{"id":2091,"date":"2008-05-16T13:50:49","date_gmt":"2008-05-16T13:50:49","guid":{"rendered":"https:\/\/www.progeriaresearch.org\/?p=2091"},"modified":"2020-12-10T12:55:01","modified_gmt":"2020-12-10T17:55:01","slug":"first-ever-progeria-clinical-drug-trial-surpasses-half-way-mark","status":"publish","type":"post","link":"https:\/\/www.progeriaresearch.org\/fr\/2008\/05\/16\/first-ever-progeria-clinical-drug-trial-surpasses-half-way-mark\/","title":{"rendered":"Le tout premier essai clinique d\u2019un m\u00e9dicament contre la prog\u00e9ria d\u00e9passe la moiti\u00e9 du parcours !"},"content":{"rendered":"<p>[et_pb_section fb_built=\u201d1\u2033 _builder_version=\u201d3.22\u2033][et_pb_row _builder_version=\u201d3.25\u2033 background_size=\u201dinitial\u201d background_position=\u201dtop_left\u201d background_repeat=\u201dr\u00e9p\u00e9ter\u201d][et_pb_column type=\u201d4_4\u2033 _builder_version=\u201d3.25\u2033 custom_padding=\u201d|||\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text _builder_version=\u201d4.6.5\u2033 background_size=\u201dinitial\u201d background_position=\u201dtop_left\u201d background_repeat=\u201dr\u00e9p\u00e9ter\u201d]<\/p>\n<p>Le PRF continue d&#039;\u00e9crire l&#039;histoire, puisque presque tous les enfants inscrits \u00e0 l&#039;essai sont venus \u00e0 l&#039;h\u00f4pital pour enfants de Boston pour leur visite d&#039;un an, marquant ainsi la moiti\u00e9 du chemin vers la fin de l&#039;essai. <a href=\"https:\/\/www.progeriaresearch.org\/fr\/clinical-trials\/\">Cliquez ici<\/a> pour plus de d\u00e9tails sur la fa\u00e7on dont vous pouvez aider.<\/p>\n<p>Des moments passionnants ! L&#039;essai clinique du m\u00e9dicament Progeria a d\u00e9but\u00e9 le 7 mai 2007 avec l&#039;arriv\u00e9e de deux enfants \u00e0 Boston, dans le Massachusetts, pour la premi\u00e8re de leurs sept visites sur une p\u00e9riode de deux ans. Lors de cette premi\u00e8re visite, ils ont subi des tests approfondis et re\u00e7u leurs premi\u00e8res doses du m\u00e9dicament. En moyenne, deux familles se rendent \u00e0 Boston chaque semaine depuis lors et en octobre 2007, l&#039;essai a \u00e9t\u00e9 enti\u00e8rement recrut\u00e9. L&#039;essai devrait se terminer en octobre 2009 et les r\u00e9sultats devraient \u00eatre publi\u00e9s en 2010.<\/p>\n<p>Au 1er octobre 2008, tous les enfants, sauf un, avaient termin\u00e9 la visite d&#039;une semaine et d&#039;un an.<\/p>\n<div><\/div>\n<div><\/div>\n<div>\n<div><strong><\/strong><\/div>\n<\/div>\n<p>[\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row column_structure=&#8221;1_4,1_4,1_4,1_4&#8243; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;][et_pb_column type=&#8221;1_4&#8243; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;][et_pb_image src=&#8221;https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2020\/12\/Megan-medal-May08.jpg&#8221; title_text=&#8221;Megan-medal-May08&#8243; admin_label=&#8221;meagan medal&#8221; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;][\/et_pb_image][\/et_pb_column][et_pb_column type=&#8221;1_4&#8243; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;][et_pb_text _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;]<\/p>\n<p><em><strong>Megan porte fi\u00e8rement sa m\u00e9daille d&#039;essai d&#039;un an, qu&#039;elle a re\u00e7ue \u00e0 la fin de son r\u00e9cent voyage \u00e0 Boston<\/strong><\/em><\/p>\n<p>[\/et_pb_text][\/et_pb_column][et_pb_column type=&#8221;1_4&#8243; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;][et_pb_image src=&#8221;https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2020\/12\/Julieta-Aug07.jpg&#8221; title_text=&#8221;Julieta-Aug07&#8243; admin_label=&#8221;Julieta&#8221; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;][\/et_pb_image][\/et_pb_column][et_pb_column type=&#8221;1_4&#8243; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;][et_pb_text _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;]<\/p>\n<p><em><strong>Julieta, d&#039;Argentine<\/strong><\/em><\/p>\n<p>[\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;][et_pb_column type=&#8221;4_4&#8243; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;][et_pb_testimonial author=&#8221;Francis Collins, MD, PhD, Director of the National Human Genome Research Institute that mapped the human genome, workshop speaker and co-discoverer of the Progeria gene.