9syle \u00bb doit \u00eatre attach\u00e9e \u00e0 la prot\u00e9ine prog\u00e9rine. Les FTI agissent en bloquant (inhibant) la fixation du groupe farn\u00e9syle sur la prog\u00e9rine. Ainsi, si le m\u00e9dicament FTI peut bloquer la fixation du groupe farn\u00e9syle chez les enfants atteints de prog\u00e9ria, la prog\u00e9rine pourrait alors \u00eatre \u00ab paralys\u00e9e \u00bb et la prog\u00e9ria am\u00e9lior\u00e9e.<\/p>\n
Pour la premi\u00e8re fois, nous avions devant nous un traitement possible pour les enfants atteints de prog\u00e9ria. Premier essai clinique d'un m\u00e9dicament contre la prog\u00e9ria<\/a> L'\u00e9tude a d\u00e9but\u00e9 en 2007 et les r\u00e9sultats de l'\u00e9tude ont \u00e9t\u00e9 publi\u00e9s en 2012, d\u00e9montrant que chaque enfant a connu une am\u00e9lioration dans un ou plusieurs domaines, y compris le syst\u00e8me cardiovasculaire vital. En mai 2014, une autre \u00e9tude a r\u00e9v\u00e9l\u00e9 que le lonafarnib augmentait la dur\u00e9e de vie estim\u00e9e d'au moins 1,6 an (qui serait augment\u00e9e plus tard, \u00e0 mesure que l'\u00e9tude progressait), et en avril 2018, une \u00e9tude publi\u00e9e dans Journal de l'Association m\u00e9dicale am\u00e9ricaine (JAMA)<\/em> Il a \u00e9t\u00e9 rapport\u00e9 que le lonafarnib seul prolongeait la survie des enfants atteints de prog\u00e9ria. \u00a0Cliquez ici<\/a> pour plus de d\u00e9tails sur l'\u00e9tude de 2012,\u00a0ici <\/a>pour plus de d\u00e9tails sur les r\u00e9sultats de 2014, et ici<\/a> pour plus de d\u00e9tails sur l\u2019\u00e9tude de 2018.<\/p>\n En mai 2018, dans la foul\u00e9e de l\u2019\u00e9tude publi\u00e9e dans JAMA<\/em>PRF et Eiger Biopharmaceuticals ont conclu un accord de collaboration et d'approvisionnement pour le d\u00e9veloppement et la poursuite de l'examen par la Food and Drug Administration am\u00e9ricaine et de l'approbation potentielle du lonafarnib pour le traitement de la prog\u00e9ria chez les enfants. Le d\u00e9p\u00f4t a \u00e9t\u00e9 compl\u00e9t\u00e9 le 23 mars 2020. <\/a>En novembre 2020, le lonafarnib est entr\u00e9 dans l\u2019histoire en tant que premier traitement \u00e0 recevoir l'approbation de la Food and Drug Administration (FDA) am\u00e9ricaine<\/a> pour la prog\u00e9ria et les laminopathies prog\u00e9ro\u00efdes.<\/p>\n Nous disposons d\u00e9sormais d\u2019un traitement contre la prog\u00e9ria, mais ce n\u2019est pas un rem\u00e8de. Nous continuons donc \u00e0 mener des recherches pour d\u00e9couvrir des m\u00e9dicaments qui seront encore plus efficaces et qui, \u00e0 terme, gu\u00e9riront la prog\u00e9ria.<\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][\/et_pb_column][\/et_pb_section][et_pb_section fb_built=\u201d1\u2033 module_class=\u201dfooter\u201d _builder_version=\u201d4.3.4\u2033 background_color=\u201d#29327a\u201d custom_margin=\u201d25px|||||\u201d custom_padding=\u201d0|0px|0|0px|false|false\u201d z_index_tablet=\u201d500\u2033 border_width_top=\u201d12px\u201d border_color_top=\u201d#00b2e2\u2033 global_module=\u201d133\u2033 locked=\u201doff\u201d global_colors_info=\u201d{}\u201d][et_pb_row column_structure=\u201d1_4,1_4,1_2\u2033 make_equal=\u201don\u201d module_class=\u201d et_pb_row_fullwidth\u201d _builder_version=\u201d4.16\u2033 width=\u201d89%\u201d width_tablet=\u201d80%\u201d width_phone=\u201d\u201d width_last_edited=\u201don|desktop\u201d max_width=\u201d89%\u201d 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global_colors_info=\u201d{}\u201d][\/et_pb_image][\/et_pb_column][\/et_pb_row][\/et_pb_section]<\/p>","protected":false},"excerpt":{"rendered":" [et_pb_section fb_built=\u201d1\u2033 fullwidth=\u201don\u201d disabled_on=\u201doff|off|off\u201d _builder_version=\u201d4.16\u2033 border_width_bottom=\u201d55px\u201d border_color_bottom=\u201d#29327a\u201d global_colors_info=\u201d{}\u201d][et_pb_fullwidth_header _builder_version=\u201d4.16\u2033 title_font_size=\u201d55\u2033 background_color=\u201d#29327a\u201d background_image=\u201dhttps:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2019\/04\/About-Header.jpg\u201d background_position=\u201dcenter_left\u201d custom_padding=\u201d9vw||9vw||true\u201d custom_padding_tablet=\u201d\u201d custom_padding_phone=\u201d|56px||\u201d custom_padding_last_edited=\u201dsur|bureau\u201d title_font_size_tablet=\u201d45px\u201d title_font_size_phone=\u201d40px\u201d title_font_size_last_edited=\u201dsur|t\u00e9l\u00e9phone\u201d z_index_tablet=\u201d500\u2033 custom_css_main_element=\u201dbackground-position: center 18% !important;\u201d global_colors_info=\u201d{}\u201d] Le m\u00e9dicament FTI [\/et_pb_fullwidth_header][\/et_pb_section][et_pb_section fb_built=\u201d1\u2033 use_custom_gutter=\u201don\u201d gutter_width=\u201d1\u2033 specialty=\u201don\u201d padding_left_1=\u201d35px\u201d padding_left_2=\u201d35px\u201d padding_2_tablet=\u201d|||0px\u201d padding_2_phone=\u201d|||0px\u201d padding_2_last_edited=\u201don|desktop\u201d module_class_1=\u201dsidebar-secondary-nav\u201d module_class=\u201dhandprint-bg\u201d _builder_version=\u201d4.16\u2033 background_image=\u201dhttps:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2019\/04\/blue-handprint-only.png\u201d parallax=\u201don\u201d parallax_method=\u201doff\u201d inner_width=\u201d100%\u201d inner_max_width=\u201d100%\u201d custom_padding=\u201d0|0px|54px|0px|false|false\u201d z_index_tablet=\u201d500\u2033 border_width_top=\u201d10px\u201d border_color_top=\u201d#8fd2ed\u201d use_custom_width=\u201don\u201d width_unit=\u201doff\u201d custom_width_percent=\u201d100%\u201d global_colors_info=\u201d{}\u201d][et_pb_column [\u2026]<\/p>","protected":false},"author":1,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"_et_pb_use_builder":"on","_et_pb_old_content":"\t\t\t\t[vc_row][vc_column][vc_custom_heading text=\"The FTI Drug Lonafarnib\" font_container=\"tag:h1|text_align:center\" use_theme_fonts=\"yes\"][vc_custom_heading text=\"The FTI lonafarnib \u2013 the first and only known drug treatment for children with Progeria\" use_theme_fonts=\"yes\"][vc_column_text]In August 2005 and February 2006, researchers published studies that supported a potential drug treatment for children with Progeria. Farnesyltransferase inhibitors (FTIs)<\/b>, originally developed to potentially treat cancer, reversed the dramatic nuclear structure abnormalities that are the hallmark of cells from children with Progeria. In addition, this FTI drug improves some signs of disease in a Progeria-like mouse model.\r\n\r\nWhy did researchers think this drug would work in Progeria?<\/b> The protein that we believe is responsible for Progeria is called progerin.\u00a0In order to block normal cell function and cause Progeria, a molecule called a \u201cfarnesyl group\u201d must be attached to the progerin protein. FTIs act by blocking (inhibiting) the attachment of the farnesyl group onto progerin. \u00a0So if the FTI drug can block this farnesyl group attachment in children with Progeria, then progerin may be \u201cparalyzed\u201d and Progeria improved.\r\n\r\nFor the first time, we had in front of us a possible treatment for children with Progeria.\u00a0<\/b>A first-ever clinical drug trial<\/b><\/a>\u00a0began in 2007, and in 2012 <\/b>the study results were published, demonstrating that every child experienced improvement in one or more areas, including the vital cardiovascular system.\u00a0 In May 2014, further study revealed lonafarnib increases estimated lifespan by at least 1.6 years (time will tell if that number increases \u2013 it just hasn\u2019t been long enough to determine)\u00a0and in April 2018 a study published in The Journal of the American Medical Association (JAMA)<\/em> reported that lonafarnib alone extended survival in children with Progeria. \u00a0Click here<\/b><\/a> for details on the 2012 study,\u00a0here<\/b> <\/a>for details on the 2014 findings, and here<\/a><\/strong> for details on the 2018 study.\r\n\r\nIn May 2018, on the heels of the study published in JAMA<\/em>, PRF and Eiger Biopharmaceuticals entered into collaboration and supply agreement for the development and pursuit of U.S. Food and Drug Administration review and potential approval of lonafarnib for the treatment of Progeria in children. Lonafarnib will mark the first therapy submitted to the FDA for the treatment of Progeria.\r\n\r\nWe now have one treatment for Progeria but it is not a cure, so we continue to drive the research to discover drugs that will be even more effective, and ultimately cure Progeria.[\/vc_column_text][\/vc_column][\/vc_row]\t\t","_et_gb_content_width":"","footnotes":"","_links_to":"","_links_to_target":""},"class_list":["post-796","page","type-page","status-publish","hentry"],"yoast_head":"\nS'inscrire<\/h2>\n
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