&#8221; admin_label=&#8221;FC quote&#8221; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;]<\/p>\n<p>\u00ab Je ne connais aucune autre maladie g\u00e9n\u00e9tique rare qui soit pass\u00e9e de la d\u00e9couverte du g\u00e8ne \u00e0 l\u2019essai clinique en moins de quatre ans \u2013 un t\u00e9moignage ph\u00e9nom\u00e9nal du travail acharn\u00e9 de la Progeria Research Foundation. \u00bb\u00a0<\/p>\n<p>[\/et_pb_testimonial][et_pb_text _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;]<\/p>\n<div><strong>Vingt-huit (28) enfants de seize pays<\/strong> Les enfants \u00e2g\u00e9s de 3 \u00e0 15 ans sont concern\u00e9s. Les enfants reviennent \u00e0 l&#039;h\u00f4pital pour enfants de Boston tous les quatre mois pour subir des tests et recevoir de nouveaux m\u00e9dicaments. Ils restent \u00e0 Boston pendant 4 \u00e0 8 jours \u00e0 chaque visite. \u00c0 la maison, leurs m\u00e9decins surveillent de pr\u00e8s les enfants et soumettent des rapports de sant\u00e9 mensuels \u00e0 l&#039;\u00e9quipe de recherche de Boston.<\/div>\n<div>\u00a0<\/div>\n<div>Pendant toute la dur\u00e9e de l\u2019essai, 1 \u00e0 2 enfants par semaine se rendront \u00e0 Boston pour participer.<\/div>\n<div>\u00a0<\/div>\n<div>Les enfants sont originaires des pays suivants\u00a0:<\/div>\n<p>[\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row column_structure=&#8221;1_2,1_2&#8243; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221; custom_margin=&#8221;|-1px||auto||&#8221;][et_pb_column type=&#8221;1_2&#8243; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;][et_pb_text admin_label=&#8221;countries 1&#8243; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;]<\/p>\n<ul>\n<li>Argentine<\/li>\n<li>Belgique<\/li>\n<li>Canada<\/li>\n<li>Danemark<\/li>\n<li>Angleterre<\/li>\n<li>Inde<\/li>\n<li>Isra\u00ebl<\/li>\n<li>Italie<\/li>\n<\/ul>\n<p>[\/et_pb_text][et_pb_image src=&#8221;https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2008\/05\/2-megans-frnt-cover.jpg&#8221; title_text=&#8221;2-megans-frnt-cover&#8221; admin_label=&#8221;2 meghans&#8221; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221; custom_margin=&#8221;|219px||||&#8221;][\/et_pb_image][et_pb_text _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221; custom_padding=&#8221;||0px|||&#8221;]<\/p>\n<p><em>\u00ab Les deux Megan \u00bb, toutes deux \u00e2g\u00e9es de 6 ans, \u00e0 Boston pour l&#039;essai clinique<\/em><\/p>\n<p>[\/et_pb_text][et_pb_image src=&#8221;https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2008\/05\/MichielHayleyJun07.jpg&#8221; title_text=&#8221;MichielHayleyJun07&#8243; admin_label=&#8221;m&#038;H&#8221; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;][\/et_pb_image][et_pb_text _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;]<\/p>\n<p><em>Michiel, 8 ans et demi, de Belgique avec Hayley, 9 ans et demi, d&#039;Angleterre en juin \u00e0 l&#039;h\u00f4pital pour enfants de Boston lors de leur premi\u00e8re visite.<\/em><\/p>\n<p>[\/et_pb_text][\/et_pb_column][et_pb_column type=&#8221;1_2&#8243; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;][et_pb_text admin_label=&#8221;countries 2&#8243; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;]<\/p>\n<ul>\n<li>Japon<\/li>\n<li>Mexique<\/li>\n<li>Pakistan<\/li>\n<li>Pologne<\/li>\n<li>Portugal<\/li>\n<li>Roumanie<\/li>\n<li>USA<\/li>\n<li>Venezuela<\/li>\n<\/ul>\n<p>[\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;][et_pb_column type=&#8221;4_4&#8243; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;][et_pb_text _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221; hover_enabled=&#8221;0&#8243; sticky_enabled=&#8221;0&#8243;]<\/p>\n<p><strong>Le<\/strong> <strong>Essai clinique d&#039;un m\u00e9dicament contre la prog\u00e9ria<\/strong><strong>:\u00a0<\/strong><strong>Qui, O\u00f9, Quand, Comment et Combien\u2026<\/strong><\/p>\n<p><strong>L&#039;essai clinique est dirig\u00e9 par le Dr Mark Kieran, PhD, <\/strong>Directeur du service de neuro-oncologie m\u00e9dicale p\u00e9diatrique du Dana-Farber Cancer Institute et du Children&#039;s Hospital de Boston ; professeur adjoint aux d\u00e9partements de p\u00e9diatrie et d&#039;h\u00e9matologie\/oncologie de la Harvard Medical School. Le Dr Kieran est un oncologue p\u00e9diatrique poss\u00e9dant une vaste exp\u00e9rience du m\u00e9dicament \u00e0 l&#039;\u00e9tude (la farn\u00e9syltransf\u00e9rase, ou FTI) chez les enfants.<\/p>\n<div>L&#039;essai clinique est un effort collaboratif. Les enfants sont suivis par des m\u00e9decins \u00e0 <strong>H\u00f4pital pour enfants de Boston, Institut du cancer Dana-Farber et H\u00f4pital Brigham and Women&#039;s,<\/strong> toutes les institutions de l&#039;Universit\u00e9 Harvard. En outre, des m\u00e9decins et des scientifiques de<strong> La facult\u00e9 de m\u00e9decine Warren Alpert de l&#039;universit\u00e9 Brown, de l&#039;UCLA et du NIH<\/strong> contribuent \u00e0 faire de cet essai un succ\u00e8s. De nombreuses personnes travaillent ensemble pour mener \u00e0 bien cette recherche.<\/div>\n<div><strong>\u00a0<\/strong><\/div>\n<div><strong>Comment en sommes-nous arriv\u00e9s l\u00e0 ?<\/strong> En 2002, l&#039;\u00e9quipe de recherche collaborative de la Fondation de recherche sur la prog\u00e9ria <a href=\"https:\/\/www.progeriaresearch.org\/fr\/2003\/04\/16\/identification-of-gene-gives-hope-to-children-with-progeria\/\" target=\"_blank\" rel=\"noopener noreferrer\">d\u00e9couvert le g\u00e8ne de la Progeria.<\/a>\u00a0\u00a0Cette d\u00e9couverte a non seulement permis de mieux comprendre la prog\u00e9ria, mais les scientifiques savent d\u00e9sormais que l\u2019\u00e9tude de la prog\u00e9ria peut nous aider \u00e0 en apprendre davantage sur les maladies cardiaques et le processus normal de vieillissement qui nous affecte tous.<\/div>\n<div>\u00a0<\/div>\n<div>Depuis la d\u00e9couverte du g\u00e8ne, le soutien des chercheurs, des cliniciens et des familles d\u2019enfants atteints de prog\u00e9ria nous a amen\u00e9s \u00e0 un autre carrefour dans la recherche d\u2019un traitement. Les chercheurs ont identifi\u00e9 un traitement m\u00e9dicamenteux potentiel pour les enfants atteints de prog\u00e9ria, appel\u00e9 inhibiteurs de la farn\u00e9syltransf\u00e9rase (IFT), et ont men\u00e9 des \u00e9tudes en laboratoire qui soutiennent un essai sur l\u2019homme avec ce m\u00e9dicament.\u00a0<a href=\"https:\/\/www.progeriaresearch.org\/fr\/2006\/02\/16\/three-studies-released-that-bring-us-closer-than-ever-to-understanding-progeria-and-to-disease-treatment\/\">Cliquez ici<\/a>\u00a0pour plus de d\u00e9tails sur la recherche.<\/div>\n<div>\u00a0<\/div>\n<div>\n<div><strong>Comment ce m\u00e9dicament agira-t-il contre la prog\u00e9ria ?<\/strong><\/div>\n<div>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 sur la prog\u00e9rine chez les enfants atteints de prog\u00e9ria, la prog\u00e9rine pourrait alors \u00eatre \u00ab paralys\u00e9e \u00bb et la prog\u00e9ria am\u00e9lior\u00e9e.<\/div>\n<\/div>\n<div style=\"text-align: center;\"><img loading=\"lazy\" decoding=\"async\" class=\"wp-image-2096 size-full aligncenter\" src=\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2017\/05\/FTI-3-cell-image-crpd.jpg\" alt=\"\" width=\"375\" height=\"89\" srcset=\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2017\/05\/FTI-3-cell-image-crpd.jpg 375w, https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2017\/05\/FTI-3-cell-image-crpd-300x71.jpg 300w\" sizes=\"(max-width: 375px) 100vw, 375px\" \/> <em><strong>Les cellules de Progeria se normalisent lorsque des FTI sont appliqu\u00e9es. Capell et al., PNAS, 2005. Cellule normale. Cellule de Progeria. Cellule de Progeria apr\u00e8s traitement par FTI<\/strong><\/em><\/div>\n<div>\u00a0<\/div>\n<div>\n<div><strong>Combien co\u00fbtera le proc\u00e8s PRF ? <\/strong>\u00a0Nous estimons que l&#039;essai co\u00fbtera entre 1 400 000 et 2 000 000 de roupies. Ce montant couvrira les tests cliniques, les traducteurs, les vols, la nourriture, l&#039;h\u00e9bergement et certains frais m\u00e9dicaux. <img loading=\"lazy\" decoding=\"async\" class=\"alignright\" src=\"https:\/\/web.archive.org\/web\/20170218013638im_\/https:\/\/www.progeriaresearch.org\/assets\/images\/donate_images\/Capsule-1_9.jpg\" alt=\"\" width=\"150\" height=\"299\" align=\"right\" hspace=\"3\" vspace=\"3\" \/>pendant cette p\u00e9riode de 2 ans et demi.<\/div>\n<div><strong>\u00a0<\/strong><\/div>\n<div><strong><em>Votre don contribuera \u00e0 la r\u00e9alisation de cet essai.<\/em><\/strong><\/div>\n<div>\n<p><em>Le PRF doit r\u00e9unir environ 1,4 million de dollars pour financer cet essai, et en juillet 2009, nous avons r\u00e9uni 1,9 million de dollars !<\/em><\/p>\n<\/div>\n<div>\n<div><strong><img loading=\"lazy\" decoding=\"async\" class=\"alignleft\" src=\"https:\/\/web.archive.org\/web\/20170218013638im_\/https:\/\/www.progeriaresearch.org\/assets\/images\/donate_images\/Circle_of_Hope_4sm.jpg\" alt=\"\" width=\"125\" height=\"130\" align=\"left\" \/><\/strong><\/div>\n<p><strong>Notre <a href=\"https:\/\/www.progeriaresearch.org\/fr\/circle-of-hope-3\/\" target=\"_blank\" rel=\"noopener noreferrer\">Cercle de l&#039;espoir<\/a> s&#039;est d\u00e9velopp\u00e9\u2026<\/strong><\/p>\n<\/div>\n<div>\u00a0<\/div>\n<div>En 2006, la campagne Circle of Hope a \u00e9t\u00e9 cr\u00e9\u00e9e pour r\u00e9colter 100 000 TP4T par an pendant 5 ans afin de maintenir \u00e0 plein r\u00e9gime notre banque de cellules, nos tests de diagnostic, nos subventions de recherche et d&#039;autres programmes. Atteindre cet objectif de collecte de fonds nous permettrait de suivre l&#039;int\u00e9r\u00eat croissant pour la recherche sur la prog\u00e9ria et de poursuivre notre rythme de progression. Qui aurait pu imaginer que moins d&#039;un an plus tard, nous serions au milieu d&#039;une campagne visant \u00e0 r\u00e9colter 100 000 TP4T2 pour financer un essai de m\u00e9dicament pour traiter la prog\u00e9ria ?! Notre campagne Circle of Hope comprend d\u00e9sormais cette initiative.<\/div>\n<div>\u00a0<\/div>\n<div>S&#039;il vous pla\u00eet, aidez-nous \u00e0 garder le <strong>Cercle <\/strong>intact, donc le <strong>espoir<\/strong> du traitement devient un <strong>r\u00e9alit\u00e9.\u00a0<\/strong><a href=\"https:\/\/progeriaresearch.donorsupport.co\/-\/XZHJVWZR\">Faire un don<\/a> aujourd&#039;hui.<\/div>\n<div><em>\u00a0<\/em><\/div>\n<div><strong>Il est temps de trouver un traitement contre la prog\u00e9ria<em>.<\/em><\/strong><\/div>\n<div>\u00a0<strong>Ensemble, nous\u00a0<em>VOLONT\u00c9<\/em>\u00a0trouve le rem\u00e8de !<\/strong><\/div>\n<\/div>\n<p>[\/et_pb_text][\/et_pb_column][\/et_pb_row][\/et_pb_section]<\/p>","protected":false},"excerpt":{"rendered":"<p>[et_pb_section fb_built=&#8221;1&#8243; _builder_version=&#8221;3.22&#8243;][et_pb_row _builder_version=&#8221;3.25&#8243; background_size=&#8221;initial&#8221; background_position=&#8221;top_left&#8221; background_repeat=&#8221;repeat&#8221;][et_pb_column type=&#8221;4_4&#8243; _builder_version=&#8221;3.25&#8243; custom_padding=&#8221;|||&#8221; custom_padding__hover=&#8221;|||&#8221;][et_pb_text _builder_version=&#8221;4.6.5&#8243; background_size=&#8221;initial&#8221; background_position=&#8221;top_left&#8221; background_repeat=&#8221;repeat&#8221;] PRF continues to make history, as nearly all the children enrolled in the trial have come to Children&#8217;s Hospital Boston for their 1-year visit, marking their half-way point to completion. Click here for details on how you can help. Exciting [&hellip;]<\/p>","protected":false},"author":1,"featured_media":11695,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_et_pb_use_builder":"on","_et_pb_old_content":"<p>[vc_custom_heading text=\"October 2008: First-Ever Progeria Clinical Drug Trial Surpasses Half-Way Mark!\" font_container=\"tag:h1|text_align:left\" use_theme_fonts=\"yes\"]PRF continues to make history, as nearly all the children enrolled in the trial have come to Children's Hospital Boston for their 1-year visit, marking their half-way point to completion. <a href=\"https:\/\/www.progeriaresearch.org\/clinical-trials\/\">Click here<\/a> for details on how you can help.<\/p><p>Exciting times! The Progeria clinical drug trial began on May 7th, 2007 with two children arriving in Boston, MA for their first of seven visits over a 2-year period. At this first visit, they were given extensive tests and their first doses of the drug. An average of two families have been flying to Boston each week since then, and in October 2007, the trial became fully enrolled. The trial is expected to end in October 2009, with results published in 2010.<\/p><div>As of October 1, 2008, all but one child has completed the week-long, 1-year visit.<\/div><div>[caption id=\"attachment_2092\" align=\"alignleft\" width=\"175\"]<img class=\"size-full wp-image-2092\" src=\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2017\/05\/Megan-medal-May08.jpg\" alt=\"\" width=\"175\" height=\"275\" \/> Megan proudly wears her 1-year Trial Medal, which she received at the end of her recent trip to Boston[\/caption]<p><strong>\u201cI know of no other rare genetic disease that has gone from gene discovery to clinical trial in under four years - a phenomenal testament to the hard work of The Progeria Research Foundation.\u201d\u00a0<\/strong><\/p><p>\u00a0<\/p>[caption id=\"attachment_2093\" align=\"alignright\" width=\"175\"]<img class=\"size-full wp-image-2093\" src=\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2017\/05\/Julieta-Aug07.jpg\" alt=\"\" width=\"175\" height=\"194\" \/> <em><strong>Julieta, from Argentina.<\/strong><\/em>[\/caption]<p>\u00a0<\/p><p><em><strong>Francis Collins, MD, PhD, Director of the National Human Genome Research Institute that mapped the human genome, workshop speaker and co-discoverer of the Progeria gene.<\/strong> <\/em><\/p><\/div><div><strong>Twenty-eight (28) children from sixteen countries<\/strong> are participating, ages 3 to 15 years.\u00a0Children return to Children\u2019s Hospital Boston every four months, for testing and to receive new drug supply, and stay in Boston for 4-8 days each visit. \u00a0While at home, their doctors keep a close watch over the children and submit monthly health reports to the Boston research team.<\/div><div>\u00a0<\/div><div>For the duration of the trial, 1- 2 children per week will travel to Boston to participate.<\/div><div>\u00a0<\/div><div>\u00a0<\/div><div>\u00a0<\/div><div>\u00a0<\/div><div><div>Children Originate from the Following Countries:<\/div><div>\u00a0<\/div><ul><li>Argentina<\/li><li>Belgium<\/li><li>Canada<\/li><li>Denmark<\/li><li>England<\/li><li>India<\/li><li>Israel<\/li><li>Italy<\/li><li>Japan<\/li><li>Mexico<\/li><li>Pakistan<\/li><li>Poland<\/li><li>Portugal<\/li><li>Romania<\/li><li>USA<\/li><li>Venezuela<\/li><\/ul><\/div>[caption id=\"attachment_2094\" align=\"alignleft\" width=\"113\"]<img class=\"size-full wp-image-2094\" src=\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2017\/05\/2-megans-frnt-cover.jpg\" alt=\"\" width=\"113\" height=\"225\" \/> \u201cThe two Megans\u201d, both 6 years old, in Boston for the clinical trial[\/caption]<p>\u00a0<\/p>[caption id=\"attachment_2095\" align=\"alignnone\" width=\"200\"]<img class=\"size-full wp-image-2095\" src=\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2017\/05\/MichielHayleyJun07.jpg\" alt=\"\" width=\"200\" height=\"177\" \/> Michiel, 8 \u00bd , from Belgium with Hayley, 9 \u00bd , from England in June at Children\u2019s Hospital Boston during their first visit.[\/caption]<p><strong>The<\/strong> <strong>Progeria Clinical Research Drug Trial<\/strong><strong>:\u00a0<\/strong><strong>Who, Where, When, How and How Much\u2026<\/strong><\/p><p><strong>The clinical trial is led by Mark Kieran MD, PhD, <\/strong>Director, Pediatric Medical Neuro-Oncology, Dana-Farber Cancer Institute and Children\u2019s Hospital Boston; Assistant Professor, Departments of Pediatrics and Hematology\/Oncology, Harvard Medical School.\u00a0Dr. Kieran is a pediatric oncologist with extensive experience with the drug under study (farnesyltransferase, or FTI) in children.<\/p><div>The clinical trial is a collaborative effort.\u00a0The children are being seen by physicians at <strong>Children\u2019s Hospital Boston, Dana-Farber Cancer Institute, and Brigham and Women\u2019s Hospital,<\/strong> all Harvard University institutions.\u00a0In addition, physicians and scientists from<strong> The Warren Alpert Medical School at Brown University, UCLA, and NIH<\/strong> are helping to make this trial a success. Many individuals are working together to perform this research.<\/div><div><strong>\u00a0<\/strong><\/div><div><strong>How did we get to this point?<\/strong> In 2002, The Progeria Research Foundation\u2019s collaborative research team <a href=\"https:\/\/web.archive.org\/web\/20170218013638\/https:\/\/www.progeriaresearch.org\/assets\/plugins\/fckeditor\/editor\/progeria_gene_discovered.html\">discovered the Progeria gene.<\/a>\u00a0\u00a0This discovery not only led to further understanding of Progeria, but scientists now know that studying Progeria can help us learn more about heart disease and the normal aging process that affects us all.<\/div><div>\u00a0<\/div><div>Since\u00a0the\u00a0gene discovery, the support of researchers, clinicians, families of children with Progeria have brought us to another crossroads in the search for a treatment. Researchers have identified\u00a0a potential\u00a0drug treatment\u00a0for children with\u00a0Progeria,\u00a0called farnesyltransferase inhibitors (FTIs), and have conducted studies in the lab that support a human trial with the drug.\u00a0<a href=\"https:\/\/www.progeriaresearch.org\/assets\/plugins\/fckeditor\/editor\/more_studies_provide_strong_support.html\">Click here<\/a>\u00a0for more details on the research.<\/div><div>\u00a0<\/div><div><div><strong>How will this drug work in Progeria?<\/strong><\/div><div>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. So if the FTI drug can block this farnesyl group attachment in children with Progeria, then progerin may be \u201cparalyzed\u201d and Progeria improved.<\/div><\/div><div>[caption id=\"attachment_2096\" align=\"alignleft\" width=\"375\"]<img class=\"wp-image-2096 size-full\" src=\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2017\/05\/FTI-3-cell-image-crpd.jpg\" alt=\"\" width=\"375\" height=\"89\" \/> Progeria cells become normalized when FTIs are applied. Capell et al., PNAS, 2005. Normal cell. Progeria cell. Progeria cell after being treated with FTI[\/caption]<\/div><div><a href=\"https:\/\/www.progeriaresearch.org\/assets\/plugins\/fckeditor\/editor\/the_fti_drug.html\">Click here<\/a> for more information on FTIs.<\/div><div><div><strong>What will the trial cost PRF? <\/strong>\u00a0We estimate the trial will cost PRF $2 million dollars.\u00a0This will pay for clinical testing, translators, flights, food, lodging, and certain medical costs <img class=\"alignright\" src=\"https:\/\/web.archive.org\/web\/20170218013638im_\/https:\/\/www.progeriaresearch.org\/assets\/images\/donate_images\/Capsule-1_9.jpg\" alt=\"\" width=\"150\" height=\"299\" align=\"right\" hspace=\"3\" vspace=\"3\" \/>during that 2 \u00bd year time period.<\/div><div>\u00a0<\/div><div><strong>\u00a0<\/strong><strong>Support the <a href=\"https:\/\/www.progeriaresearch.org\/assets\/plugins\/fckeditor\/editor\/clinical_trial.html\">Progeria Clinical Drug Trial<\/a><\/strong><\/div><div><strong>\u00a0<\/strong><\/div><div><strong><em>Your donation will help make this trial happen.<\/em><\/strong><\/div><div><strong>\u00a0<\/strong><\/div><div><p><em>PRF needs to raise approximately $2 million dollars to fund this trial, and as of July 2009, we have raised $1.9 million!<\/em><\/p><\/div><div><div><strong><img class=\"alignleft\" src=\"https:\/\/web.archive.org\/web\/20170218013638im_\/https:\/\/www.progeriaresearch.org\/assets\/images\/donate_images\/Circle_of_Hope_4sm.jpg\" alt=\"\" width=\"125\" height=\"130\" align=\"left\" \/><\/strong><\/div><p><strong>Our <a href=\"https:\/\/www.progeriaresearch.org\/assets\/plugins\/fckeditor\/editor\/circle_of_hope.html\">Circle of Hope<\/a> has expanded\u2026<\/strong><\/p><\/div><div>\u00a0<\/div><div>In 2006, the Circle of Hope campaign was created to raise $100,000 per year for 5 years to keep our Cell Bank, Diagnostics Testing, research grant funding and other programs running full speed ahead.\u00a0Meeting this fundraising goal would allow us to keep up with the increasing interest in Progeria research, and to continue our pace of progress. Who could have imagined that less than a year later, we would be in the midst of a campaign to raise $2 million to fund a drug trial to treat Progeria?!\u00a0Our Circle of Hope campaign now includes this g undertaking.<\/div><div>\u00a0<\/div><div>Please help us keep the <strong>Circle <\/strong>intact, so the <strong>hope<\/strong> of treatment becomes a <strong>reality.\u00a0<\/strong><a href=\"https:\/\/www.progeriaresearch.org\/assets\/plugins\/fckeditor\/editor\/ways_to_donate\/\">Donate<\/a> today.<\/div><div><em>\u00a0<\/em><\/div><div><strong>This is the time to find a treatment for Progeria<em>.<\/em><\/strong><\/div><div>\u00a0<strong>Together, we\u00a0<em>WILL<\/em>\u00a0find the cure!<\/strong><\/div><\/div>","_et_gb_content_width":"","footnotes":"","_links_to":"","_links_to_target":""},"categories":[2],"tags":[],"class_list":["post-2091","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-news"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v26.8 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>First-Ever Progeria Clinical Drug Trial Surpasses Half-Way Mark! - The Progeria Research Foundation<\/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.progeriaresearch.org\/fr\/2008\/05\/16\/first-ever-progeria-clinical-drug-trial-surpasses-half-way-mark\/\" \/>\n<meta property=\"og:locale\" content=\"fr_FR\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"First-Ever Progeria Clinical Drug Trial Surpasses Half-Way Mark! - The Progeria Research Foundation\" \/>\n<meta property=\"og:description\" content=\"[et_pb_section fb_built=&#8221;1&#8243; _builder_version=&#8221;3.22&#8243;][et_pb_row _builder_version=&#8221;3.25&#8243; background_size=&#8221;initial&#8221; background_position=&#8221;top_left&#8221; background_repeat=&#8221;repeat&#8221;][et_pb_column type=&#8221;4_4&#8243; _builder_version=&#8221;3.25&#8243; custom_padding=&#8221;|||&#8221; custom_padding__hover=&#8221;|||&#8221;][et_pb_text _builder_version=&#8221;4.6.5&#8243; background_size=&#8221;initial&#8221; background_position=&#8221;top_left&#8221; background_repeat=&#8221;repeat&#8221;] PRF continues to make history, as nearly all the children enrolled in the trial have come to Children&#8217;s Hospital Boston for their 1-year visit, marking their half-way point to completion. 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Exciting [&hellip;]\" \/>\n<meta property=\"og:url\" content=\"https:\/\/www.progeriaresearch.org\/fr\/2008\/05\/16\/first-ever-progeria-clinical-drug-trial-surpasses-half-way-mark\/\" \/>\n<meta property=\"og:site_name\" content=\"The Progeria Research Foundation\" \/>\n<meta property=\"article:publisher\" content=\"https:\/\/www.facebook.com\/ProgeriaResearch\/\" \/>\n<meta property=\"article:published_time\" content=\"2008-05-16T13:50:49+00:00\" \/>\n<meta property=\"article:modified_time\" content=\"2020-12-10T17:55:01+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2020\/12\/Megan-medal-May08.jpg\" \/>\n\t<meta property=\"og:image:width\" content=\"175\" \/>\n\t<meta property=\"og:image:height\" content=\"275\" \/>\n\t<meta property=\"og:image:type\" content=\"image\/jpeg\" \/>\n<meta name=\"author\" content=\"gravoc\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:creator\" content=\"@Progeria\" \/>\n<meta name=\"twitter:site\" content=\"@Progeria\" \/>\n<meta name=\"twitter:label1\" content=\"Written by\" \/>\n\t<meta name=\"twitter:data1\" content=\"gravoc\" \/>\n\t<meta name=\"twitter:label2\" content=\"Est. reading time\" \/>\n\t<meta name=\"twitter:data2\" content=\"7 minutes\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\/\/schema.org\",\"@graph\":[{\"@type\":\"Article\",\"@id\":\"https:\/\/www.progeriaresearch.org\/2008\/05\/16\/first-ever-progeria-clinical-drug-trial-surpasses-half-way-mark\/#article\",\"isPartOf\":{\"@id\":\"https:\/\/www.progeriaresearch.org\/2008\/05\/16\/first-ever-progeria-clinical-drug-trial-surpasses-half-way-mark\/\"},\"author\":{\"name\":\"gravoc\",\"@id\":\"https:\/\/www.progeriaresearch.org\/ta\/#\/schema\/person\/a3c935a81e92242c6a77ae0018542928\"},\"headline\":\"First-Ever Progeria Clinical Drug Trial Surpasses Half-Way Mark!\",\"datePublished\":\"2008-05-16T13:50:49+00:00\",\"dateModified\":\"2020-12-10T17:55:01+00:00\",\"mainEntityOfPage\":{\"@id\":\"https:\/\/www.progeriaresearch.org\/2008\/05\/16\/first-ever-progeria-clinical-drug-trial-surpasses-half-way-mark\/\"},\"wordCount\":1327,\"commentCount\":0,\"publisher\":{\"@id\":\"https:\/\/www.progeriaresearch.org\/ta\/#organization\"},\"image\":{\"@id\":\"https:\/\/www.progeriaresearch.org\/2008\/05\/16\/first-ever-progeria-clinical-drug-trial-surpasses-half-way-mark\/#primaryimage\"},\"thumbnailUrl\":\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2020\/12\/Megan-medal-May08.jpg\",\"articleSection\":[\"News\"],\"inLanguage\":\"fr-FR\",\"potentialAction\":[{\"@type\":\"CommentAction\",\"name\":\"Comment\",\"target\":[\"https:\/\/www.progeriaresearch.org\/2008\/05\/16\/first-ever-progeria-clinical-drug-trial-surpasses-half-way-mark\/#respond\"]}]},{\"@type\":\"WebPage\",\"@id\":\"https:\/\/www.progeriaresearch.org\/2008\/05\/16\/first-ever-progeria-clinical-drug-trial-surpasses-half-way-mark\/\",\"url\":\"https:\/\/www.progeriaresearch.org\/2008\/05\/16\/first-ever-progeria-clinical-drug-trial-surpasses-half-way-mark\/\",\"name\":\"First-Ever Progeria Clinical Drug Trial Surpasses Half-Way Mark! 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